{"id":"https://openalex.org/W4308398696","doi":"https://doi.org/10.1136/jitc-2022-sitc2022.0400","title":"400 PDGFR\u03b1-specific CAR T cell therapy of pediatric high-grade glioma","display_name":"400 PDGFR\u03b1-specific CAR T cell therapy of pediatric high-grade glioma","publication_year":2022,"publication_date":"2022-11-01","ids":{"openalex":"https://openalex.org/W4308398696","doi":"https://doi.org/10.1136/jitc-2022-sitc2022.0400"},"language":"en","primary_location":{"id":"doi:10.1136/jitc-2022-sitc2022.0400","doi":"https://doi.org/10.1136/jitc-2022-sitc2022.0400","is_oa":true,"landing_page_url":"https://doi.org/10.1136/jitc-2022-sitc2022.0400","pdf_url":"https://jitc.bmj.com/content/jitc/10/Suppl_2/A422.full.pdf","source":{"id":"https://openalex.org/S2754803477","display_name":"Journal for ImmunoTherapy of Cancer","issn_l":"2051-1426","issn":["2051-1426"],"is_oa":true,"is_in_doaj":true,"is_core":true,"listed_in":["cwts-core","doaj","dongbi-b","doyens","jufo-2","ki-jl-2","medline","norway-2","oarl","poland-140","russia-white-list-1","vabb-shw"],"host_organization":"https://openalex.org/P4310319945","host_organization_name":"BMJ","host_organization_lineage":["https://openalex.org/P4310319945"],"host_organization_lineage_names":["BMJ"],"type":"journal"},"license":null,"license_id":null,"version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"Regular and Young Investigator Award Abstracts","raw_type":"proceedings-article"},"volume":null,"type":"conference-abstract","indexed_in":["crossref","doaj"],"open_access":{"is_oa":true,"oa_status":"gold","oa_url":"https://jitc.bmj.com/content/jitc/10/Suppl_2/A422.full.pdf","any_repository_has_fulltext":false},"authorships":[{"author_position":"first","author":{"id":"https://openalex.org/A5069076195","display_name":"Kathryn Eckholdt","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I1343551460","display_name":"The University of Texas MD Anderson Cancer Center","ror":"https://ror.org/04twxam07","country_code":"US","type":"healthcare","lineage":["https://openalex.org/I1343551460"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Kathryn Eckholdt","raw_affiliation_strings":["MD Anderson Cancer Center, Houston, TX, USA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"MD Anderson Cancer Center, Houston, TX, USA","institution_ids":["https://openalex.org/I1343551460"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5026957968","display_name":"Sara Mazrimas","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I1343551460","display_name":"The University of Texas MD Anderson Cancer Center","ror":"https://ror.org/04twxam07","country_code":"US","type":"healthcare","lineage":["https://openalex.org/I1343551460"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Sara Mazrimas","raw_affiliation_strings":["MD Anderson Cancer Center, Houston, TX, USA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"MD Anderson Cancer Center, Houston, TX, USA","institution_ids":["https://openalex.org/I1343551460"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5010743791","display_name":"Paul J. Hauser","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I1343551460","display_name":"The University of Texas MD Anderson Cancer Center","ror":"https://ror.org/04twxam07","country_code":"US","type":"healthcare","lineage":["https://openalex.org/I1343551460"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Paul Hauser","raw_affiliation_strings":["MD Anderson Cancer Center, Houston, TX, USA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"MD Anderson Cancer Center, Houston, TX, USA","institution_ids":["https://openalex.org/I1343551460"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5031795097","display_name":"Wafik Tharwat Zaky","orcid":"https://orcid.org/0000-0002-7079-8105","observed_orcids":["https://orcid.org/0000-0002-7079-8105"]},"institutions":[{"id":"https://openalex.org/I1343551460","display_name":"The