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Nature Neuroscience: Autism subtypes identified using cross-species functional connectivity analyses

It is often assumed that phenotypic heterogeneity in autism reflects underlying pathobiological variation. However, direct evidence supporting this link is lacking. Leveraging cross-species functional neuroimaging, we show that brain dysconnectivity patterns in autism can be parsed into biologically dissociable subtypes.

The post Nature Neuroscience: Autism subtypes identified using cross-species functional connectivity analyses appeared first on Child Mind Institute.

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FDA Approves Datroway as First-Line Therapy for Triple-Negative Breast Cancer

AstraZeneca and Daiichi Sankyo have received FDA approval for Datroway (datopotamab deruxtecan), an antibody-drug conjugate that offers an unprecedented improvement in survival to a patient population with no treatment options beyond chemotherapy. 

“Datopotamab deruxtecan is the first and only medicine to significantly prolong overall survival in the first-line setting compared to chemotherapy in patients with metastatic triple-negative breast cancer who are not candidates for immunotherapy,” said Tiffany A. Traina, MD, medical oncologist at Memorial Sloan Kettering Cancer Center. “This approval will bring a much-needed treatment option for these patients.”

Datroway is an antibody-drug conjugate (ADC) that targets the TROP2 protein, which is broadly expressed in several types of solid tumors and is associated with an increased risk of tumor progression and lower survival rates in breast cancer patients. With this approval, the ADC is now commercially available in the U.S. for three indications, including HR+/HER2– breast cancer and EGFR-mutated non-small cell lung cancer. Regulators across the world are now reviewing applications to expand this approval to Australia, Canada, China, the EU, Singapore, and Switzerland. 

The FDA decision is based on Phase III results from the TROPION-Breast02 clinical trial, where Traina was one of the investigators. In this trial, Datroway improved the median overall survival by five months and reduced the risk of disease progression or death by 43% compared to chemotherapy as a first line treatment. The ADC also achieved an objective response rate of 64% compared to 30% for chemotherapy. 

“Triple-negative breast cancer is notoriously difficult to treat,” said Dave Fredrickson, executive vice president of the oncology hematology business Unit at AstraZeneca. “Patients with metastatic disease, especially those who are unable to receive immunotherapy, urgently need more effective, durable and tolerable treatment options, which extend survival. With today’s approval, we are proud to bring Datroway to a broad population of advanced triple-negative breast cancer patients and we continue to study its promise as a mainstay treatment across tumors, stages, and settings.”

Accounting for about 15% of all cases of breast cancer, triple-negative breast cancer is the most aggressive type of breast cancer, with only 15% of patients surviving five years after their diagnosis. Because it doesn’t express any of the three most common targets for established breast cancer treatments, patients with triple-negative breast cancer have limited treatment options. 

Those patients whose tumors express PD-L1 are eligible to receive checkpoint inhibitor immunotherapy as a first line treatment in addition to chemotherapy. However, as many as 70% of patients with metastatic triple-negative breast cancer are not eligible for this form of immunotherapy, whether because their tumors do not express PD-L1 or because of comorbidities. 

“For seven out of 10 patients with metastatic triple-negative breast cancer who are not candidates for immunotherapy, chemotherapy has remained the only treatment option,” said Arlene Brothers, executive director of the Triple Negative Breast Cancer Foundation. “Today’s approval of Datroway means that for the first time, these patients will have a new standard of care beyond traditional chemotherapy at the outset of their treatment.”

The post FDA Approves Datroway as First-Line Therapy for Triple-Negative Breast Cancer appeared first on Inside Precision Medicine.

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STAT+: Congress slashed Medicaid funding to providers. The Trump administration wants to cut even further

Hospitals and other providers are bracing for an end to the extra money they’ve gotten for treating Medicaid patients, one of the many cuts contained in Republicans’ sweeping 2025 tax law.

But the Trump administration disclosed this week that it plans to take the cuts to state directed payments even further, setting up what’s likely to be a showdown with provider groups. 

