A trial of great interest to the Parkinson’s community today reported top-line findings. The Nilotinib in Parkinson’s Disease (NILO-PD) study showed that nilotinib, an FDA-approved treatment for chronic myelogenous leukemia being tested for potential repurposing as a Parkinson’s drug, was safe and tolerable in its trial population but does not exert a clinically meaningful benefit or biological effect to benefit those with Parkinson’s disease.

The NILO-PD Steering Committee unanimously agreed to accelerate the announcement of these results, originally planned for 2020, in line with members’ commitment to keep the Parkinson’s community informed of new scientific findings as quickly as possible.

Gary Rafaloff of Marlboro, New Jersey, diagnosed with Parkinson’s in 2012, served as a member of the NILO-PD steering committee. “No one wanted this trial to succeed more than I did,” Rafaloff said. “Nevertheless, the results are what they are. The good news is that there are several other upcoming trials that we can focus on as we look forward to future success.”

The randomized, placebo-controlled, double-blind study was led by Northwestern University and carried out at 25 sites through the Parkinson Study Group. NILO-PD — which tested placebo, 150mg and 300mg doses of nilotinib daily over six months in 76 participants with moderate to advanced Parkinson’s — was supported by an international consortium of research and patient advocacy groups including The Michael J. Fox Foundation.

“Science is a high-risk endeavor and too often strategies, even promising ones, do not prove replicable or scalable. Unfortunately, the results we observed — as measured by change in clinical symptoms and influence on biological measures — did not support testing nilotinib in a larger study,” said principal investigator Tanya Simuni, MD. “This is not the outcome we hoped for, but we remain dedicated to pursuing better treatments by advancing other potential therapies in today’s robust Parkinson’s pipeline.”

Simuni noted that it is critical that anyone considering adding nilotinib to their Parkinson’s treatment regimen work closely with their health care provider before doing so. While nilotinib was seen to be safe and tolerable in the study population, the study was strict with health history, excluding those who may have heart issues or other health challenges. Therefore, its safety in a broader Parkinson’s population remains unproven.

Nilotinib (marketed as Tasigna) inhibits the activity of c-Abl, a protein that has been linked to cellular pathways associated with Parkinson‘s disease. The c-Abl pathway remains an important target of interest to Parkinson’s researchers, with trials of other drugs in this class ongoing.

NILO-PD leaders will share more detailed data in February at the 3rd Pan American Parkinson’s Disease and Movement Disorders Congress in Miami, Florida. The partners also plan to make data and biosamples available for further analysis to the wider Parkinson’s research community.

“When the science doesn’t pan out, it’s easy to feel deflated. As a patient, I get it,” said Michael J. Fox. “But even a study that doesn’t show the results we hoped for ultimately gets us closer to the one that will.”

Read more in the press release announcement.

Join NILO-PD Steering Committee members for a webinar on Tuesday, December 17 at 12 p.m. ET to hear more about the study’s design and top-line findings and to ask questions. Register at www.michaeljfox.org/webinars.