Amy Dockser Marcus, WE THE SCIENTISTS: How a Daring Team of Parents and Doctors Forged a New Path for Medicine
[0:02]Hi, I'm Zippy Owens, and you're listening to moms don't have time to read books.
[0:06]This 30-minute podcast features a new author interviewed by me every single day, 365 days
[0:12]a year for about 30 minutes.
[0:15]I am also the publisher for Zippy Books, which publishes 12 books a year in fiction and
[0:21]memoir.
[0:22]Our books are already out now.
[0:23]You can check it out on zippybooks.com.
[0:26]And we have a magazine called Zippy Mag where we have lots of wonderful essays and lifestyle
[0:31]features.
[0:32]That's at zippymag.com.
[0:33]We have classes.
[0:34]It's a B-Classist.com.
[0:36]And I recently opened a book store in LA called Zippy's bookshop at 1113 Montana Avenue
[0:43]at 11th Street in San Monica.
[0:45]I hope that you are able to enjoy some of our other offerings.
[0:49]But this here podcast is the basis of all of it and started in 2018.
[0:54]And no matter what I do, this is basically my favorite thing.
[0:57]Enjoy.
[1:03]Amy Marcus is the author of We the Scientists, how a daring team of parents and doctors
[1:07]forged a new path for medicine.
[1:10]Amy is a staff reporter for the Wall Street Journal.
[1:13]She won a Pulitzer Prize for Beat Reporting in 2005 for her series of stories about cancer
[1:17]survivors.
[1:18]Amy is also the author of The View from Nibo and Jerusalem, 1913, Books which grew out
[1:23]of her reporting in the Middle East as a correspondent for the Wall Street Journal.
[1:26]Welcome, Amy.
[1:27]Thanks so much for coming on Mom's Don't Have Time to Read Books to discuss We the Scientists.
[1:32]And I saw the cover behind you, hold it up in case I just, yes, how a daring here.
[1:36]I'm reading the subtitle.
[1:38]How a daring team of parents and doctors forged a new path for medicine.
[1:43]Yes.
[1:44]Okay.
[1:45]Tell us everything about this book and particularly how your own mother's diagnosis
[1:50]are inspired your hunt for answers to diagnosis and everything.
[1:54]Yeah.
[1:55]First, thank you for having me.
[1:57]I'm so honored to be here.
[1:59]We the scientist does have its origins in a personal story.
[2:05]My mother was diagnosed with a rare form of cancer, metastatic gallbladder cancer.
[2:11]I'm sorry.
[2:12]Thank you.
[2:13]Yeah.
[2:14]It was so difficult also because, you know, I'm a reporter and I cover health.
[2:21]And so I was used to searching for answers and doing research.
[2:25]And I interviewed so many doctors trying to find some effective therapy for her.
[2:30]And I learned that there were none in part because it's a very rare disease and pharmaceutical
[2:36]companies weren't pursuing drugs and doctors weren't able to study it.
[2:43]They didn't have a lot of patience and researchers didn't think of it as a disease they
[2:48]wanted to focus on.
[2:49]And unfortunately, my mother passed away after a little bit over two years with a disease.
[2:56]And it really sort of launched me into thinking about why is it so hard to advance science?
[3:04]Why aren't things moving faster?
[3:06]Why can't we get more drugs out there for more diseases?
[3:09]And I ended up taking some time off from my job as a Wall Street Journal science reporter,
[3:14]health and science reporter.
[3:16]And I traveled around the country and I met people who were trying to change the system.
[3:22]And one of the groups that I ended up meeting were a group of parents whose children
[3:26]also have a rare disease.
[3:28]It's called Neiman Pick Type C. It's a cholesterol disorder.
[3:33]And it's fatal.
[3:34]And they were trying to forge a very unusual collaboration where they would work as partners
[3:39]with scientists and researchers to try to find a drug to treat this disease.
[3:44]And I just started following them, following them and chronicling their story.
[3:48]And use that for 10 years.
[3:51]Yes, it was a very long process.
[3:54]This isn't like you did this for a semester or something like that.
[3:58]No, I mean, you know, I really got to know everyone.
[4:00]I like, you know, I met all their kids and I got to know the parents.
[4:04]And I got to know the scientists.
