Clinical research
Being tested in people in registered clinical trials. Being in trials is not evidence that a treatment works or is safe.
Ce que teste cet essai
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 ↗
Ce que signifie (et ne signifie pas) un résultat d'essai
Les résultats en phase précoce proviennent d'un petit nombre de participants et d'un suivi de courte durée. Ils peuvent évoluer avec davantage de personnes et de temps, et un essai en cours de recrutement, actif ou même terminé n'a pas établi qu'un traitement est efficace. Les critères d'inclusion sont stricts ; l'éligibilité est décidée par le site de l'essai, jamais par un site web.