{"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":null,"pages":null},"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}
Kaining Zhi, Asit Kumar, B. Raji, H. Kochat, Santosh Kumar
{"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":null,"pages":null},"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}
{"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":null,"pages":null},"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}
A. Sayed, M. Munir, Noor Eweis, Doaa Wael, O. Shazly, A. Awad, Marihan A. Elbadawy, S. Eissa
{"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":null,"pages":null},"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}
Arpan Patel, Seyed Mohammad Abedi, M. Lekkala, M. Baumgart
{"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":null,"pages":null},"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}
{"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":null,"pages":null},"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}
N. Tidbury, J. Preston, W. Ding, J. Rivera‐Caravaca, F. Marín, G. Lip
{"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":null,"pages":null},"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}
{"title":"Noninvasive biomarkers to guide intervention: toward personalized patient management in prostate cancer","authors":"M. Frantzi, E. Gómez-Gómez, H. Mischak","doi":"10.1080/23808993.2020.1804866","DOIUrl":"https://doi.org/10.1080/23808993.2020.1804866","url":null,"abstract":"ABSTRACT Introduction Prostate cancer (PCa) is one of the most frequently diagnosed malignancies worldwide and is associated with high mortality. Broad screening through prostate-specific antigen analysis, along with an aging and growing population has resulted in a vast increase in PCa incidence. As not all PCa forms are life threatening, personalized management is of paramount importance to preserve survival and quality of life for the diagnosed patients. Owing to the complexity of PCa, noninvasive biomarkers for diagnosis, stratification and monitoring, are essential to tailor intervention among patients with different disease manifestations. Areas covered In this article, we aim to provide a critical assessment of the reported noninvasive biomarkers for PCa and their applicability according to the targeted clinical context. For this purpose, a systematic review of the literature published within the last five years was performed, focusing on noninvasive biomarkers to guide initial and repeated biopsies, stratify for active surveillance, monitor biochemical recurrence and metastasis, and adjust treatment for metastatic castration resistant PCa. Expert’s opinion Evidence from clinical trials on novel drugs and latest technological advancements, indicate several clinical applications for biomarkers to tailor intervention throughout PCa progression, toward a more personalized medicine approach in PCa clinical management.","PeriodicalId":12124,"journal":{"name":"Expert Review of Precision Medicine and Drug Development","volume":null,"pages":null},"PeriodicalIF":1.2,"publicationDate":"2020-08-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1080/23808993.2020.1804866","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"43225406","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}
{"title":"A wide perspective of targeted therapies for precision medicine in autoimmune diseases","authors":"I. Miyagawa, S. Kubo, Yoshiya Tanaka","doi":"10.1080/23808993.2020.1804867","DOIUrl":"https://doi.org/10.1080/23808993.2020.1804867","url":null,"abstract":"ABSTRACT Introduction The strategy of precision medicine is achieved by the stratification of patients and targeted therapy. It has been shown that the pathological conditions of patients who are classified or diagnosed with a single disease are actually diverse in almost all autoimmune diseases. Therefore, the practice of precision medicine is important for the treatment of patients with autoimmune diseases. Areas covered At present, precision medicine has not been achieved in any autoimmune disease. This article reviewed the attempted use of precision medicine for the treatment of systemic lupus erythematosus and psoriatic arthritis. Expert opinion Although multiple molecular-targeted therapies are available for the treatment of rheumatoid arthritis, the response to each treatment strategy often differs markedly among patients, and it is unclear how they are differentially selected for each patient. The establishment of precision medicine is especially important in heterogenous autoimmune diseases (e.g., systemic lupus erythematosus, psoriatic arthritis). Various symptoms must be improved at the same time, but the treatment options are limited. Therefore, the selection of the optimal treatment for individual patients is complex. Although some predictors of the treatment response are reported, precision medicine has not been sufficiently well evaluated in real clinical practice, especially for autoimmune diseases.","PeriodicalId":12124,"journal":{"name":"Expert Review of Precision Medicine and Drug Development","volume":null,"pages":null},"PeriodicalIF":1.2,"publicationDate":"2020-07-31","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1080/23808993.2020.1804867","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"46728653","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}
{"title":"Ivosidenib for the treatment of relapsed or refractory acute myeloid leukemia with an IDH1 mutation","authors":"F. Pasquier, C. Chahine, C. Marzac, S. de Botton","doi":"10.1080/23808993.2020.1792286","DOIUrl":"https://doi.org/10.1080/23808993.2020.1792286","url":null,"abstract":"ABSTRACT Introduction Management of acute myeloid leukemia (AML) remains challenging, especially for relapsed or refractory (R/R) AML patients who display poor prognosis with conventional therapies. This underlined the need for new treatments in this population. Areas covered This review will focus on ivosidenib, an oral inhibitor of mutated isocitrate dehydrogenase 1 (IDH1) enzyme. Mechanisms of IDH1 mutations and their inhibition by ivosidenib will be cover, as well as clinical efficacy and safety of ivosidenib in R/R AML. Ivosidenib has been approved by the FDA for R/R AML patients with IDH1 mutation in July 2018 and for unfit AML patients with IDH1 mutation as first line treatment in May 2019. Expert commentary Ivosidenib induces impressive response rates in R/R AML, a population of bad prognosis. Nevertheless, primary and acquired resistances to ivosidenib have been recently described, underlining importance of the ongoing clinical trials with ivosidenib in combination with standard chemotherapy, hypomethylating agents or other targeted therapies.","PeriodicalId":12124,"journal":{"name":"Expert Review of Precision Medicine and Drug Development","volume":null,"pages":null},"PeriodicalIF":1.2,"publicationDate":"2020-07-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1080/23808993.2020.1792286","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"43294350","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}