California's $5.5 Billion Stem Cell Countdown: Two Pathways to the Ballot But Maybe One Not So Good

A $5.5 billion, proposed ballot measure today is anywhere from 58 percent to 63 percent on its way to possibly saving the California stem cell agency from financial extinction.

The agency, known formally as the California Institute for Regenerative Medicine (CIRM), is running out of  money -- the $3 billion that voters gave it in 2004. It needs the initiative to qualify for the ballot and voters to approve it or CIRM will begin closing its doors next fall. 

As of this evening, the multibillion dollar rescue proposal was nominally 63 percent along the road to qualifying. The measure had 394,237 valid signatures of registered voters, according to election officials. It needs 623,212. 

But the campaign says it really wants substantially more. A campaign official today said that its desired figure is 685,534, 110 percent of 623,212. Reaching 110 percent would avoid a "full check," which is a prolonged verification of each signature. That process might not be finished until after the deadline of June 24 for counties to get their figures to state election officials. The state schedules its verification on June 25.

A goal of reaching 685,534, however, means that the backers of the measure have only 58 percent of the signatures needed.

"If the result of the (now ongoing) random sample indicates that the number of valid signatures represents between 95% and 110% of the required number of signatures to qualify the initiative or referendum measure for the ballot, the secretary of state directs the county elections officials to verify every signature on the petition,"  according to the state's ballot initiative web site. 

"For an initiative measure, if the number of valid signatures is greater than 110 percent of the required number of signatures, the initiative measure will be eligible for the ballot," the state says. 

Critical to the process is the percentage of qualified signatures out of the 924,189 that were turned in May 12 by the measure's backers. That percentage is holding relatively steady at about 78 and has varied little as the count has progressed over several weeks. As long as validity rate stays above about 74 percent, the initiative will be sent to voters. 

"We’ve been very pleased with the validity rate so far and believe we have more than enough valid signatures to qualify," said Sarah Melbostad, a campaign spokeswoman.

The measure's validity rate is also closely aligned with five other initiatives that have qualified or nearly qualified in recent months for the fall ballot. Those initiatives have run or are running at rates that range from 73 percent to 78 percent. That is significant because the signatures are largely gathered by firms that specialize in that sort of work. 

It is reasonable to surmise that the firms, which compete for business, are more than likely to produce similar results. (For the other initiatives, see here and also here. The five initiatives mentioned had to labor under the restrictions of the Covid crisis.)

Officials have not yet verified signatures in some larger counties including San Diego, Orange and Riverside.  

Look for the latest figures tomorrow evening or early Friday on the California Stem Cell Report.

Advisory: Update on Ballot Count for California Stem Cell Measure

The state elections office reported this afternoon that later today it will post fresh figures on the qualification count for the $5.5 billion stem cell measure. A spokesman for the office said that it has not received any fresh figures from the counties since Monday afternoon. The California Stem Cell Report will carry an item on the latest figures when they come in.

Advisory on Ballot Initiative Count, Fresh Figures Not Forthcoming

State election officials have not posted a fresh count of signatures on the $5.5 billion stem cell proposal since Monday evening. We are inquiring about the matter and will post what we learn when we learn it.

California's $5.5 Billion Stem Cell Countdown: Sixty-two Percent Along the Road to the November Ballot

Backers of California's $5.5 billion stem cell research measure this afternoon received a hefty boost as their effort to place the proposal on the November ballot moved dramatically forward. 

The initiative is now 62 percent along the path to reaching voters. That figure emerged when Los Angeles County reported that it has counted 231,196 valid signatures of registered voters. The total number of valid signatures now stands 381,849. To be placed on the ballot requires 623,212. 

The percentage of valid signatures at 78 percent. If that qualification rate holds up, the proposal will be on the ballot since the campaign has submitted 924,189 "raw" signatures. Still outstanding are large counties such San Diego, Riverside and Orange.

The measure is aimed at saving the financial life of California's stem cell agency, which has nearly run through the $3 billion that voters provided it in 2004. With no infusion of additional funds, it will begin closing its doors next fall. 

Look for an updated count tomorrow evening or early Wednesday morning right here on the California Stem Cell Report. 

California's $5.5 Billion Stem Cell Countdown: Snail-like Progress Towards the Ballot

The final steps to qualify a $5.5 billion stem cell measure for the California ballot in November moved slowly this past week with the latest figures rising only marginally. 

State officials reported late yesterday that the number of valid signatures stood at 150,598, up slightly from 146,611 the previous day. 

In order to save the California stem cell agency from financial extinction, the initiative needs 623,212 valid signatures of registered voters.  Backers submitted 924,183 "raw" signatures.  

The signature disqualification rate yesterday stood at 22 percent, which is where it has been since May 22.

Large counties such as Los Angeles and San Diego have not yet reported on their verification efforts. The state deadline for counties to report their figures to the state is June 24. 

The stem cell agency is running out of cash and will begin closing its doors next fall unless it receives a substantial infusion of cash.

Look for an updated count Monday evening or early Tuesday morning right here on the California Stem Cell Report. 

California's Stem Cell Agency Backs More Covid Research in its $5 Million Round

The California stem cell agency this afternoon awarded a total of $450,000 to three researchers to fight Covid-19, which has infected nearly 91,000 persons in the Golden State and nearly 1.8 million nationally.

