Baylor College of Medicine

Baylor College of Medicine

6621 Fannin

Houston, TX 77030

United States

MYCN-induced molecular clock disruption and metabolic rewiring drive neuroblastoma

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.

Development of allogeneic off-the-shelf gamma delta T cell product for pediatric AML

Acute Myeloid Leukemia (AML)

Acute myeloid leukemia (AML) is the second most common type of leukemia in children and remains very deadly in patients with treatment-resistant disease. A novel type of therapy where patient’s infection-fighting cells (“T cells”) are engineered in a laboratory with a cancer-fighting protein called a chimeric antigen receptor, or a CAR, has shown great promise in patients with another type of leukemia that arises from B cells.

Systematic screening of receptor pairs combinations for improving solid tumor cancer immunotherapy

Neuroblastoma, Osteosarcoma

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).

Translating sarcoma antigen-directed co-stimulatory NK cells to the clinic

Ewing Sarcoma, Rhabdomyosarcoma

Despite attempts to improve treatment over the last few decades, children with advanced sarcomas of the muscle or bone continue to die from their disease. New treatments are sorely needed. Therapies that utilize the immune system to fight cancer, such as white blood cells that are engineered to target specific proteins on tumors, have shown promise in the lab. Their ability to cure patients, however, has been limited by a powerful tumor environment that inhibits the immune system.

Newborn Screening for Early Childhood Cancer Predisposition Syndromes

Brain Tumors, Atypical Teratoid/Rhabdoid Tumor (AT/RT), Glioblastoma Multiforme (GBM), Medulloblastoma, Rhabdoid Tumor, Predisposition, General Pediatric Cancer

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.

Synthetic gene expression regulator switches (SynGERS) expressed in CAR T cells to cure children with solid tumors

Wilms Tumor, Hepatoblastoma, Rhabdoid Tumor, Rhabdomyosarcoma

The immune system can recognize and eliminate cancer. Genetically engineering a unique type of immune cells called T cells can result in upto 90% complete elimination of leukemia cells, a type of blood cancer. Such strategy holds exceptional promise for children with solid tumors. However, additional genetic engineering modifications are required to overcome the solid tumor microenvironment which can stop the engineered T cells. We developed a novel system called “Synthetic Gene Expression Regulator Switches” – SynGERS.

Alex's Lemonade Stand Foundation for Childhood Cancer