New papers on Treatments & drugs
75 new papers on treatments & drugs in the last 7 days, within Medicine & health. These are the 50 Pipette rates most worth reading, with the main result in the authors' own words.
The best of the week
Multimodal hydrogel modulation of extracellular vesicle cargo to arrest post-traumatic osteoarthritis in murine models
Here we show that PMUC integrates adaptive lubrication with stress-triggered immune-metabolic modulation, offering a first‑in‑class strategy to protect joints and alleviate early OA.
Peer-reviewed journalBold claims, read criticallyClaims a big stepReal-world useImpaired regulatory T-cell-mediated immune tolerance promotes neurodegeneration in glaucoma
Together, these findings identify failure of regulatory T-cell-mediated immune tolerance as a mechanism that permits neurodegeneration in glaucoma and demonstrate that restoring immune regulation can ameliorate neuronal injury.
PreprintReal-world useImpaired regulatory T cell mediated immune tolerance promotes neurodegeneration in glaucoma
Together, these findings identify failure of regulatory T-cell-mediated immune tolerance as a mechanism that permits neurodegeneration in glaucoma and demonstrate that restoring immune regulation can ameliorate neuronal injury.
PreprintReal-world useGLP-1 Receptor Agonist Initiation Is Associated With Excess Skeletal Muscle Loss in Adults With Diabetes: A Prospective Population-Based MRI Study
ConclusionsIn this first population-based comparison of whole-body MRI before and after GLP-1 RA initiation with matched unexposed controls, initiators lost roughly 60% more muscle.
PreprintReal-world useMultichannel, biomimetic scaffolds improve functional connectivity following traumatic long-gap peripheral nerve injury
When compared to nerve autografts and FDA-approved nerve conduits in 1cm-long rat and 3cm-long rabbit sciatic gap injury, biomimetic scaffolds were equivalent or superior to nerve autografts on measures of axonal regeneration, re-connection between gastrocnemius muscle and spinal cord motor neurons, and evoked motor responses.
Peer-reviewed journalBold claims, read criticallyReal-world useCysteine supplementation reverses immune dysfunction in cancer patients with severe COVID-19
One Sentence SummaryMerlinsky et al show that cysteine supplementation can therapeutically reverse immune dysregulation in cancer patients with severe COVID-19 infection.
PreprintBold claims, read criticallyReal-world useDrug repurposing identifies candidate uterus-selective tocolytics for preterm labor
This approach established a systematic strategy for identifying uterus-selective tocolytics, and these findings support further preclinical development of repurposed drugs and drug combinations for the treatment of preterm labor to improve pregnancy and neonatal outcomes.
Peer-reviewed journalReal-world useThe nucleoside analog kamuvudine-9 shows protective and therapeutic efficacy in a mouse model of multiple sclerosis
Here, we report that in the experimental autoimmune encephalitis mouse model of MS, treatment with kamuvudine-9 (K-9), an NRTI derivative with an enhanced safety profile, prevented further neurological deficits and reversed preexisting paralysis and vision loss.
Peer-reviewed journalReal-world useAttention Is All You Need: A Universal Augmented Reality Surgical Navigation Framework
These findings support co-located action guidance as an alternative interface paradigm for surgical navigation.
PreprintReal-world useMULTIMODHAL: a multicenter phase 3 randomized controlled trial - fMRI-based symptom capture for guiding rTMS treatment of drug-resistant hallucinations in patients with schizophrenia.
Conclusions: fMRI-guided neuronavigation significantly enhances rTMS efficacy for drug-resistant AVHs, with sustained benefits up to six months and a favorable safety profile.
PreprintReal-world useA design approach for bitopic kinase inhibitors
PonatiLink-2 outperforms clinically relevant treatments in mouse models of BCR :: ABL1 -driven cancer, both in combination with dasatinib against wild-type BCR :: ABL1 and as a single agent against ponatinib-resistant disease.
