Are you keeping up with Nurown and the push for FDA approval?

[Alan Larrivee]

The link you provided was very compelling. But the FDA said there’s no evidence of effectiveness. I’m confused. Boy would I love to slow this train wreck down!

[doug-frazier]

I worked at a large pharmaceutical firm for 25 years at corporate headquarters. I saw the work that went into these FDA submissions.

As a PALS now, I understand fully that all of us would like some hope.

But when a company has put all of their resources behind a drug for years and the trials do not produce results that meet the FDA’s criteria, then this is not hope.

I don’t think it makes sense for us to push the FDA to allow us to take these. This isn’t hope and it could shorten our already short lives.

For example, Radicava and Relyvrio are not approved in Europe. They don’t allow manufacturers to meddle in the approval process.

Here we then allow them to sell these failed drugs to PALS at exorbitant prices.

I hate that there is little hope for me at age 66 but I also don’t think false hope is the answer either.

However, the manufacturers deserve a fair hearing on any questions too because drug trials are so complex.

So, I am pulling for Nurown and every other potential solution. The Nurown patient stories are moving and it would be great to see if those patients could get access to see if the results continue.

With that said, if the drug is as good as the stories relate, we should see this in the broader results as well. But they don’t show up for some reason.

In the end I think we should rely on the pure science as frustrating as that may be.

[Alan Larrivee]

I do support science and the FDA. I trust they did due diligence.

[doug-frazier]

My comment was intended to be to the overall question, not to yours specifically. I was supporting your comment. Sorry for any confusion.

[Dagmar]

I agree that too much time and money has been spent on the Nurown campaign & research. Turning stem cells into nerve cells is not what we need – – we need stem cells that can become motor neurons – – and no one has done that yet, outside of a petri dish. We have enough treatments already that “slow” progression. We need to focus on stopping progression.

[doug-frazier]

Dagmar

I submitted another reply to yours that I don’t see.

I checked forums, messages, etc.

Am I not understanding something?

Thanks

[Dagmar]

Doug, your reply on Oct 3rd is here in this discussion. did you write another more recent one? Dagmar

[doug-frazier]

Yes. I wrote one in response to yours.

[Dagmar]

Sorry, I don’t see it – – and I even looked in the back of the forum software. You’ll have to post it again.

[doug-frazier]

Thanks for checking.

It figures it was a lengthy one!

[doug-frazier]

I posted it in research topics.

I would appreciate your experienced viewpoint.

Thanks again.

[richard-l-wheeler]

I do not follow it. Waiting for proof for something that addresses the problem and not the symptom.

[Amanda]

I asked one of the doctors that I see about Nurown this week. I got the impression that his thoughts were similar to Dagmar’s. I asked about the testimonials from pALS and he said he would need to research it more. The team of medical professionals that I see are amazing and professional. I didn’t expect a hard yes or no on Nurown. My impression was, they weren’t that optimistic and they wanted to see more research and facts before saying to much.

Dagmar, I agree. We need research targeting cures or at least ways to STOP the progression, not just slow it down.

[Dagmar]

A small change (in the ALSFRS-R*) in 34% of the participants is not enough to support going forward with Nurown.

*The ALSFRS-R is not a reliable measure in my opinion. It’s good for initiating discussion when in the doctor’s office, but due to it being mostly subjective, it has the potential to be skewed.