Tag: News Approved

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STAT+: Biotech raises $42 million to run Huntington’s disease trial

Gene therapy startup Latus Bio has raised another $42 million to start its first clinical trials, where it will try to sidestep issues that have set back a more advanced competitor. 

Latus is moving two treatments through clinical trials this year. The first is for a form of Batten disease called CLN2 disease, a fatal genetic condition that causes seizures, vision loss, and cognitive problems. The company anticipates having initial clinical data by the end of the year. 

Now, Latus — founded by Beverly Davidson, chief scientific strategy officer at the Children’s Hospital of Philadelphia — is turning its attention to a second drug candidate, a gene therapy for Huntington’s disease.

Continue to STAT+ to read the full story…

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Immigration changes are driving foreign researchers to leave the U.S. — or not come to begin with 

The budding scientist had left India for the U.S. for her Ph.D., because as she saw it, no other country offered the same opportunities for researchers. Set to finish her doctorate this summer, she also had a postdoctoral fellowship lined up in America.

Now those plans have changed. New and intrusive burdens for renewing a component of her visa — which required her to make public her social media profiles for U.S. review while she was back in India — caused her to be away from the lab for two months during the crunch time of wrapping up her degree. 

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Opinion: STAT+: China’s strict new supply chain regulations could create massive problems for Western biopharma companies

A recently published paper delivers a finding that should be well known to every executive in Western pharma: China has gone from accounting for less than 8% of global clinical trials in 2010 to surpassing the United States in annual registered trial volume by 2020, reaching more than 5,000 trials per year in 2024. Eighty-eight percent of that growth, among private-sector sponsors, was driven by domestic Chinese firms, not multinationals relocating R&D.

China is not just a manufacturing base for the West’s drug supply chain. It is now a peer competitor in drug innovation — and it intends to stay that way on its own terms.

Toward that end, on April 7, China’s State Council issued Decree No. 834, the Regulations on Industrial and Supply Chain Security, effective immediately, with no transition period. Its 18 articles give Beijing sweeping new powers to investigate and sanction any foreign company whose commercial decisions are deemed to harm China’s industrial chain security. China’s 15th five-year plan has explicitly designated biotechnology and pharmaceuticals as the centerpiece of its next phase of industrial development. Decree No. 834 is the legal infrastructure through which Beijing intends to protect and leverage that ambition.

Continue to STAT+ to read the full story…

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Manual therapy ameliorates neuromuscular dysfunction in spastic model rat: involvement of the C-Fiber-mediated CaMKII pathway

BackgroundThis study investigated whether manual therapy applied to tendon organs ameliorated neuromuscular dysfunction in rats with spasticity induced by upper motor neuron injury associated with spastic cerebral palsy, and analyzed the potential involvement of the C-fiber-mediated CaMKII signaling pathway.MethodsMale rats were used to establish palsy models and divided into groups: Control, Model, Manual Therapy (MT), Capsaicin Treatment, Sham, CaMKII Inhibitor, and DMSO Solvent groups. Except for Control, all underwent pyramidal-tract destruction. After modeling, the MT group received manual therapy on the left-lower leg tendon organs. The Capsaicin group underwent sciatic nerve capsaicin treatment for C-fiber block on days 2 and 7; the Sham group had sciatic nerve exposure only. Both received daily manual therapy intervention for 14 days. The CaMKII Inhibitor and DMSO Solvent groups received intrathecal injections every 2 days (7 times total) without manual intervention. Spasticity-related behavioral indices, molecular expression, and neurotransmitter levels were assessed.ResultsManual therapy reduced the neurological deficit scores and muscle spasticity scores of model rats, improved the pathological morphology of the pyramidal tract and skeletal muscle, and regulated the expression of key molecules and neurotransmitters in the spinal cord and hippocampus. The therapeutic effects of manual therapy were significantly attenuated after C-fiber blockage, and although CaMKII inhibition could partially mimic the neuromodulatory effects of manual therapy, its efficacy in alleviating spasticity was inferior to that of manual-therapy intervention.ConclusionManual therapy appears to regulate CaMKII signaling via C-fiber afferent pathways to ameliorate neuromuscular dysfunction in a rat model of spasticity induced by pyramidal-tract lesion, thereby providing experimental evidence for the clinical application of optimized manual therapy parameters in the management of spasticity in patients with cerebral palsy.
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Sex as a biological variable in amblyopia: implications for developmental plasticity and treatment

