Childhood Cancer Research News

Welcome and thank you for visiting!


I hope to raise awareness of childhood cancer and the need for more research. Look here for updates on some of the most important childhood cancer news. My Anjuli died at the age of 4 from a still-incurable type of cancer, a brain stem glioma (aka a Diffuse Intrinsic Pontine Glioma – DIPG). I don’t want other children and families to have to go through what we did (and still are). But sadly, every single day other children and families are going through this nightmare.

Did you know that about 3,000 kids die each and every year in the U.S. from these dreadful diseases? That’s like filling the twin towers at the World Trade Center with children and then having a 9/11/01 event every year!! Not OK with you? Good. Read on and please donate to childhood cancer research. If you would like to subscribe to these updates, please email me at and I will add you to the list. And please share this site with as many people as you can. Thank you!!

~~~~~August 2022~~~~~

A new study out this month in the prestigious medical journal The Lancet, has found that nearly half of global cancer deaths are caused by preventable factors, such as smoking, alcohol use/abuse and obesity. This means that many deaths can be prevented either by avoiding these three risk factors, or making lifestyle changes to quit smoking, reduce alcohol use and lose weight. The study analyzed global data on death and disability from 2010 – 2019 in 204 countries. They analyzed 23 types of cancer and 34 risk factors. The study was funded by the Bill and Melinda Gates Foundation.

And while the study shows that millions of lives can be saved by reducing risk factors, it also shows that millions of cancer deaths are not attributable to risk factors. In my opinion, most notable among these deaths are the deaths of children from childhood cancers. Childhood cancers are not caused by lifestyle choices, and sadly, brain cancer remains the leading disease cause of death of children in the U.S.

~~~~~July 2022~~~~~

Late last month a huge new study was launched to help develop new, targeted treatments for pediatric leukemia patients across the globe. Called the Pediatric Acute Leukemia Master Trial (PedAL), this study is unique both for its global collaboration of doctors and researchers and for the molecular screening of each patient’s cancer. This screening allows doctors to identify targeted treatments for their specific cancer biology, which means patients will not have to endure months of chemotherapies that may not work against their type of leukemia. Plus, once screened, these patients can access new drugs, even those still in clinical trial.

~~~~~June 2022~~~~~

Shocking new data in the U.K. shows that brain tumors across the country have increase by 50% since 2001, the year Anjuli died. 50%!!! In response, the Brain Tumour Charity launched a new website designed to help people know the signs and symptoms of brain tumors and when they should visit their doctor. Called Better Safe Than Tumour, the website lists common brain tumor symptoms for infants – adults. If someone is experiencing two or more of the symptoms (such as frequent vomiting and headaches), they are encouraged to visit their healthcare provider sooner, rather than later. Earlier diagnosis can save some, but not all, lives.

Please share the Better Safe Than Tumour website in your networks. You could help save a life.

~~~~~May 2022~~~~~

Results of an exciting phase 3 clinical trial of a “vaccine” for glioblastomas were revealed at the recent Frontiers in Cancer Immunotherapy Conference. The DCVax-L was found to increase the lifespan of glioblastoma by an average of three months, and some for far longer. It also doubled the five-year survival percentage from 5.7% to 13%. The vaccine involves “training” a patient’s own immune cells, starting with dendritic cells, by exposing them in a lab to the patient’s tumor cells so they “learn” how to fight the tumor. Once the dendritic cells are trained, they are injected into the patient where they then train other immune cells, including T cells, and attack the tumor together.

The DCVax-L GBM vaccine was developed by renown brain cancer researcher Dr. Linda Liau at UCLA.

~~~~~December 2021~~~~~

The Musella Foundation for Brain Tumor Research & Information Inc., reports on a very interesting potential new treatment for Glioblastoma Multiforme, the deadliest type of tumor. The new treatment is only in the very early stages of testing, but it could someday become another tool to fight these horrible tumors. The treatment involves the patient drinking a special dye that sticks to brain tumor cells. The patient is then treated with focused ultrasound to kill the tumor cells that have are dyed.

By the way, the Musella Foundation is great! They help thousands of families fighting brain tumors and have been tracking research progress against these tumors for decades. Please support them with donations if you can.

~~~~~September 2021~~~~~

September is national Childhood Cancer Awareness Month. Please wear gold and do anything you can to support research for cures for childhood cancers. A great way to help fund research for cures for childhood brain tumors and brain cancers is to support the Seattle Run of Hope. Please click here to donate in memory of Anjuli.

There is good news this month in the battle against DIPG. Researchers Michelle Monje, MD & PhD and graduate student Christopher Mount recently discovered that a fatty sugar called GD2 can be found on the surface of approximately 80 percent of DIPG tumor cells. GD2 is well known in the childhood cancer research community because it is found in neuroblastoma. Finding it on the surface of most DIPG tumor cells gives researchers a new target to help treat and eventually even cure these lethal tumors.

Phase 1 clinical studies are already underway where patients’ own immune systems are being “taught” to seek and destroy cancer cells that have GD2 on them. The immunotherapy treatment, pioneered at Stanford Center for Cancer Cell Therapy, is administered directly to children’s brains via a catheter. This bypasses the blood-brain barrier and allows patients’ healthcare teams to better monitor intracranial pressure and disease activity.

It will be years before we know if this treatment will cure these tumors, but it is a new way to target DIPG. Studies like this are expensive and are funded primarily by private foundations, philanthropists and grants. Please consider donating.

~~~~~August 2021~~~~~

Great news this month in the fight against the tumor that killed Anjuli and thousands of other children – DIPG. Dr. Nicholas Vitanza, one of the world’s leading research doctors for cures for central nervous system tumors, now has his own lab dedicated to creating tumor models and finding treatments and cures for DIPG and other dreaded tumors that most often strike children. The Vitanza Lab is part of Seattle Children’s Research and will be able to draw top scientific and disease investigative talent from around the world.

I have had the pleasure of meeting and talking with Dr. Vitanza at the Seattle Run of Hope events (pre-pandemic) and I am glad that he is so dedicated to finding cures for these horrible diseases.  Please support the work of his lab if you are able.

~~~~~June 2021~~~~~

Cancer researchers at the University of British Columbia and BC Cancer have discovered the way one type of childhood cancer, called Ewing sarcoma, can spread to other parts of the body. The cancerous cells create a protein to shield their surface, allowing them to survive traveling in a person’s bloodstream, which then allows the cancer to spread to other parts of the body/metastasize. It is likely that other forms of metastatic cancer create similar protein shields so they can spread too.

This new discovery gives cancer researchers and oncologists a new target to fight so that Ewing sarcoma doesn’t spread to other parts of the body. Once cancer has spread, the chances that a patient will be cured are greatly reduced. And while not all cancers metastasize, those that do can now be studied to find out how to target their protein shields. In the future, it could be possible to prevent metastatic disease.

~~~~~April 2021~~~~~

Researchers at the Queen Mary University in London have discovered a way to deprive a certain pediatric brain tumors of a certain protein they need to grow. The protein is called BMI1 and it is found in high quantities inside a specific subtype of the medulloblastoma brain tumor. The researchers were able to “starve” these tumors in the lab by using inositol hexaphosphate, a natural compound found in most animals and plants. (Inositol was one of the natural substances we gave Anjuli in her battle against DIPG.)

“We have identified a novel way that this type of medulloblastoma is able to adapt its metabolism and grow uncontrollably. Significantly, we have also shown how this energy supply can be blocked. These exciting results bring hope of developing new targeted treatments for patients with this aggressive paediatric brain tumour,” said lead researcher and Professor Silvia Marino, at the Brain Tumour Research Centre of Excellence at Queen Mary University.

More study and lab testing remain before clinical trials in patients can start.

~~~~~February 2021~~~~~

Brain tumor scientists in Australia have discovered that a new two-drug combination dramatically slows the growth of DIPG brain tumors in the lab. One of the drugs is a new chemotherapy agent called AMXT 1501. When used to fight DIPG tumor cells in combination with another, well-known drug, the two work in combination to slow and even stop tumor growth.

One of the things that makes this study especially promising is that it is using real DIPG cancer cells that originally came from donated tumor tissue from young patients in Australia that DIPG killed. Tumor tissue donated by families is a powerful tool in the fight against DIPG. We donated Anjuli’s tumor in 2001 after she died, and there is still some of that tissue left that is helping researchers learn how to end DIPG.

Researchers in Australia hope to start human clinical trials of the new, two-drug combo later this year at Children’s Cancer Institute and Sydney Children’s.

~~~~~September 2020~~~~~

Seattle Children’s is taking CAR T cancer therapy to a whole new level by injecting incredible, engineered T cells directly into the brains of pediatric brain cancer patients via a special catheter. This way, they can get the T-cells directly to the brain without getting “stuck” at the blood-brain barrier. The T-cells come directly from the patients and are engineered to recognize cancer cells and kill them. You can learn more about this incredibly hopeful, but still experimental, treatment here.

~~~~~July 2020~~~~~

CureSearch for Childhood Cancer just announced new funding to help Dr.Ranjit Bindra at the Yale School of Medicine study the addition of a new drug to chemotherapy regimens in the treatment of high-grade pediatric glioma brain cancer. The new drug may increase the effectiveness of the chemo and work to reduce the amount of chemo needed to effectively combat the cancer. Less chemo means reduced toxicity and awful side effects for children.

The new study is being conducted at 18 research hospitals across the country and will take three years to complete.

~~~~~May 2020~~~~~

There is good news this month in the fight against the most deadly disease on earth – DIPG (diffuse intrinsic pontine glioma). Seattle researchers led by Dr. Nick Vitanza at Seattle Children’s, announced that two new chemo drugs worked effectively against DIPG cancer cells in mice. The cells came from a little girl who, like Anjuli, was diagnosed with the deadly disease at age 3. Her name was Addy Trevino and her parents donated extra DIPG cells from her tumor biopsy so they could be studied. These cells were the first to be donated that have never been treated with radiation or other chemos.

Dr. Vitanza and his team discovered that the two new drugs were effective at shrinking DIPG tumors that grew from Addy’s cells. However, they did not do well crossing the blood-brain barrier – a protective coating around the brain that keeps most things out. This issue though, can be solved with the use of other drugs that break down the blood-brain barrier enough to allow the new drugs through.

The discovery that there are two new drugs that can combat this horrible tumor does not mean that a new treatment will be ready to treat children in the near future. Years of further studies must be done, including clinical studies in humans. However, it is a very good sign to have two new drugs that can combat this horrible tumor. What a beautiful tribute to a brave little girl who got her angel wings far too soon.

~~~~~March 2020~~~~~

A recent phase 2 clinical study has found that adding a chemo to one of the most widely used standard-of-care chemos for the treatment of recurring neuroblastoma improved treatment response. The study, done in Europe, means researchers can move forward with Phase 3 clinical studies, which moves the new combination closer to being a possibly improved treatment for this deadly disease.

The added chemo, bevacizumab, was found to benefit pediatric patients on temozolomide, another chemo that is widely used in brain cancer as well as neuroblastoma. It will be many years before we know if this new combination can become a new tool to fight this disease, but a promising Phase 2 study is a step in the right direction.

~~~~~Jan. 2020~~~~~

It is time, past time really, to talk about tumor tissue donation. To those lucky enough to be outside of the childhood cancer community, talking and even thinking about tumor tissue donation can be scary and even a bit creepy. But to those of us in the childhood cancer community, tissue donation can mean the difference between life-saving cures and death.

One of the reasons why progress against pediatric brain cancer is so slow, especially when compared to the research, treatments and cures for other types of adult cancers, is because there simply is not enough tumor tissue for the researchers to study. That’s why it is so important that whenever tumor tissue can be donated, it is.

When Anjuli died in 2001, the decision to donate her entire DIPG tumor was the easiest decision to make out of the hundreds of other agonizing decisions we had to make when she was diagnosed and fighting for her life. My thinking was perhaps when researchers had some of her tumor they could study it to find pathways to cures for future DIPG patients.

I recently learned some of her tumor is still available for pediatric brain tumor researchers, and that there are researchers who want some of it right now. The easiest way for them to get it is through the Children’s Brain Tumor Tissue Consortium. So I took the steps necessary to share what remains of her tumor with the tissue bank and am hopeful that more researchers will use it to provide hope for the hundreds of DIPG patients fighting to live and for the thousands more that will be diagnosed in the future. Many thanks to Amanda Haddock, mom to David who died of this horrible disease in 2012, and head of the Dragon Master Foundation that is fighting for cures, for sharing about the CBTTC with me and for inspiring thousands to donate tissue to the CBTTC.

