CMTRF/AcuraStem Partnership Yields First Significant Milestone in Search for CMT Cure

AcuraStem compound shows positive CMT2A motor neuron survivability

ATLANTA, September 18, 2019 — One of the compounds in a series under development by AcuraStem for ALS, was administered to a neuronal cell line derived from a patient with CMT2A, and showed improved cell survival resulting in the identification as a promising new, preclinical drug candidate for CMT2A.

This is the result of a December 2018 investment made in a research partnership by The CMT Research Foundation (CMTRF), a 501(c)(3) nonprofit focused solely on delivering treatments and cures for Charcot-Marie-Tooth disease, with AcuraStem to test thousands of compounds aimed at producing effective treatments for CMT2A.

Nearly 3 million individuals of every age, race and ethnicity have a form of CMT, a progressive genetic disease of which there are many variants. About 5 percent of patients have CMT2A which can be severe, causing significant disabilities in younger children, including chronic pain. Because CMT is progressive, patients continue to lose function as they age, creating a greater degree of disability. Currently, there is no cure or any effective treatments for CMT.

“To be clear, this is the first step on a very long and difficult search for a cure for CMT2A, but it is a very positive first result,” says Susan Ruediger, CMTRF's co-founder and CEO. “In fact, AcuraStem has met three other important milestones including creating a neuronal cell line derived from a patient with CMT2A; testing the neurons for characteristics of CMT2A, including survival deficit and; showing that the cell line demonstrates improved survival when exposed to neurotrophic factors, an agent known to support nerve cells.”

The next step in the CMTRF/AcuraStem collaboration is to screen all U.S. Food and Drug Administration- and European Medicines Agency-approved drugs to ascertain if any of these existing therapeutics generate more promising results than the first candidate identified, known as AS-1. The project’s use of AcuraStem’s iNeuroRx® platform will accelerate the pace of drug screening and find drug candidates that likely have a higher probability of success in the clinic because they were discovered using patients’ own cells.

“Using FDA/EMA-approved drugs, which have already undergone rigorous clinical trial testing and have a documented positive safety and tolerability profile, we could potentially shorten the time to regulatory approval of one of these drugs as an available treatment option for CMT. Although formulation or dosing of any of these drugs may need to be modified for CMT, the first hurdle to approval — safety — has already been crossed,” adds Ms. Ruediger. “These drugs potentially could be fast-tracked to the clinic, either alone or in combination.”

About AcuraStem

AcuraStem Incorporated is a fast-growing biotechnology company striving to find cures for a number of neurodegenerative diseases via a new disruptive technological advancement called patient-based drug discovery. Formed in 2016, AcuraStem has an exceptional team of Ph.D.s, professors, tech entrepreneurs, and successful drug company veterans employing the latest scientific breakthroughs to first address one of the most challenging, but tractable diseases, ALS. AcuraStem’s first therapeutic candidate will enter clinical trials in 2020.

About CMTRF

The CMT Research Foundation (CMTRF) is focused solely on delivering treatments and cures for CMT. Founded by two patients who are driven to expedite drug delivery to people who live with CMT globally, the organization funds research and drug development. The 501(c)(3) federal tax-exempt organization is supported by personal and corporate financial gifts.

For more information, please contact kblack@acurastem.com .