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Expert Opinion on Investigational Drugs

Prithviraj Bose, Srdan Verstovsek
The Philadelphia chromosome negative myeloproliferative neoplasms (MPN) mainly comprise polycythemia vera (PV), essential thrombocythemia (ET) and myelofibrosis (MF, primary or post-PV/ET). Therapy in PV and ET focuses on minimizing thrombosis and bleeding risk, while in MF, prolongation of survival is an important goal. Different cytoreductive agents are employed in high risk PV and ET, while the JAK inhibtior ruxolitinib is the cornerstone of therapy in MF. Histone deacetylase inhibitors (HDACi) are pleiotropic agents with diverse epigenetic and non-epigenetic actions, selectively in transformed cells...
October 18, 2016: Expert Opinion on Investigational Drugs
Marcella Nebbioso, Valeria Fameli, Magda Gharbiya, Marta Sacchetti, Anna Maria Zicari, Alessandro Lambiase
The dry eye disease (DED) is a chronic multifactorial disorder of the tears that also involves the ocular surface, the lacrimal glands, and meibomian dysfunction. Furthermore, DED is often associated with Sjögren's syndrome (SS) and other autoimmune disorders. Sometimes, this chronic or subclinical condition is difficult to diagnose and treat, due to its heterogeneity. Areas covered: A literature search of relevant publications about treatment of DED was performed. All relevant articles published between 2011 and 2016 were identified through a computerized search for review and clinical trials using Pub Med database...
October 18, 2016: Expert Opinion on Investigational Drugs
Luca Semerano, Patrice Decker, Gaëlle Clavel, Marie-Christophe Boissier
No abstract text is available yet for this article.
October 17, 2016: Expert Opinion on Investigational Drugs
Nóra Török, Zsófia Majláth, Levente Szalárdy, László Vécsei
The therapeutic management of Parkinson's disease (PD) is challenging and has not been fully resolved. The main challenges include motor fluctuations and levodopa-induced dyskinesia. Moreover, no disease-modifying or neuroprotective therapy is currently available. Areas covered: This review focuses on α-synuclein aggregation inhibitors and their therapeutic role in PD, with special attention to heat shock proteins, immunotherapy (active and passive), the potential of targeting the Ser129 phosphorylation site, and the antibiotic possibilities...
October 8, 2016: Expert Opinion on Investigational Drugs
A L Moekotte, M A M Huson, A J van der Ende, S T Agnandji, E Huizenga, A Goorhuis, M P Grobusch
To date, the management of patients with suspected or confirmed Ebolavirus disease (EVD) depends on quarantine, symptomatic management and supportive care, as there are no approved vaccines or treatments available for human use. However, accelerated by the recent large outbreak in West Africa, significant progress has been made towards vaccine development but also towards specific treatment with convalescent plasma and monoclonal antibodies. Areas covered: We describe recent developments in monoclonal antibody treatment for EVD, encompassing mAb114 and the MB-003, ZMAb, ZMapp™ and MIL-77E cocktails...
October 8, 2016: Expert Opinion on Investigational Drugs
José-Manuel Gómez-Sáez
Sunitinib is an oral oxindol derivative and a potent inhibitor of vascular endothelial growth factor receptor and platelet-derived growth factor receptor and a multitargeted tyrosine-kinase inhibitor, which has antitumor and antiangiogenic activity due to the selective inhibition that can stabilize progressive metastatic disease. The aim of this review is to expose whether the drug could be considered as a new promising therapy compared with other tyrosine-kinase inhibitors. Areas covered: In seven open-label studies carried out with sunitinb, the drug showed its anti-tumoral activity in advanced differentiated thyroid carcinoma and in medullary thyroid carcinoma...
October 6, 2016: Expert Opinion on Investigational Drugs
Alexander Arlt, Heiner Schäfer
No abstract text is available yet for this article.
