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Expert Opinion on Investigational Drugs

Marco Greco, Gaia Varriale, Giangennaro Coppola, Francesca Operto, Alberto Verrotti, Maria Laura Iezzi
Epilepsy is a neurological disorder that significantly impacts the quality of life of the affected person and affects approximately 70 million people globally. Despite advances in research, nearly a third of patients have refractory or pharmacoresistant epilepsy. Even though numerous antiepileptic drugs (AEDs) have been approved over the past decade, there are no agents that halt the development of epilepsy. Thus, new and improved AEDs to prevent refractory seizures and modify the disease are necessary. Areas covered: We highlight recent advances and emerging trends in new and innovative drugs for epilepsy and seizure disorders...
November 8, 2018: Expert Opinion on Investigational Drugs
Alexandre A Jacome, Cathy Eng
Squamous cell carcinoma of the anal canal (SCCA) is a rare malignancy but incidence rates have been increasing in the last decade. Studies have demonstrated that up to 47% of patients with locally advanced disease have high-risk features for treatment failure. The potential high rates of recurrence after standard chemoradiotherapy for locally advanced disease and the lack of established care for metastatic disease, has created an urgent need for the evaluation of new drugs that will ultimately improve the efficacy of treatment...
November 1, 2018: Expert Opinion on Investigational Drugs
Davide Capodanno, Roxana Mehran, C Michael Gibson, Dominick J Angiolillo
The risk of major adverse cardiac events (MACE) remains elevated soon after a coronary event. High-density lipoprotein (HDL) cholesterol has been proposed as a target to reduce cardiovascular endpoints, but there is growing recognition that increasing the function of HDL may be more important than merely increasing its concentration. CSL112 is a reconstituted, infusible human plasma-derived apolipoprotein A-I (apoA-I) that increases cholesterol efflux capacity - an ex vivo measure of the ability of HDL to accept cholesterol from macrophages...
October 31, 2018: Expert Opinion on Investigational Drugs
Rossella Siligato, Valeria Cernaro, Chiara Nardi, Francesca De Gregorio, Guido Gembillo, Giuseppe Costantino, Giovanni Conti, Michele Buemi, Domenico Santoro
Minimal change disease (MCD) and Focal and segmental glomerulosclerosis (FSGS) are two of the major causes of nephrotic syndrome (NS) in children and adults. According to KDIGO (Kidney Disease: Improving Global Outcomes) guidelines, the treatment of adult primary MCD and FSGS should be based on immunosuppressants and antiproteinuric drugs. Recently, Rituximab, a humanized monoclonal antibody (mAb) has emerged as a potential treatment for steroid or calcineurin inhibitor-dependent patients; it has however demonstrated lower efficacy in those with nephrotic syndrome that is resistant to the above indicated drugs...
October 25, 2018: Expert Opinion on Investigational Drugs
Alexis D Leal, Anuradha Krishnamurthy, Lia Head, Wells A Messersmith
Antibody drug conjugates (ADCs) represent a developing class of anticancer therapeutics which are designed to selectively deliver a cytotoxic payload to tumors, while limiting systemic toxicity to healthy tissues. There are several ADCs which are currently in various stages of clinical development for the treatment of gastrointestinal malignancies. Areas covered: We discuss the biologic rationale and review the clinical experience with ADCs in the treatment of gastrointestinal malignancies, summarizing the pre-clinical and phase I/II clinical trial data that have been completed or are ongoing...
October 25, 2018: Expert Opinion on Investigational Drugs
José-Manuel Gómez-Sáez
Thyrotoxicosis with hyperthyroidism is treated with these classical approaches (i) antithyroid drugs to blockade thyroid hormone release and normalize thyroid hormone production and (ii) destruction of the thyroid using radioiodine or surgical removal of the thyroid. The optimal medical therapy, especially for Graves´ disease, remains a subject of debate and there has been little progress in Graves' disease therapeutics over the last decade. Areas covered: Novel treatments of thyrotoxicosis with hyperthyroidism...
October 25, 2018: Expert Opinion on Investigational Drugs
Zhaohua Zhu, Jia Li, Guangfeng Ruan, Guoliang Wang, Cibo Huang, Changhai Ding
Osteoarthritis (OA) is the leading cause of pain, loss of function, and disability among elderly, with the knee the most affected joint. It is a heterogeneous condition characterized by complex and multifactorial etiologies which contribute to the broad variation in symptoms presentation and treatment responses that OA patients present. This poses a challenge for the development of effective treatment on OA. Areas covered: This review will discuss recent development of agents for the treatment of OA, updating our previous narrative review published in 2015...
