Roanoke Times
Copyright (c) 1995, Landmark Communications, Inc.
DATE: SUNDAY, May 16, 1993 TAG: 9305160139
SECTION: NATIONAL/INTERNATIONAL PAGE: A-1 EDITION: METRO
SOURCE: SHERYL STOLBERG LOS ANGELES TIMES
DATELINE: LOS ANGELES LENGTH: Medium
The 6 pound, 12 ounce baby with thick black hair was born Tuesday evening with an immune system that does not work. Saturday, a doctor infused him with a teaspoon's worth of genes intended to cure the defect that, if left untreated, kills most of its victims before they reach their first birthday.
The treatment makes Andrew the first newborn ever to undergo a cutting-edge brand of medicine known as gene therapy. It also employs, for the first time, a twist on previous attempts at genetic engineering: the use of blood drawn from the umbilical cord that was snipped from the baby at birth.
For Andrew, the highly experimental operation means a chance to live a normal life, without drugs that cost as much as $250,000 a year. For his parents - who three years ago lost an infant daughter to Severe Combined Immune Deficiency, the same disease that plagues their newborn boy - it means the chance to have a surviving child.
For a team of four pediatric immunologists and hematologists, Saturday's effort was the culmination of six years of painstaking investigation, of late nights in the laboratory and frustrating wrong turns and finally, the rare heady rush that comes when science moves out from under the microscope and into the operating room.
And for thousands of other infants who will be born with genetic disorders that can be diagnosed in the womb - hemophilia, sickle cell anemia, Gaucher's Disease among others - it represents a step toward treatment immediately after birth, and hence freedom from the debilitating effects of those diseases.
SCID is extremely rare; each year just 100 Americans are born with the disease. The most famous victim was David, the "boy in a bubble," who lived in isolation in a Houston hospital for 12 years before his death in 1984.
But gene therapy, which offers the best hope, is still in its infancy. Just 47 gene therapy projects, most of them designed to treat cancer patients, are now either approved or under way worldwide, according to Dr. W. French Anderson, a USC genetecist who is a pioneer in the field.
Andrew's drama began Tuesday at 6:25 p.m. at Los Angeles' Hospital of the Good Samaritan, where Emery gave birth. Right away, the obstetrician snipped his umbilical cord and began drawing blood. It was crucial that as much as possible be obtained. Dr. Donald Kohn, the gene therapy expert who headed the experiment, and his colleagues were hoping for 100 cubic centimeters - about 4/10 of a cup. They got half that.
With the blood drawn, a long night of laboratory work lay ahead, as technicians began the intricate process of isolating Andrew's "stem cells" - the parent cells of all other blood cells. For weeks, they had been practicing on a prototype machine borrowed from a Seattle-based firm, 17 practice runs in all.
Most gene therapy experiments target white blood cells; the therapy must be repeated every several months when the white cells carrying the new gene die. But stem cells stay alive in the marrow, continually spawning new cells. Thus if Andrew's therapy works, he will be cured.
"The stem cell is the holy grail of this work," says Kohn, who has spent the past six years in his lab at Children's Hospital working on these techniques. "It's the cell that we want to fix. If we can genetically fix the stem cell, then all the cells that it makes will have the right gene."
But experts say the process is fraught with obstacles. Stem cells are very rare in the blood, composing less than one tenth of 1 percent of all blood cells, and are difficult to isolate. And for reasons scientists do not entirely understand, genes introduced into stem cells do not function as well as those introduced into white blood cells.
It took Children's Hospital technicians nearly six hours of careful, intense work to separate the stem cells from the rest of Andrew's blood. They began with 500 million blood cells and ended with 3 million - about one teaspoon's worth. But the number of true stem cells remains a mystery to the doctors, part of a guessing game that made Saturday's experiment just that - an experiment.
A mouse leukemia virus altered to contain the ADA gene, and "growth factors" to help the cells divide were added to the genetic brew. At midnight, the mixture was put in an incubator, where it spent the rest of the week.
It will likely be six months before doctors know if Andrew's treatment has worked. He will get twice-weekly injections of an enzyme to shore up his immune system. Until doctors are certain that drug is working, he will remain in isolation.
by CNB