Cancer drug for Parkinson’s disease enters phase 2 clinical trial

A phase 2 trial will examine the safety of nilotinib and its ability to slow disease progression. (Credit: Jeremy Atkinson/Flickr)

Researchers at Georgetown University Medical Center recently announced the launch of a phase 2 clinical trial of the cancer drug nilotinib for people with Parkinson’s disease.

Parkinson’s is one of the most common neurodegenerative brain disorders. It generally develops slowly, and typical early symptoms can include shaking, tremors, difficulty walking, and slowness. The new study will investigate the safety of nilotinib along with its ability to treat symptoms and slow disease progression.

Nilotinib is already approved by the U.S. Food and Drug Administration and is used to treat leukemia. A phase I study previously published in the Journal of Parkinson’s Disease suggested that nilotinib might also slow or even reverse the progression of Parkinson’s disease. However, the small study involved only 12 participants. It also did not include a placebo group to compare against the drug.

“The early proof of concept study conducted in 2015 and published in 2016 provided encouraging results, but we won’t know the exact effects of nilotinib on Parkinson’s disease until larger trials like this new one are complete,” says Fernando Pagan, MD, the medical director of GUMC’s Translational Neurotherapeutics Program and principal investigator of the new study in a press release.

The Phase 2 trial aims to recruit 75 patients with moderate Parkinson’s. Patients will be randomly assigned to receive either the placebo or one of two doses of nilotinib. Researchers will monitor patients over the course of 12 months to understand the long-term effects of the drug.

Novartis, the pharmaceutical company that makes nilotinib, is supplying the drug and matching placebo for the phase 2 study.

Clinical Trial
Phase 1 Trial Results
Press Release