[4:06]I mean, one of the things that's unusual in rare disease communities is everyone knows
[4:10]each other.
[4:11]I mean, you know, they're relatively small communities.
[4:15]And, you know, there are annual kind of science and parents support group meetings.
[4:21]And then, you know, the parents were so welcoming they invited me into their homes.
[4:25]I got to meet the children and the scientists let me come into their labs.
[4:29]And so I, you know, and I kept going because they eventually did find a drug that they
[4:34]wanted to develop together and they were able to get it into a clinical trial.
[4:39]And they, they ended up talking to FDA and try, you know, really participating in this
[4:44]project in a way that, you know, was more unusual.
[4:49]And so I kind of just kept going with them.
[4:53]That's amazing.
[4:54]It's like just so incredible.
[4:56]It also like blows my mind that in today's day and age, like who is it up to who decides
[5:03]which labs are worth saving, which diseases, I mean, I understand it's a numbers game.
[5:08]But when is your loved one, right?
[5:12]Like why does that mean there shouldn't be a cure or are there just way too many?
[5:17]I mean, it sounds like a naive question, but is it just impossible like it?
[5:22]It's not a naive question.
[5:24]I think it's I think it's the central question, you know, like how do we decide where we're
[5:29]going to focus our attention and our resources and our some lives more valuable than others.
[5:34]I mean, obviously, I think all lives are valuable.
[5:37]So like, and I, and I think everyone deserves if not a treatment because no one can be guaranteed
[5:42]a treatment.
[5:43]I mean, I think one of the arguments of this book is a treatment is not guaranteed, but
[5:48]we should create a system where working together, we have a better chance to develop a treatment.
[5:54]And we can, and we can partner to develop a treatment because one of the reasons why I think
[5:58]it's hard to do this is because we don't use all of the expertise that's available to us.
[6:06]Like I argue that these parents, they know their children so well, they immerse themselves
[6:13]in this disease.
[6:14]They, I mean, many doctors never see a patient in their entire careers with an even
[6:19]you know, disease type say it's very, very rare.
[6:23]There's only around 200 or so cases in the US, maybe 500 or so cases, you know, internationally.
[6:31]And so if that's the case, then we have to use all the data and all the resources.
[6:36]And that means trying to help all of us participate in science.
[6:39]Yeah, I love how you call them like, what did you say like social scientists?
[6:43]Not social science.
[6:44]Yeah, it's some great buzzword for community activists.
[6:47]There's, there's citizen scientists.
[6:49]Yes, yes, yes.
[6:50]It is in scientists.
[6:51]Yes.
[6:52]There's a broad sort of movement of citizen science, it's been around for a while.
[6:58]And even the title of the book, we, the scientists, like I intentionally chose that as
[7:05]kind of echo to we, the people, you know, that we, that we all should consider ourselves
[7:12]not just the beneficiaries of science, but also potential contributors to science.
[7:17]I mean, I feel like that the pandemic, everybody was sort of, was trying to help in their
[7:22]own way.
[7:23]Right.
[7:24]That was like another, when we were all collectively fighting for the vaccine and everybody
[7:29]was doing whatever they could, you know, obviously that affected everybody.
[7:32]But, you know, I think we saw that, I think many people who hadn't been exposed to that
[7:36]level of involvement saw even the lay person sort of going through all the documents and
[7:40]like figuring out what to do and, and all of that, it's just how to scale that for every
[7:45]disease.
[7:46]Yeah.
[7:47]And I mentioned in the book, Long COVID, you know, the patients that struggle with symptoms
[7:52]for months or even years after a diagnosis with COVID-19.
[7:56]And I definitely think that all these groups that are emerging build on what has happened
[8:01]and come before.
[8:03]And I think that during the pandemic, we were much more attuned the idea that it's a novel
[8:08]disease and we don't know a lot about it.
[8:10]And even the scientists don't know a lot about it and the doctors don't know a lot about
[8:13]it.
[8:14]Let's try to gather data from the patients.
[8:18]And, you know, I personally, one of the arguments in the book is I don't think that that
[8:22]should be confined to COVID-19 pandemic.
[8:26]I think that's an approach that we should bring to every disease because if you're living
[8:31]with a disease or someone you love is, then you have data and expertise that's incredibly
[8:38]valuable and you have something to contribute that might help accelerate the search for
[8:44]therapies and treatments.
