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@#Wet age-related macular degeneration is a severe and blinding disease with progressive central visual impairment, accounting for about 10%-15% of age-related macular degeneration. In recent years, with the deepening understanding of its pathogenesis, especially the discovery of anti-VEGF family, the clinical popularization of new drugs has brought benefits to patients. However, this treatment mode requires frequent intraocular injection, and many patients must continue to use it indefinitely to preserve their vision. Gene therapy can provide long-term stable anti-VEGF activity, which has become a new hotspot of research. Several methods of gene therapy are summarized as follows.
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In the 1960s, scientists first raised the idea of curing genetic diseases using gene therapy. This new conceptual strategy aimed to achieve a much longer therapeutic effect by introducing exogenous genetic materials into the patients. After more than five decades of ups and downs, gene therapy has been brought into a new era by those milestone breakthroughs in the 21st century. Here we reviewed and summarized the history and breakthroughs of gene therapy, including some critical clinical trials, approved drugs, and emerging gene editing techniques. We believe that with their unique advantages over traditional therapies, more gene therapies will become practical approaches to genetic diseases and benefit the entire human race.
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Humans , CRISPR-Cas Systems , Gene Editing , Genetic TherapyABSTRACT
Differentiated thyroid cancer is a common endocrine malignancy, and its incidence has increased rapidly in the past 10 years.Although most patients have a good prognosis when treated with traditional methods, it is difficult to achieve satisfactory results for a small part of refractory thyroid cancer. With the understanding of the pathogenesis of thyroid cancer gradually improving to the molecular level, targeting therapy based on mechanisms and molecular targets have become a frontier area in the treatment of refractory thyroid cancer. Up to now,a variety of molecular targets has been found to be closely related to the incidence of thyroid cancer, and their effectiveness and safety has been verified by clinical trials. A number of targeted drugs of refractory thyroid cancer have been found through a method of target-based drug design, and have come into clinical trials or clinical applications. The representative drugs, sorafenib and lenvatinib, were approved for the treatment of refractory differentiated thyroid cancer, which provides a new hope for the treatment of advanced thyroid cancer.
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Differentiated thyroid cancer is a common endocrine malignancy,and its incidence has increased rapidly in the past 10 years.Although most patients have a good prognosis when treated with traditional methods,it is difficult to achieve satisfactory results for a small part of refractory thyroid cancer. With the understanding of the pathogenesis of thyroid cancer gradually improving to the molecular level,targeting therapy based on mechanisms and molecular targets have become a frontier area in the treatment of re?fractory thyroid cancer. Up to now,a variety of molecular targets has been found to be closely related to the incidence of thyroid can?cer,and their effectiveness and safety has been verified by clinical trials. A number of targeted drugs of refractory thyroid cancer have been found through a method of target-based drug design,and have come into clinical trials or clinical applications. The representative drugs,sorafenib and lenvatinib,were approved for the treatment of refractory differentiated thyroid cancer,which provides a new hope for the treatment of advanced thyroid cancer.
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Em 2000, dada a urgência de adequação à realidade científica vigente, iniciativas de revisão dos aspectos polêmicos da Declaração de Helsinque (DoH) à publicação de sua quinta versão na qual ficou estabelecido que o uso do placebo seria aceito apenas quando nenhum tratamento existisse. A Federal Drug Administration nos E.U.A., todavia, continuou a exigir controle-placebo, adotando o Guia de Boas Práticas Clínicas. Também a Agência ReguladoraEuropeia considerou tal proibição arbitrária e, junto a outras entidades, lançaram-se numa campanha contra a DoH. Em 2002 e 2004, Notas de Clarificação publicadas na DoH provocaram grande comoção mundial, culminando na versão de 2008. Apesarda divergência dentre vários grupos, sobretudo, quanto ao uso do placebo, o texto atual assumiu uma posição neutra, flexibilizando sua aplicação. Todavia, uma vez norma ética, a atual DoH fomentou o reinício das discussões sobre a necessidade de um documento mais ampla e uniformemente aceito. Hoje, esta polêmica ainda continua em alguns países, principalmente no Brasil, onde o Conselho Federal de Medicina (CFM) e a Comissão Nacional de Ética em Pesquisa (CONEP) proíbem o uso de placebo em estudos clínicos em território nacional, quando existir qualquer tratamento disponível. Este fato eliciou grande polêmica entre agências regulatórias e pesquisadores brasileiros. Assim, dado que, desde Nuremberg, a garantia do não enfraquecimento das normas de proteção dos sujeitos de pesquisa recrutados mundo afora é razão primordial de ser da DoH, a reabertura de diálogo em níveis nacional einternacional se faz premente perante a nova revisão comemorativa de seus 50 anos em 2014...
