NG01 Cell Therapy for Multiple Sclerosis

Recruiting at 1 trial location

Overseen By Flavia Nelson, MD

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: NeuroGenesis Ltd.

Disqualifiers: Pregnancy, Clotting disorders, Cancer, others

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: 4

Duration: 9 months

45 Participants Needed

Trial Summary

Will I have to stop taking my current medications?

The trial protocol does not specify whether you need to stop taking your current medications. However, it mentions that participants should have been on their current disease-modifying therapy for at least 6 months.

What data supports the effectiveness of the NG01 Cell Therapy treatment for multiple sclerosis?

Research on similar cell therapies shows promise in treating multiple sclerosis by using stem cells to repair damaged nerve cells and reduce inflammation. For example, engineered neural stem cells have been shown to help recovery in chronic autoimmune conditions by delivering therapeutic molecules directly to affected areas in the brain and spinal cord. ^{1 2 3 4 5}

What safety data exists for NG01 Cell Therapy or similar treatments in humans?

Several studies have shown that cell therapies similar to NG01, such as those using mesenchymal stromal cells (MSCs) and neural stem cells, have been generally safe and well-tolerated in humans. For example, a study on MSC therapy in multiple sclerosis patients found it to be safe with no serious side effects, and another study confirmed the safety of human neural stem cells in clinical trials. ^{6 7 8 9 10}

How does NG01 Cell Therapy differ from other treatments for multiple sclerosis?

NG01 Cell Therapy is unique because it uses NG2(+) cells, a type of neural cell, to promote myelin repair and reduce inflammation in the central nervous system, which is different from traditional treatments that mainly focus on suppressing the immune system. ^{1 11 12 13 14}

What is the purpose of this trial?

The goal of this clinical trial is to assess the safety and efficacy of repeated intrathecal (IT) injection of NG01, autologous bone marrow derived human stromal cells, in treating Secondary Progressive Multiple Sclerosis (SPMS), compared to placebo. The study will assess the proportion of participants demonstrating improvement in walking ability, defined as a reduction in the average time to complete the Timed 25-Foot Walk (T25FW) at 6, 9, and 12 months compared to baseline. This will be analyzed by the mean change in walking speed across these time points. The study will also evaluate the incidence and nature of treatment-emergent adverse events (AEs). Participants will receive intrathecal administrations of NG01, by lumbar puncture, and will be followed up for 6 months after their fourth administration.

Eligibility Criteria

This trial is for individuals with Secondary Progressive Multiple Sclerosis (SPMS). Participants should be able to complete the Timed 25-Foot Walk test. Specific eligibility criteria are not provided, but typically include age range, disease severity, and no conflicting health conditions.

Inclusion Criteria

I have been diagnosed with Secondary Progressive Multiple Sclerosis.

My disability has worsened in the last 2 years as per EDSS or T25FW scores.

My disability level is moderate to severe but I can still walk.

Exclusion Criteria

My condition worsened or I had new symptoms in the last 2 years.

I am not pregnant, breastfeeding, and if capable of childbearing, I use effective contraception.

History of a general chronic handicapping/incapacitating disease other than MS

Timeline

Screening

Participants are screened for eligibility to participate in the trial

4 weeks

Run-in

Participants undergo a 4-month run-in period before treatment begins

16 weeks

Treatment

Participants receive 4 intrathecal administrations of NG01 or placebo, 3 months apart over a 9-month period

9 months

4 visits (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment

6 months

Treatment Details

Interventions

• NG01 Cell Therapy

Trial Overview The study tests NG01 cell therapy involving autologous bone marrow derived human stromal cells given through spinal injections versus a placebo of saline solution. It measures improvement in walking speed over time and monitors any adverse events after treatment.

Participant Groups

Group I: 50x10^6 cells Experimental Treatment 1 Intervention

15 participants with SPMS will receive 4 IT administrations of NG01 (50×10\^6 cells), 3 months apart

Group II: 100x10^6 cells Experimental Treatment 1 Intervention

15 participants with SPMS will receive 4 IT administrations of NG01 (100×10\^6 cells), 3 months apart

Group III: Placebo Placebo Group 1 Intervention

15 participants with SPMS will receive 4 IT administrations of placebo solution, 3 months apart

Who Is Running the Clinical Trial?

NeuroGenesis Ltd.

Lead Sponsor

Findings from Research

The article has been retracted due to significant concerns about image reuse and misrepresentation of data, which undermines the integrity of the research.

Despite the authors' disagreement with the retraction, the editorial office confirmed that the issues found in the article represent a serious violation of scientific publishing standards.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

RETRACTED: Neural Stem Cells Engineered to Express Three Therapeutic Factors Mediate Recovery from Chronic Stage CNS Autoimmunity Li, X., Zhang, Y., Yan, Y., et al.[2023]

References

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

RETRACTED: Neural Stem Cells Engineered to Express Three Therapeutic Factors Mediate Recovery from Chronic Stage CNS Autoimmunity [2023]

2. Iran pubmed.ncbi.nlm.nih.gov

Pathophysiological Concepts in Multiple Sclerosis and the Therapeutic Effects of Hydrogen Sulfide. [2020]

3. New Zealand pubmed.ncbi.nlm.nih.gov

Cell Therapy for Multiple Sclerosis. [2022]

4. Germany pubmed.ncbi.nlm.nih.gov

Immune Checkpoint Ligand Bioengineered Schwann Cells as Antigen-Specific Therapy for Experimental Autoimmune Encephalomyelitis. [2023]

5. Spain pubmed.ncbi.nlm.nih.gov

The effect of intravenous immunoglobulin on neuromyelitis optica. [2022]

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

Preclinical safety of human embryonic stem cell-derived oligodendrocyte progenitors supporting clinical trials in spinal cord injury. [2015]

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

Intracerebroventricular transplantation of human iPSC-derived neural stem cells (hiPSC-NSCs) into neonatal mice. [2022]

8. England pubmed.ncbi.nlm.nih.gov

Clinical safety of intrathecal administration of mesenchymal stromal cell-derived neural progenitors in multiple sclerosis. [2018]

9. Switzerland pubmed.ncbi.nlm.nih.gov

Human Neural Stem Cell-Based Drug Product: Clinical and Nonclinical Characterization. [2022]

10. Switzerland pubmed.ncbi.nlm.nih.gov

Short and Long Term Clinical and Immunologic Follow up after Bone Marrow Mesenchymal Stromal Cell Therapy in Progressive Multiple Sclerosis-A Phase I Study. [2020]

11. Singapore pubmed.ncbi.nlm.nih.gov

Myelin repair and functional recovery mediated by neural cell transplantation in a mouse model of multiple sclerosis. [2021]

12. Singapore pubmed.ncbi.nlm.nih.gov

Roles of NG2 glial cells in diseases of the central nervous system. [2021]

13. Switzerland pubmed.ncbi.nlm.nih.gov

Genetically Engineered Artificial Microvesicles Carrying Nerve Growth Factor Restrains the Progression of Autoimmune Encephalomyelitis in an Experimental Mouse Model. [2023]

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

LINGO-1-Fc-Transduced Neural Stem Cells Are Effective Therapy for Chronic Stage Experimental Autoimmune Encephalomyelitis. [2018]