Thursday, October 10, 2019

Full Text: Critics on Economic Impact Report on California Stem Cell Agency

Here is the full text of the email responses of stem cell agency critics to the report on the economic impact of the agency over the last 15 years.

State Sen. John Moorlach, R-Costa Mesa, who responded directly to questions from the California Stem Cell Report (CSCR):

CSCR: "Does the USC study bring something to light that is new to you?"
Moorlach: This study does not bring to light anything new. Doling out $3 billion to any entity or entities would generate the same economic metrics. The study is a shell.
"CIRM did not produce or sell anything of substance. It was not a stadium that created jobs around it, like restaurants, bars, and hotels.
"CIRM spent $3 billion and it's costing the taxpayers that much, plus $800 million-plus in interest."
CSCR: "Does it lead you to modify your thinking about the California stem cell program?"
Moorlach: "The new report has not modified my thoughts on CIRM. It was a boondoggle when it started and it still is. It's held up by emotional appeals, sustained by empty promises, and now rationalizing to extend its existence."
CSCR: "What is your overall view of the agency at this point?"
Moorlach: "Stem cell research is important, but best left to the private sector. This was style drift and extremely expensive, including the necessity to raise taxes with Propositions 30 and 55."
From Marc Joffe, senior policy analyst with the Reason Foundation:
"I oppose the use of state general obligation bonding authority for any purpose other than building well-conceived civil infrastructure projects. The fact that spending bond proceeds generates economic activity is not surprising and not a reason to support a new bond in 2020. Similar studies have been released in support of the ill-conceived high-speed rail project: we might not get a usable system that takes many passengers out of their cars, but at least we created a lot of jobs in the Central Valley! This is not a persuasive argument for imposing more debt on our children, who already have the challenge of paying for Baby Boomer retirements."
"Also, for what it’s worth, I don’t oppose the existence of CIRM. If it can be financed privately, that would be wonderful. I just don’t think it should be imposing burdens of future taxpayers."
From Joe Rodota, who worked for Republican Gov. Pete Wilson and Republican Gov. Arnold Schwarzenegger.
"This sentence (from the study) caught my attention 
"The estimates in the report are based on the economic stimulus created by CIRM funding and by the co-funding that researchers and companies were required to provide for clinical and late-stage preclinical projects. The estimates also include:
"Investments in CIRM-supported projects from private funders such as equity investments, public offerings and mergers and acquisitions, 
"Our (Rodota's) proposal, as outlined here, includes this language:
"In exchange for the funds they receive, companies would tender to the University of California shares of their common stock, with an estimated value as determined by the most recent outside valuation or price set by investors. These shares would become part of the UC endowment -- and the University of California be free to sell or leverage these shares, or acquire additional shares, as it sees fit.

"Under our proposal, in exchange for providing funding to private companies engaged in developing stem-cell therapies, the University of California would receive shares, alongside any shares sold to private investors. Although the report categorizes equity sold to private investors as part of the 'economic stimulus created by CIRM funding,' that equity is held by private investors, not the University of California."

Wednesday, October 09, 2019

'Handsome Dividend' -- California Stem Cell Agency and Its Economic Impact on the Golden State


This video was produced by Forty Seven, Inc., a firm in which California's stem cell agency has invested more than $15 million

California’s stem cell research program has had a “major impact” on the state’s economy, generating billions in sales revenue and creating tens of thousands of new jobs, according to a study released today.

Commissioned by the $3 billion state stem cell agency, the report comes as the program is hitting a difficult financial patch. Known officially as the California Institute for Regenerative Medicine (CIRM), the agency expects to run out of money for new awards by end of this month. CIRM is hoping that voters will refinance it with $5.5 billion via a ballot initiative in November 2020.

The 84-page study is expected to serve as a rebuttal to critics who have called the nearly 15-year-old agency a boondoggle. The report said, 
“The state’s investment in (the agency) has paid handsome dividends in terms of output, employment and tax revenues for California."
Adam Rose, USC photo
Dan Wei, USC photo
The study and a yet-to-be released companion report were commissioned at a cost of $206,000 by the agency. The economic study was prepared by Dan Wei and Adam Rose of the Price School of Public Policy at the University of Southern California

Maria Millan, CEO and president of CIRM, said in a news release that the study reflects the agency's role in building a stem cell "ecosystem" in the Golden State. 

