Inactivating a single enzyme could effectively eradicate an aggressive form of leukemia

EPFL scientists show how inactivating a single enzyme could effectively eradicate an aggressive form of leukemia. The principles could apply to other cancers as well.

T-cell acute lymphoblastic leukemia (T-ALL) is a rare type of leukemia that is more common in older children and teenagers. It affects white blood cells, which are an essential component of our immune system that fights infection.

T-ALL onset is linked to microRNAs, small non-coding RNA molecules that silence RNA and regulate gene expression. Most MicroRNAs are generated with the help of the enzyme Dicer1, which has been the focus of research for treating T-ALL. EPFL scientists now show that Dicer1 is crucial for the development of T-ALL, and inhibiting it can actually prevent the disease altogether.

The lab of Freddy Radtke at EPFL has now shown that Dicer1 is a central factor in T-ALL. The scientists tracked the development of T-ALL in genetically engineered mice where Dicer1 could be abrogated in a mouse model where a mutated cancer gene induces T-ALL.

Switching Dicer1 off at an early stage of T-ALL completely prevented the development of the disease despite the mutated gene. But even more fascinating was the fact that, in mice where Dicer1 was completely abrogated, T-ALL cells were entirely eliminated, allowing all the mice to survive.