SJD Barcelona Children’s Hospital is spearheading EPICA-DIPG, a Horizon Europe-backed consortium trialling an investigational combination immunotherapy against diffuse intrinsic pontine glioma (DIPG), a brainstem tumour with survival rates below one per cent at five years. The Phase I study integrates a multinational safety trial, minimally invasive immune-monitoring diagnostics, and a decentralised-recruitment, centralised-manufacturing model – establishing a regulatory and logistical framework potentially applicable beyond paediatric oncology. DIPG is the most common paediatric brain tumour and one of the deadliest paediatric cancers.
More than 300 new cases are diagnosed in Europe every year, usually in children aged between 5 and 10. The tumour originates in a region of the brainstem involved in essential functions such as movement, breathing and swallowing, and progresses rapidly. Its diffuse growth through healthy brain tissue makes surgical removal impossible, whilst conventional cancer treatments have proved ineffective. Focal radiotherapy remains the main treatment and can provide temporary relief from symptoms, but it is not curative.
Less than 10% of patients survive for two years after diagnosis and fewer than 1% survive for five years. The trial framework targets three major obstacles: patient safety, diagnostic precision, and operational efficiency. The immunotherapy combination, previously untested in humans, will undergo initial safety assessment in Phase I. Simultaneously, researchers will refine a minimally invasive diagnostic approach to track immune response and tumour progression in real time. This integrated approach enables coordinated treatment and evaluation across European medical centres. A defining feature is the trial’s decentralised recruitment paired with centralised production.
Specialised drug manufacturing is typically concentrated in select facilities, which may create logistical challenges for rare-disease therapies. By producing treatments at academic centres and distributing them to participating hospitals, the project evaluates whether this model can function effectively for conditions with limited patient numbers. The study’s integrated design, combining clinical testing, diagnostics, and logistical coordination, establishes a model for delivering advanced therapies across Europe. While immediate priorities focus on safety, the findings may guide future research for DIPG and comparable aggressive malignancies.