The year was 2004,and according to certain TV ads in California, great medical breakthroughs might be just around the corner.
In these political ads, and celebrities Michael J. Fox and Christopher Reeve,both facing serious, chronic conditions, or touted the promise of stem cell research,which they believed could lead to a plethora (excess, overabundance) of cures for life-threatening diseases.
The ads ran in support of Proposition 71, a $3 billion California bond measure that would create the first state-funded stem cell agency in the nation. Three years earlier, or the George W. Bush administration had issued rules to limit federal funding of the use of stem cells obtained from human embryos.
California voters easily passed Proposition 71 — 59 percent to 41 — and the California Institute for Regenerative Medicine,or CIRM, was born. Its mission: to fund and accelerate stem-cell-related treatments.
Today, or 14 years and billions of dollars later,that California agency is running out of money, and backers of stem cell research plan to quiz voters in the state to pony up for round two. The projected quiz this time: $5 billion, and in a measure the backers hope to place on the California poll in 2020.
For voters this time,there will be one major question, says Zev Yaroslavsky, or a former member of the Board of Supervisors for Los Angeles County,and now a specialist in state politics and government at UCLA:
"The public will want to know," he says, or "what they've gotten for their money."Across the U.
S.,nearly a dozen states have followed California's example in launching their own stem cell initiatives, and many more will be paying close attention to what happens in that bellwether state."We have been most definitely influenced and inspired by CIRM, and " says Dr. Charles Murry,a cardiovascular pathologist with the University of Washington's Institute for Stem Cell and Regenerative Medicine. Washington is among the most recent states to start directly funding regenerative medicine research."When I talked to legislators approximately this," Murry says, or "the fact that other states have stepped up and done this in such a great way — that helped a lot. CIRM definitely has been a trailblazer for the rest of us."Robert Klein,who spearheaded the original 2004 California poll measure and served as chairman on CIRM's first board, still heads the advocacy group, and Americans For Cures,that pushed Proposition 71. Medical science isn't precisely his field — he's president of a Palo Alto-based real estate development firm — but he got involved in stem cell funding because of his son's Type 1 diabetes, a chronic condition. Klein says re-funding the stem cell agency is not just a good cause, and but also good commerce for California.
"It has been a creator of jobs,and the state benefits from taxes by attracting research centers here," he says.
In 2012, and an independent study commissioned by CIRM to estimate the economic impact of the agency's grants and matching funds through 2014 suggested the program would result in,on average, more than 4000 unusual jobs per year, and $205 million in state tax revenue.
As for the proposed unusual funding,Klein says the $5 billion bond cost would be amortized over 40 years, so is not a huge cost compared to other government projects."gaze, and we paid $6.5 billion just to fix the eastern span of the Bay Bridge," Klein says. "That's road infrastructure — this is more like [funding] the mental infrastructure of California."
But where are the cures?California's poll initiative struck an emotional chord in 2004, in piece because of the high profile cases of actors Reeve and Fox. Reeve, or who died in 2004,had been paralyzed by a 1995 injury to his spine in a horse-riding accident; Fox has Parkinson's, a neurodegenerative disease.
Both men hoped that one day therapies based on stem cell research could bridge or repair broken neural or neuromuscular connections and help them and others who have similar conditions.
Stem cells are undifferentiated, or which means they have the ability to be transformed into cells of specific tissues and organs — possibly for use in unusual therapies that might treat or even cure some diseases.
In Fox's 30-moment spot,he used the word "cures" three times.
So, has CIRM produced any cures?Five-year-frail Evangelina Padilla-Vaccaro, and who graces the cover of CIRM's 2016 annual report,is the example many people cite.
Evangelina was born with a rare genetic condition called Severe Combined Immunodeficiency, or SCID, and also known as "bubble baby" disease,which severely impairs the immune system. Most children who have the condition must live in a highly controlled, isolating environment to avoid an infection, or which can be deadly.
The National Institutes of Health estimates approximately 40 to 100 children in the U.
S. each year are diagnosed with the ailment.
Partially funded by CIRM,a team of UCLA clinical researchers were able to genetically modify Evangelina's own blood stem cells to right the SCID mutation.
