United States – Page 27

May 18, 2026

Multiple Sclerosis Myelin Loss Revealed by Transcriptomic Analysis in Mice

More than one million people across the United States live with multiple sclerosis (MS), a disease that affects the brain, optic nerves, and spine. MS is characterized by overwhelming fatigue, muscle spasms, and vision problems, which can flare up and then subside over days, months, or even years. Studying the underlying damage to the nervous system is key to identifying new treatment paradigms for MS.

A new study published in Nature Communications titled, “A comparative transcriptomic analysis of mouse demyelination models and multiple sclerosis lesions,” compares two prevailing models, cuprizone (CPZ) and lysophosphatidylcholine (LPC), for the study of myelin loss and regeneration in an MS mouse model.

Katrina Adams, PhD, Gallagher Assistant Professor at University of Notre Dame, studies the role of the loss and regeneration of myelin on MS progression. As a fatty substance protects nerve cells, myelin envelopes the axons of the brain as they route the electrical signals that carry information throughout the nervous system. The damage and swelling that follow myelin loss in MS form distinct “lesions,” which vary in size, number and location in the nervous system.

“Our analysis of these two models of myelin loss and regeneration provides a road map based on robust scientific evidence that we hope will advance the study of MS and related diseases,” said Adams.

While both CPZ and LPC models degrade myelin, the timeline and localization of myelin loss varies. CPZ causes widespread loss of myelin over several weeks while LPC induces a lesion within days. This new research, which was funded by the National Multiple Sclerosis Society, points to specific scenarios in which one model is better suited, depending on which aspect of MS is under investigation.

“If you’re studying the myelin-producing cells and what’s happening to them in MS—are they stressed, dying or trying to repair?—CPZ is better, since the loss of myelin is more gradual,” Adams said. “For studying the immune cells that respond to the myelin loss, LPC may be better, since the immune response is more aggressive than in CPZ.”

The team also analyzed the resulting lesions from each preclinical model alongside data obtained from human MS tissue samples. Genetic maps of each type of tissue using single-cell RNA sequencing were constructed to examine the genetic changes that occurred in response to demyelination.

“By matching each model to features seen in diseased tissue from real patients, we can be sure that we’re targeting things that are actually causing disease in human patients,” Adams said. “There are so many potential paths to follow, so we want to make sure that the path chosen has direct relevance to MS patients.”

In addition to phenotypic differences, the genetic changes in diseased cells vary between the two models, an area of future exploration for the Adams research group.

Since MS flare-ups are primarily triggered by the immune system’s reaction to lesions, current clinical treatments focus on quelling this autoimmune response. The regeneration of lost myelin within MS lesions remains a promising yet unrealized drug target.

“The strategic use of these two preclinical models is essential for translating insights into therapies that might restore lost myelin,” Adams said. “We need to better understand the very process of demyelination in order to treat one of the root causes of this debilitating disorder.”

The post Multiple Sclerosis Myelin Loss Revealed by Transcriptomic Analysis in Mice appeared first on GEN – Genetic Engineering and Biotechnology News.

May 18, 2026

Reporter’s Notebook: The Day the Scientific Debate Died

When the news broke on May 5 that U.S. Food and Drug Administration (FDA) officials had blocked the publication of two major COVID-19 vaccine safety studies in 2025 after being accepted for publication in medical journals, many researchers saw more than a scientific dispute. They saw it as further evidence that America’s most powerful public health agencies were devolving into ideological warfare, institutional instability, and political distrust.

By the time FDA commissioner Martin Makary, MD, resigned a week later on May 12, 2026, there was a growing conflict among scientists, political appointees, public health officials, and outside activists not only over vaccines and public health policy but also over something even more fundamental: who gets to determine what constitutes legitimate science and whether scientific disagreement itself would still be permitted to occur in public.

A decade in the making

Makary’s departure came amid a broader transformation of the FDA and HHS from relatively stable, technocratic agencies into politicized institutions shaped by pandemic-era conflicts. Under Obama-era leaders Robert Califf, MD, and Margaret Hamburg, MD, the FDA emphasized regulatory continuity and evidence-based policymaking, while the Department of Health and Human Services (HHS) focused largely on healthcare administration and implementing the Affordable Care Act.

That changed during the first Trump administration and accelerated during COVID-19, when disputes over vaccines, masking, emergency authorizations, and therapeutics turned the FDA into a political flashpoint. Leadership turnover has increased, tensions between political appointees and career scientists have deepened, and public trust has fractured along ideological lines.

The Biden administration attempted to restore institutional stability by returning Califf to the FDA and appointing lawyer and politician Xavier Becerra, JD, to HHS, but the agencies remained mired in conflicts over pandemic policy and public health authority. Under HHS Secretary Robert F. Kennedy Jr. in the second Trump administration, those tensions intensified further through staffing and funding cuts, ideological battles over vaccines and food policy, and growing distrust inside federal health agencies.

Makary initially aligned with parts of the administration’s “Make America Healthy Again” (MAHA) agenda, particularly on food reform and criticism of segments of the pharmaceutical industry. But reports suggest he became caught between competing pressures from the White House, HHS leadership, industry groups, conservative activists, and public-health officials. He ultimately resigned amid disputes over vaping regulation, drug approvals, and broader public health policy during a sweeping restructuring of federal health agencies. Reports also indicated he was already at risk of removal and that his departure was not directly tied to controversy over the blocked COVID publication.

Instead, the move signals the White House’s continued support for Robert F. Kennedy Jr., “MAHA,” and a shift toward more centralized control over food-safety strategy, inspections, and outbreak response—changes that could affect how aggressively the FDA enforces nutrition standards and responds to contamination events. More broadly, the episode has done little to ease concerns among scientists and public-health experts that political considerations are increasingly shaping regulatory decisions and narrowing the space for independent scientific debate within federal health agencies.

Seeing double(think)

One of the two was posted online as a medRxiv preprint in 2025 by lead author Joann F. Gruber, PhD, and senior author Steven A. Anderson, PhD, and examined updated Covid-19 vaccines in adults over 65, the population most vulnerable to severe disease and death from the virus. The analysis drew on data from 7.6 million Medicare FFS beneficiaries who received a COVID-19 vaccination in 2023–2024—either the Pfizer-BioNTech (3.68 million) or Moderna (3.84 million) mRNA vaccine or the Novavax protein-based vaccine (30,000)—and found no new vaccine safety signals.

