Ten Days in March: Is the $3 Billion California Stem Cell Agency Measuring Up?

Highlights

'Success metrics'

Next week's board meeting

Discouraging public attendance

But amply open in many ways

One of the more admirable aspects of the California stem cell agency’s plan for spending its last $900 million is its attention to careful measurement of the agency’s own performance.

The “success metrics” are tucked away at the end of the agency’s new, 47-page strategic plan. They lay out the criteria for determining how well each team at the agency is doing. The metrics range from the number of conflict of interest appeals to the number of clinical trials completed at Alpha clinics. Based on decades of experience with state agencies, the California Stem Cell Report can attest that such attention to performance is rare among state departments.

The agency's measurements also include the “number of board meeting documents posted with ten-day lead time.” Which is where the March 16 meeting of the governing board of the stem cell agency comes in.

On Saturday, the agency posted on its Web site the agenda for the meeting, a good 11 days ahead of the session in Millbrae, near San Francisco International Airport. The agency plans to give away millions of dollars, perhaps tens of millions. It will review the status of its ambitious and risky clinical trial program, which has seen a sharp upsurge in the last year or two. At least that is what can be deduced from the cryptic agenda items, the longest of which consists of only 26 words.

Total missing, as of this writing, are any documents supplying the details essential for understanding what the agency is actually doing.

The board meetings are the single most important public events of the agency. The agency professes to want to see greater public turnout at the sessions. But without adequate notice and some sort of meaningful information, the public, stem cell firms, scientists and patient advocates cannot make plans to attend the meetings even if they might have a deep impact on their lives.

On Feb. 9, Kevin McCormack wrote on the agency's blog about a CIRM meeting dealing with gene editing. He said,

“(Bioethicist) Alta Charo said this is not just a question for scientists, but something that could potentially affect everyone and so there is a real need to engage as many groups as possible in discussing it

"‘How and to what extent do you involve patient advocates, members of the disability rights community and social justice community – racial or economic or geographic.  This is why we need these broader conversations, so we include all perspectives as we attempt to draw up guidelines and rules and regulations.’”

Those sentiments pretty much apply to all that the agency does. It is spending $6 billion of California taxpayers’ money, including interest, on research that is yet to produce a commercial therapy, despite the facile promises of the ballot campaign that created it in 2004. It is financing experiments in areas that are new to medicine, some of which involve serious risk and ethical considerations.

All of which might seem to be issues that would engage many persons and enterprises. But the best way to discourage involvement by those not embedded in the agency is to withhold information until it is too late to respond. If interested parties don’t know what is going on, how can they offer suggestions and criticism, some of which might be useful and help the agency succeed in its endeavors? Not to mention helping to build support for continued funding beyond 2020, when the agency expects to run out of cash.

Additionally, the agency basically hides the schedule of its public meetings on its Web site. They are virtually invisible on the agency’s home page, buried at the bottom in teeny lettering under a heading of “about CIRM” -- one of 11 subjects such as “mission” and “history.”

In many ways, CIRM is more than amply open and transparent. Its board meetings are audiocast on the Internet and by phone. Its committee sessions are now available live online as well, an improvement that began during the last few months. Transcripts of the sessions are also available online, albeit sometimes months after the sessions.

The CIRM Web site is laden with a variety of material. The agency’s excellent blog, The Stem Cellar, produces daily reports that chronicle CIRM affairs as well as research developments elsewhere.  Interested persons can sign up for email alerts on various CIRM matters. Eventually, most of background information on board meeting agendas is posted online, although sometimes only a day or two ahead of the session.

Nonetheless, CIRM can and should do a better job of telling California what its governing board is up to and what the agency is thinking and doing. One key and long neglected tool is to provide more detailed information -- well in advance -- about the issues that come before the 29-member board that hands out the cash and establishes the priorities for one of the most ambitious stem cell programs in the world.

Champion of Stem Cell Research: Nancy Reagan Hailed by California Stem Cell Leader

The chairman of California’s $3 billion stem cell research effort yesterday weighed in with a tribute to Nancy Reagan and her support for human embryonic stem cell research.

Jonathan Thomas said,

“With the passing of former first lady Nancy Reagan the California Institute for Regenerative Medicine has lost a good friend and a champion of stem cell research. Mrs. Reagan was an advocate for stem cell research for many years and her voice was an important one in helping ensure the passage of Proposition 71. Her call to protect the ability of scientists to ‘pursue medical miracle possibilities’ echoed the feelings of millions of Americans looking to stem cell research for help in battling deadly diseases. We, and patients everywhere, were fortunate to have such a great champion for the work that we do.”