The action brought to nine the number of awards made in the agency's $5 million Covid round, which now has about $2.5 million remaining. 

Today's awards involved basic research, including a project aimed at a vaccine. Maria Millan, CEO of the agency, known formally as the California Institute for Regenerative Medicine (CIRM), said in a news release. 

"“These three projects highlight three very different approaches to combatting the acute devastating health manifestations of Covid-19 as well as the debilitating sequelae that impact the ability to recover from the acute illness. Through this Covid funding opportunity, CIRM is enabling researchers to re-direct work they have already done, often with CIRM support, to quickly develop new approaches to Covid-19."

The vaccine research award (application number DISC2COVID19- 11941) went to Albert Wong of Stanford. The $149,000 grant will be used to develop a cellular response to beef up immunity. A CIRM news release said Wong's "team will use the experience it gained using CIRM funds ($3 million) to (fight) glioblastoma, a deadly brain cancer, to advance a similar approach to produce an effective cellular immune response to combat Covid-19."

CIRM directors directed Wong to apply for a supplement of up to $100,000 to cover more work to assure that his work has application to under-served and under-researched communities that have been the most seriously affected by Covid.

Another Stanford scientist, Helen Blau, was awarded $149,996 (DISC2COVID19-11920) to help prevent atrophy of the diaphragms in Covid patients on mechanical ventilation. 

Jianhua Yu of the City of Hope was awarded $150,000 (DISC2COVID19-11947) for research to turn umbical cord blood cells into natural killer cells that would target Covid. The research would leverage existing studies that the City of Hope has developed to treat cancer. 

Summaries of the reviews of their applications and scores can be found here. That web page also includes summaries of the review of the 10 applications that were rejected. 

CIRM directors are expected to meet again within the next couple of weeks to fund more Covid research under its fast-track effort to combat the disease. The agency was created 15 years ago with $3 billion in state funding, but it is running out of money and will begin closing its doors this fall unless more funds are forthcoming. 

An American Vaccine Disaster 65 Years Ago: Lessons for Today

"Remember the Cutter Incident!" The phrase is not exactly the same sort of watch word as "Remember the Alamo." 

But it does have fresh resonance today. The Cutter Incident was a vaccine disaster in America 65 years ago. The case is the keystone of a column yesterday in the Los Angeles Times that said, "History tells us that speed kills."

The comment came from Arthur Caplan, a bioethicist at NYU Langone Medical Center and a co-author of an article this week in the Journal of the American Medical Association (JAMA) that sounded a warning in the rush for a Covid-19 vaccine. 

Caplan spoke about the matter with Michael Hiltzik, a business columnist at the Times and author of "Big Science." Hiltzik's column appeared one day before the California stem cell agency is set this afternoon to consider more applications in its $5 million Covid-19 funding round. (Vaccines also came up at last Friday's meeting of the agency's governing board.)

Hiltzik wrote, 

"If anything is known for sure about the scientific battle against the novel coronavirus, it’s that the quest for a vaccine has been unprecedentedly intense, with rapid development and speedy production the paramount goals."

The Cutter Incident, Hiltzik said, is example of the perils of speed. It involved errors in a polio vaccine produced in 1955 by Cutter Laboratories. An estimated 40,000 children contracted polio from the vaccine. About 200 were permanently paralyzed. Ten died. 

Hiltzik wrote, 

"The so-called Cutter incident was 'one of the worst biological disasters in American history, exploded the myth of the invulnerability of science and destroyed faith in the vaccine enterprise,' observed Paul Offit, a vaccine expert at the University of Pennsylvania whose 2005 book is the definitive account of the episode."

The polio vaccine was halted in its tracks during the summer of 1955. Hiltzik wrote, 

"Public confidence in the vaccine was shattered. 'The summer of 1955 came and went with few children getting their shots,' historian David Oshinsky, a co-author of the JAMA article, recounted in his 2005 history of the epidemic. Major outbreaks occurred in Boston and Chicago. 'It seemed like old times, with beaches and movie theaters once again deserted and people fleeing the cities to escape the evil germs.'"

Hiltzik concluded, 

"Clinical trials won’t be the final hurdle in bringing vaccines to the world. Managing the public’s expectations will be crucial, since no vaccine for anything is 100% effective. So will ensuring that the manufacturing process is airtight.

"With anti-vaxxers and conspiracy theorists already spreading wild stories about the coronavirus, it would be a shame to give them more ammunition.

"'In this world, where there’s so much vaccine hesitancy and doubt,' Caplan said, 'you better get it right, out of the box. You can’t afford a failure. Even knowing that we’re in desperate need of a vaccine and the deaths are happening, I worry that if we don’t do it right, it’s going to be worse.'"

California's $5.5 Billion Stem Cell Countdown: Still Creeping Up Little by Little

The drive to save the California stem cell agency with a $5.5 billion bond measure gathered modest momentum today in the signature count to put the proposal on the November ballot. 

The total number of valid or projected valid signatures late this afternoon advanced to 146,611, up slightly from 98,159 yesterday. The ballot initiative needs 623,212 valid signatures. Backers submitted 924,183 "raw" signatures.  

The signature disqualification rate stood at 21 percent, about the same as yesterday's 22 percent.

