Ontology highlight
ABSTRACT: 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?×?106, 5?×?106, or 1?×?107 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 observation without any intervention. For the long-term follow-up, only one participant needed surgical treatment for a serious adverse event and the vision was restored. An enrollment of larger number of participants with less advanced RP and long-term follow-up is required to evaluate the safety and efficacy of this intervention.Trial registration
ClinicalTrials.gov, NCT01531348 . Registered on February 10, 2012.
SUBMITTER: Tuekprakhon A
PROVIDER: S-EPMC7796606 | biostudies-literature | 2021 Jan
REPOSITORIES: biostudies-literature
Tuekprakhon Aekkachai A Sangkitporn Siripakorn S Trinavarat Adisak A Pawestri Aulia Rahmi AR Vamvanij Visit V Ruangchainikom Monchai M Luksanapruksa Panya P Pongpaksupasin Phitchapa P Khorchai Areerat A Dambua Acharaporn A Boonchu Patcharaporn P Yodtup Chonlada C Uiprasertkul Mongkol M Sangkitporn Somchai S Atchaneeyasakul La-Ongsri LO
Stem cell research & therapy 20210109 1
<h4>Background</h4>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.<h4>Methods</h4>This non-randomized phase I clinical trial enrolled 14 participants, categoriz ...[more]