This technology includes the use of the small molecule metarrestin (ML246) for the treatment of several types of pancreatic cancer. A subcellular structure called the perinucleolar compartment (PNC) is frequently found in metastatic tumors and cancer stem cells. Reduction of PNC prevalence followed by medicinal chemistry was used to identify metarrestin as a compound that reduces PNC prevalence without significantly impacting cell viability. In vitro and in vivo animal work have demonstrated desirable pharmacokinetic properties as well as a reduction in metastatic burden and extended survival.
Further clinical work could establish the use of metarrestin as an oral adjuvant treatment for pancreatic cancer alone or in combination with existing chemotherapeutic agents such as gemcitabine.
Metarrestin is an orally active first-in-class and specific perinucleolar compartment inhibitor. Clinical trials using metarrestin in other cancers have shown favorable patient outcomes.