Stem Cell Therapy for ALS

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

75 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

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 in ALS patients every 3 months for a total of 4 injections over 12 months. Mesenchymal stem cells (MSCs) are a type of stem cell that can be grown into a number of different kinds of cells. In this study, MSCs will be taken from the subject's body fat and grown. CSF is the fluid surrounding the spine. The use of mesenchymal stem cells is considered investigational, which means it has not been approved by the Food and Drug Administration (FDA) for routine clinical use. However, the FDA has allowed the use of mesenchymal stem cells in this research study.

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 stem cell therapy safe for ALS patients?

Research shows that stem cell therapy, including autologous adipose-derived mesenchymal stromal cells, is generally safe for ALS patients. Most studies report no severe side effects, although some patients experienced minor issues like chest pain or shortness of breath. Long-term monitoring indicates that the treatment is well-tolerated without accelerating disease progression. ^{1 2 3 4 5}

How is the treatment with autologous adipose-derived mesenchymal stromal cells different from other ALS treatments?

This treatment uses a patient's own fat-derived stem cells, which are injected into the spinal fluid to potentially protect nerve cells and improve motor function, offering a novel approach compared to traditional ALS treatments that mainly focus on symptom management. ^{1 4 6 7 8}

What data supports the effectiveness of the treatment Autologous Adipose-derived Mesenchymal Stromal Cells for ALS?

Research in animal models of ALS shows that adipose-derived mesenchymal stem cells can improve motor function and delay disease onset by protecting motor neurons and reducing harmful inflammation. Additionally, a case study reported long-term survival in an ALS patient who received this treatment. ^{1 4 6 9 10}

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. United States pubmed.ncbi.nlm.nih.gov

Safety of intrathecal autologous adipose-derived mesenchymal stromal cells in patients with ALS. [2021]

2. United States pubmed.ncbi.nlm.nih.gov

Systemic treatment with adipose-derived mesenchymal stem cells ameliorates clinical and pathological features in the amyotrophic lateral sclerosis murine model. [2022]

3. United States pubmed.ncbi.nlm.nih.gov

Adipose-derived stem cells protect motor neurons and reduce glial activation in both in vitro and in vivo models of ALS. [2021]

4. Italy pubmed.ncbi.nlm.nih.gov

Long-term survival of a patient with amyotrophic lateral sclerosis (ALS) who received autologous adipose-derived mesenchymal stem cells. [2022]

5. England pubmed.ncbi.nlm.nih.gov

Mesenchymal stromal cell transplantation in amyotrophic lateral sclerosis: a long-term safety study. [2022]

6. Iran pubmed.ncbi.nlm.nih.gov

Safety and Efficacy of Allogeneic Adipose Tissue Mesenchymal Stromal Cells in Amyotrophic Lateral Sclerosis Patients, Single-Center, Prospective, Open-Label, Single-Arm Clinical Trial, Long-Term Follow-up. [2022]

7. England pubmed.ncbi.nlm.nih.gov

Stem cell therapy in amyotrophic lateral sclerosis: a methodological approach in humans. [2019]

8. England pubmed.ncbi.nlm.nih.gov

Lumbar intraspinal injection of neural stem cells in patients with amyotrophic lateral sclerosis: results of a phase I trial in 12 patients. [2023]

9. Austria pubmed.ncbi.nlm.nih.gov

Intrathecal application of neuroectodermally converted stem cells into a mouse model of ALS: limited intraparenchymal migration and survival narrows therapeutic effects. [2018]

10. United States pubmed.ncbi.nlm.nih.gov

Bone marrow mesenchymal stem cells from healthy donors and sporadic amyotrophic lateral sclerosis patients. [2019]