Watching and Waiting : Infant to Finally Go Home, but Success of Gene Therapy Uncertain

On occasion, when hospital nurses are not looking, Crystal Emery and Leonard Gobea slip off the surgical gloves and mask they are required to wear when they cradle their infant son and steal a kiss on the baby’s forehead, or gently stroke his tender skin.

“Sometimes,” Gobea said wistfully the other day, “you wonder if he really knows your touch. When we get him home, will he recognize us?”

Monday, the parents hope to get their answer. Andrew Gobea, the dark-haired baby boy who made medical history May 15 by becoming the first newborn ever to undergo gene therapy, is scheduled to go home--less than two months after the landmark experiment but at least four months before doctors will know if it has worked.

Doctors at Childrens Hospital Los Angeles say the baby, who was born with an immune system that did not work, is ready to be released because he is responding exceptionally well to drugs that give him immunity by delivering a crucial enzyme that his body fails to manufacture.

“He has done as well as we could have possibly expected,” Dr. Carl Lenarsky said. “He is growing and developing, and the very good news is that he is developing signs of immune function, probably because of this medicine. We think it is really too early to see evidence of immune function from the gene transplant.”

The gray-eyed baby with the hands that his dad says are destined to make him a baseball player has indeed grown considerably since his birth May 11. He was 6 pounds, 12 ounces then; he now weighs 9 pounds, 4 ounces. He has strong legs and arms, his parents say, and has demonstrated a distinct affinity for a big blue plastic bunny that squeaks when you squeeze it.

For Andrew, the impending release means he will no longer have to live in a “bubble”--a sterile isolation unit on the fourth floor of Childrens Hospital. For his parents, who come from the Imperial Valley, it will mean a break from cafeteria food and a seemingly eternal stay at the Hollywood Travelodge and a chance, finally, at a life with their son.

Said Emery: “We just want to raise him like a normal baby.”

Complete normalcy, however, will not come right away. For at least the next four months, while doctors continue to monitor the strength of his immune system, Andrew’s parents will not be able to take the baby to malls or restaurants or other crowded places where he might be exposed to infection.

“His immune system is certainly not normal,” Lenarsky said. “But we feel that there is enough of an immune system there to remove him from the bubble.”

Andrew suffers from a rare genetic disorder called ADA deficiency, in which he fails to manufacture the enzyme adenosine deaminase, which enables the immune system to fight infection. Three years ago, Emery and Gobea lost an infant daughter to the same disease. When prenatal testing revealed that Andrew, too, would be afflicted, doctors at Childrens Hospital decided to attempt the gene therapy.

The historic experiment employed a novel technique: the use of blood from Andrew’s own umbilical cord. The blood was drawn as soon as Andrew was born. That night, laboratory technicians began the intricate process of altering the blood to include genes that, doctors hope, will insert themselves into Andrew’s stem cells, the parent cells of all other blood cells.

On May 15, in an emotional procedure, Dr. Donald Kohn, a gene therapy expert at Childrens and USC, infused the genetically altered blood into Andrew. If, as doctors hope, the stem cells give rise to other cells that manufacture the correct enzyme, Andrew will be cured.

Although tests reveal that Andrew is now manufacturing lymphocytes that are crucial to the functioning of his immune system, Kohn and his colleagues cannot say if the new genes are expressing themselves. In the meantime, Andrew will make weekly visits to a doctor in San Diego County, and monthly visits to Childrens; blood drawn during those visits will later be tested for evidence of the gene.

At the same time, Kohn is monitoring the progress of another gene therapy baby in Northern California. Several days after Andrew’s birth, the same experiment was performed on Zachary Riggins by San Francisco doctors who collaborated with Kohn. Zachary, who is also responding well to drug therapy, went home July 2.

The other day, his mother sent Emery and Gobea a letter and a small, engraved gold pin. “Andrew and Zachary,” it read. “Gene Therapy, 1993.”