Zibby interviews Pulitzer Prize-winning reporter Amy Dockser Marcus about We the Scientists: How a Daring Team of Parents and Doctors Forged a New Path for Medicine, a gripping and deeply compassionate account of a fatal genetic disease and the collaboration between families and researchers to one day vanquish it. Amy talks about her mother, who died of a rare form of cancer and inspired this incredible project. She also describes the 10-year period she spent with the community of families whose children suffer from the rare and fatal Niemann-Pick Disease Type C and the unique way they contributed to the development of new treatments. Finally, she talks about her impressive career, from journalism assistant to foreign correspondent in the Middle East to ultimately falling in love with health and science beat reporting.

Transcript:

Zibby Owens: Welcome, Amy. Thanks so much for coming on “Moms Don’t Have Time to Read Books” to discuss We the Scientists. I saw the cover behind you. Hold it up in case I — I’m reading the subtitle. How a Daring Team of Parents and Doctors Forged a New Path for Medicine. Tell us everything about this book and particularly, how your own mother’s diagnosis inspired your hunt for answers to diagnoses and everything.

Amy Dockser Marcus: First, thank you for having me. I’m so honored to be here. We the Scientists does have its origins in a personal story. My mother was diagnosed with a rare form of cancer, metastatic gallbladder cancer.

Zibby: I’m so sorry.

Amy: Thank you. It was so difficult also because I’m reporter, and I cover health, and so I was used to searching for answers and doing research. I interviewed so many doctors trying to find some effective therapy for her. I learned that there were none, in part because it’s a very rare disease. Pharmaceutical companies weren’t pursuing drugs. Doctors weren’t able to study it. They didn’t have a lot of patience. Researchers didn’t think of it as a disease they wanted to focus on. Unfortunately, my mother passed away after a little bit of over two years with the disease. It really launched me into thinking about, why is it so hard to advance science? Why aren’t things moving faster? Why can’t we get more drugs out there for more diseases? I ended up taking some time off from my job as a Wall Street Journal health and science reporter. I traveled around the country, and I met people who were trying to change the system. One of the groups that I ended up meeting were a group of parents whose children also have a rare disease. It’s called Niemann-Pick type C. It’s a cholesterol disorder. It’s fatal. They were trying to forge a very unusual collaboration where they would work as partners with scientists and researchers to try to find a drug to treat this disease. I just started following them and chronicling their story.

Zibby: You did that for ten years.

Amy: Yes. It was a very long process.

Zibby: This isn’t like you did this for a semester or something like that.

Amy: No. I really got to know everyone. I met all their kids. I got to know the parents. I got to know the scientists. One of the things that’s unusual in rare disease communities is everyone knows each other. They’re relatively small communities. There are annual science and parent support groups meetings. Then the parents were so welcoming. They invited me into their homes. I got to meet the children. The scientists let me come into their labs. I kept going because they eventually did find a drug that they wanted to develop together. They were able to get it into a clinical trial. They ended up talking to the FDA and really participating in this project in a way that was more unusual. I just kept going with them.

Zibby: That’s amazing. It’s just so incredible. It also blows my mind that in today’s day and age — who is it up to who decides which lives are worth saving? Which diseases? I understand it’s a numbers game. When it’s your loved one, why does that mean there shouldn’t be a cure? Are there just way too many? It sounds like a naïve question. Is it just impossible?

Amy: It’s not a naïve question. I think it’s the central question. How do we decide where we’re going to focus our attention and our resources? Are some lives more valuable than others? Obviously, I think all lives are valuable. I think everyone deserves, if not a treatment — no one can be guaranteed a treatment. One of the arguments of this book is a treatment is not guaranteed, but we should create a system where, working together, we have a better chance to develop a treatment. We can partner to develop a treatment. One of the reason why I think it’s hard to do this is because we don’t use all of the expertise that’s available to us. I argue that these parents — they know their children so well. They immerse themselves in this disease. Many doctors never see a patient in their entire careers with Niemann-Pick disease type C. It’s very, very rare. There’s only around two hundred or so cases in the US, maybe five hundred or so cases internationally. If that’s the case, then we have to use all the data and all the resources. That means trying to help all of us participate in science.

