415: Mistrust in science and a juicy gene-editing lawsuit

0:06Welcome to The Readout Loud, a weekly biotech podcast from STAT. I'm Allison DeAngelis. I'm Adam Feuerstein. And I'm Bilene Chen.

0:17Today, why have so many Americans lost trust in science? And how have our policy leaders fueled that mistrust? We were told for three years during COVID, trust the experts, but that is not science. Science is eternal skepticism towards all orthodoxies, understanding that people in authority lie. We speak to our new Truth in Science reporter about the impact of America's growing skepticism of science. We're also going to chat about a gene therapy for Huntington's disease, a juicy lawsuit filed by a U.S. gene editing company against a Chinese competitor, and a shift in the way pharma companies are talking about their GLP-1 products.

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2:10I don't know. Maybe. Like the seasons have changed, so has Adam Feuerstein. All right. We have an interesting update this week from a company that we've talked about quite a bit this year, Unicure, and its gene therapy for Huntington's disease. Talk to us about what we found out today. Yeah. So let me start with some helpful background. Unicure is a biotech company. They're developing a one-time gene therapy for patients with Huntington's disease. That's an inherited and fatal neurodegenerative condition. You know, that work has been pretty hard, but almost exactly one year ago, Unicure delivered what looked like a breakthrough.

2:50The delivery of a high dose of estrogen therapy into the brains of 12 patients slowed the progression of Huntington's by 75 % compared to patients from a natural history study. The analysis was conducted with three years of follow-up. The benefit of that gene therapy was both clinically meaningful and statistically significant. And, you know, that was a big deal in the Huntington's community, given the dearth of effective treatments. So now let me touch on this week's news. Unicure reported the results of a follow-up analysis from the same study, the same 12 patients, conducted after four years.

3:30This time, the gene therapy slowed disease progression by 44%, and the benefit was no longer STAT-SIG. So it seems like the efficacy of the gene therapy is waning with another year of follow-up. Adam, does this raise concerns about the therapy's durability? Elaine, it does. I think that people are a little bit concerned about the durability. We've seen this with other gene therapies where over time, the efficacy of the gene therapies wanes. Now, even at four years, 44%, that's still a very meaningful slowing of disease progression for a disease like Huntington's where you see patients progress three, four years, and often patients die at that time point.

4:19So I think from that perspective, when you step back, this is still a very meaningful benefit comparing it to the patients that are being followed in this natural history study. I want to understand in the context of everything that we've been talking about with Unicure, what this data means. Because the big story with Unicure, the thing that we have been going back and forth on, is whether the FDA would be flexible in considering whether to approve this drug and what that meant for other drug developers, for other rare disease patients, and for the Huntington's disease patients. Does this data this week provide any greater context?

4:59Where does this leave us? Alice, that's a great point, a great question. And you remember a year ago when we saw these incredible three-year results, we thought, like everybody, oh, this is a slam dunk. This is going to get approved. And then remember what happened. They tried to submit it to the FDA. The FDA just said, no, you cannot submit this study based on this comparison against a natural history. This was the FDA under Macari and Vinay Prasad. Obviously, we have new leadership at the FDA now. And the company and the FDA have now reached an agreement where, indeed, they will consider this gene therapy for approval based on the three-year data.

5:38So the data that was reported last year. And I spoke to Lauren Holder. She has Huntington's disease, a very early form of the disease, very early stage. She's also very involved in the Huntington's community. And I asked her, what do you think? Now, you see three-year data. You see four-year data. Has it changed her opinion? And she said, absolutely not. She says that these four-year data are as compelling as the three-year data. And, you know, for her and for a lot of patients with Huntington's, it's a race against time. She looks at this and says, you know, if these four-year data were the only data that I ever saw, I would still want to get this gene therapy.

6:19This is something that I would want for me. This is something that I would want for other patients with Huntington's. Okay, turning to another topic in the news this week, Allison, tell us about a juicy case that's got a lot of people in the biotech industry chatting this week. Yes, there is a very juicy lawsuit that has been filed recently by the gene editing company Beam Therapeutics, alleging that one of its former scientists used its intellectual property when founding a Chinese biotech company. That company, called Yoltec, made a licensing deal earlier this year with some venture capital firms, which actually led to the creation of a new U.S.

