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Imiglucerase in the treatment of Gaucher disease: a history and perspective.

The scientific and therapeutic development of imiglucerase (Cerezyme(®)) by the Genzyme Corporation is a paradigm case for a critical examination of current trends in biotechnology. In this article the authors argue that contemporary interest in treatments for rare diseases by major pharmaceutical companies stems in large part from an exception among rarities: the astonishing commercial success of Cerezyme. The fortunes of the Genzyme Corporation, latterly acquired by global giant Sanofi SA, were founded on the evolution of a blockbuster therapy for a single but, as it turns out, propitious ul

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… As already outlined, the general stratagem of complementing lysosomal enzyme deficiencies was initiated in another laboratory at the NIH under the direction of Neufeld. 108 However, the seminal discovery of the mannose-binding lectin of the macrophage by Philip Stahl et al 109 at Washington University … Moreover, large mannosylated structures with a mean number of mannose residues exceeding the mean of 14 per molecule reported in imiglucerase had increased affinity for the mannose-binding lectin in serum – a humoral component of the innate immune system. …

Gaucher disease: clinical profile and therapeutic developments.

Gaucher disease is a rare inborn error of glycosphingolipid metabolism due to deficiency of lysosomal acid β-glucocerebrosidase; the condition has totemic significance for the development of orphan drugs. A designer therapy, which harnesses the mannose receptor to complement the functional defect in macrophages, ameliorates the principal clinical manifestations in hematopoietic bone marrow and viscera. While several aspects of Gaucher disease (particularly those affecting the skeleton and brain) are refractory to treatment, enzyme (replacement) therapy has become a pharmaceutical blockbuster.

Editorial: The Role of Complement in Health and Disease.

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… The review by Ratajczak et al. focuses on an emerging role of mannose-binding lectin- (MBL)-dependent complement activation in the release of effector cells from bone marrow (BM) in response to tissue injury, pathogens, and pharmacological inducers of hematopoietic stem/progenitor cells (HSPCs). …

Protein-Based Adjuvants for Vaccines as Immunomodulators of the Innate and Adaptive Immune Response: Current Knowledge, Challenges, and Future Opportunities.

New-generation vaccines, formulated with subunits or nucleic acids, are less immunogenic than classical vaccines formulated with live-attenuated or inactivated pathogens. This difference has led to an intensified search for additional potent vaccine adjuvants that meet safety and efficacy criteria and confer long-term protection. This review provides an overview of protein-based adjuvants (PBAs) obtained from different organisms, including bacteria, mollusks, plants, and humans. Notably, despite structural differences, all PBAs show significant immunostimulatory properties, eliciting B-cell- a

Prophylaxis and Empirical Therapy of Infection in Cancer Patients

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… lectin. … TABLE 310-2 Rates of Infectious Complications in Cancer Patients —, data not reported; AL, acute leukemia; ALL, acute lymphocytic leukemia; AnLL, acute nonlymphocytic leukemia; NHL, non-Hodgkin's lymphoma; HSCT, hematopoietic stem cell transplantation; MBL, mannose-binding lectin. …

An Update on Current Therapeutic Options in IgA Nephropathy.

Immunoglobulin A nephropathy (IgAN) remains the leading cause of primary glomerular disease worldwide. Outcomes are poor with high rates of progressive chronic kidney disease and kidney failure, which contributes to global healthcare costs. Although this disease entity has been described, there were no disease-specific treatments until recently, with the current standard of care focusing on optimal supportive measures including lifestyle modifications and optimization of the renin-angiotensin-aldosterone blockade. However, with significant advances in the understanding of the pathogenesis of I

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… Multiple complement proteins are found co-deposited with IgA immune complexes including C3, properdin, C4d, mannose-binding lectin as well as terminal complement components in IgAN [ 35 , 36 , 37 , 38 , 39 ]. However, C1q is absent in the majority of IgAN biopsy specimens [ 35 ]. … Lectin Pathway Inhibition as an Anti-Inflammatory Approach in IgAN Mannose-binding lectin-associated serine protease 2 (MASP-2) is a component of the lectin pathway that triggers C3 convertase formation. …

Comparison of different sets of immunological tests to identify treatable immunodeficiencies in adult bronchiectasis patients.

BACKGROUND: The reported prevalence of immunodeficiencies in bronchiectasis patients is variable depending on the frequency and extent of immunological tests performed. European Respiratory Society guidelines recommend a minimum bundle of tests. Broadening the spectrum of immunological tests could increase the number of patients diagnosed with an immunodeficiency and those who could receive specific therapy. The primary objective of the present study was to assess the performance of different sets of immunological tests in diagnosing any, primary, secondary or treatable immunodeficiencies in a

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… Mannose-binding lectin gene polymorphic variants predispose to the development of bronchopulmonary complications but have no influence on other clinical and laboratory symptoms or signs of common variable immunodeficiency. … Low serum mannose-binding lectin level is not associated with disease severity in non-cystic fibrosis bronchiectasis. Innate Immun 2012; 18: 787–792. doi: 10.1177/1753425912440472 …

New strategies and perspectives on managing IgA nephropathy.