University of Texas MD Anderson Cancer Center","ror":"https://ror.org/04twxam07","country_code":"US","type":"healthcare","lineage":["https://openalex.org/I1343551460"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Wafik Zaky","raw_affiliation_strings":["MD Anderson Cancer Center, Houston, TX, USA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"MD Anderson Cancer Center, Houston, TX, USA","institution_ids":["https://openalex.org/I1343551460"]}]},{"author_position":"last","author":{"id":"https://openalex.org/A5114045801","display_name":"Amer M. Najjar","orcid":"https://orcid.org/0000-0002-6414-7234","observed_orcids":["https://orcid.org/0000-0002-6414-7234"]},"institutions":[{"id":"https://openalex.org/I1343551460","display_name":"The University of Texas MD Anderson Cancer Center","ror":"https://ror.org/04twxam07","country_code":"US","type":"healthcare","lineage":["https://openalex.org/I1343551460"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Amer Najjar","raw_affiliation_strings":["MD Anderson Cancer Center, Houston, TX, USA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"MD Anderson Cancer Center, Houston, TX, USA","institution_ids":["https://openalex.org/I1343551460"]}]}],"institutions":[],"countries_distinct_count":1,"institutions_distinct_count":1,"corresponding_author_ids":[],"corresponding_institution_ids":["https://openalex.org/I1343551460"],"apc_list":{"value":2490,"currency":"USD","value_usd":2490},"apc_paid":{"value":2490,"currency":"USD","value_usd":2490},"fwci":null,"has_fulltext":false,"cited_by_count":1,"citation_normalized_percentile":null,"cited_by_percentile_year":null,"biblio":{"volume":null,"issue":null,"first_page":null,"last_page":null},"is_retracted":false,"is_paratext":false,"is_xpac":false,"primary_topic":{"id":"https://openalex.org/T11491","display_name":"CAR-T cell therapy research","score":0.9945999979972839,"subfield":{"id":"https://openalex.org/subfields/2730","display_name":"Oncology"},"field":{"id":"https://openalex.org/fields/27","display_name":"Medicine"},"domain":{"id":"https://openalex.org/domains/4","display_name":"Health Sciences"}},"topics":[{"id":"https://openalex.org/T11491","display_name":"CAR-T cell therapy research","score":0.9945999979972839,"subfield":{"id":"https://openalex.org/subfields/2730","display_name":"Oncology"},"field":{"id":"https://openalex.org/fields/27","display_name":"Medicine"},"domain":{"id":"https://openalex.org/domains/4","display_name":"Health Sciences"}},{"id":"https://openalex.org/T10129","display_name":"Glioma Diagnosis and Treatment","score":0.003800000064074993,"subfield":{"id":"https://openalex.org/subfields/2716","display_name":"Genetics"},"field":{"id":"https://openalex.org/fields/27","display_name":"Medicine"},"domain":{"id":"https://openalex.org/domains/4","display_name":"Health Sciences"}},{"id":"https://openalex.org/T10580","display_name":"Immunotherapy and Immune Responses","score":0.0003000000142492354,"subfield":{"id":"https://openalex.org/subfields/2403","display_name":"Immunology"},"field":{"id":"https://openalex.org/fields/24","display_name":"Immunology and Microbiology"},"domain":{"id":"https://openalex.org/domains/1","display_name":"Life Sciences"}}],"keywords":[{"id":"https://openalex.org/keywords/pdgfra","display_name":"PDGFRA","score":0.9710000157356262},{"id":"https://openalex.org/keywords/chimeric-antigen-receptor-t-cells","display_name":"chimeric antigen receptor T cells","score":0.9160000085830688},{"id":"https://openalex.org/keywords/car-t-cell-therapy","display_name":"CAR T-cell therapy","score":0.8849999904632568},{"id":"https://openalex.org/keywords/pediatric-high-grade-glioma","display_name":"pediatric high-grade glioma","score":0.972000002861023},{"id":"https://openalex.org/keywords/single-chain-variable-fragment","display_name":"scFv","score":0.6880000233650208},{"id":"https://openalex.org/keywords/lentiviral-vectors","display_name":"lentiviral