ProMIS Neurosciences Inc. announced a publication identifying Receptor for Activated C-Kinase 1 (RACK1) as a novel misfolded protein target for ALS and frontotemporal lobar degeneration (FTLD). The article published in the online edition of Acta Neuropathologica Communications is titled, "Targeting RACK1 to alleviate TDP-43 and FUS proteinopathy-mediated suppression of protein translation and neurodegeneration." TAR DNA-binding protein 43 (TDP-43) and Fused in Sarcoma/Translocated in Sarcoma (FUS) are ribonucleoproteins associated with pathogenesis of ALS and FTLD. Under normal conditions, TDP-43 and FUS are predominantly localized in the nucleus of cells, where they participate in the regulation of protein expression.

In disease, however, they typically become mislocalized in the cytoplasm of neurons where they form aggregates. The study authors reported that pathological FUS and TDP-43 both co-aggregate with RACK1 resulting in suppression of protein synthesis. Importantly, they showed that removal (knock-down) of RACK1 can restore protein synthesis in a cell system and alleviate neurodegeneration in a fruit fly model of disease.