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https://www.readbyqxmd.com/read/28742871/comprehensive-immune-profiling-reveals-substantial-immune-system-alterations-in-a-subset-of-patients-with-amyotrophic-lateral-sclerosis
#1
Michael P Gustafson, Nathan P Staff, Svetlana Bornschlegl, Greg W Butler, Mary L Maas, Mohamed Kazamel, Adeel Zubair, Dennis A Gastineau, Anthony J Windebank, Allan B Dietz
Amyotrophic lateral sclerosis (ALS) is a fatal neurodegenerative disease with a median lifespan of 2-3 years after diagnosis. There are few meaningful treatments that alter progression in this disease. Preclinical and clinical studies have demonstrated that neuroinflammation may play a key role in the progression rate of ALS. Despite this, there are no validated biomarkers of neuroinflammation for use in clinical practice or clinical trials. Biomarkers of neuroinflammation could improve patient management, provide new therapeutic targets, and possibly help stratify clinical trial selection and monitoring...
2017: PloS One
https://www.readbyqxmd.com/read/28710685/pericytes-extend-survival-of-als-sod1-mice-and-induce-the-expression-of-antioxidant-enzymes-in-the-murine-model-and-in-ipscs-derived-neuronal-cells-from-an-als-patient
#2
Giuliana Castello Coatti, Miriam Frangini, Marcos C Valadares, Juliana Plat Gomes, Natalia O Lima, Natale Cavaçana, Amanda F Assoni, Mayra V Pelatti, Alexander Birbrair, Antonio Carlos Pedroso de Lima, Julio M Singer, Francisco Marcelo M Rocha, Giovani Loiola Da Silva, Mario Sergio Mantovani, Lucia Inês Macedo-Souza, Merari F R Ferrari, Mayana Zatz
Amyotrophic Lateral Sclerosis (ALS) is one of the most common adult-onset motor neuron disease causing a progressive, rapid and irreversible degeneration of motor neurons in the cortex, brain stem and spinal cord. No effective treatment is available and cell therapy clinical trials are currently being tested in ALS affected patients. It is well known that in ALS patients, approximately 50% of pericytes from the spinal cord barrier are lost. In the central nervous system, pericytes act in the formation and maintenance of the blood-brain barrier, a natural defense that slows the progression of symptoms in neurodegenerative diseases...
July 14, 2017: Stem Cell Reviews
https://www.readbyqxmd.com/read/28705085/international-survey-of-als-experts-about-critical-questions-for-assessing-patients-with-als
#3
Mamede De Carvalho, Adam Ryczkowski, Peter Andersen, Marta Gromicho, Julian Grosskreutz, Magdalena Kuźma-Kozakiewicz, Susanne Petri, Maria Piotrkiewicz, Gabriel Miltenberger Miltenyi
OBJECTIVE: To define an applicable dataset for ALS patient registries we weighted specific clinical items as scored by worldwide ALS experts. METHODS: Sixty participants were invited based on relevant clinical work, publications and personal acquaintance. They rated 160 clinical items consensually agreed by the members of our project, incorporating specialists from five European Centres. Scoring scheme was defined as: 1 - essential; 2 - important; 3 - not very important...
July 13, 2017: Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration
https://www.readbyqxmd.com/read/28674995/biomarkers-in-neurodegenerative-diseases
#4
Andreas Jeromin, Robert Bowser
The past decade has seen tremendous efforts in biomarker discovery and validation for neurodegenerative diseases. The source and type of biomarkers has continued to grow for central nervous system diseases, from biofluid-based biomarkers (blood or cerebrospinal fluid (CSF)), to nucleic acids, tissue, and imaging. While DNA remains a predominant biomarker used to identify familial forms of neurodegenerative diseases, various types of RNA have more recently been linked to familial and sporadic forms of neurodegenerative diseases during the past few years...
2017: Advances in Neurobiology
https://www.readbyqxmd.com/read/28674982/motoneuron-disease-clinical
#5
Hristelina Ilieva, Nicholas J Maragakis
ALS is a neurodegenerative disease in which the primary symptoms result in progressive neuromuscular weakness. Recent studies have highlighted that there is significant heterogeneity with regard to anatomical and temporal disease progression. Importantly, more recent advances in genetics have revealed new causative genes to the disease. New efforts have focused on the development of biomarkers that could aid in diagnosis, prognosis, and serve as pharmacodynamics markers. Although traditional pharmaceuticals continue to undergo trials for ALS, new therapeutic strategies including stem cell transplantation studies, gene therapies, and antisense therapies targeting some of the familial forms of ALS are gaining momentum...
