NYTimes: Fresh and Major Attention to Immunotheraphy and Cancer

The New York Times personalized its look at immunotherapy -- Times graphic

The New York Times this weekend unveiled a dramatic, special report on immunotherapy to treat cancer, a field in which California's stem cell agency has invested tens of millions of dollars.

The Times' work will undoubtedly focus greater public attention on the use of immunotherapies and generate a fresh surge of interest on the part of patients who are unfamiliar with the expensive treatment, which is very much in its infancy. The Times wrote,

"Harnessing the immune system to fight cancer, a medical dream for more than a century, is becoming a reality. Terminally ill patients who had slim chances of surviving have gone into remission and patients with inoperable tumors are gaining months or years. But so far, the new treatments work for only a minority patients, and doctors are not sure why. The treatments are prohibitively expensive, and not all patients have access to them."

The Times did not deal with any of the work specifically backed by California. But as recently as March, the $3 billion California Institute for Regenerative Medicine(CIRM), as the stem cell agency is formally known, made another investment in the field with more than $10 million to two researchers, William Murphy of UC Davis and Lili Yang of UCLA. (See Tran1-08533 and Tran1-08635 here.)

In February, Alex Lash of xconomy reported that a new immunotherapy firm has sprung from handsome support by the stem cell agency. He wrote,

"Forty Seven(the firm's name), emerged Wednesday with a program already in clinical trials, a rarity for an academic spinout. Stanford researchers led by Irving Weissman, the director of the school’s Institute of Stem Cell Biology and Regenerative Medicine were able to begin testing the drug in humans thanks to $30 million in funding from California’s stem cell agency, known as CIRM."

Lash said that Forty Seven is named for the CD47 protein on tumor cells that its experimental cancer drugs target.

The agency also sponsored a webinar last year on immunotherapies with participation by the Food and Drug Administration and Kite Pharma of Santa Monica, Ca., and which covered such topics as safety and regulatory issues. The webinar is available here. Slides offered by Margo Roberts of Kite can be found here.

Here are links to the various Times stories.
Harnessing the immune system to fight cancer
What is immunotherapy
Immunotherapy offers hope to a cancer patient but no certainty

NY Times: Stem Cell Theory vs. Flourishing, Dubious Stem Cell Clinics

The New York Times this morning took a crack at coverage of the first-ever study of the wave of dubious stem cell clinics in America. The headline on the Times' prominently displayed story said,

"Stem Cell Therapies Are Still Mostly Theory, Yet Clinics Are Flourishing"

The starting point for the article by Gina Kolata was the study by UC Davis stem cell researcher Paul Knoepfler and bioethicist Leigh Turner of the University of Minnesota.  They reported on June 30 that nearly 600 dubious stem cell clinics have sprung up around the country. It is the first study to document the reach of these businesses. The report, published in the scientific journal Cell Stem Cell,  received heavy mainstream media coverage.

Kolata highlighted the tentative nature of stem cell research. She wrote,

 "In theory, stem cells might be a useful treatment for certain diseases that involve the loss of cells, like Type 1 diabetes, Parkinson’s or osteoarthritis. They are primitive cells that can develop into a range of mature cells and perhaps serve as replacements. But progress is slow. After a flurry of stem cell excitement two decades ago, almost all the research today is still in mice or petri dishes. The very few clinical trials that have begun are still in the earliest phase.

"The problem is that stem cell therapies are still mostly theory. So what is going on? How can there be clinics, even chains of clinics run by companies, offering stem cell treatment for almost any disease you can think of — sports injuries, arthritis, autism, cerebral palsy, stroke, muscular dystrophy, A.L.S., cancer?"

She said that the Knoepfler-Turner study showed "what can happen when regulations fall behind." 

And she noted the limits of federal regulation as well as the failure of state regulatory bodies to deal with the issue. 

Kolata's story was displayed online this morning on the main page of the New York Times. The media attention to the work by Knoepfler and Turner has significantly raised the visibility of the issues involving dubious stem cell "therapies."  A narrowly defined search on Google news this morning turned up nearly 11,000 citations, up from about 7,000 early this month.

