The Forever Fix: Gene Therapy and the Boy Who Saved It [NOOK Book]


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...

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The Forever Fix: Gene Therapy and the Boy Who Saved It

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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.

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Editorial Reviews

Publishers Weekly
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. (Feb.)
From the Publisher

"Lewis adeptly traverses the highs and lows of gene therapy and explores its past, present, and future through the tales of those who've tested its validity." -The Scientist
Times Union

An engaging and accessible look at gene therapy.
Molly Caldwell Crosby

"Ricki Lewis gives us the inspiring story of gene therapy as told through Corey's eyes--literally. Her book delves into the challenges modern medicine faces--both in its bitter disappointments and great successes--but it goes much deeper than that. With empathy and grace, Lewis shows us the unimaginable strength of parents with sick children and the untiring devotion of the physicians who work to find ‘the forever fix' to save them. But best of all Lewis gives us a story of profound hope."
Kevin Davies

"Ricki Lewis has written a remarkable book that vividly captures the breathtaking highs and devastating lows of gene therapy over the past decade while giving ample voice to all sides -- the brave patient volunteers, their parents and physicians. The Forever Fix is required reading as we dare to dream of curing a host of genetic diseases."

"Medical writer Ricki Lewis interweaves science, the history of medical trial and error, and human stories … from the death in 1999 of teenager Jesse Gelsinger, from a reaction to gene therapy intended to combat his liver disease, to radical successes in some children with adenosine deaminase deficiency."
Library Journal
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.
Kirkus Reviews
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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Product Details

  • ISBN-13: 9781429941471
  • Publisher: St. Martin's Press
  • Publication date: 3/13/2012
  • Sold by: Macmillan
  • Format: eBook
  • Pages: 336
  • Sales rank: 271,801
  • File size: 2 MB

Meet the Author

RICKI LEWIS is a Ph.D. geneticist, journalist, professor and genetic counselor. The author of one of the most widely used college textbooks in the field (Human Genetics: Concepts and Applications, now in its 10th edition), she has also written hundreds of popular pieces on science and other topics for trade and specialized magazines, including Nature, Discover, and The Scientist.

RICKI LEWIS is a Ph.D. geneticist, journalist, professor and genetic counselor. The author of one of the most widely used college textbooks in the field (Human Genetics: Concepts and Applications, now in its 10th edition), she has also written hundreds of popular pieces on science and other topics for trade and specialized magazines, including Nature, Discover, and The Scientist.
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Read an Excerpt



