Israel’s BrainStorm Offers Encouraging Phase 2 Data on Stem-Cell Therapy NurOwn to Treat ALS

Israel’s BrainStorm Offers Encouraging Phase 2 Data on Stem-Cell Therapy NurOwn to Treat ALS

Israel’s BrainStorm Cell Therapeutics has announced positive top-line Phase 2 data on NurOwn, a customized stem-cell therapy technology to treat amyotrophic lateral sclerosis (ALS).

BrainStorm has been developing and commercializing NurOwn in partnership with Ramot, the business engagement unit of Tel Aviv University. NurOwn enables the production of growth factors that support the survival of neurons, a critical step in treating neurodegenerative diseases.

 

In July 2016, BrainStorm announced positive results from its phase 2 randomized, placebo-controlled study of NurOwn in ALS patients, and presented the results at the 27th International Symposium on ALS/MND in Dublin, Ireland. That study mainly showed that NurOwn was safe and well tolerated. It also achieved multiple efficacy endpoints when given to 48 ALS patients, including the ability to halt or reverse disease progression over the course of a six-month follow-up.

Subsequently, BrainStorm received U.S. Food and Drug Administration (FDA) permission to start a Phase 3 trial. This will be a randomized, double-blind, placebo-controlled multi-dose trial conducted at multiple sites in the United States and Israel. Patient enrollment is expected to begin later this year.

“Following our end of Phase 2 meeting with the FDA, we are making final preparations to begin a Phase 3 clinical trial, which, when successful, will position us to bring to ALS patients and their families a much-needed and innovative ALS disease modifying therapy,” BrainStorm President and CEO Chaim Lebovits said in a press release.

The company plans to use cryopreservation to enable repeated doses of autologous NurOwn from a single bone marrow aspirate.

The FDA has already accepted key elements of the Phase 3 program to support a Biologic License Application (BLA) for NurOwn, which is a necessary step in commercializing the product.

Separately, BrainStorm announced plans to partner with City of Hope’s Center for Biomedicine and Genetics in the Los Angeles area to produce clinical supplies needed for its Phase 3 study. That study also involves Israel’s Tel Aviv Sourasky Medical Center.

41 comments

  1. Kim Persaud says:

    Please I will like to part of the phase 3 treatment. Please let me know how I can make this happen.
    Thank you.

    • Jose Marques Lopes, PhD says:

      Dear Kim, thank you for your comment. I would recommend that you talk with your clinician about the possibility of taking part in the phase 3 study. He/she will certainly be able to help you more efficiently than we are.

  2. Karen says:

    When you say to talk to your clinician are you referring to in the USA, can a person from USA be considered for treatment in Israel under compassionate use?

    • Jose Marques Lopes, PhD says:

      Dear Karen, the original information from BrainStorm states that the phase 3 trial will be a multi-center study, performed in both US and Israel. Thank you.

    • Jose Marques Lopes, PhD says:

      Dear José,

      The phase 3 trial will be done in Israel and the US. We are a news service and we don’t have more information beyond what you read in the article and what BrainStorm provides. I would suggest you discuss that possibility with your clinician. Thank you for your message.

  3. david joki says:

    What is the procedure to get on a trial volunteer list? Do we need to have a treating physician contact someone from this agency? Thanks.

  4. Steve Heyworth says:

    ALS patients with a ALSRS less than or equal to 35 should be aloud to take drugs/treatment that have gone though a phase 2 clinical trial. We are running out of time! Endaravone is another good example.

    • Jose Marques Lopes, PhD says:

      Dear Bev, thank you for your question. The information we have is all in the article and in my reply to Karen. I would recommend discussing the possibility of participating in the clinical trial with a clinician. Thank you!

      • Bev says:

        Karen is US citizen. Would a Canadian citizen be accepted in the US study. If not, will there be a trial in Canada soon. I will refer to my clinician when I know the answer.

        • Jose Marques Lopes, PhD says:

          Dear Bev, we are a news service and we usually don’t have more information beyond what companies provide in their press releases, their website or the clinical trials pages. This trial will be performed in the US and Israel, and we don’t have any indication of a similar study in Canada. Regarding acceptance of non-US citizens, that would depend on the eligibility criteria, which are not available yet. Thank you.

    • Cynthia Dixon says:

      I agree…although we all realize trials are necessary, this beyond horrible disease is deadly enough that it should be given extra speed with patients accessing it. I feel once it’s into phase 3, it should no longer be a placebo trial. They’ve established it’s safe by then. We are so close to a treatment. Please, help push this through.

    • Nim says:

      We would like to know about this trial as well, my husband has just been diaagnosed last week with als so I really somehow something has to happen

  5. Emma Getliffe says:

    Hi I would also like to be considered for the Phase 3 trial. I will talk to my specialist here in France 4th May and see what he says.

  6. John Tallis says:

    Amazing what you can find..
    I hope all the research comes through soon for us. Had lots of dogs over last 40yrs and wish I could get out and about walking. A son doing the London Marathon on Sunday and got 3k for MND

  7. Regina Parker says:

    Can you tell me what the criteria is to be included in the phase 3 trial? i would like to take part but gather I will need to get my neurologist to make the application.

  8. Azzar says:

    Can you please tell me when the phase 3 trials will be completed? I just turned 33 and still holding onto hope I can survive this. Thanks.

    • Jose Marques Lopes, PhD says:

      Dear Azzar, we don’t have that information yet. We will definitely report the phase 3 trial results once they are published. Thank you.

  9. Chris Gargaro says:

    My wife would like to be considered a a participant for the study. She was recently diagnosed ALS earlier this month. How does she get on the list of candidates?

    • Jose Marques Lopes, PhD says:

      Dear Chris, I would suggest discussing that possibility with your wife’s clinician. We are a news service, so I am sure he/she would be far better-qualified to provide you with that information. Thank you.

  10. HappyERdoc says:

    IMHO, everyone interested should contact Brainstorm and their congressperson! There is power in numbers!
    http://www.brainstorm-cell.com/patients-caregivers/patient-access-to-nurown/

    “Brainstorm is exploring the possible commencement of a program to make NurOwn® available to a limited number of patients with Amyotrophic Lateral Sclerosis (ALS, also known as motor neuron disease) through a regulatory pathway designated for an orphan drug with a good safety profile and strong efficacy signals. This program would potentially provide patients, who today face an unmet need, with access to this investigational treatment before it receives final marketing approval from the regulatory authorities.”

    • Edward J. Mueller MD

      What has happened with this initiative. I printed out the notice from the Brainstorm website in June, but have seen no further information on this. I think it is unethical to deny this therapy to patients when the efficacy shown in the phase 2 trial has already been presented at a major medical meeting and disseminated via a webinar. Time is critical in this disease.

      • Cynthia Dixon says:

        I totally agree. We are intelligent people here, but enough is enough. Unfortunately, this entire journey has made me feel less competent with the medical system. This is such a horrible disease. How can we make a difference?

  11. Steve Cliff says:

    This is the only real treat of hope that I’ve seen in the three years since I’ve been diagnosed. I can’t get through to Brainstorm. I’m guessing they are inundated with calls of interest.

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