Tag: research

  • As White House shifts focus toward AI and tech, U.S. science leaders defend role of the American university

    As White House shifts focus toward AI and tech, U.S. science leaders defend role of the American university

    BOSTON — This fall, the Massachusetts Institute of Technology, where 95% of students are STEM majors, enrolled nearly 500 fewer graduate students — a 20% drop compared to 2024. The reduction is a direct result of a $100 million cut in federal research awards to the institution in that same timespan, MIT President Sally Kornbluth said at the STAT Summit Wednesday in Boston. 

    Graduate students are largely supported with these grants, and “if we admit someone, we don’t want them to be partway through their Ph.D. and run out of funding, so we tend to be conservative on the front end,” Kornbluth said. “I wish I had a crystal ball and knew when things were going to turn around, but for now we’re just being cautious.”

    It’s not just MIT. Universities across the country have suspended or shrunk admissions to graduate programs in response to a tumultuous 18 months that have included unprecedented grant terminations, funding delays, and policy changes to how science is supported at the federal level. Even if the upcoming midterms usher in a power shift in Congress, it will do little to change the uncertainty faced by academic institutions regarding the reliability of the U.S. government as a science funding partner, particularly in light of a recently revealed plan for political appointees to gain more control over National Institutes of Health grantmaking decisions.

    “We will go toe-to-toe with the best of them based on merit. If there are other elements that lead to being funded or not, we don’t have control of that and that concerns me,” Kornbluth said. “I think we just have to do our very best science and feel like that in the end will win the day, and the country needs that.”

    Kornbluth was joined by Holden Thorp, editor-in-chief of the Science family of journals, to argue for the model of American research that for the last 80 years propelled the U.S. to global dominance in science, technology, and medicine. First articulated by Vannevar Bush in his 1945 report “The Endless Frontier,” it involved sustained public funding of science at research universities and federal laboratories and a commitment to open inquiry.

    While that model was showing signs of stagnation before the second Trump presidency, the last 18 months have shredded the social compact at its foundation. In its place, the administration has proposed a new blueprint for American innovation — one that is focused more narrowly on national priorities and a smaller role for the federal government in science funding. 

    At the White House’s Science: A New Golden Age summit last week, the Trump administration announced $6 billion in new national research investments, including support for the buildout of advanced computing facilities and autonomous laboratories, the development of nuclear powered spacecraft and next-generation quantum computers, and the creation of accelerated training programs in artificial intelligence and engineering. White House Office of Science and Technology Policy Director Michael Kratsios described the funding announcement as “the most transformative day for the American scientific enterprise since the reshaping of the federal research ecosystem following World War II.” 

    The investments mark the administration’s first steps to implement its vision for advancing U.S. innovation. Laid out in an OSTP report over the summer, it calls for a dramatic shift of hundreds of billions of research dollars from universities to industry — primarily AI and technology companies. 

    While there are roles for commercial entities as well as national labs to play in propelling science and innovation, Kornbluth said, what has very uniquely flourished at universities is the very basic end of the pipeline — the unpressured curiosity-driven research that has led to revolutionary discoveries including CRISPR gene editing, microRNA, and this year’s Nobel-winning work underlying the exploding field of optogenetics. “I worry that all of these moves, while they’ll have short term gains, are going to result in a serious drought in the pipeline … for us maybe five, 10 years down the line, but certainly for our children and their children’s children,” Kornbluth said. 

    It’s too soon to see the effects of these shifting priorities in research output indicators like top-tier journal publications, Thorp said. But he doubts the Trump administration’s push will do much to reverse the ongoing trend of the U.S. falling further behind its geopolitical rivals. 

    Even before the last 18 months of disruptions and delays to government investment, the U.S. was on its way to losing its place of prominence as the world’s largest public funder of science. Forecasts have put China on track to surpass the U.S. on research spending in the next two to three years. On the ground, that means buildings going up, more people going to graduate school, and a tangible sense of momentum. 

    “The atmosphere is just so much more pro-science there than it is here,” Thorp said, recounting a recent trip to China in which he sat down with presidents of several universities. 

    It’s getting results. Nine out of the top 10 academic research institutions are now in China, according to the latest data from the Nature Index, with Harvard University as the lone U.S. representative. A decade ago, American universities held half of the top 10 spots. 

