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Expert Opinion on Emerging Drugs

Vineeth Sukrithan, Lei Deng, Alexander Barbaro, Haiying Cheng
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 and escape pathways associated with these agents such as secondary mutations, downstream signaling, bypass pathways, phenotypic transformation, anti-apoptotic signaling, immune evasion, and angiogenesis...
December 20, 2018: Expert Opinion on Emerging Drugs
Alexander Weigert, Christina Martin-Higueras, Bernd Hoppe
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, regularly resulting in end-stage renal disease (ESRD) at least in patients with type I PH...
December 12, 2018: Expert Opinion on Emerging Drugs
Christopher R Cederroth, Jonas Dyhrfjeld-Johnsen, Berthold Langguth
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 NMDA receptor antagonist AM-101 from Auris Medical, the AMPA receptor antagonist BGG492 from Novartis and the Kv3 modulator AUT00063 from Autifony Therapeutics will be discussed...
December 7, 2018: Expert Opinion on Emerging Drugs
U Milenkovic, J Campbell, E Roussel, M Albersen
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 a larger patient population...
December 3, 2018: Expert Opinion on Emerging Drugs
Giorgio Lambru, Anna P Andreou, Martina Guglielmetti, Paolo Martelletti
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 pathophysiological mechanisms in order to understand the rationale of the most promising novel treatments targeting the Calcitonin-Gene-Related Peptide (CGRP) receptor and ligand and the 5 hydroxytryptamine (5-HT)1F receptor...
November 28, 2018: Expert Opinion on Emerging Drugs
Francesco Brigo, Pasquale Striano, Ganna Balagura, Vincenzo Belcastro
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 the treatment of DS...
November 27, 2018: Expert Opinion on Emerging Drugs
Anupam Batra, Eric Winquist
Disease progression despite androgen suppression defines lethal castration-resistant prostate cancer (CRPC). Most of these cancers remain androgen receptor (AR)-signaling 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 investigation of targeted agents in CRPC...
December 2018: Expert Opinion on Emerging Drugs
Win Min Oo, Shirley Pei-Chun Yu, Matthew Sean Daniel, David John Hunter
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 pathogenetic mechanism: cartilage catabolism/anabolism, pathological remodeling of subchondral bone and synovial inflammation to identify targeted disease-modifying osteoarthritis drugs, based on compounds currently in Phase II and III stages of clinical development in which x-ray and/or MRI was used as the structural outcome with/without symptomatic outcomes according to regulatory requirements...
December 2018: Expert Opinion on Emerging Drugs
Yoon Se Lee, Daniel E Johnson, Jennifer R Grandis
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...
October 30, 2018: Expert Opinion on Emerging Drugs
Marco Mula
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, Ganaxolone, Minocycline, NAX 810-2, Padsevonil and Selurampanel seem to be particularly promising in focal epilepsy...
September 2018: Expert Opinion on Emerging Drugs
Melanie K Miller, Joshua R Smith, Jacqueline J Norman, Anita H Clayton
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, ospemifene and prasterone...
September 2018: Expert Opinion on Emerging Drugs
Alessia Spagnuolo, Paolo Maione, Cesare Gridelli
The medical treatment of non-small cell lung cancer (NSCLC) has radically changed over the last 10 years thanks to new molecular-targeted drugs able to act on biological mechanisms involved in tumor development. One such mechanism is the aberrant anaplastic lymphoma kinase (ALK) activation: patients with ALK-driven NSCLC benefit from treatments that selectively inhibit its pathogenetic mechanism. Areas covered: The first-generation ALK inhibitor is crizotinib, initially used in Europe as second-line treatment for ALK-positive metastatic NSCLC patients, then approved as the standard first-line (already approved in the USA as front-line therapy)...
September 2018: Expert Opinion on Emerging Drugs
Marie Robert, Anne Patsouris, Jean-Sébastien Frenel, Carole Gourmelon, Paule Augereau, Mario Campone
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 with studies on going in the adjuvant, neoadjuvant and metastatic setting...
