ageing, stem cells

First Transplant using Induced Pluripotent Stem Cells: Treating Blindness

Induced pluripotent stem cells (iPSCs) have enormous potential for being used in tissue transplants and therapies. Why is this? It’s because iPSCs can be made using virtually any cells from a person’s body — such as skin or fat samples that wouldn’t be missed. (And since they’re the patient’s own cells, the immune system should not reject them in a transplant.) iPSCs are also pluripotent, which means they can be turned into nearly any cell type. This means that if a patient needs a certain type of retinal cell to treat an eye disease, a skin sample could be taken, turned into the desired retinal cells, and then transplanted into the patient’s eye. And this is exactly what happened for the first time last week.

retina age-related macular degeneration fundus

This image shows the retina of a person with intermediate age-related macular degeneration, an eye disease recently treated with iPSCs for the first time. (Image credit: National Eye Institute, National Institutes of Health)

Even though human iPSCs were first created in late 2007, it wasn’t until last week that they were used in a tissue transplant in a human for the first time. The person was a Japanese woman in her 70s, and the iPSC-derived cells were used to treat her age-related macular degeneration (AMD), a leading cause of blindness in the elderly. Safety studies in mice and monkeys had been previously done to ensure the iPSCs would not cause tumors or be rejected by the immune system.

The cells were grown and prepared by researchers at the RIKEN Center for Developmental Biology in Kobe, Japan. (This is also where researchers reported the discovery of the controversial STAP cells earlier this year.) To treat the patient’s AMD, her skin cells were specifically turned into retinal pigment epithelium (RPE) cells, which were then transplanted into her retina to replace RPE damaged due to the disease. The transplant was performed last Friday, September 12, at the Institute for Biomedical Research and Innovation, next to RIKEN. So far, the patient has not had any serious problems following the surgery.

A total of six patients with AMD are planned to be treated with the iPSC-derived cells in the clinical trial and will be monitored for one year.

It is particularly thrilling for me to see this research reach the clinical stage as it’s actually the same area I did my Ph.D. graduate studies in, in the laboratory of Dennis Clegg at the University of California, Santa Barbara. For my thesis, I specifically investigated how different substrates could be used to create RPE (derived from iPSCs or human embryonic stem cells) and transplant the cells into patients. If all goes well in this initial trial, I look forward to seeing more iPSC-based therapies reach the clinical spotlight.


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