New Clinical Trial Will Test Cancer Drug as Alzheimer’s Treatment

The Alzheimer’s Drug Discovery Foundation (ADDF) announces a $2.1 million grant awarded to R. Scott Turner, MD, PhD, of Georgetown University Medical Center to conduct a phase II clinical trial of low-dose nilotinib (marketed as Tasigna® for use as a cancer therapy) in patients with Alzheimer’s disease.

Nilotinib is an FDA-approved drug for the treatment of adult chronic myeloid leukemia. In preclinical studies conducted by Georgetown researchers, nilotinib reduced cognitive impairment by targeting two of the underlying causes of Alzheimer’s—neuroinflammation and misfolded proteins. Nilotinib triggers a process (called autophagy) that removes the toxic proteins tau and beta-amyloid from the brain before they accumulate into plaques and tangles.

Dr. Turner, co-medical director of Georgetown University Medical Center’s Translational Neurotherapeutics Program (TNP) and director of the Georgetown Memory Disorders Program, says, “By stimulating the brain’s normal autophagic process, which clears out these misfolded proteins in cells, we hope to prevent or slow the progression of Alzheimer’s. In fact, nilotinib may be a first—a broad-spectrum anti-neurodegenerative drug that targets all misfolded protein aggregates that accumulate in the brain of Alzheimer’s patients. By targeting both amyloid and tau, this study may point the way to a new strategy in Alzheimer’s disease treatment.”

The preclinical research was conducted by Charbel Moussa, MD, PhD, scientific and clinical research director for Georgetown’s TNP, who explains, “Nilotinib seems to activate the cell’s garbage disposal machine, reduce plaques and tangles and reverse cognitive decline in animal models of Alzheimer’s disease. We hope that this trial will clarify the effects of nilotinib in Alzheimer’s patients.” Moussa will be a co-investigator on the Alzheimer’s trial.

The trial is expected to start this year and will include 42 patients, with half randomized to receive an escalating dose of nilotinib, while the other half receives a placebo. The primary objectives of the study will be to test the drug’s safety and tolerability and to measure whether nilotinib reduces inflammation and the presence of beta-amyloid and tau in spinal fluid. Dr. Turner and his colleagues in the Georgetown Memory Disorders Program will also perform cognitive and functional abilities tests.

Dr. Howard Fillit, Founding Executive Director and Chief Science Officer of the ADDF, says: “The ADDF is proud to support a clinical trial that holds such promise for Alzheimer’s patients. This funding is part of our wider initiative to use the knowledge gained from cancer research to advance effective treatments for Alzheimer’s.”

The ADDF’s initiative “Learning from Cancer to Advance Treatments for Neurodegenerative Diseases” launched in 2015 with a conference held in partnership with the New York Academy of Sciences. Its goal is both to develop new therapies and test existing cancer therapies for their potential in treating Alzheimer’s disease. In addition to the nilotinib trial at Georgetown, this initiative includes funding for drug development projects at Oryzon Genomics, Rodin Therapeutics, and Yuma Therapeutics.

For information about volunteering for the clinical trial of nilotinib, visit Georgetown University's website.