Denmark is at the forefront in testing new medicine for children with cancer

​Rigshospitalet's unit for experimental cancer treatment for children and young people, 'Innovative Medicine for Children with Cancer', has been accredited as an 'Early Phase Centre' in a European network for experimental treatment for children and young people with cancer.

​The 'Innovative Medicine for Children with Cancer' research unit has existed since 2014, and is testing and gathering knowledge about new medicine for children and young people with cancer.  The unit and Denmark are at the forefront in testing new medicine for children with cancer and can now initiate even more trials with participants from all over the world.

"As early as two years ago, the unit applied for accreditation, but at that time there were not enough trials and patients in progress for the unit to document its abilities and become accredited," explained Karsten Nysom, a consultant at the Department of Paediatrics and Adolescent Medicine at the Juliane Marie Centre at Rigshospitalet. 

Accredited as an 'Early Phase Centre'

Since then, the unit has initiated several trials and started experimental treatment on a total of 54 children and young people. Half of the patients treated at the unit have been referred from colleagues outside Denmark, particularly the Nordic countries, but also from the rest of Europe. In November 2017, the unit was accredited as an 'Early Phase Centre' by the Innovative Therapies  for Children with Cancer in Europe (ITCC).

"Our team at the research unit and all our colleagues at Rigshospitalet who have helped us achieve this accreditation have proved that we're able to deal with very complex trials of new medicine and international collaboration on testing," explained Karsten Nysom. He stressed that the accreditation is a seal of approval for the unit, which now can participate in even more trials with new medicine for children with cancer. All this will benefit children and young people who cannot be cured by the standard treatment that helps most patients.

First in the Nordic countries

The accreditation means that, of the more than 50 similar centres in Europe, Denmark is now one of just 21 centres that are authorised to conduct Phase 1 trials of new medicine for children with cancer. Rigshospitalet is the first in the Nordic countries to have been accredited.

"There is a great need for testing new medicine for children with cancer who don't respond to the standard treatment," explained Karsten Nysom.

Systematic testing of drugs for adults

"Many drugs have yet to be studied systematically in children and young people," Karsten Nysom said. Many of the drugs being tested for children and young people with cancer have already been approved for adults, but have not been tested sufficiently on children and young people," he said. The drugs have to be tested on children and young people, because they do not always work in the same way as for adults: Cancer diseases and their biology are often different in children compared with adults, and this also applies for digestion of the drugs and the most frequent side effects. 

Karsten Nysom mentioned tazemotostat as an example of one of the drugs being tested at the 'Innovative Medicine for Children with Cancer Unit’ with participants from all over Europe. The recommended dose for adults is 800 mg morning and evening, but the trial showed that children and young people needed twice as much to reach the recommended blood values.  The children absorbed, digested and tolerated the drug differently from adults.

"This shows why testing is necessary on children and young people to ensure the best possible treatment," explained Karsten Nysom. He explained that this result is relatively rare, but no one knows until the drug has been systematically tested in a scientific trial.

About the unit
The Innovative Medicine for Children and Adolescents with Cancer Unit  was established and developed with funding from the Danish Childhood Cancer Foundation and the Danish Cancer Society. The unit is part of the NEXT partnership.

Facts about Phase 1, 2 and 3 trials

Trials of new medicine test drugs which are believed to be effective on a new type of disease or patients based on research or evidence-based treatment from other diseases or patient categories: 

In Phase 1, there is focus on investigating what dose is the best, as well as side effects and the possible effect. 
In Phase 2, focus is on finding indications of whether the drug is working, and on measuring and documenting the effect.
In Phase 3, focus is on finding out whether the new medicine is better than the previous, or whether it may bring something new into the treatment; either on its own or together with other drugs.

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