Researchers are leveraging an open science model to accelerate the development of a new treatment for a devastating and rare childhood brain cancer, aiming to make therapies more accessible and affordable.
The team is focused on diffuse intrinsic pontine glioma (DIPG), an aggressive cancer primarily affecting young children. Current treatment options are severely limited, and the life expectancy following diagnosis is roughly one year. The project specifically targets mutations in the kinase ALK2, which are linked to approximately 25% of DIPG cases. By developing an ALK2 inhibitor, researchers hope to slow the cancer's progression and extend lives, potentially as part of a future combination therapy.
This work is conducted through the Structural Genomics Consortium at the UNC Eshelman School of Pharmacy, a public-private partnership that places all its research outputs in the public domain. The open science framework means all findings and project meetings are shared publicly, allowing scientists worldwide to contribute ideas and resources. "What's so nice about the school of pharmacy is you have colleagues here with pharmaceutical industry experience, who know about the science of drug discovery, we have great lab facilities, and everyone is open to collaboration," said Professor David Drewry, who leads the medicinal chemistry effort.
The initiative is a partnership with M4K Pharma, a virtual biotech company dedicated to rethinking how medicines for childhood diseases are developed. The collaboration has already selected a clinical candidate drug. The team is now compiling the necessary data to present a package to the Food and Drug Administration, with the goal of advancing the candidate into Phase 1 clinical trials.
With critical funding from patient advocacy foundations like The Cure Starts Now, the project represents a community-driven effort against a rare disease. The researchers express a shared personal commitment to helping families, fueled by the urgent need for more treatment options. The next major milestone is formal regulatory approval to begin testing the potential therapy in children, offering a hopeful pathway toward a much-needed intervention.