But before the study could move through peer review, publication was halted by the FDA, according to a spokesperson for the HHS. As reported by the New York Times, an HHS spokesperson said the studies were withdrawn “because the authors drew broad conclusions that were not supported by the underlying data. The FDA acted to protect the integrity of its scientific process and ensure that any work associated with the agency meets its high standards.”

Both Gruber, whose work with the FDA’s Center for Biologics Evaluation and Research (CBER) began in 2017, and Anderson, a veteran of CBER having joined in 2001, left the FDA at some point in 2025. It’s worth noting that Anderson’s team posted a second preprint on influenza vaccines that mirrored the COVID-19 vaccines study, using the same patient population, on the same day, which was also posted online as a medRxiv preprint on January 5, 2025, and was accepted in the peer-reviewed journal Vaccine on March 25, 2025, and made available online April 8, 2025.

On June 25, 2025, Makary and former CBER director Vinay Prasad, MD, PhD, in conjunction with manufacturers, added class safety warnings for myocarditis and pericarditis to COVID-19 mRNA vaccines’ prescribing information. The exact timing of when the accepted Gruber and Anderson study was pulled from publication has yet to be reported. That it occurred before June 2025 to prevent contradiction with Makary’s and Prasad’s safety update to the COVID-19 mRNA vaccine is entirely possible. Anderson’s team posted a second preprint on influenza vaccines that mirrored the COVID-19 vaccines study, using the same patient population, on the same day, which was also posted online as a medRxiv preprint on January 5, 2025, and was accepted in the peer-reviewed journal Vaccine on March 25, 2025, and made available online April 8, 2025.

Both Gruber, whose work with the FDA’s Center for Biologics Evaluation and Research (CBER) began in 2017, and Anderson, a veteran of CBER having joined in 2001, no longer work at the FDA. According to their LinkedIn profiles, Gruber left in June 2025 and Anderson in December 2024.

Gold-standard science

I spoke with several leading epidemiologists to assess whether there was substance to the HHS statement. All immediately noted the lack of specificity in the agency’s criticism, particularly the vague references to “gold-standard science.”

An epidemiologist with experience conducting vaccine safety studies, requesting anonymity, told Inside Precision Medicine, “If someone wants to criticize the study, there should either be a very clear articulation of what exactly they mean when they invoke terms like ‘gold-standard science’—specifically, which methods are acceptable, which are not, and why—or they should bring to the table the scientific credibility that would justify dismissing this kind of work outright. Frankly, neither of those things has happened. Broad, nonspecific attacks like these actually undermine the critique itself.”

The epidemiologists I spoke to emphasized that the study’s methods were not novel but reflected established approaches used in vaccine surveillance and prior scientific work, including self-controlled case series and cohort studies. A Harvard University researcher familiar with the study said the framework was specifically designed for this purpose. “The system/infrastructure (FDA BEST), data source, study design, and analytic approach are all fit-for-purpose for the study question,” the Harvard researcher told me. “Self-controlled designs are robust and control for non-time-varying factors, especially in adults.”

Céline Gounder, MD, an infectious disease specialist, epidemiologist, and editor-at-large for public health at KFF Health News, said the report used one of the strongest available methods for post-market vaccine surveillance. “This study used one of the best methods we have to check if vaccines cause side effects, and it found that the updated COVID vaccines are safe,” Gounder told me. “Pulling this study from publication doesn’t protect good science. It’s not radical transparency, and it’s not gold-standard science.”

Gounder also noted that the study analyzed data from more than seven million people and found no new safety concerns. “That’s a careful conclusion backed by solid data,” she said. “Blocking a study because you don’t like the answer is censorship.”

Indeed, the consensus among interviewees was that the study appeared adequately powered and appropriately cautious in its conclusions. “This study includes a large number of people, and from what I can see, it appears adequately powered for the conclusions they’re making,” said the epidemiologist with vaccine expertise. “Importantly, the authors are framing the findings appropriately. They are not claiming more than the data support. Their framing is essentially ‘No new safety signals identified.’ That’s a careful and reasonable way to present findings like this.”

The study also openly acknowledged limitations, including possible outcome misclassification and residual uncertainty, while describing how these issues were addressed in the analysis. “Seasonality can be a concern with the study design, but it was adjusted for in the study,” said the Harvard researcher. “Claims data are well equipped for studying the exposure and outcomes of interest… In this study, misclassification was accounted for.”

Steven Goodman, MD, PhD, associate dean of clinical and translational research and professor of epidemiology and population health and medicine at Stanford University, told Inside Precision Medicine the study was informative and aligned with broader evidence supporting the low-risk profile of COVID vaccines in adults over 65. Goodman also highlighted the restraint of the authors’ interpretations. “They do not make a statement about the risk-benefit balance, which they can’t because they didn’t study the benefit, but they note that the FDA felt that the balance was positive,” he said. “Their main conclusion was, ‘Our study contributes to growing evidence on the safety of COVID-19 vaccines.’ It is hard to argue with that.”

Goodman added, “All studies have strengths and limitations, i.e., none establish a scientific truth all by themselves. But this is fundamentally good science that adds valuable information to the COVID vaccine safety picture in adults >65.”

The epidemiologists stressed that vaccine safety science depends on cumulative evidence across multiple studies, methods, and datasets. “Public health surveillance has always operated this way,” the Harvard University researcher said. “No single study claims to be the final word on a topic. You accumulate evidence across multiple studies, multiple methods, and multiple datasets and then interpret the totality of evidence together.”

The Harvard researcher added, “The results are consistent with what has been reported by others, including in other countries. There is no clear scientific reason for this work being pulled from publication.”

Truth welcomes questions

Further, many of the epidemiologists I interviewed stressed that publication does not imply unquestioned acceptance. Instead, they argued that publication serves as the mechanism through which scientific claims challenge, refine, or overturn one another.

