Small Molecule Inhibitors
Small molecule inhibitors (SMIs) are molecules designed to interfere with the function of a protein important in cancer onset and progression. LICR is building on years of comprehensive laboratory research, which has discovered and characterized many such proteins, in order to develop SMIs that have therapeutic potential.
SMIs, which can be used to inhibit protein signaling or even protein-protein interactions, are already being used to treat cancer. However, their identification and optimization is typically a long and complex process. High throughput screens, which allow investigators to rapidly assess the inhibitory effects thousands of potential SMI molecules are first required, as are more complex cell-based assays that analyze the effects of inhibiting the correct protein in more detail. Computer modeling experts and medicinal chemists may then be required to model and synthesize, respectively, slightly different variations of the SMI candidates to improve their inhibitory effect. This screening and optimization, which may take months or even years, is then followed by a comprehensive analysis of the candidate SMI's affect in tumor and biological models, before, finally, entering phase I clinical trials to test safety in humans.
LICR's most advanced projects to date are the development of SMIs that specfically target:
- - a mutated version of the JAK2 protein, which was discovered by LICR, that causes several myeloproliferative diseases
- - a family of proteins involved in epithelial-mesenchymal transition (EMT), a process required for tumor onset and progression
