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Proof of concept trials

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https://www.readbyqxmd.com/read/29332733/a-fundamental-critique-of-the-fascial-distortion-model-and-its-application-in-clinical-practice
#1
Christoph Thalhamer
INTRODUCTION: The therapeutic techniques used in the fascial distortion model (FDM) have become increasingly popular among manual therapists and physical therapists. The reasons for this trend remain to be empirically explored. Therefore this paper pursues two goals: first, to investigate the historical and theoretical background of FDM, and second, to discuss seven problems associated with the theory and practice of FDM. MATERIALS AND METHODS: The objectives of this paper are based on a review of the literature...
January 2018: Journal of Bodywork and Movement Therapies
https://www.readbyqxmd.com/read/29326851/recent-advancements-in-gene-therapy-for-hereditary-retinal-dystrophies
#2
REVIEW
Ayşe Öner
Hereditary retinal dystrophies (HRDs) are degenerative diseases of the retina which have marked clinical and genetic heterogeneity. Common presentations among these disorders include night or colour blindness, tunnel vision, and subsequent progression to complete blindness. The known causative disease genes have a variety of developmental and functional roles, with mutations in more than 120 genes shown to be responsible for the phenotypes. In addition, mutations within the same gene have been shown to cause different disease phenotypes, even amongst affected individuals within the same family, highlighting further levels of complexity...
December 2017: Turkish Journal of Ophthalmology
https://www.readbyqxmd.com/read/29324745/proof-of-concept-study-of-an-at-home-engaging-digital-intervention-for-pediatric-adhd
#3
Naomi O Davis, Jeffrey Bower, Scott H Kollins
OBJECTIVE: Pharmacological and behavioral therapies have limited impact on the distinct neurocognitive impairments associated with ADHD, and existing cognitive training programs have shown limited efficacy. This proof-of-concept study assessed treatment acceptability and explored outcomes for a novel digital treatment targeting cognitive processes implicated in ADHD. METHOD: Participants included 40 children with ADHD and 40 children without ADHD. Following psychiatric screening, ADHD ratings, and baseline neuropsychological measures, participants completed 28-days of at-home treatment...
2018: PloS One
https://www.readbyqxmd.com/read/29321602/multifunctional-microfluidic-chip-for-optical-nanoprobe-based-rna-detection-application-to-chronic-myeloid-leukemia
#4
Pedro Urbano Alves, Raquel Vinhas, Alexandra R Fernandes, Semra Zuhal Birol, Levent Trabzon, Iwona Bernacka-Wojcik, Rui Igreja, Paulo Lopes, Pedro Viana Baptista, Hugo Águas, Elvira Fortunato, Rodrigo Martins
Many diseases have their treatment options narrowed and end up being fatal if detected during later stages. As a consequence, point-of-care devices have an increasing importance for routine screening applications in the health sector due to their portability, fast analyses and decreased cost. For that purpose, a multifunctional chip was developed and tested using gold nanoprobes to perform RNA optical detection inside a microfluidic chip without the need of molecular amplification steps. As a proof-of-concept, this device was used for the rapid detection of chronic myeloid leukemia, a hemato-oncological disease that would benefit from early stage diagnostics and screening tests...
January 10, 2018: Scientific Reports
https://www.readbyqxmd.com/read/29317339/from-clinical-proof-of-concept-to-commercialization-of-car-t-cells
#5
Boris Calmels, Bechara Mfarrej, Christian Chabannon
The development of CAR T cells currently represents an exciting opportunity to convert the already published clinical successes observed in clinical trials into commercially available efficient therapies. However, the path toward successful commercialization is still hindered by many hurdles. Here, we review such issues as: the need for structured collaborations between hospital collection and clinical facilities and industry manufacturing facilities to streamline the supply chain; necessity for uniform and efficient medical procedures to cope with severe toxicities associated with CAR T cells; and absolute need to define an economical and sustainable model for manufacturers and payers...
