$10 Million More for Viacyte for a Virtual Stem Cell Cure for Type 1 Diabetes

The San Diego stem cell company that has snagged $72 million from the state of California  announced this week another $10 million infusion from the firm that created the rain gear fabric known as Gore-Tex.

The San Diego firm is Viacyte, Inc., a privately held company that has received more than any other company from the $3 billion California stem cell agency. In 2017, Viacyte became involved with W.L. Gore & Associates, Inc., a materials science company that deals with drug delivery technologies.

Gore is also the enterprise whose Gore-Tex product has shaped the current outerwear industry.

On Tuesday, Viacyte announced the $10 million investment by Gore. The funds are coming in the form of a convertible promissory note.

Paul Laikind, president of Viacyte, said this week that the collaboration with Gore "has been exceptionally productive." He said the use of Gore membranes "has proven invaluable for improving the performance of the PEC-Encap product candidate in non-clinical studies that have been shown to be reflective of the human experience."

He said the additional support "should allow us to resume clinical testing of PEC-Encap as early as the first half of 2019."

California's $72 Million Diabetes/Stem Cell Bet: A New Partner from Massachusetts

CRISPR Therapeutics is the latest firm to become involved in a California-
backed stem cell research effort. It uses gene-editing techniques to devise cures.

The California stem cell agency has invested $72 million in a San Diego firm that is pursuing a a functional cure for diabetes and which announced this week it was moving to dodge a major obstacle facing its potential therapy. 

The firm is Viacyte, Inc., a privately held enterprise that has received more funding by far than any other state-backed stem cell firm. It announced on Monday that it had hooked up with publicly traded CRISPR Therapeutics, Inc., of Cambridge, Mass., to collaborate on a gene-editing treatment that would evade the body's immune response to earlier Viacyte therapies.

In a Q&A with UC Davis researcher Paul Knoepfler, Paul Laikind, CEO of Viacyte, said,

"The advantage of an islet cell replacement therapy that has been gene-edited for immune evasion is simply that patients would not need to take immunosuppressive drugs, which can have side effects. Our main mission is to improve the lives of patients with insulin-requiring diabetes by delivering transformative new therapeutic options. The work we are doing on PEC-Direct, PEC-Encap, and now an immune-evasive approach, known as PEC-QT, are all a part of that mission."

The treatment is principally aimed at type 1 diabetes, which afflicts 1.25 million persons in this country, the stem cell agency said in a piece on its blog about the new collaboration.

Viacyte was one of the earliest firms to receive cash from the agency, officially known as the California Institute for Regenerative Medicine (CIRM). The awards began with only $48,950 in 2008 when Viacyte was known as Novocell.

The arrangement with CRISPR will provide more funding for Viacyte, $15 million from its new partner and possibly another $10 million in the form of a convertible promissory note.

CRISPR's stock closed at $48.99 yesterday, down from $55.36 last Friday. Its 52-week high was $73.90 and its 52-week low $16.16.  The firm announced on Wednesday that it was planning to sell $200 million in common stock.  (Here is a detailed presentation on the firm's strategy and research. Here is a link to one analysis of the firm as an investment.)

In the piece on Knoepfler's blog, Laikind also provided an update on Viacyte's other related research. He said,

"(T)he PEC-Encap (also known as VC-01) product candidate has a bright future! We remain enthusiastic regarding the prospects of PEC-Encap, and are actively working on it. In June, two-year data from ViaCyte’s STEP ONE clinical trial were presented at ADA 2018. Although consistent and robust engraftment has been limited in this study to date, results showed that when engraftment does occur, viable mature insulin-expressing endocrine islet cells can be formed. In some cases, insulin-expressing cells have persisted for up to two years after implantation, the longest time point investigated in the study.

"Building on insights gained during the STEP ONE study, ViaCyte is working with W.L. Gore & Associates, one of the world’s top materials science companies with expertise in medical device development and drug delivery technologies, to modify the Encaptra Cell Delivery System and improve the potential for long-term engraftment. This work has yielded positive results in non-clinical models that, based on clinical experience, have been selected to reflect the response in patients. If the progress continues as expected, we plan to resume STEP ONE trial enrollment in 2019.

"As for PEC-Direct (also known as VC-02), the Phase 1/2 clinical evaluation of that product candidate is also continuing. We are now evaluating patients in the second cohort of the trial. As you know, PEC-Direct has the potential to help the patients with type 1 diabetes with the greatest need."

California's Stem Cell Agency Says It Has Saved Lives and Is Building a Foundation to Save More

Evie Vaccaro, whose life was saved with research backed by the stem cell agency

California's $3 billion stem cell agency took its message this week to the op-ed pages of the
San Francisco Chronicle, declaring that the agency's efforts have saved lives and created a world class network of clinics to develop therapies for all.

Maria Millan, CEO of the agency, and Jonathan Thomas, chairman of its board, said in an op-ed article,

"Have we achieved all we wanted to? Of course not. The first decade of CIRM’s life was laying the groundwork, developing the knowledge and expertise, and refining processes so that we can truly accelerate progress. As a leader in this burgeoning field of regenerative medicine, CIRM needs to continue its mission of accelerating stem cell treatments to patients with unmet medical needs."

The Millan-Thomas article came in the wake of a Chronicle editorial that said the agency, formally known as the California Institute for Regenerative Medicine (CIRM), did not merit additional funding based on its results to date. The Chronicle also carried a lengthy news piece that concluded the agency has not met the expectations of voters who created it through a ballot initiative in 2004. 

The agency expects to run out of cash for new awards at the end of next year. Its hope for survival lie with a yet-to-written bond measure that would appear on the November 2020 ballot. 

Millan and Thomas said the agency is now backing 49 clinical trials and has a host of accomplishments. They said, 

"Today the therapies resulting from the institute’s work are not just changing lives — they are already saving lives.