Goodman emphasized that the unpublished manuscript was intended for scientific scrutiny and peer review and said imperfections in such work are neither unusual nor disqualifying. “Is it perfect? No, but this is a preprint, and usually the peer review and editing process improves the analyses, exploring robustness to various assumptions, the reporting, and the interpretation,” he said. “I would presume that the final version would have come out with some more qualifications, limitations, sensitivity analyses and caveats.”

The experts I contacted emphasized how suppressing publication interrupts the ordinary process through which scientific consensus develops. “This study should be out there, clearly labeled as one piece of evidence among many, with all the necessary caveats attached,” said the epidemiologist. “Then additional studies come in, more data accumulate, and eventually the field interprets the evidence in totality.”

The unnamed epidemiologist added, “For 250 years, this country has benefited from exactly that: reasonable people openly disagreeing about difficult issues. So why not say, ‘Fine, publish the study,’ and then publish an editorial alongside it explaining the caveats, limitations, and alternative interpretations? That’s how science is supposed to work. You respond to speech you disagree with by adding more speech, not by suppressing speech and certainly not by suppressing scientific speech.”

Several of the epidemiologists argued that blocking the manuscript conflicts with repeated public calls for open scientific debate from directors at agencies under the purview of HHS, notably Jay Bhattacharya, PhD, Director of the National Institutes of Health (NIH).

Goodman said that how the FDA handled this study “contrasted with Dr. Bhattacharya’s many public remarks stressing the criticality of open discussion of scientific results and his objections to suppressing science whose results one doesn’t like. The forced withdrawal of this manuscript prevented that process from occurring, shutting down the open discussion Dr. Bhattacharya has called for in innumerable forums.”

Goodman added that if officials believe the study contains fatal flaws, they should articulate those concerns publicly and subject them to scientific scrutiny, “letting the authors respond and the scientific community decide… Their own critique should be subjected to peer review.”

The fundamental process of science encourages that disputes over evidence should unfold transparently in scientific journals and public debates. “If someone has objections, they should make those objections publicly and specifically in the scientific literature where others can critique them, evaluate them, or even prove them right,” said the epidemiologist. “That’s how science advances. That’s what real science looks like: gold-, platinum-, titanium-, or whatever rare metal metaphor people want to use for standards. The scientific enterprise in this country has succeeded because ideas are tested openly, criticized openly, and refined openly. This kind of amateur hour behavior at regulatory agencies doesn’t help anybody.”

Nostrums, not normalcy

The culling of FDA scientists, be it via resignations or firings in 2025–2026, has continued since Makary’s resignation. Tracy Beth Hoeg, MD, PhD, the head of the FDA’s Center for Drug Evaluation and Research (CDER), was fired Friday (according to a social media post reported by Reuters on Sunday) and replaced by Michael Davis, MD, PhD, who had served as deputy director of CDER for about a year.

There is no concrete evidence connecting the FDA’s blocking publication of two studies accepted into medical journals to Makary’s departure. But the contradictory messaging within the agency on COVID-19 mRNA vaccines—the product of Operation Warp Speed, considered a signature accomplishment of the first Trump administration—is obvious. The collapse of confidence within institutions that once relied on scientific independence as their organizing principle. Increasingly, senior scientists and regulators appear unwilling to publicly defend decisions, studies, or processes they privately regarded as scientifically sound. That shift matters more than any single resignation.

The appointment of Kyle Diamantas as acting head of the FDA, however, is far from a course correction, marking another sharp turn away from independent scientific leadership at America’s top health regulator. A former corporate lawyer for Abbott Laboratories with no medical or research background, Diamantas rose through the agency by advancing the “MAHA” food agenda and cultivating ties to politically aligned health influencers rather than the scientific establishment. A close friend of Donald Trump Jr., the appointment of the 38-year-old Diamantas only reinforces concerns that ideological loyalty is increasingly outweighing scientific expertise inside the FDA.

For decades, FDA and HHS leadership operated with relative continuity, assuming that disputes would be resolved through open scientific debate. That assumption now appears badly weakened. The blocked vaccine safety study became symbolic not merely because of the substance of the research but also because even many scientists who believed the work was rigorous hesitated to say so publicly. When experts become reluctant to attach their names to conclusions that they consider obvious or well-supported, the problem extends beyond politics or personnel. It reflects a deeper institutional fear inside the scientific establishment itself.

Makary’s resignation earlier this month therefore represented more than another leadership change in Washington. It exposed how federal health agencies have been pulled into a culture where scientific judgments are increasingly filtered through ideological loyalty, political risk, and reputational self-preservation. The larger danger is not only instability at the FDA or HHS, but the emergence of a scientific culture in which silence becomes safer than candor. Institutions built to evaluate evidence cannot function for long under those conditions.

The post Reporter’s Notebook: The Day the Scientific Debate Died appeared first on Inside Precision Medicine.

May 18, 2026

AI Model Predicts Alzheimer’s Progression from a Single MRI Scan

Researchers at the University of California, San Francisco (UCSF) have developed an artificial intelligence model capable of predicting cognitive impairment and Alzheimer’s disease progression using only a single baseline MRI scan and basic demographic information. The approach, published in Nature Aging, could help make early Alzheimer’s assessment faster, more accessible, and less dependent on costly specialized testing.

Alzheimer’s diagnosis remains complex and resource-intensive

Alzheimer’s disease accounts for approximately 60% to 70% of dementia cases worldwide. Although structural brain changes and cognitive decline are hallmarks of the disease, accurately forecasting who will develop progressive impairment remains difficult.

Current diagnostic workflows often rely on multiple complementary techniques, including PET imaging, cerebrospinal fluid or blood biomarkers, genetic testing, and comprehensive neuropsychological assessments. While effective, these approaches can be expensive, time-consuming, and inaccessible in many healthcare settings.

MRI scans are among the most widely available clinical imaging tools for neurological assessment, but MRI data alone has historically struggled to capture the complexity and heterogeneity of Alzheimer’s disease progression when used in conventional AI frameworks.

To address this challenge, the UCSF team designed a multitask deep learning framework that combines domain-specific imaging knowledge with advanced machine learning methods to predict cognitive outcomes directly from structural MRI scans.

