The ALS Association

ALS Ice Bucket Challenge Progress
Carrie Munk
The ALS Association



University of Miami and Orphazyme Announce Successful Completion of Phase II Trial of Arimoclomol in SOD1 ALS

Washington, D.C. (December 9, 2016) — Today at the International ALS/MND Symposium in Dublin, Professor Michael Benatar, Walter Bradley Chair in ALS Research and Executive Director of the Kessenich Family ALS Center at the University of Miami Miller School of Medicine, presented data from a recently completed phase II study of arimoclomol. The study was aimed at people living with amyotrophic lateral sclerosis (ALS) with rapidly progressive disease caused by mutations in the SOD1 gene, the second most common cause of inherited ALS. The ALS Association funded this study, along with the U.S. Food and Drug Administration’s Orphan Products Development Program. Pharmaceutical company, Orphazyme ApS provided the study medication.

ALS is a progressive neurodegenerative disease that affects 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. For unknown reasons, veterans are twice as likely to develop ALS as the general population. There is no cure, and only one drug modestly extends survival by only a few months.

Data presented today demonstrate safety and preliminary evidence for efficacy of arimoclomol, a heat shock protein-based therapy targeted to a small population of people with SOD1 ALS. The heat shock response in cells is a protective system responsible for handing cell stress and are involved in proper folding of proteins. For more detailed information about the study, please read the press release out of Orphazyme and the University of Miami.

"This was one of the first trials that The ALS Association funded which was focused on a specific ALS population, those people carrying a SOD1 mutation. It set the stage for future studies in a sub population of ALS patients and it made use of the TREAT ALSTM NEALS Clinical Trials network. I am very pleased that Dr. Benatar will be working with Orphazyme to move this study as rapidly as possible to the next phase,” stated Dr. Lucie Bruijn,MBA, Ph.D., Chief Scientist of The ALS Association.

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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