Article
Neurosciences
Nirosen Vijiaratnam, Thomas Foltynie
Summary: Treatment of Parkinson's disease is currently limited to symptomatic therapies, with the goal of halting or reversing disease progression remaining unmet. Utilizing new treatment targets and biomarkers may improve outcome measurements in clinical trials. Exploring disease mechanisms and targeting peripheral interfaces may hold promise for disease modification.
Review
Immunology
Georges Jalkh, Rachelle Abi Nahed, Gabrielle Macaron, Mary Rensel
Summary: In the past decade, the therapeutic options for multiple sclerosis have greatly expanded, with newer and more effective disease modifying therapies being increasingly used early in the disease course. Despite their advantages in controlling disease activity and improving long-term outcomes, these newer therapies come with safety concerns and monitoring requirements that highlight the need for periodic re-evaluation and adjustment of monitoring strategies for optimizing treatment safety in an individualized manner.
Review
Clinical Neurology
Todd E. Golde
Summary: Scientific advances have brought optimism to the field of Alzheimer's disease, but the disease is still largely untreatable. The article reviews the major therapeutic approaches and open questions, and discusses the need to adjust our approach to ensure future success.
Review
Cardiac & Cardiovascular Systems
Jacqueline T. Vuong, Ashley F. Stein-Merlob, Arash Nayeri, Tamer Sallam, Tomas G. Neilan, Eric H. Yang
Summary: Immune checkpoint inhibitor therapy has revolutionized the treatment of advanced malignancies, but it may contribute to the development and exacerbation of atherosclerosis, highlighting the need for close monitoring and management.
JOURNAL OF THE AMERICAN COLLEGE OF CARDIOLOGY
(2022)
Review
Clinical Neurology
Dylan C. Brock, Scott Demarest, Tim A. Benke
Summary: Although trials with anti-seizure medications have not shown clear anti-epileptogenic activity in humans, advancements in genomic technology and emerging gene-mediated therapies offer hope for the successful development of disease-modifying therapies for genetic epilepsies. More than 26 potential DMTs are in various stages of development, showing promise for altering the natural history and improving the quality of life for patients with genetic epilepsy syndromes.
Article
Clinical Neurology
Steve Simpson-Yap, Edward De Brouwer, Tomas Kalincik, Nick Rijke, Jan A. Hillert, Clare Walton, Gilles Edan, Yves Moreau, Tim Spelman, Lotte Geys, Tina Parciak, Clement Gautrais, Nikola Lazovski, Ashkan Pirmani, Amin Ardeshirdavanai, Lars Forsberg, Anna Glaser, Robert McBurney, Hollie Schmidt, Arnfin B. Bergmann, Stefan Braune, Alexander Stahmann, Rodden Middleton, Amber Salter, Robert J. Fox, Anneke van der Walt, Helmut Butzkueven, Raed Alroughani, Serkan Ozakbas, Juan Rojas, Ingrid van der Mei, Nupur Nag, Rumen Ivanov, Guilherme Sciascia do Olival, Alice Estavo Dias, Melinda Magyari, Doralina Brum, Maria Fernanda Mendes, Ricardo N. Alonso, Richard S. Nicholas, Johana Bauer, Anibal Sebastian Chertcoff, Anna Zabalza, Georgina Arrambide, Alexander Fidao, Giancarlo Comi, Liesbet Peeters
Summary: Using data from 12 sources in 28 countries, this study examined the characteristics of COVID-19 severity in people with MS, particularly those taking immunosuppressive therapies. The results showed that older age, progressive MS phenotype, and higher disability were associated with worse COVID-19 outcomes. Additionally, rituximab was consistently associated with increased risk of hospitalization, ICU admission, and need for ventilation, while ocrelizumab was mainly associated with hospitalization and ICU admission.
Review
Biochemistry & Molecular Biology
Niranjan G. Kotla, Yury Rochev
Summary: The pathogenesis of inflammatory bowel disease (IBD) is characterized by gut mucosal inflammation, epithelial damage, and dysbiosis, resulting in a disturbed gut mucosal barrier. Current treatment regimens focus on symptom relief but do not address mucosal epithelial repair, barrier homeostasis, or intestinal dysbiosis. Efforts to identify new therapeutic modalities to enhance gut barrier functions are still in the early developmental stage and have not been entirely successful. In this review, we discuss conventional therapies, the potential role of gut barrier-protecting agents, and biomaterial strategies related to combination therapies that may lead to the development of new therapeutic approaches for IBD.
