Thursday, February 16, 2017

Defying Basic Medical Know-how, Stem Cell Treatments and Fake News

A racing car driver, a celebrity TV surgeon and allegations of stem cell "fake news" surfaced this week on California stem cell blogs.

It was a matter of Dr. Oz, A.J. Foyt and a company called Cell Surgical Network Corp. of Rancho Mirage, Ca., which UC Davis stem cell scientist Paul Knoepfler says is the largest affiliated group of stem cell clinics in the United States.

But first Oz and Foyt. They were the subject of an item on the The Stem Cellar, the blog of California's $3 billion stem cell agency,

Kevin McCormack, communications director for the Oakland-based agency, wrote the piece, which was headlined "TV's Dr. Oz takes on clinics offering dubious stem cell treatments."

Foyt has said he has signed up for stem cell treatment in Mexico for issues stemming from his many injuries sustained in his very successful career in auto racing. Oz this week ran an investigative piece dealing with some of the 570 clinics in this country that offer unproven treatments.

The Oz show said that complications and death have resulted in some cases from treatments at these clinics here and abroad.

McCormack's concluding sentence:
 "Perhaps someone should tell A.J. Foyt."
Michael Hiltzik, a Pulitzer Prize winning columnist with the Los Angeles Times, also had an article concerning the Oz show, which reported that the treatments being offered at many of the 570 medical clinics defy "basic medical know-how."

Hiltzik also wrote that the Oz provided a "a withering assessment of doctors who claim to be engaged in clinical trials of stem cell treatments but 'ask you to give money upfront and mortgage your house and borrow from your friends’ credit cards — that’s not how medicine should be practiced.'"

Davis' Knoepfler dealt with the Cell Surgical Network and discussed its possible use of "laboratory-proliferated stem cells" in patients, which Knoepfler indicated would require federal approval.

The matter was addressed in an email Q-and-A with the leaders of the corporation, Mark Berman and Elliott Lander.

Berman and Landers' final point:
"All we care about is our patients. Providing them with the best and safest regenerative medical care in the world is what Americans deserve. We are not interested in anyone who desires to slow or obstruct this patient care by manipulating regulators into criminalizing certain medical practices. Therefore, we continue on our mission and ignore the fake news and rumors that generate blog ratings and spread fear and mistrust."
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Tuesday, February 14, 2017

California to Hand Out $32 Million for Stem Cell Research Next Week

The California stem cell agency next week is expected to award as much as $32 million for late stage research and clinical trials involving therapies for arthritis of the knee, type 1 diabetes, an immunodeficiency affliction and ALS. 

Also on tap for the Feb. 23 governing board meeting are concept proposals for expansion of the Alpha Clinic program along with unspecified changes in the $3 billion agency's discovery, translation and clinical plans. 

Four awards are already approved by the agency's reviewers and are scheduled for routine ratification by the board. Their review summaries can be found on the agenda. The reviewers also rejected one proposal for research involving Parkinson's disease. That summary can also be found on the agenda.

More details on the concept plans are expected to be posted soon. The meeting will take place in Oakland with public teleconference locations in in San Diego, Los Angeles and two in La Jolla. Addresses can be found on the agenda. 
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Sunday, February 12, 2017

Cost of a Stem Cell Therapy? An Estimated $900,000

What is the likely cost of a freshly minted stem cell therapy? Close to $900,000. That's at least by one current estimate.

In the United States, such calculations are rare. Researchers and biotech executives shy away from discussing in public such daunting figures.

The figure emerged last week, however, in news from Japan about groundbreaking research to treat macular degeneration with reprogrammed adult stem cells.

While stem cell insiders are not keen on discussing $900,000 therapies -- at least their cost -- the public, however, is deeply interested. Development of expensive therapies is also likely to play a role in the future of California's $3 billion stem cell agency, which expects to run out of cash in 2020. Voters may look askance at publicly financed therapies that appear to be out of reach.

Exorbitant health care costs are on the minds of many. Forty-seven percent of the public said in 2016 that cost and access are the nation's most urgent health care problems, according to a Gallup Poll. Of all the nearly 4,300 items published on the California Stem Cell Report over the last 12 years, the most widely read article deals with the cost of stem cell treatments.

As of this morning, the 2013 article had recorded 21,963 page views, a standard way of measuring readership on web sites. Another related document chalked up 27,699 views on Scribd, where it was also published by the California Stem Cell Report. The figures are roughly four and five times higher than other relatively well-read pieces.

Readers do not give reasons for choosing the articles. But it is likely that their pocketbooks and hopes of affordable therapies are driving their interest.

Affordability was a big issue in the creation of the stem cell agency via a ballot initiative in 2004, Proposition 71. The agency, formally known as the California Institute for Regenerative Medicine (CIRM), has not devoted any significant attention to the matter in the last few years.