Nancy Reagan’s backing of hESC research began in the spring of 2004 with an unsuccessful effort to convince former President Bush to rescind his restrictions on federal funding of hESC research.

An Associated Press story said at the time,

"'It's hard to overstate or overestimate the power of her impassioned plea for the Bush administration to reform its stem cell policy,' said Daniel Perry, president of the Coalition for the Advancement of Medical Research. "She's given permission for very conservative, anti-abortion Republicans to disagree with Bush. It's a courageous stance against a president of her own party."

Newsweek magazine also carried a piece about her efforts.

Royalties and California Stem Cell Research: $1.1 Billion Promise Still to be Fulfilled

California’s 11-year-old stem cell research effort was born with the extravagant promise that it would generate something like  $1.1 billion in royalties that would flow into the Golden State’s coffers.

None of that bonanza has yet surfaced, but there is no doubt that scientific research generally has the potential to generate economic value for state entities. A case in point was news this morning in the Los Angeles Times that the sale of drug rights (intellectual property or IP) held by UCLA will generate “hundreds of millions of dollars.”

The story by Teresa Watanabe said the deal involved a prostate cancer drug developed at the campus. She reported,

“Royal Pharma, a New York-based pharmaceutical company, paid $1.14 billion for royalty rights to the drug known as Xtandi. It was the largest-ever technology transfer deal involving a University of California invention.”

 California’s $3 billion stem cell agency is not involved in the UCLA-Royal Pharma arrangement. However, the agency, known formally as the California Institute for Regenerative Medicine (CIRM), has acquired other bits and pieces of IP as the result of the $1.9 billion that it has given to researchers over the last decade.

So far the agency has not been able to turn that IP into cash largely because no therapies have reached the marketplace, although the agency has a number of clinical trials underway.

Back in 2005, the royalty promises made during the ballot campaign that created the stem cell agency came under fire as the result of actions by the man who headed the campaign, Robert Klein. He also served as the agency’s first chairman.

Bernadette Tansey, writing in the San Francisco Chronicle, reported,

“The billion dollars in royalties that voters were told could flow to the state if they passed California's $3 billion stem cell research funding initiative in 2004 may turn into an empty promise.”

Stuart Leavenworth, then editor of The Sacramento Bee’s editorial pages, wrote,

“In marketing this initiative, proponents said the state would receive not only miracle cures and reduced medical costs, but also up to $1.1 billion in royalties from new stem cell innovations.

“Now we are learning that this promise, at best, was misleading. At worst, it was a cynical ruse.”

The issue turned on whether tax-exempt bonds would be used to finance the agency, which depends solely on state borrowing. Federal tax laws restrict the use of such bonds. To date, however, no tax-exempt bonds have been used to support CIRM, according to last report.

The use of taxable bonds, however, raises the cost of borrowing and the cost of the research. Estimates in 2004 were that that the total cost of the agency would exceed $6 billion, including the interest on the borrowed $3 billion. No revised, upward estimate has been made public, if it exists.

The issue of the agency’s IP, its possible value and protection has surfaced only intermittently over the years. The most recent mention came last November in the agency’s plan for spending its last $900 million. (The agency expects to run out of cash for new awards in less than four years.)

The agency’s five-year plan said that it would “demand creation (from universities)  for the out-licensing of CIRM-funded technologies with a greater opportunity to achieve a financial return.” Aligned with that is a $75 million proposal to partner with specific enterprises and give them pick of the agency’s best research that does not currently have a partner. (See here and here.)

Relationships with universities can be touchy at the agency because its governing board includes top executives from virtually all of the major, academic stem cell research centers in California. Sometimes those board members can be protective of the interest of their institutions, which have received hundreds of millions of dollars in agency cash over the years.

For those who want to dig more deeply into CIRM’s IP policy, the regulations can be found here and here.

Scientists to Executive Assistants: California Stem Cell Agency Seeking to Fill Four Positions

Looking for stem cell work in warm and dry California?

The state’s $3 billion stem research effort is seeking four persons to fill slots as an executive assistant to the vice president of therapeutics, project manager for medical affairs and centers and two science officer positions, one on the discovery team and another in therapeutics.

