Media Series

Media Series

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Tuesday, 26 February 2019

Shilling for Stem Cell Treatments? A Look at the Journal Nature and an Advertising Matter

February 26, 2019 0
Shilling for Stem Cell Treatments? A Look at the Journal Nature and an Advertising Matter
For UC Davis scientist, Paul Knoepfler it was something "very strange." For Pulitzer Prize-winning Los Angeles Times columnist Michael Hiltzik, it had the earmarks of a "race to the bottom."

And it all involved a stem cell matter published by the prestigious journal Nature.

The headline on Hiltzik's column yesterday summarized the case like this. 
"Did a world-famous science journal become a shill for a questionable stem cell claim?"
Hiltzik wrote,
"Readers of Nature, one of the world�s most important scientific journals, might have been struck recently by an audacious claim appearing on its website about a possible stem cell treatment for heart attacks.

"The published item asserted that MUSE cells, a subset of stem cells, could regenerate heart tissue after acute myocardial infarctions, which are deadly sudden heart attacks. This could be a significant advance in both cardiac treatment and the use of stem cells.

"Here�s the problem. The published item wasn�t a peer-reviewed article subject to Nature�s rigorous professional vetting procedure. It was an advertisement placed by the Translational Research Center for Medical Innovation, the Japanese research lab that says it performed the reported study on MUSE cells using white rabbits.

"It looked like a Nature article, however, at least to Paul Knoepfler, a stem cell expert at UC Davis who has become one of our most assiduous debunkers of stem-cell quackery. Knoepfler thought the layout of the item might cause some readers to mistake it for a peer-reviewed paper, and promptly queried Nature. The journal responded by taking the advertisement offline Feb. 22. Knoepfler�s brief chronicle of the affair can be found on his website here."
Hiltzik concluded:
"The Food and Drug Administration has its hands full monitoring these claims and treatments. The agency has issued multiple warnings to steer the public away from such clinics and has taken administrative and legal action against some of them. The signs point to more exploitation and more danger to the public health. Important journals such as Nature shouldn�t be participating in a race to the bottom."
For the record, Knoepfler has been critical in the past about some of Hiltzik's commentary, including the columnist's views about the state stem cell agency.  

Monday, 25 February 2019

'Getting Stanford's Attention:' The Transcript of a $1.7 Million Matter

February 25, 2019 0
'Getting Stanford's Attention:' The Transcript of a $1.7 Million Matter
The California stem cell agency has posted online the 24-page transcript from last week's meeting during which the agency's governing board raised sharp questions about Stanford University and its obligation to provide co-funding on a $19 million award to one of its scientists.

The board voted unanimously last Thursday to condition additional millions for the research on receiving by May 1 the co-funding, which totals $1.7 million.

Just before casting his vote, Art Torres, vice chairman of the board and a former longtime state lawmaker, said, 
"I do think that we have put forward this motion in order to get Stanford's attention as to what their commitment has been in the past, and what it will continue to be given the nature of the arrangement that we made with them."
Stanford researcher Judith Shizuru sought $6 million for continuation of a phase one clinical trial to develop a potentially "transformative" product that would eliminate the toxic impact of chemotherapy for a number of diseases. 

The $3 billion agency reduced the amount that would be given to Shizuru to $3.75 million along with imposing the deadline for co-funding. 

The agency expects to run out of money for new awards by the end of this year. 

Thursday, 21 February 2019


February 21, 2019 0

California's Stem Cell Agency Smacks Stanford for Failing to Deliver on Financial Promises

February 21, 2019 0
California's Stem Cell Agency Smacks Stanford for Failing to Deliver on Financial Promises
The California stem cell agency today delivered an unusual and sharp rebuke to Stanford University, declaring that it needs to stand by its financial commitments to help back state-funded research. 

The message came during consideration of a request for more research cash from a Stanford researcher, Judith Shizuru. She sought $6 million for continuation of a clinical trial to develop a potentially "transformative" product that would eliminate the toxic impact of chemotherapy for a number of diseases. 

