Stem Cell Colonies' Viability Unproven
Some in NIH List of 64 Termed Young, Fragile

By Ceci Connolly and Rick Weiss
Washington Post Staff Writers
Tuesday, August 28, 2001

At least one-third of the 64 embryonic stem cell colonies approved for funding under a new Bush administration policy are so young and fragile it remains unclear whether they will ever prove useful to scientists, several researchers working on the cells said yesterday.

In fact, at least 16 of those colonies have been subject to so little research that the Swedish scientists working on them are unwilling to claim they are embryonic stem cells capable of becoming any kind of human tissue.

When President Bush announced Aug. 9 that he was willing to fund research only on existing stem cell colonies, or "lines," he and his top health adviser said more than 60 lines existed worldwide -- more than enough, they said, to launch a new and vibrant era of medical research. The existing lines, Health and Human Services Secretary Tommy G. Thompson said at the time, "are diverse, they're robust and they're viable for research."

But interviews with several scientists whose cell lines were identified yesterday by the National Institutes of Health as meeting the Bush criteria raised questions about whether many will live up to that advance billing.

At Goteborg University in Sweden, which the NIH identified as home to the world's largest collection of embryonic stem cell lines -- about 19 in all -- a key scientist called into question the NIH's numbers.

"I was a little surprised to see the NIH calling them 19 lines," said neurobiologist Peter Eriksson, part of a six-member team developing stem cell lines there. "Maybe they misinterpreted a little bit."

At most, he said, three of the 19 batches of cells could be called stem cell lines.

The largest cache of embryonic stem cells in the United States belongs to CyThera Inc., a 12-person biotechnology firm in San Diego that is at least several months from providing a colony to researchers, officials said.

"The last thing the NIH wants and the last thing we want is to give somebody a half-baked research tool that they can't use," said Michael Ross, CyThera's president and chief executive.

At Reliance Life Sciences in India, four of the seven cell lines included in the NIH tally have barely cleared the first hurdles in the long process of proving their identity and usefulness as stem cells. The three remaining lines are even younger and could easily "peter out," said Firuza Parikh, founder and director of the Bombay-based research firm.

"We are in the very initial stages," Parikh said. "We still need to characterize these lines."

William Pierce, a spokesman for the Department of Health and Human Services, said it may be too early to judge the quality of the existing cell lines and emphasized that scientists are eager to begin work on them. Even if some of the 64 lines do not last long, he said, they could still provide worthwhile information for scientists and further Bush's goal of pursuing "basic research."

"No one should be under the illusion that cures for diseases are just around the corner, for there is much fundamental work to be done," Thompson said in a written statement.

Human embryonic stem cells are primordial cells retrieved from five-day-old human embryos. The cells have the potential to turn into all kinds of tissues in the body. Since their discovery three years ago, they have attracted intense interest from biomedical researchers, who hope to fashion the cells into replacement tissues to treat diabetes, Parkinson's disease, spinal injuries and other degenerative syndromes.

Bush said he pinned his decision on assurances that at least 60 colonies of eligible cells existed -- a number far greater than anyone in the field was aware of and well beyond the handful documented in scientific journals. The president said he was unwilling to permit federal funding of research on other stem cells because that would involve the destruction of new embryos.

Yesterday, at the request of lawmakers, scientists and the media, the NIH released the first details about where the cell lines are and what criteria were used to include them. All told, the 64 lines are mostly young, temperamental colonies that may well thrive and lead to new medical discoveries, but which remain largely untested and fragile.

Administration officials acknowledged it will take some time to gather a clearer picture of the available cells but encouraged scientists to begin applying for grants. "Now it's time to go to work," Thompson said.

Reaction to yesterday's revelations was guarded as those in the pro-research camp continue to view the Bush approach as an opening -- albeit a narrow one -- to pursue lifesaving therapies. Sen. Edward M. Kennedy (D-Mass.) described the NIH list as a "first step in understanding" the new policy. But he vowed to press for more information in a hearing next week.

Tony Mazzaschi, associate vice president for research for the Association of American Medical Colleges, said, "We are very pleased NIH has provided us with the list. . . . If NIH tells us 60 lines exist, 60 lines exist."

But he said the list does not resolve "critical questions." The remaining ambiguities include whether the cell lines "are usable, whether they are available, whether they are robust," Mazzaschi said.

The Coalition for the Advancement of Medical Research, representing several patient advocacy groups, said yesterday that it "is concerned about possible restrictions placed on the use of the lines by their owners as well as about the scientific viability and status of existing lines."

Most of the 64 lines are controlled by private, for-profit companies or foreign labs. The administration has been trying to negotiate a standard agreement that would permit researchers to experiment on a cell line owned by someone else.

"At this time, we have only begun conversations with the patent holders," Ruth Kirschstein, acting director of the NIH, wrote yesterday to Kennedy.

In some cases, the embryonic stem cells were acquired from donors through methods previously prohibited by the NIH. Some lines held by the University of Wisconsin and several at Life Sciences in India were extracted from fresh embryos, a technique the NIH previously rejected as unethical because donor couples are targeted at a particularly vulnerable moment in their fertility treatment.

Eriksson, the Swedish scientist, said his group has three lines that have been alive for about six months but have yet to prove that they can turn into all the major cell types of the body -- a key test that cells must pass before they can be regarded as stem cells. The group has 12 colonies that have been alive for less than three months and which he calls "potential cell lines." Four other colonies of cells are in the freezer, he said, waiting to be analyzed.

All told, Eriksson said, "it's a little bit exaggerated to say we have 19 lines. I think [the NIH] probably kept a relatively low bar."

Many of the cell lines covered under the Bush policy are controlled by small companies that had no idea until recently that they would be expected to supply stem cells to the entire U.S. research establishment.

In general, they say they are willing to fulfill the role but caution that they are months, at least, from being able to do so. The companies are willing to make few claims about the value of their cells for long-term research, much less their usefulness in curing disease. Generally, the most they would say is that their cells can serve as a starting point.

Ross and another CyThera executive, Lutz Giebel, said years of work remain.

"I think the scientific community over the last year or so has raised a lot of unrealistic expectations in the general public," Giebel said.

Staff writers Justin Gillis, Amy Goldstein and Nicholas Johnston contributed to this report.

© 2001 The Washington Post Company