Children’s Hospital Los Angeles


Grant Amount: $61,416

Immunotherapy with Natural Killer Cells for Treatment of Neuroblastoma

Neuroblastoma is the most common extra-cranial solid tumor in children. Patients with high-risk features continue to have an expected survival of 50% despite intensive therapy.  The addition of immunotherapy utilizing the recently FDA approved anti-GD2 mAb ch14.18 Unituxin has improved outcome.  However, long-term disease free survival remains a significant problem and improving immunotherapy further is needed for complete disease eradication of disease.  Adoptive immunotherapy with natural killer (NK) cells has emerged as a promising anti-cancer treatment with broad anti-cancer potential. Until recently, the clinical efficacy and effective application of NK cell immunotherapy has been limited by the inability to obtain sufficient cell numbers for adoptive transfer. We have developed a novel expansion method for NK cells that solves this problem using artificial antigen presenting cells to express stimulatory molecules that promote sustained proliferation of NK cells in the laboratory. This expansion can be performed from blood from a simple venipuncture that can then be sent to the central laboratory. This phase 1 study will be the first study to combine cellular therapy with Unituxin in the treatment of patients during which we will identify the maximum tolerated dose of NK cells to be given with Unituxin and determine the immune effects of the combination therapy for patients with neuroblastoma. It will also be the first multi-center pediatric trial with cellular therapy and will help pave the way for other cellular therapy trials in pediatrics. The trial will be conducted through the NANT consortium (NANT.org), a multi-institution consortium (centered at CHLA) whose goal is identify new therapies for patients with relapsed and refractory neuroblastoma and to improve their outcome.  If successful, this therapy could then be utilized for all high-risk neuroblastoma patients to eradicate residual disease.  The cost of this trial exceeds $1 million, but funding from Phase One foundation would be used as a component of the budget.