"It boggles my mind," said Jeff Sheehy, a member of the agency's governing board and chairman of its Science Committee, that Stanford, which has an endowment of $27 billion, has not stepped forward to provide the co-funding. 

The amount involved is $1.7 million and is due May 1. It is connected to an earlier $19 million award to Shizuru that kicked off her clinical trial. 

Sheehy noted that Stanford and its researchers have received $379 million from the agency since 2005. It is the top recipient of funds from the California Institute for Regenerative Medicine (CIRM), as the agency is formally known. Stanford also has a member on CIRM's board. 

Steve Juelsgaard, chairman of the board's Finance Committee and a former top executive at Genentech, said that Stanford is "very well taken care of in terms of its economics." 

He said that the agency requires all institutions and businesses to sign agreements to deliver on promises of co-funding. CIRM cannot have two standards for its grantees, Juelsgaard said. 

Today was the first time that the $3 billion research agency has publicly rebuked a grantee on co-funding. Recipients of awards, be they institutions or individual researchers, are generally  treated tenderly in public.  (The full transcript of the meeting is available here.)

CIRM directors did not take issue with the quality of the research, which they described as good. Specifically, today's application sought to advance Shizuru's phase one clinical trial to develop a way to avoid the necessity of chemotherapy in a genetic affliction, popularly known as the bubble baby syndrome.

Nine researchers in the field sent letters to the CIRM board praising the work. Several called it "transformative" and said it could have use in afflictions ranging from blood cancer to diabetes. 

Shizuru applied for $6 million. However, the CIRM board reduced the award to $3.7 million and only on the condition that the previous co-funding be delivered.  

Shizuru told the CIRM board she accepted responsibility for raising the co-funding, which she said has been difficult. Board members noted that agreements on awards are signed by both the recipient researcher and his/her insitution. She said she would discuss today's action with Stanford officials.

Her application came before directors in January with a seal of approval from the agency's reviewers, who make their decisions behind closed doors. Normally, approval by reviewers means a rubber stamp by directors.

Action in January, however, was deferred until this month after directors raised questions about research delays and financial matters. The agency also publicly released a slough of information concerning Shizuru's work, most of which she provided to them.  

Here are links to key documents involving Shizuru application. 
(An early version of this item misspelled Shizuru's last name.)

Tuesday, 19 February 2019

A Peek Inside the California Stem Cell Research Machine: $25 Million, Babies, Bonds and Dwindling Cash

February 19, 2019 0
A Peek Inside the California Stem Cell Research Machine: $25 Million, Babies, Bonds and Dwindling Cash
California's stem cell agency this week dished up rare public details of an advanced effort to create "transformative" therapies that would help cure afflictions ranging from diabetes to always fatal immune disorders.

The disclosure involves Stanford University researcher Judith Shizuru, severely ill babies, toxic chemical treatments, delays in clinical trials and stem cell agency board members who are acutely aware of the $3 billion agency's rapidly dwindling resources.

Judith Shizuru
Photo by Flynn Larsen, Ludwig Institute
All this plus more is on the table Thursday at a meeting of the directors of the California Institute for Regenerative Medicine (CIRM), as the agency is formally known. 

The agenda nominally contains one item, an application by Shizuru for $6 million from CIRM. But the issues reach back to 2013 and provide insight into difficult research pathways and how the agency manages its programs. 

At the top of the matter is the fact that the state agency expects to run out of cash for new awards by the end of this year. It is pinning its hopes on a proposed $5 billion bond measure on the November 2020 ballot.  Meanwhile, directors are trying to raise privately $200 million to tide it over until then. 

Avoiding Toxic Chemotherapy

That was the backdrop Wednesday Jan. 30 when directors convened to consider application number CLIN2-11431 by Shizuru for $6 million. She is seeking a way to avoid chemotherapy treatments and their toxic side effects in the case of a rare genetic affliction often referred to as the bubble baby syndrome. 