2017: Advances in Neurobiology
https://www.readbyqxmd.com/read/28664036/virtual-brain-biopsies-in-amyotrophic-lateral-sclerosis-diagnostic-classification-based-on-in-vivo-pathological-patterns
#6
Peter Bede, Parameswaran M Iyer, Eoin Finegan, Taha Omer, Orla Hardiman
BACKGROUND: Diagnostic uncertainty in ALS has serious management implications and delays recruitment into clinical trials. Emerging evidence of presymptomatic disease-burden provides the rationale to develop diagnostic applications based on the evaluation of in-vivo pathological patterns early in the disease. OBJECTIVES: To outline and test a diagnostic classification approach based on an array of complementary imaging metrics in key disease-associated anatomical structures...
2017: NeuroImage: Clinical
https://www.readbyqxmd.com/read/28628244/phosphorylated-neurofilament-heavy-chain-a-biomarker-of-survival-for-c9orf72-associated-amyotrophic-lateral-sclerosis
#7
Tania F Gendron, Lillian M Daughrity, Michael G Heckman, Nancy N Diehl, Joanne Wuu, Timothy M Miller, Pau Pastor, John Q Trojanowski, Murray Grossman, James D Berry, William T Hu, Antonia Ratti, Michael Benatar, Vincenzo Silani, Jonathan D Glass, Mary Kay Floeter, Andreas Jeromin, Kevin B Boylan, Leonard Petrucelli
As potential treatments for C9ORF72-associated amyotrophic lateral sclerosis (c9ALS) approach clinical trials, the identification of prognostic biomarkers for c9ALS becomes a priority. We show that levels of phosphorylated neurofilament heavy chain (pNFH) in cerebrospinal fluid (CSF) predict disease status and survival in c9ALS patients, and are largely stable over time. Moreover, c9ALS patients exhibit higher pNFH levels, more rapid disease progression, and shorter survival after disease onset than ALS patients without C9ORF72 expansions...
June 19, 2017: Annals of Neurology
https://www.readbyqxmd.com/read/28619000/astrocytes-pathology-in-als-a-potential-therapeutic-target
#8
Sonja Johann
The mechanisms underlying neurodegeneration in amyotrophic lateral sclerosis (ALS) are multifactorial and include genetic and environmental factors. Nowadays, it is well accepted that neuronal loss is driven by non-cell autonomous toxicity. Non-neuronal cells, such as astrocytes, have been described to significantly contribute to motoneuron cell death and disease progression in cell culture experiments and animal models of ALS. Astrocytes are essential for neuronal survival and function by regulating neurotransmitter and ion homeostasis, immune response, blood flow and glucose uptake, antioxidant defence and growth factor release...
June 15, 2017: Current Pharmaceutical Design
https://www.readbyqxmd.com/read/28608303/assessment-of-the-factorial-validity-and-reliability-of-the-alsfrs-r-a-revision-of-its-measurement-model
#9
Leonhard A Bakker, Carin D Schröder, Michael A van Es, Paul Westers, Johanna M A Visser-Meily, Leonard H van den Berg
The amyotrophic lateral sclerosis functional rating scale-revised (ALSFRS-R) is a widely used primary outcome measure in amyotrophic lateral sclerosis (ALS) clinical practice and clinical trials. ALSFRS-R items cannot, however, validly be summed to obtain a total score, but constitute domain scores reflecting a profile of disease severity. Currently, there are different measurement models for estimating domain scores. The objective of the present study is, therefore, to derive the measurement model that best fits the data for a valid and uniform estimation of ALSFRS-R domain scores...
July 2017: Journal of Neurology
https://www.readbyqxmd.com/read/28600459/patients-with-als-show-highly-correlated-progression-rates-in-left-and-right-limb-muscles
#10
David J Rushton, Patricia L Andres, Peggy Allred, Robert H Baloh, Clive N Svendsen
OBJECTIVE: Amyotrophic lateral sclerosis (ALS) progresses at different rates between patients, making clinical trial design difficult and dependent on large cohorts of patients. Currently, there are few data showing whether the left and right limbs progress at the same or different rates. This study addresses rates of decline in specific muscle groups of patients with ALS and assesses whether there is a relationship between left and right muscles in the same patient, regardless of overall progression...
