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Biomarkers for cystic fibrosis drug development
Abstract Purpose To provide a review of the status of biomarkers in cystic fibrosis drug development, including regulatory definitions and considerations, a summary of biomarkers in current use with supportive data, current gaps, and future needs. Methods Biomarkers are considered across several are...
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Published in: | Journal of cystic fibrosis 2016-11, Vol.15 (6), p.714-723 |
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creator | Muhlebach, Marianne S Clancy, JP Heltshe, Sonya L Ziady, Assem Kelley, Tom Accurso, Frank Pilewski, Joseph Mayer-Hamblett, Nicole Joseloff, Elizabeth Sagel, Scott D |
description | Abstract Purpose To provide a review of the status of biomarkers in cystic fibrosis drug development, including regulatory definitions and considerations, a summary of biomarkers in current use with supportive data, current gaps, and future needs. Methods Biomarkers are considered across several areas of CF drug development, including cystic fibrosis transmembrane conductance regulator modulation, infection, and inflammation. Results Sweat chloride, nasal potential difference, and intestinal current measurements have been standardized and examined in the context of multicenter trials to quantify CFTR function. Detection and quantification of pathogenic bacteria in CF respiratory cultures (e.g.: Pseudomonas aeruginosa ) are commonly used in early phase antimicrobial clinical trials, and to monitor safety of therapeutic interventions. Sputum (e.g.: neutrophil elastase, myeloperoxidase, calprotectin) and blood biomarkers (e.g.: C reactive protein, calprotectin, serum amyloid A) have had variable success in detecting response to inflammatory treatments. Conclusions Biomarkers are used throughout the drug development process in CF, and many have been used in early phase clinical trials to provide proof of concept, detect drug bioactivity, and inform dosing for later-phase studies. Advances in the precision of current biomarkers, and the identification of new biomarkers with ‘omics-based technologies, are needed to accelerate CF drug development. |
doi_str_mv | 10.1016/j.jcf.2016.10.009 |
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Methods Biomarkers are considered across several areas of CF drug development, including cystic fibrosis transmembrane conductance regulator modulation, infection, and inflammation. Results Sweat chloride, nasal potential difference, and intestinal current measurements have been standardized and examined in the context of multicenter trials to quantify CFTR function. Detection and quantification of pathogenic bacteria in CF respiratory cultures (e.g.: Pseudomonas aeruginosa ) are commonly used in early phase antimicrobial clinical trials, and to monitor safety of therapeutic interventions. Sputum (e.g.: neutrophil elastase, myeloperoxidase, calprotectin) and blood biomarkers (e.g.: C reactive protein, calprotectin, serum amyloid A) have had variable success in detecting response to inflammatory treatments. Conclusions Biomarkers are used throughout the drug development process in CF, and many have been used in early phase clinical trials to provide proof of concept, detect drug bioactivity, and inform dosing for later-phase studies. Advances in the precision of current biomarkers, and the identification of new biomarkers with ‘omics-based technologies, are needed to accelerate CF drug development.