Poster Presentation Advances in Neuroblastoma Research Congress 2016

The iTHER (individualized THERapy) program; personalized cancer treatment for relapsed pediatric cancer (#223)

Jan Molenaar 1 , Paula de Vree 1 , Monique den Boer 2 , Emmy M Dolman 3 , Bianca Goemans 2 , Rogier Versteeg 3 , Huib N Caron 4 , MIchel Zwaan 2
  1. Prinses Maxima Center for pediatric cancer, Utrecht, Netherlands
  2. Erasmus MC, Rotterdam, Netherlands
  3. Oncogenomics, AMC, Amsterdam, Netherlands
  4. Pediatric cancer, AMC, Amsterdam, Netherlands

Background

A vast amount of clinical effective targeted drugs against genes involved in oncogenic processes are becoming available. In parallel the technical developments in genetic tumor cell characterization have made it possible to analyse the complete DNA sequence, the total mRNA gene expression profile of an individual’s cancer relative quickly. Thereby, a rational selection of targeted drugs matching an individual patient’s tumor characteristics is realistic in a clinically-relevant time span. This personalized cancer treatment approach is being implemented in new trial protocols from pharma as well as academia. Thereby a strong need is generated to systematically perform biological characterization of relapsed or refractory tumors for patients that might benefit from these new trials.

 

Methods and results

Therefore we have initiated in 2016, the iTHER (individualized THERapy) project which aims to realize personalized treatment for children with relapsed or refractory, incurable cancer in the Netherlands including all neuroblastoma relapse patients. Within this program we are currently testing the feasibility of 4 components:

  • Selection and inclusion of patients with relapsed or refractory pediatric cancers for whom no curable options are available.
  • Molecular genomic tumor characterization for events in actionable genes/pathways within 21 days
  • Selection and prioritization of ‘actionable events’ in a Molecular Tumor Board setting.
  • Register the follow-up and potential treatment with targeted drugs.

The feasibility phase will take 2 years which will be followed by a clinical trial phase of 3 years. This should result in full implementation of personalized cancer treatment approach in the clinical setting of centralized pediatric cancer care in the Netherlands. This program is closely linked to similar initiatives in other European countries and will be part of the E-smart ITCC ‘basket’ trial.