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

by Ricki Lewis

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 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.

Genres: Science, Nonfiction, Biology, Health

336 pages, Kindle Edition

First published March 13, 2012

Reviews

[Charlotte Larsen · Rating 3 out of 5]

The Forever Fix by Ricki Lewis is a nonfiction, scientific novel, depicting not only the triumphs and success stories associated with gene therapy procedures, but the risks and tragedies as well. The entire book is centered around the story of young Corey Haas. Corey is just a toddler when his parents Nancy and Ethan begin to notice that something is not quite right. He stares directly into bright light bulbs without flinching, and always seems to walk into things without meaning to. Not only that, but he never truly looks his parents in the eyes the way that most children do. When Nancy and Ethan decide to finally take Corey to see the doctor, he is given a pair of glasses and at first diagnosed with albinism. It is not until years later that they finally uncover his true disease; Leber congenital amaurosis (LCA for short), and Corey participates in a life-changing clinical trial for gene therapy, that will alter the course of medicine forever. When hundreds of viruses are injected into Corey’s worst eye, he begins to see the world again in all its true colors. Even the untreated eye improves slightly. However, the novel takes the reader through the difficult course of gene therapy that led up to Corey’s story of success. Whether it be the death of Jesse Gelsinger, or the little babies who accidentally develop leukemia, it is not an easy road. All of the patients who undergo gene therapy throughout the book may not see improvement, or even survive. However, without a doubt Lewis reminds the reader that their courage has contributed to the future of medicine, and for that they will not be forgotten.

[Brian · Rating 2 out of 5]

For a few years now I've been curios to learn more about gene therapy-- not specifics related to treatment, which would mean little to me since I'm not a scientist. Rather, I'm interested in the concept. Diseases that originate in the function of the cell, like diabetes, cystic fibrosis, AIDS, leukemia, and certain neurological disorders, can be treated by "reprogramming" the function or operation of those cells. (This is contrasted to a drug therapy, which focuses on the symptoms of disease.) When discussing the concept of gene therapy, a computer analogy is useful; the patient's sick organ or system is like the malfunctioning program, and the "gene therapy" is like the little piece of precision-engineered debugging software. Genetic material is introduced into the tissue/organ/system and goes to work "hijacking" the defective system. In principle, this is the same way a virus operates, but in a therapeutic setting the proteins made by the introduced genetic material work to stop malfunction or expedite proper cellular function.

The title of this book is "Forever Fix," and it points to the way gene therapy goes to the source of the disorder and cures it "forever," as opposed to drug therapy which might only affect symptoms. The author's main focus is the treatment of a form of congenital blindness, but she talks about other attempts to use gene therapy to treat other disorders. There are some good sections outlining the nuts-and-bolts science of gene therapy, and I was also very interested to learn about the way clinical trials are designed, the way research is funded (or not funded), and the way history and the law protect patient privacy and "ownership" of biological material (or don't protect it).

The goodreads blurb says this book examines the subject through a "very personal prism." I could have done without all the discussion of the patients' family life, their pets, their favorite flavors of ice cream... etc.

[Judie Dooley · Rating 5 out of 5]

I won this book on goodreads. This book was awesome. I learned so much about gene therapy and the number of diseases it is helping to cure. The little boy Cory is so brave to have gone through so much to get get his sight back.
Also it told about all the other children that it helped , and unfortunately the ones that had bad outcomes. It was well written and I was able to understand all the procedures etc with no trouble. I think every eye surgeon should read this book. Thank you for allowing me the opportunity to read it. Judie

[Hani · Rating 1 out of 5]

DO NOT READ

This book was poorly written, scattered, and vastly unenjoyable. I was forced to swallow it down by my Biology teach who I will always resent because of it. The author wrote out genetics textbook and that becomes evident within the first few chapters of the book. The woman tries so hard to garner empathy from the reader by using cheap descriptors of random children such as “beautiful”. Every child is apparently beautiful. So disgustingly horrendous I hope to forget this trash in future years but I’m afraid it will haunt me for the rest of my life, that and the dangers of fungus.

[A. · Rating 5 out of 5]

Enjoyed reading this book about gene therapy and how it helps and can help so many children.

