EVALUATION OF A NEW TREATMENT FOR ACUTE LYMPHOBLASTIC LEUKEMIA, THE MOST COMMON PEDIATRIC CANCER
This trial was co-funded by the Children Without Cancer City 2018 race.
About Childhood Leukemias
Leukemia in children shares similarities with adult leukemia but may present certain differences. Acute leukemia is a type of cancer characterized by the spread of cancerous cells originating from the bone marrow stem cells. These cells proliferate uncontrollably, invade the bone marrow, enter the bloodstream, and can thus invade the entire body. This proliferation hampers the production of all blood cells (white and red blood cells, platelets). In 2023, this disease is the most common cancer in children, accounting for approximately 30% of pediatric cancer cases.
The two main types in children are acute lymphoblastic leukemia (80% of cases) and acute myeloid leukemia. The exact causes of leukemia in children are not always clearly defined, but it is believed that they may be linked to genetic, environmental factors, or a combination of certain events occurring at the bone marrow stem cell level.
Learn more about leukemias: [link]
About the SELUDEX Project
SELUDEX is an international phase I/II therapeutic trial focusing on relapsed or therapeutically failed acute lymphoblastic leukemias. It is the most common pediatric cancer, affecting more than one child per day in France. The trial proposes a combination of Selumetinib, a new MEK inhibitor (targeted therapy), with dexamethasone for the treatment of leukemias with a RAS mutation.
This trial is unique in that it includes both children and adults and has been recently published.
Although this trial did not succeed in including all patients, it provided initial solid indications of the potential of this therapeutic combination. However, during this trial, CAR T-cell therapy was approved for these forms of relapsed or refractory leukemias. Nevertheless, the therapeutic combination developed in this trial could be an interesting solution while patients await their CAR T-cell therapy.
This project was funded through donations collected during the 2018 Children Without Cancer race.
Summary of the Trial
Promoter: University of Birmingham
Principal Investigator: Dr. Tobia Menne
Program Duration: 2018 – 2022
Recruitment: 12 patients were recruited
Countries involved: United Kingdom, France, Denmark, Ireland, Netherlands, Italy
Funding from Imagine for Margo: €90,000
Other co-funder: Cancer Research UK