TRENDS IN MOLECULAR MEDICINE
(2023)
Review
Clinical Neurology
Francesca Washington, Dawn Langdon
Summary: Research shows that patients with multiple sclerosis often struggle with treatment adherence, influenced by factors such as age, gender, emotions, cognition, and treatment satisfaction. Targeted patient support programs should be developed to improve treatment compliance.
JOURNAL OF NEUROLOGY
(2022)
Review
Biochemistry & Molecular Biology
Marina Stavrou, Irene Sargiannidou, Elena Georgiou, Alexia Kagiava, Kleopas A. Kleopa
Summary: CMT disease is a genetically heterogeneous disorder affecting the peripheral nerves, with diverse molecular genetic mechanisms discovered over the past three decades. There are currently various treatment approaches in preclinical testing and clinical trials, including disease-specific targeted therapies and treatments targeting common pathways shared by different CMT types. As promising treatments advance to clinical translation, optimizing outcome measures, novel biomarkers, and appropriate trial designs are crucial to facilitate successful testing and validation of novel treatments for CMT patients.
INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES
(2021)
Review
Neurosciences
Eiko N. Minakawa, Yoshitaka Nagai
Summary: Polyglutamine diseases are a group of inherited neurodegenerative disorders caused by abnormal expansion of CAG trinucleotide repeats. Stabilizing protein conformation and inhibiting aggregation offer promising therapeutic approaches, while gene silencing to reduce expression of expanded polyQ proteins is another potential treatment avenue. These advancements hold promise for delaying or preventing the progression of currently incurable diseases.
FRONTIERS IN NEUROSCIENCE
(2021)
Review
Clinical Neurology
Daniel M. Hartung
Summary: The high costs of multiple sclerosis disease-modifying therapies (DMTs) in the USA, averaging $70,000 a year, have created economic hardships for patients. These high prices are the main reason why the economic value of most DMTs is questionable.
CURRENT NEUROLOGY AND NEUROSCIENCE REPORTS
(2021)
Review
Chemistry, Medicinal
Win Min Oo, Christopher Little, Vicky Duong, David J. Hunter
Summary: This review discusses the need for disease modifying drugs (DMOADs) for the management of osteoarthritis (OA), the classification of clinical phenotypes or molecular/mechanistic endotypes for targeted drug discovery, and summarizes the efficacy and safety of targeted drugs in Phase 2 and 3 clinical trials targeting cartilage-driven, bone-driven, and inflammation-driven endotypes. The reasons for failures in OA clinical trials and possible steps to overcome these barriers are also briefly presented.
DRUG DESIGN DEVELOPMENT AND THERAPY
(2021)
Article
Medicine, Research & Experimental
Martin Qui, Nina Le Bert, Webber Pak Wo Chan, Malcolm Tan, Shou Kit Hang, Smrithi Hariharaputran, Jean Xiang Ying Sim, Jenny Guek Hong Low, Weiling Ng, Wei Yee Wan, Tiing Leong Ang, Antonio Bertoletti, Ennaliza Salazar
Summary: Patients with inflammatory bowel disease under immune-modifying therapies show a favorable profile of vaccine-induced T cell responses, providing potential COVID-19 protection despite reduced humoral response.
JOURNAL OF CLINICAL INVESTIGATION
(2022)
Review
Pharmacology & Pharmacy
Peter Alping
Summary: This study aimed to summarize the current evidence for rituximab as a treatment for relapsing-remitting MS (RRMS).
BASIC & CLINICAL PHARMACOLOGY & TOXICOLOGY
(2023)
Article
Economics
Jakub P. Hlavka, Bryan Tysinger, Jeffrey C. Yu, Darius N. Lakdawalla
Summary: Aduhelm is the first approved disease-modifying therapy for Alzheimer's disease. However, under current payment models, AD DMTs may pose challenges to patient access due to costs accruing before benefits do. New payment approaches may be needed to address this difference in timing.
