Association-Funded Scientists Identify Major Steps to Accelerate Therapy Development

November 13, 2014

Progress in the search for new treatments for ALS comes most quickly when researchers work together, sharing data and new ideas. That spirit of cooperation was at the center of the Investigator Research Workshop, held in October, which brought together almost 100 top ALS researchers whose work is funded by The ALS Association. Over three full days, the scientists presented their latest data, brainstormed about new approaches, and began new collaborations to accelerate the pace of treatment discovery.

“We planned this meeting as a way to bring people together,” said Association Chief Scientist Lucie Bruijn, Ph.D., M.B.A., “and we couldn’t be more pleased with the outcome.” The group included top academic scientists and clinicians from the United States, Canada, and several European countries, as well as researchers from biotech and pharmaceutical companies developing therapies for ALS. The program encompassed the newest findings from genes to clinical trials and from stem cells to drug discovery and development. A social networking evening, hosted by the Philadelphia Chapter of The ALS Association, increased the opportunities for contact and kindling of collaborations.

Among the highlights of the meeting was a set of roundtable working groups, each focused on identifying concrete steps that can be taken to move forward opportunities in genetics, disease models, data and tissue banking, biomarkers, and other key topics. Each discussion group produced a detailed set of action items that can now be used to catalyze new initiatives. “These working groups provided us with a terrific list of opportunities that we will be using to set the agenda across the spectrum of therapy development,” Dr. Bruijn said.

In addition, researchers shared details of both their newest developments and the roadblocks they have encountered along the way and sought feedback from other specialists in the field to determine the best path forward. Those new developments included potentially major advances in understanding the disease process, fuller details of which will become public in the coming months as the work is published. David Borchelt, Ph.D., of University of Florida, Gainesville, who co-chaired the meeting, said that all the new work presented “gives us important new ideas about developing new treatments for ALS. This meeting was a real success in this regard.”

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