Phase IIa Randomized Placebo Controlled Trial: Mesenchymal Stem Cells as a Disease-modifying Therapy for Idiopathic 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

45 Participants Needed

Trial Summary

What is the purpose of this trial?

This trial is testing how many doses of special donor bone marrow cells are safe and effective for slowing down Parkinson's disease. The study involves 45 patients and aims to find the best way to use these cells to help protect and repair brain cells. The goal is to see if this treatment can slow the progression of the disease. Bone marrow stromal cells have been explored as an alternative source for neural transplantation and repair in Parkinson's disease.

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.

What data supports the effectiveness of the treatment involving Mesenchymal Stem Cells (MSC)?

Research shows that Mesenchymal Stem Cells (MSC) have anti-inflammatory properties and can help in tissue regeneration, which may be beneficial in various clinical settings. They have been used to enhance bone marrow transplants and are being studied for their potential in treating heart attacks, bone regeneration, and immune-related conditions. ^{1 2 3 4 5}

Is MSC therapy safe for humans?

Research over the past 15 years suggests that mesenchymal stem cell (MSC) therapy is generally safe for humans, with transient fever being the most common treatment-related side effect. Studies have shown that unmodified MSCs have a good safety profile, although more research is needed to confirm long-term safety. ^{6 7 8 9 10}

How is the MSC treatment different from other treatments for this condition?

The MSC treatment is unique because it uses mesenchymal stem cells, which are multipotent cells capable of differentiating into various cell types, including bone, cartilage, and fat cells. This treatment focuses on enhancing the number of these stem cells and maintaining their differentiation potential, which can be particularly beneficial for conditions requiring tissue regeneration or repair. ^{2 11 12 13 14}

Research Team

Mya C Schiess, MD

Principal Investigator

The University of Texas Health Science Center, Houston

Eligibility Criteria

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

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)

Treatment Details

Interventions

• MSC
• Placebo

Participant Groups

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+

Findings from Research

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]

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 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]

References

1. England pubmed.ncbi.nlm.nih.gov

Anti-inflammatory effects of mesenchymal stem cells: novel concept for future therapies. [2022]

2. Netherlands pubmed.ncbi.nlm.nih.gov

Osteogenic differentiation of mesenchymal stem cells is impaired by bone morphogenetic protein 7. [2018]

3. Sweden pubmed.ncbi.nlm.nih.gov

[Mesenchymal stem cells. Basic science and future clinical use]. [2012]

4. England pubmed.ncbi.nlm.nih.gov

The therapeutic potential of mesenchymal stem cells. Cell- & tissue-based therapy. [2019]

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

Mesenchymal stromal cells. [2021]

6. United Arab Emirates 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. [2023]

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

Cell therapy with intravascular administration of mesenchymal stromal cells continues to appear safe: An updated systematic review and meta-analysis. [2022]

8. Switzerland pubmed.ncbi.nlm.nih.gov

Considerations in the Design of Non-Clinical Development Programmes to Support Non-Viral Genetically Modified Mesenchymal Stromal Cell Therapies. [2021]

9. England pubmed.ncbi.nlm.nih.gov

The safety of MSC therapy over the past 15 years: a meta-analysis. [2021]

10. Netherlands pubmed.ncbi.nlm.nih.gov

Platelet-derived growth factors for GMP-compliant propagation of mesenchymal stromal cells. [2018]

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

Multi-lineage potential research of bone marrow-derived stromal cells (BMSCs) from cattle. [2018]

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

Mesenchymal cell contributions to the stem cell niche. [2022]

13. England pubmed.ncbi.nlm.nih.gov

A simple and reliable protocol for long-term culture of murine bone marrow stromal (mesenchymal) stem cells that retained their in vitro and in vivo stemness in long-term culture. [2022]

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

Nonadherent cell population of human marrow culture is a complementary source of mesenchymal stem cells (MSCs). [2018]