~~~~~Oct. 2019~~~~~

As if the families of children with cancer do not have enough to worry about, along comes a new crisis – a shortage of a critical chemotherapy used in many forms of childhood cancer. Vincristine is an older, tried-and-true chemo that has been used successfully for years to treat many forms of childhood cancer, including leukemia and lymphomas. That the U.S. is now experiencing a shortage of this staple medication in the fight against childhood cancer is heartbreaking.

The shortage is a result of two issues: one of vincristine’s manufacturers, Teva, decided this past summer to stop making it (not earning enough money for the company’s shareholders), and the remaining company that makes it, Pfizer, has been plagued with what are described as “manufacturing issues.” And so, families of children with cancer and their doctors are left to worry about whether each child will receive her or his dose on time. Some children’s oncology hospital have built up some stockpiles of vincristine, but these can’t last forever.

Pfizer has issued a statement saying they have stepped up production and plan to begin shipments again later this month, but the question remains – how can situations like this be allowed to happen in childhood cancer? Our kids deserve better. Please connect with your elected representatives in Congress and let them know something must be done to make sure this doesn’t happen again. You can find your Congress people here:

~~~~~Sept. 2019~~~~~

Great news this month! Dr. Vitanza, a Seattle Children’s pediatric neuro-oncologist and researcher, is launching a new CAR T-cell trial for children with the most horrible cancer of all, diffuse intrinsic brainstem gliomas (DIPG). About 4,000 kids a year are diagnosed with brain cancer in the U.S., and for those 300 or so with DIPG there are no cures or even good treatments. It is 100% fatal. “That’s a group of children that are just widely underserved by current medicine and need new therapies,” said Dr. Vitanza. CAR T-cell therapy is immunotherapy, where a patient’s own white blood cells are engineered to recognize, find and kill cancer cells.

I recently spoke briefly with Dr. Vitanza at the Seattle Run of Hope, an annual fundraiser that raises millions for pediatric brain tumor research, about the lack of progress in fighting DIPG. Dr. Vitanza explained the main reason DIPG research lags other childhood cancer research is the lack of tumor tissue to study. It has only been in the last two decades that some families (too few) have donated their deceased child’s tumors for research, so DIPG research is decades behind other cancer research where tumors have been donated since the 1970s. We donated Anjuli’s tumor the day she died in 2001. Samples of it were flown by commercial airline that night to researchers around the country.

This new clinical trial holds a lot of hope. I will keep you posted on it.

~~~~~August 2019~~~~~

Good news for potentially curing some children of rare cancers. The St. Baldrick’s Foundation this month awarded $400,000 to continue funding the important big data work of the Treehouse Childhood Cancer Initiative at my alma mater U.C. Santa Cruz in California.

The Treehouse Initiative is the big data, non-clinical part of a research consortium that collects and analyzes genomic information about children’s and adult’s cancer cells, including not just DNA data, but also RNA data. (RNA is a molecule that processes proteins in humans.) Pediatric oncologists contact the group when they have a childhood cancer patient with a rare, difficult-to-treat disease. The researchers then comb through the data to find cancer cell similarities between the patient’s cancer and the millions of other patients’ cancers in the database. When similarities are found, specifically targeted therapies can be used to fight the rare cancer.

“We have a very unique design in that we perform clinically relevant genomic analysis while being outside of a medical institution,” said Olena Vaske, Treehouse Initiative co-founder and assistant professor of molecular, cell and developmental biology at UCSC. “We hope that our work will lead to better treatments and eventually cures for every child with cancer.”

~~~~~June 2019~~~~~

Dr. Matt Dun at the Hunter Medical Research Institute (HMRI) and the University of Newcastle in Australia, has begun a new battle against DIPG that seems to be working to prolong life in the face of this universally fatal brain stem disease. His work in this area is heroic as not only is he a childhood cancer researcher, he also, heartbreakingly, is the father of a child with DIPG.

Little Josie was diagnosed in Feb. 2018. She is still fighting today, thanks to a new drug target Dr. Dun and his colleagues are working on. The new drug, GDC-0084, crosses the blood-brain barrier and disrupts a well-known gene signaling pathway, P13-kinase, used by many cancer cells, including the ones in Josie’s DIPG.

Please watch this short, great video about Dr. Dun, Josie and this important work. You can also contribute to this research and help save Josie’s life, and the lives of thousands of other young children fighting this horrible disease.

~~~~~April 2019~~~~~

This month the Michael Mosier Defeat DIPG Foundation talked with one of the pediatric brain cancer researchers they help fund to get an update on a potentially very exciting new treatment for DIPG – the most deadly disease of all.

The researcher is Dr. David Ashley, director of the Preston Robert Tisch Brain Tumor Center at Duke University. Duke has been doing some incredible research on using inactivated polio virus to treat deadly brain tumors. The virus, after the most dangerous part of it is removed, acts as an inter-cellular delivery system to receptors on the cancer cells. Early results in mice and in petri dishes using human cells are promising.

Duke now needs to work with the FDA to determine what additional studies are required before they can move into clinical trials. This means we are still many years away from knowing if this treatment can slow or even cure DIPG in a person. However, it is still exciting to see new research happening against DIPG.

~~~~~March 2019~~~~

The Musella Foundation reported this month on a potential breakthrough in the treatment of a type of brain tumor called a diffuse midline glioma. The treatment involved an oral chemo called Onc 201, which crosses the blood-brain barrier and makes cancer cells self-destruct. There were 17 patients total and all of them responded to the treatment, including Kristin Johnson of Burlington, CT, who had undergone two brain surgeries previously. After five months of being on Onc 201, most of her tumor shrunk away.

So far the drug is only being tested on brain tumors with a certain mutation – one that is most often found in some types of pediatric brain cancer. It is also being studied in some other cancers. This is definitely a study to keep an eye on.

~~~~~Jan. 2019~~~~~

Last month the Today Show covered the miraculous survival of a young boy who had stage 4 neuroblastoma. Neuroblastoma is cancer that invades nerve tissues and it is very deadly. Roughly only 40 – 50 percent of stage 4 neuroblastoma patients survive five years.

None of the typical neuroblastoma treatments worked for Will Lacey, of Braintree, MA, so his family was told there was nothing else to do. He was only seven months old when he was diagnosed. But Dr. Giselle Sholler, oncologist at Helen Devos Children’s Hospital, tried a chemo called DFMO that is usually used to treat colon cancer.

More research was required, so Will’s family and community raised hundreds of thousands of dollars. Finally, Will was treated and the treatment worked. His tumors began shrinking. Then the company that makes DMFO decided to no longer sell the drug to Will’s family. (The U.S. needs to remove big pharma greed from childhood cancer research!) So they formed their own company and now provide it to other children with neuroblastoma who are in a similar situation. So far, the children they have helped provide treatment for are showing an 97% remission rate.

~~~~~Nov. 2018~~~~~

Neuroblastoma is an aggressive childhood cancer that starts in nerve cells. It strikes about 800 children a year in the U.S. Last month, a new study was announced that involves researchers and hospitals in three countries – the United Kingdom, Germany and the U.S.

The new phase 1 trial will attempt to boost patients’ own immune systems so they can identify and kill cancerous neuroblastoma cells. It involves the use of radioactive therapy and two other anti-cancer agents, one of which has shown promise in the fight against adult cancer, to be used in combination.

“Work in the laboratory has shown that combining these types of antibodies with radiotherapy is potentially a very powerful way of eradicating neuroblastoma tumours…,” said Dr. Juliet Gray, Associate Professor of Paediatric Oncology and pediatric oncologist at the University of Southampton in the U.K.

A cure is desperately needed for neuroblastoma because if it spreads to a child’s bones and bone marrow, it is almost always fatal. If this combination proves effective it will move on to phase 2 and phase 3 clinical studies.

~~~~~Oct. 2018~~~~~

Know thy enemy! It is a key principle for cancer researchers. They must know all there is to know about a specific cancer – its cells, its power sources, its environment and its weaknesses.

Recently, researchers at Seattle Children’s Hospital (Dr. Courtney Crane and Dr. Nick Vitanza, who I have had the pleasure of meeting at the Run of Hope, Seattle), Cincinnati Children’s Hospital, the University of Washington and others, announced results of a study that examined in-depth the microenvironment of DIPG (diffuse intrinsic pontine glioma) tumors, the cancer that kills 100% of those it strikes. This is the tumor that killed Anjuli. It strikes about 300 kids each year in our country and kills them all.

What the researchers found was that the DIPG environment is not inflammatory or highly immunosuppressive. This means that while researchers continue their search for cures for this beast, they do not need to continue to consider drugs that mainly fight inflammation or boost the immune system. Instead, they need to look at drugs that are known to specifically attack DIPG cells and find new ways of introducing those cells to the tumor and keeping them there. Targeted research brings us closer to a cure.

~~~~~Sept. 2018~~~~~

It’s Childhood Cancer Awareness month! What are you doing to raise awareness of childhood cancer? Please consider donating to the Run of Hope in Seattle that funds childhood brain tumor research. We are participating again this year. You can donate in memory of Anjuli hereRead on to see how you can contact your representative to help even more!

We are still waiting to see if the recently passed childhood cancer STAR Act (Survivorship, Treatment, Access and Research Act) will be funded. This landmark bill was finally signed into law in early June, after years of work by advocates to get it passed. It provides $30 million annually from 2019-2023 for research at the NIH and the CDC against childhood cancers. Without funding, the Act will do little to move research forward.

The good news is the Senate included full funding for the STAR Act in its recently passed budget. They also added a $2 billion funding increase to the NIH. The bad news is that now the House must pass its budget and agree to these two funding levels. If the House does approve, the budget will go to conference where a final version will be agreed upon. Then the President must sign it. As you can see, it’s a long road ahead.

If you have not yet written to your House representative letting them know you support funding the STAR Act and increased funding for the NIH, please do so now. The House will begin work on the budget next week and every voice matters in the fight against childhood cancer. Research costs money and $30 million is not a huge, budget-busting number. The United States can afford this investment in saving children’s lives! To find out who your representative is and how to contact them, visit the House website here.

~~~~~Aug. 2018~~~~~

Research scientists at the Wellcome Sanger Institute in the United Kingdom announced their discovery of how very early some childhood bone cancers start to form. The genetic changes that lead to Ewing sarcomas happen years before diagnosis. Knowing this will lead to earlier testing for this disease, which will, in turn, lead to earlier and better treatments for it.

Ewing sarcoma most commonly strikes young teenagers. Current therapies (surgery, chemo and radiation) are extremely harsh and fail in approximately one-third of treated patients. The important discovery of how early these tumors form can also help scientists better understand the causes of other childhood cancers, because the findings are based on genomics, a field of medical research that looks at a patient’s entire set of DNA, not just at individual genes.

~~~~~July 2018~~~~~

Seattle Children’s announced this month that they are conducting a phase 1 clinical trial on CAR T Cell Immunotherapy against certain types of brain tumors. A phase 1 trial is an early stage trial, and if the new therapy proves effective, many more years of testing remain.

This particular trial will offer hope to children whose brain tumors have returned or not responded to traditional therapies – surgery, chemo and radiation. The tumors to be treated must produce the HER2 factor, a protein most commonly found in breast cancer.

CAR T-cell therapy holds much promise against certain types of cancer, including several types of childhood cancer including certain forms of leukemia. The therapy takes a patient’s own cancer-fighting T-cells, modifies them in the lab so they recognize a certain receptor found on cancer cells called the chimeric antigen receptor (CAR) and then re-infuses the T-cells back into the patient so the T-cells attack and kill the cancer cells.

“While survival rates have improved, many of the children we care for have no life-saving therapy options if their disease recurs,” said Dr. Nick Vitanza, MD, a neuro-oncologist at Seattle Children’s and lead investigator for the BrainChild-01 trial. “We have to find a way to give them a life after they recur — and ultimately — be able to offer initial treatments with fewer long-term side effects.”

Seattle Children’s plans to open a similar trial for the treatment of certain solid tumors next month.

~~~~~June 2018~~~~~

There is great news this month about the completion of a remarkable and very important phase 1 study of a new way to treat DIPG, the most deadly cancer of all that is 100% fatal. This is what killed Anjuli. (See my first article about this treatment in the Feb. 2013 post.)

