Clinical Trial to Assess Whether Cancer Therapy Tasigna Can Treat Parkinson’s

Georgetown University will conduct a clinical trial of Novartis’ cancer therapy Tasigna (nilotinib) as a treatment for Parkinson’s disease.

The Phase 2 trial follows a report last year that Tasigna can improve both cognitive and movement symptoms in Parkinson’s patients.

Georgetown University Medical Center (GUMC) researchers will perform the trial (NCT02954978). They will work with colleagues at the MedStar Georgetown University Hospital, where the trial will take place. Novartis will provide Tasigna and a placebo.

The U.S. Food and Drug Administration approved Tasigna for treating chronic myeloid leukemia, but the blood cancer requires much higher doses than Parkinson’s does.

Seventy-five patients are being recruited for the Parkinson’s study. They will be randomized into three groups. Two will receive different doses of Tasigna — either 150 mg or 300 mg. One will be treated with a placebo.

The main focus of the study will be the safety and tolerability of Tasigna in people with mid-stage Parkinson’s.

Researchers will also examine the impact that low doses of Tasigna have on disease biomarkers and patient outcomes.

The team will be led by Fernando Pagan, MD, medical director of the GUMC Translational Neurotherapeutics Program and director of the Movement Disorders Clinic at MedStar.

The treatment will last 12 months. Eligible patients can take part in a one-year follow-up trial to assess the drug’s long-term effect. The follow-up will randomize patients to receive either 150mg or 300mg of Tasigna, the same doses used in the Phase 2 initial trial.

A previous study indicated that Tasigna increased levels of dopamine, which helps regulate movement and emotional responses, in the brains of 12 patients. Tasigna also reduced levels of the protein alpha-synuclein, which researchers believe contributes to destruction of neurons in Parkinson’s.

At the time researchers reported the study’s results, they warned that the findings needed to be viewed with caution.

“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,” Pagan said in a press release.

Tasigna belongs to a class of drugs called tyrosine kinase inhibitors. In recent years, accumulating evidence indicates that enzymes, called tyrosine kinases, are involved in neurodegeneration and inflammation.

Trials like the current one will shed light on whether such compounds can slow or prevent disease progression in Parkinson’s.

“I am pleased to offer this study to my patients, which demonstrates the importance of teaming Parkinson’s care with academic research. Only through clinical trials will we be able to move the field forward so that we can offer better treatments to our patients in the future,” Pagan added.

Patients and families may sign up here for more information about the trial.