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Current treatment options for treating OPA1-mutant dominant optic atrophy. 目前治疗opa1突变显性视神经萎缩的治疗方案。
IF 1.8 4区 医学
Drugs of today Pub Date : 2022-11-01 DOI: 10.1358/dot.2022.58.11.3448291
Lorenzo Ferro Desideri, Carlo Enrico Traverso, Michele Iester
{"title":"Current treatment options for treating <i>OPA1</i>-mutant dominant optic atrophy.","authors":"Lorenzo Ferro Desideri,&nbsp;Carlo Enrico Traverso,&nbsp;Michele Iester","doi":"10.1358/dot.2022.58.11.3448291","DOIUrl":"https://doi.org/10.1358/dot.2022.58.11.3448291","url":null,"abstract":"<p><p>Dominant optic atrophy (DOA) is caused by OPA1 gene mutation, and it represents one of the most frequently diagnosed forms of hereditary optic neuropathies. This neurodegenerative disorder typically occurs in the first decades of life, and it is often associated with severe visual impairment. For this reason, several treatment options have been examined for the management of DOA, including vitamin supplements, ubiquinone analogues (in particular idebenone) and, more recently, gene therapy. Among them, idebenone has shown the most promising clinical outcomes in recent real-life studies. Furthermore, gene therapy represents also a promising therapeutic approach; however, more evidence in clinical trials is needed. In this review, we will summarize and discuss all the possible treatment options for DOA, in order to identify the current optimal management in these patients, whose visual prognosis remains unfortunately poor and unsatisfactory in the everyday clinical practice.</p>","PeriodicalId":11397,"journal":{"name":"Drugs of today","volume":null,"pages":null},"PeriodicalIF":1.8,"publicationDate":"2022-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"40704844","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Mobocertinib in non-small cell lung cancer. 莫博西替尼治疗非小细胞肺癌。
IF 1.8 4区 医学
Drugs of today Pub Date : 2022-11-01 DOI: 10.1358/dot.2022.58.11.3408816
Shengwu Liu, Kristen E Lowder
{"title":"Mobocertinib in non-small cell lung cancer.","authors":"Shengwu Liu,&nbsp;Kristen E Lowder","doi":"10.1358/dot.2022.58.11.3408816","DOIUrl":"https://doi.org/10.1358/dot.2022.58.11.3408816","url":null,"abstract":"<p><p>Tyrosine kinase inhibitors (TKIs) have provided great benefit for patients with EGFR-mutant non-small cell lung cancer (NSCLC). While prior TKIs have demonstrated limited efficacy against exon 20 insertion mutations of EGFR (EGFR Ex20Ins), mobocertinib (TAK-788) is designed to specifically inhibit these Ex20Ins mutations. In a phase I/II clinical trial, mobocertinib demonstrated meaningful benefits among a cohort of platinum-pretreated patients with EGFR Ex20Ins mutant NSCLC. For this cohort, the objective response rate was 28% (95% confidence interval [CI], 20%-37%). The median progression-free survival and duration of response were 7.3 months (95% CI, 5.5-9.2) and 17.5 months (95% CI, 7.4-20.3), respectively, both by independent review committee assessment. On the basis of these results, mobocertinib was granted accelerated approval as the first TKI for treatment of this indication by the U.S. Food and Drug Administration (FDA) in 2021. This review summarizes the preclinical development of mobocertinib and the early-phase clinical data leading to its approval and discusses potential directions for mobocertinib's development.</p>","PeriodicalId":11397,"journal":{"name":"Drugs of today","volume":null,"pages":null},"PeriodicalIF":1.8,"publicationDate":"2022-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"40704841","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Olverembatinib in chronic myeloid leukemia. Olverembatinib治疗慢性髓性白血病。
IF 1.8 4区 医学
Drugs of today Pub Date : 2022-11-01 DOI: 10.1358/dot.2022.58.11.3441854
Renan Öziskender, Ahmet Emre Eşkazan
