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Expert Opinion on Investigational Drugs

Kirsten C Morley, Andrew Baillie, Wim van den Brink, Kate Chitty, Kathleen Brady, Sudie Back, Greg Sutherland, Devanshi Seth, Lorenzo Leggio, Paul S Haber
Alcoholic liver disease (ALD) is the leading cause of alcohol-related death and one of the most common forms of liver disease. Abstinence from alcohol is crucial to reducing morbidity and mortality associated with the disease. However, there are few treatments for sustaining abstinence suitable for those with significant liver disease. Areas Covered: This paper presents a rationale for the use of N-acetyl cysteine (NAC) to promote abstinence or reduce heavy alcohol consumption for patients with an alcohol use disorder, particularly in the presence of liver disease...
July 18, 2018: Expert Opinion on Investigational Drugs
Leigh C Walker, Andrew J Lawrence
Alcohol use disorder (AUD) is a complex psychiatric condition characterised by craving, compulsive seeking, loss of control of alcohol consumption, as well as the emergence of negative emotional states during withdrawal. Despite the large socioeconomic burden of AUD, therapeutic treatment options lag behind. Areas covered: This review covers pharmacotherapies currently in phase I/II clinical trials for the treatment of alcohol use disorders listed on We discuss drug therapies that modulate monoamine, GABA/Glutamate, neuropeptide and neuroimmune systems...
July 18, 2018: Expert Opinion on Investigational Drugs
Carrie van der Weyden, Martine Bagot, Paul Neeson, Phil K Darcy, H Miles Prince
Therapeutic options for mycosis fungoides and Sezary syndrome include a variety of immunomodulatory, epigenetic, and cytotoxic options, however, none has been demonstrated to be efficacious for all patients, or to deliver deep and durable responses to the majority of patients. In this review, we examine the monoclonal antibody, IPH4102, a novel agent for the treatment of cutaneous T-cell lymphoma. Areas covered: In this review, we examine data demonstrating the tissue specificity of KIR3DL2 receptor, which is highly expressed on the malignant cells in cutaneous T-cell lymphoma, including mycosis fungoides and Sezary syndrome...
July 12, 2018: Expert Opinion on Investigational Drugs
Mariangela Allocca, Daniela Gilardi, Gionata Fiorino, Federica Furfaro, Marjorie Argollo, Laurent Peyrin-Biroulet, Silvio Danese
Gut-specific homing is mainly mediated by the expression of MAdCAM-1 on endothelial cells. An increase in MAdCAM-1 expression has been shown in patients with inflammatory bowel disease (IBD). Areas covered: PF-00547659 is a fully human monoclonal antibody (mAb) towards MAdCAM-1 on endothelial cells, blocking its binding with the α4β7 integrin on leukocytes. This review discusses the available data on effectiveness and safety of PF-00547659 in IBD. Expert opinion: A phase II study in moderate to severe ulcerative colitis (UC) patients, both naïve and previously exposed to anti-TNFs, showed that PF-00547659 was superior to placebo for induction of remission, response and mucosal healing at week 12...
July 9, 2018: Expert Opinion on Investigational Drugs
Lucia Del Vecchio, Francesco Locatelli
In the last decade, concerns have been raised around the use of erythropoiesis stimulating agents (ESA) and intravenous iron in CKD patients, especially when given at high doses. Moreover, treatment with ESA is expensive. Areas covered: We searched PubMed for original articles, reviews and editorials having as a topic anaemia, chronic kidney disease, hypoxia inducible factor, hepcidin, iron, hypoxia-inducible factor prolyl hydroxylase inhibitors (HIF-PHI). HIF-PHI are a new class of small molecules activating HIF-alfa isoforms (the main mediators of the effects of hypoxia on the body)...
July 5, 2018: Expert Opinion on Investigational Drugs
Mario Cazzola, Maria Gakriella Matera, Paola Rogliani, Luigino Calzetta
Aging is the major risk factor for most of the chronic diseases. Cellular senescence is one of the main hallmarks of aging. A growing body of evidence implicates accelerated mechanisms of aging, including cellular senescence, in idiopathic pulmonary fibrosis (IPF) and chronic obstructive pulmonary disease (COPD) pathogenesis. Senolytics are pharmaceutical agents that eliminate senescent cells, thus blocking tissue degeneration and late life complications and allowing tissue regeneration. Areas covered: The potential use of senolytic drugs in respiratory medicine is examined and discussed...
July 4, 2018: Expert Opinion on Investigational Drugs
Paolo Capogrosso, Francesco Montorsi, Andrea Salonia
Introduction - The prevalence of sexual dysfunctions has increased over the last decades; despite a number of available treatments for erectile dysfunction (ED), premature ejaculation (PE) and Peyronie's disease (PD), still several unmet therapeutic needs deserve to be fulfilled. The aim of this review is to detail on phase I and II clinical trials investigating novel medical treatments for ED, PE and PD. Areas covered - We conducted a systematic review of the literature including both published and on-going phase I and II registered trials focused on medical treatment of ED, PE and PD during the last 5 years...
