Updated May 30, 2019
xCures and Cancer Commons are pleased to announce a collaboration with Oncoceutics to implement the Expanded Access policy of the company. Part of this Expanded Access 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 invesstigational 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 faciliate the process in close cooperation with the patient’s treating physicians and hospital system.
“We are pleased to see rapid progress in the development of ONC201 in H3 K27M-mutant gliomas as well as those with midline high-grade gliomas, a serious disease that principally has no satisfactory standard of care. We believe novel access approaches like this Intermediate Size Expanded Access Protocol, performed in a continuous learning system capable of generating regulatory grade data, are important to speed up progress for brain cancer patients who currently do not have effective treatment or trial options. We appreciate the support [from]... The Cure Starts Now Foundation.”
- William Hoos, Chief Operating Officer, xCures
Update on Compassionate Use
In May of 2019, The Cure Starts Now funded an additional $83,333 for a Phase II of ONC201 in support of compassionate use.