Brainstorm Nurown 'Right to Try' participant shares progress

The FDA does not consider cost as an evaluation criterion for approval.

Radicava had a Phase 3 trial dataset; NurOwn does not as yet. They are testing a new dosage regimen, different from the other trials. You can't seriously expect the FDA to let Brainstorm rush it into the wild without testing.

As I read the Advocates' last series of FDA asks, they wanted an FDA staff member as ALS liaison, someone to finish Right to Try guidance and someone to finish the long-debated ALS drug development guidance.

IMHO, 2/3 of that agenda is a waste of time due to the economic and legal realities of RTT/ALS drug development, and the 3rd is window-dressing.

If they came up with a 4th ask around NurOwn that entailed pre-Phase 3 approval, I presume Brainstorm would have the good sense to stay out of that briar patch, even if the FDA was unexpectedly willing.

NurOwn has not to date shown "highly promising results," and it was always clear they'd have to up the "dose" to have the chance to get any, plus the data told them that they needed to stay <60 years old and stick with rapid progression. That's what this Phase 3 trial is.

You can't compare NurOwn with Radicava. There's no reason to believe you couldn't do both, though, if you're in the sweet spot for both, NurOwn is approved, etc.

Rant: Meanwhile, I would love to see the Advocates focus a few pixels on approaches that we discuss here every day, that actually have better data in terms of length/quality of life. If anyone thinks that the ALS clinics are doing everything that could be done to support respiratory and nutritional support, mobility devices and assistive technology, I have a Space Needle to sell you.

You just said to date there are no promising results. Here is a quote from Matt Bellina last week which I consider highly promising:

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in terms of the FDA rushing Brainstorm into the wild I want to repost the words of the FDA commissioner who said :

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I meant trial results, Par.

If one person was enough for a trial, why would we need hundreds?

But even if I didn't...what are some of the possible reasons underlying Matt's no-doubt well-intentioned statements?

What we see in ALS and other wasting diseases is that lung capacity can wax and wane depending on who does the test, fatigue, examiner encouragement, mouthpiece seal, etc.

What we see in strength testing is again examiner and fatigue can play a role.

But even past possible reasons for changing numbers that don't mean improvement, in trials, you almost always have some people who undeniably improve even when you give them water or sugar pills. It's called the placebo effect, and it's so real that some clinicians believe it accounts for most or all of the success ascribed to antidepressants, for example.

In most trials, some patients also get worse, and some are stable.

That's why you need lots of patients to see if there is a real effect, a reason to recommend stem cell injections to PALS around the world, who are different.

I truly cannot count the number of times P/CALS and even some clinicians have felt they had reason to say, "This is it!" and it wasn't. And I can refer you to agents like minocycline that later trials after "This is it" showed they made things worse on the whole, or that burdensome, risky treatments didn't help.

Cheerleading too early doesn't help anyone.

What more is needed than this to at least allow terminal patients an option to try? This is a patient after the 3rd injection of Nurown at the FDA meeting. Remember he had to be a fast progressor to qualify. Who can argue this is a placebo effect ?


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Brainstorm has stated that it can't support RTT for NurOwn -- that's how Matt became the token example. The FDA can't make them do it, either.

It's a lot of money, and it's a legal risk that the law doesn't fully address even if a PALS wrote their own check. Etc.

The key issue-- if there is NurOwn-caused improvement, we don't know that everyone will respond the same way -- or even that they won't be harmed. I'm sure you've seen the reports on deaths caused by stem cell therapy, and harms detailed here in the forums. It's not an aspirin.

NurOwn has had every concession from the FDA. It's now up to Brainstorm and the DSMB to confirm or deny the hypothesis that the new regimen can slow progression statistically more often than placebo, with a reasonable risk profile. If you want what works to be available for PALS worldwide, this is the path.

If/as we take single cases as gospel, because there is such a craving for hope (that all of us understand), that is how the quackery that has caused tremendous suffering in diseases from Lyme to cancer, takes root.

I'm happy that Matt's happy, but none of this is a substitute for doing the rest of the work.

OK. I understand you will delete anything positive I post so I will stay quiet. The link I pasted may have been appreciated by many though.

It’s always interesting and encouraging to hear a success story, especially when it involves a disease where failure is the norm. Thank you for sharing that link.

A story or two (or three) of treatment success is going to be remembered and referenced far more than stories of no response. It’s easy for us to pin our hopes on these stories. But we need to hear about the treatment failures too.

What really matters is the big picture, I.e. how well does the treatment fare in a large double-blind placebo-controlled trial? Such a trial may also suggest which groups, or clinical characteristics, predict a better outcome. And, importantly, which adverse effects are likely to occur.

Before we rush to embrace this treatment, we need that kind of data. We also need to know how long the treatment lasts, how often it may be necessary to retreat, how much it’s going to cost, and will insurance even cover it.

A note about why some links are removed- if they include solicitation of funds/ requests for donations on the linked site, they are not allowed, per the ALS Forums Terms and Services. ( Terms of Use | ALS Support and Help Forum ) It has nothing to do with moderator opinion of a subject.

If anyone wants to read more on this subject, it is being talked about on ALSTDI forum and on some of the Facebook groups.

Whether or not it ends up being a viable treatment for some or all PALS, I am hopeful.

I promise that link had no solicitation for donations, etc. In no way do I want to create trouble or disrespect the rules of the forum. I think we have heard about things not working endlessly for years. When someone goes to a meeting with the FDA after a diagnosis of ALS where he says he has improved in this trial and is running in the hallway....should we not show it ? I know some moderators and other people may not be convinced yet, but is that against the rules ?

It is unfortunately on the page that was linked not the post but it still is against the rules. I encourage people to look at Matt Bellina’s facebook page to see what par is speaking of. Par you may describe as I did. Just don’t link. It is interesting and hopeful though we still need actual trial results.

Given your diagnosis date, par, unless you are FALS you did not live the heartbreak of the failed phase 3 dex. Interim reports were so hopeful many of us expected approval within months before the final analysis was done

I am hoping for different results for Brainstorm, the ASO trials and some others.

Btw I shared the link with someone who works at my clinic( not a provider) They were interested to watch

Par, it's pretty clear-cut here -- links to FB pages that solicit money aren't kosher.

We all want to hear and share positive news. You have shared some. And Kim has pointed to other places to find more information. Since we know of PALS who have suffered and died from jumping into new paradigms, we'll continue to provide historical and scientific context around the good news.

Best,
Laurie

Thanks. In addition to Matt Bellina, I strongly encourage people to search on google: 'Annie ALS twitter' and click on the first result to see a video of Mark Bedwell who is participating in the Nurown Phase 3 trial and has received his 3rd and final injection. The video shows him running up and down a hallway and speaking clearly.

Thanks

Matt put the fda video on his fb page yesterday if you don’t have twitter and or need one stop shopping.

For those that don't know, we should also note that the NurOwn and many other interventional trials are recruiting PALS. I linked to the US list but those in other countries can filter on theirs.