[8:46]Wow.
[8:47]I have a dear friend who's mother passed away from lung cancer and she was really involved
[8:51]for a long time.
[8:52]It's a lung cancer research foundation.
[8:53]So every year I would go to that lunch.
[8:55]And it always blew my mind that the funding for that is like such a small fraction of
[8:59]what it is for even breast cancer and that there is this, at least for that particular
[9:03]cancer, everybody, not everybody, but there is this assumption that it is almost caused
[9:08]it yourself.
[9:09]And therefore, it doesn't deserve the funding that a blameless cancer, you know, that's
[9:14]like what they would talk about is perhaps as to why the treatment is less yet.
[9:19]But again, like allocating resources and who gets to decide just seems crazy in a way.
[9:27]No, I mean, it's such an important point that you're raising because I mean, you know, fighting
[9:36]for attention, you know, to try to get people to focus on the things that matter to you.
[9:44]Sometimes do turn on factors that are outside of the realm of science or even sometimes
[9:50]numbers.
[9:51]I mean, you know, people do bring a lot of like their own value system into deciding
[9:56]these things and, you know, one of the things that I described.
[10:00]and we the scientists is that at the National Institutes of Health,
[10:04]you know, there was an institute there called NCATS
[10:08]where the director at the time, you know,
[10:12]one of his sort of visions was, you know,
[10:15]how can I collaborate with patients who have diseases
[10:18]that really don't, are often off the radar
[10:21]because there's not a lot of people,
[10:23]it was not a lot of interest or funding.
[10:25]And, you know, he got involved,
[10:28]he believed that the government should get involved.
[10:30]And I think that there is an argument to be made
[10:32]that some of the funding for biomedical research
[10:36]that comes via government institutions
[10:40]can be directed to create partnerships
[10:44]between scientists and those who wanna, you know,
[10:48]be citizen scientists, like one of the arguments
[10:50]in my book is, let's try to make partnerships here,
[10:54]including an ability to apply for research funding
[10:59]from the government just like scientists do all the time
[11:02]for the diseases they want to study.
[11:04]Totally, oh my gosh.
[11:07]Well, wait, what was telling me more about what it was like
[11:09]for you emotionally trying to be impartial observer
[11:14]of this group of families and a reporter,
[11:16]how could you not get swayed into the emotional component
[11:20]of the disease and the impact of it on the families?
[11:24]What was that like for you?
[11:26]Well, I definitely was emotional, I mean,
[11:29]I don't park my emotions at the door, that's for sure.
[11:32]I definitely try as a reporter to bring my passion
[11:37]and interest in whatever I'm covering, you know,
[11:40]to my approach.
[11:42]I spent a lot of time with everybody
[11:44]and I really came away from the feeling of, you know,
[11:47]often in narratives about race against time,
[11:52]there's a villain in a sense.
[11:54]There's a bad guy, there's, you know, sides.
[11:58]And one of the things I came away from this is,
[12:02]there are sides, there aren't villains.
[12:05]There's a system that's really not working well
[12:09]and there's so many obstacles that can just grind you down
[12:13]and seem like it makes it impossible for you to advance.
[12:17]And there's obstacles for the parents and for patients
[12:19]and there's obstacles also for scientists.
[12:22]They're always worried about like they have to,
[12:25]they have to, you know, look for their next grant.
[12:28]They need to publish a paper so that they can get tenure.
[12:31]They need to justify how they're spending their time
[12:34]and their resources.
[12:35]There's nothing wrong with that.
[12:37]Like we all justify how we spend our time and our resources.
[12:40]But I mean, I came away from it feeling like
[12:43]the people involved here are all good people
[12:47]and yet it's not working as well as it should.
[12:50]And why is that?
[12:51]And how could we make changes to make it better?
[12:54]And I really tried, that's the way I tried to come at it
[12:56]as, you know, not pitting people against one another,
[13:00]but rather trying to understand their viewpoint,
[13:04]how they ended up in the room that we were all gathered in
[13:08]and how their values informed the choices they were making
[13:12]and the arguments they were having.
[13:13]Interesting.
