Ospedale Pediatrico Bambino Gesu'

The Ospedale Pediatrico Bambino Gesu' is a leading hub for pediatric oncology, where investigators are testing innovative cellular and molecular therapies to improve survival for children with blood and solid cancers.

• Acute lymphoblastic leukemia and high‑risk B‑cell ALL
• Relapsed or refractory acute myeloid leukemia
• Neuroblastoma and other solid tumors
• Lymphoblastic lymphoma (B‑cell and T‑cell subtypes)
• Anaplastic large cell lymphoma and ALK‑positive malignancies

With 84 investigators across 25 trials, the site emphasizes safety, tolerability, and long‑term outcomes of novel agents such as CAR‑T cells and targeted kinase inhibitors.

Research in this area focuses on rare blood disorders and lymphatic conditions, aiming to evaluate new targeted drugs and gene‑based treatments for pediatric patients.

• Chronic myeloid leukemia (CML) in children
• Severe hemophilia A requiring factor replacement
• Beta (β)‑thalassemia (transfusion‑dependent and non‑transfusion‑dependent)
• Refractory systemic autoimmune diseases with hematologic involvement
• Proteinuria associated with lymphatic disorders

The program seeks to define optimal dosing and assess clinical activity while prioritizing patient safety.

The department explores therapies that modulate the immune response, including CAR‑T approaches for autoimmune conditions and novel immunosuppressive strategies.

• Refractory systemic autoimmune diseases (e.g., SLE, systemic sclerosis)
• Steroid‑refractory acute graft‑versus‑host disease
• Anti‑CD19 CAR‑T therapy for B‑cell driven autoimmunity
• CD7‑CAR T cells for relapsed/refractory T‑cell ALL/LBL
• Immune‑related adverse events after prior CAR‑T treatment

Studies aim to improve disease control while monitoring long‑term immune safety profiles.

Clinical investigations target severe epilepsy syndromes and demyelinating disorders, testing both disease‑modifying drugs and adjunctive therapies.

• Dravet syndrome and Lennox‑Gastaut syndrome
• Relapsing‑remitting multiple sclerosis in children
• Adjunctive soticlestat therapy with standard anti‑seizure regimens
• Long‑term safety of novel agents for refractory seizures
• Neurodevelopmental outcomes in treated pediatric cohorts

The goal is to assess efficacy in seizure reduction and disease progression while ensuring tolerability in young patients.

Studies address inherited lipid disorders and renal‑cardiac complications, evaluating long‑term lipid‑lowering and renoprotective agents.

• Familial hypercholesterolemia (heterozygous and homozygous)
• Inclisiran for sustained LDL‑cholesterol reduction
• Finerenone combined with ACE‑inhibitor or ARB in chronic kidney disease
• Proteinuria management in pediatric renal disease
• Cardiovascular risk monitoring in metabolic disorders

Research emphasizes safety over extended treatment periods and impact on cardiovascular outcomes.

The site conducts trials on inherited metabolic and genetic conditions, focusing on enzyme replacement and gene‑based therapies for children.

• Gaucher disease type I and type III
• Hypophosphatasia and related bone metabolism disorders
• Beta (β)‑thalassemia with novel luspatercept therapy
• Early‑onset metabolic syndromes with genetic confirmation
• Neonatal screening follow‑up studies

Objectives include establishing safe dosing, improving quality of life, and evaluating long‑term disease control.

Research targets metabolic bone disease, iron‑related disorders, and nutrition‑linked conditions, aiming to enhance therapeutic options for pediatric patients.

• Hypophosphatasia treatment with ALXN1850
• Beta (β)‑thalassemia management with luspatercept
• Nutritional support strategies for chronic kidney disease
• Metabolic monitoring in children receiving enzyme therapies
• Bone health outcomes in long‑term metabolic disease management

Studies prioritize efficacy, safety, and functional improvement in daily activities.

Investigations include connective‑tissue manifestations of autoimmune disease and rare dermatologic conditions, testing targeted immunotherapies.

• Systemic sclerosis and related skin fibrosis
• Dermatomyositis with refractory disease activity
• Autoimmune cutaneous involvement in systemic lupus erythematosus
• Novel biologic agents for pediatric connective‑tissue disorders
• Quality‑of‑life assessments in treated cohorts

The focus is on reducing disease activity while monitoring safety in a pediatric population.