Stem Cell Therapy Advances Toward Clinical Trial for Chronic Spinal Cord Injury

A stem cell therapy designed to restore function by repairing damaged nerve pathways is moving closer to clinical testing for people with chronic spinal cord injury (SCI), according to research presented at the International Society for Stem Cell Research (ISSCR) 2026 Annual Meeting.

The work, presented by Hideyuki Okano, MD, PhD, of Keio University in Japan, builds on more than a decade of research into regenerative therapies for SCI. Okano’s team pioneered the development of induced pluripotent stem cell (iPSC)-derived neural stem/progenitor cells and launched the world’s first clinical study of the approach in patients with subacute SCI.

In March 2025, the researchers announced one-year follow-up results from that four-patient first-in-human study, reporting no serious treatment-related adverse events and neurological improvements in two participants, including one who regained the ability to stand independently and begin gait training. Although those findings have not yet been published in a peer-reviewed journal, they provided the safety foundation for expanding the program to chronic SCI.

The physician-initiated trial, expected to begin recruiting patients in 2027, will enroll individuals with chronic, incomplete SCI who retain inactive but demyelinated nerve fibers. Unlike patients treated soon after injury, these individuals often have surviving nerve pathways that have lost the myelin insulation needed for efficient signal transmission.

“We have already successfully completed a world-first, first-in-human clinical study targeting patients in the subacute phase, which demonstrated a promising safety profile,” Okano said. “Our shift to the chronic phase is the next logical milestone, built upon that solid foundation. Since the cellular environment changes over time, we are evolving our strategy from just establishing safety to actively overcoming the stubborn, long-standing barriers of chronic paralysis.”

The biology of chronic injury also required a different cell therapy. While the earlier program used neural stem/progenitor cells capable of generating neurons, the new approach uses clinical-grade gliogenic neural stem/progenitor cells (gNS/PCs), which preferentially develop into astrocytes and oligodendrocytes—the support cells responsible for maintaining neurons and restoring myelin around surviving nerve fibers. Rather than attempting to regenerate entirely new neural circuits, the therapy aims to repair and restore the function of existing ones. Earlier preclinical studies demonstrated that these gliogenic cells promoted remyelination, preserved host axons, and improved locomotor recovery in animal models, providing the rationale for advancing the therapy toward human testing.

The latest preclinical findings further support that strategy. The researchers reported that the clinical-grade gNS/PCs efficiently differentiated into neurons, astrocytes, and oligodendrocytes in laboratory studies. Following transplantation into animal models of chronic SCI, the cells promoted behavioral recovery without evidence of tumor-like tissue formation while remodeling the injured spinal cord microenvironment.

The post Stem Cell Therapy Advances Toward Clinical Trial for Chronic Spinal Cord Injury appeared first on Inside Precision Medicine.

Mung Bean Pan-Genome Study Maps Key Genes for Yield, Nutrition, and Pest Resistance

Because Mung beans (Vigna radiata) are nutritious, inexpensive, have nitrogen-fixing capacity, and are relatively easy to grow with a short growing cycle, they are an important crop for food security in many parts of Asia, Africa, and other regions. Now, researchers have made a significant contribution to a landmark international study that has uncovered tens of thousands of previously hidden structural variations influencing yield, nutritional quality, insect resistance, and other relevant mung bean traits.

The study entitled, “Graph-based pan-genome reveals structural variations associated with agronomic traits in mung bean,” published in Nature Genetics, presents the world’s first graph-based pan-genome for the pulse crop (a legume grown specifically for its dry edible seeds) offering a comprehensive resource for understanding the genetic basis of key agronomic traits and accelerating crop improvement.

The international research team, co-led by researchers at the Chinese Academy of Agricultural Sciences and Murdoch University’s Centre for Crop and Food Innovation (CCFI), assembled chromosome-scale genomes from genetically diverse mung bean accessions and analyzed genomic variation across 580 global accessions. The resulting graph-based pan-genome captures more than 75,000 gene families and identifies over 66,000 structural variants, offering important insights that will help breeders target key agronomic traits and accelerate crop improvement.

More specifically, the authors note that, “integrating these structural variants and single nucleotide polymorphisms, genome-wide association studies across five environments identified candidate genes for 20 agronomic traits, underscoring the pivotal roles of these variants in driving mung bean domestication and improvement.”

Mechanistically, they add, the work demonstrates that “a 68-bp promoter insertion in VrTIFY6B and a 136-bp promoter deletion in VrPGIP1 regulate flavonoid content and confer bruchid resistance, respectively.”

