Nilotinib Effects on Safety, Tolerability, and Biomarkers in Alzheimer's Disease.

ABSTRACT:

Objective

Preclinical evidence with nilotinib, a US Food and Drug Administration (FDA)-approved drug for leukemia, indicates improvement in Alzheimer's disease phenotypes. We investigated whether nilotinib is safe, and detectable in cerebrospinal fluid, and alters biomarkers and clinical decline in Alzheimer's disease.

Methods

This single-center, phase 2, randomized, double-blind, placebo-controlled study investigated the safety, tolerability, and pharmacokinetics of nilotinib, and measured biomarkers in participants with mild to moderate dementia due to Alzheimer's disease. The diagnosis was supported by cerebrospinal fluid or amyloid positron emission tomography biomarkers. Nilotinib 150 mg versus matching placebo was taken orally once daily for 26 weeks followed by niloti

SUBMITTER: Turner RS 

PROVIDER: S-EPMC7383852 | biostudies-literature | 2020 Jul

REPOSITORIES: biostudies-literature