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 Big drug companies have failed to develop therapeutic strategies for ALS, which is perceived as an unprofitable market. In 1998, Project A.L.S. took drug discovery into its own hands, uniting researchers and clinicians in a coordinated effort to identify potentially effective compounds and FDA-approved drugs. In 1999, the Project A.L.S. drug discovery team devised the first ALS micro-assay or tiny test-against which researchers have now screened thousands of drug candidates. A high throughput screening, utilizing this and other micro-assays sponsored by the National Institutes of Health, yielded several "hits." Some of these compounds are being tried in humans.  Saving TimeCurrently, the most reliable laboratory model for ALS drug discovery is a transgenic mouse that develops the disease. The SOD1 mouse, which features the genetic mutation associated with a small number of ALS cases, is a hot commodity. Researchers were forced to wait up to 18 months to receive these mice. The backorder problem severely slowed ALS research. Established in 2003, and housed at Jackson Laboratory in Bar Harbor, Maine, the Project A.L.S. SOD1 Colony has made up for lost time, making this valuable transgenic available immediately, at no charge, to scientists all over the world. To date, the Colony has shipped 7000 SOD1s and now offers the new B6 strain. For more information, please contact Harriet Abramson, SOD1 Colony Director at info@projectals.org. Special Delivery to the Brain The human brain is protected from harmful influences by the naturally occurring Brain Blood Barrier (BBB). Unfortunately, the BBB also serves as a blockade, preventing helpful medicines from reaching the degenerating brain. A team led by Fred H. Gage and Brian K. Kaspar discovered that a viral particle could be gutted of its contents and transfected with a growth factor called IGF1. These viral particles proved an effective delivery system, overcoming the BBB and delivering IGF1, a potent growth factor, to spinal motor neurons. CLICK HERE TO VIEW GENE THERAPY VIDEO.
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