In Australia, mung bean generates over $100 million annually in export revenue. At roughly three times the price of wheat, it represents a highly profitable break crop opportunity for Australian growers; however, seasonal rainfall variability continues to drive significant year-to-year swings in the size and value of the crop.

By cataloging tens of thousands of previously invisible structural variations and linking them to agronomic traits through genome-wide association analysis, this new genomic resource gives breeders a far more complete map of the genetic variation they can work with.

Globally, where mung bean underpins the diets and incomes of millions of smallholder farmers across Asia and Africa, the study’s findings on genes governing seed nutritional compounds and resistance to bruchids, a major storage pest, have direct implications for global food security.

Rajeev Varshney FRS FAA, CCFI director, said the research represents a major advance in crop genomics and demonstrates how next-generation genomic technologies are transforming plant breeding. “Traditional reference genomes capture only part of the genetic diversity within a crop species. By constructing a graph-based pan-genome, we can now identify structural variations that were previously invisible but often have profound effects on important agricultural traits.

“These discoveries provide breeders with powerful new genomic tools to accelerate the development of higher-yielding, more nutritious and climate-resilient mung bean varieties,” Varshney continues. “The genomic resources generated through this work will support marker-assisted breeding, genomic selection and genome editing, enabling breeders to deliver improved varieties to farmers much faster.”

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What Anthropic’s latest AI discovery does—and doesn’t—show

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

Anthropic—currently the world’s most valuable AI company, with a nearly $1 trillion valuation—has a reputation for publishing strange and heady research. It’s looking into whether AI models can feel pain, for example, and will sometimes cut off chatbot conversations if it suspects users are “abusing” the model. 

One niche that Anthropic spends more time and money on than other AI companies is called mechanistic interpretability, which means looking inside the complex math of an AI model to learn why it comes up with one particular output and not another. It’s complicated stuff; there are millions of data points that might contribute to any result, and wading through them can look more like word salad than anything useful. It’s also controversial. Describing AI models with terms borrowed from psychology and neuroscience can make their behavior seem more sophisticated than we might otherwise judge it to be.

That’s why, when Anthropic announced last week that it had found a new window into its models’ “internal thoughts” as they reason through answers, there was one colleague I had to talk to. Senior editor Will Douglas Heaven, aside from having a PhD in computer science, has spent a lot of time digging into what we can say about how AI models work. I spoke with him about what we should take from Anthropic’s new (and predictably quirky) research.

What did Anthropic learn here, exactly?

Anthropic has been trying to understand how large language models (LLMs) work for a few years now. Anthropic isn’t the only one looking at this, but I think the company has made it part of its core mission more than most. Anthropic’s CEO, Dario Amodei, has said we won’t be able to control LLMs fully unless we learn more about how they work. 

So this new research is very much in that context. It goes deeper into the weird mechanisms inside LLMs than ever before. What Anthropic learned was that LLMs have a space inside them—which Anthropic calls the J-space—filled with words that don’t appear in their output but that seem to influence the way they puzzle through problems. All this was hidden until Anthropic developed a new technique to probe its model Claude, so it’s a genuine discovery. 

Sometimes these words keep track of where the LLM has got to in a particular task, sometimes they look more like flashes of recognition (for example, “protein” might pop up when you give an LLM only the letters of a protein sequence), and sometimes they represent a kind of internal commentary on the model’s decision-making. In my favorite example, Claude decided to cheat on a coding test when the word “panic” appeared.

Anthropic also found that LLMs are able to describe and manipulate the words in this space. So somehow they seem to be making use of it. 

Let’s step back for a second. I don’t think of large language models as simple, but they’re also not magic. There’s a bunch of math that learns relationships between words, right? So why is it so hard to “peer” into an LLM to know what’s going on?

Yeah, they’re not magic! I think the fact we don’t fully understand them plays into the mythmaking. And it’s worth noting that the whole narrative that Anthropic is leaning into here—that they’ve built this really mysterious technology, but don’t worry, because they’re also the ones to figure it out—very much fits with the company’s vibe. [See how Anthropic warned that its new models were so good at coding they posed a global cybersecurity risk, only for the US government to shut them down shortly thereafter.]

So yes: LLMs are just math. And yet it’s vastly complex math. Not only are today’s LLMs made out of hundreds of billions of numbers, but running them triggers a cascade of millions and millions of calculations. I wrote last year that if you printed out even a medium-size LLM on pieces of paper, it would cover a city the size of San Francisco. 

