By Steve Sternberg

Special To BioWorld Financial Watch

Not long ago, academics who studied embryonic stem cells feared that a ban on government funding for embryo research would cost them their careers, asserted Michael West, CEO of Advanced Cell Technology Inc. in Worchester, Mass.

"Back in the early '90s," he said, "you'd be in scientific meetings with cell biologists who were talking about their work and plans for the future. There'd be discussions about embryonic stem cells, but the conversations would be whispered in hallways because people were really concerned about their jobs, about being labeled embryo researchers."

No longer, he said, thanks to new National Institutes of Health (NIH) guidelines that open the door to federal funding of embryonic stem cell research. "What you see now," West said, "is an avalanche of academic researchers coming forward and saying, 'I've been wanting to do this work for the last 15 years.'"

The guidelines, released Aug. 23, still prohibit funding for the derivation of human pluripotent stem cells, but they permit funding for research that uses the cells once they've been collected. Researchers seeking government support for embryo research must assure the agency, however, that the cells were derived from unused embryos created in fertility clinics an abundant source of frozen embryos.

A new NIH committee, the Human Pluripotent Stem Cell Review Group, will review grant requests to make sure they comply with the new guidelines. The group also will hold public hearings when a researcher proposes a novel use of stem cells that has not previously been reviewed.

If the next president doesn't overturn the guidelines, they'll promote competition and collaboration between industry and academic scientists, and that's how it should be, West said. "There's no way that one company or all of the biotech companies working together could develop these cells in all of their potential applications," he said.

"There are over 200 cell types in the body. To develop all of these cells, you would need at least 200 separate programs. NIH funds thousands of researchers. So it comes down to a very simple equation. The research can only be done adequately in the public sector. We can't possibly do it alone."

Human pluripotent stem cells promise to revolutionize medicine because they are the source of a host of specialized cells that could be used to patch or replace damaged tissues throughout the body. The list of ailments that could be treated or cured using stem cell technology would fill a textbook: heart disease, stroke, burns, spinal cord injury, diabetes, osteoarthritis, rheumatoid arthritis, and Parkinson's and Alzheimer's diseases.

Indeed, experts say, stem cells even promise to change the trajectory of medical research into a whole range of diseases. Scientists who once would have been satisfied to search for ways of treating diseases now will explore ways to cure them.

Adult Stem Cells Skirt Political Controversy

"The hope for us and probably for others in this field, is moving medicine from treating disease to curative platforms. That is something that these stem cell therapies offer," said Tad Heitmann, vice president for corporate communications at Nexell Therapeutics Inc. in Irvine, Calif.

Not everyone agrees, however, that pluripotent stem cells are the way to go. Multipotent stem cells derived from adult tissues also offer great promise without the potential for political, religious and social upheaval generated by embryo research. "We view true therapeutics based on embryonic stem cells as something for the future -- if ever," said Heitmann, whose firm focuses its research efforts on hematopoietic, or blood-forming, stem cells.

A biotech analyst who asked to remain anonymous said, "You're talking about harvesting embryos. That's obviously a very contentious point, and one we are unlikely to reach consensus on anytime soon."

If the experience of those at Stem Cells Inc., of Sunnyvale, Calif., can serve as a guide, investors don't harbor the same reservations about plunging into embryonic stem cell research as some of the scientists. George Dunbar, the firm's CEO, said the NIH announcement has brought in a flood of calls from investors wanting to hear about the company and its technology.

"We've had more calls from people saying we've heard about stem cells, but we didn't know what they are and now we understand," he said. Not only that, Dunbar said, investors are more willing to bet on the technology. The firm's stock, which was "flat-lined" at about a dollar a share for most of last year, soared to $10 or $12 right after the announcement, Dunbar said. It has since settled to about $8 or $9 per share, still a dramatic improvement.

Dunbar added that the NIH guidelines are not as liberal as those recently put forth by the British Science and Ethics Commission, which recommended allowing human therapeutic cloning. But he said that should come as no surprise, because U.S. research regulations have long offered less latitude than those in European nations.

"It has always been that way," he said. "Any product usually gets to market faster in the UK and Europe than in the U.S. or Japanese markets."

Research Could Move Overseas If Politics Get Dicey

Should a future political administration choose to reverse the new guidelines, Dunbar said, it won't put him out of business; he'll simply move his research to Europe. "That will be really disappointing to U.S. patients, because they won't be able to get access to products or trials in the U.S. that are available to patients in Europe.

"I can imagine the public outcry, when high-profile public figures, like Christopher Reeves, Michael J. Fox, Janet Reno and President Reagan, come to Washington and say: 'How dare you keep us from these potential cures?'"

West said placing hopes in pluripotent stem cells is more than justified. "We have in our hands the mother of all stem cells," he said. "This cell has almost magical properties and does things that we've seen no other cells do. This is the beginning of whole new era of medical research. It needs to move forward purely for the benefit of you and me, our families and our friends. We'll all be needing important health care in the future." *

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