Dr. Mark Souweidane, Director of Pediatric Neurological Surgery at the Memorial Sloan-Kettering Cancer Center and the Weill Cornell Medical Center, announced that convection-enhanced delivery (CED) of a specific antibody via a canula directly to DIPG tumors resulted in no serious adverse effects during the trial, and there also was no toxicity limiting the dosage of the antibody used to fight the tumor. This means that the breakthrough technique to fight these cancers is safe to use in children.

There are still many more years of trials and testing needed to determine the exact dosage needed and precisely which chemos will completely destroy these monstrous tumors, but these results are a huge step forward in the fight against DIPG.

“This trial is about hope,” said Dr. Souweidane. “It’s about admitting that we have been failing these children for decades, and taking a huge departure from the standard protocols in order to provide children with more effective options, less toxicity and increased hope for the future.”


Also, early this month at the American Society of Clinical Oncology annual meeting in Chicago, results of a promising results of a phase 2 clinical trial of a new vaccine targeting glioblastoma, the second most deadly type of cancer after DIPG. Survival rates for glioblastoma are bleak and better treatments and cures are desperately needed. The new vaccine targets a protein that helps cancer cells fight traditional chemotherapies. The name of the protein is surviving and it is inside most cancer cells.

The vaccine, called SurVaxM, was tested on 55 patients who had already received traditional glioblastoma treatments (surgery, chemo and radiation). The trial was sadly not conducted on childhood glioblastoma patients, although the youngest person in this trial was 20. Progression-free survival at 12 months was almost 91% – a big improvement over progression-free survival rates of traditional therapies of just 60-65%.

SurVaxM is years away from standard use as more clinical trials are needed. However, this is a huge step forward in the fight against a disease that has so far remained virtually incurable. “The results are encouraging,” said Dr. Manmeet Ahluwalia, M.D., Director, Brain Metastasis Research Program at the Cleveland Clinic Cancer Center.

~~~~~May 2018~~~~~

The Childhood Cancer Survivorship, Treatment, Access and Research (STAR) Act has passed both houses of Congress! This new law would, if signed into law, expand childhood cancer research, expand research into treatments and therapies for childhood cancer survivors and much more. (Please see April 2018’s post for additional information.)

Also newsworthy is a brand new line of “treatment native” DIPG tumor cells, grown and developed at the Fred Hutchinson Cancer Research Center and Seattle Children’s. This is especially exciting because this is the first time ever that treatment-native cells (those not previously treated with chemo or radiation) have been grown in a lab. These deadly cells can now be used by research scientists to find new treatments for this most devastating tumor. The original cells were donated by a darling little girl stricken by this deadly tumor, Addy Trevino of Montana. You can learn more about this exciting development here.

~~~~~April 2018~~~~~

The Childhood Cancer Survivorship, Treatment, Access and Research (STAR) Act of 2017 is awaiting a vote in the U.S. House of Representatives, after passing the Senate in late March. If passed, the STAR Act would, among other things, expand childhood cancer research, improve the tracking of childhood cancer cases (important for finding clusters of childhood cancer), increase efforts to collect specimens of childhood cancer and increase research on the late effects of childhood cancer treatments on survivors. In short, this is a very important piece of legislation for everyone involved in improving and saving the lives of children with cancer.

Now is the time to contact your U.S. Congressional Representatives and urge them to co-sponsor the STAR Act (it already has more than 300 co-sponsors!) and ask them to vote for it. Please also ask them to fund it, because if the STAR Act is passed with no funding it will not help very many childhood cancer patients. Funding is key!

The bill is pending in House Energy and Commerce Committee. If your Representative sits on that committee, your voice is especially important. Click here to find out who your rep is and how to contact them, and click here to find the members of this committee.

~~~~~March 2018~~

Researchers in the United Kingdom recently announced a major breakthrough in the fight against the tumor that killed Anjuli and thousands of other beautiful children – DIPG. They found that a mutation on a certain gene (ACVR1 to be exact) is found in approximately 1/4 of DIPG tumors, is not found in any other cancers, and is the exact same mutation as the one found in every cell of a person who has Fibrodysplasia ossificans progressive (FOP). This is disease turns the soft tissues of the body into bone.

Finding this mutation means that researchers now have a new target to study for possible new treatments for children with DIPG. Specifically, the team in the UK that discovered this mutation and the link between DIPG and FOP will study whether drugs used to slow the action of the ACVR1 will also slow or stop the cancerous DIPG cells.

“This is the most exciting development in DIPG research since the disease was first diagnosed,” said Dr. Chris Jones of the Institute for Cancer Research in London. 

Years of research are required now to find out if the drugs used to fight FOP (or any other drugs) work to combat the mututation in ACVR1 that causes DIPG. If such drugs are found, they will need to be tested in years of clinical trials before a new treatment could be available.

~~~~~January 2018~~~~~

There is good news this month! A major “big data” cancer-fighting organization has received new funding to continue their important work. The California Initiative to Advance Precision Medicine just awarded $500,000 to the U.C. Santa Cruz Genomics Institute. The grant will fund continued research at the Institute’s Treehouse Childhood Cancer Initiative that supports the California Kids Cancer Comparison project.

As I reported here in April 2016, The California Kids Cancer Comparison project collects and analyzes genomic information of children’s tumor tissues, and helps pediatric oncologists by combing through the data to find tumor cell similarities between different patients’ tumors. When similarities are found, specifically targeted therapies can be used to fight the tumors, based on data about them in the database.

For this particular study, researchers will collaborate with oncologists from Stanford University and the Lucile Packard Children’s Hospital. The goal is to learn whether having a patient’s tumor analyzed by the Cancer Comparison project changes the treatment options clinicians have and whether it benefits pediatric patients and their families.

“We expect to work closely with Stanford clinical research staff using rigorous data-handling processes, institutionally approved protocols, and consent forms,” said Isabel Bjork, director of pediatric programs at the Genomics Institute. “We will have the ability to formally assess the clinical utility of genomic analysis within the clinical environment.”

~~~~~September 2017~~~~~

It’s Childhood Cancer Awareness Month! Please wear gold this month to show your support for childhood cancer. Did you know that every three minutes a child is diagnosed with cancer?! This is not okay! Research can and does save lives! We are once again doing the Run of Hope to provide funding for childhood brain tumor research. Please support us with a donation!

This month we have an exciting update on a study I mentioned here back in Jan. 2014. A new gene therapy has just been approved by the FDA for use in treating childhood leukemia patients. This is the first immune therapy approved by the FDA! It is expected to be the first of hundreds more as research scientists continue to study ways to harness a patient’s own immune system in the fight against cancer.

The new treatment uses a patient’s own white blood cells by genetically altering them so they can recognize and fight cancer cells. The white blood cells are taken from a patient, genetically changed in a lab and then re-infused back into the patient. The new treatment, called Kymriah by Novartis, the pharmaceutical company that won the FDA approval, is for children and young adults who have a certain type of leukemia, a deadly blood cancer.

The first pediatric patient to receive the treatment, Emily Whitehead of Phillipsburg, PA, was within days of dying at age 6 from leukemia. She is now 12.

The treatment carries a large risk of severe, potentially life-threatening side-effects, so it must be given in specially certified treatment centers. It has a huge price tag at $475,000. The treatment is only given once.

~~~~~August 2017~~~~~

Another group of childhood cancer researchers are embracing big data as a way to make new discoveries and increase scientific collaboration that could lead to new treatments, reduced toxicities and even cures. The new project also will include data on birth defects and seeks new discoveries that will aid in the treatment of certain defects.

The new cloud-based, database initiative is called the Kids First Pediatric Data Resource Center. It will be led by The Center for Data Driven Discovery in Biomedicine at Children’s Hospital of Philadelphia, and is funded by the National Institutes of Health Common Fund.

“The Kids First initiative provides a unique opportunity to use DNA sequencing information to gain a better understanding of the underlying causes of birth defects and childhood cancers.” said Dr. Diana Bianchi, Director of the Eunice Kennedy Shriver National Institute of Child Health and Human Development, one of seven major partners in the project.

The other partners are the Ontario Institute for Cancer Research, the University of Chicago, Children’s National Health System, the Oregon Health and Science University and Seven Bridges. The group joins a California-based effort in harnessing the power of big data to fight childhood cancer. As I reported here in April 2016, The California Kids Cancer Comparison project collects and analyzes genomic information of children’s tumor tissues, and helps oncologists by combing through the data to find tumor cell similarities between different patients’ tumors. When similarities are found, specifically targeted therapies can be used to fight the tumors, based on data about them in the database.

~~~~~July 2017~~~~~

The National Cancer Institute and the Children’s Oncology Group have opened a new clinical trial for childhood cancer patients whose tumors are not responding to standard therapies or whose cancers have returned after a period of remission. The Pediatric Molecular Analysis for Therapy Choice (MATCH) trial will screen potentially eligible patients ages 1 – 21 to see if their particular tumor has genetic changes that match genetic changes targeted by a drug in the trial. If a match is found, the patient can join the trial and receive the study medication.

The trial is unique in that it focuses on the genetic changes within tumors, not on a specific type of cancer. The trial is only for patients with solid tumors. “The trial seeks to find out whether it is effective to treat cancer in children and adolescents by targeting certain genetic changes in their tumors with specific targeted drugs, no matter the type of cancer or cancer site.” ~ NCI website, July 2017

~~~~~May 2017~~~~~

May is Brain Cancer Awareness month. If you haven’t yet worn gray this month, you still have time. In honor of those fighting brain cancer and in memory of those who fought the battle but could only be healed in Heaven, please wear gray and please tell people why you are.

Amazing things can happen when teams of medical researchers from different fields come together. A new study published earlier this month shows that an antibody that plays a role in the development of MS can also activate the immune system to target specific cancer cells in three types of cancer, including glioblastoma, a deadly type of cancerous brain tumor. The other two types are colon cancer and melanoma, a deadly type of skin cancer.

Antibodies are proteins that are activated when foreign substances, such as viruses and bacteria, enter the body. They identify and help neutralize the foreigners by activating the immune system. The study was published in the journal Science Immunology by medical research scientists at Brigham and Women’s Hospital in Boston.

“As a neurologist, I never expected I would be publishing a paper about cancer immunotherapy, but as my team studied a subpopulation of T cells that are supposed to prevent autoimmune disease, we had an idea: if cancer is the opposite of an autoimmune disease, we could turn our investigations around and think about how to restore the immune system’s ability to prevent cancer’s growth,” said Howard Weiner, co-director the Ann Romney Center for Neurologic Diseases at Brigham and Women’s Hospital.

~~~~~March 2017~~~~~

Research scientists at the Northwestern University Feinberg School of Medicine in Chicago have found a molecule that stops the growth of the tumor that killed Anjuli, the worst, most-fatal type of cancer of all. It’s called a Diffuse Intrinsic Pontine Glioma or DIPG.

“This tumor kills every single kid who gets DIPG within one year. No one survives,” said the study’s first author, Andrea Piunti, a postdoctoral fellow and one of the research scientists involved in discovering this molecule. The study was published late last month in Nature Medicine.

This molecule prevents DIPG cells from growing because it detaches proteins the cells need. So far the molecule is only being tested on mice, where it is causing human-derived DIPG cells to turn into other types of cells. Years of testing and study remain before a therapy can be developed. Encouragingly, similar molecules are being tested in the fight against leukemia.

~~~~~January 2017~~~~~

A team of medical research scientists in Buffalo, NY at the Roswell Park Cancer Institute, in conjunction with medical researchers at two other institutions, are conducting a phase II clinical study of a potential new vaccine for glioblastomas – one of the most deadly forms of brain cancer.

“Vaccines are typically thought of as things to prevent diseases like measles, polio and mumps. But vaccines are a form of immunotherapy that can also be used to treat cancer. They can be used in a therapeutic mode, rather than a preventive mode,” says Dr. Robert Fenstermaker, Chair of Neurosurgery at the Roswell Park Cancer Institute. “And cancer vaccines, in general, tend to have few serious side effects.”

The vaccine works by targeting a protein the glioblastoma cells need to survive. Once the protein is targeted, the body’s own immune system detects and attacks it. The new vaccine also has the potential to help treat myeloma, a blood cancer. It will be years before anyone knows for sure if the vaccine works against all glioblastomas and whether it will ever be available for children. But it is an important step in fight for a cure.

~~~~~Dec. 2016~~~~~

There is both good news and bad news in the childhood cancer community this month. The good news is that the 21st Century Cures Act became law earlier this month. Championed by U.S. Vice President Joe Biden, whose eldest son Beau died of brain cancer last year, the new law “will harness America’s best minds in science, medicine, and technology to tackle the biggest health challenges of our time,” he said. The new act, among many other important things, invests $1.8 billion in leading cancer research.

The bad news is that the STAR Act did not pass in the U.S. Senate. The Childhood Cancer Survivorship, Treatment, Access & Research Act did pass the U.S. House of Representatives, and would have expanded childhood cancer research and tracking of childhood cancer incidences, provided funding to help improve the health of childhood cancer survivors and increased access to childhood cancer studies for patients who have no other options. At this point the plan is for the STAR Act to be re-introduced in Congress next year. Stay tuned for more details.

