As her newborn sister was having groundbreaking cord blood transplant treatment for a rare genetic disorder that also had ravaged their brother’s brain, 5-year-old Katie Kristoff had one question for her parents: “Will this baby smile?”

Pamela and Bill Kristoff had struggled over whether to conceive a third child. Although Katie was healthy, younger brother Ryan was diagnosed with Krabbe’s disease when he was about 8 months old. Doctors said he wouldn’t live to see age 2.

Ryan’s devastated parents, in Colchester, Conn., scoured the Internet for information about Krabbe’s. They discovered the Hunter’s Hope Foundation, established in 1997 by NFL Hall of Famer Jim Kelly and his wife, Jill, after their infant son, Hunter, was diagnosed with Krabbe’s.

Through Hunter’s Hope Foundation, the Kristoffs learned that Duke University doctors had halted the progression of Krabbe’s with umbilical cord blood transplants. The catch: The earlier babies received a transplant, the better their chances for a normal life. For Ryan it was rather late.

Cord blood refers to the blood in the placenta and the part of the umbilical cord still attached to it after delivery. Hospitals usually discard this tissue. But it’s a rich source of blood-forming stem cells similar to those found in bone marrow. In Krabbe’s, those stem cells supply a critical missing enzyme, without which communication between the brain and the rest of the nervous system breaks down.

Elder brother Ryan remains severely handicapped as his transplant was unfortunately done much too late after significant brain damage. Kristoff says. “He requires lots of care.” But, she says of the transplant, “I think it did make him more comfortable. He loves to be moved around. Before, we couldn’t even hold him.” Best of all, she says, “six months ago, he started smiling again.”

The family arrived at Duke on July 5, and Alyssa Ryan Kristoff was born two weeks later. If not for Ryan, Kristoff says, Alyssa, who got a transplant at 3½ weeks old, wouldn’t have had a chance.

Although she’s extremely small for her age and her motor skills are delayed, after cord blood transplant at 31/2 weeks, Alyssa has far surpassed Ryan’s accomplishments. She still needs a feeding tube, and she may never walk. But, her mom says, she can hold her head up, grab toys and smile, smile, smile.

“The first time we heard one of her toys crinkle, we were crying,” Kristoff says. “She’s reaching milestones he never met.”

Krabbe’s disease is a rare genetic disorder. Though Krabbe’s affects only about 1 in 100,000 live births, about 1 in 5,000 babies born in the USA have some type of metabolic disorder that could be treated with a cord blood transplant, says Joanne Kurtzberg, head of the Duke Pediatric Blood and Marrow Transplant Program. Others include Tay-Sachs and adrenoleukodystrophy, the condition made famous by the 1992 film Lorenzo’s Oil.

Worldwide, 5,000 to 6,000 cord blood transplants have been performed since 1988, says Pablo Rubinstein, director of the New York Blood Center’s National Cord Blood Program, the world’s oldest and largest cord blood bank.

Any parent banking his childs cord blood is not only providing insurance for her beloved child, but in the process possibly saving other children too.

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