{"title":"Olverembatinib in chronic myeloid leukemia.","authors":"Renan Öziskender,&nbsp;Ahmet Emre Eşkazan","doi":"10.1358/dot.2022.58.11.3441854","DOIUrl":"https://doi.org/10.1358/dot.2022.58.11.3441854","url":null,"abstract":"<p><p>The introduction of tyrosine kinase inhibitors (TKIs) represents a new era in the management of chronic myeloid leukemia (CML). Despite their long clinical success, point mutations emerging before or during TKI treatment remain an obstacle for several cases. T315I is one of these point mutations in the tyrosine kinase domain of BCR::ABL1. It is a major cause of resistance against first- and second-generation TKIs and therefore lowers survival rates of a small group of patients. Olverembatinib (HQP-1351, formerly GZD-824) is a novel, orally active TKI, which acts through targeting the ATP-binding site of the BCR::ABL1 tyrosine kinase. In recent studies, olverembatinib appears to be an effective and safe treatment option for CML patients harboring T315I mutation. This article mainly focuses on the efficacy and safety data of olverembatinib along with other clinically available and potentially active drugs against T315I-mutated CML.</p>","PeriodicalId":11397,"journal":{"name":"Drugs of today","volume":null,"pages":null},"PeriodicalIF":1.8,"publicationDate":"2022-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"40704842","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Optimizing the use of Paxlovid in clinical practice. 优化Paxlovid在临床中的应用。
IF 1.8 4区 医学
Drugs of today Pub Date : 2022-11-01 DOI: 10.1358/dot.2022.58.11.3461265
M W McCarthy
{"title":"Optimizing the use of Paxlovid in clinical practice.","authors":"M W McCarthy","doi":"10.1358/dot.2022.58.11.3461265","DOIUrl":"https://doi.org/10.1358/dot.2022.58.11.3461265","url":null,"abstract":"<p><p>On December 22, 2021, the United States Food and Drug Administration (FDA) issued an emergency use authorization (EUA) for nirmatrelvir/ritonavir (Paxlovid) for the treatment of mild to moderate coronavirus disease 2019 (COVID-19). The drug is authorized for use in patients 12 years of age and older weighing at least 40 kg who have tested positive for severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) and who are at high risk for progression to severe COVID-19. Nirmatrelvir, an orally bioavailable protease inhibitor that prevents SARS-CoV-2 replication by cleaving the two viral polyproteins, is packaged with ritonavir, a cytochrome P450 (CYP)3A4 inhibitor and pharmacokinetic boosting agent that increases nirmatrelvir concentrations. Although Paxlovid has demonstrated clinical efficacy in unvaccinated patients with COVID-19, its role in the treatment of other populations is less clear. This manuscript reviews what is known about Paxlovid and explores how this drug may be used in the future to treat patients with SARS-CoV-2 infection.</p>","PeriodicalId":11397,"journal":{"name":"Drugs of today","volume":null,"pages":null},"PeriodicalIF":1.8,"publicationDate":"2022-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"40704843","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 5
Disitamab vedotin, a novel HER2-directed antibody-drug conjugate in gastric cancer and other solid tumors. diitamab vedotin，一种新的her2导向抗体-药物偶联物，用于胃癌和其他实体肿瘤。
IF 1.8 4区 医学
Drugs of today Pub Date : 2022-10-01 DOI: 10.1358/dot.2022.58.10.3408812
Yixuan Hu, Yinxing Zhu, Xiaowei Wei, Cuiju Tang, Wenwen Zhang
{"title":"Disitamab vedotin, a novel HER2-directed antibody-drug conjugate in gastric cancer and other solid tumors.","authors":"Yixuan Hu,&nbsp;Yinxing Zhu,&nbsp;Xiaowei Wei,&nbsp;Cuiju Tang,&nbsp;Wenwen Zhang","doi":"10.1358/dot.2022.58.10.3408812","DOIUrl":"https://doi.org/10.1358/dot.2022.58.10.3408812","url":null,"abstract":"<p><p>Antibody-drug conjugates (ADC), a combination of cytotoxic drugs and antibodies, have emerged as a rising star in cancer therapy. Disitamab vedotin (RC48), a novel ADC targeting human epidermal growth factor receptor 2 (HER2), is currently being explored in a variety of malignancies. Compared with conventional HER2-targeting agents, RC48 is characterized by a wider therapeutic window and less toxicity to normal tissues. In this review, we will analyze the structural elements and mechanisms of RC48. Besides, we provide a landscape on the progression of RC48 in common malignancies, focusing on RC48 in gastric or gastroesophageal junction cancer, and a brief overview of urothelial, breast and other cancers (e.g., gynecological cancer, biliary tract cancer, non-small cell lung cancer and myometrial invasive bladder cancer). Finally, we will also discuss future challenges in the development of RC48 and future directions to improve efficacy.</p>","PeriodicalId":11397,"journal":{"name":"Drugs of today","volume":null,"pages":null},"PeriodicalIF":1.8,"publicationDate":"2022-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"40434853","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 3
Asciminib in chronic myeloid leukemia. 阿西米尼治疗慢性髓性白血病。
IF 1.8 4区 医学
Drugs of today Pub Date : 2022-10-01 DOI: 10.1358/dot.2022.58.10.3441853
