Brainstorm Nurown 'Right to Try' participant shares progress

[ThroughThatValley]

This is very interesting I have to say my late sister regained some finger movement when she was far more advanced than Matt. She wasn’t getting nurown. She had been taking off label retigabine for a number of months. We hoped it was that and increased the dose but no further improvement. I think it just happened by itself.

I am not saying this is the case with Matt. I hope it isn’t

Nurown isn’t going to allow more Compassionate use I don’t think at this point they have interest in going to the fda and asking for approval either If they don’t the fda isn’t going to call them up and ask them to.

I get that is incredibly hard to see something that might help you and you can’t have it! Even more when you see the timeline and think it will be too late even if it goes through the process.

Spot on. The impression we are dying on the very last days of the war is nauseating. I remember about a documentary about the first world war. One of the last french soldier killed before the war ended had be sniped down when he was on his way trying to help a fellow soldier wounded on the no-man's-land. I remember thinking aloud: "now, how of a bad luck is that!".

 

[Lkaibel]

The Radicava phase III trial involved few subjects and questionable methodology. Personally, I think cash was the heaviest motivation in putting it out there. I would further suspect that there is some cash based motivation in holding up NurOwn.

Superficially, it would seem there were many dollars to be made off a NurOwn approval but I have learned that there is often more to these things than we see.

 

[Nikki J]

It was a small trial yes. I don’t kno what you mean questionable methodology. Doing another trial with a more limited group based on findings from the previous trial is an appropriate way to do things. Similar to another trial that limited the next phase to those with inflammation on biomarkers. We know that ALS is many different diseases. Targeted trials are valid and probably the correct aporoach. Not stratifying trial members other than by symptom time certsinly hasn’t given us answers

The trial was shorter than we see here. The Japanese FDA will not allow longer placebo trials.

I am not a particular fan of Radicava though some seem to benefit.

The different companies approach trials. Compassionate use, expanded access and seeking approval in different ways. I am sure money enters into it- they are businesses after all.

 

[ThroughThatValley]

Compassionate use, expanded access and seeking approval in different ways. I am sure money enters into it- they are businesses after all.

The use of the word "compassionate" coming out from up there in Big Pharma land is the most cynical move they've ever made. There's nothing that far from compassionate than labs and the money grinding wheels of their business.

 

[Lkaibel]

My crabby opinion is that Radicava was approved under those extremely limited trials simply to make a buck. They didn’t really care that it had shown very limited effectiveness in a tiny number of people. It had not shown substantial side effects, and in the U.S. market a crazy price could be charged for the drug.

Somehow, NurOwn is not making that grade.

 

[lgelb]

It's important to remember that there has been no completed trials -- Phase 1, 2, or 3 -- of the current NurOwn regimen. The trial that is going on now, that hasn't even recruited all the participants yet, is its first test.

So the traditional definition of Phase 1/2/3 trials doesn't apply the same way as if the dose were the same all the way through. You can't say everything is positive and Phase 3 is just dotting the i's, or that the therapy has been approved for something else and so we know it's safe and effective for at least something. Changing later trials' dosing based on early results is now used to accelerate development of many drugs, just as with NurOwn.

Please don't waste everyone's time making me the fall guy for forum rules or the fact that NurOwn has a ways to go. If I "deleted everything I don't like," the pharma-bashing would certainly make the list. I worked in that industry for many years (as well as hospitals, clinics, managed care and medical devices) and the notion that any of these is one big money-making conspiracy is far from the truth.

I might also point out that the FDA, which approved Radicava in the US, does not profit from manufacturers' revenues, and committee votes to approve drugs are made in public hearings for which the evidence presented, questions asked, etc. are available at fda.gov.

 

[Par220]

I disagree with the take you have that the new dosing regimen is something we need 200 patients to enroll for before the FDA decides to release/evaluate for a condition with this prognosis and no effective options. You present a very black and white view of policy. It turns out there is a grey area which the FDA can (and has) used to evaluate drug approval for terminal diseases.

 

[ThroughThatValley]

Sadly it appears that this protocol, even if proven efficient, will not provide help for Pals already diagnosed. If I read it correctly, more than five years, at best, will come and go before any patient will get full access to the drug. How many of us here speaking will still be in the position to recieve the tratment when it will hit the drugstore? No one can afford today to wait five, six, seven or ten more years.

 

[Nikki J]

If the trial goes well I don’t think it will be quite that long but it is true that many of us will not be here. I suspect too the key to successful treatment will be early intervention. The more advanced the disease the harder it is to fix I think. This isn’t completely a function of time slow progressing people should have a bigger window.

Do we know this is completely up to the FDA? Nurown has to initiate any requests surely? They may not wish to commit resources to a hopeless task but they may have other reasons such as logistics and infrastructure.

 

[Par220]

Agreed. The FDA can request. Brainstorm can also ask for an interim analysis without unblinding both the doctors and patients. I'm not sure why they are not requesting. It may be because they are a small company which likely is not willing to take risks. I write them regularly as well.

Let's not just assume we won't be here and accept the naysayers. Let's fight for a chance to gain access. There are drugs brought to market quickly because of advocacy. There are always people who say it's not possible. In every disease there is a turning point where therapies are developed that work. It's possible the time is now. Please don't just give up.

