Focus will be on expansion of AcuraStem’s patient-based discovery platform, iNeuroRx®, to leverage patient neurons and advanced informatics technology to identify promising drugs
ATLANTA, December 20, 2018 — The newly launched CMT Research Foundation (CMTRF), a 501(c)(3) nonprofit focused solely on delivering treatments and cures for Charcot-Marie-Tooth disease, today announced it has entered into a collaborative research partnership 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 severe, 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.
“AcuraStem’s best-in-class technology platform combines patient-derived neurons and advanced informatics to identify potential cures for neurodegenerative diseases,” says Susan Ruediger, CMTRF's co-founder and CEO. “AcuraStem will take patient-derived stem cells for CMT2A, transform them into neurons, then screen approved drugs to look for treatments and cures that are active in the patient neurons. This will greatly accelerate the process of testing and ruling in, or out, potential CMT2A treatments.”
AcuraStem’s success in using its precision medicine iNeuroRx® platform to identify therapeutic targets and promising drugs for the treatment of neurological diseases, including amyotrophic lateral sclerosis (ALS), suggests that a similar strategy potentially may be employed to identify therapeutics that could rapidly be developed or repositioned for individuals with CMT. In collaboration with the CMTRF, AcuraStem will work now to apply its iNeuroRx® platform’s application to CMT2A which, like ALS, causes the degeneration of muscle-controlling nerve cells called motor neurons.
“We believe that our iNeuroRX® platform could be the key in finding a transformative drug target for this underserved population of individuals with CMT,” said AcuraStem Co-founder and Chief Executive Officer Sam Alworth. “We have made great strides in modeling ALS, and now we are excited to build on our early successes through our collaboration with CMTRF. It’s a logical next step to extend our innovative, patient-focused platform to address CMT, another devastating, rare degenerative motor neuron disease.”
The partnership will be milestone driven and rolled out in phases, enabling CMTRF to stop funding any research that is deemed ineffective. Conversely, it also assures that if the studies produce a viable treatment that comes to market, CMTRF will benefit, enabling it to fund more promising research.
“As a partner and not just a source of funds, CMTRF has adapted elements of the venture capital model that assure when the programs we support come to market and generate a profit, CMTRF shares in that success and commits to reinvesting proceeds into additional translational research,” says Pat Livney, CMTRF's co-founder and chairman.
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.
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.