Building Bone: Harnessing Fetal Mesenchymal Stem Cells to Treat Osteogenesis Imperfecta.

Our aim is to harness the full potential of our recently developed fetal mesenchymal stem cell (fMSC) therapy.

We are currently about to change the lives of people living with Osteogenesis Imperfecta (OI), by the demonstrated 76% reduction in fracture frequency in 18 children with severe OI in the VR-supported BOOSTB4 trial.

OI is a devastating congenital disease, resulting in hundreds of fractures, some already before birth, deformed long bones, short stature and an chronic pain.The unmet medical need is striking, and we are determined to pursue our quest to take this very promising therapy to the patients.

For this to materialize some topics must be addressed, including 1) identifying the true bone-forming stem cell in the starting material, 2) investigate cell stress and interactions between fMSCs and OI-osteoblasts and, 3) evaluating efficacy of the enriched fMSCs in vitro and in an OI zebrafish model.

The project will be performed at Karolinska Institutet over 3-years.The benefits of this novel stem cell treatment are vast; it is the only treatment being developed for OI that addresses the underlying molecular defect (it supplements healthy collagen and bone), it does not require pre- or post-conditioning and treatment can be started before or at birth.

In conclusion, we have performed a successful clinical trial with impressive results and need to fill the gaps as described in this application before making this a therapy helping children with OI to get better lives.