Stem cell therapies and regulations

Today, donated tissues or organs are often used to replace damaged or diseased tissues, but there is ever-increasing demand for transplantable organs and the need for new tissues far outweighs the available supply. Stem cells, directed to differentiate into specific cell types, offer seemingly limitless possibilities for regenerative medicine and becoming a renewable source for the replacement of cells and tissues to treat diseases including heart diseases, diabetes, spinal cord injury, stroke and macular degeneration, burns and arthritis. For example, it may become possible to generate functional heart muscle cells, known as cardiomyocytes, in the GMP laboratory and then transplant those cells into patients with chronic heart disease and avoid the need for some heart transplants.

Preliminary research in laboratory animals indicates that bone marrow-derived mesenchymal stem cells, transplanted into irreversibly damaged heart, can have beneficial effects on survival and mortality. Whether these cells can generate heart muscle cells or stimulate the growth of new blood vessels that repopulate the heart tissue, or help via some other mechanism is currently unknown. For example, injected cells may accomplish repair by secreting growth factors, rather than actually incorporating into the heart. Promising results from animal studies have served as the basis for number of clinical trials in humans. In 2013 there were around 312 academia or industry sponsored clinical trials with stem cells worldwide. Hoverer, translating basic stem cell research into routine therapies is a complex multi-step process which entails the challenge related to managing the expected therapeutic benefits with the potential risks while complying with the existing regulations and guidelines. In 2007, the European Medicines Agency (EMA) adopted the Advanced Therapy Medicinal Products (ATMP) Regulation EC (No) 1394/2007, which came into force on December 30, 2008. Its scope includes somatic cell therapy medicinal products, gene therapy medicinal products and tissue-engineered products. The Regulation clarifies the regulatory path for such products and facilitates the widening of the European market to innovative new ATMPs and therefore has been a major step to stimulate the development and approval of ATMPs in Europe.