With no approved vaccine for Ebola outbreak, experts weigh testing a long shot

The latest Ebola outbreak in the Democratic Republic of the Congo, which was only confirmed to be underway at the end of last week, is already the fourth largest on record. The deadly virus is spreading in a conflict zone where recent Ebola experience has shown containment will be a challenge. There is no vaccine that targets the species of the virus that is spreading there, Bundibugyo.

But there is a tiny bit of scientific evidence that suggests the existing licensed Ebola vaccine, Merck’s Ervebo, might offer some protection against this virus, even though it is designed to target a different species of Ebola, Zaire ebolavirus. 

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Drug Target for Fragile X Syndrome Identified Through Preclinical Study

UCLA Health researchers have identified a potential drug target for treating fragile X syndrome (FXS), the most common genetic cause of intellectual disability and autism that affects roughly one in 2,000 boys.

Fragile X syndrome is caused by a mutation in a single gene, FMR1, that results in the loss of a protein critical for normal brain development and function. Headed by Carlos Portera-Cailliau, MD, PhD, professor of neurology at UCLA and member of the UCLA Brain Research Institute, the researchers, the team’s work in genetically engineered mice lacking the Fmr1 gene identified the synaptic protein EPAC2 as a potential therapeutic target for fragile X syndrome. Their study showed that blocking EPAC2 in the fragile X mouse model restored abnormal patterns of brain activity and improved several FXS-associated behavioral symptoms.

Pertera-Cailliau is senior and corresponding author of the researchers published paper in Neuron, titled “Translatome profiling reveals opposing alterations in inhibitory and excitatory neurons of fragile X mice and identifies EPAC2 as a therapeutic target.”

Fragile X syndrome is a prototypical neurodevelopmental disorder (NDD) characterized by intellectual disability, social anxiety, atypical sensory processing characterized heightened sensitivity to sensory input such as sound and touch, and a higher risk of seizures. Many also meet the criteria for an autism spectrum disorder diagnosis. “Symptoms of fragile X syndrome (FXS), the leading monogenic cause of intellectual disability and autism, are thought to arise from an excitation/inhibition (E/I) imbalance,” the authors stated.

FXS is caused by mutations in the FMR1 gene, resulting in near complete loss of the fragile X messenger ribonucleoprotein (FMRP), an RNA-binding protein in neurons that plays different roles in cell compartments including the nucleus, axons and dendrites, including regulating mRNA translation at synapses, they explained. As it is caused by a change in a single gene, fragile X syndrome has long been considered a promising candidate for targeted therapies yet clinical trials to date have not produced an effective treatment. “Since the discovery of the genetic basis of FXS in 1991, several clinical trials have been undertaken—without success—and no specific treatments for FXS are currently available,” the investigators continued. “Thus, there is an urgent need to rethink therapeutic strategies for FXS.”

For their newly reported study the researchers used genetically engineered knockout (KO) mice that lack Fmr1 to simulate fragile X syndrome. Using genetic sequencing, they found that levels of the gene EPAC2 were increased in the brain of fragile X mice. This was of potential interest as a target for therapy because the gene’s protein, EPAC2, is localized to synapses and is known to be important for learning and memory.

The researchers then demonstrated that blocking EPAC2 in the fragile X mouse model, either genetically, or using an EPAC2 inhibitor compound, restored cortical circuit function and improved multiple behavioral symptoms associated with fragile X syndrome, including heightened sensitivity to touch, difficulties with social interaction and their susceptibility for seizures. “Perhaps the most exciting result is that treatment with an EPAC2 antagonist can rescue several behavioral phenotypes in Fmr1 KO mice,” the authors stated.

“EPAC2 emerged as an attractive target because it was consistently altered across multiple types of brain cells in our analysis,” said the study’s first author Anand Suresh, PhD, a post-doctoral fellow in the laboratory of Portera-Cailliau. “When we blocked it, either genetically or with a drug compound, we saw meaningful improvements in both brain circuit function and behavior.”

EPAC2 is expressed almost exclusively in the brain, which means drugs targeting it are less likely to cause unwanted effects elsewhere in the body. Suresh said this is an important consideration as researchers continue preclinical studies. “This bodes well for future preclinical trials and safety studies in humans, as compounds that target EPAC2 should not have off-target effects,” the authors stated in their report.

For their study the UCLA investigators used an RNA sequencing technique to examine gene activity separately in two major classes of brain cells: those that excite and those that inhibit neural activity. Fragile X syndrome is thought to arise from an imbalance between these two systems. The analysis revealed striking differences in how the genetic mutation underlying Fragile X syndrome affects each cell type but also identified a small set of genes, including the one that encodes EPAC2, that were dysregulated in both.

The researchers also found that EPAC2 levels appear to rise gradually as the brain matures, suggesting it may be a particularly relevant target for older children and adults with Fragile X syndrome, rather than only in early development. They concluded, “Our results should encourage the development of novel EPAC2 inhibitors for the treatment of FXS. More generally, our study exemplifies how transcriptomic approaches in animal models of neuropsychiatric conditions can be used to prioritize potential novel therapeutic targets.”

