Research And Grants

Understanding tumor heterogeneity and characterizing treatment responses in ACVR1-mutated diffuse midline gliomas.

Diffuse midline gliomas (DMGs), sometimes called diffuse intrinsic pontine gliomas (DIPGs) are aggressive brain tumors that occur in children. There is currently no cure for these cancers. DMGs occur because genetic defects appear in some cells in the brain during childhood. Researchers and doctors have identified many of these genetic defects. However, we still do not fully understand how they lead to tumor formation. Furthermore, at the molecular level, DMGs can be quite different from one another. Children diagnosed with DMGs that look similar at first glance, may in fact have tumors that carry distinct genetic lesions. These may affect how fast the tumors grow, and how they respond to therapies.  Therefore, it is important to identify treatments that may work best for specific patients. To achieve this, we need better DMG models that reflect the disease variability. Here, we will create mouse models of different DMG subtypes. We will study how the tumors are similar or different from each other. Using our mouse models, we will test the efficacy of a drug that could be particularly useful in specific patients. Overall, we expect that our findings will help the development of better treatments for DMGs.