Poster Presentation Melbourne Protein Group Student Symposium 2013

Structurally guided small molecule targeting of the Insulin and Type 1 Insulin-like Growth Factor Receptors. (#45)

Callum Lawrence 1
  1. Walter and Eliza Hall Institute of Medical Research, Parkville, VIC, Australia

The Insulin Receptor (IR) and Type 1 Insulin-Like Growth Factor Receptor (IGF-1R) are homodimeric α2β2 receptor tyrosine kinases capable of hybrid heterodimerisation. While both IR and IGF-1R possess high affinities for their cognate ligands (insulin, and IGF-I and IGF-II, respectively), they may also bind and be activated by reciprocal ligands. Activation of both homodimeric and hybrid receptors effects the downstream PI3K-AKT and RAS-RAF-MAPK signaling pathways, having varying effects on cellular glucose metabolism, differentiation and proliferation. Aberrant signalling leads to clinical manifestations including diabetes (in the case of IR) and cancer (in the case of IGF-1R and possibly IR), making these receptors attractive pharmaceutical targets.
Recent failures of novel therapeutics targeting of IR/IGF-1R highlight the need for highly specific receptor targeting.  Such an approach has been hampered by an inadequate understanding of the way that each target engages its respective ligand. Recently it was shown that the primary insulin binding site is assembled as a tandem element comprising the first leucine-rich repeat domain (L1) of one receptor monomer in association with the C-terminal region (αCT) of the opposite receptor α-chain. Dissociation of the L1 and αCT components will thus specifically abrogate ligand binding.
To further the above idea, a number small molecules were screened for their ability to perturb the interaction between a high-affinity αCT mimetic and the L1 domain within a truncated IGF-1R system. Identified hits were subsequently confirmed in a surface plasmon resonance assay targeting the same interaction.
The agonistic or antagonistic potential of identified compounds as well as their defined binding kinetics remain to be determined. Further pharmacological refinement of lead compounds will require accurate pharmacophore generation through x-ray crystallography; of which some progress has been made.