Researcher Studies Gene Pathways Responsible for Metastatic Rhabdomyosarcoma

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One Year Research Update:

Jason Yustein, MD, PhD, is a CureSearch Young Investigator studying rhabdomyosarcoma at Baylor College of Medicine. Dr. Yustein’s research focuses on creating new models of mice that spontaneously develop rhabdomyosarcoma (RMS), which are useful because they allow researchers to analyze the genetics and molecular pathways altered in RMS development and progression. Dr. Yustein has used these models to not only understand tumor formation, but also learn more about the causes of metastatic RMS, or disease that has spread to multiple locations in the body. Outcomes for children with metastatic RMS are poor and research has not made significant progress in improving this condition over the past 20-30 years. Dr. Yustein hopes that by understanding the basic biology of how RMS becomes metastatic, he can develop more effective treatments for metastatic disease.

In his first year of research as a CureSearch Young Investigator, Dr. Yustein has made significant and exciting progress in developing mouse models and identifying the genetic differences between the local and metastatic RMS tumors. This research has led him to a novel discovery about “hypoxia-based pathways” contributing to RMS metastasis. Hypoxia is when cells are deprived of oxygen. Tumors often have low levels of oxygen because they grow faster than the blood supply can deliver oxygen. Cancer cells must adapt to hypoxic conditions. Some researchers believe that these adaptations make cancer cells resistant to chemotherapy and radiation therapy as well as contribute to metastatic mechanisms.

Dr. Yustein’s models of RMS and his insights into the growth of metastatic disease is the first step toward developing more effective treatments for this disease. CureSearch funding has enabled Dr. Yustein to pursue this novel research and we are pleased to be able to share his progress.

Rhabdomyosarcoma is cancer that develops in the skeletal muscles of children. It is the most common soft-tissue sarcoma diagnosed in children, and is most frequently found in the head, neck and extremities. Children who develop metastatic rhabdomyosarcoma, a cancer that has spread from the original tumor site to other areas of the body, have very poor outcomes. Jason Yustein, MD, PhD, from Baylor College of Medicine, believes that understanding how rhabdomyosarcoma cells become metastatic can lead to new therapies to improve the odds for these children.

Jason Yustein
  • Jason Yustein, MD, PhD
  • Baylor College of Medicine

It is believed that poor outcomes are a result of a lack of understanding of the genes and pathways involved in metastatic rhabdomyosarcoma. Dr. Yustein will explore what genes are changed when cancer spreads and what genes are critical to this process in hopes that they can be altered to prevent and treat the metastatic disease.

Dr. Yustein will conduct his research by using genetically engineered mice that have a higher likelihood of developing metastatic tumors in order to study the biology of the disease. By studying mice with rhabdomyosarcoma, he believes he can track the development of the cancer, and identify the genes responsible for the disease’s progression and subsequent metastasis. Dr. Yustein will use these findings to study critical genes and pathways that are altered in the disease development and progression. He believes these studies will provide significant advancements for understanding how these tumors become metastatic and by further understanding this process, new therapies can be developed to prevent and treat metastasis.

Dr. Yustein is funded for two years through the CureSearch Young Investigators Program. CureSearch is excited to provide regular updates on Dr. Yustein’s research.

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