After 50 years of wandering among new discovery technologies and a worrying antiquated development process, the research and development (R&D) enterprise may finally have found a way out of its wilderness through the auspices of translational medicine. For that to happen, a number of problems have to be confronted. Most prominent is the traditional problem credited with giving rise to translational medicine - the lack of a feedback loop from bench to bedside. However, there are equally significant roadblocks to be confronted at every turn along the R&D pathway, from basic research through discovery and preclinical testing and, finally, into the clinic. Translational medicine is now beginning to encompass other powerful and promising innovations such as personalized medicine, bioinformatics, advanced imaging and biomarkers to help move beyond these roadblocks. Although translational medicine has attracted significant financial support in recent years, the economics of the movement are still challenging, as public and private sector funding are both difficult to come by due to the economic downturn. Some of the dearth of monies has been redressed by foundations and public-private partnerships, which combine resources and, hence, divide the risk to the benefit of all. More recently, the inherent tension among the traditional roles of 'big pharma', biotechs and the public sector, which had waned somewhat in the wake of the Bayh-Dole legislation in the USA, has re-emerged owing to the furor over conflicts of interest. Translational medicine may help alleviate these tensions by its example of successful precompetitive collaboration, such as the Predictive Safety Testing Consortium and its effective use of project management techniques in multidisciplinary, multiphasic, multisectoral projects to bring the discipline necessary for the efficient functioning of consortia. Changing a research paradigm that has remained substantially unchanged for half a century will require considerable time, money and effort. However, there is reason to be optimistic that translational medicine is the right solution for the right problem at the right time.
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http://dx.doi.org/10.2217/pme.09.40 | DOI Listing |
J Med Chem
January 2025
School of Pharmacy, College of Pharmacy, Taipei Medical University, Taipei 110, Taiwan.
Since decades after temozolomide was approved, no effective drugs have been developed. Undoubtedly, blood-brain barrier (BBB) penetration is a severe issue that should be overcome in glioblastoma multiforme (GBM) drug development. In this research, we were inspired by linezolid through structural modification with several bioactive moieties to achieve the desired brain delivery.
View Article and Find Full Text PDFJAMA Neurol
January 2025
Department of Neurology, Xuanwu Hospital Capital Medical University, National Center for Neurological Disorders, Beijing, China.
Importance: Autoantibodies targeting astrocytes, such as those against glial fibrillary acidic protein (GFAP) or aquaporin protein 4, are crucial diagnostic markers for autoimmune astrocytopathy among central nervous system (CNS) autoimmune disorders. However, diagnosis remains challenging for patients lacking specific autoantibodies.
Objective: To characterize a syndrome of unknown meningoencephalomyelitis associated with an astrocytic autoantibody.
Sleep Breath
January 2025
Nantong Key Laboratory of Translational Medicine in Cardiothoracic Diseases, and Research Institution of Translational Medicine in Cardiothoracic Diseases, Affiliated Hospital of Nantong University, Nantong, Jiangsu, 226001, China.
Background: Our previous study have demonstrated chronic intermittent hypoxia (CIH) induced cardiomyocyte apoptosis and cardiac dysfunction. However, the molecular mechanisms are complicated and varied. In this study, we first investigated the CaMKIIγ expression and signaling pathway in the pathogenesis of cardiomyocyte apoptosis after CIH.
View Article and Find Full Text PDFJ Infect Dis
January 2025
Johns Hopkins University School of Medicine, Baltimore, Maryland, USA.
J Clin Endocrinol Metab
January 2025
Department of Kinesiology and Health, Rutgers University, New Brunswick, NJ 08901, USA.
Context: Physical activity, exercise, or both are a staple of lifestyle management approaches both for type 1 diabetes mellitus (T1DM) and type 2 diabetes (T2DM). While the current literature supports both physical activity and exercise for improving glycemic control, reducing cardiovascular risk, maintaining proper weight, and enhancing overall well-being, the optimal prescription regimen remains debated.
Evidence Acquisition: We searched PubMed and Google Scholar databases for relevant studies on exercise, insulin sensitivity, and glycemic control in people with T1DM and T2DM.
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