The ALS Association

ALS Ice Bucket Challenge Progress
Brian Frederick
The ALS Association
(202) 464-8612



Nearly $1 Million Awarded in Drug Development Contracts for New ALS Therapeutic Strategies

Washington, D.C. (February 9, 2017)— The ALS Association is pleased to announce the award of two new drug development contracts to accelerate the discovery of new treatments for ALS. The awards, totaling over $998,500, will fund preclinical development of new therapeutic strategies targeting known disease-related pathways in the central nervous system.

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.

The ALS Association’s TREAT ALS™ (Translational Research Advancing Therapy for ALS) portfolio funds research from early target identification to preclinical research and early pilot clinical trials. Within this portfolio, The Lawrence and Isabel Barnett Drug Development Program has enabled the development of novel therapeutics that are currently in clinical trials for ALS and related neurological disorders. It has been instrumental in building and fostering academic-industry partnerships and has contributed to the explosion in companies, small and large, entering the ALS field. Among other accomplishments, this milestone-based program made possible the development and clinical testing of antisense therapy against a common gene mutation in ALS, the first application of antisense therapy to any neurologic disease.

This vital program is named in honor of Lawrence and Isabel Barnett, both widely respected for their roles as a prominent entertainment executive and as actress in television and theater, respectively. They contributed their time, talent and treasure to The ALS Association after a family friend was diagnosed with ALS. Over the years, their contributions to the fight against ALS have advanced understanding of the disease through groundbreaking research.

The two new contracts continue this pioneering effort to develop novel therapies against the disease. The recipients are:

OptiKira LLC, of Cleveland, Ohio, which will validate the inhibition of IRE1α kinase/RNase as a therapeutic approach to ALS. ALS is characterized by accumulation of misfolded proteins. When this misfolding cannot be corrected, a cell death pathway is initiated. One of the triggers for this pathway is a protein called IRE1α. By blocking the activation of this trigger, survival has been prolonged in animal models of several diseases involving programmed cell death. With funding from The ALS Association, the company will perform proof-of-concept experiments to determine the potential for this approach in a mouse model of ALS, and explore novel blocking compounds to optimize important drug properties such as oral bioavailability and brain penetrance. OptiKira has received grants from the National Institutes of Health and the Harrington Discovery Institute, investment from BioMotiv and Arix Bioscience and is conducting its work with Charles River Laboratories. Funding for the project is $498,500 over 2 years. This grant is generously supported by The ALS Association Oregon and SW Washington Chapter.

Thomas Lloyd, M.D., Ph.D., of Johns Hopkins University, will test nuclear export inhibitors as a strategy for treatment in ALS due to the C9orf72 mutation. This mutation, the most common genetic cause of ALS, disrupts import of materials into the cell nucleus, which is believed to contribute to development of disease. Dr. Lloyd has shown in a fly model that blocking a critical protein required for nuclear export called exportin (XPO1) rescues neurodegeneration caused by the C9orf72 mutation. This is through a possible mechanism that prevents the loss of ALS-related protein TDP-43 from the nucleus. An XPO1 inhibitor KPT-350, which is orally bioavailable and brain-penetrant, has shown promise in animal models of traumatic brain injury, and will now be tested in multiple ALS disease models. Funding for the project is $500,000 over 2 years. This study is in collaboration with Karyopharm Therapeutics, University of Pittsburgh and Johns Hopkins University.

“We are excited to be able to fund these important projects in new therapeutic strategies,” said ALS Association Chief Scientist Lucie Bruijn, Ph.D., MBA. “Through our Lawrence and Isabel Drug Development Program, we are able to bring the most promising new ideas in therapy development quickly through the preclinical phase, to more rapidly advance the best ones into clinical trials. That is the key to getting new treatments for ALS, which we so desperately need.”

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

Powered by Blackbaud
nonprofit software