Intravitreal autologous mesenchymal stem cell transplantation: a non-randomized phase I clinical trial in patients with retinitis pigmentosa
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 intravitrea...
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doaj-0f023597343e4294b34b623d3eead7c12021-01-10T12:13:04ZengBMCStem Cell Research & Therapy1757-65122021-01-0112111510.1186/s13287-020-02122-7Intravitreal autologous mesenchymal stem cell transplantation: a non-randomized phase I clinical trial in patients with retinitis pigmentosaAekkachai Tuekprakhon0Siripakorn Sangkitporn1Adisak Trinavarat2Aulia Rahmi Pawestri3Visit Vamvanij4Monchai Ruangchainikom5Panya Luksanapruksa6Phitchapa Pongpaksupasin7Areerat Khorchai8Acharaporn Dambua9Patcharaporn Boonchu10Chonlada Yodtup11Mongkol Uiprasertkul12Somchai Sangkitporn13La-ongsri Atchaneeyasakul14Department of Ophthalmology, Faculty of Medicine Siriraj Hospital, Mahidol UniversityStem cell and Regenerative Medicine Center, Department of Medical Sciences, Ministry of Public Health, National Institute of HealthDepartment of Ophthalmology, Faculty of Medicine Siriraj Hospital, Mahidol UniversityFaculty of Medicine, Universitas BrawijayaDepartment of Orthopaedic Surgery, Faculty of Medicine Siriraj Hospital, Mahidol UniversityDepartment of Orthopaedic Surgery, Faculty of Medicine Siriraj Hospital, Mahidol UniversityDepartment of Orthopaedic Surgery, Faculty of Medicine Siriraj Hospital, Mahidol UniversityDepartment of Ophthalmology, Faculty of Medicine Siriraj Hospital, Mahidol UniversityStem cell and Regenerative Medicine Center, Department of Medical Sciences, Ministry of Public Health, National Institute of HealthStem cell and Regenerative Medicine Center, Department of Medical Sciences, Ministry of Public Health, National Institute of HealthStem cell and Regenerative Medicine Center, Department of Medical Sciences, Ministry of Public Health, National Institute of HealthStem cell and Regenerative Medicine Center, Department of Medical Sciences, Ministry of Public Health, National Institute of HealthDepartment of Pathology, Faculty of Medicine Siriraj Hospital, Mahidol UniversityStem cell and Regenerative Medicine Center, Department of Medical Sciences, Ministry of Public Health, National Institute of HealthDepartment of Ophthalmology, Faculty of Medicine Siriraj Hospital, Mahidol UniversityAbstract 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, 2012https://doi.org/10.1186/s13287-020-02122-7Retinitis pigmentosa (RP)Inherited retinal diseasesMesenchymal stem cellStem cell therapyPhase I clinical trial |
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English |
format |
Article |
sources |
DOAJ |
author |
Aekkachai Tuekprakhon Siripakorn Sangkitporn Adisak Trinavarat Aulia Rahmi Pawestri Visit Vamvanij Monchai Ruangchainikom Panya Luksanapruksa Phitchapa Pongpaksupasin Areerat Khorchai Acharaporn Dambua Patcharaporn Boonchu Chonlada Yodtup Mongkol Uiprasertkul Somchai Sangkitporn La-ongsri Atchaneeyasakul |
spellingShingle |
Aekkachai Tuekprakhon Siripakorn Sangkitporn Adisak Trinavarat Aulia Rahmi Pawestri Visit Vamvanij Monchai Ruangchainikom Panya Luksanapruksa Phitchapa Pongpaksupasin Areerat Khorchai Acharaporn Dambua Patcharaporn Boonchu Chonlada Yodtup Mongkol Uiprasertkul Somchai Sangkitporn La-ongsri Atchaneeyasakul Intravitreal autologous mesenchymal stem cell transplantation: a non-randomized phase I clinical trial in patients with retinitis pigmentosa Stem Cell Research & Therapy Retinitis pigmentosa (RP) Inherited retinal diseases Mesenchymal stem cell Stem cell therapy Phase I clinical trial |
author_facet |
Aekkachai Tuekprakhon Siripakorn Sangkitporn Adisak Trinavarat Aulia Rahmi Pawestri Visit Vamvanij Monchai Ruangchainikom Panya Luksanapruksa Phitchapa Pongpaksupasin Areerat Khorchai Acharaporn Dambua Patcharaporn Boonchu Chonlada Yodtup Mongkol Uiprasertkul Somchai Sangkitporn La-ongsri Atchaneeyasakul |
author_sort |
Aekkachai Tuekprakhon |
title |
Intravitreal autologous mesenchymal stem cell transplantation: a non-randomized phase I clinical trial in patients with retinitis pigmentosa |
title_short |
Intravitreal autologous mesenchymal stem cell transplantation: a non-randomized phase I clinical trial in patients with retinitis pigmentosa |
title_full |
Intravitreal autologous mesenchymal stem cell transplantation: a non-randomized phase I clinical trial in patients with retinitis pigmentosa |
title_fullStr |
Intravitreal autologous mesenchymal stem cell transplantation: a non-randomized phase I clinical trial in patients with retinitis pigmentosa |
title_full_unstemmed |
Intravitreal autologous mesenchymal stem cell transplantation: a non-randomized phase I clinical trial in patients with retinitis pigmentosa |
title_sort |
intravitreal autologous mesenchymal stem cell transplantation: a non-randomized phase i clinical trial in patients with retinitis pigmentosa |
publisher |
BMC |
series |
Stem Cell Research & Therapy |
issn |
1757-6512 |
publishDate |
2021-01-01 |
description |
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 |
topic |
Retinitis pigmentosa (RP) Inherited retinal diseases Mesenchymal stem cell Stem cell therapy Phase I clinical trial |
url |
https://doi.org/10.1186/s13287-020-02122-7 |
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