Michael S. Rafii, MD, PhD

Michael S. Rafii, MD, PhD, is Medical Director of the Epstein Family Alzheimer’s Therapeutic Research Institute (ATRI) and Professor of Clinical Neurology at the Keck School of Medicine of the University of Southern California. He is an internationally recognized Alzheimer’s disease clinical trialist whose work focuses on developing and evaluating therapies across the Alzheimer’s disease continuum, from the earliest presymptomatic stages through symptomatic disease.

Dr. Rafii helps lead a broad portfolio of national and international Alzheimer’s disease therapeutic trials, with particular emphasis on prevention, biomarker-guided treatment, innovative trial design, and the translation of emerging therapies into clinical practice. He is Associate Director of the NIH-funded Alzheimer’s Clinical Trials Consortium (ACTC) and Director of its Medical Safety Unit, providing medical and safety leadership across one of the world’s largest multicenter Alzheimer’s disease clinical trial portfolios.

As part of ATRI and ACTC’s broader Alzheimer’s disease research, Dr. Rafii leads programs focused on genetically determined Alzheimer’s disease in people with Down syndrome. He is Principal Investigator of the NIH-funded ACTC-Down Syndrome (ACTC-DS) program, which extends ACTC’s clinical-trial infrastructure and expertise to conduct clinical trials for Down syndrome-related Alzheimer’s disease.

Dr. Rafii previously led the Down Syndrome Biomarker Initiative (DSBI), the first study to apply modern Alzheimer’s biomarkers and multimodal neuroimaging to adults with Down syndrome. This work helped characterize the biological progression of Alzheimer’s disease in this population and contributed to the scientific foundation for subsequent prevention and treatment trials.

His work spans several of the major national programs shaping Alzheimer’s disease research and therapeutics. In addition to his leadership roles at ATRI and ACTC, Dr. Rafii is Co-Director of the Clinical Core of the Alzheimer’s Disease Neuroimaging Initiative (ADNI) and Co-Principal Investigator of the Alzheimer’s Network for Treatment and Diagnostics (ALZ-NET).

Dr. Rafii earned his MD and PhD degrees from Brown University, conducted neurogenetics research at Harvard Medical School, completed his neurology residency at the Johns Hopkins Hospital, and completed fellowship training in neurodegenerative diseases at the University of California, San Diego.

His work has been featured in The New York Times, The Washington Post, The Wall Street Journal, the Chicago Tribune, The Economist, and NPR. He has also served as a medical advisor for the Netflix documentary Remembering Gene Wilder and for the Emmy Award-winning television programs Grey’s Anatomy and This Is Us.

• Brown University School of Medicine: Aronson Prize for Excellence in Neuroscience, 2002
• The Johns Hopkins University/Sinai Hospital: Intern of the Year, 2003
• The Johns Hopkins Hospital: Preziosi Outstanding Neurology Resident Award, 2006
• The Johns Hopkins Hospital: Alpha Omega Alpha, 2006
• LuMind Down Syndrome Research Foundation: David Cox Award, 2016
• Alzheimer’s Association: Research Champion Award, 2022

• Life?s end Benefits of CannaBidiol and TetrahYdrocannabinol (LiBBY) Open-Label Extension (OLE) Study
  NIH · R01AG095004 · Aug 15, 2025 – Jul 31, 2028 · Role: Co-Principal Investigator
• Precision Medicine for Inflammatory Treatment for Alzheimer's Disease in Down Syndrome
  NIH · R01AG073979 · Sep 30, 2021 – Aug 31, 2026 · Role: Co-Principal Investigator
• Clinical trials to prevent Alzheimer's Disease in Down Syndrome
  NIH · R33AG066543 · Sep 15, 2019 – Aug 31, 2025 · Role: Principal Investigator
• Clinical trials to prevent Alzheimer's Disease in Down Syndrome
  NIH · R61AG066543 · Sep 15, 2019 – Aug 31, 2021 · Role: Principal Investigator
• ACTIVE IMMUNOTHERAPY FOR COGNITIVE DECLINE IN ADULTS WITH DOWN SYNDROME
  NIH · R01AG047922 · Aug 15, 2014 – Jun 30, 2019 · Role: Principal Investigator
• Alzheimer's Disease Cooperative Study
  NIH · U19AG010483 · Jul 1, 1997 – Feb 28, 2021 · Role: Co-Principal Investigator

• Santoro, Jonathan
• Aisen, Paul
• Rissman, Robert
• Thompson, Paul
• Schneider, Lon
• Ringman, John