A developmental and stem cell gene PROX1 regulates rhabdomyosarcoma characteristics and growth

What is it about?

Rhabdomyosarcoma is an aggressive pediatric soft tissue cancer with an unmet need for new and more specific therapy options. Despite having skeletal muscle characteristic, RMS can occur anywhere in the body, including tissues devoid of skeletal muscle. Identification of the main regulators of rhabdomyosarcoma development would provide essential information to develop new therapies. Researchers from the University of Helsinki and Wihuri Research Institute reported recently in PNAS that PROX1 gene, which is essential for development of the liver, lymphatic vessels, and skeletal muscle stem cell differentiation, is highly expressed in rhabdomyosarcoma and plays a critical role in acquisition of skeletal muscle like features. In addition, the study showed that PROX1 promotes rhabdomyosarcoma growth by increasing expression and/ or activation of fibroblast growth factor receptors. Drugs inhibiting these receptors efficiently blocked the growth of tumor cells, suggesting that this pathway could be used as a potential target for rhabdomyosarcoma treatment.

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Photo by National Cancer Institute on Unsplash

Photo by National Cancer Institute on Unsplash

Why is it important?

The current treatments for rhabdomyosarcoma is based on general cancer drugs and have not developed much over the last decades. These drugs are not specific for rhabdomyosarcoma and often have severe side effects in surviving children. Thus, to have better survival rates and less side effects from the treatments, now treatment options are urgently needed. The present study identified PROX1 as an important regulator of rhabdomyosarcoma growth and FGFR signaling as a potential specific treatment target, which should be tested in further studies.

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