New study aims to expand access to stem cell transplant using deceased donor bone marrow
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Bioengineering company Ossium Health, which is developing the first bank of bone marrow collected from organ donors, has partnered with the National Marrow Donor Program/Be The Match to increase access to lifesaving bone marrow.
The collaborative effort is aimed at expediting the industry’s ability to make deceased donor bone marrow transplants available at scale. The two companies have initiated a clinical trial to assess the safety and efficacy of Ossium’s bone marrow product. The trial, PRESERVE I, is a first-in-human study of deceased donor marrow in patients with hematologic malignancies.
“We have more than 35 years of experience managing this diverse registry of potential unrelated blood stem cell donors and cord blood units, and we successfully match over 7,000 patients per year with volunteer donors,” Erin Leckrone, MBA, vice president of clinical trials at National Marrow Donor Program (NMDP)/Be The Match, told Healio. “Yet, despite all of that, up to 45% of patients who need a transplant never receive one, primarily because they cannot identify a suitable donor.”
Leckrone and Kevin Caldwell, JD, CEO, co-founder and president of Ossium Health, spoke with Healio about the partnership, the PRESERVE I trial, and their plans to make Ossium’s bone marrow bank available to patients as soon as possible.
Healio: What inspired this partnership?
Leckrone: We are dedicated to partnering with organizations like Ossium, who have innovative products that could potentially fill the unmet needs in transplantation. There are areas of research right now within the mismatched, unrelated donor realm that are showing promise when combined with post-transplant drugs to reduce graft-versus-host disease. This partnership with Ossium is one area we’re very excited about.
Caldwell: For some of those patients, the problem is not that there isn’t anyone in the world or even in any registry who could theoretically be a donor — it’s that they are unable to get bone marrow from a donor recovered in time for them to receive a transplant. The basic idea behind Ossium is to develop a product in advance that’s available off the shelf to deliver to the patient.
To accomplish this, we need a bank of bone marrow readily available at scale, with lots of samples from a very wide variety of donors. The ideal source for that is the organ donor population. The U.S. does about 40% of all global deceased donor organ transplants — there are about 15,000 donors per year. Those donors enable about 45,000 organ transplants per year. Ossium’s initial innovation was to develop a process for obtaining bone marrow from organ donors, process it, cryopreserve it at clinical grade, test it, and make those units available for immediate deployment and infusion. This dramatically reduces wait time and enables many patients to receive a transplant who otherwise would not.
Healio: What will the PRESERVE I trial evaluate?
Caldwell: We call this study PRESERVE I for two reasons. Our goal is to preserve human life, and we use cryopreserved bone marrow to do that. This study is a first-in-human, 12-patient study using our hematopoietic progenitor cell marrow for patients with hematologic malignancies. It has a few special characteristics that are important to frame. Today, the FDA does not regulate bone marrow transplants for patients with blood cancer. In fact, the FDA doesn’t regulate transplantation at all, whether it’s a bone marrow transplant, a kidney transplant or a heart transplant.
When we first started out 7 and a half years ago, one of the things we did was to reach out to the FDA for guidance on how our product would be regulated. What the FDA ultimately decided was that Ossium’s product is not sufficiently different from living donor bone marrow that it should be treated differently from a regulatory perspective. They concluded that it was equivalent, and so this study isn’t under an IND application like we would see for a traditional drug. It’s a study that is designed to establish a dataset that can show safety, efficacy and broad comparability of our product to living donor bone marrow.
Leckrone: The phase 1 study that we are collaborating on is looking at safety and feasibility, and the phase 2 study will look at safety and efficacy. PRESERVE I will primarily be conducted for individuals diagnosed with acute myeloid leukemia and acute lymphoblastic leukemia between the ages of 18 and 55. Some of the endpoints we’re looking at beyond safety and feasibility are neutrophil engraftment by day 28 and safety events within 56 days of infusion.
Healio: If the results of the trial are favorable, how will these products be incorporated into NMDP/Be The Match’s registry?
Leckrone: We’re waiting to see the results of the clinical trial, and then we’ll be tackling that between our two organizations. We’re excited to operationalize and integrate Ossium’s registry with NMDP/Be The Match so that there is a seamless interaction for transplant centers. That’s something that will be coming once we collect additional datasets for the efficacy of the product.
Healio: What do you hope will be the long-term implications of your study?
Leckrone: If the clinical trial results are favorable, our next steps would be to determine how to operationalize this for the transplant center. There are other benefits beyond what we were saying about the additional selection of cryopreserved product to meet that unmatched need. So, it’s not only an additional option for a patient who might not have a fully matched donor available, but the Ossium product could also potentially meet other unmet needs.
Caldwell: There are three key advantages or benefits we see for patients in the long term, even patients who might otherwise have another source of bone marrow available. The first is one we’ve already alluded to, which is time. In general, the quicker patients receive a transplant, the better their long-term overall survival outcomes are. The second is optimized dosing. Because we’re able to recover far more bone marrow from an organ donor than you can ethically take from a living person who still needs their cells, the physician is in the unique position of being able to choose the exact dose they want for their patient based on their patient’s weight and other factors. The third benefit relates to a principal complication of bone marrow transplants — graft-versus-host disease. Ossium’s bone marrow has about 70% fewer naive T cells per CD34 than traditional bone marrow sources. We anticipate that this will result in a lower risk for GVHD for those patients.
Healio: Is there anything else you’d like to mention?
Leckrone: There are disparities in accessibility to transplant that really impact ethnically diverse patients, and this is an area where NMDP/Be The Match has already committed significant research efforts. Our partnership is a commitment to further closing that disparities gap. We do see — particularly in terms of mismatched, unrelated donor transplants — an opportunity for Ossium’s bone marrow product could make a key difference in the ability of ethnically diverse patients to get to transplant.
Caldwell: We are incredibly excited about the PRESERVE I study and about our partnership with NMDP/Be The Match. We see this as a foundation for a broad cell therapy platform. What we’ve built is a first-of-its kind large source of cryopreserved bone marrow that can be used as a basis for developing therapeutics that further engineer the cells and tailor them to specific indications in rare diseases.
Leckrone: We are also excited to capture this data and examine this through the clinical trial process, because it really establishes the precedent of providing transplant centers with data to guide their decision-making process. This is not based on subjective information, but on the data that is statistically relevant for this space. Both of our organizations are committed to providing data-driven options to transplant centers.
Reference:
- Ossium Health and Be The Match unveil partnership to increase bone marrow transplant accessibility and treatment efficacy (press release). Available at: https://ossiumhealth.com/ossium-health-and-be-the-match-unveil-partnership-to-increase-bone-marrow-transplant-accessibility-and-treatment-efficiency/. Posted Aug. 31, 2023. Accessed Nov. 16, 2023.
For more information:
Kevin Caldwell, JD, can be reached at Ossium Health, 80 Tehama St., San Francisco, CA 94105; email: info@ossiumhealth.com.
Erin Leckrone, MBA, can be reached at 500 N. 5th St., Minneapolis, MN 55401; email: eleckron@nmdp.org.
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