NurOwn Phase 2 Clinical Trial Results Promising, BrainStorm Cell Reports

NurOwn Phase 2 Clinical Trial Results Promising, BrainStorm Cell Reports

BrainStorm Cell Therapeutics recently presented data from its Phase 2 clinical trial, showing that the company’s NurOwn stem cells technology may halt disease progression in patients with amyotrophic lateral sclerosis (ALS). The data were presented at the 27th International Symposium on ALS/MND in Dublin, Ireland.

The multi-center, randomized, double blind, placebo-controlled, Phase 2 trial (NCT02017912), assessed the safety and efficiency of autologous (self) transplantation of neurotrophic factors-secreting mesenchymal stromal cells (MSC-NTF, NurOwn) in 48 patients with ALS. NurOwn technology uses the patient’s own  MSCs, which are isolated from the patient’s bone marrow, and expanded and induced to differentiate into MSC-NTF cells. The cells with neuro-protective properties are then transplanted back into the patient. In the study, patients were randomly assigned to receive NurOwn cells administered via combined intramuscular and intrathecal (spinal) injection (36 patients), or placebo (12 patients). The study’s primary endpoint was safety and tolerability.

During the symposium, lead investigator James Berry, MD, MPH, reported safety and tolerability measurements demonstrating that the treatment was well-tolerated without causing adverse side effects.

Secondary endpoint results, also presented by Berry during the meeting, showed that treatment with NurOwn resulted in changes in the slope of Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R) score, changes in Slow Vital Capacity (SVC) scores and muscle strength, in positive responder analysis (the percentage of subjects who improved post-treatment compared with pre-treatment), and also led to favorable results in a subgroup analysis.

The results showed specifically that in the cerebral-spinal fluid (CSF) samples that were collected from patients treated with NurOwn, there were significant increases in the levels of neuro-protective factors, and a reduction in inflammatory disease markers.

In addition, the results revealed that a greater number of patients treated with NurOwn achieved the high threshold of 100% improvement in the post-treatment versus pre-treatment slope, compared with patients treated with the placebo. Basically, these results mean that patients’ symptoms were halted or they achieved a positive improvement on their ALSFRS-R score. This effect was even more pronounced in the subgroup-analysis that excluded patients with slow disease progression.

“This Phase 2 trial demonstrated clinical meaningful improvements in disease symptoms as measured by the well-established ALSFRS-R scale,” said Berry, unit chief of the Massachusetts General Hospital ALS Multidisciplinary Clinic, in a press release. “Importantly, there is evidence that NurOwn may be halting disease progression or improving symptoms in some patients. The CSF biomarker profiles were also encouraging,” Berry said. “The significant increases in neurotrophic factors and decrease in inflammatory markers observed in the treated group post-transplant provide a biological mechanism supporting the observed clinical effect,” he added.

21 comments

    • Tim Bossie says:

      We certainly understand your question. It is hard when there is all this great news, but it is all research or experimental. We can only hope that these things are able to come to fruition very soon so people can use them.

  1. Mike Leist says:

    I have ALS, and these results are very encouraging. What will be the likely process and timeframe this treatment would be available to a wider population of patients?

    • Tim Bossie says:

      Hi Mike, we do not know any timeline right now. Of course, when we learn more about this research and availability we will be reporting it. Stay tuned!

  2. Robert Sanders says:

    I live in Oklahoma and have ALS how do I get into trials and studies.
    How soon can we get some relief ALS can be a short term, we need something quick.
    Is there one place to get updates, I go to several sites

    • Tim Bossie says:

      Hi Robert! Thank you for the comment. We do try to deliver the most relevant, and newest, research news and info on our site. 🙂

      • Kim Persaud says:

        What will it take to run a TV add about Als. So the world will know about this disease. You can get more donation that way.
        How much money and time will brainstorm needs to get Nurown to all Als patients.
        I am tired of sitting and waiting. Please answer my question.

        • Tim Bossie says:

          I really couldn’t tell you what it takes to take out an ad on TV. There was a lot of awareness during the ice bucket challenge and news programs, television shows and social media videos. We are all impatient when it comes to finding a cure for ALS and other diseases. The damage that is being done to people’s lives, and their families, needs to stop quickly. All we can do is our part in raising awareness and helping researchers do their jobs.

  3. kj kerl says:

    Daniela, the most important variable in this new study summary is missing. Please post the time period (how long) these patients were treated with MSC and how long their FRS scores trend were followed. In too many studies, patients are only followed two or three months, which is not very significant. Please tell us that their FRS’s stabilized, or improved, for over a year or more. Now THAT would be cause for celebration. Keep us posted. Thanks, Klaus in Seattle.

  4. olga torres says:

    this is great news! but we need acceleration in the process, treatment research should be a priority…I seen many people die of this horrible disease and many more to come, I was just diagnose with als at 38 and is sad that everyone suffering of this monster should wait patiently!

    • Tim Bossie says:

      Absolutely right! There does need to be some acceleration in the advancement, and approval, of treatments. However, there is a lot of movement in research – more than just a few years ago – so that we will hopefully see new things coming soon.

  5. Kim Persaud says:

    Agreed with all the comments. I read that edaravone helps to slow progression. IT is waiting for Fda approval, which i§ schedule to be approve by June 2017. I need that drugs now. What can we do speed up the approval. Japanese are using it.
    Do we get all als patients to lobby for treatments. I willing to do what ever it takes to get treatments available.
    Can this organization help us.

  6. Olga torred says:

    Thank you ! Thank you ! All als patients are desperate for a possible treatment option 😭 This is soooo scary and devastating we need hope we need life we need help !

    • Tim Bossie says:

      Yes, we all need some hope that these new procedures and treatments not only look promising, but actually deliver results for all of those who are struggling with ALS.

      • Herb Van Gundy says:

        My name is Herb, I was just diagnosed with ALS because it took five years for doctors to put all my injuries together, finally one doctor was smarter than others and had me tested. I’m still trying to wrap my brain around the whole thing,I’ve been in great shape all my life, semi pro body builder now I’m turning into a shell of what I was . My family is very supportive but they are struggling with it .the news is great to hear but like everything else for me and others it’s a pipe dream . We will probably pass away before the bureaucratic BS let’s it come to us . If somebody isn’t making money then they really don’t care about us . Thank you for the hope anyway.

        • Tim Bossie says:

          Hi Herb. Yes, it is incredibly frustrating when those we look to to help us are the ones that fail us many times. I am sorry that the doctors couldn’t give you a correct diagnosis. So much time wasted. And yes, the BS from bureaucrats can be quite thick at times. All we can ever really do is hope that something drastic will happen soon.

  7. Val nakamoto says:

    How do you become a trial patient? A friend of mines has been fighting this decease for over 2 years and his progression continues even after stem cell treatments. He keeps getting worse and his speech is really bad. He really needs help now, not sure if he’ll make it until June or July 2017.

    • Tim Bossie says:

      If you want to take place in a clinical trial when/if that trial is available in your area. Talk to your friend’s doctor or pharmacist if this trial is in your area.

  8. Kim Persaud says:

    I have been reading people stories about Als on patientlikeme. And it’s very sad. .. Most of the people are trying treatments like Lunisan, Deanna protocol which are very expensive. It breaks my heart to see people suffering and there are treatments out there which they can’t get. Someone have a heart make something happen.
    Edaravone has been approved in Japan.Can
    You help in getting the Fda to approve it before June 2017. This
    Will slow the progression.

    Thank you
    Kim

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