July 3, 2018: Expert Opinion on Investigational Drugs
Michalis V Karamouzis, Athanasios G Papavassiliou
No abstract text is available yet for this article.
June 29, 2018: Expert Opinion on Investigational Drugs
Jerry R Colca, William G McDonald, Wade J Adams
INTRODUCTION: Non-alcoholic steatohepatitis (NASH) is a serious form of non-alcoholic fatty liver disease (NAFLD) for which there are no marketed treatments. NAFLD is initiated by excess intake of nutrients and recent evidence has pinpointed the mitochondrial pyruvate carrier (MPC) as a mediator of the nutritional overload signals. Areas covered: An overview is given of MSDC-0602K, a new agent in development that modulates the MPC and as such treats the symptoms of fatty liver including dysfunctional lipid metabolism, inflammation, and insulin resistance as well as the key liver pathology including fibrosis...
June 28, 2018: Expert Opinion on Investigational Drugs
Marcus P Watkins, Nancy L Bartlett
Ubiquitous expression of CD19 on B cell non-Hodgkin lymphoma identified it as a potential target for immune-based therapies. Areas covered: This article reviews the current literature on anti-CD19 therapies currently in clinical trials including monoclonal antibodies (mAb), antibody targeted cytotoxic drug conjugates (ADC), bispecific antibodies, and chimeric antigen receptor (CAR) modified T cells. Expert opinion: Naked anti-CD19 mAbs, have shown little clinical benefit in B cell lymphomas. Despite unusual toxicity profiles with many anti-CD19 ADCs slowing development, the durable remissions in a substantial minority of patients with refractory aggressive lymphomas should encourage continued efforts in this area...
June 25, 2018: Expert Opinion on Investigational Drugs
Ferdinando D'Amico, Gionata Fiorino, Federica Furfaro, Mariangela Allocca, Silvio Danese
A new pharmacological class, janus kinases (JAK) inhibitors, has been shown to be effective and safe for the treatment of inflammatory bowel diseases (IBD). The aim of this review is to provide an overview of the JAK inhibitors currently under investigation in phase I and II clinical trials for patients with Crohn's disease (CD) and ulcerative colitis (UC), and the possible future perspectives for the treatment of IBD patients with this class of drugs. Areas covered: This review describes the JAK-STAT pathway and analyzes the efficacy and safety of new small molecules such as filgotinib, upadacitinib, TD-1473, peficitinib and Pf-06651600/Pf-06700841, showing data from phase I and II trials...
June 23, 2018: Expert Opinion on Investigational Drugs
Loris R Lopetuso, Marco Napoli, Gianenrico Rizzatti, Antonio Gasbarrini
The gastrointestinal tract acts as a functional unit organized as a semipermeable multilayer system, in which commensal gut microbiota represents the anatomical barrier. Recently, several studies have highlighted the involvement of gut microbiota in inflammatory bowel diseases (IBD) pathogenesis, in sustaining gut barrier chronic inflammation, and in conditioning disease course and therapeutical response. This evidence provides a rationale for treating patients with gut microbiota modifiers. Among these, Rifaximin represents a non-traditional antibiotic able to act as a 'eubiotic' on intestinal barrier...
June 2018: Expert Opinion on Investigational Drugs
Athanasios E Dellis, Athanasios G Papatsoris
Prostate cancer (PCa) is the most common cancer in elderly males. Androgen deprivation therapy (ADT) is still the cornerstone of initial treatment; however, the vast majority of patients develop castration-resistant prostate cancer (CRPC). Several studies with numerous androgen receptor (AR)-directed agents have emerged since the approval of abiraterone acetate and enzalutamide. One of these agents is apalutamide, which seems to be a promising AR antagonist for the treatment of CRPC. Areas covered: The authors review Phase I, II, and III studies for apalutamide, in a large spectrum of PCa (from low-risk to metastatic CRPC [mCRPC]) patients as sole treatment or in the setting of combined therapy...
June 2018: Expert Opinion on Investigational Drugs
Kelly Valla, Christopher R Flowers, Jean L Koff
Dysregulated B cell receptor (BCR) signaling has been identified as a potent contributor to tumor survival in B cell non-Hodgkin lymphomas (NHLs). This pathway's emergence as a rational therapeutic target in NHL led to development of BCR-directed agents, including inhibitors of Bruton's tyrosine kinase (BTK), spleen tyrosine kinase (SYK), and phosphatidylinositol 3 kinase (PI3K). Several drugs have become valuable assets in the anti-lymphoma armamentarium. Areas covered: We provide an overview of the BCR pathway, its dysregulation in B cell NHL, and the drugs developed to target BCR signaling in lymphoma...
