MSC-NTF (NurOwn) for ALS

Not yet recruiting at 14 trial locations

Overseen By Chief Medical Officer, Brainstorm Cell Therapeutics, MD

Age: 18+

Sex: Any

Trial Phase: Phase 3

Sponsor: Brainstorm-Cell Therapeutics

Disqualifiers: Prior stem cell therapy, Cancer, Other motor neuron diseases, others

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

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

Compensation: Varies

Number of Visits: 8

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new treatment called NurOwn (Debamestrocel - MSC-NTF) for individuals with ALS, a disease affecting nerve cells in the brain and spinal cord. The goal is to evaluate the safety and effectiveness of NurOwn compared to a placebo (a look-alike substance with no active drug) in individuals with early symptoms and moderate progression of ALS. Participants will receive either NurOwn or a placebo every 8 weeks for the first 24 weeks, followed by NurOwn for another 24 weeks. Suitable candidates have experienced muscle weakness from ALS for less than two years and can still perform daily activities like standing and walking. As a Phase 3 trial, this study represents the final step before FDA approval, offering participants a chance to contribute to potentially groundbreaking treatment advancements.

Do I have to stop taking my current medications for the trial?

The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the trial coordinators or your doctor.

Is there any evidence suggesting that Debamestrocel - MSC-NTF (NurOwn) is likely to be safe for humans?

Research has shown that NurOwn, the treatment being tested, has generally been well tolerated in past studies. For example, an earlier study with patients who have ALS (a disease affecting nerve cells in the brain and spinal cord) found NurOwn to be well tolerated. Most participants did not experience serious side effects, reporting only mild to moderate reactions such as headaches or discomfort at the injection site.

Another study found that even in patients with rapidly progressing ALS, the treatment was considered safe. This suggests that NurOwn could be a promising option for those considering joining the trial. While every treatment carries risks, the data so far indicates that NurOwn's safety profile is encouraging for potential participants. ^{1 2 3 4 5}

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

Unlike the standard treatments for ALS, which include medications like Riluzole and Edaravone, Debamestrocel - MSC-NTF (NurOwn) uses a novel approach by employing NTF-secreting mesenchymal stem cells. This treatment aims to deliver neurotrophic factors directly to damaged areas in the nervous system, potentially offering a more targeted and effective therapy. Researchers are excited because this method could not only slow disease progression but also improve motor function by addressing the underlying neuronal damage more directly than current options.

What evidence suggests that Debamestrocel - MSC-NTF (NurOwn) might be an effective treatment for ALS?

Research has shown that NurOwn, a type of cell therapy, may help treat ALS (amyotrophic lateral sclerosis). In earlier studies, people with ALS who used NurOwn lived longer. Specifically, long-term use of NurOwn significantly increased their survival. Additionally, past trials found that NurOwn could deliver healing substances directly to damaged areas, potentially slowing the disease. In this trial, participants will receive either NurOwn or a placebo. These findings suggest that NurOwn could help manage ALS symptoms and improve life expectancy. ^{2 6 7 8 9}

Are You a Good Fit for This Trial?

This trial is for people with early symptomatic ALS and moderate disease presentation. Participants will be involved in the study for a total of 48 months, receiving treatments every 8 weeks and visiting the clinic regularly for checkups.

Inclusion Criteria

≤45 points on ALSFRS-R total score at Screening Visit 1

≥2 points on each item of the ALSFRS-R at the Screening Visit 1

Participants must adhere to highly effective methods of contraception as specified in the study protocol

Exclusion Criteria

Active participation in any other ALS interventional study

I cannot lie flat for treatments or tolerate certain medical procedures.

Pregnant women or women currently breastfeeding

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

9 weeks

2 visits (in-person)

Treatment (Part A)

Participants receive NurOwn or placebo via IT injection every eight weeks

24 weeks

3 visits (in-person)

Open-label Extension (Part B)

All participants receive NurOwn via IT injection every eight weeks

24 weeks

3 visits (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Debamestrocel - MSC-NTF (NurOwn)
• Placebo

Trial Overview The trial is testing Debamestrocel - MSC-NTF (NurOwn) against a placebo to see if it's safe and effective in treating ALS. After an initial comparison phase of 24 months, all participants will receive NurOwn for another 24 months.

