Q&A: Working to deal a critical blow to brain cancer in kids

National Brain Tumor Society talks about its part in launching Project Impact and what that means to the landscape of pediatric brain tumor treatment

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On Sept. 12, the National Brain Tumor Society (NBTS) hosted an event at the National Press Club in Washington, D.C,. (also streamed live) featuring leaders in the field of pediatric brain tumor research. The purpose was to announce the launch of Project Impact: A Campaign to Defeat Pediatric Brain Tumors.
As NBTS noted in an email to DDNews, “No drug has ever been developed specifically to treat pediatric brain tumors, so why are these most vulnerable cancer patients being overlooked?” The purpose of the launch event was to give people the chance to learn why, despite so many challenges and disincentives for R&D in the pharma and biotech industry, there is new hope for these pediatric brain cancer patients and their families.
NBTS says it is the largest nonprofit dedicated to the brain tumor community in the United States and in this effort it its partnered with the St. Baldrick’s Foundation and several world-renowned experts in the field of pediatric neuro-oncology. The new project will consist of an awareness and fundraising campaign to support a major translational research and drug discovery program. Goals of the program include improving clinical outcomes for pediatric brain tumor patients and informing the development of the first-ever standard of care for treating pediatric high-grade gliomas.
We spoke with David Arons, CEO of NBTS, to find out more.
DDNews: Brain tumors have the dubious honor of being the most prevalent form of pediatric cancer as well as having taken first place away from leukemia as the deadliest form of cancer for kids under age 14. As far as that second distinction, is the change in deadliness more about brain cancer itself, or improved therapeutics in leukemia compared to brain cancer?
David Arons: The CDC’s study which found that pediatric brain tumors replaced leukemias as the deadliest cancer in children and adolescents was based on survival trends from 1999-2014. During this time, pediatric brain tumors did not become inherently more deadly. Nor did these tumors become more prevalent. As the report makes clear, the reason brain tumors replaced leukemias as the deadliest pediatric cancer is simple: children with leukemia have benefited from tremendous advances made in science and medicine over the past five decades. Their survival rates have improved, in some cases dramatically. Meanwhile, survival rates for the pediatric brain tumors that weigh most on the mortality rates in this field—pediatric high-grade gliomas—have not improved, really, since the 1970s. These tumors have never had a drug developed specifically for them, and there is no standard of care. Even chemotherapy is virtually ineffective against these tumors.
DDNews: Is the lack of research into therapeutics for pediatric brain cancer just about the difficulties of including children in clinical trials, or does it permeate other parts—perhaps even all parts—of the discovery-through-trials pipeline?
Arons: There is really a convergence of challenges that make the entire pediatric brain tumor R&D landscape very arduous. A lot of it stems from three major hurdles: first, that even though pediatric brain tumors are one of the most prevalent pediatric cancers, it is still a relatively small patient population; second, these tumors’ location, in the fragile developing brain, make treatment and research difficult; and third, ethical considerations and protections our society affords children, and particularly how we protect children from research, create an added layer of complexity.
For example, as is the case in any disease area, tissue samples and biospecimens are critical to research. At this point, really the only way to collect biospecimens needed for research purposes in pediatric brain tumors is through biopsy. However, in many situations a brain tumor’s location precludes a biopsy because of the risk of any surgical intervention in the brain. This is heightened in children because of the sensitivity of their healthy brain tissue and medical ethics about performing procedures on children that do not directly correspond to treating that child. Because of this challenge collecting tissue, combined with the fact that the small patient population limits collection already to begin with, most researchers—spread out across the country—don’t really have the samples necessary to conduct well-powered research studies or create good preclinical models. This can create a disincentive for biopharmaceutical companies from even wanting to provide their compounds for preclinical screening.
DDNews: Describe Project Impact—how it came about, its partners and its goals.
Arons: Project Impact: A Campaign to Defeat Pediatric Brain Tumors is a capital campaign. Project Impact will ultimately resource a new research program organized by NBTS called the Defeat Pediatric Brain Tumor Research Collaborative.
Project Impact, as a capital campaign or fundraising component, seeks to initially raise at least $2.5 million in specialty gifts over five years to fund the launch of scientific projects within the Defeat Pediatric Brain Tumors Research Collaborative, with an ongoing aim to ultimately invest more than $5 million, from multiple sources, in the program. The St. Baldrick’s Foundation has already come on as an initial partner for the Defeat Pediatric Brain Tumors Research Collaborative and we welcome more partners.
The goal of the Defeat Pediatric Brain Tumors Research Collaborative is to enable the development of a first-ever standard of care for treating pediatric high-grade gliomas, including DIPGs, in order to extend and improve the lives of our youngest and most vulnerable of patients.
Three years ago we became acutely aware of the barriers to progress in medical research related to the deadliest of pediatric brain tumors, including the lack of available drugs, lack of high-quality models to evaluate new drugs before clinical trials and the lack of incentives for biopharmaceutical companies to invest in childhood cancer research. So we brought biopharmas together with patient advocates, with parents, with the FDA, with researchers and asked what could we do that can make progress and improve the way research is done. The result is the newly launched collaborative that brings together leaders across phases of research to work simultaneously in a rapid learning environment aimed at moving discoveries to clinical trials and forward. The collaborative is designed to accelerate research through a platform that fosters collaboration and data, information and material sharing.
The Defeat Pediatric Brain Tumor Research Collaborative involves the following leaders:
