Thanks
@Lopez I can't see SI not continuing to treat either but not all the children were getting them for free. A good percentage (I don't know how many) are not and if you contact BMT centres, the couple of responses I got (just mentioning MSCs in general but not MSB's name), they seem to think there are just research grade ones available.
The forum I belong to is mainly for chronic GvHD sufferers but there have been a few cases that sound like acute and every so often, members are asked to pray. A little girl aged seven was on Jakafi for gut and liver aGvHD which wasn't working. I suggested to the mother she run by her daughter's treating physician the possibility of contacting MSB on compassionate grounds. I declared myself a shareholder with a personal interest in the product (for my daughter). I didn't hear back, so I don't know what happened. There was another little boy two or three years ago the forum was asked to pray for (aGvHD of the gut), I was going to suggest the same but by the time I saw the message, the child had died.
I'd like to see the data in the adults too. I think FDA knew the cells worked at least from 2020. Anyone
genuinely sceptical of Ryoncil's efficacy and a true believer in the "gold standard" of randomized, controlled studies would accept the real possibility that a study in adults might not provide further efficacy and therefore they'd spot the implication of approval for children being contingent on further data in adults as unscientific nonsense right away.
EEN, for example, is a therapy used in GvHD and IBD. It's well-known to be less effective in adults but there's no doubt whatsoever it works very well in children with small bowel involvement.
Having said that, I think Ryoncil is likely to work well in adults too. I'd also like to see the data (and the adults treated in ARDS to know about effect on fibrosis - I couldn't agree more with posters on this forum who are critical of MSB for the lack of information including raw data).
A genuine sceptic and true believer in the sanctity of the randomized, controlled study, would require a RC study in adults
and children. FDA knew that was impossible to do. There's nothing approved in under 12s. Parents would be given proper informed consent by physicians such as Dr. Prockop who would tell it how it is. If I recall right, she said after the experience of using Rux in three patients, her centre wouldn't be participating in a clinical trial. Sounds to me like she might have been asked. If something works for severe GI, it's unequivocal. They know Ryoncil works.
(20min delay)