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Stem cells and clinical trials

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https://www.readbyqxmd.com/read/30321455/therapeutic-potency-of-oncolytic-virotherapy-induced-cancer-stem-cells-targeting-in-brain-tumors-current-status-and-perspectives
#1
REVIEW
Amirhossein Bahreyni, Elnaz Ghorbani, Hamid Fuji, Mikhail Ryzhikov, Majid Khazaei, Marjan Erfani, Amir Avan, Seyed M Hassanian, Kayhan Azadmanesh
Brain tumors are the most common form of solid tumors in children and is presently a serious therapeutic challenge worldwide. Traditional treatment with chemotherapy and radiotherapy was shown to be unsuccessful in targeting brain tumor cancer stem cells (CSCs), leading to recurrent, treatment-resistant secondary malignancies. Oncolytic virotherapy (OV) is an effective antitumor therapeutic strategy which offers a novel, targeted approach for eradicating pediatric brain tumor CSCs by utilizing mechanisms of cell killing that differ from conventional therapies...
October 15, 2018: Journal of Cellular Biochemistry
https://www.readbyqxmd.com/read/30320151/multiple-integrated-non-clinical-studies-predict-the-safety-of-lentivirus-mediated-gene-therapy-for-%C3%AE-thalassemia
#2
Maria Rosa Lidonnici, Ylenia Paleari, Francesca Tiboni, Giacomo Mandelli, Claudia Rossi, Michela Vezzoli, Annamaria Aprile, Carsten Werner Lederer, Alessandro Ambrosi, Franck Chanut, Francesca Sanvito, Andrea Calabria, Valentina Poletti, Fulvio Mavilio, Eugenio Montini, Luigi Naldini, Patrizia Cristofori, Giuliana Ferrari
Gene therapy clinical trials require rigorous non-clinical studies in the most relevant models to assess the benefit-to-risk ratio. To support the clinical development of gene therapy for β-thalassemia, we performed in vitro and in vivo studies for prediction of safety. First we developed newly GLOBE-derived vectors that were tested for their transcriptional activity and potential interference with the expression of surrounding genes. Because these vectors did not show significant advantages, GLOBE lentiviral vector (LV) was elected for further safety characterization...
December 14, 2018: Molecular Therapy. Methods & Clinical Development
https://www.readbyqxmd.com/read/30305848/primary-central-nervous-system-lymphoma
#3
REVIEW
Sarah Löw, Catherine H Han, Tracy T Batchelor
Primary central nervous system lymphoma (PCNSL) is a rare and aggressive extranodal non-Hodgkin lymphoma (NHL), confined to the brain, eyes, spinal cord or leptomeninges without systemic involvement. Overall prognosis, diagnosis and management of PCNSL differ from other types of NHL. Prompt diagnosis and initiation of treatment are vital to improving clinical outcomes. PCNSL is responsive to radiation therapy, however whole-brain radiotherapy (WBRT) inadequately controls the disease when used alone and its delayed neurotoxicity causes neurocognitive impairment, especially in elderly patients...
2018: Therapeutic Advances in Neurological Disorders
https://www.readbyqxmd.com/read/30305514/-tyrosine-kinase-inhibitor-therapy-discontinuation-for-chronic-myelogenous-leukemia-to-achieve-clinical-cure-current-status-and-future-perspectives
#4
Takashi Kumagai
Tyrosine kinase inhibitors (TKIs) markedly improve the prognosis of patients with chronic myelogenous leukemia (CML) by potentially helping to achieve a deep molecular response (DMR). In many clinical trials, beginning with the French Stop Imatinib (STIM1) trial, approximately 40%-60% patients with chronic CML who sustained a long DMR could discontinue TKI therapy and achieve long-term treatment-free remission (TFR). These trials have proposed many predictive factors for successful TKI therapy discontinuation, including deeper molecular response, longer duration of DMR and lack of TKI resistance prior to the discontinuation, and greater numbers of natural killer (NK) cells during the discontinuation...
2018: [Rinshō Ketsueki] the Japanese Journal of Clinical Hematology
https://www.readbyqxmd.com/read/30304859/epigenetic-modifiers-in-myeloid-malignancies-the-role-of-histone-deacetylase-inhibitors
#5
REVIEW
Johanna S Ungerstedt
Myeloid hematological malignancies are clonal bone marrow neoplasms, comprising of acute myeloid leukemia (AML), the myelodysplastic syndromes (MDS), chronic myelomonocytic leukemia (CMML), the myeloproliferative neoplasms (MPN) and systemic mastocytosis (SM). The field of epigenetic regulation of normal and malignant hematopoiesis is rapidly growing. In recent years, heterozygous somatic mutations in genes encoding epigenetic regulators have been found in all subtypes of myeloid malignancies, supporting the rationale for treatment with epigenetic modifiers...
