Donor funds

Breathe new life into CDH research

Eight-year-old Danny Murphy, born with congenital diaphragmatic
hernia (CDH) and saved by a cutting- edge therapy, loves to strike
a “muscleman” pose.

Eight-year-old Danny Murphy dreams of growing up to be just like his idol, Arnold Schwarzenegger. But the real hero in Danny's life is Jay Wilson, MD, a surgeon at Children's Hospital Boston. In turn, Wilson's heroic work has been aided by a woman Danny has never met. Dina Zahid's generous gift to Children's gives kids like Danny, born with congenital diaphragmatic hernia (CDH), a chance for survival her own child missed.

CDH occurs in one of every 2,500 births. In the developing fetus, the diaphragm — the muscle that separates the chest and abdominal cavities — fails to close, allowing the abdominal organs to migrate into the chest, compressing the heart and lungs. While surgery can correct the diaphragm shortly after birth, the underdeveloped lungs cannot deliver enough oxygen and a CDH newborn requires immediate high-tech respiratory support.

A chance to draw breath
Twenty years ago in the U.S., only about one in five babies with CDH lived. Now more than half pull through, and at Children's, the survival rate has reached 90 percent. That's because Jay Wilson, the leading CDH expert, directs the world's only CDH clinic and has advanced research and treatment in the disease. "With Russell Jennings, MD, and the Children's Center for Fetal Diagnosis and Treatment, we've pioneered one of the most successful procedures for CDH newborns," says Wilson. "EXIT to ECMO is a heart and lung bypass performed while a baby is still attached to the umbilical cord and before he or she draws a breath." ECMO — extracorporeal membranous oxygenation — gives infant lungs precious time to rest and heal before and after corrective surgery, and, hopefully, to grow strong enough to breathe on their own. "But ECMO can be used for only a few weeks," Wilson says. "Sometimes that isn't adequate for enough lung growth."

For the past 14 years, Wilson has studied normal lung development, discovering that lung growth rate is not fixed, and can be dramatically increased both in the fetus and newborn. Searching for ways to accelerate growth before and after birth, he found that temporarily filling animal models' lungs with perfluorocarbon, an inert liquid, stretched and stimulated them to grow.

Beating the odds
By January 1996, Wilson was ready to launch a small pediatric clinical trial — just in time to help Danny Murphy. CDH had left Danny's left lung, which normally would be the size of a lime, only as big as a quarter, and his first week on ECMO wasn't enough to increase his lung capacity. "Dr. Wilson told us perfluorocarbon might help," says his mother, Helen. "We were willing to try anything to save his life." Danny spent a critical second week on ECMO, while perfluorocarbon gently expanded his tiny lungs. It worked, and after four months in the hospital, Danny was able to go home, where he spent another year attached to an oxygen tank. Today he's an eager second grader who loves reading, swimming in the family pool and being a protective big brother to his 4-year-old sister, Krista. "He's my miracle child," says Helen. "Dr. Wilson sent his lung X rays all over the world to show what perfluorocarbon treatment can do."

Despite promising results, further clinical trials were stalled when the manufacturer of perfluorocarbon went out of business and funding was depleted. Then Wilson's research received help from an unexpected source. In 1997, Dina Zahid learned her unborn child had CDH, and her doctors in London were discouraging. "When we heard about Dr. Wilson, we knew we'd have a better chance in Boston," she says. Her son Mohammed was born here January 29, 1998, and placed immediately on ECMO, but his condition was complicated by an unexpected heart ailment. Tragically, he survived only a few weeks. "We have a saying in Islam that babies who die become birds in heaven, where they wait for their parents to join them," Mrs. Zahid says. Before returning to England, she and her family made a gift of $250,000 in Mohammed's memory, to be used for further CDH research. With the gift, Wilson and his team resurrected the research, even without a source of perfluorocarbon. "Our combined clinical and research efforts increased CDH survival rates from 70 percent in 1997 to 90 percent today," says Wilson. More important, these results attracted further support from a new pharmaceutical manufacturer. This year, Children's will participate in a national multicenter perfluorocarbon trial that promises gentler and more effective therapies for thousands more CDH children.

Dina Zahid can see the seeds of her generosity flourishing. "Baby Mohammed was very much loved — it was terrible to see him suffer and not be able to comfort him in intensive care," she says. "I knew then I wanted to help increase the chance of survival for other babies with this disease." Today, her loving gift means the breath of life for someone else's child.