CMT2 accounts for roughly one-third of CMT cases and CMT2A is its most common subtype. Inherited mutations in the MFN2 gene lead to degeneration of muscle-controlling nerve cells. Symptoms typically appear before age 10.
The announcement marks the end of the second phase of a three-phase collaboration between AcuraStem and the CMT Research Foundation. In the next phase, the molecules of interest will undergo more detailed analyses to determine which make the most promising candidates for further research.
Many of the identified compounds already are approved by the U.S. Food and Drug Administration and other international regulatory agencies as treatments for other disorders.
Finding new uses for established medicines is a strategy called drug “repurposing” or “repositioning.” Pharmaceutical companies often see this as an attractive drug discovery strategy, as working with drugs that are already proven safe and well-tolerated limits risk while also saving time and money.
Should any of the newly-identified molecules enter clinical trials for CMT2A, for instance, they may skip Phase 1 testing, as a safe and well-tolerated dose already has been established and approved.
The project completed its first phase last year. This phase consisted of creating a neuronal cell line from stem cells of a CMT2A patient and verifying that those cells have the characteristics of CMT2A. Researchers also showed that the survival of these cells improves when treated with neurotrophic factors — agents known to support nerve cell growth and survival.
During that phase, researchers discovered a small molecule called AS-1 that prevented motor neurons derived from amyotrophic lateral sclerosis (ALS) patients from dying. Because of similarities in how motor neurons degenerate in both conditions, AS-1 also may prove therapeutic in both CMT2A and ALS.
The 37 compounds found in the current drug screen came from a collection of thousands that AcuraStem tested in motor neurons derived from patients and validated in animal models.
Screening takes place on AcuraStem’s proprietary iNeuroRx platform, developed by Justin Ichida, a professor at the University of Southern California and a co-founder of the company. The platform seeks to identify potential treatments by applying an artificial intelligence approach to data collected from patient-derived motor neurons.
In the long term, the use of patient-derived cells might enhance the understanding of CMT neuropathy and contribute to more approved treatments.
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