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Oct 03, 2013
 |  BC Innovations  |  Targets & Mechanisms

Alternative strategies in ALS

Biogen Idec Inc. and Evotec AG are taking opposite strategies to finding new therapies for amyotrophic lateral sclerosis, a disease with an unknown mechanism. Whereas Biogen Idec is forming partnerships with multiple academic labs to solve the biology of the disease, Evotec is partnering with the Harvard Stem Cell Institute at Harvard Universityto employ phenotypic screens that circumvent the need to understand the mechanism from the outset.

Amyotrophic lateral sclerosis (ALS) is a severe neurodegenerative disease in which progressive loss of motor neurons leads to muscle weakness, atrophy and respiratory failure. Despite recent advances in understanding the genetics of the disease,1 the biological trigger of the nerve cell loss remains unclear and therapeutic targets remain elusive.

About 10% of patients with ALS show familial inheritance, whereas the remaining 90% of cases are sporadic and have no clear genetic linkage. Both forms of the disease are phenotypically similar, suggesting that pathways identified by genetic mutations might also be disrupted in sporadic ALS.

Going phenotypic

In September, Evotec partnered with the Harvard Stem Cell Institute (HSCI) to combine the academic lab's expertise in creating induced pluripotent stem (iPS) cells from patients with ALS and the biotech's proprietary screening assays for the disease.

The deal is with Lee Rubin and Kevin Eggan, both faculty members at HSCI who have developed a technique for creating motor neurons from human fibroblasts. The duo also has generated cell lines from patients with ALS representing a variety of genetic backgrounds.

Evotec CSO Cord Dohrmann would not elaborate on the details of the endpoints of the company's ALS assays but told SciBX that the goal of the screens is to...

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