Nurown

[Dee Dee 0617]

Catalent and BrainStorm Cell Therapeutics Announce Partnership for the Manufacture of Mesenchymal Stem Cell Platform Therapy NurOwn®

SOMERSET, N.J. and New York – October 22, 2020 — Catalent (NYSE: CTLT), the leading global provider of advanced delivery technologies, development, and manufacturing solutions for drugs,...

ir.brainstorm-cell.com

Don't know where a lot of you stand where this therapy is concerned, but I am hopeful, conservatively, but hopeful

 

[Nikki J]

I am hopeful too. One of the pi s from the study gave a webinar last night in which she said they hoped results will be published by the end of next month. This also seems like a reason for optimism

 

[rmt]

We are hopeful too. This all seems like good news.

 

[Scotiaspirit]

Cautiously optimistic but still probably 1 1/2 years from approval for use in Canada

 

[Greerguy]

Brainstorm has entered into a partnership with a company for mfg in Texas once the FDA approves it.. Patents in US and Japan Europe. . We are going to have to watch what "fast track" means once results are released next month.

 

[lgelb]

Just a caution that the results scheduled for release next month are topline, not final data. The study period for efficacy is 28 weeks post-first dose. Last dose was in late June or early July and the three doses were two months apart.

Also keep in mind the trial was limited to rapidly-progressing PALS because those were the responders in Phase 2, but no one using BiPAP or feeding tubes was eligible, among other exclusions. The protocol summary is here.

The FDA has committed to an expedited statistical review but to incorporate the final data and receive/process the full BLA, which will include required assays separate from the clinical trials, even in installments, will take several months.

NurOwn approval, presuming it happens, will be a big step, but it will not be right for everyone, and there is a lot else going on. Other trials are recruiting. I encourage everyone to consider all the possibilities.

 

[Nikki J]

Nurown said the last dose of the last patient happened 7/7/20 and they had12 weeks of data collection to go. This sounds right because there were 2 prior doses 2 months apart so the last data collection would have occurred around the end of September. The first dose of the 7/7 patient would have been early March. I don’t know what will see when but they have all the data they are going to get from the trial

 

[lgelb]

Yes, what I meant is, you have to clean the data, clear up missing values, etc. on an ongoing basis, but the last phase of that process will be as the last efficacy data drops this month. So not all the data will be clean/final as of the topline.

Also, I thought a map of trials might help.

 

[Nikki J]

Still not getting you “- last efficacy data drops this month” ? Meaning what? Last efficacy visit happened in September. Of course they have to process , clean up etc and it is a lot of data and they will do topline first but all the data that will exist has been obtained

 

[lgelb]

Well, not to quibble, but to fill in missing values or clarify suspect ones, the data will change. So let's just say, the database isn't locked at this point.

Anyway, the topline will be fully directional and very likely positive, since they increased the number of doses from 1 to 3 and kept the population the same, and there were no safety concerns in the DSMB review.

I guess my point, buried in there somewhere, was that because of the expense and invasiveness of the treatment, coupled with the narrowness of the trial population, I do not expect this to be made available to as broad a population as the other two therapeutics, so PALS esp. in mid-disease stages should continue to pursue other avenues. 33 interventional trials in the US are recruiting.

 

[Nikki J]

I completely agree that eligible people should pursue trials. I have tried to encourage this. Nurown is going to be a tough sell for insurance companies and we all know what happened with Radicava but even if all goes perfectly people aren’t going to get it instantly.

 

[Dee Dee 0617]

I'm sorry I bought it up. I can't seem to find my place here. Thanks Nikki for sharing, you're a wealth of knowledge. Lgelb, I get it you lost someone, so you think you know, you don't. I wanted to be just fleetingly hopeful. I was happy that there was even an article. Nikki got it, Spirit got it, Karen would've got it. Then there is your harsh light of reality . Nikki, God bless her, tried valiantly to lessen the impact of your statements. You just wouldn't catch the rope she was throwing you in the deep end. Nope, you had to make sure the people who are actually doing the dying here, understood it, this treatment was not for us. Are you still going to be pulling the next generation of ALS diagnosed patients threads of hope away, after we're dead. You can recycle all of your righteousness every 5years. Lift a glass, of course you're the only one here who can.(paralyzed humor, in case it was lost, on your healthy self) Here's to you, your correct, this treatment is years away from being used for any of us in any meaningful manor. If you think I'm mean, insulated your tender sensibilities. You eviscerated me. I will be banned now, but you'll continue to kick over sand castles.

 

[EricInLA]

This disease is frustrating, so we all get a little room to vent - that’s the beauty of this forum. But Laurie’s opinion of Nurown does not warrant this kind of attack. As one of those dying, I don’t see her opinion - while it might be conservative - as eviscerating hope, but rather providing a dose of reality and context. Who knows, she might be wrong, but Laurie is also a wealth of knowledge and gives generously of her time on this forum. We should all appreciate that, even if we differ on certain things.

 

[Fusia]

Each of us here (PALS and CALS) is on our own path - levels of optimism about therapeutics is going to vary greatly. That's the beauty of this forum, where we can learn from each other no matter which path we are on.

I went through the ups and downs of optimism and dashed hopes for a "silver bullet" to treat Alzheimer's disease - my husband died 13 months ago after living with it for six years. I learned through that to temper hope and optimism with the reality of how complicated these diseases are.

 

[KevinM]

Dee Dee, I so understand (as do we all) the need for hope. The lack of hope with this f—king disease is emotionally crushing, and that as much as my slow but steady progression is a big chunk of my depression and anxiety.

Nurown has received so much attention among our tribe, mostly because numerous trial participants who believe they received the actual treatment have come forward to talk about their improvement or dramatic slowing of progression. I get caught up in their testimonials and admit to being (conservatively) hopeful like you. I do believe that the final data will be quite positive.

I have also occasionally been a bit deflated after reading about “exciting new, game changing” articles, only to have Laurie give much needed (for me, anyways) reality checks on timeframes and obstacles these potential therapies face. I would rather give my family an honest assessment of where things likely stand than fill them with potentially unrealistic optimism.

For me, Laurie’s practical knowledge from years In the industry and being the caregiver for her partner is invaluable to this forum, even if the news is not what we may need or want to hear. The respect I have for her continued participation in this forum years after her spouses death is great.

No, she isn’t a PALS, so I get what you are saying. But that doesn’t diminish the incredibly generous sharing of her time and continued service to the forum. I would want to put ALS as far behind me as possible if it were my partner that died from this monster, yet she does not. I for one am grateful.

As with the election, let’s try to be somewhat patient and see where the final Nurown data takes us. Maybe, as Eric said, her conservative timeline might be wrong, given the large grassroots effort to speed access. But killing the messenger—even if the message is not what we would like to hear—is not the answer.

One final thought: I love your unfiltered, heartfelt comments. Please do not stop. Your thoughts, emotions, and amazing self-reliance are too important to me and I’m sure many others here. Love and respect, Kevin