V Successes

NANT means New Therapy, New Hope

labtour 1By Carole Wegner, Ph.D., Director of Grants for The V Foundation 

Recently, I had the opportunity to meet a remarkable team of scientists, health care providers, patient advocates and a healthy young girl, Cathryn, who survived neuroblastoma and is now not only surviving, but thriving! You would never guess this beautiful, intelligent and charming 10-year-old child was severely ill several years ago.

We met at Children’s Hospital Los Angeles to discuss a program called the “New Approaches to Neuroblastoma Therapy” (NANT), which has provided innovative therapies for over 600 neuroblastoma patients from 2000-2013 who, unlike Cathryn, did not respond to standard therapies.

Dr. Judith Villablanca explains neuroblastoma is the third most common cancer in children. Children who have a particularly aggressive form of the disease have no more than a 50 percent chance of survival. For these children, the standard therapies do not work. Villablanca is the lead investigator and recipient of a $250,000 V Foundation Challenge grant, made to match funds raised by the Carousel of Possible Dreams/Friends of Cathryn fundraiser to fund several clinical NANT trials. The V Foundation funds were raised at the 2014 Dick Vitale Gala in memory of another young girl, Lacey Holsworth, who tragically lost her battle with neuroblastoma.

The power of NANT comes from its organization as a research partnership of 14 hospitals – 13 in the U.S., one in Canada. Many of these organizations are the Top-Ranked Pediatric Hospitals in the Country according to U.S. World News  & World Report for 2014-15. The hospitals work together to find new therapies and enroll patients in clinical trials.  Pre-clinical research data from NANT scientists provides the basis for NANT pilot clinical studies performed in their nationwide consortium. Validated results from these studies  have the potential to become the new standard of care for neuroblastoma. NANT provides a clinical trial incubator for innovative studies so they can be transitioned to mainstream therapies.

My visit to the NANT investigators at the Children’s Center for Cancer and Blood Diseases at Children’s Hospital Los Angeles also included a tour of the lab of Dr. Robert Seeger, one of Villablanca’s scientific collaborators. His NANT research is looking at the use of a patient’s own natural killer (NK) immune cells to boost the patient’s immune system to kill neuroblastoma cells. Experiments by Dr. Hong-wei Wu, a scientist in Seeger’s lab, in collaboration with Dr. Dean Lee and Dr. Laurence Cooper at MD Anderson Cancer Center, demonstrated patient NK cells can be removed, stimulated to proliferate in culture, frozen and thawed without any loss of their power to kill neuroblastoma cells.

Returning NK cells, which are amplified in number and specifically targeted, back to the patient is an immune system boost without the toxicity of chemotherapy. This trial is but one part of the research projects that will be funded by The V Foundation.