Austrian research team identifies new therapeutic target for leukemic cutaneous T-cell lymphoma

Leukemic cutaneous T-cell lymphoma (L-CTCL) is a rare type of skin cancer that presents varied clinical features and symptoms. In a recently published study by the University of Veterinary Medicine Vienna and the Medical Universities of Vienna and Graz, the researchers managed to integrate the genomic data from affected patients with pharmacologic studies. This innovative research approach has led to the identification of STAT3/5 and PAK kinase as potential new therapeutic targets. Despite numerous genomic sequencing efforts, no characteristic driver mutations for L-CTCL have been identified to date. Chromosomal losses and gains, however, are known to be frequent and dominant in L-CTCL. To gain new insights into the causes of the disease and to identify possible new targets for therapies, the research team took a new approach: For their study, published in EMBO Molecular Medicine, the research team integrated genomic analyses with pharmacologic interference studies to identify key mutations leading to the disease and so explore new therapeutic approaches. PAK inhibitor FRAx597 inhibits tumour growth and disease dissemination In 74% of the L-CTCL patients studied, we detected increased copy numbers of loci containing STAT3/5 oncogenes, which correlated with an increased T-cell count in the blood.