The job descriptions and requirements, along with salary ranges, can be found on this Web page.

The agency is headquartered in new offices in downtown Oakland, overlooking Lake Merritt, close to public transportation. The offices are also only a 30-minute walk from the waterfront saloon where author Jack London misspent a portion of his youth.

California's $30 Million Bet On a Cancer Treatment: New Company Formed to Bring the Therapy to Market

Highlights

Stanford, Weissman involved

Good news for CIRM

Google hooked in

A new stem cell company that targets cancer by unleashing an “eat me” trigger has emerged from a $30 million investment by the state of California.

Creation of the Palo Alto firm, which is called Forty Seven, Inc., was announced this week by its backers and its key researcher, Irv Weissman, director of Stanford University’s stem cell program.

Venture capitalists, including Google, are supporting the enterprise with $75 million. The effort

has two phase one clinical trials underway, the first step in a years-long process of testing potential therapies before the federal government approves their widespread use.

Forty Seven, named after the molecular target of the “eat-me” trigger, is not the first firm to emerge from research funded by the California Institute for Regenerative Medicine (CIRM), as the state stem cell agency is formally known. At least eight others have surfaced over the years.

But this week’s announcement attracted some national attention in a story by Alex Lash on Xconomy, an online technology information service. Lash wrote,

“This is the kind of news CIRM has yearned for in recent years: significant private cash that gives CIRM-funded projects a push to become actual products and to return value to the state…. After a decade of funding buildings, salaries, and early stem-cell related research—not to mention conflict of interest problems that included a past CIRM president and a different company (StemCells, Inc.) with Weissman’s imprimatur—CIRM and its new president acknowledged it needed to show results for the $6 billion taxpayers committed to the agency in 2004.

“This type of follow-on funding is what we’re looking for,” says CIRM spokesman Don Gibbons, who adds that the big financial awards for Stanford were in line with the agency’s mission “to get things moving early when others won’t invest.”

(See here, here and here for more on the conflicts of interest.)

Voters approved the creation of the $3 billion agency in 2004. But no therapies have emerged for widespread use despite the optimism of its early backers and campaign promises. The agency expects to run out of funds for new awards in 2020. One of the possibilities for new funding is another multi-billion dollar state bond issue. State bonds are the only significant source of cash for the Oakland-based agency.

Xconomy reported that CIRM has yet to provide its final $5.1 million to Weissman. And Lash wrote that “Forty Seven is in the process of taking charge of the grant-funded programs.” Asked this morning by the California Stem Cell Report whether the funds would be going directly to the firm, Kevin McCormack, senior director of communications, replied,

“Our grant goes to the principal investigator on the project, in this case it’s both Irv Weissman and his co-PI Ravi Majeti. Both are at Stanford.”

Weissman’s technique uses a monoclonal antibody that attaches to the CD47 protein on cancer cells them from hiding from the patient’s immune system. Lash wrote,

“But cancer cells also produce what Weissman and colleagues call an ‘eat me’ signal from a protein called calreticulin. Normal, healthy cells don’t. By cutting off the CD47 ‘don’t eat me’ signal, Hu5F9-G4 should in theory allow the calreticulin signal to prevail.”

Stanford, which has a member on the CIRM board of directors, is the No. 1 recipient of funds from CIRM. It has chalked up 98 awards totalling $308 million. Weissman has received five awards totalling $36 million. StemCells, Inc., which was co-founded by Weissman, has been awarded $9 million in for three projects.

Chinese Cloning Firm Pumps $15 Million into California Stem Cell Business

A Chinese firm that says it has the technology to clone human beings has invested $15 million in a stem cell company in Northern California.

The American firm is Cesca Therapeutics of Rancho Cordova, a suburb of Sacramento. The Chinese enterprise is BoyaLife Group, which has plans to clone one million cattle a year by 2020. In December, Rebecca Davis of Agence France-Press interviewed Xu Xiaochun, CEO of BoyaLife, and wrote:

“The Chinese scientist behind the world's biggest cloning factory has technology advanced enough to replicate humans, he told AFP, and is only holding off for fear of the public reaction.”

BoyaLife has a South Korean partner, Sooam, which was founded by Hwang Woo-Suk, the researcher who generated a global scandal in 2005 by falsly claiming to have cloned a human embryo. Sooam is involved currently in cloning pet dogs at $100,000 a pop, according to AFP.

