Thursday, April 17, 2014

NIH Action on Stem Cells: More Than 'Bureaucratic Bungling'

A national stem cell advocacy group this week ripped the National Institutes of Health (NIH) for “dismantling” its Center for Regenerative Medicine, describing the move as a "setback" for the entire field.

Bernard Siegel
GPI photo
The Genetics Policy Institute (GPI) said the action was a “huge disappointment” in an email sent out internationally by Bernard Siegel, executive director of the group, which stages the heavily attended World Stem Cell Summit.

The NIH move also has implications for the California Institute for Regenerative Medicine(CIRM), whose $3 billion program is down to its last $600 million.

The GPI was reacting to news in the journal Nature that the NIH said its center “will not continue in its present form.” The head of the NIH program, Mahendra Rao, took a job this month with the New York Stem Cell Foundation. James Anderson, director of the NIH’s Division of Program Coordination, Planning, and Strategic Initiatives, told Nature that the $52 million NIH effort was going to be rethought following a workshop in May.

Siegel said,
“The dismantling of the program appears to be a retreat by the United States from the translational imperative and a setback for the field at large. The patient community will be looking to Dr. Collins(head of the NIH) and others in NIH leadership to fulfill their commitment to stem cell development as a singular priority. Other countries are pouring in resources and moving full steam ahead. The NIH’s failure to continue the program represents more than just a case bureaucratic bungling. What we see here is a lack of vision and a public relations blunder. Years of valuable work and planning just tossed away. The scientific community and the public rightfully believes regenerative medicine will one day provide innovative treatments and cures to chronic diseases. The decision to tear down the Center for Regenerative Medicine, without first providing an alternative plan, undermines the credibility of NIH. A huge disappointment."

Asked for a response this week by the California Stem Cell Report, Amanda Fine, a public affairs specialist at the NIH, said in an email,
“The NIH Intramural Center for Regenerative Medicine has not closed.  This is a fast evolving area of science and NIH decided to step back and reassess what the field needs in 2014 and beyond and where NIH can have the greatest impact. NIH is holding a workshop tentatively scheduled for May 5 that will convene a group of experts in the field to address current obstacles to translation of cell therapies and will help prioritize a number of requirements that the larger community has articulated through white papers over the past two years.  NIH will consider the feedback from the workshop and establish a set of goals for the CRM.

Initially, Fine asked that the NIH statement not be attributed to a specific individual but later
said it could be attributed to James Anderson, who also made the statements to Nature.

Public details of the NIH program and its problems are murky. But the NIH action has implications for the California stem cell agency, which is scheduled to run out of cash for new awards in 2017. The agency is attempting to raise funds to continue beyond that point.

The agency could use the news to argue that now, more than ever, it is necessary to support stem cell research efforts in California because of weak federal support, which ebbs and flows depending on political vagaries. On the other hand, some might interpret the NIH rollback as a sign that those well-informed in the field have judged it to be less-than-ready for prime time, an argument that could be extended to California's efforts.

What the NIH action clearly does is create more uncertainty concerning progress in stem cell research. Uncertainty is an anathema to businesses that may be considering where to invest hundreds of millions of dollars.
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Wednesday, April 16, 2014

California Stem Cell CEO Search: Three to Four Candidates Remain, Fewer Tomorrow

A key panel of the directors of the $3 billion California stem cell agency today is expected to whittle down its list of candidates to become the new CEO of the organization, which is facing financial extinction in 2017.

Currently there are three or four candidates, all from the private sector, the California Stem Cell Report understands. The agency, however, did not comment on the number or nature of the candidates.

Kevin McCormack, senior director of public communications, only said this morning in an email,
“The full board is meeting to interview the short-listed candidates on April 30 at which point we hope they will be able to agree on the top candidate. If that candidate then agrees to take the job the announcement will be made at the May (29) board meeting(in San Diego).”
 
Today will mark the conclusion of two days of closed-door meetings of the directors' Presidential Search Subcommittee. The focus on industry candidates reflects the agency's push to drive stem cell research into commercialization. The effort is aimed at fulfilling the promises of the 2004 election that created the agency as well as making it more likely to find future sources of funding for the agency, which now subsists on money borrowed by the state. The agency has only $600 million left. Cash for new awards is scheduled to run out in about three years, and no therapies have been produced.


Agency President Alan Trounson announced last fall that he was leaving his $490,008-a-year post to return to Australia. He has continued to fill in on an interim basis.
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Tuesday, April 15, 2014

$124 Million Deal for Keirstead's California Stem Cell, Inc.

California Stem Cell, Inc., founded by the UC Irvine researcher made famous eight years ago by showing a once paralyzed rat apparently walking on national television, was sold this week in a deal reportedly worth at least $124 million in stock and cash.