"Lives like Evie Vaccaro, who is alive today because of a treatment CIRM is funding. Vaccaro was born with SCID, also known as “bubble baby disease,” an immune disorder that often kills babies in their first two years. Vaccaro, now 6, and dozens of other babies were given stem cell treatments thanks to the institute. All are showing improvement; some are now several years past treatment and considered cured. 

"An accident left Jake Javier from Danville paralyzed from the chest down on the eve of his high school graduation. Javier was treated in a CIRM-funded clinical trial. Today, he has regained the use of his arms and hands, is driving a car and is a sophomore at Cal Poly San Luis Obispo. Five other patients treated at the same time as Javier all have experienced improvements meaning that instead of needing round-the-clock care, they can lead independent lives."

California's $50 Million Alpha Network: Managing the Complexity of Stem Cell Clinical Trials

Clinical Leader, an online publication dealing with clinical trials, yesterday carried a piece exploring the $50 million Alpha Clinic program initiated by the California stem cell agency.

Written by Geoff Lomax, a senior officer at the agency for its strategic initiatives, the article discussed the complexity of stem cell clinical trials and the use of a network of high-powered medical centers to support the novel research and treatment, particularly involving the FDA's programs to speed use of some therapies. 

Lomax's concluding statement in the article said,

"(R)egenerative medicine therapies are treating and curing debilitating and deadly diseases. The FDA’s RMAT (regenerative medicine advanced therapy) and breakthrough designations have created a streamlined environment for product sponsors. To effectively leverage this regulatory policy environment, sponsors must conduct high-quality clinical trials that are often operationally complex.

"Clinical trial networks, capable of managing the array of regenerative medicine technologies, are well suited to manage this complexity. The CIRM Alpha Clinic network is a current highly functioning proof of the concept. Patients with unmet medical need and product sponsors will benefit from the replication of this model nationally and internationally."

The agency, formally known as the California Institute for Regenerative Medicine or CIRM, initiated the Alpha network in 2015. It has since enlarged the effort with a total of $50 million and locations at UC San Francisco, UC Davis, UCLA, UC Irvine, City of Hope and UC San Diego. 

The Clinical Leader piece said, 

"Recognizing this operational complexity and limitations in clinical experience, the California Institute for Regenerative Medicine (CIRM) pioneered the development of infrastructure to support regenerative medicine clinical trials. Launched in 2015, the CIRM Alpha Clinics are a network of six California medical centers. The clinics conduct FDA-authorized and IRB approved clinical trials. The aim of the network is to achieve greater and more efficient results than the member organizations could if they acted independently. Each of the Alpha Clinics has formed teams with specialized knowledge and experience with regenerative medicine clinical research involving human cell and tissue products. These teams work across their respective centers. As of September 1, 2018, the network is supporting 49 clinical trials — and has supported 60 clinical trials since 2015 — across a range of indications."

Lomax continued,

"This network model of conducting therapeutically diverse clinical trials lies in contrast to the traditional disease-based clinical trial networks that tend to be located in specific clinical units – oncology, cardiology, neurology, etc. The Alpha Clinic teams are horizontally integrated across the centers, so they can support the diverse range of indications where regenerative medicine therapies are currently being evaluated. This technology-based approach facilitates the conduct of clinical trials, particularly in clinical units that may have limited experience with cell and gene therapy products."

A Global Stem Cell Perspective: CIRM's Accomplishments in a Field Tougher than Rocket Science

The comments below were emailed to the California Stem Cell Report yesterday in the wake of recent news coverage of the California Institute for Regenerative Medicine (CIRM), as the state's stem cell agency is known. 

The author, who asked not to be identified, has followed the field of stem cell research worldwide since its inception. Here is the text of the remarks.

"As you know, I am usually reticent about putting in my two cents worth regarding CIRM and all the politics swirling around the subject. But the recent published items, as well as radio interviews on the subject, have made me quite uncomfortable. I was not in California when Proposition 71 came on the ballot, so I cannot speak to the hype and campaign promises made that swayed the public. But I have to take issue with some of the nonsense that has been reported as news.   

"Here are some relevant facts: 

"1. People forget that in 2004, the NIH, the major agency in the US funding biomedical research, prohibited funding most of the research needed for this field to move forward. Forget about clinical trials, even basic research was severely curtailed based on unscientific premises. This meant that while other countries could move ahead, US scientists could not step into the arena. Prop. 71 was proposed to unshackle Californian scientists so that they could compete in the research arena on a par with UK, Canada, Australia, and many other countries eager to take advantage of this new discovery.

"2. Lest people forget, the NIH rules at that time were so onerous that anything -- buildings, maintenance, and equipment -- partially or wholly funded by the federal government could not be used to conduct human embryonic stem cell research. Hence the need for CIRM to fund the building of labs, infrastructure and facilities early on. This is not an idea to mindlessly benefit academic institutions, but to allow their investigators to conduct stem cells research without using anything (buildings, equipment, personnel) paid for by federal funds. 

"3. Biomedical research is not like rocket science - it is much harder and much less predictable. Twenty years after the big push by Congress to cure Parkinsons’ Disease by building Udall Centers and mandating funding for this effort - PD is still not cured. Alzheimers Disease has been a target of the NIH for decades; just this year, they are increasing funding for this effort. Yet - no cure in sight. Add to the growing list the “War on Cancer” declared by Nixon in 1971. How much progress was made by 1981? Yet look at all the success today? The fact is that biomedical research is much harder than engineering, and we should be patient instead of whining about the slow rate of progress. It behooves the critics of CIRM to remember that success in research requires time and money. Things are just beginning to ramp up exponentially by the end of the first 10 years. 

