A Pilot Trial Of Unmatched Human Placenta-Derived Stem Cells (Hpdscs) In Conjunction With Unrelated Cord Blood Transplantation (Ucbt) In Children And Young Adults With Malignant And Non-Malignant Disease (Ind 14949)

Background: Myeloablative conditioning (MAC) and reduced toxicity conditioning (RTC) followed by UCBT (matched at 4-6/6 HLA loci) in children with malignant and non-malignant diseases is safe and effective (Wagner/Cairo et al, Blood, 1996; Geyer/Cairo et al, BJH, 2011). However, there is a low concentration of CD34+ hematopoietic progenitor cells (HPCs) in banked UCB (Cairo/Kurtzberg et al, Transfusion, 2005) compared to bone marrow or peripheral blood, leading to delayed hematopoietic reconstitution and 10-20% incidence of graft failure (Cairo et al, Blood, 1997; Cairo et al, Transfusion, 2005; Satwani/Cairo et al, BBMT, 2013). Human placenta-derived stem cells (HPDSCs) are rich in HPCs and HPC colony forming unit capabilities, low in HLA Class I and II expression, low in T-cell content, and have regenerative, anti-inflammatory, and immunosuppressive properties (Cairo et al, BMT, 2015). Mendez-Ferrer et al have demonstrated that HPDSCs enhance in-vivo engraftment when combined with UCBT in NOD-SCID animals (Mendez-Ferrero et al, Nature, 2010).