In 2000, given the urgency for the ethical guidelines adequacy to the present scientific standards, revision initiatives of the most controversial aspects of the Declaration of Helsinki (DoH). These efforts culminated with the publication of its fifth version stating that the use of placebo was acceptable when proven treatmentdoes not exist. The US Federal Drug Administration, however, continued demanding the placebo control and adopted the Good Clinical Practice Guidelines. The European regulatory agency also considered such prohibition arbitrary and, amongst other entities,started a campaign against the DoH. In 2002 and 2004, Clarification Notes published on the DoH promoted extensive controversy worldwide, concluded at its version of 2008. Despite the divergence amongst many groups, especially on placebo use, the current text assumed a more neutral positioning, flexibilising its application. Being an ethical norm, the current DoH re-started the discussions about thenecessity of a document of uniform worldwide acceptance. Today, the controversy still remains in a few countries, especially in Brazil, where governmental regulatory agencies namely the Federal Council of Medicine and the National Commission for Research Ethics posed a ban on the placebo use in clinical trials nationally,when there is available treatment, corroborating with the ongoingcontroversy amongst regulatory agencies and researchers in Brazil.Therefore, since Nuremberg, given the warranty of not weakening the protection of research subjects recruited throughout the worldconstitutes the DoH most desired aim, dialogue must be resumed both nationally and internationally, considering the upcoming revisionin 2014, at the DoH 50th anniversary...
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Humans , Helsinki Declaration , Clinical Trials as Topic/ethics , Placebos , Societies, Medical/legislation & jurisprudence , BrazilABSTRACT
The performance of CRA directly affects the quality of clinical trials. In the appraisal of the CRA performance, satisfaction of clinical institutions plays a vital role. This article presents the outcome of a satisfaction survey on 16 clinical research centers in Shandong province, and the analysis of 503 survey samples regarding their exploratory factor and confirmatory factor respectively. The purpose is to identify the main factors for the satisfaction and to build a model to evaluate the satisfaction of hospitals for CRA.
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This paper introduces the international experiments of risk management in clinical trails, responsibility of sponsor, responsibility of investigator and responsibility of other participators and to help to improve our management level of clinical trails.
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The clinical trails of Traditional Chinese Medicine Herbs in treating insomnia need to pay attention to the adaptation and the symptoms change after stopping the herb medicines.It is recomnendated that the placebo control,sleep diary have to be a base for the result evaluation,the week is basic duration of evaluation and the insomnia symptom score is the major index.
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BACKGROUND: Cardiovascular disease is the major cause of death in industrialized countries including Korea. METHODS: The methodology of cardiovascular disease clinical trials, published in 'The Korean Circulation Journal(KCJ)' and in 'The Korean Journal of Internal Medicine (KJIM)' from 1971 to 1993 were reviewed. For the study period, a total of 169 research papers in this field, includion 155 papers in the KCJ and 14 in the KJIM, were reviewed. To review the methodology behind the cardiovascular disease clinical trials, presence of a control group, set up a primary end point, determined sample size and the statistical method to be used, report on the side effects, and consent of patients were checked. In cases involving clinical trials using control groups, rendomization and blinding were also analysed. RESULTS: Among clinical trials reviewed only 6 (4.0%) of the 169 papers were used a control group, and 46.7% were set up with a primary end point. There was no clinical trial which used statitical methods to estimate the sample size. Clinical trials which reported results with statistical methods were 107 to 169 papers;most of these used the student's paired t-test. The number reporting the side effects of intervention was 143(88.7%) and papers reporting with the consent of patients was 15(8.8%). CONCLUSION: Most of clinical trials reviewed did not use appropriate method for clinical trials. Methodologically well-designed clinical trials in cardiovascular disease should be performed to guarantee the validity of study result.