NeoStem, a New York city-based firm, announced the acquisition yesterday in a move that sent its stock to $6.64 this morning, up roughly 6 percent from last Friday. Its 52-week high is $9.00 and low $5.00.

Gen News reported that the deal “will add a late-stage technology to the acquiring company’s pipeline. Through the deal, NeoStem will take over development of CSC’s Melapuldencel-T, an autologous melanoma initiating (stem) cell immune-based therapy intended to eliminate the tumor cells capable of causing disease recurrence, beginning with the launch of a pivotal Phase III trial.”
Hans Keirstead
UC Irvine photo

The president of the privately held California Stem Cell, Inc., is Hans Keirstead, who was a key figure in the 60 Minutes news show on Feb. 23, 2006. CBS reported that “if paralyzed people are ever going to walk again, it might be because of the scientist (Keirstead) in this story.” 

The piece said he had injected the rats with embryonic stem cells. Keirstead, however, has not taken that research into a clinical trial for human beings.

NeoStem's interest in the Irvine firm did not appear to involve a possible spinal therapy.

While the company's stock price jumped as a result of the acquisition, one view of the future of the NeoStem was not optimistic. The Street Web site is recommending a “sell” on the firm's stock. It said yesterday. The Street said,
“This is driven by a few notable weaknesses, which we believe should have a greater impact than any strengths, and could make it more difficult for investors to achieve positive results compared to most of the stocks we cover. The company's weaknesses can be seen in multiple areas, such as its disappointing return on equity, weak operating cash flow and poor profit margins.”

UC Davis stem cell scientist and blogger Paul Knoepfler, however, called the deal good news for the stem cell field.

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Monday, April 14, 2014

Strangling Science: Antiquated System Wastes Billions, Costs Lives

The scientific community in California and the nation is fraught with worry about declining financial support for academic research. At the same time, however, as scientists wring their hands about the lack of funding, they are going along with the waste of more than $10 billion that could be used to help relieve the cash shortage.

What this is all about is the “stranglehold” that scientific journals have on publishing the results of the research paid for almost entirely by taxpayers. That research is the foundation of the journal industry, which is both highly profitable and a powerful lobby in Washington as it seeks to maintain its franchise. The industry is also based on a 400-hundred-year-old system that not only wastes money but costs lives because of its tediously slow mechanisms.

“Tragically insane” and unnecessary is how respected UC Berkeley researcher Michael Eisen describes the whole business, which is nearly invisible to the general public. That is, until one of its members starts to seek publicly financed information that is buried behind expensive paywalls.
Michael Eisen
UCB Alumni Association photo

Scientists are the unpaid workers for journals, submitting their articles for what they hope will be professional prestige and advancement. They do so, Eisen said, “while they acknowledge that their business practices are bad for science and the world.”

Last year, Eisen spoke to the Commonwealth Club in San Francisco about the subject. His speech was carried in a somewhat truncated form in this past winter's issue of the UC Berkeley alumni magazine. One of the headlines on the article asked,
“In the age of the Internet, why is so much research inaccessible?”

What Eisen had to say is worthy of careful thought as the $3 billion California stem cell agency faces financial extinction and the NIH sees its budget under increasing pressure. It seems a dubious proposition for scientists to give away the fruits of their labor and then have to pay for critically necessary access. Here is how Eisen described the situation along with additional excerpts from his speech.
“Every year universities, governments and other organizations spend in excess of $10 billion dollars to buy back access to papers their researchers gave to journals for free, while most teachers, students, health care providers and members of the public are left out in the cold. 
“Even worse, the stranglehold existing journals have on academic publishing has stifled efforts to improve the ways scholars communicate with each other and the public. In an era when anyone can share anything with the entire world at the click of a button, the fact that it takes a typical paper nine months to be published should be a scandal. These delays matter – they slow down progress and in many cases literally cost lives.”

Eisen continued,
“Tonight, I will describe how we got to this ridiculous place. How twenty years of avarice from publishers, conservatism from researchers, fecklessness from universities and funders, and a basic lack of common sense from everyone has made the research community and public miss the manifest opportunities created by the Internet to transform how scholars communicate their ideas and discoveries.”