January 6, 2018: Drug Discovery Today
https://www.readbyqxmd.com/read/29312629/fluorescence-guided-surgery-for-cancer-patients-a-proof-of-concept-study-on-human-xenografts-in-mice-and-spontaneous-tumors-in-pets
#6
Eliane Mery, Muriel Golzio, Stephanie Guillermet, Didier Lanore, Augustin Le Naour, Benoît Thibault, Anne Françoise Tilkin-Mariamé, Elizabeth Bellard, Jean Pierre Delord, Denis Querleu, Gwenael Ferron, Bettina Couderc
Surgery is often the first treatment option for patients with cancer. Patient survival essentially depends on the completeness of tumor resection. This is a major challenge, particularly in cases of peritoneal carcinomatosis, where tumors are widely disseminated in the large peritoneal cavity. Any development to help surgeons visualize these residual cells would improve the completeness of the surgery. For non-disseminated tumors, imaging could be used to ensure that the tumor margins and the draining lymph nodes are free of tumor deposits...
December 12, 2017: Oncotarget
https://www.readbyqxmd.com/read/29306887/developing-professional-caregivers-empathy-and-emotional-competencies-through-mindfulness-based-stress-reduction-mbsr-results-of-two-proof-of-concept-studies
#7
Martin Lamothe, Pierre McDuff, Yves D Pastore, Michel Duval, Serge Sultan
OBJECTIVES: To assess the feasibility and acceptability of a mindfulness-based stress reduction (MBSR)-based intervention and determine if the intervention is associated with a significant signal on empathy and emotional competencies. DESIGN: Two pre-post proof-of-concept studies. SETTING: Participants were recruited at the University of Montreal's Psychology Department (Study 1) and the CHU Sainte-Justine Department of Hematology-Oncology (Study 2)...
January 5, 2018: BMJ Open
https://www.readbyqxmd.com/read/29296748/immune-signatures-associated-with-improved-progression-free-and-overall-survival-for-myeloma-patients-treated-with-ahsct
#8
Christine M Ho, Philip L McCarthy, Paul K Wallace, Yali Zhang, Ahmad Fora, Patrick Mellors, Joseph D Tario, Benjamin L S McCarthy, George L Chen, Sarah A Holstein, Sophia R Balderman, Xuefang Cao, Bruno Paiva, Theresa Hahn
Multiple therapeutic options exist for multiple myeloma (MM), including autologous hematopoietic stem cell transplantation (AHSCT). Measurement of minimal residual disease (MRD) and immune reconstitution is rapidly becoming an integral part of the care of MM patients. We investigated comprehensive immune profiling (IP) associated with progression-free survival (PFS) and overall survival (OS). From August 2007 to January 2014, 101 consecutive MM patients underwent peripheral blood IP and marrow MRD testing before and approximately 100 days after AHSCT...
June 27, 2017: Blood Advances
https://www.readbyqxmd.com/read/29287690/implication-of-stard5-and-cholesterol-homeostasis-disturbance-in-the-endoplasmic-reticulum-stress-related-response-induced-by-pro-apoptotic-aminosteroid-rm-133
#9
Martin Perreault, René Maltais, Lucie-Carole Kenmogne, Danny Létourneau, Jean-Guy LeHoux, Stéphane Gobeil, Donald Poirier
The aminosteroid derivative RM-133 is an effective anticancer molecule for which proof of concept has been achieved in several mouse xenograph models (HL-60, MCF-7, PANC-1 and OVCAR-3). To promote this new family of molecules toward a clinical phase 1 trial, the mechanism of action governing the anticancer properties of the representative candidate RM-133 needs to be characterized. In vitro experiments were first used to determine that RM-133 causes apoptosis in cancer cells. Then, using proteomic and transcriptomic experiments, RM-133 cytotoxicity was proven to be achieved via the endoplasmic reticulum (ER)-related apoptosis, which characterizes RM-133 as an endoplasmic reticulum stress aggravator (ERSA) anticancer drug...
December 26, 2017: Pharmacological Research: the Official Journal of the Italian Pharmacological Society
https://www.readbyqxmd.com/read/29285613/are-large-fracture-trials-really-possible-what-we-have-learned-from-the-randomized-controlled-damage-control-study
#10
Eva Steinhausen, Bertil Bouillon, Dieter Rixen
PURPOSE: Although they are considered the 'gold standard' of evidence-based medicine, randomized controlled trials are still a rarity in orthopedic surgery. In the management of patients with multiple trauma, there is a current trend toward 'damage control orthopedics', but to date, there is no proof of the superiority of this concept in terms of evidence-based medicine. The purpose of this article is to present unexpected difficulties we encountered in successfully completing our randomized controlled trial and to discuss the problematic differences between theoretically planning a trial and real-life practical experience of implementing the plan, with attention to published strategies...
