In a news release today, The ALS Association announced its financial commitment of $5 million to The Neuro Collaborative in an effort to develop and expedite therapeutic approaches for ALS. The Neuro Collaborative will combine the efforts of three leading California laboratories focused on ALS: The Svendsen lab at Cedars-Sinai in Los Angeles, the Cleveland lab at the University of California San Diego, and the Finkbeiner lab at the Gladstone Institutes, which is affiliated with UCSF.
The project is made possible by the unprecedented outpouring of support from the Ice Bucket Challenge. The Neuro Collaborative is one of four major new initiatives by The Association as a direct result of that support.
The goal of the Neuro Collaborative is to discover and develop potential new therapies for ALS, which can be delivered to pharmaceutical companies for further development in clinical trials. Early development of potential therapeutics is a major bottleneck in ALS therapy development and represents a significant opportunity for accelerating new treatments.
The Neuro Collaborative will employ a three-pronged approach, drawing on the unparalleled expertise of the three associated labs. Don Cleveland, Ph.D., of the Ludwig Institute at UC San Diego, will spearhead the development of antisense therapy against the C9orf72 gene, the most common genetic cause of ALS. Antisense therapy has been shown to be safe in people with ALS and is one of the most promising therapeutic technologies in neurodegenerative diseases. Steven Finkbeiner, M.D., Ph.D., of the Gladstone Institutes and UCSF, will further develop robotic technology for screening drugs in motor neuron cell culture.
Targets include reducing protein misfolding and increasing misfolded protein clearance mechanisms, both key problems in ALS. The ability to track the response of individual motor neurons to drugs holds the potential of rapidly finding agents that can be moved toward clinical trials. Clive Svendsen, Ph.D., of Cedars-Sinai, will develop The ALS Association and Cedars-Sinai Stem Cell and Motor Neuron Core Facility to create clinical-grade induced pluripotent stem cell (iPSC) lines from people with ALS, which will be openly shared with the ALS research community. iPSCs can give rise to the motor neurons that die in ALS and have emerged as a key research tool and potential source of therapeutic cells. iPSCs are a key source of motor neurons for drug discovery efforts, such as those at the Gladstone Institutes.
The Golden West Chapter of The ALS Association has played a key role in the planning and development of this project.
Cooperative synergies among the four new initiatives announced today (ALS ACT, New York Genome Centre, The Neuro Collaborative, and Project MinE) will increase the quantity and most importantly the value of data available for ALS research.
The ALS Association’s Board of Trustees will be meeting in mid-October to discuss and vote on additional funding decisions related to the use of Ice Bucket Challenge donations.