~~~~~Nov. 2016~~~~~

Researchers at Novocure, a for-profit company based on Jersey, one of the Channel Islands in the English Channel, announced this month the results of their long-term study of their medical device used to fight GBM brain tumors. The device, called Optune, is a cap worn by patients that creates low-level electrical waves in the brain that disrupt cancer cell division, and do not harm normal cells. The device is for use by adult patients only, but I have heard of at least one child who has been treated with it experimentally.

The research studied nearly 700 patients who wore the device and took the chemo temozolomide and compared survival rates of this group vs. patients who only took temozolomide. Patients treated with both achieved an increase in median overall survival of almost 21 months, compared with just 16 months for those on temozolomide alone. In addition, the number of patients still alive four years post diagnosis was 17% compared to just 10% of patients who only took temozolomide.

More study is needed to show effectiveness in pediatric patients.


Another international company, Israel-based Vascular Biogenics Ltd. (VBL), publicized promising study results for a drug that blocks the way cancerous tumors feed themselves. Tumors actually grow their own blood vessels that then deliver the nutrients and oxygen they need to rapidly grow. Scientists call this process angiogenesis. By blocking it, tumor cells will starve and die.

VBL has discovered a gene that targets only tumor-specific blood vessels and sends other genes to these vessels to block their growth. The new gene is delivered to the tumor via a common cold virus. When it is introduced inside of a cancer patient, not only does the new gene get to work on stopping new blood vessel development, but the patient’s own immune system attacks the tumor as well because it recognizes and responds to the virus.

“Tumors are smart,” said VBL’s CEO Dror Harats, in an interview with The Times of Israel. “They have 20 agents that they can excrete to stimulate the buildup of new blood vessels. For this reason, trying to block one or two of these factors will not be potent enough. We took another approach — when the tumor tries to build these new vessels, we activate a deadly gene, targeting only the angiogenic blood vessels.”

The company is beginning a new Phase III study and interim results are expected in the middle of next year. The study will combine VGL’s new gene with Avastin, another anti-angiogenesis drug. Years of study remain and it is unclear whether the new combination could be used in children because children’s blood vessels are still growing.

~~~~~Oct. 2016~~~~~

Children who have low-grade cancerous brain tumors may be on the verge of getting some very good news. Researchers at Dana-Farber Boston Children’s Hospital have found that a new combination of chemotherapies increased positive response to the therapy and also reduced toxicity.

The small study (only 32 patients) focused only on patients whose tumors have a certain mutation, called BRAF V600. Twenty-three patients responded to the drug – and for two of them their brain tumors completely disappeared! In 11 patients the tumors shrunk by more than half, and 13 had stable tumors for at least six months.

“The finding that dabrafenib can shrink tumors or stop them [from] growing is exciting and has led to trials with a MEK inhibitor and now the combination of drugs. This combined therapy may completely change the way we treat low-grade gliomas in children with this mutation,” said lead author Dr. Mark Kieran, Director, Pediatric Medical Neuro-Oncology, Dana-Farber Boston Children’s. “The caveat is that these targeted personalized drugs are relatively new so we need to make sure that they don’t have any long-term developmental toxicities in children.”

~~~~~Sept. 2016~~~~~

It’s September – Childhood Cancer Awareness month! Please make sure to show your support for the kids fighting cancer, those who died and those yet to be diagnosed by wearing gold this month, spreading awareness and donating to childhood cancer research.

We are once again doing the Seattle Run of Hope this year. Please support us in memory of Anjuli here:

Last month a researcher who is studying DIPGs (the most deadly brain tumor of all) announced that she and her team were able to complete part of a major study that is sequencing the genes of five different DIPG tumor samples. Dr. Marielle Filbin, of the Dana Farber Cancer Research Institute in Boston, and her team were able to generate enough data from the study to qualify for additional funding to continue this important work.

Why is this work important? For several reasons: 1) This type of tumor is the deadliest of all with a zero percentage survival rate. That’s right, zero. Everyone who gets a DIPG (and no one yet knows why some children get these horrible tumors) dies. This is the tumor that killed my Anjuli. 2) Not enough research to fight these tumors is being done, so the more we can learn about them, the better armed the scientists will be when they seek to target and exploit cellular weaknesses to destroy tumor cells.

By analyzing all of the genes in DIPG sample cells (up to 10K actives genes per cell) the researchers now have a much better understanding of all of the various cell-types and genes in different DIPG tumors, and can begin the immense undertaking of learning what drives cell (and tumor) growth, as well as what might work to stop the growth. Since the study is continuing, this knowledge will be shared with other cancer researchers and will likely benefit other types of pediatric cancer research as well.

~~~~~May 2016~~~~~

There are two stories this month, both about brain cancer. It is Brain Cancer Awareness Month after all.

The first story is from 60 Minutes. You may have seen their report on May 15 about how researchers and doctors at Duke University Medical Center are using a modified version of the polio virus to kill the most deadly cancer of all – glioblastomas. (Anjuli had a glioblastoma in her brainstem.) If you haven’t seen the story, check it out online here:

Why the polio virus? The short answer is one researcher, Dr. Matthias Gromeier, was brave enough, audacious enough and smart enough to try it. For years. And now two patients who really would not still be alive if they hadn’t tried the experimental therapy, are still alive. And one, Stephanie Lipscomb, plans to become a pediatric oncology nurse!

I think the best answer  to the “Why polio?” question is from one of the 60 Minute producers. “One of the scientists told me it takes a killer to kill a killer,” says Michael Radutzky, one of the producers. “And that’s when I started to appreciate that if you’re going to kill something as formidable and hydra-headed as cancer, you needed an agent that could do major damage.”


The second story is also about glioblastomas.  They are nearly always fatal, partly because of their ability to grow very rapidly, and also partly because of their ability to “learn” to resist the few chemotherapies that manage to cross the blood-brain barrier.

One of the reasons these deadly tumors grow so fast is because their cells are over-active, continuously sending and receiving signals to grow and invade nearby brain cells. Finding new ways to block, or turn off, this over-active cell signaling is a common target of brain cancer researchers.

Last month, researchers at the University of California at Los Angeles and at Caltech published results from their promising study that uses penny-sized chips that are fitted with tiny DNA “bar codes” that can “read” how the glioblastoma cells reacted to a specific chemotherapy. This particular drug has been found to be effective at shrinking glioblastomas upon initial treatment, but usually only works for about a month before the tumor starts growing again. The penny-sized chips found that the glioblastoma cells started resisting the drug within just two days of being hit with it.

“By sensing this adaptation so early, we were able to anticipate and treat drug resistance,” said James Heath, co-director of the UCLA Jonsson Comprehensive Cancer Center’s Nanotechnology Program.

Eventually, if the study continues to show promise in the lab, researchers could learn more about when and how to switch chemos and what drugs best fight resistance. The study has also shown promise in the fight against melanoma as well.

~~~~~April 2016~~~~~

Big data and big computing are finally being used in the fight against cancer, including childhood cancer. A recent article published by my alma mater, the University of California at Santa Cruz, announced their role in developing a childhood cancer comparison database in California for use by researchers and pediatric oncologists all over the world. The California Kids Cancer Comparison project collects and analyzes the genomic information of children’s tumor tissues, and then helps oncologists by combing through the data to find tumor cell similarities between different patients’ tumors. When similarities are found, specifically targeted therapies can be used to fight tumors, based on data about the tumors in the database.

The project has already helped a child, an eight-year-old boy with terminal lung cancer (sarcoma), who had been through all of the standard treatments, but his cancer kept returning. He was out of options and near death when his doctor sent a tumor sample to the team at UC Santa Cruz, which then analyzed it and found similarities between it and another pediatric tumor. They shared the information with the boy’s doctor, who tried the same drugs used to treat the other patient’s tumor. The new treatment worked and bought the child some time. (I couldn’t find current information about him though.)

For now, the goal of the comparison project is to help the sickest kids, the ones who have relapsed and have little chance of surviving. “Eventually we would like to bring genomics to every child with cancer at diagnosis, but the more immediate goal is difficult-to-treat patients,” said Olena Morozova, a postdoctoral scholar at U.C.S.C’s Center for Biomolecular Science and Engineering, and a lead researcher on the project.

~~~~~March 2016~~~~~

The University of British Columbia recently announced that researchers have discovered that glioblastoma cancer cells “hijack” other healthy brain cells, called astrocytes, and then “re-program” them to create conditions in the cancer patient’s brain that are conducive to tumor growth. This discovery opens up a new way to help kill glioblastomas, by protecting the other healthy brain cells so they can’t be hijacked.

“This is the first evidence that microRNA can go from glioma cells into astrocytes and reprogram them to provide an altered environment that stimulates tumor growth and invasion,” said Dr. Christian Naus, a professor in the Department of Cellular & Physiological Sciences in the Life Sciences Institute, and a researcher at the Djavad Mowafaghian Centre for Brain Health in Vancouver, Canada.

“We should consider the possibility of creating a treatment that would temporarily modify the healthy astrocytes around the tumor so the cancer cells can’t hijack them,” added Wun Chey Sin, a research associate leading the study.

~~~~~December 2015~~~~~

Know thy enemy!

Killing brain cancer requires finding all of its weaknesses and exploiting them. Because brain cancer is a particularly nasty type of cancer, made even nastier by the fact that it uses the brain’s own shield, the blood brain barrier, to protect itself (see November’s article below), doctors and patients require multiple new weapons to kill brain tumors. No potential weakness can be left unexploited.

To this end researchers here in Seattle at the Fred Hutchinson Cancer Research Center, including some of Anjuli’s doctors like Dr. James Olson, are studying patients’ brain cancer cell genomes in an effort to find ways to disrupt their ability to replicate and thrive.

This month Dr. Patrick Paddison and his colleagues at the Hutch announced they have discovered a potential “Achilles heel” in brain cancer cells. Normal neural precursor cells require two specific proteins to progress through the cell cycle. These proteins back each other up. The team at the Hutch found that if either of these certain proteins in brain cancer cells is disturbed, those cells die.

The study shows that genome editing is a viable strategy to employ to find such essential proteins, Dr. Paddison said. And this in turn, could lead to the development of more effective therapies to treat and cure brain cancer, especially glioblastoma, the most deadly type of brain cancer. Anjuli had a stage 4 glioblastoma in her brainstem.

~~~~~November 2015~~~~~~

Several groups of scientists around the world have been researching ways to use ultrasound to open up the blood brain barrier (BBB) – that important shield that protects our brains from contaminants. Opening up the BBB, or making it penetrable in other ways, has long been a huge goal of brain cancer researchers and doctors because the BBB keeps most chemotherapy drugs out of patients’ brains and away from the tumors they are meant to kill.

Earlier this month a group of scientists at Sunnybrook Hospital in Toronto, Canada announced they treated the first brain tumor patient with ultrasound, micro-bubbles and chemo. They called their trial treatment an “ultrasonic screwdriver.” The technique was first developed by researchers at Pitié-Salpêtrière Hospital in Paris, who placed ultrasound emitters inside a brain cancer patient’s brain. The Toronto team’s treatment involved no surgery and instead used external ultrasound.

The non-invasive treatment consisted of a female brain cancer patient receiving chemo and micro-bubbles into her bloodstream. Then a beam of ultrasound was focused on her tumor. This caused the micro-bubbles to vibrate and create tiny holes in a small part of her BBB. This allowed the chemo to cross into the tumor.

Complete results have not yet been announced, and the treatment is still in the trial phase. If it proves to be effective, this treatment could be used to treat other diseases, including Alzheimer’s. Researchers are already studying its effectiveness in treating Alzheimer’s in mice.

~~~~~October 2015~~~~~

Earlier this month researchers at the College of Human Medicine at Michigan State University announced they have discovered that two well-known medications used to treat other diseases can be combined to help stop of the growth of neuroblastoma, a common form of childhood cancer.

This is potentially huge news for neuroblastoma patients and their families, because the drugs are already in use and therefore are already approved by the federal Food and Drug Administration (FDA).

