This paper demonstrates that levacetylleucine modulates TFEB activity in a bidirectional manner, normalizing lysosomal function in both wild-type and NPC1 knockout cells.

bioRxiv · Dec 2, 2025 · PreprintPharmacology & Toxicology

Stereospecific rapid activation of Transcription Factor EB (TFEB) by Levacetylleucine (NALL)

Lianne C. Davis, Rebecca Brain, Grant C. Churchill, Mallory Factor, Taylor Fields, Frances M. Platt, Marc C. Patterson, Michael Leo Strupp, Antony Galione

This paper shows that NALL activates TFEB, enhancing lysosomal and autophagic function, which may explain its therapeutic effects in neurodegenerative diseases.

bioRxiv · Jun 6, 2025 · PreprintPharmacology & Toxicology

Loss of NPC1 enhances phagocytic uptake and impairs lipid trafficking in microglia

Alessio Vittorio Colombo, Lina Dinkel, Stephan A. Müller, Laura Sebastián Monasor, Martina Schifferer, Ludovico Cantuti‐Castelvetri, Jasmin König, Lea Vidatic, Tatiana Brémovà-Ertl, Andrew P. Lieberman, Silva Katusic Hecimovic, Mikael Simons, Stefan F. Lichtenthaler, Michael Leo Strupp, Susanne A. Schneider, Sabina Tahirović

This study shows that loss of NPC1 in microglia increases phagocytic uptake and impairs lipid trafficking, leading to myelin accumulation and potential therapeutic implications.

Nature Communications · Feb 24, 2021Peer-reviewedNeuroscience

Acetyl-Leucine slows disease progression in lysosomal storage disorders

Ecem Kaya, David Aitchison Smith, Claire Smith, Lauren Morris, Tatiana Brémovà-Ertl, Mario Cortina‐Borja, Paul Fineran, Karl Morten, Joanna Poulton, Barry Boland, John Spencer, Michael Leo Strupp, Frances M. Platt

This study shows that ADLL and its enantiomer ALL slow disease progression in lysosomal storage disorders through mechanisms involving glucose and antioxidant metabolism.

Brain Communications · Sep 15, 2020Peer-reviewedNeuroscience