Translational science comprises studies conducted to apply medical knowledge and innovative technologies developed in basic research in real-world settings for the prevention, diagnoses, and treatment of diseases. Translational medicine is challenging and requires input from individuals with multiple areas of expertise. Thus, programs designed to train young researchers who wish to work in drug development are needed. Here, we report the establishment of an intensive training program, called the Translational Science and Medicine Training Program (TSMTP), which provides training for young researchers in academia and pharmaceutical companies and staff members at industrial, academic, or governmental agencies throughout Japan. TSMTP disseminates information on the latest technology used by the National Center for Advancing Translational Sciences (NCATS) at the US National Institutes of Health (NIH). Through this program, young researchers can receive an overview of drug development from instructors who are experts in each step of the process, allowing them to apply this knowledge for future research.
In 2014, the Kyushu University Center for Clinical and Translational Research (CCTR) established a program to develop collaborations and harmonization strategies for translational science in the Asia-Pacific region. The program utilized connections between academia, industry and government in Japan to act as hosts to talented medical innovators who can contribute to developing new innovations in the region. The CCTR called 51 participants from ten Asia-Pacific countries to take part in an intensive program to visit facilities on the front line of medical innovation in Japan so that they may take the knowledge back to their countries and make fruitful collaborations between their institutions and Japan counterparts.
Drug repurposing is being actively pursued as a cost- and time-efficient drug discovery and development strategy. We proposed a new public-private partnership model for drug repurposing that involves academic institutions, government, investors, pharmaceutical and biotech companies. The model initially identifies promising hypothesis based on information of pharma assets on shelves, and proves the hypothesis in early clinical settings through academic and industry collaborations. We are currently developing multiple clinical assets including ART-001, a novel PI3Kα inhibitor for complex vascular malformations, based on this strategy to prove the model as a highly productive drug discovery and drug development framework.
Tensin 2 (TNS2) is a focal adhesion-localized multidomain protein expressed in various tissues. TNS2 expression significantly decreases in many tumor cell lines, and low TNS2 expression is associated with a poorer relapse-free survival in some cancers, suggesting that the loss of TNS2 may be related to tumor progression. Deregulation of Wnt/β-catenin signaling is frequently observed in colorectal cancer. In the present study, we found that TNS2 negatively regulated Wnt signaling by suppressing the nuclear translocation of β-catenin by reducing integrin-linked kinase (ILK) activity in colon cancer cell lines. To investigate the role of TNS2 in intestinal tumorigenesis in vivo, we introduced Tns2 mutation into ApcMin/+ mouse, a model of human familial adenomatous polyposis. The compound mutant mice showed a significant increase in tumor number and size in the small intestine and colon. Thus, this study may contribute to the discovery of novel mechanisms underlying cancer malignancy, and pave the way for the development of treatment strategies for intestinal cancers.
In this review, I have discussed current issues related to risk assessments of developmental and reproductive toxicities during the use of pharmaceuticals in pregnant women or women of childbearing potential. Owing to the lack of empirical information on the use of pharmaceuticals (including those approved for the market) in pregnant women, the toxicity of these drugs has been generally evaluated in animal studies. In the case of embryo-fetal developmental (EFD) toxicity, in particular, testing of small-molecule pharmaceuticals in animals (namely rodents and rabbits) is required. The body of research in animal studies affords us an opportunity to explore numerous topics in regulatory science research. However, there are three major issues: risk communication, use of animals, and reduction or deferral of EFD toxicity studies for the development of human pharmaceuticals. Although regulatory science research may not be necessary for some topics, it would contribute to the reduction or replacement of testing in animals. It is hoped that regulatory science research in this field will proceed rapidly, with the goal of safe use of pharmaceuticals for women.