Researchers at Baylor College of Medicine and Texas Children’s Cancer Center will be using an immunotherapy approach to treat neuroblastoma. The study design has been approved and a phase 1 clinical trial has received $153,000 in funding from the non-profit organization Solving Kids’ Cancer. Assistant professor of pediatrics – hematology/oncology at Baylor College of Medicine, Dr. Chrystal Louis, will be the principal investigator of the trial starting this summer.
Louis, who also serves at Center for Cell and Gene Therapy at BCM, Texas Children’s Hospital and the Methodist Hospital, explained neuroblastoma in these words:
“Neuroblastoma is one of the most common malignant solid tumors of childhood. Since children with high-risk disease continue to have poor outcomes despite intensive therapy, new treatment strategies are required. Researchers have shown that some patients with neuroblastoma can benefit from immunotherapy.”
Patho-physiology of T cells in tumor degradation:
Previous research studies have provided evidence that neuroblastoma is susceptible to antibodies targeting GD2 antigen (a protein expressed by tumor cells). In addition, cancer cells also respond to the T cell immune responses incited by the tumor vaccines. The teams of researchers were already working on the chimeric antigen receptor (CAR) that increases the strength of T cells to identify the GD2 antigen; thereby signaling the T cells to exert its anti- neoplastic activity.
Louis explained that chimeric antigen receptors have been modified to increase the anti- neoplastic potential of T cells and also lasts longer in the system. In order to ascertain the optimal safety of the therapy, researchers have introduced a safety switch that has the ability to kill 90% of the modified t cells without affecting the normal cells of the body.
Goals of the trial:
The primary goal of the phase 1 trial is to investigate the safety and anti-neoplastic capacity of GD2-CAR T cells. Dr. Louis suggested that study participants who will be able to tolerate the initial doses without developing any toxicity, and will be eligible to receive an additional dose of T cells. Similarly, any patients who experience toxicity due to T cells will be given drugs to activate the safety switch that will target and kill the modified T cells. It is believed by the team of researchers that modified GD2-CAR T cells will help in improving the survival in patients.
Dr. Louis expressed appreciation for the funding provided by Solving Kids’ Cancer in these words:
“The funding provided by Solving Kids’ Cancer has been crucial in allowing our research team to offer this novel clinical trial to patients with relapsed or refractory neuroblastoma”
Scott Kennedy, the executive director of Solving Kids’ Cancer commented:
“Immunotherapy using modified T cells has successfully treated several types of cancer, including leukemia in children. This phase 1 clinical trial will allow children with neuroblastoma to benefit from the same cutting-edge research and offer them a promising new treatment option.”
The non-profit organization strives to improve the survival rate and treatment outcomes of deadliest childhood cancers by providing funding and financial aid to novel clinical researches and devising effective therapies that are least toxic and most effective.