</description><identifier>ISSN: 1569-1993</identifier><identifier>EISSN: 1873-5010</identifier><identifier>DOI: 10.1016/j.jcf.2016.10.009</identifier><identifier>PMID: 28215711</identifier><language>eng</language><publisher>Netherlands: Elsevier B.V</publisher><subject>Advanced technologies ; Biomarker ; Biomarkers - analysis ; Cystic Fibrosis - diagnosis ; Cystic Fibrosis - genetics ; Cystic Fibrosis - metabolism ; Cystic Fibrosis - microbiology ; Cystic Fibrosis Transmembrane Conductance Regulator - physiology ; Drug Discovery - methods ; Electrophysiology ; Humans ; Infection ; Inflammation ; Microbiome ; Pulmonary/Respiratory</subject><ispartof>Journal of cystic fibrosis, 2016-11, Vol.15 (6), p.714-723</ispartof><rights>European Cystic Fibrosis Society.</rights><rights>2016 European Cystic Fibrosis Society.</rights><rights>Copyright © 2016 European Cystic Fibrosis Society. Published by Elsevier B.V. All rights reserved.</rights><lds50>peer_reviewed</lds50><oa>free_for_read</oa><woscitedreferencessubscribed>false</woscitedreferencessubscribed><citedby>FETCH-LOGICAL-c506t-d1125541a139a4d3fdfd6929f4c6bbe88f438d019f957748645c3d3509efd7e23</citedby><cites>FETCH-LOGICAL-c506t-d1125541a139a4d3fdfd6929f4c6bbe88f438d019f957748645c3d3509efd7e23</cites></display><links><openurl>$$Topenurl_article</openurl><openurlfulltext>$$Topenurlfull_article</openurlfulltext><thumbnail>$$Tsyndetics_thumb_exl</thumbnail><link.rule.ids>230,315,786,790,891,27957,27958</link.rule.ids><backlink>$$Uhttps://www.ncbi.nlm.nih.gov/pubmed/28215711$$D View this record in MEDLINE/PubMed$$Hfree_for_read</backlink></links><search><creatorcontrib>Muhlebach, Marianne S</creatorcontrib><creatorcontrib>Clancy, JP</creatorcontrib><creatorcontrib>Heltshe, Sonya L</creatorcontrib><creatorcontrib>Ziady, Assem</creatorcontrib><creatorcontrib>Kelley, Tom</creatorcontrib><creatorcontrib>Accurso, Frank</creatorcontrib><creatorcontrib>Pilewski, Joseph</creatorcontrib><creatorcontrib>Mayer-Hamblett, Nicole</creatorcontrib><creatorcontrib>Joseloff, Elizabeth</creatorcontrib><creatorcontrib>Sagel, Scott D</creatorcontrib><title>Biomarkers for cystic fibrosis drug development</title><title>Journal of cystic fibrosis</title><addtitle>J Cyst Fibros</addtitle><description>Abstract Purpose To provide a review of the status of biomarkers in cystic fibrosis drug development, including regulatory definitions and considerations, a summary of biomarkers in current use with supportive data, current gaps, and future needs. Methods Biomarkers are considered across several areas of CF drug development, including cystic fibrosis transmembrane conductance regulator modulation, infection, and inflammation. Results Sweat chloride, nasal potential difference, and intestinal current measurements have been standardized and examined in the context of multicenter trials to quantify CFTR function. Detection and quantification of pathogenic bacteria in CF respiratory cultures (e.g.: Pseudomonas aeruginosa ) are commonly used in early phase antimicrobial clinical trials, and to monitor safety of therapeutic interventions. Sputum (e.g.: neutrophil elastase, myeloperoxidase, calprotectin) and blood biomarkers (e.g.: C reactive protein, calprotectin, serum amyloid A) have had variable success in detecting response to inflammatory treatments. Conclusions Biomarkers are used throughout the drug development process in CF, and many have been used in early phase clinical trials to provide proof of concept, detect drug bioactivity, and inform dosing for later-phase studies. Advances in the precision of current biomarkers, and the identification of new biomarkers with ‘omics-based technologies, are needed to accelerate CF drug development.