Large counties such as Los Angeles and San Diego have not yet reported on their verification efforts. The state deadline for counties to report their figures to the state is June 24 -- 20 business days away. 

The stem cell agency is running out of cash and will begin closing its doors next fall unless it receives a substantial infusion of cash.

Look for an updated count tomorrow evening or early Saturday morning right here on the California Stem Cell Report. 

Rogue Stem Cell Clinics and Covid: California Moves 'Sedately' on Regulation

The state of California and its top medical regulator remain mired in a go-slow posture on regulation of "snake oil" stem cell clinics that are currently riding the Covid crisis to peddle dubious treatments to desperate patients.

The marketing surge by the clinics has drawn increased attention nationally, including in prestigious scientific journals such as Cell Stem Cell whose usual fare deals with such things as "Stem Cell Hierarchy in Colorectal Cancer."

But one article published earlier this month was titled:

 "Preying on Public Fears and Anxieties in a Pandemic: Businesses Selling Unproven and Unlicensed 'Stem Cell Treatments' for COVID-19." 

The quotes around "stem cell treatments" are deliberate. No guarantee exists that the treatments actually include stem cells. 

Leigh Turner, an associate professor of bioethics at the University of Minnesota, authored the piece. He said,

"In the midst of a global public health emergency, some businesses are taking advantage of widespread fears by marketing purported stem cell treatments for Covid-19. 

"Such businesses target prospective clients with misleading claims, expose patients to potentially risky stem cell-based products, and undermine efforts to develop evidence-based treatments for Covid-19."

Nearly two years ago, California's State Medical Board said it was going to tackle the problem of the rogue clinics. But to this date little has occurred. State legislation to develop regulations was also sidelined.

The California Stem Cell Report queried the medical board earlier this month concerning the status of its effort and whether regulations had been drafted and when a draft would be ready. In response, the board last week released the following statement:

"The board is continuing to work toward the goal of providing recommendations on stem cell and regenerative therapies and developing some guidelines that California physicians and patients can follow that will include a sample informed consent document and educational materials for the public to present to the board for review and final approval. 

"In the meantime, pursuant to Business and Professions Code section 684, healthcare practitioners providing stem cell therapy not approved by the Food and Drug Administration (FDA) and who have not obtained approval or clearance for an investigational new drug or device exemption from the FDA for the use of human cells, tissues, or cellular or tissue-based products, must provide notice to their patients indicating the lack of FDA approval, and encouraging their patients to consult with their primary care physician before treatment. Individuals concerned about a physician’s stem cell practice should file a complaint with the Board for review and appropriate action."

The issues with the clinics has drawn attention in the Los Angeles Times from Pulitzer Prize winning columnist Michael Hiltzik  who wrote recently,

"The proliferation of stem cell clinics selling untested and unlicensed therapies has been a public health crisis for years. The Covid-19 pandemic will only deepen the crisis as clinics add the coronavirus to their menu of treatment claims."

The California stem cell agency has also pushed for tighter regulation and partnered to help pass the law for disclosure notices concerning lack of FDA approval. For patients considering stem cell treatments, the agency has posted as rundown on issues concerning the treatments or clinical trials. 

(After this item was posted, the agency posted a blog item dealing with Parkinson's. In addition to information on the search for a cure or treatment, the item carried the following advice for persons seeking some sort of unregulated treatment. Here is what the agency had to say in a Q&A format,

"If you go online you can find lots of stem cells clinics, all over the US, that claim they can use stem cells to help people with Parkinson’s. Should I go to them?

("In a word, no! These clinics offer a wide variety of therapies using different kinds of cells or tissues (including the patient’s own blood or fat cells) but they have one thing in common; none of these therapies have been tested in a clinical trial to show they are even safe, let alone effective. These clinics also charge thousands, sometimes tens of thousands of dollars these therapies, and because it’s not covered by insurance this all comes out of the patient’s pocket.

("These predatory clinics are peddling hope, but are unable to back it up with any proof it will work. They frequently have slick, well-designed websites, and  'testimonials' from satisfied customers. But if they really had a treatment for Parkinson’s they wouldn’t be running clinics out of shopping malls, they’d be operating huge medical centers because the worldwide need for an effective therapy is so great.

("Here’s a link to the page on our website that can help you decide if a clinical trial or “therapy” is right for you.")

California's $5.5 Billion Stem Cell Countdown: No Big Jump Today

New totals tonight show slight progress in the drive to rescue the financially strapped California stem cell agency, adding only 6,717 signatures to qualify a $5.5 billion measure for the November ballot.   

It needs 623,212 valid signatures. Backers submitted 924,183 "raw" signatures. State election officials reported late today that the proposal now has 98,159 valid or projected valid signatures of registered voters. 

The signature disqualification rate remains at about 22 percent. 

Large counties such as Los Angeles and San Diego have not yet reported on their verification efforts. The state deadline for counties to report their figures to the state is June 24 -- 20 business days away. 

The stem cell agency is running out of cash and will begin closing its doors next fall unless it receives a substantial infusion of cash.

Look for an updated count tomorrow evening or early Friday morning right here on the California Stem Cell Report. 

Correction

An item today dealing with the count of signatures for the proposed, $5.5 billion California stem cell initiative incorrectly stated that the disqualification rate was 78 percent. It is 22 percent. The percentage of signatures that have been qualified stands at 78 percent.