Since 2024, some hospitals, doctors, nursing homes, and other types of providers have been reimbursed for Medicaid services at much higher commercial rates, thanks to a Biden-era change. The One Big Beautiful Bill Act, passed in July, directs the Centers for Medicare and Medicaid Services to gradually trim those payments beginning in 2028 until they’re close to or on par with Medicare rates. 

Continue to STAT+ to read the full story…

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AI Scientists, Stock Tumble, Patent Lawsuit, and New Partnerships

Agentic AI is growing in its applications. Google DeepMind and Edison are leveraging the growing capabilities by developing AI Scientists. These platforms are poised to streamline the scientific process, aiding human scientists with a variety of tasks. Meanwhile, despite positive data in its Phase III DMD therapy trial, Regenxbio’s stock fell for a variety of reasons. 10X Genomics and Harvard University are suing Element Biosciences over patents for a multiomics platform. Finally, Bristol Meyers Squibb is partnering with Hengrui Pharma to develop 13 early-stage programs with the potential to grow their investment to a predicted $15 billion in sales.

Listed below are links to the GEN stories referenced in this episode of Touching Base:

The State of Precision Medicine Summit
Join us June 3, 2026

Google DeepMind and Edison Are Building the AI Scientist
By Fay Lin, PhD, GEN Edge, May 19, 2026

StockWatch: Regenxbio Tumbles Despite Positive Pivotal Data for DMD Gene Therapy Candidate
By Alex Philippidis, GEN Edge, May 17, 2026

10x Genomics, Harvard Target Element’s Multiomics Platform in Patent Lawsuit
By Alex Philippidis, GEN Edge, May 12, 2026

BMS, Hengrui Pharma Partner on 13 Programs in Up-to-$15.2B Collaboration
By Alex Philippidis, GEN Edge, May 13, 2026

Touching Base Podcast
Hosted by Corinna Singleman, PhD

Behind the Breakthroughs
Hosted by Jonathan D. Grinstein, PhD

The post AI Scientists, Stock Tumble, Patent Lawsuit, and New Partnerships appeared first on GEN – Genetic Engineering and Biotechnology News.

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Synaptic alterations are preceding the axonal loss in optic atrophy of Wolfram syndrome mouse model

BackgroundWolfram syndrome is a rare autosomal recessive disorder characterized by antibody-negative early-onset diabetes mellitus, optic atrophy, sensorineural hearing loss, arginine-vasopressin deficiency, and progressive neurodegeneration of the brainstem and cerebellum. It is caused primarily by pathogenic variants in the WFS1 gene, which encodes a transmembrane endoplasmic reticulum–resident protein involved in the unfolded protein response and cellular calcium homeostasis. Although multiple rodent models of Wolfram syndrome have been developed and shown to exhibit visual defects, some studies have reported significant vision loss prior to any detectable axonal degeneration or myelin abnormalities, and the mechanisms underlying these early visual deficits remain poorly understood.ObjectiveRecent in vitro studies have demonstrated altered synaptic contacts and aberrant neurite morphology in WFS1-deficient cerebral organoids and human iPSC-derived neurons, respectively. These findings prompted us to investigate, for the first time in vivo, whether synaptic and dendritic abnormalities occur in the retina of Wfs1 knockout mice.MethodsUsing confocal microscopy, we examined retinal and optic nerve histology in Wfs1 knockout mice at 4 and 7 months of age.ResultsOur analysis reveals progressive synaptic alterations in the inner plexiform layer, driven by early presynaptic compartment failure. These changes represent the earliest detectable phenotype associated with vision loss in this model and precede overt axonal degeneration.ConclusionThese findings identify early synaptic preservation as a promising therapeutic target for vision loss in Wolfram syndrome.
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The triple-hit hypothesis of Alzheimer’s disease: blood–brain barrier breakdown, infection, and neuroimmune activation as a unified etiological framework

