In a pioneering clinical trial, a novel multifunctional CAR-T cell treatment has demonstrated remarkable efficacy and safety in HIV-1 infected patients, potentially paving the way for a functional cure for HIV/AIDS. Published in Cell Discovery, the phase I study introduced M10 CAR-T cells, engineered to incorporate endogenous broadly neutralizing antibodies (bNAbs) and the follicle-homing receptor CXCR5.

The M10 cells were designed with triple biological functions, targeting HIV-infected cells with broad cytotoxicity, neutralizing free viruses post-latency reversal, and homing to B-cell follicles. After confirming their triple bioactivity, 18 HIV-1 patients were treated with two allogeneic M10 cell infusions, 30 days apart, followed by chidamide stimulation to activate HIV-1 reservoirs. The treatment led to a significant suppression of viral rebound in 74.3% of cases and an average 67.1% reduction in viral load over a 150-day observation period.

Notably, M10 cells selectively pressured the latent viral reservoir without significant treatment-related adverse effects. These findings underscore the potential of M10 CAR-T cells as a novel, safe, and effective therapeutic option for HIV-1/AIDS, offering hope for a functional cure and an alternative to lifelong antiretroviral therapy.

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https://doi.org/10.1038/s41421-024-00658-z