Optimising drug treatment for childhood cancer patients

It is particularly difficult to treat some groups of childhood cancer patients, especially infants in their first weeks of life. It can be especially difficult to know how much chemotherapy (anti-cancer drug) to give to these children. Doctors decide what doses of chemotherapy to give without sufficient scientific information. Some patients do not receive enough drug, this reduces their chances of treatment response and survival. Some patients will get too much drug, this can cause serious long-term health problems including major organ toxicity, hearing loss, and in some cases treatment-related death. This impacts on patients and families in a hugely negative way. It also places a heavier burden on the NHS due to increased hospital visits and admissions.

Studies carried out by our research group have established the best way to treat patients with the widely used anticancer drug carboplatin. This involves measuring drug levels/exposure in individual patients and modifying drug doses based on these measurements. We have improved dosing regimens and influenced national treatment guidelines in patient groups where drug exposure is most variable. We established a national research programme to expand this work to include a wide range of drugs for treating infants with cancer. This helps us understand what factors influence drug exposure in children of all ages, and how much drug an individual patient should receive to achieve an effective drug exposure without excessive toxicity. The drug exposure information is used by doctors to support dosing decisions for individual patients.

This application is to fund this rapidly developing research programme for two years, involving the treatment of 150-200 childhood cancer patients nationally. This will benefit patients who are on the study and treated during the lifetime of the grant and will improve treatment for children in the future who will receive the most appropriate drug dosages.