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Breath from Salt: A Deadly Genetic Disease, a New Era in Science, and the Patients and Families Who Changed Medicine Forever
Cystic fibrosis was once a mysterious disease that killed infants and children. Now it could be the key to healing millions with genetic diseases of every type—from Alzheimer's and Parkinson's to diabetes and sickle cell anemia.
In 1974, Joey O'Donnell was born with strange symptoms. His insatiable appetite, incessant vomiting, and a relentless cough—which shook his tiny, fragile body and made it difficult to draw breath—confounded doctors and caused his parents agonizing, sleepless nights. After six sickly months, his salty skin provided the critical clue: he was one of thousands of Americans with cystic fibrosis, an inherited lung disorder that would most likely kill him before his first birthday.
The gene and mutation responsible for CF were found in 1989—discoveries that promised to lead to a cure for kids like Joey. But treatments unexpectedly failed and CF was deemed incurable. It was only after the Cystic Fibrosis Foundation, a grassroots organization founded by parents, formed an unprecedented partnership with a fledgling biotech company that transformative leaps in drug development were harnessed to produce groundbreaking new treatments: pills that could fix the crippled protein at the root of this deadly disease.
From science writer Bijal P. Trivedi, Breath from Salt chronicles the riveting saga of cystic fibrosis, from its ancient origins to its identification in the dank autopsy room of a hospital basement, and from the CF gene's celebrated status as one of the first human disease genes ever discovered to the groundbreaking targeted genetic therapies that now promise to cure it.
Told from the perspectives of the patients, families, physicians, scientists, and philanthropists fighting on the front lines, Breath from Salt is a remarkable story of unlikely scientific and medical firsts, of setbacks and successes, and of people who refused to give up hope—and a fascinating peek into the future of genetics and medicine.
In 1974, Joey O'Donnell was born with strange symptoms. His insatiable appetite, incessant vomiting, and a relentless cough—which shook his tiny, fragile body and made it difficult to draw breath—confounded doctors and caused his parents agonizing, sleepless nights. After six sickly months, his salty skin provided the critical clue: he was one of thousands of Americans with cystic fibrosis, an inherited lung disorder that would most likely kill him before his first birthday.
The gene and mutation responsible for CF were found in 1989—discoveries that promised to lead to a cure for kids like Joey. But treatments unexpectedly failed and CF was deemed incurable. It was only after the Cystic Fibrosis Foundation, a grassroots organization founded by parents, formed an unprecedented partnership with a fledgling biotech company that transformative leaps in drug development were harnessed to produce groundbreaking new treatments: pills that could fix the crippled protein at the root of this deadly disease.
From science writer Bijal P. Trivedi, Breath from Salt chronicles the riveting saga of cystic fibrosis, from its ancient origins to its identification in the dank autopsy room of a hospital basement, and from the CF gene's celebrated status as one of the first human disease genes ever discovered to the groundbreaking targeted genetic therapies that now promise to cure it.
Told from the perspectives of the patients, families, physicians, scientists, and philanthropists fighting on the front lines, Breath from Salt is a remarkable story of unlikely scientific and medical firsts, of setbacks and successes, and of people who refused to give up hope—and a fascinating peek into the future of genetics and medicine.
615 pages, Hardcover
First published September 8, 2020
367 people are currently reading
8,710 people want to read
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September 7, 2020
As a PhD researcher working on Cystic Fibrosis I immediately knew I had to get this ARC and read Breath from Salt, which will be published tomorrow on September 8, the 31st anniversary of the publication of the CFTR gene, mutations in which cause Cystic Fibrosis. I wanted to read it to get more background and personal stories, and I am also looking for accessible ways in which to introduce my thesis subject to my friends and family.
Breath from Salt aims to tell the story of Cystic Fibrosis from the early days before it was recognized, over the helpless days where the diagnosis was a (very) early death sentence, to the currently hopeful days where CF specific drugs that can restore the faulty protein have started to reach the market. This is the story of nothing short of a revolution - where Cystic Fibrosis helped to make sure that rare diseases (CF itself is considered the most common of the rare diseases) get the research and resources that are needed, and then again at the forefront of the treatment revolution that is called personalized medicine.
However, I suggest Breath from Salt should be re-titled An American History of Cystic Fibrosis.
