Researchers have identified a new target for immunotherapies that could treat and block the advancement of neuroblastoma, which is the most common form of cancer in infants.

Neuroblastoma is a type of cancer that commonly occurs in infants and young children. It affects the development of the nervous system, attacking immature nerve cells as early as the embryonic stage.

Neuroblastoma accounts for approximately half of all cancers in infants, so it is the most common cancerous tumor found in children younger than 1 year old. And unfortunately, despite available therapy, the long-term survival rates for children with high-risk neuroblastoma are currently lower than 50 percent.

Scientists led by Dr. John M. Maris, a pediatric oncologist at the Children’s Hospital of Philadelphia, PA, set out to identify molecules on the surface of neuroblastoma cells that would be a target for more adequate treatments.

The study was led by first author Dr. Kristopher R. Bosse, and the findingswere published in the journal Cancer Cell.

Targeting GPC2 stops neuroblastoma

“Our rationale was to identify a cell-surface molecule that an immune-based therapy could target without damaging healthy tissues,” explains Dr. Bosse.

In order to do this, his team conducted ribonucleic acid (RNA) genetic sequencing. They developed an RNA-sequencing-based pipeline, which they first used to compare RNA sequencing data from 126 neuroblastomas with RNA data from normal tissue.

With this approach, the researchers identified 296 differentially expressed genes, which they further filtered down in order to find “an optimal candidate immunotherapeutic target.”


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