ALSTDI CEO Urges More Preclinical Testing Before Human ALS Trials

Each new candidate ALS therapy that is tested in patients sparks overwhelming hope across the ALS community. However, often these drugs are tested in human trials without sufficient preclinical validation, leading to repeated failures in the clinic, states Steve Perrin, CEO of ALS Therapy Development Institute (ALSTDI) in Cambridge, Massachusetts. In a Comment in the March 26 issue of Nature, ALSTDI reports testing over one hundred compounds in ALS mouse models that had been previously identified as potential drugs for ALS. Strikingly, the researchers were unable to reproduce the reported efficacy of these candidate therapies, including several that had progressed to clinical testing and subsequently failed. Perrin reiterated the need to sufficiently characterize aspects of the mouse model that correspond to human ALS and to use mathematical simulations to help guide the statistical analyses (for guidelines on using the ALS mice see Working with ALS Mice). Perrin also underscored the need for public and private funding specifically for studies to better characterize the animal models.

Each new candidate ALS therapy that is tested in patients sparks overwhelming hope across the ALS community. However, often these drugs are tested in human trials without sufficient preclinical validation, leading to repeated failures in the clinic, states Steve Perrin, CEO of ALS Therapy Development Institute (ALSTDI) in Cambridge, Massachusetts. In a Comment in the March 26 issue of Nature, ALSTDI reports testing over one hundred compounds in ALS mouse models that had been previously identified as potential drugs for ALS. Strikingly, the researchers were unable to reproduce the reported efficacy of these candidate therapies, including several that had progressed to clinical testing and subsequently failed. Perrin reiterated the need to sufficiently characterize aspects of the mouse model that correspond to human ALS and to use mathematical simulations to help guide the statistical analyses (for guidelines on using the ALS mice see Working with ALS Mice). Perrin also underscored the need for public and private funding specifically for studies to better characterize the animal models.

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