"4. Finally, taking a world-wide view of the state of human stem cell research, CIRM has to be considered a great success. CIRM supported the first few clinical trials using human embryonic stem cell products. At the end of 2017, there were 17 clinical trials using human embryonic stem cells. Of these 11 are conducted in the US, and all 11 are initiated in California or conducted in California - this is completely due to CIRM. Of the 11 trials, 6 are directly funded by CIRM, and NONE by the NIH. So without CIRM, there will be few or no clinical trials using this new technology in this country!"

(For some of the news coverage, see here and here.)

SF Chronicle Backs Away From More Cash for California's Stem Cell Agency

The San Francisco Chronicle, which has the largest circulation of any newspaper in Northern California, today said the results generated so far by state's $3 billion stem cell agency "don’t argue for expanded public spending."

The California Institute for Regenerative Medicine (CIRM), as the agency is formally known, expects to run out of cash for new awards at the end of next year. It is seeking to raise privately $220 million to tide it over until November 2020, when it hopes that voters will approve a $5 billion bond measure for continued stem cell research.

However, in a formal unsigned editorial published on its website, the Chronicle said,

"The results to date don’t argue for expanded public spending. The science of stem cell work will need to evolve before more money is provided."

The editorial followed a four-part series  in the Chronicle looking into the state of stem cell research. The final installment last week said the agency had fallen short of the high voter expectations when they approved creation of the agency in 2004. The agency has not yet backed development of a therapy that is available for widespread use, although it currently has investments in 49 clinical trials.

The Chronicle, which says it has a readership of more than 500,000, said,

"California’s pioneering decision to spend $3 billion on stem cell research isn’t producing cures after 14 years of work. Instead, it’s generating a widening scientific field that shows potential but not results.

"It’s a frustrating shortcoming, especially as supporters of the state-sponsored research weigh another bond measure to continue the work. Promised breakthroughs used to sell Proposition 71 in 2004 aren’t panning out, a Chronicle investigation found."

California's Stem Cell Agency Touts Its "Incredible Ecosystem" of Research

The California stem cell agency today added more of its comments to last week's evaluation of its work by the San Franciso Chronicle in a lengthy piece that said the agency had not measured up to voter expectations.

The forum was The Stem Cellar, the blog of the California Institute for Regenerative Medicine (CIRM), as the nearly 14-year-old agency is formally known. 

Maria Millan, CIRM photo

CIRM CEO Maria Millan, in a Q&A, elaborated on the value proposition offered by the agency and some of the points that were raised in the Chronicle article. Here are two excerpts from the blog. 

"Q: There have been many critics who say it’s taking too long for CIRM to deliver cures, and they expected more. What is your response to these people?

"A: Many of us can relate that relief cannot come quickly enough for our relatives and friends who suffer from debilitating and devastating medical conditions— I believe that is why many of us are at CIRM, an organization whose mission is to accelerate stem cell treatments to patients with unmet medical needs. Through the years, we have enabled the creation of an incredible ecosystem of top scientists and researchers and partnered with patients and patient advocates to pursue this mission. We continually strive to improve and to become more efficient and we share the sense of urgency to harness the potential of stem cell biology to deliver relief to those in need.

"Q: Given all of the differences between CIRM and the NIH (National Institutes of Health), why do you think the reporter compared CIRM to the NIH?

"A: The NIH is the largest health research funder world-wide, has been around a lot 
longer, has a much larger budget >$30B this past year alone and the NHLBI
(National Heart, Lung, and Blood Institute) alone has a $3B annual budget—NHLBI is just one of the 27 NIH Institutes. The reason that CIRM was formed is that the advocates of Proposition 71 wanted to make sure that scientists and developers can pursue vital research opportunities that may not have access to funding by traditional funders, including the NIH. CIRM has a total budget of $3B available to fund research and support operations and we have been managing that budget since the passage of Proposition 71 in 2004. If we consider the number of stem cell trials for given available budget, CIRM has funded a disproportionately higher number of translational and clinical programs in stem cell and regenerative medicine. In fact, the NHLBI has entered into a collaboration with CIRM on their Cure Sickle Cell initiative because of CIRM’s specialization in funding and enabling cell-gene regenerative medicine research. I take this as a validation of CIRM’s value proposition in this new area– acceleration, translation, and clinical trials."

See here for more on the sickle cell initiative.

San Francisco Chronicle: California's $3 Billion Stem Cell Program Does Not Measure Up to Voter Expectations

The San Francisco Chronicle, in a long and penetrating look at California's $3 billion stem cell agency, today said the research program has fallen "far short" of the promises made by its backers during the ballot campaign that created the effort.

Written by Erin Allday and Joaquin Palomino, the article said the agency, created by Proposition 71 in 2004, "can take credit for some notable progress," including saving the lives of children with rare immune deficiency diseases. Such efforts have been well supported by the agency, formally known as the California Institute for Regenerative Medicine (CIRM).

"But as thrilling as such advances are, they fall far short of what Prop. 71’s promoters promised." Allday and Palomino wrote.

"Not a single federally approved therapy has resulted from CIRM-funded science. The predicted financial windfall has not materialized. The bulk of CIRM grants have gone to basic research, training programs and building new laboratories, not to clinical trials testing the kinds of potential cures and therapies the billions of dollars were supposed to deliver."

Allday and Palomino worked on the CIRM overview for months, along with three other major pieces on stem cell therapies, both unregulated and those backed by the stem cell agency.  They reviewed the nearly 1,000 grants awarded by the agency and tracked the results, interviewing researchers and patient advocates and quantified the results.

The Chronicle series appeared as the agency nears its financial demise. It expects to run out of cash for new awards next year. The agency hopes that voters will approve a yet-to-be-written, $5 billion ballot measure in November 2020.

The Chronicle noted, however, that much of the research financed by the agency is not likely to resonate with voters.

