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Regulatory insights on gene therapies, adeno-associated virus-based gene therapies, cell/tissue-based products, and medical care/practice in cell and gene therapies: Report from the 7th Asia partnership conference - April 25, 2024.

The 7th Asia Partnership Conference of Regenerative Medicine (APACRM) was held both in person and online on April 25, 2024, to promote the regulatory harmonization of regenerative medicine products across Asia. Recognizing domestic regulatory guidelines and their underlying rationales within each country and region is an important initial step toward achieving harmonious regulation. The 7th APACRM featured open dialogues on the non-clinical evaluations of adeno-associated virus gene therapy products, regulation of gene therapies, considerations for conducting clinical trials, and provision of

Recent advances in the bench-to-bedside translation of cancer nanomedicines.

Cancer remains a complex and challenging medical problem, driving extensive research efforts. Despite significant progress in understanding its genetic and molecular aspects, the quest for effective treatments continues. Nanomedicines have shown great potential for revolutionizing cancer treatment by offering targeted and controlled drug delivery, reducing side effects, and improving patient outcomes. Accordingly, nanomedicines have been the focus of extensive research and development for clinical translation. As of September 2024, a search on the ClinicalTrials.gov website using the term "nan

Biomarker-Driven Approaches to Bone Metastases: From Molecular Mechanisms to Clinical Applications.

Bone metastases represent a critical complication in oncology, frequently indicating advanced malignancy and substantially reducing patient quality of life. This review provides a comprehensive analysis of the complex interactions between tumor cells and the bone microenvironment, emphasizing the relevance of the "seed and soil" hypothesis, the RANK/RANKL/OPG signaling axis, and Wnt signaling pathways that collectively drive metastatic progression. The molecular and cellular mechanisms underlying the formation of osteolytic and osteoblastic lesions are examined in detail, with a particular foc

Inhibition of the fibroblast growth factor receptor (FGFR) pathway: the current landscape and barriers to clinical application.

The fibroblast growth factor/fibroblast growth factor receptor (FGF/FGFR) is a tyrosine kinase signaling pathway that has a fundamental role in many biologic processes including embryonic development, tissue regeneration, and angiogenesis. Increasing evidence indicates that this pathway plays a critical role in oncogenesis via gene amplification, activating mutations, or translocation in tumors of various histologies. With multiplex sequencing technology, the detection of FGFR aberrations has become more common and is tied to cancer cell proliferation, resistance to anticancer therapies, and n

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… FGFR3 In a study using microarray-based comparative genomic hybridization on samples from 18 patients with recurrent or metastatic adenoid cystic carcinoma (ACC), the investigators noted that 61% of tumors displayed a DNA copy number increase that was associated with loci for FGF(R)s. …

Изучение точных методов лечения иммунизированных подстрекательских заболеваний: использование бесконечного потенциала доставки нуклеиновой кислоты.

Неизлечимые подстрекательские заболевания (ИМИД) создают неизмеримое бремя для отдельных лиц и общества. В то время как традиционное использование иммунодепрессантов и лекарственных средств для борьбы с заболеваниями обеспечивает частичную помощь и контроль, их неизбежные побочные эффекты и ограниченная эффективность ставят под сомнение возможность найти лекарство. Перспективные лекарства от нуклеиновой кислоты продемонстрировали способность оказывать точное воздействие на молекулярном уровне, при этом различные классы нуклеиновых кислот выполняют регулирующие функции с помощью различных механ

Nanoparticles Based Therapeutic Approaches in Oncogenic Viral Infections: A Review.

Nanotechnology has revolutionized biomedical research by providing novel strategies for the prevention, diagnosis, and treatment of oncogenic viral infections. Nanoparticles, possessing tunable physicochemical properties, enable targeted drug delivery, enhance vaccine efficacy, and facilitate advanced viral diagnostics, addressing key limitations of conventional therapies. Oncogenic viruses contribute substantially to the global burden of cancer through persistent infections and are influenced by environmental and lifestyle factors. The objective of this review is to synthesize and critically

mRNA vaccine in cancer therapy: Current advance and future outlook.