“It’s such a big advantage when you find an existing, FDA-approved drug that acts on neuroblastoma, because it already has been shown to be safe,” said André Bachmann, lead researcher.

Neuroblastoma is cancer that forms on certain nerve cells. It causes about 15% of all childhood cancer deaths. Most patients get sick before they are two years old.

Bachmann, who worked with other researchers on the new study, including Lisette Yco, a graduate student at the College of Human Medicine, and Dirk Geerts, a cancer researcher in the Netherlands, had already discovered that a drug called difluoromethylornithine (DFMO) targeted a protein neuroblastoma cells need in order to grow. DFMO is used primarily to treat African sleeping sickness. The current study sought another drug that could work synergistically with DFMO and be even more effective in killing neuroblastoma cells.

The researchers found the drug sulfasalazine, which is used to treat rheumatoid arthritis and bowel disorders, worked similarly to DFMO. And when combined, the drugs were more than twice as effective. “Instead of one plus one equals two, it equals four,” Bachmann said. “That’s why the synergistic effect is so important. We can use the two drugs in lower doses, thus achieving the same result while minimizing side effects.”

~~~~~September 2015~~~~~

It’s Childhood Cancer Awareness Month! What are you doing to help raise awareness of childhood cancer and the need for more research to help cure these diseases? There are many things you can do including making a donation to support childhood cancer research (we are doing the Run of Hope for pediatric brain tumor research and would love your support!), wearing gold, posting about it on your social media and reaching out to your elected representatives to see if they are supporting childhood cancer research. For more ideas, go here


When people think of childhood cancer, they don’t usually think about the devastating and sometimes long-lasting, potentially life-threatening, side-effects that many of the cancer treatments cause. But thankfully some doctors and research scientists are. They are providing hope to thousands of survivors and their families.

One of the deadliest side-effects is heart damage. Sometimes kids who survive have hearts so damaged they resemble the hearts of 50-year-old, obese men who smoke! (And yes, these kids are the lucky ones.)

There’s a new study that just launched that is testing to see whether low doses of a blood pressure drug called carvedilol can protect kids’ hearts during cancer treatment. The nationwide study is just now getting underway, and it will be many years before we know if the drug helps, but it is a beginning.

“Best case scenario is that there will be a subset of patients in whom we can avert a life threatening complication that develops after treatment, without compromising their overall health-related quality of life,” said Dr. Saro Armenian, of City of Hope in Duarte, CA, who is leading the study. “That is really something that would be momentous, in terms of being able to preserve the health of our survivors and making sure that they thrive and not just survive.”

Dr. Armenian has a strong drive in the fight against childhood cancer. He lost his sister to leukemia when he was a teenager. He was her bone marrow donor. She died from complications of the transplant.


Here’s a great video about the tumor paint I keep telling you about. Tumor paint delivers tiny molecules derived from scorpions to cancer cells in the brain and lights them up! This makes it so surgeons can easily see what is tumor and what is normal brain tissue. This means surgeries with less risk and more success!

It’s a 3-minute video and two of Anjuli’s docs and one of her nurses talk about and show how tumor paint works. I love it when Dr. Ellenbogen says “If we can see the cancer while we are operating, then we can Kill the Cancer.” Tumor paint is in clinical trials for both pediatric and adult brain tumor patients.

~~~~~August 2015~~~~~

As we near Childhood Cancer Awareness Month, it is time to start thinking about advocacy. September is the month to wear gold to show your support for children with cancer, their families and the need for more research. So how about advocating for STAR?

The Childhood Cancer Survivorship Treatment Access and Research (STAR) Act, a new bill introduced in Congress recently by Sen. Jack Reed, D-Rhode Island, would, among other things, improve efforts to track the incidence of childhood cancer nationwide, expand childhood cancer research, and ensure that pharmaceutical companies make their compassionate-use* policies publicly available.

The STAR Act already has bi-partisan support, but of course, we need all Senators to get onboard and support this important legislation. So please contact your Senator and let them know you want them to support it.

“Too many young people’s lives have been cut short by cancer. These kids and their families who’ve battled this disease inspire us to take action,” said Sen. Reed.

Almost 16,000 kids in the U.S. get diagnosed each and every year. It’s up to everyone to help.

*Compassionate use refers to a pharmaceutical company allowing a very ill patient with incurable disease to take a chemotherapy that is still in clinical trial and not yet fully approved by the FDA. For some patients who have failed all other therapies, having access to some of these drugs has meant the difference between life and death. Anjuli was a compassionate-use patient for Gleevec. It did not work for her, but it has worked for thousands since.

~~~~~July 2015~~~~~

St. Baldrick’s Foundation just announced its summer research grants. Check out the reactions of some of the researchers as they found out their work has been funded.

Some of the $21.2 million that was awarded is going to Dr. Stephen Hunger of The Children’s Hospital of Philadelphia, to help support the research he and his team from around the country are doing to find a cure for the most deadly type of childhood leukemia.

High-risk acute lymphoblastic leukemia (ALL) does not respond to the treatments that most other leukemias do. Dr. Hunger and his team have been looking at 125 genes from patients with this type of leukemia for the past 10 years. They found that there is not just one unique subset of high-risk ALL patients, as previously thought, there are two, and each requires different chemotherapy because each expresses a different protein that can be targeted. This second subset of high-risk ALL disease is only now being researched because Dr. Hunger’s team, along with another team in the Netherlands, only recently found it. Now, thanks to their St. Baldrick’s grant, the team is planning to screen about 800 high-risk ALL patients each year to see if they can benefit from their new findings.

“We have a lot of anecdotal evidence of great short-term responses, but the goal of this [research] is to see if the strategy will not just get short-term responses, but will lead to higher cure rates,” Dr. Hunger said.

~~~~~May 2015~~~~~

May is National Brain Tumor Awareness Month. Read more about Anjuli at Project Violet.

Lauren Hill died last month of the same brain cancer that killed Anjuli. She was 19. And like Anjuli, she was a brain cancer warrior. Also, just like Anjuli, Lauren will never be forgotten. She inspired thousands and helped raise more than $1 million for brain cancer research, all while fighting for her life and for the last months, while in hospice care. Go here for a beautiful, short tribute to her.

We owe it to cancer warriors like Lauren and Anjuli and Sarah and Tess and Tara and DeLila and so many, many, MANY others to keep on fighting. And fighting means research. And research costs money. And since the federal government allocates less than 2% of its cancer research funding to childhood cancer, it is up to us in the private sector to keep the research moving forward. Thankfully, several non-profit organizations have been moving the needle by mobilizing thousands to support this cause. Among them are St. Baldrick’s Foundation, Alex’s Lemonade Stand, The Musella Foundation for Brain Tumor Research and more. We must never give up!


The National Institutes of Health announced May 4 that researchers from the U.S. and Europe have found an anti-cancer drug that may end up slowing the growth of Diffuse Intrinsic Pontine Gliomas (DIPG) – the most lethal cancer of all. And this discovery was made possible by testing DIPG tissue samples for children who died.

“Our results provide a glimmer of hope for treating this heartbreaking disease,” said Michelle Monje, M.D., Ph.D., assistant professor of neurology and neurological sciences, Stanford University School of Medicine, California, a senior author of the study and a specialist in DIPG. “Caring for DIPG patients drives me to find new ways to treat them.”

DIPG mostly strikes children 4 to 9 years of age (Anjuli was just 3), and kills them all, usually within nine months.

The drug is called panobinostat and is being tested in petri dishes and in mice. There is a long way to go before we will know if it will work in humans. But early results are promising.

“It’s astounding. In only six years, scientists have gone from knowing virtually nothing about this tumor to understanding its underlying genetics and finding a potential therapy,” said Jane Fountain, Ph.D., program director, at the National Institute of Neurological Disorders and Stroke (NINDS), part of NIH. “This study epitomizes collaborative medicine at work. It took a dedicated team of international scientists working with patients, families and foundations to get to this point.”

When she says collaborative medicine, she isn’t kidding. Check out the following list of organizations that support this work: The National Cancer Institute, The Cure Starts Now Foundation, Reflections of Grace Foundation, Smiles for Sophie Foundation, Cancer-Free Kids Foundation, Carly’s Crusade Foundation, Jeffrey Thomas Hayden Foundation, Soar with Grace Foundation, Accelerate Brain Cancer Cures Foundation, The Lyla Nsouli Foundation, CureSearch for Childhood Cancer, The Team Julian Foundation, Alex’s Lemonade Stand Foundation, McKenna Claire Foundation, Connor Johnson Memorial Fund, Dylan Jewett Memorial Fund, Dylan Frick Memorial Fund, Abigail Jensen Memorial Fund, Zoey Ganesh Memorial Fund, Wayland Villars Memorial Fund, Jennifer Kranz Memorial Fund, Virginia & D.K. Ludwig Fund for Cancer Research, Price Family Charitable Fund, Matthew Larson Foundation, Godfrey Family Fund in memory of Fiona Penelope, Child Health Research Institute at Stanford Anne T. and Robert M. Bass Endowed Faculty Scholarship in Pediatric Cancer and Blood Diseases, Etoile de Martin, Foundation LEMOS and Le Defi de Fortunee, Scott Carter Foundation, Semmy Foundation, Department of Defense, Marie Curie, Spanish Ministry of Health, St. Baldrick’s Foundation and Iron Matt Foundation.

~~~~~April 2015~~~~~

Scorpion venom. Polio virus. That brain cancer patients are willing to have these substances put into their brains in an attempt to survive speaks to the depths of fear brain cancer instills. Because it is so often incurable. Because it so often claims the lives of the young. Because it claims those lives in a horribly devastating way.

By now many of you have heard that the Duke University Medical Center in Durham, North Carolina, is pioneering a clinical trial using polio virus to kill brain cancer cells in the worst tumors of all – glioblastoma multiforme (GBM), led by renowned neurosurgeon Dr. Henry Friedman. Did you know that this trial and possible “cure” for some patients (not all) was funded in part by a wonderful childhood cancer research foundation? It was. Back in 2006 Matthias Gromeier, MD, at Duke Medical Center, was working on “re-engineering” the polio virus as a potential anti-brain cancer agent. Alex’s Lemonade Stand Foundation, awarded him a grant. His grant proposal specifically targeted pediatric GBMs.

~~~~~February 2015~~~~~

Brain tumors feed not only on glucose, but also on natural acetate in the body – which is produced in the body as a result of carbohydrate fermentation in the gut and from other sources too many to include here. Acetate does cross the blood brain barrier and new research by the Harold C. Simmons Cancer Center in Dallas shows brain tumor cells burn acetate for fuel.

Why is this significant? Because controlling glucose alone will never be enough to control cancer growth if the nasty cancer cells have two sources of fuel. And finding this second fuel source, acetate, gives researchers yet another target to focus on.

“We identified that glucose wasn’t the only fuel being burned,” said senior author Dr. Robert Bachoo, Assistant Professor of Neurology and Neurotherapeutics, and Internal Medicine, and a member of the Harold C. Simmons Cancer Center. “Acetate can be used to generate fuel and metabolites that can then be used to make other things that the cell needs to survive and multiply.”

The more targets, the better.


People have done many amazing, beautiful and creative things to raise money for cancer research. I recently learned of a very unusual effort that garnered more than $21,000. In Billingsborough, UK, two friends on a cricket team batted non-stop for 48 hours to raise money and to win the Guiness Book World Record for cricket batting – at the same time. Dave Newman and Richard Wells completed the challenge in honor of their friend Wayne Chessum, father of three, who is battling brain cancer. Now I know this story doesn’t directly relate to childhood cancer, but I include it here because #1 – Raising money for brain tumor research helps kids with brain cancer (brain cancer is the leading cause of cancer death in kid, and #2 – How many times have you heard of two guys winning the Guinness World Record for cricket batting while raising money to fight cancer?

~~~~~January 2015~~~~~

We know cancer cells can develop resistance to the chemotherapies used to try to eradicate them. This is especially true of brain cancer cells. Researchers are looking for ways to combat this and recent research by an international group of scientists (see below for the complete list) is working to develop a new drug that circumvents Temodar resistance. Temodar is one of the leading brain cancer chemo treatments (Anjuli was on it), so finding something that will make Temodar work better, longer, would be very important. Killing more cancer cells is always a good thing.

These researchers are from:

  • Department of Biochemistry and Molecular Pharmacology, University of Massachusetts Medical School
  • Department of Radiation Oncology, Mayo Clinic, Rochester, Minnesota
  • Institute of Cancer Therapeutics and the School of Pharmacy at the University of Bradford, Bradford, United Kingdom
  • School of Pharmacy, University of Eastern Finland, Kuopio, Finland


Sad new childhood cancer stats – the incidence of childhood cancer is increasing. Only a couple of years ago the risk of getting cancer before you turned 20 was 1 in 300. And 12,500 kids a year in the US were diagnosed with cancer every year.

But now, it is a 1 in 285 risk and almost 16,000 new cases will be diagnosed each year.

Source: American Cancer Society

~~~~~December 2014~~~~~

In the spirit of the season, I am happy to share news of a gift from the just-signed federal budget to the fight against childhood cancer. The Gabriella Miller Kids First Research Act, which was signed into law last April, was fully funded!

