Boost to Brittle Bones (BOOST2B)
|Coordinator||Karolinska Institutet - CLINTEC Karolinska Institutet|
|Funding from Vinnova||SEK 4 550 000|
|Project duration||May 2017 - June 2022|
Purpose and goal
Karolinska Institutet (KI) and XNK Therapeutics (XNK) in Sweden and Christian Medical College (CMC) in India are jointly evaluating whether mesenchymal stem cells (MSC) can be developed into a treatment of Osteogenesis Imperfecta (OI). KI manufactures the investigational drug which is exported to CMC in India who conducts the clinical trial. Prior to the clinical trial, transfer of knowledge and technologies to the clinical site in India has been carried out. This transfer of knowledge is unique and will lead to the development of better advanced cell therapies in the future.
Expected results and effects
The collaboration between KI, XNK and CMC and the clinical trial conducted in India is successful and has resulted in valuable learnings and knowledge. Three children with severe OI have been treated according to the protocol and have received 4 doses of MSC intravenously and 4 doses of MSC injected into the bones. No severe side effects have been detected. Follow-up is ongoing, and the data shows a preliminary positive effect on fracture frequency, mobility and quality of life. This successful outcome will enable more children with OI to be treated in the future.
Planned approach and implementation
The pandemic has meant great difficulties especially for hospitals, which have had to change their activities, lock-down of the society, as well as patients not being able to travel to the hospital, but also for manufacturing and access to materials and reagents. After completed risk assessments, manufacturing of the investigational drug has been able to continue, and three children have received all 4 doses of stem cells. We have also clarified requirements to enable the release of additional investigational drugs and identified tools to improve quality analyses.