| Title | Stem Cell Therapy for Age-Related Macular Degeneration Ready for Clinical Trials | |
| Date | Thursday January 17 2019, @09:57PM | |
| Author | martyb | |
| Topic | ||
| from the eye-see-what-you-did-there dept. | ||
Macular degeneration trial will be first human test of Nobel-winning stem cell technique
Curing [...] age-related macular degeneration [(AMD)], a major cause of blindness [...] was supposed to be low-hanging fruit.
The cause of AMD is well-known, the recipe for turning stem cells into retinal cells works like a charm, and the eye is "immunoprivileged," meaning immune cells don't attack foreigners such as, say, lab-made retinal cells. Yet more than a decade after animal studies showed promise, and nearly eight years since retinal cells created from embryonic stem cells were safely transplanted into nine patients in a clinical trial, no one outside of a research setting (or a rogue clinic) is getting stem cell therapy for macular degeneration.
That may change soon. Researchers in California expect to launch a Phase 2 clinical trial of stem cell therapy for age-related macular degeneration this year, while a team from the National Institutes of Health is not far behind: It is planning the first study in humans using what are called induced pluripotent stem cells, which were discovered 12 years ago and won a 2012 Nobel Prize. These cells (iPSCs, for short) are made by sending plain old adult cells back in time, biologically, until they're like embryonic stem cells — but without the ethical baggage those cells carry.
"This will be the first such study for iPSCs for any disease indication worldwide," said Kapil Bharti of the NIH's National Eye Institute. "When iPSCs were discovered in 2007, there was a lot of hype that we could easily turn them into therapies. But there were many unanswered questions" about how to safely make transplantable cells, questions that are only now being answered. "I hope this reignites the field," Bharti said.
He and NEI [National Eye Institute] colleagues reported in Science Translational Medicine [DOI: 10.1126/scitranslmed.aat5580] [DX] on Wednesday that they had used retinal cells created from human iPSCs to treat a form of macular degeneration in rats and pigs, with results promising enough that they hope to start recruiting macular degeneration patients for a clinical trial in the next few weeks. That sets up a face-off between two forms of stem cells. In their trial, scientists at the University of Southern California are starting with stem cells derived from human embryos.
Related: Stem Cell Therapy for Macular Degeneration: Conflicting Reports
Congenital Blindness Reversed in Mice
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