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https://www.readbyqxmd.com/read/28443372/cord-blood-as-a-potential-therapeutic-for-amyotrophic-lateral-sclerosis
#1
Svitlana Garbuzova-Davis, Jared Ehrhart, Paul R Sanberg
Amyotrophic Lateral Sclerosis (ALS) is a fatal neurodegenerative disease characterized by progressive motor neuron degeneration in the brain and spinal cord. Treatment options are limited due to the complexity of underlying disease factors. Cell therapy, using human umbilical cord blood (hUCB) cells may be a promising new treatment for ALS, mainly by providing a protective microenvironment for motor neuron survival. Areas covered: Composition and in vitro and in vivo differentiation of hUCB cells and the advantages of cord blood as a source of transplant cells are discussed...
April 26, 2017: Expert Opinion on Biological Therapy
https://www.readbyqxmd.com/read/28443187/treatment-with-penicillin-g-and-hydrocortisone-reduces-als-associated-symptoms-a-case-series-of-three-patients
#2
Bert Tuk, Harmen Jousma, Pieter J Gaillard
Three male Caucasian patients with ALS were admitted to the hospital due to progressive dysphagia and dysarthria. During two 21-day courses of penicillin G and hydrocortisone, these patients' dysphagia and dysarthria resolved. The patient's other ALS-associated symptoms also improved, including respiratory function, coordination, walking, and muscle strength. This is the first report of a treatment with a protocol for treating dysphagia, dysarthria, respiratory depression and other ALS-related symptoms. Furthermore, the observations are consistent with the recent hypothesis that the successful treatment of ALS symptoms with this treatment course in six patients with syphilitic ALS was not directly due to the treatment of syphilis; but that the administered penicillin G and/or hydrocortisone treated these patients' ALS symptoms due the off-target pharmacological activity of penicillin G and/or hydrocortisone...
2017: F1000Research
https://www.readbyqxmd.com/read/28412941/survival-prediction-in-amyotrophic-lateral-sclerosis-based-on-mri-measures-and-clinical-characteristics
#3
Christina Schuster, Orla Hardiman, Peter Bede
BACKGROUND: Amyotrophic lateral sclerosis (ALS) a highly heterogeneous neurodegenerative condition. Accurate diagnostic, monitoring and prognostic biomarkers are urgently needed both for individualised patient care and clinical trials. A multimodal magnetic resonance imaging study is presented, where MRI measures of ALS-associated brain regions are utilised to predict 18-month survival. METHODS: A total of 60 ALS patients and 69 healthy controls were included in this study...
April 17, 2017: BMC Neurology
https://www.readbyqxmd.com/read/28406915/predicting-functional-decline-and-survival-in-amyotrophic-lateral-sclerosis
#4
Mei-Lyn Ong, Pei Fang Tan, Joanna D Holbrook
BACKGROUND: Better predictors of amyotrophic lateral sclerosis disease course could enable smaller and more targeted clinical trials. Partially to address this aim, the Prize for Life foundation collected de-identified records from amyotrophic lateral sclerosis sufferers who participated in clinical trials of investigational drugs and made them available to researchers in the PRO-ACT database. METHODS: In this study, time series data from PRO-ACT subjects were fitted to exponential models...
2017: PloS One
https://www.readbyqxmd.com/read/28406335/clinical-efficacy-of-edaravone-for-the-treatment-of-amyotrophic-lateral-sclerosis
#5
Hideyuki Sawada
Amyotrophic lateral sclerosis (ALS) is a progressive, fatal, neurodegenerative disease. Although the pathogenesis remains unresolved, oxidative stress is known to play a pivotal role. Edaravone works in the central nervous system as a potent scavenger of oxygen radicals. In ALS mouse models, edaravone suppresses motor functional decline and nitration of tyrosine residues in the cerebrospinal fluid. Areas covered: Three clinical trials, one phase II open-label trial, and two phase III placebo-control randomized trials were reviewed...
May 2017: Expert Opinion on Pharmacotherapy
https://www.readbyqxmd.com/read/28382000/als-clinical-trials-review-20-years-of-failure-are-we-any-closer-to-registering-a-new-treatment
#6
REVIEW
Dmitry Petrov, Colin Mansfield, Alain Moussy, Olivier Hermine
Amyotrophic lateral sclerosis (ALS) is a devastating condition with an estimated mortality of 30,000 patients a year worldwide. The median reported survival time since onset ranges from 24 to 48 months. Riluzole is the only currently approved mildly efficacious treatment. Riluzole received marketing authorization in 1995 in the USA and in 1996 in Europe. In the years that followed, over 60 molecules have been investigated as a possible treatment for ALS. Despite significant research efforts, the overwhelming majority of human clinical trials (CTs) have failed to demonstrate clinical efficacy...
