Article
Biochemistry & Molecular Biology
Angela Genchi, Elena Brambilla, Francesca Sangalli, Marta Radaelli, Marco Bacigaluppi, Roberto Furlan, Annapaola Andolfo, Denise Drago, Cinzia Magagnotti, Giulia Maria Scotti, Raffaella Greco, Paolo Vezzulli, Linda Ottoboni, Marco Bonopane, Daniela Capilupo, Francesca Ruffini, Daniela Belotti, Benedetta Cabiati, Stefania Cesana, Giada Matera, Letizia Leocani, Vittorio Martinelli, Lucia Moiola, Luca Vago, Paola Panina-Bordignon, Andrea Falini, Fabio Ciceri, Anna Uglietti, Maria Pia Sormani, Giancarlo Comi, Mario Alberto Battaglia, Maria A. A. Rocca, Loredana Storelli, Elisabetta Pagani, Giuseppe Gaipa, Gianvito Martino
Summary: This study conducted a phase 1 clinical trial evaluating the feasibility, safety and tolerability of intrathecally transplanted human fetal neural precursor cells (hfNPCs) in patients with progressive multiple sclerosis (PMS). The results demonstrated that hfNPC therapy is feasible, safe and tolerable in PMS patients.
Article
Pharmacology & Pharmacy
Agustin Clemente-Moragon, Eduardo Oliver, Daniel Calle, Lorena Cusso, Monica Gomez, Jesus M. Pradillo, Raquel Castejon, Norma Rallon, Jose M. Benito, Jose C. Fernandez-Ferro, Joaquin Carneado-Ruiz, Maria A. Moro, Javier Sanchez-Gonzalez, Valentin Fuster, Marta Cortes-Canteli, Manuel Desco, Borja Ibanez
Summary: By targeting neutrophil beta(1) adrenoceptors (beta 1AR), cerebral damage in patients with ischaemic stroke can be improved, providing a novel therapeutic strategy.
BRITISH JOURNAL OF PHARMACOLOGY
(2023)
Article
Medicine, General & Internal
Xinlei Ma, Fan Yang, Jinzhi Wu, Bei Xu, Mengdi Jiang, Yiduo Sun, Chuanying Sun, Ye Yu, Danyi Xu, Lanlan Xiao, Chunyun Ren, Chunyan Chen, Zi Ye, Junyu Liang, Jin Lin, Weiqian Chen
Summary: This study explored the pathogenesis of polymyalgia rheumatica (PMR) and found that JAK signaling is involved in the disease. The study also demonstrated that tofacitinib has similar efficacy to glucocorticoids in treating PMR patients. This research provides important insights for understanding the pathogenesis of PMR and exploring alternative treatments to glucocorticoids.
Article
Dermatology
Jun-Yi Chen, Qing-Lan Feng, Hui-Hui Pan, Ding-Heng Zhu, Ren-Liang He, Cheng-Cheng Deng, Bin Yang
Summary: Oral tofacitinib has shown significant efficacy in keloid patients, reducing the volume of keloid lesions, inhibiting collagen synthesis and fibroblast proliferation, and alleviating itch and pain, improving quality of life.
Article
Biochemistry & Molecular Biology
Cristina Fernandez-Mendivil, Enrique Luengo, Paula Trigo-Alonso, Nuria Garcia-Magro, Pilar Negredo, Manuela G. Lopez
Summary: The study revealed that overexpression of HO-1 in microglial cells in aged mice leads to neurotoxic iron accumulation, oxidative stress, and cognitive decline. Inhibiting HO-1 or using an iron chelator can prevent these issues, providing new insights for treating age-related neurodegenerative diseases.
Article
Hematology
Sarah Gillessen, Annette Pluetschow, Vladan Vucinic, Helmut Ostermann, Carsten Kobe, Paul J. Broeckelmann, Boris Boell, Dennis A. Eichenauer, Jan-Michel Heger, Sven Borchmann, Michael Fuchs, Peter Borchmann, Andreas Engert, Bastian von Tresckow
Summary: Ruxolitinib showed low but favorable activity in patients with relapsed or refractory cHL, indicating potential benefit for prognosis improvement. Further investigation and improvement are still needed.