The coverage certainly has increased the likelihood of some sort of additional regulatory action. However, such moves take time, and it is not realistic to expect a crackdown anytime soon. Indeed, it may require legislative changes at the federal and state levels.

As for the ethical and medical concerns raised by Knoepfler and Turner and the Times piece, one Times reader -- identified online only as Susan -- said,  

"So what? A lot of what the scientific/medical establishment tries to sell us is just theory. Studies of this and studies of that. A study will come out and 6 months later another study will come out debunking the first study. I believe the problem is that some of these science/medical 'experts' are under the delusion that 'one size fits all.' I disagree. What may kill one person may help another."

Tracking California Stem Cell Awards: Diabetes to Dialysis

The California stem cell agency, as expected, yesterday pumped $10 million into a North Carolina firm for a kidney dialysis device and upped its stake in a San Diego diabetes therapy firm to $60 million.

The awards were first reported (see here and here) by the California Stem Cell Report last week, which will have more on the subject and other awards in the next day or two. Here is a link to the press release from the $3 billion agency. 

California Stem Cell Appeals: Zika, Parkinson's and 'Heart-on-a-Chip'

Three California researchers are asking the directors of the state's stem cell agency today to reject the decisions of its blue-ribbon scientific reviewers and grant them awards ranging up to $2 million.

The scientists are Alysson Muotri, director of the UC San Diego stem cell program; Birgitt Schuele, director of gene discovery and stem cell modeling at the Parkinson's Institute in Sunnyvale, and Kevin Healy, professor in the department of bioengineering at UC Berkeley.

Muotri is seeking $1 million from the California Institute for Regenerative Medicine (CIRM), as the stem cell agency is known. Schuele has applied for $2 million and Healy $1 million.

Alysson Muotri, UCSD photo

In a letter to CIRM's board, Muotri said he is attempting to "set up a human stem cell platform to measure the impact of the Zika virus in the nervous system and to screen potential drugs to alleviate the neurological problems associated with the infection." His application (DISC2-09095) is aimed at filling "fundamental gaps" in Zika knowledge. Muotri, who is from Brazil, wrote,

"I never debated over a grant fund decision before . However, in this case I feel I have the responsibility to alert the ICOC (the CIRM board) about neglecting studies on what we call now the ' Zika virus syndrome.' I can tell by personal experience dealing with this virus in my own lab that this is one of the most dangerous infectious agent I (have) ever seen – one can witness the virus killing brain cells in less than a day."

Birgitt Schuele, TSN photo

Schuele's letter pointed out a new study that she said demonstrated the feasibility of her research (DISC2-08953) involving a therapy for Parkinson's disease. She also included additional information that she hoped would be persuasive to the CIRM board.

Kevin Healy
photo by Laura Peterson, AAAS

In his letter, Healy said his research is aimed at developing "patient specific ‘heart-on-a-chip’ diagnostics that will have a significant impact on the early screening of drugs used to manage hypertrophic cardiomyopathy . Currently, there are no drugs that target specific disease alleles of hypertrophic cardiomyopathy."

He said the main criticism of CIRM reviewers involved the "level of maturity of cardiomyocytes" in his proposed study (DISC2-08990). But he said he proposed using the same protocol as already used by a number of currently funded CIRM researchers.

All three applications were ranked below the cutoff line for funding, which was 85. The score for Healy was 83, Muotri 77 and Schuele 72.

CIRM has funded some applications in the past with lower scores than 85. But in the last two years, it has revised its review and scoring protocols. Since then it has not overridden reviewers' negative recommendations.

The board is scheduled to act on about $30 million in awards today in a telephonic meeting with public locations throughout the state. (See here and here. ) The public can participate in the meeting at those locations, whose addresses can be found on the meeting agenda. The session will be audiocast on the Internet and through an 800-number. Complete information about the Internet access and phone number can be found on the agenda.