In December, CIRM's grant reviewers, meeting behind closed doors, approved Shizuru's application, an action that nearly invariably is rubber-stamped by directors. But last month was different, and a bit of CIRM history was brought up.

Shizuru received  a $19 million CIRM award in 2012 which has led to a phase one clinical trial with positive results for her therapy. Cost per each of the six patients so far averages $917,000, according to a CIRM document. 

However, additional patients are needed, along with more funding, before the potential product can reach the marketplace. The average cost of per patient will run about $333,333 during the final portion of the phase one trial.  Her latest application is aimed providing assistance with those costs. 

Questions about Delays and Co-Funding

Last month, questions arose among directors about months of delays in the clinical trial and a current shortfall in co-funding, among other things.

Steve Juelsgaard
Steve Juelsgaard, a former Genentech executive and chair of the directors' Finance Committee, said, 
"It's not clear to me that they're being frugal with the money that they have been given."
Juelsgaard also said,
"Delay doesn't necessarily add up to more money. They obviously spent the money on something that they didn't anticipate or under-budgeted or something. There's something more to it."
Another director, Jeff Sheehy, chair of the board's Science Committee, said,
"Financially, it seems very muddy to me."
Other directors weighed in as well, ultimately leading to a motion to delay action on the application to provide more time to find answers to questions.

At that point, Shizuru, who was in the audience, rose to respond.

"I understand CIRM's concerns, and I can see you're very thoughtful about how this money is being spent," she said, according to the transcript.

 The Sheehy-Shizuru Exchange

The Stanford researcher said that without additional funding the trial would have to suspend enrollment of additional patients, prompting this exchange between her and Sheehy.

"Shiruzu: Budgetarily we're better off using it (the remaining funding) to continue to follow the patients that we've already transplanted. From that budgetary standpoint, we should delay the trial. We should delay treating any more patients on the trial.

"Sheehy: So Stanford won't front you 1.6 million to (treat) patients if we don't give you the money today?

"Shiziru: I hesitate to say what they would do.

"Sheehy: To Stanford...they would actually put patients at risk?

"Shiziru: I'm not at liberty to say what Stanford would do."

Stanford is the No. 1 recipient of CIRM awards, chalking up $379 million over the past 14 years. It also has always had a member on the CIRM board of directors, who is not allowed to speak or vote on awards to Stanford.

Following Shizuru's comments, CIRM directors approved the motion to delay action until this week's meeting. Since the January session, Shizuru has provided the agency with more than five, single-spaced pages of explanation about her research, plus other material that has been kept under wraps for what CIRM has indicated are proprietary and legal reasons.

The information from Shizuru, as well as additional documents from CIRM, provided an unusual, public look into the agency's grant-making process as well as the hurdles encountered in advanced clinical research. In the past, reviews of applications approved by reviewers have received little or no discussion.

Shizuru's Perspective

In her material, which is available on the agenda for this week's meeting, Shiruzu said one, eight-month delay was caused by CIRM itself because of internal concerns. Another nine-month delay was caused by age-restrictions on patients.

"Significant delay" was also caused because of what might be called a supply and demand issue. The bubble baby syndrome is rare. Only one in out of 50,000 to 100,000 births results in a baby with the affliction: severe combined immunodeficiency.
Three other clinical trials are also competing for those rare patients. 

Nine letters of support were received by CIRM supporting the research, with some describing the potential result as "transformative." The letters also said Shizuru's approach could find use in a wide range of other afflictions, an expectation also agreed upon by CIRM. 

Shizuru said she is working on securing the needed co-funding for her first award, which is not yet concluded. As for commercialization, she said she and her colleagues have formed a company, whose name was not disclosed. She said she has a letter of support from one investor regarding "an intention to co-fund pending completion of due diligence."

In a final comment during the vote last month to delay consideration, one CIRM board member, a San Diego patient advocate for Parkinson's disease, praised the board's close examination of the application. David Higgins said, 

"I want to acknowledge fellow board members (for) their continued concern about spending taxpayer's money wisely because I think this is a great example of that."

