Drugs to fight acute myeloid leukaemia

Marcus Buschbeck

Bellvitge Biomedical Research Institute (IDIBELL), l’Hospitalet de Llobregat, Spain

Acute myeloid leukaemia remains one of the most devastating and lethal cancers. It is the most prevalent type of leukaemia in the adult population and affects more than 6,000 people in Spain each year. Treatment of this disease is a challenge, as tumour cells often develop resistance. Therapeutic strategies directed against multiple therapeutic targets simultaneously offer the most promising solution to overcoming this obstacle. In line with this approach, it is urgent to identify new targets and drugs based on different mechanisms of action.

In this regard, in previous studies, the team leading the project has managed to uncover a new therapeutic target from the chromatin regulatory space. Through a proof of concept, the researchers have been able to validate in vivo, in an animal model of the disease and in human cells, the effectiveness of eliminating this target by genetic means.

This novel target cannot be degraded using conventional small-molecule inhibitors. The team has therefore developed an innovative high-throughput screening method to identify and validate molecules that induce its degradation within the cell. This method is applicable to many other targets that cannot be inhibited by conventional inhibitors. Now, in the present project, they will provide proof of concept degrading molecules can be identified and that these are effective in experimental models of the disease.

Molecular glue degraders for a novel drug target in acute myeloid leukemia

Stage 1