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Effects of PB-TURSO on the transcriptional and metabolic landscape of sporadic ALS fibroblasts

Fels, Jasmine A. and Dash, Jalia and Leslie, Kent and Manfredi, Giovanni and Kawamata, Hibiki (2022) Effects of PB-TURSO on the transcriptional and metabolic landscape of sporadic ALS fibroblasts. Annals of Clinical and Translational Neurology, 9 (10). pp. 1551-1564. ISSN 2328-9503. doi:10.1002/acn3.51648.

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ALS is a rapidly progressive, fatal disorder caused by motor neuron degeneration, for which there is a great unmet therapeutic need. AMX0035, a combination of sodium phenylbutyrate (PB) and taurursodiol (TUDCA, TURSO), has shown promising results in early ALS clinical trials, but its mechanisms of action remain to be elucidated. Therefore, our goal was to obtain an unbiased landscape of the molecular effects of AMX0035 in ALS patient-derived cells.

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Manfredi, Giovanni0000-0003-3893-1348
Additional Information:We thank Amylyx Pharmaceuticals for providing the compounds used for this study. We thank Dr Hiroshi Mitsumoto (Columbia University) and the COSMOS initiative for providing the fibroblast and plasma samples utilized in this work. We acknowledge the contribution of the Weill Cornell Genomics Resource Core Facility and the Medicine Meyer Cancer Center Proteomics & Metabolomics Core Facility. This work was supported by funds from the NIH/NINDS (grants R35 NS122209 to GM and R21 NS104520 to HK and GM). Sponsored research agreement with Amylyx Pharmaceuticals.
Funding AgencyGrant Number
NIHR21 NS104520
NIHR35 NS122209
Amylyx PharmaceuticalsUNSPECIFIED
Issue or Number:10
Record Number:CaltechAUTHORS:20220916-664913000
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Usage Policy:No commercial reproduction, distribution, display or performance rights in this work are provided.
ID Code:117011
Deposited By: Olivia Warschaw
Deposited On:29 Oct 2022 22:21
Last Modified:01 Nov 2022 18:19

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