Search for a Diabetes Cure: California Stem Cell Agency Hits $60 Million Mark with ViaCyte

The California stem cell agency next week is slated to award  an additional $4 million to ViaCyte, Inc., a San Diego firm that is working with the agency to develop a "virtual" cure for diabetes.

ViaCyte has already received $56 million over the years from the California Institute for Regenerative Medicine(CIRM), as the agency is formally known. The latest award will be used to help prepare for a clinical trial for a new treatment for the highest risk diabetes patients.

In response to a query this morning from the California Stem Cell Report, Paul Laikind, president and CEO of ViaCyte, said that the new product, PEC-Direct, will deliver the same biologic component as used in the firm's current product, which is now in a clinical trial.

Laikind said PEC-Direct will allow direct vascularization, which "is expected to allow for a very robust engraftment and cellular performance."

Because the new device will require chronic immune suppression, Laikind said,

"It is being developed to treat patients with type 1 diabetes that are at high risk for acute complications such as severe hypoglycemic events associated with hypoglycemia unawareness syndrome."

(The full text of Laikind's remarks and research update can be found here.)

The agency's summary of the closed-door review of the Viacyte proposal said,

"There are over 100,000 people in the U.S. with type 1 diabetes so severe that they are at constant risk of hospitalization and/or death. Within months after administration, this product could naturally restore those patients’ blood sugar to normal healthy levels and save their lives." 

In addition to the $4 million, ViaCyte is putting up $994,343 of its own cash.

CIRM's blue-ribbon panel of scientific reviewers has already approved the award, which is part of a $30 million package of applications coming up next Thursday.  The governing board of the agency will ratify the approvals at its meeting next Thursday during a one-hour telephonic meeting.

The largest award, $10 million, will go to Humacyte, Inc., of North Carolina,  to produce an artificial vein for use in hemodialysis as reported yesterday on the California Stem Cell Report. More details of all the applications and review summaries can be found on the meeting agenda.

The session will be audiocast and carried on the Internet on a listen-only basis. Interested parties can participate in the meeting from telephonic locations throughout the state.

The main site for the meeting will be at the Sanford consortium in San Diego. Other locations include the agency's headquarters in Oakland and sites in Davis, South San Francisco(2), Napa, Los Gatos, Sacramento, Irvine, San Francisco and Los Angeles. For specific addresses and instructions for online access, see the agenda.

Text of ViaCyte's Statement on its New Proposed Diabetes Product

The California Stem Cell Report this morning queried Paul Laikind, the CEO of ViaCyte, Inc., of San Diego, about the company's upcoming $4 million award from the California stem cell agency and the award's relationship to the ViaCyte product known as VC-01. Here is the text of his response.

"ViaCyte is continuing development of VC-01 (what we now call PEC-EnCap).  PEC-EnCap is the first encapsulated allogeneic cell therapy to enter the clinic where the device is designed to protect the cells from the host adaptive immune system.

 "We have early clinical demonstration of the feasibility of the PEC-EnCap approach; the Encaptra Drug Delivery Device appears to be functioning as designed and we have shown engraftment and differentiation to beta cells is achievable at 12 weeks.  However, work is continuing to ensure a robust and consistent engraftment before we move to the second cohort of patients and seek to demonstrate efficacy.

 "We can’t overemphasize enough the importance the clinical results we are obtaining, not only for the further development of PEC-EnCap but for the stem cell-derived cell therapy field in general. This important work has, of course, received important support from CIRM. "In parallel with the development of PEC-EnCap we are preparing to initiate clinical development of VC-02 (PEC-Direct).  PEC-Direct delivers the same biologic component as PEC-EnCap, PEC-01 pancreatic progenitor cells, but does so in a device that allows direct vascularization.  This direct vascularization, based in part on what we have learned with PEC-EnCap during the clinical evaluation, is expected to allow for a very robust engraftment and cellular performance.

