Kangstem Publishes Promising Phase 1 Results for Cord-Blood Derived Stem Cell Therapy in Knee Osteoarthritis, Post-MRI Data Suggest Potential Cartilage Regeneration
The Korean biotech company published the multicenter Phase 1 results in Nature, with 24-week follow-up MRI data that look promising as they continue development.
Key Points
- Kangstem published Phase 1 data on OSCA, a combination of its cord blood-derived stem cells and an acellular cartilage matrix for knee osteoarthritis.
- In the 12-patient, multicenter dose-escalation study, OSCA was well tolerated, with no dose-limiting toxicities and only mild to moderate treatment-emergent adverse events reported in 3 patients.
- At 24 weeks, the mid-dose and high-dose groups showed stronger improvements in pain, function, and MRI-based structural measures, suggesting potential cartilage repair activity.
Kangstem Biotech, a Korean biotech company, has published results from its first multicenter Phase 1 of OSCA, a combination of its cord blood-derived stem cells and an acellular cartilage matrix for knee osteoarthritis, in Nature. They are calling this product “Furestem-OA Kit“, and it appears that it was initially tested in goats, for which they also published the results in Springer’s Tissue Engineering and Regenerative Medicine.
You can view their proposed mechanisms of action, details on the kit, before/after videos of the goats, and images of the goats’ meniscus before/after on their site here. They’re also developing products for atopic dermatitis, organoids for research, and many other interesting projects.
Results
The Phase 1 trial evaluated a single intra-articular injection of OSCA in 12 patients with Kellgren-Lawrence grade 2 or 3 knee osteoarthritis.
Patients were enrolled in low-, mid-, and high-dose cohorts, and the primary endpoint was safety/tolerability. Secondary endpoints were pain, function, and MRI-derived structural changes over 24 weeks.
The MRIs were reviewed independently by RadMD, a US-based radiology company.
Here’s what the paper reported:
- No dose-limiting toxicities were reported, but treatment-emergent adverse events occurred in 25% of patients (3 of 12), described as mild to moderate.
- At 24 weeks, the mid-dose and high-dose groups showed numerically greater improvement in pain and function than the low-dose group.
- Up to 91.3% reduction in Visual Analog Scale pain scores and a 102.7% improvement in International Knee Documentation Committee scores in higher-dose groups.
- MRI findings showed MOCART total score improvement in 70% of patients.
- WORMS cartilage integrity improved in 50% of patients.
- Kellgren-Lawrence grade remained stable in 80% of patients over the 24-week follow-up.
They also posted on LinkedIn after the trial, stating that a radiologist (assuming part of the RadMD group) said, “The WORMS results confirmed cartilage regeneration in all three subjects, such as filling in areas of cartilage were and smoothing the cartilage surface. Particularly, some subjects showed improvement in the structure of the subchondral bone that supports the cartilage, in addition to cartilage regeneration.“
These results suggest the therapy was feasible to administer and may have shown early signals of structural benefit alongside symptom improvement. Because this was an open-label Phase 1 study with a small patient group, the findings should of course be viewed as preliminary.
Discussion
The authors note that human umbilical cord blood-derived cells are a potentially useful source owing to their proliferative capacity, relatively low immunogenicity, and secretion of factors involved in tissue repair and modulation of the joint microenvironment.
The company also notes that combining the cells with cartilage acellular matrix may support a more favorable repair response than cells alone. Based on the Phase 1 safety profile and the MRI and clinical signals seen at 24 weeks, the results support further study in larger controlled trials to determine whether the structural changes translate into durable clinical benefit.
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