AI framework predicts cognition without invasive testing

Unlike many earlier Alzheimer’s prediction models, the new system does not require longitudinal imaging data, baseline cognitive testing, PET scans, or molecular biomarker analysis.

The researchers instead focused on extracting clinically meaningful information from a single baseline MRI scan. The framework was trained to perform several related tasks simultaneously, including tissue segmentation, Alzheimer’s diagnosis prediction, and estimation of both present and future cognitive performance.

A key innovation of the study was the development of a specialized image model that segments brain tissue into gray matter, white matter, and cerebrospinal fluid before generating cognitive predictions. According to the authors, this task-specific segmentation step allowed the model to learn biologically relevant spatial brain features more effectively than standard transfer-learning approaches.

Senior study author Ashish Raj, PhD, professor of radiology and biomedical imaging at UCSF, said the goal was to create a system that could be realistically implemented in routine clinical environments.

“Unlike previous approaches, our model does not require baseline cognitive assessment, specialized image pipelines, expensive PET scans, genetic analysis, or fluid proteomics, making it a fast, accurate, and easily implementable tool for most clinical settings,” Raj said in a statement.

Large imaging datasets improved robustness and generalizability

To train and validate the framework, the researchers used imaging and clinical data from the Alzheimer’s Disease Neuroimaging Initiative (ADNI), including MRI scans, demographic information, diagnoses, and cognitive assessments.

The team also incorporated MRI data from the Human Connectome Project Young Adult cohort, which contains scans from healthy younger adults with minimal age-related brain atrophy. According to the authors, exposing the model to healthy brain anatomy improved its ability to distinguish pathological neurodegeneration from normal aging.

An external validation cohort from the Dallas Lifespan Brain Study was additionally used to test the generalizability of the framework across independent datasets.

The researchers reported that the multitask framework outperformed existing AI methods, including standard transfer-learning approaches, in predicting clinically relevant outcomes. The model generated accurate predictions for Alzheimer’s diagnosis, tissue segmentation, current cognitive function, and future cognitive decline using only baseline MRI data.

The study also reported improvements in computational efficiency and processing speed compared with more complex MRI morphometry pipelines commonly used in neuroimaging research.

First author Daren Ma, MSc, a machine learning specialist in the Raj Lab at UCSF, said the framework could help clinicians identify at-risk patients earlier and streamline referrals for advanced neurological evaluation.

“We reported meaningful gains in speed and performance over other pipelines, which could prove valuable in developing a quick clinical prediction of cognitive impairment prior to referring the patient to a more advanced imaging lab and/or a full neuroradiology report,” Ma said.

Potential implications beyond Alzheimer’s disease

The researchers believe the framework could eventually be adapted for other neurodegenerative disorders characterized by structural brain changes and progressive cognitive decline.

Potential future applications include Parkinson’s disease, amyotrophic lateral sclerosis (ALS), and Huntington’s disease. The ability to estimate cognitive impairment using minimal baseline data may also prove useful in community healthcare settings where access to specialist neuropsychological testing is limited.

In addition, the model may have implications for clinical trial design. Identifying likely disease progressors early could help reduce trial size requirements and improve patient selection for studies evaluating disease-modifying therapies.

“The ability to correctly predict progressors from non-progressors using only baseline data can dramatically reduce sample sizes and cost,” Raj said.

The authors emphasized, however, that further validation will be necessary before the model can be broadly implemented in routine clinical practice. Future iterations of the framework may incorporate additional clinical measurements where available, including longitudinal MRI imaging, PET scans, genetics, and blood or cerebrospinal fluid biomarkers.

The study highlights the growing role of AI-driven imaging analysis in neurology and suggests that clinically accessible tools such as MRI may eventually support earlier and more scalable prediction of Alzheimer’s disease progression.

The post AI Model Predicts Alzheimer’s Progression from a Single MRI Scan appeared first on Inside Precision Medicine.

May 18, 2026

STAT+: Why the WakeMed – Atrium Health hospital merger matters

This is the online version of STAT’s weekly email newsletter Health Care Inc. Sign up here.

Thanks for being here! So much to read, including a new series from my colleagues Lev Facher and Isa Cueto. They delved into the perils of alcohol despite its ubiquity in American culture. The opening piece has several passages and quotes that will make you stop and think in a gratifying, educational way. Give it a read, and as always, pour your thoughts and tips my way: bob.herman@statnews.com.

Wide a-Wake(Med)

Most of you reading this do not live in North Carolina. But a hospital system merger in that state that has created local rage has national relevance.

Continue to STAT+ to read the full story…

May 18, 2026

What to expect from Google this week

This story originally appeared in The Algorithm, our weekly newsletter on AI. To get stories like this in your inbox first, sign up here.

When Google opens its doors tomorrow for its annual developer conference, I/O, it will do so as a clear third place in the foundation model race. A year ago, at Google I/O 2025, the situation looked very different: The company was still riding high from the launch of Gemini 2.5 Pro that March, and distinguishing among the top-tier large language models often felt like a subjective splitting of hairs.

But a foundation model’s reputation these days rests largely on its coding capabilities, and for months Google’s coding tools have been outgunned by Anthropic’s Claude Code and OpenAI’s Codex. Those systems are so dramatically superior to Google’s own offerings that the company has reportedly had to allow some engineers at DeepMind, its AI division, to use Claude for their work—lest they fall farther behind.

So when I arrive at the conference in Mountain View, California tomorrow, I’ll certainly be on the lookout for any efforts Google is making to claw its way back into frontrunner position. But I’m also eager to see new developments in areas where Google shapes the cutting edge, such as AI for science. The company’s moves there might receive less attention, but they will be no less consequential.

Here are three things I’ll be paying particular attention to over the next two days.

An attempted coding comeback

Google is taking its AI coding crisis seriously. According to reporting from The Information, there’s a new AI coding team at DeepMind. And the Los Angeles Times has reported that John Jumper, who shared a 2024 Nobel Prize in chemistry with DeepMind CEO Demis Hassabis for their work on the protein structure prediction software AlphaFold, is lending his talents to the efforts. I would be surprised if we don’t see a major new coding release at I/O, perhaps in the form of an update to the company’s Antigravity agentic coding platform.

