University receives $3.2M grant for childhood cancer research targeting cellular level

A $3.2 million grant from the National Institutes of Health has been awarded to researchers at the University of Houston. The funding is aimed at advancing treatment options for Rhabdomyosarcoma (RMS), a malignant soft tissue sarcoma that predominantly affects young children and accounts for 8% of all pediatric cancers. The survival rate for patients with RMS that has metastasized is approximately 20%–30%.

The project, led by Ashok Kumar, Else and Philip Hargrove Endowed Professor of Drug Discovery at the UH College of Pharmacy and director of the Institute of Muscle Biology and Cachexia, seeks to identify mechanisms and molecular targets to prevent tumor progression in RMS patients. Kumar explained, “Our project will identify key mechanisms and molecular targets to prevent tumor progression in RMS patients in future therapies.”

Researchers are focusing on a protein called TAK1 (Transforming growth factor β-activated kinase 1) found inside RMS cells, which plays a significant role in cell growth regulation. "Though the role of TAK1 in RMS or any other sarcoma has not been investigated, our preliminary results demonstrate that TAK1 is highly activated in both embryonal RMS and alveolar RMS cells and human RMS samples," said Kumar.

Kumar further elaborated on potential treatment strategies: “Blocking TAK1, either by changing the genes (genetic approaches) or using drugs (pharmacological approaches), can stop certain harmful behaviors in cancer cells.” These findings were supported by tests conducted on lab-grown cells as well as living models.

Despite these advances, several questions remain unanswered regarding how TAK1 aids in cancer growth and its prevention from allowing RMS cells to mature into normal muscle cells. According to Kumar's hypothesis based on preliminary studies, "aberrant activation of TAK1 causes tumorigenesis and growth of RMS," suggesting that inhibiting TAK1 could be a promising therapeutic approach.

Kumar expressed hope for future treatments: “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.”