In fairness, Al, this is a therapy under regulated trial -- a legitimate topic in this subsection.
My personal issue is Mark Bedwell saying things like "I don't want any other patients to die."
Whatever his improvement, whatever portion is actually attributable to the treatment/durable enough to not "even out" nearer the end, etc., it is premature and distressing to other P/CALS to imply the FDA/Brainstorm have the power to hit a switch today that they choose not to touch, and prevent death from ALS. Think about that statement. Never has NurOwn claimed that its treatment is a cure -- we have no evidence for that at all.
While I'm here, Par, I want to say something about your saying that you have been made to feel hopeless since dx. As a physician, surely you are aware that there are many means of controlling symptoms and disability beyond the FDA-approved treatments, which we would both agree are suboptimal.
Therefore, whenever we encourage P/CALS here to try "less than a cure, more than nothing" approaches from P/ROM exercise, to early use of BiPAP/mobility devices, to maintaining nutrition/hydration, we are doing something that can extend the period during which quality of life is acceptable.
So rather than feel powerless, I encourage you to use your training and voice to help yourself and perhaps others reap those potential benefits. And I am appreciative for the many P/CALS here, most with less formal medical background, who are already doing just that.
This is a very diificult emotional and often painful topic.
Optimizing quality of life is important whether there are other treatment options or not.
The hard truth is no matter how well we do that with ALS we progress.
Seeing someone who apparently has actually improved somewhat in a legitimate trial brings hope but also pain. I do think the statements of the 2 men discussed here whether you like the phrasing or not at least acknowledge that many PALS wish for the chance they have had. Brainstorm is not proven therapy at all but improvement ( even if it proves transient) as seems to be happening for some would be welcome to every PALS I know.
The discussion generated by this thread is circling round and round.
The bottom line is that the only way to get Nurown presently is through a study with fairly restrictive entry criteria, and 50% of those accepted into the study will get placebo with no hope of getting active treatment during or immediately following the study. Only one person apparently was able to get it through “Right to Try”.
The phase 3 results aren’t expected to be released until next year, with FDA approval and release possibly up to a year after that, assuming the results are favorable. So this treatment, if approved, might benefit future PALS, but it will likely be too late for many of us currently affected by ALS. We’ve been through this discussion already.
It remains to be established how often retreatment will be needed and what insurance will even approve. Even the physicians doing the study acknowledge that Nurown is a stop-gap measure, not a cure, so retreatment will likely be required to keep disease progression at bay. I strongly suspect that the people in the study who got active treatment may experience a rebound of their ALS symptoms if they don’t have immediate access to ongoing treatment following the study.
I was just responding to the question that Nikki asked - nothing more.
However, I am "following" Nurown just as I am "following" a number of other potential treatments; I have an obligation to my PALS to be informed. And since she has the C9 mutation, that obligation extends to my children and grand children.
WRT this particular advocacy, I personally find it a bit flawed, for several reasons:
- Even if it receives FDA approval, the Nurown process involves "transplantation of autologous bone marrow derived mesenchymal stromal cells (MSC), which are enriched from the patients' own bone marrow, propagated ex vivo and induced to secrete NTFs. The autologous MSC-NTF cells are back-transplanted into the ALS patient into the sites of damage, the spinal cord and the muscles."
- This is not like taking a Riluzole pill twice a day; there are a lot of logistics, legal and other variables to be ironed out. Will an expedited FDA approval for Nurown make some practitioners, providers and/or insurance companies hesitant to fully buy into this solution?
- Lastly, the costs I've seen are in the range of $180k to $260k for this treatment; unless covered by insurance in a significant way, it will be out of reach for most patients.
Having said all of the above, I hope with all my heart this treatment proves successful; beyond the hype there are many positives. Both MC-Rochester and MGH are participating sites - a strong endorsement by itself.
Ken, just to clarify one point in your post — the phase 2 study showed that the transplantation into the spinal fluid was more effective than into the muscles. So the current phase 3 study is only transplanting it into the spinal fluid.
I have stayed relatively quiet on this forum because I feel my posts were causing tension. I am not trying to create any problems.
I agree Laurie. Despite the best supportive care many of us have a minimum quality of life we are willing to accept. The hopelessness comes for me from doing what you suggested and getting nearer to that point without evidence of efficacy for a treatment (like you see in this video) until now. Just gives me hope and I know there are many who agree.
I agree there is no chance Nurown is a cure for ALS any more than lipitor is a cure for high cholesterol or insulin injections are a cure for diabetes. We're hoping it is a treatment.
As noted earlier, the FDA already has NurOwn on the fast track and it will also get priority review when the package is complete. So Brainstorm can submit the NDA in pieces (which means less likelihood of surprise down the line), They've passed their interim safety analysis and will be releasing topline efficacy results next year.
This is all separate from "Compassionate Use," a program for very small numbers of patients, and certainly not for a procedure like this.
As also noted earlier, the regimen now in trial hasn't been tried yet. It's the result of earlier trials and what was learned. That's not uncommon but underscores that it's something that needs to be tested. Some treatments previously tried for ALS did more harm than good overall, and it's not a cakewalk treatment.
To your concern, Ken, the issue with NurOwn won't be payor appetite or logistics (we do this sort of thing in certain cancers, for example) as much as waiting for the data to show if/how well/for whom it works. This trial won't tell us all that, since it's limited to rapid progressors but still with relatively high SVCs in order to increase its possible yield.
But hopefully it will work well enough in this group to merit approval, opening the door for other PALS as well, more data, more research to fine-tune the dosing and technique.
So while we await next year's results, anecdotal evidence is reason for hope, but no substitute for what P/CALS can do to help themselves/each other today and tomorrow.