GUMC receives FDA clearance to begin clinical study of cancer drug in patients with Alzheimer's disease

Georgetown University Medical Center (GUMC) today announces the U.S. Food and Drug Administration has completed its review of an investigational new drug application (IND) for the use of nilotinib in a phase II clinical trial for patients with mild to moderate Alzheimer's disease.

The FDA also informed GUMC investigators that the study can proceed. The clinical trial is expected to begin this year at Georgetown University Medical Center with its clinical partner, MedStar Georgetown University Hospital.

The clinical trial is a phase II, randomized, double blinded, placebo-controlled study to evaluate the impact of low doses of nilotinib (sold as Tasigna®) on biomarkers and clinical outcomes in people with mild to moderate Alzheimer's disease.

The rationale for using nilotinib is based on research conducted at Georgetown and involves clearing the brain of accumulated beta-amyloid (Abeta) plaques and Tau tangles. Both biomarkers are hallmarks of Alzheimer's disease. Nilotinib appears to penetrate the blood-brain barrier and turn on the "garbage disposal" machinery inside neurons (a process known as autophagy) to clear Tau and Abeta and other toxic proteins.

"In a 2015 small study at Georgetown, patients with Parkinson's and dementia with Lewy bodies were given nilotinib. As my colleagues reported, all who completed the study had a reversal in disease progression, observed both clinically and in key biomarkers — the same biomarkers seen in Alzheimer's ," explains Scott Turner, MD, PhD, co-medical director of Georgetown University Medical Center's Translational Neurotherapeutics Program and director of the Georgetown Memory Disorders Program, who will lead the Alzheimer's study. "But even before the Parkinson's study, research in the laboratory strongly supported studying this drug in people with Alzheimer's. The promising results of the Parkinson's study gives an even stronger rationale."

Charbel Moussa, MD, PhD, conducted the preclinical research that led to the discovery of nilotinib for the treatment of neurodegenerative diseases.

"When used in higher doses for chronic myelogenous leukemia (CML), nilotinib forces cancer cells into autophagy or cell death. The dose used in CML treatment is significantly higher than what we will use in our Alzheimer's study," says Moussa, scientific and clinical research director for the Georgetown Translational Neurotherapeutics Program. "When used in smaller doses once a day, as in this study, nilotinib turns on autophagy for about four to eight hours — long enough to clean out the cells without causing cell death. Toxic proteins that build up again will be cleared when the drug is given again the next day."

Moussa is an inventor on a Georgetown University patent application for use of nilotinib and other tyrosine kinase inhibitors for the treatment of neurodegenerative disease.

The Alzheimer's Drug Discovery Foundation is generously supporting the study.

"We all share a common goal of eliminating the scourge of Alzheimer's and its tremendous burden on caregivers and on society," says Turner.

Source: Georgetown University Medical Center

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