ECP + MSC Infusion for Graft-versus-Host Disease

Overseen By Molly Gallogly, MD, PhD

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: Molly Gallogly

Must be taking: Steroids

Disqualifiers: Active malignancy, Uncontrolled illness, Pregnancy, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

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

Compensation: Varies

Number of Visits: 2

Duration: 4 weeks

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests two treatments—extracorporeal photopheresis (a procedure that treats blood outside the body) and Mesenchymal Stromal Cell (MSC) infusion (a type of cell therapy)—to determine if they can safely work together to ease symptoms of Graft versus Host Disease (GvHD). GvHD may occur after a stem cell transplant when the new cells attack the body. This trial targets individuals with a severe form of GvHD that does not improve with current treatments, particularly those with skin problems or issues affecting the stomach, intestines, or liver. As a Phase 2 trial, the research focuses on measuring the treatment's effectiveness in an initial, smaller group of participants.

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

The trial information does not specify if you need to stop taking your current medications. It's best to discuss this with the trial coordinators or your doctor.

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

Previous studies have shown that Extracorporeal Photopheresis (ECP) is safe for treating Graft-versus-Host Disease (GvHD). Research indicates that patients tolerate ECP well and respond positively to the treatment. Additionally, Mesenchymal Stromal Cells (MSCs) have been used in GvHD treatments and are generally considered safe. Studies suggest that MSCs may improve symptoms without causing severe side effects. Although both treatments have been studied separately, they have demonstrated promising safety profiles. ^{1 2 3 4 5}

Why are researchers excited about this trial's treatments?

Unlike the standard treatments for graft-versus-host disease, which often rely on immunosuppressive drugs, the combination of allogeneic mesenchymal stromal cells (MSCs) and extracorporeal photopheresis (ECP) offers a unique approach. MSCs are derived from donor stem cells and have the potential to modulate the immune system, reducing harmful immune responses while promoting healing. ECP, on the other hand, works by treating the patient's blood with ultraviolet light to help control immune activity. Researchers are excited about this treatment because it targets the condition in a more holistic way, potentially offering faster relief and fewer side effects compared to traditional therapies.

What evidence suggests that this trial's treatments could be effective for Graft-versus-Host Disease?

Research has shown that both donor-derived Mesenchymal Stromal Cells (MSCs) and Extracorporeal Photopheresis (ECP) hold promise for treating Graft-versus-Host Disease (GvHD). Studies have found that MSCs can improve survival rates, with 47% of patients living six months after treatment, suggesting MSCs might help control harmful immune reactions following stem cell transplants. ECP, a well-known treatment for long-term GvHD, has also proven effective in sudden cases, with 57% of patients fully recovering. In this trial, participants will receive a combination of MSCs and ECP, which may work well together to manage GvHD symptoms. ^{3 4 6 7 8}

Who Is on the Research Team?

Molly Gallogly, MD, PhD

Principal Investigator

University Hospitals Cleveland Medical Center, Case Comprehensive Cancer Center

Are You a Good Fit for This Trial?

This trial is for people with severe Graft versus Host Disease (GvHD) after a stem cell transplant. They must have specific stages of skin, liver, or gastrointestinal involvement and may be those not responding to standard steroid treatments. Participants need stable vital signs, adequate blood counts, and suitable veins for the treatment process.

Inclusion Criteria

Hct > 27 and plts > 50,000 x10^9/L

I have received a stem cell transplant from a donor.

Participants must have the ability to understand and the willingness to sign a written informed consent document

Exclusion Criteria

My cancer is getting worse or I have a disease after a transplant.

I currently have cancer.

I cannot undergo photopheresis due to health reasons.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive a combination of Extracorporeal Photopheresis (ECP) and Mesenchymal Stem Cell (MSC) infusion for a single 28-day cycle

4 weeks

2-3 visits per week for ECP, 1-3 visits for MSC infusion

Follow-up

Participants are monitored for safety and effectiveness after treatment

1 year

What Are the Treatments Tested in This Trial?

Interventions

• Allogeneic Mesenchymal Stromal Cells (MSCs)
• Extracorporeal Photopheresis (ECP)

Trial Overview The study tests if combining two therapies—extracorporeal photopheresis (ECP) and Mesenchymal Stromal Cell (MSC) infusion—is safe and effective in treating symptoms of GvHD. ECP involves treating the blood with light after adding a drug that makes affected cells more sensitive to light; MSCs are infused to potentially repair tissue damage.

How Is the Trial Designed?

Group I: MSCs + ECP Experimental Treatment 2 Interventions

The treatment period consists of a single, 28-day cycle. Participants will be treated with ECP 2 to 3 times per week per the discretion of the treating physician. Participants will receive IV infusions of MSCs on days 1 (+ 2 days) and 8 (+/- 2 days). A third dose may be given on day 15 (+/- 2 days) if the principal investigator (PI) and treating physician determine the MSC infusions have benefited the participant. Participants will be followed for up to 1 year for assessment of endpoints.

Who Is Running the Clinical Trial?

Molly Gallogly

Lead Sponsor

Trials

2

Recruited

70+

Citations

1. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/37990219/

Real-world data suggest effectiveness of the allogeneic ... A recent real-world analysis of outcomes for 64 adult RR-aGvHD patients not treated with MSCs reports survival of 20%, 16% and 10% beyond 6, ...

2. translational-medicine.biomedcentral.com translational-medicine.biomedcentral.com/articles/10.1186/s12967-023-04731-1

Real-world data suggest effectiveness of the allogeneic ... A recent real-world analysis of outcomes for 64 adult RR-aGvHD patients not treated with MSCs reports survival of 20%, 16% and 10% beyond 6, 12 and 24 months, ...

3. ashpublications.org ashpublications.org/blood/article/142/Supplement%201/4927/504266/Real-World-Data-Suggest-Effectiveness-of

Real-World Data Suggest Effectiveness of Allogeneic ... Survival: Overall survival at 6, 12, and 24 months was 47% (38-56%), 35% (27-44%), and 29% (21-38%) for adults, and 59% (40-74%), 42% (24-58%), ...

4. nature.com nature.com/articles/s41423-023-01022-z

Current perspectives on mesenchymal stromal cell therapy ... The most commonly studied outcome is a reduction in unfavorable immune reactions after hematopoietic stem cell transplantation (HSCT), so-called ...

5. sciencedirect.com sciencedirect.com/science/article/pii/S000649712311528X

Real-World Data Suggest Effectiveness of Allogeneic ... We assessed real-world outcomes of patients with RR-aGvHD treated with the random-donor allogeneic MSC preparation “MSC-FFM,” available via Hospital Exemption ...

6. sciencedirect.com sciencedirect.com/science/article/pii/S1465324925006280

Targeted Delivery of Mesenchymal Stromal Cells (MSCs) ... This case highlights the feasibility and potential therapeutic advantage of IA MSC administration for severe GI GvHD.

7. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC8796067/

Efficacy and safety of mesenchymal stem cells treatment for ... Conclusion: MSCs treatment may be safe and effective for MDR-GVHD after HID HSCT. Keywords: graft-versus-host disease, ...

8. nature.com nature.com/articles/s41591-024-02990-z

Two-year safety outcomes of iPS cell-derived ... We now report results at the 2-year follow-up: 9 of 15 (60%) participants survived, which compares favorably with previously reported outcomes ...