Bone Marrow Banking Boosts Life-Saving Cell Therapy Development

Stem cells form the backbone of many cell-based therapies due to their unique properties, including the ability to self-renew and differentiate to many cell types. Multiple cell therapies deliver exogenous bone marrow stem cells into patients, which may trigger body repair machinery or regulate the endogenous stem cell population to achieve tissue homeostasis and regeneration. Cell-based therapies provide hope for patients suffering from many diseases, including immune system disorders, cancer, congenital thrombocytopenia, and sickle cell anemia, where conventional treatments fail to provide favorable outcomes.

In this Innovation Spotlight, Kevin Caldwell, chief executive officer, co-founder, and president of Ossium Health, discusses how Ossiums bone marrow banking platform offers a streamlined stem cell transplant process, enabling patients to undergo treatment much faster than previously possible.

Kevin Caldwell Chief Executive Officer, Co-founder, and President Ossium Health

What is the significance of bone marrow banking for therapeutics?

Bone marrow transplants, which are also referred to as stem cell transplants, have been used for over seven decades and are a curative treatment for an array of malignant and non-malignant blood and immune diseases. Over 9,000 patients receive a bone marrow transplant from a related or unrelated donor each year in the United States. While this procedure is highly effective and potentially lifesaving for patients battling blood cancers, the system for obtaining stem cells has historically relied on the generosity of unrelated living donors. This treatment option was therefore only available when a suitable and willing donor could be identified, and nearly 45 percent of patients that could benefit from a transplant were unable to receive one.

Bone marrow banking is a transformative solution that streamlines the stem cell transplant process, expanding patient access to bone marrow and improving treatment outcomes. Now, physicians have reliable and on-demand access to bone marrow stem cells that have been fully characterized and cryopreserved for long-term storage. This advancement increases donor accessibility, augmenting the conventional stem cell sources to increase patients likelihood of finding suitable matches and receiving lifesaving treatment. It also eliminates the lengthy wait and uncertainties that exist with relying on unrelated living donors, addressing one of the major critical gaps in current blood cancer treatment.

How did Ossium Health build their bank and what were some challenges that they overcame?

Although the potential of deceased donor bone marrow as a stem cell source was recognized long before Ossium came into existence, there were major barriers to entry that prevented other groups from turning this opportunity into a reality. Successfully building a bone marrow bank at the scale necessary to effectively serve patients required a team with an unlikely combination of skills. My co-founder, Erik Woods, and I assembled a small, gritty team with diverse skills that took on a wide range of challenges to get us where we are today.

Initially, building our bank required us to form critical partnerships with dozens of regional Organ Procurement Organizations to source vertebral bones from organ donors. We simultaneously developed and optimized a process for efficiently extracting and cryopreserving the bone marrow for long-term storage, built a controlled manufacturing facility, and established a robust quality system to ensure our manufacturing processes met the highest standards. We also had to raise the funds to accomplish all of this. Overcoming these challenges demanded a multidisciplinary approach, innovation at each step, and the strength to pick ourselves back up after setbacks.

How are the cells from the bone marrow bank used and what types of patients could benefit the most from this strategy?

Today, Ossiums bone marrow can benefit patients suffering from any life-threatening blood and immune diseases that require an allogeneic stem cell transplant. Several blood cancer patients have successfully received transplants through our ongoing clinical study and expanded access program, and we expect to treat hundreds more over the next few years.

Banked bone marrow also holds immense potential as a medical countermeasure. In the event of a nuclear attack on a major US city, the large number of victims suffering from acute radiation syndrome of the hematopoietic system would overwhelm the living donor system. If the need arises, bone marrow available on demand will circumvent logistical hurdles and save thousands of lives.

Looking forward, we envision far broader applications for stem cell transplants, with banked bone marrow serving as a primary stem cell source. Case studies of stem cell transplants in patients with multiple co-morbidities have demonstrated that this procedure can cure a wide range of diseases. As scientific advancements make conditioning and treatment regimens safer, this procedure will be used in a far wider range of indications, including those that are not immediately life threatening. Physicians performing hundreds of stem cell transplants per year will prefer the seamless logistics of sourcing high-quality, fully-characterized cells that are available immediately from a bone marrow bank.

What has Ossium Health learned so far from their ongoing clinical study?

Were currently conducting the PRESERVE I clinical study (NCT05589896) at transplant centers across the US. Through this study, blood cancer patients receive transplants with bone marrow stem cells from our bank. Over the course of conducting this study, weve gained valuable insights into the unmet needs of patients requiring bone marrow transplants. In multiple cases, physicians approached us with patients who urgently needed a transplant but could not participate in the study for various reasons, such as an inability to travel to an active clinical site or not meeting study eligibility criteria. In response, we developed an expanded access program to offer this life-saving treatment to patients in desperate need. We also adjusted the studys protocol to accommodate a larger and more diverse patient population, including those with a wider range of indications. As we address these immediate issues, weve also deepened our confidence in the need for this solution, strengthening our will to push forward toward our vision.

What are the benefits of this cell therapy compared to other conventional strategies for leukemia treatment?

Our approach offers numerous advantages, including higher doses, on-demand availability, and lower T cell counts.

Ossium Health developed and optimized a process for effectively extracting and cryopreserving the bone marrow for long-term storage.

Behdad Pournader

One of the primary benefits is the availability of higher stem cell doses, enabling physicians to select an optimal dose tailored to the patient. This is particularly beneficial for larger patients who often struggle to find a donor capable of providing an adequate dose. Adequate dosing is essential and has been directly linked to improved overall survival rates.

Additionally, our bank gives physicians the ability to obtain stem cells at unprecedented speeds, which allows transplants to be performed far earlier than previously possible. Lengthy searches for a living donor often cause major delays in treatment. During these months of waiting for a donor, a patients condition could decline so far as to make them ineligible for a transplant. Banked bone marrow that can be shipped almost immediately to a transplant center eliminates this issue.

Another key advantage is that the product naturally has lower levels of T cells than fresh peripheral blood stem cell grafts, reducing the risk of graft-versus-host disease (GVHD), a severe and potentially fatal complication of bone marrow transplantation.

What are the potential future opportunities for bone marrow-derived cell therapies at an industrial scale?

The future of bone marrow-derived cell therapies holds immense promise, with Ossium Health poised to play a pivotal role in scaling these treatments to meet growing demands.

One of the most exciting opportunities is the availability of allogeneic bone marrow-derived mesenchymal stem cell (MSC) therapies. MSCs have shown tremendous promise in both treatment and prevention for a range of diseases, including GVHD, but batch-to-batch inconsistencies have cast doubts around the benefits. Far more MSCs can be obtained from a single deceased donors vertebral bodies compared to living donor sources, allowing more doses per donor and reducing the variability between manufactured lots.

Another potential use of banked bone marrow is starting material for off-the-shelf allogeneic CAR T cell therapies. Autologous CAR T therapies have shown promise in select malignancies, but these products are difficult and expensive to manufacture, limiting their potential for broad use. Banked bone marrow could act as the foundation for future allogeneic CAR T therapies and other innovative cell therapies, which could make these innovative medicines far more accessible and cost effective.

Additionally, Ossium envisions a future where individuals concerned with preserving their health and vitality can bank their own bone marrow for potential autologous cell therapies. These therapies could include the full rejuvenation of their blood and immune systems, marking a new era in personalized medicine and preventive care.

With such vast potential, the industrial-scale application of bone marrow banking is set to transform healthcare on multiple fronts.

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Bone Marrow Banking Boosts Life-Saving Cell Therapy Development