Emerging Drugs for the Treatment of Amyotrophic Lateral Sclerosis: A Focus on Recent Phase 2 Trials.
Authors: Barp A, Gerardi F, Lizio A, Sansone VA, Lunetta C Abstract INTRODUCTION: Amyotrophic lateral sclerosis (ALS) is a rapidly progressive neurodegenerative disease involving both upper and lower motor neurons and resulting in increasing disability and death 3-5 years after onset of symptoms. Over 40 large clinical trials for ALS have been negative, except for Riluzole that offers a modest survival benefit, and Edaravone that modestly reduces disease progression in patients with specific characteristics. Thus, the discovery of efficient disease modifying therapy is an urgent need. AREAS COVERED: Althou...
Source: Expert Opinion on Emerging Drugs - May 29, 2020 Category: Drugs & Pharmacology Tags: Expert Opin Emerg Drugs Source Type: research

Treatments in the COVID-19 pandemic: an update on clinical trials.
Authors: Tao YY, Tang LV, Hu Y PMID: 32447996 [PubMed - as supplied by publisher] (Source: Expert Opinion on Emerging Drugs)
Source: Expert Opinion on Emerging Drugs - May 26, 2020 Category: Drugs & Pharmacology Tags: Expert Opin Emerg Drugs Source Type: research

Emerging 5-HT receptor antagonists for the treatment of Schizophrenia.
Authors: Jones MT, Strassnig MT, Harvey PD Abstract INTRODUCTION: While antipsychotics have been generally successful in treating psychosis in schizophrenia, there is a major treatment gap for negative symptoms and cognitive deficits. Given that these aspects of the disease contribute to poor functional outcomes independently of positive symptoms, treatments would have profound implications for quality of life. The 5-HT2A- receptor has been considered a potential target for interventions aimed at negative and cognitive symptoms and multiple antagonists and inverse agonists of this receptor have been tested. ARE...
Source: Expert Opinion on Emerging Drugs - May 26, 2020 Category: Drugs & Pharmacology Tags: Expert Opin Emerg Drugs Source Type: research

Emerging drugs for the treatment of L-DOPA-induced dyskinesia: an update.
Authors: AlShimemeri S, Fox SH, Visanji NP Abstract Introduction: Prolonged treatment with l-3,4-dihydroxyphenylalanine (L-DOPA) leads to the development of uncontrolled movements (L-DOPA-induced dyskinesias (LID)) in Parkinson's disease (PD). There is currently only a single approved drug for the treatment of LID, a long-acting preparation of the NMDA antagonist, amantadine, that has variable benefit and side-effects. Therefore, new treatments for LID remains an unmet in PD.Areas covered: We review the current strategies for the management of LID; the pathogenic mechanisms underlying the development of LID, which ...
Source: Expert Opinion on Emerging Drugs - May 6, 2020 Category: Drugs & Pharmacology Tags: Expert Opin Emerg Drugs Source Type: research

Emerging muscarinic receptor antagonists for the treatment of asthma.
Authors: Matera MG, Belardo C, Rinaldi M, Rinaldi B, Cazzola M Abstract Introduction: The critical role of acetylcholine in asthma pathophysiology and the increase in vagal tone are the main reasons why a LAMA is a good choice in the treatment of the asthmatic patient. Tiotropium is the only LAMA approved for use in asthma in many countries but there is a real interest in developing novel LAMAs for the treatment of asthma, or at least to extend this indication to other LAMAs already on the market.Areas covered: Trials that have studied or are evaluating the role of LAMAs already on the market in asthma and possible...
Source: Expert Opinion on Emerging Drugs - April 22, 2020 Category: Drugs & Pharmacology Tags: Expert Opin Emerg Drugs Source Type: research

Emerging drugs for the treatment of primary biliary cholangitis.
Authors: Phaw NA, Dyson JK, Jones D Abstract Introduction: Primary biliary cholangitis (PBC) is a progressive inflammatory autoimmune cholestatic liver disease. Without treatment, it may result in fibrosis and eventually end stage liver disease. In addition to the disease burden, the symptom impact on the quality of life for PBC patients is significant. Ursodeoxycholic acid, and the second-line therapy Obeticholic acid, are the only available licenced treatments. Although there has been rapid development of novel therapies in recent years for the treatment of PBC, there are very few symptoms directed therapies.Area...
Source: Expert Opinion on Emerging Drugs - April 9, 2020 Category: Drugs & Pharmacology Tags: Expert Opin Emerg Drugs Source Type: research

