Bone marrow transplants are used to replace damaged blood cells with healthy ones in patients with blood-related conditions. Credit: Terelyuk via Shutterstock.

US bioengineering company Ossium Health has treated its first leukaemia patient with cryopreserved organ donor-derived bone marrow as part of its HOPE clinical programme.

Ossium’s programme offers cryopreserved bone marrow sourced from deceased organ donors. Bone marrow transplants replace damaged blood cells with healthy ones in patients suffering from blood-related conditions such as leukaemia and lymphoma. Typically, transplants involve collecting healthy stem cells from the blood or bone marrow of a matching living donor, often a close relative.

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Speaking to Pharmaceutical Technology, Ossium’s CEO Kevin Caldwell explained that the bone marrow is cryopreserved and available for immediate shipment on-demand, eliminating the time necessary to conduct a search and secure a graft from a living donor. A faster transplant is associated with improved survival, especially for patients with high-risk malignant disease.

“Every year, 18,000 patients in the US are diagnosed with life-threatening illnesses where a bone marrow transplant is their best treatment option, but nearly 9,000 of these patients are unable to receive a transplant. Ossium’s bone marrow complements current bone marrow sources, improving donor accessibility for underserved patients,” said Caldwell.

The first patient was 68 years old with a diagnosis of acute myeloid leukaemia and received the infusion at Henry Ford Health in Detroit, Michigan. The doctors observed neutrophil engraftment by day 16 and platelet engraftment by day 21 post-transplant, with no product-related adverse events reported so far. 

Ossium’s bank could also improve accessibility to bone marrow transplants for patients that have the lowest matching rates through the living donor registry such as racial and ethnic minority groups.  

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“Our bone marrow bank will also serve patients globally, including those in underserved countries that lack a living donor bone marrow registry. Since the US is a nation of immigrants from all over the world, the racial and ethnic makeup of our US-based bone marrow bank is uniquely positioned to supply the rest of the world,” added Caldwell. 

Apart from the HOPE programme, the San Francisco-headquartered company is running the PRESERVE I (NCT05589896) study, a Phase I/II trial to evaluate the safety and effectiveness of its organ donor-derived bone marrow in patients with haematopoietic malignancies. The HOPE programme allows access to the stem cells without needing to enrol in the PRESERVE I trial.  

“This platform will revolutionise transplants, making them dramatically more accessible to underserved populations around the world,” concluded Caldwell.  

Note: This article has been updated to clarify the patient received the cryopreserved organ donor-derived bone marrow as part of the HOPE programme.