It’s impossible to make sense of any of that math without specialist tools that highlight specific parts of an LLM at specific times. You need to know where to look and how to look. And building those tools requires understanding something of that complex math in the first place. 

You’ve written elsewhere about this concept of studying LLMs the way one might study an organism’s brain. Is it fair to use “brain-like” terms when talking about how an LLM works?

I don’t love using those kinds of terms. LLMs are not brains. Talking like this is misleading because it can suggest that LLMs are capable of more human-like things than they are or that we can make assumptions about how they might behave that we shouldn’t. The whole anthropomorphization thing is also tied up with a bunch of strong ideological positions about what this technology is and what it’s going to be. 

But at the same time, we lack a good alternative vocabulary for talking about what these models are doing. I can understand why people reach for words like “think” and “understand” and “brain-like”—they’re convenient shorthand. 

Anthropic compares this new space it found inside LLMs to the space that some neuroscientists think our brains use to keep track of conscious thoughts. I asked the company how seriously we should take that comparison and it said in a statement: “Drawing these analogies was helpful to us in designing our experiments, as they allowed us to make many non-obvious experimental predictions about the J-space that turned out to be true. At the same time, it’s important to note that there are some important differences between the J-space (and language models in general) and the human brain, so we don’t mean to claim there’s a perfect correspondence.” 

What’s a problem in AI that this new concept of the J-space might be used to solve?

Anthropic has said that monitoring the J-space could be a way to catch models doing something they shouldn’t. Because words pop up in this space that don’t appear in a model’s output, they can tell you things about its behavior that you might not have noticed otherwise—such as when it is giving biased responses or when it is weighing the pros and cons of cheating. 

That’s the theory, at least. I think it’s better to think of this result as one more step on the path to understanding this technology overall than as something that will be useful by itself. 

Read more in Will’s full story about the new research. 

STAT+: Dementia study replicates promising outcomes following risk-reduction strategies

This is the web edition of STAT’s AAIC in 30 newsletter. Sign up to get it in your inbox here.

Hi there from Day 2 of AAIC. There may be 10,200 people here at the Alzheimer’s Association International Conference — up 1,700 from last year — from 115 countries, as the association’s Maria Carrillo announced at Monday’s plenary session. But by far my favorite person is the woman I saw taking a nap on a bench in a corridor, her sunhat pulled over her eyes. You are an inspiration.

One housekeeping note: Due to the vagaries of newsletter scheduling, we won’t have an edition tomorrow, but will be back in your inbox with a wrap-up edition on Wednesday. Keep your eyes on statnews.com tomorrow though and expect some conference news around 9:15 a.m. Eastern.

Latin American study praised as ‘landmark’ effort

For a disease that’s proven as intractable to treat as Alzheimer’s, it’s been known for quite some time what factors can heighten one’s risk for dementia. Poor nutrition, sleep, and cardiovascular health; a lack of physical activity and social engagement; and lower education levels all increase the likelihood that a person will develop Alzheimer’s.

A study reported at last year’s AAIC put interventions designed to reduce risk to the test. The U.S. Pointer study found that an intensive, structured program that encouraged improved diet and exercise, cardiovascular health monitoring, and other steps helped protect cognitive function among older adults at risk of dementia to a greater extent than a lower-intensity program that participants navigated on their own, even as both groups saw benefits. The study underscored that healthy habits contribute to a healthy brain.

Continue to STAT+ to read the full story…

Immune Evasion Uncovered by Virome-Wide Ubiquitin Ligase Discovery

In a new study published in Science titled, “Virome-wide ubiquitin ligase discovery reveals diverse mechanisms of immune evasion,” researchers from Harvard Medical School (HMS) have uncovered how viruses hijack cells’ garbage-disposal systems to evade immune attack. 

The study applies ORFeome, a tool that broadens the scale by which researchers study viral proteins. The advance proposes to speed basic discoveries in virology, inform the development of new vaccines and treatments to protect against emerging pathogens. 

“This library reveals how viruses manipulate human cells on a scale that simply wasn’t possible before,” said Stephen Elledge, PhD, professor of genetics and medicine at Harvard Medical School and senior author of the study. “We believe it changes virology from studying one virus at a time to discovering the common strategies and surprising innovations that viruses have evolved, providing a powerful new foundation for understanding emerging viral threats.” 

