National Brain Tumor Society is one of many disease-based organizations to join the chorus of supporters of stronger funding for the National Institutes of Health (NIH). The agency – through the National Cancer Institute (NCI) and other institutes – is the largest funder of brain tumor research in the country. Dwindling investment in this agency has broad implications to the future of medicine and disease in the United States, and has been a widely discussed topic.
But the effects the current funding climate for NIH and NCI is having on children with cancer, specifically, has not often been highlighted. With National Brain Tumor Society staff in Washington, D.C. this week participating in both the Alliance for Childhood Cancer Action Days, and Coalition Against Childhood Cancer Annual Membership Meeting, there is no better time than now to cast a light on this issue.
The potential implications to pediatric cancers from shrinking federal funding are even more amplified than in the general population. This is because:
- The small and vulnerable patient populations in pediatric oncology make childhood cancer an unattractive investment for biopharmaceutical companies.
- Whereas approximately 60% of funding for adult clinical research comes from the biopharmaceutical sector, the private sector contributes almost 0% of the investment in pediatric oncology, leaving NCI as virtually the only source of funding for clinical trials in children with cancer.
- Unlike adult cancer clinical trials – where only about 3% of the patient population participants in clinical trials – a majority of pediatric cancer patients (~60%) are treated via clinical trials.
If most children with cancer receive treatment through clinical trials…and the NCI pays for almost all of the pediatric clinical trials through its cooperative groups…what happens when the NCI has to cut funding for the cooperative groups? Given our country’s almost universal social value in protecting children, these questions should be of great public concern.
Children’s Oncology Group (COG) is the pediatric cancer clinical research arm of the NCI. COG operates under the NCI’s National Clinical Trials Network. COG’s Group Chair (Peter Adamson, MD) sent out a letter at the end of May explaining the cooperative group’s current funding situation to patient advocates:
For childhood cancer…the private sector has an almost negligible investment, resulting in virtually all research funding emanating from the NCI, with lesser but vital components of funding emerging from private foundations and other philanthropic sources…There is no second revenue stream from the biopharmaceutical industry capable of supporting a childhood cancer clinical trial program in this country, [thus] any cut from the NCI to childhood cancer research is magnified. Simply put, the clinical childhood cancer research enterprise is essentially entirely dependent upon funding from the NCI to conduct clinical-translational research. Funding to the COG has declined significantly over the past 10 years. Since 2004, adjusting for inflation, COG incurred a 30% decrease in its base funding from NCI. This steady erosion of research support, amplified further by 2014 budget cuts, will delay progress in improving the outcome for a spectrum of cancers that effect children.
In FY 2014, COG had to take a 6% cut, which equates to $2 million dollars lost. Through some creative work within the budget, COG has been able to avoid shutting down any ongoing clinical trials for now. That’s the good news. However, the group will not be able to initiate any new clinical trials to capitalize on the continually improving science happening in the post-genomic era.
Further, the NCI-supported Pediatric Preclinical Testing Platform (PPTP) will be taking a cut of about 25%, which Dr. Malcolm Smith of the NCI’s CTEP (Cancer Therapy Evaluation Program) revealed on an April 22, 2014 teleconference with advocates, is causing “some work to stop.” Though little known, PPTP is a very important component of the pediatric oncology drug development pipeline. This platform of models and assays has been one of the very few adequate pre-clinical drug-testing platforms in the pediatric drug development space. For example, when the National Brain Tumor Society hosted a working group meeting with leading pediatric cancer researchers, representative from many major biopharmaceutical companies, the NCI, and the FDA in March to discuss strategies to improve drug development for pediatric cancers (particularly pediatric high-grade gliomas) the group agreed that PPTP is one of the very limited platforms that provides quality pre-clinical data.
Dr. Maryam Fouladi, MD, Medical Director of the Neuro-Oncology Program at Cincinnati Children’s Hospital Medical Center and Director of the COG-associated Pediatric Brain Tumor Consortium (PBTC), spoke of what this means for pediatric brain tumor drug development recently via email: “[This] means that drugs that are of interest will not have appropriate preclinical testing conducted to allow us to take them forward in Phase I [trials].” And, further, that drugs that may have already gone through a Phase I via the PBTC, may not have anywhere to progress to with COG not initiating any new trials*.
Yet, unlike some other common forms of pediatric cancers that have seen progress in recent years, the survival rates for many types of pediatric brain tumors (like AT/RT, DIPG, and pediatric glioblastoma multiforme) are still staggeringly low. The ability to adequately test and move new brain tumor treatments forward is desperately needed. But advances in science will only translate into improved outcomes for children with cancer through productive R&D efforts. And until the biopharmaceutical industry begins investing more heavily in pediatric cancer, adequate clinical research for childhood cancer will only happen via the NCI. (And Industry won’t invest without what they deem reliable pre-clinical data, which right now is also coming from NCI-funded groups.)
“Clearly, [this] makes the [National Brain Tumor Society’s] pre-clinical initiative more important,” noted Dr. Foualdi, in reference to the pre-clinical component of the National Brain Tumor Society’s newest pediatric drug development initiative, Project Impact. Project Impact aims to leverage the pediatric cancer community, the biopharmaceutical industry, and government on a integrated (pre-clinical research/drug testing and clinical research policy), full-scale research and drug development project to aggressively target brain and other pediatric cancers for therapeutic development.
National Brain Tumor Society is committed to driving change in the pediatric brain tumor and cancer research and drug development landscape. Now Congress we ask to signal a commitment, too: http://www.soundoffatcongress.org/nY2f
*PBTC’s mandate is to do Phase I and pilot studies. COG is the natural outlet to move the Phase I studies PBTC does forward in larger, later stage trials and allows potential new brain tumor drugs to reach as many children as possible.