Mitochondrial transplantation as a novel therapeutic approach in idiopathic inflammatory myopathy
Authors' conclusion
PN-101 transplantation could serve as a novel treatment for IIM by enhancing mitochondrial repair and reducing inflammation in muscle tissues.
Study context
- Population
- Idiopathic inflammatory myopathy (IIM) myoblasts from patients, C2C12 perforin/granzyme B-treated myoblasts, CIM mice; adult patients with refractory PM or DM (trial)phase 1/2a trial patients
- Participants
- 9 participants
- Comparison
- PN-101 transplantation; trial outcomes vs baseline
- PN-101n = 9
Methodological detailsin vitro and in vivo IIM models, first-in-human phase 1/2a prospective clinical trial
- In vitro and in vivo IIM models
- First-in-human phase 1/2a prospective clinical trial
Main finding
As reported in the abstract
- IIM patient myoblasts showed defects in mitochondrial function and myogenesis
- PN-101 enhanced muscle differentiation and mitochondrial function in IIM myoblasts
- PN-101 enhanced intracellular ATP content, cell viability and myogenesis in C2C12 perforin/granzyme B-treated myoblasts
- In CIM mice, PN-101 reduced myositis severity via anti-inflammatory effects and restored the CIM-induced metabolic shift
- Trial: at least minimal improvement in IMACS Total Improvement Scores vs baseline
Safety
- No severe adverse drug reactions with PN-101
- Phase 1/2a trial, 9 adult patients with refractory polymyositis or dermatomyositis
Original publication
Mitochondrial transplantation as a novel therapeutic approach in idiopathic inflammatory myopathy. Ann Rheum Dis. Published 31 January 2025. doi:10.
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