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Boston Children’s Hospital

300 Longwood Avenue
Boston, MA 02115
United States

Acute lymphoblastic leukemia (ALL) is the most frequently diagnosed cancer in children and a major cause of death from disease in this age group. Despite the possibility of a cure, ALL treatments can lead to significant health problems later on. Early detection and understanding of ALL could greatly reduce the impact of cancer in children. Many genetic factors have been identified that increase the risk of developing a specific type of ALL called B-cell ALL, but how these factors contribute to the disease is still not fully understood.

Boston Children’s Hospital
Boston Children’s Hospital

In recent years, researchers have found ways to artificially reprogram our own immune system so that it recognizes specific markers and fights tumor cells. Unfortunately, certain pediatric cancers, such as acute myeloid leukemia (a type of blood cancer), are difficult to treat because the cells that make up the tumor either have the same targets as healthy cells, causing undesired effects on normal organs, or are resistant to elimination by the immune system.

Acute myeloid leukemia (AML) is the second most common childhood leukemia but is reaching acute lymphoblastic leukemia (ALL) as the leading cause of childhood leukemic mortality, primarily because of ineffective prognostic schemas and the absence of targeted therapies. While there have been significant advancements in supportive care and targeted treatments, AML shows a worse long-term prognosis across all groups when compared to other acute leukemias.

Down syndrome is one of the most common genetic disorders worldwide. It affects a relatively high proportion of newborns every year - about 1 out of every 700 babies has Down syndrome in the US according to CDC. This condition causes a spectrum of developmental issues that affect the quality of life of these children. Notably, children with Down syndrome are highly prone to blood disorders and childhood leukemia. They have an approximately 20-fold increase in the risk of overall leukemia, particularly acute myeloid leukemia and acute lymphoblastic leukemia.

Mentor Name: Kristen Long

Mentor: Alan Cantor

Alkylating agents are a class of chemotherapeutics important for children with high-risk or relapsed leukemias. These are given during what we call conditioning regimens just before bone marrow transplantation (BMT). The goal of conditioning regimens is to eliminate all leukemia cells, before the bone marrow is replaced by BMT. However, the failure of these conditioning regimens to ablate all leukemia cells is a major problem that leads to relapse after BMT. Leukemic relapse is the most common cause of treatment failure of BMT regimens, and these patients have a very poor prognosis.


The development of novel targeted therapies for cancer is a crucial priority in the field of oncology. This is particularly true for some types of leukemias that are characterized by a high risk of relapse and poor response to conventional therapies. For these diseases, it is imperative to identify and act on the essential signals that drive the malignant transformation and the survival of cancer cells.

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