The main objective of this project is to establish a Data Space for rare cancers (RC) that will make possible the re-use of existing multisource health data (cancer registry data, national registries, data from biobanks etc.) across European healthcare systems leveraging emerging interoperability technologies and AI approaches. The realized "Rare Cancer Data Ecosystem" is expected to improve the quality and the organization of RC patients care, and to increase knowledge on rare cancers advancing health research, so that all patients have equal access to high quality specialist care. The project approach will be experienced in the framework of the European reference network for rare adult solid cancers (EURACAN).

Alzheimer’s disease (AD) is a growing public health challenge in Europe’s ageing population. While Disease Modifying Therapies (DMTs) bring new therapeutic potential, fragmented care pathways, unequal diagnostic access, and healthcare limitations threaten their effectiveness and scalability. The Access AD Project is a collaborative initiative that exemplifies the public-private partnership model, bringing together government agencies and private sector organizations to addresses this unmet need by translating advanced diagnostics and monitoring approaches—such as blood-based and imaging biomarkers, digital and AI tools—into coordinated, equitable, and scalable care across real-world clinical settings in Europe. The project aims to ensure that every individual with AD can access timely, personalized, and cost-effective care, regardless of geography or socioeconomic status. ACCESS-AD builds on local, national, and EU-level efforts to create an integrated, pan-European framework for optimized AD care. The project will achieve: 1) establish robust coordination and governance to ensure efficient delivery and stakeholder alignment; 2) connect and expand fragmented clinical and research data through a shared platform, linking registries and initiatives across Europe; 3) reduce diagnostic access barriers by improving standardized imaging, blood-based, and digital biomarkers for clinical use; 4) develop AI-driven tools to support personalized treatment decisions; 5) generate harmonized real-world evidence on DMTs and emerging therapies; 6) assess health-economic, regulatory, and patient-reported impacts; 7) engage patients, professionals, and policymakers to ensure adoption and sustainability. Aligned with IHI Call 9.2, ACCESS-AD unites and engages diverse healthcare stakeholders and integrates needs and perspectives of individuals affected by dementia to deliver inclusive, connected, and personalized care centered around the AD patient. By translating innovation into real-world practice, the project will reduce inequalities, support health policy, and improve outcomes for AD patients across Europe.

Spinal cord injury is a severe and devastating neurological disorder that leaves patients with permanent paralysis of the body. No treatment is available today to regenerate interrupted nerve fibers and repair the damaged spinal cord. The incidence of spinal cord injury is about newly injured 10’000 people per year in the EU, and due to an almost normal life expectancy more than 200’000 patients are living with a spinal cord injury in the EU. The impact on the individual quality of life is high, and social costs are enormous. Recent preclinical research in animal models succeeded to greatly enhance axonal sprouting, fiber regeneration and neuroplasticity following injuries of brain and spinal cord. These results warrant translation now to patients suffering from acute spinal cord injury. A previous phase I clinical study using intrathecal application of a nerve fiber growth promoting antibody against the growth inhibitory protein Nogo-A has shown in patients with complete spinal cord injury that this treatment is safe and well tolerated. The present study will enroll patients with various degrees of complete to incomplete acute spinal cord injury for a double-blind, placebo-controlled trial to test the efficacy of this antibody therapy to improve motor outcome and quality of life of tetraplegic patients. The enrollment of patients with different degrees of spinal cord injury is considered essential to reveal drug activity and eventual proof of concept in a broad patient population. Advancements in clinical trial design, improved prediction algorithms of clinical outcomes and development of surrogate markers (in cerebro-spinal fluid/serum and by neuroimaging) will allow for scrutinizing the effectiveness of this novel treatment in an unprecedented way. A positive outcome of this trial will represent a breakthrough for the future therapy of spinal cord injuries and beyond (traumatic brain injury, stroke, multiple sclerosis).

Liver cancer in the paediatric population is rare with an incidence approximately 1-1.5 per million population. The commonest tumour seen in the childhood population is hepatoblastoma (HB), usually seen in young children and infants. Much rarer (about 10% of paediatric liver cancers) is hepatocellular carcinoma (HCC), usually seen in the teenage population and sometimes associated with underlying cirrhotic liver diseases. The ChiLTERN project relates to topic PHC 18 ‘establishing effectiveness of health care interventions in the paediatric population’. The ChiLTERN project builds on a unique opportunity to undertake a comprehensive research programme linked to an ambitious global partnership which will see the single largest clinical trial (the Paediatric Hepatic International Tumour Trial - PHITT) ever undertaken in this population of patients, with several randomised questions in six subgroups of patients. ChiLTERN will allow us to move towards an era of personalised therapy in which each patient will receive the correct amount of chemotherapy and will undergo has the best surgical operation (surgical resection or liver transplant). By using both clinical and biological information, we can assign patients more accurately to risk groups based on their survival. Using genetic tests and biomarkers, we will determine those children who may be at risk of developing long term side effects (deafness, heart failure, kidney damage). In addition, biomarkers will allow us to monitor during therapy and detect toxicities early before serious damage is done so that we can adapt treatment and prevent these problems. Finally, we will be using imaging technology tools which will help our surgeons plan liver operations more safely and effectively. Ultimately ChiLTERN will allow us to cure more children with liver cancer, expose fewer children to toxic chemotherapy and ensure their surgery is both effective and safe.