The approval of the Food and Drug Administration (FDA) means that bosutinib can now be prescribed as the first treatment option for children with CML in the United States. It is still unknown when it will become available in the Netherlands, but the data will be submitted to the European Medicines Agency (EMA) in the first quarter of 2024.
Michel Zwaan, research group leader and head of the Trial and Data Center, is proud: ‘It is very special that an academic clinical study – so not led by a pharmaceutical company – has now led to approval of a drug for children with cancer by the FDA. We hope that bosutinib will also be approved by the European regulator next year, so that all children with CML throughout Europe will have access to this drug.’
Reduced bone growth
Every year, a small number of children in the Netherlands are diagnosed with chronic myeloid leukemia (CML), a form of blood cancer. The treatment consists of a class of drugs called tyrosine kinase inhibitors. These drugs block the growth of the faulty white blood cells. Bosutinib has been used in adults with CML since 2012. A side effect of existing CML treatment is reduced bone growth –a side effect which strongly affects children with cancer, as it limits their height. Bosutinib is also a tyrosine kinase inhibitor. Research in animals has shown that this drug hinders growth less than current treatments. That is why researchers from the Princess Máxima Center and the Erasmus MC started a clinical study of the effect of bosutinib in children with CML.
The study, led by Prof. Michel Zwaan, shows that the efficacy of bosutinib is comparable to other medication approved in children with CML. Children still experienced side effects, especially stomach and intestinal symptoms, but muscle and bone pain were much less common than with current CML drugs. Zwaan: ‘More research into the effect of bosutinib is still needed. We particularly want to continue to follow children to see if the drug is also better for bone growth in the longer term.’
A total of 60 children from 67 hospitals in Europe and the US took part in the study. Based on the results, the researchers have determined the recommended dosage of bosutinib in children. As an important part of the study, various capsule dosages (25 mg and 50 mg) and administration of the drug through a nasogastric tube were investigated specifically for use in children.
Trial and Data Center
The clinical study of the effect and side effects of bosutinib started in 2016 in Erasmus MC. At the opening of the Princess Máxima Center in 2018, the researchers involved moved to Utrecht. A committed team in the Trial and Data Center in the Máxima Center has set up and coordinated the study: from submitting the study protocol in all countries and inclusion of patients, to medical advice during therapy and data collection. The research was carried out in collaboration with the Children's Oncology Group in the United States and the pharmaceutical company, Pfizer.