Blood Donor Day, an annual event on June 14, was organised for the first time in 2004 by four core international organisations: the World Health Organization, the International Federation of Red Cross and Red Crescent Societies; the International Federation of Blood Donor Organizations (IFBDO) and the International Society of Blood Transfusion (ISBT) to raise awareness of the need for safe blood and blood products, and to thank blood donors for their voluntary, life-saving gifts of blood. In acknowledgement of Blood Donor Day 2023, DDW spoke with industry experts about the significance of blood donorship in drug discovery.
Bill Block, President & CEO of Blood Centers of America
Bill Block is President and CEO of Blood Centers of America (BCA). BCA is the largest blood supply network in the US, uniquely positioned to sustain, mobilise and advocate for the nation’s blood supply. Block said: “Our 60+ independent community blood centers collect and distribute more than 5.4 million blood units annually, or 50% of the nation’s blood supply. BCA members also operate the BCA Advanced Therapies Network which is a diverse, fully integrated portfolio of blood, cell, gene and tissue materials that support the development and delivery of advanced therapies. Our network brings unparalleled and coordinated access to a vast array of members and clients with a variety of service offerings related to starting material, testing, quality control, manufacturing and clinical applications.”
When it comes to the importance of blood donorship and blood products for the development of new therapies, Block says that they are critical. He said: “A great example of this is the collection of Covid convalescent plasma (CCP) during the pandemic. BCA members and affiliates ramped up to collect over 670K doses which were used to treat patients before other therapies were available. We could not have done that without blood donors.”
He continues to say that for advanced biotherapies like CAR-T cell therapy products curing cancers, BCA centers are providing cell collections, testing, and processing to assist in the production and supply of these treatments to patients. He said: “Some of these treatments start with a patient collection and others start with donor material, while BCA sites provide donor recruitment and collection services. Either way, the BCA Advanced Therapies Network is engaged with therapeutic developers, hospitals, patients and donors to ensure these life-saving products are available to patients in our communities. In addition to the collection, processing and storing of adult blood projects, BCA is also involved in providing umbilical cord blood and other tissue products as critical components of cell therapies. BCA also provides laeukopaks for use in research and development, as well as cell-based reagents used in biotherapies manufacturing.”
BCA member testing laboratories have specialised diagnostic labs and have the regulatory processes in place to support clinical trials.
According to Block, blood and platelets are needed every two seconds in the US for emergencies such as burn and accident victims, surgeries, organ transplants, mothers during childbirth and patients receiving treatments for cancer. He said: “This need for blood is constant but currently only 3% of the population donates each year1. One of the biggest challenges facing the sustainability of our nation’s blood supply over the last decade is the loss of a significant portion of donors under the age of 30. Over the last 10 years we have lost ever 40% of the blood donors 30 and under2. To strengthen the nation’s blood supply, engagement with younger donors is essential. BCA and its members are developing innovative ways to attract younger donors through the use of technology. We recently launched a first-of-its-kind, immersive, mixed reality experience to appeal to people who have never donated before or may be apprehensive. We see the Mixed Reality for Blood Donation Experience as a way to make blood donation more enjoyable and appealing to the tech-driven Gen Z and Millennial audience. Participants will wear lightweight mixed reality headsets to enter a digital world but remain fully aware of their surroundings, ensuring a safe donation process.”
Chetan Makam, General Manager of Global Blood Solutions and Veerle d’Haenens, General Manager of Global Therapeutic Systems and Cell Therapy Technologies at Terumo Blood and Cell Technologies
Terumo Blood and Cell Technologies is a medical device company which aspires to increase patient access to healthcare, improve patient outcomes and serve unmet medical needs. Makam said: “Our products collect blood and cells from donors and patients and process these blood and cells to deliver treatments and therapies to patients. Considering that no two donors or patients are alike, getting a donation to a ready supply of transfusable blood is complex. We are experts in blood, it’s what we do. And we understand our customers processes and needs and design solutions that include devices, single use collection kits, software and other value-added services across the ecosystem.”
