Fascinating narrative science that explores the next frontier in medicine and genetics through the very personal prism of the children and families gene therapy has touched.
Eight-year-old Corey Haas was nearly blind from a hereditary disorder when his sight was restored through a delicate procedure that made medical history. Like something from a science fiction novel, doctors carefully injected viruses bearing healing genes into the DNA of Corey's eyes—a few days later, Corey could see, his sight restored by gene therapy.
THE FOREVER FIX is the first book to tell the fascinating story of gene therapy: how it works, the science behind it, how patients (mostly children) have been helped and harmed, and how scientists learned from each trial to get one step closer to its immense promise, the promise of a "forever fix," - a cure that, by fixing problems at their genetic root, does not need further surgery or medication.
Told through the voices of the children and families who have been the inspiration, experimental subjects, and successes of genetic science, THE FOREVER FIX is compelling and engaging narrative science that tells explores the future of medicine as well as the families and scientists who are breaking new ground every day.
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.
Great science, great personal stories
Ricki Lewis does a terrific job of telling the complex story of gene therapy and how it is becoming a reality for many people. Few people, even in the scientific community, are aware of how successful it has been in recent trials. The story of Corey Haas is tremendously inspirational and encouraging. In addition to Corey's story, though, I was impressed with the amazing things that several parents of very sick children have been able to accomplish in pushing the technology forward. Ricki gives them the credit they deserve, and makes the point that a few determined people really can make a difference. She also allows the scientists to seem like real people, especially the several women who have been key players in the field, which I really appreciate. The science is complicated and at times may be difficult for the layperson, but I think the book does a good job of making it as comprehensible as possible, for example using many analogies to explain the functions of different proteins and not dwelling too much on the biochemistry. The stories of the people who have been helped (and harmed) over the years are powerful enough to carry the reader through.