</description><subject>Advanced technologies</subject><subject>Biomarker</subject><subject>Biomarkers - analysis</subject><subject>Cystic Fibrosis - diagnosis</subject><subject>Cystic Fibrosis - genetics</subject><subject>Cystic Fibrosis - metabolism</subject><subject>Cystic Fibrosis - microbiology</subject><subject>Cystic Fibrosis Transmembrane Conductance Regulator - physiology</subject><subject>Drug Discovery - methods</subject><subject>Electrophysiology</subject><subject>Humans</subject><subject>Infection</subject><subject>Inflammation</subject><subject>Microbiome</subject><subject>Pulmonary/Respiratory</subject><issn>1569-1993</issn><issn>1873-5010</issn><fulltext>true</fulltext><rsrctype>article</rsrctype><creationdate>2016</creationdate><recordtype>article</recordtype><recordid>eNp9kU1v1DAQhi1ERUvhB3BBOXLJdiaOnViVKpWKj0qVOABnK2uPi9NsvLWTlfbf19GWinLoyWP7nXdmnmHsA8IKAeVZv-qNW1U5zPcVgHrFTrBteCkA4XWOhVQlKsWP2duUegBsoGnfsOOqrVA0iCfs7LMPmy7eUUyFC7Ew-zR5Uzi_jiH5VNg43xaWdjSE7YbG6R07ct2Q6P3jecp-f_3y6-p7efPj2_XV5U1pBMiptIiVEDV2yFVXW-6ss1JVytVGrtfUtq7mrQVUTommqVtZC8MtF6DI2YYqfsouDr7beb0ha3Lp2A16G33udq9D5_Xzn9H_0bdhpwXPo0mRDT49GsRwP1Oa9MYnQ8PQjRTmpDMnyFVrgCzFg9TkmVMk91QGQS-gda8zaL2AXp4y6Jzz8d_-njL-ks2C84OAMqWdp6iT8TQasj6SmbQN_kX7i_-yzeBHb7rhjvaU-jDHMePXqFOlQf9cNr0sGiUHybnkDz7yoxw</recordid><startdate>20161101</startdate><enddate>20161101</enddate><creator>Muhlebach, Marianne S</creator><creator>Clancy, JP</creator><creator>Heltshe, Sonya L</creator><creator>Ziady, Assem</creator><creator>Kelley, Tom</creator><creator>Accurso, Frank</creator><creator>Pilewski, Joseph</creator><creator>Mayer-Hamblett, Nicole</creator><creator>Joseloff, Elizabeth</creator><creator>Sagel, Scott D</creator><general>Elsevier B.V</general><scope>6I.</scope><scope>AAFTH</scope><scope>CGR</scope><scope>CUY</scope><scope>CVF</scope><scope>ECM</scope><scope>EIF</scope><scope>NPM</scope><scope>AAYXX</scope><scope>CITATION</scope><scope>7X8</scope><scope>5PM</scope></search><sort><creationdate>20161101</creationdate><title>Biomarkers for cystic fibrosis drug development</title><author>Muhlebach, Marianne S ; Clancy, JP ; Heltshe, Sonya L ; Ziady, Assem ; Kelley, Tom ; Accurso, Frank ; Pilewski, Joseph ; Mayer-Hamblett, Nicole ; Joseloff, Elizabeth ; Sagel, Scott D</author></sort><facets><frbrtype>5</frbrtype><frbrgroupid>cdi_FETCH-LOGICAL-c506t-d1125541a139a4d3fdfd6929f4c6bbe88f438d019f957748645c3d3509efd7e23</frbrgroupid><rsrctype>articles</rsrctype><prefilter>articles</prefilter><language>eng</language><creationdate>2016</creationdate><topic>Advanced technologies</topic><topic>Biomarker</topic><topic>Biomarkers - analysis</topic><topic>Cystic Fibrosis - diagnosis</topic><topic>Cystic Fibrosis - genetics</topic><topic>Cystic Fibrosis - metabolism</topic><topic>Cystic Fibrosis - microbiology</topic><topic>Cystic Fibrosis Transmembrane Conductance Regulator - physiology</topic><topic>Drug Discovery - methods</topic><topic>Electrophysiology</topic><topic>Humans</topic><topic>Infection</topic><topic>Inflammation</topic><topic>Microbiome</topic><topic>Pulmonary/Respiratory</topic><toplevel>peer_reviewed</toplevel><toplevel>online_resources</toplevel><creatorcontrib>Muhlebach, Marianne S</creatorcontrib><creatorcontrib>Clancy, JP</creatorcontrib><creatorcontrib>Heltshe, Sonya L</creatorcontrib><creatorcontrib>Ziady, Assem</creatorcontrib><creatorcontrib>Kelley, Tom</creatorcontrib><creatorcontrib>Accurso, Frank</creatorcontrib><creatorcontrib>Pilewski, Joseph</creatorcontrib><creatorcontrib>Mayer-Hamblett, Nicole</creatorcontrib><creatorcontrib>Joseloff, Elizabeth</creatorcontrib><creatorcontrib>Sagel, Scott D</creatorcontrib><collection>ScienceDirect