Our Impact: Investing Your Dollars to Make a Difference for Brain Tumor Patients & Progress Toward a Cure
Over the last 25 years, the National Brain Tumor Society (and its two legacy organizations) have funded more than $35 million in brain tumor research grants and awards to hundreds of researchers at various leading institutes in the U.S. and globally in order to find better treatments, and ultimately a cure. Your support has also enabled nonpartisan advocacy efforts which improved public policies that benefit brain tumor patients and research.
The highlights below reflect the important progress achieved over our history thanks to the support NBTS has received from generous and dedicated donors, volunteers, advocates, grantees, and supporters. These specific results have – and will continue to have – tangible impact on the research needed to improve treatments for brain tumors, as well as showcases our work in influencing and driving public policy that gives our brain tumor community a voice in our nation’s capital.
MOVING POTENTIAL NEW MEDICINES FORWARD: NBTS-funded research has led to at least 12 therapies evaluated as potential new treatments for brain tumors in clinical trials. At least seven (7) of these experimental medicines are still advancing in active clinical trials for patients with brain tumors.
- Funded the early research that enabled the development of thepoliovirustreatment currently being evaluated in clinical trials by Duke University highlighted on “60 Minutes.”
- Funded the early research that allowed MD Anderson Cancer Institute scientists to develop an approach that uses a modified version of the common cold virus to attack glioblastoma cells.This research strategy is now being evaluated in clinical trials.
- Provided funding to a company called Tocagen for the development of a first of its kind potential new treatment for glioblastoma called “Toca 511/Toca FC” that uses a one-two-punch to destroy glioblastoma cells.This approach is now in a late stage clinical trial.
IMPACT: Should any of these make it through the clinical trial process successfully – proving they are safe and effective – they would become much needed new treatments for brain tumor patients.
BUILDING OUR UNDERSTANDING OF BRAIN TUMORS & CREATING THE FOUNDATION FOR NEW, MORE PERSONALIZED TREATMENTS:NBTS-funded research has contributed, or directly led, to the discovery of a number of the most important “molecular markers,” or tumor characteristics, that are essential for determining the future course of treatment.
- Discovery of theIDH mutation, which has become a critical means for neuro-oncologists to distinguish between different type of gliomas.
- Discovery of the EGFR mutations, which are the most common mutations found in glioblastoma and represent key targets for which many GBM treatment strategies are being developed.
- Discovery of the1p/19q co-deletion, which is the key characteristic for identifying and diagnosing low-grade gliomas, including oligodendrogliomas.
- Research leading to the identification of nearly two-dozen inherited genetic variants that may influence the risk of developing a glioma.
IMPACT: Better understanding of the underlying biology of brain tumors allows researchers and doctors to “look under the hood” of a brain tumor and understand their origins and how they function in order to know what can stop them. Further, precise classification of brain tumors allows researchers and doctors to better diagnose patients, plan treatment strategies, and make sure patients get the right treatment and right clinical trials at the right time.
DEVELOPING STRATEGIES TO DEFEAT Glioblastoma (GBM):Developed to take on the most prevalent and aggressive malignant brain tumor, NBTS’ Defeat GBM Research Collaborative has contributed to breakthrough research on glioblastoma tumor biology and treatment resistance and is bringing forth a host of both new targets for future medicines to attack and drugs of interest, with 4 potential candidatesidentified for evaluation in future clinical trials.
Key Examples of the results of the Defeat GBM Research Collaborative:
- Discovered how glioblastoma tumors depend on vast amounts of cholesterol, and how shutting down this supply may halt tumor growth. Importantly, the team has identified a drug of interest to test.
- Discovered a new mechanism whereby glioblastoma cells can repair themselves after radiation treatment, and are now testing for potential drugs that could act as “radio-sensitizers,” which would make glioblastoma tumors more sensitive and less resistant to radiation.