    “Submissions from China continue to increase, not just in number but in quality,” Thorp said of the Science family of journals. To the point that, Science Advances, an open-access journal that is slightly less competitive than the flagship, is now publishing an equal number of papers from the U.S. and China. He expects the same to be true for Science within the next four to five years. “That’s the competitive effect that I don’t think the administration has very good answers for,” Thorp said. “At least when I write to them and ask them if they want to comment about it in my column, they never respond to me.”

    How these dueling visions of research in America play out will in some ways be shaped by the outcomes of the next few elections. But science happens on a longer time scale, and both Thorp and Kornbluth pointed to the need of scientists and academics everywhere, regardless of who holds power in Washington, to confront the underlying political reality of rising populism and cratering trust in institutions. 

    Kornbluth highlighted a new initiative launched this month — MIT for America — which sends students and alumni out across the country to help under-resourced high schools teach math, computer science, and other STEM classes often required for admission into the kinds of college programs required to move ahead in the increasingly AI-centric U.S. economy.  “We want to both meet people where they are, give their kids the opportunity, and also make them understand us as people who care about their communities,” she said.

    Thorp urged universities to follow MIT’s lead on getting rid of legacy admissions policies that give children of alumni an edge (which the university did decades ago). “It’s a huge part of why people are suspicious of higher education,” Thorp said. But he acknowledged that there’s only so much that institutions of all types can do to reverse the tide of populism, which has been growing for close to a century. 

    “My view is we have to accept the fact of what modernity has given us,” Thorp said. “Some people come to me as the editor of Science and think I have a big platform, and I’m sure they go to STAT and think STAT has a big platform, but compared to Mr. Beast, we’re off by three orders of magnitude. I can put all the cool videos and explainers and everything I want to on the Science social media accounts, which are the largest in academic publishing, and it’s not going to move the needle.”

  • STAT+: FDA adcomm members’ ties to industry under scrutiny

    STAT+: FDA adcomm members’ ties to industry under scrutiny

    Elaine’s stories explore the boom in new obesity treatments, new drug payment models, the ways companies are affected by FDA changes, and the emerging psychedelics drug sector. She co-writes The Readout newsletter, and co-hosts STAT’s weekly biotech podcast, The Readout Loud. You can reach Elaine on Signal at elaineywchen.70.

    Good morning. Yesterday, we got the devastating news that STAT’s infectious disease correspondent, Helen Branswell, passed away. She was a reporter I always looked up to and was also an incredibly generous colleague. Please take the time to read this tribute to her.

    The need-to-know this morning

    • Liquidia and United Therapeutics reached a settlement agreement that resolves their ongoing patent litigation over Liquidia’s treatment for pulmonary arterial hypertension. 

    Researchers in Pittsburgh are launching a novel type of GLP-1 trial — testing not only if the drug will help people with addiction, but also if it will help reduce recidivism while improving other employment, health, and housing outcomes.

    The Center for Addiction Science, Policy and Research (CASPR), a non-profit organizing the effort, and the Allegheny County Department of Human Services are planning to launch the trial in early 2027. They will enroll up to 1,200 adults with alcohol use disorder, opioid use disorder, or both, who are also at high risk of criminal-justice involvement. Half will be randomly assigned to take Novo Nordisk’s Wegovy, according to a press release issued yesterday.

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  • STAT+: Life Biosciences share glaucoma study updates for its cellular reprogramming drug

    STAT+: Life Biosciences share glaucoma study updates for its cellular reprogramming drug

    NIH funding uncertainty thwarts U.S. researchers, Argenx stumbles, and more biotech news

    Close up of an eye
    Adobe

    Jason is a general assignment reporter, with particular focus on genetic medicine and rare disease. Confidential tips can be sent on Signal at JasonMast.77.

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    This is the Readout. Please read it.

    And don’t miss us too much Monday, when the newsletter will be paused for the holiday. We’ll be back in your inbox Tuesday.

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    1. Meghana Keshavan covers biotech and contributes to The Readout newsletter.

    2. General Assignment Reporter

      Jason is a general assignment reporter, with particular focus on genetic medicine and rare disease. Confidential tips can be sent on Signal at JasonMast.77.

  • The fall of Roe dramatically changed abortion access. This researcher used Reddit to capture how

    The fall of Roe dramatically changed abortion access. This researcher used Reddit to capture how

    Betsy Pleasants was studying one historic disruption to abortion access when a more seismic one emerged.