7:02company that's directly competing against Beam. This lawsuit comes at a time of escalating tensions between American biotech companies and their Chinese counterparts and speaks to fear in the startup community that scientists that are working with their companies will leave and go start companies in China or that Chinese companies will find a way to infringe on their intellectual property and really decimate their efforts. This case is interesting because one of the executives at Yuletec was working at Beam Therapeutics, and Beam has said in this lawsuit that they actually went into their electronic records and found out that she was going into their systems on weekends, late at night, looking at things that weren't even related to her role and were what Beam considers trade secrets, and then left Beam and immediately went and worked for this company that's now competing against it.

8:01Beam. So this is my favorite story of the week, Allison, because it just strikes to the heart of so much of what we've been talking about, you know, biotech innovation and competition against China and whether it's a fair level playing field. Here's a scientist, you know, she's working for Beam. She goes in the weekends and evening, allegedly, obviously this is all allegedly, absconds with this proprietary information and goes and forms her own company in China, which then gets the backing of U.S.-based venture capitalists to form a new U.S. company that is going to compete directly against Beam.

8:42To what extent Beam is able to prove that Yoltec directly stole its intellectual property, we'll find out. But it speaks to this fear that's emanating through the industry right now. And then lastly, let's talk about a story you wrote this week, Elaine, about this shift in the way that pharma companies are talking about their GLP-1 products. Yeah, so I've been reporting on GLP-1 drugs for several years now, and I've really noticed a shift in how the pharma companies, particularly Novo Nordisk, have been framing and talking about their drugs. I remember when I first started covering these drugs, the companies were running educational campaigns trying to convince people obesity is a disease.

9:27GLP-1 drugs are medically necessary. I remember Novo even telling me they don't like people calling GLP-1s weight loss drugs. because they wanted people to focus on the health outcomes and not on weight. And this was at a time when the companies were trying to get more insurance coverage, which was a difficult endeavor because historically, drugs that cause weight loss were viewed as serving cosmetic purposes, and the companies were trying to change that perspective. But now it seems like completely different language and framing. Novo explicitly calls its GLP-1 drugs weight loss drugs in its ads.

10:02It's running campaigns with the slogans like Live Lighter, Summer Glow Up. And this comes at a time when Novo is significantly changing its commercialization strategy. Because insurance coverage has stalled, they've increasingly turned to the direct-to-consumer channel where they're selling their drugs directly to patients. So instead of focusing on health outcomes, they're focusing a lot more on serving who they call customers and appealing to the way the drugs might make them feel or look. Elaine, why does it matter how the companies talk about these drugs? I think the reality is that the way that pharmaceutical companies market and talk about their drugs has a big impact on how medicine is practiced and how we view disease.

10:49You know, just going back to the idea of mental health conditions, there's a history where mental health conditions weren't necessarily recognized as real conditions until we had medications come out to treat them. And I think obesity is going through a similar journey with the emergence of medications that would help people recognize obesity as a disease because we're saying we have these medical products to treat obesity. But with this shift of focusing much more just on the weight loss itself, less so on the health outcomes, some doctors are concerned we're going back on efforts to treat obesity as a disease.

11:28And so on one hand, people would argue that plays a role in how society views obesity and how obesity is stigmatized. But it then also goes back to insurance coverage, because ultimately the argument that obesity is a disease contributes to the argument that insurers need to cover these drugs, that these are not discretionary drugs. If insurers stopped covering diabetes drugs, there would be uproar. But we don't have that same expectation when people stop covering obesity drugs. Elaine, is it potentially dangerous to have drugs that make people lose a significant amount of weight be advertised as summer glow-up products, feel lighter, something that is pitched to the broad population and not just people who are obese?

12:15Yeah, I think it encourages people to use the drugs even if they're not overweight or obese. And it focuses on the more cosmetic aspects of weight loss rather than the health aspects and puts the drugs in the same category as, say, hair loss products or these other products that we commonly can get over-the-counter. It ultimately seems like these drugs are headed towards the over-the-counter direction. And the issue is that these are such powerful drugs and we're going to get more powerful drugs that I'm not sure if it's safe to head in that direction. Thank you, Elaine, for filling us in.

