Barcelona, 7 September 2026 – A new European research consortium called EPICA – DIPG (European Paediatric Immunotherapy Trial with Combinatory Advanced Therapy Medicinal Products for DIPG) launched under the Horizon Europe Cancer Mission, has just been launched to evaluate a novel immunotherapy strategy for children and adolescents with Diffuse Intrinsic Pontine Glioma (DIPG), a lethal paediatric brain tumour marked by progressive and irreversible neurological deterioration.
DIPG is the most frequent paediatric brainstem tumour and one of the deadliest paediatric cancers. More than 300 new cases are diagnosed each year in Europe, typically in children around seven years of age. The tumour arises in the pons, a region of the brainstem involved in essential functions including movement, breathing and swallowing, and progresses rapidly.
Its diffuse growth through healthy brain tissue makes surgical removal impossible, while conventional oncologic treatments have proven to be ineffective. Focal radiotherapy remains the principal treatment and can provide temporary symptom relief, but it is not curative. Fewer than 10% of children survive for two years after diagnosis, and fewer than 1% survive for five years.
Together, these strands will address the clinical, regulatory and logistical requirements involved in testing a complex research-stage immunotherapy at hospitals in different European countries.
Built around collaboration
EPICA-DIPG will run for five years and brings together referral paediatric oncology centres, research organisations, regulatory expertise, health technology assessment specialists, communication partners and patient organisations from Spain, Italy and the Netherlands. The recruitment of patients will involve the Pediatric Cancer Center Barcelona (PCCB) at Sant Joan de Déu Barcelona Children’s Hospital, Bambino Gesù Children’s Hospital in Italy and the Princess Máxima Center in The Netherlands.
“EPICA-DIPG is not only asking whether a novel treatment strategy can be tested, but whether the whole pathway around that strategy could be scaled up,” said Principal Investigator Andrés Morales La Madrid, PCCB at Sant Joan de Déu Barcelona Children’s Hospital. “For a rare paediatric cancer such as DIPG, the trial, the monitoring tools, the regulatory preparation and the delivery model all have to be considered together if the project is to generate useful evidence.”
The consortium also includes patient organisations from the three participating countries: Pulseras Candela, Il Coraggio dei Bambini Foundation and Vereniging Kinderkanker Nederland. This means that patient organisations, family representatives and carers will contribute to the design, revision and ongoing assessment of the clinical protocol and patient-facing materials. Their involvement is intended to ensure that the study is understandable, ethically robust and responsive to the lived realities of children and families facing a severe and rare diagnosis.
“Families bring knowledge and perspectives shaped by their experience of DIPG and the realities they face” said Alessandro Cannolicchio, President of the Il Coraggio dei Bambini Foundation. “Their involvement can help EPICA-DIPG better understand the questions and concerns families may have, communicate uncertainty openly and honestly, and ensure that information is shared with sensitivity and understanding when difficult decisions need to be made”.
A pioneering treatment strategy entering first clinical testing
EPICA-DIPG will evaluate research-stage combinatorial immunotherapy that has never been tested in humans in this form. As a Phase I study, its primary purpose is to assess safety. It will also examine feasibility and generate valuable evidence to help guide the next generation of clinical and translational research.
EPICA-DIPG will also identify and validate relevant biomarkers and develop a companion diagnostic approach for minimally invasive, real-time monitoring of immune response and disease burden during the trial.
This diagnostic work can help researchers understand whether immune changes are taking place after treatment and how the tumour may be responding. It also supports a trial design in which treatment, monitoring and interpretation need to function together across centres.
Testing an academic model for advanced therapy delivery
Advanced therapy medicinal products are complex and costly to produce, especially when patient numbers are small and specialised manufacturing capacity is concentrated in a limited number of centres.
EPICA-DIPG will test a model in which advanced therapy medicinal products are manufactured centrally at specialised academic GMP facilities and shipped to participating recruitment centres. This will allow the project to assess whether decentralised patient enrolment can operate reliably alongside centralised production, including at hospitals without their own manufacturing capacity.
EPICA-DIPG is therefore testing a research pathway in which the trial provides the setting for evaluation, the companion diagnostic work helps interpret what happens biologically during treatment, and the manufacturing and logistics model examines whether the approach can function across centres. This approach could also have relevance beyond paediatric oncology, providing valuable insights for the delivery of other therapies against other diseases.
If EPICA-DIPG produces the evidence it is designed to generate, it may help inform later-stage clinical evaluation, future companion diagnostic development, health technology assessment discussions and academic-led models for advanced therapy research in rare paediatric cancers, helping build the foundations for future research options for children and families affected by DIPG.
