Stem Cell Blowback on the Ice Bucket Challenge

Virulent opposition to research involving human embryonic stem cells is surfacing anew in the wake of the vast attention attracted by the Ice Bucket Challenge.

Sarah Pulliam Bailey wrote on Religion News Service that the objections have “gone viral” on the Internet because the beneficiary of the watery fundraising, the ALS Association, finances research involving human embryonic stem cell (hESC) lines.

For those of you who are not tuned into the icy effort, it involves celebrities and others pouring frigid water over themselves and challenging others to do likewise and donate to the ALS Association.  About $16 million has been raised so far and is still growing. Photos, news stories and blogs all chronicle the challenge on the Web.  A search this morning on the term “ice bucket challenge” turned up 57.2 million hits.

Our readers might ask how this involves the $3 billion California stem cell agency. The answer is that hESC research is the primary reason for the existence of the agency. It was created in 2004 by California voters after former President Bush restricted federal funding for research involving hESC.  Ironically Bush has participated in the challenge(see video above).

Bush’s restrictions, since lifted by President Obama, generated major controversy in the scientific community and raised fears that efforts to develop what seemed to be nearly magical therapies would wither and die. Without that concern, voters would have been unlikely to approve the measure, Proposition 71, and campaign contributors would have been unwilling to open their checkbooks.

The stem cell agency, however, has turned away from hESC research. Less than 250 of its 622 awards have gone to that field, according to the agency. Instead the agency is backing adult stem cell research, once an anathema to the organization, along with experiments involving reprogrammed adult stem cells. Leaders in the stem cell field, however, say human embryonic stem cells remain the scientific gold standard. (No figures were immediately available on the dollar value of the hESC awards.)

Continued funding of hESC research is linked to the agency’s search for alternative sources of cash as its current financing winds down. Efforts to develop a fresh stream from public sources will run up against the controversy involving hESC experiments, one of the reasons for the lack of interest by Big Pharma. The hESC opposition could also have an impact on development of private sources of funding who may not want to become embroiled in the flap, which can lead to picketing and public protests involving entities that support hESC research.

Kevin McCormack, senior director for the stem cell agency’s public communications, wrote about the ice bucket fad last week on the agency’s blog. He said it “shows how a little bit of creativity can create so much more interest in a disease, and the people suffering from it, than any amount of well-meaning, more traditional attempts at education.” And he poured a bucket of water over his head.

Nonetheless, the opponents hold their views with great passion and zeal and have little respect for hESC science. Here is what one person said today in a comment posted on the Religion News Service article. The individual was identified only as “jamadan.”

“My father died of ALS 6 years ago. My father was and my family is ‘pro-life’, meaning we believe abortion to be murder and embryonic research akin to Nazi medical testing on dead camp victims. Nothing can make us ‘accept’ abortion or anything that benefits from it. Not even our own lives. I think you’ll find that true for all Christians, who by definition, must be pro-life.” 

For other commentary involving objections to the Ice Bucket Challenge, see here, here and here.

(A footnote: This item marks the first video appearance of former President Bush and Kevin McCormack on this blog.)

Golden State Milestone: Stem Cell Agency Announces its First Phase Two Clinical Trial

The California stem cell agency today scored a first with the announcement that one of its projects – a heart disease therapy -- has now advanced to a phase two clinical trial after successfully completing a phase one safety trial.

In a press release, Jonathan Thomas, chairman of the $3 billion research effort, hailed the Allstar trial by Capricor Therapeutics, Inc., of Beverly Hills. He declared,

"This is a highly significant announcement for us at CIRM as it's the first time we have funded a therapy into a Phase 2 clinical trial.

"Heart disease claims around 600,000 American lives every year, so clearly there is a huge need for new approaches and more effective therapies. We are hopeful this is the first of many treatments to turn the tide against this disease, and that this will be the first of many projects we are funding to get to a Phase 2 trial."

The adult stem cell therapy “uses unrelated donor-derived stem cells, called cardiosphere-derived cells, that are then infused into a patient’s artery with the aim of reducing scarring caused by heart attacks,” CIRM said.

Capricor Therapeutics, which has its roots in Cedars-Sinai in Los Angeles, describes itself as as “a diversified heart failure biotechnology company.” It came into being last summer as the result of the merger of Capricor and Niles Therapeutics. The company has benefited from about $27 million in support from the stem cell agency, including $6.9 million for early work by one of its founders Eduardo Marbán at Cedars-Sinai.

The company's stock price closed at $2.48 today, down 17 cents. Its 52-week range is from $2.48 to $2.65.

The firm's CEO, Linda Marbán, said the move into phase two was a “giant leap” for the firm and the heart therapy field.

The executive chairman of the firm is Frank Litvack, a heart surgeon who was the only other candidate for chairmanship of the stem cell agency when Thomas won the job in 2011.

The announcement today is a milestone for the stem cell agency, which is aggressively seeking results that will help to generate financial support after 2017, when its current state funding runs out.

The CIRM news release said,

“The next phase will involve an estimated 300 patients who have had heart attacks, and they will be evaluated in a double-blind, randomized, placebo-controlled trial.  This will be further broken down into two groups: one will include patients 30-90 days post attack, the second will be 91 days to one year after the incident.”

