Off-the-Shelf CAR T Shows Early Signal in Progressive Multiple Sclerosis

Phase I data show B-cell depletion and mostly stable or improved disability scores, but the uncontrolled study remains small and follow-up is limited

1

The phase 1 TG-Azercel-101 study evaluated off-the-shelf, donor-derived CD19-directed CAR T-cell therapy in 26 people with progressive multiple sclerosis.
2

All participants had disability progression independent of relapse activity during the prior year despite high-efficacy treatment and had previously received anti-CD20 therapy.
3

After bendamustine lymphodepletion and a single azer-cel infusion, peripheral B-cell counts fell below 10 cells per microliter in 25 of 26 participants.
4

Among 21 participants with paired cerebrospinal fluid samples, median CD19-positive B-cell events declined by 52.3% at day 10.
5

At day 90, disability scores were stable or improved in 16 of 18 evaluable participants; no dose-limiting toxicities or grade 3 or higher cytokine release syndrome were reported.

1