Childhood Cancer Research News
Welcome to my childhood cancer awareness blog.
Thank you for visiting!
I hope to spread 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. 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 firstname.lastname@example.org and I will add you to the list. And please share this site with as many people as you can. Thank you!!
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: http://www.firstgiving.com/fundraiser/MarciaJacobs/run-of-hope-seattle-2016
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 know 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.
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: http://www.cbsnews.com/news/60-minutes-fda-breakthrough-status-duke-university-cancer-therapy/.
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.
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.
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.
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.
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.
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.”
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 http://stepupforchildhoodcancer.com/.
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.
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.
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 is National Brain Tumor Awareness Month. Read more about Anjuli at Project Violet. https://www.projectviolet.org/people
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.
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.
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?
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
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: http://curechildhoodcancer.ning.com/forum/topics/pac2-update-february-2014?xg_source=msg_mes_network
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 http://www.clinicaltrials.gov
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 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: http://www.firstgiving.com/fundraiser/MarciaJacobs/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.
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
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: http://curechildhoodcancer.ning.com/forum/topics/pac2-update-february-2014?xg_source=msg_mes_network
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? http://childhoodcancer-mccaul.house.gov/ . 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.
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.
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: email@example.com or by calling (301) 594-9896. You can also participate via the webcast, visit: http://videocast.nih.gov/ 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.
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!
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. http://www.senate.gov/general/contact_information/senators_cfm.cfm
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! http://curechildhoodcancer.ning.com/page/6th-annual-pac2-holiday-shopping-guide-2013?xg_source=msg_mes_network
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 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 https://www.projectviolet.org for more information. You will be inspired!!
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.
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.
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.
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.
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.
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
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 firstname.lastname@example.org
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 www.clinicaltrials.gov.
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. www.senate.gov and http://www.house.gov
You can write to them easily from there! Thank you!
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!
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 – http://www.anjulijacobs.com) 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!
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 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 http://www.medicalnewstoday.com/articles/240977.php
~~~~~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.