Pub Date : 2020-10-27DOI: 10.1080/23808993.2020.1816136
Nourhan Hossam, M. Matboli, H. Shehata, Marwa M. Aboelhussein, M. Hassan, S. Eissa
ABSTRACT Colorectal cancer (CRC) is characterized by high heterogeneity and a complex microenvironment that leads to high inter-patient variability. Personalized management of CRC could address this. Accumulating data highlights the interaction between the CRC microenvironment and the immune system through different cells and receptors with a focus on the toll-like receptors (TLRs). Multiple studies identified a bidirectional role played by TLRs in CRC with involvement in both carcinogenesis and therapy. Areas covered A study to highlight the interaction between TLRs and CRC microenvironment on different molecular levels was undertaken, addressing TLR gene polymorphism, TLR genetic and epigenetic deregulation and TLR ligand binders. In addition, the use of these TLRs and their interaction with CRC microenvironment were evaluated to identify novel CRC therapeutics. Expert commentary Previous literature has shown that TLRs are incriminated in CRC pathogenesis and thus research effort was directed to make use of these TLRs in drawing new therapeutic patterns for CRC. However, to date, these immune-therapeutic patterns of CRC have shown limited success in reducing tumor burden. This highlights the need for more studies that would better illustrate the interaction between TLRs and CRC microenvironment and the impact of TLR modifications to yield more efficient and precise CRC therapeutics.
{"title":"Toll-like receptor immune modulatory role in personalized management of colorectal cancer, review of literature","authors":"Nourhan Hossam, M. Matboli, H. Shehata, Marwa M. Aboelhussein, M. Hassan, S. Eissa","doi":"10.1080/23808993.2020.1816136","DOIUrl":"https://doi.org/10.1080/23808993.2020.1816136","url":null,"abstract":"ABSTRACT Colorectal cancer (CRC) is characterized by high heterogeneity and a complex microenvironment that leads to high inter-patient variability. Personalized management of CRC could address this. Accumulating data highlights the interaction between the CRC microenvironment and the immune system through different cells and receptors with a focus on the toll-like receptors (TLRs). Multiple studies identified a bidirectional role played by TLRs in CRC with involvement in both carcinogenesis and therapy. Areas covered A study to highlight the interaction between TLRs and CRC microenvironment on different molecular levels was undertaken, addressing TLR gene polymorphism, TLR genetic and epigenetic deregulation and TLR ligand binders. In addition, the use of these TLRs and their interaction with CRC microenvironment were evaluated to identify novel CRC therapeutics. Expert commentary Previous literature has shown that TLRs are incriminated in CRC pathogenesis and thus research effort was directed to make use of these TLRs in drawing new therapeutic patterns for CRC. However, to date, these immune-therapeutic patterns of CRC have shown limited success in reducing tumor burden. This highlights the need for more studies that would better illustrate the interaction between TLRs and CRC microenvironment and the impact of TLR modifications to yield more efficient and precise CRC therapeutics.","PeriodicalId":12124,"journal":{"name":"Expert Review of Precision Medicine and Drug Development","volume":"5 1","pages":"455 - 468"},"PeriodicalIF":1.2,"publicationDate":"2020-10-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1080/23808993.2020.1816136","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"42418712","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2020-10-25DOI: 10.1080/23808993.2020.1831909
M. Heiblig, Sabine Hachem-Khalife, C. Willekens, J. Micol, A. Paci, V. Penard-Lacronique, S. de Botton
ABSTRACT Introduction Isocitrate dehydrogenase 2 (IDH2) is a key metabolic enzyme that converts isocitrate to α-ketoglutarate (αKG). Somatic point mutations in IDH2 confer a gain-of-function in blast cells resulting in overproduction of D-2-hydroxyglutarate (D-2HG). High intracellular concentrations of D-2HG inhibit α-ketoglutarate-dependent dioxygenases including histone, DNA and RNA demethylases, leading to histone, DNA and RNA hypermethylation, and cell differentiation blockade. In vitro and in vivo preclinical studies have demonstrated that inhibition of IDH2-mutant enzymes with enasidenib decrease intracellular D-2HG levels, reverse epigenetic dysregulation, and release the differentiation block. The US Food and Drug Administration (FDA) approved enasidenib, a mutant-IDH2 enzyme inhibitor for patients with relapsed or refractory (R/R) IDH2-mutated AML. Areas covered We review the biology and prognostic significance of IDH2 mutations in AML and discuss the pharmacology, clinical efficacy, and toxicity profile of enasidenib. We highlight areas of ongoing preclinical and clinical research. Expert opinion Enasidenib was FDA approved due to high response rates, durability of the responses that translated into an impressive OS in that heavily pretreated population. Promising ongoing clinical trials are evaluating combination therapies with enasidenib frontline.
