New Jersey Lessons for California

While California is just embarking on a $3 billion stem cell research effort, New Jersey has already poured hundreds of millions of dollars into the biotech biz in its state. And it is considering pumping another $380 million into stem cell research.

New Jersey's efforts and its difficulties have some obvious implications for California. Reporter Clint Riley of the Record in New Jersey provided some perspective on the situation in the Garden in a couple of articles.

He reported on Sunday: "New Jersey's partnership with biotech is already running into problems. It is cloaked in secrecy and riddled with the potential for conflicts. Its goals are at times nebulous."

On Monday Riley wrote, "Secrecy is important to the biotech industry, in both its business and research incarnations. After all, this is a world where the sole ownership of ideas is the basis of profits. The picture is more complicated on the public side of the biotechnology partnership.
In particular, a closed-door culture poses significant challenges for those concerned about conflicts of interest."

His reports detail examples of where New Jersey's public interests and those of the private sector do not necessarily coincide. He also noted that New Jersey ponied up $500,000 for promotion at the 18,000-attendee BIO 2005 conference mentioned in the item below.

The articles make interesting reading for those following the affairs in California.

Stem Cell Snippets: Surfing the Big Wave to Practicing PR

Trounson's Longings – CIRM President Alan Trounson says he "absolutely" misses being in the laboratory. Trounson (photo at right) made the comment in an interview late last month on Australian radio. He told interviewer Monica Attard on ABC radio that "I still wake up at night thinking I’m in the laboratory." Trounson also talked about how grant money was luring Australian researchers away from their country. "It’s like surfing in the big surf and unless you can catch the wave you’re never going to get the thrill of it. If you are sitting there and waiting for the wave to come and ... you might get on it, you might not because there’s really not much funding...." He went on to say that "a lot of really good scientists have relocated (from various places) to California because of the money and because of the opportunity."

Liberals Criticize Grant to California Stem Cell Firm – The state is New Jersey. The firm is StemCyte of Covina, Ca. The money relatively small – only $589,000. But a New Jersey state grant in that amount inveigled the company to open a New Jersey facility, creating 12 jobs. Questionable financial decision for the state, said the New Jersey Policy Perspective group, while higher education and transportation budgets are being cut in that state.

CIRM PR Contract – The California stem cell agency is looking for some public relations assistance, specifically a communications special projects manager. Under the terms of the RFP, compensation would amount to $90,000 under a 13-month "all inclusive contract including all administrative expenses and travel." The consultant would be required to work no more than 30 hours a week on an as-needed basis. Deadline for applications is May 19 with the award date scheduled for May 26 and possibly earlier. The agency has also posted job openings for a communications manager, IP attorney, scientific officer and two administrative coordinators (one senior).

The Shifting Sands of Stem Cell Support

New Jersey voters on Tuesday sent a message to sanguine supporters of stem cell research in California: Do not assume that the public is always behind you.

Analysis this morning of the election results is a bit preliminary but news reports are characterizing the rejection of the $450 million stem cell research measure as a surprise.

And it is not good news for those in California who find reassurance in the 59 percent voter approval of Proposition 71 in 2004, the measure that created the state's $3 billion stem cell program.

We have pointed out previously that stem cell research is not well understood by the public. Support for it is weak despite often rosy polls that seem to indicate it is a motherhood issue, at least in the eyes of some at CIRM. That is not the case, as shown in a poll by the Pew Forum for Religion and Public Life. According to that survey, support dropped from 57 percent nationally two years ago to 51 percent in August this year. It also showed that 55 percent of the public had heard little or nothing about stem cell research.

The New Jersey vote signals that it is imperative for CIRM to move forward thoughtfully and effectively on its public education/PR plans and promptly fill the vacant position of chief communications officer.

The New Jersey vote showed the vulnerability of stem cell research in the political marketplace. Voters can be fickle. To forestall erosion of support in California, CIRM must move to shore up its weaknesses. Those include its penchant for closed doors and secrecy – all of which breed suspicion and provide a recipe for scandal.

Celularity and $750K from California Stem Cell Agency: Where is the Money Being Spent?

The New Jersey firm that was awarded $750,000 by the California stem cell agency to help finance a Covid-19 clinical trial has four trial sites in two states, but has not identified any in California. 

State law requires that awards from its taxpayer-funded stem cell agency be spent for work in California. 

The firm is Celularity, Inc., which is developing a product that has been fast-tracked by the federal government and which has the support of President Trump's personal attorney, Rudy Giuliani. 

According to the National Institutes of Health website this morning, Clinicaltrials.gov, Celularity has three clinical trial sites in its home state of New Jersey and one in Washington state for the Covid therapy. The information shows that all of those sites are currently recruiting patients. The estimated primary completion date of the work is Nov. 30, 2020.

Although the stem cell agency said on Sunday that a trial site existed in California, it declined to disclose the location. The agency referred questions about a site in California to the company, which has not responded to three requests yesterday and today for information.

