Shore Eye Associates Blog

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Macular Degeneration and Stem Cell Therapy


There has been recent publicity surrounding stem cell treatment with devastating results in 3 patients with age related macular degeneration.  In March, the New England Journal of Medicine published a case report of three patients who went to a clinic in Florida that purported to use fat cells from their abdomen and purified it into stem cells (N Engl J Med 2017. 376;11:1047-53.)  These stem cells were then injected into both eyes on the same day and cost the patients $5,000.  According to two of the patients, they thought they were part of a clinical trial since they found the listing on the website, although there were no documented safety profiles prior to their use.  The vision prior to treatment ranged from 20/30 to 20/200.  After treatment, these patients required multiple surgeries and lost further vision.

In contrast to the above-mentioned study, another study in the same journal illustrated extensive research prior to human clinical use. This subsequent article is an example of solid scientific research behind stem cell research for age related macular degeneration (N Engl J Med 2017. 376;11:1038-46.).  In this case, the work is based on Shinya Yamanaka, a Japanese physician who was awarded the Nobel Prize in 2012 for his discovery that adult skin cells could be reprogrammed into induced pluripotent stem cells(iPSCs). These iPSCs are then transformed into retinal pigment epithelial cells(RPE), which are disrupted in wet age related macular degeneration. As there is a concern about the safety of these stem cells, animal studies were performed prior to human clinical trials.  Once the stem cells proved to be benign, they were then surgically transplanted underneath the retina.  Even after extensive studies, they exercised extreme caution and only one patient ended up having the stem cell transplant.  One year after treatment, there was no significant improvement nor deterioration in vision.  The patient did report subjective improvement on a functional questionnaire. 

These two case reports highlight the extremes of stem cell therapy.  In the first article, the stem cell clinic was treating patients without appropriate FDA oversight and lacked preclinical research.  Funding for clinical trials should not be patient based.  Furthermore, clinical studies should not involve injections of both eyes simultaneously as it is still a trial and not proven therapy.  The safety of stem cells from peripheral blood and induced pluripotent stem cells(iPSCs) has been established, but the use of fat tissue for stem cells still lacks solid clinical evidence.  There is a great promise for stem cell treatment but until there is more regulation for stem cell therapy, the public needs to be vigilant.

For more on stem cells and the eye see our previous Stem Cell blog post.

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Wednesday, 01 December 2021

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