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  • Stem cell therapy offers hope for treating, maybe even curing, eye diseases, including blindness.
    • Two types of stem cells can be used. Human embryonic stem cells (hESC) or induced pluripotent stem cells (IPS).
    • hESCs are derived from embryos left over from fertility treatments while IPS are derived from the patient’s own body.
    • Being personalized medicine, IPS is more expensive though the advantage is it doesn’t require immunosuppressive Rx since the patient’s immune system is less likely to reject these cells.
    • OTOH, hESCs are cheaper. One batch can treat several patients though they require immunosuppressive Rx to reduce the chance of rejection by the patient’s immune system.
    • So these two types of stem cells have different trade-offs.
    • At present, hESC have been used to derive retinal pigment epithelial (RPE) cells in vitro.
  • Stargardt’s macular dystrophy (Stargardt disease), an inherited incurable disease, and age-related macular degeneration are some of the common causes of blindness in the young and the old, respectively.
    • In these diseases, loss of RPE cells contributes to vision loss.
  • Currently, clinical trials are testing whether injecting hESC-derived RPE cells directly into the eyes of such patients is 1) safe and 2) could slow/halt/reverse the disease.
    • Home – ClinicalTrials.gov shows 4 such ongoing trials, three in the US sponsored by Ocata Therapeutics and one in South Korea.
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    • Two peer-reviewed scientific papers in the Lancet suggest that this Rx is safe, tolerated and cautiously promising in young patients with Stargardt’s macular dystrophy and in older patients with macular degeneration.
  1. Schwartz, Steven D., et al. “Embryonic stem cell trials for macular degeneration: a preliminary report.” The Lancet 379.9817 (2012): 713-720. https://www.ocata.com/uploads/sc…
  2. Schwartz, Steven D., et al. “Human embryonic stem cell-derived retinal pigment epithelium in patients with age-related macular degeneration and Stargardt’s macular dystrophy: follow-up of two open-label phase 1/2 studies.” The Lancet 385.9967 (2015): 509-516. Page on ocata.com