Diz Osteoartriti İçin IL-1Ra Gen Terapisine Pratik Bir Alternatif Olarak Otolog Koşullandırılmış Serum (ACS)

Özet (abstract)

Osteoarthritis (OA) of the knee remains one of the most prevalent and disabling musculoskeletal conditions worldwide, with few disease-modifying options beyond joint replacement. The pro-inflammatory cytokine interleukin-1 (IL-1) has long been implicated in the pathogenesis of OA, and approaches to block IL-1 activity have included recombinant proteins, small molecules, autologous blood-derived biologics, and most recently, viral vector-based gene therapies delivering interleukin-1 receptor antagonist (IL-1Ra). Gene therapy platforms such as PCRX-201 (Pacira) and GNSC-001 (Genascence) represent a technical advance, aiming for durable intra-articular IL-1Ra expression from a single injection. However, these approaches are expensive, logistically complex, and face challenges of immune barriers to repeat dosing, cost of goods (COGS), and uncertain long-term safety, efficacy and cost-effectiveness.By contrast, autologous conditioned serum (ACS), marketed for human use as Orthokine (also Regenokine) and for veterinary use under trade names such as IRAP, IRAP ProEAS™, and IRAP II, delivers IL-1Ra directly to the joint using the patient's own blood. Though evidence is modest, ACS provides a mechanistically overlapping anti-inflammatory approach with lower cost, repeatability, and safety advantages. ACS is already clinically applied in Europe and in veterinary medicine in Europe and the U.S. whereas IL-1Ra gene therapy remains in mid-stage clinical trials. Until gene therapy demonstrates clear advantages in durability, disease modification, and cost-effectiveness, ACS may represent a more pragmatic and earlier option in the sequence of intra-articular treatments, ahead of where gene therapy would enter, in selected clinical contexts for targeting IL-1 in patients with knee OA.

Yazarların özeti; kaynağından alınmıştır. Connective Tissue Research, 2026 · DOI ↗