Funded by the Dick Vitale Pediatric Cancer Research Fund
Medulloblastoma is the most common brain tumor in children. While doctors can cure most of these children, the treatments are very toxic and negatively impact these patients and their families for the rest of their lives. Thus, scientists are trying to finding new therapies that are more effective and less toxic. A handful of new drugs have been tested in the last few years in patients with medulloblastoma. Most of these new drugs initially show great efficacy. Unfortunately, tumors rapidly become resistant and return more aggressively. Sadly, when these tumors come back there is no good treatment available and most of these children die. Therefore, it is very important to find drugs that can stop the growth of the tumors and prevent their reappearance. A series of experiments allowed us to find an ideal candidate therapeutic for children with medulloblastoma tumors. These compounds that target a family of proteins named BET, will reduce the size of the tumors and prevent them from growing back in the future. We believe that our research will provide a game-changing therapy for patients with medulloblastoma and restore hope in these children with cancer and their families.