That said, we shouldn’t expect anything transformative here. Googlers have access to models and products that are substantially ahead of those released to the public, yet they were still reportedly fighting over who got access to Claude Code last month. Unless the company has made astonishing progress since then, Google probably won’t make it back to the coding frontier in the next two days.

Science and health

Coding might be Google DeepMind’s weakness, but science is its conspicuous strength. It is the only frontier AI company to have earned a Nobel Prize. And as LLMs have come to dominate the AI-for-science landscape, Google has only solidified its lead. Last year, the company released multiple scientific AI tools, including the AI co-scientist, which formulates hypotheses and research plans in response to user questions and has been described as an “oracle” by one Stanford scientist, and AlphaEvolve, a system that iteratively discovers new solutions for mathematical and computational problems. If any new scientific tools are announced at I/O, they’ll be worth noting.

I’ll also be paying close attention to any moves Google makes in health and medicine. Google is doing some of the best research out there on LLM-based health tools, but OpenAI has defined the health AI conversation since the release of ChatGPT Health in January. Google has announced that it will be making its AI-powered Health Coach publicly available tomorrow, but promotional material suggests that the tool is geared more toward providing advice on topics such as fitness and diet than to addressing users’ medical concerns. Is this another area where Google has fallen behind, or is the company exercising appropriate caution in a high-stakes domain?

The drama

While Google fans congregate down in Mountain View, roughly 30 miles north in Oakland the Elon Musk v. Sam Altman trial will be wrapping up. The past few months have seen more than their fair share of AI CEO drama—before the trial, the animosity between Altman and Anthropic CEO Dario Amodei took center stage as Anthropic and OpenAI worked to negotiate deals with the US Department of Defense. But DeepMind’s Hassabis has, for the most part, steered clear of such drama. He effectively presents himself as a Nobel Prize-winning nerd, and if he has written screeds about any of his peers, they haven’t been leaked to the press or appeared in legal discovery.

That’s not to say that Google is controversy free. Last month, a group of 600 employees, many of whom work for DeepMind, sent a letter to CEO Sundar Pichai protesting an impending DoD deal. Google signed that deal the next day. Hassabis, Pichai, and all the other big names will surely do their best to skirt these and other touchy subjects while on stage, but controversies will worm their way in regardless. It will be interesting to see whether Google can maintain its veneer of neutrality.

May 18, 2026

The Download: Musk v. Altman week 3, and Trump’s tech trading

This is today’s edition of The Download, our weekday newsletter that provides a daily dose of what’s going on in the world of technology.

Musk v. Altman week 3: Musk and Altman traded blows over each other’s credibility. Now the jury will pick a side.

In the final week of the Musk v. Altman trial, lawyers attacked the credibility of the two tech leaders. Sam Altman was accused of lying and self-dealing, while Elon Musk was portrayed as a power-seeker trying to control artificial general intelligence.

The case unearthed new details about the two arch-rivals and OpenAI’s contested nonprofit status, as well as a golden trophy of a donkey’s ass awarded to an employee who challenged Musk.

Read the full story on the explosive final week of the trial.

—Michelle Kim

Michelle Kim, who’s also a lawyer, has been in court throughout the Musk v. Altman trial. Read her coverage of week 1 and week 2, plus a Q&A on what it was like in the room.

The must-reads

I’ve combed the internet to find you today’s most fun/important/scary/fascinating stories about technology.

1 Trump traded hundreds of millions in tech stocks before favorable policy moves
He bought shares in Nvidia, AMD, and Arm ahead of policy boosts. (Quartz)
+ And touted Palantir on Truth Social after buying its stock. (CNBC)
+ His crypto venture and Iran’s top exchange tapped the same networks. (Reuters $)

2 SpaceX plans to list on the Nasdaq stock exchange as soon as June 12
It wants to raise up to $75 billion at a $1.75 trillion valuation. (Reuters $)
+ BlackRock may invest up to $10 billion in the offering. (The Information $)
+ Cerebras’ blockbuster IPO has boosted hopes for the listing. (CNBC)
+ Which is set to dwarf many of the biggest IPOs on ⁠record. (Reuters)

3 Chinese AI groups have pulled ahead of US rivals in video generation
ByteDance and Kuaishou’s models lead in realism and scale. (FT $)
+ AI is fueling China’s short-drama boom. (MIT Technology Review)
+ While its AI labs are betting big on open source. (MIT Technology Review)

4 Iran says it will charge Big Tech for using undersea internet cables
The cables beneath the Strait of Hormuz carry vast digital traffic. (CNN)
+ Tech bosses met at Uber HQ on Saturday to discuss Iran’s future. (404 Media)

5 Samsung has a “last chance” to stop a massive strike over AI
Over 45,000 employees could walk out for 18 days this week. (CNBC)
+ They want a bigger share of the AI boom. (FT $)
+ Samsung and its largest labor union will resume talks on Tuesday. (Reuters $)

6 Old oil and gas wells could become a new source of clean energy
US states plan to convert them into geothermal energy assets. (Wired $)
+ A balcony solar boom is coming to the US. (MIT Technology Review)

7 The ChatGPT era has triggered a 30% surge in grades at a top university
Grades inflated in text-heavy courses but remained flat in others. (Axios)
+ Princeton has changed its honor code because of AI cheating. (WSJ $)
+ And real cheating rates may be far higher. (The Times $)

8 Ex-Google CEO Eric Schmidt was fiercely booed during an AI speech
His graduation speech praising AI agents sparked uproar. (The Verge)
+ A populist backlash is building against AI. (MIT Technology Review)

9 Arm faces a US antitrust probe over its chip tech licenses
Regulators are investigating whether it has an illegal monopoly. (Bloomberg $)
+ Qualcomm has accused Arm of anticompetitive conduct. (Reuters $)

10 ArXiv will ban researchers who submit AI slop
Offending authors face year-long bans from the pre-print server. (TechCrunch)

Quote of the day

“When someone offers you a seat on the rocket ship, you do not ask which seat. You just get on.”

—Ex-Google CEO Eric Schmidt extolls the virtues of AI agents in a graduation speech at the University of Arizona, prompting a chorus of boos.

One More Thing

a gloved hand holding up a microfluidic chip

Is this the end of animal testing?

