Alzheimer's Matters Blog

Gene Therapy, A New Frontier in Alzheimer’s

June 20, 2018

Category: Developing Drugs

Wolf Blitzer and Dr. Ron Crystal


This week on CNN, Wolf Blitzer highlighted a new gene therapy program for Alzheimer's disease. This potential new therapy would not have been possible without the support of the Alzheimer's Drug Discovery Foundation.

The ADDF began funding the gene therapy project at Weill Cornell Medical College in 2004. Dr. Ronald Crystal, a pioneer in the field, conducted the first gene therapy trials in humans. He and his colleagues at Weill Cornell have developed gene therapies for cancer, α1-antitrypsin deficiency, and Batten disease, among others. Dr. Crystal has now set his sights on Alzheimer's. He is collaborating with Dr. Gregory Petsko, a neuroscientist specializing in Alzheimer's and other neurodegenerative diseases.

Back in 1993, researchers discovered that the APOE gene was associated with the age of onset of Alzheimer's disease. People with two copies of the APOE4 variant of the gene are up to 12 times more likely to develop Alzheimer's and to get it at younger ages. (People can have APOE2, APOE3, or APOE4 variants and everyone has two copies. APOE3 is the most common and does not influence Alzheimer's risk. APOE2 appears to protect against Alzheimer's and lower risk.)

Though we have known about APOE for decades, developing a therapy to address it has been a long road. Gene therapy is difficult, but Dr. Crystal's breakthroughs for other diseases have paved the way for progress in Alzheimer's. The gene therapy he and his team developed is designed to replace the APOE4 gene with APOE2, which would significantly lower Alzheimer's risk. They expect to begin human clinical trials of their APOE gene therapy this year.

Gene therapy has enormous potential to reduce the incidence of Alzheimer's. People with the high-risk APOE4 gene account for at least 60% of all Alzheimer's cases. This therapy could potentially prevent millions from developing it.

Thanks to your support, the ADDF is helping to make gene therapy for Alzheimer's a reality.

Watch the Video


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