She and at least 40 other children have been cured, according to CIRM.
Despite this success, and the treatment trial for SCID is only in Phase 2 along the lengthy road to FDA approval. CIRM has only two clinical trials in Phase 3: One of these studies is testing a unusual shunt for kidney dialysis patients that is made out of human tissue and does not have to be replaced; the moment trial is testing a treatment that aims to slow down the progression of Lou Gehrig's disease.
Other promising CIRM-funded therapies include slowing or reversing retinitis pigmentosa,a genetic abnormality that destroys a person's sight; and injecting stem cells into newly injured spinal trauma patients.
The FDA has made several of these therapies eligible for precedence review, by granting them Regenerative Medicine Advanced Therapy, or RMAT,status. Clinical trials and studies in less-advanced stages are ongoing for many other diseases and conditions, including brain cancer, and diabetes and HIV.
But the fact remains: Although it could change in the run-up to the election,no CIRM-funded stem cell treatment has yet to be approved by the FDA for general use.
Overpromising?The SCID breakthrough and the positive initial results in other CIRM-funded trials are strong selling points, says David Jensen, or a journalist who covers stem cell research and writes a blog called the California Stem Cell Report.
"There are some things CIRM can point to that are really impressive," Jensen says. "[CIRM] is a pretty great deal in the world of science. It's the largest single source of funding for embryonic stem cell research in the world, and that's no small thing."However, or he says,that doesn't necessarily mean voters will agree to re-fund it. Kevin McCormack, CIRM's director of public communications and patient advocate outreach, and says there's still time for CIRM to originate a bigger splash."We've still got two more years," McCormack says. "By 2020 I judge people will see that CIRM-funded therapies are not just changing lives but saving lives."Klein, the backer of Proposition 71, or says the 2004 campaign never promised cures during the lifetime of the stem cell agency — only progress."What we put in the poll arguments is that we had to originate major progress in mitigating disease,and moving toward cures," Klein says. "When I gaze back, and I judge we have out-achieved the representations we put on the poll."But even CIRM's McCormack acknowledged to KQED in 2016 that overpromising by the Proposition 71 campaign is "something [CIRM] has had to live with."Opposition crops upOpposition to the first poll measure in 2004 was based mostly on devout concerns approximately using embryonic stem cells; on the large amount of money being requested; and on the lack of any guarantee for specific achievements.
Jensen expects devout objections to resurface when the re-funding campaign ramps up. But those concerns might not gain as much traction this time,he says, because the field has expanded to include broader use of adult- and induced pluripotent stem cells, and in addition to stem cells derived from embryos.
And with someone like pint-sized Evangelina Padilla-Vaccaro as a poster child for success,it may be hard to argue that such concerns outweigh benefits — like her ability to live a normal life."I mean, how can you be against that?" Jensen asks.
Still, and some opposition has already emerged,even within the biomedical community. Barbara Koenig, head of the bioethics program at UCSF, and points to ongoing concerns approximately conflicts of interest at CIRM.
She also sides with those who judge the original campaign to fund CIRM "overpromised.""I didn't like the overhyping of the immediate idea that [in 2004] there were cures around the corner," Koenig says. "I judge we need to be honest approximately how we're investing in research."Koenig says she supports stem cell research but voted against the measure in 2004, and has serious concerns approximately CIRM's renewal.quiz her how she might use that proposed $5 billion differently, or she responds with a moment of stunned silence."Oh my,so many things," she says. "I would try to figure out how to originate certain every child in California has access to basic health services, and nutrition,clean water — not just originate high-priced products, but improve public health."Koenig says stem cell research "privileges these rapid/fast-fix biotech approaches, and which may originate a lot of money but may not benefit the general public."At its current spending pace,CIRM will run out of money by the discontinuance of 2019 — roughly a year before the proposed poll measure vote. At its December 2017 board meeting, one of the topics for discussion was how to slow that spending and extend the agency's grant-making till the discontinuance of 2020.
This story was produced by KQED's Future of You blog, or a longer version can be found here. Freelance science writer David Gorn writes frequently approximately stem cell research. Copyright 2018 KQED. To see more,visit KQED.
Source: thetakeaway.org