(The public can listen to and participate in the Thursday meeting via the Internet. Instructions are on the meeting agenda.) 

Friday, 15 February 2019

California Stem Cell Opposition: Conservative Writer Declares Golden State Efforts a 'Bust'

February 15, 2019 0
California Stem Cell Opposition: Conservative Writer Declares Golden State Efforts a 'Bust'
In a preview of what is likely to be a heated ballot campaign next year,  a conservative writer declared this week that California's efforts to develop stem cell therapies are "a scientific and financial bust." 
"Back in 2004, the $3 billion California Stem Cell Research and Cures Initiative, Proposition 71, promised life-saving cures and therapies for Alzheimer�s, Parkinson�s and other diseases. The cures and therapies, in turn, would send money flowing into state coffers, so the project, in effect, would pay for itself. It didn�t exactly work out that way," said Lloyd Billingsley on two different web sites.  
"CIRM proved itself a scientific and financial bust, and almost completely off limits to state oversight."
Billingsley has written in the past about the agency, known as CIRM and formally as the California Institute for Regenerative Medicine. His latest columns appeared on the California Globe, which was founded by Ken Kurson, who has ties to the Trump family and Rudy Guiliani, and The Beacon.  

Billingsley likened the agency to the troubled bullet train project in California and efforts to solve some of California's water problems by building a tunnel under the California delta east of San Francisco. 

CIRM expects to run out of cash this year for new awards and hopes to survive with voter approval of a proposed, $5 billion bond measure on the November 2020 ballot.

It could be a hard-fought campaign, but conservatives and other likely opponents could well be diverted if President Trump is on the ballot. 

See here and here for more on Kurson, founder of the  California Globe, and here for the advisors to the Beacon web site and its parent organization.

Wednesday, 13 February 2019

Position of the California Stem Cell Agency on Gene-Edited Babies? No Go.

February 13, 2019 0
Position of the California Stem Cell Agency on Gene-Edited Babies? No Go.
California's $3 billion stem cell agency may be on the leading edge of regenerative medical research, but it is clearly opposed to the type of work that has led to the international flap over the gene-edited babies in China

Its regulations have long barred that sort of experimentation. In 2016, the agency, known formally as the California Institute for Regenerative Medicine (CIRM), convened an international conference to discuss the issues involved.

The session in Los Angeles went on for hours, generating a 223-page transcript touching on the difficulty of regulating gene editing, among a host of other difficult issues. 

One patient advocate in the audience, Adrienne Bell Cors Shapiro, noted that it is nearly impossible to control all that might happen. She said,  
"People are messy. And if you develop this technology, somebody is going to find a way to use it."
Last week the MIT Technology Review published an article about a Stanford University investigation into what two of its researchers knew about the Chinese research. The scientists also have received CIRM funds for research unrelated to the Chinese work. 

Asked for a comment, a spokeswoman for the state stem cell agency said in an email:
"CIRM�s regulations prohibit nuclear genome editing for reproductive purposes. In February 2016, CIRM convened the Scientific and Medical Accountability Standards Working Group (SWG) for a workshop on Human Gene Editing. The SWG subsequently recommended that no changes be made to CIRM�s existing prohibition on nuclear genome editing for reproductive purposes."
In 2016, Hank Greely, a law professor at Stanford who deals with bioethical issues, told the gene editing conference:
"CIRM is in the human embryo experimentation world. It funds research as long as the embryos are not implanted. It funds it with special protections and special review considerations and special informed consent considerations. I don't think CRISPR-cas9 changes that."
The controversial research in China triggered a global uproar in the scientific community. The leading stem cell research organization, the International Society for Stem Cell Research, issued a statement opposing such experimentation

He Jiankui, the Chinese scientist who performed the experiment, has lost his job in that country, according to news reports, and may be facing criminal charges. His work has not been confirmed by an independent review.