"Given the open nature of the device, patients implanted with PEC-Direct, as with other transplants, would require chronic immune suppression.  Thus it is being developed to treat patients with type 1 diabetes that are at high risk for acute complications such as severe hypoglycemic events associated with hypoglycemia unawareness syndrome.

 "This high risk patient population is the same population that would be eligible for cadaver islet transplants, a procedure that has been demonstrated as very effective but suffers from a severe lack of donor material and high cost.  PEC-Direct is expected to overcome these limitations by providing an unlimited supply of cells for transplant and a safer more optimized route of administration.

"ViaCyte remains committed to the development of PEC-EnCap which we view as a potentially transformational therapy for the majority of insulin utilizing patients with diabetes, both type 1 and type 2.  PEC-Direct represents a potentially nearer term therapy for the patients at the highest risk."

$10 Million California Stem Cell Award for Creation of a New 'Lifeline" for Kidney Disease Patients

A 2014 interview with Laura Niklason, whose firm, Humacyte, will receive a $10 million award from California. The interview was conducted at the Stem Cells Meeting on the Mesa in La Jolla, Ca.

Highlights
$10 million matched by Humacyte
Three clinical trial locations in California
Humacyte raised $150 million last year

The California stem cell agency is set to award nearly $10 million for a phase 3 clinical trial to produce a new type of "lifeline" for kidney disease patients undergoing hemodialysis.

The $10 million award to Humacyte, Inc., of Morrisville, N.C., will go for final testing and development of an artificial vein that is critical for kidney disease patients who need hemodialysis.

The $10 million will be matched by the privately held firm. The state funds can only be spent for work performed in California to test the vascular access device. Humacyte plans clinical trial sites in Sacramento, Long Beach and Irvine.  

Formal approval of the award is expected to come July 22  at a telephonic meeting of the $3 billion agency's board of directors.

The stem cell agency traditionally does not identify recipients of awards until its board formally ratifies the decisions of its reviewers, which it almost never overturns. The California Stem Cell Report determined the identity  of the recipient using public sources.

According to the National Institute of Diabetes and Digestive and Kidney Diseases,

"A vascular access is a hemodialysis patient’s lifeline. A vascular access makes life-saving hemodialysis treatments possible. Hemodialysis is a treatment for kidney failure that uses a machine to send the patient’s blood through a filter, called a dialyzer, outside the body. The access is a surgically created vein used to remove and return blood during hemodialysis."

The Humacyte device, photo Humacyte

A summary of the closed-door review of the application described the product as a "human acellular vessel" that "has the potential for less frequent clotting, abandonment and infection." The summary said current technology is "fraught with complications."

Humacyte was founded in 2004 and raised $150 million last year in venture capital, according to an article last month by Meghana Keshavan on the STAT online biomedical news service. 

In the piece, Keshaven described the process of growing the vessels. She said each has "its own little plastic sack, which serves as a sort of womb for the vessel as it grows. Each bag’s connected to a central bioreactor tank that pumps out all the nutrients it needs — a carefully crafted 'soup' of vitamins, amino acids, and chemicals called cytokines that feed cells directions about how they’re supposed to grow."

Keshaven continued,    

"The lab-grown blood vessels are currently being studied in hemodialysis, a procedure that uses implanted veins as a conduit to remove waste from the blood of patients with kidney failure. Last month, promising results from a 60-patient midstage trial of Humacyte’s product were published in The Lancet."

Humacyte currently is recruiting 350 patients and expects to have preliminary results by July of next year. In response to a question this morning, Jeffrey Lawson, chief medical officer of the firm, said, 

Shannon Dahl,
co-founder Humancyte

Juliana Blum,
co-founder Humacyte

"We currently are engaged on using at least 3 clinical sites in California that should start enrolling patients (Sacramento, Long Beach and Irvine). We also have a number of manufacturing support programs based in California."

Founders of the firm include  Laura Niklason, professor of biomedical engineering and Yale, and Shannon Dahl and Juliana Blum, vice president of the firm.

