Clinicians and scientists at Johns Hopkins All Children’s Hospital collaborated to identify the oncogene that is responsible for causing group three medulloblastomas in children.

Ranjan Perera, Ph.D., joined Johns Hopkins All Children’s in St. Petersburg, Florida, in 2018 as director of the hospital’s Center for RNA Biology. He soon began talking with neurosurgeon George Jallo and neuro-oncologist Stacie Stapleton about how they could work together.

The conversation evolved to the difficulty of distinguishing between two forms of medulloblastoma. The two forms look identical under a microscope, but one is fast growing and aggressive and has limited or no cure, while the other is less aggressive. 

“Medulloblastoma is the most common malignant brain tumor in children and remains one of our most challenging tumors to treat,” says Stapleton, chief of the Oncology Division at Johns Hopkins All Children’s. “Therefore, this type of research is critical to move us closer to an effective treatment.”

Medulloblastoma accounts for about 20% of all pediatric brain tumors with about 500 patients diagnosed in the United States each year. Through a series of investigations, including machine learning and statistical methods, the Johns Hopkins All Children’s team identified multiple medulloblastoma-specific RNA molecules, which they hope can lead to therapeutic targets for treating the disease.

“We are very excited that we were able to partner to build a strong team with a world-class neurosurgeon and a neuro-oncologist for this study,” Perera says. “Without their help and advice, we would not able to conduct this cutting-edge research. The current focus of our team is to develop lipid nano-particle based RNA therapeutics to treat children suffering from this often-fatal disease. Although it is still a long way to go, we are excited, and it is promising.”