2017: Frontiers in Aging Neuroscience
https://www.readbyqxmd.com/read/28374969/randomized-cross-over-trial-of-ventilator-modes-during-non-invasive-ventilation-titration-in-amyotrophic-lateral-sclerosis
#7
Bart Vrijsen, Bertien Buyse, Catharina Belge, Goele Vanpee, Philip Van Damme, Dries Testelmans
BACKGROUND AND OBJECTIVE: Non-invasive ventilation (NIV) improves survival, quality of life and sleep in patients with amyotrophic lateral sclerosis (ALS). Nevertheless, NIV titration is conducted in different ways. We aim to provide more insight into NIV titration by comparing the effects of a spontaneous (S) and spontaneous-timed (ST) modes on gas exchange, sleep architecture and patient-ventilator asynchronies (PVAs). METHODS: After an initial night of NIV titration, patients were randomized to S or ST mode in a cross-over design...
April 4, 2017: Respirology: Official Journal of the Asian Pacific Society of Respirology
https://www.readbyqxmd.com/read/28367604/ultra-high-field-proton-mr-spectroscopy-in-early-stage-amyotrophic-lateral-sclerosis
#8
Ian Cheong, Małgorzata Marjańska, Dinesh K Deelchand, Lynn E Eberly, David Walk, Gülin Öz
A major hurdle in the development of effective treatments for amyotrophic lateral sclerosis (ALS) has been the lack of robust biomarkers for use as clinical trial endpoints. Neurochemical profiles obtained in vivo by high field proton magnetic resonance spectroscopy ((1)H-MRS) can potentially provide biomarkers of cerebral pathology in ALS. However, previous (1)H-MRS studies in ALS have produced conflicting findings regarding alterations in the levels of neurochemical markers such as glutamate (Glu) and myo-inositol (mIns)...
April 3, 2017: Neurochemical Research
https://www.readbyqxmd.com/read/28356511/poly-gp-proteins-are-a-useful-pharmacodynamic-marker-for-c9orf72-associated-amyotrophic-lateral-sclerosis
#9
Tania F Gendron, Jeannie Chew, Jeannette N Stankowski, Lindsey R Hayes, Yong-Jie Zhang, Mercedes Prudencio, Yari Carlomagno, Lillian M Daughrity, Karen Jansen-West, Emilie A Perkerson, Aliesha O'Raw, Casey Cook, Luc Pregent, Veronique Belzil, Marka van Blitterswijk, Lilia J Tabassian, Chris W Lee, Mei Yue, Jimei Tong, Yuping Song, Monica Castanedes-Casey, Linda Rousseau, Virginia Phillips, Dennis W Dickson, Rosa Rademakers, John D Fryer, Beth K Rush, Otto Pedraza, Ana M Caputo, Pamela Desaro, Carla Palmucci, Amelia Robertson, Michael G Heckman, Nancy N Diehl, Edythe Wiggs, Michael Tierney, Laura Braun, Jennifer Farren, David Lacomis, Shafeeq Ladha, Christina N Fournier, Leo F McCluskey, Lauren B Elman, Jon B Toledo, Jennifer D McBride, Cinzia Tiloca, Claudia Morelli, Barbara Poletti, Federica Solca, Alessandro Prelle, Joanne Wuu, Jennifer Jockel-Balsarotti, Frank Rigo, Christine Ambrose, Abhishek Datta, Weixing Yang, Denitza Raitcheva, Giovanna Antognetti, Alexander McCampbell, John C Van Swieten, Bruce L Miller, Adam L Boxer, Robert H Brown, Robert Bowser, Timothy M Miller, John Q Trojanowski, Murray Grossman, James D Berry, William T Hu, Antonia Ratti, Bryan J Traynor, Matthew D Disney, Michael Benatar, Vincenzo Silani, Jonathan D Glass, Mary Kay Floeter, Jeffrey D Rothstein, Kevin B Boylan, Leonard Petrucelli
There is no effective treatment for amyotrophic lateral sclerosis (ALS), a devastating motor neuron disease. However, discovery of a G4C2 repeat expansion in the C9ORF72 gene as the most common genetic cause of ALS has opened up new avenues for therapeutic intervention for this form of ALS. G4C2 repeat expansion RNAs and proteins of repeating dipeptides synthesized from these transcripts are believed to play a key role in C9ORF72-associated ALS (c9ALS). Therapeutics that target G4C2 RNA, such as antisense oligonucleotides (ASOs) and small molecules, are thus being actively investigated...