The Act was inspired by young Gabriella Miller, who lost her battle with childhood brain cancer in 2013 at the age of 10. It will redirect $126 million a year for 10 years ($12.6 billion) from a rarely used fund that finances national political conventions to the National Institutes of Health specifically for childhood cancer research and other childhood disorders, such as autism.

Isn’t it wonderful that somehow the “powers that be” in WDC decided to put children first – instead of politicians? Could this be a “Christmas Miracle”?

I am inspired beyond words by what a hero Gabriella was and still is. Go here to learn why:

~~~~~November 2014~~~~~

Small studies continue on how to cure diffuse intrinsic brainstem gliomas, most of them directed at what gene or signaling pathway or protein to target. In other words, there are studies trying to determine where the tumor may be weakest so that treatments can be designed to target those weaknesses.

One of the ways that helps to determine these weaknesses is to do a biopsy. But it is very, very rare for a neurosurgeon to do a biopsy of a brain stem glioma because the risk of permanent serious injury or death. But a recent study has determined biopsy of these tumors does not result in surgically responsible complications or increases in tumor symptoms or death. Unfortunately this study was very small – only 22 patients at Children’s Hospital of Michigan, but it is important and does give us reason to hope.


Here is an update on Dr. Mark Souwdaine’s amazing and potentially breakthrough study of the type of cancer that killed Anjuli – a diffuse, intrinsic brain stem glioma. The treatment being studied delivers anti-cancer drugs directly to the brain stem via a canula (this bypasses the blood brain barrier completely). The two-year study is ongoing and no results have been shared yet. They are still recruiting patients and hope to finish the Phase 1 study in Dec. 2015.

Dr. Souweidane invites those interested in the study to call his office at 212-639-7056, or visit

~~~~~October 2014~~~~~

The St. Baldrick’s Foundation recently awarded another $2.5 million in grants to help researchers fund the staff and other foundational resources needed to treat more children on clinical trials. And by the way, this latest round of funding brings the total to $24.7 million in volunteer-raised dollars going to researchers via St. Baldrick’s. Volunteers do make a difference!

A new treatment to prevent the often deadly complication of graft-versus-host-disease after a bone marrow treatment is showing early signs of success. The treatment involves giving childhood bone marrow transplant patients a drug previously only used to treat rheumatoid arthritis if they develop symptoms of GVHD – and to give the drug very early after the onset of symptoms. The drug, etanercept, works by suppressing a protein called tumor necrosis factor alpha, which our bodies produce more of if there is inflammation. The study is finding that fewer children progress to the deadly stages of GVHD if they are given etanercept early, said Dr. Sung Won Choi, M.D., M.S., a St. Baldrick’s Scholar at the University of Michigan, who is leading the study.

The current “standard of care” treatment is high dose steroids, which only works about one-third of the time. There has been no advancements in the last couple of decades. “New, non-steroidal therapies for GVHD are urgently needed,” Dr. Choi said.

~~~~~September 2014~~~~

September is Childhood Cancer Awareness Month! Wear gold!

Red berries and nuts could one day help in the fight against childhood cancer. A researcher from Karlstad University in Sweden recently presented her doctoral thesis that documented how a certain phytochemical can make childhood cancer cells kill themselves.

Christina Fjaeraa Alfredsson used ellagic acid found in pomegranates, strawberries, raspberries and walnuts against neuroblastoma cells. Neuroblastoma is the third most common childhood cancer in children under the age of one. It is difficult to treat. It affects the nervous system.

“The number of tumour cells in our model system was drastically reduced after the addition of ellagic acid,” Fjaeraa Alfredsson said.

Many more years of research are needed before we will know for sure if ellagic acid can be used to fight neuroblastoma and other cancers. This is yet another reason why more money is needed for childhood cancer research. That’s one reason I am once again doing the Run of Hope. You can support me and thousands of kids with childhood brain cancer here:

~~~~~July 2014~~~~~

Many childhood cancers are genetic. Some of the gene mutations can be passed down to the children of a person with the genetic mutation.

But now, thanks to childhood cancer research into the field of childhood ovarian and lung cancer, some of these mutations can be screened for in infants soon after birth. If detected, doctors can look for the specific cancer and surgically remove it while it is in its earliest, and therefore most curable, stage.

Sometimes, the research that saves one generation comes full circle and saves the life of another. This is what happened to Megan Flynn, a survivor of a rare type of ovarian cancer when she was 17. She joined a cancer registry for ovarian and testicular cancers. Eventually she had a blood test and found that she has the genetic mutation that caused her cancer. This type of mutation is found in every cell in the body.

In 2009 childhood cancer researchers found a link between Megan’s type of genetic mutation and another type of childhood cancer – lung cancer.

Not long after joining the registry, which was started with help by a St. Baldrick’s funded scholar, Megan gave birth to a baby boy, Andrew. He was screened and was found to have the mutation. He was then given a CT scan and a cyst was found on his lung – a cyst that would have become the lung cancer caused by the same mutation that caused his mother’s ovarian cancer. It would never have been found so early if he had not been screened, because Andrew had no symptoms.

“Even after I knew it was there, I couldn’t hear it, and I couldn’t see it from looking at him,” said Kris Ann Schultz, M.D., who helped form the OTST Registry.

Andrew had surgery immediately to remove the cyst and today he is happy and healthy. He did not need chemo because his cancer was caught so early. He will continue to be monitored, of course, for several years. But his chances are good.

~~~~~June 2014~~~~~

Know Thy Enemy!!

A recent study of glioblastomas (the most deadly brain tumor of all) found that they produce multiple cancer cell types, and that the cancer cells exist in many different states, including some that are “stem-cell-like,” making them able to recreate themselves and start new tumor growth even though other parts of the tumor may have been successfully treated. This is just one reason why glioblastomas are so deadly – they are, for now at least, impossible to cure because of the immense diversity of cancer cells that must be killed.

But this news is not all bad – simply knowing now that glioblastomas contain multiple cell types and cell stages, and are unique to each individual, gives direction to brain cancer researchers. They have a clearer picture of how to attack these tumors; and that the attack must really be multiple attacks on multiple cell types and the various life-cycle stages they are in.

“This is an incurable disease. There are existing therapies that may target 99 percent of the cells, but the tumors always come back. Understanding the cellular landscape can provide a blueprint for identifying new therapies that target each of the various sub-populations of cancer cells, and ultimately for tailoring such therapies to individual patient tumors,” said co-senior author Bradley Bernstein, a senior associate member of the Broad Institute and professor of pathology at Massachusetts General Hospital and Harvard Medical School.

The study was funded by the Klarman Family Foundation, as well as the National Institutes of Health, Howard Hughes Medical Institute, the Burroughs Wellcome Fund, and the Harvard Stem Cell Institute

~~~~~April 2014~~~~~

Young Gabriella Miller, who lost her battle with childhood brain cancer, inspired the passage of a new federal law which will redirect $126 million over 10 years from a rarely used fund that finances national political conventions to the National Institutes of Health. The money is specifically for research for cures for childhood cancer and other childhood diseases and disorders, such as autism. The Gabriella Miller Kids First Research Act was signed into law April 3, 2014. I am inspired beyond words by what a hero Gabriella was and still is. Go here to learn why:

Did you know there is a childhood cancer caucus in the United States House of Representatives? Go here to see if your congressional rep is a member. If not, ask them why not? . For those of you in Washington State, Jim McDermott is a member.

Recently the National Cancer Institute convened a meeting entitled “Barriers to Drug Development in Pediatric Cancer Research.” In attendance were oncologists, government scientists and members of the childhood cancer advocacy community.

Of course, the main barrier is, plain and simple, pharmaceutical company greed. Since the childhood cancer “market” is “small” compared to breast, lung or other adult cancer markets, big pharma doesn’t believe there is enough “return on investment” to help in the fight to Save Kids’ Lives!! Yes, they look at ROI first, even though they make millions in profits every quarter!

At the meeting, the mother of a child who fought AML 27 years ago compared her daughter’s treatment back then to the treatment received by another young girl with AML last year. The drugs were the SAME! These drugs were developed in the 1950s and 1960s!! Her daughter lived, but now has severe side-effects that will last forever.

The good news that came out of the meeting is that finally, as we know, genuine progress in the fight for cures is being made, largely because of research in, and a new understanding of, the human genome and patient’s immune systems. This life-saving research is almost entirely funded by private foundations, including St. Baldrick’s, and also even small endowment funds like my daughter’s – The Anjuli Jacobs Endowment for Brain Stem Glioma Research.

~~~~~March 2014~~~~~

The University of Texas Health Science Center in San Antonio announced March 2 a new and vastly improved laboratory screening technique that helps researchers identify what to target when trying to defeat cancer cells in neuroblastomas. Neurblastomas are a common childhood cancer and account for 15% of child cancer deaths.

The new screening method was developed by Liqin Du, Ph.D., an assistant professor in the Department of Cellular and Structural Biology, and her team at the Greehey Children’s Cancer Research Institute at the University of Texas Health Science Center.

“Development of new agents for treating neuroblastoma has been greatly hampered by the lack of efficient high-throughput screening approaches,” Dr. Du said. “In our study, we applied a novel high-content screening approach that we recently developed to investigate the role of microRNAs in neuroblastoma (cell death).”

Now that researchers have identified this new target, they are better armed in developing new drugs to attack the cancerous cells.

~~~~~February 2014~~~~~

I recently found a research study that investigated whether there is an increased risk of genetic abnormalities in children born to childhood cancer survivors. Happily, the answer is no!

Also of interest this month: This Thursday there will be a meeting at the National Institutes of Health on pediatric cancer drug development – an issue of great concern in the childhood cancer community because of the abysmal lack of drug development for kids with cancer. The meeting will host panels that will discuss, among other things, the State of Pediatric Cancer Research and Advancing Pediatric Cancer Research Through Advocacy. If you are interested in attending 2/21/14 in person, please RSVP by emailing Nichelle Lewis at: or by calling (301) 594-9896. You can also participate via the webcast, visit: on Feb. 21 to watch the meeting.

And here’s an update on a drug shortage that continues for a frontline drug against childhood leukemia: The drug, daunorubicin is produced by only one company, Teva Pharmaceuticals, since the closure late last year of the only other company that made drug. At about this same time, Congress passed a law allowing compounding pharmacies to make custom generic medications that are in short supply, including pediatric cancer drugs. In exchange for the allowance to make such drugs, compounding pharmacies must first register with the FDA.

This is a very good thing, because, as many of you may remember, in the fall of 2012 several people died when a medication they used to treat severe back pain was found to be heavily contaminated with a fungus that causes meningitis. The pharmacy, the New England Compounding Center, made thousands of medications, sending chills down the spines (literally and figuratively) of thousands of patients who depended on these medications, many of which were administered in hospitals. Compounding pharmacies were not regulated by the FDA. NECC was closed and many lawsuits continue.

In the meantime, any company (if there are any) will need to begin manufacturing daunorubicin, and receive all necessary FDA approvals, soon.  Teva recently recalled daunorubicin due to the “potential presence” of  “particulate matter” in the drug.  Teva expects only a limited supply of the drug will be available through at least June of this year.

~~~~~January 2014~~~~~

She lives! That is the best way to describe a new, experimental gene therapy for leukemia patients. Eight-year-old Emily Whitehead, of Phillipsburg, PA, was just days from dying. She had endured all of the available treatments, and all failed her. Then she was selected to be the first of 22 pediatric leukemia patients in a new trial of this new gene therapy. She is alive and cancer-free today, almost two years later!

Gene therapy is one of the most promising fields of childhood cancer research. In several trials of this new therapy, nearly all of the patients who received it went into complete remission. Sadly some have relapsed, but some are still cancer free.

“It’s really exciting,” said Dr. Janis Abkowitz, blood diseases chief at the University of Washington in Seattle. “You can take a cell that belongs to a patient and engineer it to be an attack cell.”

“What we are giving essentially is a living drug” — permanently altered cells that multiply in the body into an army to fight the cancer, said Dr. David Porter, a University of Pennsylvania scientist who led one of the studies.

At least six different groups of researchers are studying this therapy, and more than 120 patients have been treated. More clinical studies are proceeding and more results will be announced in the near future. So far the results are so miraculous this may become the first gene therapy approved in the United States and the first for cancer worldwide!

