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alpha-1 antitrypsin (Alfalastin / rhAAT, GTC)

✓ Approved

rEVO Biologics · SERPINA1 · Recombinant Proteins

What is alpha-1 antitrypsin?

alpha-1 antitrypsin is a recombinant proteins developed by rEVO Biologics. It is approved for therapeutic indications via unknown.

Drug Profile

Brand NamesAlfalastin, rhAAT, GTC
CompanyrEVO Biologics
Drug ClassRecombinant Proteins
Molecular TargetSERPINA1
RouteUnknown
StatusApproved
Sources: ClinicalTrials.gov, rEVO Biologics

Mechanism of Action

Molecular Targets

alpha-1 antitrypsin acts on 1 molecular target:

SERPINA1serpin family A member 1 (PRO2275, nNIF)
Want deeper analysis?Noah AI can explain complex mechanisms and compare to similar drugs.

Therapeutic Indications

alpha-1 antitrypsin is developed for 3 unique indications across 2 therapeutic areas.

Therapeutic AreaConditionPhase
Congenital, familial and genetic disordersCongenital emphysema✓ Approved
Skin and subcutaneous tissue disordersDermatitis allergicPreclinical
Skin and subcutaneous tissue disordersDermatitis atopicPreclinical

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Dai Li L, Kong Fan-Lei FL

To explore the effects of programmed cell death protein-1 (PD-1) combined with pemetrexed (PEM) and carboplatin (CARB) chemotherapy regimen on the gut microbiota in the Lewis lung cancer model mice compared to chemotherapy alone. C57BL/6 J male mice aged 10-12 weeks were selected to establish the Lewis lung cancer model by planting tumors in the right forelimb, and were randomly divided into negative control group (NC group), chemotherapy group (PEM-CARB group), and chemotherapy combined with immunotherapy group (PEM-CARB-PD-1 group), with eight mice in each group. The total RNA of fecal bacteria was collected from the feces of mice in each group after two cycles of drug administration. 16S rRNA gene amplification and high-throughput sequencing were performed to analyze the Alpha diversity, Beta diversity, composition, and function in the gut microbiota. The Alpha diversity was not statistically different between the PEM-CARB-PD-1 group and the PEM-CARB group (Shannon index: p = 0.645; Simpson index: p = 0.879). The Beta diversity between the PEM-CARB-PD-1 group and PEM-CARB group was statistically different [weighted Unifrac Principal Co-ordinate Analysis (PCoA), p = 0.001; unweighted Unifrac PCoA, p < 0.001]. However, the Beta diversity between the PEM-CARB-PD-1 group and the NC group did not reveal statistical differences (weighted Unifrac PCoA, p = 0.690; unweighted Unifrac PCoA, p = 0.135). Compared to the PEM-CARB group, the combination of the PD-1-inhibitor affects both the "response-favorable taxa" and "response-unfavorable taxa" for immunotherapy. Notably, the PEM-CARB-PD-1 group had an increased abundance of Gram-positive bacterial phenotypes relative to the PEM-CARB group (p = 0.038). Nearly no statistically significant differences in metabolic pathways were seen between the PEM-CARB-PD-1 group and the PEM-CARB group. Combination therapy affects both "response-favorable taxa "and "response-unfavorable taxa associated with immunotherapy, and the ultimate impact remains dependent on the ratio of the two types of flora. Predicted metabolic pathway analysis using PICRUSt2 suggested that the combination regimen may not further reduce predicted functional pathway abundance beyond that observed with chemotherapy alone. However, these predictions require validation through direct metagenomic or metabolomic approaches.

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As you read this sentence, you integrate meanings of individual words into complex, higher-order representations. In addition to the core language network (LN), this dynamic process recruits other domain-general networks such as the default mode network (DMN), with which it partially overlaps anatomically but segregates from functionally. To evaluate interactions between LN and DMN, we extracted instantaneous frequency, power, and phase (4-30Hz) from intracranial electrodes in 32 participants who read sentences and wordlists. We isolated a complex low-frequency modulation, most reliably measured as a ramping of instantaneous frequency in the alpha band throughout sentences, greater than wordlists, occurring robustly across both the LN and DMN. Granger causal networks demonstrate this coincided with ramping increases in alpha-band connectivity between and within DMN and LN. We suggest that alpha-band modulations index dynamic interactions between core LN and broader domain-general networks and may be crucial for higher order semantic integration and the derivation of crossmodal knowledge from the language domain.

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Pediatric sleep disorders are common, underrecognized conditions with significant short-term and long-term consequences for physical health, neurocognitive development, mental well-being, and family functioning. This article provides a comprehensive, clinically focused overview of pediatric sleep disorders, with particular emphasis on Generation Z and Generation Alpha cohorts who are uniquely shaped by pervasive digital exposure, circadian disruption, and contemporary psychosocial stressors. Normal developmental changes in sleep architecture are reviewed to distinguish physiologic variation from pathology. Key sleep disorders including insomnia, circadian rhythm sleep-wake disorders, sleep-disordered breathing, parasomnias, and sleep-related bruxism are discussed.

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de Britto Renata R, Moura Alvorcem Leonardo de L, Marcuzzo Manuela Bianchin MB, Ribeiro Rafael T RT et al.