Corey Casper, interim president of the Infectious Disease Research Institute in Seattle, is listed as the principal investigator for the trial by Celularity. Casper spoke to stem cell agency directors on May 15 on behalf of the application. Casper also did not respond to inquiries about the location of the California work. 

Six weeks ago, when Celularity's application first came before directors of the California Institute for Regenerative Medicine (CIRM), Casper said the treatment "has a biologic plausibility for being very important and very unique in its treatment for Covid disease."

He continued,

"I think that we have adequate safeguards that have been put into the trial to really assure the safety of the participants."

Safety concerns have been raised by CIRM's grant reviewers as well as by other scientists. The reviewers, who are from outside California, originally rejected the application, giving it a score of 84, one below the cutoff point. After Celularity addressed reviewers' concerns, it scored 85 and was sent to directors who approved it last Friday with no debate.

It is CIRM's 64th clinical trial.

The Stem Cell Race: How California is Like the Cobbler's Children...Sort Of

California's stem cell effort, still yet to dispense a single research dollar, has served as lever globally to boost funding for the field in locations ranging from New Jersey to Australia.

That's one of the conclusions of the third article in a three-part series in the San Diego Union-Tribune called "The Stem Cell Race."

Written by reporter Terri Somers, the piece examines California's $3 billion effort, which has been hampered by a legal tussle over its legitimacy. Somers wrote:

"While San Diego's large stem cell research community has been waiting to tap the state funding, the Harvard University area – supported largely by philanthropists – has become the U.S. science cluster best known internationally for embryonic stem cell research.

"Also pushing ahead have been the governments of Singapore, China, Japan and several European nations, which have supported their embryonic stem cell scientists with money and favorable policies.

"'Proposition 71 is supposed to help the economy by creating jobs first, then new tools and treatments, but until it really gets moving it's just an old Jag in the garage," said Tom Okarma, chief executive of Geron, a Menlo Park stem cell research company."

Somers continued:

"New York Gov.-elect Eliot Spitzer plans to push a $1 billion 10-year stem cell initiative that mirror's California's. On Friday, New Jersey's legislature approved borrowing $270 million to fund stem cell research. And Connecticut has floated a $100 million 10-year initiative. Part of the states' impetus was the fear of losing top researchers to California and abroad."

The San Diego article also reported:

"Scientists and government officials from 15 nations have visited the stem cell institute over the past year, said Zach Hall, the institute's president. Delegations from India, Israel, the United Kingdom and China were eager to forge relationships and take home ideas and the possibility of collaborations."

Nonetheless, Somers reported that 30 "notable" scientists have come to California in the last two years because of the state effort. An informational graphic with the story showed that private and federal funds totalling $234 million have poured into the state, boosting research efforts. And she noted that CIRM now has $181 million in hand, ready to dispense on research.

"'With these loans California's funding is now six times the nation's funding through the (National Institutes of Health) and California is squarely in the global leadership of this breakthrough field of medical research,' said Robert Klein, chairman of the California Institute for Regenerative Medicine."

UC Irvine Named for Covid-19 Trial Backed by California Stem Cell Agency and Giuliani

A New Jersey firm backed by $750,000 from the California stem cell agency today announced that the California location of its Covid-19 trial will be at UC Irvine.

The firm is Celularity, Inc., and its proposed therapy is supported by Rudi Giuliani, President Trump's personal lawyer.  Celularity expects to complete its work by Nov. 30.

CIRM said the research involves "a phase I/II study of human placental hematopoietic stem cell derived natural killer cells for the treatment of adults with COVID-19."

The award was made June 26 by the agency's governing board, which expected its Covid grantees to be ready to start work within 30 days under its fast-track Covid round. Celularity had not responded to questions about the trial location prior to its news release today. The stem cell agency cannot fund research outside of California. 

The company's news release indicated there would be more locations in California, but did not disclose any. 

The UC Irvine work will be overseen by Leonid Groysman, a neurologist. Here is a link to a summary of the review of Celularity's application (CLIN2COVID19-11857 #2). 

A Look Behind the Cyberspace Curtain

It's been a merry old romp.

Three years plus and more than 1,500 items posted. That's the count for the California Stem Cell Report, which began its cyberspace explorations of the stem cell world in January of 2005.

A little more than 12 months ago, we gave you a bit of an annual report on this enterprise, which is the creation solely of yours truly, David Jensen. Along with that came our economic disclosure. Today we will give you an update in both areas.

Nothing has changed on our economic interests, which can be found here. But briefly neither my wife nor I nor any members of our immediate family hold any interest in biotech or stem cell companies or other enterprises that could stand to gain or profit or benefit from the activities of California's stem cell agency.

This blog is financed personally on an extremely low budget. Some people ask why I do it. The answer is that the California Institute for Regenerative Medicine and its related activities are interesting, important and unique.

The Golden State's $3 billion enterprise is on the cutting edge of science, business, politics, religion, medicine – not to mention life, death and even sex(certainly one or another stem cell therapy is likely to tackle that last area). How often does one have a chance to dig into all those areas?

