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Miracle cells

"Miracle cells" Continued...

Issue: "Johnny Carson: In memoriam," Feb. 5, 2005

Meanwhile, Drs. Mitchell and Faustman, who have credible data on treating strokes, kidney damage, and diabetes-some of same diseases the NIH says embryonic stem cells can cure-are denied funding. "I think people who want embryonic stem cells just don't want [alternatives] to work," Dr. Faustman said.

But the alternatives are working, miraculously. Four years ago, doctors diagnosed Steve Barsh's 1-year-old son, Spencer, with adrenoleukodystrophy. ALD, featured in the 1992 movie Lorenzo's Oil, is a degenerative brain disease that usually only affects boys. Doctors told the Barshes there was little they could do for Spencer. There was a 50 percent likelihood he would die before age 10, and a 75 percent likelihood the disease would affect his brain, most likely leaving him disabled.

The Barshes refused to accept those odds. They started the Stop ALD Foundation to drive research to find a safe therapy for the disease. The Barshes devoted themselves to the foundation until just after Spencer turned 2, when he had trouble healing from brain surgery, a complication of ALD.

"We ran out of time to do research," Mr. Barsh said. Although the foundation continued its work, and still does today, the Barshes focused on getting Spencer the best treatment available. They found Dr. Joanne Kurtzberg and her colleagues at Duke University Medical Center. With the help of a five-year grant from the NIH, doctors at Duke were using stem cells from the blood in umbilical cords to treat children with diseases like Spencer's.

The Barshes moved from Philadelphia to Durham, N.C., for seven months while doctors treated their son. ALD affects the body's ability to break down a kind of fatty acids, which leads to excess fatty acids eventually causing brain damage. Doctors transplanted umbilical cord blood stem cells into Spencer in hopes that the healthy stem cells would help his body break down the fat molecules.

The stem cells did what the doctors hoped; they stopped Spencer's ALD. But something else happened, too, something the Barshes and Dr. Kurtzberg discuss gingerly. The treatment not only stopped the disease, it also reversed the effects ALD had on Spencer's brain, contradicting the scientific notion that it is impossible to heal the brain. Today, Spencer is a normal, healthy 5-year-old boy.

With the five-year grant they received from the NIH, Dr. Kurtzberg and her colleagues successfully treated other children with ALD, leukemia, sickle cell anemia, and severe combined immune deficiency, also known as bubble-boy disease. The same year President Bush set rules for federal funding for embryonic stem-cell research, the NIH chose not to renew the cord blood transplant grant.

"The NIH said, 'Congratulations, cord blood transplants work. We fund basic research. You are now beyond that. You now need to get funding from somewhere else,'" Mr. Barsh said. There was no money left for Dr. Kurtzberg to do clinical trials, but so much left to discover.

Dr. Kurtzberg thinks that further research into diseases such as ALD could lead to a cure for adult diseases like Parkinson's. "We still have a lot to learn about understanding why cells do what they do," she said.

The federal Health Resources and Services Administration received $10 million in appropriations last year for collecting and banking umbilical cord blood. Some of that money could eventually support research. The NIH, which bankrolls innovative medical research in the United States, has funded only 30 projects involving stem cells from umbilical cords. In contrast, it has funded 634 projects involving embryonic stem cells.

Though the priority for funding umbilical cord stem-cell research is low, the promise is great. A scientist in Denmark has shown stem cells from umbilical cord blood can turn into brain, bone, cartilage, liver, and heart cells. In Lawrence, Kan., Dr. Mitchell's research has led to stem cells from the inner tissue of umbilical cords, also known as the matrix, producing nerve cells.

But her passion is getting stem cells to repair damaged kidneys. Two years ago, her nephew died of acute renal failure, a complication of leukemia. At the time, a colleague in the KU medical school was urging Dr. Mitchell to apply her stem-cell discoveries to kidney damage. "I hate to tug at your heart strings," she remembers him telling her, "but this is the kind of thing maybe your stem cells could cure."

Despite her previous rejections, Dr. Mitchell plans to apply again to the NIH this February for a grant to test her stem cells in animals with damaged kidneys. She is a single mother who went to college for the first time after she had five children, and she has a calm patience when it comes to the NIH.

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