In a clean room in his lab, Sean Moore peers through a microscope at a bit of human intestinal tissue growing on a plastic chip. It’s one of 24 so-called “organs-on-chips” his team bought three years ago. The technology is designed to mimic human biology—and could reduce the need for animal testing.

The appeal is not only ethical. Around 95% of drugs developed through animal research ultimately fail in people, and early studies suggest organ-on-a-chip systems may offer more accurate insights into how diseases behave and how drugs work. But the field still faces major technical and cost challenges before it can replace animal research.

Find out how organ-on-chip technology could reshape drug testing.

—Harriet Brown

We can still have nice things

A place for comfort, fun, and distraction to brighten up your day. (Got any ideas? Drop me a line.)

+ Listen to the captivating first recordings of whale songs from 1949.
+ Meet the feline guardians of New York’s corner stores in this photo collection.
+ A newly discovered floor plan allowed historians to pinpoint the location of Shakespeare’s only property in London.
+ A music fan spent decades secretly recording 10,000 local shows. Now the entire collection is available online.

May 18, 2026

Texas is opening up a detransition clinic

Get your daily dose of health and medicine every weekday with STAT’s free newsletter Morning Rounds. Sign up here.

Good morning. Glad you’re here. Lots of news dropped over the weekend, so let’s get to it.

Read the rest…

May 18, 2026

STAT+: The China question is tearing biotech apart

There’s a schism in America’s drug business, playing out in punchy direct messages, feisty group chats, and the occasional heated in-person exchange.

The problem is China. Fledgling startups and pharmaceutical giants alike are addicted to Chinese drugs, filling their pipelines with would-be blockbusters developed at enviable speed and bought on the cheap. They’ve spent some $60 billion on Chinese molecules in the first three months of 2026 alone, according to state figures. That’s on pace to double last year’s total, which was already 10 times larger than the one from 2021.

No one disagrees that it’s good business. And more drugs moving swiftly through development means hope for patients around the world desperately awaiting new medicines.

But, according to more than a dozen interviews with industry executives and investors, the question of whether to partner with Chinese firms — or see them as rivals — is tearing biotech apart, pitting peers and partners against one another and souring relationships in an otherwise close-knit corporate community.

Continue to STAT+ to read the full story…

May 18, 2026

StockWatch: Regenxbio Tumbles Despite Positive Pivotal Data for DMD Gene Therapy Candidate

Regenxbio (NASDAQ: RGNX) shares nosedived 43% over two days late last week, reaching 52-week lows on consecutive days, despite generating positive pivotal Phase III data for its Duchenne muscular dystrophy (DMD) gene therapy candidate RGX-202.

While the data was encouraging enough to enable discussion of Regenxbio bringing a second DMD gene therapy to the market, investors and analysts concluded it was not encouraging enough to pose a competitive threat to the developer of the first marketed DMD gene therapy, Sarepta Therapeutics (NASDAQ: SRPT), or to Solid Biosciences (NASDAQ), whose DMD gene therapy candidate SGT-003 is in Phase III as well as Phase I/II trials.

Even worse, investors were jolted by Regenxbio’s disclosure that the FDA had recommended the company conduct a randomized controlled trial (RCT) to assess RGX-202 in DMD during talks with agency officials. Regenxbio sought to reassure investors in its first-quarter earnings press release by noting past FDA guidance that externally controlled trials “may be adequate for demonstrating substantial evidence of effectiveness, especially when the treatment effect is sufficiently large enough to overcome limitations of externally controlled trials.”

Regenxbio plans to discuss its data with FDA officials at a future meeting. The agency has offered to review the RGX-202 data and alternative proposals, according to the company.

“RGX-202 pivotal data point to potential entry of second DMD gene theory, but a possibility of RCT requirement makes market entry timing unclear,” Kostas Biliouris, PhD, a managing director on the biotechnology research team of Oppenheimer & Co., wrote in a research note.

If the FDA does not insist on an RCT, RGX-202 could gain accelerated approval in 2027, Biliouris noted. Otherwise, the gene therapy is looking at not reaching the market for at least three additional years.

“Completing an RCT study as a precursor to filing or a precursor to approval means that it’s very unlikely that any new gene therapy would be approved until 2030. And I think that scenario is really untenable for the [DMD] community,” Simpson said. “It’s the opposite of regulatory flexibility.”

These regulatory and competitive concerns sent investors scrambling to sell Regenxbio shares late last week. The shares tumbled 38% from $10.04 to $6.24 Thursday, then slid another 8% Friday, sinking to $5.72 at the closing bell.

Positive microdystrophin expression

Regenxbio’s stock woes came despite the company announcing positive results from its pivotal Phase III portion of the Phase I/II/III AFFINITY DUCHENNE^® trial (NCT05693142) of RGX-202. The company said the trial met its primary endpoint as 93% of participants (28 of 30) reached at least 10% microdystrophin expression at Week 12. A 31^st participant refused a muscle biopsy and, as a result, did not have a Week 12 biopsy available for evaluation.

Microdystrophin expression averaged 71.1% across all participants, and 41.6% in older boys, aged >8 years, with 80% of participants achieving >40% microdystrophin expression, Regenxbio said.

“High unmet need remains for Duchenne patients as current options face limitations related to efficacy, safety, and access. The untreated Duchenne population continues to grow in the United States and globally. Physicians and patients need new next-generation options,” Curran M. Simpson, president and CEO, told analysts on the company’s first quarter earnings call.

Regenxbio acknowledged two reports of treatment-related serious adverse events (~6.5% of treated patients): An 8-year-old patient developed subacute myocarditis, while a 10-year-old patient showed a case of asymptomatic liver injury.

“Both were easily managed and resolved within weeks without sequelae,” Simpson told analysts.

Biliouris acknowledged RGX-202’s positive microdystrophin but said it will not likely have a material impact on Sarepta and its marketed DMD gene therapy Elevidys^® (delandistrogene moxeparvovec-rokl).

Limited likelihood

“RGX-202’s functional benefit remains unclear without RCT data, limiting the likelihood of AA [accelerated approval] given an already fully approved DMD gene therapy,” Biliouris said.

