Special Project Grant Awarded to ONC201 EAP at Oncoceutics

In 2018, Oncoceutics (now Chimerix) launched an Expanded Access Program (EAP) for a drug called ONC201, which showed promise for treating children with DIPG. The ChadTough Foundation and Michael Mosier Defeat DIPG Foundation (now joined as the ChadTough Defeat DIPG Foundation) supported this effort to make the drug available to patients who had no other treatment options, as standard therapies had little success in managing DIPG. ONC201 is a small molecule that works by targeting specific pathways involved in cancer cell growth and survival. In preclinical studies and early trials, it demonstrated anti-tumor effects, particularly against tumors with the H3K27M mutation, which is present in most DIPG cases. This mutation drives the growth of DIPG, making it especially difficult to treat with conventional methods. Through the Expanded Access Program, patients with no remaining treatment alternatives were able to receive ONC201 under a compassionate use framework while clinical trials were ongoing. This program provided early access to ONC201 for children with DIPG who were not eligible for clinical trials, offering a potential lifeline and advancing knowledge of the drug’s safety and efficacy. The support from both foundations for the EAP reflected their joint mission to accelerate research and treatment options for children with DIPG. Their backing helped ensure that more children could benefit from innovative therapies like ONC201, contributing to the broader effort to develop effective treatments for this devastating disease.