A University of Houston research team has received a $3.2 million grant from the National Institute of Health to combat Rhabdomyosarcoma (RMS), a rare and aggressive pediatric soft tissue cancer.
Led by Ashok Kumar, the Else and Philip Hargrove Endowed Professor of Drug Discovery at the University, the research focuses on blocking a key protein, TAK1, to prevent tumor growth and improve survival rates.
RMS accounts for 8 percent of childhood cancers, with a survival rate of just 20–30 percent for metastatic cases. Kumar, also the director of the Institute of Muscle Biology and Cachexia at UH College of Pharmacy, aims to stop RMS at the cellular level by preventing its uncontrolled division and failure to mature into normal muscle cells.
The research identifies TAK1 (Transforming growth factor β-activated kinase 1) as a crucial factor in RMS development. Though its role in RMS has not been extensively studied, preliminary results show TAK1 is highly active in both embryonal and alveolar RMS cells. Embryonal RMS, more common in young children, appears in the head, neck, or genitals, while the more aggressive alveolar RMS affects older children and teens, often in the arms or legs.
By blocking TAK1 using genetic and pharmacological approaches, researchers successfully halted RMS cell growth in lab and animal models. “Our project will identify key mechanisms and molecular targets to prevent tumor progression in RMS patients in future therapies,” Kumar said.
“By targeting TAK1, we aim to stop the cancer at its source and help the cells develop normally. This approach could lead to new and better treatments for RMS,” Kumar added.
The study aims to understand how TAK1 contributes to tumor growth and why it prevents RMS cells from maturing. If successful, targeting TAK1 could pave the way for innovative, life-saving treatments, providing new hope for children battling this devastating disease.
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