Batches of human embryonic stem cells that hold the promise of treating spinal-cord injuries are being grown inside a Peninsula laboratory — ready, and now federally approved, for injection into paralyzed patients.
In the worldwide race to develop stem cell therapies, biotech giant Geron Corp. in Menlo Park will be the first to begin a human clinical trial using the controversial stem cells, which are created through repeated divisions of a human embryo.
Marie Csete, chief scientist at the state-funded California Institute for Regenerative Medicine, which is based in San Francisco, described the upcoming trial involving 10 paraplegia patients as a real milestone.
“When you think about the fact that these cells have only been known for 10 years, that’s a remarkably fast timeline to develop a product,” Csete said. “I’m hoping that will be an enormous learning experience.”
Therapies derived from embryonic stem cells have been touted as a holy grail of medicine because stem cells have a unique ability to grow into any cell and divide to create, and possibly heal, any organ. The research has sparked controversy, however, because the embryo — obtained from fertility clinics — is destroyed in the process of drawing out the stem cells.
As a result, progress within the field was stifled under the George W. Bush administration, which outlawed the use of federal funds for research into embryonic stem cell lines created after Aug. 9, 2001, according to Geron President Thomas Okarma.
The federal restrictions prompted Californians to pass Proposition 71 in 2004, which provided $3 billion in funding for stem cell research at state universities and research institutions and created the California Institute for Regenerative Medicine. The embryonic stem cell research pursued with state monies, however, could not be mixed with research supported by federal dollars.
Although Geron did not draw on public funds for its work, federal stem cell policies delayed progress for the private sector because it hamstrung critical academic research, according to Okarma.
President Barack Obama said during his campaign that he was a supporter of embryonic stem cell research and last month signed an executive order lifting the Bush administration’s limitations on using taxpayer funds for the research.
In a January ruling that coincided with the first month of Obama’s presidency, the U.S. Food and Drug Administration approved a clinical trial that will see a line of Geron’s stem cells injected directly into the spinal cords of recently paralyzed patients as soon as this summer.
With the exception of war injuries, which can sever spinal cords, most of the 11,000 spinal cord injuries that occur annually in the United States are caused by bruises and contusions that short-circuit the nervous system at the site of the back injury, according to Okarma.
After Geron’s stem cells are injected into the injured spines, they will grow in number, spread throughout the wound and create new, healthy tissue to repair the spinal damage, according to Okamra. The technique has already worked in rats and mice, he said.
“These are living cells and, after they’re injected, they expand numerically, divide and migrate within the legion space,” Okarma said. “Sixty to 70 percent of the total spinal-cord injury marketplace could benefit from this therapy if it proves to be safe and effective.”
The first patients to receive the experimental therapy will need to be newly injured, with injections taking place soon after the damage has occurred.
“If we wait several months, there’s too much scarring,” Okarma said.
The patients will be drawn from up to seven medical clinics from across the country. The first ones will receive small doses to test the safety of the therapy. If the therapy proves to be safe, future trials will involve higher doses, along with less-recently injured patients, according to Okamra.
The company eventually plans to manufacture and sell off-the-shelf lines of stem cell products to treat such ailments as Alzheimer’s and heart failure, in addition to spinal-cord injuries, according to Okamra.
Research companies could give job market huge boost
Bay Area biotech companies performing stem cell research could create substantial employment opportunities for a wide range of workers as the nascent but potentially lucrative industry matures, experts say.
The counties of San Francisco, San Mateo and San Diego, along with the cities of Boston and Madison, Wis., have become major hubs of the nation’s fast-growing biotechnology sector, which includes companies working on stem cells and other areas of regenerative medicine.
The Peninsula is poised to cash in on the approaching commercialization of prospective stem cell-based cures, according to Gregory Bonfiglio, founder of Proteus Venture Partners, a venture capital firm being formed to invest in regenerative medicine.
“This is a field that’s just exploding,” he said.
Scores of stem cell companies, including StemCells Inc., VistaGen Theraputics Inc., iZumi Bio and industry heavyweight Geron Corp. — which is presided over by former Stanford scientist Thomas Okamra — are based in San Mateo County.
Additionally, Stanford University has been awarded more than $100 million in grants by the California Institute for Regenerative Medicine — more than any other state research organization, agency figures show.
“A lot of people collaborate with Stanford,” CIRM chief scientist Marie Cseste said. “A lot of things break away from Stanford.”
The stem cell industry is emerging in the Peninsula and in San Francisco’s Mission Bay, in part because of the roots the already prosperous biotech industry has established in the region, creating a science-focused corridor, according to iZumi CEO John Walker.
The South San Francisco-based company, which was founded at the end of 2007, employs 25 people, but it plans to increase its work force to 45 people by the end of the year, according to Walker.
The company made headlines this month when it announced it was teaming up with Japanese researchers to advance a newly invented process that helps turn a patient’s skin cells or other nonembryonic cells into patient-specific stem cells.
Although most iZumi employees are scientists, the company expects it will eventually need to employ professionals in other fields, such as marketing and finance, according to Walker, once it begins to commercialize techniques developed through its research.
“All of the pieces of the puzzle are in place right now in Northern California,” he said. “With the rate that this technology is advancing, I would be very surprised if we don’t see substantial job growth over the next few years.”
Getting research to human clinical trial
Menlo Park-based Geron is given the first federal approval to study its embryonic stem cell-based therapy in patients.
$200 million
Geron’s investment in embryonic stem cell research
21,000
Pages in federal application to conduct human clinical trial
5 billion
GRNOPC1 cells — the cell Geron detveloped that will be used in the human clinical trial — produced during animal studies of the research
24
Animal studies conducted before the application was filed
10
Patients who will be selected to participate in the trial
7
Medical centers that are candidates to participate in trial
$0
Public funding received by the company
Source: Geron Corp.
Bay Area companies under the microscope
Among Peninsula companies advancing stem cell technologies:
VistaGen Theraputics Inc.
City: South San Francisco
Developing: Embryonic stem cell therapies
ExelIxis Inc.
City: South San Francisco
Developing: Treatments for cancer
iZumi Bio
City: South San Francisco
Developing: Technologies to turn ordinary cells, such as skin cells, into patient-specific stem cells
Cellerant Therapeutics Inc.
City: San Carlos
Developing: Products to regulate the blood-forming system
Geron Corp.
City: Menlo Park
Developing: Embryonic stem cell therapies
Stem Cells Inc.
City: Palo Alto
Developing: Neural stem cell technologies using adult brain tissue
SanBio Inc.
City: Mountain View
Developing: Therapies based on stem cells and other regenerative medicines for neurological disorders
By: John Upton
Examiner Staff Writer
