Commentary: Stem Cell Science Needs More from Uncle Sam
By Catherine Arnst
In the late 1960s, Barnett Rosenberg, a professor at Michigan State University, was conducting experiments to determine if electromagnetic energy could stop the growth of E. coli. By pure accident, he discovered that platinum from the electrodes stopped cell growth in the bacteria. Rosenberg brought his discovery to the National Cancer Institute, whose scientists did all the work to turn it into a drug and conducted all clinical trials. Ultimately, Michigan State, which held the patent, licensed it to Bristol-Myers Squibb Co. (BMY ) for marketing. That drug, cisplatin, was approved in 1978 and remains one of the most effective cancer drugs ever.
The story of cisplatin is a case study of the value of basic research done for the joy of discovery rather than the sake of the bottom line--and illustrates why so many scientists are dismayed about President George W. Bush's recent decision to place restrictions on federal funding of stem cell research.
DEEP POCKETS. The U.S. government has long had the world's deepest pockets for basic scientific research. But the White House ruled that scientists would have access to that money only if they limit their research to 64 human embryonic stem cell lines already in existence. Many scientists fear that these restrictions will slow progress to a crawl in this promising area of medicine. "Basic science is the only way to do it," warns Robert S. Langer, professor of chemical and biomedical engineering at Massachusetts Institute of Technology. "It's absolutely essential that we have government-funded research, and let's face it, most of the important research comes from the U.S."
Plenty of countries might argue otherwise, but they would certainly agree that their annual research budgets don't come close to matching the National Institutes of Health's 2001 outlay of $18.8 billion. There is still the private sector. But commercial enterprises have never shown much interest in funding expensive basic science that won't pay off for decades--if ever--and stem cells are no exception. Rochelle K. Seide, a patent attorney who heads the biotech practice at BakerBotts LLP in New York, says she knows of no large pharmaceutical company doing work in this area. And, she adds, the small companies that hold patents on stem cell lines are unlikely to do broad-ranging studies. "If they are interested in cardiovascular therapies, they won't be doing neurological research."
Nor, says Seide, is it likely they will license their lines to potential competitors. Already, two of the private companies that developed some of the 64 stem cell lines approved by the NIH, CyThera Inc. of San Diego, and Reliance Life Sciences P.V.T. Ltd. of Bombay, have said they are not willing to make them generally available just now.
That's not surprising. If a commercial enterprise is going to spend the hundreds of millions of dollars required to derive a viable commercial product from a stem cell line, it would not be inclined to share its discoveries. "If all this work moves to the private sector, that means any positive data will not be available to other scientists for years," says Dr. Evan Y. Snyder, an associate professor of neurology at Harvard Medical School. "And the public may never hear about negative data."
EXORBITANT. And then there's the question of profit. It'll be at least a decade before any therapy based on embryonic stem cells is ready for human trials, and those could take another 10 years or more. It already costs from $150 million to $500 million to bring a new drug to market; most of those, like cisplatin, were based on basic research done in university or government labs. If stem cell research works, the resulting therapy "will be exorbitantly expensive because companies will need to recoup their investment," says Snyder. "Only the rich will be able to afford it."
A case in point is Novartis' (NVS ) breakthrough new cancer drug, Gleevec. Unlike cisplatin, Gleevec was developed completely by the company over 10 years. The drug finally reached the market in May--at a price to the patient of $2,300 per month.
The solutions to most of the world's most vexing scientific problems are far too large for any one company, or government, to solve. The human genome would not have been deciphered in a decade without the support of the U.S. government, for example, and stem cell research is much the same. Let's hope the President rethinks his position before too much time is lost.
Senior Writer Arnst covers science and medicine for BusinessWeek.