Today I talked about paediatric drug development. It’s a very exciting area nowadays because we have reached quite a good survival rate for our children that are diagnosed with cancer but unfortunately we still don’t manage to cure everybody, all the children. Also, there are some high risk tumours which survival rate is very, very low like neuroblastoma, high risk neuroblastoma, or diffuse intrinsic pontine glioma which is a brain tumour. So we want to improve the survival rate for these children and we truly think that new types of therapy and new approaches will be beneficial for these children.
The main message is really that the only way that we can do research in children is collaboration. The number of children that are diagnosed with cancer is quite low because cancer in children is a rare disease and the number of children that relapse is even lower. So we really need to collaborate with the other institutions, the other countries, the other continents in order to increase the number of patients that can access new drugs. We also need to collaborate with the parents and the patients, charities, but also with the laboratories and with industry and the regulators in order to get access to patients and to new drugs.
Could this work change clinical practice?
The point is we have quite a standard treatment for children that are first diagnosed but when they relapse there are really a variety of potential different treatments. So what we are trying to do is trying to standardise the treatment also for relapsed patients and also introducing new drugs into relapse so then eventually, if they work, can be introduced into front line and treat a higher number of patients.
The next step is really to build this platform for paediatric clinical trials which we can, in a dynamic way, introduce new treatments into the same trial, potentially from different pharmaceutical companies that will not compete together but will compete what we call the standard treatment to see whether or not we can have a benefit in a precision medicine approach. So treat a patient who has got a particular mutation activation pathway with their specific drug.
What are the obstacles and potential benefits of this work?
The obstacles are that nowadays we have many drugs, many targets and only a few patients. So really the point is we need to prioritise the list of the drugs according to the list of the patients, of the target in the patients, and we need also in amongst the different drugs of the same class to try to find out which one is the best one that we can use because we don’t have many patients and we cannot afford many clinical trials.