CK0801 Therapy Safe, Effective in Patients With Myelofibrosis

CK0801, an allogeneic T regulatory cell therapy, demonstrated safety and efficacy in patients with myelofibrosis (MF) with suboptimal responses to prior therapy in a recent study.

The phase I, dose-escalation study was led by Tapan Kadia, MD, of the University of Texas MD Anderson Cancer Center, and published in NEJM Evidence.

To determine the safety and efficacy of CK0801, the researchers evaluated nine patients with bone marrow failure syndromes such as MF (n=4), aplastic anemia (n=4), or hypoplastic myelodysplasia (n=1). Patients had a median of three prior therapies.

The starting dose levels were 1 × 10⁶ (n=3), 3 × 10⁶ (n=3), and 10 × 10⁶ (n=3) cells per kg of ideal body weight. Three patients with MF had objective responses at one year, including two patients with symptom responses, one with anemia response, and one with stable disease. Three patients with aplastic anemia had objective response and two had partial response.

Of the four transfusion-dependent patients at baseline, three achieved transfusion independence.

No infusion reactions, grade 3 or 4 severe adverse events, dose-limiting toxicities, or deaths were observed.

Reference

Kadia TM, Huang M, Pemmaraju N, et al. Phase 1 study of CK0801 in treatment of bone marrow failure syndromes. NEJM Evid. 2024. doi:10.1056/EVIDoa2300362