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.

Dr. Rafii is an internationally recognized Alzheimer’s disease clinical trialist and a leading investigator in efforts to understand, prevent, and treat the genetically determined form of Alzheimer’s disease that occurs in people with Down syndrome (DSAD).

He is Principal Investigator of the NIH-funded Alzheimer’s Clinical Trials Consortium–Down Syndrome (ACTC-DS), a clinical trials network created to accelerate the development and evaluation of Alzheimer’s therapies for people with Down syndrome.

Dr. Rafii 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 establish that the biological progression of Alzheimer’s disease in Down syndrome closely parallels that seen in other forms of Alzheimer’s disease and helped lay the scientific foundation for subsequent prevention and treatment trials in this population.

His leadership extends across the broader Alzheimer’s clinical research field. Dr. Rafii is Associate Director of the NIH-funded Alzheimer’s Clinical Trials Consortium (ACTC) and Director of its Medical Safety Unit, with responsibility for medical safety oversight across one of the world’s largest Alzheimer’s clinical trial portfolios. He 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), major national programs advancing Alzheimer’s disease clinical research, and the implementation of newly approved Alzheimer’s therapies.

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 served as 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