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

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new treatment called IMS001 for individuals with Multiple Sclerosis (MS) who haven't found relief with other medications. IMS001, a type of stem cell therapy, might alter the disease's progression. Participants will receive varying doses of IMS001 through an IV, which delivers medicine directly into the bloodstream, to evaluate its effectiveness and monitor any side effects. The trial seeks individuals with MS who haven't responded well to disease-modifying treatments and are stable enough for MRIs. As a Phase 1 trial, this research aims to understand how IMS001 works in people, offering participants the opportunity to be among the first to receive this new treatment.

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.

Is there any evidence suggesting that IMS001 is likely to be safe for humans?

Research shows that IMS001, a treatment made from mesenchymal stem cells (MSC) derived from human embryos, is being tested for safety in people with Multiple Sclerosis (MS). These MSCs might help manage the disease. Currently, limited information exists on the safety of IMS001 specifically. This trial is in its first phase, focusing primarily on assessing the treatment's safety for people.

As a Phase 1 trial, it represents the earliest stage of testing in humans. Researchers carefully monitor participants to identify any side effects. IMS001 is being tested for individuals who did not respond well or could not tolerate other treatments, making safety a top priority.

If approved, this treatment could offer a new option for patients with MS. Participation in this early trial helps scientists learn more about how well IMS001 is tolerated and what side effects might occur. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for Multiple Sclerosis?

Unlike the standard treatments for multiple sclerosis, which often include medications like interferons and monoclonal antibodies, IMS001 is an innovative approach that involves the infusion of cells intravenously. This treatment is unique because it uses a cellular therapy that may help modulate the immune system differently than traditional drugs. Researchers are excited about IMS001 because it offers different dosing options and may provide a new way to manage the disease with potentially fewer side effects. Additionally, the option for an additional high dose at six months offers flexibility in treatment, which could lead to more personalized care for patients with multiple sclerosis.

What evidence suggests that IMS001 might be an effective treatment for Multiple Sclerosis?

Research has shown that stem cells like IMS001 could help treat conditions similar to multiple sclerosis. In animal studies, stem cells derived from human embryos effectively treated models of multiple sclerosis. These cells performed better than other types of stem cells, possibly slowing the disease. They might repair damaged tissues and reduce inflammation in the body. Although researchers are still studying how IMS001 works in humans, these early results offer hope for its potential to manage multiple sclerosis. Participants in this trial will receive different doses of IMS001 to evaluate its effectiveness and safety. ^{2 4 6 7 8}

Who Is on the Research Team?

Richard Kim, MD

Principal Investigator

ImStem Biotechnology

Are You a Good Fit for This Trial?

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).

My condition did not improve after taking disease-modifying treatments.

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 for a Trial Participant

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

What Are the Treatments Tested in This Trial?

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.

How Is the Trial Designed?

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+

Published Research Related to This Trial

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]

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]

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]

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]

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 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]

Citations

1. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC4110787/

Human ESC-Derived MSCs Outperform Bone Marrow MSCs ... We demonstrate that these hES-MSC lines can effectively treat an EAE model of MS and outperform multiple lines of BM-MSCs in therapeutic activities. Results.

2. clinicaltrials.gov clinicaltrials.gov/study/NCT05532943

Study Details | NCT05532943 | Evaluate the Safety and ... This study is to identify the safety and efficacy of repeat IV(Intravenous) and IT(Intrathecal) administrations of UMSC01 in patients with MS.

3. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC11397842/

Potential of Nano-Engineered Stem Cells in the Treatment of ... Study to Evaluate the Safety, Tolerability, and Exploratory Efficacy of IMS001 in Subjects with Multiple Sclerosis, Phase-I, Recruiting, NCT04956744, 2021. MSC ...

4. clinicalresearch.com clinicalresearch.com/en/find-trials/Study/NCT04956744

NCT04956744 A Study to Evaluate the Safety, Tolerability, and Exploratory Efficacy of IMS001 in Subjects With Multiple Sclerosis.

5. nationalmssociety.org nationalmssociety.org/how-you-can-help/get-involved/participate-in-research-studies/participate-in-clinical-trials/ims001

Clinical Trial: IMS001 to Treat MS Participate in the clinical trial to determine the safety, tolerability, efficacy, and optimal dose regimen of IMS001 to treat MS.

6. connect.careboxhealth.com connect.careboxhealth.com/en-US/trial/listing/295228

A Study to Evaluate the Safety, Tolerability, and Exploratory ... This is a Phase 1 study of IMS001, given as a single dose to subjects with Multiple Sclerosis who experience inadequate response and/or ...

7. clinconnect.io clinconnect.io/trials/NCT04956744

A Study to Evaluate the Safety, Tolerability, and - ClinConnect This clinical trial is testing a new treatment called IMS001 for people with Multiple Sclerosis (MS) who haven't had good results with their current medications ...

8. rarediseaseadvisor.com rarediseaseadvisor.com/hcp-resource/multiple-sclerosis-clinical-trials/

Multiple Sclerosis Clinical Trials Safety and dose-response of vidofludimus calcium in relapsing multiple sclerosis: extended results of a placebo-controlled phase 2 Trial.