Data from Functional Precision Medicine Provides Clinical Benefit in Advanced Aggressive Hematologic Cancers and Identifies Exceptional Responders Article Swipe
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 Greinix
,
Wolfgang Gstöttner
,
Marcus Hacker
,
Bernd Hartmann
,
Alexander W. Hauswirth
,
Tim Heinemann
,
Daniel Heintel
,
Mir Alireza Hoda
,
Georg Hopfinger
,
Ulrich Jaeger
,
Lukas Kazianka
,
Lukas Kenner
,
Barbara Kiesewetter
,
Nikolaus Krall
,
G. Krajnik
,
Stefan Kubicek
,
Trang T. 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 Zielinski
,
Niklas Zojer
,
Ingrid Simonitsch‐Klupp
,
Giulio Superti‐Furga
,
Berend Snijder
,
Philipp B. Staber
·
YOU?
·
· 2023
· Open Access
·
· DOI: https://doi.org/10.1158/2159-8290.c.6549464
YOU?
·
· 2023
· Open Access
·
· DOI: https://doi.org/10.1158/2159-8290.c.6549464
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
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- Type
- preprint
- Language
- en
- Landing Page
- https://doi.org/10.1158/2159-8290.c.6549464
- OA Status
- gold
- Related Works
- 10
- OpenAlex ID
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Raw OpenAlex JSON
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https://openalex.org/W4362558496Canonical identifier for this work in OpenAlex
- DOI
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https://doi.org/10.1158/2159-8290.c.6549464Digital Object Identifier
- Title
-
Data from Functional Precision Medicine Provides Clinical Benefit in Advanced Aggressive Hematologic Cancers and Identifies Exceptional RespondersWork title
- Type
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preprintOpenAlex work type
- Language
-
enPrimary language
- Publication year
-
2023Year of publication
- Publication date
-
2023-04-03Full publication date if available
- Authors
-
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 Greinix, Wolfgang Gstöttner, Marcus Hacker, Bernd Hartmann, Alexander W. Hauswirth, Tim Heinemann, Daniel Heintel, Mir Alireza Hoda, Georg Hopfinger, Ulrich Jaeger, Lukas Kazianka, Lukas Kenner, Barbara Kiesewetter, Nikolaus Krall, G. Krajnik, Stefan Kubicek, Trang T. 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 Zielinski, Niklas Zojer, Ingrid Simonitsch‐Klupp, Giulio Superti‐Furga, Berend Snijder, Philipp B. StaberList of authors in order
- Landing page
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https://doi.org/10.1158/2159-8290.c.6549464Publisher landing page
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YesWhether a free full text is available
- OA status
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goldOpen access status per OpenAlex
- OA URL
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https://doi.org/10.1158/2159-8290.c.6549464Direct OA link when available
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Medicine, Internal medicine, Oncology, Precision medicine, Clinical trial, Personalized medicine, Cancer, Drug, Bioinformatics, Pathology, Pharmacology, BiologyTop concepts (fields/topics) attached by OpenAlex
- Cited by
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0Total citation count in OpenAlex
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10Other works algorithmically related by OpenAlex
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