EUROPEAN JOURNAL OF HAEMATOLOGY
(2022)
Article
Gastroenterology & Hepatology
Richard J. Thompson, Reha Artan, Ulrich Baumann, Pier Luigi Calvo, Piotr Czubkowski, Buket Dalgic, Lorenzo D'Antiga, Angelo Di Giorgio, Ozlem Durmaz, Emmanuel Gonzales, Tassos Grammatikopoulos, Girish Gupte, Winita Hardikar, Roderick H. J. Houwen, Binita M. Kamath, Saul J. Karpen, Florence Lacaille, Alain Lachaux, Elke Lainka, Kathleen M. Loomes, Cara L. Mack, Jan P. Mattsson, Patrick Mckiernan, Quanhong Ni, Hasan Ozen, Sanjay R. Rajwal, Bertrand Roquelaure, Eyal Shteyer, Etienne Sokal, Ronald J. Sokol, Nisreen Soufi, Ekkehard Sturm, Mary Elizabeth Tessier, Wendy L. van der Woerd, Henkjan J. Verkade, Jennifer M. Vittorio, Terese Wallefors, Natalie Warholic, Qifeng Yu, Patrick Horn, Lise Kjems
Summary: The ongoing PEDFIC 2 study evaluated the effectiveness of odevixibat in patients with progressive familial intrahepatic cholestasis. The study found that odevixibat can reduce serum bile acids and alleviate pruritus symptoms. This suggests that odevixibat may be a safe and effective treatment option for patients with progressive familial intrahepatic cholestasis.
Article
Gastroenterology & Hepatology
Anima Sharma, Akash Roy, Madhumita Premkumar, Nipun Verma, Ajay Duseja, Sunil Taneja, Sandeep Grover, Madhu Chopra, Radha K. Dhiman
Summary: Fecal microbiota transplantation (FMT) is safe and improves short-term and medium-term survival, as well as clinical severity scores, in patients with severe alcoholic hepatitis presenting as acute-on-chronic liver failure.
HEPATOLOGY INTERNATIONAL
(2022)
Article
Immunology
Chenhui Ji, Yalin Tang, Yanyan Zhang, Congcong Li, Huazheng Liang, Lu Ding, Xiaohuan Xia, Lize Xiong, Xin-Rui Qi, Jialin C. Zheng
Summary: Glutaminase 1 (GLS1) has been found to play a crucial role in neuroinflammation, particularly in depression. By targeting GLS1 in microglia, it is possible to alleviate neuroinflammation and depression-like behaviors.
BRAIN BEHAVIOR AND IMMUNITY
(2022)
Article
Clinical Neurology
Patrick Vermersch, Luis Brieva-Ruiz, Robert J. Fox, Friedemann Paul, Lluis Ramio-Torrenta, Matthias Schwab, Alain Moussy, Colin Mansfield, Olivier Hermine, Maciej Maciejowski
Summary: The study demonstrated that masitinib has benefits for patients with progressive MS, particularly for those with PPMS and nSPMS. Further phase 3 studies will be initiated to substantiate these findings.
NEUROLOGY-NEUROIMMUNOLOGY & NEUROINFLAMMATION
(2022)
Article
Pharmacology & Pharmacy
Graciela Cardenas, Raul J. Bobes, Gladis Fragoso, Nicolas I. Perez-Osorio, Marisela Hernandez, Alejandro Espinosa, Agnes Fleury, Jose Flores, Juan Pedro Laclette, Edda Sciutto, Helgi Jung-Cook
Summary: Dexamethasone and methylprednisolone are effective glucocorticoids for controlling inflammation. Intranasal administration bypasses the blood-brain barrier and allows direct access to the central nervous system. This study found that intranasal delivery of DXM and MEP has high bioavailability and is comparable to intravenous administration, suggesting it can be a non-invasive alternative for systemic drug delivery.
Article
Oncology
Peter J. DeMaria, Katherine Lee-Wisdom, Renee N. Donahue, Ravi A. Madan, Fatima Karzai, Angie Schwab, Claudia Palena, Caroline Jochems, Charalampos Floudas, Julius Strauss, Jennifer L. Marte, Jason Mark Redman, Eva Dombi, Brigitte Widemann, Borys Korchin, Tatiana Adams, Cesar Pico-Navarro, Christopher Heery, Jeffrey Schlom, James L. Gulley, Marijo Bilusic
Summary: MVA-BN-brachyury-TRICOM is a recombinant vector-based therapeutic cancer vaccine designed to induce an immune response against brachyury. The intravenous administration of the vaccine was found to be safe and tolerable, with the recommended phase 2 dose determined to be 109 infectious units every 3 weeks for three doses. The study showed promising immune activation against brachyury and other tumor-associated antigens.
