Paediatric oncology and cellular immunotherapy
Clinical research at Ospedale Pediatrico Bambino Gesu includes CAR T-cell therapy and related immuno-oncology approaches for children and young adults with relapsed/refractory acute lymphoblastic leukemia, B-cell non-Hodgkin lymphoma, neuroblastoma, and GD2-positive tumours.
- CD19-directed CAR T cells
- GD2-directed CAR T cells
- Relapsed/refractory B-cell malignancies
- Malignant central nervous system tumours
The oncology programme also extends to minimal residual disease assessment and response-focused treatment strategies in paediatric blood cancers and solid tumours.
Haematopoietic stem cell transplantation in paediatric acute lymphoblastic leukemia
Another major area of interest is the optimisation of HSCT for children, adolescents, and young adults with ALL, including donor selection, conditioning approaches, and management of transplant-related complications.
- Transplant conditioning
- Graft-versus-host disease
- Post-transplant maintenance therapy
- Relapse prevention after HSCT
Research activity in this domain also covers transplantation outcomes in complex donor settings and supportive strategies for reducing treatment-related toxicity.
Paediatric neurology and neurodevelopmental disorders
The sponsor funds studies in bipolar disorder, Down syndrome, and related neuropsychiatric conditions, with a focus on symptom control, cognition, and functional outcomes in adolescents and children.
- Manic symptoms
- Cognitive function
- Neuropsychiatric symptoms
- Developmental disability
This research area includes interest in therapies aimed at behavioural and cognitive manifestations of neurodevelopmental syndromes.
Paediatric cardiology and metabolic kidney disease
Clinical studies also address heart failure in the setting of dilated cardiomyopathy and kidney-related manifestations of Dent disease type 2, reflecting a broad interest in complex paediatric internal medicine.
- Heart rate control
- Cardiac dysfunction
- Renal tubular handling
- Proximal tubule function
These studies involve therapeutic questions in both cardiovascular medicine and inherited renal disorders.
Paediatric infectious diseases and HIV reservoir research
The sponsor is active in research on perinatally acquired HIV infection, with attention to viral reservoir biology, immune control, and treatment interruption strategies in children and adolescents.
- HIV latency
- Proviral reservoir
- Antiretroviral therapy
- Immune response profiling
This work is centred on long-term management of paediatric HIV and the biological basis of persistent infection.