The overall readership of this blog is modest by Internet standards. No megamillion counts of page views here. We are currently running about 8,000-9,000 page views a month. Our readers, however, are deeply interested in the subject and seem to represent important sectors in the world of stem cells. They include folks from the UK, Canada, Australia, Korea, India, Germany, Sweden and Singapore. Readers from virtually all the major California research institutions dip into the California Stem Cell Report from time to time. But less than half of the total are from California. Other regions represented include New York, Massachusetts, New Jersey, Wisconsin, Illinois and Texas, Readers also come from enterprises such as the National Institutes of Health and the National Academy of Sciences along with patent attorneys, lawmakers and their aides and investment companies.

This past 12 months, we have branched out somewhat, publishing freelance articles with Wired News online and a couple with The Sacramento Bee.

We also broke the story on the attempt by one of the CIRM directors to lobby the agency on behalf of a grant to his institution. That story led to an ongoing investigation by the state Fair Political Practices Commission and a new audit of CIRM. The story also apparently played something of a role in the formulation of the latest legislation targeting CIRM.

We take no particular pleasure in CIRM's missteps. We support human embryonic stem cell research and CIRM's programs generally. Our main effort is simply to present information about CIRM, which has slipped well below the radar of the mainstream media. We do have a point of view, however. Our starting point is that CIRM is a public agency first. Everything else is a poor second. Without public trust and credibility, without conduct that matches the standard for Caesar's wife, CIRM's efforts could easily become a poster child for what can go wrong. Instead of being an exemplar, CIRM could become a nightmare of conflicts and concealment because it is uniquely free from normal state oversight and is riddled with built-in conflicts of interest that are not going to disappear. As we have reported, the conflicts are so deep that more than once the vast majority of CIRM directors have been disqualified at meetings from taking part in public discussions, not to mention being barred from voting. The result is that sometimes decisions can be made by as few as four out of the 29 members on the board.

We are interested in hearing from readers, both on individual items and on the general direction of the blog. If you have suggestions for changes, improvements or whatever, please send them along to me (djensen@californiastemreport.com) or you can post them via the comment function (created by Google), which does not allow myself or others to know your identity.

As for others involved regularly in CIRM affairs and regularly trying to influence the organization, we urge them to disclose their financial interests as well(via the comment function or other means). That includes newspaper reporters, but that is not anymore likely to happen than CIRM opening the scientific grant review sessions to the public.

Rationale Behind Kuehl's Stem Cell Legislation

The new chair of the California State Senate Health Committee will "push the CIRM to live up to its promises," including insuring that all Californians will have access to any therapies developed as the result of state-funded stem cell research.

So says the January newsletter put out by Sen. Sheila Kuehl, D-Santa Monica. The newsletter carried a piece by Peter Hansel, staff director of the Senate Health Committee, that discussed the California stem cell agency at some length, including needed changes. Hansel, it should be noted, also served as a Health Committee analyst when it was chaired by Sen. Deborah Ortiz, who is now out of office.

Hansel wrote that Kuehl disagrees with arguments that CIRM should should go easy in terms of royalty requirements and affordable access to cures and therapies. He noted that CIRM has adopted some IP rules that respond to legislative concerns. Hansel continued:

"However, regulations governing the pricing of stem cell therapies in California run counter to assurances given to the Legislature. While at first proposing that grantees and licensees sell such therapies to publicly funded programs at the federal Medicaid price, the CIRM has recently reduced that to a requirement to sell at the same prices they offer them to the state’s new California Prescription Drug Discount Program, a significantly less favorable price. Similarly, the regulations for grants and loans to commercial entities propose to cap the amount of revenues coming back to the state associated with most products developed with Prop. 71 funds, as opposed to giving the state an open-ended return commensurate with its investment. By contrast, New Jersey, which also allows commercial entities to receive grants under its stem cell research program, requires such grantees to share a percentage of revenues—one percent—on an open-ended basis from licensing or commercialization of inventions (two other states that fund stem cell research, Illinois and Connecticut, do not allow for-profit entities to receive funding while another two, Maryland and Illinois have yet to develop policies for grants to for-profits). Capping returns from joint research ventures also appears to run counter to the practices of most universities who co-fund research with commercial entities and the venture capital industry.

"In addition, recent changes require grantees to grant exclusive licenses for inventions they develop to entities that agree to have plans at the time of commercialization to provide access to resultant therapies and diagnostics for uninsured patients, consistent with 'industry standards,' The current industry standard, the patient assistance programs that have been developed by the major drug companies, have been shown to be woefully inadequate in encouraging access to free or reduced price drugs for uninsured persons. Thus, it is incumbent on the CIRM to develop a more meaningful standard than this."

calif legislation, IP, affordability, access, skuehl, phansel

Another Perspective on the Trounson Flap: Columnist Lists It as Example of 'Possible Corruption'

The $3 billion California stem cell agency was mixed up with some bad company this week – part of the fallout from last month’s Trounson Affair. 

