Review
Health Care Sciences & Services
Rayner K. J. Tan, Dan Wu, Suzanne Day, Yang Zhao, Heidi J. Larson, Sean Sylvia, Weiming Tang, Joseph D. Tucker
Summary: Digital approaches have the potential to enhance community engagement in clinical trials through decentralization of processes, development of trial components through digital crowdsourcing, and the use of digital qualitative research methods. These approaches can increase diversity of participants and deepen community engagement, while also offering new possibilities for community engagement. Practical considerations, strengths, and weaknesses should be taken into account when implementing digital approaches.
NPJ DIGITAL MEDICINE
(2022)
Review
Oncology
Michael A. Vogelbaum, Ian F. Parney, J. Bradley Elder, Daniel Cahill
Summary: Neurosurgeons play a significant role in clinical trials in neurooncology, developing and leading both traditional and translational prospective trials to evaluate new therapeutic approaches. However, there are barriers that need to be addressed in order to enhance neurosurgical participation and leadership in the prospective development of novel therapeutics.
JOURNAL OF NEURO-ONCOLOGY
(2021)
Review
Biochemistry & Molecular Biology
Caroline Sevin, Kumaran Deiva
Summary: There are more than 70 known lysosomal storage disorders caused by mutations in genes encoding lysosomal hydrolases. Central nervous system involvement is a hallmark of most LSDs, but current therapies are often limited by the blood-brain barrier. Gene therapy shows promise in correcting neurological defects in LSDs by genetically corrected cells crossing to adjacent deficient cells in the brain, and both in vivo and ex vivo strategies are feasible. Promising preclinical studies have been conducted, and clinical trials are underway to assess the safety and efficacy of gene therapy in neuropathic LSDs.
FRONTIERS IN MOLECULAR BIOSCIENCES
(2021)
Article
Health Care Sciences & Services
Mei Wang, Lisa Dolovich, Anne Holbrook, Susan M. Jack
Summary: The study aimed to understand factors influencing the engagement of community hospitals in Ontario to participate in RCTs. The key barriers identified were invisibility of community hospitals to research investigators and lack of research infrastructure in most of these hospitals. Improved communication and resource sharing between academic centers and community hospitals facilitated recruitment process.
JOURNAL OF EVALUATION IN CLINICAL PRACTICE
(2022)
Review
Oncology
Hemaasri-Neya Girithar, Ananda Staats Pires, Seong Beom Ahn, Gilles J. Guillemin, Laurence Gluch, Benjamin Heng
Summary: Breast cancer is the leading cause of cancer incidence and mortality in women worldwide. Metastasis is the main cause of death in breast cancer patients, as current treatment strategies are not effective in treating metastatic tumors. Immunotherapy and the kynurenine pathway (KP) have emerged as potential new approaches for breast cancer metastasis. This review discusses the mechanisms of immune suppression and cancer growth mediated by the KP, and provides an overview of studies and clinical trials targeting KP enzymes in breast cancer.
BRITISH JOURNAL OF CANCER
(2023)
Editorial Material
Oncology
Duncan C. Gilbert, Valerie Speirs
Summary: Precision oncology relies on high-quality tissue samples, but samples from patients receiving radiotherapy are less commonly available for biomarker studies. Although biobanks with material from radiotherapy studies exist, the challenge is to increase their visibility and accessibility to researchers.
NATURE REVIEWS CLINICAL ONCOLOGY
(2021)
Article
Medicine, General & Internal
Pablo Juan-Salvadores, Marcela Sanchez Michel Gomez, Victor Alfonso Jimenez Diaz, Cristina Martinez Reglero, Andres Iniguez Romo
Summary: This study found that females were underrepresented in clinical trials and the majority of participants had a low educational level. Educational interventions can improve participants' understanding and trust in clinical trials.
FRONTIERS IN MEDICINE
(2022)
Review
Gastroenterology & Hepatology
Paul Y. Kwo, Tushar Patel
Summary: Clinical trials are crucial for advancing new therapies for liver diseases and adapting to emerging capabilities and external forces. The COVID-19 pandemic has prompted changes in clinical trial operations and opportunities for innovation in hepatology trials. Technological advances will drive future trials, incorporating digital capabilities and expanded data collection, computing, and analytics. Innovative trial designs and broader participant engagement will shape trial conduct, along with evolving regulatory needs and new stakeholders. The evolution of clinical trials presents unique opportunities to improve the lives of patients with liver diseases.
