Brain tumors are now the leading cause of cancer-related deaths among children. Year after year, Kentucky ranks among states with the highest incidence of children with brain tumors, and Kentucky currently ranks third in the nation. At Norton Children’s Cancer Institute, affiliated with the UofL School of Medicine, our goal is to be able to offer the latest precision medicine treatments to give these children the best possible outcomes.

We are leading a national clinical trial looking at whether an anti-worm drug that has been used successfully in high-risk neuroblastoma, difluoromethylornithine (DFMO or eflornithine), also will work against medulloblastoma. Medulloblastoma accounts for a fifth of all brain tumors in children, making it the most common childhood malignant brain tumor.

I am the principal investigator for this trial, underway in collaboration with the Beat Childhood Cancer research consortium, an international consortium of more than 50 universities and children’s hospitals. This study is already open at Norton Children’s Cancer Institute and is expected to have 15 participating pediatric cancer centers throughout the country initially, with the plan of expanding to 30 by the end of the year.

DFMO offers promise because medulloblastoma and neuroblastoma cells harness overlapping tumor-activating pathways. My hope is that DFMO, which belongs to a family of drugs called polyamine inhibitors, will offer a new and safe option against medulloblastoma.

This research was made possible by a grant to UofL from the Kentucky Pediatric Cancer Research Trust Fund (KPCRTF) to fund the work with Norton Children’s Cancer Institute. The KPCRTF was established in 2015 with the goal to make childhood cancer a state health priority and give all Kentucky children access to new and innovative precision cancer treatment. The KPCRTF also has made it possible for Norton Children’s Cancer Institute to collaborate with the University of Kentucky to institute a statewide molecular tumor board for childhood brain and spine tumors.

Even prior to this collaboration, Norton Children’s Cancer Institute initiated universal genomic testing of all childhood malignant brain tumors. Given that many brain tumors in children share similar cancer pathways with their adult counterparts, at Norton Children’s Cancer Institute we have been able to safely and successfully use newer Food and Drug Administration-approved non-chemotherapy targeted cancer drugs for our patients with difficult-to-treat brain tumors such as glioblastoma. By offering the latest therapies in Kentucky, our hope is that children and their families won’t have to travel to other cancer centers out of state for treatment.

Michael Angelo Huang, M.D., known to his patients and their families as Dr. Mikey, is a pediatric hematologist/oncologist and neuro-oncologist with Norton Children’s Cancer Institute.

Dr. Huang’s recently published research

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