Dana-Farber Cancer Institute

Dana-Farber Cancer Institute

44 Binney Street

Boston, MA 2115

United States

Understanding and Preventing Healthy Tissue Damage in Pediatric Cancer Patients

Background

Treatment of human cancers with chemotherapy or radiation has led to the successful eradication of malignancies in millions of patients, yet the cell death induced in healthy tissues drastically limits the use of these crucial therapies. This is especially true in pediatric patients who experience more damaging side effects from treatment. For example, children with many types of cancers are commonly treated with doxorubicin, which is an extremely effective anti-cancer agent and cures many patients.

Translating BET Bromodomain Inhibitors for Acute Myeloid Leukemia Therapy

Leukemia

Background

Acute myeloid leukemia (AML) accounts for 20% of childhood leukemia and has a long-term survival of only 50%. Thus, new therapies are urgently needed. A new class of drugs, BET bromodomain inhibitors, target proteins that regulate the architecture of DNA and hold significant promise for treatment of pediatric leukemia. At the same time, the relevance of altered metabolism in AML has also come to the forefront. Much work is still needed to effectively implement these discoveries in the clinic.

SETD2 Mutations in Prognosis and Chemotherapy Resistance in Relapsed Acute Lymphoblastic Leukemia

Leukemia

Background

Despite substantial improvements in the treatment of pediatric acute lymphoblastic leukemia, relapse still occurs in 15% of children and 50% of infants, with particular subgroups at substantially higher risk. In contrast to newly diagnosed ALL, patients with relapsed ALL have substantially worse rates of long term survival, reflecting the acquisition of or selection for leukemia cells with chemotherapy resistance.

Mechanisms of CNOT3 Tumor Suppression in T-ALL and Identification of New Drugs That Specifically Kill CNOT3-Deficient T-ALL Cells

Leukemia

Background

Acute Lymphoblastic Leukemia (ALL) is the most common leukemia among children making up 80% of all childhood leukemia cases. Among acute leukemia, T-cell acute lymphoblastic leukemia (T-ALL), which accounts for about 15% of pediatric cases, is aggressive hematologic tumors arising from progenitor cells that meant to become T cells. Understanding the biology of T-ALL may identify new targets for T-ALL therapy and open the way for the development of new drugs.

Analysis of the Deviations in the Diagnosis and Treatment Trajectories of Children with Cancer in Low-income Countries and Their Impact on Outcomes: A Comparative Study Between US and Guatemala

Rhabdomyosarcoma

Reactivating Apoptosis in Refractory Pediatric Cancer by Molecular Inhibition of MCL-1

Leukemia, Lymphoma, Neuroblastoma

Background

New treatments are urgently needed to combat relapsed and refractory pediatric cancers. A specialized group of proteins, called the BCL-2 family, regulates the natural balance between cellular life and death, ensuring that essential cells are preserved and damaged cells eliminated.

Alex's Lemonade Stand Foundation for Childhood Cancer