Alex's Lemonade Stand Foundation for Childhood Cancer

Neuroblastoma (NB) is one of the leading causes of pediatric cancer mortality. Despite advances in therapy, the survival rate for relapsed NB patients is dismal. MYCN is a major genetic driver for NB disease progression and relapse. However, targeting MYCN directly remains challenging, and novel therapeutic approaches against MYCN warrant in-depth investigations. Our lab has shown that MYCN profoundly rewires the way NB utilizes nutrients--specifically lipids--to drive oncogenesis. This function is effectively opposed by a core component of the circadian clock called BMAL1.

Cancer remains a top cause of death for children in the United States despite significant advances in treatment options and effectiveness. Even children who are cured face long-term health problems from their treatments including chemotherapy and radiation, which both damage healthy cells as well as cancer cells. However, a new approach called cancer immunotherapy uses the body's own immune system to fight cancer, specifically targeting cancer cells and leaving healthy cells unharmed. One type of immunotherapy uses a unique protein known as a chimeric antigen receptor (CAR).

Most newborns in the United States undergo "heel-stick" testing within a few days of birth. The blood collected is for newborn screening (NBS), detecting diseases important to diagnose early; cancer risk is not currently part of NBS. Children with a cancer of the retina, retinoblastoma, often have an abnormal copy of a gene called RB1, which can be detected in a heel-stick test. These children, or "carriers," have a 90% or more chance of developing retinoblastoma, usually in both eyes and in the first years of life.

NUT carcinoma is an aggressive cancer with a devasting prognosis. It is almost always fatal, with survival measured in months to – at best – two years. This cancer can be diagnosed in children of all ages – from infants to teenagers. NUT carcinoma tumors most often occur in the chest, but can also be found in the head, neck, bones, or soft tissue. NUT carcinoma tumors are caused by a genetic event in which two chromosomes break in half, swap halves, and then fuse back together. This results in a gene fusion that adds a segment of the protein “NUT” to a segment of the protein “BRD4”.

A deadly complication known as hematopoietic stem cell transplantation-associated thrombotic microangiopathy (HSCT-TMA) can occur after bone marrow transplant, a curative therapy for malignancy. HSCT-TMA results in injury to small blood vessels throughout the body, preventing red blood cells from delivering oxygen to tissues. This can damage multiple organs, especially the kidneys. The cause of the disorder is unknown and there are limited effective drug therapies, making diagnosis and treatment difficult.