Emerging therapies in β-Thalassemia: towards a new era in management.
Emerging therapies in β-Thalassemia: towards a new era in management. Expert Opin Emerg Drugs. 2020 Apr 05;: Authors: Bou-Fakhredin R, Tabbikha R, Daadaa H, Taher AT Abstract Introduction: The thalassemias constitute one of the common inherited monogenic diseases worldwide, characterized by autosomal recessive inherited defects in the production of hemoglobin. Currently available conventional therapies have many challenges and limitations. Advances in understanding the underlying pathophysiology of β-thalassemia enabled clinicians and researchers to move towards the development of novel thera...
Source: Expert Opinion on Emerging Drugs - April 8, 2020 Category: Drugs & Pharmacology Tags: Expert Opin Emerg Drugs Source Type: research

Emerging systemic drugs in the treatment of plaque psoriasis.
This article reviews systemic biologic and oral small molecule drugs currently undergoing clinical trials for the treatment of plaque psoriasis.Expert opinion: Many patients with psoriasis have mild disease, and many with mild disease do not seek medical care for their condition. Many patients with mild disease could be adequately treated with topical treatments and phototherapy; however, adherence and feasibility have often been an issue with these treatment types. There seems to be limited room for development of novel biologics, as the existing ones are extraordinarily safe, effective, and convenient with few injections...
Source: Expert Opinion on Emerging Drugs - March 22, 2020 Category: Drugs & Pharmacology Tags: Expert Opin Emerg Drugs Source Type: research

Emerging therapies for the treatment of ulcerative colitis.
Authors: Pugliese N, Roda G, Peyrin-Biroulet L, Danese S Abstract Introduction: Ulcerative colitis (UC) is a chronic idiopathic autoimmune inflammatory disorder, primarily affecting the gastrointestinal system. There are many patients affected that do not respond well to therapy and many others to which there is a loss of efficacy every year. The proportion of patients who have already experienced anti-TNF therapy is constantly increasing, making the development of new drugs with alternative mechanisms of action an important need for the treatment of UC.Areas covered: This review aims on emerging drugs in the treat...
Source: Expert Opinion on Emerging Drugs - March 10, 2020 Category: Drugs & Pharmacology Tags: Expert Opin Emerg Drugs Source Type: research

Novel targeted therapies for the management of liver fibrosis.
Authors: Trivella JP, Martin P, Carrion AF Abstract Introduction: Prolonged liver injury results in tissue damage and replacement by extracellular matrix and fibrosis. Cirrhosis represents a leading cause of mortality worldwide and imposes a major financial burden on health care systems. Fortunately, fibrogenesis has proven to be reversible if halted early, encouraging the development of novel anti-fibrotic agents that may accelerate histological restoration. Preclinical data have elucidated numerous potential therapeutic targets and many anti-fibrotic agents are currently at various stages of clinical research.Are...
Source: Expert Opinion on Emerging Drugs - February 28, 2020 Category: Drugs & Pharmacology Tags: Expert Opin Emerg Drugs Source Type: research

Pharmacology and potential role of selatogrel, a subcutaneous platelet P2Y12 receptor antagonist.
Authors: Parker WAE, Storey RF PMID: 32064955 [PubMed - as supplied by publisher] (Source: Expert Opinion on Emerging Drugs)
Source: Expert Opinion on Emerging Drugs - February 19, 2020 Category: Drugs & Pharmacology Tags: Expert Opin Emerg Drugs Source Type: research

Actinic keratosis: where do we stand and where is the future going to take us?
Authors: Cramer P, Stockfleth E Abstract Introduction: Actinic keratosis (AK) is a chronic disease which is mainly located across areas of sun exposed skin. Clinical and subclinical lesions coexist across a large area resulting in a field cancerization. As these lesions have the potential to transform into invasive squamous cell carcinoma (iSCC), treatment is crucial. With global prevalence increasing, AK is expected to be the most common in situ carcinoma of the skin.Areas covered: In this article, we cover the established algorithm of treating AK and give an insight into the drugs under development. There are six...
Source: Expert Opinion on Emerging Drugs - February 19, 2020 Category: Drugs & Pharmacology Tags: Expert Opin Emerg Drugs Source Type: research

Managing autonomic dysfunction in Parkinson's disease: a review of emerging drugs.
Authors: Kulshreshtha D, Ganguly J, Jog M Abstract Introduction: Autonomic dysfunction is an integral part of Parkinson disease (PD) complex and can be seen both in early and advanced stages. There is a paucity of medicines available to manage autonomic dysfunction in PD and this adds to the considerable morbidity associated with the illness.Areas covered: The pathophysiology and the available therapeutic options of autonomic dysfunction seen in PD are discussed in detail. The potential targets for novel regimens are reviewed and the available literature on the drugs emerging in management of autonomic dysfunction ...
Source: Expert Opinion on Emerging Drugs - February 19, 2020 Category: Drugs & Pharmacology Tags: Expert Opin Emerg Drugs Source Type: research

