What do you think? Most people carry two copies of a normal gene that codes for an enzyme, glucosylceramidase, that is involved in breaking down lipids no longer needed in cells. (Enzymes are proteins that cause specific chemical changes; they are biological catalysts.) One in 100 people in the United States carries a recessive mutation that codes for a defective glucosylceramidase enzyme. And about one in 40,000 people carries two copies of the mutation and displays the symptoms of Gaucher disease. These symptoms, caused by the accumulation of lipids in cells, include anemia, enlarged organs, swollen glands and joints, and, in severe cases, neurological problems and early death.
Enzyme replacement therapy is effective but very expensive-about \$200,000 annually-and must be continued, every 2 weeks, for life. The Israeli biotech company Protalix Biotherapeutics, working with the U.S.-based Pfizer Pharmaceuticals, has developed a process to genetically modify carrots to produce a replacement enzyme. The biopharmed enzyme will cost about 25 percent less than the standard enzyme therapies, which are grown in mammalian cell lines. Protalix is now working on treatments for other enzyme deficiency diseases. The FDA's May 2012 approval of the drug developed by Protalix alarmed some environmental activists and health
advocates, who fear that the company's genetically modified carrot is just the beginning of a wedge that will lead to an underregulated and potentially dangerous industry. There is some legitimacy to their concerns: the U.S. Department of Agriculture (USDA) does not require an environmental impact assessment for biopharmed crops; nor does it require biotech companies to share the location of their test fields or the identity of the biopharmed molecules being produced. Furthermore, the USDA is not sufficiently staffed to effectively monitor companies involved in biopharming. What do you think? Should biopharming be allowed in the United States? If so, under what conditions and with what limits? For example, should it be allowed to produce drugs for only life-threatening illnesses, or only under highly controlled conditions? Be prepared to discuss your observations and reflections in class.