IMS001 for Multiple Sclerosis

Recruiting at 2 trial locations

Overseen By Richard Kim, MD

Age: 18+

Sex: Any

Trial Phase: Phase 1

Sponsor: ImStem Biotechnology

Disqualifiers: Autoimmune disease, Myelodysplasia, Hematologic, others

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

Approved in 1 Jurisdiction This treatment is already approved in other countries

Compensation: Varies

Number of Visits: 1

Duration: 1 day

30 Participants Needed

Trial Summary

What is the purpose of this trial?

This trial tests IMS001, a treatment made from special cells, on Multiple Sclerosis patients who haven't had success with other treatments. These cells might help control or slow down the disease.

Will I have to stop taking my current medications?

The trial information does not specify if you need to stop taking your current medications. However, it mentions a history of excluded medications prior to Day 1, which suggests some medications might need to be stopped. It's best to discuss your specific medications with the trial team.

What data supports the effectiveness of the treatment IMS001 for Multiple Sclerosis?

Research shows that mesenchymal stem cells (MSCs) derived from human embryonic stem cells (hES-MSCs) can significantly reduce symptoms and prevent nerve damage in a mouse model of multiple sclerosis. These hES-MSCs are more effective than those from adult sources, suggesting they may be a promising treatment for multiple sclerosis. ^{1 2 3 4 5}

How is the treatment IMS001 different from other treatments for multiple sclerosis?

IMS001 is unique because it uses mesenchymal stem cells derived from human embryonic stem cells (hES-MSCs), which offer a stable and unlimited supply, and have shown greater effectiveness in reducing symptoms and preventing nerve damage in multiple sclerosis compared to traditional bone marrow-derived stem cells. ^{1 3 4 6 7}

Research Team

Richard Kim, MD

Principal Investigator

ImStem Biotechnology

Eligibility Criteria

This trial is for adults aged 18-65 with Multiple Sclerosis who haven't had success with standard treatments. Participants must be able to have MRIs, not have other autoimmune or blood diseases, no recent serious infections, and should not be pregnant. They also need to be free from certain medications and conditions that could affect the study.

Inclusion Criteria

Provides signed and dated informed consent in accordance with local regulations

I have been diagnosed with multiple sclerosis (MS).

I am between 18 and 65 years old.

Exclusion Criteria

Women pregnant, breast feeding, or planning to become pregnant during the study

My body weight is 120 kg or more.

I have a history of autoimmune, myelodysplasia, or blood diseases.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive a single dose of IV IMS001, with optional additional dose at Month 6

1 day

1 visit (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment

60 months

Treatment Details

Interventions

• IMS001

Trial Overview IMS001, a therapy derived from human embryonic stem cells designed to potentially alter the course of Multiple Sclerosis, is being tested. This Phase 1 trial involves giving a single dose to participants who didn’t respond well to existing disease-modifying treatments.

Participant Groups

Group I: Optional Dose Experimental Treatment 1 Intervention

High dose of cells/kg of intravenous (IV) IMS001 as a single dose infusion on Day 1 and at Month 6.

Group II: Low Dose Experimental Treatment 1 Intervention

Low dose of cells/kg of intravenous (IV) IMS001 as a single dose infusion on Day 1.

Group III: High Dose Experimental Treatment 1 Intervention

High dose of cells/kg of intravenous (IV) IMS001 as a single dose infusion on Day 1.

IMS001 is already approved in United States for the following indications:

Approved in United States as IMS001 for:

• Multiple Sclerosis (under investigation)

Who Is Running the Clinical Trial?

ImStem Biotechnology

Lead Sponsor

Trials

1

Recruited

30+

Rho, Inc.

Industry Sponsor

Trials

25

Recruited

5,800+

Findings from Research

Mesenchymal stem cells have shown promising immunomodulatory and immunosuppressive effects in experimental models of multiple sclerosis, suggesting they could help manage the disease's inflammatory nature.

Clinical trials indicate that mesenchymal stem cell therapy is safe and well-tolerated in multiple sclerosis patients, with some evidence of reducing proinflammatory responses and improving MRI inflammatory markers in the short term.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Mesenchymal stem cells in multiple sclerosis - translation to clinical trials. Dulamea, A.[2022]

Haematopoietic stem cell transplantation (HSCT) has shown promising results in treating patients with poor-prognosis autoimmune diseases like multiple sclerosis (MS), with over 600 patients treated worldwide, particularly benefiting those in the relapsing-remitting phase.

An interdisciplinary group of experts is planning a controlled clinical trial to rigorously assess the efficacy of HSCT for highly active MS, aiming to standardize patient selection and outcome measures for future studies.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

A prospective, randomized, controlled trial of autologous haematopoietic stem cell transplantation for aggressive multiple sclerosis: a position paper. Saccardi, R., Freedman, MS., Sormani, MP., et al.[2022]

A novel method to produce mesenchymal stem/stromal cells (T-MSCs) from human embryonic stem cells (hESCs) in just 11-16 days has been developed, showing potential for rapid clinical application.

