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Expert Opinion on Investigational Drugs

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https://www.readbyqxmd.com/read/28817311/beta-secretase-inhibitors-in-phase-i-and-phase-ii-clinical-trials-for-alzheimer-s-disease
#1
Charbel E-H Moussa
BACE 1 is a protease that cleaves the transmembrane amyloid precursor protein and generates amyloid-β peptides that accumulate in AD brains. No known mutations are identified in the gene encoding BACE1 in AD. However, enzyme levels are elevated in AD and a single residue mutation in amyloid precursor protein protects against protein cleavage by BACE1, suggesting BACE involvement in disease pathogenesis. Drugs that can inhibit BACE1 would theoretically prevent Aβ accumulation and halt AD onset and progression...
August 17, 2017: Expert Opinion on Investigational Drugs
https://www.readbyqxmd.com/read/28816076/current-drug-and-molecular-therapies-for-the-treatment-of-atrophic-age-related-macular-degeneration-phase-i-to-phase-iii-clinical-development
#2
Huiling Li, Sumana R Chintalapudi, Monica M Jablonski
Age-related macular degeneration (AMD) is the leading cause of vision loss among the elderly. Atrophic AMD, including early, intermediate and geographic atrophy (GA), accounts for ~90% of all cases. It is a multifactorial degeneration characterized by chronic inflammation, oxidative stress and aging components. Although no FDA-approved treatment yet exists for the late stage of atrophic AMD, multiple pathological mechanisms are partially known and several promising therapies are in various stages of development...
August 17, 2017: Expert Opinion on Investigational Drugs
https://www.readbyqxmd.com/read/28814118/considerations-for-clinical-trial-design-in-older-adults-with-cancer
#3
Enrique Soto-Perez-de-Celis, Stuart M Lichtman
No abstract text is available yet for this article.
August 17, 2017: Expert Opinion on Investigational Drugs
https://www.readbyqxmd.com/read/28770623/angiogenesis-inhibitors-in-early-development-for-gastric-cancer
#4
Mauricio P Pinto, Gareth I Owen, Ignacio Retamal, Marcelo Garrido
Angiogenesis, or the generation of new blood vessels from pre-existent ones is a critical process for tumor growth and progression. Hence, the development of angiogenesis inhibitors with therapeutic potential has been a central focus for researchers. Most angiogenesis inhibitors target the Vascular Endothelial Growth Factor (VEGF) pathway, however a number of tyrosine kinase inhibitors (TKIs), immunomodulatory drugs (IMiDs) and inhibitors of the mammalian Target-Of-Rapamycin (mTOR) pathway also display antiangiogenic activity...
August 14, 2017: Expert Opinion on Investigational Drugs
https://www.readbyqxmd.com/read/28777013/investigational-drugs-for-idiopathic-pulmonary-fibrosis
#5
Francesco Varone, Giuliano Montemurro, Francesco Macagno, Mariarosaria Calvello, Emanuele Conte, Enrica Intini, Bruno Iovene, Paolo Maria Leone, Pier-Valerio Mari, Luca Richeldi
IPF is a specific form of chronic fibrosing interstitial pneumonia of unknown cause, characterized by progressive worsening in lung function and an unfavorable prognosis. Current concepts on IPF pathogenesis are based on a dysregulated wound healing response, leading to an over production of extracellular matrix. Based on recent research however, several other mechanisms are now proposed as potential targets for novel therapeutic strategies. Areas covered: This review analyzes the current investigational strategies targeting extracellular matrix deposition, tyrosine-kinase antagonism, immune and autoimmune response, and cell-based therapy...
