Ann: Update on BLA for Graft Versus Host Disease, page-118

My memory what that there were three main concerns last time, based on staying up and listening to the whole bloody thing, was:

• only a single-armed trial
• the cells came from only a handful of donors (I think, two?), and there was nothing to show that cells from new donors would be as effective
• the science behind the assay technique was behind what the "expert" considered leading edge (hence the second bullet point above)

The recommendation was to develop the assays to be more robust and run a controlled trial, either with adults or children. I would assume that the first has been completed, but not the second, and hence rejection.

** all of the above is from my memory and may be incomplete --- I don't have the stamina or willpower to watch through all the 2020 proceedings again