Clinical research
Being tested in people in registered clinical trials. Being in trials is not evidence that a treatment works or is safe.
Qué está evaluando este ensayo
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 anyone was helped long term.
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 ↗
Qué significa y qué no significa el resultado de un ensayo
Los resultados de fases tempranas provienen de un número reducido de participantes y un seguimiento breve. Pueden cambiar con más personas y más tiempo, y un ensayo en reclutamiento, activo o incluso completado no ha demostrado que un tratamiento funcione. Los criterios de inscripción son estrictos; la elegibilidad la decide el centro del ensayo, nunca un sitio web.