Eisen decscribed the role of the scientific publications.
“I want you to note just how little the journal actually does here. 
“They didn’t come up with the idea. They didn’t provide the grant. They didn’t do the research. They didn’t write the paper. They didn’t review it. All they did was provide the infrastructure for peer review, oversee the process, and prepare the paper for publication. This is a tangible, albeit minor, contribution, that pales in comparison to the labors of the scientists involved and the support from the funders and sponsors of the research. 
“And yet, for this modest at best role in producing the finished work, publishers are rewarded with ownership of – in the form of copyright – and complete control over the finished, published work, which they turn around and lease back to the same institutions and agencies that sponsored the research in the first place. Thus not only has the scientific community provided all the meaningful intellectual effort and labor to the endeavor, they’re also fully funding the process. 
“Universities are, in essence, giving an incredibly valuable product  – the end result of an investment of more than a hundred billion dollars of public funds every year – to publishers for free, and then they are paying them an additional ten billion dollars a year to lock these papers away where almost nobody can access them.
“It would be funny if it weren’t so tragically insane.”

How does it affect the public, particularly persons with serious diseases? Eisen answered,
“This is most obviously a problem for people facing important medical decisions who have no access to the most up-to-date research on their conditions – research their tax dollars paid for. In a world where patients are increasingly involved in health care decisions, and where all sorts of sketchy medical information is available online, it is criminal that they do not have access to high quality research on whatever ails them and potential ways to treat it. 
“Astonishingly, many physicians and health care providers also lack access to basic medical research. Journal subscriptions in medicine are very expensive, and most doctors have access to only a handful of journals in their specialty. 
“But this lack of access is not just important in the doctor’s office. Scores of talented scientists across the world are blind to the latest advances that could affect their research. And in this country students and teachers at high schools and small colleges are denied access to the latest work in the fields they are studying – driving them to learn from textbooks or Wikipedia rather than the primary research literature. Technology startups often can not afford to access to the basic research they are trying to translate into useful products.”

Supporters of the journal industry argue that it is necessary because it provides for peer review of research results, thus ensuring the integrity of the science. Eisen said, however, that current peer review “poisons science.”
“Peer review is the closest thing science has to a religious doctrine. Scientists believe that peer review is essential to maintaining the integrity of the scientific literature, that it is the only way to filter through millions of papers to identify those one should read, and that we need peer reviewed journals to evaluate the contribution of individual scientists for hiring, funding and promotion.
“Attempts to upend, reform or even tinker with peer review are regarded as apostasies. But the truth is that peer review as practiced in the 21st century poisons science. It is conservative, cumbersome, capricious and intrusive. It encourages group think, slows down the communication of new ideas and discoveries, and has ceded undue power to a handful of journals who stand as gatekeepers to success in the field.
“Each round of reviews takes a month or more, and it is rare for papers to be accepted without demanding additional experiments, analyses and rewrites, which take months or sometimes years to accomplish.
“And this time matters. The scientific enterprise is all about building on the results of others – but this can’t be done if the results of others are languishing in peer review. There can be little doubt that this delay slows down scientific progress and often costs lives.”

Eisen continued,
“So, while it is a nice idea to imagine peer review as defender of scientific integrity – it isn’t. Flaws in a paper are far more often uncovered after the paper is published than in peer review. And yet, because we have a system that places so much emphasis on where a paper is published, we have no effective way to annotate previously published papers that turn out to be wrong.”

Flawed stem cell research has made international headlines in recent months. They came in the case of a peer-reviewed paper published in the world's most prestigious journal, Nature. Hundreds, if not thousands, of other examples exist that are chronicled on a Web site called Retraction Watch.
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Wednesday, April 09, 2014

Rao Joins New York Stem Cell Foundation

The New York Stem Cell Foundation announced today that Mahendra Rao, former head of the NIH Center for Regenerative Medicine, has been hired as its vice president for regenerative medicine, a new position at the organization.

Rao left the NIH March 28, apparently unhappy with its lack of funding to advance stem cell clinical trials. At one point, Rao was also being discussed as a potential successor to Alan Trounson as head of the California stem cell agency.

The agency says it is on schedule to name a new president late next month.
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California Stem Cell CEO Search: Rao Out, Closed-Door Meeting Next Week

Mahendra Rao
NIH photo
Speculation surfaced this week but was quickly squelched that Mahendra Rao, until recently the head of the federal Center for Regenerative Medicine, is a candidate to become the new president of the $3 billion California stem cell agency.

Rao's departure from the NIH's $52 million stem cell effort took many by surprise yesterday when it surfaced in an online story from Nature. The piece by Sara Reardon said that Rao left the NIH on March 28 and that the move has left NIH researchers “in the dark.”

She wrote,
“Relations seem to have soured last month owing to an NIH decision to award funding to only one project aiming to move iPS cells into a clinical trial. Rao says he resigned after this became clear. He says that he had hoped that five trials would be funded, especially because the centre had already sorted out complex issues relating to tissue sources, patents and informed consent.”