But if the agency wants to secure additional public or even private funding, it will need to make the case that its work is more than just another entry in the medical arms race.

Just yesterday, OncLive,  an oncology news site, carried a report on the skyrocketing expense of cancer drugs alone, which cost the nation $16 billion annually in 2010 and jumped to $38 billion in 2015. As for individual cancer patients, they are looking at costs of more than $150,000 a year for drugs, figures that have generated a ruckus in the cancer treatment community.

Drug costs are a small part of the total health care bill for country. But they are a litmus test for policy makers and the public. The costs are relatively straight forward compared to some other health care measures. But they are readily understandable by most families, who usually have one member or more involved in prescription purchases.

 As efforts to repeal-and-replace the Affordable Care Act gain increasing attention over the next year, the public is likely to focus even more on the costs of treatments and drugs, whether it is a $19 aspirin or a $900,000 stem cell therapy.

The "good" news, however, last week out of Japan was that the $900,000 cost of the stem cell macular degeneration treatment could be reduced to below $200,000 as the kinks are worked out and the treatment becomes more common -- if it clears its clinical trials.

As for California, CIRM  has pumped $125 million into research dealing with blindness, including macular degeneration which afflicts 1.7 million Americans. Nearly one million Americans are blind from all causes and another 2.4 million suffer significant visual impairment. More information on the state research can be found here. A CIRM video on vision issues is below.

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Thursday, February 09, 2017

Sampling Stem Cell News: $1 Million Gift, Unsettling Thoughts and Paolo Macchiarini

New-fangled pigs, $1 million donations and a recommendation to wind down the stem cell agency, it was all part of the stew of stem cell news recently.

Here is the first bite from recent bits and pieces from the media:

Eli and Edythe Broad added another $1 million to the many millions they have already contributed to stem cell research, much of it in California. The latest cash went to USC, UCLA and UC San Francisco, which have already received many millions more from the Broads. Charlie Rose also interviewed Eli in a six-minute segment that can be found here. Broad told Rose that he does not think the government is doing enough for science.

The Sacramento Bee carried an opinion piece headlined "To fulfill stem cell agency’s promise, consider winding it down." Joe Radato, who was an aide to former California Gov. Pete Wilson, and Bernard Munos of FasterCures were the authors. Instead of providing more funding for the California stem agency, they said a better approach would be to "provide funds directly to California-based companies developing new drugs to cure diseases and prolong healthy lives."

Paul Knoepfler, a UC Davis stem cell researcher and blogger, authored a piece in the Washington Post dealing with the "unsettling thought" of human-pig hybrids. He wrote that more than 100,000 people in the United States are waiting for organ transplants and that these new-fangled pigs could be a source, down the road. Knoepler said the ethical and other obstacles are like to be overcome.

The latest on former super surgeon Paolo Macchiarini was reported by Radio Free Europe Radio Liberty. He is in Russia after being fired by the Karolinska Institute in Sweden after some of his patients died following surgery involving stem cells. The story reported that his activities have been newly restricted in Russia. Macchiarini's operations, which included a 2010 procedure at UC Davis, drew wide-ranging, favorable international attention for a number of years. Sphere: Related Content

Saturday, February 04, 2017

Politics Could Be Key to Future of California's $3 Billion Stem Cell Program

The Los Angeles Times, California's largest circulation newspaper, is carrying an article this weekend that says the future of the state's $3 billion stem cell agency could "depend more on politics than science."

The assertion was carried in a column by Michael Hiltzik, a Pulitzer Prize winner and author, that popped up on the Internet last night. He provided a broad overview of the agency that was less harsh than some of his previous pieces dealing with the California Institute for Regenerative Medicine or CIRM, as the agency is formally known.

Hiltzik wrote,
"CIRM’s leadership knows that the public’s inflated expectations threaten to obscure the program’s real accomplishments. With multiple clinical trials of CIRM-funded research underway, the first government approval of treatments is expected 'in the not-too-distant future,' C. Randal Mills, the program’s president, said in an interview.
"But he acknowledged that expectations 'need to be tempered with humility at the enormity of the task before us. We don’t want to overpromise or overhype. CIRM is doing what it was set up to do, but it might be taking longer than people thought or hoped.' "
Hiltzik continued,
"Still, the program’s future may depend more on politics than science. 'If it looks like Washington is flipping off California, that could have political ramifications' at the ballot box, (Hank) Greely (director of the Center for Law and the Biosciences at Stanford)  says. Some researchers aren’t optimistic about the prospects for independent, federally funded science under the Trump administration."
The reference to the Washington involves the likelihood that the Trump Administration would impose restrictions on federal funding of human embryonic stem cell research. The administration is populated by appointees who hold anti-abortion views that are generally coupled with opposition to embryonic stem cell research on the grounds that it is tantamount to murder. 

