SAN FRANCISCO, CA (August 14, 2024) – The Pediatric Neuro-Oncology Consortium Foundation (PNOC Foundation), a platinum rated non-profit dedicated to a future where we lose no child to brain cancer, applauds the FDA approval of Dordaviprone (ONC201, now known as Modeyso™) for recurrent H3 K27M-mutant diffuse midline glioma. Over 1000 children die every year following being diagnosed with Diffuse Midline Glioma - including Diffuse Intrinsic Pontine Glioma (DIPG). These devastating tumors arise in the midline structures of the brain such as the thalamus, pons as well as spinal cord. The mean overall survival for diagnosed children with DMGs historically has been 9-12 months. The FDA approval is the culmination of years of research, collaboration, and determination by patient families, scientific researchers, clinicians, foundations, philanthropists, and pharmaceutical industry. 

The PNOC DMG working group, and especially the work of Dr. Matt Dun, Dr. Carl Koschmann, Dr. Sabine Mueller and Dr. Javad Nazarian, led some of the key preclinical discoveries and early published research that enabled a better understanding of how ONC201 works. 

PNOC co-founders Dr. Sabine Mueller and Dr. Michael Prados, as thought leaders in this field, participated in many FDA discussions as disease educators sharing insights with them on relevant clinical endpoints. The use of ONC201 was pioneered in several PNOC studies. 

The PNOC DMG working group adapted ONC201 early on into the largest transatlantic DMG platform trial using ONC201 in combination with other agents that will continue to move the field even further forward. The optimization of ONC201’s potential continues to be explored in current combination therapy studies underway in PNOC’s DMG ACT (Diffuse Midline Glioma – Adaptive Combinatorial Trial also referred to as PNOC022). Rather than a single trial, DMG-ACT is a collaborative clinical trial framework designed to accelerate the evaluation of promising treatments for DMG, including ONC201, through adaptive, multi-arm studies across multiple institutions based on multi-institutional preclinical assessments. 

“PNOC is proud of our investigators and the pediatric brain cancer community’s contributions toward this collaborative achievement,” said Dr. Sabine Mueller, PNOC’s co-founder. “We are especially grateful to the brave patients and families who participate in clinical trials, deepening our understanding of these complex tumors. Today’s approval unlocks new possibilities: It allows drug developers testing combination therapies to pursue FDA approval of their agents in combination with ONC201—a regulatory path that wasn’t possible before. Our ongoing clinical trials like our DMG platform trial (PNOC022) are already evaluating multiple ONC201-based combinations, and that data will be critical in defining the next generation of treatment for our patients."

“This breakthrough marks a powerful step forward in the fight against pediatric brain cancer,” said Bruce Campbell, PNOC Foundation co-founder. “There has never been a more promising time to support childhood cancer research. Advances in genetics, immunotherapy, artificial intelligence, and gene editing are driving science into a new era. ONC201’s FDA approval brings us one step closer to a future where we lose no child to brain cancer—but continued momentum depends on bold investment. We urge the philanthropic community to help meet this moment with the resources needed to match the pace of discovery.”

For more information contact: media@pnocfoundation.org 

To view the Jazz Pharmaceuticals news release, Click HERE.

To view the FDA news release, Click HERE. 

To learn more about the PNOC Foundation please visit www.pnocfoundation.org.

###

Photo: Dr. Carl Koschmann with a patient at Michigan Medicine's C.S. Mott Children's Hospital (Leisa Thompson / Michigan Photography)