scholarly journals Novel therapeutic approaches for the management of cystic fibrosis

2020 ◽  
Vol 15 ◽  
Author(s):  
Ryan Jaques ◽  
Arslan Shakeel ◽  
Cameron Hoyle
Keyword(s):  
Cystic Fibrosis ◽  
Recurrent Infection ◽  
Current Evidence ◽  
Therapeutic Approaches ◽  
Genetic Condition ◽  
Disease Treatment ◽  
Pharmacological Agents ◽  
Ongoing Research ◽  
Mucus Clearance ◽  
Made In

Cystic fibrosis (CF) is a genetic condition characterised by the build-up of thick, sticky mucus that can damage many of the body’s organs. It is a life-long disease that results in a shortened life expectancy, often due to the progression of advanced lung disease. Treatment has previously targeted the downstream symptoms such as diminished mucus clearance and recurrent infection. More recently, significant advances have been made in treating the cause of the disease by targeting the faulty gene responsible. Hope for the development of potential therapies lies with ongoing research into new pharmacological agents and gene therapy. This review gives an overview of CF, and summarises the current evidence regarding the disease management and upcoming strategies aimed at treating or potentially curing this condition.

scholarly journals JAK2 (and other genes) be nimble with MPN diagnosis, prognosis, and therapy

Hematology ◽  
2018 ◽  
Vol 2018 (1) ◽  
pp. 110-117 ◽  
Author(s):  
Michele Ciboddo ◽  
Ann Mullally
Keyword(s):  
Clinical Decision Making ◽  
Phenotypic Diversity ◽  
Myeloproliferative Neoplasms ◽  
Clinical Decision ◽  
Prognostic Models ◽  
Driver Mutations ◽  
Therapeutic Approaches ◽  
Ongoing Research ◽  
Substantial Progress ◽  
Made In

Abstract Now that the spectrum of somatic mutations that initiate, propagate, and drive the progression of myeloproliferative neoplasms (MPNs) has largely been defined, recent efforts have focused on integrating this information into clinical decision making. In this regard, the greatest progress has been made in myelofibrosis, in which high-molecular-risk mutations have been identified and incorporated into prognostic models to help guide treatment decisions. In this chapter, we focus on advances in 4 main areas: (1) What are the MPN phenotypic driver mutations? (2) What constitutes high molecular risk in MPN (focusing on ASXL1)? (3) How do we risk-stratify patients with MPN? And (4) What is the significance of molecular genetics for MPN treatment? Although substantial progress has been made, we still have an incomplete understanding of the molecular basis for phenotypic diversity in MPN, and few rationally designed therapeutic approaches to target high-risk mutations are available. Ongoing research efforts in these areas are critical to understanding the biological consequences of genetic heterogeneity in MPN and to improving outcomes for patients.

scholarly journals Clinical trials for treatment or prevention of COVID-19. A review of Clinicaltrials.gov.

F1000Research ◽  
2021 ◽  
Vol 9 ◽  
pp. 823
Author(s):  
Martha Fors ◽  
Paloma González
Keyword(s):  
Clinical Trials ◽  
Phase Ii ◽  
Randomized Trials ◽  
Descriptive Study ◽  
Therapeutic Approaches ◽  
Open Label ◽  
Disease Treatment ◽  
Cross Sectional ◽  
Ongoing Research ◽  
Antimalarial Agents

Background: Coronavirus disease 2019 (COVID-19) has rapidly progressed into the worst pandemic in recent years. There are currently no approved therapies to treat the disease. Several clinical trials are being conducted to evaluate therapeutic approaches. Methods: We conducted a cross-sectional descriptive study to examine the main characteristics of COVID-19-related clinical interventional trials registered with ClinicalTrials.gov  from January to March 27th, 2020. . Results: We included 519 trials, 57.6% were phase II or III, open-label and randomized trials. Disease treatment was evaluated in 75.5% of trials, while prevention was evaluated in 12.1%. A total of 243 trials were listed as recruiting, and 42.4% were not yet recruiting. Approximately 20% of the analyzed trials are investigating antimalarial agents, while 10.2% are studying the use of convalescent plasma to treat the disease. Antiretrovirals, monoclonal antibodies, the use of stem cells, nitric oxide gases and vaccines are the most commonly evaluated therapies. As of the publication of this review, none of the clinical trials had uploaded results. Conclusions: ClinicalTrials.gov is an important database that contains ongoing research trials on COVID-19This study quantifies the outcomes of COVID-19-related clinical trials.  More than 500 studies have been  analyzed finding that most of these studies are interventional clinical trials  Phase II or III evaluating drugs or biological agents for the prevention or treating COVID-19.

