Megacaryoblastic acute leukemia: understanding the mechanisms involved to improve management

Megacaryoblastic acute leukemia
  • Megacaryoblastic acute leukaemias are infantile blood cancers that are too resistant to treatments.
  • The researchers wish to explore the molecular mechanisms at the heart of the development of these cancers to discover new therapeutic targets.
  • To do this, they are developing very advanced cell models to explore the formation of tumors.
 This research is led by Thomas Mercher and his team “Genetics and modeling of childhood leukemia” at the Gustave Roussy Institute in Villejuif.

Megacaryoblastic acute leukemia: a specific entity

Thomas Mercher and his team are interested in a particular childhood leukemia, acute megacaryoblastic leukemia (LAM7). This form of leukemia is in particular diagnosed in children with Down’s syndrome, but it can also occur in children without a particular risk factor. In the latter, the prognosis of the pathology is often very dark: only 14 to 34% of them are still alive 3 years after the diagnosis, despite intensive chemotherapy protocols. Research is therefore intensifying to develop new treatments for this disease.

Proteins at the heart of cancer development

Leukemias are cancers that result from an accumulation of genetic mutations in precursor cells that will give rise to certain blood cells. The latter then multiply in an anarchic manner. Among the mutations most commonly found during leukemia are those which induce the production of abnormal proteins called “fusion proteins”. These proteins, by activating certain molecular processes, are at the heart of cancer development. Thomas Mercher and his team wish to better characterize the role of these fusion proteins as well as the molecules that regulate their activity during megacaryoblastic acute leukemia (LAM7) in order to counter their action.

Towards the creation of cellular models of pathology

To this end, researchers will first develop cell models that faithfully reproduce the genetic mechanisms behind these fusion proteins. This will involve innovative molecular biology and genetics techniques. In a second step, the team will study the deregulations caused by these fusion proteins.

Ultimately, researchers want to use these laboratory models to identify new therapeutic targets, but also to test new therapies in the disease: a first step towards better management of this form of childhood leukemia.

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