Neurology

Cell culture studies suggest inhibiting HDAC6 could help treat ALS. In ALS patient-derived motor neurons, inhibition of HDAC6 with two tool compounds or antisense oligonucleotide (ASO)-mediated knockdown decreased axon transport defects -- a cause of neuromuscular junction dysfunction -- compared with vehicle or normal HDAC6 expression. Next steps including testing HDAC6 inhibitors in mouse models of ALS.

Celgene Corp. has two oral HDAC6 inhibitors: citarinostat (ACY-241), in Phase I testing to treat melanoma, multiple myeloma (MM), non-small cell lung cancer (NSCLC) and solid tumors; and ricolinostat (ACY-1215), in Phase II testing to treat MM and in Phase I/II testing to treat lymphoma. Celgene and Regenacy Pharmaceuticals LLC also have ricolinostat in preclinical testing to treat chemotherapy-induced peripheral neuropathy (CIPN)...