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.

项目摘要/摘要 小儿基因疗法和医学伦理工作组（PGTME） - 在纽约大学格罗斯曼学校 医学伦理部医学部的计划在华盛顿特区举办为期一天的混合动力会议。 在5月中旬的标题为“基因疗法研究中的病毒载体抗体：寻求最佳实践 赞助商政策和沟通。“我们正在研究该主题，以追求可能的政策解决方案 解决针对基因治疗中使用的病毒载体抗体（AB）测试的标准和通信 （GT）研究。 2021年，PGTME举行了两次听取会议和一次有关此主题的闭门会议。 这些会议的参与者提出了许多潜在的教育和/或结构性干预措施； 但是，仍然存在可行和有效的问题。 PGTME召集潜水员 在此期间探索可能的政策建议和沟通标准化的专家小组 会议。 基因疗法（GT）干预措施目前无法逆转或“关闭”，如果它们对危害造成伤害 接受者。此外，通过病毒载体施用的GTS导致免疫后遗症 排除接受者参加将来的GT临床试验。目前，这些研究中的研究对象 只能获得一个潜在的改变人生的候选GT。考虑到有关GT的独特事实 研究，患者和/或替代决策者必须最好地理解 参加GT临床试验的潜在风险和好处以及该试验的含义 参与时间，费用，努力和机会成本。虽然有许多道德问题 围绕GT临床试验，拟议的会议仅着重于两个。首先，病人和/或护理人员 GT临床试验目前正在进行的罕见疾病患者或目前正在 预计通常会从赞助商和其他实体获得有关抗体的不一致的消息传递 在GT临床试验中筛选潜在参与者资格的一部分进行的水平测试。 其次，这些患者和/或护理人员通常无法获得适当或一致的 临床医生或试验发起人就参与的免疫原性的沟通 GT研究。 我们完全期望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试用版发起人的消息传递 以及有关抗体水平测试实用程序的临床医生，并将为旨在的协作方法奠定基础 更具道德和更有效的GT临床试验。