Using donor stem cells to strengthen bones in children with brittle bone disease
Cellular Therapy for Osteogenesis Imperfecta
['FUNDING_R01'] · UNIVERSITY OF CONNECTICUT SCH OF MED/DNT · NIH-11326776
Researchers are trying direct bone injections of donor stem cells to help infants and young children with osteogenesis imperfecta make normal collagen and build stronger bones.
Quick facts
| Phase | ['FUNDING_R01'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | UNIVERSITY OF CONNECTICUT SCH OF MED/DNT (nih funded) |
| Locations | 1 site (FARMINGTON, UNITED STATES) |
| Trial ID | NIH-11326776 on ClinicalTrials.gov |
What this research studies
If your child enrolls, they may receive low-dose whole-body irradiation followed by injections of donor mesenchymal stem/progenitor cells directly into affected bones. Doctors will look for donor cells settling into the bone, turning into bone-making cells, and producing normal type I collagen using imaging, lab tests, and mechanical measurements of bone strength. This approach was developed because past whole-body infusions had limited success, so targeting individual bones plus controlled irradiation may improve long-term engraftment. The team also plans lab studies to understand how irradiation helps the donor cells take hold so they can design safer, lasting treatments.
Who could benefit from this research
Good fit: Children from infancy through about 11 years old diagnosed with osteogenesis imperfecta, especially those with frequent fractures or needing corrective bone surgery, would be the ideal candidates.
Not a fit: People without osteogenesis imperfecta, those older than the trial age range, or those who cannot safely undergo the required irradiation or transplant procedures would likely not benefit from this approach.
Why it matters
Potential benefit: If successful, this could let affected bones make normal collagen, increase bone mass and strength, and improve outcomes of surgeries on transplanted bones.
How similar studies have performed: Previous systemic stem-cell transplants for OI showed limited results, but earlier work using direct intra-bone cell delivery with irradiation produced donor cell engraftment and improved bone mass in preclinical and early clinical reports, so this builds on promising but still early evidence.
Where this research is happening
FARMINGTON, UNITED STATES
- UNIVERSITY OF CONNECTICUT SCH OF MED/DNT — FARMINGTON, UNITED STATES (ACTIVE)
Researchers
- Principal investigator: KALAJZIC, IVO — UNIVERSITY OF CONNECTICUT SCH OF MED/DNT
- Study coordinator: KALAJZIC, IVO
About this research
- This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
- Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
- For full project details, budget, and progress reports, visit the official NIH RePORTER page below.