ORFeome and other ORF libraries are named after open reading frames, DNA sequences that encode proteins. Previous viral ORF libraries from other groups focused on individual viruses or virus families that contained 100 or 200 sequences each. The new ORFeome contains about 13,000 physical DNA sequences, or constructs, that code for about 9,000 proteins from 513 different viruses, including Andes hantavirus, Ebola virus, and Zika virus. 

“Most viruses have never been studied in detail, yet evolution has already performed countless experiments for us. This library gives us a way to read the results of those experiments across the viral world,” said Elledge, who is also a Howard Hughes Medical Institute (HHMI) Investigator. 

The team attached a genetic barcode to each ORF, allowing researchers to conduct studies of all 13,000 ORFs at once. 

“We can insert the sequences into a population of cells, ask questions like which ones cause the cells to grow better or less, and then identify those by their barcodes when the experiment is finished,” said Colin O’Leary, PhD, HMS research fellow and co-author on the study. “It hasn’t been possible before to do genetic screens like this with viral proteins.” 

The team will make the ORFeome freely available for the research community. Elledge and colleagues implemented a flexible design to enable application to other model systems and experiments. 

The team conducted genetic screens in three cell types, searching for viral proteins that affect cell proliferation, stop cells from presenting antigens to trigger the immune system to attack, or block the effects of interferon. Results uncovered more than 700 viral proteins that contribute to at least one of those actions. 

The study opens opportunities to design drugs that hinder viral activity while sparing normal function. 

The post Immune Evasion Uncovered by Virome-Wide Ubiquitin Ligase Discovery appeared first on GEN – Genetic Engineering and Biotechnology News.

Evaluating the detection of small brain lesions in magnetic resonance using deep learning

IntroductionDetecting brain lesions using Artificial Intelligence methods has been a focus of prior research, with numerous datasets supporting this task to improve clinical results. However, evaluation metrics and reported results often summarise overall performance without considering variations in lesion size. In clinical practice, the detection of small tumours is particularly critical for early diagnosis and treatment effectiveness.MethodsThis study evaluates the performance of Artificial Intelligence models on established datasets with a specific focus on lesion identification and lesion size. We introduce a novel Deep Learning model tailored to detect small brain tumours in Magnetic Resonance Imaging, integrating a clinically defined “small tumour” concept into both the training and evaluation processes.ResultsThe proposed approach demonstrates robust performance, achieving loss values ranging from 1.5 to 11.1 and Dice Scores between 96.3% and 98.1% across multiple datasets.DiscussionThe main contribution of this work is the incorporation of clinically meaningful lesion-size information into model development and assessment. These findings suggest that explicitly considering small tumours can improve the clinical relevance of Artificial Intelligence systems for brain lesion detection and support earlier diagnosis and more effective treatment planning.

Nucleus accumbens DRD2 receptor agonism attenuates escape behavior

Animals learn to approach and escape stimuli in their environment, in part through the representation of rewarding or aversive outcomes in the nucleus accumbens (NAc). The regulation of reward motivation in the NAc by dopamine signaling at DRD1 and DRD2 receptors has been the subject of extensive study. However, the process by which aversive stimuli are signaled within this system to promote motivated escape behavior is less well characterized. Conventional wisdom posits that rewarding and aversive stimuli ultimately affect DRD1 or DRD2-receptor expressing medium spiny neurons (MSNs) in an opposing manner to differentially modulate motivated behavior. However, recent studies have challenged this view and demonstrate the need to better characterize the processes that mediate aversion learning. To determine if DRD2 dopamine receptor activation disrupts escape behavior, 21 male and female Sprague Dawley rats were treated with an intra-NAc core DRD2 receptor agonist, quinpirole, while escape behavior was negatively reinforced by the termination of aversive white noise. This treatment attenuated escape, a result that is consistent with the view that aversion-induced reductions in dopamine promote escape behavior through decreased DRD2 receptor signaling in the NAc, and potential disinhibition of an aversion-sensitive striatal output circuit.

