OI is most commonly diagnosed on clinical evaluation followed by genetic testing of the patient. There is no cure for OI and the only current pharmaceutical treatments are palliative, do not completely eliminate fragility fractures or address the underlying bone brittleness and collagen defect.
Using Stem Cells to Improve Therapy
The BOOST2B project is focused on bringing the transplantation of fetal-derived mesenchymal stem cell (MSC) into the clinic as a therapy for Osteogenesis Imperfecta in toddlers. During the project we will investigate the safety and efficacy of this innovation in regenerative medicine in the treatment of OI in child patients between 1 and 4 years 11 months 29 days. Successful clinical demonstration of the improvement in growth and decreasing the fracture rate and severity of disease will result in life-long benefits for the affected individuals and their families.
In a parallel BOOSTB4 project ongoing in Europe the focus is on translating fetal-derived mesenchymal stem cell (MSC) transplantation into the clinic as a perinatal therapy for Osteogenesis Imperfecta. (BOOSTB4 https://www.boostb4.eu/)