August 08, 2005 08:00 AM US Eastern Time zone
CALABASAS HILLS, Calif.--(BUSINESS WIRE)--Aug. 8, 2005--The ALS Association (ALSA) today announced funding for a collaborative project that will screen thousands of existing compounds for possible activity as ALS therapeutics, using tiny worms living in lab dishes that recreate aspects of amyotrophic lateral sclerosis (ALS, also called Lou Gehrig's disease).
The ALSA-initiated grant gives continued support to the efforts of Richard Morimoto, Ph.D., who has created a worm that expresses the mutant gene for copper-zinc superoxide dismutase (SOD1), responsible for some inherited cases of ALS. Researchers suspect that mutant SOD1 is not folded properly and disrupts the ability of motor neurons to function.
"We are extremely pleased to announce this exciting collaboration between academia and biotech and believe this is the way forward to finding new therapies for ALS," commented Lucie Bruijn, Ph.D., science director and vice president of ALSA.
Morimoto, who is at Northwestern University in Evanston, Ill., will collaborate with Cambria Biosciences in Woburn, Mass. to carry out high throughput screening of several libraries of compounds. The collaboration will seek compounds that change the ability of a cell to either correct poorly constructed protein or consign it to the cellular trash heap.
"The collaboration with Cambria Biosciences will allow us to more rapidly identify new lead compounds that interfere with the appearance of misfolded proteins and aggregates and the associated cellular toxicity that ultimately paralyzes the animal," said Morimoto. "We are very excited that this project brings together the technical expertise of Cambria in high throughput screening with our knowledge on the new animal model system that we have established."
Leo Liu, M.D., president of Cambria Biosciences, added that, "Dr. Morimoto's work has laid the foundation for a new approach to ALS therapy discovery. We are very pleased to be able to pursue this translational research project together with ALSA and Dr. Morimoto's laboratory."
The collaborative screening effort, using the C. elegans worm model of ALS, is expected to find fresh insight into the basic process of the disease, revealing new targets at which to aim drug discovery efforts.
In close conjunction with ALSA's new initiative, Translational Research Advancing Therapy for ALS (TREAT ALS), the Morimoto and Cambria collaboration will review available compound libraries and strategize on any promising leads.
The millimeter long worm, C. elegans, is commonly used in the lab to manipulate genes and study the result in a living organism. Morimoto, an expert in Huntington's disease and on the cellular response to damaged proteins, introduced the gene for SOD1 into the worm, and was able to detect both a change in the way the protein looks in the transparent worm under the microscope, and a change in the worm's ability to move about, once the mutated SOD1 gene is expressed.
With the worm, and Cambria's expertise in high throughput screening, investigators will pan for compounds in existing collections that might have unrecognized potential as therapeutics for ALS.
ALSA is the only national not-for-profit voluntary health organization dedicated solely to the fight against ALS.
- Contacts The ALS Association Jeff Snyder, 818-587-2220 jeff@alsa-national.org
- At A Glance The ALS Association Headquarters: Calabasas Hills, CA Website: http://www.alsa.org
- CEO: Gary Leo
- Employees: 40
- Organization: Non-profit
- Source: via Business Wire Updated 02/03/2005 by company © Business Wire 2005