W. French Anderson Papers

William French Anderson was born in Tulsa, Oklahoma on December 31, 1936. He received both his undergraduate and medical degrees from Harvard, the latter in 1963. After an internship at Children's Hospital Medical Center in Boston and a post-doctoral fellowship at Harvard, Anderson began his 27-year career at NIH with a 2-year position through the USPHS surgeon service in 1965. From 1965 into 1968 he worked for Dr. Marshall Nirenberg in the National Heart Institute's (NHI) Biochemical Genetics Laboratory, where he became an integral member of Nirenberg's DNA-coding team.

From the very start of his medical training, Anderson's primary interest was to develop a means of healing by replacing faulty genes that caused a disease with normally functioning genes. In 1968 he became chief of NHI's newly-created Section of Human Biochemistry and began researching gene therapy exclusively. During this same period he also taught molecular genetics at George Washington University School of Medicine and was a faculty member of the NIH Graduate Program's Department of Genetics.

From then until the early 1980s, Anderson pursued gene repair using microinjection techniques. The results, however, proved unsatisfactory. By 1980 he had reached an impasse in his research and began exploring other medical fields, notably sports medicine.

His interest in gene therapy was revived when experimentation with gene replacement, notably by Richard C. Mulligan, demonstrated the effective use of a retrovirus as a genetic vector. Seizing upon the possibilities, Anderson determined that the most likely initial candidate for genetic transplantation would be adenosine deaminase (ADA), due to its relative simplicity. When deficient, this enzyme causes a human body's immune system to fail leading to severe combined immunodeficiency (SCID). Persons suffering from this condition rarely survive childhood and are vulnerable to nearly every pathogen in the environment.

Though experimentation with ADA in primate bone marrow proved only marginally encouraging, Anderson felt confident that the tests should be conducted in humans. Throughout 1986 and 1987 he began the groundwork to obtain approval for human ADA gene therapy from NIH, NIH's Recombinant DNA Advisory Committee (RAC), and the Food and Drug Administration (FDA).

By early 1989 Anderson, and partners Dr. Steven A. Rosenberg and Dr. Robert M. Blaese from NCI, had secured approval from NIH, the FDA, and RAC's Human Gene Therapy Subcommittee to conduct a genetic transfer experiment on 10 terminal human subjects whose treatments by all known methods had failed. The technique involved re-inserting a malignant melanoma patient's own modified tumor-infiltrating lymphocytes (TIL), which had been imbued with a distinctive marker. On May 22, 1989, Maurice Kuntz received the marker injection which Rosenberg called "the first time ever that a new gene has been introduced into a human." This successful experiment demonstrated that gene transfer in humans was safe.

Encouraged by the experiment's outcome, Anderson and his team sought approval to take the procedure a step further and initiate human gene therapy trials with an ADA/SCID patient. Shortly after receiving FDA's approval, Anderson replaced the defective ADA gene in 4-year-old Ashanthi DeSilva on September 14, 1990. The procedure was recognized as the first time a healthy gene had been infused into a human. In January, 1991, 9-year-old Cindy Cutshall was also treated. The treatments did not bring about total correction of SCID in either patient, but were therapeutically substantial enough that suppported by a regular medication routine the patients were able to enjoy relatively normal lives.

Although scientific consensus about the trial's success was mixed, the groundbreaking work by Anderson and his team in developing and demonstrating the efficacy of genetic transplantation was hailed internationally as the first steps in a new field of medical treatment. Because of this work Anderson has been called "The Father of Gene Therapy."

Anderson left the NIH in 1992 to accept a position as director of the Norris Comprehensive Cancer Center's Gene Therapy Laboratories (GTL) at USC's Keck School of Medicine, and as professor of biochemistry and pediatrics.