Evangelina Padilla-Vaccaro, now cured of a serious genetic illness thanks to a successful stem cell treatment playing recently in a public park. The bubble around her is just for fun. Courtesy of Alysia Padilla-Vaccaro hide caption
Evangelina Padilla-Vaccaro, now cured of a serious genetic illness thanks to a successful stem cell treatment playing recently in a public park. The bubble around her is just for fun.
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, celebrities Michael J. Fox and Christopher Reeve, both facing serious, chronic conditions, touted the promise of stem cell research, which they believed could lead to a plethora 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, 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, 14 years and billions of dollars later, that California agency is running out of money, and backers of stem cell research plan to ask voters in the state to pony up for round two. The projected ask this time: $5 billion, in a measure the backers hope to place on the California ballot in 2020.
For voters this time, there will be one major question, says Zev Yaroslavsky, 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, "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," 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 about this," Murry says, "the fact that other states have stepped up and done this in such a big way that helped a lot. CIRM definitely has been a trailblazer for the rest of us."
Robert Klein, who spearheaded the original 2004 California ballot measure and served as chairman on CIRM's first board, still heads the advocacy group, Americans For Cures, that pushed Proposition 71. Medical science isn't exactly 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, but also good business for California.
"It has been a creator of jobs, and the state benefits from taxes by attracting research centers here," he says.
In 2012, 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 4,000 new jobs per year, and $205 million in state tax revenue.
As for the proposed new 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.
"Look, 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 intellectual infrastructure of California."
But where are the cures?
California's ballot initiative struck an emotional chord in 2004, in part because of the high profile cases of actors Reeve and Fox. Reeve, 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, which means they have the ability to be transformed into cells of specific tissues and organs possibly for use in new therapies that might treat or even cure some diseases.
In Fox's 30-second spot, he used the word "cures" three times.
So, has CIRM produced any cures?
Five-year-old Evangelina Padilla-Vaccaro, 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, 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, which can be lethal.
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 correct the SCID mutation.
She and at least 40 other children have been cured, according to CIRM.
Despite this success, 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 new shunt for kidney dialysis patients that is made out of human tissue and does not have to be replaced; the second 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 priority 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, 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, 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 big 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, 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, says there's still time for CIRM to make a bigger splash.
"We've still got two more years," McCormack says. "By 2020 I think people will see that CIRM-funded therapies are not just changing lives but saving lives."
Klein, the backer of Proposition 71, says the 2004 campaign never promised cures during the lifetime of the stem cell agency only progress.
"What we put in the ballot arguments is that we had to make major progress in mitigating disease, and moving toward cures," Klein says. "When I look back, I think we have out-achieved the representations we put on the ballot."
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 up
Opposition to the first ballot measure in 2004 was based mostly on religious concerns about using embryonic stem cells; on the large amount of money being requested; and on the lack of any guarantee for specific achievements.
Jensen expects religious 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, 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, some opposition has already emerged, even within the biomedical community. Barbara Koenig, head of the bioethics program at UCSF, points to ongoing concerns about conflicts of interest at CIRM.
She also sides with those who think 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 think we need to be honest about how we're investing in research."
Koenig says she supports stem cell research but voted against the measure in 2004, and has serious concerns about CIRM's renewal.
Ask her how she might use that proposed $5 billion differently, and she responds with a moment of stunned silence.
"Oh my, so many things," she says. "I would try to figure out how to make sure every child in California has access to basic health services, nutrition, clean water not just make high-priced products, but improve public health."
Koenig says stem cell research "privileges these quick-fix biotech approaches, which may make a lot of money but may not benefit the general public."
At its current spending pace, CIRM will run out of money by the end of 2019 roughly a year before the proposed ballot 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 end of 2020.
This story was produced by KQED's Future of You blog, and a longer version can be found here. Freelance science writer David Gorn writes frequently about stem cell research.
Excerpt from:
Will States Continue To Fund Stem Cell Research? : Shots ...
