Using stem cells to treat multiple system atrophy
Official title: Randomized Double-Blind Placebo-Controlled Adaptive Design Trial of Intrathecally Administered Autologous Mesenchymal Stem Cells in Multiple System Atrophy
Funded at Mayo Clinic Rochester · NIH-10872108 · Data updated 2026-11-12
This study is looking at whether using your own stem cells, delivered into the spinal canal, can help slow down the progression of multiple system atrophy (MSA), and it's for people living with this challenging condition.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Mayo Clinic Rochester NIH-funded |
| Lab location | 1 site (Rochester, United States) |
| Project start | September 10, 2021 |
| Project end | May 31, 2026 |
| Funding year | 2024 |
| Award (this year) | $792,634 |
| NIH project number | 5R01FD007290-04 |
| Project ID | NIH-10872108 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals diagnosed with early-stage multiple system atrophy who are seeking potential new treatment options.
✕Less likely to help
Patients with advanced multiple system atrophy or those who do not meet the eligibility criteria for the trial may not benefit from this research.
What this research studies
This research investigates the use of autologous mesenchymal stem cells (MSCs) delivered directly into the spinal canal to treat multiple system atrophy (MSA), a severe neurodegenerative disease. The study aims to determine the effectiveness and safety of this treatment approach through a randomized, double-blind, placebo-controlled trial. Patients will receive either the stem cell treatment or a placebo, and their progress will be monitored over time to assess changes in disease progression and overall health. The research builds on previous findings that suggest MSCs may help slow the progression of MSA by increasing neurotrophic factors in the body.
Why it matters
Potential benefit: If successful, this research could provide a new treatment option that slows the progression of multiple system atrophy and improves quality of life for patients.
How similar studies have gone: Previous studies have shown promising results with similar approaches using stem cells in neurodegenerative diseases, indicating potential for success in this trial.
Where this research is happening
-
Mayo Clinic Rochester
Rochester, United States Active
Researchers
- Principal investigator: Singer, Wolfgang — Mayo Clinic Rochester
- Study coordinator: Singer, Wolfgang
Related research
Other NIH-funded projects on the same topics.
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Integrated MRI biomarkers to diagnose and monitor multiple system atrophy
This project will help doctors use MRI markers to better diagnose multiple system atrophy and track how the disease progresses.
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Clinical Trial Readiness for Multiple System Atrophy
A multi-center study to help people with multiple system atrophy by creating better clinical and biological measures to use in future drug trials.
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Single-cell brain profiling to map MSA cell changes
Researchers will map gene activity in individual brain cells from people who had multiple system atrophy to understand how the disease affects cells in key brain regions.
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Investigating how inflammation caused by alpha-synuclein affects multiple system atrophy
This study is looking at how inflammation affects multiple system atrophy (MSA) and aims to understand how the immune system might play a role in the disease, with the hope of finding new ways to help people with MSA.
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Understanding brain inflammation in multiple system atrophy
This research explores how a specific immune signal, interferon-gamma, contributes to brain damage in multiple system atrophy.
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Single-cell gene map of brains from people with multiple system atrophy
This project reads gene activity in individual brain cells from people with multiple system atrophy to find cell-specific changes linked to the disease.
About this research
- This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
- Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
- For full project details, budget, and progress reports, visit the official NIH RePORTER page.