- Discovered that circular bunches of DNA not located in chromosomes (where DNA is typically found) can be found in high levels in glioblastoma tumor cells. These pieces of “extrachromosomal DNA” are now believed to be major contributors to tumor growth and treatment resistance. This discovery has the potential to significantly change the way we treat glioblastoma, based on where cancer-fueling genes are located.
IMPACT: Collectively, these discoveries present a multitude of potential new approaches for treating glioblastoma that are now being evaluated further.
DISCOVERING A NOVEL TARGET FOR THE WORST TUMORS IN KIDS: NBTS funded research led to the molecular characterization of the most aggressive and deadly pediatric brain tumors – pediatric high-grade gliomas.
- Launched a Pediatric Molecular Profiling initiative, which helped researchers discover the Histone 3.3 mutations, key drivers of pediatric high-grade gliomas.
- This research also highlighted the molecular differences between pediatric and adult brain tumors for the very first time – a seminal moment in pediatric high-grade glioma discoveries.
IMPACT: Armed with this new knowledge, we have launched a new initiative launched called the Defeat Pediatric Brain Tumors Research Collaborative, which is attracting strong partnerships, while helping to reinvigorate the pediatric brain tumors research field around the world in the quest to develop much-needed new medicines for this disease.
Advocacy and Public Policy
PUTTING BRAIN TUMORS ON THE MAP AT THE NATIONAL CANCER INSTITUTE:NBTS Public Policy & Advocacy efforts pushed for, and helped lead to, the creation of a dedicated “Neuro-Oncology Branch”(NOB)at the National Cancer Institute (NCI).
IMPACT: The NOB has become a key place to conduct important brain tumor research and for patients to get second opinions on treatment options.
INCREASING FEDERAL DOLLARS FOR BRAIN TUMOR RESEARCH:NBTS Public Policy & Advocacy efforts have helped increase the money spent on brain tumor research by the National Institutes of Health (NIH) from$280 million in 2011 to an estimated $335 million in 2017, a $55 million, or nearly 20%, increase.
IMPACT: More money available for brain tumor research increases the pace – and odds – of future groundbreaking discoveries that contribute to the quest for cures.
CREATING (AND PROTECTING) A NEW STREAM OF RESEARCH FUNDING:NBTS Public Policy & Advocacy efforts helped make pediatric brain tumors (and now all brain tumors, including adults) eligible for research funding through the Department of Defense’s Congressionally Directed Medical Research Program. Further, NBTS helped increase the pool of money this program makes available for researchers applying for grants.
IMPACT: A vital new stream of funding for the field is now available to help advance research efforts.
AMPLIFYING THE BRAIN TUMOR VOICE & TAKING THE FIGHT TO THE HALLS OF CONGRESS:NBTS launched a Public Policy & Advocacy Program that leverages the passion, determination, and dedication of brain tumor patients, survivors, caregivers, family, and friends and channels it into effective efforts to influence public policy that impact the brain tumor community.
- Built a grassroots army of volunteer brain tumor advocates from scratch, eventually growing the program to more than 41,000 registered advocates from all over the United States, who call, write, email, and tweet their members of Congress to educate them on the needs of the brain tumor community and ask for their support on key policy issues.
- Created the first and only annual brain tumor Advocacy Day at the Capitol in D.C. to flood the halls of Congress with hundreds of brain tumor advocates from across the country. Named, “Head to the Hill,” this yearly event draws more than 300 advocates to Washington DC each year to raise awareness and advocate for policies that will help brain tumor patients, survivors, and caregivers.
IMPACT: Brain tumor advocacy has successfully pushed policymakers in D.C. to introduce and advance numerous bills that benefit brain tumor patients and brain tumor research. (SEE BELOW)
INTRODUCING AND ADVANCING NEW POLICIES:NBTS Public Policy & Advocacy efforts helped introduce key legislation that stands to benefit the brain tumor community.
- Lead the charge to introduce oral chemotherapy parity legislation, and attract supporters to the bill.
