Multi-Pole Approach to Structural Science

Warsaw
May 10 - 13, 2015

Bench-to-Bedside Design, Synthesis, Evaluation, and Development of Novel DNA Binding Agents
Waldemar Priebe

Using our own unique modular approach we have designed and synthesized libraries of distinctively different DNA binding agents and assessed their biological activity. Our design and systematic screening of such libraries allowed identifying highly apoptotic compounds with drug-like properties and unique anticancer activity. Two of our compounds with promising clinical activity (Annamycin and Berubicin-WP744) are being evaluated in humans and several other agents are in preclinical development. Design of one of our library focused on the development of novel drugs for CNS malignancies. Existence of the blood-brain barrier (BBB) that blocks anticancer drugs from reaching brain tumors is one of the main reasons for the lack of clinically efficient treatment for CNS malignancies. So far, we have designed and identified two classes of the DNA-binding agents that are able to cross BBB. Other potential clinical indications of our DNA binding drugs that are currently in preclinical development include melanomas resistant to BRAF inhibitors and pancreatic cancer, which has the lowest 5-years survival of all cancers. Selected DNA-binding agents are also being tested in combination with our separate class of protein targeting agents that we named “Modulators of Transcription.” We will discuss our different approaches to the design of DNA interactive agents, their evaluation and potential applications.