Evidence map›Paper›PMID 41596551›Full record

ArticleInternational journal of molecular sciences2026

Identification of KHS-101 as a Transcription Factor EB Activator to Promote α-Synuclein Degradation.

Haizhen Zhu, Anqi Ren, Ting Li, Tao Zhou, Ailing Li, Xin Pan, Liang Chen, Jiayi Chen

Abstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in International journal of molecular sciences, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 1 paper.

0numbers the graph read from it
0cells of the map it votes in
1citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

1 citing paper in PubMed.

  1. Review
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

8 authors.

Haizhen ZhuNanhu Laboratory, State Key Laboratory of Biomedical Analysis (SKLBA, Formerly Known as National Center of Biomedical Analysis, NCBA), Beijing 100039, China.
Anqi RenNanhu Laboratory, State Key Laboratory of Biomedical Analysis (SKLBA, Formerly Known as National Center of Biomedical Analysis, NCBA), Beijing 100039, China.
Ting LiNanhu Laboratory, State Key Laboratory of Biomedical Analysis (SKLBA, Formerly Known as National Center of Biomedical Analysis, NCBA), Beijing 100039, China.
Tao ZhouNanhu Laboratory, State Key Laboratory of Biomedical Analysis (SKLBA, Formerly Known as National Center of Biomedical Analysis, NCBA), Beijing 100039, China.ORCID 0000-0001-6222-2053
Ailing LiNanhu Laboratory, State Key Laboratory of Biomedical Analysis (SKLBA, Formerly Known as National Center of Biomedical Analysis, NCBA), Beijing 100039, China.
Xin PanNanhu Laboratory, State Key Laboratory of Biomedical Analysis (SKLBA, Formerly Known as National Center of Biomedical Analysis, NCBA), Beijing 100039, China.ORCID 0000-0002-1982-2261
Liang ChenNanhu Laboratory, State Key Laboratory of Biomedical Analysis (SKLBA, Formerly Known as National Center of Biomedical Analysis, NCBA), Beijing 100039, China.
Jiayi ChenNanhu Laboratory, State Key Laboratory of Biomedical Analysis (SKLBA, Formerly Known as National Center of Biomedical Analysis, NCBA), Beijing 100039, China.ORCID 0009-0006-0930-7924

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Neurodegenerative disorders are increasingly linked to a progressive decline in lysosomal function. Activating Transcription Factor EB (TFEB), a master regulator of lysosomal biogenesis and autophagy, has therefore emerged as a promising therapeutic strategy to enhance cellular clearance in these conditions. In this study, we identified KHS-101 as a novel TFEB activator through a high-throughput screen of blood-brain-barrier-permeable small molecules. We demonstrated that KHS-101 promotes TFEB nuclear translocation, enhances lysosomal biogenesis and proteolytic activity, and increases autophagic flux. Furthermore, KHS-101 significantly accelerates the degradation of pathogenic A53T mutant α-synuclein in a cellular model of Parkinson's disease, suggesting its potential to mitigate α-synuclein-mediated proteotoxicity and hold neuroprotective potential. Our findings identify KHS-101 as a potent TFEB activator and highlight the therapeutic potential of modulating the autophagy-lysosomal pathway for treating Parkinson's disease and related disorders.

Indexed as

alpha-SynucleinBasic Helix-Loop-Helix Leucine Zipper Transcription FactorsAnimalsAutophagyHumansLysosomesParkinson DiseaseProteolysisalpha-SynucleinBasic Helix-Loop-Helix Leucine Zipper Transcription FactorsTFEB protein, humanautophagy–lysosome pathwayKHS-101lysosome degradationParkinson’s diseaseTFEBα-synuclein

Identifiers

PMID41596551
PMCPMC12841447

What OpenQuestion holds

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Registered trials

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Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.