ALS Drug Discovery

Amyotrophic Lateral Sclerosis (ALS or Lou Gehrig’s disease) is a progressive, ultimately fatal disorder. The only drug approved to treat ALS (Riluzole) is of limited value.

But in the last decade, much has been learned about ALS and its underlying disease mechanisms. To take this knowledge and translate it into possible treatments, the Harvard NeuroDiscovery Center has launched one of the largest nonprofit ALS drug discovery programs nationwide.

Thanks to the generous support of the ALS Therapy Alliance, Project ALS and private supporters, in July 2008 we established the Collaborative ALS Drug Discovery Initiative (CADDI). The aim of this new research program is to accelerate the development of new, effective treatments for individuals with ALS.

Collaboration is key to this Program’s success. We contend that ALS is far too complex for any single laboratory to solve on its own. Our CADDI Program is designed to solicit and select exciting new drug discovery projects from across the ALS research community. Each selected project will be championed by an investigator, who will temporarily relocate to work in our Cambridge, MA laboratory – along side our laboratory’s drug discovery experts!

Launched in June 2008, the CADDI is establishing a robust pipeline of early stage ALS drug discovery projects. The goal, of course, is to drive the most promising projects forward to the point that they attract interest from industry. Such partnerships are crucial to advancing the development of promising new drugs. And, by increasing the flow of promising ALS drug discovery leads to industry, we will increase the number of mature drug discovery projects pursued by industry!

The Harvard NeuroDiscovery Center and its Laboratory for Drug Discovery in Neurodegeneration is an ideal drug discovery platform for the Collaborative ALS Drug Discovery Initiative. Since 2001, its focus has been drug discovery for ALS and other neurodegenerative diseases. The Collaborative ALS Drug Discovery Initiative takes full advantage of the Center’s state-of-the-art facilities, proven track record and 14-person industry-seasoned staff of drug discovery experts. To learn more about the Laboratory for Drug Discovery in Neurodegeneration, click here.

A Steering Committee has been established to provide senior-level oversight of the Collaborative Drug Discovery Research Initiative. Its membership is diverse, ensuring objective and broad-based input. Members include:

• Ross Stein, PhD (Co-Chair)
  Director, Laboratory for Drug Discovery in Neurodegeneration
• Robert Brown, MD, DPhil (Co-Chair)
  Chair, Department of Neurology, University of Massachusetts Medical School and Memorial Medical Center
• Adrian Ivinson, PhD
  Founding Director, Harvard NeuroDiscovery Center
• Valerie Estees
  Co-Founder and Director of Research, Project ALS
• Tom Maniatis, PhD
  Professor of Molecular and Cellular Biology, Harvard University

For our neuroscience colleague who are interested in the technical details about the Collaborative ALS Drug Discovery Initiative, click here.