Nonetheless, the article today contained ample information from the agency about its efforts, including its 49 clinical trials and some high profile results from those trials.  The piece posed the question of whether the nearly 14-year-old program has paid off. And it said,

"It’s not a question that can be answered simply. Science often can’t be measured in quantifiable outcomes. Failures aren’t just common, they’re necessary — it’s impossible to expect every dollar invested in research to lead down a traceable path toward success.... 

"It has helped make California a global leader in the field that’s come to be known as regenerative medicine. Anywhere significant stem cell research is taking place in the state, it almost surely has received support from CIRM."

The Chronicle quoted a member of the CIRM board who has been with it since its first days.

"'What was promised was not deliverable,' said longtime CIRM board member Jeff Sheehy, a former San Francisco supervisor. 'However, I would distinguish the promises from the impact and value. We have developed a regenerative medicine juggernaut.'"

The Chronicle also spoke with Bob Klein, a Palo Alto real estate investment banker who led the 2014 campaign.

"Klein...is unapologetic about the campaign he led. Indeed, as he lines up advocates and testimonials for the coming campaign, his message is familiar: Fund this research and we will save lives. Slow it down and the consequences will be grave.

"'Do you want your son to die? Are you going to wait?' Klein asked recently. 'Is that the price you are prepared to pay?'"

Today's Chronicle piece, roughly 5,000 words long, raises a host of important issues and deals with them in a nuanced and thoughtful manner. It is must reading for all those interested in California's stem cell research effort. 

(Editor's note: An earlier version of this item inadvertently omitted Palomino's name. Allday noted in an email to the California Stem Cell Report: "He played a HUGE role in putting together the CIRM story – he was basically solely responsible for collecting and analyzing the data from CIRM.")

'Better Than Hope' to 'No More Cash Without Changes' -- Overviews of California's $3 Billion Stem Cell Program

Looking for a relatively quick, current overview of the activities of the $3 billion California stem cell agency?

You can find it here on the California Stem Cell Report with the links below to recent items growing out of a state legislative hearing.

CEO Millan Makes the Case for California's $3 Billion Stem Cell Research Program

The top executive at California $3 billion stem cell agency, which expects to run out of cash for new awards next year, outlined for state lawmakers last week the benefits of the nearly 14-year-old program.

California Stem Cell Agency Touts its Economic Impact on the Golden State

The California stem cell agency last week told its story to a state Assembly committee on biotechnology, including economic figures from a study financed by the agency itself. 

'Better Than Hope' -- An Advocate's View of California's $3 Billion Stem Cell Research Program

Longtime patient advocate Don Reed appealed to California lawmakers last week to support the state's stem cell research effort, which expects to run out of money for new awards at the end of next year.

A Before and After Story of $138 Million in California Stem Cell Cash

The head of the stem cell program at UC Davis says California is leading the way into a "new era of living medicine," thanks to the efforts of the $3 billion state stem cell agency.

State Lawmakers Hear Story of a Life-Saving Treatment and the California Stem Cell Agency

A father from Folsom, Ca., earlier this month told a state legislative committee about how his baby boy was saved through a clinical trial that was being financed by the Golden State's $3 billion stem cell agency.

California's Stem Cell 'Gold Rush:' Nearly 14 Years of Prospecting

A committee of the California Legislature today examined the state's $3 billion stem cell agency. Officials of the agency and others presented their perspective. Here is the text of prepared remarks by David Jensen, publisher of this blog, who appeared at the invitation of the Legislature. This is a third-party overview that deals with pluses, minuses and policy questions.

A Harsher Look at California's Stem Cell Program: No More Cash Without Changes

California's $3 billion stem cell experiment received a host of accolades last week at a state legislative hearing, but one strong, critical voice was not heard in the proceedings.

Exploring California's $210 Million Search for a Sickle Cell Cure, Plus an Online Look at Stem Cell Over Excitement

CIRM mounted another Facebook Live event last week, which has
chalked up nearly 2,000 views.

Hype, hope and sickle cell anemia -- all were part of Internet videos last week involving the $3 billion California stem cell agency.

The San Francisco Chronicle mounted one and the stem cell agency the other. The Chronicle's production was hosted by Erin Allday, the reporter at the newspaper who wrote its ongoing series, The Miracle Cell, dealing with stem cell treatments. (The next installment is scheduled for publication on Thursday and is expected to evaluate the agency itself.)

Allday asked Maria Millan, CEO of the agency, and Paul Knoepfler, a researcher at UC Davis, about the juggling act needed when the benefits and progress of stem cell research are discussed. Both Millan and Knoepfler said it is easy to get overexcited by the progress of stem cell research.

The agency, formally known as the California Institute for Regenerative Medicine (CIRM), produced the second video, a Facebook Live event that so far has drawn more than 1,900 views. Similar figures were not available for the Chronicle video.

Featured on the CIRM video are Donald Kohn of UCLA and Mark Walters of the research institute at Children's Hospital Oakland, along with patient advocate Adrienne Shapiro. The agency has invested $210 million into efforts to find a cure for the disease that afflicts 80,000 people nationally.

Yes or No on More Money for California's Stem Cell Program? Your Chance to Vote Today

It's time to vote -- sort of -- on whether to kick in $5 billion more for California's $3 billion stem cell research program.

Recent results from poll on CIRM

I say "sort of" because the voting consists of a poll being conducted by UC Davis researcher Paul
Knoepfler on his blog, The Niche. Initial results are already in. Sixty-three percent of those responding say they would definitely vote for a ballot measure to "refund" the agency.  (That figure jumped to more than 67 percent by Sept. 1,  three days after this item appeared. You can vote by clicking on this link.)

The poll, of course, is non-scientific, and the readers of Knoepfler's blog are hardly representative of the state's voter profile. Presumably those who seek out his blog are more informed about and more interested in the California stem cell agency than the average California voter . Nonetheless, it is an interesting exercise.

