Disease
NCT ID
Source
Dose (/kg)
Route
Age
Follow-up
Type of study
Location
Cerebral Palsy
01072370
Autologous (MNC)
1 × 107
iv
2–12 years old
1 year
Phase I/II (placebo, double blinded)
Georgia Regents University
01147653
Autologous
1 × 107
iv
1–6 years old
2 years
Phase II (randomized double blinded)
Duke University, NC
01193660
Allogeneic (immuno-suppression)
3 × 107
iv
10 m-10 years
6 months
Randomized/double blind + erythropoietin
Bundang CHA Hospital, Korea
01486732
Allogeneic (immuno-suppression)
NS
iv/ia
6 m-20 years
3 months
Phase II (Randomized, placebo + rehabilitation)
Bundang CHA Hospital, Korea
01528436
Allogeneic (immuno-suppression)
D/K
iv/ia
6 m-20 years
3 months
Phase II (randomized, double-blind, placebo + rehabilitation)
Bundang CHA Hospital, Korea
01639404
Allogeneic (immuno-suppression)
D/K
iv/ia
6 m-20 years
3 months
Open-label + rehabilitation
Bundang CHA Hospital, Korea
SCI
01046786
Allogeneic (HLA matched)
1.6–6.4 × 106 (total)
Spinal
18–60 years old (>12 months after SCI)
1 year
Phase I/II (open-label, dose escalation)
University of Hong Kong
01354483
Allogeneic (HLA matched)
1.6–6.4 × 106 (total)
Spinal
18–60 (>12 months after SCI)
1 year
Phase I/II (open-label, dose escalation)
Chengdu Army Kunming General Hospital, China
01393977
Allogeneic (MSC)
D/K
spinal
20–50
3 months
Phase II (Open-label + rehabilitation)
Chinese People’s Armed Police Force, China
01471613
Allogeneic (StemCyte)
6.4 × 106 (total)
spinal
18–65 (<4 weeks since SCI)
1 year
Phase I/11 (Randomized, double-blind, placebo-controlled + lithium)
Chinese PLA Chengdu Army Kunming General Hospital
Stroke
01438593
CD34+ (StemCyte; HLA mismatch 5/6)
5 × 106 (total)
ic
35–75 years old (6–60 months post stroke)
6 months
Phase I (open label)
China Medical University, Taiwan
01673932
UCB-MNCs (HLA-matched)
1–4 × 107 (total)
ic
35–65 years old (6–60 months post stroke)
12–36 months
Phase I (open-label randomized control)
The University of Hong Kong
01700166
Autologous
D/K
iv
6 weeks–6 years old
2 year
Phase I (open label)
The University of Texas Health Science Center, Houston
AD
01297218
Allogenic (NeuroStem-AD; MSC)
3–6 × 106 (total)
ic
50–75 years old
12 weeks
Phase I (Open label)
Medipost Co., Korea
01696591
Allogenic (NeuroStem-AD; MSC)
3–6 × 106 (total)
ic
50–75 years old
24 months
Phase I (Open-label; follow-up)
Medipost Co., Korea
Neonatal hypoxia–ischemia
00593242
Autologous
5 × 107
iv
14 days old
26 months
Phase I (open label)
Duke University, NC
01649648
Autologous
D/K
iv
1–3 days old
2 years
Phase I (open label)
National University Hospital, Singapore
TBI
01251003
Autologous
<10 × 109
iv
6–18 months post TBI (Children)
2 years
Phase I/II (open label)
University of Texas, Houston
01451528
Allogeneic
D/K
iv/ia
>18 yearsold (>6 months with TBI)
6 months
Open label
CHA University
While cerebral palsy appears to be the most popular disease (6/19), four of the studies originate from the same institution and three of them appear to be Phase or type of trial variants. The other study was recently published with favorable results [217] and also uses EPO as a cotreatment. Unfortunately, there was no UCB alone group in this study, which makes it difficult to evaluate the contribution of the EPO.
Except for spinal cord injury, which is exclusively by spinal injection, in most cases, the cells will be injected intravenously. The AD studies and two of the stroke studies also involve direct injection into the brain.
While some of the studies may not be as rigorously performed as would be ideal to maximize interpretation of their effectiveness, it will be interesting to see how the trials fare with respect to treating the disease of interest. The animal studies suggest that many more clinical trials could be on the horizon.
5 Conclusions
UCB contains a heterogeneous mixture of immature cells that have shown potential for the treatment of many diseases of the CNS. This relates to their ability to modify the inflammatory response, promote angiogenesis and neurogenesis, and secrete a number of cell survival cytokines and neurotrophic factors. There are several clinical trials exploring their value in the clinic and as our understanding of these cells progress, many more trials should be performed.
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