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.................................................................................................... .................................................................... Book Review: Genetic Medicine Correcting the Code, by Larry Thompson. 1994. New York: Simon and Schuster, 378 pp., $23. n 1990 medicine entered a new Iage. Since Galen, medicine has been largely a matter of cut it out, kill it, or palliate it. Drugs are devel- oped by trial and error: throw a range of chemicals at a disease and see what works. Often one doesn't know why a given drug works-it's enough that it does. Genetic dis- eases have been among the hardest to treat. In the most successful cases, a person suffering from a disease caused by a missing or damaged gene can receive a drug that replaces the culprit protein the gene should make. Hereditary diabetes is an example. But in some cases such treat- ments gradually lose their effective- ness; in others, the quantity of drug the patient must take is enormous, painful, and inconvenient. For years doctors-and patients-have fanta- sized about how nice it would be to just fix the bad gene once and for all and cure the patient. This is the dream of gene therapy. Larry Thompson's excellent new book chronicles the path to- ward the first successful gene thera- py trials. Thoroughly researched yet highly readable, Thompson's book traverses the high wire of good, careful writing, stretched over the yawning chasms of sloppiness and sensationalism. He begins with the story of Ashanthi DeSilva, a 4-year-old girl with Severe Combined Immune De- ficiency (a form of the disease made famous by David the Bubble Boy). Ashi's form of the disease results from loss of a single gene, one that makes an enzyme called Adenosine Deaminase (ADA). Without ADA, a child's immune system cannot function. Thompson tells the story of her first gene therapy injection, when scientist Ken Culver injected into her arm cells that had come from her bone marrow and, in a test tube, had the missing gene inserted. It is done with high drama, and the prose here is perhaps a little purple. But one goes along for the ride, be- cause it is in fact a dramatic mo- ment in the history of medicine. The rest of the book is essen- tially a flashback, with Thompson relating the history of the events leading up to the momentous injec- tion. As with most historic events, the trail to this one is littered with high pressure, intense competition, big egos, a few heroes, a few villains. It's a good story, well told. It's im- portant to realize that gene therapy didn't spring fully formed out of an NIH lab in 1990. Scientists have been dreaming about curing disease by altering genes at least since the 1960s. During the 1980s serious at- tempts were made, most notably by UCLA scientist Martin Cline. My one quibble with the book is with the title. The "code" referred to is the genetic code, the ancient cipher cells use to translate DNA into protein. With a couple of obscure exceptions among unicellular pond ani- mals, the genetic code is universal. Gene therapists do not correct the code; the code isn't broken and can't be changed. When physicians correct a gene, the cells use the code to effect the change. Whether gene therapy is a med- ical fad or a revolution in the treat- ment of genetic diseases will ulti- mately be decided by the educated public. Is it "intelligent medicine" or eugenics in high-tech clothing? Gene therapy is so new there is very little material available to general audiences. Correcting the Code is an entertaining, informative introduc- tion to this important issue. 10E -Cold Spring Harbor Laboratory Board of Trustees Officers David L. Luke 111, Chairman Mary D. Lindsay, Vice-Chairman John R. Reese, Treasurer Wendy Vander Poel Hatch, Secretary Townsend J. Knight, Esq., Assistant Secretary James D. Watson, Ph.D., President Bruce Stillman, Ph.D., Director Winship Herr, Ph.D., Assistant Director G. Morgan Browne, Administrative Director Individual Trustees Charles F. Dolan Martha Farish Gerry Lita Annenberg Hazen David H. Koch Laurie J. Landeau, V.M.D. Edwin S. Marks William R. Miller )ohn J. Phelan William S. Robertson Thomas A. Saunders III Owen T. Smith, Esq. Douglas A. Warner III Henry Wendt Scientific Trustees Gunter Blobel, M.D., Ph.D. The Rockefeller University W. Maxwell Cowan, M.D., Ph.D. Howard Hughes Medical Institute Gerald R. Fink, Ph.D. Whitehead Institute for Bio. Research Thomas M. Jessell, Ph.D. Columbia University J. Anthony Movshon, Ph.D. New York University Joan A. Steitz, Ph.D. Yale University School of Medicine Shirley M.C. Tilghman, Ph.D. Princeton University Don C. Wiley, Ph.D. Harvard University Eckard Wimmer, Ph.D. SUNYat Stony Brook Honorary Trustees Bayard Clarkson, M.D. Robert L. Cummings H. Bentley Glass, Ph.D. Walter H. Page I larhor Tr.ucript • Sununer 1994 11

:........ . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . Hot and Cold (Spring Harbor) Jerry Jerome and His All-Stars brought their hot Dixieland jazz to Grace Auditorium on Jtdy 24. The benefit concert for the Cold Spring Harbor Laboratory Association's Annual Fund was called "Hot Jazz on Cold Spring Harbor." Event co-chairs Hope Reese and Jane Spingarn Annual Fund Associate Director Joan Pesek hands out picnic dinners to concert-goers. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . : Cold Spring Harbor Laboratory Cold Spring Harbor, New York 11724-2213 Address Correction Requested Non-profit Org. U.S. Postage PAID Permit -5 Cold Spring Harbor, NY :......................................... : Dr. Harmon C. i1cAilister Council for Tobacco Research USA, Inc. G00 3rd Ave. New York, NY 10022