Emerging bruton tyrosine kinase inhibitors for chronic lymphocytic leukaemia: one step ahead ibrutinib.
Authors: Molica S, Gianfelici V, Levato L Abstract Introduction: In the last few years, the expansion of therapy with pathway inhibitors has revolutionised the treatment landscape of chronic lymphocytic leukemia (CLL). As matter of fact, ibrutinib, the first-in-class Bruton tyrosine kinase (BTK) inhibitor, became a milestone in the treatment of both naïve or relapsed/refractory CLL patients. Most patients treated with such an agent achieve durable clinical response, however, a deeper response is rarely reached and continuous treatment is required. Since ibrutinib-resistant CLL clones can develop in about 20% o...
Source: Expert Opinion on Emerging Drugs - February 1, 2020 Category: Drugs & Pharmacology Tags: Expert Opin Emerg Drugs Source Type: research

Emerging drugs for the treatment of vitiligo.
This study comprehensively summarises the current concepts in the pathogenesis of vitiligo with special focus on the cytokine and signalling pathways, which are the targets for newer drugs. JAK kinase signalling pathways and the cytokines involved are the focus of vitiligo treatment in current research, followed by antioxidant mechanisms and repigmenting mechanisms. Topical immunosuppressants may be an alternative to steroids in localised vitiligo. Newer repigmenting agents like basic fibroblast growth factors, afamelanotide have been included and a special emphasis is laid on the upcoming targeted immunotherapy.Expert opi...
Source: Expert Opinion on Emerging Drugs - January 22, 2020 Category: Drugs & Pharmacology Tags: Expert Opin Emerg Drugs Source Type: research

Correction.
Authors: PMID: 31914874 [PubMed - in process] (Source: Expert Opinion on Emerging Drugs)
Source: Expert Opinion on Emerging Drugs - January 10, 2020 Category: Drugs & Pharmacology Tags: Expert Opin Emerg Drugs Source Type: research

What potential is there for LSD1 inhibitors to reach approval for AML?
Authors: Pandey MR, Wang ES PMID: 31914875 [PubMed - in process] (Source: Expert Opinion on Emerging Drugs)
Source: Expert Opinion on Emerging Drugs - January 10, 2020 Category: Drugs & Pharmacology Tags: Expert Opin Emerg Drugs Source Type: research

Emerging serine-threonine kinase inhibitors for treating ovarian cancer.
Authors: Maoz A, Ciccone MA, Matsuzaki S, Coleman RL, Matsuo K Abstract Introduction: Ovarian cancer is the leading cause of gynecologic cancer death, owing to high rates of incurable, recurrent disease after initial treatment. Serine threonine kinases (STKs) have been proposed as potential therapeutic targets in ovarian cancer because of their role in the initiation and progression of cancers. Experience in non-ovarian cancers suggests that STK inhibitors are active against tumors with specific molecular alterations.Areas covered: This review discusses STK inhibitors in active development in phase II/III clinical ...
Source: Expert Opinion on Emerging Drugs - November 24, 2019 Category: Drugs & Pharmacology Tags: Expert Opin Emerg Drugs Source Type: research

New immunotherapy in the treatment of advanced renal cancer.
Authors: Muto A, Gridelli C Abstract Introduction: Metastatic renal cell carcinoma (mRCC) in recent years has been one of the diseases in the oncology that has experienced the greatest development of targeted therapies.Area covered: On one hand, more and more refined acquisitions of molecular biology are allowing a better sub-classification of this tumor, identifying new and peculiar biological profiles of patients with metastatic renal cancer that will soon be destined for increasingly tailored treatments. On the other hand, immunotherapy has, in a short time, redesigned the perimeter of our knowledge, entering cl...
Source: Expert Opinion on Emerging Drugs - November 23, 2019 Category: Drugs & Pharmacology Tags: Expert Opin Emerg Drugs Source Type: research

Emerging drugs for the treatment of onychomycosis.
Authors: Gupta AK, Stec N Abstract Introduction: Onychomycosis is a widespread nail disease, often occurring on the feet. It is a chronic and often recurring disease, which makes it difficult to eradicate. The infection may be caused by dermatophytes, non-dermatophyte molds, and yeasts. Traditionally, systemic antifungal medications have been used to treat this infection, but in recent years topical formulations have been the focus of research. Areas covered: This review outlines the current antifungal market and novel treatments currently in development or in experimental phases. It highlights a shift from systemi...
Source: Expert Opinion on Emerging Drugs - October 30, 2019 Category: Drugs & Pharmacology Tags: Expert Opin Emerg Drugs Source Type: research

