Families facing a diagnosis of Diffuse Intrinsic Pontine Glioma (DIPG), also known as diffuse midline glioma (DMG) are often thrown into a world filled with uncertainty, fear, and questions that do not have easy answers. These tumors grow deep within the brain, in areas responsible for essential functions such as breathing, movement, and speech. Because of where they are located, surgery is often not possible. Radiation therapy remains the main treatment option and can sometimes slow the disease for a period of time, but it is not a cure.
For many families, one of the hardest realities to face is how little progress has been made over time. Despite decades of research, treatment options remain limited, and outcomes have not changed significantly. Parents often ask why this disease does not receive the same attention as others, or why promising ideas seem to take so long to reach children.
One of the reasons progress has been slow is that DIPG is rare. Traditional drug development is expensive and time-consuming, and pharmaceutical companies often focus on diseases that affect larger numbers of patients. As a result, rare pediatric cancers like DIPG are frequently underfunded, even though the need is urgent.
Because of this, families, advocates, and researchers have increasingly looked for different ways to move research forward. One approach that has gained attention is collaboration built on openness—sharing data, learning from one another, and working together across institutions rather than in isolation. This approach is often referred to as open science.
Open science focuses on sharing research findings openly so that many experts can contribute to the work. Instead of repeating the same experiments in separate labs, researchers can build on what is already known. For families, this matters because it can help research move more efficiently and make better use of limited resources.
This approach is central to a collaborative research effort involving Agora Open Science Trust, its research partners, and academic investigators. In 2024, this work was further supported by funding from the DIPG/DMG Collaborative, awarded through a research grant led by Dr. David Drewry at the University of North Carolina at Chapel Hill.
Dr. Drewry has been involved in this effort since its early stages. His work focuses on carefully evaluating potential drug compounds and helping determine which ones show enough promise to move forward. The funding from the Collaborative helped strengthen this partnership and support the careful, step-by-step work needed to evaluate possible treatments.
The research supported by this collaboration focuses on a change found in some DIPG tumors involving a protein called ALK2. About one quarter of children with DIPG have tumors with this change. Researchers believe this alteration can work together with another common mutation, known as H3 K27M, to drive aggressive tumor growth. Understanding these biological changes helps researchers think more clearly about how treatments might be designed.
Over several years, researchers working together through this open science model examined more than 800 possible drug compounds. Each compound was evaluated carefully, asking questions such as: Can it reach the brain? Does it appear safe in early testing? Does it show signs of slowing tumor growth in laboratory studies?
Out of this large group of compounds, one stood out. That compound is now known as M4K 2009. It was selected not because it was perfect, but because it showed the strongest balance of the qualities researchers were looking for. Importantly, it is designed to be taken by mouth, which could make treatment easier for children and families if it eventually reaches clinical trials.
What makes this effort different is not only the science, but how the work has been done. Academic researchers, nonprofit organizations, and industry experts have shared data and expertise openly. Many contributed time and resources because they believed in the importance of the work. This allowed the research to move forward more efficiently and with fewer barriers than traditional drug development.
Today, M4K 2009 is still in the research and development stage. The next steps include additional safety testing, developing a formulation that is easier for children to take, and preparing for future clinical trials. These steps take time, and there are no guarantees.
For families, this work does not offer immediate answers or promises. What it does offer is transparency, collaboration, and steady progress. It reflects a growing commitment to working together—researchers, advocates, funders, and families—to move closer to better options for children diagnosed with DIPG and DMG.