T-MSCs exhibit strong immunomodulatory effects, effectively inhibiting T and B lymphocyte proliferation and demonstrating significant efficacy in animal models of autoimmune diseases, making them a promising candidate for MSC-based therapies.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Immune modulatory mesenchymal stem cells derived from human embryonic stem cells through a trophoblast-like stage. Wang, X., Lazorchak, AS., Song, L., et al.[2018]

Human embryonic stem cell-derived mesenchymal stem cells (hES-MSCs) significantly reduce clinical symptoms and prevent neuronal demyelination in a mouse model of multiple sclerosis, showing greater efficacy than bone marrow-derived MSCs.

The enhanced therapeutic effects of hES-MSCs may be linked to their ability to migrate into inflamed central nervous system tissues and lower levels of IL-6 expression compared to BM-MSCs, which could hinder their effectiveness.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Human ESC-derived MSCs outperform bone marrow MSCs in the treatment of an EAE model of multiple sclerosis. Wang, X., Kimbrel, EA., Ijichi, K., et al.[2021]

Mesenchymal stem cell (MSC) transplantation shows promise in treating various autoimmune diseases, with a total clinical effective rate of 54% reported for rheumatoid arthritis (RA), where it may reduce disease activity and improve clinical symptoms.

In systemic lupus erythematosus (SLE), MSC transplantation significantly improved disease activity scores and markers like urine protein and complement C3, indicating its potential efficacy, while no serious adverse events were reported across the studies reviewed.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Efficacy and Safety of Mesenchymal Stem Cell Transplantation in the Treatment of Autoimmune Diseases (Rheumatoid Arthritis, Systemic Lupus Erythematosus, Inflammatory Bowel Disease, Multiple Sclerosis, and Ankylosing Spondylitis): A Systematic Review and Meta-Analysis of Randomized Controlled Trial. Zeng, L., Yu, G., Yang, K., et al.[2022]

The MESEMS study, involving 144 patients with active multiple sclerosis, found that intravenous infusion of autologous bone marrow-derived mesenchymal stem cells (MSCs) was safe and well tolerated, with no serious adverse events related to the treatment.

However, MSC treatment did not show a significant effect on reducing gadolinium-enhancing lesions, a marker of inflammation in multiple sclerosis, indicating that this approach may not be effective for treating active forms of the disease.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Safety, tolerability, and activity of mesenchymal stem cells versus placebo in multiple sclerosis (MESEMS): a phase 2, randomised, double-blind crossover trial. Uccelli, A., Laroni, A., Ali, R., et al.[2022]

Mesenchymal stem cells (MSC) showed a stronger ability to suppress the proliferation of myelin-induced memory T cells compared to PHA-stimulated T cells, suggesting their potential effectiveness in treating multiple sclerosis (MS).

The degree of MSC suppression was inversely correlated with patients' Expanded Disability Status Scale (EDSS) scores, indicating that measuring MSC suppression and CD119 expression in T-lymphocytes could help predict the immunosuppressive potential of MSC therapy in MS patients.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

In vitro assessment of mesenchymal stem cells immunosuppressive potential in multiple sclerosis patients. Zafranskaya, MM., Nizheharodova, DB., Yurkevich, MY., et al.[2023]

References

1. Romania pubmed.ncbi.nlm.nih.gov

Mesenchymal stem cells in multiple sclerosis - translation to clinical trials. [2022]

2. England pubmed.ncbi.nlm.nih.gov

A prospective, randomized, controlled trial of autologous haematopoietic stem cell transplantation for aggressive multiple sclerosis: a position paper. [2022]

3. England pubmed.ncbi.nlm.nih.gov

Immune modulatory mesenchymal stem cells derived from human embryonic stem cells through a trophoblast-like stage. [2018]

4. United States pubmed.ncbi.nlm.nih.gov

Human ESC-derived MSCs outperform bone marrow MSCs in the treatment of an EAE model of multiple sclerosis. [2021]

5. United States pubmed.ncbi.nlm.nih.gov

Efficacy and Safety of Mesenchymal Stem Cell Transplantation in the Treatment of Autoimmune Diseases (Rheumatoid Arthritis, Systemic Lupus Erythematosus, Inflammatory Bowel Disease, Multiple Sclerosis, and Ankylosing Spondylitis): A Systematic Review and Meta-Analysis of Randomized Controlled Trial. [2022]

6. England pubmed.ncbi.nlm.nih.gov

Safety, tolerability, and activity of mesenchymal stem cells versus placebo in multiple sclerosis (MESEMS): a phase 2, randomised, double-blind crossover trial. [2022]

7. Netherlands pubmed.ncbi.nlm.nih.gov

In vitro assessment of mesenchymal stem cells immunosuppressive potential in multiple sclerosis patients. [2023]