New therapeutic target found for aggressive pediatric cancers with few treatment options
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IMAGE: Giannicola Genovese, M.D. view more 

Credit: MD Anderson Cancer Center

Researchers at The University of Texas MD Anderson Cancer Center have discovered that malignant rhabdoid tumors (MRT), a rare pediatric cancer without effective treatments, may be sensitive to drugs that block the cancer cell’s ability to dispose of misfolded proteins. The findings provide a much-needed therapeutic target for these and other cancers caused by mutations in the SMARCB1 gene.

Based on these findings, published today in Cancer Cell, the researchers now are leading a Phase II clinical trial to investigate this approach in renal medullary carcinoma (RMC), a related adolescent cancer also characterized by SMARCB1 mutations.

Malignant rhabdoid tumors are aggressive pediatric cancers that generally manifest in children in the first year of life, explains Giannicola Genovese, M.D., assistant professor of Genitourinary Medical Oncology and corresponding author on the study.

“It’s somewhat fortunate that these are very rare cancers. With current treatments, the prognosis is ominous – just about one year,” said Genovese. “It’s an orphan disease, meaning it has no targeted treatments available. The current options include high-dose chemotherapy, radiation and surgery, which can cause significant developmental and neurological effects and increase the risk for secondary cancers

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