The state of California is preparing to make a bet of up to $40
million on a fast-moving field that promises to revolutionize
medicine and ultimately lead to personalized
stem
cell treatments that can be tailored
for a patient’s genetic makeup.
Directors of the California
stem
cell agency are meeting in Berkeley
on Wednesday to create one or two
stem
cell genomic centers that they
predict will make the state a world leader in the new field.
Scientists and businesses from biotech centers in the
Bay
Area, San
Diego and elsewhere are competing
for the money.
The move into genomics comes as the $3 billion agency struggles
to fulfill the promises of the ballot initiative campaign of 2004,
when voters approved its creation with a total of $6 billion in
state
spending, including the interest on
bonds sold to finance the endeavor. So far, no therapies or cures
have emerged from the
California
Institute for Regenerative Medicine
(CIRM), as the agency is formally known. It will run out of cash for
new awards in less than three years and needs some high-profile
results to raise more money.
Scientists and biotech businesses say they hope that genomics,
the study of genes and their relationships, can lead to a catalog of
disease genes and pave the way for new therapies that are tailored
to individual needs. Linking
stem
cell treatments, which also promise
extraordinary results, could provide even more effective treatments.
UC
Davis stem
cell researcher and blogger Paul
Knoepfler describes the
stem
cell genome effort as part of a
“revolution.”
“Genomics is going to become a key part of all of our lives
whether you like it to be or not,” he says on his blog.
“Right now, in a lot of ways, doctors are making educated
guesses as to how to treat us patients more generally,” Knoepfler
says. “By knowing our genomic information, our genotype – the
information tucked away in our genomes –they could be making far
more educated choices about treatments, and we could be making far
more informed decisions about our health.”
The
National
Institutes of Health says that genes
play a role in nine out of the 10 leading causes of death in this
country. “Genomics is helping researchers discover why some people
get sick from certain infections, environmental factors and
behaviors, while others do not,” the institute says.
The nascent field is not without controversy. The
U.S.
Food and Drug Administration
recently cracked down on the Google-backed genetics firm 23andMe of
Mountain
View, saying that it had failed to
show that its testing produced accurate results. The company last
month said it would stop providing health information with its
tests. The danger to the public, say some medical experts, is that
people might act on inaccurate or poorly understood genetic
information and unnecessarily undergo drastic or harmful procedures
intended to ward off future disease.
Such concerns haven’t slowed growth in the genomics industry,
however. Various studies say that the current annual sales of
genomic products exceed $3 billion and peg the annual growth rate at
anywhere from 10 percent to 17 percent.
The
stem
cell agency two years ago this month
sized up the situation and decided it was time to jump in. The
agency’s governing board gave the go-ahead – on a voice vote
with virtually no discussion – to the concept behind this week’s
awards. CIRM directors had already been primed at the time by a
presentation by Craig Venter, head of the La Jolla Institute bearing
his name and internationally famed for his genomics work. Venter
told the CIRM board that “there will not be any clinical
stem
cell applications without
understanding genomics.”
Venter said genomics is needed to tell whether a particular
stem
cell therapy will cause more harm
than good. Venter also told the board that he already had embarked
on a
stem
cell genome effort. He is believed
to be competing for the CIRM funding, and his talk raised eyebrows
among some researchers because it was so closely tied to the board
action.
The agency opened the door to applications from researchers and
institutions in October 2012, eight months after the talk by Venter,
who appeared at the agency’s invitation. The review of those
applications and the identities of the applicants are cloaked in
secrecy, which is the traditional way scientific grants are awarded
in this country even when they involve public funds.
A combination of out-of-state scientists and six CIRM board
members scores the grants and makes its decisions. The full,
29-member CIRM board will have the final say in a public meeting in
Berkeley on Wednesday, but it almost never departs from the
recommendations for approval by its reviewers. CIRM announces only
the names of the winners and does not release the names of rejected
applicants because it might embarrass them.
Last week, CIRM President Alan Trounson and his staff recommended
funding only one of the applications – for $33 million –
although reviewers had approved four, according to documents at the
CIRM website. No public explanation was immediately provided, except
that CIRM spokesman Kevin McCormack said the reviewers actually “did
not recommend funding all of the applications,” although that was
clearly stated on the website, as has been the practice on the
review of thousands of previous applications.
The funding round is budgeted for $40 million, but could be more
or less depending on the wishes of the board.
A number of the major educational institutions in the state are
likely to be involved in this week’s awards. Stanford University’s
name surfaced last year when a conflict-of-interest violation in the
initial grant review was reported by the California
Stem
Cell Report. CIRM grant reviewer Lee
Hood of Seattle, renowned internationally for his genomics work,
acknowledged that he had failed to disclose his conflict in
connection with a $24 million application involving Irv Weissman,
director of Stanford’s Institute for
Stem
Cell Biology and
Regenerative
Medicine. Weissman and Hood are
longtime friends and own property together in Montana.
The closed-door review also marked the first time in CIRM’s
history that reviewers, all from out of state, failed to finish with
a decision supporting any of the proposals, according to CIRM.
Reviewers’ comments were sent back to applicants, who resubmitted
their proposals for review in November in another closed-door
session. This time, Hood did not participate.
In addition to Stanford, California enterprises that have a
strong interest in genomics and that are possibly involved in the
competition include: Illumina and Sequenom of San Diego, Life
Technologies of Carlsbad, CombiMatrix of Irvine, Pacific Biosciences
of
Menlo
Park and Complete Genomics of
Mountain View, which is owned by BGI, a Chinese business that is the
largest genomics sequencing firm in the world. Others include
Scripps, the San Diego Supercomputer Center at
UC
San Diego, the Novartis Genomics
Institute and Fate Therapeutics, both of San Diego, and
UC
Santa Cruz.
UC
Davis has just begun an $18 million
genome operation in partnership with BGI, but Richard Michelmore,
director of the Davis Genome Center, said it was not involved in any
of the CIRM applications. (Ken Burtis, who is a member of the
faculty of the Davis Genome Center, is a member of the CIRM
governing board.)
The expected winner of the $33 million award is a group headed by
Stanford University’s Michael Snyder, director of its Center for
Genomics and Personalized Medicine, based on documents posted Friday
on the
stem
cell agency’s website.
David Jensen publishes the California Stem
Cell Report –
californiastemcellreport.blogspot.com --and has followed the stem
cell agency since 2005.