[13:15]Do you mind backing up and telling me how you became
[13:17]a Wall Street Journal health reporter
[13:20]and Pulitzer Prize finalists and all of that?
[13:22]Can you, like, how did you get started on everything?
[13:25]And like, what did you think you wanted to be
[13:27]when you were a kid?
[13:29]I always wanted to be a writer.
[13:31]I think I came home from school when I was eight years old
[13:33]and announced to my parents that was what I was gonna do.
[13:36]And I sort of never veered from that.
[13:37]I just, I loved writing.
[13:39]I always loved writing.
[13:40]I've been at the Wall Street Journal for many years.
[13:43]I started out as the sort of, like, lowest sort of
[13:47]entry-level job you can have.
[13:48]I was the news assistant for the legal reporters
[13:52]and I would go to the courthouse and pull their documents
[13:55]and I would bring them coffee and I would try to create charts,
[13:58]you know, that, like, illustrated their amazing reporting
[14:02]and just sort of worked my way up, you know,
[14:05]to become a reporter and I had spent many years
[14:10]as a foreign correspondent for the Wall Street Journal
[14:12]covering the Middle East and then, you know,
[14:14]got back to the States and was sort of looking around,
[14:18]you know, what should I cover next?
[14:19]What's of interest to me?
[14:22]And I tried education for a while, which I love.
[14:25]That was a great beat.
[14:26]But then there was this opening for a health reporter
[14:30]on a new section that the paper was launching
[14:33]and the editor was a good friend
[14:35]and he approached me and said, I think you love writing
[14:39]about health, it's all about life and death
[14:42]and society and, you know, ethical issues.
[14:46]I just think you'd love it.
[14:47]And I hadn't given it a lot of thought before,
[14:50]but I sort of trusted my editor's vision
[14:53]for what the beat could be and joined on the team
[14:56]and I've been with the Health and Science team, you know,
[15:00]ever since.
[15:01]Wow. I feel like that must be super exciting
[15:04]because things are constantly changing.
[15:06]I mean, and sometimes, like,
[15:08]you're diametrically opposite places.
[15:12]I mean, I feel like even everything from weight loss trends
[15:16]to, you know, there's like everything keeps going
[15:19]through all these different cycles
[15:20]and it must just be so interesting to sort of keep up
[15:23]and make sure you're always reporting facts
[15:25]when the facts and the conclusions seem to shift.
[15:30]I think one of the challenges for science is like,
[15:34]it's hard sometimes to know, you know,
[15:37]what to do, like what, you know,
[15:39]what the data says because so much about science involves
[15:43]building on the results of one experiment
[15:47]and then creating another one.
[15:48]And then you have to replicate the work
[15:50]and then you can't be sure.
[15:51]I mean, that is one of the themes also
[15:54]and we the scientists because one of the challenges
[15:57]and one reason why I've spent so many years following,
[16:01]you know, following the patients and the families
[16:03]in this book is that even when you run a clinical trial,
[16:08]even when you do everything right and you gather the data,
[16:10]sometimes either the scientists or the food
[16:14]and drug administration is still not sure
[16:18]if something is working and that's what happened
[16:21]by the way in this trial that I described in the book,
[16:24]which is that, you know, they overcame all these obstacles,
[16:28]they found a promising therapy, they created a trial,
[16:31]and then the trial results don't indicate
[16:36]that people who are getting the drug
[16:38]are doing any better than people who aren't getting the drug.
[16:41]And of course, the FDA looks at that kind of result
[16:44]and says, we're not gonna be able to approve this drug,
[16:47]you have to show us that you benefit
[16:50]from taking this drug.
[16:52]And, you know, one of the things I describe in the book
[16:54]is how because they had forged this partnership
[16:58]because they had a collaboration that setback,
[17:02]which can kill so many drug development efforts,
[17:07]you know, didn't stop this group of people from continuing.
[17:12]And they are still trying to move this drug forward.
[17:17]Many of the patients still receive the drug
[17:20]through compassionate use, which is a way
[17:23]that the FDA allows people with life-threatening diseases
[17:26]to get access to drugs.
[17:28]And they're working on another effort
[17:31]to try potentially with another company
[17:33]to launch either another trial
[17:35]or to continue gathering data
[17:37]in the hopes that this drug will eventually get approved.