December 28, 2017: European Journal of Trauma and Emergency Surgery: Official Publication of the European Trauma Society
https://www.readbyqxmd.com/read/29284397/stem-progenitor-cells-and-biodegradable-scaffolds-in-the-treatment-of-retinal-degenerative-diseases
#11
Yuyao Wang, Dandan Zhang, Bingqiao Shen, Yi Zhang, Ping Gu
Visual impairment caused by retinal degeneration is primarily attributed to the irreversible degradation of retinal neurons or the adjacent retinal pigment epithelium (RPE). No efficient clinical therapies to restore or improve visual ability are currently available. Cell therapy has been touted as a promising strategy to overcome this challenge. Progenitor/stem cells may be obtained from both ocular and non-ocular tissues. The former mainly includes retinal progenitor cells (RPCs), whereas the latter comprises embryonic stem cells (ESCs) and induced pluripotent stem cells (iPSCs), which have been utilized in stem cell replacement therapy studies ranging from proof-of-concept animal models to clinical trials in humans...
December 27, 2017: Current Stem Cell Research & Therapy
https://www.readbyqxmd.com/read/29284369/bayesian-phase-ii-optimization-for-time-to-event-data-based-on-historical-information
#12
Anja Bertsche, Frank Fleischer, Jan Beyersmann, Gerhard Nehmiz
After exploratory drug development, companies face the decision whether to initiate confirmatory trials based on limited efficacy information. This proof-of-concept decision is typically performed after a Phase II trial studying a novel treatment versus either placebo or an active comparator. The article aims to optimize the design of such a proof-of-concept trial with respect to decision making. We incorporate historical information and develop pre-specified decision criteria accounting for the uncertainty of the observed treatment effect...
January 1, 2017: Statistical Methods in Medical Research
https://www.readbyqxmd.com/read/29282731/cognitive-behavioural-therapy-for-adult-attention-deficit-hyperactivity-disorder-a-proof-of-concept-randomised-controlled-trial
#13
A J Dittner, J Hodsoll, K A Rimes, A J Russell, T Chalder
OBJECTIVE: To investigate efficacy, patient acceptability and feasibility of formulation-based cognitive-behavioural therapy (CBT) for adults with attention-deficit hyperactivity disorder (ADHD). NICE guidelines for adult ADHD recommend further research into psychological treatments. METHOD: Sixty participants with adult ADHD were randomly allocated to treatment as usual (TAU) vs. TAU plus up to 16 sessions of individual formulation-based CBT for ADHD. RESULTS: Adding formulation-based CBT to TAU for ADHD significantly improved ADHD symptoms on the Barkley Current Symptoms Scale and scores on the Work and Social Adjustment Scale...
December 27, 2017: Acta Psychiatrica Scandinavica
https://www.readbyqxmd.com/read/29275332/dual-il-17a-and-il-17f-neutralisation-by-bimekizumab-in-psoriatic-arthritis-evidence-from-preclinical-experiments-and-a-randomised-placebo-controlled-clinical-trial-that-il-17f-contributes-to-human-chronic-tissue-inflammation
#14
Sophie Glatt, Dominique Baeten, Terry Baker, Meryn Griffiths, Lucian Ionescu, Alastair D G Lawson, Ash Maroof, Ruth Oliver, Serghei Popa, Foteini Strimenopoulou, Pavan Vajjah, Mark I L Watling, Nataliya Yeremenko, Pierre Miossec, Stevan Shaw
OBJECTIVE: Interleukin (IL)-17A has emerged as pivotal in driving tissue pathology in immune-mediated inflammatory diseases. The role of IL-17F, sharing 50% sequence homology and overlapping biological function, remains less clear. We hypothesised that IL-17F, together with IL-17A, contributes to chronic tissue inflammation, and that dual neutralisation may lead to more profound suppression of inflammation than inhibition of IL-17A alone. METHODS: Preclinical experiments assessed the role of IL-17A and IL-17F in tissue inflammation using disease-relevant human cells...