{"title":"Enasidenib for the treatment of relapsed or refractory acute myeloid leukemia with an isocitrate dehydrogenase 2 mutation","authors":"M. Heiblig, Sabine Hachem-Khalife, C. Willekens, J. Micol, A. Paci, V. Penard-Lacronique, S. de Botton","doi":"10.1080/23808993.2020.1831909","DOIUrl":"https://doi.org/10.1080/23808993.2020.1831909","url":null,"abstract":"ABSTRACT Introduction Isocitrate dehydrogenase 2 (IDH2) is a key metabolic enzyme that converts isocitrate to α-ketoglutarate (αKG). Somatic point mutations in IDH2 confer a gain-of-function in blast cells resulting in overproduction of D-2-hydroxyglutarate (D-2HG). High intracellular concentrations of D-2HG inhibit α-ketoglutarate-dependent dioxygenases including histone, DNA and RNA demethylases, leading to histone, DNA and RNA hypermethylation, and cell differentiation blockade. In vitro and in vivo preclinical studies have demonstrated that inhibition of IDH2-mutant enzymes with enasidenib decrease intracellular D-2HG levels, reverse epigenetic dysregulation, and release the differentiation block. The US Food and Drug Administration (FDA) approved enasidenib, a mutant-IDH2 enzyme inhibitor for patients with relapsed or refractory (R/R) IDH2-mutated AML. Areas covered We review the biology and prognostic significance of IDH2 mutations in AML and discuss the pharmacology, clinical efficacy, and toxicity profile of enasidenib. We highlight areas of ongoing preclinical and clinical research. Expert opinion Enasidenib was FDA approved due to high response rates, durability of the responses that translated into an impressive OS in that heavily pretreated population. Promising ongoing clinical trials are evaluating combination therapies with enasidenib frontline.","PeriodicalId":12124,"journal":{"name":"Expert Review of Precision Medicine and Drug Development","volume":"5 1","pages":"421 - 428"},"PeriodicalIF":1.2,"publicationDate":"2020-10-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1080/23808993.2020.1831909","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"47392961","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2020-10-21DOI: 10.1080/23808993.2020.1834843
N. Navanandan, S. Szefler
ABSTRACT Introduction Asthma is the most common chronic illness in the pediatric population and is characterized by substantial heterogeneity in phenotypes and treatment response. A precision medicine approach is required to advance asthma care and improve asthma morbidity in children. Areas covered We present a review of currently available biomarkers for the diagnosis and management of pediatric asthma. We discuss a core set of biomarkers recognized by the National Institute of Health Asthma Outcomes Task Force including eosinophil counts, IgE levels, and fraction of exhaled nitric oxide. We also review emerging biomarkers including periostin, thymic stromal lymphopoietin, and volatile organic compounds. These biomarkers have proven effective in differentiating asthma phenotypes and predicting and monitoring treatment response, and have led to the development of game-changing biologic therapies for pediatric asthma. Expert opinion Substantial progress has been made in the identification of key biomarkers to aid in the classification and management of pediatric asthma. However, for biomarkers to be used routinely in clinical practice, further investigations are needed to expand biomarker representation of additional asthma phenotypes. A systems biology approach is also required, combining the various ‘omic’ strategies, for precision medicine to reach its full potential in pediatric asthma management.