October 5, 2016: Expert Opinion on Investigational Drugs
J Tamargo, Luis Miguel Ruilope
Voltage-gated Ca(2+) channels are the primary route of Ca(2+) entry in vascular smooth muscle cells, playing a key role in the regulation of arterial tone and blood pressure. Since the 60´s, L-type Ca(2+) channel blockers (CCBs) have been widely used for the treatment of hypertension. Areas covered. T-type Ca(2+) channels regulate vascular tone in small-resistance vessels and aldosterone secretion, and N-type channels expressed in sympathetic nerve terminals regulate the release of neurotransmitters. We performed a literature search in MEDLINE, PubMed and ClinicalTrials...
October 4, 2016: Expert Opinion on Investigational Drugs
Molly Campa, Alan Menter
INTRODUCTION: Interleukin-17 has recently been identified as a key player in the pathogenesis of psoriasis. As such, several drugs targeting IL-17 are in various stages of clinical development. AREAS COVERED: In this review, the authors describe several emerging therapies and drug candidates targeting IL-17. The authors detail many biologic injectable drug candidates as well as numerous potential oral and topical small molecule drug candidates. EXPERT OPINION: Approval of IL-17 inhibitors has significantly improved the treatment options for psoriasis patients...
September 30, 2016: Expert Opinion on Investigational Drugs
Dhaval Kolte, Wilbert S Aronow, Maciej Banach
INTRODUCTION: Cardiovascular diseases (CVD) remain the leading cause of death worldwide with an estimated 17.5 million deaths per year. Since its initial conception over a decade ago, the use of cardiovascular polypills has gained increasing momentum as a strategy to lower risk factor levels and prevent CVD. Several new data have emerged including the recent publication of the first outcomes trial using polypills. AREAS COVERED: In this review, the authors summarize the current literature on the safety, efficacy, and cost-effectiveness of polypills for primary and secondary prevention of CVD, describe the current controversies in this field, and identify important areas for future research...
September 22, 2016: Expert Opinion on Investigational Drugs
A I Fraguas-Sánchez, A Fernández-Carballido, A I Torres-Suárez
INTRODUCTION: The term "cannabinoids" designates a family of compounds with activity upon cannabinoid receptors. Cannabinoids are classified in three groups: phytocannabinoids, endocannabinoids, and the synthetic analogues of both groups. They have become a promising tool in the treatment of cancer disease, not only as palliative agents, but also as antitumor drugs, due to their ability to inhibit the proliferation, adhesion, migration, invasion, and angiogenesis of tumour cells. Two of the cancers where they have shown high anticancer activity are breast and prostate tumours...
September 16, 2016: Expert Opinion on Investigational Drugs
Marie-Françoise Heymann, Hannah Brown, Dominique Heymann
INTRODUCTION: Osteosarcomas are the main malignant primary bone tumours found in children and young adults. Conventional treatment is based on diagnosis and resection surgery, combined with polychemotherapy. This is a protocol that was established in the 1970s. Unfortunately, this therapeutic approach has reached a plateau of efficacy and the patient survival rate has not improved in the last four decades. New therapeutic approaches are thus required to improve the prognosis for osteosarcoma patients...
September 15, 2016: Expert Opinion on Investigational Drugs
(no author information available yet)
No abstract text is available yet for this article.
November 2016: Expert Opinion on Investigational Drugs
Carmen Di Giovanni, Ettore Novellino, Adriana Chilin, Antonio Lavecchia, Giovanni Marzaro
INTRODUCTION: Cell cycle and gene transcription are under the control of cyclin-dependent kinases (CDKs), whose activity depends on the binding with cyclins. Deregulated CDK activities have been reported in a majority of human cancers, representing potential therapeutic targets. AREAS COVERED: This review provides preclinical and clinical (phase I/II) updates of promising therapeutic compounds targeting CDKs published between 2013 and 2016 Expert opinion: First generation pan-CDK inhibitors showed marked toxicity in clinical trials and most compounds were discontinued...