- While working with coalition partners in the Alliance for Childhood Cancer, helped draft and advancing the Childhood Cancer STAR Act, which passed into law June 2018.
- Joined numerous other patient advocacy organizations across the disease spectrum to help successfully advocate for the passage of the 21stCentury Cures Act. The 21stCentury Cures Act make changes to the NIH and FDA to provide these agencies with new tools to speed up the development and ultimately approval of new potential treatments including those for brain tumors patients.
IMPACT: These bills would directly benefit the brain tumor community by making common treatments more affordable; accelerating pediatric brain tumor research efforts; and providing additional research funding and fostering innovation in biomedical research, respectively.
BUILDING AND FOSTERING THE BRAIN TUMOR RESEARCH FIELD:In its early days, NBTS and its legacy organizations helped build and develop the nascent neuro-oncology field.
- Provided a number of America’s best brain tumor researchers their first brain tumor research grants.
- Partnered with the American Association of Cancer Research (AACR) for years to award the NBTS-AACR “Career Development Grant” for early-career brain tumor researchers.
- Provided early support of the Central Brain Tumor Registry of the United States, which tracks brain tumor statistics and trends for the field.
- NBTS was an early supporter of the Society of Neuro-Oncology, the field’s main professional organization.
- Provided patient advocates to help build and oversee theBrain Tumor Specialized Programs of Research Excellence (SPORE) at the NCI.
- Formed the Jumpstarting Brain Tumor Drug Development Coalition and, together, led efforts to improve clinical trials through improvements in MR imaging and resulted in a new protocol enabling better use of patient data in evaluating new treatments.
- Established the first ever brain tumor Research Roundtable to bring brain tumor researchers from many sectors (industry, academia, government, and hospitals) together to tackle barriers to research.
IMPACT: This work has helped move the field from a very limited understanding of brain tumors, to the point today where many brain tumors are well studied and new treatment approaches are now being developed based on that knowledge. Additionally, these efforts have resulted in advances like the creation of a new consensus imaging protocol (basic and advanced) for use in brain tumor clinical trialsto help reduce variability and improve confidence in using imaging data for decisions about the effectiveness of investigational drugs, and whitepapers published in a major brain tumor scientific/medical journal to educate the entire neuro-oncology field on key topics.
PUSHING FORWARD NEW RESEARCH STRATEGIES:NBTS has led the charge in pursuing new and innovative research models.
- Partnered with AACR to organize a Cancer Systems Biology Think Tankat which world-renowned researchers in the field of cancer systems biology discussed ways to move the field forward to improve the efficiency and success of cancer research.
- Developed andlaunched our “Defeat” model for research collaborationwith both the Defeat GBM Research Collaborative and Defeat Pediatric Brain Tumors Research Collaborative.
- Embraced the field of Developmental Neurobiologyas an approach to developing new treatments for pediatric brain tumors that take into account a child’s developing brain.
- Sponsored two of the first workshops, which eventually led to the creation of the GBM AGILE clinical trial.
IMPACT: NBTS’ embrace of new and emerging research techniques and strategies has pushed the brain tumor research field to be more creative and find innovative ways to accelerate research and new treatment development.
MAKING A ‘MOONSHOT’ AGAINST CANCER:NBTS’ CEO was invited in 2016 to serve on the “Blue Ribbon Panel” of national experts picked to advise then Vice President Joe Biden on his National Cancer Moonshot Initiative.
IMPACT: This gave the brain tumor community a voice in this high-profile national effort.
CONNECTING PATIENTS TO CLINICAL RESEARCH:NBTS developed and launched a brain tumor-specific clinical trial finderwhich will help patients find a trial that could potentially be a good fit for them.
IMPACT: The trial finder can help patients access the latest cutting-edge treatments in development for brain tumors, as an additional treatment option, while also increase awareness of, and enrollment in, brain tumor clinical trials to help ensure as many trials as possible can accrue the necessary patients to proceed.