$630,000 challenge grant to establish ALS mouse repository at The Jackson Laboratory

Date: November 3, 2010
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Bar Harbor, Maine -- Researchers seeking ways to treat and cure amyotrophic lateral sclerosis (ALS) will soon have better access to mouse models of the disease. A $315,000 challenge grant from The Tow Foundation will enable The Jackson Laboratory to establish a national ALS mouse model repository.

The new grant provides half the $630,000 required to cover the costs of creating the repository, with the balance of funding to be provided by the ALS Association and the ALS Therapy Alliance.

"This repository will acquire new ALS mouse models and rapidly deploy them to the scientific community," explains Michael E. Hyde, Jackson's vice president for advancement and external relations. "The Tow Foundation challenge leveraged generous gifts from ATA and ALSA. Together, these three donors will help speed the development of new ALS therapies."

Creation and rapid distribution of mouse models of ALS is crucial to finding treatments. The Jackson ALS repository will be designed to eliminate or reduce the logistical barriers to scientists' donating valuable new models of ALS, as well as to ensure the models' genetic quality control and make them available to the scientific community.

ALS, a progressive neurodegenerative disease that affects nerve cells in the brain and spinal cord, afflicts an estimated 30,000 Americans, with 15 new cases diagnosed each day. As this destructive disease progresses, motor neurons degenerate and die; the brain's ability to initiate and control muscle movement is lost; paralysis strikes the arms and legs; and speech, swallowing and breathing are affected. Tragically, many ALS sufferers survive only two to five years, and 90 percent of people diagnosed with the disease die within 10 years.

The new grant is one of several important initiatives that The Tow Foundation has funded to advance ALS research. Claire Tow is an ALS patient, and her husband, Leonard Tow, has become one of the nation's most passionate advocates for the study of the disease's causes.

Research in ALS mouse models has focused primarily on mutations in the SOD1 gene. Recent discoveries point to other possible gene culprits, as well as defects in RNA processing proteins, as keys to understanding the causes of the disease. These discoveries offer new hope for therapeutic intervention.

"The ALS Association is very excited to work with The Jackson Laboratory on this important initiative," says ALS Association Chief Scientist Lucie Bruijn, Ph.D. "The opportunities that have arisen with the discovery of TDP43 and FUS in developing new models for ALS to better understand the disease and develop therapies makes this initiative very timely. Advances in this field rely heavily on collaboration and easy access to tools."

Robert H. Brown, Jr., D.Phil., M.D., chair of the Department of Neurology at the University of Massachusetts Medical School, is director and organizer of the ALS Therapy Alliance. "An ALS repository will be a vital resource for researchers," Dr. Brown says. "The Jackson Laboratory is the ideal place to carry out this effort, and the ALS Therapy Alliance is happy to help support it."

According to Cat Lutz, Ph.D., Jackson's associate director for Genetic Resource Sciences Repository and the lead scientist on the grant, a key goal is to standardize the new mouse models, in terms of their genetic background, specific mutations and physiological characteristics.

"ALS mice have been notoriously difficult to work with," she says, "with different scientists getting different results with the SOD1 mouse. It turns out that a lot of those problems were due to variability in the mice themselves. At The Jackson Laboratory we spent a lot of time and effort to stabilize the SOD1 mouse lines in terms of genetic background and the mutation itself, and now that there are new genes of interest, we want to ensure that those mouse models are stable too, right from outset."

Dr. Lutz and other Jackson Laboratory scientists will reach out to the ALS research community to identify the appropriate models for the repository, starting with a Nov. 12 workshop at the Society for Neuroscience meeting in San Diego, Calif., sponsored by The ALS Association and the National Institute of Neurological Disorders and Stroke.

Dr. Lutz adds, "The ALS research community has really gotten behind the repository concept, and we want to thank The Tow Foundation, ALSA and the ALS Therapy Alliance for their support."

The Tow Foundation's investments focus on the support of innovative programs in the areas of groundbreaking medical research, the performing arts, higher education and vulnerable children and families, with a concentrated initiative on juvenile justice reform.

The Jackson Laboratory is a nonprofit biomedical research institution based in Bar Harbor, Maine. Its mission is to discover the genetic basis for preventing, treating and curing human diseases, and to enable research and education for the global biomedical community.

Contact(s): Joyce Peterson, 207-288-6058

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