Phase 3 Trial of NurOwn Cell Therapy Fully Enrolls 200 Patients, BrainStorm Announces

Phase 3 Trial of NurOwn Cell Therapy Fully Enrolls 200 Patients, BrainStorm Announces

BrainStorm Cell Therapeutics announced that the Phase 3 clinical trial of its cell therapy candidate NurOwn in 200 amyotrophic lateral sclerosis (ALS) patients is fully enrolled and treatment is underway.

The randomized, double-blind and multicenter trial (NCT03280056) is assessing the safety and effectiveness of three administrations of NurOwn into the spinal canal. Patients will be given either NurOwn or placebo intrathecal injections every two months.

“The BrainStorm team is very excited to reach this important milestone in the development and potential commercialization of NurOwn in ALS. We have been fully dedicated to this journey since we began clinical trial enrollment in October 2017,” Chaim Lebovits, the president and CEO of BrainStorm, said in a press release.

NurOwn is a cell therapy that that takes mesenchymal stem cells (MSCs) from patients (autologous MSCs), which are able to generate various cell types. These MSCs are then converted into a cell-type — called MSC-NTFs — that produce neurotrophic factors (molecules which promote nerve tissue growth and survival). The converted cells are then returned through a transplant to the patients, either through an injection into the muscle or the spinal canal, to promote nerve tissue growth and survival.

The study’s primary goals of NurOwn’s safety and effectiveness will be measured using the ALS functional rating scale score (ALSFRS-R; a score of abilities like swallowing, speech, handwriting, walking, etc.) and compared to the placebo group. A secondary goal is assessing how biomarkers, such as cell-secreted neurothrophic factors, inflammatory factors, and cytokines, change in the blood and cerebrospinal fluid (the liquid surrounding the brain and spinal cord) following treatment with NurOwn.

“To date, a significant percentage of the 200 enrolled patients have received three treatments and we expect that all trial participants will complete planned study visits and assessments by October 2020,” Lebovits said.

This study is being carried out at the University of California, Irvine; Cedars-Sinai Medical Center; California Pacific Medical Center; Massachusetts General Hospital; the University of Massachusetts Medical School; and the Mayo Clinic.

If successful, trial results will be used to support an FDA request for approval of NurOwn as an ALS treatment, BrainStorm said.

The use of autologous MSC-NTF cells to potentially treat ALS was given orphan drug status by both the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA).

“We are very pleased to have fully enrolled our Phase 3 randomized placebo-controlled clinical trial in ALS and look forward to reporting top-line results before the end of next year. This clinical milestone is a key inflection point in the development of NurOwn,” said Ralph Kern, the chief operating officer and chief medical officer of BrainStorm.

This study is financially supported by a grant and other funding from the California Institute for Regenerative Medicine (CIRM), as well as other types of investment.

“Based on BrainStorm Cell Therapeutics’ scientific and early phase clinical trial data, CIRM awarded them $15.9 million to advance their investigational therapy NurOwn into a Phase 3 trial,” said Maria Millan, MD, president and CEO of the CIRM.

“Our support helped bring this program to three leading California medical centers. We congratulate Brainstorm Cell Therapeutics in completing enrollment, an important milestone toward developing a much-needed treatment for ALS,” Millan added.

Patricia holds a Ph.D. in Cell Biology from University Nova de Lisboa, and has served as an author on several research projects and fellowships, as well as major grant applications for European Agencies. She has also served as a PhD student research assistant at the Department of Microbiology & Immunology, Columbia University, New York.
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Margarida graduated with a BS in Health Sciences from the University of Lisbon and a MSc in Biotechnology from Instituto Superior Técnico (IST-UL). She worked as a molecular biologist research associate at a Cambridge UK-based biotech company that discovers and develops therapeutic, fully human monoclonal antibodies.
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Patricia holds a Ph.D. in Cell Biology from University Nova de Lisboa, and has served as an author on several research projects and fellowships, as well as major grant applications for European Agencies. She has also served as a PhD student research assistant at the Department of Microbiology & Immunology, Columbia University, New York.
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36 comments

  1. Kelly says:

    My mom, my everything, has no movement in her arms and legs. She aspirates on food every couple of weeks, her voice is weakening, and her hopes of getting this in time has diminished. This is great news for people who had the resources to be able to participate in the study. Unfortunately, my parents didn’t have the money or other resources to try this with a 50 percent chance of getting the placebo. To my family, October 2020 seems so very far away. I understand it takes time, but it there is always a state of panic in my family. My mom and my family need a miracle like so many others.