August 10, 2017: Expert Opinion on Investigational Drugs
https://www.readbyqxmd.com/read/28772081/investigational-phosphodiesterase-inhibitors-in-phase-i-and-phase-ii-clinical-trials-for-alzheimer-s-disease
#6
Jos Prickaerts, Pim R A Heckman, Arjan Blokland
Phosphodiesterase (PDE) inhibitors improve signaling pathways in brain circuits by increasing intracellular cyclic adenosine monophosphate (cAMP) and/or cyclic guanosine monophosphate (cGMP). In the last decade, the first clinical studies investigating selective PDE inhibitors in Alzheimer's disease (AD) have been initiated, based on their positive effects on cognitive processes and neuroprotection in numerous animal studies. Areas covered: This article reviews the clinical studies investigating the pro-cognitive/neuroprotective effects of PDE inhibitors in patients with AD, as well as in age-associated memory impaired elderly and patients with mild cognitive impairment (MCI), the prodromal stage of AD...
August 9, 2017: Expert Opinion on Investigational Drugs
https://www.readbyqxmd.com/read/28758504/investigational-drugs-in-systemic-vasculitis
#7
Adrien Mirouse, Patrice Cacoub, Anne Claire Desbois, Cloé Comarmond, Christian Pagnoux, David Saadoun
Treatment of systemic vasculitis is based on glucocorticoids (GC) in association with immunosuppressive therapy. There are still unmet needs, including earlier onset of response, more targeted therapies, reduction of relapse-risk and decrease of long-term GC and classic immunosuppressants toxicities. Areas covered: In this review, we discuss investigational drugs in early phase clinical trials for induction of remission in vasculitis. We focus on ANCA-associated vasculitis, Behçet's disease, giant cell arteritis, Takayasu arteritis, and cryoglobulinemic vasculitis...
August 4, 2017: Expert Opinion on Investigational Drugs
https://www.readbyqxmd.com/read/28741970/efficacy-and-safety-of-once-weekly-semaglutide-for-the-treatment-of-type-2-diabetes
#8
Xueying Tan, Xiaojing Cao, Minzhi Zhou, Ping Zou, Jingbo Hu
Type 2 diabetes is a chronic metabolic disease characterized by persistent hyperglycemia resulting from progressive deficient of insulin in patients with a background of insulin resistance. Current treatment algorithms recommended by American Diabetes Association/The European Association for the Study of Diabetes promote a patient-centered approach that takes into account a comprehensive consideration of pharmacological properties of drugs, including glucose-lowering action, effects on body weight, correction on multiple pathophysiologic defects, tolerability, and long-term safety...
August 2, 2017: Expert Opinion on Investigational Drugs
https://www.readbyqxmd.com/read/28745064/therapeutic-potential-of-investigational-chk-1-inhibitors-for-the-treatment-of-solid-tumors
#9
Hani M Babiker, Ali McBride, Laurence S Cooke, Daruka Mahadevan
For several decades' cancer treatment targeting DNA repair pathways incorporated both chemo- and radiotherapy only. However, over the last decade improved knowledge of DNA repair processes has paved the way for the development of novel targeted drugs abrogating DNA repair signaling. Checkpoint kinase inhibitors are exciting molecules and hold promise in the treatment of both solid and hematologic malignancies. Herein, we discuss preclinical and clinical studies with this class of molecules. Areas covered: In this review, we discuss the role of check point kinase 1 (CHK-1) in DNA repair and provide a comprehensive summary of pre-clinical and early phase clinical trials with CHK-1 inhibitors...
July 30, 2017: Expert Opinion on Investigational Drugs
https://www.readbyqxmd.com/read/28730878/investigational-drugs-in-development-for-hepatitis-d
#10
Mario Rizzetto
Treatment of chronic hepatitis D still relies on Interferon. To improve efficacy, new therapeutic strategies are in development which aim to deprive the Hepatitis D Virus (HDV) of functions of the Hepatitis B Virus and of the host required for its life-cycle. Areas covered: The therapeutic options are; 1) The inhibition of the farnesylation of the large HD-protein permissive of virion assembly with Lonafarnib, 2) The blocking of HBsAg entry into cells with Myrcludex B via the inhibition of the Sodium Taurocholate Cotransporting Receptor, to prevent the spreading of HDV to uninfected hepatocytes, 3) The reduction of subviral HBsAg particles by REP 2139, leading to diminished virion morphogenesis ...
