AxoSim Creating Model Targeted for ALS Research

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Amyotrophic lateral sclerosis (ALS) is a fatal neurodegenerative disease that affects approximately 13 per 100,000 individuals. Although many potential mechanisms for ALS have been found using animal and in vitro cell models, the translation of these discoveries into valid treatment options has been limited. There is a clear need for improved preclinical drug testing that will translate to clinical success.

AxoSim received a grant from the DoD Congressionally Directed Medical Research Program (CDMRP) to support the development of an ALS NerveSim® model to mimic the pathology of human motor nerves of ALS patients. This model utilizes AxoSim’s revolutionary NerveSim® technology platform. Using patient-derived spinal motor neurons and spinal astrocytes, AxoSim is working to establish a 3D NerveSim® culture method that will model clinically relevant phenotypes in ALS and demonstrate the efficacy of drugs in reversing those phenotypes. ALS is a notoriously difficult condition to treat and more than 50 clinical trials have yielded only two therapies for the treatment of ALS, though neither is disease-modifying. With no translatable preclinical models available to drug developers, this project aims to fulfill a significant unmet need for a clinically-relevant model of ALS.

“ALS is such a devastating disease that it is difficult to believe how few treatments are available,” says Lise Harbom, PhD, AxoSim’s Post Doc leading this ground breaking study. “It is important to remember that our end goal is ultimately to help get potential new treatments to patients faster, without the possibility of putting patients through lengthy clinical trials of drugs that may not be effective in a human setting. Providing better, human-relevant models is our way of expediting that research process.”

AxoSim is dedicated to developing a human cell-based model that can serve as an intermediary between animal studies and clinical trials and is grateful for the grant award from the DoD, to pursue this crucial research to help improve the lives of those living with ALS.