[Melissa T · Rating 4 out of 5]

*Please note I won this via Goodreads Giveaways.*

This book is really well written. Being that it deals with gene therapy, it has a lot of scientific background in it. I've taken biology a few times. Once in high school, once when getting my BA, and once when going back to school to get an associate's degree to be able to do a work exchange program. I took it so many times not because I didn't get it, but because it is easiest science to digest, IMO. So. I learned multiple times over about brain body connections, proteins, DNA, axons, neurons, etc. All of which helped me in reading this book.

Although, I will say, that this is written in such a way that even if you don't have much scientific background, it's still easy enough to understand.

I honestly thought this would be more of a third party memoir, about Corey, the boy who's treatment "saved" gene therapy. But it doesn't focus on him nearly as much as I thought it would. Instead it's more of a combination of short chapters on a variety of people and the disorders they live with, and some of them, ultimately die from.

As easy as this was to read, I do have to admit that I was less invested in Corey's story by the end. I skimmed over the last few pages of the book. He still had a way to go in his treatment from the sound of things, since they only did the gene therapy on one eye, so it would be interesting to find out where he is at in that process these days.

Even though this was less memoir and more scientific than I was hoping, it's still a great read.

[Jenny · Rating 4 out of 5]

I'd give it 3.5 stars, so I'll round up. This was gave some good information on gene therapy, its history, current applications, and everything that needs to happen during a clinical trial. However, many parts of the book were not super accessible to those who may not have a great background in genetics, as often Lewis would describe genetic parts or disorders in long paragraphs of technical jargon and then conclude with a single sentence analogy in layman's terms. There were also some long winded sections about authorship of scientific papers, which I imagine is important to scientists, but that didn't seem to be her audience for this book.

[Lily Anderson · Rating 3 out of 5]

Gene therapy is such a new concept, and even newer in execution. This book displayed very nicely how it has evolved, and gave a plethora of stories to represent each step of the process.

Even so, I found myself bored with some of the explanations. I know these are for those who aren’t well versed in the terminology of gene therapy, but sometimes it felt like the explanations were meant for kindergartners.

Regardless, this was a very interesting book, and I do recommend.

[Heidi Hisrich · Rating 5 out of 5]

Absolutely incredible book. Anyone interested in medicine and treatment of diseases (especially genetic diseases) or in how new treatments are researched and approved should definitely read this!

[Abbie · Rating 4 out of 5]

The beginning was a bit slow, but the ending was thrilling!

[Lime]

TPL challenge 2024: a book about an invention.
Since it was written more than a decade ago, it is now quite dated. Very choppy writing style.

[Natalia · Rating 3 out of 5]

This was a 3.5! Pretty well written for lay audience and cool to see some familiar names in this book. Def outdated at this point tho

[ally ♡]

let me just preface this by saying that if it weren't for school, i most likely would not have picked this up. nonfiction has always been a difficult genre to read— which feels shameful, in a way, to confess.

many believe that one can only claim themselves to be an experienced reader once they read certain "academic" novels. i personally believe that reading is an art, essentially meaning that there are no right or wrongs to going about it. if someone were to enjoy spending their time reading epic poetry on the weekends, the same respect should be given to someone who prefers reading young adult romance. after all, isn’t love cited to be the greatest experience of human life?

(however, i can admit the factuality of the genre brings up excellent, reliable talking points on a topic that otherwise would have remained unknown to me.)

the forever fix follows multiple different case studies of individuals with inherited genetic disorders seeking help through clinical trials. our frontrunner is young corey haas, who completely changed the genetic therapy world when his clinical experiment (consisting of millions of doctored viruses being injected into his eye) restored his eyesight.

now, gene therapy is such an interesting field. despite there being hundreds of examples of genetic therapy actually succeeding, the media and the general public tend to focus on the failures of the industry: inciting leukemia in newborns, the death of a mildly-ill teenager, etc. corey haas' story came as a surprise to all, and as a shining beacon of hope to all geneticists working to save their field every day. lewis' message is not to dismiss these tragedies, but to instead urge everyone to honor their losses by supporting those whom genetic therapy has successfully helped.

my biggest issue with this is that ricki lewis does not have a particularly compelling writing style. It is clear that she is primarily a geneticist before all else, but her sheer passion for the topic helps ease some of the choppiness of the text.

all in all, a nice introduction to the nonfiction genre. not sure if i'll be sticking around, but i will be checking out some of the gene therapy articles mentioned in this book!!!!

[Nigel Hey · Rating 5 out of 5]

If, like me, you grew up believing that scientists can't write, here's the proof of how wrong one can be. This is a science book so skilfully constructed that it reads like a novel - except that it is a true story about an undeniably important subject, the promise of gene therapy.

