Michael S. Rafii, MD, PhD

Professor of Clinical Neurology

Associate Director of ACTC (Alzheimer's Clinical Trial Consortium) and Director of Medical Safety

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Overview

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 Associate Director of the NIH-funded Alzheimer’s Clinical Trials Consortium (ACTC) and Director of its Medical Safety Unit where he provides medical and safety oversight to one of the world’s largest AD clinical trial portfolio.

Dr. Rafii 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). He leads research on genetically-determined Alzheimer’s disease in people with Down syndrome as Principal Investigator of ACTC’s Down Syndrome program (ACTC-DS), which leverages ACTC’s infrastructure to develop and conduct trials for this population.

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 fellowship 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.

Awards

  • 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

Research Funding

  • 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

Publications

  • Blood pressure and transcranial ultrasound predict moyamoya vasculopathy in Down syndrome. Dev Med Child Neurol. 2026 Sep 29.. View in PubMed
  • Sex-specific biological aging clocks across organs and omics. Nat Med. 2026 Sep 16.. View in PubMed
  • Late-onset seizures as a sentinel of Alzheimer’s disease progression in Down syndrome. Lancet. 2026 Sep 26; 408(10561):1154-1156.. View in PubMed
  • Multi-dimensional attention framework for personalised Alzheimer’s disease progression prediction across sporadic and genetic risk cohorts. Res Sq. 2026 Aug 03.. View in PubMed
  • Author Correction: Sleep chart of biological ageing clocks in middle and late life. Nature. 2026 Aug; 656(8128):E39.. View in PubMed
  • Correction: Serum and cerebral folate are normal in Down Syndrome Regression Disorder. Mol Autism. 2026 Jul 30; 17(1).. View in PubMed
  • Delayed Recognition and Stroke-Predominant Presentation in Down Syndrome-Associated Moyamoya Syndrome. Stroke. 2026 Sep; 57(9):2731-2743.. View in PubMed
  • Relationships between longitudinal retinal amyloid imaging and amyloid PET in the A4 Trial. Alzheimers Dement (Amst). 2026 Jul-Sep; 18(3):e70422.. View in PubMed
  • Facial feature removal in magnetic resonance imaging scans of adults with Down syndrome: A de-facing methodological study. Alzheimers Dement. 2026 Jul; 22(7):e71614.. View in PubMed
  • Recent Cochrane review has serious flaws: A perspective of clinicians and researchers from around the world. Alzheimers Dement. 2026 Jul; 22(7):e71696.. View in PubMed