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[Preprint]. 2023 Nov 17:2023.09.05.556422. [Version 3] doi: 10.1101/2023.09.05.556422

TGF-β blockade drives a transitional effector phenotype in T cells reversing SIV latency and decreasing SIV reservoirs in vivo

Jinhee Kim, Deepanwita Bose, Mariluz Araínga, Muhammad R Haque, Christine M Fennessey, Rachel A Caddell, Yanique Thomas, Douglas E Ferrell, Syed Ali, Emanuelle Grody, Yogesh Goyal, Claudia Cicala, James Arthos, Brandon F Keele, Monica Vaccari, Ramon Lorenzo-Redondo, Thomas J Hope, Francois Villinger, Elena Martinelli
PMCID: PMC10680555  PMID: 38014094

Abstract

HIV-1 persistence during ART is due to the establishment of long-lived viral reservoirs in resting immune cells. Using an NHP model of barcoded SIVmac239 intravenous infection and therapeutic dosing of the anti-TGFBR1 inhibitor galunisertib (LY2157299), we confirmed the latency reversal properties of in vivo TGF-β blockade, decreased viral reservoirs and stimulated immune responses. Eight SIV-infected macaques on ART were treated with four 2-week cycles of galunisertib. ART was discontinued 3 weeks after the last dose, and macaques euthanized 6 weeks after ART-interruption(ATI). All macaques rebounded between week 2 and 6 post-ATI, but one did not. Galunisertib led to viral reactivation as indicated by plasma viral load and immunoPET/CT with 64 Cu-DOTA-F(ab’) 2 -p7D3-probe. A decrease in cell-associated (CA-)SIV DNA was detected in lymph nodes, gut and PBMC, while intact pro-virus in PBMC decreased by 3-fold. No systemic increase in inflammatory cytokines was observed. High-dimensions cytometry, bulk, and single-cell RNAseq revealed a shift toward an effector phenotype in T and NK cells characterized by a progressive downregulation in TCF1.

In summary, we demonstrated that galunisertib, a clinical stage TGF-β inhibitor, reverses SIV latency and decreases SIV reservoirs by driving T cells toward an effector phenotype, enhancing immune responses in vivo in absence of toxicity.

One-sentence summary

TGF-β blockade drives an effector phenotype in immune cells leading to SIV latency reversal and enhanced immune responses in vivo.

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