Poster Presentation The 46th Lorne Conference on Protein Structure and Function 2021

De Novo Designed Receptor Transmembrane Domains Modulate CAR-T Cell Function (#410)

Nick Chandler 1 2 , Ashleigh Davey 1 2 , Assaf Elazar 3 , Sarel Fleishman 3 , Matthew Call 1 2 , Melissa Call 1 2
  1. Walter and Eliza Hall Institute of medical research, Parkville, VIC, Australia
  2. Department of Medical Biology, University of Melbourne, Parkville, VIC, Australia
  3. Weizmann Institute, Rehovot, Israel

Chimeric antigen receptor (CAR)-T cell therapy has revolutionized the treatment of B cell malignancies by redirecting patient T cells to destroy cancer cells using engineered receptors. However, CAR-T therapies carry significant risk of inducing cytokine release syndrome (CRS), a potentially deadly toxicity caused by excessive release of inflammatory cytokines. The ability to minimize CRS toxicity without compromising tumour cell-killing is therefore vital to the continued improvement of CAR-T therapies. We aimed to investigate the currently ill-defined relationship between CAR oligomeric state and potency, with the aim of leveraging this knowledge to predictably modulate CAR activity. Alongside de-novo protein design collaborators we generated synthetic transmembrane domain (TMD) sequences that predictably formed homo-oligomeric structures. X-ray protein crystallography was used to determine the atomic structures of dimeric and trimeric TMD peptides, confirming a close alignment with their predicted structures. Structurally validated TMD sequences were then inserted into a well-established anti-HER2 CAR construct (comprised of an anti-HER2 scFv attached via stalk/transmembrane domains to a stimulatory tail sequence) and functionally characterised. These oligomeric TMDs facilitated expression in the context of a HER2-specific CAR construct in a mouse T cell line and triggered signalling in response to HER2+ target cells. When expressed in mouse primary CD8+ T cells and co-cultured with HER2+ target cells, dimeric and trimeric CARs exhibited enhanced target cell killing compared to a reference anti-HER2 CAR and, remarkably, also produced dramatically reduced levels of inflammatory, CRS-associated cytokines. These findings present an exciting opportunity to improve both efficacy and safety of CAR T cell therapies and warrant further validation in in vivo mouse models.