Structure of P-glycoprotein reveals a molecular basis for poly-specific drug binding

SG Aller, J Yu, A Ward, Y Weng, S Chittaboina, R Zhuo… - Science, 2009 - science.org
SG Aller, J Yu, A Ward, Y Weng, S Chittaboina, R Zhuo, PM Harrell, YT Trinh, Q Zhang…
Science, 2009science.org
P-glycoprotein (P-gp) detoxifies cells by exporting hundreds of chemically unrelated toxins
but has been implicated in multidrug resistance (MDR) in the treatment of cancers. Substrate
promiscuity is a hallmark of P-gp activity, thus a structural description of poly-specific drug-
binding is important for the rational design of anticancer drugs and MDR inhibitors. The x-ray
structure of apo P-gp at 3.8 angstroms reveals an internal cavity of∼ 6000 angstroms cubed
with a 30 angstrom separation of the two nucleotide-binding domains. Two additional P-gp …
P-glycoprotein (P-gp) detoxifies cells by exporting hundreds of chemically unrelated toxins but has been implicated in multidrug resistance (MDR) in the treatment of cancers. Substrate promiscuity is a hallmark of P-gp activity, thus a structural description of poly-specific drug-binding is important for the rational design of anticancer drugs and MDR inhibitors. The x-ray structure of apo P-gp at 3.8 angstroms reveals an internal cavity of ∼6000 angstroms cubed with a 30 angstrom separation of the two nucleotide-binding domains. Two additional P-gp structures with cyclic peptide inhibitors demonstrate distinct drug-binding sites in the internal cavity capable of stereoselectivity that is based on hydrophobic and aromatic interactions. Apo and drug-bound P-gp structures have portals open to the cytoplasm and the inner leaflet of the lipid bilayer for drug entry. The inward-facing conformation represents an initial stage of the transport cycle that is competent for drug binding.
AAAS