The post Drug Target for Fragile X Syndrome Identified Through Preclinical Study appeared first on GEN – Genetic Engineering and Biotechnology News.

STAT+: Trump’s CMS to allow skimpier plans on ACA exchanges

The Trump administration is opening the floodgates for Affordable Care Act exchanges to feature plans that offer bare-bones coverage and fewer protections, but which have lower monthly premiums.

The Centers for Medicare and Medicaid Services on Friday finalized its proposal from February to pare back the requirements that ACA plans have to meet beginning for 2027, like offering standardized plans and restricting high-deductible, catastrophic plans to a small subset of members. 

The changes are aimed at making policies cheaper, in line with President Trump’s affordability agenda, but they also fit within the conservative playbook of loosening market restrictions and consumer protections in favor of offering people the freedom to choose policies that offer less coverage. The obvious drawback, health policy experts say, is that those policies put individuals and families on the hook for potentially massive bills if they actually need care. 

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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.

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.

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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.

Inside Anduril and Meta’s quest to make smart glasses for warfare

The defense-tech company Anduril has shared new details about the augmented-reality headset for the military it’s prototyping with Meta, including a vision for ordering drone strikes via eye-tracking and voice commands.

Quay Barnett, who leads the efforts as a vice president at Anduril following a career in the Army’s Special Operations Command, says his fundamental goal is to optimize “the human as a weapons system.” The vision is undoubtedly cyborg-inspired: Barnett wants drones and soldiers to see together, share information seamlessly, and make decisions as one. 

Anduril actually has two such projects in the works. The first is the Army’s Soldier Born Mission Command, or SBMC, for which the company won a $159 million prototyping contract last year to work with Meta on augmented-reality glasses to attach to existing military helmets. But Anduril has also embarked on a self-funded side quest, announced in October, to design its own helmet and headset combo called EagleEye. This is something the military has not asked for, but Anduril insists it will prefer it and purchase it in the end.

So far, both systems are years away. The Army isn’t expected to move its top choice for the SBMC program into production until 2028, if it picks one at all (the previous lead for the effort, Microsoft, was set to receive a $22 billion production contract that was ultimately cancelled when the glasses didn’t prove viable). But Barnett told MIT Technology Review about where both Anduril’s prototypes are headed.

Depending on the situation, the glasses for either prototype will overlay certain information onto a soldier’s field of view. This might be as simple as a compass or as complex as an entire map of the area, information about where nearby drones are flying, or AI-driven recognition of a target like a truck. 

The soldier would then speak to the interface in plain language—for example, to order an evacuation for someone who’s been injured or to plan a route taking into account which areas are off limits. A large language model—Anduril is in tests with Google’s Gemini, Meta’s Llama, and even Anthropic’s Claude, despite the company’s conflict with the Pentagon—will be used to help translate a soldier’s speech into commands the software can follow. And the engine for it all will be Anduril’s software Lattice, which incorporates data from lots of different military hardware into one picture. The Army announced in March that it would spend $20 billion to integrate Lattice with essentially its entire infrastructure.

Barnett’s team is designing the headset to carry out multi-step tasks. A soldier might send a drone to surveil an area and instruct it to come back once it’s found something that looks like an artillery unit; then the system would recommend courses of action, like sending a nearby drone to strike, that would have to be approved by the normal chain of command. Leading the system through this, if all goes to plan, might not even require speech; the soldier could instead communicate through tracked eye movements and subtle taps.

That’s the idea, anyway. It’s worked on early prototypes, Barnett says, but there aren’t yet versions ready for the Army to test at scale. The component parts began arriving in March. Because of federal military contracting rules, these parts—unlike Meta’s commercial smart glasses—required new supply chains that don’t rely on Chinese companies.

It’s a lot for soldiers already bogged down in information overload, says Jonathan Wong, a former US Marine who works as a senior policy researcher at RAND on Army efforts to buy new tech. Both smart glasses projects aim to create a clean interface that presents only the right information at the right time. But it’s a product that soldiers will reject if it costs more of their attention than it saves. “How much mental bandwidth do you have to be both aware of your surroundings and to operate this technology in a way that makes you and your whole unit better?” he says.

Wong recalls that as a platoon commander, for example, he had a radio that operated on three different channels at once. “The moment that two people were on different channels talking at the same time, I immediately couldn’t comprehend anything that either one of them was trying to tell me, and I was probably not aware of my own surroundings,” he says. “I think there are limits to what you can take in.”

Ideally, Barnett says, smart glasses can ease that information overload. Anduril’s approach is to get creative with ways the user can access necessary information quickly. Voice commands and eye tracking are a piece of that strategy. But even if it’s all technically feasible, it might take years of field testing to know if the system is actually useful for soldiers, Wong says. 