The agency was lumped together with some ne’er-do-wells in a column in California newspapers that was headlined:

“California state government awash in corruption, conflict of interest”

The piece was written by Tom Elias, a middle-of-the-road, longtime columnist whose work appears twice weekly in 93 newspapers with a circulation of 2.2 million. Elias wrote,

“To some, it seems almost as if California has lately become New Jersey West. Incidents of possible corruption and conflict of interest are seemingly exposed at least once a month these days, with almost no consequences for anyone involved.”

Top on his list was the business involving Trounson, who left the California stem cell agency June 30. Seven days later, he was appointed to the board of StemCells, Inc., which holds $19.4 million in awards from the agency, formally known as the California Institute for Regenerative Medicine (CIRM).

Second on Elias’ list was a case involving a consultant at another state department that was reminiscent of another situation at the stem cell agency. Elias said,

“A month earlier, this column caught the state Energy Commission earmarking more than $28 million in ‘hydrogen highway’ grants for a new company co-founded by a consultant who only months earlier drew the map determining where hydrogen refueling stations will go and then trained commission staff on how to evaluate grants.

“No conflict of interest there, the commission insisted. Right.”

The case at CIRM involved a “special advisor” by the name of  Saira Ramasastry, managing partner of Life Sciences Advisory, LLC, of Emerald Lake Hills, Ca. In 2012, she was named to the board of Sangamo BioSciences, Inc., a publicly traded firm based in Richmond, Ca. At the time of her appointment to the board, the firm shared in a $14.5 million grant from the agency. She also worked as a consultant to Sangamo.

Since then, Sangamo has received another $6.4 million from CIRM. The president of Sangamo, Edward Lanphier, told the agency last January,

“We wouldn't be where we are today without you.”

In 2013, Ramasastry received $99,662 in cash and stock options from the company, according to its filings with the Security and Exchange Commission.

First Chance for Business to Snag California Stem Cell Dollars

Attention Stem Cell Businesses! California's $3 billion wagon of cash is moving fast, and it is now time for you to jump on board.

The California Institute for Regenerative Medicine is looking for a few good businesses, and it wants to give a few million dollars to them. Even if you are headquartered in New Jersey or anywhere else in the world, it makes no difference. Only a couple of catches – you must have a "research site" located in California by Feb. 5. That site is also where the CIRM-funded research must be done.

Feb. 5 is the deadline for applications for a $25 million round of grants for research into development of new cell lines, both from IVF and alternative methods such as reprogramming. Sixteen grants are expected to be awarded with funding possibly as early as August of next year.

This is the first opportunity for businesses to seek grants from CIRM. It appears that they will be competing for stem cell dollars against academic and nonprofit researchers. It is hard to set the odds on the competition given the array of stem cell talent in the Golden State. But some CIRM directors are eager to pump money into the private sector, figuring that could pay off by developing therapies faster than going the academic route. Others are simply sympathetic to the needs of cash-starved stem cell enterprises.

The news about business grants was buried deep in the new cell lines RFA that CIRM posted with no fanfare last Thursday. John M. Simpson, the stem cell watchdog for the Foundation of Taxpayer and Consumer Rights, was quick to see it. (We also wrote about it for Wired.com in a piece that appeared Friday evening.)

Simpson issued a news release on Friday afternoon noting that biotech companies play an important role in stem cell research. But he also said that CIRM should not be in the business of writing blank checks for biotech. Simpson declared,

"We’ve already seen an example of an improper attempt by a stem cell board member to influence an award to his non-profit research institution. The possibility of abuse is even greater when the biotech industry goes after the money.”

Simpson has been active in development of intellectual property policy of the the stem cell agency, which is the key mechanism for the state to share any wealth that may result from CIRM-funded research. It is also the mechanism for assuring affordable access to CIRM-backed therapies. On Dec. 12, the agency's directors are expected to approve its IP policy for grants to business. Businesses can still weigh in with comments ahead of the meeting or at the meeting itself. Our advice is to do both, if it is important to your enterprise.

The exact IP policy that is to be considered is not yet posted as part of the agenda for the meeting, but the RFA has a link to this related document.

The reprogramming effort should come as no surprise to those who follow the agency closely. Arlene Chiu(see photo), former chief scientist at CIRM, and others were working on the new cell line proposal, including the reprogramming aspects, well before the news surfaced last month about a breakthrough in creating pluripotent cells from adult skin cells.

In October, she presented the concept for the new cell line grants to CIRM directors, declaring,

"We want to support the full spectrum of human pluripotent stem cell types and experimental approaches."

Also open to businesses is a $1 million round of planning grants to help lay the foundation for awards in a much larger series of "disease team" grants. That round will total roughly a whopping $122 million. In the case of planning grants, the RFA states that "for-profit organizations in California" may apply, but it does not specify whether they must be headquartered in the state or simply have operations in California.

Here are the key dates for the planning grants: Candidate nomination forms and letters of intent, Dec. 20; applications Jan. 31.

Key dates for the new cell grants: Candidate nomination forms and letters of intent, Jan. 10; applications, Feb. 5 as noted previously.