Makam continued, saying that blood donation and blood products are a basic requirement for healthcare, which many people don’t think about blood until they need it. He said: “The availability of safe blood is essential for surgeries, trauma patients, maternal care, and even geopolitical conflicts. However, access to safe blood is far from equitable, with many developing countries facing acute blood shortages. Developed countries, with only 15% of the world’s population, have access to more than 50% of the total blood supply. Contrast that to developing countries, with 85% of the population, which often lack a national framework for the collection, testing, and distribution of safe blood.
“Improving access to blood is crucial for the treatment of existing diseases as well as the development of new therapies. Without it, this work stops. We read every day about biotech’s and their work to develop new treatment options. One example we are following with anticipation are the two FDA approvals for the first gene therapies for sickle cell disease. However, patients need better therapy while waiting for a chance for a cure; thankfully, there are underutilised approaches such as automated red blood cell exchange that are bringing relief today and can be scaled to reach even more patients. But here, we come back to the importance of blood donors. Red blood cell exchange relies on the generosity of donors, and in particular, ethnically matched donors for this patient population.”
D’Haenens explained that Terumo works across the whole blood and cell ecosystem, from collection to processing to treatments. She said: “In the cell therapy space, we recognised the power of our work in this area and knew we could do more. We brought in more clinical and scientific people and realigned our focus toward cell therapy development, launching two products, Quantum Flex Cell Expansion and Finia Fill & Finish, in the past three years to improve cell therapy manufacturing.”
She elaborates by saying that biotechs are increasingly recognising the significance of starting materials in cell therapy development but lack experience in cell collections. She continued: “We play a role here as a global leader in apheresis with our Spectra Optia Apheresis System used for 94% of white blood cell collections in the US and 67% worldwide. We offer a Cell Collections training program geared to biotechs, teaching how to shorten, streamline and standardise the collection process which can help reduce costs, and scale cell manufacturing. Beyond the technical learning, this improves collaboration with industry partners and treatment centers to build knowledge in cell collections.”
Becky Cap, SVP, Advanced Therapies, BioBridge Global
BioBridge Global (BBG) is a San Antonio-based 501(c)(3) non-profit advanced therapies enterprise that provides transfusible transplantable products to hospitals and the full range of donor to patient services to advanced therapies developers. It enables development and production of advanced therapies by providing access to human cells and tissue, testing services and biomanufacturing and clinical trials support. BBG offers its services through its operating subsidiaries – South Texas Blood & Tissue (starting materials), QualTex Laboratories (analytical services), and GenCure (process development and manufacturing). Cap said: “BBG is committed to saving and enhancing lives through the healing power of human cells and tissue.”
Speaking on the importance of blood donorship or blood products for therapy development, Cap said: “Today, we see therapeutic developers work to finding donors with unique profiles. There is still a contingent focused on getting to an ‘ideal’ or ‘universal’ donor, but we are increasingly seeing an interest in selecting a donor that is specifically suited to the development of a defined product or product indication. If a donor already has a part of a profile you were expecting to engineer, that can limit the risks, time and costs associated with target cell modification. The challenge is finding, maintaining, and growing a group of donors that can support the ongoing needs of therapeutic developers in the allogeneic space.”
That challenge must be met in the context of a generally shrinking donor base, Cap continues. She said: “We need to consider the impact of collecting material from these donors and how it affects our ongoing need for traditional transfusible blood products for trauma, bleeding disorders, and cancer supportive care. Overall, blood donation is down across the country, especially among younger members of the community. Finding new ways to engage a broader community to support donation for blood products for transfusion and for further manufacturing is essential for increased therapy access and overall public health needs.”
According to Cap, BBG is making a concerted effort to grow, understand, and protect the donor base for research and the development of advanced therapies. She said: “With our connection to the donor network for transfusible and transplantable products, we’re adding ways current donors can participate in serving our mission. We are connecting with infrequent donors and providing them with an avenue for supporting innovative development in the advanced therapies space. We are adding collection sites to increase convenience for the donors and take advantage of the growing population of San Antonio. We are also providing ways for our donors to participate in larger scale public health projects like All of Us. These projects appeal to donors’ sense of a broader mission and help build our donor base.”
