Leaders from key cross-sections of the neuro-oncology community came together at a recent Brain Tumor Clinical Trial Endpoints Workshop to confront a critical challenge in developing and testing treatments for glioblastoma multiforme. Participants were able to adjourn the workshop with the constructs of a specific action plan to ultimately bring greater clarity to one of the central tasks of clinical trials, establishing the effectiveness of therapies.
Experts representing the U.S. Food and Drug Administration (FDA), the National Cancer Institute (NCI), medical imaging companies, contract research organizations, leading biopharmaceutical companies, and medical academic research organizations, were convened at the workshop, which was hosted by the Jumpstarting Brain Tumor Drug Development Coalition (Accelerate Brain Cancer Cure, Musella Foundation for Brain Tumor Research & Information, National Brain Tumor Society, and the Society for Neuro-Oncology). Individuals addressed how to overcome variables in medical imaging, which currently hinder the ability to accurately assess brain tumor response to investigational therapies, thus slowing drug development for this vulnerable patient population.
Objectives and Action Plan
“Historically, variability and lack of standardization in the criteria for the use of imaging endpoints to determine tumor response to treatment has impeded efforts to accelerate the evaluation and approval process of new brain cancer therapies,” said W.K. Alfred Yung, MD, Workshop Chair, and Chair of the Department of Neuro-Oncology at The University of Texas MD Anderson Cancer Center in Houston. “What we did with this workshop, and what the guidance the FDA graciously provided enables, is to speed the development of standards and tumor response criteria, so that together we can accurately assess the effectiveness of promising therapies seeking approval,” Dr. Yung said.
The Workshop featured presentations and panel discussions, as well as facilitated audience-panel deliberations on topics such as current brain tumor imaging protocols in multicenter trials, emerging techniques and technologies in brain tumor imaging, and the use of imaging measurements of tumor progression and tumor response in clinical trials. A final group discussion led to the creation of an action plan for the community to improve the accuracy and consistency of imaging data, and to launch a coordinated effort to accelerate brain tumor therapy approvals using imaging as a criteria.
The Jumpstarting Brain Tumor Drug Development Coalition will work collaboratively with the biopharmaceutical companies, medical academics, medical technology industry, NCI, and FDA to advance the agenda set forth at the first Brain Tumor Clinical Trial Endpoints Workshop. For more information, visit braintumor.org.