Combination drug therapy for childhood brain tumors shows promise in laboratory models

In experiments with human cells and mice, researchers at the Johns Hopkins Kimmel Cancer Center report evidence that combining the experimental cancer medication TAK228 (also called sapanisertib) with an existing anti-cancer drug called trametinib may be more effective than either drug alone in decreasing the growth of pediatric low-grade gliomas.

Approximately 50% of children treated with traditional therapy have their tumors regrow, underscoring the need for better, targeted treatments.

The combination therapy, when tested in tumor cell lines derived from children's gliomas, stopped the tumor cells from growing.

When Raabe and team treated tumors or cells with just one of the drugs targeting one of the pathways, the cancer cells were able to use the other pathway to survive, Raabe says.

In the new study, Raabe and colleagues tested TAK228 and trametinib in patient-derived pediatric low-grade glioma cell lines grown in the laboratory.

The combination killed some pediatric low grade glioma cells -- increasing the cells killed by nearly threefold over cells treated with each agent alone.

The investigators then gave mice implanted with human low grade glioma tumors TAK228, trametinib, the combination of the two drugs, or a combination placebo.