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The Amyotrophic Lateral Sclerosis (ALS) Association announced that it has awarded Cambria Biosciences a $3.5 million contract to develop new drug compounds for treatment of ALS, also known as Lou Gehrig's disease. This partnership builds on previously funded efforts with Cambria to develop unique neuroprotective drug compounds that prevent cell death caused by misfolded proteins that aggregate together within the cell. Mutant copper-zinc superoxide dismutase, or SOD1, is a protein that is misfolded to form aggregates in the motor neurons of some people with inherited forms of ALS.


Funded through the Association's Translational Research Advancing Therapies for ALS (TREAT ALS) clinical trials and drug discovery program, the award to Cambria Biosciences supports the identification of compounds that can block mutant SOD1 protein aggregation and cell death and the selection of the most suitable compounds to reach motor neurons and their surrounding cells. This funding allows researchers to test these compounds in mice that have been bred to express mutant SOD1, which creates an animal model that mimics the human disease. The study is a collaboration between Dr. Donald Kirsch, Senior Vice President for drug discovery at Cambria Biosciences, Dr. Richard Morimoto, an international expert in protein misfolding (which can cause cellular damage associated with neurodegenerative diseases) from Northwestern University, and Dr. Richard Silverman, a leading medicinal chemist from Northwestern University. The project team presented results to date at the 2007 International Meeting of the Society for Neuroscience in San Diego, California in a poster entitled "Protein Aggregation and the Development of ALS Therapeutics," by R. Benmohamed and other researchers.


The compounds are optimized to produce the lowest toxicity, maximum potency and efficacy, and favorable pharmacological properties, such as oral activity, to develop an investigational new drug to enter clinical testing. Cambria will retain intellectual property and commercialization rights on resulting drug candidates, and the ALS Association will be eligible to receive royalties from Cambria on net sales of any approved products.


Currently there is only one Food and Drug Administration-approved compound on the market for ALS (Riluzole) with modest effects on disease progression. There is an urgent need for the development of new compounds with properties more suitable for the treatment of neurodegenerative diseases. The compounds identified through this consortium effort act on a mechanism thought to be involved not only in ALS but also other neurodegenerative disorders, such as Parkinson's and Huntington's disease. The successful development of such compounds will have a major impact on currently untreatable neurodegenerative disorders.


ALS is a motor neuron disease that was first identified in 1869 by the noted French neurologist Jean-Martin Charcot. Although the cause of ALS is not completely understood, the 1990s have brought a wealth of new scientific understanding about the physiology of this disease. ALS is a progressive neurodegenerative disease that affects nerve cells in the brain and the spinal cord. Motor neurons reach from the brain to the spinal cord and from the spinal cord to the muscles throughout the body. The progressive degeneration of the motor neurons in ALS eventually leads to their death. When the motor neurons die, the ability of the brain to initiate and control muscle movement is lost. With voluntary muscle action progressively affected, patients in the later stages of the disease may become totally paralyzed. For most people, however, through it all their minds remain unaffected.


For more information on TREAT ALS, please see The ALS Association's Web site under the research tab and Laboratory Models in ALS


Cambria Biosciences uses its chemical genetics platform to discover and elucidate drug candidates, internally and in partnership with other companies. Further information is available at