Stem cell sector moves ahead full speed in US

Many states have followed suit, increasing funding for stem cells and challenging California’s lead as a stem cell haven. “During the Bush administration there was more opportunity in Europe than in the United States. But now we are looking at two continents that are on equal footing. There is tremendous opportunity for [international] collaboration to pool investments and talent,” said Gary Stein, professor at the University of Massachusetts Medical Center and interim director of the International Stem Cell Registry, an affiliate of the Massachusetts Stem Cell Bank http://www.umassmed.edu/10_26_07.aspx.

The Obama administration established a series of criteria for human embryonic stem cells that could be supported by federal funds, and that has made more stem cell lines available. “That made a tremendous difference”, Stein said.

Challenging California

On a state level, California became the first US magnet for stem cell research when it passed the California Stem Cell Research and Cures Act of 2004, a 10-year, $3 billion stem cell initiative funded by bonds and with money dolled out by the California Institute for Regenerative Medicine (CIRM). But other states have been following suit to challenge California, encouraged by the promise of federal support for the fledgling industry in the run-up to the US election.

The Massachusetts Life Sciences Center, for example, approved $695,000 to continue funding the registry and bank, to which it had earlier awarded $8.2 million in start-up capital (for more information, see their press release). The centre administers the state’s 10-year, $1 billion life-sciences initiative.

Over the previous decade the climate for US stem cell research was quite different, limited by both state and federal regulations and the number of available lines of embryonic stem cells available. Even in Massachusetts, a global hotbed of biological research, scientists and companies hemmed in by state regulations looked to move to California or Europe, where they could continue their research.

“There were 21 lines approved with the Bush administration, but in reality you could only work with about seven lines. You need sufficient diversity of cell lines and applications for regenerative medicine,” said Stein. “I’m confident people or companies left or carried out stem cell research outside the state of Massachusetts,” he added, though he said it is difficult to put a number on how many people took flight.

The change in federal and state acceptance of stem cell research has been dramatic and tremendous, according to Stein. Obama’s executive order reversed former President George W. Bush’s 9 August 2001 directive that banned federal funding for research into stem lines created after that date.

In Massachusetts, Governor Duval Patrick has delivered on his campaign promise to support stem cell research as part of the state’s economic recovery, Stein said. And that’s begun to bear fruit. This week, BioCell Center, an Italian company that cryopreserves human amniotic fluid-derived stem cells, set up its first US office near Boston for work on amniotic and other mesenchymal stem cells. The company already has a collaboration with Massachusetts Eye and Ear Infirmary that is seeking grant money from the Italian National Center for Research, said Kate Torchilin, CEO of the new Biocell Center Corp. in Medford, Massachusetts. And in the future, it may expand to the west coast of the US.

“Our vision is to be a global company,” said Torchilin of the decision to set up a US base. “When we looked at the US we looked for a place with a strong medical community combined with research institutions, including stem cell research.” Massachusetts was high on the list, also because it had a more favourable time zone with Italy. The company also looked at Florida, North Carolina and other locations.

She added that since Obama changed the rules of how to conduct research in embryonic stem cells, there are, “Much broader possibilities for what can be preserved and possibly used for therapy. The biggest difference is what materials can be used.” Like Stein, she said the stimulus package has increased the level of grant-making. In the last several months, funding has been higher for clinical and translational research.

An eye toward the NIH

Even with the relaxed regulations in the US, stem-cell research remains high risk and sensitive. In August, for example, Geron Corp.’s much-publicized stem cell clinical trial for its GRNOPC1 spinal cord injury therapy was put on hold by the US Food and Drug Administration pending that agency’s review of new animal study data submitted by the company.

Geron issued a statement saying the animal study showed a higher frequency of cysts than earlier studies, but the cysts were confined to the regenerating injury site and no adverse effects on the animals were seen. The company said it is answering questions for the FDA to try to get the clinical hold released. No human patients have yet been treated in the study. Both Torchilin and Stein said the hold has not had a ripple effect that has affected their stem cell work.

A glitch in the system also held back NIH challenge grant awards over the summer. Stein chaired three reviews this summer. “But we were not able to review the stem cell grants because NIH was revising its guidelines,” he said (for more information, see the NIH Challenge Grants FAQ).

Despite the federal government loosening its purse strings, US states remain in a financial pinch. California’s crisis is among the worst, but Massachusetts also has wide budgetary shortfalls. Stein said he is confident that Massachusetts will continue to commit the promised funds, as the governor has been present at life sciences events, touting the sector as a vehicle to economic recovery.

California, while stumbling under oppressive economic shortfalls, is still moving ahead, with CIRM recently announcing a stem cell collaboration with the German Federal Ministry of Education and Research.

The biggest chunk of cash may still be coming from the NIH. The American Recovery and Reinvestment Act granted $8.2 billion in extramural funding to the NIH, and states are salivating to see how much of that will be tagged for stem cells, and more specifically, for their own projects.

The University of Michigan has already received 13 federal stimulus grants worth $6.8 million for stem cell research. That state’s voters last year agreed to change state law to let people donate embryos left over from fertility treatments for scientific research. Sean Morrison, director of the university’s Center for Stem Cell Biology, said, “[This] made it possible for millions of dollars in new resources to flow into the University of Michigan to be invested into ... stem cell research."

Massachusetts also is zealously eyeing the funding. “Massachusetts gets more per capita in NIH funding than other states,” said Angus McQuilken, spokesman for the Massachusetts Life Sciences Center. “So the next big step is further distribution of that $8 billion, and what is going to stem cells.”

McQuilken added that European companies can get access to those funds through collaborations with Massachusetts and other US companies. “Co-investment is a key way to get access to the ARRA funds,” he said, encouraging those interested to contact his agency.