The phase two trial is to determine the therapy's effectiveness and further study its safety. If successful, it can move into a phase three trial to confirm its effectiveness, monitor side effects, compare it to common treatments and collect information that will allow the drug or treatment to be used safely.

Here is a CIRM video in which Eduardo Marbán discusses some of the issues involved in the therapy. Here is a link to a 2011 presentation to the CIRM governing board about the early research – a presentation that generated some excitement on the board.

California Stem Cell Agency to Court Patient Groups This Summer

Jonathan Thomas, chairman of the California stem cell agency, said this morning that he and a team from the agency will begin a round of meetings this summer with patient advocate groups throughout the state.

He said the effort is aimed at keeping the groups up to speed on developments at CIRM. While Thomas did not mention it to the agency's governing board, it is also critical that the agency have strong support from patient advocate groups as it tries to develop new sources of funding, either public or private.

The agency will run out of cash for new grants in 2017 and hopes to have a plan for the future before the board later this year. Its initial assumptions include as much as $200 million in onetime public funding with more cash coming from the private sector.

Currently the agency is funded by state bonds at a cost of about $6 billion, including interest. It spends roughly $300 million a year on grants and loans for research.

Two More Editorials: The California Stem Cell Agency Should Heed IOM Recommendations for Reform

Two other major California newspapers today said the $3 billion California stem cell agency needs to “clean up its act” if it wants to be successful in continuing its efforts at turning stem cells into cures.

The editorials appeared in the Los Angeles Times, the state's largest circulation newspaper at more than 700,000, and the San Jose Mercury News in California's Silicon Valley. The Mercury News has a reported circulation of nearly 600,000, although that figure includes other Bay Area newspaper owned by the same chain.

Both editorials focused on the 17-month evaluation of the agency by the prestigious Institute of Medicine (IOM) as did earlier editorials in The Sacramento Bee and the San Francisco Chronicle. The IOM recommended sweeping reforms at the agency that would alter its structure and target conflicts of interest. 

The Times said,

“The $700,000 spent on the study...will be wasted if the institute's oversight board fails to heed the (IOM) committee's criticisms, which echo the findings of the Little Hoover Commission and other groups over the years.”

The editorial continued,

“The 29-member board is made up almost entirely of representatives of advocacy groups and research institutions that have a direct interest in how the money is spent.”

The Times cited the California Stem Cell Report's calculations that about 90 percent of the $1.7 billion awarded by CIRM has gone to institutions linked to current and former members of its governing board. 

The Times noted an award to a Northern California firm that has stirred some criticism. The editorial said,

“The board also overrode the advice of its scientific advisors — twice on a single application when it considered a grant for a well-connected company, StemCells Inc. based in Newark, Calif. The board granted the company $20 million after Robert Klein, the driving force behind the passage of Proposition 71, which created and funded the agency in 2004, and its former head, lobbied so intensively for the company that one board member described it as 'arm-twisting.'"

The Times concluded,

“The agency has used more than half of its funding and one day will almost certainly want to ask taxpayers for more. It should remember that voters will look for evidence of public accountability as well as respected research.”

The San Jose paper sounded a similar note about the agency. Its editorial said,

“(I)f it wants to survive...it should heed the Institute of Medicine's advice to eliminate conflicts of interest on its board -- and do it before awarding the remaining $1.2 billion of the $3 billion voters approved for stem cell research.”

But the paper said the stem cell agency should not be provided any more state funding.

“Long-term funding was never the intent when Proposition 71 passed in 2004. It was supposed to kick-start research at a time when federal funding was blocked and to establish California as a major player in the rapidly advancing medical field. 

“The agency could continue to bring value to the state as an advocate and funder of research, but only if it can attract private donors, partners and investors. For that to happen, it will need a board that passes the ethics test, with more independent experts and industry executives free of conflicts. 

“At the outset, stem cell advocates took immense pride in structuring the agency to keep it relatively free of legislative interference despite the use of public money. Politicians kept their hands off, which was good. But the agency created its own inappropriate influences in the way it constituted its board. Now it needs to clean up its act.“

Politics and the Price of Defiance

The California stem cell agency last month had a bit of instruction in politics when directors chose a new chairman.

Both California Gov. Jerry Brown and state Treasurer Bill Lockyer made an extra effort – beyond their nominating letters – to assure the election of their man, Jonathan Thomas, a Los Angeles bond financier, as chairman. We noted a few days ago that such episodes are not uncommon when an enterprise is reliant on public funding.

Implicit in some of the lobbying by Brown and Lockyer was the likelihood that failure to follow their lead could endanger bond funding for CIRM.

Some might think that possibility was remote, that Tamany Hall tactics are just a part of the past. Those who favor that view might want to consider the case of Democratic Assemblyman Anthony Portantino. He voted no on the recently passed state budget in defiance of legislative leaders in his own party.

Now he is being punished. His staff is being sent home for a month of unpaid leave, and he is being threatened with a cutoff of both his mail service and office supplies, according to The Sacramento Bee. Such are examples are not uncommon in California politics in one form or another.

It is something to think about as readers ponder the wisdom of CIRM directors in electing Thomas as their new chairman.