journal
https://read.qxmd.com/read/38516735/evaluating-emerging-drugs-in-phase-ii-iii-for-the-treatment-of-amyotrophic-lateral-sclerosis
#1
REVIEW
Xiaoyan Li, Richard Bedlack
INTRODUCTION: Amyotrophic Lateral Sclerosis is a rapidly progressive motor neuron disorder causing severe disability and premature death. Owing to the advances in uncovering ALS pathophysiology, efficient clinical trial design and research advocacy program, several disease-modifying drugs have been approved for treating ALS. Despite this progress, ALS remains a rapidly disabling and life shortening condition. There is a critical need for more effective therapies. AREAS COVERED: Here, we reviewed the emerging ALS therapeutics undergoing phase II & III clinical trials...
March 22, 2024: Expert Opinion on Emerging Drugs
https://read.qxmd.com/read/38469871/emerging-drugs-for-the-treatment-of-hepatic-fibrosis-on-nonalcoholic-steatohepatitis
#2
REVIEW
Michihiro Iwaki, Masato Yoneda, Naohiro Wada, Otani Tomohiro, Takashi Kobayashi, Asako Nogami, Satoru Saito, Nakajima Atsushi
INTRODUCTION: Approved drug therapies for nonalcoholic steatohepatitis (NASH) are lacking, for which various agents are currently being tested in clinical trials. Effective drugs for liver fibrosis, the factor most associated with prognosis in NASH, are important. AREAS COVERED: This study reviewed the treatment of NASH with a focus on the effects of existing drugs and new drugs on liver fibrosis. EXPERT OPINION: Considering the complex pathophysiology of fibrosis in NASH, drug therapy may target multiple pathways...
March 12, 2024: Expert Opinion on Emerging Drugs
https://read.qxmd.com/read/38410863/an-extract-of-phase-ii-and-iii-trials-on-recent-developments-in-managing-neuropathic-pain-syndromes-diabetic-peripheral-neuropathy-trigeminal-neuralgia-and-postherpetic-neuralgia
#3
REVIEW
William C Upshaw, Lenise G Soileau, Nicholas R Storey, Kassady A Perkinson, Patrick M Luther, Noah J Spillers, Christopher L Robinson, Benjamin C Miller, Shahab Ahmadzadeh, Omar Viswanath, Sahar Shekoohi, Alan D Kaye
INTRODUCTION: Neuropathic pain (NP) conditions involve lesions to the somatosensory nervous system leading to chronic and debilitating pain. Many patients suffering from NP utilize pharmacological treatments with various drugs that seek to reduce pathologic neuronal states. However, many of these drugs show poor efficacy as well as cause significant adverse effects. Because of this, there is a major need for the development of safer and more efficacious drugs to treat NP. AREAS COVERED: In this review, we analyzed current treatments being developed for a variety of NP conditions...
February 29, 2024: Expert Opinion on Emerging Drugs
https://read.qxmd.com/read/38337150/targeting-the-pacap-38-pathway-is-an-emerging-therapeutic-strategy-for-migraine-prevention
#4
REVIEW
Lanfranco Pellesi, Messoud Ashina, Paolo Martelletti
INTRODUCTION: The pituitary adenylate cyclase-activating polypeptide-38 (PACAP-38) has emerged as a key mediator of migraine pathogenesis. PACAP-38 and its receptors are predominantly distributed in arteries, sensory and parasympathetic neurons of the trigeminovascular system. Phase 2 trials have tested human monoclonal antibodies designed to bind and inhibit PACAP-38 and the pituitary adenylate cyclase-activating polypeptide type I (PAC1 ) receptor for migraine prevention. AREAS COVERED: This review focuses on the significance of the PACAP-38 pathway as a target in migraine prevention...
February 9, 2024: Expert Opinion on Emerging Drugs
https://read.qxmd.com/read/38296815/emerging-drugs-for-the-treatment-of-irritability-associated-with-autism-spectrum-disorder
#5
REVIEW
Ahmad Shamabadi, Hanie Karimi, Razman Arabzadeh Bahri, Mohsen Motavaselian, Shahin Akhondzadeh
INTRODUCTION: Autism spectrum disorder (ASD) is an early-onset disorder with a prevalence of 1% among children and reported disability-adjusted life years of 4.31 million. Irritability is a challenging behavior associated with ASD, for which medication development has lagged. More specifically, pharmacotherapy effectiveness may be limited against high adverse effects (considering side effect profiles and patient medication sensitivity); thus, the possible benefits of pharmacological interventions must be balanced against potential adverse events in each patient...
