Functional Precision Medicine Provides Clinical Benefit in Advanced Aggressive Hematologic Cancers and Identifies Exceptional Responders

Christoph Kornauth, Tea Pemovska, Gregory I. Vladimer, Günther Bayer, Michael Bergmann, Sandra Eder, Ruth Eichner, Martin Erl, Harald Esterbauer, Ruth Exner, Verena Felsleitner-Hauer, Maurizio Forte, Alexander Gaiger, Klaus Geissler, Hildegard T. Greinix, Wolfgang Gstöttner, Marcus Hacker, Bernd Lorenz Hartmann, Alexander W. Hauswirth, Tim HeinemannDaniel Heintel, Mir Alireza Hoda, Georg Hopfinger, Ulrich Jaeger, Lukas Kazianka, Lukas Kenner, Barbara Kiesewetter, Nikolaus Krall, Gerhard Krajnik, Stefan Kubicek, Trang Le, Simone Lubowitzki, Marius E. Mayerhoefer, Elisabeth Menschel, Olaf Merkel, Katsuhiro Miura, Leonhard Müllauer, Peter Neumeister, Thomas Noesslinger, Katharina Ocko, Leopold Öhler, Michael Panny, Alexander Pichler, Edit Porpaczy, Gerald W. Prager, Markus Raderer, Robin Ristl, Reinhard Ruckser, Julius Salamon, Ana-Iris Schiefer, Ann-Sofie Schmolke, Ilse Schwarzinger, Edgar Selzer, Christian Sillaber, Cathrin Skrabs, Wolfgang R. Sperr, Ismet Srndic, Renate Thalhammer, Peter Valent, Emiel van der Kouwe, Katrina Vanura, Stefan Vogt, Cora Waldstein, Dominik Wolf, Christoph C. Zielinski, Niklas Zojer, Ingrid Simonitsch-Klupp, Giulio Superti-Furga, Berend Snijder, Philipp B. Staber

Research output: Journal article (peer-reviewed)Journal article

54 Citations (Scopus)


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 assaybased scFPM can have a significant value in clinical therapy guidance based on functional dependencies of each patient’s cancer.

Original languageEnglish
Pages (from-to)372-387
Number of pages16
JournalCancer Discovery
Issue number2
Publication statusPublished - 01 Feb 2022


  • Adult
  • Aged
  • Aged, 80 and over
  • Austria
  • Cohort Studies
  • Female
  • Hematologic Neoplasms/drug therapy
  • Humans
  • Male
  • Middle Aged
  • Molecular Targeted Therapy
  • Precision Medicine
  • Progression-Free Survival
  • Young Adult

ASJC Scopus subject areas

  • Oncology


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