Drug target discovered for neuroblastoma, a common cancer in infants

Neuroblastoma is a cancer originating in the nervous system, the most common in children under 1 year of age, accounting for %6 of all childhood cancers. Each year, among children under 15, approximately one in 100.000 is diagnosed with neuroblastoma. Children with this condition are diagnosed at an average age of around 1-2 years. Despite current treatments, long-term survival rates in children with high-risk neuroblastoma are below 50 percent. Research into neuroblastoma has intensified in recent years. In September 2017, Cancer published a scientific study reporting that a new cancer immunotherapy drug target had been discovered for neuroblastoma.

What is neuroblastoma?

Neuroblastoma is a cancer that begins in certain precursor nerve cells while the child is still in the womb (“neuro” refers to nerves, while “blastoma” indicates a cancer affecting immature or developing cells). This childhood disease is a neuroendocrine tumor and causes tumors in the adrenal glands and / or sympathetic ganglia.

The study aimed to enable our immune system cells to target only cancer cells without harming healthy cells by identifying surface proteins associated with cancer cells. Using a new technique they developed, researchers performed RNA analysis on 126 neuroblastoma samples through sequencing and compared them with healthy cells.

According to the study results;

• A total of 296 genes were identified that were active differently in cancer cells than in normal cells.
• The GPC2 (Glypican2) protein was discovered to be one of the essential molecules for the growth and spread of neuroblastoma tumor cells, and therefore potentially a good drug target. GPC2 is one of the important proteins located on the cell surface.
• Researchers designed an antibody to bind to the GPC2 protein and also attached a cancer drug to this antibody. 
• In laboratory analyses using human cell cultures and mouse models, the developed antibody-drug molecule effectively destroyed cancer cells. Normal cells were not harmed.

So what does this study mean for us?

• It is an important development for neuroblastoma, for which treatment options are limited. This study enables the development of treatments that will cause less harm to healthy cells.
• Antibody-drug molecules have begun gaining importance in cancer treatment. By adding an antibody (protein) that recognizes the cancer cell to any chemotherapy drug, only the cancer cell can be targeted.
• This study sheds light on modern genetic treatments such as CAR-T cell therapies. The cell surface protein called GPC2 is an oncoprotein with very high activity in other cancer types as well. Therefore, it may be possible to apply this study’s approach to other cancer types.
• It must be emphasized that this treatment method is still at the experimental stage. Modern medicine uses genetic and biochemical techniques to apply the most appropriate treatment. Cancer treatment is possible only through the means offered by modern medicine.

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