Intratumoral Immunotherapy Clinical Trial (IICT) Design

On behalf of the society and the SITC Intratumoral Immunotherapy Clinical Trials Manuscript Development Group, we are pleased to announce the publication of a new manuscript “Society for Immunotherapy of Cancer (SITC) recommendations on intratumoral immunotherapy clinical trials (IICT): from pre-malignant to metastatic disease” in the Journal for ImmunoTherapy of Cancer.

While intratumoral immunotherapies have the potential to boost anti-tumor immune responses to overcome immunosuppressive tumor microenvironments, phase III trials evaluating them in late-stage disease have had limited success to-date, in part due to the complex nature of these trials not fully adapted to assess local therapies. The aim of this manuscript is to provide guidance on how to design trials with the greatest likelihood of demonstrating the benefits of intratumoral immunotherapy across all stages of pathogenesis, and ultimately bring more effective treatment options to patients.

Key Recommendations:

  • Patient stratification factors 
  • Exclusion criteria
  • Study arm considerations
  • Optimal endpoints
  • Response assessment
  •  Patient populations to prioritize

Expert Panel Recommendations for Trial Design

To address challenges in intratumoral immunotherapy clinical trial design, an Expert Panel comprised of an international group of leaders from academia and industry convened to develop guidance on a variety of design elements tailored to different stages of cancer development—from pre-malignant to unresectable/metastatic—that can maximize the chance of capturing the effect of intratumoral immunotherapies.  Key elements discussed include patient stratification factors, exclusion criteria, study arm considerations, optimal endpoints, response assessment, and patient populations to prioritize.

Supplemental Guidance on Localized Therapy Administration

 A critical component for the development of intratumoral immunotherapies is identifying a standardized process for monitoring and documenting drug injection/application.  As an additional educational tool for readers, an illustrated supplement accompanies the manuscript to instruct users on the proper administration of localized immunotherapies for solid tumors and lymphomas, subcutaneous lymph nodes from lymphoma or metastatic tumors, accessible head and neck, liver, and breast lesions, and intravesical immunotherapy.

Intratumoral Immunotherapy Clinical Trials Expert Panel


Jason J. Luke, MD, FACP 
UPMC Hillman Cancer Center


Diwakar Davar, MD, MS
UPMC Hillman Cancer Center 


Aurelien Marabelle, MD, PhD
Gustave Roussy 


Robert H. Andtbacka, MD, CM, FACS, FRCSC — HiFiBiO Therapeutics

Nina Bhardwaj, MD, PhD — The Icahn School of Medicine at Mount Sinai

Joshua D. Brody, MD — The Icahn School of Medicine at Mount Sinai

Jason Chesney, MD, PhD — University of Louisville

Robert Coffin, PhD  — Replimune

Thierry de Baere, MD, PhD — Gustave Roussy

Tanja D. de Gruijl, PhD — Amsterdam University Medical Centers

Matthew Fury, MD — Regeneron Pharmaceuticals

Gregory Goldmacher, MD, PhD, MBA — Merck

Kevin J. Harrington, BSc, MBBS, MRCP, FRCR, FRCP, PhD, DIC — The Royal Marsden

Howard Kaufman, MD — Ankyra Therapeutics

Ciara M. Kelly — Memorial Sloan Kettering Cancer Center

Anuradha D. Khilnani, MD — Merck

Ke Liu, MD, PhD — Marengo Therapeutics

Sherene Loi, MD, PhD — Peter MacCallum Cancer Centre

Georgina V. Long, MBBS, PhD, BSc, FRAC — University of Sydney

Ignacio Melero, MD, PhD — Universidad de Navarra

Mark Middleton, MD, PhD — University of Oxford

Bart Neyns, MD, PhD — Universitair Ziekenhuis Brussel

David J. Pinato, MD, MRes, PhD — Imperial College London

Rahul A. Sheth, MD — University of Texas MD Anderson Cancer Center

Stephen B. Solomon, MD — Memorial Sloan Kettering Cancer Center

Philippe Szapary, MD, MSCE — Johnson and Johnson

Additional Resource

SITC Summit on Intralesional Immunotherapy: Moving Beyond Oncolytic Viruses
THURSDAY, SEPT. 19, 2024 - 10 A.M.–2 P.M. EDT

The SITC Summit on Intralesional Immunotherapy: Moving Beyond Oncolytic Viruses will highlight the current understanding of intralesional immunotherapies and their effects on the immune system and the tumor microenvironment. In situ vaccination, intralesional combination therapies, and next generation intralesional immunotherapies will also be discussed. This open-door virtual educational event will present the current state and future directions of the field and feature participation of invited stakeholders involved in intralesional immunotherapy research and development, including clinicians and researchers from academia and industry.