The Forever Fix

Starred review from December 19, 2011
In this impressive, meticulously researched study of the exciting new developments in gene therapy, geneticist and journalist Lewis (Human Genetics) looks closely at the history of setbacks plaguing the treatment of rare genetic diseases as well as recent breakthroughs. The success in reversing eight-year-old Corey Haas’s blindness from the rare inherited disorder Leber congenital amaurosis by gene therapy in 2008 has proved the “happy story” that the field needed after the well-publicized death in 1999 of Jesse Gelsinger after gene therapy to treat his inherited metabolic deficiency. Case by case Lewis chronicles the small steps in advancing treatment of rare disorders of the very young, such as severe combined immune deficiency, adrenoleukodystrophy, giant axonal neuropathy, and Canavan and Tay-Sachs diseases afflicting the Ashkenazi Jewish community—all of which have undergone years of trial and error and benefited from the advocacy of parents. Lewis traces the evolution of the idea of gene therapy by William French Anderson from the 1950s, leading to approaches in recombinant DNA technology in the 1970s with animal testing, and culminating in the first sequencing of the human genome in 2000. Issues of patent-control of certain genes inhibit testing while large pharmaceutical companies continue to drag their heels in funding research. Yet with each success, as Lewis recounts in this rigorous, energetic work, possibilities in treating HIV infection and dozens of other diseases might be around the next corner. Agent: Ellen Geiger.

January 15, 2012
Science journalist Lewis (Human Genetics: The Basics, 2010, etc.) examines the emergence of a new medical frontier. The author opens in 2008, when 8-year-old Corey Haas's attention was drawn to a hot air balloon and he cried out, "It hurts!" Just four days before he had been legally blind due to a mutation that prevented his eyes from processing visual signals; now for the first time in his life, the boy was experiencing the brilliance of full sunlight. Lewis takes readers through the tortuous process that turned young Corey into a poster boy for gene therapy nine years after the tragic death of 18-year-old Jesse Gelsinger from a similar operation. Gelsinger's operation was intended to correct a mutated gene that affected his liver function. In both cases, the mutated gene was identified, and the boys were deliberately "infected" with genetically engineered viruses "sent into the body as microscopic ferries" bearing healthy replacement genes. Gelsinger, however, had suffered from an "unusual and deadly immune-system response" to the virus. Both boys took part in experimental protocols with the informed consent of their guardians, who accepted the risk involved. Lewis chronicles the painstaking search for the specific genes responsible for genetic diseases, and she recounts the human stories behind each discovery, triumph and failure. A sidelight on this absorbing story is the development of rare genetic disorders in populations such as Jewish survivors of pogroms, whose gene pool suffered "the genetic consequences of serial strangulation." The author also looks at the struggle to make genetic testing freely available to at-risk couples contemplating marriage. A fascinating account of groundbreaking science and the people who make it possible.

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September 15, 2011

Geneticist and science journalist Lewis here explains how gene therapy works and why it's "forever"--by effecting a cure at the genetic root of a problem, it obviates the need for further treatment or surgery. Lewis illustrates by relating the story of eight-year-old Corey Haas, nearly blind from a hereditary disorder, who in a historic procedure had viruses bearing healing genes injected into the DNA of his eyes. A few days later he could see again. Exciting for the medically up-to-date.

Copyright 2011 Library Journal, LLC Used with permission.

February 1, 2012
Thanks to sound-bite-addicted popular media outlets, the phrase gene therapy possesses every bit as much charisma as the Kardashian familyand about as much misinformation. But now, thanks to geneticist Lewis, the mystery and complexity of gene therapy is unraveled in this articulate, enlightening account. She explains that while it is, or can be, a remarkable intervention in the cure or even elimination of a variety of both congenital and environmentally acquired illnesses, the path from inception to that goal is littered with errors, even tragedies. Her technique of putting human faces on those who participate in clinical trials makes the therapy's prospects at once heartening and seemingly beyond hope. The eponymous boy who saved gene therapy notwithstanding, financing the research still remains a major obstacle. It may come as no surprise that Big Pharma is more interested in treating diseases than curing them. So funding sources are few and far between, coming largely from not-for-profit foundations. Lewis' cogent, even compelling book has the potential to inspire both hope and cash.(Reprinted with permission of Booklist, copyright 2012, American Library Association.)