Acute lymphoblastic leukaemia (ALL) is the most common cancer in children. While modern treatments cure over 90% of cases, ALL is still the leading cause of cancer-related deaths in children. The biggest risk factor for poor outcomes is relapse. The standard treatment is chemotherapy, which has serious side effects, both short-term and long-term. In case of relapse, children need even stronger treatments, such as high-dose chemotherapy and stem cell transplants, to achieve long-term cure. In recent years, new treatment options have been developed to target leukaemia cells more specifically while avoiding damage to healthy cells, thus reducing side effects. One group of these treatments are monoclonal antibodies, which target specific markers on leukaemia cells and help the immune system to destroy them. Another option is CAR T-cell therapy, where T-cells (a type of immune cell) are modified to recognize and attack leukaemia cells. This is especially effective for B-cell leukaemias (BCP-ALL), which express specific markers like CD19 and CD22. New small molecules are also being developed to target specific pathways activated in leukaemia cells, especially for T-cell leukaemias, which are harder to treat with immunotherapies.
The IntReALL consortium includes most European countries and is the largest research group focused on treating childhood relapsed ALL. The consortium conducts clinical trials to add new immunotherapy and targeted treatments into standard care, aiming to create more effective and less toxic treatments. We aim also at assuring equitable access to optimal therapy in every affected child regardless of ethnic origin, gender or socio-economic status in all participating countries. The group involves medical experts from 20 countries, pharmaceutical companies providing the drugs, scientific institutes offering the infrastructure for these large studies, and patient advocates to ensure the project is carried out in the best interest of patients and their families. The trials will also collaborate with regulatory authorities like the EMA to ensure they follow the law and may help extend the approval of new drugs. Ethical concerns, especially for such vulnerable patients, are carefully considered and approved by ethics committees.
