Wednesday, June 07, 2006

Transgenic Mice and Men [the Harvard OncoMouse]

In a full-page Science advertisement in 1989, DuPont unveiled what promised to be a powerful new tool for “shortening the path to knowledge in carcinogenesis”: a transgenic mammal called the OncoMouse. Named for its possession of an inserted gene sequence conferring susceptibility to cancer, this animal quickly came to be seen as an ideal test subject for toxicology studies and new therapeutic developments in the war on cancer.

The mouse was the product of an induced mutation in a gene which encodes proteins crucial for regulating cell growth and differentiation. This was called an “oncogene” for its role in the development of tumors in many types of mammalian tissues. In order to produce the OncoMouse, scientists originally introduced a cancer-promoting gene through micro-injection of DNA into a fertilized mouse embryo. The inserted genes were modified to be expressed in the mammary tissue, so that the mouse could be used as a biological model for understanding the development of breast cancer. Further, the mice were engineered to exhibit heightened sensitivity to toxic substances, allowing researchers to study carcinogenic environmental factors.Many scientists predicted the usefulness of this new transgenic organism, but few foresaw the consequences of its private appropriation. Thirteen years later, the OncoMouse is a prime example of the hazards of mixing science and commerce, illustrating how the growth of intellectual property rights in the life sciences has created practical obstacles to basic research.
Indeed, the high prices and royalties demanded by DuPont are indicative of a larger trend of corporate impediments to science. If the recent furor between DuPont and cancer researchers over the use of the mouse technology is any indication, increasing private ownership may slow the pace of progress in health and disease research.Let’s review the history of this story. In 1988, the OncoMouse became the first animal ever given patent protection when the United States Patent and Trademark Office (USPTO) issued a patent to Harvard University geneticist Philip Leder and Timothy Stewart of the University of California, San Francisco. The ruling was broad in scope, covering the so-called vectors in which the oncogene gets copied, the fertilized mouse egg containing the foreign DNA, and the fully-developed OncoMouse and its descendants.
Moreover, the Patent Commissioner noted that the animal was “not simply a transgenic mouse with an activated MYC gene; it is any transgenic mammal, excluding human beings, that contains in all its cells an activated oncogene that had been introduced into it or an ancestor at an embryonic stage.”
A gradually developing legal precedent had paved the way for this decision. In 1980, the US Supreme Court in Diamond vs. Chakrabarty ruled that an oil-digesting bacterium was patentable. In the majority opinion, Chief Justice Warren Burger affirmed the lower court in claiming that “the fact that micro-organisms are alive is a distinction without legal significance.” He wrote that patent law extends to “anything under the sun made by man.” Reinforcing this view, in 1987 the USPTO in Ex Parte Allen extended this first patent to a multi-cellular organism, deciding in favor of a claim over a type of oyster.Following the OncoMouse patent in 1988, other countries at the forefront of cancer research adopted similar guidelines — the European Patent Office in 1992, Japan in 1994, and Canada in 1998. The fact that these patents were issued, despite widespread opposition, indicates the reluctance of patent offices around the world to consider ethical and public policy questions. While many courts have invalidated biotechnology-related patents, such decisions have usually been made on technical grounds.
DuPont and Harvard University signed a memorandum of understanding giving the company exclusive rights to license the OncoMouse and control its use by researchers. In order to secure the right to royalties from “downstream” discoveries, DuPont imposed aggressive licensing agreements on the use of OncoMouse technologies.The San Francisco Chronicle wrote on June 3, 2002 that DuPont was “impeding the war on cancer by charging high fees to companies, imposing unusually strict conditions on university scientists and pushing an overly broad interpretation of which lab mice the patents cover.” As a result, many agreements with MIT, the University of California system, and the Memorial Sloan-Kettering Cancer Center in New York have been cancelled or put on hold. Beyond the use of OncoMouse, DuPont has argued that the Harvard patent enables the company to demand a fee for the use of “knockout” mice, a line of mice with mutations or deletions of critical tumor suppressor genes.What can be made of these anti-competitive practices? Industry advocates claim that intellectual property rights are a necessary incentive for innovation, as they entitle the holder to an “appropriate value” from commercial and non-commercial uses of their product or technique. They argue that patents hedge against financial risk in developing new technologies, especially those that require large initial investments. The problem with such arguments is that patents create not only incentives for innovation, but substantial market power for those to whom the patents are issued. In early-stage technologies and industries characterized by rapid innovation, broad patents tend to reduce future investment in research. A growing number of examples point to the adverse effects of patents on technology transfer. Limited monopolies allow the holder to impose significant costs on “second comers.” In the licensing of important health- and disease-related genetics research tools, the resulting delays and lost opportunities may be costly to public health. The problem is that debate over patenting in science too often focuses on only two issues: whether patents on living things, cells, tissue and DNA are ethically acceptable, and whether these ownership claims constitute a misuse or over-extension of the patent system. Less attention has been paid to whether patents act as a barrier to scientific innovation. But evidence points to at least two corrosive effects of intellectual property in this area. First, ownership rights that cover a wide array of technologies and basic materials in any one area tend to deter future research, especially if the costs of obtaining licenses and the conditions imposed on users are prohibitive. Gene and organism patents are a “toll booth” through which future scientists must pass, and in that sense create an anti-competitive environment. In contrast to the 1980s, when biotechnology patenting focused on deliverable commercial goods, claims are today laid increasingly on “upstream” materials, such as basic research tools, which affect the conduct of a large amount of laboratory work.
In the case of the OncoMouse, DuPont’s restrictions have curbed access to a key vehicle for emerging gene-related cancer treatments (such as synthetic proteins). The higher the costs of obtaining this model organism, the more biomedical innovations will be impeded, as researchers in the early stages of their work may choose to look elsewhere, not willing to pay steep up-front costs or abide by unyielding restrictions.Second, patents reduce incentives to disseminate results of biomedical research into the public domain. Since the passage of the Bayh-Dole Act in 1980, which enabled universities and non-profits to retain intellectual property rights to federally-funded inventions, the number of patents claimed by universities has grown from 250 to 31000 per year. Biotechnology now accounts for half of all patents filed by academic scientists. In this era of corporate entrepreneurship, the primary mission of universities — the dissemination of knowledge — is increasingly at risk. Secrecy and under-communication become the norm as faculty members withhold data from the scientific community to protect proprietary interests. Whatever the ultimate fate of the OncoMouse, this and other cases should move policymakers to rethink current patent and commercial licensing laws. A full ban on the patenting of genes and organisms remains an important central goal. Other proposed reforms include clarification of the “experimental use” exemption, which would allow university researchers to freely acquire and use patented inventions; a ban on patents for specific clinically useful technologies; and new policies to ensure that licensing agreements meet standards of fairness. These measures would help to revive the openness and accessibility of science, and so ensure that intellectual property not impede important biomedical developments.

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