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.

项目总结/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 计划在华盛顿举办一个为期一天的混合会议。 5月中旬，题为“基因治疗研究中的病毒载体抗体： 赞助商政策和沟通”。我们正在研究这一主题，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的抗体（AB）测试的标准和沟通 (GT)research.于二零二一年，PGTME就此议题举行了两次聆听会及一次闭门会议。 这些会议的与会者提出了一些可能的教育和/或结构干预措施; 然而，仍然存在一些可行和有效的问题。PGTME正在召集一个多样化的 专家组，探讨可能的政策建议和通信标准化， 会议 基因治疗（GT）干预目前不能逆转或“关闭”，如果他们造成伤害的人， 收件人。此外，通过病毒载体给予GT导致免疫后遗症， 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 只能接收一个潜在的改变生活的候选人GT。鉴于这一套独特的事实GT 研究，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获益，以及此类试验的影响 在时间、费用、努力和机会成本方面的参与。虽然有很多道德问题 围绕GT临床试验，拟议的会议只集中在两个。首先，患者和/或护理人员 目前正在进行GT临床试验或正在进行GT临床试验的罕见疾病患者 预期经常收到来自赞助商和其他实体关于抗体的不一致的消息 作为筛选GT临床试验合格性潜在参与者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得到适当或一致的治疗。 来自临床医生或试验申办者的关于参与免疫原性影响的沟通 GT研究。 我们完全期待小组会议和与会者之间的其他非正式互动， 会议将有助于推进实践和政策，改善GT试验申办者的信息传递 和临床医生关于抗体水平测试的效用，并将为合作方法奠定基础， 更符合伦理道德和更有效的GT临床试验。