{"entity":{"id":"idea-bio2-functional-precision-rare","kind":"idea","name":"Test drugs on the patient's own cancer cells when there is no trial to join","aka":[],"tldr":"For rare cancers there is often no genetic clue and no trial to join. Testing drugs directly on the patient's fresh tumour cells has guided treatment with reported benefit in a randomised haematology study and in paediatric case series; turnaround and tissue quality are the barriers.","summary":"Functional precision medicine, ex vivo drug sensitivity testing on fresh patient material, guided treatment with reported benefit in a randomised haematology study and in paediatric and rare solid tumour case series. Turnaround, tissue quality and the absence of standardised reporting are the barriers. For rare cancers where genomics is uninformative, function is the only remaining evidence source.","asOf":"2026-09-08","links":[{"label":"Bottleneck evidence (Rare and paediatric cancers without markets): Gatta et al., Rare cancers are not so rare: the rare cancer burden in Europe (EJC 2011)","url":"https://doi.org/10.1016/j.ejca.2011.08.008"}],"tags":[],"related":[],"cancers":["sarcoma","cholangiocarcinoma"],"sections":[],"technologies":["functional-drug-testing","organoids","bh3-profiling","pdx-models"],"targets":[],"drugs":[],"companies":["curesponse","xilis","champions-oncology","sengine"],"institutions":[],"pathways":[],"terms":[],"trials":[],"people":[],"bottlenecks":["b-rare-cancers","b-preclinical-models","b-biomarker-validation"],"keyPapers":["paper-gatta-eur-j-cancer"],"journals":[],"dependsOn":[],"notes":[],"hypothesis":"Ex vivo sensitivity-guided therapy improves progression-free survival ratio compared with the patient's own prior line in rare cancers without actionable mutations, in at least a third of tested patients.","rationale":"Functional testing bypasses the need for a biomarker hypothesis, which is what makes it suited to rare and unclassifiable tumours. Haematology results show the approach can outperform physician choice when turnaround is fast enough.","test":"A multi-centre study in rare solid tumours reporting assay success rate, turnaround, proportion of patients whose treatment changes, and progression-free survival ratio against their own prior line.","maturity":"early-clinical","actor":"clinic","cost":"medium","horizonYears":5},"route":"/ideas/idea-bio2-functional-precision-rare/","neighbours":{"cancer":[{"id":"cholangiocarcinoma","kind":"cancer","name":"Biliary tract cancer (cholangiocarcinoma)","route":"/cancers/cholangiocarcinoma/"},{"id":"sarcoma","kind":"cancer","name":"Sarcomas (soft tissue, bone, GIST)","route":"/cancers/sarcoma/"}],"technology":[{"id":"bh3-profiling","kind":"technology","name":"BH3 profiling (functional apoptosis testing)","route":"/technologies/bh3-profiling/"},{"id":"functional-drug-testing","kind":"technology","name":"Functional (ex vivo) drug testing","route":"/technologies/functional-drug-testing/"},{"id":"organoids","kind":"technology","name":"Patient-derived organoids","route":"/technologies/organoids/"},{"id":"pdx-models","kind":"technology","name":"Patient-derived xenografts","route":"/technologies/pdx-models/"}],"company":[{"id":"champions-oncology","kind":"company","name":"Champions Oncology","route":"/companies/champions-oncology/"},{"id":"curesponse","kind":"company","name":"Curesponse","route":"/companies/curesponse/"},{"id":"sengine","kind":"company","name":"SEngine Precision Medicine","route":"/companies/sengine/"},{"id":"xilis","kind":"company","name":"Xilis","route":"/companies/xilis/"}],"bottleneck":[{"id":"b-biomarker-validation","kind":"bottleneck","name":"Biomarkers are not validated or standardised","route":"/bottlenecks/b-biomarker-validation/"},{"id":"b-preclinical-models","kind":"bottleneck","name":"Lab models that fail to predict what happens in patients","route":"/bottlenecks/b-preclinical-models/"},{"id":"b-rare-cancers","kind":"bottleneck","name":"Rare and paediatric cancers without markets","route":"/bottlenecks/b-rare-cancers/"}],"paper":[{"id":"paper-gatta-eur-j-cancer","kind":"paper","name":"Rare cancers are not so rare: the rare cancer burden in Europe","route":"/key-papers/paper-gatta-eur-j-cancer/"}]}}