Here is an author whose writing skill is such that she can take on a difficult subject and blend it with real-life, factual information so deftly that you're likely to forget you're reading a book about cutting-edge science. Words like adrenoleukodystrophy will not disturb you. Instead, you will very quickly realize that this is a book about vulnerable young people, their families and their carers. Importantly, it is also about the people on research teams and hospital staffs who believe that with time gene therapy can improve and save the lives of more youngsters in the future.

It is not necessarily a pretty story. Tragedy is built into the stories of the youngsters who did not respond to certain types of gene therapy and did not survive. Success comes in the stories of people like Corey Haas, the central character of this story, who underwent gene therapy for hereditary blindness, and his family. Corey's sight gradually returned, a success story that gave hope to the millions who suffer from “glitches” that prevent genes from doing their job. Science knows enough about genes these days that Corey's problem was traced to a gene that normally would use vitamin A to send visual signals to his brain. The challenge was to undo the genetic problem so that Corey's descent into blindness would cease and his sight would return. Incredibly it worked.

Author Ricki Lewis, a geneticist by training, explains with engaging clarity how this revolution is coming about. If you want to know the technical details of how the work is being undertaken, those details are there. If you want an enjoyable, heart-warming, and optimistic story about a family in trouble and how science helped them, it's there too. You can't miss. And incidentally, you might as well get acquainted with gene therapy now, because it's almost surely going to be one of the Big Stories of this scientific decade.

[David · Rating 3 out of 5]

fairly readable account of the false starts and subsequent improvements with gene therapy, centering on story of a young, nearly blind, boy whose vision was improved dramatically, and to a lesser extent on other anecdotes.

The nuances of the preclinical animal research were hard for me to follow, and perhaps the sense that this may be true of many readers was the motivation for including so many human interest stories. Not really a seamless fit, though, in that the one has almost nothing to do with the other. When NBC Olympics coverage delves into athletes' background stories (here's Mo Farah drinking tea at the house in Oregon where he moved to train under Alberto Salazar, and here's his wife talking about the family sacrifices involved in relocation, and.......), it may or may not be to your taste as a viewer, but at least it relates directly to their accomplishments. The life story of the kid with a rare genetic disease could be completely the opposite of what it is, and it wouldn't change the scientific developments involved in (someone else's) fixing the problem in a chicken or pig one bit.

Two interesting side issues she covers well: (a) the phenomenal efforts some parents have undertaken to raise funds for gene therapy trials relevant to their kids' disorders, in order to speed up the otherwise sluggish process of getting NIH grants for early-phase studies etc. etc.; (b) difficulty of interesting big Pharma in funding the research, partly because some of the disorders are rare but also because if it works, it works ("forever fix"). Much more profit to be made in something like cholesterol-lowering drugs, targeting a common condition with a treatment that you have to keep taking indefinitely.

[Medeeha · Rating 4 out of 5]

Personal notes:
Leber congenital amaurosis type 2-LCA2
"Four days earlier, Corey had been legally blind, heading toward a world of total darkness." -Significance of repetition.
Upenn: Pages 18,41.
"A decade from now, once researchers..."[19]
Theodore Friedmann: Page 27: UCSD
Harvard: Page 28
Charles Krauthammer-psychiatrist
Human Gene Therapy Journal
I like how you learn the exact logic behind the condition.
It's like a mystery
"moments of sudden insight when a syndrome on the whole-body level made sense at the cell or molecular level"[42[
terribly disease involving self-mutilation called Lesch=Nyhan syndrome. Wilson investigated what was wrong with the enzyme HPRT.
Wow. Wilson's cool xD "To find different mutations in the HPRT gene, Wilson traveled."[43]
"One September when Edwin was visiting, Wilson finally figured out Edwin's exact mutation and how it destroyed the key part of the HPRT enzyme."[44]
"I've got to figure a way o every ounce of energy goes to directly helping people." "dual role of the scientist-physician"[44]
Question: WHY do the HPRT genes in the nondividing brain cells of the affected child result in a child who is self-mutilating?
"Immunity" Journal. Interest in reading journals containing studies of the effects of various gene therapies[in vivo, ex vivo...etc].
OTC-deficient mice.
"Insert Quote here.."mantra"...a small group of committed people..jung"
page 198
page 213 page 220
Page

[R. · Rating 4 out of 5]

I won this book through the Goodreads Firstreads Book Giveaway program and I am required to disclose that in any review as part of the terms and conditions.