When I met nine-year-old Corey Haas, it was hard to believe that just over a year earlier, he had been well on his way to certain blindness. As a geneticist, I’d followed the incredible story of his gene therapy, and as a mom, I was curious about the young man who had made medical history.
On a dazzling Saturday morning in early December 2009, I drove forty-five minutes north to meet Nancy, Ethan, and Corey Haas, who live in the Adirondack Park. This spectacular expanse of nature is not a park in the conventional sense, but a vast swath of New York State peppered with small, older houses and log cabins nestled among towering pines, with the occasional noticeably newer vacation home of a downstater. The meandering Hudson River, only a few hundred feet across this far north, snakes through the Haas family’s small town of Hadley, where Corey’s parents grew up. The river is only a block away from their Cape Cod home, flooding their basement in bad snow years. I’d been in the area before, so I found it easily, but some of the reporters coming to interview the family over the past month had grown uneasy when the towering mountains blocked even the most powerful cell phone and GPS signals. But they made it—Ethan gives good directions.
Although it took only days for the researchers to realize that Corey’s gene therapy had worked, they had waited a year to publish the results, to be sure the effect lasted and hadn’t caused any problems. They had learned from past gene therapy fiascoes, and also knew Corey’s newfound sight would be big news. And they were right, with Good Morning America setting the slightly hyped tone by describing “revolutionary surgery that could give the gift of sight to the blind.” When the research paper appeared in The Lancet in November, the Haas family traveled to New York City for the very first time, where they made the rounds of the major TV news programs. Despite the intense interest, though, only a few journalists ventured north to meet Corey on his own turf.
The morning of my visit, the scent of the season’s first snowfall was in the air. In the Adirondacks, you don’t need a weatherman to know which way the wind blows, to quote Bob Dylan. You just have to watch the squirrels. I had to hopscotch around them as I walked up to the Haases’ front door, my hands laden with my laptop, books, and a large box. The squirrels scurried everywhere, grabbing and stashing acorns in the heart-shaped crevices at the bases of the tree trunks, occasionally dashing the wrong way and bashing into the large picture window in the living room that reached almost to ground level. The squirrels knew snow was coming.
When Corey opened the door, our eyes went first to the scampering squirrels, then to each other. He gave me a huge grin, his blue eyes dancing behind thick glasses. He took my leg and guided me to a coffee table, where I dropped my armload. As Ethan and Nancy introduced themselves, Corey began rummaging around the rocks and fossils in the box, a subset of the collection I’d begun at his age. Knowing he’d only recently been legally blind, I knelt down and tried to show him how to feel the indentations of the trilobites and brachiopods. But he proudly refused all assistance, peering closely at each fossil, as I had at his age, slowly turning each one to see what different angles might reveal.
I looked around. The décor was a welcoming blue and white, the rooms unusually tidy for the home of a small boy. His toys were neatly aligned on low shelves and stacked in corners, without the clutter that continually grows in my own home. But perhaps this was a habit from the time when Corey had to memorize where every single item in a room was in order to move about. Above the couch where I sat and typed as the family talked, a shelf held a dozen of Nancy’s 110-plus dolls. These were the special ones, the others inhabiting closets and a storage room, packed in with Nancy’s scrapbook collection. The other walls held what can only be described as a gallery of Coreys. My favorite was a photo of Corey at about one year old, with a broad smile and thick glasses, “Corey” stitched in needlepoint below, framed in red. The earliest photo on the walls, taken when Nancy and Ethan had yet to learn that Corey had inherited an eye disease, showed baby Corey stuffed into railroad overalls, a yellow shirt, and white socks, looking off to the side as if he’d rather be anywhere else. A wall in the dining room had the words live, laugh, and love spelled out in wood, with three photos beneath each—all of the resident celebrity, Corey.
As Ethan talked and sorted copies of medical reports to give me, Corey jumped up every few minutes to show his mother a rock. I glanced at them, trying, but failing, to imagine this energizer bunny of a boy stumbling around the same living room, crashing into the furniture like the squirrels outside careening from tree to tree. But that day he rocketed around the cozy living room with ease. He showed me his handheld Nintendo and how to extract a fart noise from a container of Silly Putty–like goop, and then he asked me to name the minerals in his rocks. Periodically he’d demonstrate the hardest part of the gene therapy for him—lying ramrod still on his back. I noted subtle signs of what the family had been through. Once in a while Corey would lose his footing briefly, and Nancy’s arm would instantaneously zap out to steady him, sometimes hugging him close. He didn’t squirm away. Corey’s independence was still new to all three of them. For years he could see only shadows, and in dim light, nothing at all.
On September 20, 2008, two days before Corey’s eighth birthday, and fifteen months before I met him, the Haas family had traveled to Children’s Hospital of Philadelphia—CHOP. That week, Corey underwent a battery of what had become familiar tests, plus a few new ones, and on Thursday morning was finally prepped for the procedure. Then, as Corey lay anesthetized, the eye surgeon Al Maguire snipped open a tiny flap in the left eye, the worse one, and carefully injected 48 billion doctored viruses into a tiny space just above a thin layer of colored cells that resembled patterned bathroom tile. This space and the layer of cells hug the rods and cones at the back of the eye. Corey’s colored cells weren’t doing their job of nourishing the precious rods and cones, which are called photoreceptors because they capture incoming light. His rods and cones had been slowly starving for years, gradually becoming too weak to send light signals to his brain to paint an image. As a result, before Corey lay down on the operating table, he saw only blurry, dark shapes. But that would change—sooner than anyone had even dared imagine.

