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 heart is in this because my firstborn, 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 email@example.com and I will add you to the list. And please share this site with as many people as you can. Thank you!!
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: firstname.lastname@example.org 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:
a.) FredHutchinsonCancerResearchCenter, Seattle
b.) 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 email@example.com
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
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.
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.