Stem Cell Therapy for Type 1 Diabetes

Overseen By Leah Benn, MPH

Age: 18 - 65

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 1

Recruiting

Sponsor: Medical University of South Carolina

Disqualifiers: Retinopathy, Malignancy, High blood pressure, others

Approved in 3 Jurisdictions

Trial Summary

What is the purpose of this trial?

This trial is testing if special cells from umbilical cords can help young adults with Type 1 diabetes by calming their immune system and reducing inflammation. These cells pose a lesser risk of viral contamination due to low placental transmission during prenatal life.

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.

What data supports the idea that Stem Cell Therapy for Type 1 Diabetes is an effective treatment?

The available research shows that using umbilical cord-derived mesenchymal stem cells (MSCs) can help in treating Type 1 Diabetes by potentially regenerating insulin-producing cells. One study mentions that these stem cells might help prevent further damage to the cells that produce insulin. Another study highlights that combining these stem cells with cells from a patient's own bone marrow could help manage diabetes over a long period, as seen in an 8-year follow-up. This suggests that stem cell therapy could be a promising treatment option for Type 1 Diabetes. ^{1 2 3 4 5}

What safety data is available for stem cell therapy in Type 1 Diabetes?

The safety of umbilical cord-derived mesenchymal stem cells (UC-MSCs) has been evaluated in clinical trials. One study focused on the safety and efficacy of allogeneic MSC transplantation in Type 1 Diabetes, indicating that MSCs are promising for immune disorders due to their immunoregulatory properties. Another study on Type 2 Diabetes patients showed that UC-MSC transplantation was safe over a 24-month follow-up period, suggesting potential safety in similar applications for Type 1 Diabetes. ^{1 2 6 7 8}

Is the treatment Mesenchymal Stem Cells (MSCs) promising for Type 1 Diabetes?

Yes, Mesenchymal Stem Cells (MSCs) show promise for treating Type 1 Diabetes. They can help regenerate insulin-producing cells and protect existing ones, potentially improving insulin production and reducing complications. ^{1 2 3 4 9}

Research Team

Hongjun N Wang, PhD

Principal Investigator

Medical University of South Carolina

Eligibility Criteria

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.

Trial Timeline

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

Treatment Details

Interventions

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

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.

Participant Groups

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 for the following indications:

Approved in China as Mesenchymal Stem Cells for:

• 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+

Findings from Research

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]

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]

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]

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]

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]

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]

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]

References

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

Umbilical Cord Derived Mesenchymal Stem Cells Useful in Insulin Production - Another Opportunity in Cell Therapy. [2020]

2. England 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. [2018]

3. England 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. [2022]

4. United States 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. [2022]

5. Singapore pubmed.ncbi.nlm.nih.gov

A secretory function of human insulin-producing cells in vivo. [2018]

6. England 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. [2021]

7. Greece pubmed.ncbi.nlm.nih.gov

Therapeutic efficacy of umbilical cord-derived mesenchymal stem cells in patients with type 2 diabetes. [2020]

8. England 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. [2022]

9. Netherlands 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. [2018]