JOURNAL FOR IMMUNOTHERAPY OF CANCER
(2021)
Article
Medicine, General & Internal
John A. Tayek, Andrew A. Stolz, Danh Nguyen, M. Wayne Fleischman, John A. Donovan, Joseph M. Alcorn, Daniel C-K Chao, Aliya Asghar, Timothy R. Morgan
Summary: This trial aimed to evaluate the safety and efficacy of long-acting recombinant GCSF in patients with AH in the United States. The results showed that there was no significant survival benefit at 90 days in patients treated with pegfilgrastim+prednisolone compared to those treated with prednisolone alone. The incidence of complications and adverse events were similar between the two groups.
Article
Oncology
E. Antonio Chiocca, Arnold B. Gelb, Clark C. Chen, Ganesh Rao, David A. Reardon, Patrick Y. Wen, Wenya Linda Bi, Pierpaolo Peruzzi, Christina Amidei, Dan Triggs, Leah Seften, Grace Park, James Grant, Kyla Truman, Jill Y. Buck, Nira Hadar, Nathan Demars, John Miao, Taylor Estupinan, John Loewy, Kamal Chadha, Joseph Tringali, Laurence Cooper, Rimas Lukas
Summary: The combination of VDX-regulatable IL-12 gene therapy and nivolumab in recurrent glioblastoma patients has shown promising safety and potential therapeutic effects.
Article
Oncology
Yanshuo Cao, Ming Lu, Yu Sun, Jifang Gong, Jie Li, Zhihao Lu, Jian Li, Xiaotian Zhang, Yan Li, Zhi Peng, Jun Zhou, Xicheng Wang, Lin Shen
Summary: This study evaluated the safety and efficacy of the combination treatment of surufatinib and toripalimab in patients with advanced solid tumors, and determined the recommended dose. The results showed that this combination therapy was well tolerated and demonstrated preliminary anti-tumor activity.
JOURNAL OF CANCER RESEARCH AND CLINICAL ONCOLOGY
(2023)
Review
Clinical Neurology
Nicolette Soler, Reinie Cordier, Iain E. Perkes, Russell C. Dale, Paula Bray
Summary: This study evaluated the quality and utility of proxy-reported sensory measures for children and adolescents with neurodevelopmental disorders. Only one measure met the criteria for good measurement properties.
DEVELOPMENTAL MEDICINE AND CHILD NEUROLOGY
(2023)
Article
Clinical Neurology
Davide Martino, Tammy Hedderly, Tara Murphy, Kirsten R. Mueller-Vahl, Russell C. Dale, Donald L. Gilbert, Renata Rizzo, Andreas Hartmann, Peter Nagy, Mathieu Anheim, Tamsin Owen, Osman Malik, Morvwen Duncan, Isobel Heyman, Holan Liang, Andrew McWilliams, Shauna O'Dwyer, Carolin Fremer, Natalia Szejko, Velda X. Han, Kasia Kozlowska, Tamara M. Pringsheim
Summary: There has been a significant increase in adolescents and young adults seeking urgent help for functional tic-like behaviors (FTLBs) between 2019 and 2022. An international collaborative group collected retrospective data to better understand this spectrum and its clinical differences from primary tic disorders. The study findings suggest that social media exposure may be a relevant contributing factor to FTLBs.
EUROPEAN JOURNAL OF NEUROLOGY
(2023)
Letter
Immunology
Yanick J. Crow
JOURNAL OF CLINICAL IMMUNOLOGY
(2023)
Review
Neurosciences
Shrujna Patel, Brooke A. Keating, Russell C. Dale
Summary: Mental health and neurodevelopmental disorders are common and complex conditions that affect wellbeing. There is evidence suggesting a role of maternal inflammation in these disorders, and it is now known that commonly used psychiatric drugs have anti-inflammatory properties. This review summarizes the human evidence of the anti-inflammatory mechanisms of psychiatric drugs and highlights the potential therapeutic implications.
FRONTIERS IN NEUROSCIENCE
(2023)
Article
Cell Biology
Marianna Spatola, Omar Chuquisana, Wonyeong Jung, Joseph A. Lopez, Eva-Maria Wendel, Sudarshini Ramanathan, Christian W. Keller, Tim Hahn, Edgar Meinl, Markus Reindl, Russell C. Dale, Heinz Wiendl, Douglas A. Lauffenburger, Kevin Rostasy, Fabienne Brilot, Galit Alter, Jan D. Luenemann
Summary: Using a systems-level approach, we investigated humoral immune responses in 123 patients with MOGAD. Our study revealed that age is a significant factor in determining MOG-antibody-related immune signatures. Moreover, we identified two dominant immunological endophenotypes of MOGAD, with the pro-inflammatory endophenotype associated with clinically active disease. Our findings suggest that FcyR-mediated effector functions control the pathogenicity of MOG-specific IgG and highlight the potential of FcyR-targeting therapies in treating MOGAD.