More Than a Minor Headache: The Stem Cell Snake Oil Problem vs. Legitimate Research

Just two weeks ago, the headlines from Bloomberg News offered a glowing view of the prospects for stem cell therapies and the industry. The story in the online publication, which counts nearly 9 million readers monthly, said,

"Stem cell crusader sparks new hope....Regenerative medicine could be a $120 billion industry by 2030"

But only three days later, that article was overwhelmed by a wave of stories with a much different flavor. They carried reports of unproven therapies, possibilities of fraud, lack of regulation and fearful, expensive consequences for desperate patients.

That theme continued even as recently as this past weekend as The Economist carried a piece headlined,

"A dish called hope: The flourishing, unregulated industry in expensive, experimental treatments"

So which is it? Hope for legitimate cures and oodles of cash for stem cell companies? Or "hope" for treatments that do not work and have sometimes damaged both the bodies and wallets of the patients?

The situation is more than a minor public relations headache for the supporters of stem cell research, be they patients, stem cell companies or the $3 billion California stem cell agency, which is on a campaign to ease federal regulation of stem cell clinical trials.

For all practical purposes, the public generally does not make much of distinction between the legitimate research conducted by institutions such as Stanford and the so-called "miracles" reported about the late hockey great Gordie Howe and others. It all goes into the same cognitive bag. The stories about mysterious and fearsome tumors found in little-known patients attract little widespread attention.

But UC Davis scientist Paul Knoepfler and bioethicist Leigh Turner of the University of Minnesota ripped open that bag with their study last month that reported for the first time that nearly 600 dubious stem cell clinics are peddling their unproven therapies across the country.  Their report supported the view that the stem cell field is rife with snake oil merchants. For the general public, which does not delve deeply into medical research, the perspective could well be described as, "You seen one stem cell therapy, you seen 'em all."

Does this mean that these clinics should be ignored and shoved quietly off into a corner in order to avoid besmirching legitimate efforts? Of course not. It certainly appears that the FDA and other  state regulatory bodies can do a better job. But stiffer regulations are not going to come anytime soon, despite an FDA hearing on the topic in September.

However, the situation DOES mean that folks like Knoepfler and Turner should continue to speak out along with many other scientists who do not want their efforts blackened by the snake oil men. Researchers can work with their institutions' PR departments to place op-ed pieces, find speaking engagements and gin up TV and radio interviews. Blogs, like the one produced by Knoepfler, can be started. The International Society for Stem Cell Research should revive its public education efforts to help patients and the general public understand the facts about stem cell research. It should also reinvigorate its warnings about dubious therapies, which were throttled back a few years ago, reportedly after legal threats were made.

California's stem cell agency has a special concern. It is trying to "de-risk" development of therapies  by a variety of means and lure biotech and Big Pharma into the stem cell game in a bigger way. This summer the agency is offering $75 million to entice a private partner into the state's first-ever, public-private partnership to create a stem cell cure.

The private sector has shied away from the stem cell business, as the stem cell agency's CEO, Randy Mills, has remarked on multiple occasions. One of the reasons involves the public climate and perception of the field. It is hard for companies to invest hundreds of millions of dollars or billions when the stem cell field is burdened with public perception and regulatory obstacles.

Selling the stem cell story in a realistic way is not necessarily an easy task. Nuances must be respected, but excitement is also needed. Balancing it all is a challenge for the men and women in the trenches. But without a good push, development of therapies will be slower, and, as Mills has noted, people will undoubtedly die who likely would have benefited from a more timely treatment.

A Scientist's Comment: Deal Directly with FDA Rather Criticizing It in the Media

The following comment on FDA regulation of stem cell research was submitted via email by a longtime scientist in the field, who asked to remain anonymous. 