California's $5.5 Billion Stem Cell Countdown: Nearly 15 Percent Along the Path Towards a Rescue

The latest figures on the effort to save the financially strapped California stem cell agency show that it is 14.7 percent along the way to placing a $5.5 billion rescue measure on the November ballot. 

State election officials reported late yesterday that the proposal now has  91,442 valid or projected valid signatures of registered voters. It needs 623,212 valid signatures and has submitted 924,183 "raw" signatures. 

The signature disqualification rate stands at about 22 percent. 

Large counties such as Los Angeles and San Diego have not yet reported on their verification efforts. The state deadline for counties to report their figures to the state is June 24 -- 20 business days away. 

The stem cell agency is running out of cash and will begin closing its doors next fall unless it receives a substantial infusion of cash.

(Editor's note: An earlier version of this item incorrectly stated that the disqualification rate was 78 percent. It is 22 percent. The percentage of signatures that have been qualified stands at 78 percent.)

Correction

An item on May 23, 2020, dealing with the signature count on the proposed $5.5 billion California stem cell initiative incorrectly said that Mono County had the least number of valid signatures (4). The correct county is Modoc. 

California Stem Cell Agency Backs Away from Covid Vaccine Plan; Concerns Raised About Mission Creep

Directors of the $3 billion California stem cell agency have rejected a Covid-19 proposal to extend the agency's reach beyond stem cells, expressing concerns about mission creep, public relations and dilution of cash for other important research.

The case in point last Friday was "emergency" consideration of a plan to fund Covid-19 vaccine development as part of a $5 million special round of funding. The federal government has already allocated nearly $1 billion for vaccine development. More than 100 vaccine projects are underway globally.

Shlomo Melmed of Cedars-Sinai in Los Angeles was one of the directors of the California Institute for Regenerative Medicine (CIRM) who opposed the proposal. He described the likelihood of CIRM being successful in helping to develop a vaccine was "pie in the sky."  He said, 

"We should be realistic about how that may look good on our website...but the reality is that this whole project (is) not going to have major, short-term impact barring some wonderful serendipity.

"And, and to come back to the original trust of the voters of California in CIRM, I really do think that we are deviating from our mission and ought not dilute our impact, which is so necessary and so vital."

Friday's discussion was triggered by a proposal to declare vaccine development a "vital research opportunity(VRO)" and make it eligible for state funding.

CIRM directors have approved a VRO only twice in the 15 years that the agency has existed. One was for gene therapy research. Another involved blood plasma, a possible Covid-19 treatment.

The VRO loophole is contained in the ballot initiative that created the agency in 2004, but was little noticed at the time. The same provision exists in the proposed initiative to refinance the financially strapped agency with $5.5 billion more. The initiative also contains language that would expand the scope of CIRM and, in some cases, require it to delve into major new areas. The initiative has not yet qualified for the November ballot.

Director Jeff Sheehy, an HIV/AIDs patient advocate member of the board, said,

"As an agency we're taking a much broader and much more sophisticated view of what our mission is. It's morphed. If we were being true to our original mission, we would only be doing embryonic stem cell research....(T)hat's what we the voters asked us to do in 2004."

Director Steve Juelsgaard, a former top executive at Genentech, said that the amount of funding that CIRM could provide was only "a drop in the bucket," given its financial condition. He added, "I think we're just getting way off course here."

Director Keith Yamamoto, vice chancellor of science policy at UC San Francisco, said the search for Covid treatments is "urgent and important." But he added that the agency would be "well advised to kind of stay in our lane" because of the important work that it was intended to do. He reminded directors of the hundreds of millions of dollars already allocated by other entities for vaccine work.

During an earlier part of the discussion, Jonathan Thomas, chairman of the CIRM board, said that the vaccine proposal had been the subject of robust internal discussion at the agency. He said that the idea behind it was to assist in the Covid crisis: "going a little bit above and beyond to try to help."

Thomas gave Sheehy, chairman of the board's science committee, a "shout-out" for suggesting the idea of discussing the concept of a VRO in connection with a vaccine. During his comments, Sheehy suggested that if the agency wanted to make an impact on vaccine development it would need to remove "the stem cell handcuffs." But he added he was "agnostic" on the matter.

Over the weekend, Sheehy, who has raised questions about the $5.5 billion initiative, responded at length to a follow-up inquiry by the California Stem Cell Report. He said:

"To sum up, I'm not really sure what the board is trying to accomplish with its Covid program.  I'm not sure that the board has a clear, coherent view of the scope of CIRM's research.  And I am not sure the board has a clear idea of what the scientific mission of CIRM should be in the event that new funding comes from the voters."

The full text of his remarks can be found here.

Ultimately, the CIRM board said it would vigorously support research platforms that have stem cell elements that could help in vaccine development. That position, however, was not as expansive as originally proposed. It does not require a VRO and falls within the agency's normal scope.

Directors also approved raising the funding cap on some elements within the current $5 million Covid-19 round. They are scheduled to meet again this Friday at 1 p.m. to consider more Covid applications. The online sessions are open to the public, members of which can comment on all matters. Directions are on the agenda.

Here is a link to CIRM's news release on the Friday meeting.