Messenger ribonucleic acid (mRNA) vaccines are a relatively new class of vaccines that have shown great promise in the immunotherapy of a wide variety of infectious diseases and cancer. In the past 2 years, SARS-CoV-2 mRNA vaccines have contributed tremendously against SARS-CoV2, which has prompted the arrival of the mRNA vaccine research boom, especially in the research of cancer vaccines. Compared with conventional cancer vaccines, mRNA vaccines have significant advantages, including efficient production of protective immune responses, relatively low side effects and lower cost of acquisitio

MET Signaling Pathways, Resistance Mechanisms, and Opportunities for Target Therapies.

The MET gene, known as MET proto-oncogene receptor tyrosine kinase, was first identified to induce tumor cell migration, invasion, and proliferation/survival through canonical RAS-CDC42-PAK-Rho kinase, RAS-MAPK, PI3K-AKT-mTOR, and β-catenin signaling pathways, and its driver mutations, such as MET gene amplification (METamp) and the exon 14 skipping alterations (METex14), activate cell transformation, cancer progression, and worse patient prognosis, principally in lung cancer through the overactivation of their own oncogenic and MET parallel signaling pathways. Because of this, MET driver alte

A technical note on emerging combination approach involved in the onconanotherapeutics.

Cancer is the second cause of mortality worldwide, and the currently available conventional treatment approach is associated with serious side effects and poor clinical outcomes. Based on the outcome of the exploratory preclinical and clinical studies, it was found that therapeutic response increases multiple folds when anticancer drugs are used in combination. However, the conventional combination of anticancer drugs was associated with various limitations such as increased cost of treatment, systemic toxicity, drug resistance, and reduced pharmacokinetic attributes. Hence, attempts were made

Mechanism-based prediction of drug synergy via network controllability analysis of therapeutic pathways in intractable diseases.

Identifying effective drug combinations for multidrug therapies in intractable diseases remains challenging, and drug synergy depends on regulatory mechanisms. Herein, we introduce a mechanistic framework that redefines drug combination strategies into two distinct synergy paradigms-"boost" and "complement"-and present a network-based method called SYNERGIE to predict associated combinations across diseases. For diseases lacking known therapeutic targets, we introduced network controllability analysis to estimate therapeutic pathways and target molecules. SYNERGIE integrates drug-disease and d

Rare diseases and orphan drugs: Latvian story.

BackgroundTen years have passed since Latvia became a Member State of the EU in 2004. As a result European regulations, including those related to rare diseases and orphan drugs, have been applied to Latvian legislative system. Orphan diseases have been recognized as a priority area for action in the public health system, though there are significant differences in the national healthcare services for rare diseases among the EU States. This study aims to determine situation in the field of rare diseases in Latvia and compare it with other European countries.MethodsWe used the national plan for

Current drugs for HIV-1: from challenges to potential in HIV/AIDS.

The human immunodeficiency virus (HIV) persists in latently infected CD4+T cells and integrates with the host genome until cell death. Acquired immunodeficiency syndrome (AIDS) is associated with HIV-1. Possibly, treating HIV/AIDS is an essential but challenging clinical goal. This review provides a detailed account of the types and mechanisms of monotherapy and combination therapy against HIV-1 and describes nanoparticle and hydrogel delivery systems. In particular, the recently developed capsid inhibitor (Lenacapavir) and the Ainuovirine/tenofovir disoproxil fumarate/lamivudine combination (

Theranostic Potentials of Gold Nanomaterials in Hematological Malignancies.

Hematological malignancies (HMs) are a heterogeneous group of blood neoplasia generally characterized by abnormal blood-cell production. Detection of HMs-specific molecular biomarkers (e.g., surface antigens, nucleic acid, and proteomic biomarkers) is crucial in determining clinical states and monitoring disease progression. Early diagnosis of HMs, followed by an effective treatment, can remarkably extend overall survival of patients. However, traditional and advanced HMs' diagnostic strategies still lack selectivity and sensitivity. More importantly, commercially available chemotherapeutic dr

Последние новости и достижения в области диагностики и управления клетками Плазмы.

