Researchers Discover Mechanism Behind Deadly Pediatric Brain Cancer

A team of scientists from Texas Children’s Hospital, University of Pittsburgh and Baylor College of Medicine has discovered the primary driver of a deadly pediatric brain cancer, PFA (posterior fossa type A) ependymoma. They identified male sex hormones called androgens as playing a key role in promoting PFA ependymoma growth. 

The study, whose findings appeared in the journal Nature, shows that androgens don’t play a part in the development of any other kind of brain tumor. 

The team’s work shows that when the signaling of these hormones is blocked, the growth of the tumors is blocked. This discovery creates a possibility that treatments could be developed to block these hormones to curb the proliferation of these tumors that are currently untreatable. 

Dr. Jiao Zhang, the co-first author of this significant research, explains that for a long time, scientists have been baffled by what exactly drives the growth of this cancer. He said PFA ependymoma doesn’t have any genetic drivers, so it has been difficult to develop any therapies indicated for this cancer. The team decided to investigate this disease from an angle that hadn’t been explored before in order to get some insights into what is behind the condition. 

They drew on previous research showing that the majority of patients with this cancer are male and the survival of these male patients was, on average, much lower than that of female patients. This pointed to sex differences playing a role in the proliferation of the disease. 

Studies have also shown that the brains of female patients with this disease tend to be more developed when compared to those of their male peers. The researchers therefore wanted to establish what exactly makes boys more likely to develop this cancer. This information could help in developing treatments to boost survival and quality of life of those diagnosed with PFA ependymoma. 

They used animal models and lab-grown human cancer cells to investigate whether chromosomes or sex hormones played a part in disease onset and proliferation. Their work revealed that chromosomes don’t contribute to disease onset and neither do female sex hormones. 

It is notable that in human brains that are growing normally, the cells of this cancer in people with PFA ependymoma tend to be less developed in boys and therefore primed to grow, unlike in females, where the cancer cells are more developed. Their tests showed that when boys with these cells receive androgen supplementation, the cancer cells grow and multiply rapidly. 

The team concluded that early exposure to androgens promoted this cancer’s growth and administering treatments which inhibit these androgens could slow tumor progression. 

With more companies like CNS Pharmaceuticals Inc. (NASDAQ: CNSP) engaged in work geared at developing treatments for primary brain tumors in pediatric and adult patients, we can expect more eye-opening insights about these malignancies to come to light. 

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