vectors","score":0.7480000257492065},{"id":"https://openalex.org/keywords/antitumor-efficacy","display_name":"antitumor efficacy","score":0.7089999914169312},{"id":"https://openalex.org/keywords/tumor-growth-inhibition","display_name":"tumor growth inhibition","score":0.5659999847412109},{"id":"https://openalex.org/keywords/bioluminescence-imaging","display_name":"bioluminescence imaging","score":0.7210000157356262},{"id":"https://openalex.org/keywords/receptor-tyrosine-kinases","display_name":"receptor tyrosine kinase","score":0.6430000066757202}],"concepts":[{"id":"https://openalex.org/C3875195","wikidata":"https://www.wikidata.org/wiki/Q5099363","display_name":"Chimeric antigen receptor","level":4,"score":0.7622277736663818},{"id":"https://openalex.org/C502942594","wikidata":"https://www.wikidata.org/wiki/Q3421914","display_name":"Cancer research","level":1,"score":0.5938749313354492},{"id":"https://openalex.org/C2778227246","wikidata":"https://www.wikidata.org/wiki/Q1365309","display_name":"Glioma","level":2,"score":0.5185430645942688},{"id":"https://openalex.org/C154317977","wikidata":"https://www.wikidata.org/wiki/Q376266","display_name":"Cytotoxic T cell","level":3,"score":0.5068095326423645},{"id":"https://openalex.org/C180361614","wikidata":"https://www.wikidata.org/wiki/Q7202242","display_name":"Platelet-derived growth factor receptor","level":4,"score":0.4378972053527832},{"id":"https://openalex.org/C148125776","wikidata":"https://www.wikidata.org/wiki/Q1622348","display_name":"CD28","level":4,"score":0.42240551114082336},{"id":"https://openalex.org/C86803240","wikidata":"https://www.wikidata.org/wiki/Q420","display_name":"Biology","level":0,"score":0.418254554271698},{"id":"https://openalex.org/C153911025","wikidata":"https://www.wikidata.org/wiki/Q7202","display_name":"Molecular biology","level":1,"score":0.3809899091720581},{"id":"https://openalex.org/C147483822","wikidata":"https://www.wikidata.org/wiki/Q103537","display_name":"Antigen","level":2,"score":0.3420463800430298},{"id":"https://openalex.org/C167672396","wikidata":"https://www.wikidata.org/wiki/Q417800","display_name":"CD8","level":3,"score":0.2878057360649109},{"id":"https://openalex.org/C170493617","wikidata":"https://www.wikidata.org/wiki/Q208467","display_name":"Receptor","level":2,"score":0.28536203503608704},{"id":"https://openalex.org/C2776090121","wikidata":"https://www.wikidata.org/wiki/Q193529","display_name":"T cell","level":3,"score":0.276503324508667},{"id":"https://openalex.org/C203014093","wikidata":"https://www.wikidata.org/wiki/Q101929","display_name":"Immunology","level":1,"score":0.1992005705833435},{"id":"https://openalex.org/C202751555","wikidata":"https://www.wikidata.org/wiki/Q221681","display_name":"In vitro","level":2,"score":0.1903308928012848},{"id":"https://openalex.org/C2775960820","wikidata":"https://www.wikidata.org/wiki/Q408378","display_name":"Growth factor","level":3,"score":0.1557583510875702},{"id":"https://openalex.org/C8891405","wikidata":"https://www.wikidata.org/wiki/Q1059","display_name":"Immune system","level":2,"score":0.10991927981376648},{"id":"https://openalex.org/C55493867","wikidata":"https://www.wikidata.org/wiki/Q7094","display_name":"Biochemistry","level":1,"score":0.0}],"mesh":[],"locations_count":1,"locations":[{"id":"doi:10.1136/jitc-2022-sitc2022.0400","doi":"https://doi.org/10.1136/jitc-2022-sitc2022.0400","is_oa":true,"landing_page_url":"https://doi.org/10.1136/jitc-2022-sitc2022.0400","pdf_url":"https://jitc.bmj.com/content/jitc/10/Suppl_2/A422.full.pdf","source":{"id":"https://openalex.org/S2754803477","display_name":"Journal for ImmunoTherapy of