Oral Presentation CD1-MR1 Workshop 2025

Design of cytokine engineered invariant Natural Killer T (iNKT) cells for adoptive immunotherapy of cancer (#62)

Iris Stefanetta 1 , Gloria Delfanti 1 , Alessandra Perini 1 , Giulia Lopatriello 1 , Gaia Antonini 1 , Claudio Garavaglia 1 , Giovanni Sitia 2 , Ingrid Cifola 3 , Eleonora Mangano 3 , Roberta Bordoni 3 , Clarissa Consolandi 3 , Gianluca De Bellis 3 , Paolo Dellabona 1 , Giulia Casorati 1
  1. Experimental Immunology Unit, Division of Immunology, Transplantation and Infectious Diseases, IRCCS San Raffaele Scientific Institute, Milan, Italy
  2. Experimental Hepatology Unit, Division of Immunology, Transplantation and Infectious Diseases, IRCCS San Raffaele Scientific institute, Milan, Italy
  3. National Research Council (CNR), Institute for Biomedical Technologies (ITB), Segrate, Mi, Italy

CD1d-restricted invariant Natural Killer T (iNKT) cells play a key role in tumor immunosurveillance by actively remodeling the composition of myelomonocytic cells in the tumor microenvironment, enforcing immunostimulatory conditions. iNKT cell transferred into tumor-bearing mice home to the tumor site and delay tumor progression, but their therapeutic impact is limited by progressive functional exhaustion.

To enhance their anti-tumor potential, we engineered iNKT cells to express different immunostimulatory cytokines: IL-15 (±IL-15Rα); IL-12 and IFNα, each known to support lymphocyte survival and effector function. IL-15 and IFNα supported robust expansion and preserved the TH0 effector phenotype of iNKT cells, while IL-12 promoted a TH1-skewed, pro-inflammatory profile with limited in vitro persistence. RNA sequencing of cytokine-engineered iNKT cells revealed that iNKT-IL12 cells acquired a distinct gene expression program, characterized by high metabolic activity, effector profile and terminal differentiation signatures compared to iNKT-IL-15 or iNKT-IFNα cells. In vivo, iNKT-IL12 cells demonstrated superior tumor control but induced significant systemic toxicity, including body weight loss and elevated plasma cytokines in both tumor- and non-tumor-bearing animals, which critically depended on the induction of a massive endogenous IFNg response in the recipient mice. In contrast, IL-15 and IFNα engineering conferred only moderate anti-tumor efficacy, although with good safety profiles. To mitigate IL-12-associated toxicity, we are developing a membrane-bound form of IL-12 to localize cytokine effects and avoid systemic exposure.

These findings highlight how cytokine engineering differentially shapes iNKT cell behavior and identifies IL-12 as a powerful but potentially toxic enhancer of anti-tumor activity.