Abstract
Therapies to reduce liver fibrosis and stimulate organ regeneration are urgently needed. We conducted a first-in-human, phase 1 dose-escalation trial of autologous macrophage therapy in nine adults with cirrhosis and a Model for End-Stage Liver Disease (MELD) score of 10-16 (ISRCTN 10368050). Groups of three participants received a single peripheral infusion of 107, 108 or up to 109 cells. Leukapheresis and macrophage infusion were well tolerated with no transfusion reactions, dose-limiting toxicities or macrophage activation syndrome. All participants were alive and transplant-free at one year, with only one clinical event recorded, the occurrence of minimal ascites. The primary outcomes of safety and feasibility were met. This study informs and provides a rationale for efficacy studies in cirrhosis and other fibrotic diseases.
Original language | English |
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Pages (from-to) | 1560-1565 |
Number of pages | 6 |
Journal | Nature Medicine |
Volume | 25 |
Early online date | 7 Oct 2019 |
DOIs | |
Publication status | Published - Oct 2019 |
Keywords
- regeneration
- translational research
- DIAGNOSIS
- MARKER
- DISEASE
- INJURY
- FUNCTIONAL-CHARACTERIZATION
- CELL THERAPY
- DUCTULAR REACTIONS
- TRANSPLANTATION