Stem Cells To The Rescue For Blindness: Brilliant Science At Work

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Stem Cells To The Rescue For Blindness: Brilliant Science At Work By Dr Kapil Bharti 

Researchers in California expect to launch a clinical trial of stem cell therapy for age-related macular degeneration this year. 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.

“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.

In the NEI study, scientists led by Bharti started with blood-making cells, called CD34+ cells, isolated from the blood of three AMD patients, and then turned them into iPSCs. They added growth factors and other biochemicals that shape the cells’ destiny: Although pluripotent cells can become any type of cell in the body, the specific combination of biochemicals produces one and only one. Bharti and his team created retinal pigment epithelial cells, the kind that die early in macular degeneration. Each batch took about 11 weeks.

The idea is that replacing the dead and dying RPE cells “will stop these photoreceptors from dying and stop the disease from getting worse,” said Bharti, who with two co-authors are inventors on a patent application for generating retinal cells from iPSCs. Saving RPE cells wouldn’t bring photoreceptors back from the dead, and restore lost vision. But he and his team are already developing an AMD therapy combining photoreceptors with an RPE patch. That could actually cure AMD blindness.

Source: Stat News  Jan 17 2019