Now that we have that out of the way. I can honestly say that I learned a great deal about Gene Therapy and the ways that it is currently being used as well as the potential uses for the future. Well written and researched as well as being an easy read, even if it was a little slower pace than what I am used to being a predominately science fiction/fantasy reader.

I am sold on Gene Therapy being a worthwhile pursuit in medical research although truth be told I was already a fan of the concept before reading this book. So, while it didn't sway my opinion any it did reinforce it and make me more informed as to the potential and pitfalls of Gene Therapy in general.

[E.E. Giorgi · Rating 5 out of 5]

As a disclaimer I should say that genetics is part of what I do at work, even though I don't work on gene therapy in particular. But aside from the topic, which I knew I would enjoy, I loved Dr. Lewis's writing and the way she effortlessly folded all the science into the narrative. I am grateful to Dr. Lewis because through this wonderful book she humanized scientific research, giving a voice to the struggle of not just one, but many families, and she gave a voice and face to the doctors and the researchers who face many failures for every small step forward. Science is not dry and impersonal. Science is made of people, not just the scientists, but also the people who ultimately benefit from it, and the ones who, due to unfortunate events, didn't benefit but were still part of what made the successes possible. A wonderful read and a truly touching story.

[Laura · Rating 4 out of 5]

This book nicely delineates the course and purposes of clinical trials, especially those for gene therapies. It relates the highs and lows of the last 20 years in genetic research. I learned about some rare genetic conditions that were new to me as well as some that were more familiar. As a technical writer myself, I would give the author props for clear (if not concise) writing, though occasionally the narrative meandered and it could have been better formatted. But don't let my nitpicking stop you from reading this! My husband brought it home fir me as a wild-card pick from the library and it's very good. Accurate too, from my limited knowledge, and well-sourced.

[Emily Dentler · Rating 5 out of 5]

Absolutely loved this book! As a biomedical engineer, I really appreciated the level of detail included in this story, yet it was not so technical that a lay person would not be able to understand. A great overview of the history of gene therapy, interwoven with personal details from people who have gone through the process.

[Riann · Rating 5 out of 5]

Fascinating! I learned so much about gene therapy! Its beginnings, successes, failures and applications in the future! Written by a geneticist who is able to make all the technical jargon understandable to the layperson. She also shares stories of families of people with different types of genetic diseases both rare and more common. I highly recommend this book!

[Elizabeth · Rating 5 out of 5]

I really enjoyed reading this book. Not only did I learn a lot about gene therapy but this book actually caught my attention and I finished it in one day. It took me a while to actually start reading it because I was always so busy but now I wish I could of read it a long time ago!

[Jason · Rating 3 out of 5]

A very interesting topic about trying to help people with genetic diseases but I just couldn't get into the style of writing. "So and so tears up when she remembers the day that..." or variations of that were written so often, it got kind of tiresome.

[Jim Walker · Rating 4 out of 5]

Wow. The stuff that goes on around us. The drama of lives devastated by rare, often unknown, genetic disorders. And the people who are driven to fix lives. The science, politics, economics, and dedication of people in an arena we rarely hear of. I found this book to be one of heroes.

[Jeanne · Rating 3 out of 5]

Good read about clinical trials and a wonderful story about a boy that was given his sight back by and investigator that I actually know! A bit heavy in the science at times but very interesting. A bit scary to know that all the diseases that are covered are out there and affecting so many.

[Austin Larson · Rating 4 out of 5]

The history of gene therapy in the United States told via the stories of the individual patients in the trials. Manages to stay non-technical with compelling anecdotes about patients who died in gene therapy trials and those who were cured.

[Delaney Slattery · Rating 2 out of 5]

I had to read this book for my biology class and I was dreading it at first. The farther I read the more intrigued I became. The story became more like a story and less like a textbook. Great book for any biology/genetics lover!

[Tamar Kieval · Rating 4 out of 5]

Excellent book explaining a difficult topic through compelling true stories of patients with genetic diseases and the eventual use of gene therapy to treat them despite a very tragic start which nearly derailed its use.

[Elizabeth]

As seen in Nature .

[Sheryl Duane · Rating 4 out of 5]

This book choked me up quite a bit. It was a very interesting read !