Text of Sheehy's comments re Covid-19, CIRM, Its Mission and Other Matters

The discussion at the May 23, 2020, meeting of the governing board of the California stem cell agency ranged from Covid-19 and the agency's strapped financial condition to its mission and priorities. One of the directors, Jeff Sheehy, elaborated on the matters and more when he responded to an inquiry following the meeting from the California Stem Cell Report. 

The California Stem Cell Report has a policy of running verbatim comments from stem cell agency board members and other interested parties.  If other readers would like to submit their comments on this subject or other stem cell matters, please direct them to djensen@californiastemcellreport.com.

Below is the verbatim text of Sheehy's comments.  
"First, I felt a great deal of uncertainty around CIRM proposing a COVID program.  Having worked towards stem cell therapies for HIV since I came onto the board in 2004, I felt some skepticism around stem cell approaches targeting infectious diseases (ID).  Infectious disease has not been a particularly major focus of regenerative medicine efforts and many of the approaches we have tried have struggled.  I think there could be a role, but we have a long way to go.  

"I thought, in general, a focus on ID is a bit overdue, but I was very concerned about taking funds from sickle cell, where I think there is a strong case for believing we are on track for a cure using stem cell technology, and there are well constructed clinical trials such as the one we just funded.  

"Given that CIRM is using its last of its funding, I was also concerned about the funding needed.  One of the most telling moments in Friday's meeting was the descriptions by (CIRM Director) Dr. (Keith) Yamamoto (vice chancellor for science policy at UCSF)  of the ready and ample availability of Covid funding from the NIH and other federal sources, not just in new funds but also the ability to repurpose existing grants towards Covid.

"Btw, I am not alone in my skepticism around stem cells for Covid-19.  I offer first the tweet from Dr. Sean Morrison, former head of ISSCR on March 29:

"'There is no stem cell therapy for Covid19. We would not expect stem cells to have any therapeutic value for people with Covid19.
@ISSCR'"

"Then a couple of weeks ago, Dr. Paul Knoepfler on his blog, the NICHE, offered this:
"'The idea of testing stem cells for Covid-19 may be music to the ears of some folks as an opportunity, but to me from the beginning it sounded mostly like a spaghetti on the wall road to trouble.
"'There is buzz out there that some kind of stem cells or other cells will help with COVID-19. The reality is that that’s probably not going to happen.
"'Even so, a whole range of people and firms are somewhat exaggerating and in a few cases outright hyping the odds of success. That is harming patients and the cell medicine field.'"

"I offer these two comments in support of my doubts.  But, I also feel the urgency surrounding Covid.  This is my second pandemic, having lived through HIV/AIDS and lost and continue to lose countless people that I care deeply about and living with the disease myself, having endured enormous stigma and outright hatred along with indifference from a president, seeing marginalized communities suffering death and disease disproportionately and indifference because people don't recognize the humanity of the populations most impacted, feeling triggered and re-traumatized by this second pandemic and the enormous stupidity from all points in trying to make a disease fit their self described perceptions of reality in the face of what is real and undeniable.

"I voted to support the COVID program despite my doubts due to urgency, but we know what tools work against infectious diseases -- antivirals and vaccines, and I became an enthusiastic and vocal supporter of the vital research opportunity for convalescent plasma.  Convalescent plasma is about as far removed from CIRM as a project could be--a technology at least 100 years old with zero barriers to receiving funding from any source.  But, in the absence of a vaccine or antivirals, it represented to me the best and most rational chance to make a difference in patients now. Plus, Zaia et al at City of Hope have the cell handling and manufacturing ability to address some of the factors limiting use and evaluation of convalescent plasma, e.g. potency and consistency of product to name a couple.

"After having participated in reviews, our two clinical projects are the convalescent plasma one and an ongoing $14 million trial for mesenchymal stem cells for ARDS, the lung complication suffered by critical COVID patients necessitating ventilator support.  That UCSF/UC Davis trial is already treating COVID patients at SF General and in Houston to name a couple of sites, and it was supportable because we were availing more patients of access to that therapy like we are doing with convalescent plasma. But both approaches are well supported outside of CIRM and the $750K we gave each project.  

"The $150K Discovery projects we funded are interesting, but they are very early in development and given the time and money it takes to move from discovery into translation into clinical trials, these antiviral products could likely be superseded by the host of antiviral products in trials around the world.
In that context, we did see a couple of interesting vaccine proposals that were very far afield and did not score in the fundable range.  I had a feeling -- having looked at the vaccine trials underway (and I'm not an expert btw)  -- that they seemed very much based in the most part on proven tech that a far-out, left field approach might be useful to explore.  HIV has been stubbornly resistant to vaccine development, so I'm very cautious about that field.  I was heartened to hear (CIRM Director) Dr. (Kristiina) Vuori from Sanford Burnham opine that she thought that current vaccine approaches were, to paraphrase, following usual pathways, and I thought I heard a suggestion that opening up CIRM to vaccine work might elicit something novel.

"However, the board thought otherwise and I defer to their wisdom.

"I would note that I was intrigued by the discussion around CIRM's mission.  As Dr. (Gil) Sambrano (CIRM vice president for portfolio development)  noted, stem cells are ubiquitous in the body, and I took that to mean that one could either be elastic or inelastic in accepting products.  A vaccine, for instance, would almost necessarily  impact T memory stem cells, which are a crucial part of the adaptive immune response to disease (and a vaccine).  We have begun to recognize the limits of stem cell centric therapies through our VRO for gene therapy.

