Translational research
Recent decades saw a new trend evolving across all disciplines in biomedical science. It is the translational approach, which was introduced to all aspects of the field, meaning translating the discoveries of basic research into clinical practice and patient care with a more distant aim of impacting public health. There is a big variety of terms currently used, including “translational research” (TR), “translational science”, “translational medicine” with their branched development into narrow specialist fields (Fig. 1) [1]. One of the best and widely known examples of TR is the drug development process. It shows how a basic research effort gets clinical trial opportunity and at the end the approval for practical use, later directly and positively impacting patient care. In neurology, the neurotherapeutic discovery and development is increasing in numbers being funded by governmental and private sources [2]. This model utilizes an approach when the purpose of this translational process shapes the way it is started and conducted. Finally, this approach creates a more efficient bridge between researchers working in the basic areas and those coming from the clinical practice and public health environment with a clear understanding of what they would like to have as the ultimate result of this time- and effort-taking route. In simple, the clinical researchers get open window to channel their ideas and needs to the fundamentalists.
It becomes a standard that any basic research project usually gets more attention if the investigators show clear vision of the impact it may have on patients’ health. This highly stimulates formation of various collaborations between the groups on multiple levels. Moreover, TR promotes also transnational collaborations. The latter create opportunities for specialists from underdeveloped countries to be involved in cutting-edge research consortia initiatives, while contributing to the projects by collecting biological samples or other research materials. Through this approach the consortia may benefit by getting samples from underrepresented populations, further enriching the scientific data they have and making it more representative [3]. This kind of collaboration is also helpful for increased researcher networking and mobility, with opportunities to visit sites utilizing new research methods, learning and later implementing them in their own laboratories or research centers. Moreover, the concept of TR reshapes the landscape of the biomedical field. The clinical academic departments change their staff structure and involve positions responsible for conducting basic research in connection with clinicians.
The importance of TR is rising since the outcomes of increasing volume of basic research need to be translated into clinical research and practice. However, for some reasons the accumulating vast amount of basic research was not reaching this level frequently being “under-translated”. Only in recent decades have there been improvements in this gap. The shift to positive balance had several explanations. Firstly, the research methods became far improved and brought wider opportunities. Like in many other fields, neurology also includes examples of such collaborations. Secondly, big data approach, creation of large research consortia involving many research groups under one umbrella with joint research efforts make the obtained results more representative and translative [4,5]. It is helpful for creating virtual or sometimes physical platform for basic (laboratory) and clinical researchers using modern communication technologies to create scientifically promising projects, get funding, perform the research in an honest and conscientious way. Finally, in case of successful results these get published with a more visible impact on the clinical practice outcomes, although often not immediate. More networking between these groups of researchers brings more chances to create meaningful projects in future.
Translational neuroscience
The term “translational neuroscience” (TN) is already a highly regarded standard in the major clinical fields comprising it – neurology and psychiatry [6,7]. There was and there still is (in increasing numbers) a general trend that neuroscience is one of the biggest contributors of Nobel prize winners and overall growing numbers of highly impactful publications [8,9]. This is a direct result of the abovementioned general shift towards more translational approaches, which include both research planning but also material redistribution and accessibility and human resource logistics.
Translational neuroscience is a large area seeing numerous fruitful collaborations. This is connected to the fact that TN intersects with other rapidly growing basic TR fields like genetics, molecular biology, biochemistry and immunology, to mention a few [7]. Indeed, these developments highly stimulated the neurological and psychiatric TR. There are many regional and international TN collaborations further unifying research efforts in particular fields, which could become truly global and multi-component, e.g. ENIGMA Consortium, Global Parkinson Genetics Program (GP2), etc. [10,11]. These examples show major components of research work – scientific initiative and opportunity – brought to worldwide level.
Across the general trends, neurology sees a more focused work towards precision medicine. Especially when considering specific debilitating diseases with scarce or absent opportunities for approved treatment. Added to previously mentioned progress on Parkinson’s disease (PD), also Alzheimer’s disease and cognitive health, amyotrophic lateral sclerosis, multiple sclerosis, genetic neuromuscular diseases and many others see increasing efforts pursuing treatment solutions through improved understanding oh pathophysiology [12].
