ALS Drug Discovery Meeting Forges Partnerships for Faster Drug Development

Via Scoop.itALS Lou Gehrig’s Disease
For three long and intense days in early March, The ALS Association brought together more than 120 researchers, drug developers, government officials, and others to brainstorm ways to accelerate drug discovery for the treatment of ALS. The meeting was packed with presentations, discussions, and ideas for collaboration to solve what everyone recognized is a difficult problem. Although participants didn’t minimize the difficulty of the challenge, they expressed optimism that progress is being made. “The last eight months have been the most exciting in the history of ALS genetics,” according to Don Cleveland, Ph.D., Professor of Medicine, Neurosciences, and Cellular and Molecular Medicine at the University of California at San Diego. The discovery of the C9ORF72 gene mutation, which is responsible for more than one quarter of familial ALS, has set the field abuzz with new ideas for understanding the causes of the disease. In addition, the newly discovered ubiquilin2 gene emphasizes the importance of protein aggregation as a likely mechanism in the disease. Other new findings have also been recognized for their potential importance in understanding the causes of ALS and for pointing to likely targets for drug development. These include excitotoxicity, toxic accumulation of RNA and inflammation. “The excitement is palpable among ALS researchers that we are getting closer to truly understanding the process of the disease,” said ALS Association Chief Scientist Lucie Bruijn, Ph.D., who organized the meeting. “That understanding is crucial to developing treatments that have the best hope for slowing or stopping ALS.”

Via www.alsa.org

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