A Chance to Peek Inside a $50 Million California Stem Cell Program

Video from 2017 Alpha Clinic symposium

One of the better stem cell, show-and-tell conferences is coming up this spring in San Francisco, plus it is free and open to the public.

The day-long session will provide a peek inside one of the signature efforts of California's 14-year-old, $3 billion stem cell research program, which is edging closer to ever more critical financial times. 

The meeting April 18 is the fourth annual Alpha Clinic symposium. The Alpha Clinic Network was created in 2015 by the California Institute for Regenerative Medicine(CIRM), as the stem cell agency is formally known. 

CIRM has pumped $50 million into the network, which is aimed at delivering clinical trials to patients, creating strategic relationships between science and industry and leveraging statewide resources. Sites for the network are located at the City of Hope and at UC campuses in San Francisco, Davis/Sacramento, San Diego, UCLA and UC Irvine.

For those interested in California public policy issues dealing with health, medical mattters and research, the symposium will offer an up-close opportunity to check out the impact of the state stem cell agency. 

The session promises updates on stem cell research and clinical trials on afflictions ranging from sickle cell disease to cancer along with a look at gene editing. Scientists and patient advocates will be telling their stories and detailing the research.

CIRM is scheduled to run out of cash for new awards this year. It is attempting to raise privately $220 million to bridge a financial gap before hoped-for voter approval of $5 billion more in funding in November 2020.

Geoff Lomax, CIRM's senior officer for strategic infrastructure, told the California Stem Cell Report via email last week:

"It’s an exciting time in medicine as we have the tools to literally mend cells at the DNA level. The annual symposium is an opportunity to hear from the pioneers who developed these tool, the Alpha Clinic doctors and nurses that use them and the patients that have benefited from them."

Here are links to more information about the Alpha Clinic Network: Agenda for the symposium. CIRM's Alpha Clinic page, a CIRM blog item on the April meeting. 

The session this spring is financed with $60,000 from the stem cell agency.

California Stem Cell Program Trying to Build $220 Million Financial Bridge: An Update

For many months now, California's stem cell research program, which expects to run out of cash by the end of this year, has been seeking to raise privately $220 million. But it has yet to report that it has snagged a significant chunk of that amount.

The most recent overview of the current fundraising effort came last December at a meeting of the governing board of the California Institute for Regenerative Medicine (CIRM), as the state stem cell agency is formally known. 

Jonathan Thomas
CIRM photo

Jonathan Thomas, chairman of the CIRM board, briefed the panel on the progress of the effort to create a financial bridge to the hoped-for passage of a possible $5 billion ballot measure in November 2020. 

The fund-raising effort is targeting high net-worth, potential donors and medically oriented foundations with a smorgasbord of philanthropic options. The choices can be tailored to a potential donor's appetite, including supporting specific types of research, Thomas said. 

Thomas is expected to update the board on the fundraising effort at its next meeting March 21, which will be based at its Oakland headquarters. The session will also be available on the Internet with remote hook-ups that will allow members of the public to comment and ask questions.

CIRM was created by voters in 2004 who also provided it with $3 billion in state bond funding. No additional source of cash was established.  At the beginning of this year, the agency was down to its last $144 million for new awards. It has set aside cash for administering its final awards should a new bond measure fail to pass. 

Here is the text of what Thomas told the 29-member board in December about the fund-raising drive. The text comes from the transcript of the meeting.

“So I'm going to move next to a report on the bridge fundraising, sort of a year-in-review summary. As you recall, through the end of last year (2017), we had secured 7 million from Bill Bowes and Pitch Johnson. We talked about that earlier (2015). (The funds are allocated for a "wind-down.")

“At our December board meeting last year, when we reported on the fact we were aware we're going to be out of funds potentially by the end of 2019, we talked about a couple things. One, Bob Klein (former chairman of the agency’s governing board) was here and talked about his potential intent to run a new bond measure in the november 2020 general election for $5 billion. 

We, also at that meeting, discussed the fact that we would like to be able to keep things going at a normal pace between the time we had run out of money, which we anticipated to be late 2019, and the election. And so we decided at that point that we would look to pursue bridge funding to fill that gap ideally in an amount equal to roughly the average of what we put out over the last few years.

“So the vision at that time was to raise bridge funds to not only go through 2020, but, if successful in 2020, the next actual realization of bond proceeds would be in the spring issuance by the state treasurer of bonds on behalf of all state agencies funded out of the state treasurer's office. So it would really be getting from late 2019 to spring of 2021.

“The idea was all of that money had to be raised in a staggered way through the last installment in the middle of 2020. A note that this is just referring to research funds. We have admin funds earmarked currently all the way through 2023.

