By Anuj Kalsy
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Repairing tissues and organs makes up a key goal of medicine, and many companies pursue this objective. By 2030, the regenerative-medicine market is projected to reach nearly $38 billion, based on an annual growth rate of more than 15% a year. Much of that growth comes from advances in stem cells and pathways around them.
In February, for example, researchers at IRCCS Bambino Gesù Children’s Hospital in Rome reported on a Phase III clinical trial of adding functional copies of a modified gene to hematopoietic stem cells that can be used to treat some people who have β-thalassemia and depend on transfusions. On 17 August, the U.S. Food & Drug Administration (FDA) approved this treatment, called betibeglogene autotemcel, and described it as the “first cell-based gene therapy for the treatment of adult and pediatric patients with beta-thalassemia who require regular red blood cell transfusions.”
To gain insights into the rise of this type of research and the applications of human cells for that purpose, we spoke to Anuj Kalsy, Scientific Director at Precision for Medicine, Biospecimens Solutions Division.
Ready-to-use cells
“Human primary cells have been the key material for research and production of cell therapies, but these cells have not always been easy to access,” says Kalsy. “So, there is definitely an urgent need for suitable alternatives or cellular products that are viable and of high quality, which can be used as powerful research tools by the R&D community in the life science industry.” To explore stem cells and a variety of immune-system mechanisms, Precision for Medicine gives scientists easy access to reliable and well characterized cells.
For work on cell and gene therapies, stem cells, and regenerative medicine, Precision for Medicine offers a variety of leukopak products, which include specific cell types that are collected from a patient’s peripheral blood through a special form of apheresis. “Leukapheresis removes leukocytes—the white blood cells—and returns the remainder of the cells—granulocytes, platelets, and red blood cells—to the human donor,” says Kalsy. Consequently, a leukopak provides a rich source of white bloods cells—an average of about 73% in each leukopak. Of those cells, approximately 38% are CD8+ T cells and 18% are CD4+ T cells. “T cells have gained popularity in cell and gene therapy markets due to many in the life sciences industry focusing on the development of CAR-T cell therapies,” Kalsy says.
Precision for Medicine can provide research-grade fresh, cryo-preserved, and mobilized leukopaks, which can be used in basic and clinical research. Mobilized leukopaks are manufactured using FDA-approved granulocyte colony-stimulating factor (G-CSF) drugs, such as 5-dayfilgrastim, to mobilize and acquire the highest number of CD34+ stem cells.
“Leukopaks are the preferred choice in the life science industry for large-scale research and development efforts, specifically early discovery and preclinical efforts, instead of the typical choice of PBMCs, or peripheral blood mononuclear cells,” Kalsy explains. “Research studies require assay-to-assay consistency and large numbers of cells.” Leukopaks meet both of those requirements. For example, Kalsy notes that compared to whole blood-based products, “leukopheresis can provide up to 100 times more cells from the same donor than a blood draw.” Consequently, “leukopaks are gaining a lot of popularity in the cell and gene therapy niche, as they are a great source of CD4 and CD8 T cells, B cells, and natural killer or NK cells—all of which are being used as therapeutic modalities to target human diseases,” Kalsy explains.
Precision for Medicine continues to expand its line of leukopaks. For example, the company is developing a leukopak enriched for CD34+ stem cells using in vivo mobilization procedures. These will be extremely useful to scientists studying the basic biology and potential treatment applications of such hematopoietic stem cells.
Selecting the best cells
For a specific project, Precision for Medicine can create custom leukopaks from people with certain HLA types, particular ethnicities, or even a specific disease. “The selection process involves adequate screening and having a well-qualified human donor pool, and this is achieved through our professional staff, which consists of American Red Cross trained veterans, as well as our infrastructure that utilizes modern and gold-standard equipment for processing the highest quality products,” Kalsy explains.
With research-use-only leukopaks, Precision for Medicine can acquire a custom collection of cells that meet a customer’s needs in terms of cell type, optimal yields, viability, and quality. “Our cell subset products are manufactured using industry-standard reagents, and qualified protocols, alongside doing rigorous QC analysis in the workflow,” Kalsy explains. “This ensures a high product performance in the hands of our customers.”
To meet so many crucial criteria, Precision for Medicine oversees the entire process. “We have an end-to-end chain of custody over the entire workflow, and we collect these leukopaks at our in-house, CLIA-certified blood donor and apheresis facility,” Kalsy says. Then, the samples go right to Precision for Medicine’s processing labs.
Just collecting the right cells, though, is not enough. “Our leukopaks are prepared from donors who are well characterized,” Kalsy notes. “We have a large database of recallable donors with different HLA types, and we collect demographic information, medical history, and IRB consent.”
Exploring new areas of interest
As noted, many scientists study treatments developed with CAR-T cells, but other immune cells are also gaining traction. One is NK cells, which Precision for Medicine can provide. “NK-cell therapies have been showing some promising results in very early-stage oncology studies across the globe,” Kalsy says. “Multiple companies are using NK cell therapies to enhance ADCC—antibody dependent cellular cytotoxicity—in both hematologic as well as solid tumors.”
Therapies based on NK cells could offer valuable benefits. “The cost of manufacturing NK-cell therapies is far, far lower than a CAR-T cell therapy,” Kalsy says. “Some people in the research industry call this therapy ‘the rising star in cancer treatment.’”
Precision for Medicine provides the tools that scientists need to find even more rising stars in healthcare. “We are always looking to provide innovative solutions to our customers in life sciences,” Kalsy says. “We’re always increasing the breadth of cases and sample types for all human diseases, including parallel cohorts of non-diseased samples or at-risk samples, to provide scientists with a one-stop service, from discovery through translation, to really apply biomarker-driven healthcare.”
Learn more about Precision for Medicine and its leukopak offerings.
