Mesenchymal Stem Cells for Parkinson's Disease

Overseen By Vanessa Thyne, MS

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: The University of Texas Health Science Center, Houston

Must be taking: Levodopa

Disqualifiers: Psychosis, Cognitive impairment, Cardiac disease, others

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

Approved in 5 Jurisdictions This treatment is already approved in other countries

Compensation: Varies

Number of Visits: 3

Duration: 12 months

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests whether mesenchymal stem cells (MSCs) can slow the progression of Parkinson's disease. Researchers aim to determine the safest and most effective method for administering repeat doses of these stem cells. Participants will receive either MSC infusions, a combination of MSC and placebo, or only placebo. Suitable candidates have had Parkinson's disease for 3 to 10 years, exhibit key symptoms like slow movement (bradykinesia), and respond well to current Parkinson's medications. As a Phase 2 trial, this research focuses on evaluating the treatment's effectiveness in an initial, smaller group of people.

Will I have to stop taking my current medications?

The trial does not specify if you need to stop taking your current medications, but it does require that you are on levodopa-containing medications. It's best to discuss your specific medications with the trial team.

Is there any evidence suggesting that this trial's treatments are likely to be safe?

Research has shown that mesenchymal stem cells (MSCs) are generally safe for humans. These cells, taken from bone marrow, did not cause major side effects in earlier studies. One study found that MSCs were safe for up to 150 days, suggesting potential usefulness for treatment. Another study involving patients with ALS, a different condition, also reported that MSCs were safe when used in certain ways. While specific information on Parkinson's disease is still being gathered, these findings offer promise for those considering participation in trials. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for Parkinson's disease?

Researchers are excited about using Mesenchymal Stem Cells (MSCs) for Parkinson’s Disease because these cells have the potential to modify the disease itself, not just manage symptoms. Unlike current treatments, which primarily focus on dopamine replacement to alleviate symptoms, MSCs might promote nerve repair and slow disease progression by targeting inflammation and aiding in neuroprotection. This new approach could revolutionize treatment by addressing the underlying causes of Parkinson’s, offering hope for more lasting improvements in patients' quality of life.

What evidence suggests that this trial's treatments could be effective for Parkinson's disease?

Research has shown that mesenchymal stem cells (MSCs), one of the treatments under study in this trial, could be promising for various diseases. These cells play a crucial role in regenerative medicine due to their ability to renew themselves and repair damaged tissues. Studies have found that MSCs from bone marrow can improve outcomes in different conditions, such as reducing injury size. They have also been linked to better heart function, highlighting their wide-ranging abilities. While specific research on Parkinson's disease is still developing, these findings suggest that MSCs might help slow the disease by repairing or replacing damaged cells. Participants in this trial may receive MSCs alone or in combination with a placebo, while others will receive only placebo infusions. ^{6 7 8 9 10}

Who Is on the Research Team?

Mya C Schiess, MD

Principal Investigator

The University of Texas Health Science Center, Houston

Are You a Good Fit for This Trial?

Inclusion Criteria

You have a mild to complete loss of sense of smell.

You have mild to moderate Parkinson's disease.

You have been diagnosed with Parkinson's disease between 3 and 10 years ago.

Exclusion Criteria

Active anticoagulation treatment and/or abnormal INR.

An atypical DAT scan or MRI supporting an alternative explanation for PD symptoms

Patient not on levodopa containing medications

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive 3 infusions of MSC or placebo every 4 months

12 months

3 visits (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment

6 months

Multiple visits (in-person and virtual)

What Are the Treatments Tested in This Trial?

Interventions

• MSC
• Placebo

How Is the Trial Designed?

Group I: Mesenchymal Stem Cells and Placebo Experimental Treatment 2 Interventions

2 infusions of 10 X 10\^6 MSC/kg and 1 placebo infusion, all doses administered 4 months apart.

Group II: Mesenchymal Stem Cells Experimental Treatment 1 Intervention

3 infusions of 10 X 10\^6 MSC/kg administered every 4 months.

Group III: Placebo Placebo Group 1 Intervention

3 placebo doses administered every 4 months.

MSC is already approved in United States, European Union, Canada, Japan, China for the following indications:

Approved in United States as Mesenchymal Stem Cells for:

• Graft-versus-host disease
• Crohn's disease
• Multiple sclerosis
• Type 1 diabetes
• Cardiovascular diseases

Approved in European Union as Mesenchymal Stem Cells for:

• Graft-versus-host disease
• Crohn's disease
• Multiple sclerosis
• Type 1 diabetes
• Cardiovascular diseases

Approved in Canada as Mesenchymal Stem Cells for:

• Graft-versus-host disease
• Crohn's disease
• Multiple sclerosis
• Type 1 diabetes
• Cardiovascular diseases

Approved in Japan as Mesenchymal Stem Cells for:

• Graft-versus-host disease
• Crohn's disease
• Multiple sclerosis
• Type 1 diabetes
• Cardiovascular diseases

Approved in China as Mesenchymal Stem Cells for:

• Graft-versus-host disease
• Crohn's disease
• Multiple sclerosis
• Type 1 diabetes
• Cardiovascular diseases

Who Is Running the Clinical Trial?

The University of Texas Health Science Center, Houston

Lead Sponsor

Trials

974

Recruited

361,000+

Michael J. Fox Foundation for Parkinson's Research

Collaborator

Trials

117

Recruited

537,000+

Published Research Related to This Trial

Mesenchymal stromal cells (MSCs) are promising for treating various diseases due to their immune-suppressive properties and regenerative capabilities, with unmodified MSCs showing good safety profiles in clinical settings.

Genetically engineered MSCs, while offering enhanced therapeutic potential, require thorough non-clinical safety assessments to evaluate risks associated with introducing new genes, especially when using non-viral modification methods to minimize risks of cell transformation.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Considerations in the Design of Non-Clinical Development Programmes to Support Non-Viral Genetically Modified Mesenchymal Stromal Cell Therapies. Iansante, V., Brooks, A., Coney, L.[2021]

A meta-analysis of 152 randomized clinical trials involving 9228 individuals treated with mesenchymal stromal cells (MSCs) found that fever is the only adverse event closely associated with MSC therapy, with a significant odds ratio of 1.61.

The analysis also indicated that MSC therapy may lower the incidence of tachycardia and fatigue, although these findings were not statistically significant, suggesting a generally favorable safety profile for MSC treatments.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Transient Fever: the Sole Treatment-Related Adverse Event associated with Mesenchymal Stromal Cells and Solid Clues from the Real World. Wang, Y., Mou, Q., Yi, H., et al.[2023]

A review of 62 randomized clinical trials involving 3546 participants found that mesenchymal stem cell (MSC) therapy is generally safe, with no serious adverse events like death or infection reported.

While MSC therapy was associated with some mild adverse effects such as transient fever and fatigue, it also showed a trend towards reducing the incidence of arrhythmia, suggesting a favorable safety profile compared to placebo treatments.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

The safety of MSC therapy over the past 15 years: a meta-analysis. Wang, Y., Yi, H., Song, Y.[2021]

Citations

1. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC6719501/

The Pros and Cons of Mesenchymal Stem Cell-Based ... Here, we reviewed the current knowledge about MSCs in terms of their therapeutic potential, clinical effects and safety in clinical applications.

2. nature.com nature.com/articles/s41392-025-02313-9

Mesenchymal stem cells in treating human diseases Mesenchymal stem cells (MSCs) have emerged as a highly promising strategy in regenerative medicine due to their self-renewal, ...

3. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/32141308/

Clinical therapeutic efficacy of mesenchymal stem cells ... BMSCs showed significant VAS score improvement, while ADMSCs showed significant improvement in VAS and WOMAC scores. ADMSCs tended to be superior to BMSCs.

4. sciencedirect.com sciencedirect.com/science/article/pii/S2405844024010818

Effects of bone marrow mesenchymal stromal cells-derived ... As well, MSC(M) derived EVs were effective in reducing lesion volume by an SMD of - 0.80 (95 % CI -1.20; −0.40; p < 0.01). It was observed that ...

5. stemcellres.biomedcentral.com stemcellres.biomedcentral.com/articles/10.1186/s13287-024-03829-7

a meta-analysis of randomized controlled trials | Stem Cell ... Both types of stem cells are effective in improving LVEF in patients with heart failure without any noticeable difference between the cells.

6. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC8173294/

Safety and therapeutic potential of human bone marrow ... This review aims to provide an overview of safety, homing potential and ongoing work aimed at understanding the potential of these cells in regenerative ...

7. atcc.org atcc.org/products/pcs-500-012

Bone Marrow-Derived Mesenchymal Stem Cells The cells can be used in adult stem cell differentiation, regenerative medicine, cell therapy, tissue engineering, and generation of iPS cell lines.

8. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC10704945/

Preclinical Short-term and Long-term Safety of Human ... We confirmed that hBMMSCs are safe through a 150-day study, demonstrating that this is a safe and promising therapy and offering preliminary ...

9. sciencellonline.com sciencellonline.com/en/human-bone-marrow-derived-mesenchymal-stem-cells/?srsltid=AfmBOoq5PD1krMk3fYFzjs9wxtBVAz_uXgGeJQv5J8XNh88fT7kE_djp

Human Bone Marrow-derived Mesenchymal Stem Cells Human Bone Marrow-derived Mesenchymal Stem Cells (HMSC-bm ) are isolated from human bone marrow ... Safety Data Sheets (SDS) · Distributors · Promotions · Custom ...

10. 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.