Methods and Compositions Comprising a Drosophila Model of Amyotrophic Lateral SclerosisTechnology #ua15-037
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- Patricia Estes Assistant Staff Scientist, Molecular & Cellular Biology
- Daniela Zarnescu Professor, Molecular & Cellular Biology
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- Lisa Lin Licensing Manager (520) 626-6969
Background: Amyotrophic Lateral Sclerosis (ALS) is a devastating neurological disorder characterized by the degeneration of motor neurons in the motor cortex of the brain and the spinal cord. ALS leads to paralysis and respiratory failure followed by death, usually within two to five years of diagnosis. The only FDA drug currently approved to treat ALS is Riluzole (Rilutek®), but it has only marginal effects on survival and no effect on patient quality of life. Development and discovery of new therapeutic treatments for ALS is thus desperately needed. Global sales of Rilutek® alone were estimated at $224m in 2010. However, Rilutek® has relatively low market penetration due to its relatively low efficacy. It is expected that sales of more efficacious treatments for ALS would be significantly higher. Existing drug discovery models for ALS have focused primarily on rodent models of the disease.
Invention: The present invention is a drosophila model of ALS and an accompanying drug screening method that is capable of efficiently screening candidate therapeutics for their potential efficacy in the treatment of ALS. It has already been used to identify at least one candidate therapeutic that shows promise of greater efficacy in the treatment of ALS-like symptoms than Riluzole.
Applications: This model may be used to find potential therapeutics for ALS.
Advantages: Transgenic drosophila provide a model of ALS that is less expensive and more convenient than rodent models. Putative therapeutics can be tested in vivo, and in a more rapid manner than in competing rodent models.
Lead Inventors: Daniella Zarnescu, Patricia Estes