Amblyopia is a common childhood visual disorder caused by abnormal visual experience that drives visual cortical plasticity during the sensitive period. The timing and forms of treatment with patching therapy and other therapeutic interventions have been extensively studied; however, sex has not been a primary focus in studies examining amblyopia. This mini-review synthesizes evidence for sex differences across levels of analysis, from typical visual development to animal models of amblyopia and human studies of amblyopia incidence and treatment. In addition, it highlights latent sex differences in plasticity mechanisms that may provide insights for future visual neuroscience studies of amblyopia. These findings highlight a key concept: visual outcomes may appear similar yet depend on different mechanisms in females and males, potentially influencing the durability of recovery. We discuss a framework to advance a sex-aware research pipeline for amblyopia, spanning basic, preclinical, and clinical/translational research.
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Altered glymphatic function in nasopharyngeal carcinoma following radiotherapy: novel insights from choroid plexus volume and free-water fraction analyses

Background and purposeRadiotherapy (RT) often causes delayed radiation-induced brain injury (RBI) with unclear pathophysiology; emerging evidence links this to glymphatic dysfunction, but radiation effects on cerebrospinal fluid (CSF) dynamics and interstitial fluid-CSF exchange are unstudied. Thus, we used choroid plexus (CP) volume and free-water fraction (FWF) imaging to assess glymphatic changes in Nasopharyngeal carcinoma (NPC) patients after RT.Materials and methodsIn this cross-sectional cohort of 101 NPC patients (45 pre-RT and 56 post-RT) underwent 3 T MRI, including T1-weighted and diffusion tensor imaging. Automated CP segmentation and tract-specific FWF analysis are performed. Spearman correlation models assessed radiation-dose relationships with CP volume and Whiter matter (WM) FWF.ResultsWe observed that post-RT patients exhibited significant bilateral CP enlargement (total CP: 2560.56 ± 636.72 mm3, left: 1196.92 ± 334.53 mm3, right: 1363.64 ± 365.84 mm3; all p < 0.05) and elevated FWF in critical WM tracts, including the pontine crossing tract (PCT), bilateral corticospinal tracts (CST), middle cerebellar peduncle, right inferior cerebellar peduncle, and left medial lemniscus. Radiational dose exhibit strong dose-dependent correlations with CP volume and WM FWF. Maximum doses to the brainstem (MDRT_BS) and left temporal lobe (MDRT_LT) showed the strongest associations: MDRT_LT correlated with left CP volume (r = 0.599, p < 0.001), right CP volume (r = 0.585, p < 0.001), and bilateral CST FWF (left: r = 0.414, p = 0.005; right: r = 0.354, p = 0.017). CP volume positively correlated with FWF in the PCT and CST (left CST vs. total CP: r = 0.374, p = 0.011). These associations remained significant after adjusting for age, gender, and intracranial volume (r = 0.31–0.58, all p < 0.05).ConclusionThe observed association between choroid plexus enlargement and elevated white matter free-water fraction suggests RT-associated glymphatic dysfunction in NPC, offers a novel perspective on the pathogenesis of RBI.
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Analysis of risk factors for tic disorders in children in Hubei region based on liquid chromatography-tandem mass spectrometry

ObjectiveThis study aims to investigate the correlation between trace element concentrations, 25-hydroxyvitamin D [25(OH)D] levels, and the severity of tic disorders (TD) in children from the Hubei region. Additionally, it seeks to explore the interrelationships among these monitored indicators to provide a reference for the clinical diagnosis and treatment of pediatric TD.MethodsA retrospective review was conducted on the medical records of 237 children diagnosed with TD (TD group) and 137 healthy controls, admitted to the Department of Neurology at Hubei Maternal and Child Health Care Hospital. The TD group was further divided into mild and moderate-to-severe subgroups based on the Yale Global Tic Severity Scale scores. General clinical data were collected, and serum trace element levels were measured using inductively coupled plasma mass spectrometry (ICP-MS), while serum 25(OH)D levels were quantified via liquid chromatography–tandem mass spectrometry (LC–MS/MS). Group comparisons, Spearman correlation analysis, and univariate/multivariate logistic regression analyses were performed.ResultsThe findings indicated that serum 25(OH)D levels were significantly lower in children with TD compared to controls (p < 0.001). Logistic regression analysis demonstrated that 25(OH)D was an independent protective factor against tic disorders.(p < 0.001). Compared to the control group, children with TD exhibited significantly lower levels of calcium and copper (p < 0.001), along with higher levels of iron and cadmium (p < 0.01). These differences were more pronounced in the subgroup of children older than 6 years. An age-stratified subgroup analysis revealed no significant differences in any other indicators except for cadmium between the TD and control groups among children aged 6 years or younger (p > 0.05). Spearman correlation analysis demonstrated that within the TD group, 25(OH)D levels had the most significant correlations with calcium and copper (p < 0.001). No statistically significant differences were observed in the levels of the 10 trace elements or 25(OH)D between the mild and moderate-to-severe TD subgroups (p > 0.05).ConclusionChildren with Tic Disorders in Hubei Province demonstrate a distinctive alteration in their micronutrient profile, primarily characterized by a deficiency in 25(OH)D, alongside reduced levels of calcium and copper, and elevated levels of iron and cadmium. This association is particularly pronounced in male children over the age of six. While 25(OH)D deficiency is identified as an independent risk factor for TD, its concentration does not significantly correlate with the severity of the disorder.
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Excessive Internet use and depressive symptom levels in adolescents with depressive disorders: chain mediation of social anxiety and sleep quality