July 11, 2017: Neurology
https://www.readbyqxmd.com/read/28599400/the-use-of-mesenchymal-stem-cells-mscs-for-amyotrophic-lateral-sclerosis-als-therapy-a-perspective-on-cell-biological-mechanisms
#11
Bor Luen Tang
Recent clinical trials of mesenchymal stem cells (MSCs) transplantation have demonstrated procedural safety and clinical proof of principle with a modest indication of benefit in patients with amyotrophic lateral sclerosis (ALS). While replacement therapy remained unrealistic, the clinical efficacy of this therapeutic option could be potentially enhanced if we could better decipher the mechanisms underlying some of the beneficial effects of transplanted cells, and work toward augmenting or combining these in a strategic manner...
May 25, 2017: Reviews in the Neurosciences
https://www.readbyqxmd.com/read/28569393/multidimensional-latent-trait-linear-mixed-model-an-application-in-clinical-studies-with-multivariate-longitudinal-outcomes
#12
Jue Wang, Sheng Luo
Multilevel item response theory (MLIRT) models have been widely used to analyze the multivariate longitudinal data of mixed types (e.g., categorical and continuous) in clinical studies. The MLIRT models often have unidimensional assumption, that is, the multiple outcomes are clinical manifestations of a univariate latent variable. However, the unidimensional assumption may be unrealistic because some diseases may be heterogeneous and characterized by multiple impaired domains with variable clinical symptoms and disease progressions...
June 1, 2017: Statistics in Medicine
https://www.readbyqxmd.com/read/28561886/improving-symptom-management-for-people-with-amyotrophic-lateral-sclerosis
#13
Katharine Nicholson, Alyssa Murphy, Erin McDonnell, Jordan Shapiro, Ericka Simpson, Jonathan Glass, Hiroshi Mitsumoto, Dallas Forshew, Robert Miller, Nazem Atassi
INTRODUCTION: Symptomatic management is the main focus of ALS clinical care. We aim to report the prevalence of ALS-related symptoms and characterize self-reported symptomatic management. METHODS: A symptom management survey developed by the MDA Clinical Research Network was completed by ALS registrants. Logistic regression identified potential predictors of symptom prevalence, severity, and treatment. RESULTS: 567 ALS participants reported fatigue (90%), muscle stiffness (84%), and muscle cramps (74%) as most prevalent symptoms...
May 31, 2017: Muscle & Nerve
https://www.readbyqxmd.com/read/28552237/ex-vivo-gene-therapy-for-the-treatment-of-neurological-disorders
#14
Genevieve Gowing, Soshana Svendsen, Clive N Svendsen
Ex vivo gene therapy involves the genetic modification of cells outside of the body to produce therapeutic factors and their subsequent transplantation back into patients. Various cell types can be genetically engineered. However, with the explosion in stem cell technologies, neural stem/progenitor cells and mesenchymal stem cells are most often used. The synergy between the effect of the new cell and the additional engineered properties can often provide significant benefits to neurodegenerative changes in the brain...
2017: Progress in Brain Research
https://www.readbyqxmd.com/read/28539871/protein-quality-control-and-the-amyotrophic-lateral-sclerosis-frontotemporal-dementia-continuum
#15
Hamideh Shahheydari, Audrey Ragagnin, Adam K Walker, Reka P Toth, Marta Vidal, Cyril J Jagaraj, Emma R Perri, Anna Konopka, Jessica M Sultana, Julie D Atkin
Protein homeostasis, or proteostasis, has an important regulatory role in cellular function. Protein quality control mechanisms, including protein folding and protein degradation processes, have a crucial function in post-mitotic neurons. Cellular protein quality control relies on multiple strategies, including molecular chaperones, autophagy, the ubiquitin proteasome system, endoplasmic reticulum (ER)-associated degradation (ERAD) and the formation of stress granules (SGs), to regulate proteostasis. Neurodegenerative diseases are characterized by the presence of misfolded protein aggregates, implying that protein quality control mechanisms are dysfunctional in these conditions...
2017: Frontiers in Molecular Neuroscience
https://www.readbyqxmd.com/read/28527102/studies-of-environmental-risk-factors-in-amyotrophic-lateral-sclerosis-als-and-a-phase-i-clinical-trial-of-l-serine
#16
Walter G Bradley, R X Miller, T D Levine, E W Stommel, P A Cox
β-N-Methylamino-L-alanine (BMAA) has been linked to Guam ALS/PDC and shown to produce neurodegeneration in vitro and in vivo (Drosophila, mice, rats, primates). BMAA misincorporation into neuroproteins produces protein misfolding and is inhibited by L-serine. Case-control studies in Northern New England indicate that living near to water-bodies with cyanobacterial blooms increases the risk of developing amyotrophic lateral sclerosis (ALS). The distribution of addresses of ALS cases in New Hampshire, Vermont, and Florida was compared to that of controls...
