The ALS Therapy Development Institute (ALS TDI), LifeArc, and Axol Bioscience launched the Patient induced pluripotent stem cell (iPSC)-based Research to Improve Sporadic ALS Modeling (PRISM) initiative, a collaborative effort to expand access to patient-derived stem cell models.

ALS is a heterogeneous disease. While 10-15% of cases are linked to inherited mutations, nearly 85% are sporadic, according to a PRISM ALS official, who adds that much of ALS drug discovery has relied on models representing a limited number of rare genetic subtypes. This mismatch has constrained target discovery, limited therapeutic testing across patient populations, and contributed to the high failure rate of clinical trials, maintains the spokesperson.

This initiative plans to provide a high-quality and accessible source of sporadic ALS/MND models for use in research. PRISM ALS aims to develop, evaluate, and make available a diverse panel of well-characterized, patient-derived induced pluripotent stem cell (iPSC) models that capture both genetic and sporadic forms of ALS.

For researchers and drug developers, those standardized, human-relevant models are expected to allow them to better understand disease mechanisms, identify therapeutic targets, and evaluate treatments across distinct biological subtypes. For people living with ALS, it might lead to the development and testing of therapies in models that more closely mirror their own biology, increasing the likelihood that discoveries will translate into meaningful treatments.

The stem cells used in PRISM ALS are derived from samples contributed by people living with ALS through ALS TDI’s ALS Research Collaborative (ARC) Study, the longest-running longitudinal patient study in ALS.

“We know that ending ALS will require delivering the right treatments to the right individuals,” says Fernando Vieira, MD, CEO, and CSO of ALS TDI. “By characterizing iPSC-derived motor neurons from sporadic ALS and making these cells broadly accessible, PRISM ALS will facilitate global drug discovery. This program is only possible thanks to the people living with ALS who contributed samples and data through the ARC Study.”

“We’re delighted to participate in this consortium to develop multiple iPSC-derived end point cell types from sporadic ALS iPSC lines that reflect for the first time, real-world variability across age, sex, and genotype,” notes Sapna Vyas, head of scientific programs at Axol Bioscience. “By leveraging Axol’s scalable manufacturing infrastructure, we will facilitate access to standardized iPSC-derived cells that empower researchers to stratify patients, assess subgroup responses to therapies, and reduce late-stage clinical trial failures.”

“Our hope is that the stem cell models we produce can unleash a new generation of treatments that could be effective against this disease by slowing its progression and, ultimately, curing it,” adds Paul Wright, head of motor neuron disease (MND) at LifeArc. “We need to do more for people living with MND/ALS, and PRISM ALS brings together leading organizations to help make that happen.”

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