The beginning was great! I was rather excited to be reading this, so I probably bothered a lot of people with my constant status updates. While I knew the main parts of story here, they are usually only summarized in review papers like "Dr. Dorothy Andersen was the first to describe CF in 1938...". Breath from Salt however was adding the personal touch and anecdotes to these early discoveries that was exactly what I was hoping to find. Interspersed was the touching story of a family struggling with CF in a time there were so little options. Sad as it was to read about it, it also highlighted how far we have come from those days.
Next up, there was a lot of basic science to discover. After being able to recognize and diagnose Cystic Fibrosis, the next question was: what is going wrong in Cystic Fibrosis? This is essential to know in order to figure out how to fight the disease. Its clustering in families made clear there had to be a genetic cause, the salty sweat (people with CF taste salty) pointed towards a defect in salt transport. The unraveling of how (which part of the DNA was mutated) and what (which functions where the cells missing because of it) was super interesting to read for me. All of this is science that was performed before I was born, let alone read about CF, and it was great to see it all together in one place.
The gene hunt culminates with the discovery of the CFTR gene which was published on September 8, 1989 in three papers in Science. From there on the focus is on remedies for the disease. And after a very short stint into gene therapy the focus becomes the collaboration of the Cystic Fibrosis Foundation and Aurora (later: Vertex) in the search for so-called CFTR modulators, which would restore the faulty CFTR protein. The story of the CFF was interwoven throughout the earlier parts as well, showing how it became a large and successful patient organization which had the means to hire a company to start working on Cystic Fibrosis (and not for example cancer or diabetes or any other much more common diseases).
This was the part I knew best already - since it was all more recent. It was interesting to read how these molecules which I work with on a daily base (VX-770; VX-809/661; VX-445) came to be. However, I was a little bit disappointed because it no longer focused on other scientific discoveries besides the Vertex development of CFTR modulators. One of the amazing things in the first parts was how it showed that science is a group effort. Discoveries are made because groups at different labs work together and build onto the results from one another to find the gene, discover the protein, and what the mutations do to disrupt it. There was very little non-Vertex related science in the latter part of the book. There is a very short mention that CFF funds some other pharmaceutical companies as well, but that is about it. I suggest this website if you are interested in all ongoing clinical trials into new treatments: https://www.cff.org/Trials/pipeline
Which brings me to my suggested title. There is little to no mention of Europe. Cystic Fibrosis is at least as common in Europe as it is in Northern America, and based on reading this book one might suggest there is no research at all across the pond. As a European-based CF researcher I found this a little unfair. Especially near the end when rare mutations (CF can be caused by different mutations, some of them are only found in a handful of people with CF, and there are no medicines yet for these mutations) are discussed I feel the European effort should have been acknowledged.
Because it does mention how CFF are engineering rare mutations in their models, and Vertex' All in For CF Program, but it doesn't talk for example about HIT-CF (https://www.hitcf.org/) the large ongoing European project with teams from the Netherlands, Belgium and Portugal where new drugs from multiple companies are tested directly on cells derived from people with these rare mutations. It doesn't discuss the human intestinal organoid model (stem cells taken from a small, minimally invasive biopsy and then grown in a petridish where they form some kind of a mini-organ), which correlates very well with clinical data from people with CF. And since it is a minimally invasive procedure these are available from many, many patients and really allow to perform personalized medicine. (You can find the original paper describing the assay over here: https://www.nature.com/articles/nm.3201) 500+ pages and the only reference to organoids at all (it was lung organoids in this case) was to say that some researcher in the Netherlands (not mentioned by name) received some funding to study a certain type of mutation.
Since a picture says more than a thousand words and this assay is something I also perform, I will include a little movie from my own results demonstrating the assay over on my blog https://www.urlphantomhive.com
These things obviously only bothered me because I already knew a lot about the subject (and I am from Europe). As an introduction I think it would work really well, not just into Cystic Fibrosis but also into the ideas and challenges of personalized medicine. Or for anyone who likes to read science books. For me, the recap of all the 'old' science was great, and I wouldn't have minded if the focus had remained more on the science part rather than the business part after the discovery of the gene. I found it rather readable and accessible, but I can't really vouch for people unfamiliar with many of the scientific terms used.
I would certainly recommend Breath of Salt - just keep in mind that while it covers a lot already, there is still a lot more going on.
