Hematologic Malignancies Research

Ongoing Research

Researchers at the Center for Childhood Cancer Research have developed a unique mouse model system to develop new strategies that induce immune-mediated protection against ALL disease progression. 

Researchers at the Center for Childhood Cancer Research (CCCR) are studying perturbations in leukemia cell signal transduction networks for therapeutic intervention in high risk subtypes of childhood leukemias.

Many researchers at the Center for Childhood Cancer Research are involved with clinical trials sponsored by the Children’s Oncology Group.

Researchers at the Center for Childhood Cancer Research are currently evaluating the use of antibody drug conjugates and targeted small molecule drugs as treatments for acute myeloid leukemia (AML) in a Phase III clinical trial.

Researchers are developing and evaluating chimeric antigen receptor (CAR)-modified T-cells to treat children with acute lymphoblastic leukemia (ALL). 

Researchers are testing the efficacy of the JAK1/JAK2 inhibitor ruxolitinib with chemotherapy in a phase 2 clinical trial as a treatment for children with newly-diagnosed ALL.  

Clinical trials are underway at the Center for Childhood Cancer Research to evaluate the use of sirolimus as a treatment for refractory pediatric autoimmune diseases.

Researchers at the Center for Childhood Cancer Research are using zebrafish models to understand the role of MLL in the normal development of the nascent hematopoietic system from its origins in the embryo. 

Providing fellowship trainees with innovative educational materials and experiences may help them to learn differently and augment their engagement in the process. One example includes providing them with on-demand access to educational materials that can help them build and maintain the skillset necessary for the communication of difficult information to families of children with cancer.

Studies currently in progress at the Center for Childhood Cancer Research are evaluating the antiviral agent ribavirin as a targeted agent with lower toxicity than conventional therapy to treat MLL-rearranged infant ALL.

Researchers are analyzing clinical samples and medical outcomes data from AML clinical trials to establish possible molecular links between specific AML germline or somatic mutations and disease progression and treatment responsiveness. 

Identifying the underlying genetic causes of high-risk acute lymphocytic leukemia can provide clues to potential therapeutic targets.

Researchers at the Center for Childhood Cancer Research are creating computer-based methods to collect, analyze and share clinical data to improve pediatric cancer care and treatment.

Ongoing clinical trials are improving survival for pediatric acute lymphocytic leukemia.

Researchers at the CCCR performed chart review for 12 different AEs for pediatric patients enrolled in U.S.-based COG Phase III clinical trials for pediatric acute myeloid leukemia (AML). Results from this analysis showed the current system of AE reporting on cooperative group oncology trials had modest sensitivity and a demonstrable false positive rate.
 

Treating patients with Philadelphia chromosome-like acute lymphoblastic leukemia with a combination of chemotherapy and tyrosine kinase inhibitors may result in higher cure rates.

Researchers at the Center for Childhood Cancer Research are studying the ability of mitoxantrone and related agents to potentiate immune responses to pediatric ALL.  

Studies underway at the Center for Childhood Cancer Research are investigating the role of the DNA cutting enzyme TOP2 in the etiology of both infant leukemias with MLL (KMT2A) gene translocations, as well as chemotherapy-related leukemias with translocations of the MLL gene and other translocations.

Clinical studies conducted by physician scientists at the CCCR have demonstrated that administration of tocilizumab — a recombinant humanized anti-IL6 receptor monoclonal antibody that interferes with secretion of the cytokine IL6 — can be an effective treatment for pediatric patients who develop cytokine release syndrome (CRS) after CART or BiTE therapy.  

Researchers at the Center for Childhood Cancer Research are investigating new immunotherapies using chimeric antigen receptor (CAR)-modified T cells to treat children with relapsed and refractory AML.  

Researchers at the Center for Childhood Cancer Research are investigating the use of immunostimulatory synthetic DNA oligonucleotides that contain CpG motifs (CpG ODNs) to induce anti-leukemia immune responses in pediatric ALL. 

Early stage clinical trials in partnership with the Children’s Oncology Group (COG) are being developed to evaluate the efficacy and safety of a combination of ruxolitinib with standard chemotherapy to treat high-risk ALL with dysregulated Jak /STAT signaling mutations.