Science, medicine, abundant enthusiasm, and hope are components of the pioneering work in regenerative medicine and cell therapies. Helping a body repair itself from injury, disease, or genetic maladies is a vision not only shared by scientists, clinicians, and patients but also by the managers and staff of companies that are manufacturing the products. Discoveries in molecular biology and genetics have recently led to a better understanding of how healthy and diseased cells work. Cells that can reproduce more copies of themselves (i.e. “stem cells”) can be cultured and chemically or genetically coaxed to become specialized cell types better suited to repair wounds or nerves, torn livers, broken bones and damaged hearts. Some patients have already benefited from such therapies, while others wait. The field of regenerative medicine is in its infancy but will become standard medical care as a future multi-billion dollar industry.
Laboratory research provides a foundation for the proposed use and characteristics of a potential product. However, the clinical use can only be realized by defining the specialized processes and steps for manufacturing, packaging and storage. Essential elements are similar to other types of manufacturing: product development, quality by design, well-defined standard operating procedures, quality systems, raw material sourcing, staff training, and quality control release of products meeting specifications. Unique needs for cell therapy products are safety, sterility, dose, and characterization. Also, many cell and tissue therapies come from donated tissues that are the starting raw materials for processing to therapeutic tissues or cells, so a special respect and understanding about the donor and donor families, as well as the procurement entities, are needed. Meeting these various needs requires specialized training of personnel at all levels, from the laboratories to the boardroom. Our academic institutions do not focus on this type of work, so each company must invest in training.
How is quality regulated? Depending on the type of product, where it came from, and where it will be sold, there are specific regulatory standards defined by US and international laws that relate to all aspects of source tissue assays, reproducible manufacturing and quantity scale-up, quality, labeling, shipping, pre-clinical testing, clinical studies, and marketing. Each is a challenge and an opportunity. As a practical matter, the “Wow!” factors of scientific research discoveries and potential clinical applications must be balanced with the realities of product development, and getting the manufactured and packaged “bio-widget” to a market with ready end-users and appropriate pricing that captures the costs but still assures a profit margin. It must also consider the packaging and route of administration. Will the product be swallowed, injected, or rubbed on? What other components will be in the end-product? Do all components meet regulatory standards? Is the product stable? What are the storage needs? Most cell therapy products are stored frozen below minus 150°C, requiring specialized packaging, labeling and shipping.
New products develop from laboratory-to-industry-to–clinic, not laboratory-to-clinic. Something that “worked” in a laboratory may elude scale-up or reproducible manufacturing, release testing, storage, practical packaging, or stability. Such problems can lead to an unacceptably high cost of goods, lost product lots, and possibly recalled product. Such catastrophes are best avoided by early teaming partnerships of the inventor with selected scientists and business, quality and regulatory specialists who understand all stages from development through manufacturing and distribution.
Sometimes cell and tissue therapy products are manufactured for personal use or are processed in an operating suite for transplantation. That is, the individual donating the tissue, or a tissue from which cells will be isolated, will also be the recipient of the tissues or cells. These transplants are autologous (“self”), in contrast to transplants from other donors, which are allogeneic. Common autologous transplants are blood and bone marrow, skin, and blood vessels. Many of these are done as standard clinical practices that are not FDA regulated. Others, such as isolating cells from tissues and then injecting the cell product, are “more than minimal manipulation” and are FDA regulated so that the processes meet proper manufacturing standards.
Some doctors insist on bypassing mandatory FDA regulations and do manufacturing in their offices without quality systems and methods for defining doses, sterility, and testing of product characteristics for final release, putting their patients at risk. Breaches of ethics and integrity may also come from those who put profits first, preying on sick, damaged and desperate people, and charging excessive fees for injections or storage of “stem cells,” which may not even be cells. As an example, scientific research has shown that no more than a few million cells exist per cc of human or animal fat tissue. However, some companies market that they are better than their competitors, claiming their products contain billions of cells per cc which is a scientific impossibility, unless the company is capable of creating life!
The art and beauty of science will foster innovations in regenerative medicine and cell therapies that are currently just ideas and imagination. However, it will be commitments to safety, quality, manufacturing and ethical practices that will sustain the reality of the new therapies. Each CEO has the corporate responsibility to assure these commitments, and to understand the science behind a company’s products or services. Leadership comes by insisting that each individual on the team agrees and does quality work to a level that they would be confident to put the product into their child, spouse, parent, grandparent, themselves or anyone they love.
Today’s cell therapy products come from many tissues including skin, fat, birth tissues, liver, blood and bone marrow. They must undergo laboratory tests and clinical trials before they will be approved as products for treating patients with arthritis, neural disorders, infectious diseases, cancer, genetic disorders or other maladies. Regenerative medicine is not the future. It is now.
Mary Pat Moyer, Ph.D., is the INCELL (1993) Founder, CEO and Chief Science Officer. With 45 years’ experience and numerous entrepreneur and science awards, Moyer is a human cells and microbiology expert, and leads INCELL’s manufacturing, product development and contract services business. INCELL is currently storing autologous stem cells and cancer patients’ tumor cells for personalized medicine and developing and manufacturing multiple types of cell and tissue products, including stem cells, for itself and its clients.
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