Intravitreal autologous mesenchymal stem cell transplantation: a non-randomized phase I clinical trial in patients with retinitis pigmentosa
Tuekprakhon A., Sangkitporn S., Trinavarat A., Pawestri AR., Vamvanij V., Ruangchainikom M.
Clinical Trial with a reported sample of 14, published in Stem Cell Res Ther (2021) — summary generated from the PubMed abstract.
Several human studies show positive signals, while research methods and sample sizes continue to develop.
- Level A · Stronger Clinical Evidence
- Level B · Emerging clinical evidence with positive signals
- Level C · Early human research exploring benefits
- Level D · Scientific groundwork from lab and animal studies
- Emerging · Emerging topic under active research
This page is generated from the PubMed record. The Thai description is an automated summary of bibliographic fields and the abstract, not a full translation, and is not medical advice.
- Study type
- Clinical Trial
- Journal
- Stem Cell Res Ther (2021)
- Reported sample size
- 14
- Source database
- Europe PMC
- PMID
- 33422139
- PMCID
- PMC7796606
- DOI
- 10.1186/s13287-020-02122-7
- NCT
- NCT01531348
- Citations
- 60
Abstract (original English)
Background Retinitis pigmentosa (RP) is a progressive inherited retinal disease with great interest for finding effective treatment modalities. Stem cell-based therapy is one of the promising candidates. We aimed to investigate the safety, feasibility, and short-term efficacy of intravitreal injection of bone marrow-derived mesenchymal stem cells (BM-MSCs) in participants with advanced stage RP. Methods This non-randomized phase I clinical trial enrolled 14 participants, categorized into three groups based on a single dose intravitreal BM-MSC injection of 1 × 10 6 , 5 × 10 6 , or 1 × 10 7 cells. We evaluated signs of inflammation and other adverse events (AEs). We also assessed the best corrected visual acuity (BCVA), visual field (VF), central subfield thickness (CST), and subjective experiences. Results During the 12-month period, we noticed several mild and transient AEs. Interestingly, we found statistically significant improvements in the BCVA compared to baseline, although they returned to the baseline at 12 months. The VF and CST were stable, indicating no remarkable disease progression. We followed 12 participants beyond the study period, ranging from 1.5 to 7 years, and observed one severe but manageable AE at year 3. Conclusion Intravitreal injection of BM-MSCs appears to be safe and potentially effective. All adverse events during the 12-month period required observa
What this study does not prove
- • This study does not prove SVF is an approved treatment or a replacement for standard care.
Evidence level
Several human studies show positive signals, while research methods and sample sizes continue to develop.
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