Thanks to the publisher and Netgalley for providing me with a free copy of this book in exchange for an honest review!
Find this and other reviews on https://www.urlphantomhive.com
Breath from Salt aims to tell the story of Cystic Fibrosis from the early days before it was recognized, over the helpless days where the diagnosis was a (very) early death sentence, to the currently hopeful days where CF specific drugs that can restore the faulty protein have started to reach the market. This is the story of nothing short of a revolution - where Cystic Fibrosis helped to make sure that rare diseases (CF itself is considered the most common of the rare diseases) get the research and resources that are needed, and then again at the forefront of the treatment revolution that is called personalized medicine.
However, I suggest Breath from Salt should be re-titled An American History of Cystic Fibrosis.
The beginning was great! I was rather excited to be reading this, so I probably bothered a lot of people with my constant status updates. While I knew the main parts of story here, they are usually only summarized in review papers like "Dr. Dorothy Andersen was the first to describe CF in 1938...". Breath from Salt however was adding the personal touch and anecdotes to these early discoveries that was exactly what I was hoping to find. Interspersed was the touching story of a family struggling with CF in a time there were so little options. Sad as it was to read about it, it also highlighted how far we have come from those days.
Next up, there was a lot of basic science to discover. After being able to recognize and diagnose Cystic Fibrosis, the next question was: what is going wrong in Cystic Fibrosis? This is essential to know in order to figure out how to fight the disease. Its clustering in families made clear there had to be a genetic cause, the salty sweat (people with CF taste salty) pointed towards a defect in salt transport. The unraveling of how (which part of the DNA was mutated) and what (which functions where the cells missing because of it) was super interesting to read for me. All of this is science that was performed before I was born, let alone read about CF, and it was great to see it all together in one place.
The gene hunt culminates with the discovery of the CFTR gene which was published on September 8, 1989 in three papers in Science. From there on the focus is on remedies for the disease. And after a very short stint into gene therapy the focus becomes the collaboration of the Cystic Fibrosis Foundation and Aurora (later: Vertex) in the search for so-called CFTR modulators, which would restore the faulty CFTR protein. The story of the CFF was interwoven throughout the earlier parts as well, showing how it became a large and successful patient organization which had the means to hire a company to start working on Cystic Fibrosis (and not for example cancer or diabetes or any other much more common diseases).
This was the part I knew best already - since it was all more recent. It was interesting to read how these molecules which I work with on a daily base (VX-770; VX-809/661; VX-445) came to be. However, I was a little bit disappointed because it no longer focused on other scientific discoveries besides the Vertex development of CFTR modulators. One of the amazing things in the first parts was how it showed that science is a group effort. Discoveries are made because groups at different labs work together and build onto the results from one another to find the gene, discover the protein, and what the mutations do to disrupt it. There was very little non-Vertex related science in the latter part of the book. There is a very short mention that CFF funds some other pharmaceutical companies as well, but that is about it. I suggest this website if you are interested in all ongoing clinical trials into new treatments: https://www.cff.org/Trials/pipeline
Which brings me to my suggested title. There is little to no mention of Europe. Cystic Fibrosis is at least as common in Europe as it is in Northern America, and based on reading this book one might suggest there is no research at all across the pond. As a European-based CF researcher I found this a little unfair. Especially near the end when rare mutations (CF can be caused by different mutations, some of them are only found in a handful of people with CF, and there are no medicines yet for these mutations) are discussed I feel the European effort should have been acknowledged.
Because it does mention how CFF are engineering rare mutations in their models, and Vertex' All in For CF Program, but it doesn't talk for example about HIT-CF (https://www.hitcf.org/) the large ongoing European project with teams from the Netherlands, Belgium and Portugal where new drugs from multiple companies are tested directly on cells derived from people with these rare mutations. It doesn't discuss the human intestinal organoid model (stem cells taken from a small, minimally invasive biopsy and then grown in a petridish where they form some kind of a mini-organ), which correlates very well with clinical data from people with CF. And since it is a minimally invasive procedure these are available from many, many patients and really allow to perform personalized medicine. (You can find the original paper describing the assay over here: https://www.nature.com/articles/nm.3201) 500+ pages and the only reference to organoids at all (it was lung organoids in this case) was to say that some researcher in the Netherlands (not mentioned by name) received some funding to study a certain type of mutation.
