Patrick's $1b offer a temporary boost
By Scott Allen, Globe Staff
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."
Scott Allen can be reached at allen@globe.com. 