Article
Ethics
Michael K. Gusmano
Summary: First-in-human clinical trials involving xenotransplantation raise ethical concerns regarding participant selection, therapeutic misconception, long-term monitoring and withdrawal rights, equitable access, and mistrust. Clinical investigators and regulators have an obligation to engage with underrepresented communities to address these issues.
HASTINGS CENTER REPORT
(2022)
Article
Medicine, General & Internal
Jennifer Le-Rademacher, Heather Gunn, Xiaoxi Yao, Daniel J. Schaid
Summary: Clinical trials are important for evaluating the safety and efficacy of new interventions. Traditional explanatory trials focus on exploring the biological causes and effects, while pragmatic trials consider real-world conditions and aim to optimize interventions in diverse clinical practices. It is essential to maintain scientific rigor, increase trial efficiency, ensure generalizability of results, and address health disparities through technology and decentralized approaches.
MAYO CLINIC PROCEEDINGS
(2023)
Article
Psychology, Multidisciplinary
Fleur Goedkoop, Daniel Sloot, Lise Jans, Jacob Dijkstra, Andreas Flache, Linda Steg
Summary: This study found that community factors, such as community identification and interpersonal contact with other community members, uniquely influence people's involvement in community energy initiatives, while personal sustainable energy motivations and perceptions of the community's sustainable energy motivation did not have a unique relationship with initiative involvement.
FRONTIERS IN PSYCHOLOGY
(2022)
Article
Immunology
W. Watson Buchanan, Colin A. Kean, K. D. Rainsford, Walter F. Kean
Summary: A clinical trial is a research method that investigates the therapeutic intervention's efficacy, both short-term and long-lasting, as well as its potential toxicity. The history of clinical trials can be traced back to Babylonian King Nebuchadnezzar's court around 600 BC, as described in the Book of Daniel. However, it is in the past 500 years that significant written documentation and interpretation of therapeutic benefits have been made. Examples such as Lind's demonstration on the effectiveness of oranges and lemons for scurvy treatment in 1747, and Edward Jenner's unethical experiment on inoculation in 1796, showcase the importance of clinical trials. Ethical considerations, rigorous clinical observations, statistics, scientific purity of therapeutic agents, and safety testing have contributed to the foundation of modern clinical trials.
INFLAMMOPHARMACOLOGY
(2023)
Article
Clinical Neurology
Matteo Garibaldi, Tommaso Nicoletti, Elisabetta Bucci, Laura Fionda, Luca Leonardi, Stefania Morino, Laura Tufano, Girolamo Alfieri, Antonio Lauletta, Gioia Merlonghi, Alessia Perna, Salvatore Rossi, Enzo Ricci, Jorge Alonso Perez, Tommaso Tartaglione, Antonio Petrucci, Elena Maria Pennisi, Marco Salvetti, Gary Cutter, Jordi Diaz-Manera, Gabriella Silvestri, Giovanni Antonini
Summary: In a large cohort of DM1 patients, muscle involvement was characterized by fat replacement, muscle atrophy and STIR positivity, which correlated with disease severity and duration. The study also identified STIR hyperintensity as an early marker of muscle involvement, providing potential therapeutic targets for future clinical trials.
EUROPEAN JOURNAL OF NEUROLOGY
(2022)
Article
Medicine, General & Internal
Shirley V. Wang, Sebastian Schneeweiss, RCT-DUPLICATE Initiative
Summary: Nonrandomized studies using insurance claims databases can provide real-world evidence on the effectiveness of medical products. By emulating the design parameters of randomized clinical trials and quantifying the agreement between database studies and RCTs, this study highlights the importance of closely replicating trial design and measurements for reliable conclusions.
JAMA-JOURNAL OF THE AMERICAN MEDICAL ASSOCIATION
(2023)
Article
Medicine, Research & Experimental
Lucy Ellen Selman, Clare Clement, Margaret Douglas, Keith Douglas, Jodi Taylor, Chris Metcalfe, J. Athene Lane, Jeremy Horwood
Summary: The study revealed various challenges in patient and public involvement (PPI) in clinical trials, including recruitment difficulties, maintaining professional boundaries, and lack of training. Successful PPI requires early planning, power sharing, clear roles, and collaboration and communication with public contributors.