SJD Barcelona Children’s Hospital is spearheading EPICA-DIPG, a Horizon Europe-backed consortium trialling an investigational combination immunotherapy against diffuse intrinsic pontine glioma, 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.
The first-in-human EPICA-DIPG trial is aimed at tackling diffuse intrinsic pontine glioma (DIPG), an aggressive and fatal paediatric brain tumour. It will evaluate a combination immunotherapy and the system required to produce, transport, administer and monitor it across centres in different countries.
SJD Barcelona Children’s Hospital will lead, through the SJD Pediatric Cancer Centre Barcelona, a European research consortium called EPICA-DIPG (European Paediatric Immunotherapy Trial with Combination Advanced Therapy Medicinal Products for DIPG), supported by the Horizon Europe Cancer Mission, to evaluate a novel immunotherapy strategy for children and adolescents with DIPG.
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 brain-stem 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. Fewer than 10 per cent of patients survive for two years after diagnosis and fewer than 1 per cent survive for five years.
In this context, EPICA-DIPG brings together three interconnected areas of work: a multinational Phase I clinical trial; a complementary diagnostic approach for the minimally invasive monitoring of the immune response and response to treatment; and an accessible manufacturing model for the cross-border production and delivery of advanced therapies.
Together, these strands will address the clinical, regulatory and logistical requirements involved in testing a complex investigational immunotherapy in hospitals across different European countries.
A pioneering treatment in early-stage clinical trials
EPICA-DIPG will evaluate an investigational combination immunotherapy that has never been tested in humans in this form. As a Phase I study, its primary objective is to assess safety. It will also examine feasibility and generate valuable evidence to help guide the next generation of clinical and translational research.
It will also develop and validate a complementary diagnostic approach for minimally invasive, real-time monitoring of the immune response and disease burden during the trial.
This diagnostic work may help researchers understand whether immunological changes occur following treatment and how the tumour may be responding. It also supports a trial design in which treatment, monitoring and interpretation must work together across different centres.
An academic model for the delivery of advanced therapies
Advanced therapy medicines are complex and costly to produce, particularly when patient numbers are low and specialised manufacturing capacity is concentrated in a limited number of centres.
EPICA-DIPG will trial a model in which advanced therapy medicinal products are manufactured centrally at specialised academic facilities and dispatched to participant recruitment centres. This will enable the project to assess whether decentralised patient recruitment can yield good results alongside centralised production, even in hospitals without their own manufacturing capacity.
For this reason, this trial serves as a framework for evaluating diagnosis, treatment, and also the manufacturing model and logistics between centres. This approach could also have relevance beyond paediatric oncology, providing valuable insights into the administration of other therapies for other diseases.
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