scholarly journals Who is making clinical trials for treatment or prevention of COVID-19? A review of Clinicaltrials.gov, May 2020

F1000Research ◽  
2020 ◽  
Vol 9 ◽  
pp. 823
Author(s):  
Martha Fors ◽  
Paloma González
Keyword(s):  
Nitric Oxide ◽  
Clinical Trials ◽  
Randomized Trials ◽  
Descriptive Study ◽  
Therapeutic Approaches ◽  
Open Label ◽  
Disease Treatment ◽  
Cross Sectional ◽  
Ongoing Research ◽  
Antimalarial Agents

Background: Coronavirus disease 2019 (COVID-19) has rapidly progressed into the worst pandemic in recent years. There are currently no approved therapies to treat the disease. Several clinical trials are being conducted to evaluate therapeutic approaches. Methods: We conducted a cross-sectional descriptive study to examine the main characteristics of COVID-19-related clinical interventional trials registered with ClinicalTrials.gov until May 15th, 2020. Results: We included 519 trials, most of which were phase II or III, open-label and randomized trials. Disease treatment was evaluated in 75.5% of trials, while prevention was evaluated in 12.1%. A total of 243 trials were listed as recruiting, and 42.4% were not yet recruiting. Approximately 20% of the analyzed trials are investigating antimalarial agents, while 10.2% are studying the use of convalescent plasma to treat the disease. Antiretrovirals, monoclonal antibodies, the use of stem cells, nitric oxide gases and vaccines are the most commonly evaluated therapies. As of the publication of this review, none of the clinical trials had uploaded results. Conclusions: ClinicalTrials.gov is an important database that contains ongoing research trials on COVID-19, a disease that is of vital importance. This study quantifies the outcomes of COVID-19-related clinical trials. The safety and effectiveness of many therapeutic approaches are investigated to fight this disease.

Natural Compounds and Drug Discovery: Can Cnidarian Venom Play a Role?

2019 ◽  
Vol 19 (2) ◽  
pp. 114-118
Author(s):  
Gian Luigi Mariottini ◽  
Irwin Darren Grice
Keyword(s):  
Biological Activity ◽  
Marine Species ◽  
Natural Compounds ◽  
Therapeutic Agents ◽  
Industrial Applications ◽  
Bioactive Molecules ◽  
Sea Anemones ◽  
Therapeutic Approaches ◽  
Ongoing Research ◽  
Biological Compounds

Natural compounds extracted from organisms and microorganisms are an important resource for the development of drugs and bioactive molecules. Many such compounds have made valuable contributions in diverse fields such as human health, pharmaceutics and industrial applications. Presently, however, research on investigating natural compounds from marine organisms is scarce. This is somewhat surprising considering that the marine environment makes a major contribution to Earth's ecosystems and consequently possesses a vast storehouse of diverse marine species. Interestingly, of the marine bioactive natural compounds identified to date, many are venoms, coming from Cnidarians (jellyfish, sea anemones, corals). Cnidarians are therefore particularly interesting marine species, producing important biological compounds that warrant further investigation for their development as possible therapeutic agents. From an experimental aspect, this review aims to emphasize and update the current scientific knowledge reported on selected biological activity (antiinflammatory, antimicrobial, antitumoral, anticoagulant, along with several less studied effects) of Cnidarian venoms/extracts, highlighting potential aspects for ongoing research towards their utilization in human therapeutic approaches.

scholarly journals Immune Sensing and Potential Immunotherapeutic Approaches to Control Chromoblastomycosis

