Rare Disease Report

ALS and FTD Novel Small Molecule Therapy Receives Fast Track SBIR Grant

JUNE 18, 2018
Krista Rossi
Today, AcuraStem announced that its amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD) novel small molecule therapy, AS2015, has been granted a 3.7-million-dollar Small Business Innovation Research (SBIR) Fast-Track grant by the National Institute of Neurological Disorders and Stroke (NINDS) to continue research for the treatment’s development.  

“The team at AcuraStem is grateful for this grant as it will allow us to progress more rapidly toward a treatment, and set us on a path toward a cure for ALS,” stated AcuraStem president and cofounder Justin Ichida, PhD, in a recent statement. “The award is intended to facilitate the advancement of a new drug candidate for ALS leveraging patient neurons and engaging an entirely new target that focuses on the endosomal trafficking pathway.”

ALS is a rare nervous system disease characterized by weakening muscles and limiting physical function. FTD are a group of disorders characterized by damage to the brain’s frontal and temporal lobes, which cause forms of dementia. Both ALS and FTD are caused by expansion repeats in the gene C9ORF72.

“ALS affects people when they’re in the prime of their lives,” commented Qing Liu, PhD, AcuraStem CSO and co-Founder. “Traditionally, scientists have only been able to study a small population of ALS patients, relying heavily on animal models. This explains why there are no effective treatments for this disease, and why the available drugs only extend a patient’s life by a few months. We are disrupting this approach.”

The Fast Track SBIR grant assists in supporting AcuraStem’s integrated drug discovery approach, which seeks to leverage the induced motor neuron cellular models developed at AcuraStem Dr Ichida's lab at the University of Southern California (USC).

The NINDS SBIR program aims to assist researchers in commercializing early-stage scientific innovations. A beneficial feature of small-molecule drugs is that they treat neurodegenerative diseases, are easier to administer to patients, are often less expensive, and more easily penetrate the blood brain barrier.

The program is believed to be important in leveraging relevant human disease and toxicity models as early as possible during the preclinical drug discovery phases since small molecule drug approval is marred with hundreds to thousands of failed programs

iNeuroRx, AcuraStem’s innovative precision platform, uses nerve cells obtained directly from patient stem cells in conjunction with artificial intelligence to forecast drug efficacy. The technology is drastically changing patient outcomes.

The platform helps patients and clinicians assess disease progression and assess existing approved therapeutics to determine the most efficacious treatments to assist in slowing the progression of ALS. The platform also provides research scientists to utilize artificial intelligence analysis in combination with stem cell biology to accurately forecast personalized cures.

“AcuraStem’s objective is to advance a therapeutic portfolio to the clinic to treat ALS patients where limited therapies currently exist,” added Sam Alworth, AcuraStem’s CEO and co-Founder. “AS-1 is our lead program, and we are initiating multiple ALS therapeutic programs aimed at targeting the endosomal trafficking pathway in neurons.”


 

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