13:09Since the COVID pandemic, America's trust in public health and science has eroded, especially when it comes to childhood vaccines. Here's testimony from Polly Tommy of Children's Health Defense at a Senate hearing on vaccine safety last year. These childhood vaccinations, the people of America are telling us they are not safe and effective. We are seriously concerned at Children's Health Defense about the disinformation and misinformation out there being told these vaccines are safety trials. The safety studies are so good, yet all we see is death and destruction over and over again. Much of this mistrust in science is now driving policy decisions in the Trump administration.

13:53For example, the federal recommendations for children's vaccinations have been dramatically reduced. We wanted to understand the impact of this increasing mistrust of science and why it's happening. We're joined today by Stats' new Trust in Science reporter, Nick Florco. Welcome back to the podcast. Glad to be back. We are glad to have you, Nick. You have returned, like the prodigal son, to take over a new reporting beat that Stat created called Trust in Science. Now, I have to say, Nick, when I posted about your new job on X, the replies to my posts were a mix of people mocking me and us for taking on this controversial issue.

14:35Many of them were angry and used bad words. there were also people applauding us. And I think that speaks to the divisiveness that you'll have to address. So maybe tell us a little bit in your own words, what exactly is the trust in science beat? And why are you doing this now? Well, I think readers are going to see that the trust in science beat is frankly, a lot of different things. Trust in science touches everything we cover at STAT. One day I might be covering vaccines. The next might be peptides. The next it could be sunscreen. The next it could be medical malpractice. You know, I've always consider myself an investigative reporter, so you're going to see investigations digging into people who benefit off of sowing distrust for sure.

15:14But also a real goal of this job is for me to connect with people who do distrust science, because I think that's how we actually figure out why people distrust science and how to fix it. And I'm under no illusion that that's going to be an easy job, but I think somebody's got to do it. Nick, you wrote a deeply personal essay, and you talked about how you wanted to approach this new trust in science role. I just want to read something that you wrote. Far too much coverage of declining trust in science is predicated on the assumption that skeptics are just a small minority of ill-informed people.

15:50But we may be asking the wrong question. The more illuminating question isn't whether people trust scientists, but rather what happens when scientific knowledge collides with emotions like fear, confusion and frustration. Elaborate on that for us. One of the things I think the scientific literature gets wrong about this topic is they often just ask folks, do you trust scientists? And inevitably, a large percentage of people say yes, but I think that's the wrong question. And that's really where my own experience comes in. I trust scientists. I think their heart is very much in the right place, but that doesn't mean And I always make scientific decisions.

16:29And if I'm personally willing to try a drug or a supplement, despite there not being good data that it works, that's a form of distrust in science, too. And I think that's one that a lot more folks can connect with. It really comes less from not believing science and more from just these other emotions that we often don't talk about. And I think that's what's motivating a lot of folks. And when it comes to parents, I mean, they've been told to be scared of basically everything. And even if they don't think that there's a real cabal of scientists aiming to hurt them, it's reasonable to assume that some would take extraordinary measures to try to protect their child from everything they can.

17:08I think those are the types of stories we really need to tell because I think that tells us a lot more about what leads people down this path as opposed to just saying, do people trust the scientists? I think that's just like three levels above where we need to be. You know, measles seems like a very real and serious consequence of this mistrust in science, which then leads to a mistrust of public health policy. I wonder if that's how you see it. And are there other real tangible examples that you think come to mind that galvanize what you want to do with this beat? I mean, measles is absolutely a symptom of our declining trust in science.

17:46Consider the fact that Andrew Wakefield's discredited study tying measles vaccines to autism was retracted in 2010. But polls keep showing that that belief actually keeps going up over the last few years. So clearly there's a problem there. But in terms of other examples, one that I'm fascinated by is peptides. Because when we think of people who are distrustful of science or distrustful of vaccines, the thought that somebody would inject a experimental compound into their body for benefit seems kind of crazy. But there's clearly something going on there where folks see those types of products as trustworthy and they see traditional pharmaceuticals as not.

18:27And those are the types of things that I want to untangle to try to unravel how people justify these things in their heads and what that means for public health and for the industry. Now, talking about vaccinations and this rising distrust in the measles vaccine, what does it say about the state of things that you have a continued following for Andrew Wakefield's discredited study and now Robert F. Kennedy Jr. as the head of Health and Human Services? Does it legitimize the mistrust of science? Yeah, I mean, I think it absolutely does. I mean, Andrew Wakefield, the British doctor who published the now retracted study in The Lancet claiming that the measles vaccine causes autism.