Attention-deficit/hyperactivity disorder and chronic pain: a scoping review of epidemiology, clinical phenotypes, mechanisms, and treatment

IntroductionEmerging evidence suggests a potential association between attention-deficit/hyperactivity disorder (ADHD) and chronic pain; however, extant findings are dispersed across disciplines and have not yet been comprehensively synthesized. This scoping review aimed to systematically map the epidemiological evidence, clinical phenotypes, proposed neurobiological mechanisms, and reported therapeutic interventions related to comorbid ADHD and chronic pain.MethodsA comprehensive literature search was conducted in PubMed, PsycINFO, and the Cochrane Library from database inception to December 28, 2025. Human studies examining the association between ADHD (diagnosed or symptom-defined) and chronic or recurrent pain were also included. Fifty studies met the eligibility criteria, including observational studies (cross-sectional and longitudinal), case reports and series, and one interventional study.ResultsEpidemiological studies have consistently reported significant associations between ADHD symptoms or diagnoses and chronic pain in both the general population and clinical samples. Higher ADHD symptom burden was associated with greater pain severity and pain-related functional impairment. Comorbidity was observed not only in widespread pain syndromes, such as fibromyalgia, but also in site-specific conditions, including chronic low back pain, orofacial pain, and migraine. Proposed mechanisms involve dopaminergic and noradrenergic dysfunction affecting motor regulation, sensory processing, and descending pain modulation systems. Several case-based reports have described improvements in pain outcomes after ADHD-targeted pharmacotherapy. However, controlled trials remain scarce.DiscussionCurrent evidence suggests that ADHD traits may be relevant in a subset of individuals with chronic pain, particularly those with treatment-resistant presentations. Although causality and treatment efficacy remain unconfirmed, consideration of neurodevelopmental characteristics may enhance clinical assessments and inform future research on individualized mechanism-based treatment strategies.

Family caregivers’ involvement in home-based recovery for patients with schizophrenia: a qualitative study in Beijing, China

BackgroundFamily caregivers play critical roles in supporting the home-based recovery of patients with schizophrenia, but they often encounter substantial challenges and receive insufficient systemic support. Understanding caregivers’ involvement in home-based recovery is essential for aligning community mental health services with families’ capacities and needs. This study aimed to explore family caregivers’ involvement and adaptive processes in supporting home-based recovery of patients with schizophrenia in China.MethodsA qualitative study using interpretative phenomenological analysis was conducted through semi-structured interviews. Family caregivers were purposively recruited from four community health service centers (CHSCs) across urban and rural areas of Beijing. All interviews were audio-recorded, transcribed verbatim, anonymized, and analyzed iteratively to identify themes and subthemes.ResultsA total of 20 family caregivers were recruited, including 11 from two urban districts and 9 from a rural district in Beijing. Five themes were identified: Caregivers’ redefinition of recovery as stability rather than cure; Routine recovery involvement in medication management and symptom monitoring; Experienced tensions between the patient’s independence and relapse prevention; Bearing family obligation and personal strain in sustained caregiving involvement; and Uncertainty in sustaining caregiving and the patients’ future stability. Caregivers reported persistent challenges in supporting patients’ independent living, participation in family activities, communication, and social interaction.ConclusionFamily caregivers gradually develop their capacity to support home-based recovery, but continue to encounter complex challenges while receiving limited support from CHSCs. Strengthening recovery-oriented family support within community mental health services, particularly through accessible psychoeducation and rehabilitation guidance, may enhance caregivers’ capacity to support patients’ independence and social participation, thereby promoting sustainable home-based care and long-term functional recovery.

Case Report: A novel de novo heterozygous truncating mutation in MED12L identified in a Chinese autistic boy

BackgroundAutism spectrum disorder (ASD) is a highly heterogeneous neurodevelopmental disorder. A previous study by Nizon et al. indicated that some children with intellectual disability (ID) carrying de novo MED12L mutations exhibited mild to moderate autistic features. However, the relationship between MED12L and ASD remains unclear.Case presentationHere we reported a male child with severe autistic features carrying a novel de novo heterozygous truncating mutation of MED12L (NM_053002.5:c.586C>T, p.(Arg196Ter)). He was diagnosed with ASD according to ICD-11 and DSM-5 criteria. Clinical examination indicated that this child exhibited severe autistic features and several dysmorphic features, including a flat nasal bridge, bulbous nasal tip, thin upper lip, and triangular face. Magnetic resonance imaging (MRI) of the brain revealed an enlarged perivascular space in the right temporal lobe.ConclusionThis case demonstrates that this de novo heterozygous truncating mutation in MED12L may be involved in the development of ASD, and haploinsufficiency of MED12L may be associated with severe autistic features. Obvious clinical manifestations and dysmorphic features in this child with a truncating mutation in MED12L expand the phenotypic spectrum of MED12L-related cases and warrant further functional studies to elucidate the relationship between MED12L and ASD.