- 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 (
), 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.
shows 4 such ongoing trials, three in the US sponsored by and one in South Korea.
- 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.
- shows 4 such ongoing trials, three in the US sponsored by and one in South Korea.
- Schwartz, Steven D., et al. “Embryonic stem cell trials for macular degeneration: a preliminary report.” The Lancet 379.9817 (2012): 713-720.
- 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.
- News reports about hESC Rx trials for Stargardt’s macular dystrophy and age-related macular degeneration in the UK: