Clinical research
Being tested in people in registered clinical trials. Being in trials is not evidence that a treatment works or is safe.
Cosa sta testando questo trial
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 ↗
Cosa significa (e cosa non significa) il risultato di un trial
I risultati delle fasi iniziali provengono da un numero ridotto di partecipanti e da un follow-up breve. Possono cambiare con più persone e più tempo, e una sperimentazione in fase di arruolamento, attiva o anche completata non ha dimostrato che un trattamento funzioni. I criteri di arruolamento sono rigidi; l'idoneità è stabilita dal centro che conduce la sperimentazione, mai da un sito web.