Such a system would mark a major escalation in how closely soldiers rely on imperfect AI systems. While computer vision models used to identify objects have long been employed by militaries, and chatbots have recently entered decision-making during the war in Iran, these technologies have not yet made their way to most frontline soldiers. A smart glasses system tasked with identifying threats and recommending strikes would introduce massive new risks of errors. 

Anduril is not the only one competing to develop smart goggles for combat. Rivet, which specializes in wearable sensors for the military, received a $195 million prototyping contract the same time, and in March the Israeli defense-tech company Elbit received its own $120 million contract. This all comes after Microsoft lost its role leading the Army’s smart glasses effort, following a Pentagon audit that found the Army wasn’t properly testing the glasses, a mistake that could have wasted $22 billion.

For both Anduril’s prototypes, the company is testing a new system for digital night vision, which uses electronic sensors and algorithms to boost low levels of light. It’s been a promised technology for decades but has tended to work too slowly for practical use and produce grainy images. Anduril says it has found improvements over previous prototypes through techniques rooted in both new generative AI and older machine learning. 

Much of the other hardware for both projects is being built by Meta, including the displays and the waveguides that send visuals to the user’s eye without blocking the view. That might be a surprise to anyone who knows the backstory: In 2017, Facebook (now Meta) ousted Anduril founder Palmer Luckey following an internal conflict involving his support for Donald Trump. The two are now back in the augmented-reality business together, while Mark Zuckerberg has also adopted a friendlier posture toward the second Trump administration.

For the Army initiative, this suite of smart glasses, night vision, and sensors will be attached to the helmets and other gear soldiers already wear, with a separate battery pack. The EagleEye version will instead incorporate the tech into the helmet itself. Even if the Army doesn’t prefer EagleEye in the end, Barnett says, Anduril will attempt to sell the system to foreign militaries.

Multiple challenges must still be overcome. Unlike Meta’s Ray-Ban glasses, the prototypes have to operate in an environment full of dust, explosions, and smoke. Adding the computing power and battery life they need also means more weight for soldiers already carrying upwards of 100 pounds. Then the technology has to work in environments without ubiquitous 5G cell connections; powerful computer vision and AI models will need to run locally on the device.

For the Army to want to buy it at scale, “it’s got to work, and it’s got to be pretty seamless,” Wong says. “It’s a high bar.”

Genetic and clinical investigation of insulin-degrading enzyme in Parkinson’s disease within the Chinese Han population

IntroductionGrowing evidence suggests a mechanistic link between type 2 diabetes mellitus and Parkinson’s disease (PD), with insulin-degrading enzyme (IDE) implicated in both insulin and amyloid-β metabolism, as well as α-synuclein degradation. However, the role of IDE in PD pathogenesis remains insufficiently defined. This study aimed to investigate the association of IDE gene polymorphisms and serum IDE levels with sporadic PD in a Chinese Han population.MethodsFourteen single nucleotide polymorphisms (SNPs) within the IDE gene were genotyped in 463 patients with sporadic PD and 576 age- and sex-matched healthy controls (HCs). An independent cohort of 100 PD patients and 100 HCs was used to quantify serum IDE concentrations. Correlations between IDE levels and clinical features were assessed. Logistic regression was employed to identify independent factors associated with PD.ResultsAmong the examined SNPs, rs11187007 showed a nominal allelic association with PD (P = 0.046), which did not survive the Bonferroni correction. Serum IDE concentrations were significantly higher in PD patients than in HCs (P = 0.015). Elevated IDE levels were negatively correlated with Mini-Mental State Examination scores (R = –0.230, P = 0.027) and positively associated with more severe symptoms. Logistic regression indicated that elevated serum IDE levels were associated with PD.ConclusionOur findings highlight that elevated serum IDE correlates with PD, suggesting a role for IDE in neurodegeneration, warranting further mechanistic and longitudinal studies to evaluate its potential as a therapeutic target in PD.

From autophagy–lysosomal deficits to neurodegeneration in Niemann-Pick type C1 disease: implications for age-related neurodegenerative disorders

Niemann-Pick type C1 (NPC1) disease is a neurodegenerative lysosomal storage disorder caused by loss-of-function mutations in the NPC1 gene. NPC1 deficit primarily disrupts lipid homeostasis and subsequently drives cellular degeneration through mechanisms involving impaired autophagy and mitophagy, mitochondrial dysfunction, and, recently demonstrated NAD depletion that links autophagy impairment to neuronal death. Emerging evidence also highlights the activation of innate immune signaling leading to neuroinflammation. In this review, we synthesize current mechanistic insights and describe how these molecular deficits are interconnected to drive neuronal death in NPC1 disease. We also discuss how these pathological processes parallel those observed in major age-related neurodegenerative pathologies such as Alzheimer’s and Parkinson’s disease. Finally, we highlight emerging therapeutic strategies that can potentially ameliorate these cellular deficits, offering avenues for mitigating neurodegeneration in NPC1 disease and other related neurodegenerative disorders.