Belly-Button Brothers
When their 11-month-old son Layne died abruptly of liver failure after an infection with Epstein-Barr virus in 1994, Theresa and Scott LaRue were devastated. Layne, it turned out, had a rare inherited disorder that severely compromised his immune system. And when doctors at the UCLA Medical Center tested the three other LaRue children, two were found to be similarly afflicted.
As the disease's tongue-twisting name implies, instead of simply dispatching an invading virus--like the one that killed Layne--X-linked lymphoproliferative syndrome (XLP) allows white blood cells (including lymphocytes) to grow unchecked, destroying vital organs like the liver, lymph glands and spleen. Inherited by males on their X chromosomes, it usually means death by age 10.
The LaRues could have opted for bone-marrow transplants. But these are painful, require precise genetic matches that can take months to find and often fail. "The doctors basically told us [transplants] would either kill them or save them," says Theresa. So they chose an experimental alternative: transfusing the youngsters with a type of stem cell harvested from a newborn's umbilical cord and placenta. Unlike their more controversial cousins, embryonic stem cells, which are harvested from aborted fetuses and can develop into almost any cell, cord blood cells are used to rebuild blood and immune systems--exactly what the LaRue boys needed. In effect, says UCLA's Dr. E. Richard Stiehm, "we transplant another baby's immune system into the sick child's body."
There were risks, however. The new cells take longer to establish, exposing the child to infection. Also, as Stiehm explains, if the transplant doesn't work, "you don't have a second chance," because you're unlikely to find matching cord blood.
In early 1995 the LaRues let the UCLA doctors proceed with their son Blayke, then eight months old. "It was a horrible decision," Theresa says, and for a while they regretted it. Blayke languished in the hospital for two years. First, his new immune system began attacking his spleen. Surgery solved that problem, but he was still so sick he had to be fed intravenously for many more months.
In February 1997 it was four-year-old brother Garrett's turn. His ordeal was mercifully briefer. After four months, including 10 days of chemo, Garrett was out of the hospital--with a temporarily bald pate but a spanking-new immune system. Heartened, the UCLA doctors did a cord transplant on a third boy, Billy Bodine, 11, to correct a similarly inherited immune deficiency called X-linked hyper-immunoglobin M syndrome.
Last week, after two years of post-transplant observation, the UCLA doctors felt confident enough to pronounce all three boys cured. "They're as healthy as anyone," says Stiehm, who sees them as proof that cord blood can save many more young lives. --By Frederic Golden. Reported by Dan Cray/Los Angeles
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