Furtive First

Genetic jump from mice to man

Just this past spring, scientists at U.C.L.A. announced that they had inserted foreign genes into the bone-marrow cells of mice, the first attempt at using new genetic-engineering techniques with living animals. But experiments on humans, experts said, were still years away. Not so. Last week it was disclosed that the great divide between research in mouse and in man had been quietly crossed.

In July, U.C.L.A. Hematologist Martin Cline and colleagues at Jerusalem's Hadassah Hospital on Mount Scopus and at a clinic of the University of Naples performed gene transfers on two female patients. Both had severe thalassemia, an inherited blood disorder in which the bone marrow produces red cells with defective hemoglobin (the molecule that carries oxygen to body tissues). Victims need frequent blood transfusions, but this leads to a buildup of iron in the body, particularly the heart, that can eventually cause death.

Cline and his collaborators treated their patients by removing a small amount of bone marrow and mixing it with genes capable of directing production of normal hemoglobin. The genes had been manufactured by bacteria altered by recombinant-DNA techniques. The marrow cells, now bearing the new genes, were then injected back into the patients. There is as yet no sign that their reconstituted marrow cells are producing healthy hemoglobin. But the story of the experiment, which was broken by the Los Angeles Times, has raised questions about whether the effort was premature. U.S. regulations require investigators to get approval for tests with humans from special committees at their own institutions. Cline had submitted a proposal for such work to U.C.L.A. last year, but its committee turned him down, citing the lack of study with animals. The decision came at the end of July, just days after the human experiments had been done abroad.

Cline says that safety guidelines similar to those in the U.S. were followed in Israel and Italy. But, observed one Israeli hematologist, "if this type of research is forbidden in the U.S., a world model for such work, I would hesitate to approve it in my own country." In Washington, the National Institutes of Health's Office for Protection from Research Risks has asked U.C.L.A. for a full report. Declares Cline: "After consideration of all the scientific and moral aspects, I'd do it again."

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