{"title":"Personalized asthma management in pediatric patients based on treatment response","authors":"N. Navanandan, S. Szefler","doi":"10.1080/23808993.2020.1834843","DOIUrl":"https://doi.org/10.1080/23808993.2020.1834843","url":null,"abstract":"ABSTRACT Introduction Asthma is the most common chronic illness in the pediatric population and is characterized by substantial heterogeneity in phenotypes and treatment response. A precision medicine approach is required to advance asthma care and improve asthma morbidity in children. Areas covered We present a review of currently available biomarkers for the diagnosis and management of pediatric asthma. We discuss a core set of biomarkers recognized by the National Institute of Health Asthma Outcomes Task Force including eosinophil counts, IgE levels, and fraction of exhaled nitric oxide. We also review emerging biomarkers including periostin, thymic stromal lymphopoietin, and volatile organic compounds. These biomarkers have proven effective in differentiating asthma phenotypes and predicting and monitoring treatment response, and have led to the development of game-changing biologic therapies for pediatric asthma. Expert opinion Substantial progress has been made in the identification of key biomarkers to aid in the classification and management of pediatric asthma. However, for biomarkers to be used routinely in clinical practice, further investigations are needed to expand biomarker representation of additional asthma phenotypes. A systems biology approach is also required, combining the various ‘omic’ strategies, for precision medicine to reach its full potential in pediatric asthma management.","PeriodicalId":12124,"journal":{"name":"Expert Review of Precision Medicine and Drug Development","volume":"5 1","pages":"439 - 446"},"PeriodicalIF":1.2,"publicationDate":"2020-10-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1080/23808993.2020.1834843","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"43820455","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2020-10-18DOI: 10.1080/23808993.2020.1831910
C. Werno, Kamran Honarnejad, B. Polzer
ABSTRACT Introduction Circulating tumor and disseminated cancer cells can be detected after surgical removal of the primary tumor in non-metastatic patients. Despite being considered the prime targets of adjuvant therapy, they have not been implemented into clinical decision making yet. Areas covered Here we review the recent progress in understanding the biology of circulating tumor cells and disseminated cancer cells as well as the technical challenges associated with quantification, isolation, and preclinical model development. We highlight the first examples of clinical studies in which metastasis founder cells have been used as surrogate markers in adjuvant cancer patients and address the current hurdles in implementing these in routine clinical application. Finally, we provide a perspective on how the combination of technologies to detect and isolate metastasis founders, single-cell multi-omics, development of preclinical models, and their drug responses in specific niches could improve personalized adjuvant treatment strategies. Expert opinion The specific target cells of adjuvant cancer treatment are metastasis founder cells that remain in the body of the patients after surgical removal of the primary tumor. We, therefore, believe that the success of adjuvant therapies will be improved by implementing circulating and disseminated cancer cells in future clinical decision making.
