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Stem cell discovery promises health care revolution

April 13, 2009

 Scientists are using a new stem-cell technique that may someday revolutionize care for disorders as diverse as diabetes, Alzheimer's disease and muscular dystrophy.

Discovered by the Japanese scientist Shinya Yamanaka, the method creates stem cells without using and destroying human embryos. By studying cells created from people with inherited disorders, scientists are observing, in ways never before possible, how diseases progress and react to treatments, said Doug Melton, a Harvard University researcher.

"This is the breakthrough the stem-cell field has been waiting for," said Beth Seidenberg, a partner at Kleiner Perkins Caufield & Byers, the Menlo Park, Calif.-based venture-capital firm that helped start Google Inc.

Unlike embryonic cells, the cells created using the Yamanaka method opened a path to test drugs for genetic diseases, Seidenberg said.

The approach used in a study conducted at the Gladstone Institute in San Francisco is being adopted by labs in the United States, Europe and Asia and helped persuade GlaxoSmithKline Plc to invest $25 million in a joint venture with the Harvard Stem Cell Institute.

Since human embryonic stem cells were first isolated in a laboratory in 1998, they have fired the imagination of doctors, scientists and patients, who envision a day when new tissues or body parts might be grown to replace diseased ones. The cells are pluripotent, meaning they can be turned into any other type of cell, such as those that make up skin, nerves or neurons.

Embryonic cells also stir controversy. Obtaining them destroys the embryos they come from, placing the research along the same ethical fault line as abortion. Former President George W. Bush limited federal support for the science-a policy overturned this month by his successor, Barack Obama.

While politicians and citizens debated the morality of using embryonic cells, Yamanaka, a professor at Kyoto University in Kyoto, Japan, developed a technology that may make the argument moot. Yamanaka, who has two daughters, started his effort 10 years ago, after peering at a tiny embryo through a microscope and reflecting that it might form a child if it weren't used to make stem cells, he said in an interview.

"I saw that if we could make pluripotent stem cells without using human embryos, that would be ideal," he said.

In 2006, he scored his first success. Using a virus to insert four genes into the skin cells of mice, he started a process that returned the cells to a primordial state able to form any other cell in the body. Yamanaka named them induced pluripotent stem, or IPS, cells. The next year, he repeated the feat with human cells.

Yamanaka and researchers elsewhere are now racing to find better ways to achieve the same effect. They would like to get rid of the virus, which can cause the genes to lodge permanently in the structure of the cell and may trigger the growth of tumors.

Yamanaka's technique exploits a basic fact of human biology-that every cell in a person contains the genetic instructions that set that person's traits, from hair color to inherited disease. By taking skin cells from a person with a disease and turning them into cells in the heart, brain or pancreas that are affected by a genetic disease, researchers can experiment with disorders at their earliest stages, Melton of Harvard said.

IPS cells are now being created in laboratories because making them is far simpler than getting cells from embryos, said Jeanne Loring, founding director of the Center for Regenerative Medicine, part of the Scripps Research Institute in La Jolla, Calif.

"Every stem-cell researcher I know has made about a dozen," Loring said.

She estimates that researchers have made 300 different so-called lines of IPS cells, a number that may double this year. Each line is a colony of cells descended from the first ones made. Scientists keep them alive in culture and the cells keep replicating.

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