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Boston Children’s Hospital

300 Longwood Avenue
Boston, MA 02115
United States

Mentor Name: Leonard Zon

Boston Children’s Hospital

Choroid plexus carcinoma (CPC) is a rare, aggressive brain cancer arising from the choroid plexus (ChP), a highly complex tissue within the brain's ventricles that produces cerebrospinal fluid. CPC is most often diagnosed in young children, and the only hope for survival is usually total removal of the tumor through surgery. However, this surgery is often complicated due to large tumor size at diagnosis, tumor invasion of nearby brain tissue, and complicated networks of blood vessels in CPC that bleed significantly in the operating room.

Although there have been significant strides made in treating pediatric cancer, a large number of children still succumb to their disease. Moreover, survivors can have detrimental late effects from their chemotherapy and radiation including abnormal growth and development and acquisition of secondary cancers. Therefore, improved and less toxic treatments are urgently needed. A common event leading to pediatric cancer is the breakage of two chromosome and swapping of genetic material between ends.

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.

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