Stewart Mark D, Kalos Michael, Coutinho Vicki, Better Marc, Jazayeri Jonathan, Yohrling Jennifer, Jadlowsky Julie, Fuchs Miriam, Gidwani Shalini, Goessl Carsten, Hanley Patrick J, Healy Jane, Liu Wen, McKelvey Brittany A, Pearce Laura, Pilon-Thomas Shari, Andrews Hillary S, Veldman Monica, Vong Judy, Weinbach Susan P, Allen Jeff D
Friends of Cancer Research, Washington, DC, USA.
Next Pillar Consulting, Philadelphia, PA, USA.
Cytotherapy. 2024 Jul;26(7):778-784. doi: 10.1016/j.jcyt.2024.03.009. Epub 2024 Mar 16.
Significant advancements have been made in the field of cellular therapy as anti-cancer treatments, with the approval of chimeric antigen receptor (CAR)-T cell therapies and the development of other genetically engineered cellular therapies. CAR-T cell therapies have demonstrated remarkable clinical outcomes in various hematological malignancies, establishing their potential to change the current cancer treatment paradigm. Due to the increasing importance of genetically engineered cellular therapies in the oncology treatment landscape, implementing strategies to expedite development and evidence generation for the next generation of cellular therapy products can have a positive impact on patients.
We outline a risk-based methodology and assessment aid for the data extrapolation approach across related genetically engineered cellular therapy products. This systematic data extrapolation approach has applicability beyond CAR-T cells and can influence clinical development strategies for a variety of immune therapies such as T cell receptor (TCR) or genetically engineered and other cell-based therapies (e.g., tumor infiltrating lymphocytes, natural killer cells and macrophages).
By analyzing commonalities in manufacturing processes, clinical trial designs, and regulatory considerations, key learnings were identified. These insights support optimization of the development and regulatory approval of novel cellular therapies.
The field of cellular therapy holds immense promise in safely and effectively treating cancer. The ability to extrapolate data across related products presents opportunities to streamline the development process and accelerate the delivery of novel therapies to patients.
随着嵌合抗原受体（CAR）-T细胞疗法的获批以及其他基因工程细胞疗法的发展，细胞疗法作为抗癌治疗领域取得了重大进展。CAR-T细胞疗法在各种血液系统恶性肿瘤中已显示出显著的临床疗效，确立了其改变当前癌症治疗模式的潜力。由于基因工程细胞疗法在肿瘤治疗格局中的重要性日益增加，实施加速下一代细胞治疗产品开发和证据生成的策略可能会对患者产生积极影响。
我们概述了一种基于风险的方法和评估辅助工具，用于跨相关基因工程细胞治疗产品的数据外推方法。这种系统的数据外推方法不仅适用于CAR-T细胞，还可影响多种免疫疗法的临床开发策略，如T细胞受体（TCR）或基因工程及其他基于细胞的疗法（如肿瘤浸润淋巴细胞、自然杀伤细胞和巨噬细胞）。
通过分析制造工艺、临床试验设计和监管考量方面的共性，确定了关键经验教训。这些见解有助于优化新型细胞疗法的开发和监管批准。
细胞疗法领域在安全有效地治疗癌症方面具有巨大潜力。跨相关产品外推数据的能力为简化开发过程和加速向患者提供新型疗法提供了机会。