Studies of Alzheimer’s disease (AD) have long been dominated by the amyloid cascade hypothesis, although mounting evidence suggests that amyloid-β (Aβ) deposition might be a late downstream event, rather than the initiating trigger of AD. Here, I propose a unifying Triple-Hit Hypothesis in which AD develops through a sequential interaction among three causative processes that have been individually implicated before in the onset of Alzheimer’s disease: (1) early blood–brain barrier (BBB) breakdown, (2) entry or reactivation of microbial agents within the brain, and (3) maladaptive innate immune responses that produce chronic neuroinflammation and Aβ accumulation. Converging data from human imaging, neuropathology, infectious disease studies, genetics, and vascular medicine could suggest that these three processes, which were in some previous studies linked to AD, might not be independent but rather that their temporal synergy could drive disease progression. This new conceptual framework integrates long-standing but fragmented lines of evidence and, if confirmed by experimental studies, offers the possibility for the development of new diagnostic biomarkers, new therapeutic entry points, and prevention strategies for AD. I argue that understanding AD as a disorder of the blood-brain barrier, immunity, and host–pathogen interactions should be taken into account in future research on the etiology and clinical progression of AD.
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Reading speed, visual deficits, and cerebral white matter integrity in veterans with and without mild traumatic brain injury

IntroductionSince 2001, approximately 17.3% of enlisted personnel have experienced a traumatic brain injury (TBI) according to the United States military. Visual deficits (e.g., convergence insufficiency or pursuit abnormalities) are reported as chronic, persistent symptoms of TBI, which can impact daily activities such as reading, computer work, and driving.MethodsIn the present study, diffusion-weighted imaging (DWI) data and behavioral and survey data related to visual function were analyzed for 63 combat veterans with and without mild TBI (mTBI). We also tested the hypothesis that white matter damage, measured as either decreased fractional anisotropy of white matter or “potholes” evident in the DWI data, would predict visual behaviors (reading speed, smooth pursuit catch-up saccades, and/or convergence insufficiency).ResultsOur key finding is that scores on the Convergence Insufficiency Symptom Survey (CISS) predicted whether the use of a color overlay would increase reading speed for participants with mTBI, but not for control participants. General linear model analyses found a relationship between smooth pursuit catch-up saccades and the cumulative number of white matter “potholes” found in white matter across the cerebrum. However, the sample size was too small to conclude that these correlations were uniquely related to TBI status.DiscussionThese findings point toward the importance of additional research to determine exactly how mTBI is associated with reduced reading speed and why altering the color of the page improves performance for individuals with mTBI and convergence insufficiency.
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Beyond the auditory: anxiety bridges sleep disturbances and depressive symptoms to tinnitus handicap

BackgroundTinnitus is closely associated with psychological factors including anxiety, depression, and sleep disturbances. However, how these variables interact to influence tinnitus severity remains poorly understood, warranting further exploration.MethodsThis retrospective study analyzed data from 285 patients with tinnitus, utilizing the Tinnitus Handicap Inventory (THI), Self-Rating Depression Scale (SDS), Self-Rating Scale of Sleep (SRSS), and Self-Rating Anxiety Scale (SAS) as assessment instruments. Univariate and multivariate regression analyses were performed to identify psychosocial factors associated with THI scores. Structural equation modeling (SEM) was employed to examine the mediating role of anxiety—specifically, whether sleep disturbances and depression exacerbate tinnitus handicap indirectly through heightened anxiety. Subgroup analyses were further conducted to evaluate the stability of this psychological mediation pathway across different demographic and clinical subgroups (e.g., age, gender, disease duration).ResultsTinnitus severity was significantly positively correlated with scores on the SRSS, SDS, and SAS (all P < 0.05). Multivariate regression analysis identified age, sleep quality (SRSS), and anxiety (SAS) as independent predictors of increased Tinnitus Handicap Inventory (THI) scores. Structural equation modeling further confirmed that SAS partially mediated the relationship between SRSS and THI (mediation effect: 31.7%) and fully mediated the relationship between SDS and THI. Subgroup analyses revealed that the mediating effect of anxiety was more pronounced in middle-aged and elderly individuals, females, and patients with left-sided tinnitus.ConclusionAnxiety serves as a central mediating mechanism linking sleep disturbances and depressive symptoms to tinnitus severity. The present findings demonstrate that anxiety not only directly exacerbates tinnitus-related handicap but also mediates the influence of sleep and mood disturbances on tinnitus distress. These findings underscore the critical role of anxiety in personalized tinnitus treatment.