Stem cell good news for the holidays: biotechs, FDA, organoids

I can see many reasons to be jolly on the regenerative medicine front as we get deeper into the holiday season as there is quite a lot of stem cell good news even if there is also some not so good news.

In today’s post the focus is on the good news of late.

kidney organoid good news
Atsuhiro Taguchi and Ryuichi Nishinakamura, part of Figure 4, Cell Stem Cell 2017, kidney organoids in development.

Semma Therapeutics, a stem cell diabetes biotech, raised more than $110 million. I’m eager to see how the next few years play out with the healthy competition between them, ViaCyte, & others in this area. The end result of this good news now will hopefully be at least one safe, effective new diabetes treatment in the future within less than a decade. You can read my recent interview with ViaCyte leadership here.

More on the biotech front, as Amgen invests big bucks into direct reprogramming firm Fortuna, which focuses in part on the development of autologous neural stem cells for a variety of conditions.

The promise of cell-gene therapy combos is exemplified by the recent effort that restored basically the entire epidermis of a young boy with epidermolysis bullosa, who would have otherwise died. You can read about my take on the Nature paper reporting this finding here. It’s one study, but I see it as good news. You have to start somewhere with a new, transformative approach.

Recent FDA actions and words on stem cells are good news on a number of levels in terms of clarity and strength, which I will optimistically say are likely only the beginning of a different phase in our field. See more of my thoughts and information on this here. I believe that new Commissioner Scott Gottlieb is serious about differentiating between good citizens in this arena, whose path we should facilitate, and those on the other end of the spectrum who are after patient’s money and willing to risk their customers’ health and even lives. You can see text of Gottlieb’s broader testimony last week before Congress on 21st Century Cures here.

There are unfortunately usually too many steps from discoveries to the bedside to help patients, but each step is something to be happy about as long as we have a balanced view and don’t succumb to hype. New work on the organoid front suggests the idea of “kidneys in a dish” is a step closer to a future reality. The scope of kidney disease is so profound that more effort is needed in this area. You can see the beautiful data above (part of Figure 4 from their Cell Stem Cell paper) from the work by Atsuhiro Taguchi and Ryuichi Nishinakamura mentioned in the overview article.

What’s your stem cell good news for the holidays?