Stem Cell Educator Therapy for Type 1 Diabetes

Recruiting at 1 trial location

Overseen By Boris Veysman, MD

Age: Any Age

Sex: Any

Trial Phase: Phase 2 & 3

Sponsor: Throne Biotechnologies Inc.

Must not be taking: Immunosuppressants, Chemotherapy, Anticoagulants

Disqualifiers: Coronary artery disease, Active infection, Autoimmune diseases, 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

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

Compensation: Varies

Number of Visits: 2

Duration: 1 day

50 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

The trial aims to test a new treatment called Stem Cell Educator (SCE) therapy for individuals with Type 1 diabetes, a condition where the immune system attacks insulin-producing cells in the pancreas. The treatment uses a person's own blood cells, treated with special stem cells, to help the immune system stop attacking these cells. Participants will either receive SCE therapy or continue their regular insulin treatment. This trial suits those diagnosed with Type 1 diabetes within the last two years and currently managing their condition with continuous glucose monitoring. As a Phase 2 trial, the research measures the treatment's effectiveness in an initial, smaller group, offering participants a chance to contribute to important advancements in diabetes care.

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

The trial requires that you stop using immunosuppressive medications at least one month before enrollment. Other medications are not specifically mentioned, so it's best to discuss with the trial team.

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

Research has shown that Stem Cell Educator (SCE) therapy is safe for people. Studies with both humans and animals have demonstrated this. Over 200 patients have received SCE therapy, which safely corrected immune system issues and improved pancreatic function in people with Type 1 diabetes. This treatment presents no major safety concerns and avoids the ethical issues associated with some other stem cell therapies. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Stem Cell Educator Therapy is unique because it uses stem cells to reprogram the immune system, potentially addressing the root cause of Type 1 Diabetes rather than just managing symptoms. Unlike standard treatments like insulin therapy, which requires regular injections to control blood sugar levels, this therapy involves a one-time treatment that may restore the body's ability to produce insulin naturally. Researchers are excited because this approach could lead to long-term remission and significantly improve quality of life for people with Type 1 Diabetes.

What evidence suggests that Stem Cell Educator Therapy might be an effective treatment for Type 1 diabetes?

Research has shown that Stem Cell Educator (SCE) Therapy, which participants in this trial may receive, can help treat Type 1 diabetes (T1D). In earlier studies, SCE therapy showed promising results by improving immune system function. Patients who received SCE therapy demonstrated lasting improvements, such as higher C-peptide levels, indicating increased insulin production. They also had lower HbA1C levels, reflecting better long-term blood sugar control, and required less daily insulin. The therapy uses a patient's own immune cells and "teaches" them to stop attacking insulin-producing cells. This process helps the body regain its ability to produce insulin and manage blood sugar levels. ^{1 3 4 5 6}

Who Is on the Research Team?

YONG ZHAO, MD,PhD

Principal Investigator

Throne Biotechnologies Inc.

Are You a Good Fit for This Trial?

This trial is for adults and children over 14 with Type 1 Diabetes, diagnosed within the last two years. Participants must have a certain level of C-peptide, use continuous glucose monitoring, consent to study requirements including birth control for women, and not be on immunosuppressants or have other autoimmune diseases.

Inclusion Criteria

I am willing to use birth control as recommended until 6 months after treatment ends.

Your C-peptide level is higher than 0.3 ng/ml when you haven't eaten.

I am 14 years old or older.

Exclusion Criteria

Your liver enzymes (AST or ALT) are higher than a certain limit.

I am on a blood thinner that is not aspirin.

You are pregnant or breastfeeding.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive one treatment with Stem Cell Educator (SCE) therapy

1 day

1 visit (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment, including measurements of immune markers and glucose control

12 months

Visits at baseline, 1, 3, 6, 9, and 12 months

What Are the Treatments Tested in This Trial?

Interventions

• Stem Cell Educator Therapy

Trial Overview Stem Cell Educator therapy is being tested in this trial. It involves circulating a patient's blood through a device where immune cells are 'educated' by cord blood stem cells to potentially reverse autoimmunity in Type 1 Diabetes patients.

How Is the Trial Designed?

Group I: Treatment of T1D with Stem Cell Educator therapy Experimental Treatment 1 Intervention

Recruited T1D subjects will receive one treatment with SCE therapy.

Group II: Conventional insulin therapy Experimental Treatment 1 Intervention

Control group will receive conventional insulin therapy.

Stem Cell Educator Therapy is already approved in China, United States for the following indications:

Approved in China as Stem Cell Educator Therapy for:

• Type 1 Diabetes

Approved in United States as Stem Cell Educator Therapy for:

• Type 1 Diabetes

Who Is Running the Clinical Trial?

Throne Biotechnologies Inc.

Lead Sponsor

Trials

7

Recruited

340+

Hackensack Meridian Health

Collaborator

Trials

141

Recruited

42,900+

Published Research Related to This Trial

Stem Cell Educator therapy, developed using cord-blood-derived multipotent stem cells, has shown clinical safety and efficacy in treating Type 1 diabetes (T1D) through international multi-center studies over the past 10 years.

This therapy works by correcting autoimmunity and inducing immune tolerance through various mechanisms, including T-cell modulation and suppression of activated B cells, making it a promising option for restoring pancreatic β-cell function in T1D patients.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Stem Cell Educator therapy in type 1 diabetes: From the bench to clinical trials. Zhao, Y., Knight, CM., Jiang, Z., et al.[2022]

Stem cell therapy (SCT) using autologous adipose-derived insulin-secreting mesenchymal stromal cells (IS-AD-MSC) combined with bone marrow-derived hematopoietic stem cells (BM-HSC) is a safe treatment for type 1 diabetes mellitus (T1DM), showing sustained improvements in blood sugar control without adverse effects in a study of 20 patients.

