Alzheimer's Matters Blog

Trial of First Broad-Spectrum Drug for Neurodegeneration Seeking Volunteers

April 28, 2017

Category: Developing Drugs

Diana Shineman and R. Scott Turner


Recently, I led a panel discussion of prominent Alzheimer’s researchers as part of our Seventh Annual Great Ladies Luncheon in Washington, D.C. Along with the our Senior Director of Scientific Affairs Dr. Diana Shineman, the panel included Dr. Michela Gallagher of Johns Hopkins and Dr. R. Scott Turner from Georgetown.

Dr. Turner told the captivated audience that he is now recruiting patients for a phase 2 clinical trial, which is being supported by the Alzheimer’s Drug Discovery Foundation (ADDF). The trial will test nilotinib—a drug already FDA-approved for leukemia—as a treatment for Alzheimer’s disease.

This exciting study was borne out of a wider initiative. In 2015, the ADDF convened a meeting at the New York Academy of Sciences, “Learning from Cancer to Advance Drug Development for Neurodegeneration.” We knew that cancer survivors were less likely to develop Alzheimer’s, and that the two diseases shared some common mechanisms. The meeting brought together experts to share what they knew and map out a path to use that combined knowledge to quickly develop new Alzheimer’s drugs.

Just two short years later, Dr. Turner says nilotinib could become the “first broad-spectrum neurodegenerative drug.” It works by spurring the removal of many misfolded, toxic proteins from the brain, including tau, beta-amyloid, TDP-43, and Lewy bodies. Because it may remove many of these toxic proteins, rather than just one as the current cohort of vaccines in development do, it has the potential to be more effective in the treatment of Alzheimer’s disease, as well as Parkinson’s and Lewy body dementia, among others.

Thanks to ADDF funding, Dr. Turner and his team are already recruiting patients for the nilotinib trial. To learn more, visit Georgetown’s clinical trial recruitment website or email the trial coordinator. 

Howard Fillit, MD is the Founding Executive Director and Chief Science Officer at the ADDF