PressRelease
“Made-to-measure”drugs and therapies for children
An international symposium in Florence to discuss research in paediatrics
The Meyer Children’s Hospital at the forefront in surgery for treating epilepsy
Monday 16 March 2015 – Florence is the world centre of research in paediatrics thanks to the international symposium entitled “Clinical research in paediatrics”, organised by the University of Florence, the Meyer Children’s Hospital of Florence and the Paediatric Ethics Committee of the Regione Toscana, and sponsored by the Fondazione Internazionale Menarini.
Under discussion were the new European regulations concerning clinical research, especially in the field of paediatrics, and the modern surgical procedures for treating epilepsy.
As far as research is concerned, the drugs used today in children are often administered without having undergone any specific registration procedures, and are only based on studies conducted on adult patients. It is therefore important to carry out clinical research in paediatrics. “In Tuscany a paediatric ethics committee has been set up as a section of the single regional committee. This is a signal that the Regione is sending out in research in paediatrics”, explains Alessandro Mugelli, President of the Paediatric Ethics Committee of the Regione Toscana and professor of the Department of Neuroscience, Pharmaceutical Area and Health of Children at the University of Florence.
“The implementation of research in paediatrics is currently faced with numerous problems because we are dealing with a fragile population that requires painstaking attention. The result however, is that currently half of the pharmaceutical products that are used in children have not been studied in patients of this age”, continues Mugelli. “The purpose of this convention is to encourage research in paediatrics, both by companies and independent bodies, for creating the conditions so that this research can be conducted in the best possible manner, in view of being able to generate valid pharmaceutical products and treatments for taking care of such an important age bracket of our population”.
Also intervening, Luca Pani, Director of AIFA, the Italian Medicines Agency, who illustrated the changes that research must undergo in paediatrics in the near future. "The new structure of the paediatric regulations at a European Level regarding clinical trials will become fully operational as of 2014 but it has already been active since July 2014. Among the innovations, special attention is paid to paediatrics, especially the so-called PIPs (paediatric investigation plans), a change in the role of the ethics committees, with the presence of a representative of the civil society and total transparency, because all the material goes on line and can be consulted free of charge”, explains Pani. “We must not forget that paediatrics foresee numerous precautions and advanced skills, similar to those required for special studies such as those on orphan drugs or cutting edge treatments, like the biotechnological ones. In fact, the whole world is striving to increase research in paediatrics and we are observing efforts made in countries everywhere, from Europe to the USA, from South America to Japan and China, because we need to have standardised regulations and to pay special attention to children, also for apologising and repaying them for all these years during which exclusion criteria have been applied in clinical research for subjects under the age of eighteen”.
As regards clinical practice, on occasion of the symposium the results reached in the diagnosis and treatment of epilepsy were presented, starting from the definition of the so-called phenotypes, that is, the clinical symptoms linked to a specific genetic cause. “It is important to establish exactly when this process is triggered in order to intervene with targeted therapies that are able to stop the involutive phenomenon”, explains Renzo Guerrini, Director of the Operating Unit Complex of the Meyer Clinic of Paediatric Neurology. “It is also essential to make an early diagnosis in the aim of preventing the onset of other cases in the same family, or if necessary, give precise instructions to families at the risk of recurrence”.
Also at the symposium, Flavio Giordano, Neurosurgeon at the Meyer Children’s Hospital of Florence presented the results achieved in the surgical treatment of epilepsy. “Surgery for epilepsy consists of performing operations to remove the parts of the brain which are responsible for forms of epilepsy that are not sensitive to pharmacological treatment. The purpose of this type of surgical procedure is to remove not only the diseased parts of the brain, like for example in the case of cancer diseases, but it also aims at re-establishing the normal functioning of the brain", explains Giordano. “To date, over two hundred surgical procedures have been carried out in Florence and the results of resection surgery are very positive. Pure resection procedures can be performed, that is, the actual removal of the epileptogenic areas. In these cases it is possible to remove the whole section of the malformation that causes the attacks, and disappearance of the epileptic symptoms is achieved in 70-80% of the children operated. Otherwise, more complex procedures can be resorted to for disconnecting parts of the brain, as well as neuromodulation procedures in which the tissue is not removed, and instead devices are applied for delivering current at an intracerebral level in an attempt to remodulate the epileptogenic circuits that fail to respond to the disease. This is palliative surgery that is used when resection surgery is not possible because the malformation is too large, bilateral, or involves zones that cannot be sacrificed, such as the language or motor areas. However, even when removal is not complete, thanks to a series of intrinsic mechanisms in these malformations it is still possible to observe a “shutdown” of epilepsy and significant improvements are also achieved in these cases. It is vital to remember that the sooner surgery for epilepsy is performed, the longer the results will be maintained over time and the fewer the relapses, with the result that the child’s psychomotor development becomes stable”.
Lastly, it must be pointed out that the greatest number of these interventions are performed with the highest specialisation in Florence, and only in 3-4 other centres in Italy, and for this reason each year the Meyer Children’s Hospital in Florence accepts dozens of small patients from other regions.
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