MSC Therapy for Asthma

Recruiting in Palo Alto (17 mi)

Overseen by Edwin Horwitz, MD

Age: 18 - 65

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 1

Recruiting

Sponsor: Emory University

Must not be taking: Corticosteroids, Macrolides, Anticonvulsants, others

Disqualifiers: Chronic lung disease, Smoking, Diabetes, others

No Placebo Group

Trial Summary

What is the purpose of this trial?

This trial is testing the safety of using special stem cells to treat children and young adults with severe asthma that doesn't improve with regular treatments. The stem cells are given through an IV and may help reduce inflammation in the airways. The study aims to find out if this new treatment is safe and effective.

Do I need to stop my current medications for the trial?

The trial requires that you stop using oral or injectable corticosteroids and certain other medications that interact with corticosteroids at least two weeks before the screening visit. Nasal corticosteroids can be used during the trial, and you should not start new asthma therapies until 7 days after the γMSC infusion.

What data supports the effectiveness of the treatment Interferon gamma-primed mesenchymal stromal cells (MSCs) for asthma?

Research shows that low doses of interferon-gamma (a protein that helps regulate the immune system) can reduce inflammation in the airways of mice with asthma. This suggests that treatments involving interferon-gamma, like the one in the trial, might help manage asthma symptoms by reducing airway inflammation. ^{1 2 3 4 5}

Is MSC therapy generally safe for humans?

The safety of mesenchymal stem cells (MSCs) for asthma treatment has not been confirmed in the studies reviewed, as they primarily focus on animal models and do not provide specific safety data for humans. ^{6 7 8 9 10}

How is the treatment with Interferon gamma-primed mesenchymal stromal cells (MSCs) different from other asthma treatments?

This treatment is unique because it uses mesenchymal stromal cells (MSCs) that are primed with interferon gamma, which may help modulate immune responses in asthma by reducing inflammation and airway hyperreactivity, offering a novel approach compared to traditional asthma therapies that primarily focus on symptom relief. ^{1 3 4 11 12}

Research Team

Edwin Horwitz, MD

Principal Investigator

Emory University

Eligibility Criteria

This trial is for individuals aged 18-30 with moderate-to-severe persistent asthma diagnosed in childhood and evidence of atopy. Participants must not have other chronic lung diseases, be on new biologic asthma therapies, or have used oral/injectable corticosteroids within two weeks prior to screening. Women must use birth control if applicable.

Inclusion Criteria

My asthma started when I was a child.

I have moderate-to-severe asthma.

I am between 18 and 30 years old.

Exclusion Criteria

I have been on a stable allergy shot regimen for at least 3 months.

I haven't taken drugs like carbamazepine or erythromycin in the last two weeks.

I have a history of eye issues or conditions worsened by steroids.

Trial Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

1 visit (in-person)

Treatment

Participants receive a single intravenous infusion of γMSCs at either 2x10^6 cells/kg or 5x10^6 cells/kg

1 day

1 visit (in-person)

Observation

Participants are observed for adverse reactions and safety assessments post-infusion

7 to 30 days

Multiple visits (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment, including lung function tests and analysis of inflammatory markers

1 year

Up to 12 visits (in-person)

Treatment Details

Interventions

• Interferon gamma-primed mesenchymal stromal cells (MSCs) (Mesenchymal Stromal Cells)

Trial Overview The study tests the safety and optimal dose of two types of mesenchymal stromal cells (MSCs) treatments: one derived from cord tissue (cMSCs), another primed with interferon gamma from bone marrow (γMSCs). It's a Phase I trial with children receiving escalating doses to assess tolerance.

Participant Groups

Group I: Infusion of γMSCs Experimental Treatment 3 Interventions

Escalating doses Dose escalation design with two dose levels. The low dose level involves a single intravenous infusion of γMSCs at 2x106 cells/kg. The high dose level involves a single intravenous infusion of γMSCs at 5x106 cells/kg.

Find a Clinic Near You

Research Locations Nearby Select from list below to view details:

Children's Healthcare of Atlanta Atlanta, GA

Who Is Running the Clinical Trial?

Emory University

Lead Sponsor

Trials

1735

Recruited

2,605,000+

Dr. George Painter

Emory University

Chief Executive Officer since 2013

PhD in Synthetic Organic Chemistry from Emory University

Dr. R. Donald Harvey

Emory University

Chief Medical Officer

MD from Emory University School of Medicine

The Marcus Foundation

Collaborator

Trials

19

Recruited

2,200+

Ossium Health, Inc.

Industry Sponsor

Trials

11

Recruited

200+

Findings from Research

Inhaled IFN-λ significantly reduces Th2 and Th17 inflammation in the lungs of asthmatic mice, suggesting its potential as a therapeutic option for controlling allergic asthma.

The treatment not only improved lung histopathology and normalized lung resistance but also increased IL-10 secretion, indicating a regulatory effect on inflammation even after asthma has developed.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Inhaled delivery of recombinant interferon-lambda restores allergic inflammation after development of asthma by controlling Th2- and Th17-cell-mediated immune responses. Won, J., Jo, A., Gil, CH., et al.[2023]

References

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

Mucosal IFN-gamma gene transfer inhibits pulmonary allergic responses in mice. [2008]

2. Germany pubmed.ncbi.nlm.nih.gov

[Effect of recombinant gamma interferon on allergic skin reaction and bronchoconstriction]. [2011]

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

Low doses of exogenous interferon-γ attenuated airway inflammation through enhancing Fas/FasL-induced CD4+ T cell apoptosis in a mouse asthma model. [2021]

4. Netherlands pubmed.ncbi.nlm.nih.gov

Inhaled delivery of recombinant interferon-lambda restores allergic inflammation after development of asthma by controlling Th2- and Th17-cell-mediated immune responses. [2023]

5. New Zealand pubmed.ncbi.nlm.nih.gov

Long-Term Use of Maintenance Systemic Corticosteroids is Associated with Multiple Adverse Conditions in a Large, Real-World Cohort of US Adults with Severe Asthma. [2022]

6. England pubmed.ncbi.nlm.nih.gov

Therapeutic administration of bone marrow-derived mesenchymal stromal cells reduces airway inflammation without up-regulating Tregs in experimental asthma. [2019]

7. Korea (South) pubmed.ncbi.nlm.nih.gov

Mesenchymal stem cell transfer suppresses airway remodeling in a toluene diisocyanate-induced murine asthma model. [2022]

8. Australia pubmed.ncbi.nlm.nih.gov

Pollen-induced antigen presentation by mesenchymal stem cells and T cells from allergic rhinitis. [2020]

9. Greece pubmed.ncbi.nlm.nih.gov

Mesenchymal stem cells abrogate experimental asthma by altering dendritic cell function. [2018]

10. Korea (South) pubmed.ncbi.nlm.nih.gov

Evaluation of Human MSCs Treatment Frequency on Airway Inflammation in a Mouse Model of Acute Asthma. [2021]

11. England pubmed.ncbi.nlm.nih.gov

Inhaled delivery of Interferon-lambda restricts epithelial-derived Th2 inflammation in allergic asthma. [2020]

12. Netherlands pubmed.ncbi.nlm.nih.gov

Interferon-alpha inhibits airway eosinophilia and hyperresponsiveness in an animal asthma model [corrected]. [2021]