Global initiative to tackle gaps in ALS/MND drug discovery 

A global initiative has launched to close the gap in ALS/MND drug discovery. 

The ALS Therapy Development Institute (ALS TDI), LifeArc, and Axol Bioscience announced the launch of ‘Patient induced pluripotent stem cell (iPSC)-based Research to Improve Sporadic ALS Modelling’ (PRISM ALS), a collaborative effort to expand access to high-quality, patient-derived stem cell models that better reflect the biological complexity of amyotrophic lateral sclerosis (ALS). 

The initiative aims to develop, evaluate, and make available a diverse panel of well-characterised, patient-derived induced pluripotent stem cell (iPSC) models that capture both genetic and sporadic forms of ALS. 

For researchers and drug developers, those standardised, human-relevant models could allow them to better understand disease mechanisms, identify therapeutic targets, and evaluate treatments across distinct biological subtypes.  

For people living with ALS, the effort could mean therapies are developed and tested in models that more closely mirror their own biology, increasing the likelihood that discoveries will translate into meaningful treatments. 

“We know that ending ALS will require delivering the right treatments to the right individuals,” said Dr Fernando Vieira, CEO and Chief Scientific Officer at ALS TDI.  

“By characterising iPSC-derived motor neurons from sporadic ALS and making these cells broadly accessible, PRISM ALS will facilitate global drug discovery. This programme is only possible thanks to the people living with ALS who contributed samples and data through the ARC Study.” 

ALS is a heterogeneous disease and, while 10-15% of cases are linked to inherited mutations, nearly 85% are sporadic. Much of ALS drug discovery has relied on models representing a limited number of rare genetic subtypes, constraining target discovery, limiting therapeutic testing across patient populations, and contributing to the high failure rate of clinical trials. 

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