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Parkinson's DiseaseMarch 2020Summary reviewed July 2026

A Cancer Drug Slowed Parkinson's but Caused Serious Side Effects at High Doses

Scientists tested nilotinib, a cancer drug, in 75 people with Parkinson's to see if it could slow the disease. The drug reached the brain and changed several brain chemicals linked to Parkinson's, including reducing harmful protein clumps.

What the trial was testing

The trial enrolled 75 patients with parkinson's disease. The study was sponsored by Georgetown University and tracked outcomes across the full group of patients who matched the trial's eligibility profile.

It was mid-stage testing (phase 2/3). Trials at this stage are designed to produce evidence regulators and physicians can act on — not just observations to follow up later.

What the results showed

The lower dose reduced alpha-synuclein clumps, a hallmark of Parkinson's, in brain fluid.

JAMA neurology · 2020 · NCT02954978

These findings — that nearly half of patients on 300mg had serious side effects versus 16% on placebo — were published in the JAMA neurology and represent the headline result of the study.

Researchers tracked outcomes across 75 patients enrolled in the trial. The result was consistent enough across the group that the team felt confident reporting it.

What this means for patients

For patients with parkinson's disease, this result changes the calculus on what to ask their care team about. Whether it changes day-to-day care depends on factors like disease subtype, prior treatments, and where the patient is in their care journey.

What you can do now

This was a mid-stage study showing nilotinib can reach the brain and affect chemicals involved in Parkinson's, but more serious side effects occurred with the drug compared to placebo. The treatment is not FDA-approved for Parkinson's. Larger studies are needed to know if it actually slows the disease. Talk to your doctor about proven Parkinson's treatments.

Eligibility for the treatments mentioned above depends on specific test results and clinical history. Bring this summary, the trial name, and your most recent labs or pathology report to your next visit.