Lab-grown cells make doping agent EPO and cure anaemia in mice

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DENNIS KUNKEL MICROSCOPY/SCIENCE PHOTO LIBRARY

Transplants grown from stem cells in the lab can help replenish the blood and have been used to cure anaemia in mice. The discovery could lead to treatments for people with anaemia caused by kidney disease.

In the US, 30 million people have chronic kidney disease. Because the kidney makes erythropoietin (EPO), a hormone that triggers the production of red blood cells, people whose kidneys degenerate can develop anaemia – not having enough red blood cells to carry oxygen around the body.

EPO, which has notoriously been used as a doping agent in competitive sports, can be produced commercially and used as an anaemia treatment, but it is expensive. Such treatment requires a person to undergo at least three infusions of EPO a week, which is impractical for many, and the treatment can sometimes cause heart problems.

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Now researchers have made cells that produce EPO instead, and used them to treat mice. The team did this using stem cells from human cord blood, inducing them to become pluripotent stem cells capable of becoming any type of cell. By treating these with various growth factors, they were able to coax them into becoming cells that make EPO.

Smart cells

The team then transplanted these cells into the kidney cavities of mice with a form of kidney anaemia. Within four weeks, EPO levels in the blood of treated animals were 20-fold higher than in controls.

“Just one transplant of the human EPO-producing cells treated kidney anaemia in mice, keeping their haemoglobin levels in the normal range for the remaining 7-month lifespan of the animals,” says Kenji Osafune, of Kyoto University in Japan, who led the team.

“This is a very clever strategy that exploits ‘smart cells’ as factories for therapeutic proteins,” says George Daley of Boston Children’s Hospital. “Cells have the capacity to regulate protein production to suit demand, and offer considerable advantages over periodic injections of therapeutic proteins,” he said.

Journal reference: Science Translational Medicine, DOI: 10.1126/scitranslmed.aaj2300

Read more: Human blood stem cells grown in the lab for the first time

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