Device for slowing ALS progression wins FDA breakthrough status
MyoRegulator delivers gentle electrical current to spinal cord, nerves
- MyoRegulator, a neuromodulation device, has been granted FDA breakthrough status for the treatment of ALS.
- The noninvasive device delivers gentle electrical currents to nerves and the spinal cord to slow ALS progression.
- It's now in follow-up testing in the U.S. in a small clinical trial.
An experimental, noninvasive device for slowing the progression of functional symptoms in people with amyotrophic lateral sclerosis (ALS) has been awarded breakthrough device designation by the U.S. Food and Drug Administration (FDA).
Called MyoRegulator, the neuromodulation device from Pathmaker Neurosystems aims to reduce excessive nerve cell activation, which is thought to contribute to neurodegeneration in ALS. The device, designed for at-home use, works by providing a gentle electrical current to specific areas of nerves and the spinal cord.
This FDA designation is intended to expedite the development of new medical devices that may offer effective treatments for life-threatening or debilitating diseases, such as ALS. It provides manufacturers with prioritized review and more opportunities to interact with the regulatory agency during the review process.
“The breakthrough device designation represents a significant de-risking and accelerates our path to market for this treatment,” Nader Yaghoubi, MD, PhD, Pathmaker’s cofounder and CEO, said in a company press release announcing the new status.
“This designation further validates our pioneering approach to ALS, as well as the tremendous unmet medical needs of people and families living with ALS,” Yaghoubi said.
ALS is caused by the progressive dysfunction and loss of motor neurons, the specialized nerve cells that control voluntary movements. This leads to symptoms such as muscle weakness, and difficulties with moving, swallowing, and breathing.
The exact mechanisms that drive neuronal loss in ALS are still not fully understood. However, motor neuron hyperexcitability, where nerve cells fire electrical signals more frequently than they should, and the accumulation of toxic protein clumps inside motor neurons are thought to play a role.
Electrical signs delivered via electrodes placed on skin
MyoRegulator delivers a gentle electrical current to specific areas of the spinal cord and peripheral nerves — those outside the brain and spinal cord — to modulate the activity of spinal motor neurons. The electrical signals, delivered through electrodes placed on the skin of the spine and both arms, may also activate cellular mechanisms for protein breakdown.
By reducing hyperexcitability and boosting the degradation of faulty proteins in motor neurons, the device is expected to preserve motor neuron health and slow ALS progression. MyoRegulator, which earlier this year secured three new patents in the U.S. and Japan covering its use in people with ALS, is being developed for use at home.
In preclinical studies using a mouse model of ALS, the device was shown to slow disease progression and extend survival.
It also was tested in ALS patients in a first-in-human Phase 1 clinical trial (NCT06165172) at a single site in Massachusetts. That trial evaluated the device’s safety and tolerability, and assessed preliminary signs of effectiveness.
Five individuals were treated with the device three times a week for about six weeks, with one week of rest after the first week. The participants were followed for four weeks after the last treatment.
That study has been completed, and the company plans to present its results at upcoming clinical conferences and to publish them.
Small trial now testing neuromodulation device in US
Pathmaker has also launched a second U.S.-based trial, called CALM (NCT06649955), to further test the MyoRegulator. The trial, involving up to 15 adults with ALS, may still be recruiting patients at its single site, the Beth Israel Deaconess Medical Center, in Boston.
Participants will receive the treatment three times a week for four weeks, followed by a six-month follow-up. The trial’s main goal is to gather additional data on the device’s safety and feasibility. Secondary goals include assessing changes in patients’ disability, quality of life, muscle strength, lung function, and neuronal excitability.
The study is expected to end in November of next year.
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