Physicians at Riley Children’s Health at Indiana University (IU) Health have performed the first pediatric bone marrow transplant using Ossium’s off-the-shelf bone marrow. This milestone extends a treatment approach that has shown encouraging results in adults and is a meaningful step in expanding donor options for children.

The 14-year-old patient was diagnosed with acute myeloid leukemia (AML) in August 2025. In January 2026, he received a first transplant using living donor stem cells, but the cells failed to engraft, meaning they did not establish themselves in his bone marrow and begin producing healthy blood cells. “When the first transplant failed and there were no remaining donor matches, we were facing a race against time,” said Dr. Jodi Skiles, medical director of Riley’s pediatric stem cell transplant program and associate professor of clinical pediatrics at Indiana University School of Medicine. Without a new donor quickly, the patient’s prognosis was grave.

Dr. Skiles turned to Ossium’s bank of cryopreserved bone marrow recovered from organ donors. The marrow is manufactured, cryopreserved, and held in a banked inventory, making it available for on-demand shipment within days rather than the months typically required to search for and secure a living unrelated donor graft. Ossium’s process yields more cells per donor than can be practically obtained from a living donor, allowing physicians to select an optimal cell dose. Together, immediate availability and high cell dose make the platform particularly relevant in urgent scenarios such as graft failure, donor attrition, and rapidly progressing disease.

“Because these stem cells are banked and ready to use, we were able to act in days instead of months,” Dr. Skiles said. “This treatment has the potential to dramatically expand access to transplant for children who previously had few or no remaining treatment options, giving us a way to close critical gaps in care and offer hope to families facing impossible circumstances.”

The patient received a transplant with organ donor bone marrow in March 2026 through Ossium’s HOPE Program. The cells engrafted within weeks, and the patient is now in remission.

“His story is a huge advancement for science,” Dr. Skiles said. “The fact that he survived and is thriving and got to be outpatient relatively quickly is shocking.”

Extending that capability to pediatric patients carries particular weight. For children with aggressive blood cancers, an off-the-shelf, ready-when-needed graft source offers a path to transplant that did not previously exist. Pediatric transplant cases have historically been among the most time-sensitive because pediatric disease often progresses fast, and a failed or delayed transplant can foreclose options within weeks. A graft that is characterized, banked, and ready to ship directly addresses that timeline.

The need extends well beyond leukemia. Each year, approximately 4,000 children in the US are diagnosed with blood diseases that could be successfully treated with an allogeneic bone marrow transplant, yet nearly half never receive one. In children, allogeneic transplant, which uses healthy blood-forming cells from a donor, is often the only curative option for a range of malignant diseases like acute lymphoblastic and myeloid leukemias, but also non-malignant conditions including sickle cell disease, transfusion-dependent thalassemia, severe aplastic anemia, inherited bone marrow failure syndromes such as Fanconi anemia, severe combined immunodeficiency and other primary immunodeficiencies, and certain inborn errors of metabolism. Transplant is curative for many of these diseases when a suitable donor can be found in time, yet donor access remains limited. Only about a quarter of patients have a matched sibling donor, and those who must rely on unrelated living donor registries face delays and uncertainty. A centralized bank with grafts available on demand adds donor options and removes the procurement delay that can be decisive for a young patient with rapidly progressing disease.

“Treating our first pediatric patient is a defining moment for Ossium,” said Kevin Caldwell, Ossium’s CEO, Co-Founder and President. “No child should run out of time waiting for a donor, and this milestone moves us closer to a world where none has to.”

This case also reinforces what the broader clinical experience has shown: organ donor bone marrow can engraft reliably and perform across a range of urgent and routine scenarios. This pediatric case was the 21st transplant performed with Ossium’s organ donor bone marrow, a total that has since grown to 28 patients, all of whom successfully engrafted. Treating the first pediatric patient turns possibility into precedent, laying the foundation for many more children to receive this life-saving therapy.

See more on this patient’s journey and what this advancement means for the field:

• Riley Children’s Health: Deceased donor stem cells save teen’s life
• Indiana Public Media: Bloomington resident is first pediatric patient to receive successful stem cell transplant from deceased donor
• WISH-TV: Indiana teen becomes first pediatric patient to receive bone marrow transplant from deceased donor
• ABC57: IU Health completes world’s first successful pediatric bone marrow stem cell transplant using deceased donor
• WBIW: Riley Children’s Health performs world’s first successful pediatric bone marrow transplant using deceased donor stem cells