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Odds high, funds sparse for stem cell researchers

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Patrick’s $1b offer a temporary boost

For all the hype and hope surrounding stem cell research, most of the companies trying to develop treatments from these powerful cells live in a place Governor Deval Patrick this week called the “valley of death.” It is a harsh place where neither the federal government nor private investors provide much support and small firms with limited funding struggle to figure out how to harness stem cells’ extraordinary power.

No one knows that better than Dr. Thomas Okarma , whose company, Geron Corp., hopes next year to start the nation’s first human tests of a treatment derived from embryonic stem cells. The California company has already spent years developing OPC1 as a possible therapy for spinal cord injuries. Researchers have had to inject 2,000 animals to show it was safe, grow the cells 75 times over to prove they could do it, and invent a needle to inject cells into the injury site. Even if human tests go well, federal approval probably won’t happen for years. If tests go badly, foes of embryonic stem cell research are sure to pounce.

“The challenge . . . is not for the faint of heart or the light of purse,” said Okarma, who says his company has invested more than $100 million in stem cell research to date.

The $1 billion life sciences initiative Patrick announced this week could provide a boost for stem cell companies that locate or start up in Massachusetts, in part by providing stable support through the many years it will take to realize the promise of stem cell treatments. Saying he intended to make the state the “capital of stem cell research on the planet,” Patrick wants to set up an embryonic stem cell bank at the University of Massachusetts that would greatly reduce storage costs for the delicate cells and expand researchers’ access to different types.

He proposed grants for lab equipment that could be used to work with the embryonic stem cells scientists are banned from studying with federally supported lab instruments, and grants to keep promising researchers from leaving the state.

The proposal would also provide a hand up from the “valley of death” in the form of short-term funding for companies to develop ideas until they can attract private investors, and possibly an actual “incubator” building where new stem cell companies could be housed. Many details of the 10-year plan remain to be decided, including how the money would be divided between stem cell research and other sciences, but stem cell researchers have been overwhelmingly positive about the assistance.

“This is really a long-term investment in Massachusetts not losing its premier role in life sciences,” said John Auerbach, state public health commissioner.

But, even with the burgeoning aid from Massachusetts and other states, biotech executives at the BIO 2007 conference in Boston this week made it clear that researchers still face daunting hurdles in learning how to grow and manage both adult and embryonic stem cells and to get them to do something medically useful. Embryonic stem cell research is less than a decade old, they pointed out, and new fields of medicine typically take 20 years or more to produce results. One danger, several top stem cell scientists said, is that cash-strapped firms will rush into human testing before they answer basic questions, with potentially disastrous results.

Many stem cell pioneers “are dealing with things they don’t know enough about to begin with, and then they’re adding stuff to it,” said Nancy Parenteau of Vermont-based Parenteau BioConsultants, referring to the growth enhancers and other chemicals that are used to manipulate stem cells. She noted that some researchers don’t even know where the stem cells go once they’re injected into a patient. She said the Massachusetts initiative could be great for the whole field if the state spurs more research on how stem cells work and why.

The Massachusetts investment could also discourage firms from following the lead of Advanced Cell Technology, a stem cell company that moved its corporate headquarters from Worcester to Alameda, Calif., a year and a half ago, partly to take advantage of California’s Proposition 71, which made $3 billion available for stem cell research in that state over the next decade.

William M. Caldwell IV, Advanced Cell Technology chairman, called Patrick’s move “long overdue,” after former governor Mitt Romney’s opposition to embryonic stem cell research. Now, Caldwell said, Massachusetts could have “the most user-friendly state from the standpoint of research and commercialization in the country.” The company still has researchers in Worcester, and Caldwell said his firm may expand activities there.

Compared with most states, Massachusetts has a vibrant stem cell research community, led by the Harvard Stem Cell Institute, which has 45 principal faculty members and more than 600 employees involved in stem cell research at Harvard-affiliated hospitals and labs. But the number of companies in the state trying to develop treatments from adult or embryonic stem cells remains tiny, a problem Caldwell sees across the country. “It’s like a desert,” he said.

Embryonic stem cells have inspired hope and controversy since University of Wisconsin researchers first isolated them in 1998. The cells are the body’s master cells, capable of becoming any kind of tissue, raising the possibility that they could be coaxed to create replacement tissue for diseased and damaged organs. But some argue that the harvesting of stem cells from fertilized human embryos is unethical because it requires the destruction of the embryo . In August 2001, President Bush sided with critics, banning US funding for research on embryonic stem cells harvested after that date.

As a result, unlike those in other new scientific fields, embryonic stem cell researchers got almost no boost from the federal government: Nationally, they received only $122 million from the National Institutes of Health from 2002 to 2006, roughly the amount Geron alone has spent on stem cell research. NIH did provide $799 million over the same period for stem cells taken from adults, but the requirement that older and newer embryonic stem cells be strictly segregated discouraged many researchers from entering the stem cell field at all. And overall NIH funding for stem cells has not increased for three years, resulting in a steady rise in the percentage of studies that are rejected.

For Geron, dwindling federal support means that whenever the company needs a question answered it has to pay for the research. Fortunately for the company, animal tests of its stem cell therapy for spinal damage, OPC1, have produced some of the most remarkable results yet in stem cell research, consistently restoring rats’ ability to use their hind legs. Geron is now ready to ask the US Food and Drug Administration for permission to begin testing the treatment in humans.

But Goldstein, who was not involved in the OPC1 research, said the clarity of the Geron findings is a rare exception in a field full of ambiguities.

The fear among researchers is that poorly-thought-out experiments could go so badly that the results damage the field much the way the 1999 death of Jesse Gelsinger, a healthy teenager, cast a shadow over another promising treatment, gene therapy.

“The ideological right is not asleep,” said Robert Klein , chairman of the Independent Citizens Oversight Committee in California, which oversees public stem cell funding. He cautioned that opponents of embryonic stem cell research will attempt to exploit setbacks in human trials. “There will be failures as well as successes. We must be patient.”

By Scott Allen, Globe Staff

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