The question posed by Knoepfler's poll is not trivial. The agency, formally known as the California Institute for Regenerative Medicine(CIRM), expects to run out of cash for new awards at the end of next year. It hopes voters will approve a proposed ballot measure in November 2020, giving it another $5 billion.

If you would like to know more about the case to be made for the agency, search this blog (the California Stem Cell Report) using the term "assembly biotech hearing."  That will take you to seven brief brief articles related to a legislative informational hearing on the work of the agency over the last 13 years.

State Lawmakers Hear Story of a Life-Saving Treatment and the California Stem Cell Agency

Pawash Kashyap and son, Ronnie

A father from Folsom, Ca., earlier this month told a state legislative committee about how his baby boy was saved through a clinical trial that was being financed by the Golden State's $3 billion stem cell agency.

"It was a blessing day for us," said Pawash Kashyap, who appeared before the Assembly Select Committee on Biotechnology,  which held a hearing Aug. 15 dealing with the California Institute for Regenerative Medicine (CIRM), as the agency is formally known. Kashyab referred to the occasion when he and his wife, Upasana, received a call from UC San Francisco warning him of the immune deficiency that afflicted their baby, Ronnie.

Researchers told them that the child could well die if he suffered from even a slight infection. The immune deficiency is sometimes known as the "bubble baby" disease, which has sometimes meant children were encased in plastic bubbles to stave off infections.

The problem was detected by routine newborn screening. Kashyap told legislators of their concern after learning the bad news.

"We Googled it, and nothing good was coming out of it."

Ultimately, the Ronnie was treated successfully. His father said that Ronnie has experienced potentially infectious crowds in malls and elsewhere and that he is doing "fantastically well."

Ronnie is now the "cover baby" on CIRM's annual report, which was praised by one lawmaker, Assemblyman Todd Gloria, D-San Diego. He said the report told the nearly 14-year, CIRM story in understandable and compelling language.

The full hearing can be seen here and downloaded. Kashyap's brief remarks begin at 55:36 into the video (he spoke without a prepared text). An audio file is also available at the same URL.

Here are links to the remarks at the hearing by CIRM CEO Maria Millan, CIRM Vice Chair Art Torres, patient advocate Don Reed, Jan Nolta, head of the UC Davis stem cell program, and David Jensen, publisher of this web site.

Article Postponed on Folsom baby and the Stem Cell Agency

Due to circumstances beyond the control of the California Stem Cell Report, the promised article on a legislative appearance by a Folsom father of an infant involved in a stem cell agency clinical trial has been delayed until Monday.

The father appeared at a legislative hearing earlier this month, along with his wife and child. The baby was afflicted with an immune deficiency disorder that would have been fatal. But he was treated in a clinical trial backed by the stem cell agency and is now healthy.

A Harsher Look at California's Stem Cell Program: No More Cash Without Changes

California's $3 billion stem cell experiment received a host of accolades last week at a state legislative hearing, but one strong, critical voice was not heard in the proceedings.

That came from the Biopolitical Times, a blog operated by the Center for Genetics and Society of Berkeley, a longtime foe of the agency. 

In a piece written by Pete Shanks, the agency was taken to task for a number of reasons. And he argued that it should not receive additional funds as it is presently constituted.

Shanks wrote, 

"At one time, CIRM had a deserved reputation for funding buildings , some of them at private universities, and was heavily criticized for that, but the $270 million “major facilities” budget approved in 2008 has all been spent. Some of the conflict of interest scandals are largely in the past, though ripples persist , and some of the institutional ones remain; several universities that receive large grants are still represented on the board . But there has been a new regime in place (“CIRM 2.0”) for several years.

"Things have improved, though not enough."

Shanks noted that the agency has failed to finance any therapies that are available for widespread use. He noted that the interest expense on state bonds that support the agency boost the cost to taxpayers to $6 billion from the $3 billion in awards.

He said the hearing last week was largely "a promotional vehicle." (Shanks' piece was published on Aug. 14, the day before the hearing by the Assembly Select Committee on Biotechnology.)

Shanks concluded:

"Going forward, there are two separate questions to consider: Is continued state funding of stem cell research at a rate of roughly half a billion dollars a year the best use of state funds?

"If it is, should those funds be spent through CIRM as it is presently constituted?
Is continued state funding of stem cell research at a rate of roughly half a billion dollars a year the best use of state funds? If it is, should those funds be spent through CIRM as it is presently constituted? 

"The first question is debatable; the second deserves a flat “No.” There is something obviously wrong when an agency is funded by public money but never has to submit a budget to the legislature, and can even go 13 years without appearing before an oversight committee. Two major reports, in 2009 by the Little Hoover Commission and in 2012 by the then Institute of Medicine (now part of the National Academies of Sciences, Engineering, and Medicine), both concluded that the governance structure of CIRM is seriously flawed."

A Before and After Story of $138 Million in California Stem Cell Cash

UC Davis graphics and captions

The head of the stem cell program at UC Davis says California is leading the way into a "new era of living medicine," thanks to the efforts of the $3 billion state stem cell agency.

Jan Nolta, the stem cell chief at Davis,  told the Assembly Select Committee on Biotechnology last week that the demise of the agency would lead to what some in her program are calling "cirm-amageddon," a play on the word armageddon and the initials of the stem cell agency's formal name..

The nearly 14-year-old agency is officially known as the California Institute for Regenerative Medicine (CIRM). It expects to run out of cash for new awards at the end of next year. CIRM is pinning its hopes for survival on yet-to-be-written ballot measure that would give it another $5 billion in state bonds if voters approve it in November 2020.

Nolta told lawmakers about the specialized facilities that are needed to deliver "living medicines" and highlighted the growth of the UC Davis stem cell program, which was virtually non-existent in 2004 when the agency was created.