    In spring 2022, as a graduate student at UC Berkeley School of Public Health, Pleasants was researching the effects of the Covid-19 pandemic on abortion services when a leaked draft opinion indicated that the U.S. Supreme Court would overturn Roe v. Wade. The court finalized its decision that June, ending the nearly 50-year-old constitutional right to abortion and ushering in an era of rapid change and increased state restrictions.

    Working with her thesis committee, Pleasants, who is interested in expanding access to abortion and other reproductive health care, adapted her dissertation to study conversations on the social network Reddit. Her new focus was tracking emerging barriers to abortion access in real time and examining the role of online resources in connecting people with evidence-based health information and services.

    “When it became clear that Roe v. Wade was going to be overturned, we all agreed that we had to shift the project,” said Pleasants, now a postdoctoral research associate at the Center for Women’s Health Research at the University of North Carolina. The Supreme Court decision “is something that required a response, and this project was positioned to offer timely insights into what this policy change means in people’s lives.”

    Pleasants and her colleagues plumbed thousands of posts from the Reddit forum r/abortion using natural language processing (NLP) — a type of computational analysis used to identify patterns in human language — alongside a qualitative close reading of a subset of posts. Through this dual approach, grounded in safeguards for user privacy, Pleasants is defining a framework for using artificial intelligence tools without losing sight of the context, complexity, and people behind the data.

    “We are in a complex policy moment for reproductive health,” said Pleasants, who was recently named a STAT Wunderkind. This type of research “is an act of documenting in an effort to work towards evidence-based policy change over time, and to advocate for the change that’s needed to promote access to reproductive health care.”

    Findings by Pleasants and her colleagues reaffirmed well-established barriers to access, including high costs and limited appointment availability, while also revealing lesser-known challenges, such as delays in receiving abortion medications by mail, low credibility of online ordering platforms, and concerns about the legal risks of seeking abortion or related medical care.

    “Embracing this opportunity and new methods was visionary,” said Ushma Upadhyay, professor in the Department of Obstetrics, Gynecology, & Reproductive Sciences at the University of California San Francisco, who mentored Pleasants during her doctoral research. “At the time, I didn’t know of any other studies using NLP to study online discourse on abortion. Most researchers will just take a random sample of a manageable number that they can analyze, and so this approach was really great because Betsy was able to get a more complete picture.”

    For scientists like Pleasants, the value of NLP goes beyond making large-scale research more efficient. It offers a broader view of how people communicate and behave at a scale that would otherwise be difficult to capture.

    “Computational research is like a stepping stool over a tall fence that lets you look at the other side and see everything that’s around you,” said Danny Valdez, associate professor of public health at the Indiana School of Public Health-Bloomington, who has employed NLP to study vaccine misinformation, addiction and recovery, and cancer care. “NLP, machine learning, and, increasingly, AI methods give you a 1,000-foot overview of how people communicate when they need help. You can learn a lot about the pulse of the nation, which helps us understand and inform where future policy could be headed.”

    A new model for studying online discourse

    Growing up in North Carolina, Pleasants got her first window into how people access health information, sparking an interest that would eventually shape her career. Even in Durham, a liberal core city of North Carolina’s Research Triangle, she recalled receiving abstinence-only sex education from a gym teacher. The contrast between the information she received at school and at home made her aware of how unevenly health knowledge and resources could be distributed.

    “I saw peers and other people in the community navigating not having good sex education, not having access to desired health services, and stigma,” said Pleasants. “All of these things really shaped the capacity they had to make decisions for themselves about their reproductive health.”

    Those early observations would become a throughline in Pleasants’ career, from earning her undergraduate degree at the University of North Carolina to completing her master’s and doctorate at UC Berkeley, and ultimately returning to UNC for her postdoctoral research.

    Pleasants entered college with an eye on becoming a physician, but was instead drawn to medical anthropology and broader discussions of how history, politics, and culture have shaped, and continue to shape, the U.S. health care system.

    “A big question that came up for me was: ‘Do I want to work within that system or do I want to critically engage with it?’” said Pleasants. “So, I ended up on this trajectory of research with an interest in asking questions of how we offer health services, particularly for women, and seeking to understand how we can make that better for all people as much as possible.”

