Josh Neman, PhD

Medulloblastoma is a pediatric brain tumor that can recur near its original location or spread to other areas of the brain and spinal cord. These recurrent and metastatic tumors are especially difficult to treat and are associated with poor outcomes, underscoring the importance of research into why they develop.

When medulloblastoma returns after surgery, cancer cells that remained after the original treatment may have evolved and adapted, developing resistance to chemotherapy and radiation. These tumors can grow more aggressively than the original cancer, while available treatment options remain limited. Similarly, treating cancer that has spread to the spine poses significant challenges because surgery, radiation, and chemotherapy can carry substantial risks.

Building on their understanding of medulloblastoma, brain development, and neuroscience, Dr. Neman's research team is pursuing two goals: improving understanding of why medulloblastoma recurs locally and developing safer targeted treatments for patients whose tumors have metastasized.

The team hypothesizes that medulloblastoma cells use a protein called GABA transaminase, also known as ABAT, to survive the stressful conditions created by radiation and chemotherapy. This survival mechanism may allow residual tumor cells to persist and eventually cause the cancer to return.

The researchers have also found evidence that metastatic medulloblastoma cells depend on ABAT for growth. Based on these findings, they are investigating a potential ABAT-targeting treatment called NEO216 to determine whether it could offer a safe and effective therapeutic approach for metastatic disease.

By studying the biological mechanisms behind recurrence and developing treatments aimed at metastatic tumors, this research seeks to address two major causes of mortality in children with medulloblastoma. The ultimate goal is to reduce the burden of treatment, improve quality of life, and increase survival for children and families facing this disease.