Alzheimer’s disease, as a major type of neurodegenerative dementia, had decades of breakthroughs, including specific biomarker identification, their presentation to clinical practice, improvement and increased utilization through changing analysis procedure from cerebrospinal fluid to blood sampling [13]. Development of amyloid clearance-related treatments revolutionized the disease image from a primarily palliative to potentially treatable condition [14].
Going back to PD, there is an interesting situation when researchers accumulated deep understanding of the disease pathology and biomarkers but still have lack of identified disease-modifying treatments. This is especially imperative because one of the most powerful PD biomarkers is rapid eye-movement sleep behavior disorder (RBD), which is known to precede the development of PD motor clinical features for many years as isolated RBD [15]. And to make it simple, once the PD-modifying treatment is found, it could be started as early as 20 years before disease onset. The latter may serve as a fascinating example of TN research in progress with us hopefully lucky to see its breakthrough in the coming years. Researchers in this and other TN fields are eagerly waiting for this sort of change to happen to act, very much like Formula 1 team members waiting for their racing cars at the pit stops – ready and determined to repair the damage.
Overall, considering recent developments, TN is offering continuously increasing impact on clinical practice and patient outcomes, proving that shifting to this vector of development was one of the most significant redirections of research organization in recent decades.
References
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- Mott M, Koroshetz W. Bridging the gap in neurotherapeutic discovery and development: the role of the national institute of neurological disorders and stroke in translational neuroscience. Neurotherapeutics. 2015;12(3):651-654.
- Kaiyrzhanov R, Zharkinbekova N, Guliyeva U, et al. Elucidating the genomic basis of rare pediatric neurological diseases in Central Asia and Transcaucasia. Nat Genet. 2024;56(12):2582-2584.
- Kerestes R, Laansma MA, Owens-Walton C, et al. Cerebellar volume and disease staging in Parkinson’s disease: an ENIGMA-PD Study. Movement Disorders. 2023;38(12):2269-2281.
- Quidé Y, Jahanshad N, Andoh J, et al. ENIGMA-Chronic Pain: a worldwide initiative to identify brain correlates of chronic pain. Pain. 2024;165(12):2662-2666.
- Gomez CM. Translational neuroscience: A neurologist’s translation. Curr Neurol Neurosci Rep. 2006;6(2):85-87.
- Marková IS. Translational neuroscience and psychiatry: A conceptual analysis. Evaluation Clinical Practice. 2018;24(4):791-796.
- Yeung AWK, Goto TK, Leung WK. The changing landscape of neuroscience research, 2006–2015: a bibliometric study. Front Neurosci. 2017;11:120.
- Sourkes TL. Introduction: neuroscience in the Nobel perspective. J Hist Neurosci. 2006;15(4):306-317.
- Thompson PM, Stein JL, Medland SE, et al. The ENIGMA Consortium: large-scale collaborative analyses of neuroimaging and genetic data. Brain Imaging Behav. 2014;8(2):153-182.
- Blauwendraat C, Noyce AJ, Mata IF, et al. Tackling a disease on a global scale, the Global Parkinson›s Genetics Program, GP2: A new generation of opportunities. Am J Hum Genet. 2025;112(9):1988-2000.
- Sahakian BJ, Bruhl AB, Cook J, et al. The impact of neuroscience on society: cognitive enhancement in neuropsychiatric disorders and in healthy people. Philos Trans R Soc Lond B Biol Sci. 2015;370(1677):20140214.
- Hunter TR, Santos LE, Tovar-Moll F, De Felice FG. Alzheimer’s disease biomarkers and their current use in clinical research and practice. Mol Psychiatry. 2025;30(1):272-284.
- Hering H, Bussiere T, Liu CC, et al. A manifesto for Alzheimer’s disease drug discovery in the era of disease-modifying therapies. Mol Neurodegeneration. 2025;20(1):88.
- Miglis MG, Adler CH, Antelmi E, et al. Biomarkers of conversion to α-synucleinopathy in isolated rapid-eye-movement sleep behaviour disorder. Lancet Neurol. 2021;20(8):671-684.