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We the Scientists: How a Daring Team of Parents and Doctors Forged a New Path for Medicine
A Pulitzer Prize–winning reporter’s moving narrative of a group of patient advocates who are revolutionizing the way medical research is conducted.
For more than half a century, medical advances have been driven by investigators launching experiments inside labs. Science is often conducted in isolation and geared toward the long view. This is the story of a group of people who tried to force the lab doors parents whose children had been diagnosed with a rare and fatal genetic condition known as Niemann-Pick disease type C. The disease prevents cells from processing cholesterol, which leads to the progressive loss of the brain’s and the body’s ability to function. Recognizing that there would never be a treatment in time to save their children if things stayed the same, the parents set up a collaboration with researchers and doctors in search of a cure.
Reconciling different views of science took work. The parents, doctors, and researchers didn't always agree—among themselves or with each other. But together they endeavored to accelerate the development of new drugs. The parents became citizen scientists, identifying promising new treatments and helping devise experiments. They recorded data about the children and co-authored scientific papers sharing findings. They engaged directly with the FDA at each step of the drug approval process. Along the way, they advanced the radical idea that science must belong to us all.
Amy Dockser Marcus shows what happens when a community joins forces with doctors and researchers to try to save children’s lives. Their extraordinary social experiment reveals new pathways for treating disease and conducting research. Science may be forever changed.
For more than half a century, medical advances have been driven by investigators launching experiments inside labs. Science is often conducted in isolation and geared toward the long view. This is the story of a group of people who tried to force the lab doors parents whose children had been diagnosed with a rare and fatal genetic condition known as Niemann-Pick disease type C. The disease prevents cells from processing cholesterol, which leads to the progressive loss of the brain’s and the body’s ability to function. Recognizing that there would never be a treatment in time to save their children if things stayed the same, the parents set up a collaboration with researchers and doctors in search of a cure.
Reconciling different views of science took work. The parents, doctors, and researchers didn't always agree—among themselves or with each other. But together they endeavored to accelerate the development of new drugs. The parents became citizen scientists, identifying promising new treatments and helping devise experiments. They recorded data about the children and co-authored scientific papers sharing findings. They engaged directly with the FDA at each step of the drug approval process. Along the way, they advanced the radical idea that science must belong to us all.
Amy Dockser Marcus shows what happens when a community joins forces with doctors and researchers to try to save children’s lives. Their extraordinary social experiment reveals new pathways for treating disease and conducting research. Science may be forever changed.
256 pages, Hardcover
Published February 14, 2023
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Displaying 1 - 24 of 24 reviews
February 20, 2023
The story of the family’s dedication to the collaborative project was emotional and evocative. But the author’s research into the scientists’ investment in the collaboration was underdeveloped and does not appear to be wholly in good faith. Anyone worth their salt in this domain would discuss patient centered outcomes research (PCOR) and how patient/family and physician/scientist collaboration has been monumental in this field; the author does not touch on this at all, which points to either incomplete research or intentional disregard…
March 19, 2024
Let me start by saying: I really wanted to like this book. The topic is compelling and the stories of families and parents are heartbreaking. As an aspiring physician, this gives critical insight into a side of medicine that is not discussed often enough.
That being said, the flaws:
1. Hearing the word “cyclodextrin” one more time might put me over the edge. The back half of the book was just an infomercial.
2. I have no clue who any of the people were. Everyone blended together.
3. The stylistic choices of how to describe people and events felt so off. Sometimes the author would spend two pages on how a certain doctor was a boy scout, other times she would completely skip over some critical interaction between major players or how the drug impacted the children. The flow needed work.
4. And here is the crux of my entire review: the right questions weren’t asked nor answered. The attention paid to the “paradigm shift” (loved the T. Kuhn shoutout) was skimpy. If cyclodextrin is readily available for compassionate use cases, why push so hard for FDA approval? Doesn’t the absence of scientific proof that it is working mean anything? If parents wanted to keep giving it to their children, nothing was stopping them, as I understood it.
With that question left unanswered, it naturally made me ask why the emphasis on that drug instead of allowing research to proceed on other promising leads like Tocopherol that was just dropped in the middle of the story? There may be a good reason, but without addressing it I’ll never know.