    Analyzing r/abortion while at Berkeley allowed Pleasants to capture perspectives often overlooked by conventional research methods. The platform’s relative anonymity gave people space to speak candidly about personal experiences they might hesitate to share elsewhere. And because most community members had not interacted with the health care system, their experiences would often be missed by practice-based analysis.

    “Following the reversal of Roe v. Wade, a lot of people were turning to Reddit for support,” said Upadhyay.

    Pleasants and her collaborators used NLP to analyze more than 7,000 posts from the months before, during, and after the leak of the Supreme Court’s draft decision, identifying 55 distinct topics. Human oversight was integral throughout, both to validate the computational approach and to guide deeper qualitative analysis. The team closely examined posts within the identified topics to understand not only what people were discussing, but how they were navigating their experiences.

    The research revealed a wide range of barriers to abortion care, from state bans, high costs, and long travel distances to stigma and lack of social support. It also surfaced emerging challenges, including delays receiving abortion medications by mail, uncertainty about the legitimacy of online services, and concerns about potential legal risks. The qualitative analysis provided further insight into the impacts of these barriers, including anxiety and isolation and, in some cases, the need to self-manage abortions.

    The emphasis on human supervision also extended to protecting the privacy of Reddit users. As NLP and AI tools become more widely integrated into public health research, their use has fueled broader discussion, both within the research community and among the public, about the ethical use of online data. Pleasants said the stakes are particularly high in reproductive health research.

    “We are studying issues that are, at minimum, sensitive and stigmatized, and in some cases even pose risks for prosecution,” she said. “This becomes even more complex when you’re working with publicly available data where people may not be aware that their posts could be used for research.”

    To protect Reddit users, the team employed a “heavy disguise” process, replacing potentially identifiable posts with composite quotations that combined and rephrased similar experiences. The researchers then tested these composites using online searches and plagiarism-detection tools to help ensure they could not be traced back to their original sources.

    Together, these practices have established a model for studying sensitive online discourse at scale.

    “As this technology keeps getting faster and faster and faster, you do have very talented and forward-thinking researchers like Betsy, who are slowing things down and looking at how to make this as rigorous and concrete as possible through a very firm qualitative lens,” said Valdez, who was not involved in Pleasants’ research, though both have received funding from the Society of Family Planning. “We need work like Betsy’s that’s already acknowledged the difficulties of social media mining … and to show very high regard for the content that people are leaving on social media.”

    Today, Pleasants said her research, which has now expanded to include contraception and misinformation online, has taken on greater urgency as shifting policies increasingly constrain access to reproductive health care. She noted that researchers, advocates, and clinicians in the field have long worked amid scrutiny and uncertainty.

    “The importance of the research doesn’t lessen just because policies make the work harder. Rather, the opposite seems true right now,” she said.

  • STAT+: Roche goes beyond licensing in deal with Chinese biotech

    STAT+: Roche goes beyond licensing in deal with Chinese biotech

    DOJ signals it will start scrutinizing drugmakers, PhrMA sues Trump administration, and more biotech news

    gantenerumab Alzheimer's disease Roche
    Sebastien Bozon/AFP via Getty Images

    Elaine’s stories explore the boom in new obesity treatments, new drug payment models, the ways companies are affected by FDA changes, and the emerging psychedelics drug sector. She co-writes The Readout newsletter, and co-hosts STAT’s weekly biotech podcast, The Readout Loud. You can reach Elaine on Signal at elaineywchen.70.

    Want to stay on top of the science and politics driving biotech today? Sign up to get our biotech newsletter in your inbox.

    Good morning. Let’s get straight to the news.

    The need-to-know this morning

    Roche goes beyond licensing in deal with Chinese biotech

    Over the past few years, pharma companies have flocked to China to license existing drug candidates. Roche is taking a new approach, partnering with Chinese startup Defand Therapeutics to discover new medicines.

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    1. National Biotech Reporter

      Elaine’s stories explore the boom in new obesity treatments, new drug payment models, the ways companies are affected by FDA changes, and the emerging psychedelics drug sector. She co-writes The Readout newsletter, and co-hosts STAT’s weekly biotech podcast, The Readout Loud. You can reach Elaine on Signal at elaineywchen.70.