"Many advertised  'stem cell' treatments use hematopoietic or blood stem cells which are run under a different set of regulations from pluripotent stem cells since they are umbrella-ed under regulations designed to cover blood transfusions and bone marrow transplants.  Pluripotent stem cells are very new, with many unknowns and the potential for triggering tumor growth.   In contrast, clinical use of blood stem cells has been ongoing for over 50 years with special regulations (or lack thereof) grandfathered in.   Unfortunately, the newly touted 'treatments' are not for homologous use of blood stem cells, are unlikely to be therapeutic and may even be dangerous. 

"The FDA has published new draft guidances addressing these issues that will be discussed in open forum this September.  I think it best to bring concerns about FDA regulations to these meetings and to initiate change by talking directly with the agency rather than criticizing them in the press or through Congress.  Like their oversight over the IRS and other federal agencies, Congress’s actions are counterproductive when they add broad, unfunded mandates while cutting funding at the same time.  But perhaps this is their way of shrinking the government and simultaneously dinging them for not doing their work.  A sure route to failure."

Monty Python, $3 billion and Stem Cells in the Golden State

Inquisition scene from Monty Python, photo CIRM/Daily Mail

The headline today from California was nearly irresistible:

"The Spanish Inquisition and a tale of two stem cell agencies"

Not only that, but the headline appeared over an article involving Monty Python, the $3 billion California stem cell agency and the lesser,  $38 million United Kingdom's Regenerative Medicine Platform(UKRMP). 

The "Inquisition" article ran on the California agency's blog. The author was Kevin McCormack, senior director of communications for the California Institute for Regenerative Medicine(CIRM), as the agency is formally known. 

His topic? How the UK and California efforts have performed, according to an article by Stanford's Irv Weissman and Fiona Watt of King's College London, who is a member of the CIRM scientific advisory board, currently "on hold," according to McCormack.  The Watt-Weissman piece was recently published and buried behind a paywall in Cell Stem Cell.

McCormack noted that the article cited location and politics as important in both operations. He wrote, 

"CIRM was created by the voters of California in 2004, largely in response to President George W. Bush’s restrictions on the use of federal funds for embryonic stem cell research. UKRMP, in contrast was created by the UK government in 2013 and designed to help strengthen the UK’s translational research sector....

"Inevitably the two agencies took very different approaches to funding, shaped in part by the circumstances of their birth – one as a largely independent state agency, the other created as a tool of national government.

"CIRM, by virtue of its much larger funding was able to create world-class research facilities, attract top scientists to California and train a whole new generation of scientists. It has also been able to help some of the most promising projects get into clinical trials. UKRMP has used its more limited funding to create research hubs, focusing on areas such as cell behavior, differentiation and manufacturing, and safety and effectiveness. Those hubs are encouraged to work collaboratively, sharing their expertise and best practices."

Irv Weissman, photo wapiti-waters

Weissman and Watt said it was "not unexpected" that CIRM still has not produced a therapy for widespread use. They cited the sometimes decades-long time frame for transforming basic research into a therapy.

Which is where Monty Python comes in, said McCormack. The two scientists picked up a famous line from the British comedy series:

“Nobody expects the Spanish Inquisition – because our chief weapon is surprise.”

Fiona Watt, photo Watt Lab

McCormack wrote,

"They use that to highlight the surprises and uncertainty that stem cell research has gone through in the more than ten years since CIRM was created. They point out that a whole category of cells, induced pluripotent stem (iPS) cells, didn’t exist until 2006; and that few would have predicted the use of gene/stem cell therapy combinations. The recent development of the CRISPR/Cas9 gene-editing technology shows the field is progressing at a rate and in directions that are hard to predict; a reminder that that researchers and funding agencies should continue to expect the unexpected."

Influential Scientific Journal Rips Effort to Loosen Stem Cell Research Rules

The prestigious journal Nature today editorialized against easing federal regulation of stem cell treatments, a major blow to the campaign by the California stem cell agency to speed such therapies to the marketplace.

The headline on the piece said,

"FDA should stand firm on stem cell treatments."