Since a picture says more than a thousand words and this assay is something I also perform, I will include a little movie from my own results demonstrating the assay over on my blog https://www.urlphantomhive.com
These things obviously only bothered me because I already knew a lot about the subject (and I am from Europe). As an introduction I think it would work really well, not just into Cystic Fibrosis but also into the ideas and challenges of personalized medicine. Or for anyone who likes to read science books. For me, the recap of all the 'old' science was great, and I wouldn't have minded if the focus had remained more on the science part rather than the business part after the discovery of the gene. I found it rather readable and accessible, but I can't really vouch for people unfamiliar with many of the scientific terms used.
I would certainly recommend Breath of Salt - just keep in mind that while it covers a lot already, there is still a lot more going on.
Thanks to the publisher and Netgalley for providing me with a free copy of this book in exchange for an honest review!
Find this and other reviews on https://www.urlphantomhive.com
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July 13, 2020
August 26, 2020
This was initially an impulse read based on the cover; however, as someone who has an interest in knowing more about diseases, I was fascinated. The author manages to balance the voice between the discussing Cystic fibrosis as well as other diseases including the future of medicine. It was a bit of an intense topic; however, and could be broken a bit better.
I received an ecopy of this through Netgalley; however, all opinions are my own.
I received an ecopy of this through Netgalley; however, all opinions are my own.
December 20, 2020
This is a very interesting, but overly long book. I'm glad I stuck it out until the end, although sometimes it was a slog.
The book tells the story of the long effort to develop drugs that would ameliorate the suffering and extend the lives of those born with cystic fibrosis, which, we learn, is a genetic disease.
The book is overly long because it discusses three sub-topics in depth:
o the lives of several individuals born with the disease -- the most interesting aspect of the book
o the science behind the drug development -- interesting, but told in somewhat unnecessary detail, and a reader who doesn't have much of a science background would probably find this to be tough going
o the fund-raising efforts of the Cystic Fibrosis Foundation, which frankly was of almost no interest to me.
So worth reading, with these caveats
The book tells the story of the long effort to develop drugs that would ameliorate the suffering and extend the lives of those born with cystic fibrosis, which, we learn, is a genetic disease.
The book is overly long because it discusses three sub-topics in depth:
o the lives of several individuals born with the disease -- the most interesting aspect of the book
o the science behind the drug development -- interesting, but told in somewhat unnecessary detail, and a reader who doesn't have much of a science background would probably find this to be tough going
o the fund-raising efforts of the Cystic Fibrosis Foundation, which frankly was of almost no interest to me.
So worth reading, with these caveats
February 1, 2022
So impactful to see our lived experience presented this way. Our son was involved in two of the trials, and is now benefitting from the triple. So grateful for the efforts of Joe and all the others who came before us and made this possible
May 27, 2021
Bijal Trivedi's book is an epic tale of Cystic fibrosis disease and the several decades of science, research and development that went into developing a cure. I love the book because she makes the complex science and financing easier to digest. More importantly, she follows so many people and narrates so many stories of courage and heart break - of patients, families, physicians and researchers - constantly placing drug development in context of what it meant for the people involved.
This isn't a casual read. I'd suggested pairing this with something light-hearted or taking breaks while reading this book.
This isn't a casual read. I'd suggested pairing this with something light-hearted or taking breaks while reading this book.
June 19, 2021
This was a massive book to read at 615 pages, and much of it was like a medical textbook. I felt it was worth the effort, however, to read the personal stories from families that were scattered within the informative material. I’ve been interested about CF since 1986, when the t.v. movie “Alex, The Life of a Child” debuted, based on the book written by Frank DeFord. The story of this child’s struggle with her disease, which took her life at the age of eight, has been a very inspirational to me. The stories related by the families in “Breath From Salt” were certainly no less moving.
July 30, 2024
I finally finished this behemoth of a book! I normally have a strict policy of not mixing work and reading, but this was strongly recommended to me and on a topic I wanted to learn more about. And it was really good!
This essentially walks through, in incredible detail, the clinical and scientific history of studying and treating cystic fibrosis in the past century. All the way from the first (female!) pathologist who discovered the disease, to the recent drugs that have improved life expectancy by decades for many patients. I was pretty surprised, but I actually really enjoyed learning about all of the historical research. It was really interesting to learn about the genetic techniques that people were using in the '80s, for example. The section where they describe the race to identify the CFTR gene was particularly exciting to read.