Emerging antibiotics for Community-Acquired Pneumonia.
Authors: Liapikou A, Cilloniz C, Palomepue A, Torres T Abstract Introduction: Community-acquired pneumonia is the most common infection leading to hospitalization and death in all age groups, especially in elderly populations. Increasing antibiotic resistance among the common bacterial pathogens associated with community-acquired pneumonia, especially Streptococcus pneumoniae and staphylococci, has made its empirical treatment increasingly problematic, highlighting the need for effective antibiotic therapy. Areas covered: We searched PubMed and ClinicalTrials.gov for English-language reports of phase III clinical t...
Source: Expert Opinion on Emerging Drugs - October 30, 2019 Category: Drugs & Pharmacology Tags: Expert Opin Emerg Drugs Source Type: research

Emerging drugs for treating methicillin-resistant Staphylococcus aureus.
Authors: Bassetti M, Russo A, Carnelutti A, Wilcox M Abstract Introduction: In clinical practice, methicillin-resistant Staphylococcus aureus (MRSA) represents a major threat and has been associated with high rates of inadequate antibiotic treatment and significant increases in morbidity, mortality and overall healthcare costs. The association between the prescription of an inappropriate or delayed antibiotic and impaired clinical outcomes has been widely described. Areas covered: To address the threat of MRSA, many new therapeutic options with a peculiar activity against MRSA have been recently developed and appro...
Source: Expert Opinion on Emerging Drugs - October 10, 2019 Category: Drugs & Pharmacology Tags: Expert Opin Emerg Drugs Source Type: research

Emerging drugs for the treatment of noninfectious uveitis.
This article discusses existing local and systemic applied treatments for ocular inflammation including corticosteroids, non-biologic, and biologic disease-modifying anti-rheumatic drugs (DMARD). Potential drugs being studied in clinical trials are introduced for both local and systemic use. Expert opinion: Treatment options for uveitis continue to expand. Still, more efforts and research are needed to better understand the mechanisms potentially leading to clinical trials. PMID: 31498689 [PubMed - as supplied by publisher] (Source: Expert Opinion on Emerging Drugs)
Source: Expert Opinion on Emerging Drugs - September 11, 2019 Category: Drugs & Pharmacology Tags: Expert Opin Emerg Drugs Source Type: research

An update on emerging drugs in osteosarcoma: towards tailored therapies?
Authors: Hattinger CM, Patrizio MP, Magagnoli F, Luppi S, Serra M Abstract Introduction: Current treatment of conventional and non-conventional high-grade osteosarcoma (HGOS) is based on the surgical removal of primary tumor and, when possible, of metastases and local reccurrence, together with systemic pre- and post-operative chemotherapy with drugs that have been used since decades. Areas covered: This review is intended to summarize the new agents and therapeutic strategies that are under clinical evaluation in HGOS, with the aim to increase the cure probability of this highly malignant bone tumor, which has not...
Source: Expert Opinion on Emerging Drugs - August 15, 2019 Category: Drugs & Pharmacology Tags: Expert Opin Emerg Drugs Source Type: research

Emerging protein kinase inhibitors for the treatment of multiple myeloma.
Authors: Lind J, Czernilofsky F, Vallet S, Podar K Abstract Introduction: Significant advances have been made during the last two decades in terms of new therapeutic options but also of innovative approaches to diagnosis and management of multiple myeloma (MM). Indeed, proteasome inhibitors, immunomodulatory drugs, and monoclonal antibodies have fundamentally changed the landscape of MM treatment. However, while patient survival has been significantly prolonged, most patients relapse. Including the milestone approval of the first kinase inhibitor imatinib mesylate for chronic myeloid leukemia in 2001, 48 small mole...
Source: Expert Opinion on Emerging Drugs - July 23, 2019 Category: Drugs & Pharmacology Tags: Expert Opin Emerg Drugs Source Type: research

Emerging drugs for primary Sj ögren's syndrome.
Emerging drugs for primary Sjögren's syndrome. Expert Opin Emerg Drugs. 2019 Jun;24(2):121-132 Authors: Gandolfo S, De Vita S Abstract Introduction: Primary Sjögren's syndrome (pSS) is an autoimmune systemic disease characterized by a complex and not yet completely elucidated etiopathogenesis, where autoimmune manifestations coexist with different degree of lymphoproliferation, resulting in multiple possible scenarios extremely heterogeneous from patient to patient. Although considerable progress has been made in the identifications of potential novel therapeutic targets in recent years, the ...
Source: Expert Opinion on Emerging Drugs - July 11, 2019 Category: Drugs & Pharmacology Tags: Expert Opin Emerg Drugs Source Type: research

