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Rare disease patients protection: theoretical discussion and institutional construction of orphan drug designation in China.

Orphan drugs play a vital role, often described as the "public goods of public goods." As a result, they demand robust policy support. Among these, the designation of orphan drugs serves as the pivotal and core element of the national incentive mechanism for such drugs. The designation of orphan drugs is a prerequisite and a key system to determine whether pharmaceutical enterprises are eligible to access policy support, which directly determines the effective output of orphan drugs and the fairness of public policies. However, there is still an urgent need to explore this system in China theo

Engineering precision nanoparticles for drug delivery.

In recent years, the development of nanoparticles has expanded into a broad range of clinical applications. Nanoparticles have been developed to overcome the limitations of free therapeutics and navigate biological barriers - systemic, microenvironmental and cellular - that are heterogeneous across patient populations and diseases. Overcoming this patient heterogeneity has also been accomplished through precision therapeutics, in which personalized interventions have enhanced therapeutic efficacy. However, nanoparticle development continues to focus on optimizing delivery platforms with a one-

Вирусы и "Мозг-отношение к беде".

Неврологические расстройства, некоторые из которых связаны с вирусными инфекциями, растут из-за старения и увеличения численности населения. Несмотря на крепкую защиту центральной нервной системы, некоторые вирусы эволюционировали, чтобы пробить их, что часто приводит к ужасным последствиям. В этом обзоре мы рассказываем о различных способах проникновения различных вирусов в ЦНС и о последствиях таких вторжений. Последствия могут проявляться в виде острых заболеваний, таких, как энцефалит, менингит, или приводить к долгосрочным последствиям, таким, как нервно - мышечная дисфункция, как это име

The relationship between acceptance, fatigue, and subjective cognitive impairment in hematologic cancer survivors.

Background/Objective: Cancer and its treatment can have a detrimental impact on psychological well-being. Acceptance as the basis of acceptance and commitment therapy (ACT) has shown beneficial effects on depression and anxiety. However, its relationship to fatigue and cognitive impairment has not been investigated. A protective effect of acceptance may open up a new target for psychological intervention. Method: A cross-sectional postal survey was undertaken. 922 hematological cancer survivors (≥ 2.5 years post diagnosis) were recruited through two regional cancer registries in Germany. Accep

Psychological distress, chronic conditions and quality of life in elderly hematologic cancer patients: study protocol of a prospective study.

BackgroundSimilar to most solid tumors, the incidence of hematologic malignancies has been rising. Although the median age at diagnosis is about 70 years, little is known about psychosocial aspects and comorbid conditions in elderly patients with hematologic cancers. The main objectives of our study are to assess the prevalence of psychological distress, chronic conditions, functional disabilities, and quality of life in both elderly hematologic cancer patients aged ≥70 years and an age-matched comparison sample of the general population.MethodsWe conduct a prospective study with three measuri

Promising Therapeutic Strategies for Hematologic Malignancies: Innovations and Potential.

In this review we explore innovative approaches in the treatment of hematologic cancers by combining various therapeutic modalities. We discuss the synergistic potential of combining inhibitors targeting different cellular pathways with immunotherapies, molecular therapies, and hormonal therapies. Examples include combining PI3K inhibitors with proteasome inhibitors, NF-κB inhibitors with immunotherapy checkpoint inhibitors, and neddylation inhibitors with therapies targeting the tumor microenvironment. Additionally, we discuss the potential use of small molecules and peptide inhibitors in hem

Иммуномодулирующие свойства месенхимальных стволовых клеток и их внеклеточных везиков: потенциальные клинические применения в аутоиммунных заболеваниях.

Мезенхимальные стволовые клетки (МСЦ)/мезенхимические стромальные клетки и их внеклеточные везикли (ЭМ), в частности экзосомы, появились в качестве терапевтических кандидатов в регенеративной медицине в силу их многопотенции, низкой иммуногенности и обширных иммунодуляторных свойств. В настоящем описательном обзоре содержится сводная информация об иммуномодуляции МСЦ- и МСЦ-В и о ее потенциале в плане перевода на другие языки. four основные аутоиммунные заболевания: множественный склероз (МС), тип 1 - сахарный диабет (T1D), системный волшебный эриматоз (SLE) и ревматоидный артрит (RA).Механист

Bispecific Antibodies for the Management of Relapsed/Refractory Multiple Myeloma.