{"title":"Predicting therapy response by analysis of metastasis founder cells: emerging perspectives for personalized tumor therapy","authors":"C. Werno, Kamran Honarnejad, B. Polzer","doi":"10.1080/23808993.2020.1831910","DOIUrl":"https://doi.org/10.1080/23808993.2020.1831910","url":null,"abstract":"ABSTRACT Introduction Circulating tumor and disseminated cancer cells can be detected after surgical removal of the primary tumor in non-metastatic patients. Despite being considered the prime targets of adjuvant therapy, they have not been implemented into clinical decision making yet. Areas covered Here we review the recent progress in understanding the biology of circulating tumor cells and disseminated cancer cells as well as the technical challenges associated with quantification, isolation, and preclinical model development. We highlight the first examples of clinical studies in which metastasis founder cells have been used as surrogate markers in adjuvant cancer patients and address the current hurdles in implementing these in routine clinical application. Finally, we provide a perspective on how the combination of technologies to detect and isolate metastasis founders, single-cell multi-omics, development of preclinical models, and their drug responses in specific niches could improve personalized adjuvant treatment strategies. Expert opinion The specific target cells of adjuvant cancer treatment are metastasis founder cells that remain in the body of the patients after surgical removal of the primary tumor. We, therefore, believe that the success of adjuvant therapies will be improved by implementing circulating and disseminated cancer cells in future clinical decision making.","PeriodicalId":12124,"journal":{"name":"Expert Review of Precision Medicine and Drug Development","volume":"5 1","pages":"413 - 420"},"PeriodicalIF":1.2,"publicationDate":"2020-10-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1080/23808993.2020.1831910","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"42656223","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2020-10-12DOI: 10.1080/23808993.2020.1812382
Kaining Zhi, Asit Kumar, B. Raji, H. Kochat, Santosh Kumar
ABSTRACT Introduction Extracellular vesicles (EVs) are a family of natural nanosize vesicles that transport biological cargos, including DNA, RNA, protein, and lipids. In recent years, EVs have attracted a lot of attention for their capability to function as a drug delivery system (DDS). While clinical trials have been conducted, the techniques to formulate, process, and quality control EVs-based drug products still have the potential to be improved, especially in large scale production. Areas covered We will introduce and discuss EVs biology, their potential role in CNS pathologies under different conditions, and recent methods of isolation. We will then provide a detailed discussion on current benchtop formulation methods, quality control methods, and regulatory affairs for EVs-based drug products. Expert opinion Despite being a hot topic, EVs-based drug products have not received any approval from regulatory agencies. In this review, we will provide general guidance to help EVs-based drug products to ‘move from bench to bedside’.
{"title":"Formulation, manufacturing and regulatory strategies for extracellular vesicles-based drug products for targeted therapy of central nervous system diseases","authors":"Kaining Zhi, Asit Kumar, B. Raji, H. Kochat, Santosh Kumar","doi":"10.1080/23808993.2020.1812382","DOIUrl":"https://doi.org/10.1080/23808993.2020.1812382","url":null,"abstract":"ABSTRACT Introduction Extracellular vesicles (EVs) are a family of natural nanosize vesicles that transport biological cargos, including DNA, RNA, protein, and lipids. In recent years, EVs have attracted a lot of attention for their capability to function as a drug delivery system (DDS). While clinical trials have been conducted, the techniques to formulate, process, and quality control EVs-based drug products still have the potential to be improved, especially in large scale production. Areas covered We will introduce and discuss EVs biology, their potential role in CNS pathologies under different conditions, and recent methods of isolation. We will then provide a detailed discussion on current benchtop formulation methods, quality control methods, and regulatory affairs for EVs-based drug products. Expert opinion Despite being a hot topic, EVs-based drug products have not received any approval from regulatory agencies. In this review, we will provide general guidance to help EVs-based drug products to ‘move from bench to bedside’.","PeriodicalId":12124,"journal":{"name":"Expert Review of Precision Medicine and Drug Development","volume":"5 1","pages":"469 - 481"},"PeriodicalIF":1.2,"publicationDate":"2020-10-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1080/23808993.2020.1812382","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"42001510","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2020-10-10DOI: 10.1080/23808993.2020.1825937
S. Cooke, P. Beer
For learnings from academic research to impact health outcomes, they must be embedded into routine clinical practice. Research innovations such as the human genome project, next-generation sequenci...