  • Breast cancer can behave differently in older women. This physician is making more tailored treatments a reality

    Breast cancer can behave differently in older women. This physician is making more tailored treatments a reality

    Of all the factors that can increase a person’s risk of breast cancer, age is among the most significant. Almost half of people diagnosed with breast cancer are at least 65, and about 20% are 75 or older. Despite these rates, older women are frequently excluded from clinical trials; one study of breast cancer trials found that only 9% of participants were 65 or older, leaving physicians with a poorer understanding of how drugs behave in older adults and fewer guidelines with which to treat them.

    There’s increasing evidence that these patients could benefit from more tailored treatments. Neil Carleton, a cancer biologist and first-year resident physician at Brigham and Women’s Hospital, said that breast tumors can develop and grow differently in older patients than in younger ones because of hormone levels and inflammation. 

    “It can be a very heterogeneous disease,” Carleton said. But “historically, we’ve kind of treated all breast cancer the same,” he added — in a way that’s “age-agnostic.” 

    Carleton, who was recently named a STAT Wunderkind, wants to change that. After an M.D.-Ph.D. spent studying how clinicians can better treat tumors in older adults, he is now working to ensure that these patients receive care that is right for them, whether that means dialing back treatment or increasing it.

    “We typically think an older adult may be a little bit more frail” or less able to tolerate standard surgeries and therapies, he said. “But I don’t even think it’s about that. I think it’s more about appropriately treating.”

    Crafting the ‘nudge’

    As an undergraduate at Carnegie Mellon University, Carleton spent two summers working in a Johns Hopkins prostate cancer lab and was surprised to see surgeons making a point to attend the department’s journal club. On Wednesday mornings, as the urology group — basic scientists and physicians alike, in all stages of their careers — filed into the conference room to discuss scientific papers in the field, Carleton began to see how medicine and research were intertwined. 

    He’d been thinking about medical school since high school in Pittsburgh, when a formative biology teacher veered his lessons away from photosynthesis and toward anatomy, physiology, and new biotechnology — the kind of science that explained how human health really worked. The idea of a physician-scientist was completely new, and it felt like the right fit.

    “I knew you could be a doctor, and I knew you could do research. I had no idea that you could kind of do both,” Carleton said.

    When he started his M.D.-Ph.D. at the University of Pittsburgh in 2018, Carleton began working in the lab of Adrian Lee and Steffi Oesterreich, a husband-and-wife team of breast cancer biologists who were enthusiastic about new ideas and had a robust track record of translational research. Lee remembers the intensity with which Carleton wanted to be involved in that kind of work. “We can go on doing basic research forever, but we would like to eventually impact patient care,” Lee said. “And you could tell that he wanted to do that.” 

    At the time, there were reports from surgeons and in the scientific literature that older women with breast cancer were being overtreated. The three started talking about the patients that would come to launch Carleton’s career and a body of work in Lee and Oesterreich’s lab. In 2020, under the guidance of Lee, Oesterreich, and surgeon Priscilla McAuliffe, Carleton set out to quantify how many patients were receiving a surgery called a sentinel lymph node biopsy. 

    When removing a tumor from a patient’s breast, surgeons sometimes make an incision in the armpit and take a small amount of lymph node tissue to see if the cancer has spread there. Though professional society guidelines recommended against performing this biopsy on women 70 or older with early-stage cancer, other groups had reported that it was still being done.

    In data from the university medical center, Carleton found that about 65% of patients were still receiving biopsies. Rates actually continued to increase after the Society of Surgical Oncology adopted the guidelines in 2016, even though patients who received the biopsy were not more likely to survive or avoid recurrence. But “it can be very hard for surgeons to stop doing a surgery that’s kind of been ingrained in the paradigm for a while,” Carleton said.

    How, then, could he and his colleagues convince surgeons to reconsider? They designed a “nudge” within the electronic health record — a pop-up that would appear in a provider’s schedule for a qualifying patient that would remind them to consider omitting the biopsy. They implemented the nudge in eight clinics across the community and, in a clinical trial, tracked biopsy rates. The year after the nudge, they decreased by almost half.

    McAuliffe, now a breast cancer surgeon and clinical investigator at Emory University, said that surgeons encounter a lot of extraneous information in the electronic health record — but the nudge was simple. “I think that it reminded them without being a burden to them,” she said.

    One surgeon, when surveyed, said that the nudge “did exactly what its job was,” urging physicians to think about biology rather than reflexively recommending the biopsy. “I had to keep in mind that complete care doesn’t always equate with better care,” they said.

