Infant gets gene transplant It may be answer to rare disease


LOS ANGELES -- A 5-day-old baby was sleeping soundly and in good condition after undergoing an experimental gene

transplant that may cure his rare blood disorder and give hope to newborns with other genetic diseases.

Andrew Gobea was diagnosed before birth with severe combined immunodeficiency (SCID), or "bubble baby disease" -- genetic disorder that leaves the body helpless to fight off infection.

On Saturday, doctors at Childrens Hospital of Los Angeles injected into the sleeping newborn a teaspoon of blood cells newly enriched with a gene whose absence left Andrew unable to make infection-attacking white blood cells.

Although a similar genetic therapy has been used on six older children in other countries during the past six months, Saturday was the first time that the procedure was administered to a newborn, said Carl Lenarsky, Andrew's doctor and an immunology researcher at Childrens Hospital.

"The beauty of it is that it is being done before the child gets sick," Dr. Lenarsky said.

Doctors hope that the procedure will cure Andrew's disease, so rare that only 50 to 100 babies are born with it each year in the United States.

Without the procedure, the dark-haired Andrew, who weighed 6 pounds, 12 ounces at birth, would have faced a risky bone marrow transplant, or weekly injections that cost hundreds of thousands of dollars a year, Dr. Lenarsky said.

With no treatment, Andrew probably would die in a year, he said.

Beyond the significance of the treatment for Andrew, Childrens Hospital researchers say newborns with many other genetic diseases -- sickle cell anemia and hemophilia among them -- could benefit from the ground-breaking technology.

The gene transfer also may be used to treat people with AIDS, Dr. Lenarsky said. "It probably wouldn't be a cure, but it might prolong remission," he said.

During the procedure on Saturday, Childrens Hospital doctors permitted Andrew's father, Leonard Gobea, 20, an unemployed laborer from the Imperial Valley town of Holtville, to push the syringe into his new son.

"It made me feel real good. I just wanted to do something," said Mr. Gobea, who wants his son to grow up to be a baseball player.

Andrew was born Wednesday afternoon at the Hospital of the Good Samaritan in Los Angeles to Gobea and Crystal Emery, 19, who both live in Imperial County. Imperial County is in the southeast corner of the state, bordering Arizona and Mexico.

The parents have been together since junior high school, Ms. Emery said. They are not married and live separately with their parents.

Both are carriers of a defective gene that led to Andrew's condition. Three years ago, the couple had a baby girl who suffered from the same condition. The infant died at 5 months after a receiving a bone marrow transplant.

Before Ms. Emery became pregnant with Andrew, the couple knew there was a one-in-four chance that the infant would inherit the potentially fatal disease.

An amniocentesis confirmed their fears.

"I just wanted a baby real bad. I just knew there was something they [the doctors] could do," Ms. Emery said.

In what doctors termed a startling coincidence, a second baby with the same disease was born Friday at the University of California, San Francisco, and will undergo gene therapy identical to Andrew's, Dr. Lenarsky said.

Immediately after the birth, umbilical cord blood cells from newborn Zachary Riggins of Exeter in Central California were flown to Childrens Hospital of Los Angeles, where doctors will add the missing gene and return the fluid to the University of California, San Francisco.

Doctors knew to test Zachary for SCID because he has a 2 1/2 -year-old brother who has the same disorder, officials said. Zachary is expected to be injected with the blood today.

Doctors have been researching the procedure for six years, but it was approved only last week by the Food and Drug Administration, Dr. Lenarsky said.

As a supplement, Andrew will receive twice-weekly injections of enzymes until he is healthy enough to survive on his own, said Dr. Kenneth Weinberg, a Childrens Hospital researcher.

Andrew will remain in isolation in the bone marrow transplant unit at Childrens Hospital for three to six months, until doctors determine that his immune system works well, Dr. Lenarsky said.

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