Pharmacogenetic and pharmaco-miR biomarkers for tailoring and monitoring myasthenia gravis treatments

Abstract

ABSTRACT Introduction Inter-individual variation in drug efficacy and tolerability in myasthenia gravis (MG), an autoimmune disorder mostly treated by chronic immunosuppression, highlights the need of specific, safe and tailored precision medicine approaches. By targeting the disease-effector molecules, biological drugs are the most promising therapeutic agents to treat MG. A number of pharmacogenetic biomarkers associated with response to immunosuppressive drugs in MG have been identified, and microRNAs (miRNAs) are emerging as reliable markers for predicting or monitoring treatment response in individual patients. Areas covered This review provides an overview of pharmacogenetic and pharmaco-miR biomarkers associated with immunosuppressive treatment response in MG and other autoimmune diseases, pointing out the need of pharmacogenetic/-miR profiling for the recently developed biological drugs as an important step toward precision medicine. Expert opinion Extension of pharmacogenetic and pharmaco-miR data, and their entry into the clinical practice, hold the promise to greatly revolutionize MG therapy with clinically relevant drugs, including conventional and biological drugs, or their combination. High-throughput technologies, covering DNA and RNA, have the potential to disclose valuable pharmacogenomic/-miRNomic profiles able to guide the choice of the different drugs in individual patients, or biomarker-defined patients’ subgroups, thus significantly improving MG treatment in a cost/effective manner.