In addition to all the good science discussions, the book also includes tons of stories of patients and family members. It also goes into lots of detail on the fundraising efforts and the cystic fibrosis association. Overall, this was an incredibly interesting and valuable read and I would definitely recommend it to anyone with at least one month of time!
This essentially walks through, in incredible detail, the clinical and scientific history of studying and treating cystic fibrosis in the past century. All the way from the first (female!) pathologist who discovered the disease, to the recent drugs that have improved life expectancy by decades for many patients. I was pretty surprised, but I actually really enjoyed learning about all of the historical research. It was really interesting to learn about the genetic techniques that people were using in the '80s, for example. The section where they describe the race to identify the CFTR gene was particularly exciting to read.
In addition to all the good science discussions, the book also includes tons of stories of patients and family members. It also goes into lots of detail on the fundraising efforts and the cystic fibrosis association. Overall, this was an incredibly interesting and valuable read and I would definitely recommend it to anyone with at least one month of time!
April 5, 2021
I was hesitant to pick this book up. It's over 600 pages and I've never had an interest in science non-fiction. I am so glad I ended up reading this! Trivedi made me feel connected to every individual and family affected by CF. I cried on several accounts. But at the heart of this book is hope. It made me appreciate how much science has advanced and excited me for the future of science!
June 4, 2021
My best friend has CF and I've been trying to stay on top of the information for the last 20 years. Additionally, I love reading nonfiction, science, and history books. So I should be a pretty good audience for this book but it was way way way way too long and full of unmemorable details. I finished it out of sheer stubbornness. I mostly recommend this book if you're 1) interested in how chloride transport works in our cell (middle of book), or 2) interested in innovating funding for disease cures (middle of book).
January 1, 2021
This is an engaging book describing a turning point in medicine. I couldn't put it down. I cried tears of sadness, frustration, and joy while reading about the struggle to treat cystic fibrosis. Five stars and two thumbs up!
December 3, 2020
I rarely read nonfiction, but was intrigued by this book because I have two relatives with cystic fibrosis (CF), and I spent a couple of years working for a small biotech company that was developing drugs to treat CF (without success, unfortunately). Although I've worked in the pharmaceutical industry for years, my role has been administrative/technical, and I do not have a science background. This book presented the science behind the disease and drug development process in a way most lay people can understand, even if a bit challenging at times. It's a wonderful, inspiring story of how patients, families, health care providers, scientists, industry, and nonprofits can join forces to accomplish the extraordinary. This is a great book for anyone wanting to understand more about disease and drug development and why it can take so long and be so costly to find effective treatments. Because of the amazing work the people of this story accomplished, ~90% of CF patients now have a treatment option. I have faith that if the Cystic Fibrosis Foundation continues to fund and promote cutting-edge research with dedication and passion equal to their past, they will reach their goal of finding treatments for all patients and ultimately a cure.
January 29, 2021
Just finished the last page of this book and I am left with such a deep appreciation for every single person mentioned in this book, from the parents, to the scientists to the doctors and everyone past and present at the Cystic Fibrosis Foundation. Having been diagnosed with CF at age 3, I had heard how the term ‘sixty five roses’ came about, I had learned the biology that goes into passing on the CF gene, and just recently following the CFTR modulators mentioned towards the end of the book. But I had no idea of the in-depth history that went into studying and diagnosing the disease in the early half of the 1900’s. I had no idea how the CF foundation actually came about. I had no idea that the scientists who studied the disease and made so many breakthroughs were such pioneers.
I have such empathy for all of those with CF who have come before me. It is because of them and those who participated in all of the clinical trials throughout the years/decades that I am alive and breathing right now.
This book is not only about the CF patients and the many scientific breakthroughs; it’s also a book about hope. Hope for a future free of cystic fibrosis. The events in this book are a testament that someday that is possible.
I have such empathy for all of those with CF who have come before me. It is because of them and those who participated in all of the clinical trials throughout the years/decades that I am alive and breathing right now.
This book is not only about the CF patients and the many scientific breakthroughs; it’s also a book about hope. Hope for a future free of cystic fibrosis. The events in this book are a testament that someday that is possible.