Glutaric aciduria type 1 (GA1) is a cerebral organic aciduria caused by deficient activity of glutaryl-CoA dehydrogenase (GCDH). Patients present with acute striatal degeneration and develop progressive cortical leukodystrophy whose pathophysiology is only partially known. As treatment for GA1 is limited, we evaluated the impact of JP4-039, a mitochondria-targeted reactive oxygen species (ROS) and electron scavenger, on redox homeostasis, mitochondrial quality control, and glucose metabolism in the cortical and striatal brain tissues of GCDH-deficient (Gcdh-/-) mice. Both tissues exhibited increases in lipid peroxidation, ROS levels, and the activities of superoxide dismutase, catalase, and glutathione S-transferase. Furthermore, glutathione reductase activity was increased, and glutathione peroxidase was reduced in the striatum, while Nrf2 mRNA levels were elevated in the cortex. Notably, most of these altered endpoints of redox homeostasis were prevented by treatment with JP4-039. Peroxisome proliferator-activated receptor gamma coactivator 1-alpha (PGC1α) expression was reduced in the cortex of Gcdh-/- mice, whereas voltage-dependent anion channel (VDAC) and dynamin-related protein 1 (DRP1) expression were increased in the striatum, signaling a disturbance of mitochondrial quality control. JP4-039 mitigated the DRP1 change. The cerebral cortex displayed reduced glucose metabolism, increased lactate levels, and elevated activities of hexokinase, pyruvate kinase, and lactate dehydrogenase (LDH), which JP4-039 mitigated. GLUT3 expression was reduced in the cerebral cortex, but JP4-039 did not change this effect. Our data suggest that redox imbalance and dysregulated mitochondrial quality control and of the glycolytic pathway contribute to the pathophysiology of GA1, and that JP4-039 may offer therapeutic benefit.

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PubMedHNO2026-07-25

The European Board Examination in Otorhinolaryngology as a high-quality test instrument: psychometric measures in a long-term comparison.

Neudert Marcus M, Meric Hafiz Aysenur A, Ward Victoria V, Simo Ricard R et al.

The European Board Examination in Otorhinolaryngology, Head and Neck Surgery (EBEORL-HNS) is a Europe-wide specialty examination consisting of a written multiple-choice test and a standardized oral examination. Only limited psychometric analyses have been published to date. This study aimed to evaluate examination data from recent years and assess the formats regarding difficulty, reliability, and pass rates. Aggregated data from 2013-2024 were analyzed. Mean scores, standard deviations, item difficulty, item discrimination, Cronbach's alpha, and Angoff's score were calculated. The written and oral parts were examined descriptively and comparatively. The written examination demonstrated a mean difficulty of 67% (proportion of correctly answered items), a pass rate of 82%, and Cronbach's alpha of 0.83 (internal consistency). The passing cutoff (according to the Angoff method) was 59 points on average. The oral part showed a mean difficulty of 70%, a pass rate of 74%, and an average score of around 3.0 on a four-point scale. Year-to-year variability largely reflected differences in the international candidate cohorts. The findings confirm that the EBEORL-HNS demonstrates stable psychometric quality. Compared to national specialty examinations, it offers high transparency and clearly defined quality criteria. Variations in performance appear more related to heterogeneous training backgrounds than to shortcomings of the assessment tools.

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Cross-cultural adaptation and validation of the Arabic version of the Edmonton Frail Scale among Saudi community-dwelling older adults: A cross-sectional study.

Alshahrani Amirah M AM, Alqahtani Sarah H SH, Alotaibi Turki M TM, Almutairi Faten F FF et al.

Frailty is a clinical condition characterized by increasing vulnerability in older adults, resulting from declines in various physiological systems. The Edmonton Frail Scale (EFS) is a multidimensional tool for assessing frailty aspects including general health, functional independence, social support, medication usage, nutrition, mood, continence, cognition, balance, and mobility. Since the EFS has not been translated and validated among Arabic-speaking populations, the aim of this study was to translate, validate, and culturally adapt the EFS into Arabic for the Saudi geriatric population while examining its reliability and validity among community-dwelling older adults. A cross-sectional study was conducted between February and May 2024 in the Riyadh region of Saudi Arabia among community-dwelling older adults. One hundred eighty-two participants (100 male and 82 female) aged 60 and above from the King Salman Social Center were included. The EFS was translated from English into Arabic following standardized guidelines. Internal consistency using Cronbach alpha was assessed among the full sample (n = 182), while test-retest reliability using the intraclass correlation coefficient was assessed with 20 participants over a 1-week interval. Concurrent validity of the Arabic EFS (A-EFS) using Spearman rank correlation coefficient was tested against other related measures in all participants, including the Arabic (Saudi) version of the Tilburg Frailty Indicator, Arabic version of the Montreal Cognitive Assessment, Arabic version of Activities of Daily Living, and grip strength using a digital dynamometer, to identify correlations. In a sample of 182 community-dwelling older adults (mean age 65.6 ± 5.2 years; 100 males and 82 females), the A-EFS demonstrated good reliability and validity. Internal consistency was acceptable (Cronbach alpha = 0.61), with item correlations ranging from 0.54 to 0.65. For test-retest reliability, the intraclass correlation coefficient was 0.89 (95% confidence interval = 0.79-0.94). Moderate correlations were observed with grip strength (r = -0.508, P < .01) and Tilburg Frailty Indicator (R = 0.503, P < .01), and a weak correlation with Activities of Daily Living and Montreal Cognitive Assessment (r = -0.330, -0.346; P < .01) respectively. The A-EFS is a reliable, valid, and culturally sensitive tool for assessing frailty among Saudi older adults in both research and clinical contexts, with the potential to support informed clinical decision-making in routine practice. Future studies should focus on establishing the validity and reliability of the A-EFS across diverse settings.

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