CIRM Dwarfed in Biotech Patent Fight

The battle to protect the biotech industry from generic drug makers, a war in which CIRM is a dogface, is going swimmingly, according to an article this week by The Associated Press.

In a piece headlined “The influence game: Biotech drug lobbying war,” Alan Fram wrote that the weapons include “millions of dollars in lobbying, thousands in campaign contributions and uncounted visits to members of Congress. And one noteworthy letter.”

His conclusion?

“The lobbying battle has so far been one-sided. The Senate health committee voted 16-7 for a 12-year (patent) protection period last month, while (Rep. Henry) Waxman's House energy panel voted 47-11 for 12 years of protection last Friday on an amendment by Rep. Anna Eshoo, D-Calif., who has numerous biotech firms in or near her Silicon Valley district.”

CIRM is a decidedly bit player on this stage, given the vast stakes and expenditures involved. The agency has written a letter – NOT the one cited by Fram – after directors, over the last few months, debated whether a state-funded agency should lobby to protect the biotech industry. That letter in turn generated another letter from U.S. Sen. Dianne Feinstein. (Both can be found here.)

CIRM also has hired a Washington lobbying firm, the Podesta Group, an unusual step for a state agency, paying it $240,000 for 10 months work. But Podesta's assignments from CIRM seem to involve more than patent protection.

All of CIRM's efforts are hardly a dribble on the health care reform playing field, where all the action is taking place. The Associated Press article, distributed nationally, spells out how money talks when it comes to locking up intellectual property. Fram described a “huge disparity” in cash. He wrote,

“Representing biotech companies, the Biotechnology Industry Organization has spent $3.7 million lobbying so far this year. Their ally, (the Pharmaceutical Research and Manufacturers of America), has spent $13.1 million — the second most of any group that lobbies in Washington.

“The main group opposing them, the Generic Pharmaceutical Association, has spent $1.1 million lobbying this year. Another group, a coalition of generic drug companies, insurers and large employers, has spent another $180,000, though most of its members — like AARP and the General Motors Corp. — are more focused on the overall (health reform) bill and are devoting few resources to the generic fight.”

Fram continued,

“All that money has let the biotech industry launch a lobbying blitz, with targets including lawmakers from biotech-heavy states like California, Maryland, Massachusetts, New Jersey and North Carolina.

“The biotech organization has brought its CEOs to Washington, and has run print and radio ads in the states of pivotal lawmakers. The pharmaceutical association has helped organize lobbying by universities that conduct biotech research and venture capitalists who invest in such firms, and paid for a Duke University study that concluded biotech firms need 12 to 16 years of protection from generic competitors to break even.”

All of which brings us to a business maxim of Jack Welch, the fabled executive who once led General Electric. He did not mess with enterprises in which GE could not be No. 1 or No. 2. Even today, his Web site says that maxim “stopped the decades-long practice of sprinkling money everywhere.”

The California stem cell agency is not even close to being No. 1 or No. 2 in the biotech patent protection game. That is something for CIRM directors to think about as they consider their priorities later this month when they deal with their strategic plan. Their grant portfolio is just about to hit $1 billion. They have only a tiny staff to oversee those expenditures and to hand out another $2 billion over the next few years. They will soon launch a new $210 million grant round, the largest research round ever for CIRM. And they will enter new, uncharted waters with a risky $500 million biotech lending program. A sharper focus on essentials may be in order.

Ebert on WARF and the Patent Challenge

Lawrence Ebert, a patent attorney in New Jersey, has a different take on WARF and the stem cell patent matter. Among other things, he faults The Sacramento Bee and challenges the challenge to the patents:

"Patent claims are not invalidated because earlier researchers paved the way for later researchers. PubPat's anticipation argument for human embryonic stem cells is based on a prior reference which is not enabled as to human embryonic stem cells. To look in a different area, the work of Galileo (and others) may have paved the way for the Wright Brothers, but no one achieved three dimensional flight control before the Wright Brothers, or taught how three dimensional flight control could be achieved. PubPat's obviousness argument is based on the assertion: recipe for mouse embryonic stem cells renders obvious recipe for human embryonic stem cells. If this were true, it probably would not have taken 15 years between mouse and human."

Covid Clinical Trial Location Still Missing on $750,000 California Stem Cell Grant

The California stem cell agency stipulates that winners of research awards in its fast-track, Covid-19 round must be ready to start work within a month of approval, but a New Jersey firm that snagged $750,000 three weeks ago has yet to report the location of its California work.

The  firm is Celularity, Inc. Its proposed therapy is backed by Rudi Giuliani, the president's personal lawyer, who has also touted other possible Covid therapies, including one that has been rejected by the Food and Drug Administration.

The $750,000 is intended to finance a clinical trial someplace in California. The stem cell agency funds only California work.

In creating the Covid round, CIRM said award winners must be "ready to start work within 30 days of approval and propose achieving a clear deliverable within six months." It has been 21 days since the Celularity award was approved. 

The California Stem Cell Report has asked the firm repeatedly for the location of the California site but it has not responded with this routine information. The agency itself also has not disclosed the location and defers to the company.  