January 31, 2024: Expert Opinion on Emerging Drugs
https://read.qxmd.com/read/38293894/how-will-tovorafenib-change-our-treatment-of-pediatric-low-grade-glioma
#6
EDITORIAL
Inci Yaman, Eric Bouffet
No abstract text is available yet for this article.
January 31, 2024: Expert Opinion on Emerging Drugs
https://read.qxmd.com/read/38226593/cannabinoid-treatment-for-the-symptoms-of-autism-spectrum-disorder
#7
REVIEW
Adi Aran, Dalit Cayam Rand
INTRODUCTION: Autism spectrum disorder (ASD) is a neurodevelopmental disorder affecting approximately 3% of school-age children. The core symptoms are deficits in social communication and restricted and repetitive patterns of behavior. Associated problems in cognition, language, behavior, sleep and mood are prevalent. Currently, no established pharmacological treatment exists for core ASD symptoms. Risperidone and aripiprazole are used to manage associated irritability, but their effectiveness is limited and adverse events are common...
January 16, 2024: Expert Opinion on Emerging Drugs
https://read.qxmd.com/read/38193477/how-promising-are-the-latest-monoclonal-antibodies-targeting-amyloid-%C3%AE-for-the-treatment-of-early-alzheimer-s-disease
#8
REVIEW
Jordan Beveridge, Eileen Kaniecki, Aniketh Naidu, Bret David Silverglate, George Grossberg
INTRODUCTION: Monoclonal antibodies targeting amyloid-β are the first disease-modifying treatments for Alzheimer disease to have received FDA-approval. There are three different drugs approved or pending FDA-approval: aducanumab, lecanemab, and donanemab. These three drugs are each in different stages of regulatory approval by the FDA. AREAS COVERED: We discuss the development of these drugs, the data regarding their clinical efficacy, their dosing regimens, and side effects...
January 9, 2024: Expert Opinion on Emerging Drugs
https://read.qxmd.com/read/38193191/novel-and-emerging-drugs-for-the-treatment-of-crohn-s-disease-a-review-of-phase-ii-and-iii-trials
#9
REVIEW
Panu Wetwittayakhlang, Talat Bessissow, Peter L Lakatos
INTRODUCTION: Crohn's disease (CD) is a chronic inflammatory bowel disease characterized by unpredictable flare-ups and periods of remission. While several therapeutic options, such as anti-tumor necrosis factor (TNF), anti-integrin, and interleukin (IL) 12/23 inhibitors, as well as IL-23 and Janus kinase (JAK) inhibitors, have been approved for CD treatment, a substantial number of patients fail to respond adequately or experience a loss of response over time. In recent years, the scientific community has been actively investigating novel agents to address these challenges and improve the management of CD...
January 9, 2024: Expert Opinion on Emerging Drugs
https://read.qxmd.com/read/38180809/review-of-phase-2-3-trials-in-polymyalgia-rheumatica-and-giant-cell-arteritis
#10
REVIEW
Pratheeshaa Nageswaran, Saad Ahmed, Hasan Tahir
INTRODUCTION: GCA (giant cell arteritis) and PMR (polymyalgia rheumatica) are two overlapping inflammatory rheumatic conditions that are seen exclusively in older adults, sharing some common features. GCA is a clinical syndrome characterized by inflammation of the medium and large arteries, with both cranial and extracranial symptoms. PMR is a clinical syndrome characterized by stiffness in the neck, shoulder, and pelvic girdle muscles. Both are associated with constitutional symptoms...
January 5, 2024: Expert Opinion on Emerging Drugs
https://read.qxmd.com/read/38112023/are-clinical-trials-into-emerging-drugs-for-the-treatment-of-alpha-1-antitrypsin-deficiency-providing-promising-results
#11
EDITORIAL
Joshua De Soyza, Anita Pye, Alice M Turner
No abstract text is available yet for this article.
