Expanding access to bone marrow and cellular therapies for children with life‑threatening diseases
Official title: BMT Core - Pediatric Transplantation and Cellular Therapy Consortium (PTCTC): Providing Clinical Trial Access For Children With Life Threatening Diseases
Funded at National Marrow Donor Program · NIH-11170039 · Data updated 2026-11-12
This program helps children with serious blood and genetic conditions get personalized bone marrow and cellular therapies through a network of transplant centers and trials.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | National Marrow Donor Program NIH-funded |
| Lab location | 1 site (Minneapolis, United States) |
| Project start | August 15, 2024 |
| Project end | June 30, 2031 |
| Funding year | 2025 |
| Award (this year) | $164,548 |
| NIH project number | 5UG1HL174426-02 |
| Project ID | NIH-11170039 on NIH RePORTER |
Who could benefit from this research
✓Could help
Children (pediatric patients) with life‑threatening malignant or non‑malignant blood or genetic disorders who are candidates for allogeneic hematopoietic cell transplantation are the primary candidates.
✕Less likely to help
Patients who are not candidates for allogeneic transplant (including many adults, those treated without transplantation, or those medically ineligible) are unlikely to benefit directly from this program.
What this research studies
If your child has a serious blood or inherited genetic condition, this consortium connects families with pediatric transplant centers offering allogeneic bone marrow and cellular therapies and clinical trial options. The program focuses on personalizing pre-transplant chemotherapy because children metabolize these drugs differently, which can cause too much toxicity or too little effect with standard dosing. It collects clinical data and biological samples across centers to guide safer, individualized dosing and care. The consortium also coordinates access to trials and standardizes care across participating sites to improve outcomes.
Why it matters
Potential benefit: If successful, this work could lower transplant-related toxicity and improve cure rates by making chemotherapy dosing and trial access more personalized and widely available for children.
How similar studies have gone: Multicenter transplant consortia and efforts at individualized dosing have improved outcomes in some pediatric transplant settings, but tailoring chemotherapy exposure remains an active area of research.
Where this research is happening
-
National Marrow Donor Program
Minneapolis, United States Active
Researchers
- Principal investigator: Stefanski, Heather E — National Marrow Donor Program
- Study coordinator: Stefanski, Heather E
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.