July 28, 2017: Expert Opinion on Investigational Drugs
https://www.readbyqxmd.com/read/28721753/nintedanib-in-ovarian-cancer
#11
Saira Khalique, Susana Banerjee
Advanced ovarian cancer remains an unmet clinical need. Angiogenesis is considered a therapeutic target in ovarian cancer, with bevacizumab, a monoclonal antibody against VEGF, being the first drug to show a progression-free survival benefit. Nintedanib is an oral tyrosine kinase inhibitor targeting VEGF receptor 1-3, FGFR 1-3 and PDGFR α and β, which has entered phase III trial development in ovarian cancer. Areas covered: This article reviews the preclinical and clinical efficacy of nintedanib in ovarian cancer, its pharmacokinetic and pharmacodynamics profile, safety issues, together with an overview of clinical trials carried out so far...
July 19, 2017: Expert Opinion on Investigational Drugs
https://www.readbyqxmd.com/read/28714740/do-stat3-inhibitors-have-potential-in-the-future-for-cancer-therapy
#12
Andrea L A Wong, Jayshree L Hirpara, Shazib Pervaiz, Jie-Qing Eu, Gautam Sethi, Boon-Cher Goh
No abstract text is available yet for this article.
July 17, 2017: Expert Opinion on Investigational Drugs
https://www.readbyqxmd.com/read/28778139/inhaled-or-nebulised-ciprofloxacin-for-the-maintenance-treatment-of-bronchiectasis
#13
Manjit K Cartlidge, Adam T Hill
Inhaled or nebulised antibiotics are a major topic of ongoing research interest in reducing exacerbations in bronchiectasis. There are no licenced inhaled or nebulised antibiotics currently in bronchiectasis. Areas covered: Inhaled or nebulised ciprofloxacin as a long-term treatment in bronchiectasis. Expert opinion: Results from the Phase III ongoing trials on inhaled or nebulised ciprofloxacin will be key for the outcome of the drugs but additionally, cost-effectiveness and longer-term studies will be necessary to determine the viability of the drug...
September 2017: Expert Opinion on Investigational Drugs
https://www.readbyqxmd.com/read/28726523/investigational-therapies-for-the-treatment-of-narcolepsy
#14
REVIEW
Stefano de Biase, Annacarmen Nilo, Gian Luigi Gigli, Mariarosaria Valente
Narcolepsy is a chronic sleep disorder characterized by a pentad of excessive daytime sleepiness (EDS), cataplexy, sleep paralysis, hypnagogic/hypnopompic hallucinations, and disturbed nocturnal sleep. While non-pharmacological treatments are sometimes helpful, more than 90% of narcoleptic patients require a pharmacological treatment. Areas covered: The present review is based on an extensive Internet and PubMed search from 1994 to 2017. It is focused on drugs currently in development for the treatment of narcolepsy...
August 2017: Expert Opinion on Investigational Drugs
https://www.readbyqxmd.com/read/28718331/entinostat-for-the-treatment-of-breast-cancer
#15
REVIEW
Dario Trapani, Angela Esposito, Carmen Criscitiello, Luca Mazzarella, Marzia Locatelli, Ida Minchella, Saverio Minucci, Giuseppe Curigliano
Breast cancer accounts for 29% of malignant tumors. It is an heterogenous disease covering a spectrum of different molecular subtypes. Epigenetic aberrations may affect gene expression through DNA and histone proteins modifications thus promoting tumor progression and resistance to anti- tumor treatment. Area covered: This article explores the potential role of entinostat in the treatment of breast cancer. The clinical trials evaluating entinostat are discussed, highlighting preclinical data and early-phase clinical studies results...