Researchers leading each Core include:
  • Discovery Core (Molecular Diagnostics and Target Discovery): Dr. Stefan Pfister, German Cancer Research Center
  • Biomarker Core (Identification and Validation—Tissue & Serum): Dr. Nada Jabado, Montreal Children’s Hospital
  • Preclinical Modeling and Drug Screening Core: Dr. Suzanne Baker, St. Jude Children’s Research Hospital
  • Smart Trials (N-of-1 Clinical Trials): Collaboration with the Pacific Pediatric Neuro-Oncology Consortium
The Collaborative is then overseen by a scientific director who leads a strategic scientific advisory council (SSAC). Serving as scientific director will be Dr. Roger Packer of Children’s National Health System. The rest of the SSAC includes luminaries in the world of pediatric neuro-oncology:
  • Dr. Susan Blaney: Deputy Director of the Texas Children’s Cancer and Hematology Centers, executive vice chair of the Department of Pediatrics at Baylor College of Medicine (BCM), vice president of clinical and translational research at BCM and the Martha Ann and Harold M. Selzman, M.D. Endowed Chair at the Institute for Clinical and Translational Research at BCM
  • Dr. Richard Gilbertson: Li Ka Shing Chair of Oncology and director of the Cambridge Cancer Centre in England
  • Dr. Scott Pomeroy: Neurologist-in-chief and chairman of the Department of Neurology at Boston Children’s Hospital, a consultant in pediatric neuro-oncology at the Dana-Farber Cancer Institute and the Bronson Crothers Professor of Neurology at Harvard Medical School
  • Dr. Raphaël Rousseau: Group medical director  and global franchise head for pediatrics at Genentech, a member of the Roche Group
  • Dr. Robert Wechsler-Reya: Director of the Tumor Initiation and Maintenance Program at Sanford-Burnham Medical Research Institute
The model/concept we are using for research in the Defeat Pediatric Brain Tumor Research Collaborative was designed based on consensus feedback from a large cross-section of the pediatric neuro-oncology community. Going back to 2012-2013, a number of small-group, large-group and individual meetings were held to assess the landscape, followed by a number of workshops and working group meetings in 2014 and 2015. 
DDNews: In what ways do you think Project Impact can truly move the needle on pediatric brain tumor research? Where do you see having the most impact? What kinds of new models of collaboration and data-sharing within the pediatric neuro-oncology community do you envision?
Arons: To begin with, we feel we have the right people, working on the right projects, being advised by the right experts. 
Then we believe we have an infrastructure in place that makes it easier for these experts to work together and share data and results without some of the typical constraints.
The research program is based on NBTS’ “Defeat” program model, which is a novel business approach to science. The “Defeat” model is defined by two major characteristics:
  1. A collaborative scientific research structure that consists of multiple interrelated “Cores” that work on critical areas of research simultaneously and share data, information, and materials between them in real-time. The “Cores” design allows new findings in one area of the collaborative to quickly move on to its next stage of research without barriers or typical delays. This speeds the whole research process significantly.
  2. A research management model that facilitates the collaborative’s operational and administrative needs so that researchers can spend more time in the lab and less time doing paperwork.
The researchers involved are all at the top of the field. They all have a collaborative spirit based on science and a common goal, and they are all leading specific scientific efforts in which they are specialists and leverage their individual strengths and collective expertise, at the same time. Then NBTS provides the infrastructure to move forward and foster collaboration and sharing.
DDNews: The capital campaign is important, of course, to fund the Project Impact efforts—and that will clearly be an ongoing effort, but how is it shaping up so far?
Arons: The public phase of the capital campaign was just announced on Sept. 12 at the National Press Club in Washington, D.C. This came after a few months of a “silent phase,” in which we did a lot of what could be described as “foundation building.” This included setting up a leadership structure for the campaign, developing levels of leadership gifts, investment and partnerships, and some of the other mechanics and materials we’ll need now in the public phase. Now we’re really ready to start building the rest of the house.
DDNews: In addition to the capital campaign, there is also the awareness initiative. Could you describe the plans for that in terms of strategies and next steps?
Arons: Traditionally, it has been challenging to fundraise in the pediatric brain tumor space. Historically, we just don’t see the major eight-, nine-, even 10-figure gifts specifically for pediatric brain tumor research that you hear about fairly often in the greater biomedical science and research philanthropic arena. And, again, because of the smaller patient population there isn’t a big natural pool of potential donors that have been impacted and are ready to give. This, of course, is a good thing, in general. But it does make fundraising more difficult. This necessitates that we create broader awareness in the general public about the plight these children and their families face. We are attempting to make the case through all the channels, vehicles, and tactics available to us (digital, social, earned and owned media) that funding pediatric brain tumor research—and specifically new approaches to it, like ours—should be a national priority. This is very arguably the biggest problem in pediatric oncology, because of the high mortality rates, that fact that it is one of the two most prevalent types, and the lack of progress seen over past decades, even compared to other cancer types. We want to make sure these kids see the progress they deserve.
A video of the event for the project launch can be viewed by clicking here.
A video about Project Impact can be viewed by clicking here.

David Arons is the CEO of National Brain Tumor Society (NBTS). He also serves as the NBTS’ chief public policy and advocacy officer. He previously served as the director of government relations for the American Cancer Society in Minnesota and was the co-founder and co-director of the Center for Lobbying in the Public Interest. He serves on the National Cancer Institute’s Council of Research Advocates and Clinical Trials Advisory Committee. In 2016, Arons was named to the Blue Ribbon Panel of experts selected to help advise the National Cancer Moonshot being led by Vice President Joe Biden.

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