Tasigna (nilotinib), an approved leukemia medication being tested as a repurposed treatment for Parkinson’s disease, failed to show any clinically meaningful benefits in a Phase 2 trial, the study’s steering committee announced.
Although an announcement of the study’s topline data was originally planned for 2020, the trial’s steering committee unanimously agreed to disclose the findings earlier to inform the public and the scientific community as soon as possible.
Despite being safe and well-tolerated, Tasigna — approved to treat the blood cancer chronic myelogenous leukemia (CML) — failed to show any meaningful change in clinical symptoms or biological effect in patients with Parkison’s disease, the researchers said.
“No one wanted this trial to succeed more than I did. If I had qualified, I would have participated as a volunteer,” Gary Rafaloff, who was diagnosed with Parkinson’s in 2012 and is one of the members of the study’s steering committee, said in a press release.
“Instead I spent over two years working with the other committee members to ensure that the trial was designed and administered with the utmost rigor. 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,” Rafaloff said.
Tasigna, developed by Novartis, is approved by the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) to treat adults with CML, a type of blood cancer that typically affects older adults.
The medicine blocks the activity of a protein called BCR-ABL that is known to support cancer development. But this protein also is intimately linked to several mechanisms in the brain, such as oxidative stress and alpha-synuclein-induced neurodegeneration, which play critical roles in Parkinson’s and other brain disorders.
As such, the researchers began investigating whether Tasigna could be repurposed to treat Parkinson’s disease. Drug repurposing refers to the process of testing a medication with established safety in conditions other than those for which it was originally intended.
In a previous Phase 2 study (NCT02954978), treatment with a single dose of Tasigna improved the brain’s ability to use dopamine in people with Parkinson’s by reducing inflammation and levels of toxic alpha-synuclein.
Investigators from Northwestern University, in collaboration with the Parkinson Study Group (PSG), then initiated a second Phase 2 study (NCT03205488) called NILO-PD. The trial was designed to test the safety, tolerability, and efficacy of two doses of Tasigna — two capsules of 150 mg or 300 mg taken once daily — compared with a placebo, over a period of six months. A total of 76 patients with moderate-to-advanced Parkinson’s were recruited from 25 clinical sites throughout the U.S.
Topline data now revealed by the steering committee of NILO-PD have shown that while Tasigna was found to be safe and well-tolerated by the study’s participants, it failed to show any clinically meaningful benefits among those with Parkinson’s.
NILO-PD investigators will share detailed data from the study during the 3rd Pan American Parkinson’s Disease and Movement Disorders Congress, taking place February 14-16, 2020, in Miami. The researchers also are planning to make all data and biosamples available to the Parkinson’s research community for future analyses.
The clinical study was supported by several research and patient advocacy groups, including the Michael J. Fox Foundation for Parkinson’s Research, the Cure Parkinson’s Trust, the Van Andel Institute, the Parkinson Alliance and the Demoucelle Parkinson Charity.
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