Background and general objectives

Acute myeloid leukemias in children and adolescents are rare and poorly known cancers.

Despite advances in the past 30 years, the prognosis of pediatric AML remains poorer than that of other childhood leukemia subtypes.

To progress, research is essential.

Rresearch projects of the CONECT-AML network aim to explore the following themes:

  1. AML is most commonly an adult disease. The fact that they arise in children or adolescents raises the following question: would AML in children and adolescents be linked to a genetic predisposition?
  2. Standard treatment for AML includes chemotherapy, sometimes combined with a bone marrow transplant for high-risk or relapsed patients. The transplant is a heavy treatment, source of long-term sequelae. Progress is needed to reduce the relapse rate, improve survival and limit the potential sequelae of treatment. Former patients should be followed in order to better detect potential sequelae and reduce their long-term impact
  3. The genetic study of leukemic cells allows us to better describe the transformation mechanisms and to identify new therapeutic approaches. The study of bone marrow samples from patients with a known genetic predisposition may help to better describe the sequence of genetic abnormalities that are decisive for leukemic transformation. This work will enable to define the best therapeutic target to eradicate the leukemic cell, or to target the first “key” stage of transformation before the onset of the disease.
  4. The Next Generation Sequencing techniques (NGS), associated with artificial intelligence, are revolutionary tools, bringing great hope, which will enable to accelerate research and develop the medicine of the future. This medicine will better take into account the individual characteristics of each disease and each patient, to more accurately guide physicianstowards the best treatment.
  5. The study of leukemia samples for research purposes faces experimental difficulties. Leukemia samples are sometimes difficult to collect. The quantity available is sufficient to establish the diagnosis, but does not always enable to keep enough material for multiple researches. In order to solve this problem, new techniques can enable, from a few leukemic cells of the patient, to amplify them, in order to have unlimited material available for research. The development of these new experimental models (leukemic cells in culture, or PDX for “Patient Derived Xenograft”) will allow: to have tumor material without quantity limitation; to study leukemic cells in a “humanized” environment; and to be able to test the sensitivity of leukemic cells to the various available drugs.
  6. The generated knowledge d, especially in genetics, aims to improve patient care. However, it can have difficult psychological consequences for patients and their families, especially in the context of predisposition. An ethical reflection of this research will be carried out.
  7. Patients and their families request information about the disease and research. CONECT-AML website aims to make research on AML in children and adolescents more accessible and understandable.