The development of novel anticancer agents has mainly been aimed at adults. However, children often have different types of cancer or different mutations and therefore cannot always profit from these new agents. In pediatric AML the introduction of novel medicines is urgently needed. The current front-line therapy results in significant short-term and long-term toxicity, and approximately 40% of children relapse and have poor outcomes. As AML is relatively rare in children, the number of patients that can take part in clinical research is limited. Therefore, international collaboration and prioritization is essential.
In a strategy forum of the ACCELERATE platform several stakeholders – including academia, industry, regulatory authorities of Europe and the US, and patient representatives – discussed the most promising drugs and targets to focus clinical research on in order to make progress. High priority was given to the development of CD123 as a target for cellular and targeted therapy. Another conclusion was that adolescents with mutations that are rare in children should be allowed to take part in adult trials of inhibitors of these mutations.
In addition, the new consortium PedAL/EUpAL will coordinate and organize collaboration between study groups in Europe, North America, Australia and New Zealand. One result of this collaboration is a so-called platform trial, in which data of several experimental agents are collected to determine which are the most promising for further development.
With this initiative the researchers hope to accelerate the development of innovative medicines for children and adolescents with AML, eventually improving treatment and increasing cure rate.