The BAF complex inhibitor pyrimethamine reverses HIV-1 latency in people with HIV-1 on antiretroviral therapy

Sci Adv. 2023 Mar 17;9(11):eade6675. doi: 10.1126/sciadv.ade6675. Epub 2023 Mar 15.

Abstract

Reactivation of the latent HIV-1 reservoir is a first step toward triggering reservoir decay. Here, we investigated the impact of the BAF complex inhibitor pyrimethamine on the reservoir of people living with HIV-1 (PLWH). Twenty-eight PLWH on suppressive antiretroviral therapy were randomized (1:1:1:1 ratio) to receive pyrimethamine, valproic acid, both, or no intervention for 14 days. The primary end point was change in cell-associated unspliced (CA US) HIV-1 RNA at days 0 and 14. We observed a rapid, modest, and significant increase in (CA US) HIV-1 RNA in response to pyrimethamine exposure, which persisted throughout treatment and follow-up. Valproic acid treatment alone did not increase (CA US) HIV-1 RNA or augment the effect of pyrimethamine. Pyrimethamine treatment did not result in a reduction in the size of the inducible reservoir. These data demonstrate that the licensed drug pyrimethamine can be repurposed as a BAF complex inhibitor to reverse HIV-1 latency in vivo in PLWH, substantiating its potential advancement in clinical studies.

Publication types

  • Randomized Controlled Trial

MeSH terms

  • CD4-Positive T-Lymphocytes
  • HIV Infections* / drug therapy
  • HIV-1* / physiology
  • Humans
  • Pyrimethamine / pharmacology
  • Pyrimethamine / therapeutic use
  • RNA
  • Valproic Acid / pharmacology
  • Virus Activation
  • Virus Latency

Substances

  • Pyrimethamine
  • RNA
  • Valproic Acid