Review
Biochemistry & Molecular Biology
Celia Bonilla, Mercedes Zurita
Summary: Traumatic brain injury (TBI) causes physical damage to brain tissue leading to neurological disabilities, and research on using stem cells to treat TBI is increasing.
Article
Psychology, Multidisciplinary
Kevin J. Munro, William M. Whitmer, Antje Heinrich
Summary: Clinical trials are crucial for evaluating interventions in preventing, diagnosing, or treating health conditions, with a focus on improving practice in healthcare. The lack of high-quality evidence in hearing health practice highlights the importance of understanding key elements defining trial quality, such as design, blinding, and outcome measures. The complexity of outcome measures within hearing health emphasizes the need to consider how measurement instruments impact interpretation, accuracy estimation, and the meaningfulness of differences to individuals with hearing loss.
FRONTIERS IN PSYCHOLOGY
(2021)
Article
Cell Biology
Mehdi Ghasemi, Charles P. Emerson, Lawrence J. Hayward
Summary: Facioscapulohumeral muscular dystrophy (FSHD) is a debilitating disease with variable age of onset, severity, and progression. Research progress in understanding the underlying mechanisms and developing novel therapies targeting DUX4 expression has accelerated in recent years, providing hope for future clinical trials to improve patient outcomes.
Review
Neurosciences
Zubair Ahmed
Summary: Traumatic brain injury (TBI) is a major cause of morbidity, disability, and mortality globally. Experimental treatments targeting biochemical and cellular changes after TBI are currently being evaluated in clinical trials, but the results vary. This study aims to identify the drugs currently being evaluated for TBI in clinical trials.
Article
Clinical Neurology
Marcus W. Koch, Jop Mostert, Pavle Repovic, James D. Bowen, Bernard Uitdehaag, Gary Cutter
Summary: The study compared the frequency of progression and improvement events in SPMS trials using data from the placebo arms of two large RCTs. The EDSS showed the highest rates of improvement over time, with the smallest difference between progression and improvement rates, followed by the T25FW and 9HPT. The findings suggest random variation and measurement error in all investigated outcome measures, with the T25FW and 9HPT showing less than the more established outcome measure EDSS.
Review
Biochemistry & Molecular Biology
David Gustafsson, Andrea Klang, Sebastian Thams, Elham Rostami
Summary: BDNF plays a crucial role in neural repair and regeneration, making it a focus of TBI research. Studies have shown varied effects on BDNF expression based on different experimental setups and interventions. Clinical research emphasizes the significance of BDNF polymorphism in outcomes, highlighting its protective role.
INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES
(2021)
Letter
Multidisciplinary Sciences
Eduardo F. Motti
Summary: This is a letter to the editor.
Article
Clinical Neurology
Pamela L. Wolters, Ana-Maria Vranceanu, Heather L. Thompson, Staci Martin, Vanessa L. Merker, Andrea Baldwin, Carolina Barnett, Kimberley S. Koetsier, Cynthia M. Hingtgen, Christopher J. Funes, James H. Tonsgard, Elizabeth K. Schorry, Taryn Allen, Taylor Smith, Barbara Franklin, Stephanie Reeve, REiNS Int Collaboration
Summary: The study reviewed and recommended patient-reported outcome measures for assessing multidimensional domains of quality of life for patients with neurofibromatosis types 1, 2, and schwannomatosis, with a focus on generic and disease-specific tools for use in clinical trials.
Article
Clinical Neurology
Pamela L. Wolters, Ana-Maria Vranceanu, Heather L. Thompson, Staci Martin, Vanessa L. Merker, Andrea Baldwin, Carolina Barnett, Kimberley S. Koetsier, Cynthia M. Hingtgen, Christopher J. Funes, James H. Tonsgard, Elizabeth K. Schorry, Taryn Allen, Taylor Smith, Barbara Franklin, Stephanie Reeve
Summary: The REiNS Collaboration recommends the use of generic and disease-specific PRO measures to assess multidimensional domains of QoL for NF clinical trials in children and adults. Further research is needed to evaluate the effectiveness of these measures in both medical and psychosocial trials.