CELL REPORTS MEDICINE
(2023)
Article
Clinical Neurology
Rachel Sze Hui Wong, Shekeeb Mohammad, Bindu Parayil Sankaran, Rosie Junek, Won-Tae Kim, Tiffany Wotton, Beena Devanapalli, Sushil Bandodkar, Shanti Balasubramaniam
Summary: This study reports the clinical manifestations and genetic variants of hyperphenylalaninemia and biogenic amines deficiency caused by DNAJC12 deficiency. Urine, cerebrospinal fluid neurotransmitter studies, and genetic testing can differentiate between different related diseases.
BRAIN & DEVELOPMENT
(2023)
Review
Clinical Neurology
Katarina Ostojic, Isra P. Karem, Simon P. Paget, Alison Berg, Betty-Jean Dee-Price, Raghu C. Lingam, Russell C. Dale, Valsamma Eapen, Sue Woolfenden
Summary: Social determinants of health (SDH) have both positive and negative impacts on health and social outcomes. Understanding how SDH affects children with cerebral palsy (CP) is crucial for improving health equity, optimizing health outcomes, and supporting their families. This review examines the impact of SDH on CP children globally. In high-income countries, socioeconomic disadvantage is associated with more severe comorbidities, spastic bilateral CP, and reduced community participation. In low- and middle-income countries, socioeconomic disadvantage is linked to malnutrition, poor housing conditions, lack of sanitation, and poverty.
DEVELOPMENTAL MEDICINE AND CHILD NEUROLOGY
(2023)
Review
Clinical Neurology
Sudarshini Ramanathan, Fabienne Brilot, Sarosh R. Irani, Russell C. Dale
Summary: This article reviews the key pathogenic mechanisms underlying the development of autoimmune diseases in the central nervous system, including non-modifiable risk factors, interactions between environmental factors and epigenetics, disease mechanisms associated with the loss of immune tolerance, the role of neuroimmune interfaces, and novel therapeutic approaches based on the immunopathogenesis.
NATURE REVIEWS NEUROLOGY
(2023)
Article
Medicine, General & Internal
Catherine Morgan, Nadia Badawi, Roslyn N. Boyd, Alicia J. Spittle, Russell C. Dale, Adrienne Kirby, Rod W. Hunt, Koa Whittingham, Kerstin Pannek, Rachael L. Morton, William Tarnow-Mordi, Michael C. Fahey, Karen Walker, Kristina Prelog, Catherine Elliott, Jane Valentine, Andrea Guzzetta, Shannon Olivey, GAME Study Team, Iona Novak
Summary: This is a randomized controlled trial on early intervention for cerebral palsy (CP) children. The trial aims to investigate the efficacy of an early and sustained Goals-Activity-Motor Enrichment approach on improving motor and cognitive skills in infants with suspected or confirmed CP. The study requires 150 participants per group and the outcomes will be disseminated through peer-reviewed journal publications, international conferences, and consumer websites.
Article
Medicine, General & Internal
Katarina Ostojic, Simon Paget, Tanya Martin, Betty-Jean Dee-Price, Sarah McIntyre, Hayley Smithers Sheedy, Laurel Mimmo, Heather Burnett, Timothy Scott, Alison Berg, Anne Masi, Michele Scarcella, Jack Calderan, Sheikh Azmatullah, Masyitah Mohamed, Mackenzie Woodbury, Alunya Wilkinson, Karen Zwi, Russell Dale, Valsamma Eapen, Raghu Lingam, Iva Strnadova, Susan Woolfenden
Summary: This study aims to codesign a social prescribing programme, in order to address social determinants of health concerns faced by children with cerebral palsy and their families in Australia. The study will involve young adults with cerebral palsy, parents/caregivers of children with cerebral palsy, and clinicians in the design process, and will be guided by the biopsychosocial ecological framework.