Peer-reviewed journalReal-world useA Single Suprachoroidal Injection of AXT107 (Gersizangitide), a Multimodal, Long-Acting Integrin-Disrupting Peptide, in Patients with Neovascular Age-Related Macular Degeneration: Results of the Phase 1/2a DISCOVER Trial
Conclusions In this first-in-human study, a single suprachoroidal injection of AXT107 up to 0.5 mg was well tolerated through Week 40, with no drug-related serious adverse events, no clinically significant intraocular inflammation, and no sustained IOP elevations.
PreprintReal-world useSenescence-directed nanotherapy ameliorates fibrosis and overcomes immune exclusion in cancer
These findings establish SMNPs as a generalizable strategy to target pathogenic senescent cell subsets across fibrosis and cancer.
Peer-reviewed journalBold claims, read criticallyReal-world useEffects of Virtual Co-working Environment Using a Work-Support Application on Subjective Workload, Mood, and Fatigue: A Randomized Crossover Trial
ConclusionsThe gogh condition was associated with a decrease in post-task perceived workload, negative mood, and fatigue.
PreprintReal-world usePatch nonwear accounts for more than half of apparent nonadherence in a digital medicine system
Of 322 days without a detected ingestion, 53.7% had insufficient patch wear.
PreprintReal-world useImmune checkpoint blockade reshapes drug-associated toxicity: a pharmacovigilance atlas of drug-ICI interactions
Conclusions and RelevanceICIs were associated with a structured cross-organ toxicity landscape, phenotype-specific modification of drug-associated adverse events, and distinct temporal patterns across checkpoint pathways.
PreprintReal-world usePsilocybin and Contrasting Musical Compositions: Effects on Music-Evoked Emotional Responses in Healthy Adults
These findings suggest that psilocybin and contrasting musical compositions independently influence different aspects of music-evoked emotional responses, with limited interaction effects, and point to potential implications for protocol design and music selection in psychedelic settings.
PreprintEtuvetidigene Autotemcel for the Treatment of Wiskott–Aldrich Syndrome
CONCLUSIONS: Our results show that the effects of etu-cel are consistent with a sustained clinical benefit in persons with Wiskott-Aldrich syndrome.
Peer-reviewed journalReal-world useComparative Effectiveness of Spinal Manipulation for Low Back Pain: A Bayesian Hierarchical Analysis Incorporating Natural History
ConclusionsThe apparent effectiveness of LBP treatment depends strongly on the natural course against which treatment-associated improvement is evaluated.
PreprintReal-world usePharmacokinetics, subject satisfaction, and tolerability of a novel oral iron formulation based on naturally occurring microalgae Spirulina platensis
DiscussionThis first report of oral Monurelle(R) Ferro Naturale in humans shows this novel formulation to provide a favourable PK profile with respect to serum iron concentrations, with excellent tolerability and high subject satisfaction.
PreprintReal-world useiTBS induces a suppression-rebound pattern in human cortical excitability
Our results suggest iTBS promotes a dynamic process of cortical suppression followed by rebound increases in excitability.
PreprintPharmacokinetics, subject satisfaction, and tolerability of a novel oral iron formulation based on naturally occurring microalgae Spirulina platensis
DiscussionThis first report of oral Monurelle(R) Ferro Naturale in humans shows this novel formulation to provide a favourable PK profile with respect to serum iron concentrations, with excellent tolerability and high subject satisfaction.
PreprintReal-world useSurface-displayed cholesterol enhances penetration and anti-tumor activity of a platinum nanodrug by targeting NPC1L1-expressing cancer associated fibroblasts
Herein, by selectively targeting NPC1L1-cancer associated fibroblasts, surface-displayed cholesterol enhances the delivery of a platinum nanodrug (PtD-Chol) in stroma-rich tumors, restoring anti-tumor efficacy.
Peer-reviewed journalBold claims, read criticallyReal-world useVarenicline as a Repurposable Drug Candidate for ADRD Prevention
Varenicline use was associated with a reduced risk of ADRD (HR 0.516, 95% CI: 0.353-0.754) and the composite outcome ADRD or death (HR 0.657, 95% CI: 0.501-0.861).
PreprintReal-world useAir pollutant multiomics improves functional annotation of SNPs associated with lung disease
We identified airway epithelial transcriptional mechanisms associated with exposure and disease risk using a functional genomics pipeline that combined unbiased nascent RNA sequencing, genetic prioritization, and gene-regulatory element correlation.