Giovanni Manfredi Assanto, Emilia Scalzulli, Massimo Breccia
{"title":"Asciminib in chronic myeloid leukemia.","authors":"Giovanni Manfredi Assanto,&nbsp;Emilia Scalzulli,&nbsp;Massimo Breccia","doi":"10.1358/dot.2022.58.10.3441853","DOIUrl":"https://doi.org/10.1358/dot.2022.58.10.3441853","url":null,"abstract":"<p><p>Despite the fact that, in the last years, life expectancy of chronic myeloid leukemia (CML) patients has reached that of the normal population, a significant proportion of CML patients is likely to fail treatment with first- or second-generation tyrosine kinase inhibitors (TKIs). Failure to first-line treatment is commonly due to molecular resistance or unbearable toxicity. New specific compounds are tested in this setting to fulfill this unmet clinical need in CML; of these, asciminib has shown efficacy based on allosteric inhibition which allows to overcome resistance and off-target toxicity. This review aims to cover how asciminib will change the therapeutic scenario of CML, highlighting its mechanism of action, pharmacokinetics, efficacy and toxicity. Asciminib will be a possible option as third-line therapy for patients carrying resistant mutations, such as T315I, and/or not eligible for treatment with other TKIs.</p>","PeriodicalId":11397,"journal":{"name":"Drugs of today","volume":null,"pages":null},"PeriodicalIF":1.8,"publicationDate":"2022-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"40434852","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 3
Aducanumab for the treatment of Alzheimer's disease. Aducanumab用于治疗阿尔茨海默病。
IF 1.8 4区 医学
Drugs of today Pub Date : 2022-10-01 DOI: 10.1358/dot.2022.58.10.3422314
Matteo Tagliapietra
{"title":"Aducanumab for the treatment of Alzheimer's disease.","authors":"Matteo Tagliapietra","doi":"10.1358/dot.2022.58.10.3422314","DOIUrl":"https://doi.org/10.1358/dot.2022.58.10.3422314","url":null,"abstract":"<p><p>Alzheimer's disease (AD) is the most frequent neurodegenerative condition, the most common cause of dementia, and a leading cause of disability and death globally. Mounting evidence supported accumulation of amyloid β (Aβ) as the primary cause of AD pathology and sprouted a number of candidate treatments engaging Aβ from its production to its clearance, yet no amyloid-based drug candidate had been proven effective. Alternative pathomechanisms have been proposed, but still current treatments are limited to symptomatic therapy. Aducanumab (BIIB-037) is a fully human monoclonal IgG1 antibody that selectively binds aggregated forms of Aβ, inhibits its template activity and promotes clearance of Aβ deposits. Three early terminated trials in humans are available. Overall, conflicting results exist over measures of clinical efficacy, despite an objective decrease in Aβ burden. Amyloid-related imaging abnormalities emerge as the most significant treatment-related adverse event. Here, we provide a comprehensive review of the available evidence on aducanumab, a drug that recently received a debated accelerated approval for the treatment of mild AD by the U.S. Food and Drug Administration (FDA).</p>","PeriodicalId":11397,"journal":{"name":"Drugs of today","volume":null,"pages":null},"PeriodicalIF":1.8,"publicationDate":"2022-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"40434851","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 29
Somapacitan: a long-acting growth hormone derivative for treatment of growth hormone deficiency. Somapacitan:一种治疗生长激素缺乏症的长效生长激素衍生物。
IF 1.8 4区 医学
Drugs of today Pub Date : 2022-10-01 DOI: 10.1358/dot.2022.58.10.3419556
David M Paton
{"title":"Somapacitan: a long-acting growth hormone derivative for treatment of growth hormone deficiency.","authors":"David M Paton","doi":"10.1358/dot.2022.58.10.3419556","DOIUrl":"https://doi.org/10.1358/dot.2022.58.10.3419556","url":null,"abstract":"<p><p>Growth hormone deficiency (GHD) is characterized by inadequate HG production from the anterior pituitary gland. Adult patients with GHD have increased fat mass, an abnormal lipid profile, decreased lean body mass and bone mineral density, decreased muscle strength and exercise endurance, and a diminished quality of life. Adult GHD (AGHD) has been treated with GH replacement therapy by daily subcutaneous injections. However, the administration of daily injections can be burdensome for some patients and affect treatment adherence. Somapacitan is a new long-acting human GH (hGH) analogue that is administered once a week by subcutaneous injection. It was first approved by the U.S. Food and Drug Administration (FDA) as GH replacement therapy for adults with GHD, becoming the first hGH therapy for AGHD administered by once-weekly injection, compared with other approved hGH therapies for AGHD administered by daily injection. This article reviews the pharmacokinetic, clinical and safety data that led to the approval of somapacitan as the first long-acting GH therapy for adults with GHD.