Observational studyCancer discovery2022
Functional Precision Medicine Provides Clinical Benefit in Advanced Aggressive Hematologic Cancers and Identifies Exceptional Responders.
4 registry-linked trialsOpen access · bronzeAbstract readObservational Study
In one paragraph
Observational study in Cancer discovery, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to 4 registered trials, which are not on this map. Cited by 137 papers.
0numbers the graph read from it
0cells of the map it votes in
137citing papers in PubMed
20.6field-weighted citation impact, top 1% of its field
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Observational Analysis of the Association of Drug Activity Measured in Viable Tumour Tissues ex Vivo and Clinical Response in Ovarian Cancer: A Basic Research Study
Pharmacoscopy-guided Clinical Standard-of-care in Relapsed/Refractory Acute Myeloid Leukemia, a Randomized Phase-2 Clinical Trial
Ex Vivo Drug Response Evaluation for Next Generation Care of Brain Metastases
Methods of Identifying Effective Off-Guideline Treatments for Advanced Cancer Patients (CC N-of-1)
Who cites it
137 citing papers in PubMed, 200 citations in OpenAlex.
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- Genomic Subtype Influences BH3 Mimetic Drug Sensitivity and Synergy with Cytotoxic Chemotherapeutics in T-cell Acute Lymphoblastic Leukemia.Clinical cancer research : an official journal of the American Association for Cancer Research · 2026Article
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- Patient-derived organoids from metastatic colorectal cancer mirror tumor heterogeneity and predict patient survival and drug sensitivity.Cell reports. Medicine · 2026Article
- Functional Precision Oncology Approach Using Nanoliter Droplet Array for Drug Sensitivity Testing in Lung Cancer.Advanced healthcare materials · 2026Article
- Ex vivo drug screening and clustering of bladder cancers for pre-clinical treatment prediction.Communications medicine · 2026Article
- A multi-center study on the consistency of drug sensitivity testing in patients with acute myeloid leukemia.NPJ precision oncology · 2026Article
- Functional drug screening of tumor organoids on an active-matrix digital microfluidic chip for cancer precision medicine.Microsystems & nanoengineering · 2026Article
- Midostaurin response in AML is shaped by a progenitor-like cell state selectively targeted by SMAC mimetics.NPJ precision oncology · 2026Article
77 more citing papers are in PubMed but not listed here.
Corrections and comments
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Authors and funding
70 authors at 20 institutions in 7 countries.
Christoph Kornauth *Department of Medicine I, Division of Hematology and Hemostaseology, Medical University of Vienna, Vienna, Austria.
Tea Pemovska *Department of Medicine I, Division of Hematology and Hemostaseology, Medical University of Vienna, Vienna, Austria.ORCID 0000-0003-2951-4905
Gregory I Vladimer *CeMM Research Center for Molecular Medicine of the Austrian Academy of Sciences, Vienna, Austria.ORCID 0000-0003-4205-7585
Günther BayerDepartment of Pathology, Medical University of Vienna, Vienna, Austria.
Michael BergmannDepartment of Surgery, Medical University of Vienna, Vienna, Austria.ORCID 0000-0001-8529-1166
Sandra EderDepartment of Internal Medicine and Hematology/Oncology, Klinikum Klagenfurt, Klagenfurt, Austria.
Ruth EichnerCeMM Research Center for Molecular Medicine of the Austrian Academy of Sciences, Vienna, Austria.
Martin ErlAbteilung für Innere Medizin, Krankenhaus der Barmherzigen Brüder Salzburg, Salzburg, Austria.
Harald EsterbauerDepartment of Laboratory Medicine, Medical University of Vienna, Vienna, Austria.
Ruth ExnerDepartment of Surgery, Medical University of Vienna, Vienna, Austria.
Verena Felsleitner-HauerDepartment of Medicine and Oncology, LKH Wiener Neustadt, Wiener Neustadt, Austria.
Maurizio ForteDepartment of Medicine I, Division of Hematology and Hemostaseology, Medical University of Vienna, Vienna, Austria.
Alexander GaigerDepartment of Medicine I, Division of Hematology and Hemostaseology, Medical University of Vienna, Vienna, Austria.
Klaus GeisslerMedical School, Sigmund Freud University, Vienna, Austria.
Hildegard T GreinixDepartment of Internal Medicine, Division of Hematology, Medical University of Graz, Graz, Austria.
Wolfgang GstöttnerDepartment of Otorhinolaryngology, Head and Neck Surgery, Medical University of Vienna, Vienna, Austria.
Marcus HackerDivision of Nuclear Medicine, Department of Biomedical Imaging and Image-Guided Therapy, Medical University of Vienna, Vienna, Austria.
