The Defeat GBM Research Collaborative is the National Brain Tumor Society’s largest and boldest research initiative, which aims to double the percentage of glioblastoma (GBM) patients – the most common and deadliest form of brain cancer- that survive five years or longer. The collaborative is made up of four interrelated research cores that encompass 1) Target Discovery 2) Drug Development 3) Biomarker Development and 4) Innovative Clinical Trials.
Since the commencement of research in 2014, the Defeat GBM Research Collaborative has developed an integrated scientific and drug discovery program focused on moving research from laboratory to clinical readiness. Now two years in, NBTS, together with Dr. Alfred Yung – Scientific Director of Defeat GBM – decided that it was a good time to convene a special meeting (beyond the regular monthly web-conference calls that already take place), to bring together all the investigators for an in-person retreat style meeting. The meeting was held on April 20-21, 2016 in Scottsdale, Arizona with the purpose of reviewing progress, as well as challenges, in achieving core project aims, and to discuss strategy and priorities for the collaborative’s future work. The meeting was not intended to be just a progress report but rather an opportunity to discuss how to leverage the collaborative’s synergies to improve patient outcomes and move towards the overall goal.
“We couldn’t actually organize ourselves without NBTS,” said Dr. Webster Cavenee ofLudwig Cancer Research.
The researchers who attended included: Dr Al Yung (MD Anderson Cancer Center), Dr Web Cavenee (Ludwig Institute for Cancer Research), Dr Tim Cloughesy (UCLA), Dr John De Groot (MD Anderson Cancer Center), Dr Frank Furnari (Ludwig Institute for Cancer Research), Dr Amy Heimberger (MD Anderson Cancer Center), Dr Frederick Lang (MD Anderson Cancer Center; by webconference) , Dr Ingo Mellinghoff (Memorial Sloan Kettering Cancer Center), Dr Paul Mischel (Ludwig Institute for Cancer Research), Dr Roel Verhaak (MD Anderson Cancer Center), Dr Robert Strausberg (Ludwig Institute for Cancer Research), Dr Erik Sulman (MD Anderson Cancer Center), NBTS CEO David Arons (and President of CURE GBM LLC), and Scott Memmott (Honorary Board Member of NBTS and CURE GBL LLC). In addition, a number of NBTS staff from the Research and Advancement departments attended.
During the first day of the retreat, the group spent six hours discussing research progress to date and how to maximize collaboration to integrate, progress, and translate research findings toward the goal of moving into the clinic. In broad terms, key scientific achievements include:
- A unique platform of preclinical in vitro and in vivo patient-derived glioblastoma models, that recapitulate the original tumors, has been established for basic research and drug discovery and testing projects. This platform is continuously being expanded and characterized and is accessible for all the researchers.
- Identification of potential precision medicine therapeutic targets & biomarkers for GBM.
- Identification of actionable therapeutic and biomarker strategies to overcome resistance and inhibit tumor growth.
- Preclinical drug development leading to the identification of potential clinical candidates.
- Planning of innovative biomarker enriched early phase clinical trial.
- 16 scientific and peer-reviewed publications.
“When we all get together in the same room, really good things happen,” said Dr. Frank Furnari of Ludwig Cancer Research, San Diego
In addition to the Defeat GBM researchers, Dr. Amy Heimberger (MD Anderson Cancer Center), a leading immuno-oncology scientist was invited as a special guest of the group. Dr. Heimberger participated in the meeting to discuss the future incorporation of research aimed at advancing the science and development of transformative immunotherapeutics for GBM patients. There was unanimous support and excitement for integrating immunotherapy research into the future Defeat GBM research portfolio.
The second day of the meeting included around five hours of discussion on different topics relating to governance, data sharing, engagement with pharmaceutical companies and future directions.
A full report of the meeting will be forthcoming on the NBTS website soon. In the meantime, the key takeaways from the two-day retreat include:
- A portfolio of novel and actionable therapeutic targets, and potential clinical candidates, has been developed.
- Several next steps involving close collaboration across different research cores were identified with the goal of advancing top scientific findings to go/no-go decisions for clinical trials in GBM patients.
- There was strong support for establishing a new ‘Early Clinical Trials Team’ within the Defeat GBM collaborative to enable decisions on the suitability of compounds/biologics for further phase I and/or II clinical trials
- Future research will include immuno-oncology projects.
- Future research will incorporate increased collaboration with biopharma companies.
“The Defeat GBM Collaborative research effort has already proven that its unique research model can deliver value to all stages of translational research,” said David F. Arons, NBTS’s CEO.