Bispecific antibodies (BsAbs) are artificially engineered antibodies that can bind simultaneously to the CD3 subunit within the T-cell receptor complex and an antigen on tumor cells, leading to T-cell activation and tumor cell killing. BsAbs against BCMA or GPRC5D have shown impressive clinical activity in heavily pretreated patients with relapsed/refractory multiple myeloma (RRMM), with some agents having already received regulatory approval after the third (by the European Medicines Agency, EMA) or fourth (by the Food and Drug Administration, FDA) line of therapy; the results of early-phase

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

Immunotherapy in Acute Myeloid Leukemia: A Literature Review of Emerging Strategies.

In the last twenty years, we have witnessed a paradigm shift in the treatment and prognosis of acute myeloid leukemia (AML), thanks to the introduction of new efficient drugs or approaches to refine old therapies, such as Gemtuzumab Ozogamicin, CPX 3-5-1, hypomethylating agents, and Venetoclax, the optimization of conditioning regimens in allogeneic hematopoietic stem cell transplantation and the improvement of supportive care. However, the long-term survival of non-M3 and non-core binding factor-AML is still dismal. For this reason, the expectations for the recently developed immunotherapies,

Совпадение в полном тексте источника
… Cross-priming of CD8(+) T cells in vivo by dendritic cells pulsed with autologous apoptotic leukemic cells in immunotherapy for elderly patients with acute myeloid leukemia Exp. Hematol. 2011 39 424 433.e2 10.1016/j.exphem.2011.01.001 21216276 25. Dong M. Liang D. Li Y. Kong D. Kang P. Li K. …

Network analysis used to investigate the interplay among somatic and psychological symptoms in patients with cancer and cancer survivors: a scoping review.

PurposePatients with cancer often experience multiple somatic and psychological symptoms. Somatic and psychological symptoms are thought to be connected and may reinforce each other. Network analysis allows examination of the interconnectedness of individual symptoms. The aim of this scoping review was to examine the current state of knowledge about the associations between somatic and psychological symptoms in patients with cancer and cancer survivors, based on network analysis.MethodsThis scoping review followed the five-stage framework of Arksey and O'Malley. The literature search was condu

Новый взгляд на механизмы нейродегенерации в экспериментальном аутоиммунном энцефаломелите и многоразовом склерозе: ранняя и критическая роль платец в нейро/аксональной потере.

Множественный склероз (MS) — аутоиммунное расстройство центральной нервной системы (CNS) с ограниченными возможностями лечения. Для этого заболевания характерны различия в патофизиологии между серым веществом (GM) и белым веществом (WM). Преобладающей отличительной чертой ВМ является периваскулярная доска, связанная с потерей функции мозгового барьера (ББД), инфильтрацией лимфоцитов, микроглиальной реактивностью, обезвоживанием и аксонной травмой и должным образом обработанная иммуномодулятивными препаратами. С другой стороны, механизмы, лежащие в основе ущерба ГМ, остаются неясными, что имеет

Удельное микробное соотношение в кишечном микробиоме связано с множественным склерозом.

Гут микробиота дисбиоз связан с множественным склерозом (MS), однако причинно-следственная связь между конкретными кишечными бактериями и патогенезом MS остается слабой. Поэтому мы описали микробиом стула людей с MS (PwMS) и здоровым управлением (HC) с использованием метагеномической последовательности дробовика. PwMS продемонстрировал отличный микробиом по сравнению с HC, при этом виды Crevotella copri (PC) и Blautia были движущими силами микробных сообществ в HC и PwMS, соответственно. В результате применения к мышам видов MS-ding Blautia (Blautia Wexlerae; BW) повысились уровни кишечных мар

Diabetes and obesity: the role of stress in the development of cancer.

