Dr. Bianca Goemans and Dr. Karin Langenberg, pediatric oncologists and researchers, are involved in the iTHER (individualized THERapy) study that has been ongoing for the past five years. Bianca Goemans says: ‘This study involved children with a cancer diagnosis that is difficult to treat, so for which there is no standard treatment available. We wanted to map the DNA and RNA of cancer cells. If you know exactly what that looks like, you can find leads for a targeted medicine. We call this precision medicine.’
Results
This study has shown that these DNA and RNA analyses are possible and that in the majority of patients (83%) changes were shown in their cancer cells that could potentially be used as a target for treatment. Bianca Goemans: ‘Unfortunately, few patients were ultimately treated with such a targeted medicine, partly because the medicine was often not (yet) available for children or because children were already too sick to participate in a clinical trial. That's why we want to do these analyses earlier in the course of the disease so that children may still qualify for a targeted treatment.’
More clinical trials
Karin Langenberg: 'The pediatric oncologist now looks at the tumor in greater detail and can therefore offer even more accurate treatment in consultation with parents and child. For children with tumors that are very difficult to treat or with tumors that have recurred, we now know earlier which studies with new medicines are appropriate. We hope to be able to offer these clinical trials while the patient is still in good clinical condition. In addition, we can develop new trials with targeted drugs on the basis of the findings.'
'In any case, the most important result after the iTHER study is, that DNA and RNA analysis of the tumor will now be available for all newly diagnosed patients at the Princess Máxima Center,' Bianca Goemans concludes. 'The study was successful because we were able to put the results of our research into practice, in order to improve treatment plans and make them more precise.'
European collaboration
In collaboration with several European countries, the iTHER study demonstrated that disease-free survival improves in certain groups when treated with the targeted drugs. Karin Langenberg: 'In addition, in 16% of patients, cancer-related DNA abnormalities were also found in the healthy cells. These can be important not only for treatment, but also for early detection of a new cancer in the child or family members. In the iTHER-study a start was made on testing tumor cells for their sensitivity to drugs, in a follow-up study we are going to accelerate this research (which previously took weeks to months).'
One thing is for sure, both pediatric oncologists say: 'The great thing of iTHER is that it is not specifically relevant to one group, but to áll children with cancer.'
The scientific article can be found here: Langenberg KPS, et al. Eur J Cancer. 2022 Nov;175:311-325. Implementation of paediatric precision oncology into clinical practice: The Individualized Therapies for Children with cancer program 'iTHER'