Current Status, Issues and Future Prospects of Personalized Medicine for Each Disease

Abstract

In recent years, with the advancement of next-generation sequencing (NGS) technology, gene panel tests have been approved in the field of cancer diseases, and approaches to prescribe optimal molecular target drugs to patients are being developed. In the field of rare diseases, whole-genome and whole-exome analysis has been used to identify the causative genes of undiagnosed diseases and to diagnose patients’ diseases, and further progress in personalized medicine is expected. In order to promote personalized medicine in the future, we investigated the current status and progress of personalized medicine in disease areas other than cancer and rare diseases, where personalized medicine is most advanced. We selected rheumatoid arthritis and psoriasis as the inflammatory disease, in addition to Alzheimer’s disease. These diseases have high unmet needs for personalized medicine from the viewpoints of disease mechanisms, diagnostic biomarkers, therapeutic drugs with diagnostic markers and treatment satisfaction. In rheumatoid arthritis and psoriasis, there are many therapeutic options; however, diagnostic methods have not been developed to select the best treatment for each patient. In addition, there are few effective therapeutic agents in Alzheimer’s disease, although clinical trials of many candidate drugs have been conducted. In rheumatoid arthritis and psoriasis, further elucidation of the disease mechanism is desired to enable the selection of appropriate therapeutic agents according to the patient profile. In the case of Alzheimer’s disease, progress in preventive medicine is desired through the establishment of an early diagnosis method as well as the research and development of innovative therapeutic agents. To this end, we hope for further research and development of diagnostic markers and new drugs through progress in comprehensive data analysis such as comprehensive genomic and transcriptomic information. Furthermore, new types of markers such as miRNAs and the gut microbiome are desired to be utilized in clinical diagnostics.

A State-of-the-Art Roadmap for Biomarker-Driven Drug Development in the Era of Personalized Therapies

“…There is accumulating scientific evidence now especially in oncology that using clinically validated biomarkers to select patients for targeted therapies in precision medicine trials result in better outcomes. Our working group also performed an in-depth review on the medical needs for personalized approaches in non-oncology diseases [ 95 ].…”

Pharmacogenomics of Monoclonal Antibodies for the Treatment of Rheumatoid Arthritis

“…However, pharmacogenomic studies on the association between the new monoclonal antibody drugs and various genetic polymorphisms are insufficient. Moreover, factors involved in immunity and inflammation are very diverse, and there is currently no clear direction for personalized medicine [ 183 ]. Therefore, further research is essential, and this will increase the safety and efficacy of the newly developed monoclonal antibody drugs, enabling more complete precision medicine.…”

Current advances in our understanding of circular RNA (circRNA) in Alzheimer’s disease (AD); the potential utilization of synthetic circRNAs as a therapeutic strategy in the clinical management of AD

“…Currently the "precision medicine" approach has been to investigate early pathophysiological changes of AD to fully capture the complexity of the disease, and has been essential to develop timely screening, detection, diagnostic, prognostic and therapeutic interventions in significantly heterogeneous AD patient populations. Importantly "precision medicine" delineates which therapeutic approach or treatment strategy would be the most effective for a specific individual, at a specified disease stage, across multiple medical research fields that include molecular-genetics, imaging technologies, neuroscience, neurology and psychiatry and the identification of miRNA-and/or mRNA-based biomarkers in the CSF and blood (Hampel et al, 2019;Lemercier et al, 2021;Giampietri et al, 2022;Jellinger 2022;Yamamoto et al, 2022). "Precision medicine" achieves the greatest success when multiple interdisciplinary diagnostic parameters are integrated to yield the most accurate diagnostic analysis of the AD patient who can be subsequently treated using an individualized combination therapy approach or multi-target-directed methodologies.…”