Phase 3 Trial of NurOwn, Cell Therapy for ALS, Gets Thumbs Up in Safety Review by Independent Board

Phase 3 Trial of NurOwn, Cell Therapy for ALS, Gets Thumbs Up in Safety Review by Independent Board

The Phase 3 clinical trial testing BrainStorm Cell Therapeutics‘ cell therapy candidate NurOwn in amyotrophic lateral sclerosis (ALS) patients is continuing as planned after a second safety assessment by the trial’s independent Data Safety Monitoring Board (DSMB) found no reasons to stop, the company announced.

The DSMB’s recommendation comes after a pre-specified interim analysis of the first 106 ALS patients treated repeatedly with NurOwn in this randomized, placebo-controlled clinical trial.

“After reviewing all of the safety data as of September 30th, the DSMB has recommended the study continue without any changes in the protocol. We did not identify any significant safety concerns,” Carlayne Jackson, MD, a professor of Neurology and Otolaryngology UT Health San Antonio and the DSMB chairperson, said in a press release.

DSMBs consist of research experts who monitor the progress of a clinical trial and review safety and efficacy data while the study is ongoing. This panel can recommend that a trial be stopped early because of safety concerns or evidence a therapy is not working as intended, or if the trial’s main goals have already been reached.

NurOwn consists of mesenchymal stem cells (MSCs; stems cells able to generate various cell types) collected from a patient’s bone marrow. These MSCs are expanded and matured into a specific cell type — called MSC-NTF — by growing them under conditions that induce them to secrete high levels of neurotrophic factors (NTFs) that support the growth, survival, and maturation of nerve cells.

MSC-NTF cells also deliver immune system regulating cytokines, small proteins important in cell signaling or messaging, to sites of damage, BrainStorm reports. It is thought this will help to slow or stabilize disease progression.

The double-blind Phase 3 trial (NCT03280056), fully enrolled at its six U.S. sites, is investigating use of NurOwn in 200 ALS patients whose symptoms became evident within two years of the study’s start. Patients are randomized 1:1 to either NurOwn or placebo, given via intrathecal (spinal canal) injection every two months.

The study’s primary measures of safety and efficacy are being determined using the ALS functional rating scale score (ALSFRS-R; a score of abilities like swallowing, speech, handwriting, walking, etc.) in patients after 28 weeks of treatment compared to placebo.

A secondary goal is assessing how biomarkers, such as cell-secreted neurothrophic factors, inflammatory agents, and cytokines, change in the blood and cerebrospinal fluid (the liquid surrounding the brain and spinal cord) after treatment with NurOwn.

BrainStorm is expecting to have topline trial data by the end of 2020, which will potentially support the submission of a Biologics License Application (BLA) to the U.S. Food and Drug Administration requesting approval.

“We are very pleased with the DSMB recommendation that the Phase 3 clinical trial continue without any protocol modification. This represents an important clinical trial advancement for BrainStorm and for the development of NurOwn as an innovative cellular therapy approach for ALS patients,” added Ralph Kern MD, BrainStorm’s chief operating officer and chief medical officer.

This clinical trial is being funded by a grant from the California Institute for Regenerative Medicine (CIRM CLIN2-0989), and other types of investment.

NurOwn was given orphan drug status by both the FDA and the European Medicines Agency (EMA) as a potential ALS treatment.

Iqra holds a MSc in Cellular and Molecular Medicine from the University of Ottawa in Ottawa, Canada. She also holds a BSc in Life Sciences from Queen’s University in Kingston, Canada. Currently, she is completing a PhD in Laboratory Medicine and Pathobiology from the University of Toronto in Toronto, Canada. Her research has ranged from across various disease areas including Alzheimer’s disease, myelodysplastic syndrome, bleeding disorders and rare pediatric brain tumors.
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Inês Martins holds a BSc in Cell and Molecular Biology from Universidade Nova de Lisboa and is currently finishing her PhD in Biomedical Sciences at Universidade de Lisboa. Her work has been focused on blood vessels and their role in both hematopoiesis and cancer development.
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Iqra holds a MSc in Cellular and Molecular Medicine from the University of Ottawa in Ottawa, Canada. She also holds a BSc in Life Sciences from Queen’s University in Kingston, Canada. Currently, she is completing a PhD in Laboratory Medicine and Pathobiology from the University of Toronto in Toronto, Canada. Her research has ranged from across various disease areas including Alzheimer’s disease, myelodysplastic syndrome, bleeding disorders and rare pediatric brain tumors.
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23 comments

  1. Fatimah says:

    Hello what a good news.
    my mother diagnosed with AlS recently and I tried to follow any news regarding this trail. they are doing great work and once I added a comment about the trial and one of the Brainstorm team contacted me personally to answer my enquiries. Stem cells is a great revolution and we hope to get great result an approval soon.

    Fatimah

  2. Mark Bedwell says:

    Since the trail is safe let’s get this approved now please. I have finished the trail and completed it and the worst thing that I have had was a headache. We need to push the FDA to approve this quickly because a lot of people will die if we wait to the end of the trail. Please give us Pals a chance to live. Thank you

  3. Area Man says:

    We know it’s safe. All previous phases proved that. There are no side effects of using your own stem cells, save for a headache if you don’t lie on your back for a couple hours after the intrathecal injection. My god, this is like watching grass grow. There has to be a way to speed up this process and make this treatment available to PALS.

  4. You guys are murderers. This treatment is the only that will save lives and all you care about is money and power. Shame on you! My husband has als 6 years now. How dare you let all of these patients with als go through this devastating disease. You have known it was safe and effective for so long. How do you sleep at night. Do something humane. Let the treatment go public. Do your job. Humanitarians. Right! Becky Monson

  5. Karin says:

    Why can’t we get this fast tracked for approval if its save? What’s the worst that can happen, we might die? All of us Pals are dying anyway.
    By the time this gets approved 1000 of us have lost the fight against ALS.

  6. Gary says:

    This is great news. FDA, please stop injecting sugar water into the 100 patients who are getting the Placebo. This is cruel and a waste of time and money. Give all 200 patients NurOwn and measure their progress. A sample of 200 is better than 100. We do not need to subject 100 “qualified” patients a placebo! Also, let’s open this therapy to “Right to Try”, no more “right to deny”.

  7. Jackie says:

    My daughter had just given birth to her second child when she was diagnosed 2 yrs ago please let anyone who wants to try this, her children want their mum back. 😢

  8. Walid Hosny says:

    Hello, I am Egyptian,42 years old, otolaryngologist,ALS patient, diagnosed at April 2019, and the symptoms progress rabidly with me, I hope that I started this new medicine with you as no time to wait till you finished all levels of trials, I hope that you feel what I complain as a ALS patient and as a doctor, please help me, thank you

  9. Pat says:

    Good news thanks.but why we have to wait till the end of 2020 .Als didn’t wait.from now till 2020 thousands of people is going to die.please approve them now ..we all want them now .Als is aggressive deases

  10. Matt Monahan says:

    I have been helping care for my father in law who has been fighting ALS for over three years. Please make this available to help him fight this disease! We would be willing to take it now!

  11. Umit says:

    Is there a study on the estimated number of ALS patients who will die by the end of 2020 ?In an untreated disease, behaving this way is not ethical.

  12. Bob jane says:

    I wish hope and pray the folks behind NurOwn allow doctors all over the world to have the technology so that countless lives could be saved/ improved. Realising money makes the world go round, health of sufferers from als is a worthwhile cause.

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