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

Thomas U Marron; Matthew D Galsky; Bachir Taouli; Maria Isabel Fiel; Stephen Ward; Edward Kim; David Yankelevitz; Deborah Doroshow; Emma Guttman-Yassky; Benjamin Ungar; Saurabh Mehandru; 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

doi:10.1038/s41591-022-01681-x

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

Nat Med. 2022 Apr;28(4):626-629. doi: 10.1038/s41591-022-01681-x.

Authors

Thomas U Marron^{1

2

3

4

5

6

7

8

9}, Matthew D Galsky^{10

11

12

13

14}, Bachir Taouli^{10

12

15

16

17

18}, Maria Isabel Fiel^{10

12

15

16

19}, Stephen Ward^{10

12

15

16

19}, Edward Kim^{10

12

15

16

18

20}, David Yankelevitz^{10

12

21

18

20}, Deborah Doroshow^{10

11

12

13

21}, Emma Guttman-Yassky^{22

23

24}, Benjamin Ungar^{22

23

24}, Saurabh Mehandru^{22

23

25}, Benjamin J Golas^{10

12

26}, Daniel Labow^{10

12

26}, John Sfakianos^{10

12

14}, Sujit S Nair^{10

12

14}, Dimple Chakravarty^{10

12

14}, Michael Buckstein^{10

12

15

16

27}, Xiaoyu Song^{10

12

28}, Effi Kenigsberg^{10

23

29

30}, Sacha Gnjatic^{10

12

23

13

21

29}, Brian D Brown^{10

12

23

29

30}, Joseph Sparano^{10

12

13

29}, Ashutosh Tewari^{10

12

14}, Myron Schwartz^{10

12

23

15

16

26}, Nina Bhardwaj^{10

22

11

12

23

13

21

14

29}, Miriam Merad^{31

32

33

34

35

36

37

38

39

40}

Affiliations

¹ The neoAdjuvant Research Group to Evaluate Therapeutics (TARGET), Icahn School of Medicine at Mount Sinai, New York, NY, USA. [email protected].
² Immunotherapy and Novel TargEt Research Across Clinical Teams (INTERACT), Icahn School of Medicine at Mount Sinai, New York, NY, USA. [email protected].
³ Early Phase Trials Unit, Tisch Cancer Institute, Icahn School of Medicine at Mount Sinai, New York, NY, USA. [email protected].
⁴ Tisch Cancer Institute, Icahn School of Medicine at Mount Sinai, New York, NY, USA. [email protected].
⁵ Precision Immunology Institute, Icahn School of Medicine at Mount Sinai, New York, NY, USA. [email protected].
⁶ Division of Hematology and Medical Oncology, Icahn School of Medicine at Mount Sinai, New York, NY, USA. [email protected].
⁷ Center for Thoracic Oncology, Tisch Cancer Institute, Icahn School of Medicine at Mount Sinai, New York, NY, USA. [email protected].
⁸ Center of Excellence for Liver and Bile Duct Cancer, Icahn School of Medicine at Mount Sinai, New York, NY, USA. [email protected].
⁹ Liver Cancer Program, Division of Liver Diseases and RM Transplant Institute, Icahn School of Medicine at Mount Sinai, New York, NY, USA. [email protected].
¹⁰ The neoAdjuvant Research Group to Evaluate Therapeutics (TARGET), Icahn School of Medicine at Mount Sinai, New York, NY, USA.
¹¹ Early Phase Trials Unit, Tisch Cancer Institute, Icahn School of Medicine at Mount Sinai, New York, NY, USA.
¹² Tisch Cancer Institute, Icahn School of Medicine at Mount Sinai, New York, NY, USA.
¹³ Division of Hematology and Medical Oncology, Icahn School of Medicine at Mount Sinai, New York, NY, USA.
¹⁴ Department of Urology, Icahn School of Medicine at Mount Sinai, New York, NY, USA.
¹⁵ Center of Excellence for Liver and Bile Duct Cancer, Icahn School of Medicine at Mount Sinai, New York, NY, USA.
