Structural insight into substrate and inhibitor discrimination by human P-glycoprotein

ABCB1 is a key multidrug exporter in humans that uses ATP to extrude a large array of chemically and structurally diverse compounds across cellular membranes. A recent “Science” paper by the Locher group (IMBB) sheds lights on the chemistry governing substrate and inhibitor binding in human ABCB1.

Science paper Alam
ABCB1 sits in the membranes of most cell types and uses ATP to shuttle various xenobiotic compounds such as the anti-cancer drug paclitaxel/Taxol (shown here as green spheres) across diverse blood-organ barriers. Antigen binding fragments of the inhibitory antibody UIC2 (shown here in blue) bind to the external surfaces of ABCB1 and can prevent drug extrusion.

ABCB1/P-glycoprotein actively extrudes xenobiotic compounds across the plasma membrane of diverse cells, which contributes to cellular drug resistance and interferes with therapeutic drug delivery. We determined the 3.5Å cryo-EM structure of substrate-bound human ABCB1 reconstituted in lipidic nanodiscs, revealing a single molecule of the chemotherapeutic compound paclitaxel bound in a central, occluded pocket. A second structure of inhibited, human-mouse chimeric ABCB1 revealed two molecules of zosuquidar occupying the same drug-binding pocket. Minor structural differences between substrate- and inhibitor-bound ABCB1 sites are amplified towards the NBDs, revealing how the plasticity of the drug-binding site controls the dynamics of the ATP-hydrolyzing NBDs. Ordered cholesterol and phospholipid molecules suggest how the membrane modulates the conformational changes associated with drug binding and transport.

Link to the publication in external page Science.
 

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