Discovering New Ways to Approach the Treatment of Rare Brain Tumors

Until a few years ago, there were only a handful of known survivors of atypical teratoid rhabdoid tumor (AT/RT), a rare cancer that affects the brain and central nervous system. When researchers at Dana-Farber/Boston Children’s Cancer and Blood Disorders Center studied how these few survivors were treated, they found three had been given an unusual chemotherapy regimen. They decided to try that therapy with several new and relapsed patients.

discoveries into new brain tumor treatments
Brain tumor experts Keith Ligon, MD, PhD, and Mark Kieran, MD, PhD

“We had two kids with newly diagnosed AT/RT and two that had relapsed, and three of them did very well,” says Mark Kieran, MD, PhD, director of Medical Neuro-Oncology at Dana-Farber/Boston Children’s. “Basically, we doubled the number of known survivors of this disease.”

After this initial success, physicians at Dana-Farber/Boston Children’s launched a national trial on the chemotherapy regimen. Over half the children in that trial became long-term survivors.

According to Kieran, the therapy may also see applications in treating adult breast, prostate, lung and colon cancers.

Learn more about this treatment in the video below and at