This board originated from the idea that an international platform of experts that facilitates knowledge sharing about disease, actionable events, and available clinical studies leads to better prioritization of matching therapeutic options to the treating physician, and subsequently to increased patient referral to targeted therapy. Eventually, they successfully set up a complete international pediatric network. From its start, the initiative was funded through Oncode’s Clinical Proof of Concept (CPoC) programme. The base-laying set-up of the project is now finalized, and with the additional funding it has received, it is ready to continue into its execution phase.

Pediatric patients with high-risk relapsed/refractory hematological malignancies have a dismal prognosis. Only 15% of these patients receive targeted therapy. That is due to both the diversity and complexity of underlying genetic lesions. It is also because of a limited number of available clinical trials and the low numbers of patients per country, and the complexity involved in choosing the best trial for the patient.

Through an Oncode-funded clinical Proof-of-Concept project, the team at the Princess Máxima Center initiated the International Leukemia Target Board (iLTB) - as a prospective multi-center study for targeted treatment advice for relapsed / refractory pediatric hemato-oncology patients. They developed the iLTB in collaboration with the Innovating Therapies for Children with Cancer (ITCC) consortium, and many other European stakeholders involved in the promotion of research and clinical care for pediatric hematological malignancies. Together they explored needs and possible solutions.

At the Princess Máxima Center, the team collaborated with the data managers and legal experts of the Trial and Data Center. Together they built a study database for patient registration, wrote a protocol and governance document, and set up a legal contract with each participating country’s national coordinating center.

In September 2022, the protocol was officially signed, and in December the Princess Máxima Center was the first opened study site. Since January 2023, weekly iLTB patient discussions take place with an international panel of both pre-clinical and clinical experts and in a GDPR compliant environment. Targeted treatment and immunotherapeutic options for refractory and relapsed children with hematological malignancies from all over Europe are being discussed. The potential treatment options are discussed by the experts panel and the treating physician. In parallel, a phase I/II clinical trial with multiple new agents (targeted treatment and immunotherapeutic) arms for hematological malignancies (HEM-iSMART) was developed and submitted to the regulatory authorities.

The CPoC study enabled the Máxima team to engage and start the international platform in a sustainable way, including a registry that enables to follow up patients, document treatment decisions, and clinical outcomes. The next phase of rolling out the iLTB in many European countries has now recently been secured by additional funding from the Fights Kids Cancer (FKC) foundation, which also includes funding to start up the HEM-iSMART clinical study for which the iLTB serves as an entry point.

Monique den Boer says: “Improving clinical outcome for children with cancer requires international collaboration due to the limited number of patients in each country. Oncode made it possible to think ‘bigger’ and to reach out to our international peers with a well-thought concept to realize the iLTB’.”