In the wake of the story, the California Stem Cell Report learned that Rao is not a current candidate for the California stem cell agency position. He is also not expected to take a fulltime research position in California. Rao did not respond to an email inquiry about the matter. (The New York Stem Cell Foundation announced later that it has hired Rao as vice president for regenerative medicine.)

Rao's departure from the NIH was reported as early as March 3 in a little-noticed press release from Cesca Therapeutics of Rancho Cordova, Ca.. The Sacramento-area firm was formerly known as Thermogenesis. The company said that Rao was joining its board of directors April 1 following his “planned departure” in March from the NIH.

Yesterday, Stemedica Cell Technologies of San Diego announced that Rao was joining its scientific advisory board. Stemedica was mentioned in a New York Times story last September involving international medical tourism. The company's Web site says it “is currently supporting clinical trials in Kazakhstan and Mexico and anticipates supporting similar initiatives in several other countries in 2013.”

Meanwhile, the agency is still on schedule to hire a new CEO by the end of May, according to Kevin McCormack, senior director of public communications. The current president, Alan Trounson, announced last fall that he was leaving to return to his family in Australia.

The agency's presidential search committee has scheduled a two-day, closed-door meeting for next Tuesday and Wednesday to screen candidates. According to the agency's timetable, the teleconference session is intended to produce the finalists for the job, who will undergo additional interviews either late this month or early next month. A vote by the agency's 29-member governing board is expected at its meeting in San Diego May 29.

Next week's meeting does provide for comment from members of the public, who can attend the public portion of the meeting. The main location is in Los Angeles, The address can be found on the meeting agenda.
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Monday, March 31, 2014

California's $40 Million Stem Cell Genomics Award Still Hanging Fire

The California stem cell agency's $40 million award to a Stanford-Salk group for stem cell genomics research is not yet a done deal.

The award was approved more than two months ago by the agency's board. However, the proposal is now caught up in a staff review of the nitty-gritty of its details.

 Agency employees, as a routine matter, go through all board-approved applications to be sure all matters are in order, ranging from commitments on matching funds to overall considerations concerning the recipient's research budget.

Sometimes changes are made as the result of the staff review. In rare cases, the recipient declines the award. (See here and here for two stories about recipient rejection of the California agency's cash.)

The California Stem Cell Report earlier today inquired about the status of the stem cell genomics award.

Kevin McCormack, senior director of public communications for the agency, said it had not cleared the staff review. Asked whether there were particular issues, McCormack replied,
“No. Some things just take time.”


In the wake of the award, which was approved on a 6-1 vote by the 29-member board, the agency initiated an examination of the grant review process in the genomics round.

Board member voting on the award was limited because of widespread conflicts of interest among the agency's governing board.
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Monday, March 24, 2014

The Long Odds Story of Stem Cell Research

It was the sort of story that Californians did not see during the ballot campaign 10 years ago that created the state's $3 billion stem cell agency.

The story line then was of hope and the imminent prospect of cures for a host of diseases that afflicted half of California's population.

Today in the San Francisco Chronicle, Stephanie Lee wrote of biotech companies that go for more than two decades without ever developing a commercial product. Lee's poster child was Geron Corp., which abandoned stem cell research in 2011 along with a $25 million loan from the stem cell agency.

Lee wrote,
“A company that goes 24 years without ever selling a product may sound unusual. But in biotechnology, it's not that uncommon. 
“Take Geron Corp. in Menlo Park, which has struggled to develop a therapy - any therapy - since its founding in 1990.”
Lee noted that Geron has an accumulated deficit of $893 million in debt since its founding and is now facing a number of lawsuits from unhappy investors.
“Faced with 10- to 15-year timelines and uncertain regulatory outcomes, companies and investors might plow hundreds of millions of dollars into therapies that will never see the light of day.”
Besides Geron, Lee also mentioned Isis Pharmaceuticals and Dendreon Corp. as examples of biotech firms with a lengthy development history.

She wrote,
“Overall, the odds of getting a drug to market aren't much better than gambling in Las Vegas. Only 1 in 5,000 to 10,000 compounds discovered in the lab gains FDA approval.”
Those odds, we should note, do not reflect development of stem cell therapies, but rather simpler substances. The expectation is that odds will go higher for stem cell therapies.

All of which is not the best news for the stem cell agency, although the information is not all that new. It was out there during the 2004 stem cell campaign, but the stem cell community did not want to talk about it, and the mainstream media largely did not report it.

The figures remained unchanged today, although a more positive aura surrounds biotech than has existed for several years. Nonetheless, the story of long odds and fruitless research is one that bedevils the stem cell agency. It will run out of funds for new awards in 2017 and is looking for ways to avoid its financial demise. Creating a new narrative, one with a brighter future, is the agency's task as it tries to develop new funding.
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