Raymond S. Turner, MD, PhD: Safety Results for Nilotinib in Alzheimer

“Our working hypothesis is that the drug is triggering autophagy, therefore, would help remove or digest any intraneuronal protein aggregate including neurofibrillary tangles, Lewy bodies, alpha-synuclein, aggregates, etc.”

At the virtual 2020 Alzheimer’s Association International Conference (AAIC) annual meeting, July 26—29, Raymond S. Turner, MD, PhD, presented phase 2 results on nilotinib, a drug whose previous past involved patients with Parkinson disease (PD). The purpose of the study was to measure and assess the safety, tolerability, and pharmacokinetics of nilotinib, while measuring biomarkers in patients with mild-to-moderate dementia due to Alzheimer disease (AD).

Patients in the study were randomized 1:1 to nilotinib 150 mg once orally or matching placebo for 26 weeks, followed by nilotinib 300 mg compared to matching placebo for another 26 weeks. At the end of the study, the treatment was able to reduce amyloid burden in both the temporal and frontal lobes compared to placebo.

Turner, the director of the Memory Disorders Program and a professor of neurology at Georgetown University Medical Center, sat down with NeurologyLive to discuss the safety findings as well as other notable data points from the study.