BrainStorm Cell Therapeutics – NUROWN – Disappointing news but…

[Mario]

Something is not right. In October Brainstorm claimed to have great results on this trial and after a month says otherwise.
don’t joke about people’s lives, they’ve given false hopes.

[Cindy]

So as explained to me by a doctor in this study the placebo group performed better than expected. Placebo group did not decline as quickly as expected. Those on NurOwn did improve. So statistically unsuccessful but scientifically successful. Also biomarkers were identified in the spinal fluid. NurOwn not over yet. They are figuring out how to proceed. Cautiously optimistic.

[Duane]

Cindy, I just don’t believe him/her.  It’s a case of smoke and mirrors.

BrainStorm Cell Therapeutics stock dropped significantly today, due to some disappointing results of a phase 3 clinical trial for its leading candidate treatment (NUROWN). The President of Brainstorm said : “We are committed to advancing discussions with the FDA to identify regulatory pathways that may support NurOwn in ALS.”
It’s stock crashed today. Down 67%. Reason: release of negative top-line results from a phase 3 clinical trial for its leading candidate treatment. NurOwn failed to achieve statistically significant efficacy results.

Read this short article and see what you think about the whole affair. Just know they have been working on this for 10+ years!

https://www.biopharmadive.com/news/als-cell-therapy-brainstorm-trial-setback/589205/

https://www.nasdaq.com/articles/why-brainstorm-cell-therapeutics-stock-imploded-today-2020-11-17

[Dagmar]

This statement caught my eye: “…the placebo group performed better than expected. Placebo group did not decline as quickly as expected.”

Perhaps money and time should be directed instead to studying the placebo group – – and what they have in common that slowed their decline?

[Duane]

The Pharma people seem to have an excuse for just about everything.  They have a lot invested and so it is understandably so.  Now it seems the are on a salvage mission.  I agree with Dagmar that money should be spent on people who decline slower.  Although I do not necessarily believe  ‘the placebo group performed better than expected’.  They may just be using that as an excuse, just as they are now saying the number of advanced cases was more than they expected so to lead to this diminished result.   I wish the results were otherwise, but they are not.  It went through extensive, well-controlled clinical studies especially phase 3.

[nicole lievano]

ALS and every disease in the world originate from an emotional shock. If placebo group got to slower decline, it was because the patients believed they were getting the treatment and not placebo, therefore I believe there should be psychological trials for this because the pharma is clearly lost about ALS. I agree with Dagmar, if Nurown results are not what everybody expected, money should be spent on studying the placebo group.

I have experienced that when I´m stressed symptoms automatically rise.

[Duane]

What annoys me is that Pharma took 10+ years to reach this conclusion that should have been reached in half that time at most. I too think that there are Psychological aspects to this disease.

[Cindy]

Well I for one found myself I low spirits after all the hype then disappointing results. It’s the same with Amylyx. Do we really have to wait for another trial?  And the Right to Try- where has this gotten us?

[Duane]

The best we can hope for is AMX0035 which probably has a positive effect to some degree from what I’ve read.  I use Riluzole and that combined with 0035 and something in the future may help.  0035 has GI side effects for some people (diarrhea).

[Lynn]

So am I reading this correctly?

”I agree with Dagmar that money should be spent on people who decline slower.”

Are you saying those with fast progression should be put aside and only those with slow progression should be considered for treatment??

I’m sorry, this comment just seems a bit calloused.

[Dagmar]

I think the original comment got misconstrued…

Research dollars should be dedicated to studying both fast and slow progressors – – but in the case of Nurown’s research, it is worth looking into the placebo group as they seemed to have a slower progression. Every stone needs to be overturned.

[Dean]

When I first read this article, I was disappointed, sad, mad, and let down to only name a few of my emotions.  Thinking back to when I received all my testing to confirm if I had ALS,  I got a spinal tap… The strange thing was the day after my spinal tap I got a CSF leak from moving furniture the following day (Horrible headache).  The surprisingly strange thing is my ALS symptoms seem to slow down slightly after the leak which finally clotted after several days.  Maybe having the CSF taken out every couple months has something to do with the slow progression group?  Just a thought!  Throw everything against the wall and see what sticks.  I also agree that mental stress effects my progression dramatically.  Our brains and emotions play a part in this decease for sure.  I pray we all get a working treatment in our life time and eventually a cure.  God Bless all pALS!