Article
Clinical Neurology
Anat Mirelman, Andrew Siderowf, Lana Chahine
Summary: This study reviewed the available literature on the subtle motor features in the prodromal phase of Parkinson's disease (PD) and aimed to provide insights for possible clinical trials. The findings revealed significant progress in understanding the evolution of motor features before PD diagnosis. Digital measures hold promise for improving measurement precision and can be used outside the clinic in naturalistic environments. Future research should focus on advancing digital sensor technology and analysis, as well as developing methods for clinical diagnosis of PD in a clinical trial context.
Review
Biochemistry & Molecular Biology
Zin Z. Khaing, Jessica Y. Chen, Gevick Safarians, Sohib Ezubeik, Nicolas Pedroncelli, Rebecca D. Duquette, Tobias Prasse, Stephanie K. Seidlits
Summary: Spinal cord injury (SCI) often leads to loss of sensory and motor function, significantly impacting patients' quality of life. There are currently no available therapies for repairing spinal cord tissue. This review focuses on clinical trials of neuroprotective therapeutics that aim to mitigate secondary injury during the acute and subacute phases of SCI, including surgical interventions, pharmacological agents, and cell-based therapies. Combinatorial therapies and relevant considerations are also summarized.
INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES
(2023)
Article
Medicine, General & Internal
Mart Kals, Kevin Kunzmann, Livia Parodi, Farid Radmanesh, Lindsay Wilson, Saef Izzy, Christopher D. Anderson, Ava M. Puccio, David O. Okonkwo, Nancy Temkin, Ewout W. Steyerberg, Murray B. Stein, Geoff T. Manley, Andrew I. R. Maas, Sylvia Richardson, Ramon Diaz-Arrastia, Aarno Palotie, Samuli Ripatti, Jonathan Rosand, David K. Menon
Summary: This study is the first genome-and transcriptome-wide association studies of genetic effects on outcome in traumatic brain injury (TBI). While no genetic variants with genome-wide significance were found, the overall heritability estimate is consistent with the hypothesis that common genetic variation substantially contributes to inter-individual variability in TBI outcome.
Review
Oncology
Simona Secondino, Costanza Canino, Domiziana Alaimo, Marta Muzzana, Giulia Galli, Sabrina Borgetto, Sabrina Basso, Jessica Bagnarino, Chiara Pulvirenti, Patrizia Comoli, Paolo Pedrazzoli
Summary: Cell therapy approaches in solid tumors are currently challenging due to lack of significant therapeutic response and on-target off-tumor toxicity. Novel cell therapy programs are exploring advanced therapy medicinal products and combination therapy for improved outcomes. This review focuses on current clinical research in this setting, which will likely contribute to future advancements in cancer treatment.
Article
Clinical Neurology
Niall J. Bourke, Celia Demarchi, Sara De Simoni, Ravjeet Samra, Maneesh C. Patel, Adam Kuczynski, Quen Mok, Neil Wimalasundera, Fareneh Vargha-Khadem, David J. Sharp
Summary: Long-term outcomes of paediatric traumatic brain injury are difficult to predict. Traumatic brain injury can lead to reductions in brain volume and is associated with cognitive, emotional, and behavioral impairments. This study investigates the impact of brain injury on healthy brain development and shows that volumetric abnormalities are common after moderate/severe traumatic brain injury in both grey and white matter regions.
Article
Pharmacology & Pharmacy
Maria Daglas, Phan H. Truong, Linh Q. Miles, Sydney M. A. Juan, Shalini S. Rao, Paul A. Adlard
Summary: Iron plays a detrimental role in the injured brain, and the use of an iron chelator like deferiprone may be a promising therapeutic approach to improve survival, functional outcomes, and quality of life in TBI patients.
BRITISH JOURNAL OF PHARMACOLOGY
(2023)
