Background: Early-onset colorectal cancer is on the rise in the United States, and many of these cases are due to inherited causes of cancer. The earliest sign of this cancer risk can start in childhood with polyps in the gastrointestinal tract that may develop as early as infancy. These polyps are harbingers of cancer risk and require intensive surveillance and screening of affected family members. Most of the patients with Juvenile Polyposis Syndrome (a specific type of childhood polyposis) do not have a known cause of their polyps or early-onset cancer.

Background: Pediatric gliomas have a relative five-year survival rate of below 30%, and represent the greatest cause of cancer-related deaths in pediatric patients. Current treatments such as chemotherapies, radiation, and/or surgery have limited success, as such, immunotherapy may be an ideal treatment option for these patients. Previously, we have created a form of immunotherapy which consists of immune cells known as T cells. These T cells are engineered to express a chimeric antigen receptor (CAR) which has the ability to seek out and kill tumor cells.

Background: Neurons are nerve cells that populate the brain and are responsible for transmitting chemical signals to regulate several human functions. A deadly form of pediatric cancer, known as medulloblastoma, occurs when these neurons start to proliferate uncontrollably. Developing nerve cells, known as neural stem cells, can transform and get ‘stuck’ at a certain stage in brain development. Medulloblastoma is hypothesized to arise from alterations to the DNA template of such neural stem cells, known as mutations.

Background: Our immune system helps fight off infections, but it also helps prevent us from getting cancer. In order to grow and survive, cancers must find ways to avoid detection by the immune system. Several therapies have been developed that help boost the patient’s immune system ability to fight cancer. Some of the newer forms of these therapies have been  effective in a subset of cancers, resulting in cures for patients previously thought to have terminal disease. However, these therapies have largely been ineffective in solid tumors in children.

Background: Acute lymphoblastic leukemia (ALL) is the most common type of childhood cancer with more than 3,000 children/adolescents under the age of 20 diagnosed with ALL each year in the United States. It mostly afflicts children between the ages of three and five. ALL is a disease that affects a type of white blood cells called lymphocytes that help the body fight infection and disease. ALL is characterized by the presence of large number of immature/non-functional lymphocytes which accumulate in the spleen, bone marrow and lymph nodes.