- Jake Diekman throws BP session for Rangers | MLB.com - MLB.com - August 6th, 2017 [August 6th, 2017]
- Cerebral palsy: Symptoms, diagnosis, treatment - December 5th, 2017 [December 5th, 2017]
- Hypothalamus - the body's thermostat | ASU - Ask A Biologist - December 6th, 2017 [December 6th, 2017]
- When Stroke Affects the Thalamus - Stroke Connection ... - December 28th, 2017 [December 28th, 2017]
- The Endocrine System: Hypothalamus and Pituitary - December 28th, 2017 [December 28th, 2017]
- Hypothalamus - Anatomy, Blood supply and Function | Kenhub - December 28th, 2017 [December 28th, 2017]
- Ulcerative Colitis - Cedars-Sinai - January 6th, 2018 [January 6th, 2018]
- Ulcerative colitis - Treatment - NHS.UK - January 7th, 2018 [January 7th, 2018]
- Business Directory | Uptown Waterloo Business Improvement Area - January 8th, 2018 [January 8th, 2018]
- Spinal Cord Injuries - emedicine.medscape.com - February 15th, 2018 [February 15th, 2018]
- Spinal Cord Injuries - spine.org - February 15th, 2018 [February 15th, 2018]
- Journal of Clinical & Experimental Cardiology - Open Access Journals - April 10th, 2018 [April 10th, 2018]
- Cardiology Treatment - Fortis Healthcare - April 10th, 2018 [April 10th, 2018]
- Biology Junction - April 12th, 2018 [April 12th, 2018]
- Managing Right Ventricular Failure in PAH: An Algorithmic ... - April 14th, 2018 [April 14th, 2018]
- Ticagrelor vs Clopidogrel After Fibrinolytic Therapy in ... - April 22nd, 2018 [April 22nd, 2018]
- Cardiovascular Pharmacotherapeutics: William H. Frishman ... - May 13th, 2018 [May 13th, 2018]
- 13 Super Foods Good for Eczema - Top Eczema Treatments - May 15th, 2018 [May 15th, 2018]
- Diagnosis & Treatment of Parkinson's - ParkinsonsDisease.net - May 30th, 2018 [May 30th, 2018]
- Arizona Heart Rhythm Center - June 10th, 2018 [June 10th, 2018]
- Ayurvedic Medicines for Ulcerative Colitis | Natural ... - June 21st, 2018 [June 21st, 2018]
- Weinstein Cardiovascular Development and Regeneration Meeting - June 30th, 2018 [June 30th, 2018]
- Ulcerative Colitis Information Centre - July 31st, 2018 [July 31st, 2018]
- Sudden Death in Dogs - Pet Health Network | Pet Health ... - August 7th, 2018 [August 7th, 2018]
- Coronary Heart Disease - Ischaemic heart disease - British ... - August 21st, 2018 [August 21st, 2018]
- Cardiac Rehabilitation: Overview, History and Definition ... - August 25th, 2018 [August 25th, 2018]
- Difference between Cardiac Arrest and Respiratory Arrest ... - August 25th, 2018 [August 25th, 2018]
- ECM for Pericardial Closure - Aziyo - August 25th, 2018 [August 25th, 2018]
- Adult Mental Health | Georgia Department of Behavioral Health ... - September 6th, 2018 [September 6th, 2018]
- Mental Health | ECLKC - September 6th, 2018 [September 6th, 2018]
- Mental Health - The Church of Jesus Christ of Latter-day Saints - September 6th, 2018 [September 6th, 2018]
- Center for Vascular Biology - Home | UConn Health - September 22nd, 2018 [September 22nd, 2018]
- # Diabetes Stem Cell Research Diabetes Care Measures - September 30th, 2018 [September 30th, 2018]
- Cardiac Rhythm Disturbances | The Patient Guide to Heart ... - November 2nd, 2018 [November 2nd, 2018]
- Arrhythmia | Cleveland Clinic - November 2nd, 2018 [November 2nd, 2018]
- Hyperbaric Oxygen Therapy for Wound Healing | Johns ... - November 13th, 2018 [November 13th, 2018]
- NIMH Home - November 24th, 2018 [November 24th, 2018]
- What is an interventional cardiologist? | Heart Surgeries ... - November 26th, 2018 [November 26th, 2018]
- Molecular Cardiology Research Institute Boston | Tufts ... - November 29th, 2018 [November 29th, 2018]
- Lower vascular plant | botany | Britannica.com - December 7th, 2018 [December 7th, 2018]
- Welcome to the ABG Tutorial Welcome to Hansen - December 12th, 2018 [December 12th, 2018]
- Cardiac catheterization - Wikipedia - December 12th, 2018 [December 12th, 2018]
- Stem Cell Research & Therapy | Home page - December 22nd, 2018 [December 22nd, 2018]
- Journal of Stem Cell Research and Therapy- Open Access ... - December 22nd, 2018 [December 22nd, 2018]
- Stem-cell therapy - Wikipedia - December 22nd, 2018 [December 22nd, 2018]
- Cardiology and Interventional Cardiology - December 23rd, 2018 [December 23rd, 2018]
- Rhode Island Interventional Cardiology | Cardiovascular ... - December 23rd, 2018 [December 23rd, 2018]
