Miracle-Gro for Cell Cultures?
Cell-based therapies, in which cells from the body are multiplied in the laboratory then introduced back into patients, have the potential to treat a broad range of diseases and hereditary conditions. In particular, the therapeutic use of mesenchymal stromal cells (stem cells that can differentiate into a variety of cell types) holds tremendous promise for patients with graft versus host disease, stroke, diabetes, osteoarthritis, chronic ocular disorders, Parkinson's disease, Crohn's disease, and many others.
Until recently, the only way to make enough cells for these treatments in the lab was to expose the cells to growth supplements derived from the serum of animals. The problem is that animal supplement proteins are xenogenic and may cause the human body to see the cell therapy as foreign and mount an aggressive immune response to remove these cells from the body. This can seriously limit the potential of any cellular therapy, says Ian Copland, PhD, assistant professor of hematology and medical oncology. There are also concerns about the potential transmission of animal-born diseases (such as prion diseases,) impurities and contaminants, and antibiotics, when such growth supplements are used.
Due to these drawbacks, researchers turned toward fracturing human platelets to release growth supplements, but this process presents its own challenges, such as cellular debris, clotting factors, and diminished cell functionality.
"While cellular therapies hold lots of promise for treating disease, the technologies and tools that enable their large-scale adoption and application are currently lacking," says Cliff Michaels, licensing associate in the Office of Technology Transfer (OTT). The OTT is working with Copland and Jacques Galipeau, MD, FRCP, professor of hematology and medical oncology, to improve growth supplement technology.
Copland and Galipeau sought to refine the manufacturing of human platelet lysate, and developed a method that removes debris and problematic factors while preserving the functionality of multiple cell types. The technology was approved for use by the FDA in March 2012 and is currently being used in an Emory initiated Phase I clinical trial where a person’s own cells—autologous mesenchymal stromal cells, specifically—are administered to individuals with moderate to severe Crohn's disease. This lysate is also being used to launch several additional cell therapy trials at Emory for conditions with unmet medical need.
This product, Multiplate FDTM, has proven superior to fetal bovine serum as a growth supplement by multi-investigators at Emory and throughout the U.S. "We believe it’s superior to just about anything out there," says Copland.
The number of patients who could benefit from cell therapy is estimated at 100 million in the U.S. alone, so the need for a reliable growth supplement that can be quickly and cheaply produced is high.
"Cellular therapy and MSC therapies can open new hope to many patients who currently have run out of therapeutic options. Our ability to help satisfy unmet medical need is extremely exciting" says Copland. "And the fact that our cell therapies can harness the body’s natural regenerative capacity give us real hope for long-term benefits for difficult to treat auto- and alloimmune disorders."
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