Innovative Therapies for Children with Cancer in Europe

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    for Children with Cancer in Europe
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What is behind the phrase "Innovative therapies"?

Traditional treatment for paediatric solid tumours primarily include surgery, radiation therapy and chemotherapy, used either individually or in combination.


Although generally effective in slowing down the progression of the disease and prolonging a cancer patient’s life, traditional cancer therapies have significant limitations both in terms of efficacy and safety.

  • Surgical removal of solid tumours generally cannot ensure the eradication of all malignant cells, especially when the cancer has metastasised.
  • Radiation therapy often results in severe side effects and has only limited utility in treating widespread metastases.
  • Chemotherapy often provides only temporary relief from most types of solid tumours, and the disease frequently reappears or resumes its progression within months or years after therapy. Moreover, subsequent chemotherapeutic treatment can become less effective as the patient’s tumour cells often develop resistance to the drug regimen over time.

All the therapeutic schemes have been evaluated through many years to assess their efficiency. What we call now “traditional therapies” were before tested as a “new therapy” in previous trials. The majority of children do well with standard cancer treatments. However, when standard treatments fail, an innovative therapy may be beneficial to children and offer hope to patients and families. “Innovative therapy” is defined as when a practitioner proposes to use a new treatment : phase I or II give access to patient to get a new drug, before having it on a market framework.


During the last two decades, a new generation of drugs has become available. They target molecules in cancer cells which are thought to play a role in causing the cell to behave like a cancer cell. The development of these novel targeted drugs is based on a rational approach, using extensive knowledge of the biology of human cancers followed by stepwise development of compounds targeting specific molecules in cancer cells.


Therapeutic agents which more specifically adhere to the tumour cell to block or destroy it without affecting other cells, allow treatment with less severe side effects and/or chronic application in order to control the disease while providing good quality of life. A large number of these new compounds are undergoing pre-clinical evaluation and early clinical development in adults. The challenge is now to select and prioritise among all the new anti-cancer compounds entering clinical development in adults, those which should be developed for paediatrics.