- Interventional Cardiology Certification Policies | ABIM.org - December 23rd, 2018 [December 23rd, 2018]
- Interventional Cardiology - Boston Scientific - December 23rd, 2018 [December 23rd, 2018]
- 9 Benefits of Human Growth Hormone (Along with Side ... - December 27th, 2018 [December 27th, 2018]
- Growth hormone deficiency - Wikipedia - December 27th, 2018 [December 27th, 2018]
- Dr. Lorrie Kirshenbaum, Cardiac Gene Biology | St ... - January 28th, 2019 [January 28th, 2019]
- Cardiomyopathy - CardioSmart - February 8th, 2019 [February 8th, 2019]
- Centenary Institute | Medical Research, Life Saving Research - February 24th, 2019 [February 24th, 2019]
- Cardiovascular Medicine | Department of Medicine - March 29th, 2019 [March 29th, 2019]
- Rapid Test Market 2019 with Recent Trends, Revenue, Demand and Top Manufactures- Bureau Veritas, SGS SA, Intertek Group plc, Eurofins Scientific -... - October 11th, 2019 [October 11th, 2019]
- Medical Imaging Equipment Market Research Report 2019: Global Industry Analysis, Business Development, Size, Share, Trends, Future Growth, Forecast To... - October 11th, 2019 [October 11th, 2019]
- Analysis on the Global DNA Read, Write & Edit Market, 2017-2019 and Forecast to 2024 - Yahoo Finance - October 11th, 2019 [October 11th, 2019]
- Cardiac Surgery Instruments Market is Expected to Tread a Steady Growth Trajectory by Clocking a CAGR of 5.9% from 2017 to 2022 - Health News Office - October 24th, 2019 [October 24th, 2019]
- PEDIATRIC MEDICAL DEVICES MARKET IS PROJECTED TO EXPAND AT A CAGR OF 8.0% FROM 2018 TO 2026 - Health News Office - November 1st, 2019 [November 1st, 2019]
- myTAIHEART Test Provides Evidence for Injury from Biopsy of Heart Transplant Recipients Cardiology2.0 - Cardiology2.0 - November 1st, 2019 [November 1st, 2019]
- Mental health and your heart - British Heart Foundation - November 19th, 2019 [November 19th, 2019]
- BUZZ-U.S. STOCKS ON THE MOVE-Cango, Gulfport Energy, Abiomed, Standard Diversified, Wanda Sports, Eidos - Nasdaq - November 19th, 2019 [November 19th, 2019]
- Cost Analysis of COPD Exacerbations and Cardiovascular Events in SUMMIT - AJMC.com Managed Markets Network - November 19th, 2019 [November 19th, 2019]
- Hundreds walk to defeat ALS in Ancient City - St. Augustine Record - November 22nd, 2019 [November 22nd, 2019]
- Living with Lung Cancer: The Silver Linings - Curetoday.com - November 22nd, 2019 [November 22nd, 2019]
- Building the human spinal cord atlas - SynBioBeta - November 22nd, 2019 [November 22nd, 2019]
- The Week That Wasn't: Keto and the Flu, Human Cyborg, Duvet Disease - Medscape - November 22nd, 2019 [November 22nd, 2019]
- ALS advance offers hope for those diagnosed with deadly disease - WRAL.com - November 22nd, 2019 [November 22nd, 2019]
- ALS Stem Cell Therapy Developer Seeks Amendment to its AstroRx Trial - ALS News Today - November 22nd, 2019 [November 22nd, 2019]
- Former Boston Globe writer, current NHL executive Snow diagnosed with ALS | College Hockey - USCHO - December 23rd, 2019 [December 23rd, 2019]
- AB Science announces the publication of new results in the peer-reviewed journal Glia that further support masitinib's potential mode of action in ALS... - December 23rd, 2019 [December 23rd, 2019]
- Christmas in the City thrills homeless families, takes on poignancy with founders ALS diagnosis - The Boston Globe - December 23rd, 2019 [December 23rd, 2019]
- Amylyx Pharmaceuticals Announces AMX0035 Demonstrated Statistically Significant Treatment Benefit for People with ALS in the CENTAUR Trial - Business... - December 23rd, 2019 [December 23rd, 2019]
- RADICAVA (edaravone) - December 23rd, 2019 [December 23rd, 2019]
- List of Amyotrophic Lateral Sclerosis Medications (5 Compared ... - December 23rd, 2019 [December 23rd, 2019]
- Neuropore Completes Phase 1 Clinical Trial in Healthy Volunteers with NPT520-34, a Therapeutic Candidate Aimed at Treating Parkinson's Disease and... - January 14th, 2020 [January 14th, 2020]
- Comprehensive Analysis on Prostacyclin Market based on types and application - Technology Magazine - January 18th, 2020 [January 18th, 2020]
- N.J. karate legend who inspired thousands loses battle with ALS - NJ.com - January 18th, 2020 [January 18th, 2020]
- Denali Therapeutics Announces Broad Pipeline Progress Including Positive Results From Its LRRK2 Program for Parkinson's Disease - Associated Press - January 18th, 2020 [January 18th, 2020]