Why are we investigating stem cell therapy for OI?
Stem cells are undifferentiated cells that are able to develop into specialized cell types, or divide to make more stem cells. We hope that by giving unaffected stem cells early in life to a baby with OI, they will be able to help make normal bone and reduce the bone brittleness. Studies in animal models of OI suggest that stem cells help to reduce the number of fractures and help the bones become stronger.
Researchers within the BOOSTB4 consortium have previously given stem cells to children with OI. The children received an infusion of the stem cells either whilst they were in the womb, and/or after they were born. Clinically, they did well after the stem cell infusions, growing better and having fewer fractures and less pain than would have been expected. By 2017 the treated children were between 1 and 15 years old. No side effects have been detected so far. However, the number of babies treated so far in the world is fewer than ten.
The BOOSTB4 clinical trial has therefore been set up to investigate stem cell therapy as a prenatal treatment for OI. The main thing we want to confirm in this trial is whether the stem cell infusion is safe to use in pregnant women and their babies with OI. We also want to see if the stem cell infusion improves the health of children with OI. Specifically, we want to investigate if the number of fractures and chronic pain can be reduced and growth can be improved. If this study is successful we aim to develop the stem cell infusion as a future clinical treatment for OI.
You can read about one of the children who received stem cells before birth in this newspaper article:
Journal article about two of the previous patients given stem cells in the womb: