Stem Cell Therapy for Type 1 Diabetes

Overseen By Leah Benn, MPH

Age: 18 - 65

Sex: Any

Trial Phase: Phase 1

Sponsor: Medical University of South Carolina

Disqualifiers: Retinopathy, Malignancy, High blood pressure, others

Approved in 3 Jurisdictions This treatment is already approved in other countries

Compensation: Varies

Number of Visits: 6

Duration: 1 day

60 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new treatment using stem cells to assist individuals with type 1 diabetes, a condition where the body cannot regulate blood sugar independently. Researchers aim to determine if these special cells, derived from umbilical cords, can safely enhance the body's ability to manage blood sugar. The trial includes two groups: one receiving the stem cells (Mesenchymal Stem Cells or MSCs) and the other receiving a placebo (inactive substance). Individuals diagnosed with type 1 diabetes within the last six months and exhibiting certain diabetes-related signs may be suitable for this study. As a Phase 1 trial, this research focuses on understanding the treatment's effects in people, offering participants the chance to be among the first to receive this innovative therapy.

Do I have to stop taking my current medications for this trial?

The trial protocol does not specify whether you need to stop taking your current medications. However, you must be willing to comply with 'intensive diabetes management' as directed by your clinician.

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

Research has shown that mesenchymal stem cells (MSCs), like those tested in this study, are generally safe for people with diabetes. In a recent study, MSCs from Wharton's jelly, part of the umbilical cord, proved safe for individuals with new-onset type 1 diabetes. Another study on type 2 diabetes patients confirmed that MSCs from human umbilical cords were well-tolerated and did not cause major safety issues.

A larger review of different studies also found that using umbilical cord MSCs is generally safe for managing diabetes. Although this current trial is in its early stages, previous research on similar treatments has shown promise regarding safety. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for type 1 diabetes?

Researchers are excited about mesenchymal stem cell (MSC) therapy for Type 1 diabetes because it offers a novel approach compared to traditional treatments like insulin therapy. Unlike insulin, which manages blood sugar levels, MSCs have the potential to modulate the immune system and promote the regeneration of insulin-producing cells. This could address the root cause of the disease rather than just its symptoms. Additionally, MSCs are administered intravenously, which allows them to travel throughout the body and target areas of inflammation and tissue damage. This innovative mechanism could lead to long-term improvements in managing Type 1 diabetes.

What evidence suggests that mesenchymal stem cells might be an effective treatment for type 1 diabetes?

Research has shown that certain stem cells, such as the Mesenchymal Stem Cells (MSCs) used in this trial, may help treat type 1 diabetes (T1D). Participants may receive MSCs, which can protect the insulin-producing cells in the pancreas, crucial for managing T1D. Some studies found that this therapy can improve blood sugar control and reduce insulin needs in people with diabetes. While many studies showed positive results, a few did not observe significant changes. Overall, the evidence suggests that these stem cells could be a helpful treatment for T1D. ^{3 6 7 8 9}

Who Is on the Research Team?

Hongjun N Wang, PhD

Principal Investigator

Medical University of South Carolina

Are You a Good Fit for This Trial?

This trial is for individuals aged 18-30 recently diagnosed with Type 1 Diabetes, having some remaining insulin-producing cell function and positive for specific diabetes-related autoantibodies. They must be mentally stable, able to manage their diabetes intensively, and have normal blood counts and liver function. Excluded are those with severe infections, certain eye diseases, extreme body weights, high blood pressure or lipids, pregnancy or intent to become pregnant during the study.

Inclusion Criteria

I am willing to follow a strict diabetes management plan.

Before the study starts, patients must have some remaining ability for their pancreas to produce insulin.

I am mentally stable and can follow the study's procedures.

Exclusion Criteria

My blood sugar levels are very high or I often have very low blood sugar.

I am currently being treated for a severe infection.

