CAR-T lymphocytes helped a fraction of sarcoma patients in a phase 2 trial

A scientific group has presented preliminary results of a Phase II trial of CAR-T-lymphocytes for the treatment of certain types of sarcoma. The drug showed an acceptable level of safety and was effective in a proportion of patients, as reported in an article from The Lancet. In it Brian Van Tine (Brian Van Tine) from Washington University in St. Louis with colleagues from the UK, Spain, Canada, USA and France published data from the main cohort of open non-randomized trials SPEARHEAD-1, which included 52 patients with HLA-A*02 aged 16-75 years in 23 clinical centers in North America and Europe. 44 had metastatic or inoperable synovial sarcoma and eight had myxoid round cell liposarcoma; tumor cells expressed the MAGE-A4 antigen. All participants, after several courses of chemotherapy and prior lymphodepletion, received a single intravenous dose of aphamitresgen autoleucel (aphami-Cel), a preparation of autologous T lymphocytes with a chimeric antigenic receptor (CAR) for this antigen. The dose ranged from 1.0 × 109 to 10.0 × 109 modified cells.

The median follow-up period was 32.6 months. Response to therapy was recorded in 37 percent of participants (39 percent for synovial sarcoma and 25 percent for myxoid round cell liposarcoma). Cytokine release syndrome developed in 71 percent of patients, but only one case reached third-degree severity. The most frequent severe adverse event was cytopenia. No therapy-related deaths occurred.