KevinM
Senior member
- Joined
- Mar 30, 2019
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- Tallahassee
Hi all. Got some time this chilly morning (in Florida? In April? WTF?), and I want to ask for opinions on a couple of NurOwn Ph. 3 participants and the functional improvement they continue to report to this day. Both were diagnosed more than three years ago.
A week ago I posted about the upcoming release of the Pentagon’s declassified report of recent unidentified aerial objects that defy easy explanation, and this seems to fall into the same difficult to explain category. It’s another of my long-winded posts, but you know me when I get on a roll.
So we all know what happened with the Phase 3 data, and the FDA’s rejection. Most of us are probably familiar with the subset of participants with FRS >35 that responded much better than those with lower scores. One physician analyzing the data hypothesized that had that subset been larger—26 participants got NurOwn, with 9 showing strong responses (35%), while 32 got the placebo with 5 showing response (15%) though with much less of a positive response than the ones who got the actual treatment—it very likely would have reached statistical significance, at least for that group.
This is all water under the bridge now because NurOwn will not get approved, but I rehashed this because both of the individuals that have posted several videos showing pretty dramatic improvement (at least as they report it) were in this group. The big question is whether they received the treatment or the placebo, which I would give anything to know.
So here is where my logic breaks down a bit, and it has been fueled a bit by an ongoing give and take between a PALS that has lost significant function in a year, and one of these two NurOwn trial participants. Bottom line is the PALS with fast progression is of the strong opinion that this person either has exceedingly slow progression or was misdiagnosed, and that NurOwn had nothing to do with his lack of progression/improvement. He argued that the trial was designed with injections every two months because the Phase 2 trial showed that efficacy would begin to wane after that time, and it has been about a year since these two received their last injection. Thus they should not still be getting any effect from the treatments. This assumes they did not receive the placebo, which is quite possible.
I know that everyone’s progression is different, and we have some exceedingly slow progressors on this forum that were diagnosed five or more years ago and are still functioning pretty darn well. But both of these folks have posted videos showing long term improvement (not a temporary “bump”) in certain functions, one with hand strength and the other with leg strength.
A recent video shows one of the fellows quickly going up and down stairs, even going up them backwards, which he claims was not possible three years ago. The other fellow is lifting heavy blocks with a hand that he claims had very little strength before he started the trial.
I’d love to hear opinions as to why and how actual functional improvement has apparently occurred with these two, at least as they report. I know, I know, it’s only two people, but their cases have gotten a lot of visibility and many PALS point to them as proof that Nurown is potentially transformative for some PALS.
I have my doubts about misdiagnosis, because I presume that in order to qualify for the trial they had to have confirmed ALS. That would leave one of two possibilities—either they have extremely slow progression, they are overstating their “improvements,” and NurOwn (or the placebo) really had no effect; or NurOwn actually worked long term to halt progression and even restore some function. I don’t know where they were diagnosed, their actual progression (as determined by their neurologist, not them) before the trial, etc., so there are many questions that aren’t readily available. I’d just love to hear your thoughts. Slowly warming up, Kevin
A week ago I posted about the upcoming release of the Pentagon’s declassified report of recent unidentified aerial objects that defy easy explanation, and this seems to fall into the same difficult to explain category. It’s another of my long-winded posts, but you know me when I get on a roll.
So we all know what happened with the Phase 3 data, and the FDA’s rejection. Most of us are probably familiar with the subset of participants with FRS >35 that responded much better than those with lower scores. One physician analyzing the data hypothesized that had that subset been larger—26 participants got NurOwn, with 9 showing strong responses (35%), while 32 got the placebo with 5 showing response (15%) though with much less of a positive response than the ones who got the actual treatment—it very likely would have reached statistical significance, at least for that group.
This is all water under the bridge now because NurOwn will not get approved, but I rehashed this because both of the individuals that have posted several videos showing pretty dramatic improvement (at least as they report it) were in this group. The big question is whether they received the treatment or the placebo, which I would give anything to know.
So here is where my logic breaks down a bit, and it has been fueled a bit by an ongoing give and take between a PALS that has lost significant function in a year, and one of these two NurOwn trial participants. Bottom line is the PALS with fast progression is of the strong opinion that this person either has exceedingly slow progression or was misdiagnosed, and that NurOwn had nothing to do with his lack of progression/improvement. He argued that the trial was designed with injections every two months because the Phase 2 trial showed that efficacy would begin to wane after that time, and it has been about a year since these two received their last injection. Thus they should not still be getting any effect from the treatments. This assumes they did not receive the placebo, which is quite possible.
I know that everyone’s progression is different, and we have some exceedingly slow progressors on this forum that were diagnosed five or more years ago and are still functioning pretty darn well. But both of these folks have posted videos showing long term improvement (not a temporary “bump”) in certain functions, one with hand strength and the other with leg strength.
A recent video shows one of the fellows quickly going up and down stairs, even going up them backwards, which he claims was not possible three years ago. The other fellow is lifting heavy blocks with a hand that he claims had very little strength before he started the trial.
I’d love to hear opinions as to why and how actual functional improvement has apparently occurred with these two, at least as they report. I know, I know, it’s only two people, but their cases have gotten a lot of visibility and many PALS point to them as proof that Nurown is potentially transformative for some PALS.
I have my doubts about misdiagnosis, because I presume that in order to qualify for the trial they had to have confirmed ALS. That would leave one of two possibilities—either they have extremely slow progression, they are overstating their “improvements,” and NurOwn (or the placebo) really had no effect; or NurOwn actually worked long term to halt progression and even restore some function. I don’t know where they were diagnosed, their actual progression (as determined by their neurologist, not them) before the trial, etc., so there are many questions that aren’t readily available. I’d just love to hear your thoughts. Slowly warming up, Kevin