Emerging angiogenesis inhibitors for non-small cell lung cancer.
Authors: Malapelle U, Rossi A Abstract INTRODUCTION: Angiogenesis represent a complex process crucial during embryo development, wound healing, and collateral formation for improved organ perfusion. Numerous stimulatory and inhibitory pathways through their balance regulate angiogenesis and vascular homeostasis. Targeting the pathways implicated in the regulation of angiogenesis and neo-angiogenesis play an important role in cancers research, treatment and patients' outcome. Antiangiogenic strategies, including monoclonal antibodies binding vascular endothelial growth factor (VEGF) or the corresponding receptor and...
Source: Expert Opinion on Emerging Drugs - May 17, 2019 Category: Drugs & Pharmacology Tags: Expert Opin Emerg Drugs Source Type: research

Emerging drugs for essential thrombocythemia.
Authors: Masarova L, Verstovsek S Abstract INTRODUCTION: Despite our recent progress in the understanding of essential thrombocythemia (ET) pathogenesis, the therapeutic management of this disease has remained largely unchanged in the past decades. Treatment has mostly focused on decreasing the risk of complications, especially prevention of thrombotic or hemorrhagic events. Areas covered: Over recent years, the treatment options of ET have been expanding with some novel agents on the horizon. The classes of agents described in this review include targeted and immunomodulatory agents, such as JAK1/2 inhibitors, int...
Source: Expert Opinion on Emerging Drugs - May 7, 2019 Category: Drugs & Pharmacology Tags: Expert Opin Emerg Drugs Source Type: research

Emerging drugs for progressive supranuclear palsy.
Authors: Giagkou N, Stamelou M Abstract INTRODUCTION: Progressive supranuclear palsy (PSP) is a common cause of atypical parkinsonism and a rapidly progressive disease that greatly burdens both patients and caregivers. Drugs with disease-modifying potential, targeting mechanisms implicated in the disease's pathogenesis are currently tested in Phase 1 and 2 trials. If proven efficacious, these compounds might provide substantial benefits not only to patients with PSP, but to patients with other tauopathies as well. Areas covered: Drugs in Phase 1 and 2 trials in PSP, and Phase 2 trials in other tauopathies (Alzheime...
Source: Expert Opinion on Emerging Drugs - April 23, 2019 Category: Drugs & Pharmacology Tags: Expert Opin Emerg Drugs Source Type: research

Emerging PEGylated non-biologic drugs.
Authors: Park EJ, Choi J, Lee KC, Na DH Abstract INTRODUCTION: PEGylation is a well-established technology for improving the therapeutic value of drugs by attaching polyethylene glycol (PEG). The first PEGylated enzyme products appeared on the market in the early 1990s; currently, more than 18 PEGylated products have been approved by Food and Drug Administration, which encompass various classes of drug molecules, such as enzymes, interferons, granulocyte colony-stimulating factors, hormones, antibody fragments, coagulation factors, oligonucleotide aptamers, synthetic peptides, and small organic molecules. Areas cov...
Source: Expert Opinion on Emerging Drugs - April 10, 2019 Category: Drugs & Pharmacology Tags: Expert Opin Emerg Drugs Source Type: research

Factor XI Inhibition fulfilling the optimal expectations for ideal anticoagulation.
Authors: Székely O, Borgi M, Lip GYH Abstract INTRODUCTION: Thromboembolic diseases are leading cause of mortality accounting for an estimated 1 in 4 deaths all over the world. Anticoagulation remains the mainstay of prevention and treatment of venous thromboembolic disorders. Conventional anticoagulants have been efficiently used over the last decades, but their clinical use encounters safety and convenience issues. To overcome these limitations, research have focused on development of new targets for anticoagulation leading to a relatively new class of drugs, non-vitamin K antagonist oral anticoagulants, s...
Source: Expert Opinion on Emerging Drugs - March 10, 2019 Category: Drugs & Pharmacology Tags: Expert Opin Emerg Drugs Source Type: research

What role do pharmaceuticals play in the treatment of Peyronie's disease and is there a need for new emerging drugs?
Authors: Milenkovic U, Ilg MM, Cellek S, Albersen M PMID: 30845848 [PubMed - as supplied by publisher] (Source: Expert Opinion on Emerging Drugs)
Source: Expert Opinion on Emerging Drugs - March 10, 2019 Category: Drugs & Pharmacology Tags: Expert Opin Emerg Drugs Source Type: research