Since then Davis has received $138 million from CIRM and has 24 stem cell trials underway "thanks to CIRM."

Nolta was lured to California after the passage of the ballot measure creating the agency. She said that the agency's programs have been a big draw for scientists from throughout the nation.

"If you want to do stem cells, this is where you come."

The full hearing can be seen here and downloaded. Nolta's remarks begin at 1:26:19 into the video (she spoke without a prepared text). An audio file is also available at the same URL.

Here are links to the remarks at the hearing by CIRM CEO Maria Millan, CIRM Vice Chair Art Torres, patient advocate Don Reed and David Jensen, publisher of this web site. Tomorrow the California Stem Cell Report will carry an item on the remarks of the father of an infant whose life was saved as the result of a clinical trial supported by CIRM. (This story has been postponed until Aug. 27.)

'Better Than Hope' -- An Advocate's View of California's $3 Billion Stem Cell Research Program

Don Reed 

Longtime patient advocate Don Reed appealed to California lawmakers last week to support the state's stem cell research effort, which expects to run out of money for new awards at the end of next year.

He said,

"Today, thanks to the 7.2 million voters who authorized the California Institute for Regenerative Medicine, or CIRM, we have something better than hope; we have results, accomplishments, people made well—and a systematic way to fight chronic disease."

Reed was among the persons testifying last week before the state Assembly Select Committee on Biotechnology.

He  is vice president of Americans for Cures, a nonprofit group founded by Robert Klein, a real estate investment banker and who ran the ballot initiative campaign that created the $3 billion California stem cell agency. Klein was also the agency's first chairman and may lead another ballot initiative in November 2020 to renew the agency with an additional $5 billion.

Here are links to the remarks at the hearing by CIRM CEO Maria Millan, Art Torres, vice chairman of the CIRM board, and David Jensen, publisher of this web site.  Tomorrow the California Stem Cell Report will carry a look at the remarks by Jan Nolta, head of the stem cell program at UC Davis.  

The full hearing can be seen here and downloaded with closed captioning. An audio file is also available at the same URL. Here is the text of Reed's remarks.
(Some readers have reported that they cannot download Reed's comments shown below. However, I cannot detect any problem with that process. If you would like a PDF of Reed's remarks, please email me at djensen@Californiastemcellreport.com.)

   2018-08-15 Reed Assembly Biotech Hearing by DavidJensen on Scribd

California Stem Cell Agency Touts its Economic Impact on the Golden State

Art Torres at Assembly hearing last week

The California stem cell agency last week told its story to a state Assembly committee on biotechnology, including economic figures from a study financed by the agency itself. 

Art Torres, a former state legislator and now vice chairman of the agency's board, kicked off the proceedings Aug. 15 with a brief overview. He also said that agency-backed projects have "brought in over $2.7 billion in investments to those projects from other sources (industry partners, co-funding and additional follow-on funding such as non-CIRM grants or philanthropy."

Torres said the add-on funding was the product of the $2.6 billion that has been actually awarded during the nearly 14-year lifespan of the agency, known formally as the California Institute for Regenerative Medicine (CIRM). The agency was provided with $3 billion in state bond funding in 2004. Cash for new awards is expected to run out by the end of next year.

Here are links to the remarks at the hearing by CIRM CEO Maria Millan and David Jensen, publisher of this web site. Tomorrow the California Stem Cell Report will carry the text of remarks by Don Reed, a longstanding patient advocate for stem cell therapies. 

(Toda,y, Aug. 21, the stem cell agency reprinted Jensen's remarks on its blog, noting that "at CIRM we know that not everyone agrees with us all the time, or supports all the decisions" of the agency.) 

The full hearing can be seen here and downloaded with closed captioning. An audio file is also available at the same URL.

Here is the text of Torres' remarks.

   California stem cell agency: Testimony by Vice Chair Art Torres to state Assembly Biotech Committee 2018-08... by DavidJensen on Scribd

CEO Millan Makes the Case for California's $3 Billion Stem Cell Research Program

Maria Millan, CEO of CIRM, at state Assembly hearing last week

The top executive at California $3 billion stem cell agency, which expects to run out of cash for new awards next year, outlined for state lawmakers last week the benefits of the nearly 14-year-old program.

Maria Millan, president and chief executive officer of the California Institute for Regenerative Medicine (CIRM), as the agency is officially known, laid out how the agency funds "the most innovative, high risk but high reward" stem cell research in the state.

Millan highlighted some of the 49 clinical trials backed by CIRM. She said,

"Every single trial represents a tremendous breakthrough approach that was unimaginable and even the subject of science fiction just years ago."

She also discussed the agency's relationship with the stem cell industry in backing research during a risky financial stage called "the valley of death." She said,

"CIRM supports and de-risks through this stage, where industry and traditional investors are not yet ready to come in. By accelerating development and allowing these early stage trials to proceed with CIRM funding, we have enabled the projects to gain visibility and interest leading to an increase in industry investments."

Her presentation was generally well received by the five lawmakers who were in attendance at various times during the meeting of the Assembly Select Committee on Biotechnology, chaired by Kevin Mullin, D-San Mateo.

The full hearing can be seen here and downloaded with closed captioning. An audio file is also available at the same URL. Tomorrow the California Stem Cell Report will carry the text of remarks by Art Torres, vice chairman of the agency and a former state legislator.  This writer was also invited to appear at the session. My remarks can be found here.

Here is the full text of Millan's comments.
Remarks by Maria Millan, President of CIRM, To California Assembly's Select Committee on Biotechnology 2018... by DavidJensen on Scribd

A California Stem Cell Tale: The Search for a Treatment for Paralyzing Spinal Injuries

Ed Wirth of Asterias, photo by Gabrielle Luri, SF Chronicle 

The San Francisco Chronicle, in a lengthy and dramatic article this week, reported on the story of the first clinical trial in the United States for a human embryonic stem cell treatment, a tale that continues today and well into the years ahead. 