"I then asked myself, does CIRM hews to its original mission per Prop. 71?  I looked up the original funding mission for the Grants Working Group :

"'(C) In order to ensure that institute funding does not duplicate or supplant existing funding, a high priority shall be placed on funding pluripotent stem cell and progenitor cell research that cannot, or is unlikely to, receive timely or sufficient federal funding, unencumbered by limitations that would impede the research. In this regard, other research categories funded by the National Institutes of Health shall not be funded by the institute....Notwithstanding subparagraph (C), other scientific and medical research and technologies may be funded by the institute if at least two-thirds of a quorum of the members of the Scientific and Medical Research Funding Working Group recommend to the ICOC that such a research proposal is a vital research opportunity.'

"With the federal ban ended, all of CIRM's research is really a vital research opportunity by default at this point.  Per COVID and ample federal funding, any funding in that arena is about as far afield as one could get.

"Even with fetal tissue research, which, btw, has fallen off the radar in the media and with some in Congress and the Administration, I can guarantee no one is talking about banning that.  You simple cannot develop vaccines or antivirals without mice with humanized immune systems created by using fetal tissue.

"To sum up, I'm not really sure what the board is trying to accomplish with its COVID program.  I'm not sure that the board has a clear, coherent view of the scope of CIRM's research.  And I am not sure the board has a clear idea of what the scientific mission of CIRM should be in the event that new funding comes from the voters.

"I would note as an aside that CIRM has never submitted its scientific program, including all grants made and their impact, to a rigorous, independent scientific review.  

"In short, I don't think we really know at CIRM where we've been and where we want to go.  We have anecdotes...."

(Sheehy’s own ellipses in the last paragraph)

The $5.5 Billion California Stem Cell Countdown: One-Thirteenth Along the Way

Supporters of a $5.5 billion, proposed ballot initiative this morning are 13.3 percent along the way to qualifying the measure for the November ballot and possibly saving the California stem cell agency from financial extinction. 

The proposal now has 82,723 valid or projected valid signatures of registered voters, which state election officials say is the key category. The measure needs 623,212 in signatures in that category to qualify.

The total of "raw," unverified signatures statewide is 924,183.  At this point, only 77.57 percent of the signatures are valid. But major counties such Los Angeles and San Diego have not yet completed their validation process. 

Twenty-two of the state's 58 counties have reported their counts. The  highest number of valid signatures has come from Sacramento with 26,761. The lowest number is from Modoc with four. 

The stem cell agency was created by California voters in 2004 when they approved a ballot initiative that also provided it with $3 billion of borrowed money but no other funding. The agency is running out of cash and will begin closing its doors next fall unless more substantial funding is forthcoming. 

Look for an update on the count Tuesday evening or early Wednesday right here on the California Stem Cell Report. 

Editor's note: An earlier version of this item incorrectly stated that Mono County had the lowest number of valid signatures. It was actually Modoc.

$5.5 Billion California Stem Cell Countdown: Total Raw Signatures Now in With 924,213

One key step is now complete in the effort to qualify a $5.5 billion stem cell initiative for the November ballot in California and save its stem cell research program from running out of money.

The "raw" count of signature is finished with a total of 924,213, according to state election officials. That is almost exactly the same as the 925,000 figure that  campaign backers said they had submitted weeks ago.

The proposed ballot measure needs 623,212 valid signatures of registered voters to qualify. The count of valid or projected valid signatures stands at 75,484 as of late yesterday afternoon. The rate of rejected signatures is running at 22 percent. 

A number of populous counties have yet to report on their validation figures including Los Angeles, with 298,147 raw signatures, and San Diego, with 99,899. 

Counties have until June 24 to complete their count.

Look for an update later today or early tomorrow on the count right here on the California Stem Cell Report. 

Parkinson's and the Unseen Offspring of California's Stem Cell Agency

On the surface, Aspen Neurosciences, Inc., a tiny firm in La Jolla, Ca., does not seem to have much of a connection to California's $3 billion stem cell agency.

It is not alone. A good chunk of the collateral benefits of agency is all but invisible. While the agency's web site is chock-a-block with videos, statistics, testimonials and pie charts, not everything significant rises to the top. Most of the information can be classified as high profile. Aspen, however, is barely a footnote for the agency. 

 

Yet the firm, over the last six months, has received a $76 million validation from the private sector, more than any business has received from the California Institute for Regenerative Medicine (CIRM), as the stem cell agency is formally known. Viacyte, Inc., of San Diego, is No. 1 among the business beneficiaries of CIRM, collecting $72.3 million.  

The lack of visibility of some benefits from CIRM may not seem all that important to some. But when plans are afoot to ask California voters for $5.5 billion to forestall the financial demise of the stem cell agency, voter perceptions of the agency become paramount. 

Aspen's story has elements that could resonate with many voters. It involves a well-regarded woman scientist, pursuit of millions of dollars, failures and success and patients with stories of their terrible times with a dreadful affliction.  

The tale begins within walking distance of what is now Aspen's headquarters in La Jolla. Aspen is located just down the street from the Scripps Research Institute, where Jeanne Loring was director of the Center for Regenerative Medicine and who also earlier co-founded a firm that morphed into Viacyte. 