Zibby: I love how you call them — what did you say? Social scientists? Not social scientists. You had some great buzzword for community activists.

Amy: They’re citizen scientists.

Zibby: Yes, yes, yes. That was it. Citizen scientists. Sorry.

Amy: There’s a broad movement of citizen science. It’s been around for a while. Even the title of the book, We the Scientists, I intentionally chose that as a kind of echo to “We the people.” We all should consider ourselves not just the beneficiaries of science, but also potential contributors to science.

Zibby: I feel like with the pandemic, everybody was trying to help in their own way when we were all collectively fighting for the vaccine. Everybody was doing whatever they could. Obviously, that affected everybody. I think many people who hadn’t been exposed to that level of involvement saw even the layperson going through all the documents and figuring out what to do and all of that. It’s just how to scale that for every disease.

Amy: I mention in the book, long COVID, the patients that struggle with symptoms for months or even years after a diagnosis with COVID-19. I definitely think that all these groups that are emerging build on what has happened and come before. I think that during the pandemic, we were much more attuned to the idea that it’s a novel disease. We don’t know a lot about it. Even the scientists don’t know a lot about it. The doctors don’t know a lot about it, so let’s try to gather data from the patients. One of the arguments in the book is, I don’t think that should be confined to COVID-19 pandemic. I think that’s an approach that we should bring to every disease. If you’re living with a disease or someone you love is, then you have data and expertise that’s incredibly valuable. You have something to contribute that might help accelerate the search for therapies and treatments.

Zibby: I have a dear friend whose mother passed away from lung cancer. She was really involved with the Lung Cancer Research Foundation. Every year, I would go to that lunch. It always blew my mind that the funding for that is such a small fraction of what it is for even breast cancer and that there’s this — at least for that particular cancer, everybody — not everybody, but there is this assumption that you’ve almost caused it yourself. Therefore, it doesn’t deserve the funding that a blameless cancer — that’s what they would talk about, perhaps, as to why the treatment is less. Again, allocating resources and who gets to decide just seems crazy, in a way.

Amy: It’s such an important point that you’re raising. Fighting for attention to try to get people to focus on the things that matter to you sometimes do turn on factors that are outside of the realm of science or even sometimes numbers. People do bring a lot of their own value system into deciding these things. One of the things that I describe in We the Scientists is that at the National Institutes of Health, there was an institute there called NCATS where the director at the time, one of his visions was, how can I collaborate with patients who have diseases that really are often off the radar because there’s not a lot of people or there’s not a lot of interest or funding? He got involved. He believed that the government should get involved. I think that there is an argument to be made that some of the funding for biomedical research that comes via government institutions can be directed to create partnerships between scientists and those who want to be citizen scientists. One of the arguments in my book is, let’s try to make partnerships here, including an ability to apply for research funding from the government just like scientists do all the time for the diseases they want to study.

Zibby: Totally, oh, my gosh. Tell me more about what it was like for you emotionally trying to be an impartial observer of this group of families, and a reporter. How could you not get swayed into the emotional component of the disease and the impact of it on the families? What was that like for you?

Amy: I definitely was emotional. I don’t park my emotions at the door. That’s for sure. I definitely try, as a reporter, to bring my passion and interest in whatever I’m covering to my approach. I spent a lot of time with everybody. Often, in narratives about a race against time, there’s a villain, in a sense. There’s the bad guy. There’s sides. One of the things I came away from this is there aren’t sides. There aren’t villains. There’s a system that’s really not working well. There are so many obstacles that can just grind you down and seem like it makes it impossible for you to advance. There’s obstacles for the parents and for patients. There’s obstacles also for scientists. They’re always worried about, they have to look for their next grant. They need to publish a paper so that they can get tenure. They need to justify how they’re spending their time and their resources. There’s nothing wrong with that. We all justify how we spend our time and our resources. I came away from it feeling like the people involved here are all good people, and yet it’s not working as well as it should. Why is that? How could we make changes to make it better? That’s the way I tried to come at it, not pitting people against one another, but rather, trying to understand their viewpoint, how they ended up in the room that we were all gathered in, and how their values informed the choices they were making and the arguments they were having.