October 9, 2018: International Journal of Molecular Sciences
https://www.readbyqxmd.com/read/30303022/cost-effectiveness-of-pembrolizumab-versus-brentuximab-vedotin-for-patients-with-relapsed-or-refractory-classical-hodgkin-s-lymphoma-a-united-states-payer-perspective
#6
Samuel Large, Robert Hettle, Arun Balakumaran, Elise Wu, Rebekah H Borse
AIMS: Patients with classical Hodgkin's lymphoma (cHL) who have relapsed after or are ineligible for autologous stem cell transplantation (ASCT) have limited treatment options and generally a poor prognosis. Pembrolizumab was recently approved in the United States for the treatment of such patients having demonstrated clinical benefit and tolerability in relapsed/refractory cHL; however, the cost-effectiveness of pembrolizumab in this population is currently unknown. MATERIALS AND METHODS: A three-state Markov model (progression-free [PF], progressed disease, and death) was developed to assess the cost-effectiveness of pembrolizumab (200 mg) versus brentuximab vedotin (BV; 1...
October 10, 2018: Journal of Medical Economics
https://www.readbyqxmd.com/read/30302229/interview-with-dr-philip-mccarthy
#7
Philip McCarthy
Philip McCarthy speaks to Roshaine Wijayatunga, Senior Editor: Oncology Philip McCarthy completed his MD at Tufts University School of Medicine, Boston, MA, USA and his Internal Medicine Residency at Yale, New Haven Hospital, New Haven, CT, USA. His Fellowship was completed at Brigham and Women's Hospital and the Dana Farber Cancer Insitute, Harvard University, Boston, MA, USA. His research interests are devoted to developing novel intensive and reduced intensity allogeneic (allo) and autologous (auto) hematopoietic stem cell transplant (HSCT) approaches for the treatment of hematologic disorders, leading to improved patient outcomes and decreased toxicity...
December 2017: International Journal of Hematologic Oncology
https://www.readbyqxmd.com/read/30301368/advances-in-stem-cell-therapy-for-erectile-dysfunction
#8
Serap Gur, Asim B Abdel-Mageed, Suresh C Sikka, Wayne J G Hellstrom
Stem cell (SC) application is a promising area of research in regenerative medicine, with the potential to treat, prevent, and cure disease. In recent years, the number of studies focusing on SCs for the treatment of erectile dysfunction (ED) and other sexual dysfunctions has increased significantly. Areas covered: This review includes critical ED targets and preclinical studies, including the use of SCs and animal models in diabetes, aging, cavernous nerve injury, and Peyronie's disease. A literature search was performed on PubMed for English articles...
October 9, 2018: Expert Opinion on Biological Therapy
https://www.readbyqxmd.com/read/30300241/is-whole-brain-radiotherapy-still-a-standard-treatment-for-primary-central-nervous-system-lymphomas
#9
Uwe Schlegel, Agnieszka Korfel
PURPOSE OF REVIEW: In primary central nervous system lymphomas (PCNSL), optimal therapy remains to be established, and the role of whole-brain radiotherapy (WBRT) is a matter of debate. With radiation alone, transient responses and clinical improvement are frequent, but long-term disease control is exceptional. WBRT has been considered possible consolidation therapy after high-dose methotrexate (HDMTX)-based initial chemotherapy. This strategy has been questioned due to a high risk of delayed neurotoxicity after combined treatment...
October 8, 2018: Current Opinion in Neurology
https://www.readbyqxmd.com/read/30300216/optimizing-clinical-use-of-biologics-in-orthopaedic-surgery-consensus-recommendations-from-the-2018-aaos-nih-u-13-conference
#10
Constance R Chu, Scott Rodeo, Nidhi Bhutani, Laurie R Goodrich, Johnny Huard, James Irrgang, Robert F LaPrade, Christian Lattermann, Ying Lu, Bert Mandelbaum, Jeremy Mao, Louis McIntyre, Allan Mishra, George F Muschler, Nicolas S Piuzzi, Hollis Potter, Kurt Spindler, John M Tokish, Rocky Tuan, Kenneth Zaslav, William Maloney
Concern that misinformation from direct-to-consumer marketing of largely unproven "biologic" treatments such as platelet-rich plasma and cell-based therapies may erode the public trust and the responsible investment needed to bring legitimate biological therapies to patients have resulted in calls to action from professional organizations and governing bodies. In response to substantial patient demand for biologic treatment of orthopaedic conditions, the American Academy of Orthopaedic Surgeons convened a collaborative symposium and established a consensus framework for improving and accelerating the clinical evaluation, use, and optimization of biologic therapies for musculoskeletal diseases...
