Wednesday, 02 May 2012 12:37
May 2, 2012
U.S. regulators made a historic announcement recently, approving for the first time ever a drug created in a genetically engineered plant cell.
The U.S. Food and Drug Administration (FDA) approved the drug this week in a move backers contend could help spur the continued development of the emergent "bio-pharming" sector, the Nature News Blog reports. Federal regulators' approval of Elelyso could also signal a shift in the FDA's approach to testing and bringing drugs to the market.
The medication allays the symptoms associated with Gaucher disease, an uncommon lysosomal storage disorder. Those afflicted with the syndrome can experience a range of painful symptoms, including bone infections, anemia and discomfort. The drug did not emanate from U.S. laboratories, but, rather, in Israel, which is a major force in the worldwide technology and biopharmaceutical sectors.
Scientists at Protalix Biotherapeutics oversaw the drug's development, as engineering research at the company increasingly focused on ascertaining a new method for generating the human enzyme that is not present in patients with Gaucher disease. The company was able to produce the enzyme in carrot cells by injecting a gene responsible for encoding the protein into the cells, according to the news provider.
In clinical trials, researchers said that patients who were treated with the enzyme developed in the carrot cells, known as taliglucerase alfa, responded well to the therapy. Overall, scientists determined that the plant-derived medicine was at least as effective in allaying symptoms associated with the disease as Cerezyme, a treatment currently available on the market.
Cerezyme is made from hamster cells, and it is one of the only medications physicians prescribe for patients with Gaucher disease. According to data from the U.S. National Library of Medicine, approximately one in 50,000 to 100,000 people in the general population suffer from the affliction. What's more, the syndrome generally is more common among those of Central and Eastern European descent, as well as those with a Jewish heritage.
"It's wonderful to have another option available," said Rhona Buyers, the executive director of the National Gaucher Foundation. "People whose symptoms had been controlled for years were having bone issues and terrible fatigue, some went the hospital," she added.
The approval of the new enzyme therapy is welcome news to patients with Gaucher disease, as shortages of therapies for the syndrome limited treatment options between 2009 and 2011, according to Buyers.
On a molecular level, Elelyso is similar to treatments available on the market. Scientists noted its composition is reminiscent of Cerezyme's, which is manufactured in the U.S. by the pharmaceutical company Genzyme. Unlike Cerzeyme, however, Elelyso is less expensive to manufacture because the genetically engineered carrots do not carry any of hefty maintenance costs associated with animal cells.
Additionally, because Elelyso is generated from carrot cells, its production is not vulnerable to the viruses and other kinds of toxins that can disrupt development in animal cultures. Popular Science reports that although the engineering tools used in the development of plant-based medical therapies are not new, the FDA had historically been loath to approve such treatments. Its latest decision, though, underscores how the federal organization is shifting its approach as new data becomes available.
The approval of Elelyso could signal a future in which drugs derived from genetically engineered plants become commonplace in the U.S., experts said, although scientists affirmed such an outcome is anything but assured.
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