I also thought would have to be early intervention, but Matt Bellina is a definite ALS diagnosis with symptoms starting many years ago. He reports significant improvement. I think the reality is no one knows 100% what the body is capable of if an effective treatment is given. There will always be people who steal your hope without knowing for sure. Misery loves company.

 

[Par220]

Matt mentioned on his facebook a few days ago that he can now use his left hand and that he can even grab a dumbbell.

 

[KarenNWendyn]

That’s fantastic for Matt. I’m happy for him.

Meanwhile, for those of us who can’t get this treatment (I.e. pretty much all of us who aren’t in the study on actual treatment), hearing this news just reinforces the idea that there’s something out there that MAY help, but we can’t get. It’s like dangling a juicy beef bone in front of a salivating dog and saying, “Want this? Well sorry, you can’t have it now. Maybe in a year or two if you’re a good doggy.”

If the data from the phase 3 trial looks good, we’ll have to wait a year or two or three, and many of us will either be dead or our motor cortices and anterior horns will be so scarred down with gliosis that it won’t matter.

So quite honestly (to be blunt here), I’d rather not hear about every little improvement this one guy may be making until the phase three study is complete, we have actual data on a larger scale, the FDA gives us the green light, we know about reimbursement and things like side effects and how often to retreat, and there’s the possibility of real hope.

 

[lgelb]

The FDA already has Brainstorm on the fast track in every respect, Par. As noted earlier, they are allowed to submit their NDA in pieces and will get priority review when the package is complete. They've passed their interim safety analysis and will be releasing topline efficacy results next year.

So I'm not sure what the "grey area" that you speak of is. If you are talking about Compassionate Use, CU is by definition for very small numbers. This is all separate from the point about the regimen's being untested as yet, which has its own legal, financial and humanistic implications.

Third-party payors don't fund rx outside (1) approved clinical trials and (2) treatments already approved for the use under discussion or something else. And they don't reimburse everything even in those categories. This is reality, and yeah, it's pretty black and white, across the world.

But I would never suggest that PALS should curl up and prepare for death because scientific progress in treating ALS has been glacial. The opportunity to have a better day tomorrow, while experiencing something of perceived value, is within reach for most PALS reading; the choice to reach for that day is yours alone.

 

[Par220]

I was diagnosed in 2011 and since then have looked at forums with titles like 'ALS Drug Trials' searching for hope and positive news. It's been a very depressing and hopeless search for a lot of years. I'm sorry if some don't like to hear about this, but my hope is there are some like minded people on here like me who want to push senators/FDA/Brainstorm for access to try something that is showing promise.

Maybe you are right. Maybe it won't work for all of us and I and many others will die before we get a chance.... But maybe not.

I will only say Matt continues to improve daily and posted a video today of himself weightlifting on his facebook. I'm truly sorry if my posts are bothersome to anyone, but these are things I have been waiting to hear since 2011.

 

[Par220]

I'm trying to do what I can and do my part. I've always been a private person regarding my fight with ALS because I was made to feel hopeless from the day I was diagnosed.

I never bring this up, but I was a physician in my former life (pre-ALS) diagnosed at 35. I trained at a highly prestigious academic hospital and went on to practice what I felt was the highest quality form of medicine. This means beyond book knowledge we focus on Evidence Based Medicine (clinical trials/statistics) and the current standard of care.

As a physician turned patient it was so clear how poor medicine training is for dealing with terminal diseases. It's a sad reality that for most physicians practicing for many years it's just a job and the life of the patient and their family impacted by the news is a temporary thought until they are done charting/ billing. In addition for an ALS specialist they feel they have done a good job.

Why is that? Because the current standard of care is supportive (bipap, wheelchair, ineffective long term treatments like riluzole/edaravone). Statistically the vast majority give you no hope whatsoever telling you the 5 year mortality, signing advanced directives and preparing to die. The Evidence Based Medicine pertains to trials which they will tell you what is available and if you qualify.

I know how physicians are trained and the vast majority (likely 99%) of even neurologists are not even aware of results related to Nurown, Copper-atsm, etc.). The very few who do or may know and/or are involved in the trial will not say because physicians are taught not to jeopardize the integrity of ongoing trials with opinion/bias.

As an example a physician friend of mine 1 year ago spoke on my behalf to the head of a multidisciplinary ALS clinic chief physician who I was told was a "force to be reckoned with" in terms of an ALS specialist. I had him ask what if anything he has heard about the copper-atsm trial. He responded he had never heard of it.

The reason I am very vocal now is because I know for the first time since I was diagnosed there is real hope with treatments which exist now. I believe it's a turning point.

So where does this leave those of us living with ALS? I believe our position is clear. The only people who can make a change NOW is us and those willing to advocate for us (friends, family, media) to put pressure on the FDA/government and drug/biotech companies like Brainstorm.

I just hope more ALS patients realize this and get involved in whatever way they can. Don't accept people who say nothing can be done. Please don't let even your physician tell you otherwise and don't expect or wait for them to advocate for accelerated approval or interim analysis of these treatments. They are not trained to think outside of a very well defined box.