May 19, 2017: Neurotoxicity Research
https://www.readbyqxmd.com/read/28527045/amyotrophic-lateral-sclerosis-like-superoxide-dismutase-1-proteinopathy-is-associated-with-neuronal-loss-in-parkinson-s-disease-brain
#17
Benjamin G Trist, Katherine M Davies, Veronica Cottam, Sian Genoud, Richard Ortega, Stéphane Roudeau, Asuncion Carmona, Kasun De Silva, Valerie Wasinger, Simon J G Lewis, Perminder Sachdev, Bradley Smith, Claire Troakes, Caroline Vance, Christopher Shaw, Safa Al-Sarraj, Helen J Ball, Glenda M Halliday, Dominic J Hare, Kay L Double
Neuronal loss in numerous neurodegenerative disorders has been linked to protein aggregation and oxidative stress. Emerging data regarding overlapping proteinopathy in traditionally distinct neurodegenerative diseases suggest that disease-modifying treatments targeting these pathological features may exhibit efficacy across multiple disorders. Here, we describe proteinopathy distinct from classic synucleinopathy, predominantly comprised of the anti-oxidant enzyme superoxide dismutase-1 (SOD1), in the Parkinson's disease brain...
July 2017: Acta Neuropathologica
https://www.readbyqxmd.com/read/28480639/pyrimethamine-significantly-lowers-cerebrospinal-fluid-cu-zn-superoxide-dismutase-in-amyotrophic-lateral-sclerosis-patients-with-sod1-mutations
#18
MULTICENTER STUDY
Dale J Lange, Mona Shahbazi, Vincenzo Silani, Albert C Ludolph, Jochen H Weishaupt, Senda Ajroud-Driss, Kara G Fields, Rahul Remanan, Stanley H Appel, Claudia Morelli, Alberto Doretti, Luca Maderna, Stefano Messina, Ulrike Weiland, Stefan L Marklund, Peter M Andersen
OBJECTIVE: Cu/Zn superoxide dismutase (SOD1) reduction prolongs survival in SOD1-transgenic animal models. Pyrimethamine produces dose-dependent SOD1 reduction in cell culture systems. A previous phase 1 trial showed pyrimethamine lowers SOD1 levels in leukocytes in patients with SOD1 mutations. This study investigated whether pyrimethamine lowered SOD1 levels in the cerebrospinal fluid (CSF) in patients carrying SOD1 mutations linked to familial amyotrophic lateral sclerosis (fALS/SOD1)...
June 2017: Annals of Neurology
https://www.readbyqxmd.com/read/28465220/the-six-minute-walk-6mwt-as-a-measure-walking-capacity-wc-in-ambulatory-individual-with-amyotrophic-lateral-sclerosis-ambals
#19
Mohammed Sanjak, Velma Langford, Scott Holsten, Nigel Rozario, Charity G Moore Patterson, Elena Bravver, William L Bockenek, Benjamin R Brooks
OBJECTIVE: To determine the validity of the 6MWT as an outcome measure to evaluate WC in ambALS. DESIGN: Observational study. Participants performed 6MWT, 25FWT, TUG, lower extremity maximum voluntary isometric contraction (MVIC), ALSFRS-R, and forced vital capacity (FVC). SETTING: Multidisciplinary ALS clinic at an academic medical center. PARTICIPANTS: AmbALS (N=186) who ambulate without (stage I) or with (stage II) assistive device...
April 29, 2017: Archives of Physical Medicine and Rehabilitation
https://www.readbyqxmd.com/read/28456383/adult-onset-spinal-muscular-atrophy-an-update
#20
REVIEW
R Juntas Morales, N Pageot, G Taieb, W Camu
Spinal muscular atrophy (SMA) refers to a group of disorders affecting lower motor neurons. The age of onset of these disorders is variable, ranging from the neonatal period to adulthood. Over the last few years, there has been enormous progress in the description of new genes and phenotypes that throw new light on the molecular pathways involved in motor neuron degeneration. Advances in our understanding of the pathophysiology of the most frequent forms, SMA linked to SMN1 gene mutations and Kennedy disease, has led to the development of therapeutic strategies currently being tested in clinical trials...
April 26, 2017: Revue Neurologique
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