Celularity updated the information today that it files with the National Institutes of Health on the trial. However, the locations of the trial still did not show any in California. 

Covid Research Backed by California's Stem Cell Agency: 15 Awards, Including Help with Three Clinical Trials

The California stem cell agency is down to its last $272,357 in its special, $5 million effort to help finance research involving Covid-19.

The research round began in May and has approved 15, fast-track research projects, including participation in three clinical trials. The latest awards were approved last Friday by agency directors. 

• Evan Snyder of Sanford Burnham Prebys Medical Discovery Institute received $250,000  to create lung organoids to test two drug candidates for treatment of the virus.
• John Zaia of the City of Hope received an additional $250,000 for his previously approved study dealing the use of convalescent plasma treatments. 
• Steven Dowdy at UC San Diego was awarded $150,000 to study a more effective way of delivering a genetic medicine, called siRNA, into the lungs of infected patients.

Summaries of the CIRM application review on the grants can be found here, including the six rejected.  

Approval of the latest awards came as a New Jersey firm that two weeks ago received a $750,000 award still has not disclosed the location of its clinical trial site in California. By law, the stem cell agency can only finance work within California.

The awardee is Celularity, Inc., a business whose proposed therapy is backed by Rudi Giuliani, the president's personal lawyer, who has also touted other possible Covid therapies, including one that has been rejected by the Food and Drug Administration.

In creating the Covid round, CIRM said award winners must be "ready to start work within 30 days of approval and propose achieving a clear deliverable within six months." It has been 16 days since the Celularity award was approved. 

The California Stem Cell Report has asked the firm repeatedly for the location of the California site but it has not responded. The agency itself also has not disclosed the location and defers to the company.

A View from the East

For another perspective on the California Stem Cell Report and whether it follows the party line of the California stem cell agency, curious readers can take a look at the Ipbiz blog produced by Larry Ebert in New Jersey.

On Tuesday, Ebert described this report as a "fantasyland." Today he suggested the report was something of a tool of California's stem cell agency.

California patient advocate Don Reed also weighed in. His comments are contained at the end of the Tuesday item.

2009 CGS Forecast: Petri Dishes -- Not Stem Cell Therapies

The usual season for prognostications for the coming year is the end of December. But Jesse Reynolds of the Center for Genetics and Society in Oakland, Ca., weighed in last week with a couple.

Writing on his group's Biopolitical Times, Reynolds said,

"With the end of stem cell research as a political vehicle, its advocates are likely to temper expectations. They'll not just move out the goalposts on the timeline towards treatments, but the touted uses of stem cells will shift from potential cellular therapies to models of human diseases in Petri dishes and better drug testing methods. These new purposes will win fewer votes than "your own personal biological repair kit," but they are also much more realistic."

Reynolds also predicted there will be no outpouring of federal cash for hESC in the near future. He made a different case than we did in our item below. He said,

"Even when President-elect Obama removes the Bush restrictions, federal funds will be available only to work with embryonic stem cell lines, not to create new ones. Grants for the latter are restricted by the Dickey-Wicker amendment, which would be left in place by both the repeatedly-vetoed stem cell bill and Obama's platform."

Reynolds also made his case for the death of hESC research as a political vehicle. He said,

"...(T)he real message from this election cycle is the end of embryonic stem cell research as a relevant political issue. It was huge in 2004, present but marginal in 2006, and seemed comatose with the 2007's failure of New Jersey's stem cell funding initiative. In this cycle, the topic made barely a peep.

"Hopefully now work can proceed in concert with a level-headed conversation about the true potential of stem cell research and the real challenges posed by human reproductive and genetic biotechnologies."

Two Days of Stem Cells: Founder Flight to Hyperventilation

Christopher Thomas Scott, executive director of Stanford's Program on Stem Cells in Society, is scheduled to set the scene Monday for a two-day international conference on stem cells in San Francisco.

"The business issues are profound," he says, "including access to patients, fragmented intellectual property and a new calculus of investment risk that includes whether the research is illegal and how to mitigate against 'founder flight' as entrepreneurs seek permissive jurisdictions to launch their businesses."

We asked Scott, who is co-chair of the conference sponsored by Burrill & Company, for a preview of his remarks. Here is what he supplied.

"No one can deny the promise of regenerative medicine. But the field has its shaky spots: an astonishingly young science, polarized politics, and fraught with ethical worry. Yet stem cell biology has been on a tear lately. In just a handful of years, the science has moved from hunting stem cells to the arcane secrets of signal transduction. The hyperventilation about which stem cells--embryonic or adult--will be clinically useful is largely lost on scientists. The questions facing them are more elemental: can stem cells be chemically reprogrammed to earlier, more powerful versions of themselves? On which branch of the family tree does a new stem cell rest? What gene signals cause a stem cell to make more stem cells, or change into the next cell type down the line? The last question is on every researcher’s mind, because signal pathways are critical to understand how a certain type of cell can be made from an embryonic stem cell line, or how millions of adult stem cells can be made from a just a few to treat disease.

