A recent and somewhat controversial new Proceedings of the National Academy of Sciences study has many researchers wondering whether mice are an appropriate model for human disease. The study found that some mouse models of disease may not recapitulate the response observed in human disease. This discovery could have relevance for ALS research. Drug testing for potential ALS therapies often begins in the SOD1 G93A mice – the gold standard for preclinical ALS studies – and Prize4Life has been a longtime advocate for the value of well-designed pre-clinical studies using mice. While the new study provided unexpected results, it will likely not significantly affect current ALS research efforts in the short term. However as researchers learn more about the molecular underpinnings of ALS these findings provides some sobering “food for thought” with regard to the current system of drug development, especially in the complex and challenging neurodegenerative disease space.
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