By Raja Mishra, Globe Staff
It was the White House's first major science policy decision. In a nationally televised speech in August, President Bush agreed to provide government funding for human embryonic stem cell research, hoping to spur new treatments for afflictions such as Alzheimer's disease and diabetes. However, he strictly limited researchers to 78 existing stem cell batches in order to avoid funding the destruction of human embryos necessary to obtain the cells, saying that amount was adequate to jump-start research.
Nine months later, a Globe survey found that nearly three-quarters of the 78 stem cell batches that met Bush's conditions for support remain unavailable to US researchers, caught in a web of political, financial, and scientific complications that continue to present obstacles to research progress.
Some of the 14 institutions worldwide with stem cell batches, or lines, lack the resources to ship cells to scientists. Others simply have not grown and nurtured their cells to the stage needed for research. Meanwhile, the government of India has forbidden the export of 10 stem cell batches until it develops a national policy for doing business on this new medical frontier.
And several private labs that own embryonic stem cell lines said that acting as a de facto cell distributor for the US government did not make financial sense, despite all the talk that these highly versatile cells found in week-old embryos hold enormous healing potential.
''It's not necessarily in the interest of our shareholders to spend money on'' distributing embryonic stem cells, said Dr. Allan Robins, chief scientific officer of BresaGen, an Australian biotech company with a US headquarters in Athens, Ga., that controls four stem cell lines on Bush's list.
The survey provides evidence of the steep learning curve faced by the federal government in this controversial new wing of biology. So far, no federal grants dedicated to medical research using embryonic stem cells have been awarded. Only nine grant applications are pending, with decisions expected later this month.
''We had hoped that there would be lots and lots of grants; the sooner more people get started the better off we'll all be,'' said Dr. Robert Goldstein, chief scientific officer of the Juvenile Diabetes Foundation, a private nonprofit organization that funds $2 million of embryonic stem cell research on its own.
The stem cell shortage hampers short-term research progress, said scientists, especially for university-based scientists who cannot easily afford to buy cells privately. But the entire field remains in its earliest stages, they said, and it's not surprising that disarray exists as the US government attempts to become a world coordinator of embryonic stem cell science. Most of the 78 lines could be available to scientists within a year, allowing research to accelerate, they predicted.
Indeed, many of the institutions currently unable to supply cell lines to researchers are working to rectify the situation. For instance, Dr. Jihwan Song at Pochon CHA University College of Medicine in Seoul said their two batches should be ready in ''two to three months.''
In addition, the wheels of the US government's scientific machine are clearly beginning to turn. The National Institutes of Health recently allowed 12 taxpayer-funded researchers to modify existing grants to include limited embryonic stem cell work. On April 26, the agency approved a $3.5 million, two-year infrastructure grant intended to help four institutions with cell batches to get supplies in the hands of researchers.
''I think it will pick up over time,'' said Dr. George Daley, a stem cell researcher at the MIT-affiliated Whitehead Institute, who has an NIH grant proposal pending. ''But access to the cells has to increase.''
Stem cells appear within an embryo several days after fertilization. They divide and transform into every body tissue. Medical researchers hope to use them to repair damaged tissue caused by dozens of afflictions, such as Parkinson's disease, spinal injuries, and heart disease. The cells can replicate indefinitely, allowing a single embryo to give rise to a long-lasting batch. Scientists often get the cells from excess embryos from fertility clinics, which otherwise would have been disposed. But the embryos perish after their stem cells are plucked out, raising serious ethical questions for many.
Stem cell science intertwines with another controversial technique: cloning. Advocates of human embryonic cloning hope to clone patient cells, then remove stem cells from the new embryo to create replacement tissue genetically matched to patients, largely eliminating the frequent problem of tissue rejection.
For the moment, the stem cell political debate lies dormant, but the Senate plans later this spring to vote on a bill that would criminalize embryo cloning. The merits and promise of stem cell science appear certain to play prominent roles in the debate, but judging the field's promise is difficult given the early stage of much of the research.
''We've understood from the beginning that progress in this field would be slow at the outset,'' said NIH spokesman Donald Ralbovsky. ''It takes a long time to scale up.''
When asked about the Globe survey findings, he said: ''We feel that there are a sufficient number of lines available. ... There are enough right now to cover the work that's being done.''
However, there are clear signs that researcher requests for stem cells are going unmet at the 14 companies and institutions that control the 78 stem cell lines. BresaGen has four healthy stem cell lines at its Georgia facility. But the company has had to ignore the 70-plus inquiries it has received from scientists around the world.
''We don't have the resources to deal with them,'' said chief scientific officer Robins.
BresaGen was one of the recipients of the NIH infrastructure grant, and Robins said he will use the money to hire three full-time technicians to care for and, eventually, distribute the cells.
The situation is more complex at San Diego-based CyThera, where company officials and Dr. Jeanne Loring, their former scientific collaborator, disagree over what to do with their nine frozen lines.
''My goal now is to get the cells in the hands of someone who can do something with them,'' said Loring, adding that she froze the cells at a very immature stage of development and that it's too early to say if they can be used in research. ''They could all be no good,'' she said.
CyThera officials did not return messages.
Another company that owns stem cell lines, Geron Corp. of Menlo Park, Calif., can legally share its seven lines only with Geron-affiliated scientists because of an agreement with the University of Wisconsin's patent agency. The Wisconsin Alumni Research Foundation won the patent to embryonic stem cell harvesting after Wisconsin scientist James Thompson produced the first-ever human embryonic stem cells in 1998. Geron funded his work.
A subsidiary of the Wisconsin patent agency, WiCell Inc., remains the only US institution on the NIH list actively supplying researchers. Forty-four scientists at 36 institutions got cells from them, according to a Wisconsin Alumni Research Fund spokesman, and 69 more scientists will be getting supplies shortly.
Although US researchers prefer the simplicity of obtaining cells in the United States, most of the lines on the federally approved list are in other countries, forcing them to deal with additional complications.
For instance, three cell lines at Maria Biotech Co. Ltd., in Seoul, cannot be handed out until the Korean government gives a green light, said Keidy Yang, general manager.
Similarly, 10 cell lines in India cannot be exported without approval from the Indian government. ''We've gotten a few requests, but we've told them to wait,'' said Dr. Mitradas M. Panicker, who manages three lines for the National Centre for Biological Sciences in Bangalore.
NIH officials visited India in November to push the government to open the lines to foreign researchers, and one US official said they expected an agreement later this year.
This story ran on page A1 of the Boston Globe on 5/12/2002.
© Copyright 2002 Globe Newspaper Company.