The Jumpstarting Brain Tumor Drug Development Coalition recently hosted the Brain Tumor Clinical Trial Endpoints Workshop where leaders from the neuro-oncology community met to establish improved pathways for therapy evaluation.
The seminal workshop focused on confronting key challenges in developing and assessing treatments for glioblastoma multiforme (GBM), one of the most common primary malignant brain tumors. GBM represents about 17% of all primary brain tumors and about 60-75% of all astrocytomas. The tumor can be difficult to treat and its exact cause is still unknown.
One of the key challenges tackled in the workshop was how to advance use of imaging-related endpoints in clinical trials. Dr. W.K. Alfred Yung, workshop chair, and chair of the department of neuro-oncology at MD Anderson Cancer Center, said, “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.”
Participants in the seminal workshop included the US Food and Drug Administration (FDA), the NCI, brain tumor patient advocates, leading neuro-oncologists, and biopharmaceutical representatives. The resulting action plan from the workshop included the following constructs:
N. Paul TonThat, CEO of the National Brain Tumor Society, said, “All the key stakeholders were aware of the current limitations and lack of clarity in brain tumor imaging endpoints, but we needed to get all of the leaders into one room to actually begin moving the conversation forward.” The National Brain Tumor Society said the prospect of faster approvals not only speeds new brain tumor therapies entry into the market, but may also entice pharma companies to invest in brain tumor research.
Dr. Richard Pazdur from the FDA’s Office of Hematology and Oncology Products said, "The FDA wants to foster the development of new and effective treatments for patients with brain tumors. We are happy to participate in conversations with the neuro-oncology community to discuss the evaluation process for brain tumor therapies.”