Spinal Cord Stimulation for Spinal Muscular Atrophy

Overseen By Sydney Bader, MS

Age: Any Age

Sex: Any

Trial Phase: Academic

Sponsor: Marco Capogrosso

Must be taking: SMN inducing therapies

Must not be taking: Anticoagulants, Anti-spasticity, Anti-seizure

Disqualifiers: Spinal deformation, Joint contractures, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Approved in 3 Jurisdictions This treatment is already approved in other countries

Compensation: Varies

Number of Visits: Unknown

Duration: 4 weeks

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial explores whether spinal cord stimulation (SCS), a technique typically used for pain relief, can improve movement in people with spinal muscular atrophy (SMA), specifically types 2, 3, or 4. Researchers aim to boost motor function by placing small leads near the spinal cord. The trial seeks participants with SMA who have noticeable upper body movement challenges, cannot stand independently, but can sit independently and use their hands for basic tasks like picking up a coin. As an unphased trial, this study offers a unique opportunity for participants to contribute to groundbreaking research that could enhance the quality of life for those with SMA.

Will I have to stop taking my current medications?

If you are taking anticoagulant, anti-spasticity, or anti-seizure medications, you will need to stop them at least 4 weeks before the lead implantation and cannot take them during the treatment phase of the study.

What prior data suggests that spinal cord stimulation is safe for spinal muscular atrophy?

Research has shown that spinal cord stimulation (SCS) has been tested in people before. In some studies, SCS improved muscle strength and movement in adults, suggesting its potential safety. Other studies found that non-invasive spinal cord stimulation is safe and can enhance arm and hand function in people with spinal cord injuries.

The SCS technology used in this trial has FDA approval for treating pain, indicating its safety for that purpose. This trial will use these approved devices to assess their impact on movement in people with spinal muscular atrophy (SMA). Overall, past research and FDA approval for other uses suggest that SCS is likely well-tolerated. However, this trial will provide more specific information about its safety and effectiveness for SMA. ^{1 2 3 4 5}

Why are researchers excited about this trial?

Spinal cord stimulation is unique because it offers a new way to manage spinal muscular atrophy (SMA) symptoms by directly targeting the nervous system. Unlike traditional treatments that often focus on genetic therapies or supportive care, this method uses electrical impulses to stimulate the spinal cord, potentially improving motor function and quality of life for patients. Researchers are excited about this treatment because it could provide an alternative for those who may not benefit from existing therapies, offering a non-invasive option with the potential for immediate and adjustable results.

What evidence suggests that spinal cord stimulation is effective for improving motor deficits in spinal muscular atrophy?

Research has shown that spinal cord stimulation (SCS), which participants in this trial will receive, can help people with spinal muscular atrophy (SMA) move better. One study found that SCS improved muscle strength and endurance, leading to better movement and control. Another study reported that adults experienced better strength, endurance, and walking ability after SCS treatment. These findings suggest that SCS could be a useful way to help with movement problems in SMA, even though it is mainly used for pain relief. ^{1 2 3 4 6}

Who Is on the Research Team?

Marco Capogrosso

Principal Investigator

University of Pittsburgh

Are You a Good Fit for This Trial?

This trial is for individuals aged 16 or older with Type 2, 3, or 4 Spinal Muscular Atrophy (SMA) who have motor deficits in the upper body. Participants will have a spinal cord stimulator implanted temporarily to test if it improves their motor functions.

Inclusion Criteria

I started my SMA treatment with Spinraza or risdiplam over 6 months ago.

I have been diagnosed with 5q-autosomal recessive SMA.

I have SMA and cannot stand or walk on my own.

Exclusion Criteria

I do not have severe behavioral or cognitive issues that prevent me from participating.

