PMID- 33422139
OWN - NLM
STAT- MEDLINE
DCOM- 20210705
LR  - 20220531
IS  - 1757-6512 (Electronic)
IS  - 1757-6512 (Linking)
VI  - 12
IP  - 1
DP  - 2021 Jan 9
TI  - Intravitreal autologous mesenchymal stem cell transplantation: a non-randomized 
      phase I clinical trial in patients with retinitis pigmentosa.
PG  - 52
LID - 10.1186/s13287-020-02122-7 [doi]
LID - 52
AB  - 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 x 10(6), 5 x 10(6), or 1 x 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 
      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.
FAU - Tuekprakhon, Aekkachai
AU  - Tuekprakhon A
AD  - Department of Ophthalmology, Faculty of Medicine Siriraj Hospital, Mahidol 
      University, 2 Wanglang Road, Bangkok Noi, Bangkok, 10700, Thailand.
FAU - Sangkitporn, Siripakorn
AU  - Sangkitporn S
AD  - Stem cell and Regenerative Medicine Center, Department of Medical Sciences, 
      Ministry of Public Health, National Institute of Health, 88/7 Tivanon Road, 
      Muang, Nonthaburi, 11000, Thailand.
FAU - Trinavarat, Adisak
AU  - Trinavarat A
AD  - Department of Ophthalmology, Faculty of Medicine Siriraj Hospital, Mahidol 
      University, 2 Wanglang Road, Bangkok Noi, Bangkok, 10700, Thailand.
FAU - Pawestri, Aulia Rahmi
AU  - Pawestri AR
AD  - Faculty of Medicine, Universitas Brawijaya, Malang, Indonesia.
FAU - Vamvanij, Visit
AU  - Vamvanij V
AD  - Department of Orthopaedic Surgery, Faculty of Medicine Siriraj Hospital, Mahidol 
      University, Bangkok, Thailand.
FAU - Ruangchainikom, Monchai
AU  - Ruangchainikom M
AD  - Department of Orthopaedic Surgery, Faculty of Medicine Siriraj Hospital, Mahidol 
      University, Bangkok, Thailand.
FAU - Luksanapruksa, Panya
AU  - Luksanapruksa P
AD  - Department of Orthopaedic Surgery, Faculty of Medicine Siriraj Hospital, Mahidol 
      University, Bangkok, Thailand.
FAU - Pongpaksupasin, Phitchapa
AU  - Pongpaksupasin P
AD  - Department of Ophthalmology, Faculty of Medicine Siriraj Hospital, Mahidol 
      University, 2 Wanglang Road, Bangkok Noi, Bangkok, 10700, Thailand.
AD  - Research Department, Faculty of Medicine Siriraj Hospital, Mahidol University, 
      Bangkok, Thailand.
FAU - Khorchai, Areerat
AU  - Khorchai A
AD  - Stem cell and Regenerative Medicine Center, Department of Medical Sciences, 
      Ministry of Public Health, National Institute of Health, 88/7 Tivanon Road, 
      Muang, Nonthaburi, 11000, Thailand.
FAU - Dambua, Acharaporn
AU  - Dambua A
AD  - Stem cell and Regenerative Medicine Center, Department of Medical Sciences, 
      Ministry of Public Health, National Institute of Health, 88/7 Tivanon Road, 
      Muang, Nonthaburi, 11000, Thailand.
FAU - Boonchu, Patcharaporn
AU  - Boonchu P
AD  - Stem cell and Regenerative Medicine Center, Department of Medical Sciences, 
      Ministry of Public Health, National Institute of Health, 88/7 Tivanon Road, 
      Muang, Nonthaburi, 11000, Thailand.
FAU - Yodtup, Chonlada
AU  - Yodtup C
AD  - Stem cell and Regenerative Medicine Center, Department of Medical Sciences, 
      Ministry of Public Health, National Institute of Health, 88/7 Tivanon Road, 
      Muang, Nonthaburi, 11000, Thailand.
FAU - Uiprasertkul, Mongkol
AU  - Uiprasertkul M
AD  - Department of Pathology, Faculty of Medicine Siriraj Hospital, Mahidol 
      University, Bangkok, Thailand.
FAU - Sangkitporn, Somchai
AU  - Sangkitporn S
AD  - Stem cell and Regenerative Medicine Center, Department of Medical Sciences, 
      Ministry of Public Health, National Institute of Health, 88/7 Tivanon Road, 
      Muang, Nonthaburi, 11000, Thailand. somchai.s@dmsc.mail.go.th.
FAU - Atchaneeyasakul, La-Ongsri
AU  - Atchaneeyasakul LO
AUID- ORCID: 0000-0002-5320-7123
AD  - Department of Ophthalmology, Faculty of Medicine Siriraj Hospital, Mahidol 
      University, 2 Wanglang Road, Bangkok Noi, Bangkok, 10700, Thailand. 
      atchanee@hotmail.com.
LA  - eng
SI  - ClinicalTrials.gov/NCT01531348
PT  - Clinical Trial, Phase I
PT  - Journal Article
PT  - Research Support, Non-U.S. Gov't
DEP - 20210109
PL  - England
TA  - Stem Cell Res Ther
JT  - Stem cell research & therapy
JID - 101527581
SB  - IM
MH  - Humans
MH  - Intravitreal Injections
MH  - *Mesenchymal Stem Cell Transplantation/adverse effects
MH  - *Mesenchymal Stem Cells
MH  - Retina
MH  - *Retinitis Pigmentosa/genetics/therapy
MH  - Transplantation, Autologous
PMC - PMC7796606
OTO - NOTNLM
OT  - Inherited retinal diseases
OT  - Mesenchymal stem cell
OT  - Phase I clinical trial
OT  - Retinitis pigmentosa (RP)
OT  - Stem cell therapy
COIS- The authors declare that they have no competing interests.
EDAT- 2021/01/11 06:00
MHDA- 2021/07/06 06:00
PMCR- 2021/01/09
CRDT- 2021/01/10 20:25
PHST- 2020/11/11 00:00 [received]
PHST- 2020/12/23 00:00 [accepted]
PHST- 2021/01/10 20:25 [entrez]
PHST- 2021/01/11 06:00 [pubmed]
PHST- 2021/07/06 06:00 [medline]
PHST- 2021/01/09 00:00 [pmc-release]
AID - 10.1186/s13287-020-02122-7 [pii]
AID - 2122 [pii]
AID - 10.1186/s13287-020-02122-7 [doi]
PST - epublish
SO  - Stem Cell Res Ther. 2021 Jan 9;12(1):52. doi: 10.1186/s13287-020-02122-7.