    The science behind a paradox

    While in the clinic, Carleton began studying a years-old question in the lab. Most breast cancer cases in older women are estrogen-receptor positive (ER+), meaning that tumor cells have receptors that bind to estrogen and trigger cancer growth. By 85, 91% of women with breast cancer have ER+ disease, even though postmenopausal women have lower levels of circulating estrogen. Carleton wanted to know how those tumors managed, seemingly paradoxically, to thrive.

    Looking at the gene expression in tissue from patients and healthy controls, Carleton and his colleagues found that the patients produced more of an enzyme involved in converting estrone — a form of estrogen that circulates in the bloodstream in postmenopausal women — to estradiol, its more potent version, within the tumor’s environment. 

    Scientists had known for 20 years that tumors made their own estrogen but didn’t know how it was done, Lee said. The enzyme Carleton identified was more than a mechanistic insight — it created a druggable target. In 3D models the lab made from patient cells, inhibiting the enzyme blocked estrogen conversion.

    Currently, doctors often treat postmenopausal women with ER+ tumors with inhibitors of a different enzyme. The drugs cause side effects like fatigue and joint pain, which lead many patients to stop treatment prematurely. Though the work is still early, Carleton hopes that drugs targeting the process they identified, administered through a gel or patch, could treat or even prevent breast cancer with fewer side effects. “I think it opens up possible therapeutic interventions,” he said.

    ‘Refining what we do’

    When looking through patient data early in his degree, Carleton noticed that some women ages 70 and older were forgoing surgery — not only the biopsy, but also a procedure to remove the main tumor in the breast. In this group, only 3% died of breast cancer after six years, and about 91% had their tumors controlled with primary endocrine therapy, which blocks estrogen receptors on ER+ breast cancer to prevent its growth. 

    If you could identify those patients for whom endocrine therapy might be enough treatment and monitor them, both the patient and their surgeon might be more comfortable with scaling back surgery, Lee said. “Precision medicine doesn’t mean doing more,” he said. “It means refining what we do, and sometimes refining what we mean we do means doing less.”

    While others might have missed the dozens of patients that hadn’t had any surgery among the thousands that did, Lee said, Carleton has a knack for noticing small details. And in September 2021, he uncovered a way to potentially track disease in patients who might not need surgery.

    Sitting in a talk at a local conference, Carleton learned about circulating tumor DNA (ctDNA), which cancer cells release into the bloodstream and can be measured to survey tumor growth. Natera, a molecular diagnostics company, had designed an assay that seemed perfect for older patients who’d declined surgery in favor of endocrine therapy: It required only a blood sample and could be done through a mobile blood-drawing service that came to their homes. Carleton and Lee approached Natera and, in the months afterward, designed a clinical trial to test whether they could use ctDNA to watch and identify patients at risk of progression.

    “It’s not an easy trial to recruit to — to say to someone, ‘Hey, you have breast cancer, but we’re not going to cut it out,’” Lee said. 

    But Carleton “has an amazing ability to kind of get the message across and encourage people,” Lee said, and put in hours with patients and physicians to earn their trust. Carleton was surprised by how many older adults were thrilled to join a trial that would guide not only their care, but the treatment of others going forward. “I think it was kind of one of the first times they were even approached to join a study,” he said.

    Over the next few years, the team studied patients who decided against surgery with Natera’s assay and ultrasound imaging. Patients with detectable ctDNA at six months were 30 times more likely to progress in their disease during the study, suggesting ctDNA was a marker of potential progression. And indeed, no patients with undetectable ctDNA before endocrine therapy progressed.

    The ‘closer’

    Today, as a first-year resident in Boston, Carleton spends most of his time treating patients. But he stays engaged with research: with the students in Lee and Oesterreich’s lab continuing to study the estrogen-converting enzyme, and with projects like designing a larger ctDNA trial. Lee and Oesterreich, with whom he still has regular check-ins, call him a “closer” — someone who takes on projects and sees them through to completion, synthesizing complex data from basic science and clinical practice into an understandable whole. “He has the complete package,” Lee said.

    Ultimately, Carleton hopes this work can help patients receive care without frequent trips to the clinic — turning breast cancer “into more of a chronic disease,” he said, where a patient might just take a daily pill and have regular imaging or ctDNA tests.

    Of course, Carleton said, not every patient will want that, and people with aggressive disease may need every intense treatment, test, and appointment. But better tools can help doctors identify: Who can be the patient they send home?

