National Brain Tumor Society Announces Multitude of New Discoveries to Enable Potential Future Brain Tumor Treatments

National Brain Tumor Society shares updates on the portfolio of progress being made through its subsidiary, Cure GBM, LLC, and its flagship program, the Defeat GBM Research Collaborative

National Brain Tumor Society (NBTS), the largest nonprofit dedicated to the brain tumor community in the United States, today, through its subsidiary Cure GBM LLC, provided a comprehensive update of the extensive discoveries and progress made over the course of the first half of its five-year flagship research program, the Defeat GBM Research Collaborative (Defeat GBM).

NBTS launched Defeat GBM to connect a team of leading brain cancer experts through a collaborative infrastructure and utilize a multidisciplinary, team science approach to accelerate treatment development for patients with glioblastoma (GBM) – the most common, complex, treatment-resistant, and deadliest type of brain cancer. The five-year survival rate for GBM patients is only approximately five percent (5%).

Now, halfway through Defeat GBM’s five-year, $10 million commitment, the collaborative has contributed to breakthrough findings on GBM tumor biology and is bringing forth a host of both new therapeutic targets and potential drugs of interest for evaluation in the clinic.

“The Defeat GBM program is reaching a tipping point, with researchers nearing the juncture of moving a number of novel therapeutics into the clinical testing phase,” said Dr. W.K. Alfred Yung of MD Anderson Cancer Center and Defeat GBM’s Scientific Director. “Our team is anxious and excited to continue our momentum and develop a multitude of potential new treatment approaches for patients in desperate need of new options.”

Collectively, in just two-and-a-half years the Defeat GBM team has:

• Developed, characterized, and validated more than 70 new laboratory models based on tumor tissue taken from GBM patients, which more faithfully mimic the behavior of human glioblastoma tumors, for use in preclinical drug screening.
• Screened more than 1,000 chemical compounds and existing drugs (and 75,000 combinations thereof) for potential tumor-fighting activity against GBM tumors in the new laboratory models.
• Through discovery research, identified 19 potential novel therapeutic targets that may be exploited as possible new GBM treatment strategies to be developed in the future.
• Through both new discovery efforts and drug screens, identified 16 categories of drugs, combinations, or treatment approaches of interest to study further to determine if they could ultimately be effective treatments for GBM patients.
  • Of the 16, eight (8) classes of drugs have been prioritized for further evaluation and analysis of their tumor-fighting potential.
  • Four (4) of the eight, have been further categorized as clinical candidates with hopes of rapid translation to early clinical trials for patients.
• Identified nine (9) possible new biomarkers to help predict and monitor patient response to GBM treatments.
• Published 29 papers and reviews in high-impact scientific/medical journals, which have collectively been cited more than 300 times by other journal publications, demonstrating their importance to providing critical new information to the brain tumor research field.

These advancements could help better match the right treatments to the right patients (precision medicine) as well as help determine early in the course of future clinical trials if a drug candidate is effectively slowing, stopping, or shrinking tumor growth.

“One of things that’s been quite terrific about the Defeat GBM program is that there is a structure and set of collaborative arrangements in place that allow investigators to go from discovery, potentially, to things we can actually do about it in the clinic,” said Dr. Paul Mischel a primary investigator within the collaborative from Ludwig Cancer Research. “One of the reasons that the scientific process is often slow in turning knowledge into outcomes for patients is because the ecosystem of science is complicated. It’s very hard to be able to have the pieces in place to interact rapidly. Defeat GBM is like a catalyst – it puts together the pieces that allow progress to move much more quickly. The integration of fundamental discovery, translation, and clinical work is really critical and a key aspect of agency that the National Brain Tumor Society is doing to empower scientists and clinicians to make a difference. It’s been really an extraordinary and interactive program and, I think, way ahead in its thinking about how science needs to be done in order to make a difference for patients.”

View the full Defeat GBM 2017 Progress Report here.

“The Defeat GBM Research Collaborative has demonstrated the ability to produce actionable discoveries as well as subsequently determine and develop viable strategies to qualify potential new targeted therapies for evaluation in early-phase clinical trials. As the stakes gets higher with each new step forward, so too does the cost and the resources needed to meet the challenges and opportunities ahead,” said David F. Arons, JD, Chief Executive Officer, National Brain Tumor Society, and President of CURE GBM LLC, the subsidiary which administers the collaborative. “The Defeat GBM Research Collaborative is thus positioned to expand via partnerships, sponsors, and dedicated supporters that share in the common mission of forever changing the trajectory of this terrible disease.”

About the Defeat GBM Research Collaborative

Defeat GBM Research Collaborative is a multi-disciplinary research program taking a precision medicine approach to develop new treatments for glioblastoma (GBM) patients. Four cores made-up of world-class research teams work in concert to accelerate the translation of discovery to the clinic. The cores operate without “borders” allowing pre-published data to be shared among participating institutions and scientists, speeding the research process, with the ultimate goal of helping double the percentage of GBM patients who survive five-years or more beyond their diagnosis.