Rapamycin treatment for Alzheimer’s disease and related dementias: a pilot phase 1 clinical trial
Rapamycin is not detectable in the CSF before or after treatment, but several Alzheimer's disease and inflammatory biomarkers increase after treatment. Our results highlight the need to better understand the biological effects and clinical impact of repurposing rapamycin for Alzheimer's disease.
Authors
- Mitzi M. Gonzales (ORCID: https://orcid.org/0000-0002-8467-0938)
- Chen‐Pin Wang (ORCID: https://orcid.org/0000-0002-1670-2838)
- Sudha S. Seshadri (ORCID: https://orcid.org/0000-0001-6135-2622)
- Gabriel A. de Erausquin (ORCID: https://orcid.org/0000-0001-6423-4461)
- Leslie A. Linehan
- Randall John Bateman (ORCID: https://orcid.org/0000-0002-7729-1702)
- Yingxin He (ORCID: https://orcid.org/0000-0003-2543-4553)
- Valentina R. Garbarino (ORCID: https://orcid.org/0000-0002-0973-7186)
- Nicolas Musi (ORCID: https://orcid.org/0000-0003-4242-2888)
- Marisa Lopez‐Cruzan (ORCID: https://orcid.org/0000-0003-0285-1012)
- Candice E. Van Skike (ORCID: https://orcid.org/0000-0002-9258-7771)
- Tiffany F. Kautz (ORCID: https://orcid.org/0000-0001-7680-4708)
- Miranda E. Orr (ORCID: https://orcid.org/0000-0002-0418-2724)
- Xuemei Song (ORCID: https://orcid.org/0000-0002-9020-2022)
- Verónica Galván (ORCID: https://orcid.org/0000-0002-4501-8568)
- Ellen Kraig
- Dean L. Kellogg (ORCID: https://orcid.org/0000-0002-6550-3296)
Institutions
- Boston University (US)
- Cedars-Sinai Medical Center (US)
- The University of Texas at San Antonio Health Science Center (US)
- Washington University in St. Louis (US)
- Oklahoma City VA Health Care System (US)
- South Texas Veterans Health Care System (US)
- Wake Forest University (US)
- University of Oklahoma Health Sciences Center (US)
- Salisbury University (US)
Publication Details
- Journal
- Communications Medicine
- Published
- 2025-05-20
- DOI
- https://doi.org/10.1038/s43856-025-00904-9
- Citations
- 19
- Primary Topic
- Cholinesterase and Neurodegenerative Diseases
- Type
- article
- Field-Weighted Citation Impact
- 26.50
Funders
- U.S. Department of Veterans Affairs
- Foundation for Barnes-Jewish Hospital
- Cure Alzheimer's Fund
- GHR Foundation
- University of Texas Health Science Center at San Antonio
- National Institute on Aging
- National Heart, Lung, and Blood Institute
- National Institute of Neurological Disorders and Stroke
- National Center for Advancing Translational Sciences