{"title":"Bridging the gaps between cancer genomics, computational solutions and healthcare delivery","authors":"S. Cooke, P. Beer","doi":"10.1080/23808993.2020.1825937","DOIUrl":"https://doi.org/10.1080/23808993.2020.1825937","url":null,"abstract":"For learnings from academic research to impact health outcomes, they must be embedded into routine clinical practice. Research innovations such as the human genome project, next-generation sequenci...","PeriodicalId":12124,"journal":{"name":"Expert Review of Precision Medicine and Drug Development","volume":"5 1","pages":"409 - 411"},"PeriodicalIF":1.2,"publicationDate":"2020-10-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1080/23808993.2020.1825937","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"43558214","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2020-08-16DOI: 10.1080/23808993.2020.1801346
A. Sayed, M. Munir, Noor Eweis, Doaa Wael, O. Shazly, A. Awad, Marihan A. Elbadawy, S. Eissa
ABSTRACT Introduction Bladder cancer is a common global cause of morbidity and mortality, with many patients not responding to -or not tolerating- traditional chemotherapeutic regimens. Recent studies have allowed us to utilize the genetic profile of tumors to better target therapy as is required, as well as innovate novel therapeutic interventions usable when more traditional options prove futile. Areas covered The development of novel interventions in bladder cancer, particularly immune checkpoint inhibitors, as well as using genetic markers to guide both traditional and novel therapeutic interventions. We also discuss the utility of these markers in diagnosing and prognosticating bladder cancer patients. Expert opinion Biomarker-guided therapy could revolutionize bladder cancer care in several ways: not only do novel therapeutic agents provide alternate treatment options for more difficult cases, but it can also increase the efficacy of more traditional treatment options. In addition, it may have a role in the early diagnosis and detection of bladder cancer, as well as predicting the course and prognosis of these patients. Unresolved challenges include how to best optimize therapy with novel agents as regarding duration and patient selection, as well as investigations as to whether using gene-guidance results in clinically improved patient outcomes.
{"title":"An overview on precision therapy in bladder cancer","authors":"A. Sayed, M. Munir, Noor Eweis, Doaa Wael, O. Shazly, A. Awad, Marihan A. Elbadawy, S. Eissa","doi":"10.1080/23808993.2020.1801346","DOIUrl":"https://doi.org/10.1080/23808993.2020.1801346","url":null,"abstract":"ABSTRACT Introduction Bladder cancer is a common global cause of morbidity and mortality, with many patients not responding to -or not tolerating- traditional chemotherapeutic regimens. Recent studies have allowed us to utilize the genetic profile of tumors to better target therapy as is required, as well as innovate novel therapeutic interventions usable when more traditional options prove futile. Areas covered The development of novel interventions in bladder cancer, particularly immune checkpoint inhibitors, as well as using genetic markers to guide both traditional and novel therapeutic interventions. We also discuss the utility of these markers in diagnosing and prognosticating bladder cancer patients. Expert opinion Biomarker-guided therapy could revolutionize bladder cancer care in several ways: not only do novel therapeutic agents provide alternate treatment options for more difficult cases, but it can also increase the efficacy of more traditional treatment options. In addition, it may have a role in the early diagnosis and detection of bladder cancer, as well as predicting the course and prognosis of these patients. Unresolved challenges include how to best optimize therapy with novel agents as regarding duration and patient selection, as well as investigations as to whether using gene-guidance results in clinically improved patient outcomes.","PeriodicalId":12124,"journal":{"name":"Expert Review of Precision Medicine and Drug Development","volume":"5 1","pages":"347 - 361"},"PeriodicalIF":1.2,"publicationDate":"2020-08-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1080/23808993.2020.1801346","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"41961030","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2020-08-16DOI: 10.1080/23808993.2020.1799710
Arpan Patel, Seyed Mohammad Abedi, M. Lekkala, M. Baumgart
ABSTRACT Introduction Head and neck squamous cell carcinomas (HNSCC) are a heterogeneous group of malignancies which make up approximately 5% of all cancers worldwide. Molecular profiling of HNSCC tumors has identified a large number of genetic and epigenetic alterations that contribute to carcinogenesis, though genomic testing currently has a limited role in the treatment of HNSCC. Areas covered Molecular testing and use of targeted therapies are generally limited to the treatment of those with recurrent or metastatic disease or within clinical trials. Given the significant morbidity associated with the treatment of HNSCC, there is growing interest in identifying molecular alterations that drive carcinogenesis and have potential as clinical biomarkers and therapeutic targets. This article aims to review the current literature regarding clinical implications for common molecular alterations in HNSCC tumors and the developing role for genomic analysis in the treatment of this disease. Expert opinion Improved understanding of how molecular alterations contribute to carcinogenesis is essential to guide further research and to develop improved treatment strategies. While there are many promising treatment strategies under investigation, ongoing research is needed before genomic testing and targeted therapy will routinely be incorporated into clinical care for most patients.
