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Our goal: A decisive step towards systematic prioritization of new cancer therapies for children in Europe and beyond through a better use of, and access to, existing genomic and clinical data

Despite the enormous progress made in recent decades in the treatment of childhood cancer, the chances of survival for patients with high-risk diseases or after a relapse are still very poor. Because the spectrum of cancers in children is fundamentally different from that in adults, it would be of great value to develop new cancer drugs specifically for children. However, due to the small 'market size', this unfortunately is rarely a priority for the pharmaceutical industry. Instead, there is currently much greater potential in prioritizing cancer drugs that were primarily developed for use in adult cancer for possible promising applications in pediatric cancer. This opportunity has been further enhanced by recent legislative changes in Europe and the USA, as medications primarily developed for adults must now be evaluated for their use in children if the mechanism of action of these medications is also relevant to pediatric cancer. Currently, there are approximately 1000 cancer drugs in development for adults. Since not all of these new drugs can (or should) be tested in clinical trials in children, the greatest current challenge in pediatric oncology is to prioritize this multitude of medications for clinical use based on existing data. Various types of existing data can be used for this purpose, including:

  1. Data on the molecular fingerprints of patients with high-risk or relapsed tumors, which are
    now routinely collected in globally active precision oncology programs.
  2. Treatment data for each of these patients whose tumor has such a molecular fingerprint
    and who have been treated based on this with an experimental therapy, for example, as part
    of a so-called single patient use trial.

So far, these data are not centrally and systematically evaluated but are usually analyzed regionally or at most nationally, which unfortunately contributes little to gaining general insights due to the rarity of each individual type of cancer in children. Therefore, a global collaborative approach, such as the one we have chosen with the ITCC Data Integration Platform, is essential for pediatric oncology. To comply with various data protection regulations in different countries, the raw data is not transferred; instead, the analysis algorithms are coordinated and then applied locally to the respective datasets. Such a relatively new and technically challenging approach requires a variety of infrastructural, technical, procedural, legal, and ethical prerequisites, which are to be established in this project. However, they are also utilized as quickly as possible to specifically address academic and industrial inquiries regarding the suitability of new cancer drugs for pediatric cancer. Furthermore, this unique global cooperation allows for the handling of previously impossible scientific questions using over 5000 molecular datasets of pediatric cancer with integrated clinical data. Through the integral participation of the leading consortium for early clinical studies in children with cancer in Europe(Innovative Therapies for Children with Cancer; www.itcc-consortium.org) in this consortium, once the infrastructure is in place, clinical trials for children in Europe, Australia, and Canada can be prioritized and rolled out, thus offering children with previously incurable cancer a new chance. Experienced patient representatives have been closely involved in the conception and implementation of the project from the outset to ensure that the patient perspective is always kept in mind as the guiding principle for the orientation of the platform.