SMA Participant: Subject has severe behavioral or cognitive problems that preclude participation in the study

I have severe joint stiffness that limits my movement for study tasks.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive spinal cord stimulation with percutaneous spinal cord stimulation leads implanted in the cervical epidural space for up to 29 days

4 weeks

Regular visits for monitoring and adjustments

Follow-up

Participants are monitored for safety and effectiveness after treatment, including explantation of the leads

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Spinal Cord Stimulation

Trial Overview The study tests whether a Spinal Cord Stimulator can help improve upper limb motor function in people with SMA Types 2, 3, or 4. Up to six subjects will receive an implant near the cervical spinal cord for up to 29 days.

How Is the Trial Designed?

Group I: Spinal Cord Stimulation Experimental Treatment 1 Intervention

All patients will receive FDA-approved percutaneous spinal cord stimulation leads implanted in the cervical epidural (C4-T1 vertebra) space. The leads will be connected to external stimulators (either FDA-approved or human-grade research stimulator with safety features) during research activities.

Spinal Cord Stimulation is already approved in United States, European Union, Canada for the following indications:

Approved in United States as Spinal Cord Stimulation for:

• Failed back surgery syndrome (FBSS)
• Adhesive arachnoiditis
• Peripheral causalgia/neuropathy
• Reflex sympathetic dystrophy (RSD)
• Phantom limb/stump pain
• Ischemic pain of vascular origin
• Complex regional pain syndrome
• Pain after an amputation
• Visceral abdominal pain and perineal pain

Approved in European Union as Spinal Cord Stimulation for:

• Chronic pain
• Neuropathic pain
• Spinal cord injury pain
• Failed back surgery syndrome (FBSS)
• Adhesive arachnoiditis
• Peripheral causalgia/neuropathy
• Reflex sympathetic dystrophy (RSD)

Approved in Canada as Spinal Cord Stimulation for:

• Chronic pain
• Neuropathic pain
• Spinal cord injury pain
• Failed back surgery syndrome (FBSS)
• Adhesive arachnoiditis
• Peripheral causalgia/neuropathy
• Reflex sympathetic dystrophy (RSD)

Who Is Running the Clinical Trial?

Marco Capogrosso

Lead Sponsor

Trials

3

Recruited

40+

Roche-Genentech

Industry Sponsor

Trials

27

Recruited

3,800+

Published Research Related to This Trial

In a pilot study involving 7 individuals with chronic spinal cord injury, the use of transcutaneous electrical spinal cord stimulation (TSCS) combined with functional training led to improvements in upper and lower extremity function, with all participants showing progress on the Capabilities of Upper Extremity Test (CUE-T).

Notably, two participants improved their ASIA impairment scale classification, and five individuals experienced enhanced sensation, demonstrating that TSCS can facilitate recovery even after a plateau in rehabilitation progress, without any serious adverse events.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Transcutaneous Electrical Spinal Cord Stimulation to Promote Recovery in Chronic Spinal Cord Injury. Tefertiller, C., Rozwod, M., VandeGriend, E., et al.[2022]

Cervical transcutaneous spinal cord stimulation (tSCS) can effectively activate sensory fibers at lower stimulation intensities when the cathode electrode is positioned at the C7 or T1 vertebra, compared to C6, which may enhance rehabilitation outcomes for upper-limb motor recovery after spinal cord injury.

Using smaller electrode sizes not only lowers the activation threshold for sensory fibers but also optimizes the recruitment of these fibers before α-motor fibers, suggesting a strategic approach to improve hand muscle activation during tSCS therapy.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Optimizing sensory fiber activation during cervical transcutaneous spinal stimulation using different electrode configurations: A computational analysis. de Freitas, RM., Capogrosso, M., Nomura, T., et al.[2022]

In a study involving 15 healthy participants, transcutaneous electrical spinal cord stimulation (tSCS) significantly increased quadriceps motor evoked potentials (Q-MEP) during active stimulation compared to baseline and sham stimulation, indicating its potential for enhancing motor function.

