A new global initiative launched today aims to fill a critical gap in ALS/MND drug discovery, impacting both the researchers developing treatments and the people urgently awaiting them. Current cell models used to test treatments do not reflect the diverse nature of the disease.
ALS Therapeutic Development Institute (ALS TDI), LifeArc, and Axol Biosciences announced the launch of the Patient-Induced Pluripotent Stem Cell (iPSC)-Based Sporadic ALS Modeling Improvement Study (PRISM), a joint effort to expand access to high-quality patient-derived stem cell models that better reflect the biological complexity of amyotrophic lateral sclerosis (ALS).
ALS is a heterogeneous disease. 10-15% of cases are associated with inherited mutations, but almost 85% are sporadic. However, much of ALS drug discovery has relied on models representing a limited number of rare genetic subtypes. This discrepancy limits target discovery, limits testing of treatments across patient populations, and contributes to high failure rates in clinical trials.
This unprecedented effort provides a reliable, high-quality, and easily 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, these standardized, human-relevant models allow them to better understand disease mechanisms, identify therapeutic targets, and evaluate treatments across different biological subtypes. For people living with ALS, this means that treatments can be developed and tested in a model that more closely reflects their own biology, increasing the likelihood that discoveries will lead to meaningful treatments.
The stem cells used in PRISM ALS are derived from samples donated by people living with ALS through the ALS TDI’s ALS Research Collaborative (ARC) Study, a long-term longitudinal study of ALS patients.
For over a decade, ALS TDI has built one of the most comprehensive collections of ALS-specific iPSCs currently available. These cells come directly from people living with ALS, many of which also contribute detailed clinical data, creating an unparalleled resource for understanding how the disease behaves and responds to treatment.
This research was only possible thanks to the more than 1,800 ALS patients who chose to participate in the ARC study and provide their samples and data to advance the research.
We know that eradicating ALS requires providing the right treatment to the right individuals. PRISM ALS characterizes iPSC-derived motor neurons from sporadic ALS and makes these cells widely available to facilitate global drug discovery. This program is only possible thanks to people living with ALS who have provided samples and data through the ARC study. ”
Dr. Fernando Vieira, ALS TDI CEO and Chief Scientific Officer
By enabling standardized production at scale, collaboration ensures quality, consistency, and reproducibility across the laboratory. The goal is to accelerate progress across the ALS field by providing robust, human-relevant tools that better reflect the biological diversity and complexity of the disease.
Sapna Vyas, Head of Scientific Programs at Axol Biosciences, said: “We are pleased to join this consortium to develop multiple iPS cell-derived endpoint cell types from sporadic ALS iPS cell lines that, for the first time, reflect real-world variation across age, gender, and genotype. By leveraging Axol’s scalable manufacturing infrastructure, we will facilitate access to standardized iPS cell-derived cells that will allow researchers to stratify patients, assess subgroup responses to therapy, and reduce late-stage clinical trial failure.” ”
Paul Wright, Head of MND at LifeArc, added: “Our hope is that the stem cell models we create can unlock a new generation of potentially effective treatments for this disease by slowing its progression and ultimately curing it. We need to do more for people living with MND/ALS, and PRISM ALS brings together leading organizations to make that happen.”
Together, the partners aim to make these models a widely accessible, high-quality resource for researchers in academia and industry working to advance ALS treatments.
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ALS Treatment Development Research Institute