Bernd Lorenz HartmannLandeskrankenhaus Feldkirch, Abteilung für Innere Medizin, Feldkirch, Austria.ORCID 0000-0001-8831-3746
Alexander W HauswirthDepartment of Medicine I, Division of Hematology and Hemostaseology, Medical University of Vienna, Vienna, Austria.
Tim HeinemannDepartment of Biology, Institute of Molecular Systems Biology, ETH Zürich, Zürich, Switzerland.ORCID 0000-0002-4618-789X
Daniel HeintelDivision of Medicine I, Klinik Ottakring, Vienna, Austria.
Mir Alireza HodaDepartment of Thoracic Surgery, Medical University of Vienna, Vienna, Austria.
Georg HopfingerThird Medical Department, Centre for Oncology and Haematology, Klinik Favoriten, Vienna, Austria.
Ulrich JaegerDepartment of Medicine I, Division of Hematology and Hemostaseology, Medical University of Vienna, Vienna, Austria.
Lukas KaziankaDepartment of Medicine I, Division of Hematology and Hemostaseology, Medical University of Vienna, Vienna, Austria.
Lukas KennerDepartment of Pathology, Medical University of Vienna, Vienna, Austria.ORCID 0000-0003-2184-1338
Barbara KiesewetterDepartment of Medicine I, Division of Oncology, Medical University of Vienna, Vienna, Austria.ORCID 0000-0002-5490-2371
Nikolaus KrallCeMM Research Center for Molecular Medicine of the Austrian Academy of Sciences, Vienna, Austria.
Gerhard KrajnikDepartment of Medicine I, Universitätsklinikum St. Pölten, St. Pölten, Austria.
Stefan KubicekCeMM Research Center for Molecular Medicine of the Austrian Academy of Sciences, Vienna, Austria.ORCID 0000-0003-0855-8343
Trang LeDepartment of Medicine I, Division of Hematology and Hemostaseology, Medical University of Vienna, Vienna, Austria.
Simone LubowitzkiDepartment of Medicine I, Division of Hematology and Hemostaseology, Medical University of Vienna, Vienna, Austria.
Marius E MayerhoeferDepartment of Biomedical Imaging and Image-Guided Therapy, Medical University of Vienna, Vienna, Austria.
Elisabeth MenschelThird Medical Department, Hematology & Oncology, Hanusch Hospital, Vienna, Austria.
Olaf MerkelDepartment of Pathology, Medical University of Vienna, Vienna, Austria.
Katsuhiro MiuraDivision of Hematology and Rheumatology, Department of Medicine, Nihon University School of Medicine, Tokyo, Japan.
Leonhard MüllauerDepartment of Pathology, Medical University of Vienna, Vienna, Austria.ORCID 0000-0002-3399-078X
Peter NeumeisterDepartment of Internal Medicine, Division of Hematology, Medical University of Graz, Graz, Austria.ORCID 0000-0002-2761-5334
Thomas NoesslingerThird Medical Department, Hematology & Oncology, Hanusch Hospital, Vienna, Austria.
Katharina OckoPharmacy Department, Vienna General Hospital, Vienna, Austria.
Leopold ÖhlerInternal Medicine I, Department of Oncology, St. Josef Hospital, Vienna, Austria.
Michael PannyThird Medical Department, Hematology & Oncology, Hanusch Hospital, Vienna, Austria.
Alexander PichlerDepartment of Medicine I, Division of Hematology and Hemostaseology, Medical University of Vienna, Vienna, Austria.
Edit PorpaczyDepartment of Medicine I, Division of Hematology and Hemostaseology, Medical University of Vienna, Vienna, Austria.
Gerald W PragerComprehensive Cancer Center Vienna, Medical University of Vienna and Vienna General Hospital, Vienna, Austria.
Markus RadererComprehensive Cancer Center Vienna, Medical University of Vienna and Vienna General Hospital, Vienna, Austria.
Robin RistlSection for Medical Statistics, Center for Medical Statistics, Informatics and Intelligent Systems, Medical University of Vienna, Vienna, Austria.
Reinhard RuckserSecond Medical Department, Klinik Donaustadt, Vienna, Austria.
Julius SalamonDepartment of Medicine, Landesklinikum Waidhofen a.d. Ybbs, Waidhofen-Ybbs, Austria.
Ana-Iris SchieferDepartment of Pathology, Medical University of Vienna, Vienna, Austria.
Ann-Sofie SchmolkeDepartment of Medicine I, Division of Hematology and Hemostaseology, Medical University of Vienna, Vienna, Austria.
Ilse SchwarzingerDepartment of Laboratory Medicine, Medical University of Vienna, Vienna, Austria.
Edgar SelzerDepartment of Radiation Oncology, Medical University of Vienna, Vienna, Austria.
