Chronic myeloid leukemia (CML) is a rare form of blood cancer in children. In the Netherlands, a few children receive this diagnosis every year. Bosutinib is so-called tyrosine kinase inhibitor (TKI) and has been used in adults with CML since 2012. Earlier research in mice showed that this drug hinders length growth less than current drugs. Therefore, researchers from Erasmus MC started a clinical study to investigate the effect of bosutinib in children with CML. When the Princess Máxima Center opened in 2018, the researchers involved moved with it to Utrecht.
Bosutinib: a new option
In the study, which involved 60 children from 67 hospitals in Europe and the U.S., bosutinib was found to be similarly effective to other TKIs. Muscle and bone pain were much less common than with other drugs. However, nausea or diarrhea were more common. The comparable effectiveness is important because it allows the choice of a drug with which the child experiences the fewest side effects. This is especially relevant because the treatment often needs to be taken for several years.
Children with CML who are under treatment at the Princess Máxima Center and other European hospitals have access to bosutinib as a treatment immediately upon diagnosis, if reimbursed by the insurer. The approval of bosutinib offers a new treatment option for children with CML, with possibly less impact on their growth and development. The latter is still part of follow-up research, as it requires long-term follow-up.
Prof. Michel Zwaan is a pediatric oncologist, research group leader and co-leads the Trial and Data Center that coordinated the study: 'The approval of bosutinib by the EMA is an important step forward in the care of children with this rare form of cancer. As a pediatric oncologist, I am pleased that we now have an additional treatment option that may better meet the needs of children, with hopefully fewer side effects.'
Collaboration and commitment lead to approval
The approval of bosutinib by the EMA is the result of intensive cooperation between the Princess Máxima Center and Erasmus MC. 'It is very special that an academic clinical study - i.e. not led by a pharmaceutical company - has now led to approval of a drug for children with cancer by the EMA,' says Zwaan. 'It is not often that academic parties coordinate this type of study.'
A committed team in the Trial and Data Center at Máxima set up and coordinated the study: from submission of the study protocol in all countries and inclusion of children, to medical advice during therapy and data collection. The study was conducted in collaboration with the Children's Oncology Group in the United States and was funded by pharmaceutical company Pfizer.