Genetics, Communication, and the Future of Alzheimer’s Prevention: A Conversation with Dr. Michael Rafii
From leading international clinical trials to advising Hollywood on accurate portrayals of Alzheimer’s, Dr. Michael Rafii is advancing both the science and the story of this complex disease. In Episode 3 of The Alzheimer’s Research Podcast, Dr. Rafii joins the conversation to explore how genetic research—particularly in individuals with Down syndrome—is unlocking critical insights into Alzheimer’s prevention.
Dr. Rafii is Professor of Clinical Neurology at the Keck School of Medicine and Medical Director of the Alzheimer’s Therapeutic Research Institute (ATRI). His research focuses on developing treatments for Alzheimer’s disease, including the genetic form that occurs in people with Down syndrome (DS). As Principal Investigator of the NIH-funded Alzheimer’s Clinical Trials Consortium – Down Syndrome (ACTC-DS), Dr. Rafii leads a global network of more than 20 expert sites working to better understand and prevent the disease.
In this episode, Dr. Rafii offers a compelling look at how Down syndrome provides researchers with a unique window into Alzheimer’s. Due to the presence of a third copy of chromosome 21—home to the amyloid precursor protein (APP) gene—virtually all individuals with Down syndrome will develop the hallmarks of Alzheimer’s disease by their 40s, with dementia symptoms often appearing in their 50s. This predictable timeline makes early detection and intervention research possible in a way not seen in sporadic cases of Alzheimer’s.
Dr. Rafii also discusses the evolution of Alzheimer’s research over the course of his career, including the role of advanced imaging and blood-based biomarkers in helping scientists visualize pathology, measure treatment response, and track disease progression. Finally, he emphasizes the need to communicate complex medical information in a way that is clear, compassionate, and accessible to families, caregivers, and the public.
Join us for a conversation that bridges clinical science, medical safety, and human connection—because the path to prevention begins with understanding. Listen now!