Last year, Cesca lost a bid for $11 million from the California stem cell agency. The president of the firm, Robin C. Stracey, told this writer at the time that it expected to partner with another enterprise rather than try again for California stem cell agency funding.

The company’s application to the state research agency sought funds for a phase three clinical trial for a process to treat critical limb ischemia, which can lead to the loss of limbs.

Dale Kasler of The Sacramento Bee reported today that financially strapped Cesca could be taken over by BoyaLife. He reported that the company said this week that BoyaLife intended “to become a majority shareholder” in Cesca.

Kasler wrote,

“However, a change in control isn’t imminent, and Cesca officials said they welcome BoyaLife’s investment as a vote of confidence in their company.”

The New York Times reported last November that BoyaLife is planning the world’s largest animal cloning center in China this year. It quoted Xu as saying,

“And I can tell you all that cloned beef is the tastiest beef I have ever had.”

It was not clear, as of this writing, what role, if any, Cesca would have in cloning animals.

Cancer and Stem Cells: A Look at the Safety of iPS cells

The safety of reprogrammed adult stem cells was much in the news during the past few days as a result of work at the Scripps Research Institute that was funded by California’s $3 billion stem cell agency.

The press release from Scripps triggered a spate of stories quoting Jeanne Loring, who led the work. The Scripps release said,

“A new study led by scientists at The Scripps Research Institute (TSRI) and the J. Craig Venter Institute (JCVI) shows that the act of creating pluripotent stem cells for clinical use is unlikely to pass on cancer-causing mutations to patients.

“The research, published February 19, 2016, in the journal Nature Communications, is an important step in assessing patient safety in the rapidly developing field of stem cell therapies.”

The stem cell agency took a deep dive into the research today on its blog, The Stem Cellar, by Karen Ring, a former stem cell researcher who is the agency’s web site and social media manager. Ring wrote,

“It’s good news that reprogramming methods are relatively safe, but the fact that maintaining and expanding iPS cells in culture causes cancerous mutations is still a major issue that scientists need to address.

“Jeanne Loring recognizes this important issue and says that the next steps are to use similar genomic analyses to assess the safety of reprogrammed iPS cells before they are used in patients.”

Scripps’ press release reported that funds from five different stem cell agency grants were used in the research. Additional funds were also provided by a variety of sources.

Inside STAP: New Yorker's Long Look at the Flap and its Implications

Over the weekend, the New Yorker published online a bang-up and thorough account of the STAP stem cell scandal of 2014. which stretched across the Pacific from Japan to Boston.

The subhead on the story said,

“Rivalries, intrigue, and fraud in the world of stem-cell research”

The piece was authored by Dana Goodyear, a writer for the New Yorker who also teaches writing at the University of Southern California.

UC Davis stem cell researcher Paul Knoepfler, who carried on his blog early and lengthy pieces on the STAP flap, today said of the article:

“It’s a long, fascinating look inside of STAP, the tangled and ultimately tragic scientific implosion that created and then brought down two Nature papers and some careers.”

Goodyear’s article brought out much fresh material, including a more detailed look at the history of the STAP research than has been previously published. The piece also contained probably enough scientific detail to satisfy the experts in the field.

But Goodyear also included thoughts on the stem field in general, issues related to scientific journals, hyper-competitiveness among researchers, replication of research and more. Here are a couple of excerpts from the article, which we highly recommend:

“The promises of stem-cell research lie at the core of human desires—to understand our origins and to cheat death—and there is a great deal of money and prestige at stake. It is a ruthlessly competitive field, susceptible to fantasy and correspondingly sensitive to bunglers. Human embryonic stem cells were first cultured in 1998; nearly twenty years later, basic assumptions about cell behavior are still routinely overturned. Andrew McMahon, a top researcher at the Broad Center for Regenerative Medicine and Stem Cell Research, at the University of Southern California, told me, “It’s not unusual to see something and not be able to explain it.” In reporting results, researchers must often craft a narrative to make sense of mysterious phenomena. What to ignore and what to privilege—that discernment can be the difference between brilliance and quackery, and between fame and obscurity.”

