Mesenchymal stem cell therapy reduces fractures in brittle bone disease
Original story from Karolinska Institutet (Solna, Sweden).
A new study from Karolinska Institutet and Karolinska University Hospital shows that stem cell therapy can reduce fractures by up to 78% in children with the rare condition osteogenesis imperfecta (OI), also known as congenital brittle bone disease.
The BOOSTB4 study represents the world’s first clinical trial to combine both prenatal and postnatal stem cell therapy for OI. Two-year results were recently presented at the 15th International Conference of Osteogenesis Imperfecta in Hong Kong (29-31 October 2025), revealing remarkable outcomes: more than half of the treated children experienced no fractures at all during their second year following treatment. Some participants received treatment even before birth through in utero therapy.
“For families living with OI, every fracture is a major strain. Seeing that more than half of the treated children had no fractures at all during the second year is tremendously encouraging. It gives us the strength to continue developing the treatment,” says Cecilia Götherström, coordinator of the study and docent at the Department of Clinical Science, Intervention and Technology, Karolinska Institutet.
Joint effort behind the study
The study, which launched in 2020 and is now in its follow-up phase, was conducted at Karolinska University Hospital using cells produced at Vecura, a GMP-certified manufacturing facility for advanced therapy medicinal products. Vecura operates as a central component of the Karolinska ATMP Center, established in 2023 through a joint initiative between Karolinska Institutet and Karolinska University Hospital to coordinate research, production, and clinical application of advanced therapies.
Stem cells can change the course of the disease
The results of the BOOSTB4 study shows that treatment with mesenchymal stem cells, which have strong bone‑forming capacity, can not only reduce the number of fractures but also potentially alter the course of the disease. This makes the therapy a possible disease‑modifying treatment – something that has so far been entirely lacking for OI.
“BOOSTB4 is the result of a unique multidisciplinary European collaboration where research, clinical practice and advanced cell therapy come together. It demonstrates that when research and clinic work hand in hand, we can change the lives of our patients,” says Cecilia Götherström.
Osteogenesis imperfecta is a hereditary disease that leads to fragile bones, impaired growth and other serious symptoms. At present, there is no approved treatment that addresses the root cause of the disease and existing care focuses primarily on fracture prevention and mobility improvement, making this stem cell therapy breakthrough particularly significant for affected families.
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