Article
Clinical Neurology
Carolina G. Nicoletti, Fabrizia Monteleone, Girolama A. Marfia, Alessandro Usiello, Fabio Buttari, Diego Centonze, Francesco Mori
MULTIPLE SCLEROSIS JOURNAL
(2020)
Article
Clinical Neurology
Claudio Liguori, Roberta Ruffini, Enrica Olivola, Agostino Chiaravalloti, Francesca Izzi, Alessandro Stefani, Mariangela Pierantozzi, Nicola Biagio Mercuri, Nicola Modugno, Diego Centonze, Orazio Schillaci, Fabio Placidi
PARKINSONISM & RELATED DISORDERS
(2019)
Article
Neurosciences
Francesca Grassivaro, Ramesh Menon, Massimo Acquaviva, Linda Ottoboni, Francesca Ruffini, Andrea Bergamaschi, Luca Muzio, Cinthia Farina, Gianvito Martino
JOURNAL OF NEUROSCIENCE
(2020)
Review
Urology & Nephrology
Davide Viggiano, Carsten A. Wagner, Gianvito Martino, Maiken Nedergaard, Carmine Zoccali, Robert Unwin, Giovambattista Capasso
NATURE REVIEWS NEPHROLOGY
(2020)
Article
Immunology
Emanuela Colombo, Claudia Bassani, Anthea De Angelis, Francesca Ruffini, Linda Ottoboni, Giancarlo Comi, Gianvito Martino, Cinthia Farina
FRONTIERS IN IMMUNOLOGY
(2020)
Article
Clinical Neurology
Laura Starost, Maren Lindner, Martin Herold, Yu Kang T. Xu, Hannes C. A. Drexler, Katharina Hess, Marc Ehrlich, Linda Ottoboni, Francesca Ruffini, Martin Stehling, Albrecht Roepke, Christian Thomas, Hans R. Schoeler, Jack Antel, Juergen Winkler, Gianvito Martino, Luisa Klotz, Tanja Kuhlmann
ACTA NEUROPATHOLOGICA
(2020)
Article
Biochemistry & Molecular Biology
Emanuela Colombo, Rosaria Pascente, Daniela Triolo, Claudia Bassani, Anthea De Angelis, Francesca Ruffini, Linda Ottoboni, Giancarlo Comi, Gianvito Martino, Cinthia Farina
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
Multidisciplinary Sciences
Erica Butti, Stefano Cattaneo, Marco Bacigaluppi, Marco Cambiaghi, Giulia Maria Scotti, Elena Brambilla, Francesca Ruffini, Giacomo Sferruzza, Maddalena Ripamonti, Fabio Simeoni, Laura Cacciaguerra, Aurora Zanghi, Angelo Quattrini, Riccardo Fesce, Paola Panina-Bordignon, Francesca Giannese, Davide Cittaro, Tanja Kuhlmann, Patrizia D'Adamo, Maria Assunta Rocca, Stefano Taverna, Gianvito Martino
Summary: In this study, the researchers provide evidence that adult neural stem/precursor cells in the brain regulate striatal function and support cognitive functions through the secretion of IGFBPL1.
NATURE COMMUNICATIONS
(2022)
Article
Neurosciences
Emanuela Colombo, Anthea De Angelis, Claudia Bassani, Francesca Ruffini, Linda Ottoboni, Livia Garzetti, Annamaria Finardi, Gianvito Martino, Roberto Furlan, Cinthia Farina
Summary: This study investigates the immunomodulatory properties of astrocytes in different age, sex, and species. It shows that adult astrocytes are more efficient in inhibiting T lymphocyte activation compared to neonatal astrocytes. Furthermore, astrocytes derived from reprogrammed fibroblasts do not interfere with T cell proliferation.
Meeting Abstract
Clinical Neurology
S. Bezukladova, F. Ruffini, L. Ottoboni, E. Brambilla, M. Absinta, F. Fagiani, C. Peri, A. Mandelli, V. Murtaj, S. Taverna, X. Gao, S. Baranzini, P. Panina-Bordignon, G. Martino
MULTIPLE SCLEROSIS JOURNAL
(2022)
Meeting Abstract
Clinical Neurology
F. Fagiani, E. Pedrini, E. Brambilla, F. Ruffini, P. Podini, A. Quattrini, L. Smirnova, G. Martino, P. Panina, M. Absinta
MULTIPLE SCLEROSIS JOURNAL
(2022)
Meeting Abstract
Clinical Neurology
V. Murtaj, D. Cittaro, F. Ruffini, F. Giannese, G. Tonon, G. Martino, P. Panina-Bordignon
MULTIPLE SCLEROSIS JOURNAL
(2022)