The points here are important to consider and find a middle ground. To just say that parents should be able to conduct their own research on human test subjects seems like a knee-jerk response to the availability of information thanks to the internet. There’s a reason all physicians go to medical school, to look at one part of the body in isolation is to narrow the view and potentially miss harmful impacts on other organs. Parents can become experts, certainly! Many do. The narrative proposed of niche experts throwing off the shackles of professional scientists makes me feel uneasy, even with the ethical components considered.
Again, I may be missing the point. But if that’s the sense I had when I finished the last page, what does that say about the book?
That being said, the flaws:
1. Hearing the word “cyclodextrin” one more time might put me over the edge. The back half of the book was just an infomercial.
2. I have no clue who any of the people were. Everyone blended together.
3. The stylistic choices of how to describe people and events felt so off. Sometimes the author would spend two pages on how a certain doctor was a boy scout, other times she would completely skip over some critical interaction between major players or how the drug impacted the children. The flow needed work.
4. And here is the crux of my entire review: the right questions weren’t asked nor answered. The attention paid to the “paradigm shift” (loved the T. Kuhn shoutout) was skimpy. If cyclodextrin is readily available for compassionate use cases, why push so hard for FDA approval? Doesn’t the absence of scientific proof that it is working mean anything? If parents wanted to keep giving it to their children, nothing was stopping them, as I understood it.
With that question left unanswered, it naturally made me ask why the emphasis on that drug instead of allowing research to proceed on other promising leads like Tocopherol that was just dropped in the middle of the story? There may be a good reason, but without addressing it I’ll never know.
The points here are important to consider and find a middle ground. To just say that parents should be able to conduct their own research on human test subjects seems like a knee-jerk response to the availability of information thanks to the internet. There’s a reason all physicians go to medical school, to look at one part of the body in isolation is to narrow the view and potentially miss harmful impacts on other organs. Parents can become experts, certainly! Many do. The narrative proposed of niche experts throwing off the shackles of professional scientists makes me feel uneasy, even with the ethical components considered.
Again, I may be missing the point. But if that’s the sense I had when I finished the last page, what does that say about the book?
This entire review has been hidden because of spoilers.
April 4, 2023
An intriguing story about paradigm shifts and the power of parents of a terminally ill child. Lots of talking points with heartwarming stories about families who go outside their comfort zones to work with drug companies, the NIH, and doctors to push for more research for rare diseases. This should be added to the required reading for all students in medical school.
I listened to this book and it was performed well.
I listened to this book and it was performed well.
June 17, 2023
Too slight for me. I’ve read much more substantive books on this topic of patient scientific advocacy, particularly Act Up in the 1980s, and was irritated at what little mention they got. Also, this author stunk at showing the passage of time. Kids went from toddlers to teenagers in a chapter and you couldn’t tell until she mentioned the age at which they died in the chapter after that.
March 22, 2023
This is the story of how a group of parents - whose children were diagnosed with a fatal disease called Neimann Pick Type C - encouraged scientist to collaborate with them to find a cure for their children. Scientist - who normally are tight lipped about their experiments and work - chose to work with parents to try a save the lives of these childrens. Thus leading to the first "citizen scientists" that paved the way for other parent groups to collaborate with scientist on othe diseases. While the cure for NPC has still not been found - the work of the parents along side of the scientist made progress that might not have otherwise been made if they did not work together pulling their resources and research toward one goal.
This was a good book. I love a good non-fiction and one that has patient stories are among my favorites. My husband has done with with NPC and we are very familiar with the rare disease world with our daughter having one of those rare diseases herself. Our own foundation has been working closely with scientist on our daughter's disease for the last decade and we are grateful that the scientist in our case are so willing to work with us to find a cure.
Well written, well researched and heart wrenching, this book is worth a read. The stories of these families are tragic. Children with NPC live around a maximum of 20 years, and most of those years are spent severely disabled. Children who are born normally quickly start to lose function in their early years and their parents watch them wither away for years. The work that these parents and researchers are doing to save these children is harrowing.
This was a good book. I love a good non-fiction and one that has patient stories are among my favorites. My husband has done with with NPC and we are very familiar with the rare disease world with our daughter having one of those rare diseases herself. Our own foundation has been working closely with scientist on our daughter's disease for the last decade and we are grateful that the scientist in our case are so willing to work with us to find a cure.
Well written, well researched and heart wrenching, this book is worth a read. The stories of these families are tragic. Children with NPC live around a maximum of 20 years, and most of those years are spent severely disabled. Children who are born normally quickly start to lose function in their early years and their parents watch them wither away for years. The work that these parents and researchers are doing to save these children is harrowing.
