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Neoadjuvant clinical trials provide a window of opportunity for cancer drug discovery

Nature Medicine volume 28, pages 626–629 (2022)Cite this article

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An Author Correction to this article was published on 18 July 2022

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Window-of-opportunity trials, during which patients receive short-duration pre-surgical therapies, provide a platform for understanding the therapies’ mechanisms of action, but will require a paradigm shift in trial design, specimen collection and analysis.

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Fig. 1: A paradigm shift in clinical trials.
Fig. 2: Iterative-design trials enable and inform rapid informative trials.
Fig. 3: Communication and collaboration across medical silos.

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Acknowledgements

The TARGET and INTERACT teams comprise hundreds of physicians, researchers, coordinators and administrators, and the contributions of all these members is absolutely integral to the success of endeavors discussed here. We would like to acknowledge all these collaborators, as well as the support of S. Burakoff, R. Parsons, K. Goodman, F. R. Hirsch and D. Charney.

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Authors and Affiliations

  1. The neoAdjuvant Research Group to Evaluate Therapeutics (TARGET), Icahn School of Medicine at Mount Sinai, New York, NY, USA

    Thomas U. Marron, Matthew D. Galsky, Bachir Taouli, Maria Isabel Fiel, Stephen Ward, Edward Kim, David Yankelevitz, Deborah Doroshow, Benjamin J. Golas, Daniel Labow, John Sfakianos, Sujit S. Nair, Dimple Chakravarty, Michael Buckstein, Xiaoyu Song, Effi Kenigsberg, Sacha Gnjatic, Brian D. Brown, Joseph Sparano, Ashutosh Tewari, Myron Schwartz, Nina Bhardwaj & Miriam Merad

  2. Immunotherapy and Novel TargEt Research Across Clinical Teams (INTERACT), Icahn School of Medicine at Mount Sinai, New York, NY, USA

    Thomas U. Marron, Emma Guttman-Yassky, Benjamin Ungar, Saurabh Mehandru, Nina Bhardwaj & Miriam Merad

  3. Early Phase Trials Unit, Tisch Cancer Institute, Icahn School of Medicine at Mount Sinai, New York, NY, USA

    Thomas U. Marron, Matthew D. Galsky, Deborah Doroshow, Nina Bhardwaj & Miriam Merad

  4. Tisch Cancer Institute, Icahn School of Medicine at Mount Sinai, New York, NY, USA

    Thomas U. Marron, Matthew D. Galsky, Bachir Taouli, Maria Isabel Fiel, Stephen Ward, Edward Kim, David Yankelevitz, Deborah Doroshow, Benjamin J. Golas, Daniel Labow, John Sfakianos, Sujit S. Nair, Dimple Chakravarty, Michael Buckstein, Xiaoyu Song, Sacha Gnjatic, Brian D. Brown, Joseph Sparano, Ashutosh Tewari, Myron Schwartz, Nina Bhardwaj & Miriam Merad

  5. Precision Immunology Institute, Icahn School of Medicine at Mount Sinai, New York, NY, USA

    Thomas U. Marron, Emma Guttman-Yassky, Benjamin Ungar, Saurabh Mehandru, Effi Kenigsberg, Sacha Gnjatic, Brian D. Brown, Myron Schwartz, Nina Bhardwaj & Miriam Merad

  6. Division of Hematology and Medical Oncology, Icahn School of Medicine at Mount Sinai, New York, NY, USA

    Thomas U. Marron, Matthew D. Galsky, Deborah Doroshow, Sacha Gnjatic, Joseph Sparano, Nina Bhardwaj & Miriam Merad

  7. Center for Thoracic Oncology, Tisch Cancer Institute, Icahn School of Medicine at Mount Sinai, New York, NY, USA

    Thomas U. Marron, David Yankelevitz, Deborah Doroshow, Sacha Gnjatic, Nina Bhardwaj & Miriam Merad

  8. Center of Excellence for Liver and Bile Duct Cancer, Icahn School of Medicine at Mount Sinai, New York, NY, USA

    Thomas U. Marron, Bachir Taouli, Maria Isabel Fiel, Stephen Ward, Edward Kim, Michael Buckstein, Myron Schwartz & Miriam Merad

  9. Liver Cancer Program, Division of Liver Diseases and RM Transplant Institute, Icahn School of Medicine at Mount Sinai, New York, NY, USA

    Thomas U. Marron, Bachir Taouli, Maria Isabel Fiel, Stephen Ward, Edward Kim, Michael Buckstein, Myron Schwartz & Miriam Merad

  10. Department of Urology, Icahn School of Medicine at Mount Sinai, New York, NY, USA

    Matthew D. Galsky, John Sfakianos, Sujit S. Nair, Dimple Chakravarty, Ashutosh Tewari & Nina Bhardwaj

  11. Department of Diagnostic, Molecular and Interventional Radiology, Icahn School of Medicine at Mount Sinai, New York, NY, USA

    Bachir Taouli

  12. BioMedical Engineering and Imaging Institute, Icahn School of Medicine at Mount Sinai, New York, NY, USA

    Bachir Taouli, Edward Kim & David Yankelevitz

  13. Department of Pathology, Icahn School of Medicine at Mount Sinai, New York, NY, USA

    Maria Isabel Fiel & Stephen Ward

  14. Division of Interventional Radiology, Icahn School of Medicine at Mount Sinai, New York, NY, USA

    Edward Kim & David Yankelevitz

  15. Department of Dermatology, Icahn School of Medicine at Mount Sinai, New York, NY, USA

    Emma Guttman-Yassky & Benjamin Ungar

  16. Division of Gastroenterology, Icahn School of Medicine at Mount Sinai, New York, NY, USA

    Saurabh Mehandru

  17. Division of Surgical Oncology, Icahn School of Medicine at Mount Sinai, New York, NY, USA

    Benjamin J. Golas, Daniel Labow & Myron Schwartz

  18. Department of Radiation Oncology, Icahn School of Medicine at Mount Sinai, New York, NY, USA

    Michael Buckstein

  19. Department of Population Health Science and Policy, Icahn School of Medicine at Mount Sinai, New York, NY, USA

    Xiaoyu Song

  20. Department of Oncological Sciences, Icahn School of Medicine at Mount Sinai, New York, NY, USA

    Effi Kenigsberg, Sacha Gnjatic, Brian D. Brown, Joseph Sparano, Nina Bhardwaj & Miriam Merad

  21. Department of Genomics and Genetics, Icahn School of Medicine at Mount Sinai, New York, NY, USA

    Effi Kenigsberg & Brian D. Brown

Authors
  1. Thomas U. Marron
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Contributions

T.U.M. and M.M. authored the manuscript, with close collaboration, editorial comments and input from the other authors listed, who are founding members of the TARGET and INTERACT programs.

Corresponding authors

Correspondence to Thomas U. Marron or Miriam Merad.

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Competing interests

The authors have no competing interests in regards to the content of this commentary. They receive industry funding from partners to fund window studies, including Regeneron, Bristol Myers Squibb, Boehringer Ingelheim, Oncovir and Merck.

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Marron, T.U., Galsky, M.D., Taouli, B. et al. Neoadjuvant clinical trials provide a window of opportunity for cancer drug discovery. Nat Med 28, 626–629 (2022). https://doi.org/10.1038/s41591-022-01681-x

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