NANT News

NANT News

NANT Statement regarding potential loss of NIH indirect funding

 Children battling neuroblastoma and other pediatric malignancies depend on federal funded research for their survival and quality of life. More effective, less toxic treatments are driven by federal investment in basic, translational and clinical science. Cancer is the leading cause of death by disease in children and we owe it to young patients fighting for their lives to invest in their future. We call on our leaders in Washington, DC to first “do no harm” to pediatric patients when considering changes to the National Institutes of Health’s (NIH) indirect cost system. Second, we urge policy makers to increase our nation’s investment in childhood cancer research at the National Cancer Institute (NCI) in the Fiscal Year 2025 appropriations process. Our patients deserve nothing less. 

In 2024 about 1,040 children in the United States between the ages of 0 and 14 are expected to die from cancer. This is out of an estimated 9,620 new cases of cancer in this age group and does not include older teenagers and young adults typically treated at children’s hospitals. A single child’s death from cancer means the loss of about 70 life years. Continued research to change these outcomes is the only solution. This is strongly affirmed by the fact that survival of children with cancer has increased from 58% in the mid-1970’s at the time when NCI cancer centers were formed as an integral part of the National Cancer Program to now 85%. We are concerned that the actions of the current administration will slow, halt, or even regress this progress, causing more deaths for children with cancer. 

For example, neuroblastoma is predominantly a cancer of childhood and the most common cancer affecting infants, with greater than 90% of patients diagnosed at less than 9 years of age. Despite substantial advancements in understanding and treating neuroblastoma, it still accounts for 15% of all childhood cancer deaths. The New Approaches to Neuroblastoma Therapy (NANT) consortium was founded in 2000 to accelerate novel therapies for this disease to both improve cure and minimize detrimental late effects of therapy. NIH and NCI funding has enabled fundamental discoveries of neuroblastoma biology and mechanisms of resistance to therapy and translation of these findings into novel therapeutics that have changed the treatment paradigm for patients with neuroblastoma. The rarity of any childhood cancer requires multi-site and even international clinical trial implementation to accomplish advancement. This is an extremely expensive undertaking that requires additional funding from industry or non-profit organizations to complete these transformative clinical trials and the associated correlative laboratory research. It is therefore notable that NIH/NCI-funded NANT research enabled novel discoveries that were then leveraged to successfully compete for necessary additional funding, yielding $20 of additional funding for every NIH/NCI dollar received. Without the ability to compete for these additional funds, advancement of cures and improved survivorship for our patients would not be possible. 

The "STING" trial (Sequential Temozolomide, Irinotecan, NK cells and GD2 mAb) 

is now open to patient enrollment

Chemoimmunotherapy with dinutuximab, temozolomide, irinotecan and GM-CSF has been shown to be active against high-risk neuroblastoma that has returned or not responded to treatment.  The STING study is trying to learn if this treatment can work better by adding Natural Killer (NK) cells. NK cells use the body's own immune system to kill neuroblastoma cells.  However NK cells are only present in the body in small numbers and don't work very well against tumor because neuroblastoma releases chemicals (incdluing TGF-beta) that weaken the NK cells.

The STING trial uses a newer process to make specially chosen donated NK cells which may work better than the patient's own NK cells (called TGF-beta imprinted NK cells).  These donor NK cells may be better killers of neuroblastoma tumor cells because they were exposed to TFG-beta while they are being prepared and grown in numbers in a laboratory. These special NK cells have been collected from donors and stored for use. They will be given to patients after the chemoimmunotherapy infusion is completed.

For more information on this trial, see the NANT 2021-01 page.