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ChadTough Defeat DIPG Foundation Funds Second Year of Innovative International Clinical Trial for DMG/DIPG

ChadTough Defeat DIPG Foundation has announced it has awarded over $500,000 for the second year of the Diffuse Midline Glioma – Adaptive Combinatorial Therapy (DMG-ACT), bringing the total amount funded for this trial to over $1 million.  The DMG-ACT, a collaboration through the Pacific Pediatric Neuro-Oncology Consortium (PNOC) and the DIPG Centre of Expertise in Zurich, is a biology-informed, adaptive, and progressive trial, designed to deliver more rapid progress for children suffering from DMG.

Two aspects of DMG-ACT are particularly important and set it apart from most prior experimental therapies for children facing DMG/DIPG.  First, the feedback loop between preclinical space and clinic is constant, meaning that clinicians treating patients and researchers in the lab are sharing information in real time and then determining any necessary adaptations to the treatment protocol.  Second, the DMG-ACT involves combination therapies (the use of more than one therapeutic agent) as distinct from historical use of single therapy trials.

DMGs are fast-growing, cancerous tumors found in the midline of the brain, and they include diffuse intrinsic pontine glioma (DIPG) tumors. Over the past five decades, clinical trials have not resulted in improved outcomes for children diagnosed with these tumors.  The overall projected survival for diagnosed children is just 9-12 months.  However, in recent years, due to knowledge gained from studying postmortem tumor specimens, scientists have gained a new understanding of the biology of these tumors.  Researchers now know that DMGs, including DIPGs, require a personalized therapy approach to improve the outcome for diagnosed children.

Governed by 18 institutions across Australia, Europe and North America, the DMG-ACT protocol is different from typical clinical trials in that it studies smaller groups of patients (15 to 20).

“Initially, our main concern is if drugs are really getting into the tumor of these devastating diffuse midline gliomas. If we do not see any early signs of survival impact, we will also look at drug penetration to decide if an arm should be adjusted or closed,” explained Dr. Sabine Mueller, pediatric neuro-oncologist of University of California, San Francisco and Clinical Program Head at DIPG Centre of Expertise in Zurich.

Through this unprecedented trial setup, scientists across the world share their latest preclinical data, allowing for more rapid progress in the care of patients suffering from high-risk pediatric brain tumors compared to traditional approaches.

“The first year was critical for finalizing the trial plan and establishing the lab correlated tests that will help us understand if the drugs are working,” said Michigan Medicine pediatric oncologist Dr. Carl Koschmann, who serves as a PNOC site co-principal investigator.  “These trials are now very progressive in design and we’re hopeful they will push the field forward.”

In addition to exploring personalized therapies, many critical studies will be performed (biopsies, blood draws, and lumbar punctures) for real-time analysis at labs around the world to learn which treatments are working and why.

“Simply put,” said Dr. Koschmann, “the DMG-ACT would not have been possible without the support of the ChadTough Defeat DIPG Foundation. For those of us involved, we feel this will finally provide the jumps in improved survival for DMG that was seen in leukemia 40 to 50 years ago.”