Skip to main content
. 2018 Apr 24;96(6):469–481. doi: 10.1007/s00109-018-1638-5

Table 2.

A summary of the advantages and disadvantages of various cell sources which can be used to generate induced hepatocytes for hepatocyte transplantation

Study name Cell source Condition Intervention Primary outcome Study phase Location Start and end date References
Umbilical cord mesenchymal stem cell transplantation combined with plasma exchange for patients with liver failure Umbilical mesenchymal stem cell (UC-MSC) Liver failure Conventional treatment only (antiviral drugs, lowering aminotransferase and jaundice medicine)
Conventional treatment plus UC-MSC transplantation (via peripheral vein slowly for 30 min 1 × 105/kg, once a week, four times) or plasma exchange (2000 mL every 3 days, three times) or both
Survival rate and time (time frame 48 weeks) Phase I and II Department of Infectious Diseases, The Third Affiliated Hospital of Sun Yat-Sen University Guangzhou, Guangdong China November 2012–March 2015 [84]
Safety and efficacy of human umbilical cord-derived mesenchymal stem cells for treatment of HBV-related liver cirrhosis Umbilical mesenchymal stem cell (UC-MSC) Liver cirrhosis
End-stage liver disease
Conventional treatment or UC-MSC transplantation (1 × 106 cells/kg via hepatic artery) One-year survival rate (time frame 1-year treatment) Phase I and II Xijing Hospital of Digestive Disease Xi’an, Shaanxi, China September 2012–September 2015 [85]
Phase II safety study of two dose regimens of HepaStem in patients with ACLF (HEP101) Human liver-derived mesenchymal stem cell (HepaStem) Acute-on-chronic liver failure Low-dose cohort—two dose regimens of HepaStem will be given, differing in cell quantity per infusion. The low dose regimen will be given to the first cohort (first six patients included in the study).
High-dose cohort—given to the second cohort after evaluation of the safety of the first cohort (stepwise approach)
Occurrence of adverse events (AEs) up to day 28 of the active study period (time frame up to 28 days post-first infusion day) Phase II Hȏpital Erasme, Brussels, Belgium
UZ Antwerpen, Edegem, Belgium
KU Leuven, Leuven, Belgium
CHU de Liège, Liège, Beligium
Cliniques St. Luc, Woluwe-Saint Lambert, Belgium
Hȏspital Beaujon, Clichy, France
Hȏpital de la Croix Rousse, Lyon, France
Hȏpital Paul Brousse, Villejuf, France
December 2016–September 2018 [86]
Bone marrow stem cells as a source of allogenic hepatocyte transplantation in homozygous familial hypercholesterolemia Bone marrow stem cells Familial hypercholesterolemia Bone marrow stem cell transplantation. 6 × 108 to 1 × 109 cells infused through the portal vein over 30 min, done once Serum cholesterol and LDL level (time frame 6 months) Phase I Digestive Disease Research Center, Shariati Hospital, North Kargar Ave., Tehran, Iran, Islamic Republic June 2007–June 2008 [87]
Study to evaluate the efficacy of HepaStem in urea cycle disorders of paediatric patients (HEP002) Human liver-derived mesenchymal stem cell (HepaStem) Urea cycle disorders HepaStem administered in maximum four infusion days, spread over 8 weeks, with 2/3-week interval between infusions. Target total dose 5 × 107/kg body weight Efficacy as determined by de novo ureagenesis (C13 tracer method) (time frame 6 months post-first infusion day) Phase II Cliniques Universitaires Saint-Luc, Brussels, Belgium, Hȏpital Jeanne de Flandre, CHRU Lille, Lille, France
Instytut–Pomnik Centrum Zdrowia Dziecka, Warszawa, Poland
Hospital Materno Infatil de Badajoz, Badajoz, Spain
Hospital Universitari Vall d’Hebron de Barcelona, Barcelona, Spain
Hospital Materno Infantil de Málaga, Málaga, Spain
October 2014–March 2017 [88]
Safety and tolerance of immunomodulating therapy with donor-specific MSC in paediatric liver-donor liver transplantation (MYSTEP1) Bone marrow-derived MSCs Paediatric liver transplantation Two doses of 1 × 106 MSCs/kg body weight Number of participants with MYSTEP-score grade 3 and grade 2 (toxicity of MSC infusion), number of participants with occurrence of any severe adverse events, graft function after liver transplantation, number of participants with abnormal liver tests) Phase I University Children’s Hospital, Tubingen, Germany July 2013–Januray 2019 [89]
Therapeutic strategy and the role of mesenchymal stromal cells for ABO incompatible liver transplantation Mesenchymal stem cells Liver transplantation Six doses of 1 × 106/kg body weight MSCs are given, intravenously Efficacy 1-year graft survival rate Phase I The Third Affiliated Hospital, Sun Yat-Sen University, Guangzhou, Guangdong, China February 2014–March 2017 [90]
Human mesenchymal stem cell transfusion is safe and improves liver function in acute-on-chronic liver failure patients Umbilical cord mesenchymal stem cell (UC-MSC) Acute-on-chronic liver failure Conventional treatment and 0.5 × 106/kg body weight UC-MSCs are given, intravenously at baseline, 4 weeks, and 8 weeks
Conventional treatment and saline was used for the control group
Liver functionality tested over 48 weeks
72-week survival rate
Phase I and II Beijing 302 Hospital Beijing, Beijing, China March 2009–March 2014 [91]
Safety study of HepaStem for the treatment of urea cycle disorders (UCD) and Crigler-Najjar syndrome (CN) (HEP001) Human liver-derived mesenchymal stem cell (HepaStem) Urea cycle disorders
Crigler Najjar syndrome
HepaStem low dose 12.5 × 106/kg body weight
HepaStem intermediate dose 50 × 106/kg body weight
HepaStem high dose 200 × 106/kg body weight
Safety of HepaStem in paediatric patients suffering from urea cycle disorder and Crigler-Najjar syndrome Phase I and II Saint Luc University Hospital, Brussels, Belgium
Universitair Ziekenhuis (US) Antwerpen, Edegem Belgium
CHU Bicetre, Le Kremlin Bicȇtre Cedex, France
Hȏpital Jeanne de Flandre, CHRU Lille, Lille Cedex, France
Hȏpital des Enfants, CHU de Toulouse, Toulouse, Cedex 9, France
Rambam Medical Center, Meyer Children’s Hospital, Haifa, Israel
Hadassah Ein-Kerem Medical Center of Israel, Jerusalem, Israel
Schneider Children’s Medical Center of Israel, Petach Tikva, Israel
Ospedale Pediatrico Bambino Gesu di Roma, Roma, Italy
Birmingham Children’s Hospital London, London, United Kingdom
March 2012–April 2015 [92]
Macrophage therapy for liver cirrhosis Autologous macrophages Liver cirrhosis Autologous activated macrophages infusion via peripheral vein for 30 min.
Standard medical care used as the control
Liver function (MELD score) at 3 months Phase I and II Edinburgh Royal Infirmary Little France Crescent Edinburgh EH16 4SA United Kingdom August 2016–August 2021 [93]

Information from Clincialtrials.gov [80]