He added that RGX-202’s safety profile could deteriorate once the gene therapy reaches the market and is being administered to patients, as happened with Elevidys after some 800 had been treated with the therapy, prompting Sarepta to halt shipments of Elevidys for non-ambulatory patients and pause a Phase III trial.

The halt—plus a label update limiting Elevidys use to ambulatory patients—explains why the gene therapy’s net product revenue plunged 73% year-over-year in Q1, to $102 million from $375 million. Elevidys generated $898.7 million in 2025 revenue—it ranks second on GEN’s just published A-List of Top 10 Best-Selling Gene Therapies—which was 9.5% above 2024’s $820.8 million.

The Q1 sales decline has sent Sarepta’s stock into decline: From $23.06 on May 6, before releasing Q1 results after that day’s closing bell, Sarepta shares have slumped 22.5%, to $17.88 on Friday.

Elevidys sparked a showdown with the FDA last summer when the agency briefly demanded Sarepta also pause Elevidys shipments to ambulant patients following the second patient death tied to Elevidys, then reversed itself after, according to news reports, pleas to Congress, the FDA, and President Donald Trump by conservative leaders and DMD patient advocates—who launched a Change.org petition that garnered 1,900 signatures.

Competitive advantage

Despite the slumping sales and resulting stock decline, Biliouris noted that Sarepta and Elevidys have a significant competitive advantage over challengers: A 3-1/2 year first to market advantage, with statistically significant functional benefits reported from randomized trials, as well as what the analyst called “compelling” three-year positive topline follow-up data from ambulatory DMD patients in the 52-patient active arm in Part 1 of Sarepta’s EMBARK trial (Study SRP-9001-301, NCT05096221).

That data showed significant improvements in North Star Ambulatory Assessment (NSAA), Time to Rise (TTR), and 10-meter walk/run (10MWR).

“Even if RGNX secures AA, we expect minimal impact given the large DMD market size (can accommodate multiple companies) and potential Elevidys monopoly in the non-ambulatory market,” projected for 2027 and later, Biliouris wrote.

According to Sarepta, Duchenne affects approximately 1 in 3,500 to 5,000 males born worldwide—some 300,000 people worldwide, according to research and patient care group Cure Duchenne. In the United States, about 15,000 young men and a few young women live with DMD, according to Parent Project Muscular Dystrophy estimates. A 2019 study found that most people with DMD become non-ambulatory around ages 10–12 and need assisted ventilation at around 20 years of age.

Andrew Tsai, equity analyst with Jefferies, said Sarepta’s three-year data, including muscle MRI data, has only begun to be promoted by the company this year. Since it can take six months to go from “start form” initiating the treatment process to infusion with Elevidys, Tsai reasoned, “we expect momentum to rebuild progressively/steadily in Q3/Q4, restoring confidence in the ambulatory DMD oppty.”

Some ~80% of ambulatory DMD patients remain untreated, Tsai noted, while Sarepta told investors in its Q1 earnings presentation that more than 1,300 patients have been treated with Elevidys in commercial settings or clinical trials as of May 5.

“While Elevidys’ safety perception has changed, we think marketing efforts on muscle MRI data, long-term three-year EMBARK data, and no deaths in ambulatory DMD could entice patients/caregivers and physicians to adopt Elevidys more, widening the moat,” Tsai wrote.

Maury Raycroft, PhD, a colleague of Tsai and equity analyst with Jefferies, wrote that Regenxbio’s data “reinforces microdystrophin as a surrogate, which is constructive for SLDB [Solid Biosciences].”

Playing to strengths

However, Raycroft added that Regenxbio’s safety events (notwithstanding immunosuppression) and limited regulatory clarity absent a pivotal RCT “play into SLDB’s strengths,” such as its use of a steroid-only prophylactic immunomodulation regimen (no safety issues to date) and its ongoing Phase III IMPACT DUCHENNE trial (NCT07160634), which is an RCT, thus a derisking factor from a regulatory standpoint.

“We believe RGNX is relying on and will require reg[ulatory] flexibility, which incorporates add’l risk and limitations, especially w/ FDA leadership in flux,” Raycroft wrote. “We caught up w/ SLDB, who also pointed out that RGNX could run into challenges to run an RCT given their immunosuppressive regimen.”

That regimen consisted of sirolimus, eculizumab, and steroids that included prednisone, researchers from Regenxbio and clinical partners reported in a poster presented at the International Congress of the World Muscle Society, held October 7–11, 2025, in Vienna.

On May 7, Solid announced it had dosed the first patient in the IMPACT DUCHENNE trial in Australia, at the Children’s Hospital at Westmead. The multi-country, placebo-controlled, randomized, double-blind trial has a pre-specified primary endpoint of change from baseline at 18 months in time to rise from supine (TTR) velocity, based on a Type C meeting with the FDA.

“With the initiation of a randomized, placebo-controlled clinical trial, we are reinforcing our conviction in SGT-003 and our long-standing commitment to generating well-controlled, high-quality data,” Gabriel Brooks, MD, Solid’s chief medical officer, said in a statement.

Solid shares dipped 2% on news of the dosing, from $7.20 to $7.07. Since then, the shares have yo-oed, climbing 9% to $7.72 on May 12 but sliding 10% since then, to $6.92 on Friday.

In its Phase I/II INSPIRE DUCHENNE study (NCT06138639), SGT-003 has also been administered to 46 patients, with approximately 30 participants dosed as of year-end 2025, Solid said.

“Families living with Duchenne continue to face difficult treatment decisions in a setting of significant unmet medical need,” Brooks added. “Solid remains focused on helping inform the Duchenne community of potential additional treatment options through the responsible and rigorous clinical evaluation of SGT-003.”

uniQure, Replimune gain as Makary exits FDA

Two gene therapy developers saw their stocks enjoy significant gains after Martin A. Makary, MD, resigned as FDA commissioner.

Makary’s resignation on May 12 capped nearly a week of speculation that he was about to exit the agency after a turbulent 13-month tenure. That tenure was marked in part by the elimination of 3,500 FDA positions as part of the Elon Musk-led Department of Government Efficiency (DOGE)-directed federal job cuts—as well as more frequent rejections of biologics license applications (BLAs) for new therapies, particularly gene therapies in rare disease indications.

