Clinical Trial Supported by $2.1M ADDF Grant to Test Cancer Treatment in Alzheimer’s Patients

Clinical Trial Supported by $2.1M ADDF Grant to Test Cancer Treatment in Alzheimer’s Patients
The Alzheimer’s Drug Discovery Foundation (ADDF) announced that a Georgetown University Medical Center researcher has been awarded a $2.1 million grant to conduct a Phase 2 clinical trial of low-dose nilotinib (Tasigna, a cancer therapy) in patients with Alzheimer’s disease. The grant was awarded as part of the ADDF’s initiative “Learning from Cancer Advance Treatments for Neurodegenerative Diseases,” launched in 2015 with the goal of developing new therapies and testing existing cancer therapies for their potential in treating Alzheimer’s. Nilotinib is approved by the U.S. Food and Drug Administration (FDA) as a treatment for adult chronic myeloid leukemia (CML). Scott Turner, MD, PhD, who won the grant, will lead the Phase 2 trial evaluating nilotinib's safety and tolerability in about 42 Alzheimer's patients. Other objectives include measuring whether the drug reduces inflammation and the presence of beta-amyloid and tau in spinal fluid, and evaluating patient response to tests of functional and cognitive abilities. The study is expected to begin this year, with patients randomized to receive an escalating dose of nilotinib or placebo in a 1:1 ratio. In previous preclinical studies of nilotinib, a team of Georgetown researchers found that the drug targeted neuroinflammation and misfolded proteins — two of the underlying causes of Alzheimer’s — to potentially reduce cognitive impairment. Nilotinib was seen to trigger a process that removes the toxic proteins tau and beta-amyloid from the brain before they gather into plaqu
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