January 23, 2021
An unbelievably well written account of changing the trajectory of a cystic fibrosis diagnosis. The author does a phenomenal job of explaining the science thoroughly without making this a textbook on cystic fibrosis. This is mostly done through the stories of the major players in the US based Cystic Fibrosis Foundation (necessary for a 500 page novel about CF), which is also what keeps the book from being the full CF story.
Despite the vital role of the stories in keeping the reader engaged, CF is not a disease where progress was made by a few groups in the US and Canada alone. It's a bit of a disservice to the other research teams involved, but still worth the read as an informative and accurate account of the disease in the US, while being filled with lessons on how to bring people together to achieve something great.
Despite the vital role of the stories in keeping the reader engaged, CF is not a disease where progress was made by a few groups in the US and Canada alone. It's a bit of a disservice to the other research teams involved, but still worth the read as an informative and accurate account of the disease in the US, while being filled with lessons on how to bring people together to achieve something great.
August 8, 2022
I have always been interested in Cystic Fibrosis. I have a close friend who has a daughter with CF and have watched her over the years struggling with this disease. A year or so ago she began a new medication ( Trikafta) that has changed her life.
This book is the history of the development of the drugs for CF. It is also the story of how a foundation took it upon themselves to find a treatment for this deadly disease. The CF Foundation has raised billions of dollars on their own.
These treatments nor the study of the genes causing CF were funded by the foundation .
The first drug was introduced in 2012 then the next in 2015, 2018, and finally 2019. The last drug developed was Trikafta, the drug that changed my friends daughter life in ways they could not imagine.
The Foundation is still working on drugs for those with rare mutations and will not stop until they do.
This book is the history of the development of the drugs for CF. It is also the story of how a foundation took it upon themselves to find a treatment for this deadly disease. The CF Foundation has raised billions of dollars on their own.
These treatments nor the study of the genes causing CF were funded by the foundation .
The first drug was introduced in 2012 then the next in 2015, 2018, and finally 2019. The last drug developed was Trikafta, the drug that changed my friends daughter life in ways they could not imagine.
The Foundation is still working on drugs for those with rare mutations and will not stop until they do.
September 14, 2020
This is an incredible, well-researched, and thorough look at the past and present of cystic fibrosis. As a person with CF, I learned so much about myself and the condition I live with every day. I highlighted probably 50 passages and spent the last ten days asking my family “Did you know this? What about this?” and telling them cool things I learned. I would compare this to The Gene- it is undeniably science writing, but it is perfectly accessible for someone without a science or medicine background. I’ve recommended it to dozens of people, and just cannot day enough positive things about the book. I am just thrilled that a book about the history of CF exists, and that it’s this phenomenal book.
I received a review copy from the publisher, which in no way affected my review.
I received a review copy from the publisher, which in no way affected my review.
April 27, 2021
What a privilege to have this story written in this format at this time. Not many people can say, "I need to know everything about the history and development of this specific rare thing from discovery to the present day, AND I want it in a clear and digestible format." and then HAVE THAT EXACTLY. Took me months to get through the first half and a week to finish the second.
Such a beautiful story of perseverance and sacrifice. The word grateful doesn't do my feeling justice, but I am immensely grateful for every single person mentioned in this story and the author for telling it.
Such a beautiful story of perseverance and sacrifice. The word grateful doesn't do my feeling justice, but I am immensely grateful for every single person mentioned in this story and the author for telling it.
January 7, 2025
I picked up this book thinking about it was cystic fibrosis (CF), and it was – but it was also about effective philanthropy and how to jumpstart innovation on a seemingly incurable disease.
What fascinated me was seeing how much science truly is a conversation: the original description of CF, for example, emerged when a physician noticed missed patterns in patients previously diagnosed with gluten intolerance. Physicians tried one thing, then another; individually each contributing the smallest piece yet aggregating the information that laid the groundwork for eventual effective treatments. You see how what was once thought impossible, gets broken down step by step as we learn more.
Then there’s the fundraising. CF absolutely wouldn’t have been cured had there not been 60+ years of fundraising by dedicated families. But raising money wasn’t enough. There came a point when the foundation had to reduce paying for direct patient care – over the protest of families – because they recognized only research would yield a cure. What felt helpful in the short term, was stopping them from succeeding in the long term. There was the recognition they had to push science to do things that seemed crazy, because venture capital alone – too desirous for guaranteed profits – wouldn’t go there on their own. In a way, this story is about effective altruism before EA was a thing. They wanted to do what worked.
