Stem Cell Therapy for Ulcerative Colitis

Overseen By Jessica Friton

Age: 18+

Sex: Any

Trial Phase: Phase 1

Sponsor: Mayo Clinic

Must be taking: 5-ASA, Thiopurines, Anti-TNF, others

Must not be taking: Investigational drugs

Disqualifiers: Cancer, Hepatitis, HIV, others

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

Compensation: Varies

Number of Visits: 2

Duration: 1 day

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new treatment using stem cells from a person’s own fat to determine if it can safely aid those with ulcerative colitis, a condition causing painful colon inflammation and frequent discomfort. The trial targets individuals whose symptoms have not improved with at least two standard treatments. Those with ongoing moderate to severe ulcerative colitis, despite trying different medications, might be suitable candidates for this study. As a Phase 1 trial, the research focuses on understanding how the treatment works in people, offering participants the opportunity to be among the first to receive this innovative therapy.

Will I have to stop taking my current medications?

The trial allows participants to continue taking certain medications like corticosteroids, 5-ASA drugs, thiopurines, MTX, antibiotics, anti-TNF, and anti-integrin therapy. However, if you are on an investigational drug, you must stop it at least 30 days before starting the trial.

Is there any evidence suggesting that this treatment is likely to be safe for humans?

Research has shown that using stem cells from fat tissue is generally safe for treatments. One study found that patients with mild inflammation tolerated these stem cells well, with no major safety issues. Another review suggests that this therapy is safe and effective for adults with inflammatory bowel diseases, such as Crohn's disease.

However, some research indicates that stem cells from patients with inflammatory or autoimmune conditions might not effectively calm the immune system. Despite this, current evidence suggests the treatment is safe overall. As a Phase 1 trial, the main goal is to ensure the treatment's safety for humans. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Unlike the standard treatments for ulcerative colitis, which often include anti-inflammatory drugs and immunosuppressants, this new approach uses autologous mesenchymal stem cells derived from a patient's own adipose (fat) tissue. Researchers are excited because these stem cells have the potential to regenerate damaged tissue and modulate the immune response, addressing the root cause of the disease rather than just alleviating symptoms. Additionally, the innovative delivery method via intra-arterial delivery directly to the inferior mesenteric artery offers targeted treatment, which could enhance effectiveness and reduce side effects.

What evidence suggests that this treatment might be an effective treatment for ulcerative colitis?

Research has shown that stem cells taken from fat tissue, known as adipose-derived mesenchymal stem cells (AD-MSCs), might help treat ulcerative colitis by calming the immune system. Studies have found that these stem cells can reduce gut inflammation, a major issue for people with ulcerative colitis. One study showed that a similar stem cell treatment healed abnormal connections between organs, called fistulas, in 82% of patients. Another review found that stem cell therapy improved outcomes for people with inflammatory bowel disease (IBD), which includes ulcerative colitis. These early findings suggest that AD-MSCs, which participants in this trial will receive, could be a promising option for managing ulcerative colitis. ^{1 2 3 6 7}

Who Is on the Research Team?

William Faubion, MD

Principal Investigator

Mayo Clinic

Are You a Good Fit for This Trial?

This trial is for men and women aged 18-65 with moderate to severe ulcerative colitis that hasn't improved after trying at least two FDA-approved treatments. Participants must have a stable hemoglobin level, normal blood clotting, and agree to use contraception. Excluded are those with certain medical conditions like vasculitis, cancer history (except localized skin cancers), recent investigational drug use, partial colectomy, or significant allergies.

Inclusion Criteria

Ability to comply with protocol

I have tried at least 2 standard treatments for UC without success.

Hemoglobin must be greater than 8

Exclusion Criteria

Pregnant patients or trying to become pregnant or breastfeeding

Inability to give informed consent

I have had a C. Diff infection in the last 30 days.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive intra-arterial delivery of 15 million or 30 million adipose-derived mesenchymal stem cells

1 day

1 visit (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment

24 months

Visits on Day 1, Week 1, Week 2, Week 8, Week 24, Week 52, and Week 104

What Are the Treatments Tested in This Trial?

Interventions

• Adipose derived, autologous mesenchymal stem cells

Trial Overview The study is testing the safety and effectiveness of using adipose-derived mesenchymal stem cells (cells from one's own body fat) in treating ulcerative colitis. Researchers will deliver these cells through an angiographic procedure to see if they can help heal ulcers in the colon.

How Is the Trial Designed?

Group I: Autologous mesenchymal stem cells Experimental Treatment 1 Intervention

Adipose derived, autologous mesenchymal stem cells (AD-MSCs) at a dose of 15 million or 30 million cells will be administered via intra-arterial delivery with interventional radiology to the inferior mesenteric artery in subjects with medically refractory ulcerative colitis.

Who Is Running the Clinical Trial?

Mayo Clinic

Lead Sponsor

Trials

3,427

Recruited

3,221,000+

Citations

1. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC8484792/

The Therapeutic Efficacy of Adipose Tissue-Derived ... Adipose-derived MSC injection for 8 weeks led to complete healing of fistula in 82% patients (27/33) (Lee et al., 2013). In another study, bone ...

2. frontiersin.org frontiersin.org/journals/cell-and-developmental-biology/articles/10.3389/fcell.2022.822772/full

Adipose-Derived Stem Cells From Patients With Ulcerative ... In this study, we examined the ADSCs from UC patients (P-ADSCs) and healthy individuals (H-ADSCs) in terms of phenotype, colony formation, ...

3. sciencedirect.com sciencedirect.com/science/article/pii/S1465324925001707

SAFETY AND EFFICACY OF MESENCHYMAL STEM ... This review suggests that MSC therapy is safe and effective in improving clinical outcomes for adults with IBD, particularly in those with Crohn's disease- ...

4. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC8894714/

Adipose-Derived Stem Cells From Patients With Ulcerative ... Adipose-derived stem cells (ADSCs) are able to modulate the immune response and are used for treating ulcerative colitis (UC).

5. stemcellres.biomedcentral.com stemcellres.biomedcentral.com/articles/10.1186/s13287-025-04549-2

Multi-function of adipose-derived stem cells on gut disorder ADSCs effectively treat gastrointestinal diseases like IBD and colorectal cancer, and show potential for liver fibrosis and gastric cancer. ADSC ...

6. trialsjournal.biomedcentral.com trialsjournal.biomedcentral.com/articles/10.1186/s13063-024-08128-3

Safety and efficacy of autologous adipose tissue-derived stem ... The transplantation of AD-MSCs in aging-related low-grade inflammation patients was safe. Two doses of cell infusion reduced the patient's ...

7. sciencedirect.com sciencedirect.com/science/article/abs/pii/S1090023315003275

Safety and efficacy of allogeneic adipose tissue-derived ... The aim of this study was to determine the safety and feasibility of adipose tissue-derived MSC (ASC) therapy in dogs with inflammatory bowel disease (IBD).