Cap says that the company’s relationships with hospitals are allowing them to think more broadly about how to extend its service and mission. She concluded: “Our hospital network needs the services we provide for the patients receiving autologous therapies. With our relationships and proximity to hospitals, we can be a strong connection point for developers, obtaining the patient material, providing the associated logistics, some degree of processing up to full manufacturing and testing, and resupply to the hospitals for infusion.”
NHS Blood and Transplant
NHS Blood and Transplant (NHSBT) is part of the UK’s largest public sector organisation and provides an invaluable service coordinating blood and organ donation. NHSBT also provides several specialist services including support for stem cell transplant, apheresis, a stem cell donor registry and a cord blood bank. NHSBT manufactures a range of advanced cell and gene therapies from a number of laboratories across England under license from the MHRA, and works with experts in hospitals, universities and commercial organisations to develop the next generation of healthcare treatments.
A spokesperson from NHSBT said: “In 1937, the UK created the world’s first blood bank. Since then, NHSBT has worked tirelessly to provide all the blood products required alongside a number of related activities. The NHSBT donors also generously provide bone marrow, peripheral blood stem cells and cord blood stem cells for the treatment of blood cancers. Recently NHSBT has also been collecting and supplying starting material for more advanced therapies such as CAR-T treatments for lymphoma. Blood and stem cell donors are often also willing to provide products for ethically approved research and development. For example, the development of new CAR-T treatments.”
The spokesperson continued by stating that NHSBT supports many steps of the stem cell transplantation and advanced therapies supply chains, including donor identification and tissue typing services, cell collection, processing, freezing and storage, through to cell expansion and genetic modification. They said: “NHSBT are also part of the ground-breaking RESTORE project where red blood cells are being manufactured from adult stem cells with a view to providing renewed hope for patients with difficult-to-match blood types.”
With regards to what NHSBT is doing in this area, the spokesperson said that as a government funded NHS provider of blood, organs, stem cells, apheresis and ATMP therapies, operating under a single quality managements system, they have a unique perspective and a responsibility to work proactively with others to identify and drive improvements across the system. The spokesperson said: “NHSBT are continuing to grow, as of 2023 we offer:
- An award winning Clinical Biotechnology Centre: Manufacturing GMP-grade plasmids and viral vectors to support academics and commercial companies with gene therapies
- Supporting stem cell transplants and the manufacture of novel cell therapies including gene modified cells Cell Therapy laboratories strategically located around the UK
- Several ATMP storage and Distribution sites
- Eight Therapeutic Apheresis Services providing approximately 10,000 procedures a year for patients and donors.;
- supporting 5 ATMP clinical trials and the delivery of 3 licensed ATMPs
- A network of over 5,000 clinicians, academics, and scientists on hand to answer your questions and guide your project.”
NHSBT continue to work with academics, hospitals, and industry, to discover, develop and expand ground-breaking novel treatments.
David Wellis, CEO of Excellos
Excellos is a cell and gene therapy CDMO, serving the R&D, clinical and commercial needs of its partners, which was seeded inside of the San Diego Blood Bank and was spun-out as a for-profit entity in late 2021. Wellis said: “Excellos is unique in that we have paired access to large and diverse blood donor pools with decades of experience in biologics manufacturing, to provide the highest performing products and services to the industry.”
When it comes to blood donorship or blood products for therapy development, Wellis said that blood donors and the products they donate are critical to the development of new therapies, as they are step one in the supply chain. He said: “Blood donors are the essential source of raw material for R & D, as well as clinical and commercial allogeneic cell therapies. The different types of white blood cells they donate, such as T-cells, natural killer cells and tumor-infiltrating lymphocytes, are the starting material for hundreds of cell therapies in development. Their cells can be engineered into the therapy itself or be used to create cell banks of starting material for later use.”