On the difficulties in replicating research findings:

“Many people believe this is partly the fault of the scientific journals. Along with the influential role that Nature has in shaping the trajectories of ideas, technologies, and careers, it is essentially a commercial enterprise. The editors like big stories, and for the right ones they take risks. Some observers complain that incentives to publish have a distorting effect, causing scientists to oversell data; a cutthroat culture sometimes leads researchers to publish intentionally incomplete or vague protocols. The perceived conflict between good science and prestige has become so pointed that, two years ago, Randy Schekman, a Nobel Prize-winning biologist, announced in the Guardian that he would no longer publish in Nature, Cell, or Science, which, he wrote, ‘aggressively curate their brands, in ways more conducive to selling subscriptions than to stimulating the most important research.’”

Leukemia Treatment: California Stem Cell Agency Awards $3.8 Million to San Diego's Angiocrine Bioscience

CIRM graphic

The California stem cell agency today approved a $3.8 million award to a San Diego firm to help develop a better way to treat such afflictions as leukemia with cord  blood transplants despite concerns that the effort was too complex.

Shahin Rafii, Cornell photo

The funds will go to Angiocrine Bioscience, Inc., whose key technology is licensed from Weill Cornell Medical College and was invented and developed by Shahin Rafii, a professor of medicine at the New York school. He remains on the company's scientific advisory board.

Directors voted 13-0 to approve the award, but not before two directors raised questions. Joe Panetta, president of the San Diego-based industry group, Biocom, wondered about difficulties with the manufacturing process raised by the agency’s blue-ribbon reviewers, who earlier approved the application behind closed doors.

Steve Juelsgaard, former executive vice president of Genentech, also raised questions. If the effort flounders, he said, 

“We need to be able to put the brakes on.”

CIRM officials said that the research would be closely monitored with clear milestones that needed to be achieved before cash continued to flow to the company.

The agency’s press release said Angiocrine

“plans to develop a product called AB-110, which blends an expanded mix of stem cells from cord blood with genetically modified endothelial cells, the kind of cell that forms the lining of blood vessels, to improve the success rate of cord blood transplantation.

“The hope is that AB-110 will reduce the complications that can occur with a cord blood transplant – such as viral infections or pneumonia – and increase the likelihood the transplanted cells will successfully engraft, meaning they start growing and creating new, healthy, blood cells.”

No members of the public spoke at the teleconference meeting which had 12 public locations around the state. The next meeting of the agency’s directors will be March 16 near San Francisco International Airport and will be a face-to-face session with likely a handful of remote public locations.

Speak Up! A Chance to Sound Off to California Stem Cell Agency

If you would like to speak directly to the governing board of the $3 billion California stem cell agency, here’s your chance on Thursday.

The  directors are holding a teleconference meeting at 11 a.m. PST with public locations at 12 different sites around the state. The purpose of the meeting is to approve a $3.8 million application for work on process to manufacture a cord blood treatment for such afflictions as leukemia and lymphoma.

Approval is expected to be routine since the agency’s blue-ribbon reviewers, meeting behind closed doors, have already decided the application should be funded. The identity of recipient, as usual, is being withheld until after the board ratifies the decision by reviewers.

Every board meeting has a point at which any person can address the board on any issue. The board sets a three-minute time limit but has been very flexible in the past.

The teleconference locations are in Oakland, Redwood City, Napa, South San Francisco, Beverly Hills, Fresno, San Diego, Elk Grove, Los Gatos, San Francisco (two different locations there) and Irvine. Specific addresses can be found on the meeting agenda,

You may want to double-check in advance with the agency on the addresses. Only one lists a room number. Others may need to include that detail as well.

LA Times on California's Stem Cell Agency, Biotech Gold Rush and Genetically Altering Human Embryos

The Los Angeles Times, which largely ignores the $3 billion California stem cell agency in its news columns, is carrying a piece this weekend that says the agency is considering “work so controversial that the federal government won’t pay for it.”

The reference is to the possibility of the state of California financing research that involves the editing of genes in human embryos, which the agency began to explore at some length at a Feb. 4 meeting in Los Angeles.

The Feb. 12 Times story, written by Melody Petersen, is straight news piece that recaps the controversy about the possibility of making heritable changes in human beings through the use of CRISPR technology. She also covered how the agency intends to proceed with its review.

Peterson described how businesses are embracing CRISPR,

“It has set off a biotech gold rush as scientists imagine its commercial uses and found start-up companies that are attracting hundreds of millions of dollars in venture capital.”