The story involves paralyzing spinal injuries, incurable afflictions, walking rats, Geron, Asterias Biotherapeutics and BioTime and the economics of development of a stem cell therapy. That is not to mention the California stem cell agency, which has pumped more than $20 million into the effort.

The story was written by Erin Allday and is the third installment in a series involving stem cell research and treatments, both legitimate and illegitimate.

Her story began like this:

"Amid the controlled chaos of the operating room, Edward Wirth stood to the side, watching the surgeon slice open the back of the young man on the table....

"The surgeon nudged aside skin and a thin layer of muscle and clamped them out of the way. He chipped away bony vertebrae, exposing the shiny, smooth rope of the spinal cord. His scalpel slid into the membrane surrounding it.

"Wirth stepped forward, just beyond the surgeon’s shoulder, close by for these last steps. The surgical team moved a robotic arm holding a syringe into place. It was loaded with millions of immature support cells that had been meticulously crafted from stem cells, the cells that are the foundation of the human body, able to transform and reproduce indefinitely."

Wirth is a scientist who has been involved with the research since its early days at Geron of Menlo Park, Ca.. He has put in more than 20 years on the effort. Asterias Biotherapeutics of Fremont, Ca., which is associated with BioTime of Alameda, is currently handling the effort to create a treatment for spinal cord injuries.

The $3 billion California Institute for Regenerative Medicine (CIRM), as the agency is formally known, has provided a total of $20.7 million for the effort, first with Geron and now with Asterias.

Allday detailed the ups and downs, financial and scientific, of the research. She peered into the future and wrote that "it will take years to produce a marketable, federally approved therapy -- assuming their research makes it that far."

Nonetheless, she reported,

"Clinical trials like the one run by Asterias are scientists’ most ambitious attempts to harness the potential of what remains an elusive medical marvel. And the work at Asterias exemplifies both the incredible progress that’s been made in stem cell research and the great distance the field has yet to go before life-changing therapies are widely available.

"Riding on Asterias’ success isn’t just the fate of a single company or the careers of scientists like Wirth. Potentially, tens of thousands of patients every year could benefit directly from its therapy — and millions more if its research leads to further advances."

The fourth and final article in the Chronicle series is now scheduled to appear on Sept. 6  and will take a look at the California stem cell agency.

California's Stem Cell 'Gold Rush:' Nearly 14 Years of Prospecting

A committee of the California Legislature today examined the state's $3 billion stem cell agency. Officials of the agency and others presented their perspective. Here is the text of prepared remarks by David Jensen, publisher of this blog, who appeared at the invitation of the Legislature.

(On Aug. 21, the stem cell agency reprinted the text below on its blog, noting that "at CIRM we know that not everyone agrees with us all the time, or supports all the decisions" of the agency.) 

   2018-08-15 Jensen Assembly Biotech Committee Testimony by DavidJensen on Scribd

ACT to Astellas: A 'Looping' Tale of the Stem Cell Industry and Research

Ever wonder what happened to Advanced Cell Technology, Inc. (ACT), the Massachusetts stem cell company that migrated -- with some considerable ballyhoo -- to California in the wake of the creation of the Golden State's $3 billion stem cell agency?

ACT landed in the San Francisco Bay area in 2006 with hopes of snagging millions from the agency, which had been created two years earlier. At one point in the company's life, Gov. Jerry Brown even approvingly toured the company's operation in Alameda. But little has been heard from the company in recent years.

Now comes UC Davis stem cell researcher and blogger Paul Knoepfler. Last week, he published a brief update on the doings of the company once known as ACT and its changing corporate identities.

Michael D. West, AgeX photo

But first a little more on ACT's history and the company's not-so-good times in California. It is a tale  that loops around Geron, Inc., which launched and then abandoned the first U.S. clinical trial involving human embryonic stem cells, and companies called
Asterias and BioTime. One thread that strings them all together is a stem cell pioneer named Michael D. West.

When ACT announced its move to the Bay Area, visions of millions of research dollars danced in the heads of its executives. The move was heralded by the state's governmental leaders, Then State Treasurer Phil Angelides said,

"California voters overwhelmingly approved Proposition 71 (which created the stem cell agency) to ensure that California remains the hub of groundbreaking scientific innovation that has the potential to cure and treat debilitating and life-threatening ailments. Advanced Cell Technology's move to California sends a powerful message that this promise can be realized - bringing high-skilled jobs and revenues to our state, and most importantly, offering hope to millions of patients and their families."

West, then president of ACT, said the company was committed to being a world leader in regenerative medicine.

That was in 2006.  Eight years later, ACT, once the only company in the United States with an ongoing clinical trial involving human embryonic stem cells, changed its name to Ocata Therapeutics.  In 2016 it was purchased by Astellas Pharma of Japan for $379 million. By then, ACT had abandoned its then nominal operations in California after its multiple applications for California state funding failed to win approval (see here and here).

West left ACT in 2007. He is now co-CEO of BioTime, Inc., of Alameda, and is a member of the board of directors of Asterias Therapeutics, Inc., of Fremont, Ca. Both companies are faring much better with the state stem cell agency, formally known as the California Institute for Regenerative Medicine (CIRM).

In 2013, Asterias picked up the human embryonic stem cell trial that Geron gave up on in 2011. West founded Geron in 1990 but left the company in 1998. Asterias has received $14.3 million to support the spinal injury trial that Geron abandoned.

BioTime also had better luck than ACT. It received a $4.7 million award from CIRM for work on human pluripotent stem cells. 

Last week's report from UC Davis scientist Knoepfler said that Astellas began a phase 1B trial in July to continue the macular degeneration work of ACT/Ocata. Knoepfler described the latest effort as a "small bit of encouraging news."

