Childhood cancer is fortunately rare. But that makes it difficult to include enough eligible children in a study to ensure reliable outcomes. Because all children with cancer in the Netherlands are treated at Máxima, setting up and conducting often international studies goes faster.
Expertise in one place
The trial and data center (TDC) of the Máxima plays a central role in the design, execution and data analysis of clinical trials. Some 120 experts work together here. As a result, the TDC plays a leading role in Europe in the field of clinical research - from early phase studies and registration studies for new drugs to studies to improve the standard treatment and quality of life of children. This pooling of knowledge and the large number of children in one place make the Máxima an accelerator of pediatric cancer research.
Early-stage studies
In total, more than 149 clinical trials are now underway at the Máxima Center. Of these, about one in four is a so-called phase 1 or 2 drug study. This first looks at the safety and best tolerated dosage of a new drug. Then, the second phase studies whether a treatment has an effect on the tumor. Pediatric oncologists and junior group leaders Dr. Paco Bautista, Dr. Reineke Schoot and Dr. Jasper van de Lugt are working together to expand these studies and find new treatments for children with cancer. Bautista explains, 'Together with colleagues in Europe, we determine which studies are most promising for children with cancer. Researchers and doctors work closely together on this, so that new insights reach the treatment room faster.'
International collaboration
The Máxima is active within the European ITCC network of clinical trials as one of the academic sponsors, coordinating several studies. In doing so, the Máxima also develops new study formats, for example for studies on very rare forms of childhood cancer where, with the participation of a small number of children, reliable research results must be obtained. Dr. Harm van Tinteren is a statistician and co-directs the trial and data center. He says: 'We coordinate closely with the European Medicines Authority (EMA) so that they agree with the innovative forms of study.'
Registration studies for new drugs
To make new drugs available for standard care, registration studies are needed. These studies require a lot of additional information to be recorded. Prof. Dr. Michel Zwaan is a pediatric oncologist, research group leader and co-directs the trial and data center: 'The Máxima Center has now successfully completed several registration studies, including audits by the EMA and the American FDA. For example, the drug bosutinib, for children with chronic myeloid leukemia, was recently approved after a study coordinated from the Máxima.' Sixty hospitals from 19 countries participated in this study. The drug inotuzumab ozogamicin was also approved by two registration authorities - in the United States and Japan - based on study data from the Máxima. Other examples of registration studies at Máxima include the studies on the drugs brigatinib, quizartinib and crizotinib.
Moving forward faster together
At the Máxima, studies are usually conducted in collaboration with international study groups. Within the PedAl consortium, a new phase 1 registration study recently started for children with leukemia, AML and ALL, with a specific genetic abnormality. In this, the menin inhibitor, ziftomenib, is being studied in combination with chemotherapy. The drug venetoclax, already widely used in adults with AML, is also being investigated. This phase 3 study has now been opened in 21 countries. The TDC is coordinating the study from the sponsor role in Europe. It is setting up the study, coordinating the output and analyzing and processing the data obtained.
Thanks in part to the efforts of the trial and data center of the Máxima, children have faster access to promising treatments. And with positive results, those drugs can become available to children worldwide. This way, the Máxima Center brings its mission closer: to cure every child with cancer, with optimal quality of life.