In partnership with xCures, Cancer Commons and the Musella Foundation, The Cure Starts Now is pleased to announce a renewal of $100,000 for the Oncoceutics Expanded access of ONC201, bringing the total contribution by The Cure Starts Now to over $267,000.
Part of this program is an intermediate size Expanded access protocol for ONC201 in patients with H3 K27M-mutant glioma entitled “ONC018: Expanded access to ONC201 for Patients with H3 K27M-mutant and/or Midline High Grade Gliomas” that was recently accepted by the U.S. Food and Drug Administration (FDA).
The H3 K27M mutation has been identified as an important prognostic indicator in aggressive midline gliomas that involve specific parts of the brain, including the thalamus, pons, or spinal cord. In 2016, the World Health Organization categorized any midline brain tumor that contains the H3 K27M mutation as the highest grade (IV) because the mutation confers such a poor prognosis. Beyond palliative radiation, no medical therapy has been shown to provide clinical benefit for patients with this mutation in their tumor. Pediatric patients are particularly impacted by this mutation, especially those with DIPG where 70-80% of the patients have the mutation.
ONC201 is an investigational, novel small molecule with a unique and novel mechanism of action that is currently under clinical investigation in several Phase II trials. Ongoing clinical trials with ONC201 include trials in adult and pediatric patients with high-grade gliomas that harbor an H3 K27M mutation that can be identified by immunohistochemistry or gene sequencing (e.g. FoundationOne CDx). H3 K27M-mutant glioma is a molecularly-defined disease with a dismal prognosis. Oncoceutics’ clinical trials are being carried out at numerous leading cancer centers across the United States, including Massachusetts General Hospital, Dana Farber Cancer Institute, NYU Langone, MD Anderson Cancer Center, Levine Cancer Institute, Miami Cancer Institute, University of California San Francisco, Emory University and the University of Michigan. In addition, several other leading cancer centers across the United States are in the process of joining the program.
Given the rare incidence and prevalence of patients suffering from H3 K27M-mutant gliomas and the severe clinical symptoms that these patients often sustain, disabling them from travel to cancer centers, the options to enroll patients on clinical studies are limited. The intermediate-size expanded access program is intended to give options to patients that do not qualify for currently available therapies, allowing the company to make the investigational drug therapy available to patients that may be eligible for the expanded access program. xCures and Cancer Commons will work together with Oncoceutics in those cases where patients cannot participate in ongoing clinical research programs. The collaborators will work to facilitate the process in close cooperation with the patient’s treating physicians and hospital system.
The Cure Starts Now involvement is also designed to further open the data collected by the trial to other clinical trial reviews in the hopes of aiding families seeking options in treatment.