  • STAT+: In small trial, optogenetics restores some vision lost to retinitis pigmentosa

    STAT+: In small trial, optogenetics restores some vision lost to retinitis pigmentosa

    Nobel-recognized technology is being explored for safety and clinical potential

    Optogenetics involves genetically engineering specific brain cells to increase their sensitivity to light.CAROL AND MIKE WERNER/SCIENCE PHOTO LIBRARY

    Annalisa (Nalis) Merelli is focused on boys’ and men’s health: She looks into reproductive health, trending treatments, screening approaches, mental health, and everything in between.

    Retinitis pigmentosa is a genetic disorder that causes degeneration of the light-sensing cells in the retina. Generally diagnosed in childhood or early adolescence, patients gradually lose peripheral and night vision, often becoming legally blind by young adulthood. 

    With the exception of a subset of patients who present a specific genetic mutation and can be treated with gene therapy, there is no cure for the condition. Cases are addressed by measures to slow degeneration and preserve what is left of the vision.

    Optogenetics  — the technology just recognized with a Nobel Prize — could offer hope, according to a new paper published on Wednesday in the New England Journal of Medicine. 

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    1. Annalisa (Nalis) Merelli is focused on boys’ and men’s health: She looks into reproductive health, trending treatments, screening approaches, mental health, and everything in between.

  • STAT+: Forbion raises $2.6 billion, its largest fundraise to date

    STAT+: Forbion raises $2.6 billion, its largest fundraise to date

    Promising Duchenne therapies will test FDA flexibility, Doudna-founded Caribou shutting down, and more biotech news

    Small people stand on a giant hundred-dollar bill
    Adobe

    Elaine’s stories explore the boom in new obesity treatments, new drug payment models, the ways companies are affected by FDA changes, and the emerging psychedelics drug sector. She co-writes The Readout newsletter, and co-hosts STAT’s weekly biotech podcast, The Readout Loud. You can reach Elaine on Signal at elaineywchen.70.

    Want to stay on top of the science and politics driving biotech today? Sign up to get our biotech newsletter in your inbox.

    Good morning. Today, STAT released our 2026 Wunderkinds, a group of early-career researchers recognized for doing innovative work in health and medicine. I had the pleasure of speaking with one NIH researcher who has been figuring out how GLP-1 drugs work in the brain. But check out all of the profiles here!

    The need-to-know this morning

    • Cerevance said its experimental pill solengepras reduced Parkinson’s patients’ daily off-time, alongside other symptoms, in a Phase 3 trial. The privately held company, which will now talk to regulators about a potential approval, touts the pill as one of the first potential Parkinson’s drugs that does not act directly on dopamine, sparing patients certain side effects.
    • Henri B. Kagan and Kenso Soai were awarded the 2026 Nobel Prize in chemistry for discovering how to manipulate “mirror image” molecules, paving the way for manufacturing more precise and safer drugs.

    Doudna-founded Caribou is shutting down

    Caribou Biosciences is shutting down after failing to raise enough money to fund a late-stage clinical trial of its off-the-shelf CAR-T therapy for lymphoma.

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    1. National Biotech Reporter

      Elaine’s stories explore the boom in new obesity treatments, new drug payment models, the ways companies are affected by FDA changes, and the emerging psychedelics drug sector. She co-writes The Readout newsletter, and co-hosts STAT’s weekly biotech podcast, The Readout Loud. You can reach Elaine on Signal at elaineywchen.70.

  • 2026 Nobel Prize in chemistry awarded for ‘mirror image’ molecule research, with impact on pharmacology

    2026 Nobel Prize in chemistry awarded for ‘mirror image’ molecule research, with impact on pharmacology

    Henri B. Kagan and Kenso Soai were awarded the 2026 Nobel Prize in chemistry on Wednesday for discovering how to manipulate “mirror image” molecules, paving the way for manufacturing more precise and safer drugs. 

    Kagan, 95, of the Université Paris-Sud in France, and Soai, 76, of Tokyo University of Science in Japan, solved a chemical mystery: Some molecules exist in the world that are mirror images of one another. This is a phenomenon known as chirality, from the Greek word for hand (as hands are mirror images of one another). Amino acids, the building blocks of proteins, are among chiral molecules, and a living organism can only contain one of the versions. The other mirror molecule is found rarely elsewhere in nature. More than half of clinically employed drugs are chiral, so understanding how this imbalance of two mirrored molecules occurs had crucial relevance for pharmacology.

