Stem Cell Treatment for Multiple Sclerosis

(MS Trial)

Overseen By Maureen Gerwin

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: Hope Biosciences Research Foundation

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

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new treatment using stem cells to help people with Multiple Sclerosis (MS), specifically the relapsing-remitting type. Researchers aim to determine if multiple doses of these stem cells, known as HB-adMSCs, can safely reduce MS symptoms. Individuals diagnosed with this type of MS for at least six months and who completed a previous related trial might be suitable candidates. Participants should be stable on their current MS treatment and must have stored their stem cells with Hope Biosciences. As a Phase 2 trial, the research focuses on measuring the treatment's effectiveness in an initial, smaller group of people.

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.

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

Research has shown that stem cell treatments, such as HB-adMSCs, may be safe for people with multiple sclerosis (MS). For instance, studies found that injections of mesenchymal stem cells (MSCs) can safely reduce brain lesions in some patients, resulting in fewer damaged areas in the brain and potentially alleviating symptoms.

Additional research supports the safety of MSCs for treating diseases like MS. In some studies, patients did not experience major side effects, indicating that the treatment is generally well-tolerated.

Since the trial is in Phase 2, early evidence suggests potential safety, but further research is needed to confirm these findings. The results so far are promising, but the treatment continues to be studied to ensure safety for everyone. ^{1 2 3 4 5}

Why are researchers excited about this trial?

Unlike the standard treatments for multiple sclerosis, which often include disease-modifying therapies like interferons and monoclonal antibodies, the investigational treatment HB-adMSCs is derived from autologous adipose (fat) tissue stem cells. Researchers are excited about these stem cells because they have the potential to repair and regrow damaged tissues, targeting the disease in a way that traditional therapies do not. This approach could offer a more regenerative solution, potentially slowing or even reversing the progression of the disease, which is a significant leap forward from just managing symptoms.

What evidence suggests that HB-adMSCs might be an effective treatment for Multiple Sclerosis?

Research has shown that HB-adMSCs, a type of stem cell therapy, may help treat multiple sclerosis. In this trial, participants will receive autologous adipose-derived HB-adMSCs. Studies have found that patients with mild to moderate relapsing forms of the disease experienced significant improvements in both physical and mental health. Reports indicate that this treatment enhanced patients' quality of life by achieving important health goals. Early findings suggest that regularly receiving high doses of these stem cells can lead to these positive changes. Overall, evidence suggests HB-adMSCs could effectively manage multiple sclerosis symptoms. ^{1 3 6 7 8}

Who Is on the Research Team?

Djamchid Lotfi, MD

Principal Investigator

Hope Biosciences Research Foundation

Are You a Good Fit for This Trial?

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

Randomized into the placebo group and completed participation in the HBMS01 clinical trial (IND 027633)

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 for a Trial Participant

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

What Are the Treatments Tested in This Trial?

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.

How Is the Trial Designed?

Group I: Treatment Experimental Treatment 1 Intervention

Autologous adipose-derived HB-adMSCs

Who Is Running the Clinical Trial?

Hope Biosciences Research Foundation

Lead Sponsor

Hope Biosciences

Lead Sponsor

Trials

27

Recruited

470+

Published Research Related to This Trial

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]

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]

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]

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT06800404

NCT06800404 | "HB-adMSCs for the Treatment of Patients ... This clinical trial is designed as an open label, single center, phase 2 study to assess the efficacy and safety of multiple autologous adipose derived ...

2. rarediseaseadvisor.com rarediseaseadvisor.com/news/phase-2-trial-promising-results-stem-cell-therapy-ms/

Phase 2 Trial for Stem Cell Therapy in MS Yields Positive ... Specifically, the results showed significant improvements in both physical and mental health for patients with mild to moderate relapsing- ...

3. neurologylive.com neurologylive.com/view/adipose-derived-mesenchymal-stem-cell-therapy-meets-primary-end-point-phase-2-trial-relapsing-remitting-ms

Adipose-Derived Mesenchymal Stem Cell Therapy Meets ... HB-adMSC therapy significantly improved quality of life in RRMS patients, meeting primary efficacy endpoints in physical and mental health ...

4. hopebio.org hopebio.org/artifact?id=a1TRi000000QCLhMAO

Hope Biosciences Research Foundation “The results of this trial are groundbreaking for multiple sclerosis. They clearly demonstrate that high doses of fresh HB-adMSCs delivered on a regular ...

5. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC5955528/

Adipose-derived mesenchymal stem cells (AdMSC) for the ... Adipose-derived mesenchymal stem cells (AdMSC) for the treatment of secondary-progressive multiple sclerosis: A triple blinded, placebo controlled, randomized ...

6. clinicaltrials.gov clinicaltrials.gov/study/NCT02326935

Multi-Center Study Safety of Adipose Derived ... The intent of this clinical study is to answer the questions: 1) is the proposed treatment safe and 2) is treatment effective in improving the disease pathology ...

7. sci.amegroups.org sci.amegroups.org/article/view/33787/html

Safety and efficacy of stem cell therapy for treatment of neural ... The results have revealed that MSCs injection safely decreases the brain lesions and disease severity and consequently improves the quality of life in these ...

8. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC3036569/

Safety and Immunological Effects of Mesenchymal Stem Cell ... Safety and immunological effects of mesenchymal stem cell transplantation in patients with multiple sclerosis and amyotrophic lateral sclerosis.