2020 ◽  
Vol 7 (1) ◽  
pp. 3
Author(s):  
Leandro C. D. Breda ◽  
Isabela G. Menezes ◽  
Larissa N. M. Paulo ◽  
Sandro Rogério de Almeida
Keyword(s):  
Social Stigma ◽  
Treatment Strategies ◽  
Specific Treatment ◽  
Treatment Interruption ◽  
Lower Limbs ◽  
Disease Treatment ◽  
Immune Sensing ◽  
Skin Immunology ◽  
Anti Fungal ◽  
Made In

Chromoblastomycosis (CBM) is a neglected, chronic, and progressive subcutaneous mycosis caused by different species of fungi from the Herpotrichiellaceae family. CBM disease is usually associated with agricultural activities, and its infection is characterized by verrucous, erythematous papules, and atrophic lesions on the upper and lower limbs, leading to social stigma and impacts on patients’ welfare. The economic aspect of disease treatment is another relevant issue. There is no specific treatment for CBM, and different anti-fungal drug associations are used to treat the patients. However, the long period of the disease and the high cost of the treatment lead to treatment interruption and, consequently, relapse of the disease. In previous years, great progress had been made in the comprehension of the CBM pathophysiology. In this review, we discuss the differences in the cell wall composition of conidia, hyphae, and muriform cells, with a particular focus on the activation of the host immune response. We also highlight the importance of studies about the host skin immunology in CBM. Finally, we explore different immunotherapeutic studies, highlighting the importance of these approaches for future treatment strategies for CBM.

scholarly journals LAT1 and ASCT2 Related microRNAs as Potential New Therapeutic Agents against Colorectal Cancer Progression

Biomedicines ◽  
2021 ◽  
Vol 9 (2) ◽  
pp. 195
Author(s):  
Francisca Dias ◽  
Cristina Almeida ◽  
Ana Luísa Teixeira ◽  
Mariana Morais ◽  
Rui Medeiros
Keyword(s):  
Colorectal Cancer ◽  
Amino Acid ◽  
Cancer Progression ◽  
Cell Metabolism ◽  
Tumor Development ◽  
Amino Acid Transporters ◽  
Epigenetic Alterations ◽  
Therapeutic Approaches ◽  
Colorectal Cancer Progression ◽  
Made In

The development and progression of colorectal cancer (CRC) have been associated with genetic and epigenetic alterations and more recently with changes in cell metabolism. Amino acid transporters are key players in tumor development, and it is described that tumor cells upregulate some AA transporters in order to support the increased amino acid (AA) intake to sustain the tumor additional needs for tumor growth and proliferation through the activation of several signaling pathways. LAT1 and ASCT2 are two AA transporters involved in the regulation of the mTOR pathway that has been reported as upregulated in CRC. Some attempts have been made in order to develop therapeutic approaches to target these AA transporters, however none have reached the clinical setting so far. MiRNA-based therapies have been gaining increasing attention from pharmaceutical companies and now several miRNA-based drugs are currently in clinical trials with promising results. In this review we combine a bioinformatic approach with a literature review in order to identify a miRNA profile with the potential to target both LAT1 and ASCT2 with potential to be used as a therapeutic approach against CRC.

scholarly journals Systems Biology and Bile Acid Signalling in Microbiome-Host Interactions in the Cystic Fibrosis Lung

Antibiotics ◽  
2021 ◽  
Vol 10 (7) ◽  
pp. 766
Author(s):  
David F. Woods ◽  
Stephanie Flynn ◽  
Jose A. Caparrós-Martín ◽  
Stephen M. Stick ◽  
F. Jerry Reen ◽  
...  
Keyword(s):  
Cystic Fibrosis ◽  
Bile Acids ◽  
Host Response ◽  
Signal Molecules ◽  
Therapeutic Approaches ◽  
Related Factors ◽  
Host Interactions ◽  
Important Host ◽  
Respiratory Microbiota

