November 28, 2020

The Niche

Knoepfler lab stem cell blog

Stem cell wish list for 2017

Last week I posted my list of 2017 predictions for the stem cell field. Today a couple of days into 2017 I’m more focused on hope than realism. What would I wish for in the stem cell and regenerative medicine arena in the coming year?stem-cell-wish-list

More stem cell clinical trial data posted and published. There are few things as exciting as stem cell and regenerative medicine clinical trials across the full spectrum of stem cell types including adult, embryonic, and IPSC. But we need to have actual trial data be peer reviewed and published or at least posted. Clinical trial updates only by press release are not helpful to patients or the field.

Clinicaltrials.gov adapts. This vital resource of trial listings adjusts to new realities. It either filters its listings to screen out for-profit entries that aren’t real trials or it provides more practically useful information such as at a minimum clear indications of whether a listing has an IND (this doesn’t need to violate any confidentiality rules) and whether the listing requires payment as an inclusion criteria. See my interview with the leader of Clinicaltrials.gov.

FDA speaks clearly. Whatever the FDA does or does not do in terms of actual stem cell & regenerative medicine-related actions in 2017, it is clear about it. This year I hope the FDA provides concrete, consistent explanations in the public domain that don’t require an FDA-ese jargon dictionary to try to understand.

FDA and its CBER center are consistent with good & bad citizens of the stem cell arena. The FDA has a tough job overall and its CBER branch focusing on biologics including stem cells has its own specific challenging task set. However, for years CBER has held different players in the stem cell arena to different rules and expectations. Paradoxically, essentially the better a citizen you are, the more the FDA expects from you. On the flip side, if you are for instance a stem cell clinic with no intention of following the rules (no BLA, no IND, no pre-IND, no expertise in stem cells, no data, etc.) CBER has historically generally left you alone.

What the heck? That’s got to change.

Of course the FDA and CBER are not police, but by analogy imagine if police devoted fewer resources and tended to take less action proportionately as people behaved worse and worse, and were conversely strictest with the most law-abiding citizens? It makes no sense.

FDA accelerated review or fast track, etc. of stem cell product. We can all hope that there is a strong enough stem cell/regenerative medicine product and that the FDA considers it expeditiously enough (e.g. under Cures Act provisions) that we can see in 2017 a stem cell product get into the fast track somehow.

Less hype on all sides. If one can get away with hype and perhaps most likely only pay a price like a correction or retraction at most just years down the road on the academic side or no price at all in the direct-to-consumer arena of clinics, some people are going to continue hyping up a storm. However, hype is bad all around and is selfish. I’d wish to see less of it in the stem cell world in 2017. Scientists and the media can talk about new, outstanding findings (see below in next section) without invoking miracles or overly aspirational language.

Loads of exciting papers. You can have your great published science without hype. For this year I expect we’ll see dozens of very cool stem cell papers that are at the same time not hyped. As a scientist, for me reading new, interesting data in papers is a real joy.

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