Translational science: Newly emerging science in biology and medicine — Lessons from translational research on the natriuretic peptide family and leptin —

Abstract

Translation is the process of turning observations in the laboratory, clinic, and community into interventions that improve the health of individuals and the public, ranging from diagnostics and therapeutics to medical procedures and behavioral changes. Translational research is defined as the effort to traverse a particular step of the translation process for a particular target or disease. Translational science is a newly emerging science, distinct from basic and clinical sciences in biology and medicine, and is a field of investigation focused on understanding the scientific and operational principles underlying each step of the translational process. Advances in translational science will increase the efficacy and safety of translational research in all diagnostic and therapeutic areas. This report examines translational research on novel hormones, the natriuretic peptide family and leptin, which have achieved clinical applications or for which studies are still ongoing, and also emphasizes the lessons that translational science has learned from more than 30 years' experience in translational research.

Distinguishing between translational science and translational research in CTSA pilot studies: A collaborative project across 12 CTSA hubs

“…A checklist was provided to project submitters to characterize the pilot study data source. Multiple categories could be selected, including (1) Questions relating to principles of translational science A set of questions was constructed based on the 20 principles of TS posted on the NCATS website in September 2022 (see Table 1; full descriptions provided in Supplementary Materials). The set of questions was developed in three iterative steps.…”

“…In the last decade of the 20 th century, translational research started to gain momentum in biology and medicine [1]. While the scientific community, policymakers, and the general public value and support basic research whose primary goal is to build the scientific basis for the development of novel therapies, there have been longstanding concerns that much basic research has little immediate impact on clinical practice and public health interventions [2][3][4].…”

Practice guideline for lipodystrophy syndromes—clinically important diseases of the Japan Endocrine Society (JES)

“…Leptin replacement therapy is approved for partial lipodystrophy syndromes as well as generalized lipodystrophy syndromes since 2013 in Japan [1,69]. In 2019, a highly sensitive enzyme-linked immunosorbent assay (ELISA) for leptin (Cosmic Corp., Tokyo, Japan), which is more sensitive than a RIA for leptin (Millipore, St. Charles, Missouri, U.S.A.), was approved by the PMDA for the diagnosis of generalized lipodystrophy syndrome [69] and its cost has been covered by health insurance from June, 2021 in Japan. Evidence has accumulated in Japan, which indicates that leptin replacement therapy for partial lipodystrophy syndromes is useful for patients with low-normal serum leptin levels before treatment [69].…”

“…In 2019, a highly sensitive enzyme-linked immunosorbent assay (ELISA) for leptin (Cosmic Corp., Tokyo, Japan), which is more sensitive than a RIA for leptin (Millipore, St. Charles, Missouri, U.S.A.), was approved by the PMDA for the diagnosis of generalized lipodystrophy syndrome [69] and its cost has been covered by health insurance from June, 2021 in Japan. Evidence has accumulated in Japan, which indicates that leptin replacement therapy for partial lipodystrophy syndromes is useful for patients with low-normal serum leptin levels before treatment [69]. These results indicate that generalized lipodystrophy is an absolute leptin deficiency, while partial lipodystrophy is a relative leptin deficiency.…”

Why scientists, academic institutions, and investors fail in bringing more products to the bedside: the Active Compass model for overcoming the innovation paradox

“…Translational research involves the multidisciplinary integration of basic research, patient-oriented research, disease-oriented research, and population-based research, aimed at improving diagnosis and prognosis [ 8 ]. Much translational research is based on physician-scientists’ bringing of ideas from bench to bedside [ 9 , 10 ]. Physician-scientists invest significant time in scientific research, mainly inpatient or disease-oriented research, and spend less time in direct clinical practice [ 11 , 12 ].…”