[nicole lievano]

I´ve been following Nurown for a while because I joined a group in Facebook named World Fight Against ALS, they are fighting for Nurown´s approval by the FDA, even if it only worked for a specific group of pALS. It could be the first treatment for this disease after 155 years and we should be happy for it. Brainstorm is already in active discussions with the FDA and it could take years, this group started a movement  calling for Expedited Approval of this treatment around the world, of course starting by the FDA since phase 3 was conducted in the USA. I signed to back this movement and encourage you all to do the same at https://www.change.org/p/fda-approval-for-life-saving-nurown-for-als-patients

I’m not into the group best favored by this treatment, but ALS has opened my eyes to the meaning of life, it has been an Infinite learning opportunity <span lang=”en”>opportunity towards love and brotherhood. </span>

Changing subject, I recently read about a spanish doctor who is also a pALS, he has done research and tested alternative medicine treatments on his own body since western medicine has not found a cure. A few years ago he tried chlorine dioxide, a mineral substance that has caused controversy since it´s an ingredient for many cleaning products. It improved his condition being able again to eat and swallow without choking, sleeping on a flat bed, improving his speech and movement. Of course all under a controlled protocol because it could be fatal. I don’t think it is approved by the FDA for human consumption, but wanted to share with you all this information,

[Dagmar]

Nicole – – yes, it is frustrating to see promising clinical trials producing poor results.

But I caution you in passing along unproven, alternative treatments: reading about a Spanish doctor (with no name) who had a patient (no name) with undocumented ALS, who used a highly dangerous product – – that improved all his symptoms… if it sounds too good to be true, it is. 🙂

Again, I know it’s frustrating.

Here’s a reminder (with a little humor): http://alsandwellness.blogspot.com/2016/04/auntie-ellis-gets-cured-als-humor.html

[Duane]

I have decided that I will only use a drug/treatment if it approved by the EMA (European Medicines Agency) and the FDA since the FDA has a reputation for being too ‘easy-peasy’.

[nicole lievano]

I´m sorry you all,  Dagmar you´re right. I´ll try to find the article, but it´s in spanish.

Dagmar,  could I send it to you before so you can read it and see if it can be published?

If so, please tell me where to, email. Thanks.

[Dagmar]

Not a problem Nicole  🙂 – – I’ll send you a private message with my email.

[Siok Tan]

The announcement from Brainstorm is certainly very disappointing. My husband has ALS for 6 years, and we were so hopeful for positive results from the trial. Looking at what little results/data were announced, it looks like the trial did succeed in treating the patients in the treatment group. If we only look at the data from the treatment group, the expected treatment response rate of 35% was attained showing that the treatment works! The issue lies with the data from the placebo group who did better than expected. Many reasons could have resulted in this. Firstly, patients who participate in trials somehow have a tendency to assume they are on the treatment group. I am a clinical scientist who runs clinical trials (not ALS trials), and there had been many instances when our patients “believe” that they have the treatment, only to discover that they are on the placebo group (we did a survey with our patients and were able to show that patients tend to think they are on the treatment group). With the ongoing hype about how NurOwn is able to reverse the disease, the placebo patients who believe they are on the treatment group will “think” they are feeling better. The next issue lies with the fact on how their progression are measured. Both primary and secondary endpoints in this trial rely on this one questionnaire ALSFRS, which can be rather subjective. There is currently no objective way to measure ALS progression (unlike some other diseases where you can do scans to objectively see how the disease has progressed). However, one way that allow us to look at the data objectively is to look at the biomarkers measured in the trial, which according to Brainstorm, shows “statistically significant increase in the levels of neurotrophic factors and reduction in neurodegenerative and neuroinflammatory biomarkers that was not observed in the placebo treatment group”. This means biologically, things are going the way it should be in the body! The last thing we need to remind ourselves is the fact that for some patients, final follow-up visits were done remotely due to COVID-19 pandemic. I don’t know if this will affect the data, but if the person administering the questionnaire is unable to physically see the patient, will that allow the patient to provide inaccurate data without the research coordinator being there to “judge” if this was true? One interesting thing that can be done is to ask the patients if they thought they were in the treatment or placebo group, before they are unblinded.

I am sorry this is such a long message, but I wanted to bring some points across. Please don’t lose heart, as NurOwn is still a possibility. My question is, how and when. Does anyone know if any actions were taken to persuade FDA to take actions ASAP? I know there is a bill that needs to be passed to allow ALS patients access to experimental drugs, but other than that I have not heard of any petition or actions. Does anyone have additional information that they can share? Thanks.

[nicole lievano]

Hello Siok Tan, the group I mentioned in the past post, is also asking pALS to send a letter to many congressmen. In their Facebook page you can find the letter and the emails to send it.

[Duane]

‘Life saving Nurown’ ??  I don’t think so, since the the science doesn’t support that.  I don’t sign any petition because they need to do the work necessary to determine if it is truly efficacious, and to what degree, without all the background noise.

[Amanda]

This is a great discussion with a lot of information from our community.  I hope we continue to see research and information about NuOwn and any other possible treatment for ALS.  Please continue to provide scientifically based information about treatments.  If you have questions or need help just let us know!!