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National Brain Tumor Society Provides Funding for the Development Novel Therapy with Potential to Treat Adult and Pediatric Brain Cancers

Al's Comment:

 From our friends at the NBTS.  There was a fascinating presentation at SNO that showed GBM patients with low EGFR levels might do better than those with High EGFR levels with the experimental drug Onc-201.  I am happy to see they are going to test that in a trial. I wish them luck.  Hopefully they will find out if it is EGFR that makes the difference or it is just that the low EGFR status is linked to a high DRD2 level - which is the target of Onc201.  If the EGFR makes a difference, this opens the door to combination treatments targeting EGFR along with Onc-201 to hopefully help all patients.

Posted on: 01/27/2020

National Brain Tumor Society Provides Funding for the Development Novel Therapy with Potential to Treat Adult and Pediatric Brain Cancers
Leading brain tumor patient advocacy organization commits to providing more than $200,000 to support a phase II clinical trial of ONC201
National Brain Tumor Society (NBTS), a leader in brain tumor patient advocacy, today announces a commitment of at least $200,000 to support a forthcoming phase II clinical trial of the investigational therapy, ONC201, with the potential for multiple years of funding. The future clinical trial will evaluate the safety and efficacy of ONC201 as a single-agent treatment for a subset of adult patients with high-grade glioma that have low expression of the epidermal growth factor receptor (EGFR). 
“National Brain Tumor Society is committed to rapidly improving clinical outcomes for all brain tumor patients by fostering the development of new and potentially more effective treatments, beyond the current, limited options,” said Kirk Tanner, PhD, Chief Scientific Officer, National Brain Tumor Society. “Funding support for this upcoming trial of ONC201 stems from our interest in understanding the full potential of this compelling investigational drug, which has shown early signs of efficacy in both adult and pediatric high-grade glioma patients whose tumors harbor H3K27M mutation, to an additional subpopulation who can be identified through biomarker analysis. In doing so, we hope to extend the number of patients who could conceivably benefit from ONC201.”
In preclinical studies, low expression of EGFR was found to correspond with an associated elevation in the expression of dopamine receptor D2 (DRD2) in glioma cells. Tumors with elevated DRD2 expression were demonstrated in earlier studies to be highly sensitive to ONC201, which reduces the effects of overexpressed DRD2. Indeed, preliminary data from clinical trial patients with recurrent glioblastoma that harbor these characteristics suggest that treatment with ONC201 may translate in improved survival endpoints relative to patients who do not express these biomarkers.
The trial will be led by Clark Chen, MD, PhD, Lyle French Chair in Neurosurgery and Head of the Department of Neurosurgery, University of Minnesota Medical School, along with Joshua Allen, PhD, Senior Vice President of Research and Development at Oncoceutics, Inc. ONC201, which is being developed by Oncoceutics, a drug discovery and development company, also continues to be evaluated in multiple, parallel clinical trials of adult and pediatric high-grade glioma patients whose tumors harbor H3K27M mutation, including diffuse intrinsic pontine glioma (DIPG) patients.
About National Brain Tumor Society
National Brain Tumor Society invests in, mobilizes, and unites the brain tumor community to discover a cure, deliver effective treatments, and advocate for patients and care partners. We are the largest patient advocacy non-profit solely dedicated to the brain tumor community and a leader in collaborating with the neuro-oncology field. Headquartered in Newton, Massachusetts, our organization raises funds to invest in accelerating brain tumor treatments, prepare the community to navigate their unique brain tumor experience, and convene stakeholders while changing public policy to improve the lives and survival of brain tumor patients. Visit us at


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