March 29, 2017: Science Translational Medicine
https://www.readbyqxmd.com/read/28331770/traditional-food-items-in-ogimi-okinawa-l-serine-content-and-the-potential-for-neuroprotection
#10
REVIEW
Paul Alan Cox, James S Metcalf
PURPOSE OF REVIEW: Ogimi village is renowned for its aging population. We sought to determine if the l-serine content of their diet could account for their neurological health. RECENT FINDINGS: The most frequently consumed food items, including tofu and seaweeds, are rich in the dietary amino acid l-serine. l-serine content of the Ogimi diet >8 grams/day for Ogimi women significantly exceeds the average American dietary intake of 2.5 grams/day for women >70 years old...
2017: Current Nutrition Reports
https://www.readbyqxmd.com/read/28302022/glial-cell-a-potential-target-for-cellular-and-drug-based-therapy-in-various-cns-diseases
#11
Shakeeb Ahmed, Azka Gull, Tahir Khuroo, Mohd Aqil, Yasmin Sultana
Glial cells are integrated part of neurovascular unit of blood brain barrier (BBB). They undergo mitosis and mainly classified as astrocytes, oligodendrocytes, microglia, ependymal cells and nerve glial antigen 2 cells. Being a most versatile glial cell, astrocytes provide structural support to neurons, maintain brain homeostasis, take part in neuronal communication, and perform some housekeeping functions. Oligodendrocytes myelinate the neuronal axons for proper transmission of nerve impulse and microglia are brain immune cells...
March 16, 2017: Current Pharmaceutical Design
https://www.readbyqxmd.com/read/28293476/evaluating-the-levels-of-csf-and-serum-factors-in-als
#12
Jie Guo, Xuan Yang, Lina Gao, Dawei Zang
OBJECTIVES: The aim of this study was to identify CSF and serum factors as biomarkers that may aid in distinguishing ALS patients from control subjects and predicting ALS progression as well as prognosis. METHODS: Serum and CSF samples from 105 patients with ALS and 56 control subjects were analyzed for 13 factors using ELISA. The revised ALS functional rating scale (ALSFRS-r) was used to evaluate the overall functional status of ALS patients, and we also followed up with ALS patients either by phone or with clinic visits for five years after enrollment in this study...
March 2017: Brain and Behavior
https://www.readbyqxmd.com/read/28277881/drugs-in-clinical-development-for-the-treatment-of-amyotrophic-lateral-sclerosis
#13
REVIEW
Ana Martinez, Maria Del Valle Palomo Ruiz, Daniel I Perez, Carmen Gil
Amyotrophic Lateral Sclerosis (ALS) is a fatal motor neuron progressive disorder for which no treatment exists to date. However, there are other investigational drugs and therapies currently under clinical development may offer hope in the near future. Areas covered: We have reviewed all the ALS ongoing clinical trials (until November 2016) and collected in Clinicaltrials.gov or EudraCT. We have described them in a comprehensive way and have grouped them in the following sections: biomarkers, biological therapies, cell therapy, drug repurposing and new drugs...
April 2017: Expert Opinion on Investigational Drugs
https://www.readbyqxmd.com/read/28267691/vorapaxar-and-amyotrophic-lateral-sclerosis-coincidence-or-adverse-association
#14
REVIEW
Victor L Serebruany, Seth D Fortmann, Daniel F Hanley, Moo Hyun Kim
BACKGROUND: Vorapaxar, a novel antiplatelet thrombin PAR-1 inhibitor, is currently approved for post myocardial infarction and peripheral artery disease indications with concomitant use of clopidogrel and/or aspirin. The vorapaxar safety profile was acceptable. However, aside from heightened bleeding risks, excesses of solid cancers and diplopia, there were more amyotrophic lateral sclerosis (ALS) diagnoses after vorapaxar. STUDY QUESTION: To assess the Food and Drug Administration (FDA) reviews on the potential association of vorapaxar with ALS...
March 2017: American Journal of Therapeutics
https://www.readbyqxmd.com/read/28265751/peripheral-nerve-diffusion-tensor-imaging-as-a-measure-of-disease-progression-in-als
#15
Neil G Simon, Jim Lagopoulos, Sita Paling, Casey Pfluger, Susanna B Park, James Howells, Thomas Gallagher, Michel Kliot, Robert D Henderson, Steve Vucic, Matthew C Kiernan
Clinical trial design in amyotrophic lateral sclerosis (ALS) remains hampered by a lack of reliable and sensitive biomarkers of disease progression. The present study evaluated peripheral nerve diffusion tensor imaging (DTI) as a surrogate marker of axonal degeneration in ALS. Longitudinal studies were undertaken in 21 ALS patients studied at 0 and 3 months, and 19 patients at 0, 3 and 6 months, with results compared to 13 age-matched controls. Imaging metrics were correlated across a range of functional assessments including amyotrophic lateral sclerosis functional rating scale revised (ALSFRS-R), lower limb muscle strength (Medical Research Council sum score, MRCSS-LL), compound muscle action potential amplitudes and motor unit number estimation (MUNE)...