~~~~~November 2013~~~~~

Here we go again…..Another drug used to help child cancer patients (leukemia patients) is in desperately short supply! Daunorubicin is a first-line drug for kids with AML. Last year a similar shortage happened with methotrexate, another critical drug in the fight against childhood leukemia. They help save children’s lives!! And yet drug-makers continue to drop off their production of them because they do not generate millions in profits. They are not “designer drugs,” or drugs used by millions of patients, thus big pharmaceutical companies have been moving away from their production. Once again it will take action by Congress to get these companies to continue manufacturing these life-saving drugs. Please contact your senators today. Go here to find out who your senators are. 

Here’s a great idea to help with your Christmas shopping this year. You can give meaningful gifts that truly make a difference for childhood cancer patients and the researchers working to find cures for the diseases that kill them. Go here to shop for gifts, and a great cause!

~~~~~October 2013~~~~~

Early in October doctors announced that a new kind of ultrasound can be used to diagnose and monitor children’s cancers. This means that CT scans do not have to be used so often. CT scans deliever very high doses of radiation and even have been found to greatly increase a child’s risk for developing cancer! Reducing or even eliminating the use of CTs will be an important step in not only diagnosing and treating children with cancer, it  perhaps will reduce the incidence of children getting cancer from too much CT radiation exposure.

Contrast-enhanced ultrasound (CEUS) imaging “is emerging as a very good and safe mechanism for evaluating the effectiveness of cancer therapy in children without having to use CT, which exposes the child to ionizing radiation,” Dr. Jamie Coleman, a physician at St. Jude Children’s Research Hospital in Memphis. He collaborated on the study with Dr. Beth McCarville, the principal investigator.

Added benefits are that CEUS is easy to use and inexpensive, Coleman said.

The study is only in Phase 1 trials and there are many years ahead before CEUS will become (we can only hope) widely used.

~~~~~September 2013~~~~~

September is National Childhood Cancer Awareness Month!

This month there is an exciting announcement by Dr. James Olson, one of the doctors who took care of Anjuli, and who is one of the pre-eminent childhood cancer researchers in the world. It’s Project Violet – a new way to speed the development of new drugs and therapies to fight pediatric cancers.  Children with brain tumors, especially children with the type of tumor that killed Anjuli, brainstem gliomas, inspired the creation of Project Violet.

The program is led by some of the brightest young researchers in the field. It is focusing on natural elements in both plants and animals that have “learned” through thousands of years of evolution how to protect themselves and fight diseases. These compounds are called optides, and by harnessing their unique abilities, Dr. Olson and his team can study thousands of optides to speed the development of new drugs and treatments.

A great example of an optide and its potential to help cure disease is “Tumor Paint.” Dr. Olson discovered it by studying, of all things, scorpion venom. It glows. He and his team were eventually able to harness this capability and make the “paint.” It is applied to brain tumors and surrounding tissue during brain surgery. It clearly delineates tumor tissue from healthy brain tissue. This means surgeons have a much greater chance of removing every last spec of tumor, and protect healthy tissue. Tumor Paint is now being successfully used to help save children’s lives!

You can “adopt” a drug candidate for just $100 and become a “citizen scientist” to help fund the way to a potential cure through a new way of conducting research – a way that will eventually fulfill the promise of cures for these dreadful diseases.

Go here for more information. You will be inspired!!

~~~~~August 2013~~~~~

A new study shows that some cancer cells, including glioblastoma cancer cells (the kind that killed Anjuli) can be programmed to commit suicide. The study, headed by Wafik El-Deiry, an oncologist at Pennsylvania State University in Hershey, shows that a small molecule that already lives in people with healthy immune systems causes this suicidal effect when boosted.

The molecule, called TIC10, stimulates a gene that makes a protein – called TRAIL for short. What makes TRAIL so effective against brain tumors is that it is small enough to cross the blood-brain barrier, which blocks most other chemotherapy drugs. Once TRAIL is activated inside a cancer cell, it dies. And there’s an additional exciting benefit – the cells surrounding the cancer cells are also stimulated to better fight cancer cells!

“We didn’t actually anticipate that this molecule would be able to treat brain tumors — that was a pleasant surprise,” says El-Deiry.

~~~~~July 2013~~~~~

There is exciting news this month for an aggressive form of childhood cancer that strikes tissues throughout the body. Called Rhabdomyosarcoma, (rhabdo), it usually occurs in children under six years old, and is often deadly.

But now, fruit flies are coming to the rescue! Yes, fruit flies. Dr. Rene L. Galindo, MD, PhD, Assistant Professor of Pathology, Molecular Biology, and Pediatrics at UT Southwestern Medical Center in Dallas, has discovered that gene in the flies can actually turn cancerous cells back to normal! It can also prevent healthy cells from becoming infected! He tested this not only in the flies, but also in humans and got the same results!

The next step is human clinical trials. If the trials prove successful, the new treatment could be a cure! And the treatment, called gene silencing would be much, much less harsh than years of chemo, radiation and surgery.

~~~~~June 2013~~~~~

Targeted gene therapy, a relatively new but now preferred weapon in the fight against childhood (and adult) cancer, finally can now target certain brain tumors, a new genetic study shows. Gene therapy that fine-tunes chemos for an individual’s specific brain tumor greatly improves survival!

The study was conducted at Children’s Hospital of Philadelphia (CHP). “By better understanding the basic biology of these tumors, such as how particular mutations in the same gene may respond differently to targeted drugs, we are moving closer to personalized medicine for children with cancer,” said the study’s co-first author, Angela J. Sievert, MD, MPH, an oncologist at CHP. Dr. Sievert believes the study could build a foundation for multicenter clinical trials.

Unfortunately the new research only focused on low-grade gliomas.

~~~~~May 2013~~~~~

Researchers have learned that beta-blockers (drugs normally used to treat high-blood pressure and other cardiac ailments) might enhance the ability of chemos to fight neuroblastoma (cancer in nerve cells) in children. In a study by the Children’s Cancer Institute of Australia, beta-blockers slowed the growth of neuroblastoma cancer cells when used with chemos, in mice. The study was reported last week in the British Journal of Cancer.

The study lead author Dr. Eddy Pasquier, of the Tumour Biology and Targeting Program at CCIA, said: “When combined with these beta-blockers, vincristine was four times more effective than when used alone.”

Work is continuing to see if someday the combination can be used to successfully treat patients.


And sadly, here is the update to the FDA’s quest to develop a list of the top diseases the pharmaceutical industry will focus on for the next five years. Incredibly, childhood cancer DID NOT make the list. In fact, no major childhood diseases made the list. In my own opinion, such ……stupidity, frankly, will rob thousands of children of their lives and will devastate thousands of families.

~~~~~April 2013~~~~~

The Children’s Hospital of Philadelphia and the University of Pennsylvania will announce this week a new therapy for acute lymphoblastic leukemia (ALL), one that recently achieved two complete remissions in relapsed disease.  Relapsed ALL is particularly deadly.

In the trial, two young girls’ immune systems were reengineered to attack and kill leukemia cells by destroying a certain protein. This type of treatment is called immunotherapy and is rapidly advancing in research centers and some hospitals nationwide. One of the girls, Emily Whitehead, age 7, reached full remission and is still cancer free today about a year after treatment. The other girl, age 10, also had a complete remission, but sadly her cancer re-emerged with different proteins not targeted in the study.

The doctors plan to target multiple proteins as the research moves forward. “The emergence of tumor cells that no longer contain the target protein suggests that in particular patients with high-risk ALL, we may need to broaden the treatment to include additional T cells that may go after additional targets,” said. Dr. Stephen A. Grupp, one of the co-leads of the study. “However, the initial results with this immune-based approach are encouraging, and may later even be developed into treatments for other types of cancer.”

Dr. Grupp is director of Translational Research for the Center for Childhood Cancer Research at The Children’s Hospital of Philadelphia, and a professor of Pediatrics at the Perelman School of Medicine at the University of Pennsylvania. The study also was led by Michael Kalos, Ph.D., an adjunct associate professor in the department of Pathology and Laboratory Medicine at the same school.

~~~~~March 2013~~~~~

March has been filled with desperately needed childhood cancer research awards to researchers and hospitals nationwide. These donations come from the St. Baldrick’s Foundation, an organization dear to my heart as I have had the privilge to co-chair several events that have raised more than $100K. St. Baldrick’s is the largest private funder of childhood cancer research. When added together, all of the private childhood cancer research foundations support more research than the National Institutes of Health! Please support them.

Some of the St. Baldrick’s awards:

$110K to various young scientists and researchers who are breaking new ground. Some went to researchers at:

Fred Hutchinson Cancer Research Center,

Seattle Children’s Hospital Los Angeles

c.)        Dana-Farber Cancer Research Institute, Boston

d.)        University of TexasM.D.AndersonCancerCenter, Houston

e.)        University of Hawaii, JohnA.BurnsSchool of Medicine

f.)         VanderbiltUniversitySchool of Medicine, Nashville

  • $168,115 as an infrastructure grant to the Cardinal Glennon Children’s MedicalCenter in St. Louis
  • $50K in research funds to Advocate Children’s Hospital in Oak Lawn, Ill
  • $50K in research funds to WayneStateUniversity in Detroit


Here’s the latest status on the FDA final list of the diseases the pharmaceutical industry will focus on for the next five years – it is still not published. According to Stephanie Yao, spokeswoman for the FDA, there is not ETA on when it will be done.


Lastly, Childhood Cancer Action Days in Washington, DC are June 17-18. Families struck by childhood cancer and advocates for increased funding will meet with members of Congress. The goal is to inspire them to act on behalf of childhood cancer patients, (those fighting now, those who lost their battles and those yet to be diagnosed) in any legislative issues, not the least of which is the budget.  For more info: contact Maureen Lilly at 202.336.8375 or

~~~~~February 2013~~~~~

There is a HUGE announcement this month in the fight against the most deadly form of childhood cancer – a brainstem glioma. This is what killed Anjuli and so many other precious young children. It strikes the very young and kills all of them. It is an abysmal diagnosis.

The breakthrough is a new SURGICAL method of delivering anti-cancer drugs directly to the brain stem!! This has been impossible until now!

Dr. Mark Souweidane, Director of Pediatric Neurological Surgery at the Memorial Sloan-Kettering Cancer Center and the Weill Cornell Medical Center, is the Principal Investigator. He has received FDA approval for a clinical trial for young patients diagnosed with Diffuse Intrinsic Pontine Glioma (DIPG). The trial will use convection-enhanced delivery, (CED) via a canula to deliver chemo right to the glioma! It thus penetrates the blood-brain barrier that prevents most drugs from reaching these horrific tumors.

The two-year study will involve only a small group of patients, but it is at least a chance of survival for these kids and possibly for others if the trial proves successful. It will take two years to complete.

The chemo is an antibody (produced by mice) that attacks many kinds of tumors. It will be combined with a radioactive drug that will kill the cancer cells that the antibody binds to.

Despite some other childhood cancers, DIPG research has been woefully under-funded, and that has meant almost zero progress in the fight against it. But Dr. Souweidane has worked on these beasts for more than 10 years and has finally found this potentially life-saving treatment. There are precious few other researchers working to fight DIPG. About 300 children are killed by it every year in the U.S.

“This trial is about renewed hope,” says Dr. Souweidane. “It’s a departure from the standard, ineffective, therapy, and has the potential to create a whole new paradigm in brain tumor treatment. Delivering drugs intravenously hasn’t worked because of the blood-brain barrier – to get even a small amount of medicine to the tumor we need high doses of chemotherapy, which is toxic to the rest of the body. But placing the agent outside the blood vessels, directly into the tumor, greatly reduces that toxicity while maximizing the attack on the tumor itself.”

Dr. Souweidane credits the financial support of private childhood cancer foundations that have enabled him to reach this milestone. Private childhood cancer research foundations are (and have been) the most important/major funders of research against childhood cancer. The National Institutes of Health spends only three percent of its cancer research budget on childhood cancer and the American Cancer Society, which unscrupulously uses children in its advertising, spends only two percent.

Dr. Souweidane invites those interested in the study to call his office at 212-639-7056, or visit

 ~~~~~January 2013~~~~~

In just the past couple of days, a chemo drug Gleevec was approved to treat a very deadly form of pediatric leukemia. This is the first time Gleevec was approved for these patients. Anjuli was on Gleevec under “Compassionate Use” (experimental use) back in 2001 as a last ditch effort to save her, but it did not help. Since then much research has been done, leading to this breakthrough. Gleevec blocks the proteins that cause the development of certain cancer cells. In the small clinical trial that led the FDA to approve this use of Gleevec, 70% of the children given it for the longest time in the study survived for up to 4 years! This is a huge improvement over previous treatments for this type of ALL. (Gleevec is made by Novartis.)


