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ALS Drug Discovery Meeting Forges Partnerships for Faster Drug Development

March 13, 2012

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.”

ALS is not alone in its search for treatments, said Christopher Austin, M.D., Scientific Director of the Center for Translational Therapeutics at the National Institutes for Health, who gave the keynote address. “There are over 4,000 diseases whose genetics are known, but fewer than 250 currently have any treatments. So the question is: how can we translate genomic insights into therapeutics?”

One big advantage with ALS is the large number and high caliber of researchers involved in searching for therapeutics. However, that alone cannot lead quickly to new drugs, a point made by multiple speakers throughout the meeting. Basic research identifies potential “targets,” those molecules or processes in cells where an intervention might help. With a target in sight, the next step is to find a “hit,” a molecule that can interact with the target to alter the course of the disease.

Nevertheless, a hit is not the same as a successful drug. The process of turning a hit into a drug that can be tested in humans is so fraught with difficulty it is sometimes called “the valley of chaos.” Successfully crossing that valley has become a major challenge in drug development, several speakers said.

Recognizing that problem, government agencies and The ALS Association have focused attention and resources on bridging the gap between basic research and clinical trials. In its support for biotechnology companies that focus on drug development, The Association’s TREAT ALS program is critical for this effort. Programs at the National Institutes of Health (NIH) and the National Institute for Neurologic Disorders and Stroke (NINDS) are funding similar efforts.

“The goal is to bring new discoveries forward as quickly as possible,” continued Dr. Bruijn. “By forming partnerships among all the stakeholders in the drug development process, from the bench to the bedside, we can more rapidly take the most promising ideas and get them into the clinic for testing.”

The meeting was meant to facilitate exactly those partnerships, and if participants’ comments were any indication, it succeeded. “It was an exhausting meeting,” one said at the end, “but it was one of the best meetings of its kind I’ve been to.”

More detailed reports about the meeting will be posted in the coming weeks. Check back here for links and additional details.

Along with The ALS Association, the following organizations sponsored the workshop: The ALS Society of Canada, The ALS Association Greater Philadelphia Chapter, The Alan Phillips Discovery Awards, Biogen Idec, Bristol-Myers Squibb, Cytokinetics, Isis Pharmaceuticals, Knopp Biosciences, NINDS, Office of Rare Disease Research, NIH, and Pfizer Neuroscience.