19:10I mean, Dr. Wakefield, I shouldn't call him a doctor at this point because he's lost his medical license. Andrew Wakefield was sharing the stage with Robert F. Kennedy Jr. at a recent Children's Health Defense Conference in Washington, D.C. And by the way, he was treated like a celebrity there. So when you have a disgraced doctor sharing a stage with the Secretary of Health and Human Services, it absolutely legitimizes these concerns, at least to a subset of people. Nick, you recently spoke to a sociologist who studies families and the immunization decisions that they make for their children. What did you learn from that conversation?

19:46That sociologist, Dr. Jennifer Reich, her take is really that parents who reject vaccines are actually acting logically based on the parenting advice that they've been given. I mean, basically, parents are told that they are the protectors of their children. They know what is right for their families. They're told to do their research on X number of products. And so parents who reject vaccines have internalized this to mean, well, I need to be skeptical of vaccines, too. And this is coupled with the fact that the messages from the modern healthcare system are essentially, you can prevent disease.

20:19You can eat well. You can exercise. You can do X, Y, and Z to essentially make your body more resilient if you get sick. And so some parents are of the mistaken belief that if they feed their kids well, if they raise a healthy kid and an infectious disease should strike, that they won't be seriously harmed anyway unless they don't need the vaccine. Now, there's real issues with that logic, but it does make sense how parents would get there based on the information that they've been fed. Nick, I want to touch a bit further on that conversation with Dr. Jennifer Reich, because I thought it was very interesting that she pointed out that a lot of people experiencing vaccine hesitancy might actually be experiencing it as a logical outgrowth of a health care system that's really pushing Americans into being savvy consumers and very active decision makers in their health.

21:07that's also at a time where pharmaceutical companies are treating patients like customers. Is this a double-edged sword? Should the pharmaceutical industry and the healthcare system think twice about its messaging? Yeah, I mean, it's a tension that I've been thinking about a lot as well. I mean, as a reporter, obviously, I really believe in the importance of a well-informed public. I want people to do their own research. And generally, I think people taking care of their own health is a great thing. I think the issue is that people are often being oversold with how much they can control. And I think part of what I want to do here is untangle how real-world data gets turned into this narrative and who benefits from it.

21:52I mean, if we're thinking about should the pharmaceutical industry be talking about people as customers, I don't necessarily think that is a bad thing. I just think that they need to not oversell what patients can control. Returning to that point about people doing their own research and making their own health decisions, kind of empowering themselves in that way, you know, obviously people go online. There's a lot of different places to get information, but there's a lot of places now where misinformation is rife. It's all over the internet. And I wonder how much of this job covering trust and science do you think is going to be about correcting or debunking misinformation?

22:34And is that even possible these days? I'm still thinking through how much we can actually correct misinformation. There's some really interesting studies out there that show that when you present parents with scary images of kids who have been infected with measles and say, get your kid vaccinated, it pushes some of them further away towards vaccination. To me, that signals that maybe the best thing to do is not to just be the person that is fact-checking everything RFK says or everything someone else in this alternative health space says. I do think there's a place for it, but I don't see it as my sole remit.

23:14And really, I think there's more value in figuring out how that misinformation evolves and who is behind it. Because I do think that if you can point out how somebody is benefiting from the information that they are feeding you that might be misinformation, that might have more of an impact than just telling them the information is wrong. That's a really interesting point, Nick, because that's a conversation that we've been having within Stats Newsroom, that how much should we be reiterating the evidence around vaccine safety or some of the well-established data around different products when that is coming up in the news and being questioned by some of the people in power.

23:54And I think there's no good answer. I don't know that there is really a set way of doing these things. Yeah. And I mean, to further complicate the conversation and to risk maybe contradicting myself, I do also think on the other side, there is a sense among folks who feel like they've been harmed by vaccines or by other medical products that those folks are just not listened to whatsoever. And I think we need to be figuring out a more intentional way to talk about those folks and talk to those folks. Because I think we need to have a realization that no medical product is safe all of the time.

24:35People do get harmed by medical products. And when they get harmed by medical products or someone in their family or their friends gets harmed, that is of course going to create skepticism. And I think we've, as media overall, have stayed away from a lot of those stories because you don't want to just be platforming misinformation and making people fearful. But at the same time, what we're doing is clearly not working. I mean, at this Children's Health Defense Conference, it could have been a conference about the media. Every speaker was talking about how the media can't be trusted. They won't pay attention to us being harmed by COVID vaccines.

