Analysis of the impact of Tominersen (IONIS-HTTRX/RG6042) in the treatment of Huntington’s disease: A systematic review

Huntington’s disease (HD) is a congenital pathology of a hereditary, neurodegenerative and progressive character, with a high mortality rate. This pathology is caused by repeated expansions of a single CAG codon in the gene encoding huntingtin. To date, several attempts to prevent and delay such a mutation have been carried out in patients. However, a promising drug, Tominersen, formerly called IONIS HTTRx/ RG6042, is in the testing phase and has shown a favorable therapeutic response, which acts directly on the messenger RNA of the gene encoding huntingtin (HTT). The drug, whose route of administration is intrathecal in bolus, intercepts and destroys the messenger before the corrupted protein can work. The present study aims to analyze the impact of Tominersen in the treatment of patients with HD and to evaluate its prognosis. Methods: The present study is a systematic literature review, in which an electronic search was performed in the PubMed, SciELO, Web of Science and Google Scholar databases. Results: In one of the double-blind randomized clinical trials (n = 46), 34 were assigned to receive the drug. The group that received Tominersen showed a decrease in the concentration of mutant HTT in cerebrospinal fluid after 28 days. In another study (n = 791), the GENERATION HD1 clinical trial is currently taking place and will last for 25 months. Conclusion: Tominersen decreases the concentration of HTT, that is, it suppressed the protein responsible for HD. Studies related to the drug are still very recent and require extra attention.