March 6, 2017: Journal of Neurology
https://www.readbyqxmd.com/read/28242130/kennedy-s-disease-1234-scale-preliminary-design-and-test
#16
Ming Lu, Haixiao Guo, Dongsheng Fan
Kennedy's disease (KD), also known as spinal and bulbar muscular atrophy (SBMA), is a rare x-linked genetic disorder which is characterized by muscle weakness and atrophy. In previous clinical trials, KD patients had been assessed using the ALSFRS scale, which was specifically designed for ALS patients. However, the progression of KD is very slow, and thus, the ALSFRS does not accurately reflect changes in the clinical condition of KD patient. Here, we developed the KD 1234 scale which designed specially for KD...
February 24, 2017: Journal of Clinical Neuroscience: Official Journal of the Neurosurgical Society of Australasia
https://www.readbyqxmd.com/read/28241758/clinical-trials-from-the-patient-perspective-survey-in-an-online-patient-community
#17
Pronabesh DasMahapatra, Priya Raja, Jeremy Gilbert, Paul Wicks
BACKGROUND: Developing new medicines relies on the successful conduct of clinical trials. As trial protocols become more arduous, it becomes harder to recruit and retain patient volunteers, although recent efforts such as OMERACT and I-SPY2 show that partnering with patients can be beneficial. We sought to describe drivers and barriers to trial participation, as well as condition-specific trial preferences. METHODS: An online survey was fielded via the patient-powered research network PatientsLikeMe to 1,621 members living with nine selected chronic health conditions...
February 27, 2017: BMC Health Services Research
https://www.readbyqxmd.com/read/28236105/selection-and-prioritization-of-candidate-drug-targets-for-amyotrophic-lateral-sclerosis-through-a-meta-analysis-approach
#18
Giovanna Morello, Antonio Gianmaria Spampinato, Francesca Luisa Conforti, Velia D'Agata, Sebastiano Cavallaro
Amyotrophic lateral sclerosis (ALS) is a progressive and incurable neurodegenerative disease. Although several compounds have shown promising results in preclinical studies, their translation into clinical trials has failed. This clinical failure is likely due to the inadequacy of the animal models that do not sufficiently reflect the human disease. Therefore, it is important to optimize drug target selection by identifying those that overlap in human and mouse pathology. We have recently characterized the transcriptional profiles of motor cortex samples from sporadic ALS (SALS) patients and differentiated these into two subgroups based on differentially expressed genes, which encode 70 potential therapeutic targets...
April 2017: Journal of Molecular Neuroscience: MN
https://www.readbyqxmd.com/read/28229508/meditation-training-for-people-with-amyotrophic-lateral-sclerosis-a-randomized-clinical-trial
#19
F Pagnini, A Marconi, A Tagliaferri, G M Manzoni, R Gatto, V Fabiani, G Gragnano, G Rossi, E Volpato, P Banfi, A Palmieri, F Graziano, G Castelnuovo, M Corbo, E Molinari, N Riva, V Sansone, C Lunetta
BACKGROUND AND PURPOSE: Studies investigating psychological interventions for the promotion of well-being in people with amyotrophic lateral sclerosis (ALS) are lacking. The purpose of the current study was to examine the use of an ALS-specific mindfulness-based intervention for improving quality of life in this population. METHODS: A randomized, open-label and controlled clinical trial was conducted on the efficacy of an ALS-specific meditation programme in promoting quality of life...
February 23, 2017: European Journal of Neurology: the Official Journal of the European Federation of Neurological Societies
https://www.readbyqxmd.com/read/28197175/can-cannabinoids-be-a-potential-therapeutic-tool-in-amyotrophic-lateral-sclerosis
#20
REVIEW
Sabrina Giacoppo, Emanuela Mazzon
Amyotrophic lateral sclerosis (ALS) is the most common degenerative disease of the motor neuron system. Over the last years, a growing interest was aimed to discovery new innovative and safer therapeutic approaches in the ALS treatment. In this context, the bioactive compounds of Cannabis sativa have shown antioxidant, anti-inflammatory and neuroprotective effects in preclinical models of central nervous system disease. However, most of the studies proving the ability of cannabinoids in delay disease progression and prolong survival in ALS were performed in animal model, whereas the few clinical trials that investigated cannabinoids-based medicines were focused only on the alleviation of ALS-related symptoms, not on the control of disease progression...
December 2016: Neural Regeneration Research
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