Poster Presentation RACI Biomolecular Division Conference 2013

Preparation and evaluation of peptidomimetic scaffold targeting MCL-1 (#60)

Jean-Marc Garnier 1 , Nathan Toovey 1 2 , Jonathan B Baell 1 2 3 , Guillaume Lessene
  1. Walter and Eliza Hall Institute, Parkville, VIC, Australia
  2. The University Of Melbourne, Parkville, Victoria, Australia
  3. Monash Institute of Pharmaceutical Sciences, Parkville, Victoria, Australia

Proteins belonging to the BCL-2 family are key regulators of apoptosis.1 The subtle interplay between pro-survival and pro-apoptotic members of this family dictates the fate of cells. Upon intra- and extra-cellular stress signals, the BCL-2 proteins mediate apoptotic signals leading to cytochrome c release, caspase activation and final destruction of essential cellular components. Deregulation of the balance between these proteins leads to a wide range of diseases: for example, up-regulation of the pro-survival proteins is a key step towards tumour formation and progression and is also linked to resistance to current anti-cancer treatments.2  Targeting the BCL-2 proteins with small molecules represents a highly attractive new paradigm in anti-cancer therapy. 3

Key discoveries at the Walter and Eliza Hall Institute have contributed to a better understanding of the role played by the BCL-2 proteins in the apoptotic pathway. By developing molecular probes targeting members of this family, we aim to investigate the role of pro-survival proteins in cancer progression using chemical biology strategies.4  Within the BCL-2 family of protein, MCL-1 has remained a considerable challenge for medicinal chemists with only one publication reporting clear evidence of binding through X-ray crystallography.5  With the aim to reproduce the interaction between the selective peptide ligand NOXA-BH3 and MCL-1, we designed and prepared a series of peptidomimetics based on a benzoylurea scaffold. We present the efficient synthesis of these compounds as well as their affinity for MCL-1 as measured by two distinct binding assays. These compounds exhibit potencies in the micromolar range, and therefore represent potential starting point for further investigations towards potent MCL-1 inhibitors.

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  2. Strasser, A.; Cory, S.; Adams, J. M. EMBO J. 2011, 30, 3667-3683
  3. Lessene, G.; Czabotar, P.; Colman, P. Nat Rev Drug Discov. 2008, 989-1000
  4. Lessene, G. et al Nat. Chem. Biol. 2013, 9, 390-397
  5. Friberg, A. Et al J. Med. Chem. 2013, 56, 15-30