Beyond CIRM's medical and scientific work, she said that the agency is "promoting economic growth in California  by attracting scientific talent and additional capital, and by creating an environment that supports the development of businesses and commercial enterprises in the state."

The report summarized CIRM's economic impact in four points. 
  • "$10.7 billion of additional gross output (sales revenue)
  • "$641.3 million of additional state/local tax revenues
  • "$726.6 million of additional federal tax revenues
  • "56,549 additional full-time equivalent jobs, half of which offer salaries considerably higher than the state average."
CIRM's news release on the report characterized the study as "independent." CIRM said it showed that the agency's efforts had a "major impact" on the state's overall economy, which totalled $3 trillion in 2017. 

The agency cited the assistance it has provided to create companies that ultimately will make CIRM-financed therapies available to the public at large.

While the agency's spending has not yet led to a widely available therapy, it is backing 56 clinical trials, which is the last stage before a treatment can be approved for widespread use. About 86 percent of clinical trials fail to result in a product, according to 2018 figures.

As an example of a fruitful collaboration, the agency cited Orchard Therapeutics of the United Kingdom, which plans to seek to qualify soon for speedier federal approval of its treatment for a version of  "bubble boy syndrome," a fatal immune deficiency. CIRM has awarded the firm $8.5 million.

The treatment was developed by Donald Kohn at UCLA with the help of $52 million in CIRM cash During clinical trials, it has saved the lives of more than 50 babies. Kohn said in a statement,
"I think one of the greatest strengths of CIRM has been their focus on development of new stem cell therapies that can become real medicines."
Orchard has offices in the San Francisco Bay area and plans to build a 150,000-square-foot manufacturing facility in Fremont. 

Also cited as an example was a company called Forty Seven, Inc., of Menlo Park, Ca., which is developing cancer therapies. Mark Chao, founder of the firm, said CIRM's support was "instrumental to our early successes."

The economic study also explored the "deal flow" funding that has aided commercialization of research. The study said it is expected that 
"...CIRM's past and current funding will attract increasing amounts of industry investment and lead to additional spending injections into the California economic in the years to come."
The companion report to today's economic study involves "health dividends" provided by the agency. That report is expected to be released next week. 

The agency has commissioned other economic studies in the past including one in 2012 that also lauded the agency. The request for proposals to perform that 2012 study said it must execute "a vibrant and aggressive strategy to support the goals and initiatives of CIRM.” 

Queried by the California Stem Cell Report, Kevin McCormack, a spokesman for CIRM, said four enterprises were solicited to develop this year's study. Three declined. The contract for the latest study stipulated that USC had control of the content. The latest study also laid out the methodology in considerable detail, something missing from the 2012 report. 

Below is a May 31, 2019, 58-second video of the president of Orchard, Mark Rothera, discussing the company's work. More brief, 2019 videos of Rothera from same interview sequence can be found hereherehere and here. The videos were taken by the Alliance for Regenerative Medicine

Thursday, October 03, 2019

California's $72 Million Diabetes Wager: ViaCyte Announces Major "Firsts" for Its Stem Cell Therapy


Vox Pop video/Viacyte

One of California's bigger stem cell bets -- $72 million -- turned up this morning with a strong positive report that included a couple of "firsts" in its search for a virtual cure for diabetes.

The announcement came from ViaCyte, Inc., of San Diego. The California stem cell agency has pumped $72 million into the company, making the firm the top for-profit recipient of state stem cell largess. 

The news comes as the agency, known formally as the California Institute for Regenerative Medicine (CIRM), is running out of funds and hoping that voters will give it $5 billion more. A major research score would be a big plus for that ballot initiative effort. 

The agency's president, Maria Millan, described the ViaCyte announcement as important and encouraging. 

ViaCyte is developing a tiny device that is implanted in a person's body and that generates insulin as needed. It is aimed primarily at type one diabetes, which afflicts more than one million Americans 

