Bold New Program Focuses on Accelerating
Drug Discovery for Lou Gehrig’s Disease

Roberta Friedman, PhD, ALS Association Research Department Information Coordinator  

[QUICK SUMMARY: The ALS Association’s new initiative TREAT ALS [Translational Research Advancing Therapy for ALS] combines efficient new drug discovery with priorities set for existing drug candidates, to accelerate clinical testing of compounds with promise for the disease. “Now tangible progress will be turned towards patients to produce treatment success,” according to Lucie Bruijn, Ph.D., The ALS Association’s science director and vice president. “Translational research and clinical trials will find the drugs which will prevent, halt, or significantly slow down disease progression.”]

The ALS Association’s new initiative TREAT ALS (Translational Research Advancing Therapy for ALS) combines efficient new drug discovery with priorities set for existing drug candidates, to accelerate clinical testing of compounds with promise for the disease (Click here to view a multimedia presentation describing TREAT ALS).

TREAT ALS Multimedia Presentation

“Our ultimate goal is to capitalize on scientific and technological progress to accelerate drug discovery and realize effective new therapy,” said Lucie Bruijn, Ph.D., ALS Association science director and vice president. “We understand far more about the biological basis of the disease and this knowledge has enabled design of laboratory models of ALS that have yielded innovative ideas and novel treatment strategies.

Already partnering with many organizations around the world including The National Institutes of Health (NIH), the largest single investor in research globally, The ALS Association brings together an expert team of scientific and business advisors to steer this initiative. “As part of this initiative we will support the development of novel compounds for large scale, U.S. Food and Drug Administration approved clinical trials and engage in small pilot trials of existing FDA-approved drugs,” said Bruijn.

Click here to view current funded projects in TREAT ALS.

Noted biologist Tom Maniatis, Ph.D., Thomas H. Lee Professor of Molecular and Cellular Biology of Harvard University, is head of the ALS Association-initiated program and has played a leading role in shaping this new focus. “As we continue to aggressively push forward on ALS research it is important to maintain our focus on the discovery and development of new ALS drugs,” Maniatis said. “TREAT ALS is an exciting initiative that will translate advances in research in academia and industry into drug development and clinical trials. I look forward to working with an outstanding group of scientists and neurologists on the TREAT ALS steering committee to intensify the search for an ALS cure.”

TREAT ALS aims to move good ideas from the research arena more rapidly into the clinic for clinic trials and then patient treatment.  This initiative does not currently provide new therapies and is designed as a research initiative, not a short term treatment initiative. The ALS Association periodically sends out Requests for Proposal (RFP) for investigators to design small pilot studies for clinical trials in ALS. TREAT ALS has already provided funding for several clinical projects.  For a list of those projects, click here and select TREAT ALS from the 'Category' drop-down box. See how TREAT ALS fits into The ALS Association's overall research strategy.