{"title":"Genomic-based treatment of patients with head and neck cancer","authors":"Arpan Patel, Seyed Mohammad Abedi, M. Lekkala, M. Baumgart","doi":"10.1080/23808993.2020.1799710","DOIUrl":"https://doi.org/10.1080/23808993.2020.1799710","url":null,"abstract":"ABSTRACT Introduction Head and neck squamous cell carcinomas (HNSCC) are a heterogeneous group of malignancies which make up approximately 5% of all cancers worldwide. Molecular profiling of HNSCC tumors has identified a large number of genetic and epigenetic alterations that contribute to carcinogenesis, though genomic testing currently has a limited role in the treatment of HNSCC. Areas covered Molecular testing and use of targeted therapies are generally limited to the treatment of those with recurrent or metastatic disease or within clinical trials. Given the significant morbidity associated with the treatment of HNSCC, there is growing interest in identifying molecular alterations that drive carcinogenesis and have potential as clinical biomarkers and therapeutic targets. This article aims to review the current literature regarding clinical implications for common molecular alterations in HNSCC tumors and the developing role for genomic analysis in the treatment of this disease. Expert opinion Improved understanding of how molecular alterations contribute to carcinogenesis is essential to guide further research and to develop improved treatment strategies. While there are many promising treatment strategies under investigation, ongoing research is needed before genomic testing and targeted therapy will routinely be incorporated into clinical care for most patients.","PeriodicalId":12124,"journal":{"name":"Expert Review of Precision Medicine and Drug Development","volume":"5 1","pages":"401 - 408"},"PeriodicalIF":1.2,"publicationDate":"2020-08-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1080/23808993.2020.1799710","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"46972429","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2020-08-04DOI: 10.1080/23808993.2020.1804865
P. Cavalcante, R. Mantegazza, P. Bernasconi
ABSTRACT Introduction Inter-individual variation in drug efficacy and tolerability in myasthenia gravis (MG), an autoimmune disorder mostly treated by chronic immunosuppression, highlights the need of specific, safe and tailored precision medicine approaches. By targeting the disease-effector molecules, biological drugs are the most promising therapeutic agents to treat MG. A number of pharmacogenetic biomarkers associated with response to immunosuppressive drugs in MG have been identified, and microRNAs (miRNAs) are emerging as reliable markers for predicting or monitoring treatment response in individual patients. Areas covered This review provides an overview of pharmacogenetic and pharmaco-miR biomarkers associated with immunosuppressive treatment response in MG and other autoimmune diseases, pointing out the need of pharmacogenetic/-miR profiling for the recently developed biological drugs as an important step toward precision medicine. Expert opinion Extension of pharmacogenetic and pharmaco-miR data, and their entry into the clinical practice, hold the promise to greatly revolutionize MG therapy with clinically relevant drugs, including conventional and biological drugs, or their combination. High-throughput technologies, covering DNA and RNA, have the potential to disclose valuable pharmacogenomic/-miRNomic profiles able to guide the choice of the different drugs in individual patients, or biomarker-defined patients’ subgroups, thus significantly improving MG treatment in a cost/effective manner.
