Could spinal cord stimulation treat SMA in the future?

Last updated June 27, 2025, by Agata Boxe

Researchers have been testing spinal cord stimulation, a new approach to treating spinal muscular atrophy (SMA), which involves electrically stimulating the sensory spinal nerves through an implanted device.

Earlier this year, they published findings from a pilot study involving three ambulatory patients with SMA type 3. The researchers observed improvements in lower limb function, including strength and walking ability, in all three participants.

This early trial focused primarily on safety and lasted four weeks. No side effects were reported. The team now hopes to conduct longer-term studies, spanning one to two years, to further evaluate the benefits.

More recently, the same technique was applied to three nonambulatory individuals — two with SMA type 2 and one with SMA type 3 — this time targeting arm and hand function. However, results from that group have yet to be published.

SMA News Today spoke with study co-author Marco Capogrosso, PhD, an assistant professor of neurosurgery at the University of Pittsburgh, in Pennsylvania, about this developing research and the future potential of spinal cord stimulation for SMA and beyond.

The interview was excerpted and edited for clarity.

AB: What is spinal cord stimulation, and what does it involve?

MC: Spinal cord stimulation is a technology first developed in the 1970s, and has grown in popularity since then. Today, it’s a well-established, minimally invasive procedure that is not entirely different from what is done to, for example, deliver epidural anesthesia. […]

It involves implanting electrodes into the epidural space, which is the space between the bone and the spinal cord. So, the electrodes don’t penetrate the spinal cord or the neural tissue; they just sit on top of it.

Originally, this technology was designed to relieve pain. Researchers discovered that electrically stimulating the spinal axons responsible for the sensation of touch could reduce pain, a bit like how rubbing a sore spot or wound can sometimes make it feel better.

Today, spinal cord stimulation is used as a treatment for refractory pain syndrome, the syndrome of pain that does not go away with other treatments, such as opioids.

These are all clinically approved devices, and it’s actually the most common implant procedure in the nervous system, with over 50,000 people in the United States receiving it each year.

So, this technology exists for this reason, and we just leveraged it to do what we wanted to do in SMA.

Is this procedure painful?

Whether or not the procedure is painful is subjective. There is an incision that is made on the skin, and there are sutures afterward.

The stimulation, per se, is not painful, nor is the fact that there is an electrode sitting near the bone. However, every surgical procedure comes with discomfort and pain associated with that.

Compared to other procedures that people with SMA may undergo, like spinal fixation procedures, this one is far less invasive.

Do you think that spinal cord stimulation will one day become widely accessible to people with SMA? If so, when do you think it may happen?

I believe that spinal cord stimulation will one day become widely accessible for people with SMA, and we are working toward that goal.

There are many challenges along the way, not just scientific ones. As long as this technology continues to perform well in our studies, we shouldn’t have trouble obtaining the necessary FDA [U.S. Food and Drug Administration] approval.

However, because these devices already exist and their prices are set, companies that sell them have to be willing to invest in securing approval for use in rare diseases, which poses a significant challenge.

What’s your next step with this research?

Our next step, strictly speaking, is to do the next clinical trial. But we’re working with the SMA Foundation and a lot of other people, including the device manufacturer, to ensure the trial includes outcomes that are meaningful for further development.

We are not interested in just running a trial and showing that it works. We are trying to build a pathway to make this treatment available as soon as possible — in less than 10 years, but I’m hoping in five. Ultimately, however, that depends on the results of the study.

We definitely want a short-term turnaround for this therapy because it already exists. It’s already proven to be biologically safe. It shouldn’t take 20 years to bring this to the market. It should be much faster. And I know that the regulators are sensitive to rare diseases and the specific challenges that they have. So, I’m really hopeful that we can do this in a reasonable time.

Why do you think the regulators are sensitive to rare diseases in particular?

Because there’s a fundamental business challenge: if a device costs a significant amount to develop and only a few hundred people have the disease, no one wants to invest the money because there is no market for it.

They’re trying to find workarounds to basically convince companies that it is worth it. Hopefully, the more we start treating these rare diseases, the more the system will find a solution.

Is there anything else you would like to add?

For people with SMA, this could be very interesting because this isn’t a drug; it’s something that can be done on and off, and it seems to be tapping into the most important deficits, which are strength and fatigue. If we can confirm that, I think that would be really important to finally see some sizable benefits for these people.

On a bigger scale, SMA is an example of the fact that you can use neuro-technologies to treat neurodegenerative diseases. What this study has demonstrated is that when you have a neurodegenerative disease like ALS [amyotrophic lateral sclerosis] or SMA, what happens is that you always think that you get a deficit because neurons die, and that’s certainly the case.

However, there is another component of the disease: neural circuit dysfunction. If we can stop the death of cells with a drug, we can then correct the circuit dysfunction with this technology and combine it with existing therapeutics to really make an impact in this disease.

Our hope is to apply this same strategy to other neuromuscular diseases in the future, like ALS, as more effective drugs become available. For now, we’ll keep working on SMA and keep building from there.

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