Open Access Titles</collection><collection>Elsevier:ScienceDirect:Open Access</collection><collection>Medline</collection><collection>MEDLINE</collection><collection>MEDLINE (Ovid)</collection><collection>MEDLINE</collection><collection>MEDLINE</collection><collection>PubMed</collection><collection>CrossRef</collection><collection>MEDLINE - Academic</collection><collection>PubMed Central (Full Participant titles)</collection><jtitle>Journal of cystic fibrosis</jtitle></facets><delivery><delcategory>Remote Search Resource</delcategory><fulltext>fulltext</fulltext></delivery><addata><au>Muhlebach, Marianne S</au><au>Clancy, JP</au><au>Heltshe, Sonya L</au><au>Ziady, Assem</au><au>Kelley, Tom</au><au>Accurso, Frank</au><au>Pilewski, Joseph</au><au>Mayer-Hamblett, Nicole</au><au>Joseloff, Elizabeth</au><au>Sagel, Scott D</au><format>journal</format><genre>article</genre><ristype>JOUR</ristype><atitle>Biomarkers for cystic fibrosis drug development</atitle><jtitle>Journal of cystic fibrosis</jtitle><addtitle>J Cyst Fibros</addtitle><date>2016-11-01</date><risdate>2016</risdate><volume>15</volume><issue>6</issue><spage>714</spage><epage>723</epage><pages>714-723</pages><issn>1569-1993</issn><eissn>1873-5010</eissn><notes>ObjectType-Article-2</notes><notes>SourceType-Scholarly Journals-1</notes><notes>ObjectType-Feature-3</notes><notes>content type line 23</notes><notes>ObjectType-Review-1</notes><notes>Contributed equally to the manuscript</notes><abstract>Abstract Purpose To provide a review of the status of biomarkers in cystic fibrosis drug development, including regulatory definitions and considerations, a summary of biomarkers in current use with supportive data, current gaps, and future needs. Methods Biomarkers are considered across several areas of CF drug development, including cystic fibrosis transmembrane conductance regulator modulation, infection, and inflammation. Results Sweat chloride, nasal potential difference, and intestinal current measurements have been standardized and examined in the context of multicenter trials to quantify CFTR function. Detection and quantification of pathogenic bacteria in CF respiratory cultures (e.g.: Pseudomonas aeruginosa ) are commonly used in early phase antimicrobial clinical trials, and to monitor safety of therapeutic interventions. Sputum (e.g.: neutrophil elastase, myeloperoxidase, calprotectin) and blood biomarkers (e.g.: C reactive protein, calprotectin, serum amyloid A) have had variable success in detecting response to inflammatory treatments. Conclusions Biomarkers are used throughout the drug development process in CF, and many have been used in early phase clinical trials to provide proof of concept, detect drug bioactivity, and inform dosing for later-phase studies. Advances in the precision of current biomarkers, and the identification of new biomarkers with ‘omics-based technologies, are needed to accelerate CF drug development.</abstract><cop>Netherlands</cop><pub>Elsevier B.V</pub><pmid>28215711</pmid><doi>10.1016/j.jcf.2016.10.009</doi><tpages>10</tpages><oa>free_for_read</oa></addata></record> |
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subjects | Advanced technologies Biomarker Biomarkers - analysis Cystic Fibrosis - diagnosis Cystic Fibrosis - genetics Cystic Fibrosis - metabolism Cystic Fibrosis - microbiology Cystic Fibrosis Transmembrane Conductance Regulator - physiology Drug Discovery - methods Electrophysiology Humans Infection Inflammation Microbiome Pulmonary/Respiratory |
title | Biomarkers for cystic fibrosis drug development |
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