Cell Therapy News/Articles, page-15608

My own view is that Dr Marks becomes more of a political animal every day.

I may be unreasonably cynical here, but I view CBER as having deliberately promoted & advanced consideration for approval of gene therapies in priority to non-haematopoietic cellular therapies (apart from CAR-T cells). And, although I have not a shred of hard evidence to support this opinion, I just think that in 2023 the FDA still feels more 'comfortable' dealing with innovative therapies that are as close as possible to a chemotherapy cocktail i.e. a drug. Genetically modified therapies seem to appeal to those who demand & seem satisfied by scaleable quantification of effect, molecule by molecule, at all costs (even when there's no superiority effect over placebo).

So, you might say 'but what about CAR-T cell therapies, eh?' After all, they make it through the FDA's filters. That's true, and Marks does love talking about them, but think about why they can be held up as an exception. Firstly, we're talking about oncologic treatments, & cancer brings out the greatest willingness in regulators to bend rules to pursue a higher goal in patient interests. But at a deeper level, in at least one sense the mechanism of action of chymeric antigen receptors is really IMO being 'programmed' by corporate pharma to degrade and take away from the natural mode of operation of T cells - because we "know so much better" than our existing evolved human cellular biologies - and it seems that we end up with a cell, in name, that appears to be largely used simply as a vector for the tuned receptor. A delivery vehicle, if you will.

I know, this is overly simplistic, doesn't qualify as systematic analysis & may just be plain wrong.

But to me there is an administrative arrogance & apparent contempt for human life in Marks saying there may be a need for his administration to “accept some level of uncertainty” at the time of approval around questions like "side-effects over long-term" and "safety during administration". The poor patient might reasonably say, 'But isn't it your job at the FDA - even if you can't be reasonably sure of an effect, at the very least to find out what is safe to administer?".

Then there's the zealotry with which embryonic & other cell therapy promoters have been aggressively pursued by Marks and his CBER administration at the FDA - while delaying guidance on the development of cellular therapies & failing to resource themselves properly to manage the future. I wonder to myself how that top-level view can not breed a 'good therapy' and 'bad therapy' approach in lower level FDA regulatory staff?

I have to admit I watch Dr Marks public actions with a lot of concern.

The sooner we get this approval through, the better.