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New Study Suggests Placental Stem Cell Therapy for Inherited Skin Blistering Disease

Review of “Efficacy of Human Placental Derived Stem Cells in Collagen VII Knockout (Recessive Dystrophic Epidermolysis Bullosa) Animal Model” from STEM CELLS Translational Medicine by Stuart P. Atkinson

Mutations in the COL7A1 gene, which encodes type VII collagen (C7), lead to the disruption of the dermal-epidermal junction (DEJ) and the development of the inherited skin blistering disease severe recessive dystrophic epidermolysis bullosa (RDEB) (2). The associated high risk of developing aggressive cutaneous squamous cell carcinomas and a lack of effective treatments has motivated the search for new cell-based therapeutic approaches. Preclinical and clinical studies demonstrated the feasibility of allogeneic hematopoietic progenitor cell (HPC) transplantation following myeloablative chemotherapy [1, 2], although this therapeutic option suffers from significant complications.

Treatment with human placental‐derived stem cells (HPDSCs), a combination of hematopoietic stem cells (HSCs), megakaryocytic precursors, and non-hematopoietic stem cells depleted of red blood cells, non-viable cells, and tissue debris [3] has recently emerged as a potentially exciting alternative treatment option. Now, new research from the laboratory of Mitchell S. Cairo (New York Medical College, Valhalla, New York, USA) reports that HPDSCs can improve overall survival and functional outcomes in a col7a1−/− mouse model of RDEB without the need for preconditioning or immunosuppression [4].

Liao et al. assessed the consequences of intrahepatic administrations of HPDSCs, discovering that a single treatment elevated the median lifespan of model mice from two to seven days, while an additional treatment significantly extended the median lifespan to eighteen days. The authors discovered that HPDSCs engrafted in the skin and gastrointestinal tract (organs affected by RDEB) following intrahepatic administration, increased DEJ adhesion, and deposited C7 protein for up to four months post-administration without raising an anti-C7 immune response.

Overall, HPDSCs appear to display potent regenerative activity in this animal model of RDEB, which, when combined with the observed safety of HPDSC therapy in humans with malignant and non-malignant diseases [3], promotes future clinical investigation in patients with severe RDEB. Indeed, the authors finally report that their findings have served as the basis for a translational pilot phase I trial to investigate the safety and feasibility of administration of HPDSC alone or in combination with UCB transplantation in patients with RDEB.

For more on the future of HPDSC therapy for inherited skin blistering diseases and more, stay tuned to the Stem Cells Portal!


  1. Tolar J, Ishida-Yamamoto A, Riddle M, et al., Amelioration of epidermolysis bullosa by transfer of wild-type bone marrow cells. Blood 2009;113:1167-1174.
  2. Wagner JE, Ishida-Yamamoto A, McGrath JA, et al., Bone Marrow Transplantation for Recessive Dystrophic Epidermolysis Bullosa. New England Journal of Medicine 2010;363:629-639.
  3. Cairo MS, Tarek N, Lee DA, et al., Cellular engineering and therapy in combination with cord blood allografting in pediatric recipients. Bone Marrow Transplantation 2015;51:27.
  4. Liao Y, Ivanova L, Sivalenka R, et al., Efficacy of Human Placental-Derived Stem Cells in Collagen VII Knockout (Recessive Dystrophic Epidermolysis Bullosa) Animal Model. STEM CELLS Translational Medicine 2018;7:530-542.