The BMJ Awards 2017: Cancer Care

New cancer drugs for children

Overall survival among children with cancer is good, but progress has been slower in those with high risk malignancies and with relapsed or refractory disease. There are serious long term consequences for a substantial number of those who do survive. A decade ago, before a dedicated paediatric drug development unit opened at the Royal Marsden Hospital, very little was being done to tackle these issues.

Drug trials in children have been avoided because of fears about toxicity. These were unwarranted, says Lynley Marshall, consultant in paediatric and adolescent drug development at the trust. “It’s really unusual to see toxicity in children that isn’t seen in adults. Very young children apart, they can tolerate doses as high, or even higher, than adults can.”

This makes it possible to carry out trials in children of drugs already licensed for adults, or moving towards a licence. “Sometimes drugs have been given a waiver from paediatric testing because they don’t seem relevant to children—breast cancer drugs, for example. Children don’t get breast cancer, but the drugs may nevertheless be useful in paediatric tumours.”

Since the centre started, enrolment has risen from fewer than 5 patients a year to 30, and trial design has become more sophisticated. Networking with others in the UK and abroad has multiplied the effort. “Those enrolled have to meet very strict eligibility criteria. This is a fragile population so many of those who come to us are screened out.” The unit has cost around £5.5 million (€6.35m; $6.85m) over its 10 year life—“very good value for an innovative programme whose reach …