He continued: “Because the starting material for cell therapies is sourced from blood donors, a significant amount of variability in the development of therapies, and ultimately in clinical response, is commonly observed. While these therapies show incredible promise for curing both blood-borne and tumor-based cancers, there still exists too many partial responses and recurrence of cancer following therapy. The industry is beginning to see the need to better understand multiple properties of the cells themselves to improve the efficacy and safety of cell therapies.”
With regards to Excellos, Wellis said that Excellos was named to indicate its vision – providing better cells for better therapies, addressing the issues he mentions above. He said: “We have developed a deep cell characterisation platform that goes beyond typical cell and donor screening to create a comprehensive immune cell profile for each donor. Besides looking at donor characteristics, our platform, which we call Excellos 360TM, includes the assessment of metabolic and effector potential of cells, and genetic analysis. This deep cell analysis can be applied at all key manufacturing steps to ensure the optimisation of purity and potency of the therapy being developed.”
According to Wellis, Excellos 360TM data has the potential to help improve the potency of cell-based treatments to reduce clinical response variability, to increase the probability of success of clinical trials, improve safety profile by making therapies effective in lower dosages, increase efficacy by improving both donor and cell selection, improve clinical outcomes, and lower the cost of goods associated with cell and gene therapy manufacture. He concluded: “These analyses will allow therapeutic developers to accelerate the development and production of immunotherapy treatments, increasing their use and accessibility.”
Resolution Therapeutics
Resolution Therapeutics is developing macrophage cell therapy products to treat end stage liver disease (cirrhosis), caused by chronic inflammation of the liver from metabolic disorders, alcohol or viral hepatitis. A spokesperson from the company said that this is the only chronic disease still on the rise in western countries, affecting millions of people worldwide. They said: “For patients with cirrhosis, the only therapeutic option is liver transplantation, a complex surgical procedure limited by complications and a shortage of donors. The company is pioneering the use of regenerative macrophages to treat cirrhosis, with potential further applications in a wide range of life-threatening conditions.”
According to the spokesperson, blood donorship is hugely important for the development of new and potentially transformative macrophage cell therapies. They said: “These therapies are complex and time consuming to manufacture, and the success of the medicine relies on a robust, reliable, and safe manufacturing process. To carry out the necessary research and development of manufacturing process, the company relies on blood products. These are used in manufacturing runs to test different parameters and equipment configurations, new technologies or unit operations and to gain a detailed understanding of how the regenerative macrophages are made to the target specifications. For the company’s autologous cell therapy product, Resolution Therapeutics relies on blood products to identify different donor characteristics that may introduce variability into the process. This helps the researchers prioritise which unit operations and quality attributes to focus their efforts on.”
Resolution Therapeutics was founded following a productive collaboration between Edinburgh’s Centre for Regenerative Medicine, the Scottish National Blood Transfusion Service (SBNTS), and Syncona, Europe’s leading sustainable biotech venture builder. The company spokesperson shared: “Since its inception, the company has closely collaborated with SNBTS to obtain blood donations from healthy volunteers and cirrhotic patients, in order to develop and validate our manufacturing process. All of the blood products used are helping build a process that will enable personalised therapies to be tailored made for each individual cirrhosis patient treated with regenerative macrophage cell therapy.”
Priya Baraniak, Chief Business Officer, OrganaBio
OrganaBio is a fully-integrated solutions provider to cell and gene therapy developers. Via two wholly owned, FDA-registered tissue procurement businesses, OrganaBio provides advanced therapy developers with clinically relevant, well characterised birth tissue- and adult peripheral blood-derived starting materials (e.g. umbilical cord blood, leukopaks, HSCs, MSCs, PBMCs, NK, and T cells). In addition, the OrganaBio team’s expertise in cell isolation, process development, GMP manufacturing, assay development, and analytical testing provides partners with access to a suite of services accelerating their advanced therapy development pipelines. Thus, Baraniak believes that OrganaBio is uniquely positioned to support its partners from early discovery through clinical translation and commercialisation.
