Childhood Cancers : Rare diseases, but the major cause of death by disease in children over 1 year old

In Europe 12,000 children annually are affected by cancer. Despite high cure rates (ca. 70%), cancer is still a life-threatening disease in children and remains the major cause of death from disease beyond the age of one year: 3000 children die each year from cancer in Europe.
In addition, cure is often only achieved at a substantial "cost" (major organ toxicity, developmental abnormalities and second tumours). These long-term sequelae constitute a significant healthcare burden and can reduce both life expectancy and quality of life for childhood cancer survivors.

There is, therefore, an unequivocal and urgent need for more effective and less toxic treatments to improve both qualitative and quantitative outcomes for children with cancer.

Poor access to innovative therapies for children with cancer in Europe

In the last two decades, a new generation of drugs that target molecules with a role in oncogenesis has become available. The development of these novel targeted drugs is based on a rational approach, from a starting point of extensive knowledge of the biology of human cancers, followed by a stepwise development of compounds targeting specific molecules in cancer cells. A large number of new compounds are in pre-clinical evaluation and early clinical development in adults. Some of them have already been registered for market use.
Access for children to innovative therapies developed by pharmaceutical companies has so far been extremely limited in Europe, one reason being that paediatric oncology does not represent a large, and hence financially attractive, area for drug marketing.

Major European initiatives to enhance drug development in children

The European Commission has recently issued a New Regulation called “Medicines for Children” This Regulation stipulates evaluation in children before a new drug can obtain a marketing registration from EMEA. Moreover, marketing rights will be extended (presumably for at least 6 months) when an appropriate research file is available showing that clinical studies have been performed in children.
Recent similar regulatory initiatives in the US have significantly increased drug research and development in the paediatric population of any type of drugs, including anticancer compounds.
The challenge is now to select and prioritise among all new anticancer compounds entering a clinical development in adults those needing a paediatric development, and then to provide the resources to conduct their clinical evaluation in a timely fashion, with respect to the ethical and medical needs of children suffering from cancer.

Goals of ITCC (Inovative Therapies for Children with Cancer)

ITCC aims to provide a combined biological and clinical drug evaluation programme to select and apply the most promising novel compounds for their use in childhood cancer, with the following objectives:

n To select and prioritise anticancer compounds being developed by pharmaceutical companies for use in adults, that are likely to be active in paediatric cancers, through a comprehensive pre-clinical R&D drug evaluation programme. ITCC has designed a rational mechanism-based strategy for compound prioritisation based on target gene and protein expression profile, target validation and pre-clinical evaluation in relevant in vitro and in vivo models.

Biology and Preclinical Evaluation Programme:

Demonstration of proof of concept through Phase I/II trials of novel agents, by establishing :

♦ a clinical trials network with critical mass (numbers of investigator sites and patients),

♦ access to contemporary technologies (biology, imaging)

♦ use of new methodology and designs.

Clinical and Translational Programme:

Improving information access and ethical aspects of clinical research in children with life-threatening diseases.

Parents, patients and their representatives are involved throughout the project, and the International Confederation of Childhood Cancer Parent Organisations (ICCCPO) is a full-partner of the ITCC Consortium.

ICCCPO initiative

In the Fall of 2006, representatives from parent organizations from the five participating countries (France, Germany, Italy, United Kingdom and Netherlands) met in Amsterdam to discuss how to proceed. A parent committee will be formed within ITCC to work towards the goals that were agreed on at the meeting, e.g. reviewing the protocols, dissemination of information, involvement in the ITCC website and lobbying.

More about ITCC can be found at http://www.igr.fr/itcc

ITCC-Consortium
Contact: Marianne Naafs-Wilstra
icccpo@vokk.nl