CHIP-AML22: improving the diagnosis and treatment of paediatric acute myeloid leukemia (AML)

Paediatric acute myeloid leukemia (AML) is a rare but severe blood cancer. 

The EU4Health project CHIP-AML22 is working to improve treatment outcomes for children with AML by advancing targeted treatments that reduce the level of toxicity and guarantee more equal outcomes across Europe.  

In 2024, HaDEA talked to representatives of the project to discover its objectives and ambitions. Closer to its completion, we explore CHIP-AML22's main achievements and results with prof. Cornelis Jan H Pronk, project coordinator.

What are the project's main achievements and what are you proud of? 

What we’re the proudest of is the fact that CHIP-AML22 now exists as a working European platform, one shared protocol used across more than 60 hospitals in 16 countries. Therefore, every child with AML in Europe is diagnosed, treated and monitored to the same high standard. In particular, we have: 

• built and validated a common, highly sensitive test for residual leukaemia that’s already running in seven countries; 
• taken the trial from its first patient in September 2023 to around 190 children registered today; 
• shown that the platform can host targeted-drug trials.  

Early and preliminary results look safe, with survival in line with our previous protocol. 

How do the new treatments and methods differ from existing ones, and what benefits do they bring to patients? 

Childhood AML treatment is already at the limit of what a child’s body can tolerate and after the first of five chemotherapy courses, almost all children are hospitalised with neutropenic fever. It is not possible to add more chemotherapy, as toxicity would reach unacceptable and sometimes fatal levels. So, instead, we add targeted drugs matched to each child’s leukaemia, which improve the odds without piling on toxicity. Each high-risk group identified by the project is matched to a targeted medicine in addition to standard chemoterapy. At the same time, we’re testing whether some children can safely have less treatment, while an additional medication protects the heart from potential damage. The benefit is straightforward: more effective and gentler treatment, with the right intensity for each child. 

What difference could these advances ultimately make for children with AML and their families? 

While survival in childhood AML has reached about four in five children, relapse is the main cause of death for the remaining ones. Our aim is to reach nearly all high-risk children (45-50% of whom currently relapse) with a targeted treatment, and to spare survivors the long-term harm that intensive chemotherapy can cause to the heart, fertility and learning skills. For a family, that’s the difference between saving their child’s life and guaranteeing the fullest possible life for that child. 

Why is cross-European collaboration so important here, and how can CHIP-AML22 help ensure equal access to treatment? 

AML in children is so rare that no country has enough patients to make progress alone. Together, we diagnose about 150 pediatric AML patients a year, so collaboration is the only way forward. This is also how we pursue fairness: the aim is that a child in a small country gets the same advanced diagnosis and monitoring as one in a large centre. In addition, this should not be dependent on the economic level in a particular country. Importantly, our project has exposed where equal access breaks down, where a proven drug can’t be used in some countries because of licensing or reimbursement rules. That’s a clear message for policymakers: adjusting rules on drug licensing, availability and reimbursement across borders matters as much as science. 

Are there any particular challenges the project has faced? 

Our biggest challenges have been practical rather than scientific. Launching a complex, twelve-country trial through the new EU Clinical Trials Information System (CTIS) took longer than planned and the project needed a one-year extension. Moreover, getting modern drugs to every child has been hard: one company withdrew a drug we had planned to use, and another drug that’s already on the market can’t be used in some countries because it isn’t reimbursed for this purpose. These access barriers are a real obstacle, and one of the project’s most important findings. 

What has EU funding made possible that would have been difficult otherwise? 

EU funding (around €2.4 million) is what turned a shared ambition into real, lasting infrastructure. Before it, advanced genetic testing reached fewer than one in five children and many countries couldn’t access newer drugs. The grant covered the work of teams in almost every country to build diagnostics to one common standard, the central database and quality systems, and the targeted drug trials themselves. Without it, we simply couldn’t offer every child the best available care within a reasonable time. 

Background  

EU4Health is the fourth and largest of the EU health programmes. The EU4Health programme goes beyond an ambitious response to the COVID-19 crisis to address the resilience of European healthcare systems. The programme provides funding to national authorities, health organisations and other bodies through grants and public procurement, contributing to a healthier Europe.  

HaDEA manages the vast majority of the total EU4Health budget and implements the programme by managing calls for proposals and tenders from 2021 to 2027.