The ALS Association

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Carrie Martin Munk
The ALS Association
(571) 319-3047



First ALS Association/NCRI Clinical Pilot Study Will Test the Anti-inflammatory Drug Tocilizumab for ALS

Washington, D.C. (November 8, 2013) — As part of an initiative to encourage the use of biomarkers in Phase II clinical trials to strengthen trial outcomes, The ALS Association and the Neurological Clinical Research Institute (NCRI) of Massachusetts General Hospital are pleased to announce a partnership for funding a clinical pilot study of tocilizumab. 

ALS (amyotrophic lateral sclerosis), often referred to as Lou Gehrig’s Disease, is a progressive neurodegenerative disease that affects neurons (nerve cells) in the brain and the spinal cord. Eventually, people with ALS lose the ability to initiate and control muscle movement, which often leads to total paralysis and death within two to five years of diagnosis. Studies all over the world, many funded by The Association, are ongoing to develop effective treatments and cures for ALS.

The study will test whether tocilizumab, an anti-inflammatory drug, can slow disease progression in people with ALS. It will be led by Shafeeq Ladha, M.D., of the Barrow Neurological Institute in Phoenix, Ariz., and Richard Barohn, M.D., of Kansas University Medical Center in Kansas City, Kan. The MGH NCRI will provide study project and data management. The State University of New York, Upstate, will provide study monitoring and outcome measure training. Sites will be selected from the Northeast ALS Consortium (NEALS), a nationwide group of ALS clinical researchers with a long history of partnership with The ALS Association.

“There is an urgent need for better ALS treatments and therapeutic agents,” said Lucie Bruijn, Ph.D., Chief Scientist for The Association. “This pilot study will determine whether this drug, already approved for rheumatoid arthritis, can offer benefit in ALS.”

The study is funded through a partnership agreement between The ALS Association and NRCI to develop novel, high-potential treatments for people with the disease. “The NCRI and The ALS Association are extremely well-positioned to work with both academic and industry partners to rapidly bring the best ALS science into clinical testing,” said Merit Cudkowicz, M.D., Chief of Neurology at Massachusetts General and Director of NCRI. NCRI serves as the coordinating center for NEALS.

Clinical studies funded through the program must include measurement of pharmacodynamic markers, also known as biomarkers, to determine whether the treatment has altered its intended molecular target and to collect tissue samples for detailed analysis of response to therapy. In the tocilizumab study, researchers will measure inflammatory gene changes in blood cells and cerebrospinal fluid. Inflammation is thought to be a central part of the ALS disease process.

About The ALS Association
The ALS Association is the only national non-profit organization fighting Lou Gehrig’s Disease on every front.  By leading the way in global research, providing assistance for people with ALS through a nationwide network of chapters, coordinating multidisciplinary care through certified clinical care centers, and fostering government partnerships, The Association builds hope and enhances quality of life while aggressively searching for new treatments and a cure.  For more information about The ALS Association, visit our website at

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