California’s government-run stem-cell research agency, on course to spend $3 billion in taxpayer money to find treatments for some of the world’s most intractable diseases, is pushing to accelerate human testing before its financing runs out.
For the California Institute for Regenerative Medicine, time is growing short to fund research that demonstrates the potential of stem cells to help treat everything from cancer to heart disease to spinal cord injuries.
The agency, created by voters in 2004, has given out more than half of its $3 billion from state bonds and must spend the rest by 2017. The largest U.S. funding source for stem-cell research outside the federal government, it’s under pressure to show results to attract new money from pharmaceutical companies, venture capitalists or even more municipal bonds.
“We need to figure out how to keep them going,” said Jonathan Thomas, a founding partner of Saybrook Capital LLC in Los Angeles, and chairman of the institute’s board, which meets today. “We could do public-private partnerships, venture philanthropy, a ballot box.”
Embryonic stem cells have the potential to change into any type of cell in the body. They are among the first cells created in embryos after conception. Scientists hope they may replace damaged or missing tissue in the brain, heart and immune system.
California voters approved the bonds after President George W. Bush banned the use of federal funds for research on embryonic stem cells. Since then, other types of stem cells have been shown to act like embryonic cells, relieving some of the debate over the ethics of destroying human embryos to use the cells.
The agency’s funding decisions have included a grant of $20 million to a team led by Irv Weissman at the Stanford University School of Medicine, seeking a cure for cancer.
Weissman’s team is working on an antibody manufactured with stem cells that allows a cancer patient’s own immune system to destroy a tumor, instead of relying on toxic radiation or chemotherapy. The antibody counteracts a protein called CD47, which creates what scientists call a “don’t eat me” shield around the cancer. Once that cloak is removed, the patient’s immune system recognizes the cancer and attacks the tumor, shrinking or eliminating it.
Tests on humans are to begin early next year. The antibody has already worked in mice against breast, colon, ovarian, prostate, brain, bladder and liver cancer.
Two other research projects funded by the California agency are in human trials now -- one targeting HIV, the virus that causes AIDS, and another that regrows cardiac tissue in heart-attack victims.
California paid $97 million from its general fund for the bonds this year, according to the state Finance Department. The nonpartisan Legislative Analyst’s Office has projected debt service costs of about $3 billion, in addition to the $3 billion principal.
The institute, with headquarters a block away from AT&T Park on San Francisco’s waterfront, has awarded grants totaling $1.8 billion to 73 institutions since 2007. Much of the early financing went to build labs and supply them with equipment. The agency now wants to devise ways to round up more money so that it can continue making grants after 2017.
“We’re getting some fantastic pre-clinical results,” Thomas said. “But science takes time.”
An advisory board has recommended that the agency push forward as quickly as possible with human testing on its most promising six to eight projects.
Brock Reeve, executive director of the Harvard Stem Cell Institute, said a rush could waste money by going too far down paths with false promise.
“There have been a lot of clinical trials in the stem cell field broadly that haven’t panned out,” Reeve said.
Brock Reeve is the half-brother of the actor Christopher Reeve, who suffered a spinal-cord injury in 1995 that left him quadriplegic. The star of the 1978 film “Superman” recorded a television commercial in support of California’s stem-cell initiative that was broadcast days after his death in 2004 at age 52.
Some of the money that Christopher Reeve campaigned for went to Menlo Park, California-based Geron Corp. (GERN), which was awarded $25 million by the state agency. It shelved its testing of spinal-cord stem-cell therapy in 2011 because of research costs and regulatory complexities.
The research was the first clinical trial using human embryonic stem cells approved by the U.S. Food and Drug Administration. Geron later sold its stem-cell program for $35 million in cash and stock to Alameda, California-based Biotime Inc. (BTX)
Nevertheless, the stem-cell agency’s go-commercial push is warranted, said Hank Greely, a professor specializing in biomedical issues at Stanford Law School who heads California’s Human Stem Cell Research Advisory Committee.
“It’s bold but I think worth trying,” Greely said
“Ultimately, companies will be essential to the development and widespread use of cell-based therapies,” he said in an interview. “The ‘valley of death’ between promising research and actual biomedical products is a well-known problem. This seems like a good test of one possible way to avoid it.”
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