Clinical research
Being tested in people in registered clinical trials. Being in trials is not evidence that a treatment works or is safe.
本试验正在测试什么
The first American trial to give patients T cells edited with CRISPR. Three edits were made in each patient's own cells before they were returned. The main finding was that it could be done safely and that the edited cells survived for months — which was the question at that stage, not whether it cured anyone.
A first-in-human Phase 1 study at the University of Pennsylvania of autologous T cells with TRAC, TRBC and PDCD1 knocked out by CRISPR-Cas9 and an NY-ESO-1-directed transgenic receptor introduced, in refractory myeloma and sarcoma. Published in Science, it reported feasibility, an acceptable safety profile and persistence of edited cells for months.
Sources
- Science (Stadtmauer et al.) · 2020
CRISPR-engineered T cells in patients with refractory cancer ↗
试验结果意味着什么，又不意味着什么
早期试验结果来自少量参与者和较短的随访周期，随着人数增加和时间延长，结论可能发生变化。正在招募、进行中甚至已完成的试验，并不代表某种疗法已被证实有效。入组标准十分严格，是否符合条件由试验机构决定，而非由网站判断。