December 23, 2017: Annals of the Rheumatic Diseases
https://www.readbyqxmd.com/read/29261476/user-experience-of-an-innovative-mobile-health-program-to-assist-in-insulin-dose-adjustment-outcomes-of-a-proof-of-concept-trial
#15
Hang Ding, Farhad Fatehi, Anthony W Russell, Mohan Karunanithi, Anish Menon, Dominique Bird, Leonard C Gray
BACKGROUND: Many patients with diabetes require insulin therapy to achieve optimal glycemic control. Initiation and titration of insulin often require an insulin dose adjustment (IDA) program, involving frequent exchange of blood glucose levels (BGLs) and insulin prescription advice between the patient and healthcare team. This process is time consuming with logistical barriers. OBJECTIVE: To develop an innovative mobile health (m-Health) mobile-based IDA program (mIDA) and evaluate the user adherence and experience through a proof-of-concept trial...
December 20, 2017: Telemedicine Journal and E-health: the Official Journal of the American Telemedicine Association
https://www.readbyqxmd.com/read/29247101/therapist-guided-internet-delivered-cognitive-behavioural-therapy-supplemented-with-group-exposure-sessions-for-adolescents-with-social-anxiety-disorder-a-feasibility-trial
#16
Martina Nordh, Sarah Vigerland, Lars-Göran Öst, Brjánn Ljótsson, David Mataix-Cols, Eva Serlachius, Jens Högström
OBJECTIVES: Social anxiety disorder (SAD) is one of the most common psychiatric disorders in youth, with a prevalence of about 3%-4% and increased risk of adverse long-term outcomes, such as depression. Cognitive-behavioural therapy (CBT) is considered the first-line treatment for youth with SAD, but many adolescents remain untreated due to limited accessibility to CBT. The aim of this study was to develop and evaluate the feasibility and preliminary efficacy of a therapist-guided internet-delivered CBT treatment, supplemented with clinic-based group exposure sessions (BIP SOFT)...
December 14, 2017: BMJ Open
https://www.readbyqxmd.com/read/29241652/acute-administration-of-roflumilast-enhances-immediate-recall-of-verbal-word-memory-in-healthy-young-adults
#17
M A Van Duinen, A Sambeth, P R A Heckman, S Smit, M Tsai, G Lahu, T Uz, A Blokland, J Prickaerts
The need for new and effective treatments for dementia remains indisputably high. Phosphodiesterase inhibitors (PDE-Is) have proven efficacy as cognitive enhancers based on their positive effects in numerous preclinical studies. Especially the PDE4 subfamily is of interest due to its expression in the hippocampus, the key structure for memory formation. The current study investigates the memory enhancing effects of the clinically approved PDE4-I roflumilast in a test battery including the Verbal Learning Task (VLT) combined with electroencephalography (EEG) recording...
December 11, 2017: Neuropharmacology
https://www.readbyqxmd.com/read/29235363/phenotypic-screening-approaches-for-chagas-disease-drug-discovery
#18
Eric Chatelain, Jean-Robert Ioset
Introduction- Chagas disease, caused by the parasite Trypanosoma cruzi, is a global public health issue. Current treatments targeting the parasite are limited to two old nitroheterocyclic drugs with serious side effects. The need for new and safer drugs has prompted numerous drug discovery efforts to identify compounds suitable for parasitological cure in the last decade. Areas covered- Target-based drug discovery has been limited by the small number of well validated targets - the latest example being the failure of azoles, T...
December 13, 2017: Expert Opinion on Drug Discovery
https://www.readbyqxmd.com/read/29232473/front-line-therapy-of-advanced-epithelial-ovarian-cancer-standard-treatment
#19
C Marth, D Reimer, A G Zeimet
Paclitaxel and carboplatin combination chemotherapy has remained the standard of care in the front-line therapy of advanced epithelial ovarian cancer during the last decade. Maintenance chemotherapy has not been proven to impact on overall survival. Acceptable alternatives include weekly paclitaxel plus 3-weekly carboplatin, the addition of bevacizumab to 3-weekly carboplatin and paclitaxel, and intraperitoneal chemotherapy. In particular, anti-angiogenic therapy has been identified as the most promising targeted therapy, and the addition of bevacizumab to first-line chemotherapy followed by a maintenance period of bevacizumab in monotherapy has shown to prolong progression-free survival...
November 1, 2017: Annals of Oncology: Official Journal of the European Society for Medical Oncology
https://www.readbyqxmd.com/read/29217712/cantos-a-gigantic-proof-of-concept-trial
#20
Borja Ibañez, Valentin Fuster
No abstract text is available yet for this article.
December 8, 2017: Circulation Research
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