Плазменные клеточные дискразии (ПХД) охватывают разнородную группу расстройств, начиная от асимптоматических свойств-прекурсоров, таких, как моноклональная гаммопатия неопределенного значения (MGUS) и заморозка множественной миеломы (SMM) и кончая открытой множественной миеломой (MM), первичной плазменной лейкемией и системным амилоидозом. Этот обзор основан на структурированном поиске литературы, опубликованной между PubMed, Scopus, Cochrane Bibrary и Web of Science. 2015 и 2025, дополненные референцной проверкой и основными международными руководящими принципами в отношении миэломы для обесп

Множественная миелома высокого риска: от молекулярной классификации к новаторскому лечению с помощью Моноклональных антител и режиссёрской терапии Т-Келл.

Большие генетические миеломы по-прежнему являются серьезной терапевтической проблемой, поскольку пациенты, имеющие неблагоприятные генетические аномалии, такие, как дель(17p), TP53 мутации и biallelic del(1p32), по-прежнему сталкиваются с плохими результатами, несмотря на недавние терапевтические достижения. В рамках этого обзора изучаются меняющиеся определения и молекулярные характеристики HRMM с уделением особого внимания последним обновленным данным о стратегиях стратификации и лечения рисков. Новая генетическая классификация, предложенная на 2025 Совещание ЕММА обеспечивает более высокую

Targeting aberrant DNA methylation in mesenchymal stromal cells as a treatment for myeloma bone disease.

Multiple myeloma (MM) progression and myeloma-associated bone disease (MBD) are highly dependent on bone marrow mesenchymal stromal cells (MSCs). MM-MSCs exhibit abnormal transcriptomes, suggesting the involvement of epigenetic mechanisms governing their tumor-promoting functions and prolonged osteoblast suppression. Here, we identify widespread DNA methylation alterations of bone marrow-isolated MSCs from distinct MM stages, particularly in Homeobox genes involved in osteogenic differentiation that associate with their aberrant expression. Moreover, these DNA methylation changes are recapitul

Utilization of translational bioinformatics to identify novel biomarkers of bortezomib resistance in multiple myeloma.

Multiple myeloma (MM) is an incurable malignant neoplasm hallmarked by a clonal expansion of plasma cells, the presence of a monoclonal protein in the serum and/or urine (M-spike), lytic bone lesions, and end organ damage. Clinical outcomes for patients with MM have improved greatly over the last decade as a result of the re-purposing of compounds such as thalidomide derivatives, as well as the development of novel chemotherapeutic agents including first and second generation proteasome inhibitors, bortezomib (Bz) and carfilzomib. Unfortunately, despite these improvements, the majority of pati

A comprehensive tool for tumor precision medicine with pharmaco-omics data analysis.

Background: Cancer precision medicine is an effective strategy to fight cancers by bridging genomics and drug discovery to provide specific treatment for patients with different genetic characteristics. Although some public databases and modelling frameworks have been developed through studies on drug response, most of them only considered the ramifications of the drug on the cell line and the effects on the patient still require a huge amount of work to integrate data from various databases and calculations, especially concerning precision treatment. Furthermore, not only efficacy but also th

Risk and Response-Adapted Treatment in Multiple Myeloma.

Myeloma therapeutic strategies have been adapted to patients' age and comorbidities for a long time. However, although cytogenetics and clinical presentations (plasmablastic cytology; extramedullary disease) are major prognostic factors, until recently, all patients received the same treatment whatever their initial risk. No strong evidence allows us to use a personalized treatment according to one cytogenetic abnormality in newly diagnosed myeloma. Retrospective studies showed a benefit of a double autologous transplant in high-risk cytogenetics according to the International Myeloma Working

MYCN Impact on High-Risk Neuroblastoma: From Diagnosis and Prognosis to Targeted Treatment.

Among childhood cancers, neuroblastoma is the most diffuse solid tumor and the deadliest in children. While to date, the pathology has become progressively manageable with a significant increase in 5-year survival for its less aggressive form, high-risk neuroblastoma (HR-NB) remains a major issue with poor outcome and little survivability of patients. The staging system has also been improved to better fit patient needs and to administer therapies in a more focused manner in consideration of pathology features. New and improved therapies have been developed; nevertheless, low efficacy and high