What side effects are associated with this type of intervention?

The most common treatments for Multiple System Atrophy (MSA) include various forms of regenerative therapies, such as adipose-derived autologous mesenchymal stem cells. Known side effects of mesenchymal stem cell treatments include infections and infestations, which were the most frequently reported adverse events. In a study, 25% of adverse events were related to infections, and no serious adverse events were directly linked to the treatment. Overall, mesenchymal stem cell therapy was found to be safe and well-tolerated, although it did not show significant efficacy in treating active forms of multiple sclerosis, which may be indicative of similar outcomes in MSA.

What prior FDA approvals exist?

There are no FDA-approved treatments specifically for Multiple System Atrophy (MSA) mentioned in the provided context. The current study focuses on the use of adipose-derived autologous mesenchymal stem cells, a regenerative therapy aimed at slowing or halting disease progression. This approach is still under investigation, and no similar regenerative therapies have been approved by the FDA for MSA to date.

What other types of research exist?

Promising novel alternatives to adipose-derived autologous mesenchymal stem cells for Multiple System Atrophy patients include: 1) Induced pluripotent stem cell (iPSC)-derived therapies, which have shown potential in neurodegenerative diseases; 2) Gene therapy approaches targeting specific genetic mutations or pathways involved in MSA; 3) Antisense oligonucleotide (ASO) therapies, which are being explored for other neurodegenerative conditions and could be adapted for MSA; 4) Exosome-based therapies, which utilize cell-derived vesicles to deliver therapeutic molecules and have shown promise in preclinical studies.

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Stem Cell Therapy

Stem Cell Therapy for Multiple System Atrophy

Phase 2

Recruiting

Led By Wolfgang Singer, MD

Research Sponsored by Mayo Clinic

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Clinical diagnosis of MSA, fulfilling consensus criteria for probable MSA

UMSARS I (omitting question 11) between 5 and 17, and able to walk unaided (i.e. able to walk at least 50 yards without the use of a cane or walker, and without other support such as holding on to an arm or touching walls).

Must not have

Medications that could affect clinical evaluations are permitted but need to be withdrawn at least four half-lives prior to study visits. Those include medications used to treat motor symptoms, such as levodopa and other anti-Parkinsonian medications

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 12 months

Summary

This trial uses stem cells from a patient's own fat, injected into the spinal fluid, to treat early-stage Multiple System Atrophy (MSA). The goal is to slow down the disease by supporting and protecting nerve cells.

Who is the study for?

Adults aged 30-70 with Multiple System Atrophy (MSA) who can walk unaided and have a life expectancy of at least 3 years. They must have normal cognition, not be pregnant or breastfeeding, agree to birth control if applicable, and haven't taken investigational drugs recently. Excluded are those with MRI contraindications, unstable medical conditions, certain medication use, or severe heart, liver or kidney diseases.

What is being tested?

This trial tests whether injecting patients' own mesenchymal stem cells into their spinal fluid is safe and effective for treating MSA. It's a randomized study where some get the real treatment while others get a placebo without knowing which one they received.

What are the potential side effects?

Potential side effects may include reactions at the injection site in the spine such as pain or infection; immune responses since stem cells are involved; and possible unknown risks due to the innovative nature of this therapy.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I have been diagnosed with probable multiple system atrophy (MSA).

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I can walk by myself for at least 50 yards and my UMSARS I score is between 5 and 17.

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I can walk by myself for at least 50 yards and my UMSARS I score is between 5 and 17.

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My cognitive function is normal, scoring 26 or higher on the MOCA test.

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I am between 30 and 70 years old and can consent to treatment.

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I have been diagnosed with probable multiple system atrophy (MSA).

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

Select...

I can stop my Parkinson's medication 4 half-lives before study visits.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 12 months

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~12 months

This trial's timeline: 3 weeks for screening, Varies for treatment, and 12 months for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Change in UMSARS total (= UMSARS I + UMSARS II) score

Secondary study objectives

Change in COMPASS select score

Change in UMSARS I score

Change in UMSARS II score

+2 more

Trial Design

3Treatment groups

Active Control

Placebo Group

Group I: Arm 2Active Control2 Interventions

25 million mesenchymal stem cells administered intrathecally every 6 months for 2 injections (placebo injections at 3 month and 9 month timepoints)

Group II: Arm 1Active Control1 Intervention

25 million mesenchymal stem cells administered intrathecally every 3 months for 4 injections

Group III: Arm 3Placebo Group1 Intervention

Placebo (lactated Ringer's) administered intrathecally every 3 months for 4 injections

0 / 7Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Multiple System Atrophy (MSA) is a progressive neurodegenerative disorder with no current treatments to slow or halt its progression. Adipose-derived autologous mesenchymal stem cells (MSCs) are being studied for their potential regenerative and neuroprotective effects. MSCs can differentiate into various cell types and secrete neurotrophic factors that support neuron survival and reduce inflammation. This is particularly important for MSA patients as it offers a potential therapeutic approach to preserve neuronal function and slow disease progression, addressing the critical need for effective treatments in this debilitating condition.

Therapeutic efficacy of multipotent adult progenitor cells versus mesenchymal stem cells in experimental autoimmune encephalomyelitis.Neuroprotective effects of mesenchymal stem cells on spinal motoneurons following ventral root axotomy: synapse stability and axonal regeneration.