SPECIAL SEMINAR

"From Drug Repositioning to Structure- Based Drug Design to Tackle Acute Lymphoblastic Leukemia"

Xavier MORELLI

Responsable d’équipe Directeur de Recherche CNRS

Centre de Recherche en Cancérologie de Marseille (CRCM)

Abstract

The discovery and development of new drugs is a very highly costly and slow process, while repositioning old drugs to treat new diseases is increasingly becoming an attractive approach. We will present in this conference a new concept, “drug repositioning to drug design approach”, where the repurposing of a drug (on a new target) can be used as a starting point to accelerate the development of a new drug. We have applied this methodology to a nucleotide kinase involved in the development of acute lymphoblastic leukemia (ALL). Our lead compound doubles median survival in a human leukemia mouse model as well as in a patient-derived xenograft model, demonstrating therapeutic potential for the treatment of ALL (Saez-ayala et al. Nature Communications 2023).