  2. Fatimah Ali says:

    We are waiting for the conclusive result .
    Great work and brain storm team showed great cooperation with our inquiries as an ALS patients ir relatives.great team work
    Thank you
    Fatimah Ali

    • Cindy says:

      2 years ago when this trial began I met all the criteria except age. I was 63 and the cut off age is 60. I’m not sure I understand why. But I hold out hope that this treatment will be a successful one for those participating. And that the FDA won’t take too long in making it available for all PALS no matter how old.

  3. Manuel Lopez says:

    I’m on radicava Now which was fast tacked at phase two testing. Hopefully Nurown will get the ok before next year. Most of us won’t make it till then.

    • P. Sharma says:

      My father is also suffering from ALS both his arms are affected. Doctor has advised Radicava for 6 months from Nov 2019 please suggest if its effective in reducing the progress.

  4. Steven says:

    Top line results in October 2020 is not acceptable FDA approval should have been granted a few months ago at 100 patients.

  5. Mary Christopher says:

    Are there any PLS patients enrolled in the study? Any thoughts on the effectiveness of this drug on PLS patients?

  6. This sounds very promising; however I thought I read that phase III would wrap up July 2020 now you’re saying October 2020. My husband is complete bedridden and fading away day by day. He has a feeding tube and a trach. Will this work for him once it becomes available? We have to small daughters and looking for a miracle. Praying daily this is the answer to our prayers. We have a huge team of prayer warriors believing and trusting for God to perform a miracle not just in the life our house hold, but others that are suffering with this awful disease.

  7. Narinder Ahuja says:

    My husband passed away within nine months of diagnosis. He had the bulbar kind. By the time he reconciled with the diagnosis, pneumonias created havock to his speach, swallowing and finally breathing. I wish I knew more about ALS then. Much education is needed about this fast degenerative disease.

    • Karen Riss says:

      So sorry for your loss, but do you mind giving more information? My Best friend’s Husband was recently diagnosed with Bulbar ALS but currently has few symptoms outside of speech,coughing, excess saliva…limited tongue movement. It was frightening to hear of your Husband going so quickly. Any information is better than none. Thanks in advance!

  8. Michelle Coiro says:

    What is the criteria to been enrolled? Is there an age cut off? Certain things the ALS patient needs to be able to do or not do? Does how far along they are play into this?

  9. Yana K says:

    It is so needed for all ALS patients who do not have this luxury of time! Shall we get the joint petition to get the treatment approved earlier before all 200 patients finished with the trial treatment. At the end of the day it is up to each patient to decide if she or he is ready to accept the risk.

  10. Gary M Sylvestre says:

    Why are any patients being injected with a placebo? This seems to be a barbaric and unnecessary FDA requirement! When this trial is over with, who is going tell the unfortunate ALS patients that they were just pincushions for a placebo? All 200 patients should be given the full treatment. There are 10,000 ALS patients who are not getting this treatment, they could be used as a baseline. Why does the FDA require this study to be a Double-blind study? Are ALS patients spontaneously reversing and curing their disease with sugar pills and saline injections? The FDA needs to update and streamline some of their Testing and drug approval processes. One size does NOT fit all!

  11. Thirulok Thilagar says:

    My dad has ALS and I can’t watch him degrade day by day. Any possibility of this treatment available to patients outside US?

  12. jan berkhey says:

    will the treatment given to patients in the Netherlands? and is the treatment available for all motorneuron deseases?

  13. Lynn B says:

    My husband has ALS! How can I get him to be part of this study. He is 51. Still walking and fighting everyday! Please we want to be part of this study. It needs to get approved faster!!

  14. Carolina Landey says:

    My friend is suffering from ALS, she’s no longer able to speak or swallow. She can still walk and move her arms. How can we get her in this clinical trial, she’s willing to try anything. She’s 52 yo woman with 3 children. Please advice.

  15. Rita Guerra says:

    Please if there is any way the FDA can approve this and make it affordable to the unfortunate my hubby is 48yrs old and this is devastating please please approve this or start another trial program we will take any hope!

  16. A. Strangis says:

    Whatever you can do to speed up Stage 3 and get this to the market. So grateful there is something with a positive result for Thurman Maynard and others. Please push the FDA with those stories so it can get on the market sooner. Thanks.

  17. Taylor Lyn Tyler says:

    My Dad has bulbar ALS and needs access sooner! He’s still walking and sort of talking but it’s getting harder and harder for him. Please, is there anything to be done to speed up approval or access? The study should have taken more than just 200.

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