September 2018: Expert Opinion on Emerging Drugs
Lisa K Stamp, Tony R Merriman, Jasvinder A Singh
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)...
September 2018: Expert Opinion on Emerging Drugs
Marco Mantero, Paola Rogliani, Mario Cazzola, Francesco Blasi, Marta Di Pasquale
Management of LRTI is becoming more frequently challenging since the emergence of multidrug resistance bacteria and the increase of severe viral infection, reducing the number of available effective drugs. The clinical evaluation of new therapeutic associations is mandatory to cope with the increases in resistance, in association with better infection control and antimicrobial policies. Areas covered: We searched Pubmed in English language of phase I, II, III clinical trials and approved treatments for LRTI, between 2006 and 2016...
September 2018: Expert Opinion on Emerging Drugs
Robert D Pesek, Sandeep K Gupta
Eosinophilic esophagitis (EoE) is rare but incidence and prevalence is increasing. EoE is characterized by eosinophilic inflammation of the esophagus causing gastrointestinal symptoms such as abdominal pain, vomiting, reflux, dysphagia, and food impactions. If untreated, remodeling and fibrosis of the esophagus can occur and stricture formation may result. Current treatment options are limited to food-restriction diets or medications including proton pump inhibitors (PPIs) or swallowed corticosteroids. Significant progress has been made in understanding the underlying mechanisms of EoE allowing for development of drugs that target specific points in EoE pathways...
June 2018: Expert Opinion on Emerging Drugs
Thomas D Rodgers, Patrick M Reagan
The B-cell receptor (BCR) pathway is a crucial aspect of mature lymphocytes and is maintained in B-cell neoplasms. Many small module inhibitors targeting kinases within the BCR pathway are approved, with others in development, offering alternative treatment options to standard chemoimmunotherapy. Areas covered: This review covers both approved inhibitors and investigational inhibitors of spleen tyrosine kinase (SYK), Bruton's tyrosine kinase (BTK), and phosphoinositide-3-kinase (PI3K) in the treatment of B-cell lymphomas...
June 2018: Expert Opinion on Emerging Drugs
Mohammad A Rai, Sam Pannek, Carl J Fichtenbaum
There are 36.7 million people living with HIV with 20.9 million having access to antiretroviral therapy (ART). Nucleoside/nucleotide reverse transcriptase inhibitors (NRTIs) remain the 'backbone' of ART. However, the currently available nine NRTIs and five non-nucleoside reverse transcriptase inhibitors (NNRTIs) have significant side effects and resistance profiles. Areas covered: We summarize the mechanisms of resistance and other limitations of the existing NRTIs/NNRTIs. GS-9131, MK-8591, Elsulfavirine and Doravirine are four new agents that are furthest along in development...
June 2018: Expert Opinion on Emerging Drugs
Jasper Crijnen, Theo M De Reijke
Bladder cancer (BC) is a severe health burden: and has high recurrence and progression rates. Standard treatment starts with TURB followed by intravesical chemotherapy with Mitomycin C or immunotherapy with BCG. However, successful management still remains a challenge, because approximately 30% of patients have recurrence or progression within 5 years, and treatment has considerable side effects. Anticipating on the upcoming BCG shortage emphasizes, moreover, the necessity to develop and study novel treatments...
June 2018: Expert Opinion on Emerging Drugs
Shambhu Aryal, Steven D Nathan
Idiopathic pulmonary fibrosis is a chronic, progressive fibrotic lung disease of unknown etiology associated with a high morbidity and mortality. The hallmark of the disease is impaired healing after alveolar epithelial injury in the setting of a genetic predisposition. Development of two new drugs has changed the landscape of the treatment of IPF but more work is needed to improve outcomes and improve survival. Areas covered: The development of two antifibrotic agents, nintedanib and pirfenidone has been an exciting landmark in the treatment of IPF...
June 2018: Expert Opinion on Emerging Drugs
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