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Research grant hopes to improve the outcomes of pediatric patients with rhabdomyosarcoma

St. Baldrick’s Grant Aims to Better Understand Aggressive Pediatric Cancer Type

Research grant hopes to improve the outcomes of pediatric patients with rhabdomyosarcoma

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Written by Cancer Center on July 29, 2020

University of Colorado Cancer Center member and associate professor of Pathology Paul Jedlicka, MD, PhD, has received the St. Baldrick’s Research Grant with generous support from Marlee’s Smile. His research will focus on better understanding the mechanisms behind rhabdomyosarcoma, a common and aggressive cancer type in children. The goal of the research is to identify new approaches to interfering with disease progression.

Genetic changes linked to aggressive nature of rhabdomyosarcoma


Paul Jedlicka, MD, PhD

Rhabdomyosarcoma is a rare disease but, unfortunately, is common in kids. It arises in cells that would normally develop into skeletal muscles, or the muscles that help move our body. It tends to be very difficult to treat. 

“It can be a very aggressive disease, especially a type that is caused by a genetic change that creates an abnormal cancer-driving protein in the cell, called ‘PAX3/FOXO1’, or ‘P3F’ for short,” explains Jedlicka. “P3F-driven rhabdomyosarcoma shows a strong tendency to spread to other parts of the body, which is what typically leads to death from the disease.”

Knowledge may lead to better prognosis

P3F is hard to target with drugs. However, the protein uses parts, or “machinery”, within the cancer cell to help spread the disease. Rather than targeting the protein itself, Jedlicka hopes that there may be a way to target this “machinery” that helps spread the disease.

“In recently published work, we have found new parts of this machinery that help P3F cause rhabdomyosarcoma to spread to other parts of the body. With the support of this grant, we want to better understand how this new machinery works and how it could be targeted to interfere with rhabdomyosarcoma spread,” says Jedlicka. ”This work could identify new ways to inhibit the aggressive nature of this disease and improve patient outcomes.”

If Jedlicka and his team are able to identify new targetable mechanisms for P3F-driven rhabdomyosarcoma, it could change the prognosis for these patients significantly.

About Marlee’s Smile

Marlee’s Smile was founded in honor of 12-year-old, Marlee Pack. Diagnosed with alveolar rhabdomyosarcoma, she relapsed three times in four years. After the final relapse, Marlee had to make a decision no child should, would she continue painful, toxic treatments or enter hospice care. She passed away on February 23, 2019. The organization’s mission is to change the lives of kids with cancer, one smile at a time in two ways. This first is by giving a custom Build-A-Bear to every child fighting cancer, as well as to their siblings in honor of Marlee’s generous heart as she knew the comfort of a furry friend. Second, by funding targeted research of pediatric cancers, specifically sarcomas to honor Marlee’s dying wish that no child should have to suffer the pain and hopelessness of current cancer treatments. 

About St. Baldrick’s Foundation

St. Baldrick’s is a volunteer-driven and donor-centered charity dedicated to funding childhood cancer research. A child is diagnosed with cancer every three minutes worldwide. One in five children diagnosed in the United States will not survive. Of those that survive, 80% will have severe or life-threatening conditions as a result of treatment. That is why the St. Baldrick’s Foundation continues its commitment to fund the most promising research initiatives to find cures and provide kids with less toxic treatments, ensuring a healthier life for survivors.

St. Baldrick’s Foundation Past Support for the CU Cancer Center: