Antibodies to Viral Vectors in Gene Therapy Research: Seeking Best Practices for Sponsor Policies and Communications

基因治疗研究中的病毒载体抗体：寻求赞助商政策和沟通的最佳实践

• 批准号: 10683623
• 负责人: Alison S. Bateman-House
• 金额: $ 2.07万
• 依托单位: NEW YORK UNIVERSITY SCHOOL OF MEDICINE
• 依托单位国家: 美国
• 财政年份: 2023
• 资助国家: 美国
• 起止时间: 2023-04-01 至 2024-03-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/10683623
• 关键词: Address; Advocate; Antibodies; Area; Authorization documentation; Benefits and Risks; Candidate Disease Gene; Caregivers; Childhood; Clinical; Clinical Trials; Clinical and Translational Science Awards; Communication; Consensus; Development; Discipline; District of Columbia; Education; Eligibility Determination; Ethical Issues; Ethics; Faculty; Family; Fostering; Funding; Future; Future Generations; Gene therapy trial; Genetic Diseases; Heritability; Hybrids; Immunologics; Individual; Intervention; Life; Marketing; Medical Ethics; National Center for Advancing Translational Sciences; National Human Genome Research Institute; Output; Participant; Patients; Policies; Rare Diseases; Research Personnel; Research Subjects; Standardization; Test Result; Testing; Therapeutic Intervention; Therapeutic Studies; Time; Training; United States Food and Drug Administration; Viral; Viral Vector; antibody test; authority; career; collaborative approach; collaborative environment; design; eligible participant; gene therapy; gene therapy clinical trial; immunogenic; immunogenicity; improved; interest; medical schools; meetings; member; opportunity cost; outreach; policy recommendation; programs; provider communication; screening; surrogate decision maker; symposium; trend; working group

Project Summary/Abstract The Pediatric Gene Therapy and Medical Ethics Working Group (PGTME)—housed in NYU Grossman School of Medicine’s Division of Medical Ethics—is planning to host a one-day hybrid conference in Washington D.C. in mid-May entitled, “Antibodies to Viral Vectors in Gene Therapy Research: Seeking Best Practices for Sponsor Policies and Communications.” We are researching this topic in pursuit of possible policy solutions addressing standards of and communication around antibody (AB) testing to viral vectors used in gene therapy (GT) research. In 2021, PGTME held two listening sessions and one closed-door meeting on this topic. Participants from these meetings raised a number of potential educational and/or structural interventions; however, there remain questions around which are feasible and effective. PGTME is convening a diverse group of experts to explore possible policy recommendations and communication standardization during this conference. Gene therapy (GT) interventions cannot currently be reversed or “turned off,” should they cause harm to the recipient. Furthermore, GTs administered by means of a viral vector result in immunologic sequelae which preclude recipients’ participation in future GT clinical trials. Thus, at present, research subjects in these studies are only able to receive one potentially life-altering candidate GT. Given this unique set of facts about GT research, it is essential that patients and/or surrogate decision-makers have the best possible understanding of the potential risks and benefits of participating in a GT clinical trial, as well as the implications of such trial participation in terms of time, expense, effort, and opportunity costs. While there are numerous ethical issues surrounding GT clinical trials, the proposed conference focuses on only two. First, patients and/or caregivers of individuals with rare diseases for which GT clinical trials are currently underway or are currently anticipated often receive inconsistent messaging from sponsors and other entities about the antibody level testing done as part of screening a potential participant for eligibility in a GT clinical trial. Secondly, these patients and/or caregivers often do not receive appropriate or consistent communication from clinicians or trial sponsors about immunogenic implications of participating in GT research. We fully anticipate that the panel sessions and other informal interactions between the participants of this conference will contribute to advancing practices and policies that improve messaging from GT trial sponsors and clinicians about antibody level testing utility and will set the basis for collaborative approaches aimed at more ethical and more efficient GT clinical trials.

项目概要/摘要 儿科基因治疗和医学伦理工作组 (PGTME)——设在纽约大学格罗斯曼学院 医学伦理学部正计划在华盛顿特区举办为期一天的混合会议 五月中旬，题为“基因治疗研究中病毒载体的抗体：寻求最佳实践” “赞助商政策和沟通。”我们正在研究这个主题，以寻求可能的政策解决方案 解决基因治疗中使用的病毒载体抗体（AB）测试的标准和沟通 （GT）研究。2021年，PGTME就此主题举行了两次聆听会和一次闭门会议。 这些会议的与会者提出了一些潜在的教育和/或结构性干预措施； 然而，PGTME 召开多元化会议的可行性和有效性仍存在疑问。 专家组将在此期间探讨可能的政策建议和通信标准化 会议。 基因治疗 (GT) 干预措施目前无法逆转或“关闭”，如果它们对人体造成伤害 此外，对于接受者来说，通过病毒载体施用GT会导致免疫学后遗症， 排除接受者参与未来的 GT 临床试验，因此，目前这些研究中的研究对象。 考虑到这组关于 GT 的独特事实，他们只能获得一个可能改变生活的候选 GT。 研究中，患者和/或代理决策者必须尽可能了解 参与 GT 临床试验的潜在风险和益处，以及此类试验的影响 参与的时间、费用、精力和机会成本。 围绕 GT 临床试验，拟议的会议仅关注两个问题：第一，患者和/或护理人员。 GT 临床试验目前正在进行或正在进行的罕见疾病个体 通常预计赞助商和其他实体会收到关于抗体的不一致的消息 作为筛选潜在参与者是否有资格参加 GT 临床试验的一部分而进行的水平测试。 其次，这些患者和/或护理人员通常没有接受适当或一致的治疗 来自群体或试验申办者关于参与的免疫原性影响的沟通 GT研究。 我们完全预计本次会议参与者之间的小组会议和其他非正式互动 会议将有助于推进实践和政策，以改善 GT 试验申办者的信息传递 以及关于抗体水平测试实用性，并将为旨在实现以下目标的协作方法奠定基础： 更道德、更高效的 GT 临床试验。