Zibby: Interesting. Do you mind backing up and telling me how you became a Wall Street Journal health reporter and Pulitzer Prize finalist and all of that? How did you get started on everything? What did you think you wanted to be when you were a kid?

Amy: I always wanted to be a writer. I came home from school when I was eight years old and announced to my parents that was what I was going to do. I never veered from that. I loved writing. I always loved writing. I’ve been at The Wall Street Journal for many years. I started out as the lowest entry-level job you can have. I was the news assistant for the legal reporters. I would go to the courthouse and pull their documents. I would bring them coffee. I would try to create charts that illustrated their amazing reporting and just worked my way up to become a reporter. I had spent many years as a foreign correspondent for The Wall Street Journal covering the Middle East and then got back to the States and was looking around. What should I cover next? What’s of interest to me? I tried education for a while, which I loved. That was a great beat. Then there was this opening for a health reporter on a new section that the paper was launching. The editor was a good friend. He approached me and said, “I think you’d love writing about health. It’s all about life and death and society and ethical issues. I just think you’d love it.” I hadn’t given it a lot of thought before. I trusted my editor’s vision for what the beat could be and joined on the team. I’ve been with the health and science team ever since.

Zibby: Wow. I feel like that must be super exciting because things are constantly changing, and sometimes in diametrically opposite places. I feel like even everything from weight loss trends to — everything keeps going through all these different cycles. It must just be so interesting to keep up and make sure you’re always reporting facts when the facts and the conclusions seems to shift.

Amy: One of the challenges for science is it’s hard sometimes to know what to do, what the data says because so much about science involves building on the results of one experiment and then creating another one. Then you have to replicate the work. Then you can’t be sure. That is one of the themes, also, in We the Scientists. One of the challenges and one reason why I’ve spent so many years following the patients and the families in this book is that even when you run a clinical trial, even when you do everything right and you gather the data, sometimes either the scientists or the Food and Drug Administration is still not sure if something is working. That’s what happened, by the way, in this trial that I describe in the book. They overcame all these obstacles. They found a promising therapy. They created a trial. Then the trial results don’t indicate that people who are getting the drug are doing any better than people who aren’t getting the drug. Of course, the FDA looks at that kind of result and says, we’re not going to be able to approve this drug. You have to show us that you benefit from taking this drug. One of the things I describe in the book is how because they had forged this partnership, because they had a collaboration, that setback, which can kill so many drug development efforts, didn’t stop this group of people from continuing. They are still trying to move this drug forward. Many of the patients still receive the drug through compassionate use, which is a way that the FDA allows people with life-threatening diseases to get access to drugs. They’re working on another effort to try potentially with another company to launch either another trial or to continue gathering data in the hopes that this drug will eventually get approved. So many drugs just fail for all the reasons that you notice, which is, it’s really hard to know if what you’re seeing is true or not.

Zibby: Oh, my gosh. How do the families feel about this book coming out?

Amy: I haven’t spoken to everyone. I’ve had a lot of great feedback from many people that I interviewed in the book and other people in the community. I hope that they’ll feel that the book honors them because I feel that the book honors them, honors their partnership and their effort. The people I’ve heard from have said that it meant a lot to them to be able to have their story known. When you write about a community — it’s a huge community. It’s a rare disease, but as a community, it’s a huge community. There were so many scientists involved. There were so many parents involved. In one chapter in the book, one of the parents calls the drug that they eventually moved forward into the trial, the community’s drug. That’s how they view it, the community’s drug, a community effort. You can’t tell a story about a community without trying to focus on certain people within the community so those who are reading the book can follow the narrative. I wish I could’ve mentioned every single name of every single person. I chose some families and some scientists. Really, I hope that they understand that the book is about a community that came together.