October 8, 2018: Journal of the American Academy of Orthopaedic Surgeons
https://www.readbyqxmd.com/read/30300133/erectile-dysfunction-treated-with-intracavernous-stem-cells-a-promising-new-therapy
#11
Lauro S Gómez-Guerra, J Iván Robles-Torres, Alejandra Garza-Bedolla, Consuelo Mancías-Guerra
In the past decades, great interest has been shown in the development of new therapies for erectile dysfunction. Stem cell therapy has generated promising results in numerous preclinical trials in animal models, which is why has led to the development of the first clinical trials in humans. The main cause involved in the pathophysiology of erectile dysfunction is vascular damage related to endothelial and neuronal injury. The interest in stem cell therapy is justified by their capability to differentiate into specific damaged tissues, including endothelium and nervous tissue, and induction of the host own cell proliferation...
July 2018: Revista Internacional de Andrología
https://www.readbyqxmd.com/read/30299399/advances-in-site-specific-gene-editing-for-primary-immune-deficiencies
#12
Caroline Y Kuo
PURPOSE OF REVIEW: Conventional gene therapy has been a successful, curative treatment modality for many primary immune deficiencies with significant improvements in the last decade. However, the risk of leukemic transformation with viral-mediated gene addition still remains, and unregulated gene addition is not an option for certain diseases in which the target gene is closely controlled. The recent bloom in genome modification platforms has created the opportunity to site-specifically correct mutated DNA base pairs or insert a corrective cDNA minigene while maintaining gene expression under control of endogenous regulatory elements...
October 6, 2018: Current Opinion in Allergy and Clinical Immunology
https://www.readbyqxmd.com/read/30299351/successful-treatment-of-an-adolescent-male-with-severe-refractory-evans-syndrome-using-bortezomib-based-therapy
#13
Tristan Knight, Yaddanapudi Ravindranath, Michael U Callaghan
Evans syndrome is defined by bilineal autoimmune cytopenia, typically coombs positive hemolytic anemia and thrombocytopenia. Corticosteroids are the mainstay of treatment, with rituximab and/or mycophenolate mofetil often used in steroid-refractory cases. However, no treatment methodology has ever evaluated by a randomized clinical trial. We present a 15-year-old boy with Evans syndrome and common variable immunodeficiency who experienced a severe, refractory flare 16 months postsplenectomy. After failing to respond to multiple other agents, he achieved a durable response to a bortezomib-based regimen...
October 5, 2018: Journal of Pediatric Hematology/oncology
https://www.readbyqxmd.com/read/30299232/clinical-tracking-of-cell-transfer-and-cell-transplantation-trials-and-tribulations
#14
Jeff W M Bulte, Heike E Daldrup-Link
Cell therapy has provided unprecedented opportunities for tissue repair and cancer therapy. Imaging tools for in vivo tracking of therapeutic cells have entered the clinic to evaluate therapeutic cell delivery and retention in patients. Thus far, clinical cell tracking studies have been a mere proof of principle of the feasibility of cell detection. This review centers around the main clinical queries associated with cell therapy: Have cells been delivered correctly at the targeted site of injection? Are cells still alive, and, if so, how many? Are cells being rejected by the host, and, if so, how severe is the immune response? For stem cell therapeutics, have cells differentiated into downstream cell lineages? Is there cell proliferation including tumor formation? At present, clinical cell tracking trials have only provided information on immediate cell delivery and short-term cell retention...
October 9, 2018: Radiology
https://www.readbyqxmd.com/read/30298387/darvadstrocel-a-review-in-treatment-refractory-complex-perianal-fistulas-in-crohn-s-disease
#15
Lesley J Scott
Darvadstrocel (Alofisel® ) consists of a suspension of expanded human allogeneic adipose-derived mesenchymal stem cells (eASCs). It is the first mesenchymal stem cell (MSC) advanced therapy approved in the EU for the treatment of complex perianal fistulas in adult patients with non-active/mildly active luminal Crohn's disease, when fistulas have shown an inadequate response to ≥ 1 conventional or biologic therapy. In the pivotal phase 3 ADMIRE-CD trial in this difficult-to-treat patient population, after standard-of-care fistula conditioning, add-on therapy with a single dose of darvadstrocel (120 million eASC) administered into the tissue surrounding complex perianal fistulas was significantly more effective than placebo (saline), with the darvadstrocel group having a higher combined remission rate (i...