¹⁶ Liver Cancer Program, Division of Liver Diseases and RM Transplant Institute, Icahn School of Medicine at Mount Sinai, New York, NY, USA.
¹⁷ Department of Diagnostic, Molecular and Interventional Radiology, Icahn School of Medicine at Mount Sinai, New York, NY, USA.
¹⁸ BioMedical Engineering and Imaging Institute, Icahn School of Medicine at Mount Sinai, New York, NY, USA.
¹⁹ Department of Pathology, Icahn School of Medicine at Mount Sinai, New York, NY, USA.
²⁰ Division of Interventional Radiology, Icahn School of Medicine at Mount Sinai, New York, NY, USA.
²¹ Center for Thoracic Oncology, Tisch Cancer Institute, Icahn School of Medicine at Mount Sinai, New York, NY, USA.
²² Immunotherapy and Novel TargEt Research Across Clinical Teams (INTERACT), Icahn School of Medicine at Mount Sinai, New York, NY, USA.
²³ Precision Immunology Institute, Icahn School of Medicine at Mount Sinai, New York, NY, USA.
²⁴ Department of Dermatology, Icahn School of Medicine at Mount Sinai, New York, NY, USA.
²⁵ Division of Gastroenterology, Icahn School of Medicine at Mount Sinai, New York, NY, USA.
²⁶ Division of Surgical Oncology, Icahn School of Medicine at Mount Sinai, New York, NY, USA.
²⁷ Department of Radiation Oncology, Icahn School of Medicine at Mount Sinai, New York, NY, USA.
²⁸ Department of Population Health Science and Policy, Icahn School of Medicine at Mount Sinai, New York, NY, USA.
²⁹ Department of Oncological Sciences, Icahn School of Medicine at Mount Sinai, New York, NY, USA.
³⁰ Department of Genomics and Genetics, Icahn School of Medicine at Mount Sinai, New York, NY, USA.
³¹ The neoAdjuvant Research Group to Evaluate Therapeutics (TARGET), Icahn School of Medicine at Mount Sinai, New York, NY, USA. [email protected].
³² Immunotherapy and Novel TargEt Research Across Clinical Teams (INTERACT), Icahn School of Medicine at Mount Sinai, New York, NY, USA. [email protected].
³³ Early Phase Trials Unit, Tisch Cancer Institute, Icahn School of Medicine at Mount Sinai, New York, NY, USA. [email protected].
³⁴ Tisch Cancer Institute, Icahn School of Medicine at Mount Sinai, New York, NY, USA. [email protected].
³⁵ Precision Immunology Institute, Icahn School of Medicine at Mount Sinai, New York, NY, USA. [email protected].
³⁶ Division of Hematology and Medical Oncology, Icahn School of Medicine at Mount Sinai, New York, NY, USA. [email protected].
³⁷ Center for Thoracic Oncology, Tisch Cancer Institute, Icahn School of Medicine at Mount Sinai, New York, NY, USA. [email protected].
³⁸ Center of Excellence for Liver and Bile Duct Cancer, Icahn School of Medicine at Mount Sinai, New York, NY, USA. [email protected].
³⁹ Liver Cancer Program, Division of Liver Diseases and RM Transplant Institute, Icahn School of Medicine at Mount Sinai, New York, NY, USA. [email protected].
⁴⁰ Department of Oncological Sciences, Icahn School of Medicine at Mount Sinai, New York, NY, USA. [email protected].

Abstract

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.

MeSH terms

Antineoplastic Agents* / therapeutic use
Breast Neoplasms* / drug therapy
Clinical Trials as Topic
Drug Discovery
Female
Humans
Neoadjuvant Therapy
Neoplasms* / drug therapy

Substances

Antineoplastic Agents

Abstract

MeSH terms

Substances

Grants and funding