[17:40]But so many drugs just fail for all the reasons,
[17:44]you know, that you notice, which is it's really hard
[17:47]to know if what you're seeing is true or not.
[17:50]Oh my gosh.
[17:51]So how do the families feel about this book coming out?
[17:54]You know, I haven't spoken to everyone.
[17:57]I've had a lot of great feedback from many people
[18:00]that I interviewed in the book and other people in the community.
[18:04]I hope that they'll feel that the book honors them
[18:08]because I feel that the book honors them,
[18:11]honors their partnership and their effort.
[18:14]The people I've heard from have said
[18:16]that it meant a lot to them
[18:18]to be able to have their story known.
[18:23]You know, when you write about a community,
[18:26]you know, it's a huge community.
[18:28]It's a rare disease, but as a community,
[18:30]it's a huge community.
[18:32]And there were so many scientists involved
[18:34]and there were so many parents involved.
[18:36]I mean, in one chapter in the book,
[18:38]one of the parents calls the drug
[18:40]that they eventually moved forward into the trial,
[18:43]the community's drug.
[18:44]And that's how they view it, the community's drug,
[18:46]a community effort.
[18:48]And so you can't tell a story about a community
[18:53]without sort of trying to focus on certain people
[18:57]within the community.
[18:58]So those who are reading a book
[19:00]can follow the narrative.
[19:01]And I wish I could have like mentioned every single name
[19:05]of every single person.
[19:06]I chose some families and some scientists,
[19:09]but really I hope that they understand
[19:11]that the book is about a community that came together.
[19:15]Do you feel like a sense of loss now that you're not
[19:19]working with them?
[19:21]Do you not?
[19:22]I feel like you're so invested.
[19:25]Well, you spend a lot of time on a story.
[19:27]You definitely get invested.
[19:30]I mean, I don't think I'll ever stop reporting
[19:33]on this particular story in the sense
[19:35]that I feel committed to following them in their efforts
[19:40]and to see if the drug does eventually get approved.
[19:43]But what I think I'm really committed to most of all
[19:47]is the idea that the model that they helped develop
[19:51]and advance, the one that they really believed in,
[19:54]I'd like to draw attention to the model
[19:57]because I believe
[20:00]that although I focused on a narrative
[20:03]about a specific group of people,
[20:05]this book is about all of us.
[20:08]All of us are patients one day
[20:11]or we're helping people that we love for patients.
[20:15]And so we all have an investment
[20:17]in trying to figure out how to make the system work better
[20:19]and how to develop drugs more quickly and efficiently.
[20:23]And so like I feel like the broader issue
[20:26]of the need to create a system
[20:28]that will work for all of us, we the scientists.
[20:31]That's what my focus of my work will continue
[20:35]to be long past this book.
[20:37]And what do you think your mom would think about all this?
[20:40]I mean, she was always proud of whatever I did, you know?
[20:44]She was my mom.
[20:45]She didn't really require me to do very much
[20:48]for her to be excited.
[20:49]She just liked being with me.
[20:50]So, but she was a big fan and she understood.
[20:54]I did write a story about her
[20:56]and the frustrations of trying to find an effective therapy
[20:59]for her for the Wall Street Journal.
[21:02]And she was a very private person.
[21:04]You know, she really didn't want anyone to know
[21:08]about her story.
[21:09]And when I came to her and said, mom,
[21:12]would you be okay about me writing about our family's journey
[21:14]and your story in the newspaper?
[21:17]She said to me, I'm only gonna say yes
[21:20]because I trust you with my story.
[21:23]And I know you wanna help other people
[21:24]and I wanna help other people too.
[21:26]So I think that she would have liked that,
[21:29]that I'm still trying to use my family's experience
[21:34]and her and her life and the values, I think,
[21:37]she imparted to me to try to help other people.
[21:41]Oh, it's so nice.
[21:42]I know you're a facts-based scientist,
[21:45]science reporter and all that.
[21:47]Do you believe that on some level your mom knows what's happening?
[21:52]I mean, no life, I'm a very emotional person.
[21:54]Yeah, I mean, I spent my,
[21:57]fortunately for me, I get to spend a lot of time
[21:59]with families and with, you know,
[22:01]people who are living with disease.
