By Randall Osborne

Editor

In biotech, the issue of how far to keep public enterprise apart from private scientific research is becoming almost as hot as the separation between church and state.

The large-scale debate began with Celera Genomics. When sequencing trailblazer Craig Venter founded that company to compete with the government-funded Human Genome Project in mapping the human genome, eyebrows rose.

There was nothing illegal about it. But, while market players lined up to get a piece of Celera, bystanders clashed over whether the federal effort might be better left to work at its own pace with taxpayer dollars, rather than being required to race a heavily funded private push.

The questions specific to Celera, of course, eventually fell away. Big profit was to be made by the company's investors, and it was difficult anyhow to argue with the notion that, when critical human genome data is at stake, philosophical battles seem silly especially since both entities promised to make their raw information public, sooner or later.

Enter Boston University. Although not allied with Celera, the academic institution finds itself at the crux of the "public vs. private" skirmish, a spot once occupied by Celera.

Boston University said it has aligned with a handful of venture capitalists who have committed $21 million to form a company called Framingham Genomic Medicine Inc., using patient data from the 52-year Framingham Heart Study, which was funded mainly by the government through the National Heart, Lung and Blood Institute.

To some, it sounds like trouble.

Just as the Human Genome Project made public the map data as it gained them, the Framingham study has offered free of charge its findings on 10,000 subjects in that Massachusetts city.

Not all of the Framingham study is "public" in the most liberal sense. Although information such as smoking habits, red-meat consumption and balding patterns is readily available, scientists must submit paperwork for more detailed and significant data, including DNA samples from 5,000 Framingham patients, and family histories. But it's theirs for the asking.

Framingham Genomic Medicine aims to use data from the heart study, which will be digitized along with findings from the human genome map, to figure out DNA variations that make cardiovascular disease more likely, along with variations that make drugs fail.

And, of course, the company aims to make money.

The financial implications for other firms are plain. Will those involved in cardiovascular research, in order to compete, have to find a university that sponsors government-funded experiments like Framingham's?

If so, they're out of luck. Nothing like it exists.

Boston University's claim is that it wants only to make the widest and best use of the heart data, and needs cash from venture capitalists to make that happen. Skeptics worry that, if the newly formed company gets exclusive access (or just wider access, without requirements imposed until now), then public money in effect will have funded a private firm. Couldn't this damage the chances of others, and flatten competition overall?

It's not an issue, said Fred Ledley, chief scientific officer of Framingham Genomic Medicine and, so far, the company's only employee. Anything done by the university as part of its contract with the government as a sponsor of the study belongs in the public domain, he said. On the other hand, any findings derived from the Framingham data that is, findings that came out of research done by the university through a government grant belong to the university itself.

"The whole foundation of the biotech industry is that, when a university gets a grant and does research, it has the right actually the obligation to commercialize that for public benefit," Ledley said.

With 15 schools and colleges, including the School of Medicine and the Center for Advanced Biotechnology, Boston University is hardly a stranger to agreements with private enterprise. Its Community Technology Fund (CTF) is designed partly to help start spinout firms from academic inventions.

In June, UK-based Oxford BioMedica, which licensed the family of cytochrome P450 enzymes for cancer gene therapy, began clinical trials of the technology, which was developed by Boston University, Massachusetts General Hospital (MGH), and the Dana-Farber Cancer Institute. Including the BioMedica deal negotiated by MGH and Boston University, the British firm is paying an up-front fee, milestones and royalties.

Such deals let entities such as those who started the P450 work avoid violating the rule adopted earlier this year by the American Society of Gene Therapy, which bars members from getting involved in human experiments by firms in which they have equity. The rule is another safeguard against public and private overlap (and against experiments that are less than maximally safe which has become a particular concern in gene therapy trials).

In February, the Boston University School of Medicine paid Sequenom Inc. an undisclosed sum to use the company's high-throughput, single nucleotide polymorphism system, launched late last year, for its work in mutation analysis.

But the new company is by far the largest foray by Boston University into the realm of private enterprise. It is the one that's drawn the most scrutiny, too, especially in the popular press, which already has misreported the facts of the venture, said university spokesman David Lampe.

"We're not selling the data," Lampe said. "It really can't be sold. What we're selling is an analysis." The company will use high-throughput analysis technology to digitize the data and analyze it for pharmaceutical and biotech customers.

"A lot of it's in the form of things like boxes of X-rays in basements. We'll update the entire database to make it more accessible than it ever has been," he said.

Ledley said the Framingham study "began before computers were invented, and I don't mean that facetiously. There's probably half a million images of X-rays and electrocardiograms" to be digitized, he said. "For 50 years, we've had physicians making notes in the margins."

Former CEO of Variagenics Inc., a pharmacogenomics company based in Cambridge, Mass., Ledley said the new firm (in which investigators at Boston University are not allowed to invest) has pledged to make no exclusive deals; to establish a charitable trust for the town of Framingham; and to form an ethical advisory board so that the data remain confidential.

The company will spend $10 million to $12 million upgrading research tools, which Ledley expects will be available in nine months. About $20 million will be spent creating a "dense genotype for each subject in the study," he said, and then providing the analysis service can begin.

"I believe every pharmaceutical company will have to have this," he said.

Meanwhile, officials from the university and the government will be sitting down to decide what belongs to whom that is, which data are original to the heart study as part of the contract work done by the university, and which findings are "derived."

Even university spokesman Lampe admits the task may be daunting.

"There are other twists and turns," he said. "Suppose you genotype some of the tissue samples. Who owns that data? In a sense it's derived, and in a sense, it belongs to the heart study."

Everyone's goal, he insisted, is to preserve and continue the study, keeping its data complete as well as readily available. "It's the goose that laid the golden egg," Lampe said. "We want to make sure it keeps laying golden eggs."

Ledley said backers of the new firm are hoping for gold, too. They include Boston University's CTF; Abingworth Venture Management Ltd.; Domain Associates; Venrock Associates; and S.R. One Ltd. The first round of funding, which totaled $6 million, was led by Oxford Bioscience Partners.

"This is a big-concept company," Ledley said. "Celera and Incyte [Pharmaceuticals Inc.] have gene sequences, and that's very important, but what people are invested in is the relevance of those. You need complementary databases that integrate the same attention to detail about people's lives that you have concerning the chemical structure of their genes. It's what people really want out of genomics." *

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