"2006 was a watershed year in other ways. Most Americans support embryonic stem cell research, and so does Congress. Despite a vote in the House and Senate that would overturn a restrictive presidential mandate, it wasn't enough to override George Bush's first-ever veto. California pushed through a thicket of lawsuits to shake loose billions of dollars for regenerative medicine. Now, finally, there is light at the end of that tunnel. Legislation in other states is moving so quickly it's difficult to keep track: just last week, Illinois, Iowa, Minnesota signed laws to permit all types of stem cell research. As political winds fanned the flames stateside, stem cells went international, creating a different kind of global warming. In a mighty push, Australia overturned a ban on nuclear transfer. The world's researchers had a banner year, with Japan, Germany, Norway and others announcing major discoveries. Not all the offshore news was good, however. The heat created a conflagration with the biggest scientific fraud in memory, the South Korean scandal.

"One thing is certain--international politics and the legal landscape has altered the way we do biomedical research. Thomas Friedman's "global flattening" doesn't apply here. A mosaic of legislation and national policy means uneven terrain for funding, infrastructure and accessibility to embryos and lines. The business issues are profound, including access to patients, fragmented intellectual property and a new calculus of investment risk that includes whether the research is illegal and how to mitigate against "founder flight" as entrepreneurs seek permissive jurisdictions to launch their businesses. The vacuum in Washington has shattered the state legislative landscape. In one state, a scientist can go to jail for doing embryonic stem cell research. In another, embryos can't be used for research, but it is fine to ship them in across the border. And who would have predicted this in 2001, the year of Bush's pronouncement: once funding from California, Wisconsin, New Jersey, Connecticut, Illinois, Maryland, Massachusetts, and other states is fully unleashed, it will surpass by a wide margin any dedicated federal dollars, restricted or otherwise.

"With all the moving parts, it made sense to assemble a group of experts and scholars from many disciplines to address issues at the interface of science, business, economics, law, and policy. I was delighted when Burrill & Company asked me to develop an agenda that would explore these connections. As a rule, stem cell conferences tend to be monolithic, in part because the reach of regenerative medicine is too broad to be addressed in two or three days. But to my knowledge, no conference tackles these questions from an international perspective. I'm excited to learn what this stellar group has to say, and how the glimmering edge of biology's most promising frontier will look in 2007 and beyond."

We will attending the conference both days. Watch for continuing coverage of the event.

California's $5.5 Billion Stem Cell Campaign and Covid-19: How Science and Politics Marry

Editor's note: The following item written by the producer of the California Stem Cell Report appeared yesterday on the Capitol Weekly online news service. 

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The California stem cell agency has just finished pumping $5.3 million into the fight to save the lives of COVID-19 victims. And — in a ballot-box bonus — its efforts are already surfacing in the ballot campaign to rescue the agency from its own demise.

The agency is running out of money. It will begin closing its doors this fall without major financial support that it hopes will come from Proposition 14, a $5.5 billion bond measure on the November ballot.

The $5.3 million in the fast-track, Covid program is tiny by comparison. But the awards are likely to play a heftier role than might be anticipated based on their size. Stories about the Covid awards have appeared on the campaign website in a move to tap into the deep public concern about the coronavirus.

The Covid research piggybacks on the federal “warp speed” drive to develop Covid treatments and is aimed at demonstrating that the agency is part of what CIRM officials describe as a worldwide, “all-hands-on-deck” effort.

Three clinical trials have been funded, which involve actual patients as opposed to laboratory research. If the trials are successful, patients who have benefited are likely to surface in campaign video advertising aimed at refinancing the agency, formally known as the California Institute for Regenerative Medicine (CIRM).

CIRM sorely needs research results that will resonate with voters. The 2004 ballot campaign that created the $3 billion program raised expectations that stem cell therapies were right around the corner. But the agency has yet to produce a stem cell treatment that is widely available to the public.

The agency launched its Covid round on March 27 when CIRM board chairman Jonathan Thomas declared, “The coronavirus is creating an unprecedented threat to all of us, and, as one of the leading players in regenerative medicine, we are committed to doing all we can to develop the tools and promote the research that will help us respond to that threat.”

Indeed, failure by CIRM to respond to the COVID-19 threat — it could be argued — would be a breach of CIRM’s public responsibilities.CIRM’s Covid research financing is a case where an urgent medical need marries nicely with ballot-box politics. The agency is forbidden by law to campaign for the ballot measure. But its charter also allows it to pursue a wide range of research possibilities.

Late last month, CIRM directors capped off the round by approving two awards and adding nearly $300,000 to the original authorization of $5 million. All of the awards have been made under an emergency process that requires awardees to be ready to begin research within 30 days and achieve “a clear deliverable within six months.”

The final awards went for basic level research. Earlier, CIRM approved funds to assist in the three clinical trials, which are the final stage before a treatment can be approved for widespread public use. The trial awards range from $1 million to $701,000 each, a bargain price for joining a trial. Most of CIRM’s previous clinical trial awards run into many millions.