[22:03]And so you just working with people
[22:05]is really the pleasure of, I think, of being a journalist.
[22:08]But yeah, I like to think that she might know.
[22:11]Yeah, of course, I mean, I don't have any proof.
[22:13]But I have to say that I felt,
[22:17]I tried to drop on her experiences
[22:20]and my family's experiences.
[22:23]When I was writing the book,
[22:24]I tried to bring the compassion
[22:27]that so many people showed her and our family
[22:29]during her experiences with cancer.
[22:33]I tried to bring that same compassion,
[22:35]you know, to how I approach other families
[22:39]who are also wrestling with disease.
[22:41]I mean, none of us can ever know
[22:43]of fully what someone else is going through.
[22:46]But I do know what it's like to sit
[22:48]on the other side of the table from a doctor
[22:51]who tells you that, you know, they're sorry,
[22:55]but there's nothing.
[22:56]Or, you know, you should go home
[22:58]and try to enjoy your time together.
[23:00]Or, you know, we're doing our best,
[23:03]but we're, you know, the odds of this
[23:04]doing much are very low.
[23:05]Like, and I do, and I think also most of us,
[23:10]or in many of us, you know, know what that feels like.
[23:13]And so that's why I think so many of the people
[23:15]I encountered with this writing this book,
[23:18]the doctors, the scientists, the government officials,
[23:21]the FDA regulators, the parents, the families,
[23:25]that, you know, the people that have,
[23:26]who have NPC disease,
[23:29]there were such so many acts of kindness,
[23:32]so many examples of compassion in the book
[23:35]and to each other and to help each other get through it,
[23:40]that it meant so much to me,
[23:42]as someone who had been a beneficiary
[23:44]also of acts of compassion and kindness
[23:46]by people who tried to help us.
[23:48]That's amazing.
[23:50]That's so nice.
[23:51]So what next?
[23:53]What are you doing?
[23:54]What are you gonna embark on another long-term project?
[23:57]Are you like shorter stories?
[23:59]What are you thinking going forward?
[24:01]I love writing books and I enjoyed writing this book,
[24:03]but I'm also a, I'm a beat reporter at the Wall Street Journal
[24:07]and I love, I love beat reporting.
[24:09]I believe in the value of immersing yourself in a topic.
[24:14]And I think I'm on the greatest beat
[24:16]you could possibly ask for, health and science.
[24:19]I mean, it's a beat that really intersects with everything,
[24:24]you know, with money, with power, with compassion,
[24:28]you know, with how we feel and our wellness.
[24:32]And it's something where it doesn't matter
[24:36]sort of what your background is or your beliefs are
[24:40]or, you know, what experiences you have,
[24:43]health is something that touches,
[24:45]I mean, we learned this in the pandemic,
[24:46]if we didn't know it all before,
[24:48]if we didn't know it before,
[24:49]health is something that touches all of us.
[24:51]And so, you know, I'm committed to continuing to write stories
[24:57]on, you know, about health and science
[24:58]that will make a difference.
[25:00]Amazing.
[25:01]Well, if you end up being able to prove anything
[25:04]about the afterlife, you know,
[25:06]can't wait to hear me that story.
[25:08]And in the meantime, you know,
[25:09]thanks for bringing all this really important information
[25:12]to all of us and trying to make systemic changes
[25:15]to make people's lives better.
[25:16]I mean, what more can you ask for?
[25:17]It's pretty awesome.
[25:19]Thank you so much.
[25:19]It's so great to talk to you about all of this.
[25:22]Thank you, you too.
[25:23]All right, keep it up.
[25:25]And you choose me for that.
[25:27]Okay, bye, bye, bye.
[25:31]Bye.
[25:32]Thanks for listening to this episode of Moms.
[25:34]Don't have time to read books.
[25:36]Don't forget to follow me on Instagram,
[25:38]at Zibi Owens and at Moms.
[25:39]Don't have time to read books.
[25:41]Also sign up for my newsletter at Zibi Owens.com
[25:43]and sign up for my virtual book club
[25:45]and meet lots of authors on Zoom every other week.
[25:48]Thanks so much to Steve and Ryan,
[25:49]at Textures Sound for the Sound Editing.
[25:51]And thank you to Morning Moon Productions
[25:53]for providing this fantastic intro and out trim music.