While she was at Scripps, she embarked on research aimed at Parkinson's Disease, stimulated and aided by a patient advocacy group called Summit for Stem Cell. It is an energetic group and once collected $120,000 in a fundraising effort that took nine Parkinson's patients on a hike at Machu Pichu in Peru. 

Summit members also trekked to meetings of the governing board of the stem cell agency to build a case -- sometimes with tears and trembling voices -- for more money and more speed on Parkinson's research. (See here also.)

Loring filed for grants for her research as well. She ultimately collected nearly $22 million from CIRM, but the agency did not follow through on additional applications. Loring cast about for other possibilities, and she and Andres Bratt-Leal, also of Scripps and Summit for Stem Cell, co-founded Aspen Neurosciences. 

Loring strongly supports renewing the financially endangered stem cell agency with $5.5 billion. She has told the California Stem Cell Report, 

"All of my CIRM awards contributed to (Aspen). We used those funds to develop our genomics tools and databases and to develop improved methods for handling the cells." 

Loring is now Aspen's chief scientific officer. Bratt-LealHer is vice president of research and development. Their ongoing work now at Aspen is aimed at creating the "first autologous neuron replacement therapy to treat Parkinson's disease," Aspen said in a news release. 

Aspen expects to use its private financing to launch a phase one trial for the therapy, which begins with a patient's own skin cells. They are reprogrammed to become pluripotent and converted into neurons. The neurons are transplanted into a patient to replace the ones that Parkinson's has destroyed. 

Whether Aspen can produce a commercial treatment that is widely available remains to be seen. Whether CIRM's supporters can produce a $5.5 billion victory in November remains to be seen as well. It is likely to depend entirely on how voters see the benefits of CIRM's work over the last 15 years -- invisible and otherwise. 

(Note to readers: If you know of other unseen offspring of the stem cell agency -- good or bad -- please send a note to djensen@californiastemcellreport.com. Your emails will be held in confidence. Thank you.)

(After this item was posted, the agency posted a blog item dealing with Parkinson's. In addition to information on the search for a cure or treatment, the item carried the following advice for persons seeking some sort of unregulated treatment. Here is what the agency had to say in a Q&A format,

"If you go online you can find lots of stem cells clinics, all over the US, that claim they can use stem cells to help people with Parkinson’s. Should I go to them?

("In a word, no! These clinics offer a wide variety of therapies using different kinds of cells or tissues (including the patient’s own blood or fat cells) but they have one thing in common; none of these therapies have been tested in a clinical trial to show they are even safe, let alone effective. These clinics also charge thousands, sometimes tens of thousands of dollars these therapies, and because it’s not covered by insurance this all comes out of the patient’s pocket.

("These predatory clinics are peddling hope, but are unable to back it up with any proof it will work. They frequently have slick, well-designed websites, and  'testimonials' from satisfied customers. But if they really had a treatment for Parkinson’s they wouldn’t be running clinics out of shopping malls, they’d be operating huge medical centers because the worldwide need for an effective therapy is so great.

("Here’s a link to the page on our website that can help you decide if a clinical trial or “therapy” is right for you.")

"Emergency" Meeting Tomorrow Morning; California Stem Cell Agency Wants to Expand Scope of its Covid Awards

With only 24 hours notice, California's $3 billion stem cell agency today called a meeting for tomorrow to significantly expand the scope of its Covid-19 round to include vaccine development and to permit "supplemental funding."

If the changes are approved tomorrow, scientists could file their applications up until next Tuesday to be considered in the next application review session, probably within 10 days to two weeks.  They would have an opportunity to submit another application about two weeks later if all the Covid funds have not been awarded.

Agency directors allotted $5 million for the Covid round. They have awarded about $2 million.

Here is what the agency listed this afternoon to describe the proposed changes:

• "Increase scope of early stage opportunities (DISC and TRAN) to include vaccine development studies as a potential vital research opportunity.

 • "Include opportunity for studies on novel ideas and biological mechanisms by opening the DISC1 program announcement.

 • "Allow supplemental funding for active COVID-19 clinical trial awards to expand their efforts.

 • "Include requirement for outreach to underserved populations in all clinical trial proposals 

• "Increase award limit on DISC2 projects and adjust language on 6-month deliverables."

The DISC term generally refers to basic research. The TRAN term refers to translational research.

The full concept plan can be found here.

Normally the agency would not fund vaccine research because it does not involve stem cells. Under the proposal for tomorrow, it would be officially deemed a "vital research opportunity," a loophole in state stem research cell law that allows such funding.  The agency has already invoked the provision in a handful of other instances. 

The supplemental funding change would permit researchers who have already received funding under the Covid-19 round to ask the agency for more cash for their applications. The change would allocate up to $1 million for such awards. 

Under normal conditions the agency's governing board could not meet legally with only 24 hours public notice. The normal legal requirement is 10 days. However, the board is invoking emergency provisions to lift the 10-day notice. 

The 11 a.m. meeting will be available for public participation by the public via phone connections and the Internet. Details are on the agenda.

$5.5 Billion California Stem Cell Countdown: No Significant Changes Today

California election officials this afternoon reported no significant changes to the count to determine whether a $5.5 billion stem cell ballot initiative will qualify for the November ballot. 