Peterson also wrote about the likelihood of heritable changes in embryos, interviewing Marcy Darnovsky, executive director of the Center for Genetics and Society in Berkeley. She quoted Darnovsky as saying,

"This is not safe. It's still way too early to try such an experiment on a human being."

Peterson continued,

“The agency's current regulations say that no money can be used to transfer a genetically modified human embryo to a woman to start a pregnancy. But some experts worry that agency-funded researchers could later turn to other sources to finance the reproductive stage of their work.

"'If you have genetically modified embryos in labs around the state,’ Darnovsky said, ‘what's to stop them from being used to initiate a pregnancy?"

Peterson quoted Kevin McCormack, senior director for communications at the stem cell agency, as saying, "So far we have not funded any research that involves CRISPR, nor have we received any proposals for funding using that technology. But that's probably just a matter of time."

The stem cell agency has longed struggled with a lack of news media attention. It would like to spread the word about what it considers its good works, such as the well over $500 million it has pumped into enterprises in the immediate Los Angeles area. The LA Times is an especially important medium because it is the largest circulation newspaper in the state, has an enormous Internet presence and helps to drive what is covered by other outlets.

But given the state of the media nowadays and the shrinkage in science reporting, about all the agency can really expect for now is coverage when something extraordinary happens or when there is the likelihood of something extraordinary happening such as genetic changes being made in human embryos.

A Nobel Laureate's 'Unsettling Note' From California's Human Gene Editing Conference

The Center for Genetics and Society, which has been monitoring human gene editing for the past few years, weighed in this week on the possibility that the state of California will beef up its role in financing research involving the sometimes controversial process.

The Berkeley-based center has taken cautionary approach to the field. The article this week on the center’s blog by Marcy Darnovsky, executive director of the group, continued in that vein.

Darnovsky attended the session last week during which the $3 billion California stem cell agency decided to embark on a thorough examination of the topic with the possibility of moving forward on gene editing of human embryos, a field that the federal government does not fund.

Darnovsky provided a summary of the session and cited “an unsettling (if unsurprising) note” from David Baltimore. He is a Nobel Laureate, former president of Caltech and a former member of the governing board of the California Institute for Regenerative Medicine(CIRM), as the stem cell agency is formally known. Baltimore also has been active in sounding go-slow recommendations on human embryo modification.

The concluding paragraph in Darnovsky piece said, however,

“Finally, an unsettling (if unsurprising) note about David Baltimore, who has played an influential role in the current controversy about germline gene editing and who chaired the organizing committee for last December’s International Summit. In previous comments about human gene editing, Baltimore has talked about responsible science; at the CIRM meeting, he came out explicitly in support of human germline modification. In his invited presentation, he said – as if this were a matter of scientific fact – that the desire for biologically related children is genetically hard-wired. He acknowledged that people at risk of transmitting genetic disease can already almost always have unaffected children in a variety of ways, and that therefore germline gene editing would at best benefit very few. But, he continued, ‘there are circumstances where it is the only opportunity for doing what a patient wants....To me, that’s sufficient reason to bring it to clinical use.’"

Darnovsky’s article also discussed testimony by Charis Thompson of UC Berkeley, who raised a number of policy issues.

Darnovsky wrote that they included “reminders that CIRM is mandated to serve not only patients with unmet medical needs, but also the taxpayers and voters of California; that disability justice experts as well as patient advocates should be consulted about gene editing directions; that CIRM should ensure that the work it funds does not exacerbate health disparities; and that if evidence of health disparities or eugenic trends emerges,’real consequences’ must ensue. She concluded by saying that ‘It is not `anti-science’ to note that historically, slopes are indeed slippery,” and that “California deserves – and can have – both the best science and the best ethics.’”

Darnovsky additionally reported on comments by Jeff Sheehy, a member of the CIRM board. She wrote that Sheehy was concerned that the agency might “have little recourse if grantees used other funds to initiate a pregnancy. ‘Where does our reach start and end?,’ he asked. ‘Does it start at the purpose of the proposed research? Do we just say you can’t implant?’ Sheehy suggested that if CIRM approves any grants for research that would produce modified human embryos, it include as a contractual requirement that those embryos cannot be used to initiate a pregnancy, whatever the funding source for that final (and trivial) step.”

CRISPR Roundup: Weapons of Mass Destruction to Gene Spills

The technique called CRISPR and gene editing are making the news again this week, as they are likely to do for quite some time. So here is a quick roundup of stories and links for further examination.

