New CAR T-cell therapy designed by researchers has shown effective results against neuroblastoma in children. This proof-of-principle study was conducted at the UCL Great Ormond Street Institute for Child Health (GOS ICH) and the UCL Cancer Institute, published in the journal Science Translational Medicine.
Neuroblastoma is a rare form of cancer that commonly affects specialized nerve cells (neuroblasts) in children. Neuroblasts are developmental cells that give rise to neurons - brain cells.
The aggressive form of neuroblastoma affects almost 100 children every year in the UK with a survival rate between 50-60 percent. Current treatment strategies include surgical excision, chemotherapy with a stem-cell transplant, radiotherapy, and antibody therapy.
The researchers explored the efficacy of modified CAR T-cell therapy in Neuroblastoma. It is a type of immunotherapy where the patient's immune cells - T-cells are withdrawn from their blood and manipulated to recognize cancer cells. Though this treatment proves beneficial in blood cancers like leukemia and lymphoma, its efficacy is lacking in solid tumors due to the similarity in cancer antigens with that of normal cells.
This modified treatment was targeted against a particular protein called GD2 which is highly found in all neuroblastoma cells but at minimal levels in normal cells.
The study was conducted on 12 children with relapsed (recurring) or refractory (no response to treatment) neuroblastoma as Cancer Research UK-funded phase I clinical trial. It was observed that the therapy showed a drastic reduction in tumor size of some patients with no adverse effects noted in healthy cells. But the result was only short-lasting.
"New treatments are needed for high-risk neuroblastoma and with more research, we hope to develop this further into a treatment that results in lasting responses and increases the number of patients that can be cured", says Lead author, Dr. Karin Straathof, Research group leader at UCL GOS ICH and Consultant Paediatric Oncologist at Great Ormond Street Hospital NHS Trust.
The evidence thereby promises a potent efficacy of modified CAR T-cell therapy at a sufficient dose. This can help scientists to develop a new cure for an aggressive type of cancer.