Small molecule ADAR1 inhibitor for prostate cancer

A synthetic nucleoside analog inhibitor of the adenosine deaminase ADAR1 could help treat prostate cancer by preventing translation of the oncogene MTDH, thus decreasing prostate cancer growth and metastasis, without inducing Aicardi-Goutières syndrome, which is caused by ADAR1 loss-of-function mutations.

In a bioinformatic analysis of The Cancer Genome Atlas data, ADAR1 expression was higher in patient prostate cancer samples than in non-cancerous tissue from individuals without prostate cancer, and prostate cancer patients with higher ADAR1 expression had shorter overall survival than prostate cancer patients with lower ADAR1 expression. In a separate analysis of public data sets, ADAR1 expression was higher in patient prostate cancer samples than in non-cancerous prostate samples. In prostate cancer patients, ADAR1 protein expression was higher in tumors than in adjacent normal tissues. ...