The most common type of leukaemia in children is acute lymphoblastic leukaemia. Thanks to many years of dedicated medical research, the cure rate for acute lymphoblastic leukaemia today is now around 85%. Our aim is to increase this to 100%, while at the same time reducing the side effects of treatment.
Key to achieving this aim is having the right tools in the laboratory – tools that not only allow us to test new treatments against cancer cells, but give as good an indication as possible that these treatments will work in children. This is where laboratory models of disease come in.
Our laboratory model of acute lymphoblastic leukaemia
Over a number of years, Professor Richard Lock and his Leukaemia Biology team have successfully developed a laboratory model of acute lymphoblastic leukaemia which closely mimics the disease in children – more so than any other model developed elsewhere.
Our living model, which is based on specially bred mice growing human leukaemia cells, is highly clinically-relevant, making it possible to carry out meaningful experimental work without involving children. Having a reliable model to test drugs in is really important, because it ensures that only the safest, most effective drugs proceed to testing in children."
- Professor Richard Lock
An internationally-recognised resource
Since 2005, our Institute has been the leukaemia testing site for the Pediatric Preclinical Testing Consortium, established by the USA’s National Cancer Institute. The purpose of the consortium is to conduct preclinical testing of potential drugs for childhood cancer, to identify which of these should be fast-tracked to clinical trials in children.
At our Institute, which is the only testing site outside of the USA, we use our experimental model of acute lymphoblastic leukaemia to evaluate up to 10 new treatment drugs and drug combinations per year.
Testing using our models of leukaemia has led to several drugs being promoted to clinical trials in children. One promising drug to come out of the program is VTP-50469, which our testing showed was especially active against infant leukaemia. VTP-50469 (now SNDX-5613) is now being evaluated in a clinical trial in patients with relapsed or refractory acute leukaemia.
Our testing also showed that the drug OBI-3424 was particularly effective against a very aggressive subtype of leukaemia called T-cell acute lymphoblastic leukaemia (T-ALL), and this drug is currently being evaluated in a clinical trial for relapsed/refractory T-ALL.