Can a Multiple Sclerosis Drug Be Used to Treat Brain Cancer?

A study from the University of California San Diego School of Medicine suggests that adding a multiple sclerosis (MS) drug to targeted cancer therapy may serve as a viable treatment for glioblastoma. The research team’s findings were recently published in Science Translational Medicine.

Glioblastoma is one of the most aggressive forms of brain cancer, and affects surrounding brain tissue, making it arduous to treat with surgery. Even in cases when chemotherapy and radiation eradicate most glioblastoma cells, they may not affect the cancer stem cells.

“We used to think we’d find a single magic bullet to treat everyone with glioblastoma,” said senior author Jeremy Rich, MD, professor of medicine at UC San Diego School of Medicine and director of neuro-oncology and director of the Brain Tumor Institute at UC San Diego Health in a press release. “But now we realize that we need to find out what drives each patient’s unique tumor, and tailor our treatments to each individual.”

In this study, the researchers tested MS drug teriflunomide, which works by blocking pyrimidine-forming enzymes on animal-model glioblastoma samples donated by patients who underwent surgery. The researchers discovered in lab testing that teriflunomide inhibits glioblastoma cell survival, self-renewal and tumor. Moreover, when treated with teriflunomide alone, the patient-derived tumors slightly diminished and the mice bearing them survived for a moderately longer duration, compared to mock-treated mice.

The team also tested two targeted cancer therapies – BKM-120, an inhibitor that effectively treats glioblastoma cells driven by lack of an enzyme called PTEN, and lapatinib, an inhibitor that treats cancers driven by mutations in the Epidermal Growth Factor Receptor (EGFR). Their results showed that BKM-120 treatment alone, tumors moderately decreased in size and mice survived even longer, compared to either mock-treated mice or teriflunomide-treated mice.

Researchers ‘Exited’ About Results

“As scientists, we are often looking at small snapshot of what a cancer stem cell is doing,” added Dr. Rich. “As a clinician, I also try to look at the bigger picture. I’m not looking for just one or two drugs to help my patients, because I think it’s going to take a whole personalized cocktail of many different drugs to really get the cancer cells on the run.”

Dr. Rich continued by stating that his team are “excited about these results, especially because we’re talking about a drug that’s already known to be safe in humans.” However, he cautioned that “this laboratory model isn’t perfect — yes it uses human patient samples, yet it still lacks the context a glioblastoma would have in the human body, such as interaction with the immune system, which we know plays an important role in determining tumor growth and survival. Before this drug could become available to patients with glioblastoma, human clinical trials would be necessary to support its safety and efficacy.”