The autologous SCT group demonstrated better long-term glycemic control compared to the allogenic SCT group, indicating that using a patient's own cells may be more effective for managing T1DM.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Insulin-secreting adipose-derived mesenchymal stromal cells with bone marrow-derived hematopoietic stem cells from autologous and allogenic sources for type 1 diabetes mellitus. Thakkar, UG., Trivedi, HL., Vanikar, AV., et al.[2022]

Stem cell transplantation (SCT) significantly reduces HbA1c levels and improves C-peptide levels in patients with type 1 diabetes (T1DM), based on a meta-analysis of 29 studies involving 487 patients.

SCT shows promising safety with a mortality rate of 3.4%, and the transient insulin independence rate was 9.6 per 100 person-years, indicating potential for improved metabolic control in T1DM patients, especially with specific stem cell combinations.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Efficacy and safety of stem cells transplantation in patients with type 1 diabetes mellitus-a systematic review and meta-analysis. Wu, Q., Zheng, S., Qin, Y., et al.[2021]

Stem Cell Educator™ therapy, which uses human cord-blood-derived multipotent stem cells, has shown clinical safety and efficacy in treating Type 1 diabetes (T1D) and other autoimmune diseases through immune education.

This novel approach not only targets the islet β cells but also aims to protect the islet nervous system, potentially restoring normal islet function and offering a safer alternative to traditional immune therapies.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Revisiting the Pathogenesis of Type 1 Diabetes: Importance of Neural Input to Pancreatic Islets and the Therapeutic Capability of Stem Cell Educator TM Therapy to Restore Their Integrity. Zhao, Y., Veysman, B.[2023]

The Stem Cell Educator therapy, which uses cord blood-derived multipotent stem cells to modulate the immune system, has shown promising results in reversing autoimmunity in patients with long-standing Type 1 diabetes (T1D).

In a clinical trial, a single treatment led to the regeneration of pancreatic islet β cells and improved metabolic control, highlighting a potential new approach to treating T1D that differs from conventional methods.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Stem cell educator therapy and induction of immune balance. Zhao, Y.[2021]

Human umbilical cord blood cells (HUCBCs) can differentiate into pancreatic islet β-cells and have been effective in lowering blood glucose levels in diabetic animal models, indicating their potential for diabetes treatment.

While autologous HUCBC transfusions in type 1 diabetic children have not shown benefits, 'Stem Cell Educator' therapy has demonstrated promise in achieving long-term blood glucose control in both type 1 and type 2 diabetes patients.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Human umbilical cord blood cells and diabetes mellitus: recent advances. Reddi, AS., Kothari, N., Kuppasani, K., et al.[2019]

In a study of 59 children with newly diagnosed type 1 diabetes, those with higher levels of very small embryonic-like stem cells (VSEL) and a higher VSEL/HSC ratio showed better maintenance of beta cell function, as indicated by stimulated C-peptide secretion.

Lower levels of hematopoietic stem cells (HSC) at diagnosis were associated with a greater likelihood of achieving partial remission within the first 3 to 6 months, suggesting that HSC and VSEL may play important roles in preserving beta cell function in type 1 diabetes.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Circulating Hematopoietic (HSC) and Very-Small Embryonic like (VSEL) Stem Cells in Newly Diagnosed Childhood Diabetes type 1 - Novel Parameters of Beta Cell Destruction/Regeneration Balance and Possible Prognostic Factors of Future Disease Course. Jamiołkowska-Sztabkowska, M., Grubczak, K., Starosz, A., et al.[2022]

Stem cell therapy shows promise for treating type 1 diabetes by potentially regenerating beta cells and preserving insulin production, with clinical trials ongoing since 2003 in various countries.

The Diabetes Control and Complications Trial (DCCT) highlighted that higher C-peptide levels, which indicate better insulin secretion, are associated with a lower risk of chronic complications in type 1 diabetes, emphasizing the importance of beta cell preservation in treatment strategies.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Stem cell therapy for type 1 diabetes mellitus: a review of recent clinical trials. Couri, CE., Voltarelli, JC.[2021]

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT03390231

NCT03390231 | Stem Cell Educator Therapy in Diabetes In this project, the optimized SCE therapy for type 1 diabetes (T1D) and T2D will be tested in a prospective, single-arm, open-label, single-center study to ...

2. clinicaltrials.gov clinicaltrials.gov/study/NCT01350219

Stem Cell Educator Therapy in Type 1 Diabetes Here, the investigators develop a novel Stem Cell Educator therapy by using CB-SC and explore the therapeutic effectiveness of Educator therapy in T1D patients.

3. thronebio.com thronebio.com/sce-therapy

Stem Cell Educator Therapy In an open-label, Phase 1 and Phase 2 study, 15 individuals with T1D received one treatment of Educator Therapy. Their median age was 29 years (range, 15 to 41) ...

4. thejdca.org thejdca.org/publications/report-library/archived-reports/2020-reports/practical-cure-update-stem-cell-educator.html

Practical Cure Update: Stem Cell Educator : 2020 Reports The therapy attempts to stop the T1D autoimmune attack by modifying a person's white blood cells, so they no longer attack insulin-producing beta cells.

5. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/35108619/

Stem Cell Educator therapy in type 1 diabetes Educator therapy is the leading immunotherapy to date to safely and efficiently correct autoimmunity and restore β cell function in T1D patients.

6. clinicaltrials.gov clinicaltrials.gov/study/NCT03390231?term=stem%20cell%20diabetes&viewType=Table&rank=7

NCT03390231 | Stem Cell Educator Therapy in Diabetes Several mechanistic studies with clinical samples and animal models have demonstrated the proof of concept and clinical safety of SCE therapy. They suggest SCE ...