Christian SillaberDepartment of Medicine I, Division of Hematology and Hemostaseology, Medical University of Vienna, Vienna, Austria.
Cathrin SkrabsDepartment of Medicine I, Division of Hematology and Hemostaseology, Medical University of Vienna, Vienna, Austria.
Wolfgang R SperrDepartment of Medicine I, Division of Hematology and Hemostaseology, Medical University of Vienna, Vienna, Austria.
Ismet SrndicCeMM Research Center for Molecular Medicine of the Austrian Academy of Sciences, Vienna, Austria.
Renate ThalhammerDepartment of Laboratory Medicine, Medical University of Vienna, Vienna, Austria.
Peter ValentDepartment of Medicine I, Division of Hematology and Hemostaseology, Medical University of Vienna, Vienna, Austria.
Emiel van der KouweDepartment of Medicine I, Division of Hematology and Hemostaseology, Medical University of Vienna, Vienna, Austria.ORCID 0000-0001-7072-5152
Katrina VanuraDepartment of Medicine I, Division of Hematology and Hemostaseology, Medical University of Vienna, Vienna, Austria.ORCID 0000-0002-2217-4998
Stefan VogtDepartment of Medicine and Oncology, LKH Wiener Neustadt, Wiener Neustadt, Austria.
Cora WaldsteinDepartment of Radiation Oncology, Medical University of Vienna, Vienna, Austria.ORCID 0000-0001-8808-1850
Dominik WolfDepartment of Internal Medicine V, Department of Hematology and Oncology, Medical University of Innsbruck, Innsbruck, Austria.
Christoph C ZielinskiVienna Cancer Center, Medical University of Vienna, Vienna, Austria.
Niklas ZojerDivision of Medicine I, Klinik Ottakring, Vienna, Austria.
Ingrid Simonitsch-Klupp *Department of Pathology, Medical University of Vienna, Vienna, Austria.
Giulio Superti-Furga *CeMM Research Center for Molecular Medicine of the Austrian Academy of Sciences, Vienna, Austria.
Berend Snijder *Department of Biology, Institute of Molecular Systems Biology, ETH Zürich, Zürich, Switzerland.ORCID 0000-0003-3386-6583
Philipp B Staber *Department of Medicine I, Division of Hematology and Hemostaseology, Medical University of Vienna, Vienna, Austria. philipp.staber@meduniwien.ac.at.
Medical University of Vienna · ATVienna General Hospital · ATAustrian Academy of Sciences · ATHanusch Hospital · ATETH Zurich · CHFachhochschule Wiener Neustadt · ATLudwig Boltzmann Institute for Cancer Research · ATMedical University of Graz · ATBaidu (China) · CNComprehensive Cancer Center Vienna · ATDonauspital · ATInnsbruck Medical University · ATKlinikum Klagenfurt · ATLandeskrankenhaus Feldkirch · ATMemorial Sloan Kettering Cancer Center · USNihon University · JPOrdensklinikum Linz Barmherzige Schwestern · ATSigmund Freud Privatuniversität Wien · ATStatistics Austria · ATTirol Kliniken · AT
Funding
Austrian Science Fund FWF I 4154Austrian Science Fund FWF I 4156
Abstract
Personalized medicine aims to match the right drug with the right patient by using specific features of the individual patient's tumor. However, current strategies of personalized therapy matching provide treatment opportunities for less than 10% of patients with cancer. A promising method may be drug profiling of patient biopsy specimens with single-cell resolution to directly quantify drug effects. We prospectively tested an image-based single-cell functional precision medicine (scFPM) approach to guide treatments in 143 patients with advanced aggressive hematologic cancers. Fifty-six patients (39%) were treated according to scFPM results. At a median follow-up of 23.9 months, 30 patients (54%) demonstrated a clinical benefit of more than 1.3-fold enhanced progression-free survival compared with their previous therapy. Twelve patients (40% of responders) experienced exceptional responses lasting three times longer than expected for their respective disease. We conclude that therapy matching by scFPM is clinically feasible and effective in advanced aggressive hematologic cancers. SIGNIFICANCE: This is the first precision medicine trial using a functional assay to instruct n-of-one therapies in oncology. It illustrates that for patients lacking standard therapies, high-content assay-based scFPM can have a significant value in clinical therapy guidance based on functional dependencies of each patient's cancer.
Indexed as
AdultAgedAged, 80 and overAustriaCohort StudiesFemaleHematologic NeoplasmsHumansMaleMiddle AgedMolecular Targeted TherapyPrecision MedicineProgression-Free SurvivalYoung Adult
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Read underepoch 390
Registered trials
NCT06068738ClinicalTrials.gov
NCT06138990ClinicalTrials.gov
NCT06620380ClinicalTrials.gov
NCT07343024ClinicalTrials.gov
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.