PreprintSafety, Feasibility, and Preliminary Clinical Findings of an Oral Polypore Fungi Combination with Mild-to-Moderate COVID-19: A Randomized, Placebo-Controlled Phase I Clinical Trial
ConclusionsFoTv was safe, feasible, reduced viral cellular infection, and may reduce symptoms of COVID-19 associated with viral transmission.
PreprintBold claims, read criticallyReal-world useVarenicline as a Repurposable Drug Candidate for ADRD Prevention
Varenicline use was associated with a reduced risk of ADRD (HR 0.516, 95% CI: 0.353-0.754) and the composite outcome ADRD or death (HR 0.657, 95% CI: 0.501-0.861).
PreprintReal-world usePhotoinitiator-free gelatin hydrogel microneedles for acute gouty arthritis treatment
Overall, this initiator-free, photo-crosslinkable GelLA-based MNs system offered a safe, efficient, and user-friendly strategy for local drug delivery in the management of acute gouty arthritis.
Peer-reviewed journalReal-world useTherapeutic blockade of IL-1β in PWH reduced the HIV reservoir via mechanisms of enhanced CD8 and CD4 effector function and reversal of monocyte dysfunction
These findings establish a mechanistic rationale for IL-1{beta} blockade as a therapeutic intervention that effectively target the HIV reservoir by restoring functional HIV specific adaptive immune responses and reversing monocyte driven inflammatory dysfunction.
PreprintBold claims, read criticallyReal-world usePreclinical and clinical evaluation of systemic danegaptide for the treatment of nonproliferative diabetic retinopathy
Here, we describe the effect of an orally available medication, danegaptide, in regulating retinal vascular permeability in preclinical animal models of disease and report safety and early efficacy data from an early-stage phase 1b clinical trial in patients with NPDR and mild center-involved macular edema.
Peer-reviewed journalReal-world useClinical Commissioning of the First Ultra-Compact Gantry-less Upright Proton Therapy System
This study reports the comprehensive clinical commissioning and benchmarking of a novel ultra-compact, gantry-free, upright PT system.
PreprintClaims a big stepReal-world useSupportive Strides: Co-designing Community-Based Walking Programs with Embedded Socialisation and Peer Support for People after Stroke
ConclusionsThe co-designed Supportive Strides community-based walking program is ready for testing to improve physical activity and social connection after stroke.
PreprintReal-world useEasy to readSupportive Strides: Co-designing Community-Based Walking Programs with Embedded Socialisation and Peer Support for People after Stroke
ConclusionsThe co-designed Supportive Strides community-based walking program is ready for testing to improve physical activity and social connection after stroke.
PreprintReal-world useEasy to read'We need this now': Acceptability and usability of a pediatric antiretroviral microarray patch prototype in South Africa and Uganda
Overall, the pediatric ARV microarray patch prototype was perceived as highly acceptable and usable across diverse end users, findings that generally align with studies of microarray patches for other use cases (e.g., contraception, HIV pre-exposure prophylaxis, vaccine delivery).
PreprintReal-world useEasy to readA non-retinoid triazolopyrimidine RBP4 antagonist for the treatment of Stargardt disease
Here, we report the pharmacological characterization of AKR-XI-85 and its analogs as promising non-retinoid small-molecule RBP4 antagonists.
PreprintReal-world useShort-Term Changes in Dermal Density Following Topical Application of a Progerinin-Containing Serum: An Exploratory Clinical Study
Four weeks of topical application of the Progerinin-containing serum was therefore associated with measurable improvements in multiple skin parameters, with a particularly pronounced and progressive increase in ultrasound-assessed dermal density.
PreprintReal-world useComparative effectiveness of digital health interventions for low back pain: systematic review and network meta-analysis of randomised controlled trials
Conclusions Among adults with low back pain, VR showed the largest estimated post-intervention effects versus control for pain intensity, physical function, pain-related fear avoidance, and health-related quality of life, although comparative effects differed across outcomes.
