Ring the bell for a stem cell milestone, specifically for iPS cell work.
There’s been a whole lot of commotion about the NEJM article yesterday documenting the experiences of three women with macular degeneration who were blinded by non-FDA approved stem cell eye injections of fat stem cells at a business in Florida, but in the same issue of the journal there also was some encouraging stem cell news that came in the form of essentially a mirror image of the bad news paper. We can call it the “stem cell good news-bad news” issue of NEJM.
Masayo Takahashi iPS cell paper
The good news was the publication of the first paper on clinical use of IPS cell-derivatives in a human patient. A big milestone. This groundbreaking manuscript comes from the pioneering team in Japan led by stem cell scholar Dr. Masayo Takahashi. I’ve written extensively in the past about the work of Takahashi and her team with IPS cells, and she received my Stem Cell Person of the Year Award back in 2014.
In the new paper they detail their data from the clinical study using sheets of retinal pigmented epithelial cells (RPEs) made from IPS cells in this case derived from the patient herself for autologous use. Remarkably in Figure 1C (above) you can see the actual transplanted RPE sheet in the eye of the patient (see dark area indicated by white arrow). The most encouraging part of this study was that the patient’s vision remained stable (rather than declining as expected) following the treatment. Was that due to the transplant? We can’t be sure.
Also, this is just a beginning as it is just one patient, but it is very exciting and represents a big milestone for the IPS cell and broader stem cell field, providing real hope for patients with vision loss along with parallel ESC-based clinical trial work as well.
RPE work
This paper contrasts so much with the report from the other one in the same issue on the terrible outcomes from the stem cell clinic’s use of fat stem cells in the eye. While the use of fat stem cells themselves is highly questionable in my view for this application, the biggest differences between the two approaches is that the Takahashi team work was extremely rigorous, careful, based on extensive preclinical studies, had governmental approval, and was in essence science-based clinical medicine.
For instance, the Takahashi team was appropriately cautious with Patient 2 since the cells exhibited some genomic changes. At least in part for that reason, moving forward this clinical work will primarily focus on allogeneic use of IPS cells via an IPS cell bank being developed by Shinya Yamanaka.
We can also look to other future IPS cell-based trials coming on-line including for Parkinson’s Disease and other conditions, which are likely to be allogeneic as well in Japan, but probably autologous here in the U.S.
I love a good stem cell milestone!
I think this is wonderful and amazing. The results right now are so encouraging. Nothing could make me happier than to have these trials work out and really lead to a treatment that would be available to everyone. I have been very careful about getting too excited about the Takahashi trials up to the this point, and I’m going to keep being careful and skeptical, because that’s what we *must do* if we’re going to evaluate evidence scientifically. I have not jumped on a bandwagon, and I absolutely will not. I think it’s important to clarify this point. We can’t do that for ANY trial, whether it’s scientifically well supported or whether it isn’t.
I’ve gotten some strange reactions because I insist on being skeptical rather than a bandwagon jumper when it comes to ALL medical work, trials, studies, possibilities, etc etc etc. But that’s the attitude we have got to take. This is the exact treatment that I would need for idiopathic macular degeneration. It’s in remission right now, but who knows what’s going to happen in the future. This could be a lifeline for me. So there is every temptation to jump the gun and believe it’s definitely going to work. I won’t do that, but I *will* say this is exciting and there seems to be every reason to be optimistic about future successes.
What cells were reprogrammed from the patient to make the RPEs?
@Pete, Skin fibroblasts. In the lung run it may prove beneficial to use starting cells with less mutational load to begin with from the sun, etc.
Paul