The unsigned editorial declared that those who contend that the Food and Drug Administration (FDA) is holding back "effective therapies" are peddling a false narrative. Nature declared,

"The claim that regulation is too harsh wrongly implies that the FDA is holding back therapies that work. Critics point to decades of preclinical and clinical work with stem cells and the pipelines of stem-cell treatments. With circular logic, they argue that, because the treatments have not been approved, there is something wrong with the approval system."

The $3 billion California stem cell agency has been lobbying for months for changes in FDA regulation. Randy Mills, president of the California Institute for Regenerative Medicine(CIRM) as the agency is formally known, said back in December that "patients are dying" because the FDA is  being "so careful about safety."

Mills has said the fast-track process in Japan is worth considering. However, Nature said,

 "It is not a fit and proper model to export, chiefly because it grants 'conditional approval' to treatments with minimal safety data and little attention to efficacy."

Nature was also critical of the Regrow Act, a legislative effort to overturn the FDA's current processes. CIRM has not taken a position on that bill.

Nature concluded,

"The FDA should strive to keep this debate on the proper topic — how to create a more efficient system that still scientifically evaluates whether treatments are safe and efficacious. To fall short would be a setback for science, and for patients."

The journal's editorial did not mention CIRM or Mills by name. It did cite the recent article by UC Davis stem cell researcher Paul Knoepfler and Leigh Turner, an associate professor of bioethics at the University of Minnesota, documenting the existence of 570 dubious stem cell clinics in the United States.

'False Hopes, Sizable Profits' -- The Nation's Largely Unregulated Stem Cell Clinics

Locations of stem cell clinics identified in Knoepfler/Turner study
Graphic by Cell Stem Cell 

The biggest stem cell story in the country this week reported that at least 570 dubious stem cell clinics exist throughout the nation that could pose a danger to both the pocketbooks and health of patients.

California led the nation with 113 clinics, with Beverly Hills having the most (18) of any city. The clinics promise to cure or ease afflictions ranging from autism to Alzheimers through the use of scientifically untested treatments.

The study is the first effort to get a handle on the size of an industry that has been little regulated by the Food and Drug Administration (FDA) but appears to promise almost miraculous results.

Results of the study were reported yesterday in the Washington Post, the Los Angeles Times, Fox News, The Sacramento Bee and many other outlets.  A search at midday today on Google turned up 7,230 citations, including articles in France, Mexico and Italy.

One of the authors of the study from the journal Cell Stem Cell is Paul Knoepfler, a stem cell scientist at UC Davis.  He has long campaigned for more action dealing with some of these clinics. In September 2015, he wrote on his blog about what he called "predatory clinics."

"These clinics prey on vulnerable patients and their families. The clinics use hope as a marketing tool. A weapon.

"As the number of such clinics has mushroomed in the US and elsewhere the risk to both patients and to the larger stem cell community proportionately rises too. We are in a situation today where the dangers from such clinics have never been higher."

Co-author of the study, Leigh Turner of the University of Minnesota, said in an article in The Bee by Adam Ashton,

"The problem is that so much of the information that’s provided by the industry, so many of the marketing claims being made, they’re not particularly accurate. They’re not trustworthy, and they don’t have any meaningful science behind them."

In a story by Karen Kaplan in the Los Angeles Times, Turner said,

"There is an obvious need for the FDA, FTC (Federal Trade Commission), state medical boards and other regulatory bodies to play a more effective role in regulating the marketplace for stem cell interventions.' Without it, these clinics are able "to peddle false hope for sizable profits.'"

Randy Mills, president of California's $3 billion stem cell agency, said in The Bee that the Knoepfler/Turner study "highlighted a 'broken system' at the FDA that lets some clinics proceed with almost no oversight while requiring others to undergo studies that could tie up their services for decades. He said there are likely far more clinics offering stem cell therapies than Knoepfler and Turner identified, with many of them operating discreetly.

"'There’s so much stuff going on today, but it’s going on because that’s the system the FDA set up and that’s the system the FDA incentivizes,' he said."

