Muscular Dystrophy Association Gives AcuraStem $300K Boost to Develop ALS Therapy

A $300,000 grant from the Muscular Dystrophy Association (MDA) will back AcuraStem’s preclinical development of an amyotrophic lateral sclerosis (ALS) patient-derived stem cell therapy that has the potential to treat a wide range of ALS patients.

The grant will allow the biotechnology company AcuraStem to begin proof-of-concept studies of its AS-1 program in ALS mouse models, and to develop biomarkers for the orally delivered, blood-brain-penetrating therapeutic candidate.

MDA is awarding the two-year MDA Venture Philanthropy (MVP) grant to a team led by Justin Ichida, PhD, co-founder and CEO of AcuraStem. He and his collaborators engineered an innovative precision medicine platform called iNeuroRx to enhance the ability to pinpoint treatments that potentially could slow ALS progression.

The drug discovery platform for neurodegenerative diseases combines artificial intelligence with nerve cell data from patients’ stem cells. Using this cutting-edge approach, the scientists identified their first preclinical therapy candidate, AS2015, also known as AS-1. The hope is to advance the candidate into human clinical trials for ALS treatment.

“We are encouraged by the progress AcuraStem and its founders have made in discovering a novel ALS drug target using patient-derived stem cells,” Amanda Haidet-Phillips, MDA scientific portfolio director, said in a press release. “MDA’s investment in biotech-driven research through its venture philanthropy program ensures that promising therapies can move swiftly from conceptualization in the laboratory to testing in people with ALS.”

Ichida’s team, whose research was published in the journal Nature Medicine last year, discovered that stopping the action of a specific protein in ALS patient-derived motor neurons, helped prevent their death in vitro.

Although the target was discovered in cells from a patient with C9-ALS — a familial form of the disease — it may also be used in other forms of ALS, including the more common sporadic ones.

In ALS patients, motor neurons degenerate or die, ultimately resulting in an inability to control movement. While most ALS cases are not inherited, a mutation in the C90RF72 gene, where one segment of the gene is repeated too often, is the most common cause of C9-ALS. The mutation can also cause frontotemporal lobar degeneration and other neurological disorders.

So that they can evaluate their therapeutic candidates under development, commercial and academic AcuraStem partners have access to this technology as well.

“MDA’s support of our work is essential to further advance our AS-1 small molecule program into the clinic,” said Paul August, AcuraStem’s co-founder and board chairman. “With this funding, we’ll continue to move forward our novel approach aimed at transforming outcomes for ALS patients by leveraging their own motor neurons to evaluate AcuraStem’s innovative drug program aimed at enhancing neuron survival and health.”

MVP grants are awarded to scientists developing treatments for neuromuscular diseases to help smooth the path during high-risk stages.

In June, AcuraStem was awarded $3.7 million by the National Institute of Neurological Disorders and Stroke to support development of AS-1 to treat C9orf72 gene-related ALS and frontotemporal dementia.