Phase 1 Study of CK0801 in Treatment of Bone Marrow Failure Syndromes
Background
An inflammatory bone marrow microenvironment contributes to acquired bone marrow failure syndromes. CK0801, an allogeneic T regulatory (Treg) cell therapy product, can potentially interrupt this continuous loop of inflammation and restore hematopoiesis.
Methods
In this phase 1 dose-escalation study of CK0801 Treg cells, we enrolled patients with bone marrow failure syndromes with suboptimal response to their prior therapy to determine the safety and efficacy of this treatment for bone marrow failure syndromes.
Results
We enrolled nine patients with a median age of 57 years with an underlying diagnosis of aplastic anemia (n=4), myelofibrosis (n=4), or hypoplastic myelodysplasia (n=1). CK0801 was administered in the outpatient setting with no infusion reactions, no severe adverse reactions, and no dose-limiting toxicity. At 12 months, CK0801 induced objective responses in patients with myelofibrosis and aplastic anemia, and three of four transfusion-dependent patients at baseline achieved transfusion independence. No increases in infections, leukemia transformations, or deaths were observed.
Conclusions
In previously treated patients, CK0801 demonstrated no dose-limiting toxicity and showed evidence of efficacy, providing proof of concept for targeting inflammation as a therapy for bone marrow failure.
Value: CK0801 showed evidence of efficacy and safety in treating bone marrow failure syndromes, providing a potential new therapy option for patients with suboptimal response to prior treatments.
Practical Solutions: The outpatient administration of CK0801 with no severe adverse reactions and no dose-limiting toxicity offers a practical and safe treatment option for patients with bone marrow failure syndromes.
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