How Is the Trial Designed?

Group I: Debamestrocel - MSC-NTF (NurOwn) Experimental Treatment 1 Intervention

NTF-secreting mesenchymal stem cells (MSC-NTF cells) are a novel cell-therapeutic approach aimed at effectively delivering NTFs directly to the site of damage in ALS patients

Group II: Placebo Placebo Group 1 Intervention

Placebo is comprised of Dulbecco Modified Eagle Medium (DMEM)

Who Is Running the Clinical Trial?

Brainstorm-Cell Therapeutics

Lead Sponsor

Trials

6

Recruited

290+

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT06973629

NCT06973629 | Efficacy and Safety of MSC-NTF (NurOwn) ... This is a multicenter, Phase 3b study to assess the efficacy and safety of NurOwn in participants with early symptomatic ALS and moderate disease presentation ...

2. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC6937497/

NurOwn, phase 2, randomized, clinical trial in patients with ALS This phase II study provides Class I evidence. Amyotrophic lateral sclerosis (ALS) is a fatal neurodegenerative disease characterized by degeneration and death ...

3. alsnewstoday.com alsnewstoday.com/news/long-term-nurown-use-shows-survival-benefit-als-analysis/

Long-term use of NurOwn shows survival benefit for ALS Long-term treatment with the cell therapy NurOwn (debamestrocel) was found to significantly extend survival in people with amyotrophic lateral sclerosis (ALS)

4. practicalneurology.com practicalneurology.com/news/cell-therapy-for-als-shows-promise-in-increasing-survival-at-five-years/2475217/

Cell Therapy for ALS Associated with Increased Survival at 5 ... Cell Therapy for ALS Associated with Increased Survival at 5 Years ... Nine out of 10 people with amyotrophic lateral sclerosis (ALS) treated with ...

5. ir.brainstorm-cell.com ir.brainstorm-cell.com/2024-10-28-BrainStorm-Cell-Therapeutics-Presented-Positive-Survival-Data-from-NurOwn-R-Expanded-Access-Program-at-2024-Annual-NEALS-Meeting

News - BrainStorm Cell Therapeutics The primary efficacy endpoint will be a comparison of change in ALSFRS-R from baseline to week-24 (i.e. at the end of Part A) for debamestrocel ...

6. clinicaltrials.gov clinicaltrials.gov/study/NCT06973629

NCT06973629 | Efficacy and Safety of MSC-NTF (NurOwn) ... This is a multicenter, Phase 3b study to assess the efficacy and safety of NurOwn in participants with early symptomatic ALS and moderate disease presentation ...

7. als-mnd.org als-mnd.org/support-for-pals-cals/drugs-in-development/brainstorm-nurown/

BrainStorm Cell Therapeutics – NurOwn The phase 2 trial data involved 48 ALS patients (36 treated and 12 placebo) and was published in December 2019 in the journal Neurology, titled “A single-dose ...

8. ir.brainstorm-cell.com ir.brainstorm-cell.com/2020-11-17-BrainStorm-Announces-Topline-Results-from-NurOwn-R-Phase-3-ALS-Study

BrainStorm Announces Topline Results from NurOwn® Phase ... Results from the trial showed that NurOwn® was generally well tolerated in this population of rapidly progressing ALS patients.

9. cirm.ca.gov cirm.ca.gov/our-progress/awards/phase-3-randomized-placebo-controlled-multicenter-study-evaluate-efficacy-safety-repeated-administrations-nurown-patients-als/

A Phase 3, Randomized, Placebo-controlled Multicenter Study ... A Phase 3, Randomized, Placebo-controlled Multicenter Study to Evaluate Efficacy & Safety of Repeated Administrations of NurOwn® in Patients with ALS.