Leukemias are neoplasms of the bone marrow characterized by rapid proliferation of immature white blood cells and their appearance in large numbers in the blood.
Acute leukemias make up almost 97 percent of all childhood leukemias. The most common types include acute lymphoblastic leukemia (ALL) and acute myeloid leukemia (AML). Although chronic leukemias in children are generally rare, chronic myelogenous leukemia (CML) occurs in up to 3 percent of pediatric leukemia patients.
Researchers at the Center for Childhood Cancer Research are using state-of-the-art genomic, immunotherapeutic and targeted molecular approaches to develop new therapies to treat pediatric leukemias and related hematologic malignancies.
The efforts of investigators at the CCCR have resulted in:
- Identification and characterization of molecular targets that can be used to develop new targeted cancer therapies
- Development of pioneering immunotherapies including chimeric antigen receptor (CAR) T-cells to treat ALL, AML and CML
- Clinical trials to evaluate targeted cancer agents and CAR T-cells as treatment options for children with pediatric leukemias
- The use of clinical informatics and outcomes data to optimize treatment delivery and enhance medical outcomes for children with hematologic malignancies
While current treatment regimens for pediatric leukemias can achieve long-term cure rates (60 percent to 70 percent) relapsed/treatment refractory disease is responsible for poor clinical outcomes of a majority of children with leukemia. The ability of researchers at the Center for Childhood Cancer Research to quickly translate laboratory-based discoveries into clinically relevant therapies offers enormous promise for children with leukemias and other hematologic malignancies.