Emerging drugs for the treatment of hypercholesterolemia.
Authors: Bove M, Cicero AFG, Borghi C Abstract Introduction Despite the consolidated role of statins and ezetimibe to treat hypercholesterolemia, often the desirable Low-Density-Lipoprotein-cholesterol (LDL-C) values are not achieved, with a consequent increase of the residual cardiovascular (CV) risk. Areas covered In this review, we summarize the main pharmacological characteristics of new lipid-lowering drugs, such as Proprotein Convertase Subtilisin/Kexin type 9 (PCSK9) Inhibitors, Cholesteryl-Ester-Transfer Protein inhibitors, Microsomal Triglyceride Transfer Protein inhibitors, ATP citrate lyase inhibitors, A...
Source: Expert Opinion on Emerging Drugs - March 8, 2019 Category: Drugs & Pharmacology Tags: Expert Opin Emerg Drugs Source Type: research

Emerging drugs for the treatment of Clostridium difficile.
Authors: Cammarota G, Gallo A, Ianiro G, Montalto M Abstract Introduction Clostridium difficile, or Clostridioides difficile, (C difficile) infection represents the most common cause of healthcare-associated infection. Over the last decades, the incidence and severity of C difficile infection is rapidly increasing, with a significant impact on morbidity and mortality, and burden on health care system. Orally administered vancomycin and fidaxomicin are the therapeutic options of choice for initial C difficile infection and fecal microbiota transplant for the recurrence infection. Furthermore, in recent years several...
Source: Expert Opinion on Emerging Drugs - March 8, 2019 Category: Drugs & Pharmacology Tags: Expert Opin Emerg Drugs Source Type: research

Standard treatment and emerging drugs for managing synovial sarcoma: adult's and pediatric oncologist perspective.
Authors: Baldi GG, Orbach D, Bertulli R, Magni C, Sironi G, Ferrari A Abstract INTRODUCTION: in this review we discuss the standard of care for both pediatric and adult synovial sarcoma (SS), the prognostic differences between them, and the treatments available for localized and advanced diseases. We also overview the biology and the recent drugs under consideration in clinical trials on SS. Areas covered: we focus on new targeted therapies being investigated for advanced SS, especially anti-angiogenic drugs, and immunotherapy. We review all the published data and ongoing trials dedicated to SS or to soft tissue sa...
Source: Expert Opinion on Emerging Drugs - March 8, 2019 Category: Drugs & Pharmacology Tags: Expert Opin Emerg Drugs Source Type: research

Emerging drugs for treating the acute respiratory distress syndrome.
Authors: Shaw TD, McAuley DF, O'Kane CM Abstract Introduction The acute respiratory distress syndrome (ARDS) is a common and catastrophic condition, with a high mortality rate and economic burden on society. Despite 50 years of study, there is no specific pharmacological therapy for ARDS. Areas covered This review outlines the definitions, epidemiology, risk factors and pathophysiology of ARDS. The priority of developing a clinically-relevant model for ARDS to test pre-clinical candidates is discussed, together with the limitations of current models. The scientific rationale of emerging therapeutic candidates is ou...
Source: Expert Opinion on Emerging Drugs - March 8, 2019 Category: Drugs & Pharmacology Tags: Expert Opin Emerg Drugs Source Type: research

Emerging drugs for EGFR-mutated non-small cell lung cancer.
Authors: Sukrithan V, Deng L, Barbaro A, Cheng H Abstract INTRODUCTION: Epidermal growth factor receptor (EGFR) tyrosine kinase inhibitors (TKIs) represent the standard of care for patients with metastatic non-small-cell lung cancer (NSCLC) harboring sensitizing EGFR mutations. However, these agents are associated with inevitable treatment resistance. Newer generations of TKIs are under development that may prevent or overcome resistance and enhance intracranial activity. Areas covered: In this review, we will discuss newer generations of EGFR TKIs for EGFR-mutated NSCLC. We will also address resistance mutations a...
Source: Expert Opinion on Emerging Drugs - December 22, 2018 Category: Drugs & Pharmacology Tags: Expert Opin Emerg Drugs Source Type: research

An update: emerging drugs for tinnitus.
Authors: Cederroth CR, Dyhrfjeld-Johnsen J, Langguth B Abstract INTRODUCTION: During the last decade, a number of candidate drugs for the treatment of tinnitus have emerged with the hope of alleviating the burden of millions of sufferers with a persisting ringing in their ears. Knowledge of the pathophysiologic mechanisms has progressed remarkably in the recent years, which has led to the identification of potential new drug targets for the treatment of tinnitus. However, pharmacologic interventions are still limited. Areas covered: In this editorial results from recent Phase 3 and Phase 2a trials investigating the...
Source: Expert Opinion on Emerging Drugs - December 15, 2018 Category: Drugs & Pharmacology Tags: Expert Opin Emerg Drugs Source Type: research

