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 leader in the development of treatments for the genetic form of Alzheimer’s disease that occurs in persons with Down syndrome (DSAD).

He is Principal Investigator of the NIH-funded Alzheimer’s Clinical Trials Consortium–Down Syndrome (ACTC-DS), an international clinical trials network dedicated to accelerating the development 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 neuroimaging to adults with Down syndrome. This work helped demonstrate that the biological progression of Alzheimer’s disease in Down syndrome closely parallels other genetic and sporadic forms of the disease and contributed to the scientific foundation for subsequent prevention and treatment trials.

His leadership extends across the broader Alzheimer’s clinical trials field. Dr. Rafii is Associate Director of the NIH-funded Alzheimer’s Clinical Trials Consortium (ACTC) and leads its Medical Safety Unit, which oversees the safety and conduct of one of the world’s largest Alzheimer’s disease clinical trial portfolios. He is also 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 research and treatment.

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.

Dr. Rafii’s work has been featured in The New York Times, The Washington Post, The Wall Street Journal, Chicago Tribune, The Economist, and NPR. He has also served as medical advisor for the Netflix film Remembering Gene Wilder and 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