Basic Medicine

Molecular Medicine and Therapy

Innovative Drug Development ~From Drug Discovery to Investigator-driven Clinical Trials in Academia~

Medical Sciences Course

  • Master / Doctoral Degree


MIYATA, ToshioMIYATA, Toshio
MIYATA, Toshio

Professor, M.D. Ph.D.

*Concurrent Position

Research Theme

  • Development of inhibitors to carbonyl stress
  • Development of inhibitors to plasminogen activator inhibitor-1
  • Regulatory science
Research Keywords:

drug discovery and development, exploratory clinical trial, microdose test, PAI-1, carbonyl stress

Technical Keywords:

in silico drug design, structural optimization, target validation, preclinical study, clinical study

Laboratory Introduction

"Drug discovery" pursued by pharmaceutical companies follows a stereotyped pattern and operates within a very narrow scope. For example, the same statin-type antihyperlipidemic and sartan-type antihypertensive drugs may be extensively developed and formulated by drug companies, whereas orphan drugs or drugs that target diseases which are overlooked by drug companies but are nonetheless of significant social impact (such as kidney diseases) are far from receiving the attention they deserve in new drug development.
Our unit focuses on "academically driven drug discovery" via a cross-disciplinary approach involving inputs from biology, structural biology, pharmacology, chemistry, and computer engineering.
Our research platform employs an array of state-of-the-art technologies including in silico hit discovery, lead optimization, pre-clinical trials, human clinical trials (microdose trials, early-phase exploratory clinical trials, POC trials) to complement rational drug discovery and development.
They should improve insights into human physiology/pharmacology and document the drug candidate's characteristics and therapeutic target relevant to disease. Such an approach should alter the balance of risks during the drug development dramatically, enable us to pursue many more promising compounds than is currently affordable, and develop them with a much greater probability of success.

Figure 1. From drug discovery to investigator-driven clinical trials in academia

Figure 1. From drug discovery to investigator-driven clinical trials in academia

Recent Publications

  • Eren M, et al. PAI-1-regulated extracellular proteolysis governs senescence and survival in Klotho mice. Proc Natl Acad Sci USA. 111(19):7090-7095,2014
  • Miyata T, et al. Drug discovery in renal disease ?towards a more efficient framework. Nature Review Nephrology. 10(5):290-6,2014
  • Remuzzi G, et al. Kidney failure: aims for the next 10 years and barriers to success. Lancet. 382(9899):353-362,2013
  • Miyata T, Suzuki N, van Ypersele de Strihou C. Diabetic nephropathy: Are there new and potentially promising therapies targeting oxygen biology? Kidney Int. 84(4):693-702,2013
  • Mori T, Miyata T, Ito S. Oxidative/Carbonyl stress in the renal circulation and cardiovascular renal injury, p.305-320. In Oxidative Stress in Applied Basic Research and Clinical Practice Studies on Renal Disorders, edited by Miyata T, Eckardt KU, Nangaku M. 1st ed, vol 1. Springer Science+Business Media, New York, NY, 2011