Cancer","issn_l":"2051-1426","issn":["2051-1426"],"is_oa":true,"is_in_doaj":true,"is_core":true,"listed_in":["cwts-core","doaj","dongbi-b","doyens","jufo-2","ki-jl-2","medline","norway-2","oarl","poland-140","russia-white-list-1","vabb-shw"],"host_organization":"https://openalex.org/P4310319945","host_organization_name":"BMJ","host_organization_lineage":["https://openalex.org/P4310319945"],"host_organization_lineage_names":["BMJ"],"type":"journal"},"license":null,"license_id":null,"version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"Regular and Young Investigator Award Abstracts","raw_type":"proceedings-article"}],"best_oa_location":{"id":"doi:10.1136/jitc-2022-sitc2022.0400","doi":"https://doi.org/10.1136/jitc-2022-sitc2022.0400","is_oa":true,"landing_page_url":"https://doi.org/10.1136/jitc-2022-sitc2022.0400","pdf_url":"https://jitc.bmj.com/content/jitc/10/Suppl_2/A422.full.pdf","source":{"id":"https://openalex.org/S2754803477","display_name":"Journal for ImmunoTherapy of Cancer","issn_l":"2051-1426","issn":["2051-1426"],"is_oa":true,"is_in_doaj":true,"is_core":true,"listed_in":["cwts-core","doaj","dongbi-b","doyens","jufo-2","ki-jl-2","medline","norway-2","oarl","poland-140","russia-white-list-1","vabb-shw"],"host_organization":"https://openalex.org/P4310319945","host_organization_name":"BMJ","host_organization_lineage":["https://openalex.org/P4310319945"],"host_organization_lineage_names":["BMJ"],"type":"journal"},"license":null,"license_id":null,"version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"Regular and Young Investigator Award Abstracts","raw_type":"proceedings-article"},"sustainable_development_goals":[{"id":"https://metadata.un.org/sdg/3","score":1.0,"display_name":"Good health and well-being"}],"study_designs":[],"awards":[],"funders":[],"has_content":{"pdf":true,"grobid_xml":true},"content_urls":{"pdf":"https://content.openalex.org/works/W4308398696.pdf","grobid_xml":"https://content.openalex.org/works/W4308398696.grobid-xml"},"referenced_works_count":0,"referenced_works":[],"related_works":["https://openalex.org/W4416085462","https://openalex.org/W4376280107","https://openalex.org/W4213448910","https://openalex.org/W3110911453","https://openalex.org/W2609041270","https://openalex.org/W4388783629","https://openalex.org/W7205249007","https://openalex.org/W4409629841","https://openalex.org/W3212595109","https://openalex.org/W7166725445"],"abstract_inverted_index":{"Background":[0],"Platelet-derived":[1],"growth":[2,172,309],"factor":[3],"receptor":[4,11,39,46],"alpha":[5],"(PDGFR\u03b1)":[6],"is":[7,20,502],"a":[8,14,40,63,69,79,88,130,223,261,329],"tyrosine":[9],"kinase":[10],"that":[12,317],"plays":[13],"pivotal":[15],"role":[16],"in":[17,120,129,241,279,306,328,347,369,404,445,459],"tumorigenesis":[18],"and":[19,25,73,100,140,170,199,210,234,291,354,376,421,444,451,462,468,483],"associated":[21],"with":[22,87,98,160,252,277,442,447],"tumor":[23,171,243,335],"proliferation":[24],"progression.":[26,336],"Pediatric":[27],"gliomas":[28],"are":[29],"known":[30],"to":[31,68,77,247,350,387,415,428,479,490],"express":[32],"high":[33],"levels":[34],"of":[35,62,113,152,179,208,226,264,295,310,334,339,372,391,409,471,496],"PDGFR\u03b1,":[36],"designating":[37],"the":[38,148,177,218,248,278,293,300,308,362,370,378,389,417,499],"viable":[41],"target":[42,229],"for":[43,365,380,402,423,466,498],"chimeric":[44],"antigen":[45,102],"(CAR)":[47],"T":[48,94,115,162,188,215,237,258,270,297,320,342,367],"cell":[49,238,368],"therapy.":[50],"Methods":[51],"Lentiviral":[52],"vectors":[53],"encoding":[54],"PDGFR\u03b1-specific":[55,92,206],"CAR-coding":[56],"sequences":[57],"were":[58,96,145,437,457,463,477,486],"constructed":[59],"by":[60,182],"fusion":[61],"single":[64],"chain":[65],"Fv":[66],"(scFv)":[67],"human":[70],"CD8a":[71],"stalk":[72],"transmembrane":[74],"domain":[75,85,221],"linked":[76],"either":[78],"CD28":[80,219,301],"or":[81,302,473],"CD137":[82,303],"intracellular":[83],"signaling":[84,220],"fused":[86],"terminal":[89],"CD3\u03b6":[90],"domain.":[91],"CAR":[93,187,193,236,269,319,341],"cells":[95,105,116,136,163,189,216,259,271,298,321,343],"expanded":[97],"IL-2":[99],"artificial":[101],"presenting":[103],"K562":[104],"(aAPC)":[106],"expressing":[107,137,217,299],"truncated":[108],"PDGFR\u03b1.":[109],"The":[110,124,493],"cytotoxic":[111],"function":[112],"PDGFR\u03b1-CAR":[114,161,214,296,366],"was":[117,127,164,173],"assessed":[118],"using":[119,155],"vitro":[121,202,280],"killing":[122],"assays.":[123],"therapeutic":[125,363],"efficacy":[126,294],"evaluated":[128,290],"mouse":[131],"KNS42":[132,135,209,230],"glioma":[133,212,312,375],"model.":[134],"firefly":[138],"luciferase":[139],"green":[141],"fluorescent":[142],"protein":[143],"(ffLuc-GFP)":[144],"engrafted":[146],"into":[147],"right":[149],"parietal":[150],"lobe":[151],"NSG":[153],"mice":[154,250,485],"screw-guided":[156],"injections.":[157],"Intratumoral":[158],"treatment":[159,371],"carried":[165],"out":[166],"one":[167],"week":[168],"later,":[169],"monitored":[174,464],"weekly":[175],"over":[176,191],"course":[178],"nine":[180],"weeks":[181,196],"bioluminescence":[183],"imaging.":[184],"Results":[185],"All":[186,434,475],"demonstrated":[190,205,316],"75%":[192],"expression":[194],"two":[195],"following":[197],"transduction":[198],"expansion.":[200],"In":[201,286],"cytotoxicity":[203,227,281],"assays":[204],"lysis":[207],"PDGFR\u03b1-U87":[211],"cells.":[213,231,313],"exhibited":[222,260,322],"higher":[224,262],"level":[225,263],"against":[228],"Both":[232],"PDGFR\u03b1-CD28":[233,318,340],"-CD137":[235],"products":[239],"resulted":[240],"decreased":[242],"progression":[244],"when":[245],"compared":[246,292],"control":[249],"injected":[251],"saline":[253],"(figure":[254,283],"1).":[255,284],"However,":[256],"PDGFR\u03b1-CD28-CAR":[257],"antitumor":[265],"activity":[266],"than":[267],"PDGFR\u03b1-CD137":[268],"(97%":[272],"vs":[273],"85%":[274],"inhibition),":[275],"consistent":[276],"observations":[282],"Conclusions":[285],"this":[287],"study,":[288],"we":[289],"costimulatory":[304],"domains":[305],"controlling":[307],"pediatric":[311,373],"We":[314,385,412],"have":[315],"more":[323,330],"immediate":[324],"anti-tumor":[325],"response":[326],"resulting":[327],"significant":[331],"long-term":[332,356],"reduction":[333],"Repeated":[337],"administration":[338],"may":[344],"be":[345],"warranted":[346],"future":[348,381],"studies":[349,360,436],"further":[351],"improve":[352],"outcomes":[353],"preserve":[355],"responses.":[357],"Our":[358],"preclinical":[359],"demonstrate":[361],"potential":[364],"high-grade":[374],"set":[377],"stage":[379],"clinical":[382],"translation.":[383],"Acknowledgements":[384],"wish":[386,414],"acknowledge":[388,416],"staff":[390],"Small":[392],"Animal":[393,449],"Imaging":[394],"Facility":[395],"(SAIF)":[396],"at":[397,439],"MD":[398,440],"Anderson":[399,441],"Cancer":[400],"Center":[401],"assistance":[403],"conducting":[405],"all":[406,425],"imaging-related":[407],"aspects":[408],"our":[410,429],"studies.":[411,431],"also":[413],"Department":[418],"Veterinary":[419],"Medicine":[420],"Surgery":[422],"facilitating":[424],"procedures":[426],"related":[427],"animal":[430,435,481,500],"Ethics":[432],"Approval":[433],"conducted":[438],"approval":[443,497],"accordance":[446],"Institutional":[448],"Care":[450],"Use":[452],"Committee":[453],"(IACUC)":[454],"guidelines.":[455,492],"Mice":[456],"housed":[458],"pathogen-free":[460],"conditions":[461],"daily":[465],"welfare":[467],"any":[469],"signs":[470],"discomfort":[472],"pain.":[474],"efforts":[476],"made":[478],"minimize":[480],"suffering,":[482],"moribund":[484],"humanely":[487],"euthanized":[488],"according":[489],"IACUC":[491],"ID":[494],"number":[495],"study":[501],"00001237-RN02.":[503]},"counts_by_year":[{"year":2024,"cited_by_count":1}],"updated_date":"2026-11-02T09:45:03.842371","created_date":"2022-11-11T00:00:00"}