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Science & Medicine: Sabotaging the molecular engines of some pediatric cancers

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Roberto Martinez
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TPR

Ewing sarcoma and rhabdomyosarcoma are cancers that overwhelmingly impact children. They're soft tissue cancers that are thankfully rare because treatment options are limited, according to pediatric cancer researcher Yogesh Gupta, PhD, an associate professor at Greehey Children's Cancer Research Institute at UT Health San Antonio.

“The drug treatment regimen has not been changed in the last three decades,” Gupta said.

Kids with these types of cancers will typically endure chemotherapy and radiation, and the aggressive treatment regimen can leave them with lifelong heart issues, problems with bone growth and development, impaired fertility, cognitive challenges, and an increased risk of secondary cancers.

Yogesh Gupta, PhD Associate Professor Department of Biochemistry and Structural Biology The University of Texas Health Science Center at San Antonio
Yogesh Gupta, PhD
Associate Professor
Department of Biochemistry and Structural Biology
The University of Texas Health Science Center at San Antonio

So even if the tumor is gone after successful treatment by chemotherapy, patients usually suffer and have to struggle with the side effects for the rest of their lives," Gupta said.

Gupta’s research focuses on RNA epigenetics in pediatric cancers and infectious diseases, and in his lab, they’re trying to come up with better treatments for these kids. One of the medications they're studying works by sabotaging what he calls the “molecular engines” of certain tumor-producing proteins.

“It binds to a specific pocket of this molecular engine, and this pocket is very important for the function of the protein,” Gupta said. “So by blocking this pocket of the molecular engine, we hope that the protein may no longer be able to do its tumor-promoting function in these cancers.”

The study of this new drug is in its earlier stages. It’s promising, in a petri dish, but has yet to be tested in an animal model. It’s far from human trials. Still, this is exciting progress in the treatment of these devastating cancers that he hopes will be encouraging to everyone in the cancer community.

“My message to the families and the patients suffering from these devastating diseases is that there is hope. Our therapy and our approach provide hope that one day we will have safer and less toxic therapies for these patients,” Gupta concluded.

Gupta received a grant for this research from the William and Ella Owens Medical Research Foundation.

Science & Medicine is a collaboration between TPR and The University of Texas Health Science Center at San Antonio that explores how scientific discovery in San Antonio advances the way medicine is practiced everywhere.