Hamburg Center for Child Healthcare Innovation, Translation and Treatment. This is the title of the project that led to the successful application of Kinder-UKE in Hamburg to member of the new center of Child and Youth Health.

The coherence in the scientific scope of HCC.HIIT lies in the strict focus on severe rare and complex diseases and research is well aligned with this overall objective. The core elements are intertwined and research activities are mutually strengthened. Therapeutic innovation originates from an in house drug development program, industrial cooperation and approval trials as well as innovative transplantation strategies.

Immune education provides new targets for therapeutic innovation and addresses immunological aspects around transplantation.

Psychosocial & mental health focusses on intervention for families with children with severe diseases along the difficult journey from diagnosis to successful treatment. Coherence was further strengthened by following the Reviewer’s advice to streamline our application and fully focus on partners at the UKE campus.

Within HCC.HIIT we seek to decipher the molecular mechanisms of disease, we established a complexomics platform and a protein interaction platform, where data are processed using multi-layer interactome analysis. We use our high-throughput drug development platform to purse own small molecule screening campaigns for protein misfolding diseases and prompted approval of the first pharmacological chaperone on the markets. The system is fully scalable, is currently provided to biotech companies for drug reposition projects and will be made available to partners in a future center. These activities are part of the pediatric University Center of Innovative Treatments where we will be running more than 65 clinical phase III and IV trials this year. Among these, 26 gene therapy trials can be offered to patients suffering from untreatable diseases.

As a leading center we perform around 40 single and combined solid organ transplantations per year. 40% of these are new indications in rare inborn diseases. Hematopoietic stem cell transplantation in inborn errors of metabolism constitutes a new life-saving treatment option. More than half of 50 hematopoietic stem cell transplantations per year serve non-malignant indications. Cellular gene therapy uses gene modified stem cells as a permanent source for enzyme replacement or for correction of immune effector function.

Immune education covers imprinted, inborn and inflicted factors. We study the susceptible window from preconception to prenatal life in the PRINCE cohort with 750 families; we perform early diagnosis, provide individualized treatment and run a national registry of 200 patients with hemophagocytic lymphohistiocytosis. We offer cellular therapies, gene therapeutic strategies and pharmacovigilance studies.

The Family Research group provides innovative family-oriented prevention and therapy. We built nationwide networks with more than 40 Partners including health insurances to improve the psychosocial burden of families affected by severe somatic diseases.

UKE harbors the nationwide largest research and care institutions for addictive disorders and gender minorities.

Our research section Child Public Health contributes longstanding experience in developing instruments to assess patient-reported outcomes. We conduct large population-based studies in cooperation with the World Health Organization and the German National Public Health Institute to develop public health and policy strategies.

All these activities foster a holistic life approach and are premises to form a joint research platform with the future German Center of Mental Health.

The 4 core elements are interlinked by 3 cross sectional disciplines.

Healthcare Research thoroughly validates innovative treatment approaches and will help to implement these into routine care.

Systems Medicine uses statistical and machine learning approaches to decipher mechanisms, biomarkers, and novel therapeutic avenues. Semantic data integration will support secure storage and dissemination of the data between partnering sites.

Biometry & Epidemiology consults during study preparation, data collection, and analysis.

What are the most important strengths we can offer to a future center?

The strong expertise in therapeutic innovation for rare diseases covers the whole chain of drug development from basic research over clinical approval trials to commercialization and includes 3 high-throughput platforms. We dispose of a strong record in developing small molecules acting as pharmacological chaperones, in cell bases medicine and we are a leading center for gene therapy approval studies.

Hamburg is one of the leading German and European Centers for Pediatric Transplantation with a particular focus on new indications requiring single and combined transplantations.

Since 20 years we develop successful programs for career and faculty development. The double-armed ped.tracks promotes structured training for pediatric Clinicals and Pediatric Scientists. The Pro.Ps program support junior faculty members to pave the way to professorship. Our experience will foster the foundation of a DZKJ Academy.

Our partner site will most benefit from

Access to large cohorts of healthy children and of those with multifactorial diseases and the opportunity to establish registries where these are not available.

We would like to benefit from and to participate in establishing comprehensive e-health and digital medicine programs. This matches well with our Systems Medicine approach.

The most important benefit for all selected partner sites will be a nationwide network that acts as a true Team. We will center on cooperation, join forces and infrastructures to invent and implement medical innovation with the ultimate goal to improve medical care for children in Germany and beyond. As enthusiastic teamplayers we believe in power of unity and consider this a most promising opportunity.