Copyright © 2012 by Ricki Lewis

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Table of Contents

Preface ix

Part I The Best That Can Happen

1 Meeting Corey 3

2 The Road to a Diagnosis 7

3 What's Wrong with Corey? 13

Part II The Worst That Can Happen

4 The Breakthrough Myth 27

5 Jesse and Jim 35

6 Tragedy 52

Part III Evolution of an Idea

7 The SCID Kids 71

8 Setbacks 94

9 Lorenzo and Oliver 108

Part IV Before Gene Therapy

10 Hannah 135

11 Lori 148

Part V After Gene Therapy

12 Amazing Women 177

13 The Jewish Genetic Diseases 185

14 The Patent Predicament 196

15 Chasing Moonbeams 204

Part VI Corey's Story

16 Kristina's Dogs 225

17 Lancelot 237

18 Success! 253

19 Back to Chop 268

20 The Future 290

Epilogue 305

Notes 307

Acknowledgments 319

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Reading Group Guide

1. How does gene therapy differ from other types of treatments?

2. Why did it take Corey's parents, and the other parents in the book, months to realize that their children had medical problems?

3. What can a parent do when she knows that something is wrong with her child, but health care professionals do not take her seriously?

4. Imagine your young child is diagnosed with a rare inherited disease. List ways to learn about the disease, find clinical trials, and raise funds.

5. What factors would you consider in deciding whether your very young child should have an experimental treatment?

6. Informed consent guidelines from the Food and Drug Administration (FDA) state that age seven is old enough for a child to give consent to undergo an experimental procedure. Do you think that this age is an appropriate cut-off? Why or why not?

7. Paul Gelsinger, whose eighteen-year-old son Jesse died in a gene therapy experiment (Chapter 4-6), sent the author this e-mail after reading The Forever Fix:

I am glad that gene therapy is making headway. I have always felt (maybe even known) that it was a viable technology. I hope that it pans out as you hope, but please, oh please, don't make the same mistake I did and believe everything you hear or see. I am thankful that there is a great deal more honesty and oversight in the field now as a result of what happened to Jesse. . . .I guess that's his real legacy. Feel free to tell anyone that I am glad there have been some breakthroughs and that I hope many more follow.

Would you be able to be as forgiving as Paul Gelsinger? What did he mean by the mistake of believing everything? That is, which particular facts were misconstrued or miscommunicated in Jesse's sad case?

8. Lori Sames, Hannah's mom, is reaching out to all the families she can find whose children have giant axonal neuropathy (GAN). Participation of families is important in research to develop tests and treatments, but some families prefer to keep to themselves. To what extent should an activist parent like Lori try to convince hesitant families to join the group seeking a treatment?

9. How can social media help families with rare diseases?

10. What complementary therapies might ease the daily lives of children with certain inherited conditions? How can complementary therapies help or hinder the progress of FDA-sanctioned clinical trials?

11. Gene therapy for HIV/AIDS is safe and appears to be working, after nearly a dozen years in three clinical trials. Yet drugs (anti-retrovirals) have been very successful in controlling the infection. What are the pros and cons of a "forever fix" for HIV/AIDS? How does the situation for HIV/AIDS differ from that of a rare disease such as giant axonal neuropathy?

12. What criteria should researchers use to select diseases to approach with gene therapy? Knowledge of mechanism? Prevalence? Whether a famous person has it? How involved the parents are? Possibility of translation to more common conditions?

13. Why were the first gene therapies done on very rare diseases?

14. Did reading the book alter your view of the use of animals in biomedical research?

15. Why won't the gene therapies described in the book be passed to the children of the treated patients?

16. Which do you think is more dangerous to use in a gene therapy trial to deliver the genes, adenovirus or HIV? Why?

17. Under what circumstances would you have or allow your child to have gene therapy?

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