@roka
You asked :
"Study only went for two weeks, showed really good results, but are the effects sustained over a long time period?
Translating to humans could show a short strong response, but then what?"
You are absolutely correct that sheep have a high predictive utility in models for humans.
I believe the two week study design was
enough to get a measure of outcomes they were looking for. Especially since measurable improvements started appearing within just two days after the sheep got the treatment.
The most important take away is this:
"All of the histopathologic components ameliorated by MPC treatment are
key features associated with progressive joint disease and destruction in patients with active RA".
Reading the tea leaves, I am thinking that such measurable (i.e., quantifiable) outcomes will be discussed with the FDA in the Phase 3 (for humans) trial design. Sheep don't talk. Can't take pain assessment questionnaires. Humans can, of course, and they have done so during the 12 week Phase 2 trial. With a measurable/quantifiable addition to the trial in humans, it would get rid of any chance of subjectivity and make FDA approval much easier and most importantly, RA therapy would get an
accelerated approval under the FDA RAT program.
Incorporating new measures in Phase 3 that were not in Phase 2 would be something quite unusual, imo. But.......we really don't know what Dr. Itescu has up his sleeve though. But if Dr. Itescu believes it is for the better, then great!
My belief is that this study on sheep was for a specific purpose, one I already outlined, not just an academic exercise. LOL.
Kudos to Mesoblast as well as the folks at the Univ of Melbourne. This study also demonstrated the 10-fold more effectiveness of STRO-1 positive MPCs. Never underestimate the improvements Mesoblast is making.
@Rappa: My apologies if the red and blue font was irritating. With so much info coming at us, I thought it would be effective to highlight some key words. I do like Red, White and Blue - I am sure you do too........take another look at the US and Australian flags
.
(20min delay)