An Effective Therapy Combination for Treating Medulloblastoma in Children

Researchers at the University of Colorado Anschutz Medical Campus have discovered a drug combination that may offer a better prognosis for children diagnosed with MYC amplified Medulloblastoma, an often deadly form of brain cancer. The research was conducted in collaboration with the German Cancer Consortium (DKTK) University Hospital Dusseldorf.

A new drug combination may offer an improved prognosis for children with MYC amplified Medulloblastoma, a form of brain tumor which is often deadly in the pediatric population. The analysis was conducted in concert with the German Cancer Consortium (DKTK) University Hospital Dusseldorf.

“An oncogene called MYC is amplified in these tumors making them very susceptible to recurrence. In addition, there’s a greater risk of it spreading to other areas of the brain and down the spine,” said Siddhartha Mitra, PhD, assistant professor of Hematology-Oncology and Bone Marrow Transplant at the University of Colorado School of Medicine and University of Colorado Cancer Center member via a press release. “The five-year survival rate of this cancer is less than 45 percent. We wanted to discover better treatment options for these kids.”

The researchers discovered that two drugs which have already cleared phase I safety trials in other solid tumors  have a significant impact on these brain tumors. They used the epigenetic drug tacedinaline to ubblock what are referred to  as ‘don’t eat me pathways’ that prevent macrophages in the immune system from consuming a tumor.

“When we used tacedinaline to unblock those pathways, and then added anti-CD47, a drug which makes macrophages become super eaters, the tumor became extremely appetizing to the macrophages enticing them to eat the tumor that was unblocked,” Mitra said. “You are essentially harnessing the body’s own immune system by giving it a jumpstart, much like a medical version of PacMan.”

Dr. Mitra commented that while traditional therapies like chemotherapy have previously targeted the tumor growth pathways this is the first time pathway immune evasion pathways are being targeted in these types of devastating brain tumors.

“Traditional adult cancer drugs don’t work well in kids because children are still developing and their normal cells divide at a rapid pace,” said Mitra. “This drug combination could potentially help not only minimize the negative impacts of traditional cancer treatment in kids, but also give patients diagnosed with MYC amplified Medulloblastoma a better chance at survival.”