California Lawmakers Schedule Update Next Week on the State's $3 Billion Stem Cell Agency

The parents of this child will be testifying next week at a state legislative hearing

For the first time in many years, a California state legislative committee is going to take a look at the state's $3 billion, 13-year-old stem cell research program.

An informational hearing is scheduled for next week, Aug. 15, by the Assembly 
Select Committee on Biotechnology, chaired by Kevin Mullin, D-San Mateo. His district includes a number of biotech firms. No legislation is up for action, and the hearing is not intended to necessarily lead to legislation.

Scheduled to appear before committee are Jonathan Thomas, chairman of the governing board of the agency, and Maria Millan, president and CEO of the agency, formally known as the California Institute for Regenerative Medicine (CIRM).  

The last time a CIRM CEO appeared before a state legislative committee was in 2005 when Zach Hall, then interim CEO testified. 

Also on tap is Jan Nolta, head of the stem cell program at UC Davis, which now stands as the No. 5 recipient of CIRM funds with $138 million. From Folsom will come a couple, Pawash Priyank and Upsana Thakur, whose infant child is now alive as the result of experimental research backed by the agency. The publisher of the California Stem Cell Report, David Jensen, is also scheduled to appear.

The stem cell agency is unique in California history and is a global leader in funding of stem cell research. It operates outside of the normal state budgetary process with its state bond funding flowing directly to it without the usual state budgetary oversight exercised by the governor and the legislature. That is all due to the ballot initiative that created the agency in 2004 and altered the state constitution.

The agency is scheduled to run out of cash next year. It is hoping that voters will approve $5 billion more for it in November 2020.

Next week's hearing, which will be in room 444 in the Capitol, will be available live on the Internet on the day of the  hearing via this page.  It will be archived as well and will be found via this link.

(To see the text of testimony at the hearing, search this blog on the term "assembly biotech hearing.")

(Editor's Note: An earlier version of this item misspelled the last name of Kevin Mullin.)

The 'Renegade Outposts' of the Stem Cell Industry: A Sweeping Examination by the SF Chronicle

Chronicle graphic

"Merchants of hope" was the headline today on a lengthy look by the San Francisco Chronicle at what it called the flourishing but costly, unproven and largely unregulated stem cell clinics in the United States and California.

Written by Erin Allday, the piece said,

"They are renegade outposts operating with little legitimacy and oversight at the frontier of what is otherwise a highly promising field of medicine."

It was the second installment in the Chronicle's look at stem cell treatments, both dubious and not-so-dubious. The first was published last month.

Today's piece took a lengthier look at the dubious efforts than has been done in other recent,  mainstream media examinations of the field, which is drawing increasing scrutiny from federal regulators. Allday wrote,

"None of the treatments the clinics offer have been shown to be safe or effective. None have been approved by the FDA. They’re not backed by decades of laboratory and animal studies or by rigorous testing in humans.

"Yet for many patients desperate for care, these clinics fill the void between the long-anticipated potential and the real-world limitations of stem cells. They cater to people whose needs reach beyond the powers of current medicine — people who want to believe in the almost mythic powers of stem cells, who feel corporate health and science have forsaken them.

"People who turn instead to these merchants of hope."

The article also touched briefly on the $3 billion California stem cell agency. Allday said, 

"The California Institute for Regenerative Medicine (CIRM) — the state agency created by Proposition 71 in 2004 to fund stem cell research — has raised concerns about the thriving consumer therapy market. CIRM officials and many of the scientists they fund worry that the selling of unproven treatments could undermine their own work and cast doubt on the enormous potential of regenerative medicine."

Allday continued,

"'It really hurt our recruitment,' said Jane Lebkowski, president of research and development at Regenerative Patch Technologies in Portola Valley. 'We got a lot of questions: What happened in Florida? Am I going to go blind too?'

"Most patients, Lebkowski noted, don’t understand the difference between the fat stem cells that a for-profit clinic is using and the complex product her company is developing, which uses embryonic stem cells to develop a kind of retinal cell that’s damaged in macular degeneration.

"That’s part of the reason why scientists like Lebkowski and organizations like CIRM, which gave $17 million to the Regenerative Patch trial, have pressured the FDA to crack down on the consumer market."

The next installment in the series will deal with stem cell research and is scheduled for publication Aug. 16.

California Stem Cell Cybercast: Multiplying the CIRM Story

Here is today's CIRM Facebook event. The number in the lefthand corner is not static.
It changes as more people view the cybercast.

California's 13-year-old stem cell research agency staged another event in cyberspace today, chalking up more than 1,200 online views for its exploration of the search for cures and therapies for Lou Gehrig's disease.

It was the second go at Facebook Live for the California Institute for Regenerative Medicine (CIRM), as the agency is formally known.

The online event, which features disease experts and questions from viewers, bypasses traditional media and features a concrete way of measuring the event's reach both today and two weeks from today. It could be a significant communications tool for the $3 billion agency, which expects to run out of funds for new awards by the end of next year. The agency is hoping voters will approve another $5 billion in funding in November 2020.

Today's event garnered 819 viewers during its initial hour. By the time of this writing that figure had climbed to 1,262 as others found the event on Facebook.

The first Facebook event for CIRM, which discussed stroke therapy, has now recorded more than 7,500 views.

Live Online Event Tomorrow from California Stem Cell Agency: Ask the Experts about ALS

California's $3 billion stem cell agency tomorrow will offer up a live, online and interactive look at ALS and the research it is financing to help find a cure for the affliction.

The agency has pumped more than $79.4 million into efforts aimed at ALS, which is often referred to Lou Gehrig's disease. Three researchers funded by the agency will be online tomorrow and will address questions from viewers. Here are more details here about the agency's FaceBook Live event at noon PDT, including instructions on how to view it and take part.