Acetylon Data Suggest HDAC6 May Not Be Ideal ALS Target

Acetylon Pharma has developed a next-generation Class II-selective inhibitor of HDAC6 and reports preclinical data showing that it holds promise for treating multiple myleoma. The studies show that HDAC6 inhibition impaired chaperone mediated refolding and aggresome mediated clearance of misfolded proteins, promoting apoptotic destruction of the cancerous cell. While HDAC6 has been considered a therapeutic target for ALS, the mechanistic studies carried out in the multiple myleoma model, suggest that HDAC6 inhibition may instead aggravate disease progression in ALS and other neurodegenerative diseases associated with misfolded proteins.

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