The study found no significant changes in tibialis anterior motor evoked potentials (TA-MEP) or isometric strength, suggesting that tSCS specifically targets certain muscle responses, potentially reflecting its mechanism of action at the interneuronal or motoneuronal level.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Transcutaneous Spinal Cord Stimulation Enhances Quadriceps Motor Evoked Potential in Healthy Participants: A Double-Blind Randomized Controlled Study. Megía-García, Á., Serrano-Muñoz, D., Taylor, J., et al.[2020]

In a double-blinded study involving 2 male patients with incomplete spinal cord injury, anodal transcutaneous spinal direct current stimulation (tsDCS) improved all measured outcomes, including gait and balance, while cathodal tsDCS showed mixed results.

Both types of tsDCS were able to modulate corticospinal excitability, indicating that this non-invasive method could be a promising approach for enhancing rehabilitation in patients with spinal cord injuries.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Spinal direct current stimulation with locomotor training in chronic spinal cord injury. Abualait, TS., Ibrahim, AI.[2021]

Noninvasive transcutaneous spinal cord stimulation (tSCS) has been shown to effectively induce muscle activation in individuals with spinal cord injury, based on a systematic review of 13 studies involving 55 participants.

While tSCS appears to enhance voluntary movement, muscle strength, and function, further research is needed to determine the optimal electrode placement and current intensity for maximum efficacy.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Transcutaneous Spinal Cord Stimulation and Motor Rehabilitation in Spinal Cord Injury: A Systematic Review. Megía García, A., Serrano-Muñoz, D., Taylor, J., et al.[2021]

Transcutaneous spinal cord stimulation (tSCS) shows potential for generating motor activity in individuals with spinal cord injury, but the overall quality of the studies reviewed was poor-to-fair, indicating a need for more rigorous research.

The review identified significant variability in stimulation parameters and outcome measurements across the 25 studies analyzed, highlighting the necessity for standardized methods to improve the reliability and comparability of tSCS research.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Transcutaneous spinal cord stimulation and motor responses in individuals with spinal cord injury: A methodological review. Taylor, C., McHugh, C., Mockler, D., et al.[2022]

Electrical spinal cord stimulation (ESCS) provides significant and sustained pain relief for patients with chronic painful diabetic neuropathy, with median pain scores showing a marked reduction when the stimulator is on compared to when it is off.

The procedure has a low complication rate, with only two late complications reported among the patients, indicating that ESCS is a safe long-term treatment option for managing chronic pain.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Electrical spinal cord stimulation in the long-term treatment of chronic painful diabetic neuropathy. Daousi, C., Benbow, SJ., MacFarlane, IA.[2019]

Citations

1. rnel.pitt.edu rnel.pitt.edu/scs-sma

Spinal Cord Stimulation in Spinal Muscular Atrophy This research study is to explore the potential benefits of electrical spinal cord stimulation in muscle strength and endurance for people living with Spinal ...

2. upmc.com upmc.com/media/news/020525-spinal-cord-stimulation

Spinal Cord Stimulation Restores Neural Function, Targets ... Spinal cord stimulation improves muscle strength in individuals with spinal muscle atrophy.

3. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC9966619/

First Use of Non-Invasive Spinal Cord Stimulation in Motor ... Spinal cord stimulation may be an effective rehabilitation method in patients treated with nusinersen. More research is needed. Keywords: spinal ...

4. curesma.org curesma.org/wp-content/uploads/2025/03/Prat-Ortega-Epidural-spinal-cord-stimulation-in-SMA-Nature-Medicine-2025.pdf

First-in-human study of epidural spinal cord stimulation in ... Here we show that epidural spinal cord stimulation (SCS) improved motoneuron function, thereby increasing strength, endurance and gait quality, in three adults ...

5. nature.com nature.com/articles/s41591-024-02940-9

Non-invasive spinal cord electrical stimulation for arm and ... These results demonstrate the safety and efficacy of ARC EX Therapy to improve hand and arm functions in people living with cervical SCI.

6. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC11200420/

Non-Invasive Spinal Cord Stimulation for Motor ... The median duration of drug treatment was over 20 months. The application of tSCS was performed during physical therapy for 20–40 min per day for ~12 days.