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About Transplant Oncology
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Clinical Trials
Genzyme Oncology
Topoisomerase I Inhibitor

Topoisomerases are enzymes that play a pivotal role in facilitating DNA replication. Topoisomerase I inhibitors based on camptothecin are an established class of agents playing important roles in the management of several types of cancer.

Genzyme’s Topoisomerase I inhibitor is a novel non-camptothecin small molecule. The structure of the compound is designed to eliminate the lactone ring that is a metabolic liability inherent in the camptothecin compounds. The drug has shown improved properties over the camptothecins with respect to mechanism of action which may provide the basis of the observed unique patterns of in vitro and in vivo preclinical activity.

Genzyme is currently conducting a multi-center phase I clinical trial in patients with advanced solid tumor malignancies.

Future clinical investigation of the drug will include study of the effect of the compound on multi-drug resistant tumors and will also explore the development of biomarkers that might predict response to the drug.