Tiny patients with leukaemia but big shot to help them achieve a cure

This project was selected in the framework of our European call: FIGHT KIDS CANCER 2022. It is jointly financed by Imagine for Margo, Kriibskrank Kanner Fondatioun and KickCancer.

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Leukaemia is a cancer of the blood where the patient’s own system turns malignant: the white blood cells grow anarchically and surpass the red blood cells in the blood stream – causing extreme fatigue, bruising and ultimately, the patient’s death.

Acute lymphoblastic leukaemia or ALL is the most common type of blood cancer – lymphoblastic refers to the type of white blood cells that has mutated. Today, about 90% of the children with ALL will survive. As a result, childhood ALL can almost be considered as a curable disease. Unfortunately, this is not true for all patients diagnosed with ALL, especially not for infants, i.e., children under 1 year of age.

This project pursues two objectives. First of all, it aims at understanding the mechanisms which make infant ALL so aggressive. It has already been demonstrated that most of the babies diagnosed with ALL carry a leukaemia-specific mutation in the MLL gene. Understanding the MLL gene is thus the key to better treatments.

Second, it will attempt to identify new treatment options by targeting these mechanisms and providing evidence to show that these newly identified treatments could be efficient when applied to patients in the clinic.

The findings of this project will be discussed with the clinical trial groups that set up clinical studies in ALL: the INTERFANT/UKALL consortium.

The project’s findings could be used to improve the risk stratification between the patients and test the identified treatments in an international clinical setting. This could allow more personalised, effective, and kind treatments for infants with ALL in the future.