Clinical research
Being tested in people in registered clinical trials. Being in trials is not evidence that a treatment works or is safe.
O que este estudo está testando
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 ↗
O que um resultado de estudo significa — e o que não significa
Resultados de fases iniciais vêm de um número pequeno de participantes e acompanhamento de curto prazo. Eles podem mudar com mais pessoas e mais tempo, e um ensaio que está recrutando, ativo ou até concluído ainda não estabeleceu que um tratamento funciona. Os critérios de inclusão são rigorosos; a elegibilidade é decidida pelo centro do ensaio, nunca por um site.