Knoepfler noted a bit of irony yesterday concerning the study in a posting on his blog, The Niche. He wrote, 

"I’m glancing over at the (Scientific American) article (on the study) just now and BOOM I see an ad for a stem cell clinic appear at the top and it is even one of the ones in the database we created for our article. The ad is focused on selling stem cells to treat autism in children, an area that raises a lot of questions.

"How can it be a good thing to have an ad for a stem cell clinic right next to the article about the challenge of stem cell clinics in America? The way the web works these days, perhaps it isn’t so surprising that articles having anything to do with stem cells will often be accompanied by ads for stem cell clinics. This one just really stuck out to me today because of the context."

Google and other media outlets routinely place advertisements for these stem cell clinics on many Web sites and blogs, including this one. Some of the newspapers carrying stories about the Knoepfler study also carry ads for such clinics. 

California Stem Cell Hooha: Testy Media Exchange Triggered by Criticism of the State's Agency

A sharp exchange erupted this week on a mainstream California media site after the state's $3 billion stem cell agency was taken to task for lobbying the federal government to ease its regulation of research on stem cell therapies.

The flap involved allegations of lies and questionable motives. Along the way certain death was mentioned, and one writer was accused of "stealing hope" from seriously ill persons. Personal animus charges were raised along with questions about ill-informed rhetoric.

The hooha was set off by an op-ed piece by UC Davis stem cell scientist Paul Knoepfler who wrote an op-ed in the San Francisco Chronicle (see here and here). It chastized the agency and its president, Randy Mills, for a risky and "political" effort to change regulations by the federal Food and Drug Administration. Knoepfler said patients could be put at risk.

The piece triggered harsh comments that were carried by the Chronicle at the end of the article. In addition to Knoepfler, individuals involved in the exchange included Jeanne Loring, head of the stem cell program at the Scripps Institute in La Jolla; Judy Roberson, a Sacramento patient advocate for Huntington's disease, and a person identified only as "Jeff." He wrote,

"What's Knoepler's real beef?....Poor Paul is not getting the money from CIRM he used to get. So his attitude is 'screw patients,' I want my lab funded like it used to be. Nauseating!!!"

CIRM is the abbreviation for the formal name of the state stem cell agency, officially known as the California Institute for Regenerative Medicine. Its board includes Jeff Sheehy, who told the California Stem Cell Report that he is not the Jeff commenting on Knoepfler's article.

The anonymous "Jeff" said, 

"Has any of (Knoepfler's) research led to a development candidate that might lead to a treatment in the future? Or is he just a stem cell scientist opining about something he knows very little or nothing about(the FDA)? Maybe he 'heard' something from somebody and that gave him just the hook he needed to attack CIRM and Mills, both of whom he obviously has personal animus towards."

Loring largely sided with Knoepfler. She also wrote,

"Jeff, why are you questioning Paul's motives? What are your motives? Do you work for CIRM? As you know, a person's profession doesn't define his/her sphere of knowledge. If something is important to you, you can research it and gain expertise."

Roberson, who favors the CIRM effort to loosen regulations to bring therapies into widespread use, said patient safety is important. She also said,

"But for patients and their families with a 100 percent fatal genetic disease like Huntington's disease, we can withstand some risk because we are facing a gut-wrenchingly long, gruesome death anyway. We have no chemotherapy, no surgery, no radiation and sadly, no hope. We have NOTHING! And we'll continue to have nothing for many more decades if the FDA continues doing business as usual."

Another reader, William Barnes, said,

"I suggest that Dr. Knoepfler consider how it feels to be suffering and near certain death and be denied a possible cure because Dr. Knoepfler doesn't think it's in their best interest....Let the individual patient decide! It’s their life! Why should the decision about someone’s life, possibly your loved one, be made by a distant and dispassionate academic?"

In addition to his research, Knoepfler produces a blog, The Niche, on stem cell science, regulation and policy. He took up the blog after a nasty bout with prostate cancer when he was in his 40s. Knoepfler is also the author of "Stem Cells: An Insider's Guide," a book aimed at helping patients understand stem cell science.