Evidence So Far
More than 15 years ago, a trial was performed in the USA where six children were treated after birth with two infusions of adult mesenchymal stem cells. One treatable allergic reaction (hives) was observed after one of the 12 infusions. No other side effects were noted. The children did well with improved growth and reduced number of fractures. Researchers within the BOOSTB4 consortium have previously given fetal mesenchymal stem cells to four children with OI. The children received an infusion of the stem cells after they were born or as an unborn baby while they were in the womb. The children have received further postnatal booster doses of the same stem cells. Clinically, they did well after the stem cell infusions, growing better and having fewer fractures and less pain than would have been expected. Looking through a microscope at bone samples, we could see that the stem cells had engrafted (settled) into the bone and become bone producing cells. By 2018 the treated children were between 3 and 16 years old. No side effects have been detected so far. However, the number of children treated so far in the world is only ten.
Studies in animals suggest that stem cells given early in life reduce the number of fractures and the bones become stronger. We therefore believe that an early intervention could lead to better results.
1. Le Blanc K, Gotherstrom C, Ringden O, Hassan M, McMahon R, Horwitz E, et al. Fetal mesenchymal stem-cell engraftment in bone after in utero transplantation in a patient with severe osteogenesis imperfecta. Transplantation. 2005;79(11):1607-14.
2. Gotherstrom C, Westgren M, Shaw SW, Astrom E, Biswas A, Byers PH, et al. Pre- and postnatal transplantation of fetal mesenchymal stem cells in osteogenesis imperfecta: a two-center experience. Stem cells translational medicine. 2014;3(2):255-64.