The real power in this book is illustrating how to tackle a seemingly intractable problem, and how many people it took working on different pieces of it even when they knew their efforts might not pay off. The state legislator who spent over a decade passing laws that let science innovation flourish; the scientists who spent year after year making incremental tweaks to drug compounds; the physicians who treated CF back when there were literally no options but still did research that paved the way for eventual cures decades later. The fundraisers, who figured out how to raise $175M, finished, and immediately started again. The parents who raised money and advocated – sometimes even after their children had died, so other parents wouldn’t have to experience the same loss. It gives me hope – and a roadmap – for fixing other problems we once thought impossible; it gives me courage to ask people to keep going and working for the long haul.
One thing I continue to ponder is who bears the ultimate costs of CF. Despite receiving extensive funding from the Cystic Fibrosis Foundation (and the foundation being the ones to request they even work on it), the company (Vertex) that finally produced the first breakthrough drug turned around and charged $294,000 per month for the drug. Vertex argued patients didn’t truly pay that, because insurance covers the cost – but who pays for insurance? You have a situation where patients fundraise for years, the money gets donated to a for-profit company, the for-profit company charges outrageous of money, and society turns around and gives them that money to increase their profits. Now, Vertex was critical in the success of this drug, and perhaps this is how capitalism works – but I think it’s important to be aware of as we think about innovation.
What fascinated me was seeing how much science truly is a conversation: the original description of CF, for example, emerged when a physician noticed missed patterns in patients previously diagnosed with gluten intolerance. Physicians tried one thing, then another; individually each contributing the smallest piece yet aggregating the information that laid the groundwork for eventual effective treatments. You see how what was once thought impossible, gets broken down step by step as we learn more.
Then there’s the fundraising. CF absolutely wouldn’t have been cured had there not been 60+ years of fundraising by dedicated families. But raising money wasn’t enough. There came a point when the foundation had to reduce paying for direct patient care – over the protest of families – because they recognized only research would yield a cure. What felt helpful in the short term, was stopping them from succeeding in the long term. There was the recognition they had to push science to do things that seemed crazy, because venture capital alone – too desirous for guaranteed profits – wouldn’t go there on their own. In a way, this story is about effective altruism before EA was a thing. They wanted to do what worked.
The real power in this book is illustrating how to tackle a seemingly intractable problem, and how many people it took working on different pieces of it even when they knew their efforts might not pay off. The state legislator who spent over a decade passing laws that let science innovation flourish; the scientists who spent year after year making incremental tweaks to drug compounds; the physicians who treated CF back when there were literally no options but still did research that paved the way for eventual cures decades later. The fundraisers, who figured out how to raise $175M, finished, and immediately started again. The parents who raised money and advocated – sometimes even after their children had died, so other parents wouldn’t have to experience the same loss. It gives me hope – and a roadmap – for fixing other problems we once thought impossible; it gives me courage to ask people to keep going and working for the long haul.
One thing I continue to ponder is who bears the ultimate costs of CF. Despite receiving extensive funding from the Cystic Fibrosis Foundation (and the foundation being the ones to request they even work on it), the company (Vertex) that finally produced the first breakthrough drug turned around and charged $294,000 per month for the drug. Vertex argued patients didn’t truly pay that, because insurance covers the cost – but who pays for insurance? You have a situation where patients fundraise for years, the money gets donated to a for-profit company, the for-profit company charges outrageous of money, and society turns around and gives them that money to increase their profits. Now, Vertex was critical in the success of this drug, and perhaps this is how capitalism works – but I think it’s important to be aware of as we think about innovation.
April 24, 2021
I am so impressed with Trivedi's ability to take a complex medical topic and explain it in a way that readers can follow. This was a very comprehensive book, covering cystic fibrosis from the time it was officially recognized as a disease through today. It was a business book, a science book, and a grief book, all rolled into one. I was captivated and am now very knowledgeable about CF.
A student of mine, a girl in 8th grade, died from CF in 2010. As I listened to the book, I thought to myself how this happened just 11 years ago.... Has that much really changed since then, that she might have lived if these discoveries had happened sooner? Sadly for her and other children, the answer is yes, but fortunately for everyone today with CF, there is a better chance at a future.
I was amazed at the effort and innovation that went into the creation of the new CF medicines. The many people you meet in this book, especially the O'Donnell family, are simply awe inspiring. This book is long and extremely detailed, but it's worth the effort.
A student of mine, a girl in 8th grade, died from CF in 2010. As I listened to the book, I thought to myself how this happened just 11 years ago.... Has that much really changed since then, that she might have lived if these discoveries had happened sooner? Sadly for her and other children, the answer is yes, but fortunately for everyone today with CF, there is a better chance at a future.
I was amazed at the effort and innovation that went into the creation of the new CF medicines. The many people you meet in this book, especially the O'Donnell family, are simply awe inspiring. This book is long and extremely detailed, but it's worth the effort.
September 26, 2024
Wow. I am gobsmacked at the amount of work that must have gone in to writing this book. It is so impressive. I loved how Bijal Trivedi told the story of cystic fibrosis from so many angles - the families, the Foundation, the drug development. I was literally on the edge of my seat during the chapter where the breakthrough that ended up as Kalydeco happened. As a CF social worker, it was so cool to do a deep dive on how we got where we are now. I have so, so much respect for the scientists and the work they did to create these medicines, and for the parents, the relentless parents of kids with CF, that did the impossible.
June 8, 2021
Truly excellent book on the diagnosis and treatment of rare genetically associated disease (the book addresses cystic fibrosis, but many of the patterns apply more broadly), covering everything from the evolving diagnostic journey, foundation creation/fundraising to draw attention and research, clinical trial design, multiple avenues of therapeutic approaches, lab automation for combinatorial drug discovery, and so much more. Highly recommended for anyone working in or curious about the genetic origins of disease.
April 8, 2022
I’ve been working on this book for quite some time. It’s a slow, non-fiction read. The way the science behind the life changing developments in CF treatments was uncovered by a relentless team of scientists and fundraisers (all of whom were touched by the disease), was an incredible story that hooked me. The way individual stories were woven throughout kept me deeply engrossed in the scientific journey to find a cure. Would highly recommend if you enjoy epidemiology and science!!
February 14, 2021
This thick book was an incredible journey through the world of cystic fibrosis. I am quite familiar with CF but found myself gasping at new discoveries (even though I already knew about them!) and even cried along with all of the CFF when these life-changing drugs were approved. Though at times the book was a little heavy into the science side of things for my liking, I do think that was critical to understanding to the narrative and appreciated the author's layman's terms and illustrations to describe these more technical parts. A fascinating and important read.
March 21, 2021
An interesting and well researched book. I learned much more about cystic fibrosis, the science of the disease, and the search for treatments and hopefully someday a cure. I knew only of the mucus and lung issues of the disease, I had never heard of or considered how cystic fibrosis would affect the other organs. It was emotional reading the personal stories of individuals and families impacted by the terrifying diagnoses.
I feel like you need some knowledge of biochemistry, genetics, and related subjects really helps when reading parts of this book.
I feel like you need some knowledge of biochemistry, genetics, and related subjects really helps when reading parts of this book.
August 8, 2022
Perhaps a too comprehensive coverage of the history of cystic fibrosis. The author narrated a broad range of topics, such as the basic science of CF, the scientific discovery pathways, and the fundraising journey. While covering all grounds, each chapter read scattered and unrelated. Description of certain personalities felt cliche and over-the-top.
March 8, 2022
Wow. Wow. Wow. Amazing telling of a fantastic success story of a huge group of folks focused on a central goal. What an achievement! These drugs, piggybacking on decades of innovative discovery, are changing the landscape for patients with CF. Heartwarming.
January 2, 2025
Incredible… such a thorough book on this disease. I am so inspired by and grateful for the parents who have fought for years to find answers to save our children’s lives. Thank God for modern medicine!!
January 25, 2021
Wow! Not knowing much about the development of these drugs meant this book read like a mystery novel. I couldn’t wait to find out how the story ended.
Want to read
February 17, 2022I started listening to Breath from Salt today. It’s extremely compelling and drew me in from the very start.
December 25, 2023
Worth the 5+ months I spent on this.
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