GEORGE STEINMETZ FOR TIME W. French Anderson: Director of Gene Therapy Laboratories and professor of biochemistry and pediatrics at the U.S.C School of Medicine

The Pioneers of Molecular Biology: W. French Anderson
	As an undergraduate, he asked the question at the heart of gene therapy
	By Leon Jaroff

Posted Sunday, February 9, 2002; 10:31 a.m. EST
W. French Anderson was still a senior in high school when he heard that "some people in England" had figured out the structure of DNA and published their reportin the journal Nature. That journal was hard to come by in Tulsa, Okla., but Anderson persevered, and a few weeks later was excitedly reading and rereading the Watson and Crick paper.

"I marveled at it," says Anderson, "but I really didn't understand it. I had no idea, for example, what X-ray diffraction was." But he was a precocious student and he won a scholarship to Harvard, where he audited a graduate chemistry course given by John Edsall, an expert in proteins. At one of Edsall's 1958 seminars, a visiting British scientist was lecturing about efforts to determine the structure of the hemoglobin molecule, which transports oxygen in the bloodstream.

Suddenly a thought occurred to Anderson, the only undergraduate in attendance. He raised his hand, and blurted out the idea that spawned gene therapy: "If you know the structure of normal hemoglobin, you could certainly know the structure of sickle hemoglobin." And because both of those structures are produced by genes, he argued, "you have a normal gene that makes normal hemoglobin and a mutant gene that makes sickle hemoglobin." Why, he wondered, "couldn't you take the gene for the normal hemoglobin and give it to a patient with sickle cell anemia and cure the disease?"

The room fell silent. Looking at the student disdainfully—"he bored a hole right through me," Anderson remembers—the lecturer admonished him: "This is a serious scientific meeting. Don't waste our time." "I just shrank," Anderson says. "I felt like crawling under the table. As soon as this thing was over, I was going to try to just sneak out." But as he stood to go, he felt a hand on his shoulder. It was John Edsall, who had been sitting behind him.

"Interesting idea," said Edsall, and walked off. Anderson's humiliation evaporated. "By God," he thought, "if John Edsall thinks it's an interesting idea, then it's an interesting idea, and that's what I'm going to do." From that moment on," he says, "gene therapy was my obsession and my lifetime goal."

Anderson has kept his eye on that goal ever since. He won approval for and helped conduct the first human gene therapy trial in 1990, treating two young girls with an immune system disorder, and has since been involved in 18 different gene therapy trials. He founded and still edits the influential journal, Human Gene Therapy, and served as the discipline's leading spokesman when it suffered a series of setbacks, including the death of a young research subject three and a half years ago and the emergence last year of a leukemia-like illness in a patient treated for the so-called bubble-boy disease.

"Every powerful technology has a downside," says Anderson, "and there will be setbacks along the way." Gene therapy clearly has more pitfalls than he had anticipated, but he is confident it will shake off its growing pains and play a major role in 21st century medicine.

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