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Gene Therapy Race : Egos, Prizes on the Line for Scientists

First of two parts. Next: The question of selective advantage.

December 13, 1987|BARRY SIEGEL | Times Staff Writer

BETHESDA, Md. — Late in the morning last Monday, two scientists faced each other, one sitting, one standing, in a small conference room here at the National Institutes of Health.

W. French Anderson, 50, director of the laboratory of molecular hematology at the National Heart, Lung and Blood Institute, had been explaining to an NIH advisory committee why he wanted to make the first authorized attempt to change a sick human being's genes. Now he was leaning back in his chair, fielding questions from a committee member, Richard C. Mulligan, 33, an MIT associate professor and lab director at the Whitehead Institute for Biomedical Research in Cambridge.

"I am curious, French," Mulligan said, pacing before a chalkboard. "Do you think it would work if you tried it?"

Anderson chuckled.

"That's the question," he said. "Do we think that it will work? We've asked that ourselves during pizza sessions many times. An even better question is, if it was my own child--"

"Let's keep it to the--" Mulligan began to interrupt.

"No one really knows," Anderson quickly replied.

In that momentary exchange lies a story about science and scientists, politics and ambition, ethics and philosophy.

While most of his colleagues and competitors believe gene therapy is still years in the future, Anderson wants to begin experiments with human beings. His campaign is forcing consideration of questions long simmering in the complicated, hybrid world of scientists, ethicists, lawyers and government regulators.

When is the right time to make a transcendent leap, and how to decide? Must we fully understand the basic science involved and feel certain we will cure the patient, or should we accept uncertainty and view the first attempts as part of the experimental research?

How do we balance the dangers in premature application against the waste of undue delays?

Classic Conflict

Do we have a workable means to settle public policy in this complex field?

These issues involve divisions among many types of people over all sorts of matters--scientific data, scientific philosophy, moral judgments, politics, public opinion. But the conflict at its most basic can be seen as a classic one between two types of lab investigators: the clinicians and the basic researchers--that is, the medical doctors and the molecular biologists.

Making such a distinction risks oversimplifying matters--the differences do not always break along such clear lines--but the two groups do tend to look at issues differently. Anderson is a medical doctor. Mulligan is a molecular biologist.

From afar, scientists in white coats in a lab seem all of a group. Closer up, the differences seem more apparent than the similarities.

The doctor's training in medical school gears him to act, to treat patients, to apply science, to get results. He thumps chests and listens to hearts. The molecular biologist earns his Ph.D. in a lab, amid test tubes and vials and slides. There he learns technique and patience and a distrust for anything the hard data cannot prove. He seeks not results so much as a general understanding into the workings of genes and cells and viruses.

How to do science? This is the eternal tension in the lab between these two groups. This is the present tension coloring the debate about gene therapy.

The story of how Anderson jumped into the race to do human gene therapy, and forced consideration of such issues, provides an unusual look at the world of scientists working on the cutting edge of a historic, pioneering field.

Tortured Lurches

Theirs is a world of conflict and confusion more than consensus and certainty. What seems an unchecked march from ignorance to knowledge most often involves tortured, random lurches in the dark that eventually find the light. Their science is nothing so precise and objective that it can be agreed upon by all. The truth depends on where the scientists stand and who they are and in which direction they direct their gaze.

It is difficult at times to separate the scientists' intellectual differences from matters of ego and ambition and politics. Competition, after all, is the engine that drives the quest.

They compete for grants--funding is their lifeblood, and funding is scarce. Profits from commercial applications also loom, albeit in the distant future. Anderson directs a private lab, Genetic Therapy Inc., funded by a venture capital arm of an investment banking firm. Most other figures in the hot molecular biology field have some sort of commercial connection.

Above all, though, they compete for recognition. The prospect of a Nobel Prize hovers over everything. Altering defective genes promises to provide cures for all manner of disease now considered untreatable, diseases that only recently have been linked to genetic causes. Molecular medicine offers a new way to attack illness. When gene therapy comes it will represent medicine's second great revolution in this century, following the emergence of antibiotics in the 1950s.

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