Also, here is another opportunity for you to get involved and make a difference for childhood cancer research. Please ask your senators and congressional reps to sponsor a new bill called “Trevor’s Law” (SB S.76). The bill is largely the result of a courageous young man, Trevor Schaefer, who beat the odds and survived brain cancer. It is designed to authorize federal agencies to form partnerships with states and universities/colleges to investigate childhood cancer “clusters,” where greater than average numbers of children have been diagnosed with cancer. Please help in this effort. You can find your senators and congressional reps easily by going to these websites. and

You can write to them easily from there! Thank you!

~~~~~December 2012~~~~~

There have been so many new and outstanding developments in the world of childhood cancer research this month it has been hard to choose one to highlight. Many of these developments have been widely reported in the media, so I am choosing one that has not been.

That said, it is an important advancement! Researchers at the University of New South Wales’ Australian Centre for Nanomedicine have announced a potential break-through in the search for less-toxic treatments and possibly a step toward a cure for neuroblastoma in children. For the first time in the world, they developed a nanoparticle that greatly increases the effectives of chemo, while allowing for the dosage to be dramatically lowered. This reduces the side-effects of chemo, and makes treatments easier to endure.

“By increasing the effectiveness of these chemotherapy drugs by a factor of five, we can significantly decrease the detrimental side-effects to healthy cells and surrounding tissue,” Dr. Cyrille Boyer said.

The nanoparticle will next be tested on lung and colon cancer cells. The hope is to eventually proceed to testing in humans. The findings have been published in the journal Chemical Communications.


I also want to provide an update on the FDA’s push to create the final list of drugs to be focused on by pharmaceutical companies for the next four years. The list will only include 20 drugs, although federal legislators are pushing for more.

Public hearings have been held, but as you know, the wheels of government turn slowly. According to a transcript from one of the hearings, there is no promise to complete the list by the end of the year, (surprise, surprise) but they want to “do it soon.” Once finalized, the list will be posted on the FDA’s website.

You can be sure I will be monitoring this. I will let you know when something happens, so stay tuned!

~~~~~November 2012~~~~~~

St. Jude Children’s Research Hospital – Washington University Pediatric Cancer Genome Project will announce tomorrow they have identified a fusion gene that causes almost 30% of a subtype of Acute Myeloid Leukemia that has a very poor prognosis.

Acute megakaryoblastic leukemia (AMKL), a subtype of AML, accounts for about 10% of patients with AML.  The finding provides evidence of a mistake that causes a significant percentage of such cases in children. The discovery paves the way for desperately needed treatment advances.

The discovery will “help guide treatment and more effective therapeutic interventions for this aggressive childhood cancer,” said James Downing, M.D., St. Jude scientific director and the paper’s corresponding author. The first author is Tanja Gruber, M.D., Ph.D., an assistant member in the St. Jude Department of Oncology.

The study is part of the Pediatric Cancer Genome Project, a three-year partnership between St. Jude and Washington University. The project is sequencing the complete normal and cancer genomes of 600 children and adolescents with some of the most aggressive and least understood cancers.

“Whole genome sequencing has allowed us to detect alterations in cancer cells that were previously unknown. Many of these changes contribute directly to the development of cancer,” Gruber said. “Such sequencing also provides the deeper understanding of the disease that is critical for developing more effective, less-toxic targeted therapies.”


Other news this month:

     The Food and Drug Administration is currently developing its disease-focus list for the agency’s Patient-Focused Drug Development Initiative. The initiative will work with, and encourage, pharmaceutical companies to develop new treatments for childhood cancer. The list will be short. If childhood cancer is not on the list, it will not be included and the pharma industry will once again ignore drug development for kids who are suffering and dying. Only two new drugs have been developed for kids with cancer in the last 20 years! You can make a difference! Send a personalized letter to the FDA to ask them to please put childhood cancer on the list. You can copy and past my letter and simply personalize it to suit your situation. Please do it very soon! This is critically important to saving kids’ lives!
Add salutation: Margaret Hamburg, MD; Commissioner; FDA, 10903 New Hampshire Ave., Silver Spring, MD 20993
As the mother of a young child who died of an incurable brain stem cancer (Anjuli 11/24/96-6/22/01 – I ask you to please include childhood cancer research in the permanent list of the Food and Drug Administration’s disease-focus areas for the agency’s Patient-Focused Drug Development Initiative.
As I am sure you know, childhood cancer is the leading cause of disease-related death among children and adolescents in the United States. Every year, approximately 13,500 children are diagnosed with cancer and 2,500 children die from these diseases. Unfortunately, the causes of most childhood cancers are unknown and the diseases cannot be prevented. Additionally, there are also 350,000 childhood cancer survivors currently living in the United States, and each of these children, adolescents, and young adults have unique physical and mental health care needs that will impact them for the rest of their lives. About two-thirds of childhood cancer survivors will experience serious, late, long-term impacts of their cancer treatments, including second cancers, physical and intellectual developmental issues, heart and lung damage, osteoporosis, financial pressures, psychosocial complications, employment and fertility problems, and many other issues.
And because children with cancer are relatively young when diagnosed, they experience a much greater loss of lifetime productivity and economic contributions compared to adults with cancer.
Unfortunately, despite the severity of childhood cancers and the long-term side effects of childhood cancer treatments, pediatric cancer has not been adequately addressed. The relatively small population of children with cancer provides little market incentive for the biopharmaceutical industry to develop new pediatric oncologic therapeutics. Only two new drugs have been approved for childhood cancers in the last two decades! Surprisingly, the importance and compassion of saving children’s lives is not enough for these companies! Today, children with cancer are treated with drugs that were developed for adults several decades ago! This is unacceptable for a country as generous and strong as the United States. We should be a leader in this field!

~~~~October 2012~~~~~

Earlier this month, Ian Pollack, M.D. of Children’s Hospital of Pittsburgh was awarded a prestigious award from the National Brain Tumor Society for his work on a potential vaccine therapy for children with a type of brain tumor called a glioma. This is the type of tumor that killed Anjuli. The award was presented at the 2012 Congress of Neurological Surgeons’ annual meeting in Chicago.

The small study of 27 young patients examined a peptide vaccine designed to stimulate an immune response to a protein fragment on their tumors’ cells. Over the three-month course of the study, 15 children had stable disease, three had “sustained partial responses,” and one had a longer-term disease-free response after under going surgery. Sadly, three children had rapidly progressing disease. An immune-response analysis was completed in seven the patients; and found six had definite immune responses.

“This was the first study of its type that examined peptide vaccine therapy for children with brain tumors like this, and the fact that we are now seeing tumor shrinkage is extremely encouraging in moving forward with this therapy,” Dr. Pollack said.

Dr. Pollack is chief, Pediatric Neurosurgery at Children’s Hospital’s Brain Care Institute and co-director of the University of Pittsburgh Cancer Institute’s Brain Tumor Program. Regina I. Jakacki, M.D., director of Pediatric Neuro-Oncology, also directed the study. The team is hoping to advance this study to a multi-center trial within the Pediatric Brain Tumor Consortium.

~~~~~September 2012~~~~~

September is childhood cancer awareness month! Wear gold!

Earlier this year, childhood brain cancer researchers announced the discovery of unique genetic mutations that lead to the formation of the deadliest type of cancer, an intrinsic brainstem glioma, the type of cancer that killed Anjuli. These mutations were previously thought not to be associated with cancer. Suzanne Baker, Ph.D., co-leader of the St. Jude Neurobiology and Brain Tumor Program and a study co-author, said: “We are hopeful that identifying these mutations will lead us to new selective therapeutic targets, which are particularly important since this tumor cannot be treated surgically and still lacks effective therapies.” Intrinsic brainstem gliomas account for 10%-15% of all pediatric brain tumors.

The breakthrough is due in large part to the Pediatric Cancer Genome Project, a three-year, $65 million mission to sequence major pediatric cancers that was launched in 2010 at St. Jude Children’s Research Hospital. The study “suggests these particular mutations give a very important selective advantage, particularly in the developing brainstem and to a lesser degree in the developing brain, which leads to a terribly aggressive brain tumor in children, but not in adults. This discovery would not have been possible without the unbiased approach taken by the Pediatric Cancer Genome Project. The mutations had not been reported in any other tumor, so we would not have searched for them in (these tumors).” Baker said.

Now that these mutations have been found, researchers can better target their efforts to find new therapies to help the children afflicted with this most deadly of all cancers.

Credit: Petra Rattue. (2012, January 31). “Childhood Brain Tumors Linked To Newly Discovered Mutations.” Medical News Today. Retrieved from

~~~~~Updates from Summer 2012~~~~~

The St. Baldrick’s Foundation announced in August that one of its many grants to childhood cancer researchers has helped Dr. Charles Mullighan, M.D., Ph.D., associate at the St. Jude Department of Pathology, to find a new treatment for a type of leukemia (Ph-like ALL) that is a subgroup of the most common form of childhood cancer. It has a high rate of relapse and poor survival. It makes up 15% of all childhood ALL.

The new development, for the first time, identifies the genetic alterations found in the growth of leukemia cellsin this type of cancer. What’s more is they found that existing drugs, Gleevec (which my Anjuli took as part of compassionate use when it first was developed) and dasatinib, currently used in therapies for other types of leukemias, could block the cells’ growth.


In July, President Obama signed into law the FDA Reform Act, which, among other things, will help address the severe and life-threatening drug shortages that have plagued many types of important, though less-expensive, drugs needed by children in their fights against childhood cancer, and also by other patients fighting other deadly diseases. One of these drugs, methotrexate, is a common drug therapy for childhood cancer patients and it’s scarcity sent chills down many patients’, parents’ and doctors’ spines.

What caused the shortages?  There is little profit for drug companies to develop drugs for childhood cancers. This is why very few childhood cancer drugs have been approved by the FDA in the last 20 years! But now, as a result of the Creating Hope Act, which is part of the FDA Reform Bill, drug companies will receive vouchers to expedite the review process for their profitable drugs if they research and develop drugs for rare diseases, such as childhood cancers. It should be noted that the Creating Hope Act was substantially championed by Nancy Goodman, Founder and Executive Director of Kids v Cancer, in honor of her son, Jacob Froman, who died from medulloblastoma (a type of brain cancer) at age 10.


In June, St. Baldrick’s introduced Dr. Lionel Chow, MD, PhD, one of their scholars, who is dedicated to research to fight the most-deadly type of tumor that killed my Anjuli, a brain stem glioma, also known as a pontine glioma. These tumors cannot be operated on due to their location, so these patients have an awful prognosis. Sadly, the current treatments, while sometimes life prolonging, do not save lives. Research aimed at these tumors is critical.

Dr. Chow uses laboratory models that strongly resemble these tumors to study their biology. These models can be used to help design and test different ways to treat high-grade glioma. I would like to add that Dr. James Olson, MD PhD, at the Seattle Cancer Care Alliance, who is pivotal in the Anjuli Jacobs Endowment, is also researching these gliomas and has used donated tumor tissue from patients, including Anjuli, who have died.


Also in June, St. Baldrick’s announced a significant breakthrough in the fight against osteosarcoma, a disease that often strikes children, usually teens and tweens, when their bones are growing rapidly. Since it’s normal for adolescents to experience “growing pains”, they are often diagnosed late, when their cancer is aggressive. And despite improvements in chemo and surgery, 40% of these patients die. But this recent study, funded in part by St. Baldrick’s, has identified a new gene called Sox2 that osteoscarcomas require. When Sox2 is blocked, osteosarcoma cells start acting like normal bone cells, according to Dr. Alka Mansukhani, MS PhD, NYU, who led the research.

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