Stem Cell Treatment for Multiple Sclerosis

(MS Trial)

Overseen By Maureen Gerwin

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: Hope Biosciences

Must be taking: MS therapy

Must not be taking: Antibiotics, Antivirals, Antifungals, others

Disqualifiers: Pregnancy, Active malignancy, Substance use, 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

Compensation: Varies

Number of Visits: 6

Duration: 32 weeks

10 Participants Needed

Trial Summary

What is the purpose of this trial?

This protocol is part of a clinical study to evaluate efficacy and safety of multiple intravenous administrations of HB-adMSCs for the treatment of Multiple Sclerosis.

Will I have to stop taking my current medications?

The trial does not specify if you need to stop taking your current medications, but you must be stable on your MS therapy for at least 6 months before joining and cannot start new MS therapies during the trial.

What data supports the effectiveness of the treatment HB-adMSCs for multiple sclerosis?

Research on hematopoietic stem cell transplantation (HSCT) for multiple sclerosis shows that it can lead to long-term remission and reduce disease activity, suggesting that similar stem cell treatments like HB-adMSCs might also be effective. ^{1 2 3 4 5}

Is stem cell treatment generally safe for humans?

Research on stem cell treatments, including those derived from induced pluripotent stem cells (iPSCs), suggests they are generally safe in humans, with studies showing no tumors or significant immune reactions in animal models. Additionally, certain stem cell mobilization methods, like Plerixafor, have been used safely in clinical trials for conditions like sickle cell disease. ^{6 7 8 9 10}

How is the stem cell treatment HB-adMSCs different from other treatments for multiple sclerosis?

The stem cell treatment HB-adMSCs is unique because it involves using stem cells, which are special cells that can develop into different types of cells, to potentially repair damage in the nervous system caused by multiple sclerosis. This approach is different from conventional treatments that mainly focus on managing symptoms rather than repairing damage. ^{5 11 12 13 14}

Research Team

Djamchid Lotfi, MD

Principal Investigator

Hope Biosciences Research Foundation

Eligibility Criteria

This trial is for adults aged 18-75 with Relapsing Remitting Multiple Sclerosis, able to walk (EDSS score 3.0-6.5), and have banked stem cells with Hope Biosciences. They must not be pregnant or planning pregnancy, agree to use contraception, and have stable MS therapy for 6 months. Exclusions include recent infections, substance abuse, active cancer, other medical conditions that pose risks or interfere with the study.

Inclusion Criteria

I can walk but have some disability due to my condition.

Previously banked their mesenchymal stem cells with Hope Biosciences

I am not pregnant and will use birth control during and for 6 months after the study.

Exclusion Criteria

I haven't had an infection needing treatment in the last 30 days.

Known addiction or dependency or current substance use or abuse

I am currently pregnant or breastfeeding.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

up to 4 weeks

Treatment

Participants receive multiple intravenous administrations of HB-adMSCs

32 weeks

6 visits (in-person) at weeks 0, 4, 8, 16, 24, and 32

Follow-up

Participants are monitored for safety and effectiveness after treatment

20 weeks

Treatment Details

Interventions

• HB-adMSCs

Trial Overview The trial tests the safety and effectiveness of multiple intravenous doses of autologous HB-adMSCs in treating Multiple Sclerosis. Participants previously in a placebo group can join this study which involves patients who've stored their own mesenchymal stem cells at Hope Biosciences.

Participant Groups

Group I: Treatment Experimental Treatment 1 Intervention

Autologous adipose-derived HB-adMSCs

Who Is Running the Clinical Trial?

Hope Biosciences

Lead Sponsor

Trials

27

Recruited

470+

Findings from Research

In a case series of 10 patients with relapsing-remitting multiple sclerosis (MS) treated with autologous hematopoietic stem cell transplantation (HSCT), 5 patients achieved sustained complete remission after 10 years, indicating the potential long-term benefits of this treatment.

Among those in remission, 3 patients showed signs of MS resolution, as evidenced by normalized intrathecal IgG production and cerebrospinal fluid neurofilament light levels, suggesting that HSCT may effectively halt disease progression in some individuals.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Sustained remission in multiple sclerosis after hematopoietic stem cell transplantation. Tolf, A., Fagius, J., Carlson, K., et al.[2020]

A CRISPR-Cas9 gene correction strategy demonstrated up to 60% correction of the sickle cell disease-causing mutation in patient-derived hematopoietic stem cells, showing promising efficacy for potential treatment.

Preclinical studies in mice showed that the corrected cells engrafted successfully without signs of abnormal blood cell formation or tumor development, indicating a favorable safety profile for this gene therapy approach.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Development of β-globin gene correction in human hematopoietic stem cells as a potential durable treatment for sickle cell disease. Lattanzi, A., Camarena, J., Lahiri, P., et al.[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]

References

1. Denmark pubmed.ncbi.nlm.nih.gov

Sustained remission in multiple sclerosis after hematopoietic stem cell transplantation. [2020]

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

Hematopoietic stem cell transplantation for multiple sclerosis: current status and future challenges. [2013]

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

Long-Term Clinical Outcomes of Hematopoietic Stem Cell Transplantation in Multiple Sclerosis. [2021]

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

Continued disease activity in a patient with multiple sclerosis after allogeneic hematopoietic cell transplantation. [2009]

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

Long-term results of stem cell transplantation for MS: a single-center experience. [2022]

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

Development of β-globin gene correction in human hematopoietic stem cells as a potential durable treatment for sickle cell disease. [2022]

7. Italy pubmed.ncbi.nlm.nih.gov

Plerixafor enables safe, rapid, efficient mobilization of hematopoietic stem cells in sickle cell disease patients after exchange transfusion. [2021]

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

Bone Marrow as a Hematopoietic Stem Cell Source for Gene Therapy in Sickle Cell Disease: Evidence from Rhesus and SCD Patients. [2022]

9. England pubmed.ncbi.nlm.nih.gov

Hematopoietic stem cell gene therapy: dead or alive? [2012]

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

Assessment of safety and immunogenicity of MHC homozygous iPSC-derived CD34+ hematopoietic progenitors in an NHP model. [2022]

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

Autologous hematopoietic stem cell transplantation in multiple sclerosis: A meta-analysis. [2022]

12. 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]

13. England pubmed.ncbi.nlm.nih.gov

Safety and efficacy of hematopoietic stem cells mobilization in patients with multiple sclerosis. [2016]

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

Effect of Nonmyeloablative Hematopoietic Stem Cell Transplantation vs Continued Disease-Modifying Therapy on Disease Progression in Patients With Relapsing-Remitting Multiple Sclerosis: A Randomized Clinical Trial. [2020]