Is there a proven stem cell therapy for vision loss?
When I started The Niche back in early 2010, one major area of hope was that there might one day be a stem cell therapy for vision loss or even blindness.
Maybe even a cure for blindness.
While we’re not there yet as a field, there are multiple lines of clinical research that seem quite promising. It’s exciting to follow the research in this area. Before we jump into that please take The Niche poll on what is the biggest regenerative medicine good news story of the year.
Novel stem cell therapy for vision loss
World-first stem-cell treatment restores vision in people, Nature. This is exciting in the sense of using iPS cell-based approach to treat damaged corneas. It’s more specifically for limbal stem-cell deficiency (LSCD) specifically.
Here’s the research article in The Lancet: Induced pluripotent stem-cell-derived corneal epithelium for transplant surgery: a single-arm, open-label, first-in-human interventional study in Japan. Larger, more controlled trial work should help clarify the prospects of this approach, but it seems very promising. I’m trying to learn more about a biotech called RAYMEI, which seems to have a role in this research.
There have been other exciting findings on stem cell treatments for damaged corneas and for vision more generally. Since 2010 things have come a long way. The fact that researchers are generating encouraging data using a variety of cell types including adult stem cells and iPS cells, increases the odds of a proven therapy being realized. In another 14 years, the FDA (or similar overseas agencies) may approve several such stem cell therapies for vision loss.
There is long-term hope on other fronts related to sensory systems such as hearing loss: Long-term hope on stem cells for deafness. Vision has gotten the most attention and funding though.
More stem cell reads including on RFK Jr.
- This scientist treated her own cancer with viruses she grew in the lab, Nature. What do you all think about this kind of self-experimentation?
- Patient iPSC models reveal glia-intrinsic phenotypes in multiple sclerosis, Cell Stem Cell.
- Kennedy’s F.D.A. Wish List: Raw Milk, Stem Cells, Heavy Metals, NY Times. I’m quoted in this one on the stem cell part of the story. I’ve written before about RFK and stem cells, including his tweet claiming the FDA is suppressing stem cells. I’m concerned he may be a powerful advocate for unproven stem cell clinics and similar firms. He could interfere with FDA oversight. With RFK Jr. now nominated to be HHS Secretary (in charge of NIH, FDA, etc.), you might want to check out my recent piece on Kennedy and stem cells.
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Reconsidering the 14-day rule in human embryo research: Advice from the Dutch Health Council, Cell Stem Cell. “The Dutch Health Council has advised to extend the 14-day rule to 28 days and to subsume integrated stem cell-based embryo models under the same legislative regime as natural embryos.” So much to think about here including on the proposed regulations on human embryo models.
EuroGCT plus Slovenia
Finally, I just wanted to recommend that you check out the great online resource EuroGCT.
What is EuroGCT?
It is a European-centric web project that has expert, trustworthy information on cell and gene therapies. While it is primarily centered on events in Europe, its resources apply globally. I helped in a small way with some of the review of content. EuroGCT is one of the “good guys” out there in cyberspace in the stem cell and regenerative space.
Speaking of Europe, a report from Slovenia reached out about unproven stem cell and other cell therapies being sold there. Here’s his article. Tisoči evrov za terapijo z matičnimi celicami: napredna medicina ali placebo? N1 in Slovenia. This one is in Slovenian but you can use Google translate on it. The piece is worth a read given the broader lack of a window into what is going on with stem cells in my European countries.
Per Google Translate, it starts: “Many Slovenian private medical centers offer therapies with stem cells. Most often, these are self-paying orthopedic procedures, where they try to help patients with wear and tear of cartilage by injecting stem cells into the joints, for which they charge several thousand euros. While Slovenian orthopedists defend their practice, many foreign experts and associations warn that there is insufficient evidence for the effectiveness of this type of treatment. Slovenian legislation in this area is deficient.”
Blast from the past
How many scientist bloggers does it take to change a light bulb? Way back in 2010 I was writing about how few stem cell blogs are out there. Unfortunately, not much has improved. I’m still doing The Niche (this website, which has a blog) but the main other blogs out there are now from stem cell clinics. One more change, though, is that after 14 years there is relatively little discussion or debate over human embryonic stem cell research, which was a main topic in 2010.
We’ll see if that changes in coming years.
Thanks so much for the update on this medical field, Dr. Knoepfler! So many patients are desperately waiting and hoping for good news. What do you think of Dr. Masayo Takahashi’s work with the robotic AI system that autonomously induces the differentiation of (iPSC-RPE) cells? Do you think that AI might speed up the process so that we do not need to wait another fourteen years for any actual clinical treatments that patients can walk into a hospital and get done? https://pubmed.ncbi.nlm.nih.gov/35762203/
Dear Admin:
Thanks for this needed correction. My mistake for a cursory search of the report for “mutation”, “cancer”, and “tumor,” instead of “tumour.” Very good to know that this risk was well addressed by these authors. Of course, the final test of safety is whether the transplants remain “tumour” cell free in patients with more time.
Regards,
James @ Asymmetrex
Dear Admin:
I find it disappointing that after all the previous protestations and promises that actively dividing iPSC-derived cells would not be implanted into patients, that is exactly what the authors of the Lancet paper are doing. Of course, they are not alone in this pursuit. Similar studies are being pursued with iPSC-derived mesenchymal cells. Cancer is a time-bomb that may tick for many years before it explodes. I suppose one can argue with a risk-benefit analyses that improved function (in this case vision no less!) can justify accepting a future risk of a devastating and potential life-ending cancer. However, we should all be concern when investigators in this clinical research space, and others who will be more enabled to enter it now and regulators (!) begin to ignore this very real risk altogether. The Lancet paper has no mention of “mutations,” “tumors,” or “cancers.” And given what we know about the many genetic alterations in iPSCs, implanting their actively dividing derivatives into patients is a real and future danger.
James @ Asymmetrex®
Your comment is somewhat confusing. Did you read the paper? They didn’t use undifferentiated iPSCs.
See “Allogeneic human iPSC-derived corneal epithelial cell sheets (iCEPSs) were transplanted onto affected eyes.”
and “The approach is based on our development of a self-formed, ectodermal, autonomous, multizone (SEAM) cultivation protocol that effectively produces precursor cells of ocular tissues from human iPSCs.”
There’s a difference between calling something “iPSCs” and referring to their derivatives. You seem to do both in your comment.
They also mention “Tumourigenesis tests for iCEPS included subcutaneous transplantation in immunodeficient mice (NOG/Shi-scid, IL-2RγKO Jic), karyotype tests, measurement of undifferentiated iPSC marker (LIN28A) expression, serial passage culture tests, and a genome analysis (appendix pp 13–14, 20–24). None of these investigations yielded results suggestive of tumourigenicity.” and ” We found no safety issues (eg, clinical immunological rejection or tumour formation) throughout the whole 2-year observational period in any of the patients.”
I don’t see any indication they were failing to be careful.
Nice response, Paul.