He now says the rate of decline for some patients in the BCLI trial " Worst patient in P2a was on IM only and averaged -2.8 ppm. That's bad, but not an outlier in terms of safety concern."
The CEO said 2.6 a 2.7, and now he reports 2.8. Yes, 0.1 is splitting hairs, but he is picking and choosing data to share. My point is that he has changed sides for some reason. Maybe, it's because cur has a legitimate analysts in Johnathon Aschoff from Brean... Just thinking out loud, IMO.
He asked a question about ALS progression in the BCLI trial. Here was the reply from
Tony Fiorino, BCLI's CO "anks for the question, Jason. So we have looked at that and we don't see anything that would be, for us, of any concern. I think I mentioned the most rapid rate of decline that we saw in the two studies for the post treatment periods was about 2.6 or 2.7 ALSFRS points per month. And that was in IM subject. In fact the three, the three most rapid regressers [ph] post treatment were in subjects who only received only IM administration, which was used in the phase 1 study. And we know that we don’t really see much systemic benefit or systemic effects from IM treatment alone. So particularly within the IT treated subjects, between the two studies we really are not seeing any signs of acceleration of disease".
Looks like Jason is inconsistent. He states in his blog "A full standard deviation (± 1.25 points per month) to the downside tells us that 84% of placebo patients will decline at a rate of less than 2.44 points per month, and Neuralstem notes in its investor presentation that non-responders declined at a rate of 3.04 points per month (company actually noted -0.1 points per day). Based on our analysis of the PROACT numbers, this puts the Neuralstem non-responders in the 4th percentile. That is a major concern."
I get that .34 points are unfavorable for an ALS patient, but three of BCLI patients progressed faster than the numbers provided by Jason above...
I am confused why he would congratulate BCLI today as the superior treatment. Anyone care to comment?
I believe that there is a big factor that is over looked in not only this trial but in all ALS trials. This factor my explain why you have responders and non responders. My Neurologist, Jonathan Glass, has said to me multiple times "Ted I may have found something that helps your form of ALS, but I have no idea what your form of ALS is nor anybody else's." There in lies the rub. The majority of Neurologist agree that ALS is a spectrum of diseases.
As a patient advocate I attend numerous conferences and have the opportunity to learn from many experts in the field. At one conference I had the pleasure to speak with Dr. James Berry from Mass. General. I think he put it perfectly when he told me that "diagnosing a patient with ALS is equivalent to every time there you have a broken radio diagnosing it with radio disease." I found that interesting. Let's extrapolate on that statement. Let's imagine that we were not sophisticated to know the cause's of radio disease. Now if we were to put a trial together for radio disease and gathered 100 broken radio's and decided to replace the speakers in order to treat radio disease in our trial, what would the results be? Back to reality we are sophisticated that the results would be based on dare I say random luck. We could cure 10, 20 or 30 radio's suffering from radio disease but the odds of curing 100 percent of them would be nearly statistically impossible.
In my humble and lay opinion the same is true of ALS. After phase I they changed the protocols for phase II to closer align with patients who symptomatically looked like me. Limb onset, not bulbar onset, closer to symptom onset and I am sure other factors as well.
Until science and medicine are sophisticated enough to understand and be able to identify for each patient through bio markers the cause of ALS, Doctors, Scientists and companies like Neuralstem are flying blind. Until then if you can have a trial where 47 % of the people respond, for a disease like ALS where there is no hope, that is promising. After all if we changed 100 speakers inflicted with radio disease I doubt 47 of them would react positively to the "treatment".
To be clear I do not own Neuralstem stock, but am a believer in the company, the science and the great people at Neuralstem for obvious reasons.
In about 2-3 weeks, Dr. Fava will submit the MR I data from the phase 1 for publication, and the expectation when published is to see difference in the hippocampus between drug and placebo that support the purported mechanism and drug screening technology. Thus far in humans, there is a statically significant difference in high alpha frequency brain waves, as evidenced via quantitative EEG.
Human neural stem cell treatments are showing promise for reversing learning and memory deficits after chemotherapy, according to UC Irvine researchers. Just published by Medical Xpress, not sure if it's the same study as the one on the Neuralstem presentation. However, it's from UC Irvine as well.