Novel therapeutic approaches in primary hyperoxaluria.
Authors: Weigert A, Martin-Higueras C, Hoppe B Abstract INTRODUCTION: Currently, three types of primary hyperoxaluria (PH I-III) are known, all based on different gene-mutations affecting the glyoxylate metabolism in the liver. Disease hallmark is an increased endogenous oxalate production and thus massively elevated urinary excretion of oxalate and other type-specific metabolites. Hyperoxaluria induces the formation of calcium-oxalate kidney stones and/or nephrocalcinosis. In addition to that, a chronic inflammasome activation by hyperoxaluria per se, often leads to an early deterioration of kidney function, regul...
Source: Expert Opinion on Emerging Drugs - December 13, 2018 Category: Drugs & Pharmacology Tags: Expert Opin Emerg Drugs Source Type: research

An update on emerging drugs for the treatment of erectile dysfunction.
Authors: Milenkovic U, Campbell J, Roussel E, Albersen M Abstract INTRODUCTION: Erectile dysfunction is an extremely frequent and extensively studied condition, currently affecting the lives of tens of millions of men around the globe. The extensive knowledge of its pathophysiology has led to the development of phosphodiesterase 5-inhibitors, which can facilitate sexual intercourse in a large number of patients. However, an ever-increasing number of patients is unresponsive to these drugs due to underlying comorbidities or previous surgery. Different molecular pathways need to be addressed to provide treatment for ...
Source: Expert Opinion on Emerging Drugs - December 7, 2018 Category: Drugs & Pharmacology Tags: Expert Opin Emerg Drugs Source Type: research

Emerging drugs for the treatment of Dravet syndrome.
Authors: Brigo F, Striano P, Balagura G, Belcastro V Abstract INTRODUCTION: Dravet syndrome (DS) is an early-onset genetic developmental epileptic encephalopathy characterized by multiple seizure types which are refractory to antiseizure medication. There is an unmet need for effective and tolerable drugs to control different seizure types in DS types, with the aim of improving quality of life and preventing neurological impairment. Areas covered: Narrative review of efficacy and tolerability of fenfluramine, cannabidiol (CBD), verapamil and modulators of serotonin signaling pathways (lorcaserin or trazodone) in th...
Source: Expert Opinion on Emerging Drugs - November 30, 2018 Category: Drugs & Pharmacology Tags: Expert Opin Emerg Drugs Source Type: research

Emerging drugs for migraine treatment: an update.
Authors: Lambru G, Andreou AP, Guglielmetti M, Martelletti P Abstract INTRODUCTION: Migraine is a very frequent and disabling neurological disorder. The current treatment options are old, generally poorly tolerated and not migraine-specific, reflecting the low priority of migraine research and highlighting the vast unmet need in its management. Areas covered: Advancement in the understanding of migraine pathophysiological mechanisms and identification of novel potentially meaningful targets have resulted in a multitude of emerging acute and preventive treatments. Here we review the known putative migraine pathophys...
Source: Expert Opinion on Emerging Drugs - November 30, 2018 Category: Drugs & Pharmacology Tags: Expert Opin Emerg Drugs Source Type: research

Emerging cell cycle inhibitors for treating metastatic castration-resistant prostate cancer.
Authors: Batra A, Winquist E Abstract Introduction Disease progression despite androgen suppression defines lethal castration-resistant prostate cancer (CRPC). Most of these cancers remain androgen receptor (AR)-signalling dependent. Therapy for metastatic CRPC includes abiraterone acetate, enzalutamide, docetaxel, cabazitaxel, sipuleucel-T, and radium-223. However, survival remains modest for men with progressive disease despite AR-targeted therapy and docetaxel, and therefore novel treatments are needed. Areas covered Recent evidence of genomic heterogeneity and sensitivity to PARP inhibitors supports investigati...
Source: Expert Opinion on Emerging Drugs - November 15, 2018 Category: Drugs & Pharmacology Tags: Expert Opin Emerg Drugs Source Type: research

Disease-modifying Drugs in Osteoarthritis: Current Understanding and Future Therapeutics.
Authors: Oo WM, Yu SC, Daniel MS, Hunter DJ Abstract INTRODUCTION: Osteoarthritis (OA) is a leading cause of pain and disability among adults with a current prevalence of around 15% and a predicted prevalence of 35% in 2030 for symptomatic OA. It is increasingly recognized as a heterogeneous multi-faceted joint disease with multi-tissue involvement of varying severity. Current therapeutic regimens for OA are only partially effective and often have significant associated toxicities. There are no disease-modifying drugs approved by the regulatory bodies. Areas covered: We reviewed the opportunities within key OA path...
Source: Expert Opinion on Emerging Drugs - November 13, 2018 Category: Drugs & Pharmacology Tags: Expert Opin Emerg Drugs Source Type: research