August 2017: Expert Opinion on Investigational Drugs
https://www.readbyqxmd.com/read/28707483/investigational-drugs-in-development-for-focal-segmental-glomerulosclerosis
#16
REVIEW
Howard Trachtman
Focal segmental glomerulosclerosis is an important cause of end stage kidney disease and is a paradigm for the study of glomerular scarring. There are no FDA approved treatments for this condition. Current therapies, assessed based on reduction in proteinuria, are generally effective in a subset of patients which suggests that FSGS is a heterogeneous group of glomerular disorders or podocytopathies that converge on a common histopathological phenotype. Areas covered: We searched for investigational drugs agents that target different pathophysiological pathways using the key words 'FSGS' and 'podocyte' in American and European clinical trial registers (clinicaltrials...
August 2017: Expert Opinion on Investigational Drugs
https://www.readbyqxmd.com/read/28691539/investigational-antiarrhythmic-agents-promising-drugs-in-early-clinical-development
#17
REVIEW
Jordi Heijman, Shokoufeh Ghezelbash, Dobromir Dobrev
Although there have been important technological advances for the treatment of cardiac arrhythmias (e.g., catheter ablation technology), antiarrhythmic drugs (AADs) remain the cornerstone therapy for the majority of patients with arrhythmias. Most of the currently available AADs were coincidental findings and did not result from a systematic development process based on known arrhythmogenic mechanisms and specific targets. During the last 20 years, our understanding of cardiac electrophysiology and fundamental arrhythmia mechanisms has increased significantly, resulting in the identification of new potential targets for mechanism-based antiarrhythmic therapy...
August 2017: Expert Opinion on Investigational Drugs
https://www.readbyqxmd.com/read/28689433/experimental-and-early-investigational-drugs-for-androgenetic-alopecia
#18
REVIEW
Hongwei Guo, Wendi Victor Gao, Hiromi Endo, Kevin John McElwee
Treatments for androgenetic alopecia constitute a multi-billion-dollar industry, however, currently available therapeutic options have variable efficacy. Consequently, in recent years small biotechnology companies and academic research laboratories have begun to investigate new or improved treatment methods. Research and development approaches include improved formulations and modes of application for current drugs, new drug development, development of cell-based treatments, and medical devices for modulation of hair growth...
August 2017: Expert Opinion on Investigational Drugs
https://www.readbyqxmd.com/read/28685626/lurasidone-for-the-treatment-of-irritability-and-anger-in-autism-spectrum-disorders
#19
REVIEW
Lynn McClellan, Kelli C Dominick, Ernest V Pedapati, Logan K Wink, Craig A Erickson
Autism Spectrum Disorder (ASD) is a heterogeneous neurodevelopmental disorder characterized by deficits in social interaction and communication as well as restricted patterns of behaviors and interests. Irritability marked by tantrums, self-injury and aggression occurs frequently in youth with ASD, causing significant parent and caregiver distress. Atypical antipsychotics have been the most studied drug class targeting irritability in ASD. Risperidone and aripiprazole are Food and Drug Administration (FDA)-approved atypical antipsychotics for treatment of irritability in youth with ASD...
August 2017: Expert Opinion on Investigational Drugs
https://www.readbyqxmd.com/read/28679291/promising-investigational-drug-candidates-in-phase-i-and-phase-ii-clinical-trials-for-mesothelioma
#20
REVIEW
Alice Guazzelli, Emyr Bakker, Kun Tian, Constantinos Demonacos, Marija Krstic-Demonacos, Luciano Mutti
Malignant mesothelioma is a rare and lethal malignancy primarily affecting the pleura and peritoneum. Mesothelioma incidence is expected to increase worldwide and current treatments remain ineffective, leading to poor prognosis. Within this article potential targets to improve the quality of life of the patients and assessment of further avenues for research are discussed. Areas covered: This review highlights emerging therapies currently under investigation for malignant mesothelioma with a specific focus on phase I and phase II clinical trials...
August 2017: Expert Opinion on Investigational Drugs
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