Study: Finding Treatments to Prevent Rhabdomyosarcoma Recurrence

Rhabdomyosarcoma is a rare form of cancer that affects children. It was recently studied at St. Jude Children’s Research Hospital, where a team of medical professionals examined cells that persist even after treatment and lead to recurrence. Want to read their full study? You can find it published in Developmental Cell.

What is Rhabdomyosarcoma?

Before we can dive into recurrence and potential treatments to prevent it, we should first understand what this rare pediatric disease is. Rhabdomyosarcoma is a rare form of childhood cancer that begins in the soft tissue. While it most commonly affects children, people of any age may be impacted. It leads to varying symptoms depending on where in the soft tissue the tumor forms, but possible symptoms include blood in the urine, issues with bowel movements, trouble urinating, swelling in the eyes, headaches, bleeding in the throat, nose, and ears; and masses or bleeding in the vagina or rectum.

Medical professionals do not know what causes this cancer, although they have identified a number of risk factors, such as a family history of cancer, Noonan’s syndrome, and Costello syndrome. In terms of treatment, a combination of therapies is typically required. Options include radiation therapy, chemotherapy, surgery, and clinical trials.

About the Study

Now that we know what rhabdomyosarcoma is, let’s talk about the study. The researchers wanted to find treatments that could prevent this rare cancer from recurring after patients have undergone treatment. This would greatly improve outcomes for children whose cancer recurs, as they have already had to endure treatment (which can take a toll on the body), and recurrent rhabdomyosarcoma is harder to treat.

In order to find potential treatments, the research team examined a population of malignant cells that live on after therapy and lead to recurrence. These cells are the least developed, and when they survive treatment, they are able to move through the muscle development program and form another tumor. The researchers theorized that if these cells could be killed off in the initial treatment, then there would be no recurrence.

Through the St. Jude Childhood Solid Tumor Network (CSTN), they were able to analyze matched patient samples and orthotopic patient-derived xenografts. They evaluated these data through epigenetic profiling and single-nucleus RNA-sequencing. They discovered that the malignant cell population displayed many properties of the beginning stage of muscle development. Another finding was these cells’ dependence on the epidermal growth factor receptor (EGFR) signaling.

Armed with these results, the researchers found that EGFR inhibitors may be a viable treatment option that will kill all cancerous cells and prevent recurrence. In fact, their findings recommend adding an EGFR inhibitor to a patient’s treatment regimen.

Want to learn more? Read the source article from Medical Xpress.

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