January 13, 2024
I generally like narrative nonfiction and books about unlikely alliances. We the Scientists delivers both. Sometimes inspiring, sometimes disheartening, always compelling, Amy Dockser Marcus provides a detailed journey of patients, parents, physicians, scientists, advocates, business leaders, and bureaucrats as they all try their best - COLLECTIVELY - to find a cure for the rare Niemann-Pick disease. The collective part is emphasized because those siloed cohorts rarely work together. This book tells the story of a time when they did and what they were able to accomplish together. It reads like a long New Yorker article.
[Potential spoiler] I feel like she was going to tell this story no matter the outcome of her efforts. She was all in for those kids with Niemann-Pick disease and that's wonderful. Unfortunately, those outcomes, at least to me, were underwhelming. I suppose the lesson is about the journey of collective action rather than the results of the collaboration. It felt like the book stopped vs. the story ended because all the buildup remained unresolved.
In the end, I'm thrilled that millions more people are aware of Niemann-Pick disease and the potential power of bringing patients, parents, and other advocates into major medical policy and research decisions.
[Potential spoiler] I feel like she was going to tell this story no matter the outcome of her efforts. She was all in for those kids with Niemann-Pick disease and that's wonderful. Unfortunately, those outcomes, at least to me, were underwhelming. I suppose the lesson is about the journey of collective action rather than the results of the collaboration. It felt like the book stopped vs. the story ended because all the buildup remained unresolved.
In the end, I'm thrilled that millions more people are aware of Niemann-Pick disease and the potential power of bringing patients, parents, and other advocates into major medical policy and research decisions.
August 22, 2025
A moving, inspirational look inside the collaboration between citizens and professional scientists. I do agree this is the way science should be done. Especially in the realm of drug development-which the book focuses on- what is the point if the drug isn’t benefitting the person it’s made for? Shouldn’t it be made in collaboration with them? At the same time, there is a reason being a scientist takes years of schooling and research. There’s a reason it’s a profession. I don’t know that I’m ready to say citizen scientists should be able to make the same decisions that professional scientists do. But I do think that there needs to be a faster way for drug development to happen, similarly to what we experienced with the COVID-19 pandemic. A vaccine within a year? How are we able to do that for a novel, unheard of respiratory illness but not for diseases like Alzheimer’s and cancers that have been around for centuries? Granted, these diseases are much more complex, but the time we’ve had to research them compared to COVID and the results we’ve had for both are astonishing. That’s where I think this book makes a very strong point
June 2, 2023
This is a short book that lacks the narrative punch of other medical creative nonfiction, like And the Band Played On or The Perfect Predator (Strathdee). True, it's not about uncovering a disease process like these are, but there were still some high-stakes events and conversations going on which didn't get the compelling framing they deserved. I would've gladly read a book twice as long had it included more detail about, well, just about everything. For example, like another reviewer noted, there's a greater context to this patient-inclusive medical research movement than just NPC disease and long Covid, and I would've liked to hear about some other such efforts and if they ran into similar pitfalls or were able to make progress. I feel like the book read more like an extended (ok, very extended) magazine article than a long-form manuscript.
That said, as someone with a common and well-researched chronic illness (type 1 diabetes), I now better appreciate what folks with rare diseases are up against vis a vis drug discovery, etc.
That said, as someone with a common and well-researched chronic illness (type 1 diabetes), I now better appreciate what folks with rare diseases are up against vis a vis drug discovery, etc.
May 31, 2024
Amy Dockser Marcus, a medical ethicist and journalist, tells the story of a pioneering effort between parents of children with Niemann-Pick disease type C, a rare genetic problem where cholesterol builds up in tissue, including brain tissue, causing its sufferers to progressively lose cognitive and muscle function. Breaking the model where researchers choose their own scientific areas to study, here the parents, scientists, and clinicians collaborated to attempt to deliver results that would help children with NPC in the short term, rather than 10 years in the future. The answers were of limited use, as most all the children died anyway, but in establishing a collaborative model they got the attention of the FDA, who helped bring rigor to the process. A short, stirring book.
