Washington, DC
February 28, 2003

Biopharming Could Reap Benefits But Must Be Carefully Regulated

Pew Initiative/FDA/USDA conference explores emerging biotech sector: looks at benefits, risks and adequacy of current regulatory framework Government plans to release guidance document as early as
March

Biopharming, an emerging sector of the biotechnology industry that involves engineering plants with
genes that allow them to produce pharmaceutical substances, could provide a cost-effective and abundant source of drugs, but both the industry and government regulators must take steps to minimize the risks associated with the technology, according to some of the nation's top biotech experts who spoke at a conference sponsored last year by the Pew Initiative on Food and Biotechnology, the Food and Drug Administration and the U.S. Department of Agriculture.

The proceedings of the conference, "Pharming the Field: A Look at the Benefits and the Risks of Bioengineering Plants to Produce Pharmaceuticals," were released today by the Pew Initiative. Government regulators are also expected to release a proposal soon to tighten restrictions on growing crops genetically modified to produce pharmaceuticals.

Representatives from government, the biotech and food industry, scientists, patient advocates and public interest groups gathered in July to examine ongoing efforts to develop genetically modified (GM) crops capable of producing specific human antibodies, enzymes and other biological materials. The conference also explored whether the technology poses risks to humans, animals and the environment (particularly if drugs are produced in food or feed crops), and what steps industry and government regulators are taking and should take to minimize these risks and prevent these products from entering
into the food supply.

Biologics, which are fast becoming a significant share of all pharmaceuticals in development, are manufactured today with material grown in mammalian cell cultures (usually Chinese hamster ovary cells) -- a process that can be costly and inefficient. For this reason, alternative production systems are in development, such as biopharming, which could help speed new treatments for a range of conditions to market that are currently unavailable. Engineering food crops, such as corn, to act as pharmaceutical factories may help to fulfill that promise.

However, using food crops such as corn for non-food uses poses unique problems on several levels. The food industry, environmental groups and consumer advocates at the conference raised questions about how these crops would be "contained" so that they did not inadvertently get consumed by humans or animals, or spread into the surrounding ecosystem. Indeed, several months after the conference, GM corn that contained a protein for a pig vaccine made by ProdiGene Inc., a Texas biopharming firm, accidentally contaminated soybeans intended for human consumption. The soybeans were destroyed before reaching the consumer market, but the incident highlighted concerns about the regulatory system's capability to monitor this new frontier.

"Agricultural biotechnology is a powerful tool that continues to stretch the bounds of the imagination," said Michael Rodemeyer, executive director of the Initiative. "Scientists can now take food crops like corn and turn them into protein factories for drug production -- a process that promises to transform the way medicines are made. But this power is not without some risk. Therefore, it's important that we find ways to reap the benefits of these products while safeguarding the food supply and the environment. We can't afford any more near-misses."

Some of the key points that emerged from the conference included:

A growing number of cutting-edge treatments for arthritis, herpes, cancer and infectious diseases could be manufactured using biopharming. Manufacturing these drugs today via mammalian cells is very expensive and there is not enough capacity to meet current, not to mention future, needs. For example, there are four pharmaceutical products requiring human antibodies that now consume three-quarters of current fermentation capacity. Some 20 to 50 products in the pipeline could be delayed by lack of manufacturing capacity. Biopharming could solve these issues by not only lowering costs (it is estimated that biopharming could cut costs by one-eighth), but also by allowing companies to rapidly ramp up capacity by merely planting more acreage (as opposed to having to build new manufacturing facilities/plants, which cost $600 million on average and take 5 to 7 years to build).

The food industry has serious concerns about biopharming containment. The food industry is unwilling
to accept any amount of co-mingling between pharma plant products and food products. Ideally, the industry would prefer that the risk of contamination be eliminated by having biotech companies only use non-food crops, such as tobacco and grasses, for biopharming. But, if food crops such as corn must be used, other measures for containment should be explored such as: engineering the plants so that the desired proteins are not expressed in the food part of the plant (i.e. in roots and other parts); using the "terminator" technology to engineer sterility into these plants; using visual markers to distinguish these crops from food; and cultivation in areas far removed from food crops.

Government regulators are trying to keep pace with the technology. The government regulators have instituted requirements for pharma plant cultivation that are more rigorous than those applied to other GM varieties. For example, unlike many other GM crops, pharma plants will always be under USDA regulatory oversight. In addition, the USDA and FDA intend to coordinate their oversight efforts. FDA will set its own standards for field operations, as it plans to give field cultivation of pharma plants the same sort of regulatory scrutiny that it applies to conventional drug manufacturing facilities. (It is expected that the government's soon-to-be released guidance will lay out these oversight issues in more detail).

There are questions about regulatory transparency. There is a tension between the desire for public
disclosure that would facilitate an independent evaluation of potential health and environmental hazards, and a company's right to protect confidential business information.

The full report is available in PDF format at http://pewagbiotech.org/events/0717/ConferenceReport.pdf