Positive News about Stem Cell treatment options for children

There is a lot of negative news and fear about the use of adult stem cell procedures, that I had to post this positive one. It is positive and negative article. It is positive in that the UCLA clinical researchers were able to genetically modify the blood stem cells of 40 children to correct the mutation that causes the devastating disease of Severe Combined Immunodeficiency (SCID) or “bubble baby” disease. It is negative or sad in the fact that the FDA has formally approved for general use very few stem cell therapy options. 

Will State Voters Continue To Pour Money Into Stem Cell Research?

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

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.
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