Zibby: Do you feel a sense of loss now that you’re not working with them? I feel like you’re so invested.

Amy: You spend a lot of time on a story, you definitely get invested. I don’t think I’ll ever stop reporting on this particular story in the sense that I feel committed to following them and their efforts and to see if the drug does eventually get approved. What I think I’m really committed to most of all is the idea that the model that they helped develop and advance, the one that they really believed in, I’d like to draw attention to the model because I believe that although I focused on a narrative about a specific group of people, this book is about all of us. All of us are patients one day, or we’re helping people that we love who are patients. We all have an investment in trying to figure out how to make the system work better and how to develop drugs more quickly and efficiently. I feel like the broader issue of the need to create a system that will work for all of us, we the scientists, that’s what my focus of my work will continue to be long past this book.

Zibby: What do you think your mom would think about all this?

Amy: She was always proud of whatever I did. She was my mom. She didn’t really require me to do very much for her to be excited. She just liked being with me. She was a big fan. I did write a story about her and the frustrations of trying to find an effective therapy for her for The Wall Street Journal. She was a very private person. She really didn’t want anyone to know about her story. When I came to her and said, “Mom, would you be okay about me writing about our family’s journey and your story in the newspaper?” she said to me, “I’m only going to say yes because I trust you with my story. I know you want to help other people. I want to help other people too.” I think that she would’ve liked that, that I’m still trying to use my family’s experience and her life and the values she imparted to me to try to help other people.

Zibby: That’s so nice. I know you’re a facts-based science reporter and all that. Do you believe that, on some level, your mom knows what’s happening?

Amy: I’m a very emotional person. Fortunately for me, I get to spend a lot of time with families and with people who are living with disease. Working with people is really the pleasure of being a journalist. I like to think that she might know. Yeah, of course. I don’t have any proof. I have to say that I tried to draw upon her experiences and my family’s experiences when I was writing the book. I tried to bring the compassion that so many people showed her and our family during her experiences with cancer. I tried to bring that same compassion to how I approach other families who are also wrestling with disease. None of us can ever know fully what someone else is going through. I do know what it’s like to sit on the other side of the table from a doctor who tells you that they’re sorry, but there’s nothing. You should go home and try to enjoy your time together. We’re doing our best, but the odds of this doing much are very low. I think also, most of us or many of us know what that feels like. That’s why I think so many of the people I encountered writing this book, the doctors, the scientists, the government officials, the FDA regulators, the parents, the families, the people who have NPC disease, there were so many acts of kindness, so many examples of compassion in the book and to each other and to help each other get through it. It meant so much to me as someone who had been a beneficiary also of acts of compassion and kindness by people who tried to help us.

Zibby: That’s amazing. That’s so nice. What next? What are you doing? Are you going to embark on another long-term project? You like the shorter stories? What are you thinking going forward?

Amy: I love writing books. I enjoyed writing this book. I’m a beat reporter at The Wall Street Journal. I love beat reporting. I believe in the value of immersing yourself in a topic. I think I’m on the greatest beat you could possibly ask for, health and science. It’s a beat that really intersects with everything, with money, with power, with compassion, with how we feel and our wellness. It’s something where it doesn’t matter what your background is or your beliefs are or what experiences you have. Health is something that touches — we learned this in the pandemic if we didn’t know it before. Health is something that touches all of us. I’m committed to continuing to write stories about health and science that will make a difference.

Zibby: Amazing. If you end up being able to prove anything about the afterlife, can’t wait to read that story. In the meantime, thanks for bringing all this really important information to all of us and trying to make systemic changes to make people’s lives better. What more can you ask for? It’s pretty awesome.

Amy: Thank you so much. It’s so great to talk to you about all of this.

Zibby: Thank you. You too. Keep it up.

Amy: You too, Zibby. Take care.

Zibby: Bye, Amy.

Amy: Bye.

WE THE SCIENTISTS: How a Daring Team of Parents and Doctors Forged a New Path for Medicine by Amy Dockser Marcus

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