Hiltzik's column noted changes at the agency that make it significantly different than its earlier days, including a step-up in funding of clinical trials, the success of which could pay an important role in the success of a new funding measure. 

He wrote, 
"A new funding campaign could give the program a much-needed reboot. The ballot measure could restructure CIRM as an 'ordinary agency of the state' subject to legislative oversight, open meetings laws and other good-government statutes, says Marcy Darnovsky, executive director of the Berkeley-based Center for Genetics and Society."
"If it returns to the ballot, CIRM would have a chance to reconsider its administrative structure, the inflated expectations it gave voters in 2004, its embedded conflicts of interest and even whether it should be limited to funding research into stem cells. All these features of Proposition 71 (which authorized the agency) have created complications during the program’s first decade."
Hiltzik's column is scheduled to appear in print on Sunday, a day on which the Times says it has 2.4 million readers.

Here are links to two other recent overviews of the agency, including one last month on the California Stem Cell Report and Stat News.
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Wednesday, January 25, 2017

Clarifying the Path to Cash: California Looking for Money from Its Stem Cell Investments

CIRM chart
The $3 billion California stem cell agency is moving to revise its rules for royalties and revenues that may be derived from its research, simplifying them while focusing more sharply on likely cash-generating products.

The proposal comes before the agency's Intellectual Property  (IP) Subcommittee Thursday at a 10 a.m. meeting that has a number of locations throughout California where the public can participate.

A document prepared for the meeting said the complexity of the existing IP regulations has led to disagreements, created an excessive administrative burden and treated for-profit and non-profit enterprises differently.

John M. Simpson of Consumer Watchdog in Santa Monica, Ca., who participated extensively in the early development of the IP rules, praised the proposed changes.

Responding to an inquiry from the California Stem Cell Report, he said:
"The proposed changes in the IP regulations should simplify oversight for CIRM and make expectations for all awardees clearer.  It puts nonprofit and commercial entities on the same footing with regard to their revenue sharing responsibilities. Most importantly the new rules will emphasize getting revenue for the state  from  companies who actually commercialize the results of CIRM-funded research.  That’s exactly as it should be. 
“Nonetheless, despite the overblown promises of Prop. 71 campaigners, the state as yet to realize any revenue from research CIRM has funded.  There could be a little money this year."
"This change in the IP rules makes sense and is the best way forward,  but realistically I doubt the state will ever see significant revenue from the research it has funded."
Proposition 71 created the California stem cell effort, known officially as the California Institute for Regenerative Medicine or CIRM. One of the promises of the 2004 campaign was that it would lead to as much as $1.1 billion in revenues to the state. No royalties have yet been announced. 

Telephonic locations for the public exist in Irvine, Napa, South San Francisco, San Diego and San Francisco in addition to the agency's headquarters in Oakland. Specific addresses can be found on the agenda, which also includes directions for a listen-only audiocast.
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Monday, January 23, 2017

From Cystic Fibrosis to Zika: California Awards $21 Million for Stem Cell Research

The California stem cell agency last week approved nearly $21 million for research to tackle afflictions ranging from cystic fibrosis to Zika.

Awards were made to only 11 researchers although the agency's reviewers had earlier approved 14 awards totaling $25.5 million. The governing board, however, had allotted only $21 million for the awards, and it decided to stick to its budget.

The awards are part of an effort by the agency to finance projects that can move forward rapidly within the next two years.

Six scientists sent letters to the board prior to the meeting discussing details of their research and dealing with concerns of the agency's reviewers, who approved awards earlier in a closed-door session, prior to ratification by the board.

Here are links to letters of each researcher: Rosa Bacchetta, Stanford, application number DISC2-
09526; Tejal A. DesaiUC San Francisco, DISC2-09559; Mark Mercola, Stanford, DISC2-09542 (rejected); Julie Sneddon, UC San Francisco, DISC2-09635; Jin Nam, UC Riverside, DISC2-09645 (rejected), and Matthew Porteus, Stanford, DISC2-09637.

Reviewers rejected any application that scored below 85. All of the three applications denied by the board stood right at the cutoff line with scores of 85. Mercola was the only one of the three to write a letter to the board. The agency did not disclose the names of the other two on its web site since they had not written a letter, which is a public record. The Nam application scored 84 and was not recommended for funding by reviewers.

Here is a link to a document that contains summaries of the reviews of all applications scored at 65 or above.

Here is a link to the agency's press release that contains the names of all the winners and a one line summary of their research. More details can be found in the summaries of the reviews. Sphere: Related Content