December 19, 2023: Expert Opinion on Emerging Drugs
https://read.qxmd.com/read/38129984/recent-advances-in-uveitis-therapy-focus-on-selected-phase-2-and-3-clinical-trials
#12
REVIEW
Zheng Xian Thng, Albert John Bromeo, S Saeed Mohammadi, Anadi Khatri, Anh N T Tran, Amir Akhavanrezayat, Ngoc T T Than, Khiem S Nguyen, Woong-Sun Yoo, Azadeh Mobasserian, Christopher Chi Mong Or, Quan Dong Nguyen
INTRODUCTION: Uveitis is a heterogeneous group of ocular conditions characterized by inflammation of the uveal tract. It is a leading cause of blindness in developed countries and exerts significant psychological, social, and economic impact on both patients and the larger society. While there are numerous pharmacotherapy options, posterior segment noninfectious uveitis remains a significant challenge to treat due to its severity, chronicity, and high recurrence rates. AREAS COVERED: The index review highlights the unmet needs of uveitis pharmacotherapy and its research and the shortcomings of existing ocular and systemic therapeutic options for noninfectious uveitis...
December 2023: Expert Opinion on Emerging Drugs
https://read.qxmd.com/read/38100555/cancer-drugs-with-high-repositioning-potential-for-alzheimer-s-disease
#13
REVIEW
Jad Majeed, Marwan N Sabbagh, Min H Kang, J Josh Lawrence, Kevin Pruitt, Sarah Bacus, Ellie Reyna, Maddy Brown, Boris Decourt
INTRODUCTION: Despite the recent full FDA approval of lecanemab, there is currently no disease modifying therapy (DMT) that can efficiently slow down the progression of Alzheimer's disease (AD) in the general population. This statement emphasizes the need to identify novel DMTs in the shortest time possible to prevent a global epidemic of AD cases as the world population experiences an increase in lifespan. AREAS COVERED: Here, we review several classes of anti-cancer drugs that have been or are being investigated in Phase II/III clinical trials for AD, including immunomodulatory drugs, RXR agonists, sex hormone therapies, tyrosine kinase inhibitors, and monoclonal antibodies...
December 2023: Expert Opinion on Emerging Drugs
https://read.qxmd.com/read/38099328/a-review-of-phase-ii-and-iii-drugs-for-the-treatment-and-management-of-endometriosis
#14
REVIEW
Umberto Perrone, Giulio Evangelisti, Antonio Simone Laganà, Stefano Bogliolo, Marcello Ceccaroni, Alberto Izzotti, Claudio Gustavino, Simone Ferrero, Fabio Barra
INTRODUCTION: Endometriosis is an estrogen-dependent disease that gives rise to pelvic pain and infertility. Although estroprogestins and progestins currently stand as the first-line treatments for this condition, demonstrating efficacy in two-thirds of patients, a significant portion of individuals experience only partial relief or symptom recurrence following the cessation of these therapies. The coexistence of superficial, deep endometriosis, and ovarian endometriomas, as three distinct phenotypes with unique pathogenetic and molecular characteristics, may elucidate the current heterogeneous biological response to available therapy...
December 2023: Expert Opinion on Emerging Drugs
https://read.qxmd.com/read/37996401/new-drugs-targeting-calcitonin-gene-related-peptide-for-the-management-of-migraines
#15
REVIEW
Claudio Tana, Francesco Cipollone, Maria Adele Giamberardino, Paolo Martelletti
INTRODUCTION: Significant advances in migraine research have contributed to the development of new drugs for the treatment of migraine. Monoclonal antibodies (mAbs) against Calcitonin Gene-Related Peptide (CGRP) or its receptor and CGRP receptor antagonists (gepants) have been associated with a good safety profile and resulted in an overall efficacy in reducing the number of monthly migraine days both in episodic and chronic forms of migraine. AREAS COVERED: The results from main investigation studies (phase 2 or 3) of CGRP-targeting drugs (both anti-CGRP mAbs and gepants) are reported in this expert-opinion review...
