Could This Cancer Drug Help Alzheimer’s Patients?

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The Alzheimer’s Drug Discovery Foundation (ADDF) has just awarded a $2.1 million grant to R. Scott Turner, MD, PhD, of Georgetown University Medical Center.

The grant is to conduct a phase II clinical trial of low-dose nilotinib, an FDA-approved drug for the treatment of adult chronic myeloid leukemia, in patients with Alzheimer’s disease.

Nilotinib is marketed as Tasigna® for use as a cancer therapy, and in preclinical studies conducted by Georgetown researchers, it reduced cognitive impairment by targeting two of the underlying causes of Alzheimer’s—neuroinflammation and misfolded proteins.

Nilotinib triggers a process that is called autophagy, which removes the toxic proteins tau and beta-amyloid from the brain before they accumulate into plaques and tangles.

Charbel Moussa, MD, PhD, scientific and clinical research director for Georgetown’s TNP, conducted the preclinical research. He will also be a co-investigator on the Alzheimer’s trial.

Moussa 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.”

The trial will include 42 patients and is expected to start this year. Half the randomized patients will receive an escalating dose of nilotinib, while the other half will receive 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. 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.”