IgA nephropathy is an inflammatory renal disease characterised by the deposition of IgA in the glomerular mesangium and is the most commonly reported primary glomerulonephritis worldwide. Thirty to forty percent of patients with the disease develop progressive renal function decline, requiring renal replacement therapy within two decades of diagnosis. Despite this, accurate individual risk stratification at diagnosis and predicting treatment response remains a challenge. Furthermore, there are currently no disease specific treatments currently licensed to treat the condition due to long standi

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… Mannose-binding lectin associated serine protease 2 (MASP-2) is a key component of the lectin pathway and promotes C3 convertase formation and subsequent downstream inflammatory effects. … Endo M Ohi H Ohsawa I Fujita T Matsushita M Fujita T Glomerular deposition of mannose-binding lectin (MBL) indicates a novel mechanism of complement activation in IgA nephropathy Nephrol Dial Transplant 1998 13 8 1984 1990 9719152 10.1093/ndt/13.8.1984 104. …

Biomarkers in the diagnosis of lysosomal storage disorders: proteins, lipids, and inhibodies.

A biomarker is an analyte indicating the presence of a biological process linked to the clinical manifestations and outcome of a particular disease. In the case of lysosomal storage disorders (LSDs), primary and secondary accumulating metabolites or proteins specifically secreted by storage cells are good candidates for biomarkers. Clinical applications of biomarkers are found in improved diagnosis, monitoring disease progression, and assessing therapeutic correction. These are illustrated by reviewing the discovery and use of biomarkers for Gaucher disease and Fabry disease. In addition, rece

Enhanced sialylation of bone marrow mesenchymal stromal cells contributes to immune remodeling through macrophage polarization in multiple myeloma patients.

INTRODUCTION: In multiple myeloma (MM), the immune system is disrupted, partly due to tumor-modified bone marrow mesenchymal stromal cells (BM-MSCs). These MSCs show altered glycosylation, including increased sialylation, which may promote immune evasion through Siglec interactions. METHODS: We quantified sialic acids and Siglec ligands on healthy donors-, MM-, T-MM-(treated multiple myeloma) and MGUS (monoclonal gammopathy of undetermined significance)-MSCs, and examined how different culture conditions (myeloma coculture, conditioned medium, hypoxia, inflammation, 3D culture) affect MSC gly

Navigating hepatotoxicity of antibody-drug conjugates: from mechanistic insights to clinical and postmarketing evidence.

Antibody-drug conjugates (ADCs) are rapidly developing targeted cancer therapeutic agents that combine specific monoclonal antibodies with cytotoxic agents. Currently, 17 ADCs are approved for the global market for treating hematological and solid tumors with more than 100 ADCs are in phase III clinical trials. ADC-induced hepatotoxicity is a significant concern with unclear mechanisms, and the incidence of hepatic adverse events (AEs) varies across different ADCs. Most hepatic AEs are moderate; however, some ADCs can cause life threatening or fatal AEs. The management of hepatic AEs is limite

Дополнительные обновления в оптическом неврите.

Оптический неврит (ON) является воспалительным состоянием зрительного нерва, связанным с демиелинизирующими заболеваниями, такими, как множественный склероз, спектральный расстройство нейромиелита, и болезнь, связанная с антителами, миэлина олигодендроцита. Система дополнения имеет решающее значение для патогенеза, нарушения зрительного нерва в крови, воспаления и повреждения тканей. В настоящем обзоре рассматриваются дополнительные пути активации - классические, альтернативные и лектиновые - и их роли в развитии. Ключевые белки, такие, как C3, C5, и компоненты конечной трассы, выделены в каче

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… Lectin pathway The lectin pathway is activated by mannose-binding lectin (MBL) and associated serine proteases, such as MASP-2, which recognize carbohydrate patterns on microbial surfaces or damaged host cells. …

Identification of six hub genes and analysis of their correlation with drug sensitivity in acute myeloid leukemia through bioinformatics.

