KCA is carrying out several projects aimed at improving best practice in child cancer care. Current practice-improving research projects include:
Pharmacokinetic assessment, aimed at reducing toxicity and enhancing efficacy, in child cancer patients undergoing therapy
Many anticancer drugs have significant toxicity, limiting how they can be used and to what effect. Methods used to calculate drug dosage are generally inadequate, especially in babies, young children and adolescents. By studying the pharmacokinetics (dose v concentration relationship) and pharmacodynamics (concentration v effect relationship) of anticancer drugs, this project aims to provide knowledge that helps clinicians improve drug dosing, leading to optimal therapy with improved efficacy and reduced toxicity.
Development of a KCA Early Phase Clinical Trials Centre for child cancer patients
This project centres on the creation of a new child cancer clinical trials centre, where pharma- and investigator-initiated clinical trials are centrally managed. At the Centre, the pre-clinical capabilities of KCA’s research institute members will be combined with the clinical research skills of its hospital members, to allow research to go ‘from bench to bedside’ (from the lab to the clinic) and back again. The aim is initially to conduct early phase clinical trials designed to evaluate new drugs developed by KCA.
Factors determining success of early phase clinical trials in child cancer patients
There are a relatively small number of children with cancer available to participate in early phase clinical trials and, combined with the expense and difficulty of running trials, this makes it imperative that the drugs selected for trials have the highest chance of success. Currently, it is very difficult to predict which new early-phase anticancer drugs are most likely to be successful and therefore worthy of clinical trials. The aim of this project is to identify which pre-clinical factors determine the success of early phase clinical trials, and provide rational guidelines to help streamline future drug development.
Predictive clinical and genetic factors for the side-effects of chemotherapy in children treated for acute lymphoblastic leukaemia
Acute lymphoblastic leukaemia (ALL) is the commonest childhood cancer and, while children with ALL have a high chance of cure (more than 80%), many suffer serious side-effects during treatment. This project will look for clinical predictors of side-effects, such as genetic polymorphisms, which can be identified before the side effect occurs. If successful, this will lead to clinicians being able to predict which of their patients are at particularly high risk of severe side effects, before treatment begins, and institute preventative measures.
Short telomere syndromes as a marker of treatment toxicity
Telomeres are the short regions of DNA at the end of each chromosome, which protect the ends of the chromosome from deteriorating. Abnormally short telomeres are known to predispose to some cancers, as well as a range of other diseases. This research project will try to establish whether child cancer patients with short telomeres are also prone to treatment side effects, such as hypersensitivity to radiotherapy or chemotherapy, or increased late effects of cancer treatment.
Molecular profiling for factors predicting sensitivity or resistance to specific therapies in relapsed child cancer
Treatment options for children who relapse with cancer are currently very limited. To find effective alternatives for these children, a more detailed understanding of the molecular pathways responsible for treatment sensitivity or resistance of the cancer is required. This knowledge can then be used to target these pathways. This project proposes to carry out ‘molecular profiling’ of individual patient tumours, to identify personalised treatment options (targeted molecular therapies) based on currently available pharmaceuticals.