June 2018: Expert Opinion on Investigational Drugs
Rita Khoury, Noam Grysman, Jake Gold, Kush Patel, George T Grossberg
Despite recent advances in Alzheimer's disease (AD) research, no breakthrough treatments have been discovered. Cholinesterase inhibitors and the NMDA-receptor antagonist memantine are currently the two approved symptomatic treatments for AD. 5-HT6 receptor antagonism has recently emerged as a promising treatment strategy to improve cognition in AD, with a modest side-effect profile. Areas covered: 5-HT6 receptors, exclusively found in the central nervous system, modulate primarily GABA and glutamate levels, facilitating the secondary release of other neurotransmitters including dopamine, noradrenaline, and acetylcholine, all of which are compromised in AD...
June 2018: Expert Opinion on Investigational Drugs
Dejana Ružić Zečević, Marko Folić, Ziyad Tantoush, Milan Radovanović, Goran Babić, Slobodan M Janković
The anticonvulsant activity of cannabinoids attracted much attention in the last decade. Cannabinoids that are currently investigated with the intention of making them drugs for the treatment of epilepsy are cannabidiol, cannabidivarin, Δ9-tetrahydrocannabivarin, and Δ9-tetrahydrocannabinolic acid. Areas covered:In this review, the authors look at the results of preclinical and clinical studies with investigational cannabinoids. Relevant literature was searched for in MEDLINE, SCOPUS, EBSCO, GOOGLE SCHOLAR, and SCINDEX databases...
June 2018: Expert Opinion on Investigational Drugs
Karl Heinz Weiss, Anna Członkowska, Peter Hedera, Peter Ferenci
Wilson disease (WD) is a genetic disorder in which excess toxic copper accumulates in the liver, brain, and other tissues leading to severe and life-threatening symptoms. Copper overload can be assessed as non-ceruloplasmin-bound copper non-ceruloplasmin-bound copper (NCC) in blood. Current therapies are limited by efficacy, safety concerns, and multiple-daily dosing. Areas covered: This article reviews the literature on WTX101 (bis-choline tetrathiomolybdate), an oral first-in-class copper-protein-binding agent in development for the treatment of WD...
June 2018: Expert Opinion on Investigational Drugs
Sarah S Ch'Ng, Andrew J Lawrence
Alcohol use disorders (AUDs) are one of the leading causes of preventable death in the developed world. In the U.S., only three FDA-approved pharmacotherapies for AUDs currently exist, but at a population level they display poor efficacy, low compliance rates, and adverse side effects. Therefore, identifying novel neurobiological targets for pharmacological treatment of AUDs is of urgent concern. Areas covered: We discuss recent preclinical data on investigational drugs that have been assessed for their therapeutic potential in AUDs...
May 2018: Expert Opinion on Investigational Drugs
Alejandro Schcolnik-Cabrera, Alma Chávez-Blanco, Guadalupe Domínguez-Gómez, Lucia Taja-Chayeb, Rocio Morales-Barcenas, Catalina Trejo-Becerril, Enrique Perez-Cardenas, Aurora Gonzalez-Fierro, Alfonso Dueñas-González
Cancer cells have increased glycolysis and glutaminolysis. Their third feature is increased de novo lipogenesis. As such, fatty acid (FA) synthesis enzymes are over-expressed in cancer and their depletion causes antitumor effects. As fatty acid synthase (FASN) plays a pivotal role in this process, it is an attractive target for cancer therapy. Areas covered: This is a review of the lipogenic phenotype of cancer and how this phenomenon can be exploited for cancer therapy using inhibitors of FASN, with particular emphasis on orlistat as a repurposing drug...
May 2018: Expert Opinion on Investigational Drugs
Gian Benedetto Melis, Manuela Neri, Bruno Piras, Anna Maria Paoletti, Silvia Ajossa, Monica Pilloni, Maria Francesca Marotto, Valentina Corda, Alessandra Saba, Elena Giancane, Valerio Mais
The medical strategy to antagonize myoma size and related-symptoms is to reduce estrogen and progesterone activity on myomas. This can be obtained with the GnRH agonist (GnRHa) or with compounds that antagonize progesterone stimulatory activity on myomas. Selective progesterone receptor modulators (SPRMs) bind progesterone receptor (PR), leading to both agonist and antagonist effects. The result of SPRMs's action is tissue-specific and it depends on the particular affinity and strength of each SPRM. Area covered: Ulipristal acetate (UPA) is the first SPRM registered for myoma treatment...
May 2018: Expert Opinion on Investigational Drugs
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