PreprintReal-world useHigh-intensity progressive resistance training to prevent falls in community-dwelling adults aged 80 years and older with reduced muscle strength: protocol for the ReFit randomised controlled trial
Therefore, this study aims to determine whether 12 months of high-intensity progressive resistance training reduces fall rates in community-dwelling adults aged [≥]80 years with reduced muscle strength.
PreprintReal-world useEvaluation of an AI-Powered Patient Education Application in Bariatric Surgery: A Prospective Mixed-Methods Feasibility Study
ConclusionsBariBot was feasible to implement and was associated with high perceived usability and acceptability postoperatively after bariatric surgery.
PreprintReal-world useA Generalizable, Governance-Embedded Pathway for Assessing Patient-Level Opportunities for Genetic Medicines
ConclusionFindings to date support feasibility and high uptake of a structured, multidisciplinary, medical genetics-led model for assessing patient-level opportunities for genetic medicines.
PreprintReal-world useRotational perturbation training improves muscle coordination during reactive standing balance in trained and untrained conditions in children with spastic cerebral palsy
Our findings suggest that reactive balance control can be modified through perturbation training, and that training-induced adaptations generalize to untrained perturbation conditions.
PreprintReal-world usePATIENT PROFILING AND OUTCOME OF ANTIHYPERTENSIVE TREATMENT. An Exploratory Analysis of the VALUE Trial.
ConclusionsWe conclude that composite patient profiles may predict BP responses to antihypertensive treatment more accurately than single factors.
PreprintReal-world usePiloting a data-extraction form for verbatim terminology: a blinded inter-reviewer agreement study before a surgical evidence map
ConclusionsA form that extracts terminology or verbatim text should be piloted with a set-overlap measure, a rule for empty sets and a defined admissible source, against a threshold fixed in advance.
PreprintPiloting a data-extraction form for verbatim terminology: a blinded inter-reviewer agreement study before a surgical evidence map
ConclusionsA form that extracts terminology or verbatim text should be piloted with a set-overlap measure, a rule for empty sets and a defined admissible source, against a threshold fixed in advance.
PreprintTENS for Movement-Evoked Pain in Fibromyalgia: A Randomised Double-Blind Controlled Trial
ConclusionThese results suggest that a single session of high-frequency TENS during resistance exercise can produce short-term reductions in MEP in women with fibromyalgia.
PreprintReal-world useComputer-Assisted Systematic Chemical-Space Mapping of a First-in-Class Peripherally Restricted α2AAR Agonist through Scaffold-Seeded Enumeration
These findings support scaffold-seeded computational enumeration as a practical strategy for systematic chemical-space mapping around a first-in-class lead and for identifying additional pharmacologically active structural regions for further optimization.
PreprintReal-world useEvaluating prescriber preferences for the treatment of indeterminate Chagas disease (EPPIC study): an international survey
A 70% efficacy threshold emerged as a critical inflection point for prescribing willingness, while prevention of mother-to-child transmission, improved tolerability, and greater accessibility were identified as key factors that could support the adoption of regimens with moderate efficacy.
PreprintReal-world useEnhanced HIV-1 Control After Antibody Therapy is Associated with Autologous Antibodies and Reservoir Clearance in the RIO trial
Finally, comparison of pre-infusion and pre-rebound HIV-1 proviral reservoir showed a decrease in intact but not defective proviruses in Arm A but not in Arm B. Together, these findings suggest that baseline humoral immunity and prolonged exposure to LS-bNAbs may shape post-treatment viral rebound dynamics in individuals that initiate ART during primary infection.
PreprintReal-world usePerioperative transcutaneous auricular vagus nerve stimulation for the prevention of postoperative nausea and vomiting after gynecologic laparoscopic surgery: protocol for a randomized, double-blind, sham-controlled trial
Conclusions This trial aims to provide rigorous evidence on the efficacy and safety of perioperative taVNS for PONV prevention in women undergoing gynecologic laparoscopy.
PreprintReal-world useEvaluating the Real-World Safety of Atypical Antipsychotics in Paediatric Autism Spectrum Disorder: Lessons from Saudi Arabia's Pharmacovigilance System.
ConclusionsThis study highlights disproportionality signals associated with AAP use in paediatric patients.
PreprintReal-world use