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Review
. 2019 Jan 23;11(476):eaar4289.
doi: 10.1126/scitranslmed.aar4289.

Rapamycin and Alzheimer's disease: Time for a clinical trial?

Affiliations
Review

Rapamycin and Alzheimer's disease: Time for a clinical trial?

Matt Kaeberlein et al. Sci Transl Med. .

Abstract

The drug rapamycin has beneficial effects in a number of animal models of neurodegeneration and aging including mouse models of Alzheimer's disease. Despite its compelling preclinical record, no clinical trials have tested rapamycin or other mTOR inhibitors in patients with Alzheimer's disease. We argue that such clinical trials should be undertaken.

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Conflict of interest statement

Competing interests: M.K. serves on the Scientific Advisory Board of resTORbio Inc. V.G. is an inventor on U.S. patent applications 13/128,800, 14/435,306, and 61/790,485 regarding the use of encapsulated rapamycin for treating a variety of conditions; V.G. also consults for Rapamycin Holdings Inc.

Figures

Fig. 1
Fig. 1. mTOR links aging and AD.
The top part of the figure depicts the involvement of mTOR in key processes common to aging. These hallmarks of aging contribute, in different degrees, to an increased risk for AD and other age-associated neurological diseases, as well as cognitive decline during normative aging. Rapamycin and other pharmacological interventions that attenuate mTOR activity may be beneficial for delaying the progression of AD and other age-associated dementias, as well as for preserving brain function in the healthy elderly.

Comment in

References

    1. Qiu C, Kivipelto M, von Strauss E, Epidemiology of Alzheimer’s disease: Occurrence, determinants, and strategies toward intervention. Dialogues Clin. Neurosci 11, 111–128 (2009). - PMC - PubMed
    1. Kaeberlein M, Rabinovitch PS, Martin GM, Healthy aging: The ultimate preventative medicine. Science 350, 1191–1193 (2015). - PMC - PubMed
    1. López-Otín C, Blasco MA, Partridge L, Serrano M, Kroemer G, The hallmarks of aging. Cell 153, 1194–1217 (2013). - PMC - PubMed
    1. Kaeberlein M, Translational geroscience: A new paradigm for 21st century medicine. Transl. Med. Aging 1, 1–4 (2017). - PMC - PubMed
    1. Johnson SC, Sangesland M, Kaeberlein M, Rabinovitch PS, Modulating mTOR in aging and health. Interdiscip. Top. Gerontol 40, 107–127 (2015). - PubMed

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