Mesenchymal Stem Cells for Lupus

Recruiting in Palo Alto (17 mi)

+8 other locations

Overseen by Gary S. Gilkeson, MD

Age: 18+

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 2

Waitlist Available

Sponsor: Medical University of South Carolina

Prior Safety Data

Trial Summary

What is the purpose of this trial?

The purpose of this study is to evaluate the efficacy and safety of mesenchymal stem cells (MSCs) obtained from umbilical cords for the treatment of adults with systemic lupus erythematosus (SLE). The goal of this study is to determine if patients receiving an MSC infusion plus standard of care respond better than patients receiving placebo infusion plus standard of care.

Do I have to stop taking my current medications to join the trial?

The trial protocol does not specify if you must stop all current medications. However, you cannot change or add to your immunosuppressant regimen within 3 months of the Baseline visit, and you must not have used prednisone >0.5 mg/kg/day within 1 month of the Baseline visit. Also, you should not have received certain biologic therapies like belimumab within 2 months or rituximab within 6 months of the Baseline visit.

What data supports the idea that Mesenchymal Stem Cells for Lupus is an effective treatment?

The available research shows that umbilical cord-derived mesenchymal stem cells (MSCs) have been tested in patients with severe and difficult-to-treat lupus. These studies found that MSCs were safe and had positive effects on the condition. For example, one study reported clinical improvements in patients who had not responded to other treatments for at least six months. Another study highlighted the potential of MSCs to help regulate the immune system, which is important in managing lupus. Overall, these findings suggest that MSCs could be a promising option for people with lupus who have not had success with other treatments.

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What safety data is available for mesenchymal stem cell treatment in lupus?

The long-term safety of umbilical cord-derived mesenchymal stem cells (UC MSCs) for treating systemic lupus erythematosus (SLE) has been demonstrated in a 6-year follow-up study. In this study, nine patients with refractory SLE received UC MSCs infusions and were monitored for adverse events and changes in health markers. Only one patient experienced mild dizziness and warmth shortly after infusion, which resolved quickly. No other adverse events were reported, and there were no significant changes in blood counts, liver function, electrocardiogram, chest radiography, or serum tumor markers over the 6-year period. This suggests a good safety profile for UC MSCs in SLE patients.

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Is the treatment using Mesenchymal Stem Cells (MSCs) promising for lupus?

Yes, the treatment using Mesenchymal Stem Cells (MSCs) is promising for lupus. Studies show that these cells, derived from umbilical cords, have a good safety profile and can help improve symptoms in patients with severe and hard-to-treat lupus. They have shown positive effects in regulating the immune system, which is beneficial for managing lupus.

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Eligibility Criteria

Adults aged 18-65 with active Systemic Lupus Erythematosus (SLE), evidenced by specific antibody tests and a minimum SLEDAI score, can join. They must have been on standard therapy for lupus nephritis if present. Exclusions include severe kidney issues, certain infections like HIV or TB, recent cancer (except some skin cancers/cervical carcinoma in situ), pregnancy/breastfeeding without contraception use, major organ transplants, unstable medical conditions, high steroid use recently or changes in immunosuppressants.

Inclusion Criteria

Historical presence of at least 4 of 11 of the ACR Classification Criteria

I am between 18 and 65 years old.

My lupus is active despite treatment, as shown by specific lupus activity scores.

Exclusion Criteria

I have lupus affecting my kidneys and need dialysis.

I haven't had cancer in the last 5 years, except for certain skin cancers or cervical pre-cancer.

My lupus affects my brain and thinking.

Participant Groups

The trial is testing the effectiveness of two different doses of Mesenchymal Stem Cells from umbilical cords against a placebo. All participants will continue their standard lupus treatments. The main goal is to see if those who get the stem cells show more improvement than those who get the placebo.

Group I: Low Dose Mesenchymal Stem Cells (MSCs) Experimental Treatment 1 Intervention

Mesenchymal Stem Cells (MSCs) 1 x 10\^6 cells/kg in Plasma-Lyte A solution

Group II: High Dose Mesenchymal Stem Cells (MSCs) Experimental Treatment 1 Intervention

Mesenchymal Stem Cells MSCs 5 x 10\^6 cells/kg in Plasma-Lyte A solution

Group III: Plasma Lyte A Solution Placebo Group 1 Intervention

Placebo Infusion (Plasma-Lyte A solution only)

High Dose Mesenchymal Stem Cells (MSCs) is already approved in China, United States for the following indications:

🇨🇳 Approved in China as Mesenchymal Stem Cells for:

• Systemic Lupus Erythematosus (SLE)
• Lupus Nephritis

🇺🇸 Approved in United States as Umbilical Cord-Derived Mesenchymal Stem Cells for:

• Refractory Systemic Lupus Erythematosus (SLE)

Find a Clinic Near You

Research Locations Nearby Select from list below to view details:

Medical University of South Carolina Charleston, SC

University of California - San Diego San Diego, CA

Northwestern University Chicago, IL

The Feinstein Institute for Medical Research Manhasset, NY

More Trial Locations

Who Is Running the Clinical Trial?

