Functional Precision Medicine Provides Clinical Benefit in Advanced Aggressive Hematologic Cancers and Identifies Exceptional Responders
| Autor: | Leopold Öhler, Philipp B. Staber, Julius Salamon, Edit Porpaczy, Gerhard Krajnik, Gregory I. Vladimer, Harald Esterbauer, Ulrich Jaeger, Christian Sillaber, Gerald W. Prager, Katrina Vanura, Stefan Kubicek, Peter Valent, Michael Panny, Alexander W. Hauswirth, Edgar Selzer, Verena Felsleitner-Hauer, Emiel van der Kouwe, Nikolaus Krall, Marius E. Mayerhoefer, Bernd Lorenz Hartmann, Alexander Gaiger, Sandra Eder, Klaus Geissler, Mir Alireza Hoda, Dominik Wolf, Simone Lubowitzki, Peter Neumeister, Barbara Kiesewetter, Ruth Exner, Giulio Superti-Furga, Thomas Noesslinger, Elisabeth Menschel, Katsuhiro Miura, Wolfgang Gstöttner, Reinhard Ruckser, Ingrid Simonitsch-Klupp, Hildegard Greinix, Ana-Iris Schiefer, Ann-Sofie Schmolke, Cora Waldstein, Georg Hopfinger, Christoph C. Zielinski, Wolfgang R. Sperr, Marcus Hacker, Trang Le, Robin Ristl, Christoph Kornauth, Lukas Kazianka, Tea Pemovska, Günther Bayer, Cathrin Skrabs, Markus Raderer, Ruth Eichner, Alexander Pichler, Lukas Kenner, Maurizio Forte, Renate Thalhammer, Stefan Vogt, Leonhard Müllauer, Katharina Ocko, Niklas Zojer, Berend Snijder, Ilse Schwarzinger, Olaf Merkel, Martin Erl, Daniel Heintel, Michael Bergmann, Tim Heinemann, Ismet Srndic |
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| Rok vydání: | 2021 |
| Předmět: |
Oncology
Drug 0303 health sciences medicine.medical_specialty business.industry media_common.quotation_subject Cancer Disease Drug profiling Precision medicine medicine.disease 3. Good health 03 medical and health sciences 0302 clinical medicine 030220 oncology & carcinogenesis Internal medicine Medicine Personalized medicine Personalized therapy business 030304 developmental biology media_common |
| Zdroj: | Cancer Discovery, 12 (2) |
| ISSN: | 2159-8290 2159-8274 |
| DOI: | 10.1158/2159-8290.cd-21-0538 |
| Popis: | 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. See related commentary by Letai, p. 290. This article is highlighted in the In This Issue feature, p. 275 |
| Databáze: | OpenAIRE |
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