BackgroundAcute myeloid leukemia (AML) is a common hematopoietic malignancy and have unsatisfactory prognosis. Our study aimed to identify hub genes in AML and explore potential biomarkers through integrated bioinformatics.MethodsMicroarray datasets were analyzed to screen the differentially expressed genes (DEGs). Functional enrichment analysis was performed, and protein-protein interaction (PPI) network was generated by the STRING (11.0) database and Cytoscape (3.7.2) software. Hub genes were screened and verified through GEPIA2 and GEO microarray database. Sensitivity of AML cell lines with

Infections After High-Dose Chemotherapy and Autologous Hematopoietic Stem Cell Transplantation

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… Table 4.1 Risk factors for infection after high-dose chemotherapy and autologous hematopoietic stem cell transplantation MBL mannose-binding lectin, TLR Toll-like receptors, CMV cytomegalovirus a Most common disease-related immunosuppression include: hypogammaglobulinemia (multiple myeloma, low-grade … In a series of 113 autologous HSCT for multiple myeloma, patients homozygous for wild-type mannose-binding lectin (MBL) 2 were at lower risk to develop septicemia compared with patients carrying the variant MBL2 [ 2 ]. …

The Complosome: An Emerging Intracellular Complement Network in Cancer Development and Therapy.

The complement system is traditionally recognized as a major effector of innate immunity, essential for pathogen clearance, inflammation and the maintenance of tissue homeostasis. In recent years, however, its role in cancer has been substantially redefined. Beyond its canonical extracellular activity, complement has emerged as a multifaceted regulator of tumor biology, acting not only within the tumor microenvironment but also intracellularly through the recently described intracellular complement system (complosome). While extracellular complement primarily shapes immune responses and the tu

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… The lectin pathway is similar to the classical pathway and is activated by MBL (mannose-binding lectin), ficolins, and collectins, thereby activating MASP-1 or MASP-2 (mannan-binding lectin-associated serine proteases-1 or -2) [ 6 , 25 , 64 , 70 ]. … Mannose-Binding Lectin (MBL) and MBL-Associated Serine Protease-2 (MASP-2) in Women with Malignant and Benign Ovarian Tumours. Cancer Immunol. …

Targeting Notch-1 positive acute leukemia cells by novel fucose-bound liposomes carrying daunorubicin.

Complete remission by induction therapy in acute myelogenous leukemia (AML) can be achieved due to improvements in supportive and optimized therapy. However, more than 20% of patients will still need to undergo salvage therapy, and most will have a poor prognosis. Determining the specificity of drugs to leukemia cells is important since this will maximize the dose of chemotherapeutic agents that can be administered to AML patients. In turn, this would be expected to lead to reduced drug toxicity and its increased efficacy. We targeted Notch-1 positive AML cells utilizing fucose-bound liposomes

Otitis media in Greenland. Studies on historical, epidemiological, microbiological, and immunological aspects.

This thesis describes the different aspects of otitis media (OM) in the population of Greenland viewed in a historical and modern clinical p … unity-based children in Nuuk. The hypothesis is based on a possible association between findings of mannose-binding lectin genotypes, early Epstein-Barr virus infections and episodes of AOM, rAOM, or nasopharyngeal colonization with potentially pathogenic bacteria. However, the study does not support any of this hypothesis. In chapter 6, future studies are suggested and …

Structure and function of therapeutic antibodies approved by the US FDA in 2025.

In 2025, the 50th anniversary of the invention of hybridoma technology, the United States Food and Drug Administration (US FDA) approved a total of 44 new molecular entities (NMEs), of which 10 were therapeutic antibody-based molecules. Additionally, the US FDA approved 31 small molecules (of which one was a peptide), and 3 new nonantibody therapeutic proteins, but no new chimeric antigen receptor (CAR)-T-cell therapeutics. Of the new antibody-based biologics, two were antibody-drug conjugates, one was a T-cell engager-bispecific antibody, two were Fc-modified, half-life extended antibodies, a

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… There are at least nine families of Lectin pathway PRMs include Mannose-Binding Lectin (MBL), which binds mannose, glucose, and other sugars, Ficolins, which target acetylated residues rather than just sugars, and Collectins, which are soluble proteins that also recognize microbial oligosaccharides [ …

Applications of Aggregation-Induced Emission Materials in Immunology: From Diagnostics to Immunotherapy.

Aggregation-induced emission (AIE) materials, due to their unique ability to significantly enhance optical emission in aggregated states, have demonstrated vast potential in immunology, particularly in early disease diagnosis and immunotherapy. AIE materials can serve as highly efficient fluorescent probes for biomarker detection, providing critical insights for early diagnosis. Additionally, they can act as cancer vaccines, enhancing the effectiveness of immunotherapy through photodynamic therapy, photothermal therapy, or activation of immune cells. This review highlights the applications of

Membranous nephropathy: pathogenesis and treatments.

Membranous nephropathy (MN), an autoimmune disease, can manifest at any age and is among the most common causes of nephrotic syndrome in adults. In 80% of cases, the specific etiology of MN remains unknown, while the remaining cases are linked to drug use or underlying conditions like systemic lupus erythematosus, hepatitis B virus, or malignancy. Although about one-third of patients may achieve spontaneous complete or partial remission with conservative management, another third face an elevated risk of disease progression, potentially leading to end-stage renal disease within 10 years. The i