Transcription factor EB: an emerging drug target for neurodegenerative disorders

Ju Xian Song*, Jia Liu, Yimin Jiang, Zi Ying Wang, Min Li*

*Corresponding author for this work

Research output: Contribution to journalReview articlepeer-review

28 Citations (Scopus)


The discovery of transcription factor EB (TFEB) as a master regulator of the autophagy–lysosomal pathway (ALP) has triggered increasing numbers of studies that aim to explore the therapeutic potential of targeting TFEB to treat neurodegenerative disorders (NDs) such as Alzheimer's disease and Parkinson's disease. So far, the findings are exciting and promising. Here, we delineate the dysfunction of the TFEB-mediated ALP in NDs, and we summarize small molecules that have been identified as TFEB activators, along with their protective effects in NDs. We discuss the molecular mechanisms and targets, and the pros and cons of these TFEB activators from the perspective of drug development. Specific and potent small-molecule TFEB activators with ideal brain bioavailability could provide a method for treating NDs.

Original languageEnglish
Pages (from-to)164-172
Number of pages9
JournalDrug Discovery Today
Issue number1
Early online date22 Oct 2020
Publication statusPublished - Jan 2021

Scopus Subject Areas

  • Drug Discovery
  • Pharmacology


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