Diabesity is a condition where an individual has both diabetes and obesity, which can lead to severe complications including cardiovascular disease, a leading cause of mortality. Recently, cancer has become a leading cause of excess hospitalizations, and both diabetes and obesity are associated with a higher risk of developing several types of cancer. In this review, we propose that chronic stress significantly increases this association. Managing diabetes and obesity is challenging as they both cause significant distress. The relationship between stress and cancer is interconnected, with anxi

Neurologic adverse events of cancer immunotherapy.

Cancer immunotherapy encompasses a wide range of treatment modalities that harness the anti-tumor effects of the immune system and have revolutionized oncological treatment in recent years, with approval for its use in more and more cancers. However, it is not without side effects. Several neurological adverse events have been recognized associated with immune checkpoint inhibitors (ICI) and chimeric antigen receptor (CAR) T-cell therapy, the two main classes of cancer immunotherapy. With the increase in the prevalence of oncological diseases and this type of therapy, it is improbable that neu

Sex Differences in Cancer Immunotherapy-Clinical Evidence and Mechanisms With a Focus on NSCLC.

Accumulating evidence suggests that the immune system shows subtle but relevant differences between men and women. These differences may have an impact on cancer development and TME composition as well as responses to and adverse events elicited by immunotherapies. Several, albeit not all, clinical trials indicate a greater benefit from mono-immunotherapies over chemotherapies for male patients than for female patients, especially in non-small cell lung cancer and melanoma. Vice versa, female patients might benefit more from chemo-immunotherapies. In human as well as animal models, sex differe

The Evolving Landscape of Immune Regulation and Immunotherapy in Cholangiocarcinoma and Biliary Tract Cancer.

Cholangiocarcinoma (CCA) is an aggressive and molecularly heterogeneous malignancy characterized by a profoundly immunosuppressive tumor microenvironment (TME) and historically limited therapeutic options. Recent advances have redefined the treatment paradigm, with phase III trials establishing chemoimmunotherapy as a standard of care and multi-omic profiling elucidating the interplay between tumor genomics, stromal architecture, and immune regulation. Despite these gains, durable clinical benefit remains confined to a minority of patients, reflecting convergent mechanisms of primary and acqui

Anti-PEG Antibodies and Their Biological Impact on PEGylated Drugs: Challenges and Strategies for Optimization.

Polyethylene glycol (PEG) has been widely utilized in optimizing therapeutics due to its excellent biocompatibility and chemical stability. However, multiple dosing of PEGylated drugs may result in toxicity due to PEG accumulation in tissues, leading to the formation of anti-PEG antibodies (APAs), which can accelerate drug clearance, reduce efficacy, and alongside enhanced side effects, such as allergic reactions. Notably, pre-existing APAs have also been detected in individuals with no prior exposure to PEGylated drugs, raising additional clinical concerns. This review summarizes the mechanis

Adeno-associated virus as a delivery vector for gene therapy of human diseases.

Adeno-associated virus (AAV) has emerged as a pivotal delivery tool in clinical gene therapy owing to its minimal pathogenicity and ability to establish long-term gene expression in different tissues. Recombinant AAV (rAAV) has been engineered for enhanced specificity and developed as a tool for treating various diseases. However, as rAAV is being more widely used as a therapy, the increased demand has created challenges for the existing manufacturing methods. Seven rAAV-based gene therapy products have received regulatory approval, but there continue to be concerns about safely using high-dos

Computational proteomics to enhance personalized treatment of COVID-19 and Long COVID.

The coronavirus disease 2019 (COVID-19) pandemic, caused by severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2), has led to significant global health burden, including both acute infections and persistent post-acute sequelae, also known as Long COVID (LC) in survivors. While clinical management has reduced case-fatality rates, a substantial proportion of patients develop LC, a heterogeneous syndrome with long-term symptoms. This complex continuum requires therapeutic strategies for both the acute and chronic phases. Plasma proteomics has emerged as a powerful tool in precision medicin