October 2016: Expert Opinion on Investigational Drugs
Brian Tomlinson, Miao Hu, Yuzhen Zhang, Paul Chan, Zhong-Min Liu
INTRODUCTION: Obesity is a worldwide problem predisposing to type 2 diabetes mellitus (T2DM), hypertension, cardiovascular disease, cancer and other comorbidities. Lifestyle modification is the first line intervention but adjunctive pharmacotherapy is often required. The GLP-1 receptor agonists (GLP-1RAs) were developed primarily for T2DM and they also reduce body weight. Liraglutide was approved for the treatment of obesity and other GLP-1RAs are likely to be suitable for this indication...
October 2016: Expert Opinion on Investigational Drugs
Sabina Antonela Antoniu
INTRODUCTION: In asthma, most commonly, 'conventional' anti-inflammatory medications represented by inhaled corticosteroids and leukotriene inhibitors are effective. In some patients however additional inhibition of the airways inflammation is necessary. Such compounds might be molecules inhibiting specifically certain inflammation pathways and lebrikizumab an anti IL-13 molecule might represent a relevant example as a potential asthma therapy. AREAS COVERED: Discussion of the rationale for the use of lebrikizumab in asthma...
October 2016: Expert Opinion on Investigational Drugs
Jed Asher Lusthaus, Ivan Goldberg
INTRODUCTION: Intraocular pressure (IOP) is the most significant modifiable risk factor to prevent onset or progression of glaucoma. Glaucoma prevalence continues to increase, emphasizing the need for improved ocular hypotensive treatment options. To try to improve on both tolerance and IOP control of currently available therapies, different receptors or mechanisms are being explored to reduce IOP more effectively and to improve tolerance. AREAS COVERED: We review synthetic topical and oral drugs in early development for the management of ocular hypertension and glaucoma...
October 2016: Expert Opinion on Investigational Drugs
Guglielmo Borgia, Alberto Enrico Maraolo, Antonio Riccardo Buonomo, Riccardo Scotto, Ivan Gentile
INTRODUCTION: Hepatitis C virus (HCV) infection is a leading cause of liver cirrhosis, hepatocellular carcinoma and liver-related death worldwide. Currently, the anti-HCV armamentarium encompasses several direct-acting antivirals (DAA) that achieve very high response rates and have an excellent tolerability profile. However, they do not represent a final solution for HCV global eradication for at least these two reasons: i) some patients harbour resistant strains to DAAs and cannot benefit from currently available treatments; ii) the cost of these drugs remains very high...
October 2016: Expert Opinion on Investigational Drugs
Andrew D Goodman, Tirisham Gyang, Andrew D Smith
INTRODUCTION: Multiple sclerosis (MS) is an autoimmune disorder of the central nervous system (CNS) characterized by inflammatory demyelination and progressive axonal loss. Clinically, this is manifest as relapsing and remitting neurological symptoms and progressive accumulation of disability. Ibudilast is a nonselective phosphodiesterase inhibitor which works by blocking the cleavage of cyclic adenosine monophosphate (cAMP). It has been found to have anti-inflammatory and neuroprotective properties in animal studies and in-vitro studies; it is currently being studied in progressive MS...
October 2016: Expert Opinion on Investigational Drugs
Gregory N Gan, Antonio Jimeno
INTRODUCTION: Cancer treatment is moving away from conventional cytotoxic drugs and towards agents that target specific proteins and mechanisms important to cancer development or survival. The Hedgehog Pathway (HhP) is a signal transduction pathway and its constitutive activation is tumorigenic in basal cell carcinoma (BCC). The HhP enables phenotypic flexibility, and channels tumor-stroma interactions. As a result, it is over-expressed in numerous cancers as well as in the tumor microenvironment and may represent a promising therapeutic target...
October 2016: Expert Opinion on Investigational Drugs
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