ALS Association lists year's Top 10 research achievements
Topping the list and partly funded by Ice Bucket Challenge donations was the discovery by a global team of NEK-1, a gene now understood to be a common contributor to ALS.
Additionally, a neurological imaging team at Massachusetts General Hospital successfully scanned an ALS patient to measure brain inflammation. The U.S. Food and Drug Administration accepted and is now reviewing an application for Edaravone to treat the disease intravenously; while a team led by Dr. Aaron Gitler identified a new treatment for ALS related to mutations in the C9orf72 gene.
Cedars-Sinai researchers obtained FDA approval for a stem cell-gene therapy clinical trial; another group, Brainstorm Cell Therapeutics, obtained significant results from a U.S.-based NurOwn (stem cell therapy platform) Phase II trial and plans Phase III for this year.
In a win for big data, an IBM revealed five new ALS genes in collaboration with Phoenix’s Barrow Neurological Institute.
Collaboration between U.S. and Irish scientists yielded a successful Phase II trial for arimoclomol in SOD1-type ALS, related to a chromosome 21 gene. Finally, international cooperation also resulted in two programs, ALS ONE and NeuroLINCS, based on ALS Association funding to continue research.
In October, the ALS Association pledged $3.5 million in Ice Bucket Challenge funds to a new nationwide precision medicine initiative in nine U.S. universities and hospitals.