Cell Therapy News/Articles, page-13150

Thanks JB, I just went over the FDA ODAC meeting again over the weekend. MSB and Dr Kurtzberg and the patients presented such a compelling case that we got 9:1 for efficacy. All other drugs for SR-GVHD also got approval without doing a randomized control trial. It's really one A*Hole from the CMC panel didn't like all the evidence. However, he did suggests that we Knock-down TNFR1 and see if the ability to down-regulate IL2R expression is reduced.

I hope that MSB did this experiment, at least in vitro and look at its ability to inhibit PBMC activation. Because everything else that MSB has shown (as stated above by @JB1975) is correlation only. I call the CMC scientist an A*Hole because 1) the MOA of a lot of drugs are not known, worst FDA approved Aduhelm, which showed disconnect between drug and MOA. 2) that A*hole is not a clinician, so he wants to give very sick kids inferior products (products with lower vs higher level of TNFR1) and see if those received lower TNFR1 receptors died 3) The A*hole forgot that this is salvage therapy + on-top of standard of care (a salvage therapy means that nothing else works, otherwise it would have been used).

I hope that triple A*hole clear his sh!T all at once (number 3) and stop blocking a salvage therapy that save kids' lives. It's only ~200 patients per year and MSB is happy to do a phase 3 on adult.