New therapeutics against T-cell acute lymphoblastic leukemia
A protein named c-Myc is causally implicated in over 50% of all human cancers. Preliminary work supports two proteins (POZ domain protein and c-Myc cofactor Miz-1) as a new drug target to disable c-Myc function. We have identified small molecules which act on this target and will now serve develop a series of optimized and effective molecules as cancer therapeutics.
To optimize these molecules, a combination of computational design and medicinal chemistry (synthesis and biological evaluation) will be carried out. Molecules that demonstrate high potency will be tested for chemical properties such as solubility in water (ie plasma) and for biological effects such as the effect of cell lines. The best compounds will then be scaled up, formulated, tested for required properties (e.g., toxicity, metabolism) and then in vivo. The results of the biological assays will guide the design and synthesis of molecules with improved potency, efficacy and pharmaceutical profile.