Those rejections were carried out by the agency’s Center for Biologics Evaluation and Research (CBER) during the two tenures of Vinayak (Vinay) Prasad, MD, as Center director. Prasad resigned the first time in August 2025 after less than three months at the CBER helm, after he led the FDA’s confrontation with Sarepta over patient deaths tied to Elevidys (see Regenxbio item, above). The second resignation was announced in March and took effect on April 30, after he led the FDA’s hardline stance and public criticism against uniQure (NASDAQ: QURE)’s Huntington’s disease (HD) gene therapy candidate AMT-130.

While uniQure stock roller-coastered after Prasad’s second resignation, the stock jumped 21% in the four trading days between May 8, when an unnamed-source report about Makary being fired first surfaced in The Wall Street Journal, and May 13, the day after he resigned. uniQure rose 14.5% from $24.15 to $27.66 the day of the WSJ report, plateaued on May 10, dipping two cents to $27.64, then resumed their climb, rising 5% to $29.10 the following day before inching up another 0.2% to $29.17 on Wednesday.

An even bigger winner among stocks, however, was Replimune Group (NASDAQ: REPL). The developer of oncolytic immunotherapies saw its shares rocket 59% after news surfaced of Makary exiting the FDA.

Replimune has found itself in the FDA’s crosshairs over its biologics license application (BLA) for its lead product candidate RP1 (vusolimogene oderparepvec) in combination with nivolumab to treat advanced melanoma, instead issuing two complete response letters (CRLs)—one in April 2025, the other last month.

On April 10, the FDA rejected Replimune’s BLA for a second time, issuing a complete response letter (CRL) contending that the data set upon which the agency’s breakthrough therapy designation was awarded was not sufficient to allow for RP1 approval—an assertion Replimune vehemently rejects.

Replimune responded to the second BLA by criticizing the FDA for an inconsistent review process, saying the agency contradicted earlier guidance to the company and assessed the resubmitted BLA through a different review team that replaced the team that previously interacted with the company.

Replimune also defended the combination therapy’s data in the Phase II IGNYTE trial (NCT03767348)—a 34% response rate with a median duration of 24.8 months and a favorable safety profile, the basis of the combo’s breakthrough therapy designation.

Following the first news report of a Makary firing in the works, Replimune shares jumped 22% from $3.34 to $4.07. After slipping 8% to $3.74 the following trading day (May 11), Replimune rose 9% to $4.09 the following day after Makary resigned—then vaulted 30% to $5.30 on Wednesday.

“Broadly, we see multiple options for experienced leaders who could help stabilize the Agency following the many leadership transitions, and believe the tendency toward the administration’s “Right to Try” could draw a next leader who is more permissive on drug approvals near-term positive on the space,” Brian Abrahams, MD, head of global healthcare research with RBC Capital Markets, wrote in a research note.

Abrahams put forward six possible permanent successors to Makary:

Kyle Diamantas, current interim FDA commissioner; previously FDA deputy commissioner for human foods and senior counselor to Health and Human Services Secretary Robert F. Kennedy Jr.
Stephen Hahn, MD, CEO of Nucleus RadioPharma and a former FDA commissioner in President Donald Trump’s first administration (December 2019–January 2021).
Brett Giroir, MD, CEO of Altesa Biosciences; previously assistant secretary for health in Trump’s first term and an acting FDA commissioner (2019).
Sara Brenner, MD, HHS senior counselor for public health as of April 16; previously FDA principal deputy commissioner and acting FDA commissioner (January–April 2025).
Houman Hemmati, MD, PhD, a board-certified ophthalmologist and co-founder of Optigo Biotherapeutics, who is under consideration for CBER director.
Richard Pazdur, MD, a 26-year FDA veteran who retired in December 2025 after serving three weeks as CDER director (November–December 2025); previously founding director, FDA Oncology Center of Excellence (2017–2025).

“If Makary’s ouster indeed stemmed from political disagreements (vapes, abortion), the next Commissioner could harbor more ideological views—which could compromise perceived Agency credibility—and just by virtue of having another change, this would likely exacerbate the mixed messages companies have been receiving around FDA’s bar for their drugs, one of the key regulatory challenges the sector has faced,” Abrahams cautioned.

Leaders and laggards

Innate Pharma (Euronext Paris: IPH) shares jumped 35% from €1.23 ($1.42) to €1.66 ($1.92) on Wednesday after the Marseille, France-based developer of cancer drugs based on innate immunity and antibody engineering reported first-quarter results that beat analyst expectations. Innate finished Q1 with earnings per share of -0.1522, vs. the consensus forecast of -0.1616, on revenue of €2.6 million ($3.022 million) that was more than double (117% above) the €1.2 million ($1.395 million) reported in Q1 2025, thanks to partial or entire recognition of the proceeds received under collaboration agreements with AstraZeneca (NYSE, London Stock Exchange, and NASDAQ Stockholm: AZN) and Sanofi (Euronext Paris: SAN). Innate’s American depositary shares (ADSs) (NASDAQ: IPHA) rocketed 64% from $1.32 to $2.17 Wednesday.

Reviva Pharmaceuticals Holdings (NASDAQ: RVPH) shares plummeted 56% from 80 cents to 35 cents on Wednesday after the central nervous system (CNS), inflammatory, and cardiometabolic disease drug developer disclosed in a regulatory filing that the Nasdaq Hearings Panel had delisted the company’s stock, suspending it from trading on the exchange as of Thursday. Reviva said its shares will instead begin trading that day on the OTCQB Venture Market under its existing symbol. The Panel told Reviva that it failed to comply with Nasdaq’s minimum bid price of $1 per share required for continued listing on the Nasdaq Capital Market. Reviva disclosed the delisting the same day it reported first quarter results: The company narrowed its quarterly net loss year-over-year, finished Q1 with a net loss of approximately $3.2 million ($0.46 per share) vs. approximately $6.4 million ($2.61 per share) in the year-ago quarter.

The post StockWatch: Regenxbio Tumbles Despite Positive Pivotal Data for DMD Gene Therapy Candidate appeared first on GEN – Genetic Engineering and Biotechnology News.