</p>","PeriodicalId":11397,"journal":{"name":"Drugs of today","volume":null,"pages":null},"PeriodicalIF":1.8,"publicationDate":"2022-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"40455550","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Difelikefalin for pruritus associated with renal disease. 异花铁素治疗与肾脏疾病相关的瘙痒。
IF 1.8 4区 医学
Drugs of today Pub Date : 2022-09-01 DOI: 10.1358/dot.2022.58.9.3425323
Lucio Manenti, Paride Fenaroli
{"title":"Difelikefalin for pruritus associated with renal disease.","authors":"Lucio Manenti,&nbsp;Paride Fenaroli","doi":"10.1358/dot.2022.58.9.3425323","DOIUrl":"https://doi.org/10.1358/dot.2022.58.9.3425323","url":null,"abstract":"<p><p>Chronic kidney disease-associated pruritus (CKD-aP) has been recognized for over a century. The complex pathophysiology of CKD-aP makes it challenging to find an effective treatment; the proposed therapeutic options come from anecdotal reports and small clinical trials, which at best compare the test drug against placebo. Gabapentinoids have shown relevant efficacy but there are serious safety concerns about their possible central nervous system toxicity. Recently difelikefalin, a κ-opioid receptor agonist, has been the first Food and Drug Administration (FDA)-approved drug for moderate-severe CKD-aP treatment. Approval from other regulatory agencies is expected in 2022. In this article, preclinical, pharmacokinetic and safety studies on difelikefalin are reported, but a great part of the data derive from meeting abstracts and non-peer-review communications and this is a possible cause for concern regarding bias in publication. A review of published and unpublished studies about difelikefalin in CKD-aP treatment is provided. Currently, two published large trials show that difelikefalin offers a new therapeutic opportunity to treat CKD-aP, a condition that leads to both worse survival and quality of life in hemodialysis patients.</p>","PeriodicalId":11397,"journal":{"name":"Drugs of today","volume":null,"pages":null},"PeriodicalIF":1.8,"publicationDate":"2022-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"40357709","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Efficacy of vosoritide in the treatment of achondroplasia. 伏索里肽治疗软骨发育不全的疗效观察。
IF 1.8 4区 医学
Drugs of today Pub Date : 2022-09-01 DOI: 10.1358/dot.2022.58.9.3422313
David M Paton
{"title":"Efficacy of vosoritide in the treatment of achondroplasia.","authors":"David M Paton","doi":"10.1358/dot.2022.58.9.3422313","DOIUrl":"https://doi.org/10.1358/dot.2022.58.9.3422313","url":null,"abstract":"<p><p>Achondroplasia is the commonest form of dwarfism and results from a mutation in the fibroblast growth factor receptor 3 (FGFR3) gene on chromosome 4p16.3. The mutation is at nucleotide 1138 resulting in a G-to-A transition (134934.0001). This condition is characterized by full penetration meaning that everyone with this genetic mutation will exhibit the phenotypic characteristics of achondroplasia. It is a gain-of function mutation that causes increased inhibition of cartilage formation. C-type natriuretic peptide (CNP) acts on the growth plate through the natriuretic peptide receptor-B (NPR-B) causing the transformation of guanosine 5'-triphosphate into cyclic guanosine monophosphate. However, CNP cannot be used in the treatment of achondroplasia because it is rapidly degraded by neutral endopeptidase. Vosoritide is a modified recombinant human CNP and has a half-life 10 times that of CNP. Clinical trials have demonstrated that vosoritide is effective in significantly increasing the annualized growth velocity in children with achondroplasia before the fusion of the epiphyses.</p>","PeriodicalId":11397,"journal":{"name":"Drugs of today","volume":null,"pages":null},"PeriodicalIF":1.8,"publicationDate":"2022-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"40357711","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 1
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