Medical University of South Carolina Lead Sponsor

References

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

Umbilical cord mesenchymal stem cell transplantation in active and refractory systemic lupus erythematosus: a multicenter clinical study. [2022] In our present single-center pilot study, umbilical cord (UC)-derived mesenchymal stem cells (MSCs) had a good safety profile and therapeutic effect in severe and refractory systemic lupus erythematosus (SLE). The present multicenter clinical trial was undertaken to assess the safety and efficacy of allogeneic UC MSC transplantation (MSCT) in patients with active and refractory SLE.

2. England pubmed.ncbi.nlm.nih.gov

Safety, immunological effects and clinical response in a phase I trial of umbilical cord mesenchymal stromal cells in patients with treatment refractory SLE. [2022] Reports of clinical improvement following mesenchymal stromal cell (MSC) infusions in refractory lupus patients at a single centre in China led us to perform an explorative phase I trial of umbilical cord derived MSCs in patients refractory to 6 months of immunosuppressive therapy.

3. China pubmed.ncbi.nlm.nih.gov

[Therapeutic effects of umbilical cord mesenchymal stem cells transplantation on systemic lupus erythematosus]. [2018] To observe the efficacy of umbilical cord mesenchymal stem cells (UC-MSCs) transplantation for the patients with refractory systemic lupus erythematosus (SLE).

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

Umbilical cord mesenchymal stem cell transplantation in severe and refractory systemic lupus erythematosus. [2022] Umbilical cord (UC)-derived mesenchymal stem cells (MSCs) have shown marked therapeutic effects in a number of diseases in animal studies, based on their potential for self-renewal and differentiation. No data are available on the effectiveness of UC MSC transplantation (MSCT) in human autoimmune disease. This study was undertaken to assess the efficacy and safety of allogeneic UC MSCT in patients with severe and treatment-refractory systemic lupus erythematosus (SLE).

5. China pubmed.ncbi.nlm.nih.gov

The regulation of the Treg/Th17 balance by mesenchymal stem cells in human systemic lupus erythematosus. [2022] Umbilical cord (UC)-derived mesenchymal stem cells (MSCs) have shown immunoregulation of various immune cells. The aim of this study was to investigate the mechanism of UC MSCs in the regulation of peripheral regulatory T cells (Treg) and T helper 17 (Th17) cells in patients with systemic lupus erythematosus (SLE).

6. Italy pubmed.ncbi.nlm.nih.gov

Long-term safety of umbilical cord mesenchymal stem cells transplantation for systemic lupus erythematosus: a 6-year follow-up study. [2022] The aim of this study is to assess the long-term safety of allogeneic umbilical cord mesenchymal stem cells (UC MSCs) transplantation for patients with refractory systemic lupus erythematosus (SLE). Nine SLE patients, who were refractory to steroid and immunosuppressive drugs treatment and underwent MSCs transplantation in 2009, were enrolled. One million allogeneic UC MSCs per kilogram of body weight were infused intravenously at days 0 and 7. The possible adverse events, including immediately after MSCs infusions, as well as the long-term safety profiles were observed. Blood and urine routine test, liver function, electrocardiogram, chest radiography and serum levels of tumor markers, including alpha fetal protein (AFP), cancer embryo antigen (CEA), carbohydrate antigen 155 (CA155) and CA199, were assayed before and 1, 2, 4 and 6 years after MSCs transplantation. All the patients completed two times of MSCs infusions. One patient had mild dizzy and warm sensation 5 min after MSCs infusion, and the symptoms disappeared quickly. No other adverse event, including fluster, headache, nausea or vomit, was observed. There was no change in peripheral white blood cell count, red blood cell count and platelet number in these patients after followed up for 6 years. Liver functional analysis showed that serum alanine aminotransferase, glutamic-oxalacetic transaminase, total bilirubin and direct bilirubin remained in normal range after MSCs infusions. No newly onset abnormality was detected on electrocardiogram and chest radiography. Moreover, we found no rise of serum tumor markers, including AFP, CEA, CA125 and CA199, before and 6 years after MSCs infusions. Our long-term observational study demonstrated a good safety profile of allogeneic UC MSCs in SLE patients.

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

hUC-MSC transplantation therapy effects on lupus-prone MRL/lpr mice at early disease stages. [2023] The efficacy of human umbilical cord mesenchymal stem cell (hUC-MSC) transplantation in treating systemic lupus erythematosus (SLE) has been confirmed by small-scale clinical trials. However, these trials focused on severe or refractory SLE, while few studies focused on mild SLE. Therefore, this study focused on the therapeutic effects of hUC-MSC transplantation in early-stage or mild MRL/lpr lupus model mice.