October 20, 2018: Expert Opinion on Investigational Drugs
Juan Antonio Moreno, Carmen Gomez-Guerrero, Sebastian Mas, Ana Belen Sanz, Oscar Lorenzo, Marta Ruiz-Ortega, Lucas Opazo, Sergio Mezzano, Jesus Egido
Diabetic nephropathy (DN) is the leading cause of chronic kidney disease (CKD) and end-stage renal disease (ESRD). Beyond the new anti-diabetic drugs that possess markedly cardiovascular and renal protective effects, no novel direct therapies for DN have become available on the market in the last twenty years. Recently well-designed clinical trials for the treatment of DN, with attractive pathogenetic rationale, e.g. bardoxolone and atrasentan, were canceled or stopped because of safety concerns or lack of reaching the end points, respectively...
October 18, 2018: Expert Opinion on Investigational Drugs
Anisha Guda, Steven R Feldman, Lindsay Strowd
Psoriasis is a common immune disorder for which there are many FDA-approved therapies. Older oral drugs had limited efficacy and considerable side effects. Biologics have been a major advance but require self-administered injections, and many patients prefer oral options. Because many patients fear needles, oral drugs are a welcome option. This review provides an update on oral drugs in Phase II development for psoriasis. Areas covered: A literature review was performed to identify these drugs by using search terms such as 'psoriasis', 'agents', 'drugs', and 'phase 2 development'...
October 6, 2018: Expert Opinion on Investigational Drugs
Juan Manuel Sepúlveda, Pilar Sánchez-Gómez, María Ángeles Vaz Salgado, Ricardo Gargini, Carmen Balañá
Standard treatment of newly diagnosed glioblastoma (GB) is surgery with radiotherapy and temozolomide, but tumors will recur with a median overall survival of only 15 months. It seems imperative to explore new possibilities of treatment based on targetable alterations known to be present in GB. Among others, Epidermal Growth Factor Receptor or EGFR (HER1) mutations or amplifications are the most prevalent alterations in GB. In fact, around 40% of GB cases show amplification of EGFR gene, and half of these patients carry the EGFRvIII mutation, a deletion that generates a continuous activation of the tyrosine kinase domain of the receptor...
October 2018: Expert Opinion on Investigational Drugs
Artemis Matsou, Eleftherios Anastasopoulos
Prostaglandin F2α analogs were the first prostaglandin agonists introduced for glaucoma treatment. Thanks to their efficacy and favorable tolerability they set a high bar in competition, with a resultant paucity in new hypotensive drug development for many years. However, the scientific community has shown recently a new interest in exploring new options for glaucoma treatment, generating a remarkable incentive in the marketplace for new drugs. Areas covered: This article reviews agents targeting prostaglandin receptors that are currently being investigated for glaucoma treatment...
October 2018: Expert Opinion on Investigational Drugs
Robin Koller, Ghyslain Mombo-Ngoma, Martin P Grobusch
Ganaplacide (previously known as KAF156) is a novel antimalarial compound part of the imidazolopiperazine family. Areas covered: At the time of writing, a total of eight studies addressing its preclinical and clinical development have been published on this compound, which is currently in phase 2 of clinical development, alongside lumefantrine in a novel soluble formulation as combination partner. This review provides an overview and interpretation of the published pre-clinical and clinical data of this possible next-generation antimalarial drug...
October 2018: Expert Opinion on Investigational Drugs
Enrique Soto-Perez-De-Celis, Kah Poh Loh, Capucine Baldini, Nicolò Matteo Luca Battisti, Yanin Chavarri-Guerra, Nienke A De Glas, Tina Hsu, Arti Hurria
One-third of breast cancer (BC) cases worldwide occur in women aged 65 years and older, with 10 to 15% overexpressing the human epidermal growth factor receptor 2 (HER2). Although several HER2-targeted therapies have been developed, the lack of data regarding their use in older patients hampers evidence-based decision-making for this population. Areas Covered: We review current evidence on the efficacy and safety of HER2-targeted therapies in older adults with BC, focusing on approved therapies such as trastuzumab, lapatinib, pertuzumab, ado-trastuzumab-emtansine, and neratinib...