March 31, 2023
We the Scientists is an inspiring and thought-provoking book that explores the incredible journey of parents and doctors who banded together to find a cure for a rare and deadly genetic disease. After her mother died from a rare form of cancer, the author traveled around the country and met a group of parents whose children also had the same rare disease. They were trying to forge a very unusual collaboration where they would partner with scientists and researchers to find a drug to treat this disease. The author skillfully weaves together the personal stories of the families affected by the disease and the scientific discoveries that led to a breakthrough treatment. Through her meticulous research and vivid storytelling, the author highlights the power of collaboration, perseverance, and the human spirit in the face of adversity.
It blew my mind that in today's day and age when we have all this technology available, deciding which lives are worth saving and which diseases are worth finding the cure for is a numbers game. One of the author's arguments was to set up partnerships here, including the ability to apply for research funding from the government just like scientists do all the time for the diseases they want to study. She also raises important questions about the future of medicine and the role of patient advocacy in shaping it.
To listen to my interview with the author, go to my podcast at:
https://www.momsdonthavetimetoreadboo...
It blew my mind that in today's day and age when we have all this technology available, deciding which lives are worth saving and which diseases are worth finding the cure for is a numbers game. One of the author's arguments was to set up partnerships here, including the ability to apply for research funding from the government just like scientists do all the time for the diseases they want to study. She also raises important questions about the future of medicine and the role of patient advocacy in shaping it.
To listen to my interview with the author, go to my podcast at:
https://www.momsdonthavetimetoreadboo...
July 6, 2025
I understand why some people would not think this book is good, but I believe they are missing the essence. This book is not about the parents or children with NPC and the progression of their diseases; it is neither a biography nor a scientific article, nor a medical guide for scientists. It's a path for a non-technical person to see a different reality. This book lacks many aspects, like a precise chronology or more tangible conclusions. Still, it's brilliant in one element: opening the borders of science, and giving any person the hope that science can help them, even when the science itself tells them not.
March 14, 2024
Reading this book is a good reminder of how much goes into a pharmaceutical drug before the medication is allowed to be recommend in the doctors office. These parents are an amazing group of people and I could see myself in each of their shoes. We are all in it together and it is people like these families who have paved the way for future generations. Thank you for putting this together to show us what is possible.
June 20, 2024
I like the idea of patient led research and making research more applicable but the story is incomplete without at least crediting all the basic researchers and scientists. Also, confused about the ending… the book is focused on the patients and families with NPC but at the end it does not answer how all this patient led research has moved the field? Maybe I misunderstood. Overall, kinda disappointed in this book.
March 12, 2023
While most non-fiction books, whose focus are on medicine and clinical trials with little information about the patients behind the research, this book leans more towards the humanitarian side of the research and how parents pushed to get their children seen and treated by professionals with a drug that may or may not elongate their lives.
May 7, 2023
Eyes-opening and insightful for me as a scientist. A model to bring science into practicality. There are so many stories that touch my heart, showing the hard work of all parties, especially the parents. For the good of human health, be it a rare or common disease, layman and experts should be together in this.
October 6, 2023
Having a granddaughter with a rare disease this book was very insightful. The disease in this book was different than my granddaughters but the process of finding the drugs is very similar. I'm glad to see scientists and parents working together to find and test drugs that may be effective against these rare diseases.
January 3, 2025
I think about this book all the time. The system is not as efficient as you would want it to be, but the doctors, scientists, funders and, most importantly, the patient advocates are all doing the best they can.
Read
December 18, 2025Intimate account of families confronting rare childhood disease; parents set up a collaboration with researchers and doctors to try to speed up finding a cure. This changed some ways of treating disease but unfortunately did not find a cure
April 19, 2023
B- Audible
April 28, 2023
This book was a very interesting look into a partnership that combined scientists, patients, and families coming together to take a new approach to the medications needed for rare disease treatments. The personal stories of the parents and patients kept it interesting while the issues with the current model of drug development and the issues it creates for children of this rare disease were explained fully.
June 22, 2023
I loved this book and it is super relevant for patient centric clinical trials. Was sad tho 😞
Fav quote: “Working together, science can reach its fullest potential”
😵💫
Fav quote: “Working together, science can reach its fullest potential”
😵💫
September 17, 2024
such a emotional read! highly reccomend
March 27, 2024
Beautiful story or what real people can do to make a change in disease treatment.
August 16, 2023
Moving book focused on the story of patients and doctors working together to make progress against a fatal disease. This opened my eyes to a world I am not much involved in, I look to increase my knowledge on this and other areas where this has been done. The international aspect is interesting, as there are so many stipulations in USA
Displaying 1 - 24 of 24 reviews