USC and Children’s Hospital Los Angeles to investigate a potential cellular therapy for treatment of cleft lip and palate

Cleft lip and/or palate (CLP) is the most common craniofacial birth defect,¹ frequently presenting with an alveolar cleft in the bone of the gum line.² The most common method of alveolar cleft repair involves autogenous iliac crest bone grafts, which comes with challenges of multiple surgeries and the invasive nature of obtaining graft material.² Mesenchymal stem cells (MSCs) from umbilical cord tissue may be a promising alternative as a potential graft source based on their capacity for self-renewal, multipotent differentiation and proliferation, non-invasive collection, and lack of ethical issues.³

To determine whether MSCs may serve as an effective graft source for alveolar cleft repair, researchers and physicians at the University of Southern California and Children’s Hospital Los Angeles hope to investigate whether the use of autologous MSCs from cord tissue may be an effective source of cells for alveolar repair in children with CLP. CBR's Newborn Possibilities Program offers no-cost processing and storage of newborn stem cells, including umbilical cord tissue, from children with qualifying conditions including CLP. Families referred to the Division of Plastic and Maxillofacial Surgery at Children’s Hospital Los Angeles based on the identification of CLP on prenatal ultrasound are invited to apply for CBR’s Newborn Possibilities Program® prior to delivery. Newborn stem cells from these children are collected at birth and cryopreserved for potential future use in alveolar cleft repair surgeries when patients reach 7-8 years of age, the age at which alveolar cleft repair surgeries typically take place.²

In a previous collaborative effort between these researchers and CBR’s Research and Development team, a mouse model was used to assess the effectiveness of MSCs from umbilical cord tissue in regenerating bone for alveolar cleft repair.⁴ Umbilical cord tissue was collected, prepared, and implanted into the mouse model to assess the regenerative capabilities of cord tissue MSCs. Relative to controls, histological and radiographic analyses confirmed significant bone regeneration in mice treated with cord tissue MSCs on a polylactic-co-glycolic acid (PLGA) scaffold.⁴ This model demonstrated successful bone fill of calvarial defects in mice, providing preliminary evidence that the use of this MSC graft adjunct may provide some benefit in alveolar cleft repair for patients with CLP, but more studies are needed.  

We continue to enroll and follow patients with CLP in the CBR Newborn Possibilities Program, providing no-cost storage of newborn cells for use in later research and creating a pool of potential candidates for future cleft repair using autologous MSCs. We have demonstrated the isolation and characterization of MSCs from composite cord tissue after long-term cryopreservation⁵ and look forward to the potential for use of these stem cells in reconstructive surgeries.

This research plan was presented as a poster this fall at the 2024 Cord Blood Connect meeting in Miami, Florida. To learn more about the Newborn Possibilities Program including who might qualify outside of this condition, visit our family programs page.