{"title":"Pharmacogenetic and pharmaco-miR biomarkers for tailoring and monitoring myasthenia gravis treatments","authors":"P. Cavalcante, R. Mantegazza, P. Bernasconi","doi":"10.1080/23808993.2020.1804865","DOIUrl":"https://doi.org/10.1080/23808993.2020.1804865","url":null,"abstract":"ABSTRACT Introduction Inter-individual variation in drug efficacy and tolerability in myasthenia gravis (MG), an autoimmune disorder mostly treated by chronic immunosuppression, highlights the need of specific, safe and tailored precision medicine approaches. By targeting the disease-effector molecules, biological drugs are the most promising therapeutic agents to treat MG. A number of pharmacogenetic biomarkers associated with response to immunosuppressive drugs in MG have been identified, and microRNAs (miRNAs) are emerging as reliable markers for predicting or monitoring treatment response in individual patients. Areas covered This review provides an overview of pharmacogenetic and pharmaco-miR biomarkers associated with immunosuppressive treatment response in MG and other autoimmune diseases, pointing out the need of pharmacogenetic/-miR profiling for the recently developed biological drugs as an important step toward precision medicine. Expert opinion Extension of pharmacogenetic and pharmaco-miR data, and their entry into the clinical practice, hold the promise to greatly revolutionize MG therapy with clinically relevant drugs, including conventional and biological drugs, or their combination. High-throughput technologies, covering DNA and RNA, have the potential to disclose valuable pharmacogenomic/-miRNomic profiles able to guide the choice of the different drugs in individual patients, or biomarker-defined patients’ subgroups, thus significantly improving MG treatment in a cost/effective manner.","PeriodicalId":12124,"journal":{"name":"Expert Review of Precision Medicine and Drug Development","volume":"5 1","pages":"317 - 329"},"PeriodicalIF":1.2,"publicationDate":"2020-08-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1080/23808993.2020.1804865","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"46297234","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2020-08-03DOI: 10.1080/23808993.2020.1804864
N. Tidbury, J. Preston, W. Ding, J. Rivera‐Caravaca, F. Marín, G. Lip
ABSTRACT Introduction Atrial fibrillation is the most common sustained cardiac rhythm disorder, which currently affects 1–2% of the global population. Furthermore, the incidence and prevalence of atrial fibrillation is rising. Biomarkers have the potential to improve clinical management of patients and therefore reduce the burden on health systems in the future. Areas covered A variety of pathways and mechanisms have been associated with atrial fibrillation. This paper provides an overview of a range of blood-based, imaging and genetic biomarkers that are associated with mechanisms and outcomes in atrial fibrillation and their potential use in a clinical setting. Expert commentary Atrial fibrillation is becoming increasingly prevalent. Current biomarkers associated with atrial fibrillation such as those involved in myocardial stress, inflammation, hemostasis and fibrosis do not currently provide much additional practical value beyond recommended scores based only on clinical risk factors.
{"title":"Utilizing biomarkers associated with cardiovascular events in atrial fibrillation: informing a precision medicine response","authors":"N. Tidbury, J. Preston, W. Ding, J. Rivera‐Caravaca, F. Marín, G. Lip","doi":"10.1080/23808993.2020.1804864","DOIUrl":"https://doi.org/10.1080/23808993.2020.1804864","url":null,"abstract":"ABSTRACT Introduction Atrial fibrillation is the most common sustained cardiac rhythm disorder, which currently affects 1–2% of the global population. Furthermore, the incidence and prevalence of atrial fibrillation is rising. Biomarkers have the potential to improve clinical management of patients and therefore reduce the burden on health systems in the future. Areas covered A variety of pathways and mechanisms have been associated with atrial fibrillation. This paper provides an overview of a range of blood-based, imaging and genetic biomarkers that are associated with mechanisms and outcomes in atrial fibrillation and their potential use in a clinical setting. Expert commentary Atrial fibrillation is becoming increasingly prevalent. Current biomarkers associated with atrial fibrillation such as those involved in myocardial stress, inflammation, hemostasis and fibrosis do not currently provide much additional practical value beyond recommended scores based only on clinical risk factors.","PeriodicalId":12124,"journal":{"name":"Expert Review of Precision Medicine and Drug Development","volume":"5 1","pages":"331 - 345"},"PeriodicalIF":1.2,"publicationDate":"2020-08-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1080/23808993.2020.1804864","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"48062056","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
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