What is the mechanism of action of this treatment?

The most common treatments for Multiple Sclerosis (MS) include disease-modifying therapies (DMTs) and cell-based therapies. DMTs, such as interferons and monoclonal antibodies, work by modulating the immune system to reduce inflammation and prevent immune cells from attacking the myelin sheath, which is crucial for protecting nerve fibers. Cell-based therapies, like the DUOC-01 cells derived from human umbilical cord blood, aim to repair and regenerate damaged tissues by promoting remyelination and providing neuroprotection. These mechanisms are vital for MS patients as they help slow disease progression, reduce relapse rates, and improve overall neurological function, thereby enhancing the quality of life.

What side effects are associated with this type of intervention?

Cell-based therapies for Multiple Sclerosis, such as those derived from human umbilical cord blood, are still under investigation, but potential side effects may include immune reactions, infections, and complications related to the administration procedure. Other common treatments for MS, such as interferon beta and dimethyl fumarate, can cause flu-like symptoms, injection site reactions, gastrointestinal issues, and liver enzyme abnormalities. Hematopoietic stem cell transplantation (HSCT) has shown promise but carries risks of serious infections, graft-versus-host disease, and treatment-related mortality.

What prior FDA approvals exist?

The FDA has approved several treatments for Multiple Sclerosis, including disease-modifying therapies (DMTs) such as interferon-beta, glatiramer acetate, and oral formulations like dimethyl fumarate. While there is ongoing research into cell-based therapies like DUOC-01, which uses cells derived from human umbilical cord blood, there are no specific FDA-approved treatments of this kind for MS as of now. Current approved treatments primarily focus on immunomodulation and reducing relapse rates rather than cell-based regenerative approaches.

What other types of research exist?

Promising novel alternatives to DUOC-01 cells for Multiple Sclerosis patients include: 1) Hematopoietic Stem Cell Transplantation (HSCT), which has shown significant benefits in reducing disease progression and relapse rates in MS patients. 2) Mesenchymal Stem Cells (MSCs), which have demonstrated potential in promoting healing and reducing inflammation in clinical trials. Both therapies are being actively researched and have shown encouraging results in treating MS.

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Stem Cell Therapy

DUOC-01 for Multiple Sclerosis (DUOC for MS Trial)

Phase 1

Recruiting

Led By Beth Shaz, MD

Research Sponsored by Joanne Kurtzberg, MD

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

EDSS score at screening 3.0-6.5 that was not acquired within the last 6 months

Diagnosis of primary progressive MS according to 2017 revised McDonald criteria

Must not have

Intractable seizures

Active, chronic disease of the immune system other than MS

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 1 year post infusion

Awards & highlights

No Placebo-Only Group

Summary

This trial tests the safety of injecting special cells from umbilical cord blood into the spinal fluid of adults with a severe form of multiple sclerosis. The goal is to see if these cells can help repair nerve damage and reduce inflammation. About 20 participants will be monitored over time to check for any improvements and side effects. Umbilical cord blood cells have been studied for their potential to treat neurological disorders, including spinal cord injuries, due to their ability to produce neurotrophic factors and modulate immune responses.

Who is the study for?

Adults aged 18-65 with Primary Progressive Multiple Sclerosis (PPMS) and an EDSS score of 3.0-6.5 are eligible for this trial. Participants must have stable MS, a matched umbilical cord blood unit available, and agree to effective contraception during the study plus six months after treatment. Exclusions include prior transplants or cell therapies within three years, other progressive neurological disorders, recent malignancies except certain skin cancers, active immune diseases besides MS, MRI or lumbar puncture contraindications, severe infections, ventilatory support need, recent suicidal behavior or ideation.

What is being tested?

The trial is testing DUOC-01 cells administered intrathecally (into the spinal canal) to treat PPMS. DUOC-01 is derived from human umbilical cord blood cells aimed at treating neurodegenerative conditions. This Phase 1a open-label single-center study will enroll about 20 participants and explore changes in MS scores, brain MRI results, and blood biomarkers.

What are the potential side effects?

As this is a Phase 1a safety trial for DUOC-01 cells in PPMS patients; specific side effects are not listed but may include typical risks associated with intrathecal procedures such as headache or back pain and potential immune reactions due to stem cell therapy.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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My disability score is between 3.0 and 6.5, and it has been stable for the last 6 months.

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I have been diagnosed with primary progressive MS.

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I have a matching umbilical cord blood unit stored.

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I will continue my current disease treatments while participating in the study.

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I am between 18 and 65 years old.

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My condition has not worsened in the past 3 months.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I have seizures that medication cannot control.

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I have a chronic immune system disease other than MS.

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I am not currently receiving treatment for another cancer, nor am I on immunosuppressants for conditions other than MS or undergoing cytotoxic chemotherapy.

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I have not had an organ, tissue, or stem cell transplant or cell therapy in the last 3 years.

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I need help with breathing (ventilator support).

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I have a bleeding disorder.

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I have a progressive neurological disorder that is not MS.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 1 year post infusion

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~1 year post infusion

This trial's timeline: 3 weeks for screening, Varies for treatment, and 1 year post infusion for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Incidence of IT administration adverse events

Incidence of adverse events attributed to the investigational product

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups

Experimental Treatment

Group I: DUOC-01Experimental Treatment1 Intervention

Intrathecal Infusion of DUOC-01 and hydrocortisone. Cohort 1: 10 million cells Cohort 2: greater than 10 to 25 million cells Cohort 3: greater than 25 to 50 million cells

0 / 13Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

The most common treatments for Multiple Sclerosis (MS) include disease-modifying therapies (DMTs) and cell-based therapies. DMTs, such as interferons and monoclonal antibodies, work by modulating the immune system to reduce inflammation and prevent immune cells from attacking the myelin sheath, which is crucial for protecting nerve fibers. Cell-based therapies, like the DUOC-01 cells derived from human umbilical cord blood, aim to repair and regenerate damaged tissues by promoting remyelination and providing neuroprotection. These mechanisms are vital for MS patients as they help slow disease progression, reduce relapse rates, and improve overall neurological function, thereby enhancing the quality of life.

Proteome analysis of post-transplantation recovery mechanisms of an EAE model of multiple sclerosis treated with embryonic stem cell-derived neural precursors.Disease-modifying treatments for progressive multiple sclerosis.Spotlight on anti-CD20.