Stem Cell Therapy for ALS

Not currently recruiting at 5 trial locations

Overseen By Megan Means, CCRP

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: Mayo Clinic

Must be taking: Riluzole, Edaravone

Must not be taking: Immunosuppressants, Herbal medications

Disqualifiers: Autoimmunity, Malignancy, Infection, others

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

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: 4

Duration: 12 months

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial explores whether a stem cell treatment can help people with ALS, a disease that weakens muscles over time. The treatment uses special cells taken from the patient's own body fat and injects them into the fluid around the spine. The researchers aim to determine if this approach is safe and effective when administered every three months. Individuals who have experienced muscle weakness from ALS for less than two years might be suitable candidates for this trial. As a Phase 2 trial, the research focuses on measuring the treatment's effectiveness in an initial, smaller group of participants.

Will I have to stop taking my current medications?

The trial requires that you either be on a stable dose of riluzole or not have taken it for at least 30 days before joining. You also cannot use Radicava® (edaravone) within 30 days of screening or during the study. If you're using immunosuppressants, you must stop at least 4 weeks before the screening. You must also avoid starting any new supplements during the study.

Is there any evidence suggesting that this treatment is likely to be safe for humans?

Research has shown that using stem cells from a person's own body fat is generally safe for people with ALS. Previous studies found that injecting these cells into the fluid around the spine is well-tolerated. In one study with ALS patients, this treatment did not cause serious side effects, and most patients handled it well. So far, research supports that this approach is safe and suitable for clinical trials like this one. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for ALS?

Researchers are excited about using autologous adipose-derived mesenchymal stromal cells (aaMSCs) for ALS because this approach is quite different from current treatment options, which mainly include medications like riluzole and edaravone. Unlike these drugs, which aim to slow the progression of symptoms, aaMSCs offer a regenerative approach by potentially repairing damaged cells and reducing inflammation in the nervous system. The treatment is also unique because it involves injecting these cells directly into the spinal canal (intrathecally), which could allow for more targeted and effective delivery to the affected areas. This innovative mechanism and delivery method offer hope for not just slowing down the disease, but potentially improving patient outcomes in new ways.

What evidence suggests that this treatment might be an effective treatment for ALS?

This trial will investigate the use of Autologous Adipose-derived Mesenchymal Stromal Cells (aaMSCs) for treating ALS. Studies have shown that stem cells from fat tissue can be safe and may offer benefits. In one study, researchers took these stem cells from patients' own fat and injected them into the fluid around the spine. The results suggested that this method might help protect nerve cells from damage, which is crucial for slowing ALS progression. Other research has also highlighted the potential of these cells to protect the brain and spinal cord from further harm. While these findings are promising, more research is needed to fully understand the treatment's effectiveness for ALS patients. ^{2 4 6 7 8}

Who Is on the Research Team?

Nathan Staff, MD

Principal Investigator

Mayo Clinic

Anthony J Windebank, MD

Principal Investigator

Mayo Clinic

Are You a Good Fit for This Trial?

This trial is for U.S. residents over 18 with ALS, diagnosed per World Federation of Neurology criteria, who can travel to the study site and follow the protocol. They must have had a stable dose of riluzole or none for at least 30 days before joining. Women should be post-menopausal, have had a hysterectomy, or use two birth control methods.

Inclusion Criteria

I have been diagnosed with ALS according to World Federation of Neurology criteria.

I have been diagnosed with ALS based on tests, after ruling out other causes for my weakness.

I have experienced a gradual increase in muscle weakness for less than 2 years.

Exclusion Criteria

Autoimmunity, including Crohn's disease or rheumatoid arthritis

Enrolled in an investigational drug trial within 30 days of baseline visit

I rely on a machine to help me breathe.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive intrathecal injections of autologous adipose-derived mesenchymal stromal cells every 3 months for a total of 4 injections over 12 months

12 months

4 visits (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Autologous Adipose-derived Mesenchymal Stromal Cells

Trial Overview The trial tests the safety and effectiveness of injecting patients' own mesenchymal stem cells from body fat into their spinal fluid every three months (4 times total). These cells are thought to potentially repair nerve damage caused by ALS.

How Is the Trial Designed?

Group I: Mesenchymal Stromal Cells Experimental Treatment 1 Intervention

Autologous Adipose-derived Mesenchymal Stromal Cells (aaMSCs) will be administered intrathecally at a single dose in a volume of 5-10 mL, all patients will receive 5 x 10\^7 intrathecal aaMSCs at the first injection (Visit 4). Subsequent doses may be reduced to 1 x 10\^7 or increased to 1 x 10\^8, based on Dose Modification Rules.

Who Is Running the Clinical Trial?

Mayo Clinic

Lead Sponsor

Trials

3,427

Recruited

3,221,000+

State of Minnesota Regenerative Medicine Minnesota

Collaborator

Trials

1

Recruited

80+

Citations

1. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/41132886/

Adipose-derived mesenchymal stem cells for the treatment ... Adipose-derived mesenchymal stem cells for the treatment of Amyotrophic Lateral Sclerosis. A phase I/II safety and efficacy clinical trial.

2. neurology.org neurology.org/doi/10.1212/WNL.0000000000204894

Results from a Phase 2 Clinical Trial of Repeated ... In this Phase 2 open label clinical trial, we aim to evaluate the safety and efficacy of repeated intrathecal autologous adipose-derived mesenchymal stromal ...

3. frontiersin.org frontiersin.org/journals/neurology/articles/10.3389/fneur.2025.1655124/full

Adipose-derived mesenchymal stem cells for the treatment ... Studies involving patients with multiple sclerosis have also demonstrated safety and signs of efficacy, suggesting neuroprotection (46–48). Also, a clinical ...

4. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/27784774/

Safety of intrathecal autologous adipose-derived ... - PubMed Conclusions: Intrathecal treatment of autologous adipose-derived mesenchymal stromal cells appears safe at the tested doses in ALS. These ...

5. sciencedirect.com sciencedirect.com/science/article/pii/S0024320524003515

Adipose-derived mesenchymal stromal cells in clinical trials Adipose-derived mesenchymal stem cells combined with extracellular vesicles may improve amyotrophic lateral sclerosis. Front. Aging Neurosci ...

6. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC5123559/

Safety of intrathecal autologous adipose-derived ... This phase I study provides Class IV evidence that in patient with ALS, intrathecal autologous adipose-derived mesenchymal stromal cell therapy is safe.

7. neals.org neals.org/als-trials/nct03268603

A Phase II Study of Intrathecal Autologous Adipose-derived ... The purpose of this study is to determine the safety and efficacy of intrathecal treatment delivered to the cerebrospinal fluid (CSF) of mesenchymal stem cells ...

8. sciencedirect.com sciencedirect.com/science/article/pii/S2352320421000596

Safety and efficacy of bone marrow derived-mesenchymal ... Our study revealed that the concurrent IT and IV application of BM-MSCs in patients with ALS is a safe procedure.