The study of the respiratory microbiota has revealed that the lungs of healthy and diseased individuals harbour distinct microbial communities. Imbalances in these communities can contribute to the pathogenesis of lung disease. How these imbalances occur and establish is largely unknown. This review is focused on the genetically inherited condition of Cystic Fibrosis (CF). Understanding the microbial and host-related factors that govern the establishment of chronic CF lung inflammation and pathogen colonisation is essential. Specifically, dissecting the interplay in the inflammation–pathogen–host axis. Bile acids are important host derived and microbially modified signal molecules that have been detected in CF lungs. These bile acids are associated with inflammation and restructuring of the lung microbiota linked to chronicity. This community remodelling involves a switch in the lung microbiota from a high biodiversity/low pathogen state to a low biodiversity/pathogen-dominated state. Bile acids are particularly associated with the dominance of Proteobacterial pathogens. The ability of bile acids to impact directly on both the lung microbiota and the host response offers a unifying principle underpinning the pathogenesis of CF. The modulating role of bile acids in lung microbiota dysbiosis and inflammation could offer new potential targets for designing innovative therapeutic approaches for respiratory disease.

scholarly journals The Pivotal Player: Components of NF-κB Pathway as Promising Biomarkers in Colorectal Cancer

2021 ◽  
Vol 22 (14) ◽  
pp. 7429
Author(s):  
Matthew Martin ◽  
Mengyao Sun ◽  
Aishat Motolani ◽  
Tao Lu
Keyword(s):  
Colorectal Cancer ◽  
Biological Response ◽  
Significant Progress ◽  
Therapeutic Approaches ◽  
Successful Development ◽  
Diagnostic Screening ◽  
Proteomic Data ◽  
Large Groups ◽  
Made In

Over the last several decades, colorectal cancer (CRC) has been one of the most prevalent cancers. While significant progress has been made in both diagnostic screening and therapeutic approaches, a large knowledge gap still remains regarding the early identification and treatment of CRC. Specifically, identification of CRC biomarkers that can help with the creation of targeted therapies as well as increasing the ability for clinicians to predict the biological response of a patient to therapeutics, is of particular importance. This review provides an overview of CRC and its progression stages, as well as the basic types of CRC biomarkers. We then lay out the synopsis of signaling pathways related to CRC, and further highlight the pivotal and multifaceted role of nuclear factor (NF) κB signaling in CRC. Particularly, we bring forth knowledge regarding the tumor microenvironment (TME) in CRC, and its complex interaction with cancer cells. We also provide examples of NF-κB signaling-related CRC biomarkers, and ongoing efforts made at targeting NF-κB signaling in CRC treatment. We conclude and anticipate that with more emerging novel regulators of the NF-κB pathway being discovered, together with their in-depth characterization and the integration of large groups of genomic, transcriptomic and proteomic data, the day of successful development of more ideal NF-κB inhibitors is fast approaching.

scholarly journals Hepatocyte Turnover in Chronic HCV-Induced Liver Injury and Cirrhosis

2015 ◽  
Vol 2015 ◽  
pp. 1-8 ◽  
Author(s):  
Nikolaos P. Karidis ◽  
Ioanna Delladetsima ◽  
Stamatios Theocharis
Keyword(s):  
Current Evidence ◽  
Hepatocellular Injury ◽  
Ongoing Research ◽  
Molecular Events ◽  
Parenchymal Liver ◽  
B Virus ◽  
Multistep Process ◽  
Differential Gene ◽  
Chronic Hcv ◽  
Hepatocyte Death

Chronic hepatitis C virus (HCV) infection may eventually lead to progressive liver fibrosis and cirrhosis through a complex, multistep process involving hepatocyte death and regeneration. Despite common pathogenetic pathways present in all forms of liver cirrhosis irrespective of etiology, hepatocyte turnover and related molecular events in HCV-induced cirrhosis are increasingly being distinguished from even “similar” causes, such as hepatitis B virus- (HBV-) related cirrhosis. New insights in HCV-induced hepatocellular injury, differential gene expression, and regenerative pathways have recently revealed a different pattern of progression to irreversible parenchymal liver damage. A shift to the significant role of the host immune response rather than the direct effect of HCV on hepatocytes and the imbalance between antiapoptotic and proapoptotic signals have been investigated in several studies but need to be further elucidated. The present review aims to comprehensively summarize the current evidence on HCV-induced hepatocellular turnover with a view to outline the significant trends of ongoing research.

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