25:11They're trying to cover it up. And I wanted to stand up and yell, no, we're not covering it up. We just don't know how to cover it. And I think that's what I'm trying to figure out right now. Two questions, Nick, you know, that relate to that topic. I mean, you know, how important is it for you to be open-minded to all sides of this trust in science issue? And I guess getting back to the fact that, look, reporters and media companies that cover science are as mistrusted as the scientists themselves. So I wonder how that is impacting the way you do this job. Yeah, I mean, it's the number one thing I'm thinking about all the time.

25:47It's a hard balance, but a balance that I'm continually mindful of, of not being dismissive of people's concerns, but also stat as a science-based publication, we are going to report on the science and what is true and what is known at that moment. And so I'm constantly balancing those things in my head in terms of what stories I cover and how I write about those stories. But really, I mean, even if someone believes something that is not true that has happened to them or someone in their life, my number one goal is to not be dismissive of that person. I don't want to be dismissive of that person's lived experience.

26:25And frankly, if I am dismissive in any of my stories, I want readers to call me out on it because I think we need to be having an open dialogue about what that looks like. But yeah, it's top of mind. The media is definitely as distrusted as pharma and all these other folks in the healthcare supply chain, and we got to figure out a way to fix that. Let's circle back to COVID really quickly, because I think so many of us, so many pharmaceutical companies, so many of the organizations existing in the shadow of everything that happened during the pandemic. The pandemic very clearly was a tipping point for declining trust in science and particularly around public health policy.

27:06Is there a way back from that? I am honestly still trying to figure it out. Frankly, the reason why I'm somewhat pessimistic is when you look at the data around mistrusting COVID, it's a lot worse than people realize. A poll from the Kaiser Family Foundation has just been stuck in my head since I started this job. And it found that nearly 30 % of people surveyed said that it was either probably true or definitely true that COVID vaccines killed more people than the virus did itself. I mean, the fact that that's where we are tells me that things are way worse than people being pissed off about having to wear a mask or their kid not going to school.

27:44There's a fundamental distrust of the science behind the vaccine and what the vaccine did. And it's going to be hard to come back from that. We literally had a vaccine that basically took us back from the brink of a once-in-a-lifetime pandemic, and it was done under President Trump. So, I mean, where do we go from here if this is what has come out of that? Like, we actually had the clearest example of why a well-resourced, well-functioning drug industry is an important thing, and it led to backlash against that industry. How do we make sense of that? Yeah, Nick, Trump 1.0 did a lot of work in COVID in propelling vaccines for the virus forward.

28:30Trump 2.0, this administration feels like it is markedly different in its approach to science. So now we're doing this trust in science beat. What's changed? I do think that it is really the fact that the skepticism around science is now the official perspective of the federal government. This was all brewing during COVID. It was clearly a huge issue. But now RFK is not speaking as a member of Children's Health Defense. He's speaking as the Secretary of Health and Human Services. And so that's what makes this moment really different. And I also think that moment echoes through all of trust in science because the polling now shows that Democrats are saying that they don't trust the CDC and they don't trust government agencies, which during COVID, they were saying, trust the scientists, trust the scientists, trust the scientists.

29:23So there's all these reverberations now, like who distrusts science, who trusts science. It's just our whole world is turned upside down. So there's just so much to cover here. And while I want to explore ways to fix the issue, I think my job as a journalist is really to challenge people to think differently about this issue because what I think we're doing now clearly isn't working. It's a good time to be in this beat to be able to take on this topic at this moment. Nick, it is always a pleasure to have a conversation with you. There's so much to discuss, so I'm sure you're going to be back on the podcast soon.

29:59Thanks so much for joining us this week. Yeah, love to be back. Thanks so much.

30:07That does it for another episode of The Read Out Loud. Our podcast is produced by Rachel Gottbaum. Our senior producer is Delissa Ambrose. Our executive producer is Rick Burke. And our theme music is by Brian Joel. And we'd love to hear from you. Tell us what you like about this week's episode, what you didn't like, and whether you think weight loss drugs will ever be over the counter. You can do all that by sending us an email at readoutloud at statnews.com. And if you like what we do, leave a review or rating on Apple Podcasts or wherever you get your podcasts. See you next week, folks.