ViaCyte issued a news release on the developments at major, national stem cell conference in Carlsbad, Ca. The headline on the release said, 
"First demonstration of insulin production in patients from a stem cell-derived islet replacement therapy"
The release said,
"Preliminary data show that implanted cells, when effectively engrafted, are capable of producing circulating C-peptide, a biomarker for insulin, in patients with type 1 diabetes."
Paul Laikind, CEO and president of the firm, declared,
“ViaCyte is the first and only company in human clinical trials with a stem cell-derived islet replacement therapy candidate, and we are now the first to demonstrate production of C-peptide in patients receiving implanted stem cell-derived islets. These data show that our PEC-01 cells are functioning as intended when appropriately engrafted. “While there is still more work to be done, this is an important milestone. We plan to present additional data in the near future.”
Laikind continued,
“ViaCyte has achieved a number of firsts in this field. Now with the first demonstration of insulin production in patients who have received PEC-Direct, we are confident we can be the first to deliver an effective stem cell-derived islet replacement therapy for type 1 diabetes.”
Asked for comment, CIRM's Millan said,
"This is encouraging news. We are very aware of the major biologic and technical challenges of an implantable cell therapy for Type 1 Diabetes, so this early biologic signal in patients is an important step for the ViaCyte program."
ViaCyte is scheduled to present its findings later today at the Cell & Gene Meeting on the Mesa. That session can be seen live on the Internet 1:45 p.m. PDT. 

(An earlier version of this item contained a slightly different quote from Millan. CIRM re-submitted the latest quote, which adds information.)

Monday, September 30, 2019

Measure to Add $5 Billion-plus to California Stem Cell Agency Yet to be Filed

The official kickoff for a proposal to refinance California's nearly 15-year-old stem program with $5.5 billion has been delayed for at least a few more weeks. 

Backers of the proposed bond initiative told the California Stem Cell Report last week that the ballot measure will not be filed with state election officials until later in October. Previously, they had said it would be filed by the end of this month. 

Melissa King, executive director of Americans for Cures, said there was no major reason for the delay in filing the measure. She said it was just a matter of "wanting to get everyone's views heard, etc."

To qualify for the November 2020 ballot, backers will need to gather more than 600,000 signatures of registered voters. 

Americans for Cures is a stem cell advocacy group controlled by Robert Klein, who ran the initiative campaign in 2004 that created the California Institute for Regenerative Medicine (CIRM), as the agency is formally known.
Klein was also the first chairman of CIRM. 

The agency expects to run out of cash for new awards this year and has no further source of major funding. 

Thursday, September 26, 2019

Pluses and Minuses: The Cases For and Against $5.5 Billion More for California's Stem Cell Agency

BURLINGAME, Ca. -- It was a case of dueling op-ed arguments -- one describing the state's $3 billion stem cell agency as a waste of money and the other touting its success and its current and future impact on human lives.

The articles appeared online on the web site of the San Diego Union-Tribune on the eve of a daylong conference here to lay out possibilities for the agency over the next several years. 

The articles and the meeting come at a critical point for the agency, known formally as the California Institute for Regenerative Medicine (CIRM). The nearly 15-year-old enterprise expects to run out of cash for new awards this year. It is hoping for a $5.5 billion infusion in November 2020 if California voters approve another bond measure for the agency.

The op-ed articles embodied many of the arguments -- pro and con -- that are likely to surface in the ballot measure campaign next year, a campaign that is expected to cost its supporters $50 million. 

In the San Diego newspaper, the case for giving the agency more billions was made by Larry Goldstein, a professor at UC San Diego; Aileen Anderson, a professor at UC Irvine, and Malin Burnham, chairman of the Burnham FoundationThe case against CIRM was made by state Sen. John Moorlach, R-Costa Mesa.

Moorlach argued that the agency has "produced close to no results." He cited articles in the journal Nature and the San Francisco Chronicle as evidence. Moorlach called CIRM a "dry hole." He wrote.
"Not only was the $3 billion for the research a bust, but taxpayers will be paying interest on the principal until 2039. California Treasurer Fiona Ma’s office told me the cost of the interest on the $2.59 billion of principal already spent will be $836.6 million. Interest rates lower than anticipated in 2004 kept that below the original $3 billion estimate."
Goldstein and his co-authors argued that more than 50 children have had their lives "given back" as the result of clinical trials funded by CIRM. They said, 
"CIRM funding has established an impressive pipeline of new stem cell-based therapies being tested in 78 human trials directly funded by CIRM or based on CIRM-funded research. CIRM funding has also led to over 3000 published medical discoveries." 
They continued, 
"In 2020, Californians can continue their commitment to the best forms of stem cell research and therapy development. While there are no guarantees in medical research, if prior achievement is any indication, the next initiative will push many breakthrough therapies across the finish line. Considering the potential benefits to Californians and the opportunities to improve lives and alleviate suffering, there is little to lose, and an incredible amount to gain."

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