Probably the most provocative story was the addition of gene editing by the nation’s top intelligence chief to a list of threats posed by weapons of mass destruction. Antonio Regalado wrote about the declaration in the MIT Technology Review but said specifics were not cited. Regadalo noted, however, that “scientists have previously speculated about whether CRISPR could be used to make ‘killer mosquitoes,’ plagues that wipe out staple crops or even a virus that snips at people’s DNA.”

Out here in California, the state's stem cell agency wrote about its gene editing review on its blog, The Stem Cellar. Kevin McCormack, senior director of communications, offered this quote from one agency governing board member, Jeff Sheehy:

“Do we need to think about the rights of the embryo donor? If they have a severe inheritable disease and the embryo they donated for research has been edited, with CRISPR or other tools, to remove that potential do they have a right to know about that or even access to that technology for their own use?”

Charles Piller of STAT caught up with the state stem cell agency’s examination of gene editing with a national overview. An excerpt:

“Among state agencies that support stem cell research — including in Texas, Connecticut, New York, and Maryland — only California’s has publicly contemplated human embryo gene editing. The Cancer Prevention and Research Institute of Texas and Bioinnovation Connecticut have not yet considered funding such experiments, their spokespersons said. The Maryland Stem Cell Research Fund has taken no position on this issue, and New York officials could not be reached for comment.”

Paul Knoepfler, a stem cell researcher at UC Davis, wrote on his blog about how CRISPR has

“...set the table for some novel kinds of technological problems for which we aren’t at all prepared including one that I call the ‘gene spill.’ ...We should be very concerned about the possibility that a self-propagating genetic modification could end up out in the real world via a technology called ‘gene drive’ in such a way that it spins out of control. That would be a gene spill.”

Ben Fidler of Exconomy had a piece this morning on the business side of CRISPR based on a conference in Boston. An excerpt:

“The big scare with CRISPR is off-target cuts; that the molecular scissors snip the wrong part of a person’s DNA and cause unintended effects. (Intellia Therapeutics CEO Nessan) Bermingham called this a 'very important question' but said CRISPR technology has come a long way.”

"All In' -- Good News for California's $56 Million Investment in Search for Diabetes Cure

A $56 million bet by the state of California on a diabetes cure is showing additional promise that it will ultimately pay off.

The latest indication came last week when Big Pharma’s Johnson & Johnson went “all in” on ViaCyte, Inc., which has received $56 million from the California stem cell agency during the last 11 years, surpassing the amount that UC Berkeley has been awarded during the same period.

J&J has already pumped $20 million into the San Diego firm, which is conducting a clinical trial for a virtual cure for type 1 diabetes. The effort is showing promising results, according to the stem cell agency.

Last week, in a sign that ViaCyte has was moving along nicely,  J&J turned over its Janssen BetaLogics group to ViaCyte.

BetaLogics was a competitor to ViaCyte and a hedging ploy by J&J.  Business columnist John Carroll of Fierce Biotech described the merger of the two as a major commitment by Johnson. He wrote: 

“J&J goes all in with ViaCyte.”

Carroll reported,

"'We needed to hedge our bet to make sure that we would be the leaders in this space,' says Diego Miralles, J&J's global head of innovation in San Diego. ‘It's clear that ViaCyte has pulled ahead.’”

Randy Mills, president of the $3 billion stem cell agency, said in a news release that “the latest clinical data from ViaCyte are very encouraging and a clear sign of progress.”

The company has been working for years on its product, which is based on human embryonic stem cells. The treatment involves insertion of a tiny device beneath the skin of a patient to produce  insulin when needed.

Linda Johnson of The Associated Press wrote,

"'This one is potentially the real deal,' said Dr. Tom Donner, director of the diabetes center at Johns Hopkins University School of Medicine. 'It's like making a new pancreas that makes all the hormones' needed to control blood sugar.

"Donner, who is not involved in the research, said if the device gives patients normal insulin levels, 'it's going to prevent millions of diabetics from getting dangerous complications.'"

Paul Knoepfler, a stem cell researcher at UC Davis, wrote on his blog that the J&J move was “great news” for ViaCyte. He said the folding of BetaLogics and recent results from the phase one clinical trial “solidify ViaCyte’s leadership.” Knoepfler also is not involved with the ViaCyte research.

Before the therapy can be marketed widely, it must clear both a phase two and phase three trial, which could take a few years.