October 8, 2018: BioDrugs: Clinical Immunotherapeutics, Biopharmaceuticals and Gene Therapy
https://www.readbyqxmd.com/read/30298218/stem-cell-therapy-in-cerebrovascular-disease
#16
REVIEW
Michael I Nahhas, David C Hess
PURPOSE OF REVIEW: The purpose of this article is to provide a review of state-of-the-art cellular therapy in cerebrovascular diseases by discussing published and ongoing clinical trials. RECENT FINDINGS: In spite of the challenge in translating the success of cellular therapy in acute strokes from preclinical models to clinical trials, early phase clinical trial have recently shown promise in overcoming these challenges. Various stem cell types and doses are being studied, different routes of administration are under investigation, as well as defining the optimal time window to intervene...
October 9, 2018: Current Treatment Options in Neurology
https://www.readbyqxmd.com/read/30295953/multistate-modeling-and-simulation-of-patient-trajectories-after-allogeneic-hematopoietic-stem-cell-transplantation-to-inform-drug-development
#17
David A James, Jennifer Ng, Jiawei Wei, Marc Vandemeulebroecke
We present a case study for developing clinical trial scenarios in a complex progressive disease with multiple events of interest. The idea is to first capture the course of the disease in a multistate Markov model, and then to simulate clinical trials from this model, including a variety of hypothesized drug effects. This case study focuses on the prevention of graft-versus-host disease (GvHD) after allogeneic hematopoietic stem cell transplantation (HSCT). The patient trajectory after HSCT is characterized by a complex interplay of various events of interest, and there is no established best method of measuring and/or analyzing treatment benefits...
September 17, 2018: Biometrical Journal. Biometrische Zeitschrift
https://www.readbyqxmd.com/read/30294976/available-and-future-treatments-for-atypical-parkinsonism-a-systematic-review
#18
REVIEW
Davide Vito Moretti
AIMS: Success in treating patients with atypical parkinsonian syndromes, namely progressive supranuclear palsy (PSP), cortico-basal degeneration (CBD), multiple system atrophy (MSA), Parkinson's disease with dementia (PDD), and Lewy body dementia with (LBD), remains exceedingly low. The present work overviews the most influential research literature collected on MEDLINE, ISI Web of Science, Cochrane Library, and Scopus for available treatment in atypical parkinsonisms without time restriction...
October 7, 2018: CNS Neuroscience & Therapeutics
https://www.readbyqxmd.com/read/30294285/current-perspectives-on-role-of-msc-in-renal-pathophysiology
#19
REVIEW
Min Fan, Jing Zhang, Hong Xin, Xiaozhou He, Xuemei Zhang
In the course of the development and worsening of kidney disease, the treatments available are expensive and may cause adverse effects such as immune rejection, inadequate renal resources, or post-operative complications. Therefore, there is an urgent to develop more effective treatments. The advent of mesenchymal stem cells (MSCs) represents a new direction in this context. The current use of MSCs for the treatment of kidney disease has mostly involved experimental studies on animals and only a few clinical trials have been conducted...
2018: Frontiers in Physiology
https://www.readbyqxmd.com/read/30293546/interferon-%C3%AE-mediated-secretion-of-tryptophanyl-trna-synthetases-has-a-role-in-protection-of-human-umbilical-cord-blood-derived-mesenchymal-stem-cells-against-experimental-colitis
#20
Insung Kang, Byung-Chul Lee, Jin Young Lee, Jae-Jun Kim, Seung-Eun Lee, Nari Shin, Soon Won Choi, Kyung-Sun Kang
Mesenchymal stem cells (MSCs) are multipotent adult stem cells that present immunosuppressive effects in experimental and clinical trials targeting various rare diseases including inflammatory bowel disease (IBD). In addition, recent studies have reported tryptophanyl-tRNA synthetase (WRS) possess uncanonical roles such as angiostatic and anti-inflammatory effects. However, little is known about the function of WRS in MSC-based therapy. In this study, we investigated if a novel factor, WRS, secreted from MSCs has a role in amelioration of IBD symptoms and determined a specific mechanism underlying MSC therapy...
October 8, 2018: BMB Reports
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