Here are the results as of 4:55 p.m. PDT today:

Raw signature count -- Unchanged at 917,722, San Mateo County still not reporting

Valid signatures -- 5,202, up from 3,803 yesterday

Disqualification rate of signatures -- Nearly 23 percent, down from 24 percent yesterday

The proposal needs 623,212 valid signatures of registered voters to qualify for the ballot. The proposal is aimed at refinancing the California stem cell agency, which is running out of money. 

Sickle Cell Trial, Backed by $2 Million from State of California, Will be Based in Los Gatos

More details emerged today about a $2 million sickle cell award and clinical trial involving the California stem cell agency, including the location of the trial in Los Gatos and the involvement of a Stanford University researcher. 

The award was approved last Friday by the agency. It went to a Canadian firm, ExCellThera, Inc.,  which is partially matching the state award with $857,143. With the addition of ExCell, the stem agency is now involved in 63 clinical trials. 

                                                                                                                  

The company said yesterday that the Phase 1 trial, which involves safety, will use its ECT-001 product, and will be conducted at the Lucile Packard Children's Hospital. It will be led by Sandeep Soni of Stanford University. The safety phase trial is aimed at children and young adults.

Guy Sauvageau, CEO and founder of ExCellThera, said in a news release.

"The current standard of care for severe sickle cell disease is a blood stem cell transplant, which is only available to patients who have a matched donor. 

“However, the unique properties of ECT-001 cell therapy enable patients without a matched donor to receive treatment, with a greatly reduced risk of post-transplant complications. This study introduces a new indication into the ongoing evaluation of our ECT-001 technology, which we hope will make this life-saving therapy available to a far greater number of  severely ill patients."

The trial has not yet been posted on clinicaltrials.gov. It is not clear whether recruitment of patients is  underway.  Here is a link to a summary of  the stem cell agency's review of ExcellThera's application (CLIN2SCD-11674).

$5.5 Billion California Stem Cell Countdown: Proposal Inches Towards Ballot

Efforts to place a $5.5 billion stem cell measure on California's November ballot edged slowly ahead yesterday with a slight increase in the number of valid voter signatures, which are critical to qualifying the proposal for presentation to voters. 

The proposal, which is aimed at saving the financial life of the state stem cell agency, needs 623,212 valid signatures of registered voters to make the ballot. Tuesday's total was 71,873, up from 45,407 the previous day. 

The total of unverified, raw signatures was unchanged at 917,722. Only one county, San Mateo, has not reported its raw totals.

The overall rejection rate for signatures stood at 23 percent, also nearly unchanged. Disqualification rates are higher in the more populous counties so far. If campaign has actually submitted 925,000 signatures, as it has claimed, it will need a disqualification rate statewide of no more than 32.6 percent to reach the required number. 

Without an infusion of substantial cash, the stem cell agency, which is running out of money, will begin closing its doors next fall.

County officials have until June 24 to complete their validation of signatures and submit the figures to the state. 

Look for an update on the signature count this evening or early tomorrow right here on the California Stem Cell Report. 

The $5.5 Billion Stem Cell Count: A Bright Spot and a Not-So-Bright Spot

The effort to save the $3 billion California stem cell agency from financial extinction received some bad news and some good news late today, both of which could be expected. 

At stake is whether a $5.5 billion bond measure will be placed on the November ballot. The funds would be used to refinance the agency, which is running out of money and will begin closing its doors beginning next fall without an infusion of cash.

Qualifying the measure for the ballot requires 623,212 valid signatures of registered voters.  

Late today, the updated count of "raw," unvalidated signatures rose to 917,222 from 824,777 last Friday. The campaign for the ballot initiative has said it submitted 925,000 signatures to county election officials. Only one county, San Mateo, has not yet completed its raw count. 

The bad news is that the interim percentage of invalid signatures now stands at 24 percent, up from 18 percent. It is common to see large percentages of signatures disqualified during the verification process, sometimes as high as 50 percent. 

Today's rate rose because Sacramento reported that it disqualified 25.2 percent of the 35,792 raw signatures turned in by the campaign, The statewide rate will change as more counties report their validation numbers.

If campaign has actually submitted 925,000 signatures, it will need a disqualification rate statewide of no more than 32.6 percent to reach the required number. 

County officials have until June 24 to complete their validation of signatures and submit the figures to the state. 

Look for an update on the signature count Tuesday evening or early Wednesday right here on the California Stem Cell Report. 

The $5.5 Billion Stem Cell Count: Stagnant at 824,777 'Raw' Signatures

The signature count to determine whether a $5.5 billion stem cell research initiative will come before California voters next fall remains unchanged this morning at 824,777 "raw" signatures. 

Also stagnant is the number of valid signatures -- 12,440. 

The proposal needs 623,212 valid signatures to qualify, but many of the "raw" signatures are likely to be disqualified. Only those from registered voters are accepted. The current disqualification rate is running at 18 percent, according to state election officials.  That rate involves smaller counties and could change. Also changing will be the number of valid signatures as county officials wade through the petitions. 

The raw total this morning is up from 760,601 earlier in the week. The campaign says it has submitted 925,000 "raw" signatures to county election officials. 

Known officially as the California Institute for Regenerative Medicine (CIRM), the state agency is running out of the $3 billion provided by voters in 2004. It will begin shuttering its doors late this year without a financial infusion. 

Look for an update on the signature count Monday evening right here on the California Stem Cell Report.