New Therapies for Childhood Cancer
By Madeline B. Torres M.D.
Arriving at the National Institute of Health (NIH) as a surgical oncology research fellow this summer felt like winning the golden ticket into Willy Wonka’s factory. I entered with high hopes to tackle one of the multiple roadblocks to the treatment of one of the many adult cancers. Little did I know that my path would lead me to investigate therapy options for the treatment of neuroblastoma. Neuroblastoma is the most common solid childhood cancer outside of the brain1, with an incidence of 9.5 cases per million children; that is approximately 700 new cases per year diagnosed in the U.S. alone. Standard of care includes surgery and adjuvant chemotherapy. Recent studies have shown a new promising target for neuroblastoma therapy, Glypican-2 (GPC2), a cell surface oncoprotein that may play a role in neuroblastoma growth and development. In studies by Li et. al. and Bosse et. al., neuroblastoma tissues showed high expression of GPC2 but it was not detectable in normal tissues, making it a promising therapeutic target.
However, neuroblastoma isn’t the only childhood cancer. The five most commonly diagnosed cancers in are: leukemia, brain and central nervous system tumors, neuroblastoma, Non-Hodgkin lymphoma and Wilm’s tumor. There are multiple subtypes of leukemia, the most common are Acute Lymphocytic Leukemia (ALL) and Acute Myelogenous Leukemia (AML), both have a high 5-year survival rate ranging from 80% for ALL and 60-70% for AML. Despite the high 5 year survival rate, some children are resistant to treatment needing more research to develop new treatments. Tisagenlecleucel, is a recent breakthrough in treatment for chemotherapy resistant acute lymphoblastic leukemia (ALL) recently approved by the FDA. Kymriah is a type of Chimeric Antigen Receptor (CAR) T cell therapy, a form of adoptive cell transfer, a type of immunotherapy. CAR T cell therapy is often called a “living drug,” it consists of giving patients genetically engineered T cells (cells are usually obtained from a donor) designed to recognize and kill tumor cells. CAR T-cells therapy unlike Tumor Infiltrating Lymphocyte (TIL) therapy does not require the use of the patient’s own cells to create the medication.
Despite all these advances, scientists continue to work tirelessly to find new therapies for childhood cancers. September is childhood cancer awareness month. This month, I ask that you consider contributing to developing new therapies for childhood cancer. You can call your state representative and ask them to continue to fund childhood cancer research. Over the next two years, I hope to make the best of my time at the NIH and make a small contribution to the treatment of neuroblastoma.
Madeline B. Torres, M.D. is a research fellow in surgical oncology at National Cancer Institute (NCI) in Bethesda, Maryland. She completed two years of general surgery residency at Penn State Milton S. Hershey Medical Center in Hershey, PA. Dr. Torres was born and raised in El Salvador. She immigrated to the United States with her mother and brother at the age of nine. She then went on to obtain her B.S. in chemistry from the University of Colorado at Denver and earned her medical degree from the University of Utah School of Medicine. She became involved with AWS during medical school after working with AWS members Amalia Cochran M.D. and Leigh Neumayer M.D. whom she considers mentors. Her interests include: surgical education, surgical oncology, work-life balance and encouraging women and minorities to pursue surgery and careers in medicine.
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