Article
Clinical Neurology
Susanna Asseyer, Nasrin Asgari, Jeffrey Bennett, Omer Bialer, Yolanda Blanco, Francesca Bosello., Anna Camos-Carreras., Edgar Carnero Contentti., Sara Carta, John Chen, Claudia Chien, Mashina Chomba, Russell C. Dale, Josep Dalmau, Kristina Feldmann, Eoin P. Flanagan, Caroline Froment Tilikete, Carolina Garcia-Alfonso, Joachim Havla, Mark Hellmann, Ho Jin Kim, Philipp Klyscz, Frank Konietschke, Chiara La Morgia, Marco Lana-Peixoto, Maria Isabel Leite, Netta Levin, Michael Levy, Sara Llufriu, Pablo Lopez, Itay Lotan, Alessandra Lugaresi, Romain Marignier, Sara Mariotto, Susan P. Mollan, Cassandra Ocampo, Frederike Cosima Oertel, Maja Olszewska, Jacqueline Palace, Lekha Pandit, Jose Luis Peralta Uribe, Sean Pittock, Sudarshini Ramanathan, Natthapon Rattanathamsakul, Albert Saiz, Sara Samadzadeh, Bernardo Sanchez-Dalmau, Deanna Saylor, Michael Scheel, Tanja Schmitz-Huebsch, Jemal Shifa, Sasitorn Siritho, Pia S. Sperber, Prem S. Subramanian, Alon Tiosano, Adi Vaknin-Dembinsky, Alvaro Jose Mejia Vergara, Adi Wilf-Yarkoni, Luis Alfonso Zarco, Hanna G. Zimmermann, Friedemann Paul, Hadas Stiebel-Kalish
Summary: This is a global study aimed at investigating the effect of early high-dose corticosteroid treatment on optic neuritis. The study will collect various data to evaluate treatment strategies and the accuracy of diagnostic criteria.
FRONTIERS IN NEUROLOGY
(2023)
Article
Medicine, General & Internal
Jingya Yan, Kavitha Kothur, Shekeeb Mohammad, Jason Chung, Shrujna Patel, Hannah F. Jones, Brooke A. Keating, Velda X. Han, Richard Webster, Simone Ardern-Holmes, Jayne Antony, Manoj P. Menezes, Esther Tantsis, Deepak Gill, Sachin Gupta, Tejaswi Kandula, Hugo Sampaio, Michelle A. Farrar, Christopher Troedson, P. Ian Andrews, Sekhar C. Pillai, Benjamin Heng, Gilles J. Guillemin, Anna Guller, Sushil Bandodkar, Russell C. Dale
Summary: CSF metabolites, including neopterin, quinolinic acid, and KYN/TRP, were found to be useful biomarkers for the diagnosis and monitoring of neuroinflammation. These findings provide insights into the role of inflammatory metabolism in neurological disorders and offer opportunities for improved management of these diseases.
Review
Clinical Neurology
Tamara Pringsheim, Amit Batla, Ali Shalash, Jitendra Kumar Sahu, Carlos Cosentino, Darius Ebrahimi-Fakhari, Jennifer Friedman, Jean-Pierre Lin, Jonathan Mink, Alexander Munchau, Daniela Munoz, Nardo Nardocci, Belen Perez-Duenas, Zomer Sardar, Chahnez Triki, Hilla Ben-Pazi, Laura Silveira-Moriyama, Monica Troncoso-Schifferli, Kyoko Hoshino, Russell C. Dale, Victor S. C. Fung, Manju A. Kurian, Emmanuel Roze
Summary: The International Parkinson and Movement Disorders Society (MDS) established a working group to provide recommendations for the transition process from pediatric to adult healthcare systems for patients with childhood-onset movement disorders. Through a multi-round, web-based Delphi survey, the group developed recommendations related to team composition and structure, planning and readiness, goals of care, and administration and research.
MOVEMENT DISORDERS CLINICAL PRACTICE
(2023)
Article
Immunology
Kenza Rhzioual Berrada, Alexandre Belot, Benedicte Neven, Camille Ohlmann, Francois Tronc, Gillian Rice, Guillaume Thouvenin, Jean-Christophe Dubus, Julie Mazenq, Marie-Louise Fremond, Nathalie Stremler, Severine Soummer-Feuillet, Vincent Cottin, Philippe Reix
Summary: STING-associated vasculopathy with onset in infancy (SAVI) is a rare autoinflammatory disease characterized by fever attacks and skin and respiratory manifestations. Respiratory involvement occurs in 80% of cases and may require lung transplantation. Three patients with SAVI who underwent lung transplantation have been reported, but their diagnosis was made after transplantation, preventing targeted therapy.
JOURNAL OF CLINICAL IMMUNOLOGY
(2023)
Article
Clinical Neurology
Nimra Feroze, Tasneem Karim, Katarina Ostojic, Sarah Mcintyre, Elizabeth H. Barnes, Byoung Chan Lee, Nadia Badawi, Shekeeb S. Mohammad, Simon Paget, Russell C. Dale, Deepak Gill, Kavitha Kothur
Summary: This study reveals important clinical associations between epilepsy, its resolution, and treatment response in children with cerebral palsy (CP), providing valuable knowledge to aid in counseling families and identifying distinct prognostic groups for effective medical surveillance and optimal treatment.
DEVELOPMENTAL MEDICINE AND CHILD NEUROLOGY
(2023)
