Connor: A Brighter Future

Six-year-old Connor has hemophilia B. Two weeks before he was born, the FDA approved use of a recombinant DNA lotting factor that would dramatically improve treatment for this genetic disease that causes uncontrolled bleeding.

WINTER 2001 - Leslie Woods has two uncles diagnosed with hemophilia B -- a genetic disease that can result in uncontrollable bleeding from an injury. The hemophilia gene is usually transmitted from mothers to sons, and because of her family history, Leslie knew she had a 50-50 chance of being a carrier.

"When our son Connor was born in 1997, we decided to have his umbilical cord blood tested," says Leslie.

"We found out that he does, in fact, have moderate hemophilia B, which means that his blood contains abnormally low levels of a clotting protein called factor IX."

Connor's pediatrician is Kim Harvey, M.D., a member of the Stanford Department of Pediatrics.

"As soon as Connor was diagnosed at birth, Dr. Harvey made sure that we were called by the Packard hematology team that day," Leslie remembers.

It wasn't long before Connor was placed under the care of Bertil Glader, M.D., Ph.D., who has been treating pediatric blood diseases for more than 20 years.

Hemophilia comes in two forms. Patients with hemophilia A have low levels of clotting factor VIII. Patients with hemophilia B, a more rare type, have low levels of factor IX.

"Just two weeks before Connor was born, the Food and Drug Administration approved the release of recombinant DNA factor IX," says Leslie. "Before that, he would have needed blood transfusions and been at risk for contracting hepatitis C."

According to Leslie, the treatment of hemophilia is dramatically better than when her two uncles were growing up.

"If they had a laceration, their parents would be up all night while they bled and bled," she remembers. "If they lost too much blood, they'd get a transfusion."

Leslie also hopes that gene therapy to treat hemophilia, under study at Packard and Stanford, will work and some day be available to children, but her feelings for Packard go well beyond its renown for cutting-edge research.

"When you get down to it, all that medical technology is valuable," says Leslie. "But equally important is the fact that I feel comfortable at Packard. Dr. Glader is so caring and concerned about Connor and our family. And the nursing staff, they're just there for us. They make us feel that we're in this together. All the technology can't replace that."