An update: emerging drugs to treat squamous cell carcinomas of the head and neck.
Authors: Lee YS, Johnson DE, Grandis JR Abstract INTRODUCTION: Subsequent to the 2006 FDA approval of cetuximab, a variety of molecular targeting agents have been evaluated in head and neck squamous cell carcinoma (HNSCC). The treatment outcomes of recurrent and/or metastatic (R/M) HNSCC, in particular, remain dismal. The 2016 FDA approval of PD-1 immune checkpoint inhibitors has expanded the treatment options for R/M HNSCC, and highlights the potential for immune-based therapies. Areas covered: We will review the clinical application of EGFR-targeted agents, alone and in combination with other drugs. Molecular tar...
Source: Expert Opinion on Emerging Drugs - November 2, 2018 Category: Drugs & Pharmacology Tags: Expert Opin Emerg Drugs Source Type: research

Emerging PARP inhibitors for treating breast cancer.
Authors: Robert M, Patsouris A, Frenel JS, Gourmelon C, Augereau P, Campone M Abstract INTRODUCTION: Some breast cancers harbor defects in DNA repair pathways, including BRCA1 and BRCA2 mutations, leading to a genomic instability. Compromised DNA-damage repair response is found in 11 to 42% of triple negative breast cancers, with a frequency varying according to family history and ethnicity. The oral PARP inhibitors are a promising strategy in breast cancer exploiting Homologous Deficient Recombination deficiency (HRD) by a synthetic lethal approach. Several PARP inhibitors have currently reached early phase trials...
Source: Expert Opinion on Emerging Drugs - September 27, 2018 Category: Drugs & Pharmacology Tags: Expert Opin Emerg Drugs Source Type: research

Evolution in the treatment landscape of non-small cell lung cancer with ALK gene alterations: from the first- to third-generation of ALK inhibitors.
Authors: Spagnuolo A, Maione P, Gridelli C PMID: 30251885 [PubMed - as supplied by publisher] (Source: Expert Opinion on Emerging Drugs)
Source: Expert Opinion on Emerging Drugs - September 27, 2018 Category: Drugs & Pharmacology Tags: Expert Opin Emerg Drugs Source Type: research

Expert opinion on existing and developing drugs to treat female sexual dysfunction.
Authors: Miller MK, Smith JR, Norman JJ, Clayton AH Abstract INTRODUCTION: Female sexual dysfunction (FSD) is a highly prevalent, yet commonly underdiagnosed and undertreated condition. This paper reviews the diagnostic terminology for FSD, and basic sexual physiology in women. The Food and Drug Administration (FDA) approved drugs for FSD are discussed, followed by investigational drugs for FSD currently in phase 2 or 3 clinical trials, reasons for failure of drug development, and potential future drug targets. Areas Covered: A literature review was conducted for available treatments for FSD: flibanserin, estrogen,...
Source: Expert Opinion on Emerging Drugs - September 27, 2018 Category: Drugs & Pharmacology Tags: Expert Opin Emerg Drugs Source Type: research

Emerging drugs for focal epilepsy.
EMERGING DRUGS FOR FOCAL EPILEPSY. Expert Opin Emerg Drugs. 2018 Sep 25;: Authors: Mula M Abstract INTRODUCTION: Epilepsy is one of the most serious neurological conditions, affecting almost 50 million people around the world. Despite more than 20 antiepileptic drugs (AEDs) available, seizures are still uncontrolled in one third of patients. Areas covered: The present paper reviews current compounds in preclinical and clinical development for the treatment of focal epilepsies and new potential molecular targets recently identified. Expert Opinion: 1OP-2198, Cannabidavirin, Everolimus, FV-082, Ganaxolon...
Source: Expert Opinion on Emerging Drugs - September 27, 2018 Category: Drugs & Pharmacology Tags: Expert Opin Emerg Drugs Source Type: research

Expert opinion on emerging urate-lowering therapies.
Authors: Stamp LK, Merriman TR, Singh JA Abstract INTRODUCTION: There has been a resurgence in gout therapeutics in the last decade, not only for the management of gout flares, but also for the treatment of hyperuricemia. This editorial summarizes new, emerging therapies for people with gout. Areas Covered: We review several new therapies for gout, including those that are focused on lowering serum urate (levotofisopam, ulodesine, verinurad, merbarone, KUX-1151, UR-1102, FYU-981, SEL-212), or treating gout flares (canakinumab, bucillamine) or both (arhalofenate, diacerein). Expert Opinion: Among therapies with both...
Source: Expert Opinion on Emerging Drugs - September 26, 2018 Category: Drugs & Pharmacology Tags: Expert Opin Emerg Drugs Source Type: research