Pharmacological Activation of Non-canonical NF-κB Signaling Activates Latent HIV-1 Reservoirs <i>In Vivo</i>.

Pache, Lars; Marsden, Matthew D; Teriete, Peter; Portillo, Alex J; Heimann, Dominik; Kim, Jocelyn T; Soliman, Mohamed S A; Dimapasoc, Melanie et al. · Cell Rep Med · 2020

basic_science · Level V

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Abstract

"Shock and kill" strategies focus on purging the latent HIV-1 reservoir by treating infected individuals with therapeutics that activate the latent virus and subsequently eliminating infected cells. We have previously reported that induction of non-canonical nuclear factor κB (NF-κB) signaling through a class of small-molecule antagonists known as Smac mimetics can reverse HIV-1 latency. Here, we describe the development of Ciapavir (SBI-0953294), a molecule specifically optimized for HIV-1 latency reversal that was found to be more efficacious as a latency-reversing agent than other Smac mimetics under clinical development for cancer. Critically, this molecule induced activation of HIV-1 reservoirs <i>in vivo</i> in a bone marrow, liver, thymus (BLT) humanized mouse model without mediating systemic T cell activation. This study provides proof of concept for the <i>in vivo</i> efficacy and safety of Ciapavir and indicates that Smac mimetics can constitute a critical component of a safe and efficacious treatment strategy to eliminate the latent HIV-1 reservoir.

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