Stem-cell advance reported

Scientists have created the first personalized stem cells for patients with a genetic disease by rewinding their skin cells to an embryonic state, according to a study published Thursday in the online edition of Science.

The researchers then converted some of those stem cells into the two kinds of brain cells that cause their crippling disease, amyotrophic lateral sclerosis (ALS), commonly known as Lou Gehrig's disease.

This is the first time scientists have coaxed embryonic-like cells from adult patients suffering from a genetic-based disease, then induced the cells to form the specific cell types needed to study and treat the disease.

"It's a big step forward," said Stephen Duncan, a stem-cell researcher at the Medical College of Wisconsin in Wauwatosa, who was not involved in the research.

"We're on the threshold of a new era of technology ... where we might have some definitive strategies to better treat or cure diseases like ALS or other neurodegenerative diseases," said Medical College neurologist Paul Barkhaus.

The new cells were derived from skin removed from an 82-year-old woman and her 89-year-old sister, who share a rare genetic mutation that causes about 2 percent of ALS cases.

The scientists from Harvard University and Columbia University focused on the rare form of ALS in part to test whether cells from elderly patients could be reprogrammed, said biologist Kevin Eggan of the Harvard Stem Cell Institute.

"It's possible to use these cells to make the actual cell type that is destroyed in that person's disease," said Eggan, the study's senior author. "It takes the study of disease out of the patient, where it's very difficult, and into the Petri dish."

Future research should find safer reprogramming methods, scientists say. In the meantime, the new reprogrammed stem cells will be valuable for understanding and combating the damage ALS does.

Information from the Milwaukee Journal Sentinel is included in this report.