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Expert Opin Biol Ther. 2013 Sep;13(9):1215-9. doi: 10.1517/14712598.2013.799129. Epub 2013 May 14.

It may be possible to delay the onset of neurodegenerative diseases with an immunosuppressive drug (rapamycin).


Rapamycin might have beneficial effects, some of them acting via autophagy, in several cellular, fly and mouse models of degenerative diseases such as Alzheimer's disease (AD), Parkinson's disease (PD), Huntington's disease (HD) and dominant spinocerebellar atrophy (SCA). Other more sophisticated mechanisms have been described such as intervention in cell signaling and cell transcription particularly related to PD. Yet AD, PD, HD and SCA3 are chronic degenerative diseases, and chronic administration of rapamycin at advanced clinical stages may result in deleterious systemic effects due to chronic inhibition of mammalian target of rapamycin (mTOR). Studies are needed to uncover more specific inhibitors of particular mTOR signaling pathways, and to establish realistic guidelines for treatment at early stages of neurodegenerative processes.

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