CIRM now counts a total of 64 clinical trials that it is helping to finance. Two of the Covid trials are taking place at the University of California, San Francisco-UC Davis and the City of Hope in the Los Angeles area. The third involves a New Jersey firm called Celularity, Inc., which has failed to respond to multiple questions about the California location of its trial. The agency is limited to funding activities within the state. CIRM directors approved the Celularity award on June 26, more than 30 days ago.

The Celularity award has another interesting angle. It involves a treatment supported by President Trump’s personal attorney, Rudi Giuliani. 

You can find more information about all the awards in a series of items on CIRM’s blog, The Stem Cellar, using the search term “covid.”   Five CIRM/Covid stories, including CIRM blog items about the Covid round, have been placed on the campaign website by the campaign staff. 

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​Read all about California's stem cell agency, including Proposition 14,  in David Jensen's new book. Buy it on Amazon:  California's Great Stem Cell Experiment: Inside a $3 Billion Search for Stem Cell Cures. Click here for more information on the author.

Stem Cell Research State by State

Stateline.org today carried a detailed overview of embryonic stem cell research efforts state by state, including the case of a woman who unsuccessfully tried to donate a leftover embryo from her IVF treatment in Michigan.

She was told she had to go to another state because Michigan law bans research on human embryos.

Writer Christine Vestal put together the piece, which goes into some detail on each state with links to the agencies that do the work.

Here is an excerpt:

"Seven states — California, Connecticut, Illinois, Maryland, New Jersey, New York and Wisconsin — are providing seed money for the fledgling science, and Massachusetts Gov. Deval Patrick (D) in May called on lawmakers in his state to follow suit.

"Six other states — Arkansas, Indiana, Louisiana, Michigan, North Dakota and South Dakota — ban the research. Three states — Iowa, Massachusetts and Missouri — have affirmed its legality but do not offer funding.

"In Florida and Texas, lawmakers are deadlocked on the issue. Most states have steered clear of it altogether."

Diabetes to Arthritis: California Awards $33 Million for Clinical Stage Stem Cell Work

The California stem cell agency today approved nearly $33 million for clinical stage research projects testing treatments for type 1 diabetes, arthritis of the knee, ALS and an immunodeficiency affliction.

The awards were quickly approved with little discussion during a meeting at the Oakland headquarters of the California Institute for Regenerative Medicine or CIRM, as the agency is formally known.

The award likely to have an impact on the most people -- if it is successful -- is a relatively small, $2.3 million award to the Cellular Biomedicine Group,  a Chinese firm with operations in Cupertino, Calif. The stem cell agency by law only finances work in California. The research would also be supported by $572,993 in co-funding.

The project is aimed at treating osteoarthritis of the knee. More than 51 million people in the United States suffer from arthritis, which is particularly common in the knee.

The goal of the research is to regenerate knee cartilage through the use of a mesenchymal progenitor cell treatment, according to the agency's application review summary. The funding would go to manufacture the product and complete work to secure Food and Drug Administration approval for a phase one safety trial. A treatment for the public would likely be years in the future.

Here are the other winners today of California stem cell cash with links to the summaries of the reviews.

Caladrius Biosciences of New Jersey won $12.2 million for a clinical trial for young people ages 12-17 for newly diagnosed type 1 diabetes. The firm plans to use regulatory T cells from the patients themselves to treat the disease. Caladrius has a California location in Mountain View. (Caladrius' press release can be found here.)

St. Jude's Research Hospital in Memphis, Tenn., was awarded $11.9 million for a phase one/two trial to treat infants with X-linked severe combined immunodeficiency. The trial would aim at enrolling at least six patients suffering from the catastrophic affliction. The treatment would use the patients own bone marrow stem cells after the cells were specially handled. The agency said in a press release that St. Jude's is working with UC San Francisco. (St. Jude's press release can be found here.)

Cedars-Sinai Medical Center in Los Angeles was awarded $6.2 million for a phase 1/2A trial to test a treatment for ALS, which has no treatment or cure. The CIRM review summary said a "huge unmet need" existed. About 20,000 persons in the United States suffer from the affliction.

CIRM's press release did not identify the researchers involved in any of the awards.

The agency is on a push to support more clinical trials, which are the last and most expensive research prior to the possibility of winning federal approval for widespread use of a therapy.

Currently the agency is participating in 27 trials and is planning on adding 37 more in the next 40 months. The agency is expected to run out of funds for new awards in June 2020 and has no source of future financing.

The awards were previously approved behind closed doors by the agency's out-of-state reviewers, who do not disclose publicly their economic or professional interests. The agency's directors rarely overturn a positive decision by the reviewers.

All of the winners have links to two or more members of the 29-member CIRM governing board. Those members are not allowed to vote on applications where they have conflicts of interest.
About 90 percent of the funds awarded by the board since 2005 have gone to institutions that have ties to members of the board, past or present, according to calculations by the California Stem Cell Report.

(Editor's note: An earlier version of this item incorrectly said the total amount of awards was $37 million.)