BackgroundAdolescents with depressive disorders are at elevated risk for adverse mental health outcomes, and excessive Internet use has been increasingly linked to greater symptom severity. Therefore, this study aimed to examine the chain mediating roles of social anxiety and sleep quality in the association between excessive Internet use and depressive symptoms among adolescents with depressive disorders.MethodsA cross-sectional design was used. A total of 266 Chinese adolescents with clinically diagnosed depressive disorders (M = 15.79 years, SD = 1.85; 71.4% female) were assessed using the Internet Addiction Test, Zung Self-Rating Depression Scale, Social Anxiety Scale for Children, and Pittsburgh Sleep Quality Index. Correlation analyses and bootstrapping methods were conducted using SPSS and the PROCESS macro to examine the chain mediating effects of social anxiety and sleep quality.ResultsThe total indirect effect of excessive Internet use on depressive symptoms accounted for 65.66% of the total effect. Specifically, the indirect effects via social anxiety and sleep quality accounted for 24.10% and 26.51% of the total effect, respectively. In addition, the chain mediating effect of social anxiety and sleep quality was significant, accounting for 14.76% of the total effect.ConclusionExcessive Internet use was positively associated with more severe depressive symptoms among adolescents with depressive disorders, both directly and indirectly through the chain mediating effects of social anxiety and sleep quality. These findings highlight potential targets for preventing and intervening in excessive Internet use among this population.
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Late-life difficult-to-treat depression and dementia subtypes: a naturalistic cohort study using electronic health records

IntroductionLate-life difficult-to-treat depression (LL-DTD) and dementia frequently coexist in later life, but it remains unclear whether clinical and sociodemographic characteristics, as well as medication exposure patterns, differ across dementia subtypes among older adults with both conditions.MethodsWe analysed anonymised electronic health records from a south London catchment area. We included patients aged ≥60 years at first recorded depression diagnosis with a dementia diagnosis. LL-DTD was defined as inadequate response to ≥2 antidepressant trials. Dementia diagnoses were classified as Alzheimer’s disease (AD), vascular dementia (VD), mixed AD/VD, dementia with Lewy bodies (DLB), or other/unspecified dementia. Around the index depressive episode, we captured sociodemographics, depressive symptoms, physical comorbidity, and medication indicators. We used multivariate logistic regression to examine cross-sectional correlates distinguishing dementia subtypes (with AD as reference) within the LL-DTD and dementia sample. We conducted stratified analyses comparing non-AD versus AD dementia by the temporal order of dementia and depression diagnoses.ResultsAmong 890 older adults with LL-DTD and dementia, AD was the most common subtype (33.9%), followed by mixed AD/VD (22.5%), VD (22.2%), other/unspecified dementia (15.2%), and DLB (6.2%). Depressive symptom profiles and psychotropic treatment history were broadly similar across subtypes. Compared with AD, VD was associated with greater functional impairment, while greater physical comorbidity burden was more evident in VD and mixed AD/VD.DiscussionSomatic multimorbidity and functional impairment provided the clearest clinical separation between subgroups, while depressive symptom patterns and medication exposure appeared largely non-specific across dementia subtypes. This underscores the importance of multimorbidity and physical health burden in understanding heterogeneity of dementia outcomes in LL-DTD.