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Current Drug Development Efforts

Current therapeutic options are limited as children are largely treated with older drugs that were approved in some cases 30-40 years ago. These are often referred to as “cytotoxic drugs”, meaning that these act on the mechanism of cell division and kill preferentially fast-diving cells (such as cancer cells).  For this reason, cytotoxic drugs may have serious side effects as they also act on other fast dividing cells such as blood cells, intestinal cells and, generally speaking, on developing tissues.

Children, as developing human beings, are particularly sensitive to these side effects, which may affect their lives even after cure. It is well documented that childhood cancer survivors are likely to suffer health problems later in life, as a result of their current cancer treatments.

Recurrent problems include:

  • Disability/Developmental problems
  • Hearing loss
  • Organ damage (heart, kidney, lung, etc.)
  • Secondary malignancies
  • General loss of lifespan expectancy

These facts underlie a strong need for more effective and safer drugs to treat cancer in children

The introduction of new therapies into the treatment of paediatric cancers may have dramatic effects and hugely improve the outcome for those patients. For example a subset of children affected by Acute Lymphoblastic Leukaemia had historically a very poor prognosis, with survival rates (defined as 3 year event free survival) of approximately 35%. After the introduction of a new targeted drug (Imatinib/Gleevec®) into the protocol, survival rates jumped to 80%. If a similar outcome could be achieved with the use of newer drugs for the other most malignant childhood cancers, the overall clinical improvement, in terms of lives saved, would be huge.

Unfortunately these “new generation” drugs are not systematically evaluated in childhood cancers and when this eventually happens and drugs are approved for paediatric use, it will be several years after approval for adult diseases. Please see the table below for some examples:

Drugs being studied in EU

Approval  date for Adults (EU)

Expected Paed. Approval (EU)













By looking at the epidemiological and clinical statistics it is clear that thousands of lives could be saved if better drugs were available for paediatric use over the time periods above.

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