Bisphosphonate drugs inhibit bone resorption but also appear to have actions outside the skeleton by mechanisms that are poorly understood. Clinical and epidemiological studies suggest that nitrogen-containing bisphosphonate (N-BP) therapy is associated with reduced risk of infection and mortality from pneumonia. To better understand how N-BPs may have beneficial effects in the lung we employed various approaches including multicolour flow cytometry, mass spectrometry and single-cell RNA sequencing, and identified alveolar macrophages (aMac) as targets of N-BPs.
Flow cytometric analysis of bronchoalveolar lavage cells, isolated from mice after a single intravenous injection of a fluorescently‑tagged analogue of the N-BP zoledronate (AF647-ZOL), revealed drug uptake by >97% of aMac (CD11blo/-CD11chiCD64+) and 34% of interstitial (CX3CR1+CD11bhi) macrophages. AF647-ZOL was still detectable in aMac one week after administration. Intracellular accumulation of unprenylated proteins and build-up of the metabolite isopentenyl diphosphate (IPP) are hallmarks of the pharmacological actions of N-BPs. Consistent with intracellular uptake of labelled ZOL, using mass spectrometry and a customised biochemical assay we detected a clear build-up of IPP and unprenylated Rab GTPases in aMac 48 hours after one intravenous dose of ZOL. Importantly, single-cell RNA sequencing of aMac, isolated from mice challenged intranasally with LPS endotoxin, revealed enhanced gene expression of cytokines and chemokines (including Il1b, Cxcl2, Cxcl3, Ccl3 and Ccl4) in a subpopulation of aMac in ZOL‑treated mice compared to controls. This was accompanied by higher levels of these cytokines and chemokines in bronchoalveolar lavage fluid from ZOL‑treated mice after LPS-challenge, as measured by multiplex cytokine assays.
These studies dispel the dogma that N-BPs act only in bone and clearly demonstrate that they also affect lung-resident macrophages. In addition to preventing bone loss, we propose that N-BP treatment may boost the initial immune response of aMac to pulmonary infections, therefore conferring protection from pneumonia.