PathMaker launches first human trial to test noninvasive ALS device
Phase 1 trial of MyoRegulator funded by grants from MDA, CERF and NIH
PathMaker Neurosystems has launched a Phase 1 clinical trial that will test its experimental noninvasive nerve-modulating device in people with amyotrophic lateral sclerosis (ALS).
This is the first time that the device — called the MyoRegulator and designed to slow disease progression in ALS — will be formally tested in humans. The newly launched trial is being carried out in conjunction with Spaulding Rehabilitation Hospital in Charlestown, Massachusetts.
“We are tremendously excited to launch this first-in-human trial of MyoRegulator in ALS,” Nader Yaghoubi, MD, PhD, co-founder and CEO of PathMaker, said in a company press release.
“Initiating clinical evaluation of our technology in ALS is not only a significant milestone for our company, but a step towards meeting the medical needs of people living with this disease,” Yaghoubi added.
In a webpage about the trial, researchers note that “there are currently a limited number of treatments available for ALS, most of which are pharmaceutical in nature. This study could open a path for a non-invasive, non-pharmaceutical option.”
Device from PathMaker seeks to slow disease progression
ALS is caused by the progressive dysfunction and death of motor neurons, which are the specialized nerve cells that control movement. The loss of motor neurons disrupts the brain’s ability to send signals to the body to trigger voluntary movements, ultimately leading to symptoms like weakness and paralysis.
Although it’s not known exactly what drives motor neuron dysfunction in ALS, one process that’s thought to play a role is motor neuron hyperexcitability — the tendency for nerve cells to fire, or send electrical signals, more frequently than they should. Hyperexcitability and abnormal nerve firing can put strain on motor neurons, driving damage in the long run.
The PathMaker MyoRegulator device aims to reduce hyperexcitability in ALS motor neurons, with the ultimate goal of helping to preserve motor neuron health to slow disease progression.
The device uses disposable electrodes that are applied to the skin to deliver a gentle electrical current to specific parts of the spinal cord, with the aim of reducing hyperexcitability in spinal motor neurons.
“Our non-invasive approach seeks to suppress spinal motor neuron hyperexcitability, which is a key characteristic of the ALS disease process that has been found in both sporadic and familial variants of ALS,” Yaghoubi said.
The Phase 1 trial is a single-arm and open-label study, meaning that all participants will get the same treatment and all will know what treatment they’re getting, with no placebo or inactive treatment option. It will enroll adult ALS patients at Spaulding who will be required to make 16 visits to the hospital over a 10-week period.
ÂOur non-invasive approach seeks to suppress spinal motor neuron hyperexcitability, which is a key characteristic of the ALS disease process that has been found in both sporadic and familial variants of ALS.
The study’s main goal is to assess the safety and feasibility of using the MyoRegulator device in people with ALS. Researchers also aim to collect preliminary data on the efficacy of the device in ALS.
PathMaker is collaborating in the trial with leading experts in the field, including Leon Morales-Quezada, MD, PhD, director of the Integrative Rehabilitation Lab at Spaulding, and Sabrina Paganoni, MD, PhD, professor of physical medicine and rehabilitation at Spaulding and a physician-scientist at Massachusetts General Hospital.
The trial is being financially supported by the Muscular Dystrophy Association (MDA), with grants also awarded by the Cullen Education and Research Fund (CERF) and the National Institutes of Health (NIH).
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