Sickle Cell Cured by Stem Cells in Mice

Dec. 7, 2007 -- Scientists have the first evidence that those "reprogrammed stem cells" that made headlines last month really have the potential to treat disease: They used skin from the tails of sick mice to cure the rodents of sickle cell anemia.

At issue: Turning adult cells into ones that mimic embryonic stem cells, master cells that can turn into any type of tissue. When scientists announced last month that they had successfully engineered embryo-like stem cells from human skin, it was hailed as a possible alternative to ethically fraught embryo research.

But no one yet knew whether those reprogrammed cells could create functioning tissue just like natural embryonic stem cells can.

Thursday, scientists in Alabama and Massachusetts reported a key next step when they used the technique to give mice with sickle cell anemia a healthy new blood supply.

The study, published in the journal Science, doesn't bring this potential therapy closer to people just yet. Big hurdles remain, including a risk of cancer from the reprogramming method.

But without the mouse work, scientists didn't know "whether all the recombined machinery will work or not," explained lead researcher Tim Townes, molecular genetics chief at the University of Alabama, Birmingham. "It's the first example of actually completing the cycle and curing a disease."

Townes had created a strain of mice bearing the human genes for sickle cell, a devastating inherited disease of deformed red blood cells that can't carry enough oxygen.