Dear Henry

'U' scientists to test stem cell treatments in children
Josephine Marcotty, Star Tribune

Published February 8, 2004

Adult stem cells discovered in bone marrow by University of Minnesota researchers soon will be used for the first time as an experimental treatment in people, according to university scientists.

Researchers at the university's Stem Cell Institute want to find out if they can repair damaged tissue in patients who have had radiation and chemotherapy.

The stem cells were first identified in 2002 by a team led by the institute director, Dr. Catherine Verfaillie. The researchers injected genetically marked stem cells into mice that successfully formed new liver, intestine and lung cells.

Now Verfaillie and Dr. John Wagner, a professor of pediatrics at the university, want to find out if they do the same thing in 16 children with a rare inherited blood disease called Fanconi anemia, which is usually fatal.

"That would be huge," Wagner said, because it would show that the stem cells have the potential to replace unhealthy cells all over the body.

Fanconi anemia leads to bone marrow failure. Children born with it are treated with radiation and chemotherapy to destroy their defective bone marrow.

It is replaced with healthy blood-making cells from a donor. However, the radiation and chemotherapy leave the children with damage to their lungs, liver and intestines.

Wagner intends to extract stem cells from the same donor and grow them in a lab. He plans to inject the adult stem cells in the children when he replaces their bone marrow. He hopes the stem cells will transform themselves into other cells to repair the damaged organs.

While the risks of the procedure are unknown, researchers did not observe dangerous side effects in animal experiments.

If the procedure works, it will have enormous implications for all cancer patients who have radiation and chemotherapy treatments, Wagner said.

"And we hope it can be even bigger than that," he said. Eventually, he thinks he can seed patients' bone marrow with their own genetically repaired stem cells, curing the disease without a bone marrow transplant, he said.

Josephine Marcotty is at

>marcotty@startribune.com