Ques. on Brainstorm (NurOwn Trial)

[Thomas933]

Hello: I am a cALS for my husband. I have questions about the Brainstorm Cell Therapeutics (Trial of NurOwn for ALS):

1) What are the pros and cons of this trial?
2) It seems to have promising anecdotal info. -- why aren't more people doing it?
3) When will results be published?
4) When could this treatment be available?

Thank you so much for helping. I've gotten a lot of differing opinions on this and am always looking for more.

 

[Nikki J]

It is discussed elsewhere here too.

It is promising yes.

Not everyone will qualify there are age and symptom limits

It is placebo and invasive. Not everyone is willing to do that combo especially as an open label extension does not so far seem likely. You can’t use radicava or have a feeding tube and there are other medical exclusions

I rather thought there were waiting lists to get in at one point? I do think not everyone qualified who tried. I have never explored it for myself ( symptom length disqualifies and hoping for gene therapy anyway )

Once the last patient is completelt through the trial they will collate results and present/ publish which takes a few months usually.

Assuming positive results it would go to the FDA If it looks good they might push it through fast but again we are talking months for that process

Are you considering it? I likely would do it if I were SALS and qualified

 

[KimT]

I would have done it but symptom length disqualifies me, too.

There are Facebook groups discussing it and other forums (ALSTDI, for one.) If you do a search here, you'll probably find more discussions.

I think it looks promising....sure hope it is.

 

[Thomas933]

Thank you Nikki -- that is good info. We are not considering it right now because my pALS is trying Radicava and the Centaur trial. Centaur is open label and he wants to get the medicine even if it's delayed by 7 mos.

I just hate passing this one by but it simply does not seem like a match for us at this time.

It's so unsettling to think there is something out there that could help people and they cannot try it. It's just not right....Plus, people who get placebo should be able to get the treatment after the trial is over.

Thanks again, hope you are having a good day.

 

[Thomas933]

Kim - I'm curious about your statement that it looks promising . May I ask where you are seeing that? I just feel like I am late to the Brainstorm party and I am playing catch up.

Thank you.

 

[KarenNWendyn]

There was a previous thread on this.

https://www.alsforums.com/forum/als-drug-trials/41146-nurown-stem-cell-phase-3-study.html

I seriously considered participating in the trial and discussed it at length with a study coordinator. My summary of that discussion can be found within that previous thread.

I decided against participating because of how far I lived from the nearest study site, the number of visits involved, 50% chance of getting placebo, and (very importantly) no guarantee of being able to receive active treatment at the conclusion of the study. That was a real dealbreaker for me. Most placebo controlled trials have what is called an open-label extension where all participants (including the ones that got placebo) will get active treatment.

The study coordinator explained to me Brainstorm chose not to have an open label extension because the bone marrow samples of the placebo patients are thrown away once they are received due to the tremendous cost of storing bone marrow samples at extremely cold temperatures.

Also, if you need treatment for complications caused by the study, it’s on your dime. You are also financially responsible for the initial visit which is to see if you even qualify for the study.

The phase 2 study, done in Israel, looked promising, which is why it went to a phase 3 trial.

 

[Par220]

The Phase 2 study was actually done here in the US

 

[lgelb]

Two Phase 2 studies were done in Israel, one in the US.

 

[KimT]

Kim - I'm curious about your statement that it looks promising . May I ask where you are seeing that? I just feel like I am late to the Brainstorm party and I am playing catch up.

Thank you.

Thomas,

Go to ALSTDI discussion forum. One person was given the treatment who wasn't in the actual study. He is posting over there. There is an ongoing discussion that will quickly catch you up.