Leukemia and lymphoid malignancies
Research activity includes acute myeloid leukemia, acute lymphoblastic leukemia, B-cell acute lymphoblastic leukemia, mixed phenotype acute leukemia, and relapsed or refractory T-cell and B-cell lymphoblastic lymphoma. The portfolio also covers infant leukemia, FLT3-ITD-positive AML, KMT2A-rearranged ALL, and molecularly defined subgroups of pediatric hematologic malignancies.
- Acute myeloid leukemia and relapsed/refractory AML
- Acute lymphoblastic leukemia and infant ALL
- Relapsed/refractory lymphoblastic lymphoma
- Molecularly targeted leukemia subtypes
Interest extends to CAR T-cell therapy, molecularly guided treatment, WT1-directed vaccination, and infection prophylaxis in children with leukemia.
Neuro-oncology and central nervous system tumors
The sponsor supports studies in diffuse midline glioma, including diffuse intrinsic pontine glioma, as well as other recurrent primary malignant CNS tumors and craniopharyngioma. This includes work in children and young adults with newly diagnosed, progressive, or recurrent disease.
- Diffuse midline glioma
- Diffuse intrinsic pontine glioma
- Recurrent malignant CNS tumors
- Craniopharyngioma
Research themes include targeted oncology agents, radiation-associated treatment strategies, and tumor tissue validation in pediatric brain tumors.
Neuroblastoma and other solid tumors
Clinical research focuses on neuroblastoma, high-risk neuroblastoma, and additional pediatric solid tumors, including inflammatory myofibroblastic tumor and ALK-positive malignancies. Imaging and therapeutic studies address disease detection and tumor-directed treatment in these settings.
- High-risk neuroblastoma
- ALK-positive ALCL
- Inflammatory myofibroblastic tumor
- Other pediatric solid tumors
Key areas include GD2-targeted approaches, molecular imaging, ALK inhibition, and neuroblastoma lesion detection.
Precision oncology and molecularly targeted therapy
The research portfolio includes pediatric cancers with actionable alterations such as JAK-STAT pathway changes, MAPK/SRC pathway alterations, ALK abnormalities, and KMT2A, NUP98, or NPM1 rearrangements. The sponsor also supports studies in FLT3-ITD-positive AML and molecular stratification for relapsed or refractory disease.
- Pathway-directed leukemia and lymphoma therapy
- ALK-targeted treatment
- FLT3- and menin-directed approaches
- Genomically defined pediatric cancers
Areas of interest also include pharmacokinetics, tolerability, and biomarker-linked treatment selection.
Supportive care, infection, and hyperinflammation
Beyond tumor-directed therapy, the sponsor funds studies on chemotherapy-induced nausea and vomiting, antimicrobial prophylaxis, sepsis, and hyperinflammation in pediatric oncology patients. These studies address complications encountered during intensive cancer treatment and critical illness.
- Delayed chemotherapy-induced nausea and vomiting
- Infection prophylaxis in AML
- Sepsis in pediatric cancer
- Hyperinflammatory complications
Additional interest includes supportive care optimization during hematopoietic cell therapy and intensive pediatric oncology treatment.