On the topic of blood donorship or blood products for development of new therapies, Baraniak said: “Blood components such as plasma and platelets have a long history in saving lives for patients with traumatic injuries, liver disease, autoimmune conditions, and clotting factor deficiencies/bleeding disorders. More recently, blood-derived cells have become critical starting materials for cell and gene therapies. As such, blood donors are a vital part of the stakeholder value chain in advanced therapies. Hematopoietic stem cells (HSCs) from cord blood have been used for the treatment of pediatric cancers and other blood and immune diseases that affect the bone marrow. HSCs, Natural Killer (NK), T cells, B cells, macrophages, and other cells found in adult peripheral blood are also demonstrating unprecedented success in treating a variety of cancers, autoimmune diseases, and other conditions that have affected quality of life and life expectancy for generations. We stand on the precipice of a transformative era in the standard of medical care, and blood-derived cells and other components (such as plasma and platelets) will continue to fuel these modern medical miracles. These scientific advances would not have been but for the millions of blood donors who have and continue to fuel this research.”
OrganaBio provides advanced therapy developers with access to robust, reliable, clinically relevant, high-quality blood-derived starting materials for their research, process development, product development, and clinical manufacturing programs. Baraniak said: “At OrganaBio, we are proud to be a part of the network providing this crucial material to cell therapy developers through our two tissue collection establishments: GaiaGift (birth tissues) and HemaCenter (adult leukapheresis).”
GaiaGift is dedicated to collecting valuable birth tissue from consenting, healthy mothers, including umbilical cord blood, which contains precious hematopoietic stem cells and immune cells. Baranbiak said: “By providing an ethical and robust supply of birth tissue and a number of isolated cell types from this tissue, GaiaGift ensures these precious resources can be used to their full potential by cell therapy developers.”
HemaCenter, on the other hand, focuses on leukapheresis. Baranbiak said: “Through leukapheresis, specific blood components like plasma and white blood cells can be selectively collected and isolated in a customisable fashion for advanced therapy development. HemaCenter’s curated, recallable donor pool ensures a reliable supply of these vital blood components, contributing significantly to the outcome of patient care.”
Through the provision of umbilical cord blood units, leukopaks, and cells derived from these tissues, OrganaBio provides critical resources to therapeutics developers in order to improve patient outcomes and save lives.
Alistair Irvine, Chief Executive Officer, Scarlet Therapeutics
Scarlet Therapeutics is developing a platform that generates novel red blood cell-based therapeutics to potentially treat a wide range of diseases. Initially targeted at the rare metabolic diseases, hyperammonemia and hyperoxaluria, this approach could also be used more broadly to target other metabolic diseases, cancer and autoimmune diseases. Scarlet is developing a platform for the production of these tRBCs and are initially looking at using them to treat some metabolic diseases in which there is a build-up of a toxic product in the body. These tRBCs will remove these toxic products.
According to Irvine, blood donorship and blood products are vital for the developing of many new therapies and for the supply of current therapies. He said: “Blood donations are used in many ways including:
- To provide material for blood transfusions
- To provide the material from which many blood products are manufactured to treat many diseases including haemophilia, thrombocytopenia, immune deficiencies and the side-effects of some cancer treatments
- Use in research of blood related diseases, such as sickle cell disease, immunodeficiencies and hemoglobinopathies
- Development of new therapies based on different types of blood cells, such as CAR-T therapies for cancer and red blood cell therapies such as those being developed by Scarlet.”
Scarlet is investigating the use of red blood cells to treat diseases, and the company call these therapeutic red blood cells (tRBCs). Irvine said: “We believe red blood cells are an exciting new treatment methodology since they are:
- Pervasive – they go all parts of the body
- Enduring – they last for around 4 months in the body
- Safe – there is a lot of experience with red blood cell transfusions that has shown it is a very safe procedure and red blood cells do not have a nucleus and do not divide and therefore there is no risk of cancer (unlike with some other cell therapies)
- Smart – they can be made to perform additional therapeutic activities in addition to their normal activity of carrying oxygen around the body. Also any proteins within the red blood cell are hidden from the immune system.”
References
- Statista 2022
- BCA Gold Data
