Landmark study to find cures for sarcomas

Children's Research Institute

Children’s doctors are participating in a study to discover what causes soft tissue sarcoma and Ewing’s sarcoma and develop treatments for children.

Doctors and researchers at Children’s Medical Center and UT Southwestern Medical Center are off and running on a one-of-a-kind cancer research study. The study has the potential to discover the unknown drivers of soft tissue sarcoma and Ewing’s sarcoma and develop tailored treatments for children with these diseases.

The Cancer Prevention and Research Institute of Texas (CPRIT) recently awarded a $6.9 million grant to the team* of four led by Stephen Skapek, M.D., medical division director for the Center for Cancer and Blood Disorders at Children’s and professor of pediatrics and director of the division of Pediatric Hematology/Oncology at UT Southwestern to undertake the study. The team will collaborate with researchers at Baylor College of Medicine/Texas Children’s Hospital and the UT Health Science Center at San Antonio over the next five years.

Soft tissue sarcoma is thought of as “an orphan disease,” Dr. Skapek said, explaining that there have been few advancements in treatments for sarcomas in the last 20 years compared to other cancers. He and his team believe their research study, which was the only new multi-investigator research award funded this year by CPRIT for pediatric cancer, has the potential to identify the unknown drivers of soft tissue and Ewing’s sarcoma that will lead to personalized treatment of the diseases.

“We are confident that we will get where no one else has been with this disease in the next three to five years,”
— Stephen Skapek, M.D.

Presently, the treatment for soft tissue and Ewing’s sarcomas is surgery, followed by radiation and/or chemotherapy. While chemo has proven effective in patients with Ewing’s sarcoma, it has not proven effective for patients with soft tissue sarcoma. And unfortunately, with both diseases, if the cancer cells spread around the body, only 10-20% of these patients will survive.

The team* plans to collect between 300-500 samples from childhood sarcoma patients living in Texas — the most ever for this disease — as well as a subset of samples from young adult patients over the five year study. They will focus on sequencing genetic material to find abnormalities in the genes on a scale never done before. The abnormalities will be run through functional models to find the drivers of these diseases.

“Our research study has the potential to quickly impact clinical care of children with sarcomas because our emphasis is on bringing basic science research into everyday clinical practice,” said Dinesh Rakheja, M.D., Interim Division Director of Pathology and Laboratory Medicine at Children’s and Interim Chief of Pediatric Pathology at UT Southwestern.

Even more exciting, is the prospect that Dr. Skapek expects the team’s study will lay the groundwork for similar studies of other childhood cancers, including childhood brain tumors, neuroblastoma, genitourinary malignancies, and childhood leukemia, which could lead to personalized cancer therapies for these diseases as well.

*The Team

  • Stephen Skapek, M.D.,  is the  medical division director for the Center for Cancer and Blood Disorders (CCBD) at Children’s and professor of pediatrics and director of Pediatric Hematology/Oncology at UT Southwestern.
  •  Jim Amatruda, M.D., Ph.D, is an attending physician for the CCBD at Children’s and assistant professor of pediatrics at UT Southwestern.
  • Rene Galindo, M.D., Ph.D,is a consultant on pediatric sarcomas at Children’s and assistant professor of Pathology at UT Southwestern.
  • Patrick Leavey, M.D., Dr. Leavey is an attending  physician and fellowship program director of the CCBD at Children’s and associate professor of pediatrics at UT Southwestern.
  •  Dinesh Rakheja, M.D., is Interim Division Director of Pathology and Laboratory Medicine at Children’s and Interim Chief of Pediatric Pathology at UT Southwestern.

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