You have signs of retinopathy in your eyes.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive an infusion of 2.5 x 10^6 MSC per kg or placebo on Day 1

1 day

1 visit (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment, with assessments at 0, 1, 3, 6, and 12 months

12 months

5 visits (in-person)

Long-term monitoring

Participants may continue to be monitored for long-term safety and efficacy

Additional 6 months

What Are the Treatments Tested in This Trial?

Interventions

• Mesenchymal Stem Cells (MSCs)
• Placebo Infusion (Plasmalyte A with 0.5% human serum albumin)

Trial Overview

The trial is testing the safety and effectiveness of using umbilical cord-derived mesenchymal stem cells (UC-MSCs) compared to a placebo in treating new-onset Type 1 Diabetes. The aim is to see if these cells can help manage the disease by preserving insulin production.

How Is the Trial Designed?

Group I: Group A Treatment Experimental Treatment 1 Intervention

2.5 x 10\^6 MSC per kg will be infused intravenously on Day 1

Group II: Group B Placebo Placebo Group 1 Intervention

Plasmalyte with 0.5% Human Serum Albumin will be infused intravenously on Day 1

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

Approved in China as Mesenchymal Stem Cells for:

• Type 2 Diabetes Mellitus

Approved in United States as Umbilical Cord-Derived Mesenchymal Stem Cells (UC-MSCs) for:

• Clinical Trials for Type 1 Diabetes Mellitus and Type 2 Diabetes Mellitus

Approved in European Union as Mesenchymal Stem Cells for:

• Clinical Trials for Type 1 Diabetes Mellitus and Type 2 Diabetes Mellitus

Who Is Running the Clinical Trial?

Medical University of South Carolina

Lead Sponsor

Trials

994

Recruited

7,408,000+

National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)

Collaborator

Trials

2,513

Recruited

4,366,000+

Published Research Related to This Trial

In a study involving 29 patients with established type 1 diabetes, those who received umbilical cord mesenchymal stromal cell (MSC) and autologous bone marrow mononuclear cell (aBM-MNC) transplantation showed significantly lower rates of chronic complications like peripheral neuropathy (7.1% vs. 46.7%) and diabetic nephropathy (7.1% vs. 40.0%) compared to the control group after 8 years.

The co-transplantation treatment was associated with a lower overall incidence of complications, with only 14.3% of patients in the SCT group developing any complications compared to 73.3% in the control group, indicating a potential long-term safety and efficacy of this stem cell therapy.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Prevention of chronic diabetic complications in type 1 diabetes by co-transplantation of umbilical cord mesenchymal stromal cells and autologous bone marrow: a pilot randomized controlled open-label clinical study with 8-year follow-up. Wu, Z., Xu, X., Cai, J., et al.[2022]

The transplantation of umbilical cord mesenchymal stromal cells (UC-MSC) combined with autologous bone marrow mononuclear cells (aBM-MNC) in 42 patients with established type 1 diabetes (T1D) was found to be safe and well-tolerated, showing no significant adverse effects over the one-year follow-up period.

Patients receiving the stem cell treatment experienced significant improvements in insulin secretion and metabolic control, including a 105.7% increase in C-peptide levels and a 12.6% reduction in HbA1c, while control subjects showed declines in these measures.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Umbilical Cord Mesenchymal Stromal Cell With Autologous Bone Marrow Cell Transplantation in Established Type 1 Diabetes: A Pilot Randomized Controlled Open-Label Clinical Study to Assess Safety and Impact on Insulin Secretion. Cai, J., Wu, Z., Xu, X., et al.[2022]

Human umbilical cord blood-derived mesenchymal stem cells (UCB-MSCs) can be successfully induced to differentiate into insulin-producing cells (IPCs), which were shown to produce human insulin in diabetic mice after transplantation.

While IPCs increased serum human insulin levels in the treated mice, the overall regulation of blood glucose and body weight remained ineffective, indicating that further research is needed to enhance their therapeutic potential in diabetes management.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

A secretory function of human insulin-producing cells in vivo. Hu, YH., Wu, DQ., Gao, F., et al.[2018]

Adipose Tissue-derived Mesenchymal Stem Cells (AT-MSCs) significantly enhance insulin secretion from transplanted islets and improve blood glucose levels in diabetic mice, demonstrating their potential to improve outcomes in islet transplantation.

The study shows that AT-MSCs help reduce immune cell infiltration and promote a more favorable immune environment by increasing regulatory cytokines and Treg populations, suggesting they could protect transplanted islets from rejection.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Immunomodulatory and protective effects of adipose tissue-derived mesenchymal stem cells in an allograft islet composite transplantation for experimental autoimmune type 1 diabetes. Mohammadi Ayenehdeh, J., Niknam, B., Rasouli, S., et al.[2018]

Transplantation of umbilical cord-derived mesenchymal stem cells (UCMSCs) in six patients with type 2 diabetes (T2D) showed significant improvements in β-cell function, as indicated by increased fasting C-peptide levels and sustained improvements over a 24-month follow-up period.

Three out of six patients became insulin-free for periods ranging from 25 to 43 months after UCMSC transplantation, demonstrating the potential efficacy of this treatment approach without any observed safety issues during the follow-up.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Therapeutic efficacy of umbilical cord-derived mesenchymal stem cells in patients with type 2 diabetes. Guan, LX., Guan, H., Li, HB., et al.[2020]

Human umbilical cord mesenchymal stromal cells (hUC-MSCs) can be effectively differentiated into insulin-producing islet-like cell clusters using in vitro-synthesized PDX1 mRNA, showing potential for diabetes treatment.

The differentiated cells not only expressed key pancreatic transcription factors and insulin but also demonstrated functional insulin secretion in response to glucose, indicating their capability to mimic pancreatic islet function.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Reprogramming human umbilical cord mesenchymal stromal cells to islet-like cells with the use of in vitro-synthesized pancreatic-duodenal homebox 1 messenger RNA. Wang, XL., Hu, P., Guo, XR., et al.[2021]

Umbilical cord-derived mesenchymal stem cells (UCMSCs) can be successfully differentiated into functional islet-like cells in vitro, showing potential for regenerating insulin-producing cells in Type 1 Diabetes Mellitus (T1DM).

These differentiated cells express key pancreatic markers and release insulin in response to glucose, indicating their functionality and suggesting that UCMSCs could provide a reliable source for cell therapy in diabetes treatment.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Umbilical Cord Derived Mesenchymal Stem Cells Useful in Insulin Production - Another Opportunity in Cell Therapy. Sarang, S., Viswanathan, C.[2020]

In a study involving 53 participants with type 1 diabetes, those receiving repeated infusions of allogeneic mesenchymal stromal cells (MSCs) showed a 40.7% rate of clinical remission, significantly higher than the control group receiving standard insulin therapy.

The MSC treatment was found to be safe, with no severe side effects reported, and it demonstrated potential for better preservation of β cell function, particularly in adult-onset type 1 diabetes patients.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

One repeated transplantation of allogeneic umbilical cord mesenchymal stromal cells in type 1 diabetes: an open parallel controlled clinical study. Lu, J., Shen, SM., Ling, Q., et al.[2022]

Wharton's jelly mesenchymal stem cells (WJ-MSCs) showed superior ability to differentiate into insulin-producing cells compared to umbilical cord blood mesenchymal stem cells (UCB-MSCs) in laboratory tests.

In a study involving diabetic rats, WJ-MSCs were more effective at controlling blood glucose levels after transplantation, suggesting they could be a better option for diabetes cell therapy.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

A comparison of Wharton's jelly and cord blood as a source of mesenchymal stem cells for diabetes cell therapy. El-Demerdash, RF., Hammad, LN., Kamal, MM., et al.[2018]

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