Last year, we announced the formation of a six-lab collaboration at the direction of Project ALS to focus on the cell process known as autophagy, or a cell’s ability to clear itself of unwanted proteins. Autophagy Team director, Tom Maniatis, and his laboratory, have led efforts to identify what goes wrong with autophagy in ALS, and how we might fix it. The original team includes leading labs from Columbia, Cornell, University of California San Francisco, and the New York Genome Center. One major focus of this year’s work has been the gene TBK-1, a player in the cell clearing process. Project ALS and Dr. Maniatis recently added collaborators from the New York Structural Biology Center to generate helpful 3-D models of TBK-1 and other autophagy-related genes and targets for use by the team. More recently, Project ALS recruited the eminent Dr. Erika Holzbauer, from the University of Pennsylvania, who is focused on the dynamics of the active transport of cargoes—unwanted protein aggregates—in ALS neurons.
(photo: Drs. Steven Altschuler and Lani Wu of UCSF, researchers on the Project ALS Autophagy Team)
In 2020, Cathy and Richard called Project ALS after hearing about the development of a novel antisense oligonucleotide (ASO) therapy for people living with FUS ALS. So was born NextGen ALS, a Weber family initiative in collaboration with Project ALS that is committed to raising funds for targeted research toward treatments and cures for genetic forms of ALS.