December 2023: Expert Opinion on Emerging Drugs
https://read.qxmd.com/read/37909462/apomorphine-for-parkinson-s-disease-pharmacologic-and-clinical-considerations
#16
REVIEW
Maxwell J Wagner, Charles P Daniel, Connor J Plaisance, Grant E Borne, Shahab Ahmadzadeh, Sahar Shekoohi, Alan D Kaye
INTRODUCTION: In Parkinson's disease, dopamine depletion in the basal ganglia leads to symptoms including bradykinesia, gait abnormalities, and cognitive impairment. Even with treatment, the disease course leads to decreases in the amount of dopamine produced and released into the synapse. As dopamine production falls and the treatment course is insufficient to match the metabolic supply and demand, acute 'off' periods develop that cause reemergence of symptoms. Apomorphine is used to reverse these 'off' periods and restore function in patients with Parkinson's...
December 2023: Expert Opinion on Emerging Drugs
https://read.qxmd.com/read/37256917/systemic-mastocytosis-2023-update-on-diagnosis-and-management-in-adults
#17
REVIEW
Alessandro Costa, Emilia Scalzulli, Ida Carmosino, Marcello Capriata, Claudia Ielo, Chiara Masucci, Mauro Passucci, Maurizio Martelli, Massimo Breccia
INTRODUCTION: Systemic mastocytosis (SM) is a complex and heterogeneous disease, characterized by the clonal accumulation of mast cells in one or more organs. In 2022 both the World Health Organization (WHO) and the International Consensus Classification (ICC) modified the diagnostic and classification criteria of SM. Moreover, the identification of new clinical and molecular variables has improved prognostic tools and led to increasingly individualized therapeutic strategies. AREAS COVERED: The aim of this review is to present the updates introduced by the International Consensus Classification in diagnostic criteria of SM...
December 2023: Expert Opinion on Emerging Drugs
https://read.qxmd.com/read/37185047/emerging-drugs-for-the-preventive-treatment-of-migraine-a-review-of-cgrp-monoclonal-antibodies-and-gepants-trials
#18
REVIEW
Marcello Silvestro, Ilaria Orologio, Mattia Siciliano, Francesca Trojsi, Alessandro Tessitore, Gioacchino Tedeschi, Antonio Russo
INTRODUCTION: Migraine is a leading cause of years lived with disability and preventive strategies represent a mainstay to reduce health-related disability and improve quality of life of migraine patients. Until a few years ago, migraine prevention was based on drugs developed for other clinical indications and relocated in the migraine therapeutic armamentarium, characterized by unfavorable tolerability profiles. The advent of monoclonal antibodies against Calcitonin Gene-Related Peptide (CGRP) and gepants, CGRP receptor antagonists, has been a turning point in migraine prevention owing to advantageous efficacy, safety and tolerability profiles...
December 2023: Expert Opinion on Emerging Drugs
https://read.qxmd.com/read/37144331/combined-chemo-immunotherapy-in-advanced-non-small-cell-lung-cancer-feasible-in-the-elderly
#19
REVIEW
Francesca Casaluce, Cesare Gridelli
INTRODUCTION: Lung cancer is the leading cause of cancer-related death worldwide, with non-small cell lung cancer (NSCLC) as the most common subtype. In the past decades, immunotherapy deeply changed paradigms for care of newly diagnosed advanced NSCLC patients without oncogenic driver mutations. An immunotherapy-based regimen alone or in combination to chemotherapy was crowned as the preferred option to choice, according to worldwide guideline. AREAS COVERED: Newly diagnoses of advanced NCSLC occurred predominantly in elderly patients, representing over half of patients treated in daily practice...
December 2023: Expert Opinion on Emerging Drugs
https://read.qxmd.com/read/37127914/emerging-treatment-for-sj%C3%A3-gren-s-disease-a-review-of-recent-phase-ii-and-iii-trials
#20
REVIEW
Robert I Fox, Carla M Fox, Sara S McCoy
INTRODUCTION: Sjögren's Disease, SjD, is a systemic autoimmune disorder characterized by reduced function of the salivary and lacrimal glands. Patients suffer from dryness, fatigue, and pain and may present with or without extra-glandular organ involvement. Symptoms limit SjD patients' quality of life and are the most difficult to improve with therapy. SjD patients are heterogeneous and clustering them into biologically similar subgroups might improve the efficacy of therapies. The need for therapies that address both the symptoms and extra glandular organ involvement of SjD presents an unmet opportunity that has recently attracted a growing interest in the pharmaceutical industry...
December 2023: Expert Opinion on Emerging Drugs
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