    As the Nobel Committee puts it, imagine needing to make copies of a set of keys, but when you try to make them, you get both the key that correctly fits the lock, and a mirror image of it. It’s very hard to distinguish which is which, so you risk trying the wrong key into the lock. 

    The stakes are much higher if that key is a drug: when chemists develop new drugs, they often end up with some of their chiral versions, too. Sometimes, the mirror molecule does something completely different from what it was originally intended to — take novrad, a cough medicine, and darvon, a painkiller, which are chiral molecules (hence their mirror names, too). Other times the mirror molecule can cause serious side effects. 

    Still, drugs are typically sold as so-called “racemates” containing an equal mixture of both molecules, and prior to Kagan and Soai ,it was pretty much the only option, as chemists had not found a way to produce a molecule without its chiral version in equal parts. 

    That changed with the scientists’ “discovery of non-linear effects and autocatalysis in asymmetric organic synthesis,” as the Nobel Committee described it. In 1986, Kagan found a way to manipulate chemical reactions so that they produced more of one type of mirror images. Seventeen years later, Soai was finally able to develop an experiment that created only one of the two variants, creating a chiral molecule out of a non-chiral combination of molecules for the first time “since the dawn of life.” 

    “This is probably the coolest experiment in organic chemistry,” said Peter Somfai, a member of the Nobel Committee for Chemistry, referencing the Soai reaction.

    From a pharmaceutical point of view, this opened the door to the creation of chiral drugs containing only the necessary version of the molecule, achieving greater precision and lower side effects. 

    “I’m very excited to receive this very nice news about the Nobel Prize,” said Soai, calling into the award ceremony at the Karolinska Institutet in Stockholm, Sweden, from his native Hiroshima, Japan. “Of course this is one of the most exciting days of my life. I am very glad to share the award with professor Kagan.” 

    There have been 202 award winners since the first chemistry prize was awarded in 1901, of which 8 have been women. However, only 200 individuals won the award, as both Frederick Sanger and Barry Sharpless have been awarded twice. The youngest recipient was 35, the oldest 97. 

    The award has been given 118 times: 63 times to a single individual, and has been shared among two winners 26 times, and among three (the maximum allowed by the Nobel statute) 29 times. 

    The winners will receive 12 million Swedish Kronor (SEK), or roughly $1.2 million.  

    The medicine and physics prizes were awarded earlier this week.

  • STAT+: AstraZeneca opens new Boston site 

    STAT+: AstraZeneca opens new Boston site 

    Utah pilots AI service to prescribe acne treatment, drug price disclosures are coming, and more biotech news

    HECTOR RETAMAL/AFP via Getty Images

    Elaine’s stories explore the boom in new obesity treatments, new drug payment models, the ways companies are affected by FDA changes, and the emerging psychedelics drug sector. She co-writes The Readout newsletter, and co-hosts STAT’s weekly biotech podcast, The Readout Loud. You can reach Elaine on Signal at elaineywchen.70.

    Allison covers what hot startups are getting up to, and who’s financing them. She is also co-host of the weekly biotech podcast, “The Readout Loud.” You can reach Allison on Signal at AllisonDeAngelis.01.

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    Good morning. From DC to Boston to Utah, we’ve got news from all over today.

    The need-to-know this morning

    • BridgeBio said the FDA granted priority review to its oral treatment for children with achondroplasia, a common form of dwarfism. An approval decision on the drug, called infigratinib, is expected by Feb. 4, 2027. 
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    Trump admin says drug price disclosures are coming

    Trump administration officials said yesterday they will start developing a system for health insurers to disclose prescription drug prices, and a “hopeful” final plan will come out by next May.

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    1. National Biotech Reporter

      Elaine’s stories explore the boom in new obesity treatments, new drug payment models, the ways companies are affected by FDA changes, and the emerging psychedelics drug sector. She co-writes The Readout newsletter, and co-hosts STAT’s weekly biotech podcast, The Readout Loud. You can reach Elaine on Signal at elaineywchen.70.

    2. Biotech Startups and Venture Capital Reporter

      Allison covers what hot startups are getting up to, and who’s financing them. She is also co-host of the weekly biotech podcast, “The Readout Loud.” You can reach Allison on Signal at AllisonDeAngelis.01.