October 2018: Expert Opinion on Investigational Drugs
Christoph Stein
Opioids are the oldest and most potent drugs for the treatment of severe pain, but they are burdened by detrimental side effects such as respiratory depression, addiction, sedation, nausea, and constipation. Their clinical application is undisputed in acute (e.g. perioperative) and cancer pain, but their long-term use in chronic pain has met increasing scrutiny and has contributed to the current 'opioid crisis.' Areas covered: This article reviews pharmacological principles and research strategies aiming at novel opioids with reduced side effects...
October 2018: Expert Opinion on Investigational Drugs
Pedro Isaacsson Velho, Michael A Carducci
Despite the heterogeneity of prostate cancer (PCa), androgen stimulation is fundamental to its development, growth, and lethality. Therefore, the blockade of androgen receptor (AR) signaling is critical to controlling the disease, even after progression with castrate levels of androgens. Areas covered: We review the current understanding of new ways to block the AR, using novel antiandrogen inhibitors, which act on different parts of the AR signaling pathway in PCa. We also review new approaches, such as the use of poly(ADP-ribose) polymerase (PARP) inhibitors, targeting both the AR and the DNA repair pathway, potentially adding synergy and improving efficacy and the combination of AR inhibitors and immunotherapy...
October 2018: Expert Opinion on Investigational Drugs
Andrea Chiricozzi, Diego Peroni, Giampiero Girolomoni
Atopic dermatitis (AD) is the most common cutaneous inflammatory disease in adults and children. In the last few years, the pathogenesis of AD has been profoundly revised, and this has led to the identification of novel druggable targets and the development of new agents targeting specific molecular pathways. Despite the high prevalence of AD in the pediatric population, the clinical development of new treatments, either topical or systemic, has been focused on the adult population in recent years; only a limited number of these new agents have been tested in pediatric cohorts...
September 2018: Expert Opinion on Investigational Drugs
Yu-Long Lan, Zhen-Long Yu, Jia-Cheng Lou, Xiao-Chi Ma, Bo Zhang
Glioblastoma is a debilitating disease that is associated with poor prognosis and a very limited response to therapies; thus, molecularly targeted therapeutics and personalized therapy are urgently needed. The Na+ /K+ -ATPase sodium pump is a transmembrane protein complex that has recently been recognized as an important transducer and integrator of various signals. The sodium pump α1 subunit, which is highly expressed in most glioblastomas compared with that in normal brain tissues, is an emerging cancer target that merits further investigation...
September 2018: Expert Opinion on Investigational Drugs
Amit Mahipal, Anuhya Kommalapati, Sri Harsha Tella, Alexander Lim, Richard Kim
Surgical resection remains the mainstay of potentially curative treatment in the early stages of cholangiocarcinoma, whereas for the advanced stage, systemic chemotherapeutics and experimental targeted therapies are the primary treatment options. The molecular heterogeneity of the tumor is based on location, liver dysfunction, and relative rarity of the disease and confers challenges for clinical trial enrollment. The advancements in the understanding of molecular pathogenesis of cholangiocarcinoma have led to the development of targeted therapies that are currently being evaluated in the clinical trials...
September 2018: Expert Opinion on Investigational Drugs
Daniel Kerr, Lubomir Sokol
Blastic plasmacytoid dendritic cell neoplasm (BPDCN) is a rare and aggressive myeloid malignancy that contributes to <1% of all hematologic neoplasms. Before the introduction of various targeted agents, the therapeutic approach was based on regimens used for acute lymphoblastic or myeloid leukemia and non-Hodgkin's lymphoma (e.g. hyperCVAD (hyperfractionated cyclophosphamide, vincristine, doxorubicin and dexamethasone alternating with high dose methotrexate and cytarabine) and CHOP (cyclophosphamide, doxorubicin, vincristine, prednisone) -based regimens) followed by allogeneic stem cell transplantation for eligible patients...
September 2018: Expert Opinion on Investigational Drugs
Natalia Muñoz-Durango, Magdalena S Pizarro-Ortega, Emma Rey-Jurado, Fabián E Díaz, Susan M Bueno, Alexis M Kalergis
The human respiratory syncytial virus (hRSV) is the main cause of acute lower respiratory tract infection in susceptible population worldwide, such as young children and the elderly. Although hRSV is a major public health burden, there are no licensed vaccines and the only available therapy is palivizumab. During life, reinfections with hRSV are common, suggesting that the virus can impair the development of an efficient host immune response. This feature has hindered the development of efficient therapies...
September 2018: Expert Opinion on Investigational Drugs
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