“The strategy at that point to pursue the vision was to identify the most likely parties interested in medical research both in California and in the rest of the country. We spent a great deal of time doing that.
“Secondly, to tailor the asks to specific candidates that we felt would be the most successful in terms of what we were pitching. Thirdly, to approach those candidates either directly or through third-party intermediaries well known to the candidates. And the theory there is not only is how you ask is important, but who gets you in the door to ask is vital.

“So we've spent a lot of time analyzing who the correct third-party intermediary would be who would have individual and direct contact with the candidates in question. We've had weekly meetings of our fund-raising team, consisting of myself, Maria Millan (CIRM president and CEO), Maria Bonneville (vice president of administration for CIRM), Scott Tocher (CIRM general counsel), and Eliana Barnett (senior executive assistant in CIRM's office of president), to discuss strategy as we continue to update. And we have coordinated with Bob Klein's office and with Melissa King (executive director of Americans for Cures) of Bob Klein's office on these discussions. (Klein founded Americans for Cures.)

“The plan to implement the strategy is to offer a menu of fund-raising options including charitable gifts, the loan product that we discussed that tied to the election, program-related investments, and other derivatives of those ideas. We developed that menu of options in consultation with legal counsel, bond counsel for the state, the state treasurer's office, the state controller's office, and other outside parties with relevant input, such as those that have had programs that we might wish to emulate. Note that we're asking for a variety of things, including unrestricted gifts or loans or gifts or loans to specific projects or conditions or whatever.

“The idea with the gift would be, or loan, if you put money in, not only would it enable the bridge period, but it would allow for giving us the most credible shot of getting an election passed in 2020, at which time, whatever your particular interest would be, if the measure passed, you would have a tremendous leveraging effect. 

“So if you were to put in, for example, 50 million and the measure passed, you've have a hundred-to-one leverage that would result from that, a good chunk of which could go towards whatever your specific interest was.

“So as we've been developing those ideas, we've been refining the asks as we've gone along in terms of what feedback we get as to what sounds more appealing or less appealing.

“To date, implementing that plan, which is connected to the strategy, we've met or had confidential calls with dozens of stakeholders, including ultra high net worth individuals whether individually or in groups.

“I referenced an event that Bob and I did in the summer in the Palo Alto area for a number of family offices. We've also talked, met with major foundations, with corporations who have an interest in the medical research space, and with numerous third-party intermediaries of the kind I described earlier. The way it sort of is broken down, been looking at funding either CIRM generally or with respect to specific projects, which I've sort of taken the lead on. Maria Millan has done a lot of great work in developing project-specific related asks for different initiatives that CIRM either has or would consider having that would get the fundraisers in the game.

“To date, as you might expect, a number of the meetings that we've had, the people we have talked to have, as far as the pitches go, either declined respectfully, others are ongoing as we speak. Since the last board meeting, we've continued our strategy discussions of third-party intermediaries. We've had several approaches to specific ultra high net worth individuals either in person or through these intermediaries. As before, a number of those have declined. However, there are a number of those discussions which are ongoing.

“We've calendared a number of meetings with potential stakeholders between now and the next board meeting, including meetings with major foundations, ultra high net worth, again, either in person or through third-party intermediaries. In addition, we have two group meetings similar to the one we had in the summer with family offices scheduled for the first quarter, which are one in San Diego and one in Los Angeles.

“So that is sort of where we are at this point. We continue to look for our anchor investor. And if we can do that, the strategy is when you get the anchor investor on board, that anchor investor typically has a number of friends that he or she can then rope into the fold. We're also going to focus more beyond the anchor investment to the smaller potential gifts. We've spent a great deal of our time on the anchor effort. And we will be, as we have successes, reporting in real time back to the board and in the next in-person meeting in March.

“So that's a review of the year. Are there questions? Thank you. That is most definitely a subject to be continued and continued and continued.”

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

For UC Davis scientist, Paul Knoepfler it was something "very strange." For Pulitzer Prize-winning Los Angeles Times columnist Michael Hiltzik, it had the earmarks of a "race to the bottom."

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

The headline on Hiltzik's column yesterday summarized the case like this. 

"Did a world-famous science journal become a shill for a questionable stem cell claim?"

Hiltzik wrote,

"Readers of Nature, one of the world’s most important scientific journals, might have been struck recently by an audacious claim appearing on its website about a possible stem cell treatment for heart attacks.

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

"Here’s the problem. The published item wasn’t a peer-reviewed article subject to Nature’s rigorous professional vetting procedure. It was an advertisement placed by the Translational Research Center for Medical Innovation, the Japanese research lab that says it performed the reported study on MUSE cells using white rabbits.

"It looked like a Nature article, however, at least to Paul Knoepfler, a stem cell expert at UC Davis who has become one of our most assiduous debunkers of stem-cell quackery. Knoepfler thought the layout of the item might cause some readers to mistake it for a peer-reviewed paper, and promptly queried Nature. The journal responded by taking the advertisement offline Feb. 22. Knoepfler’s brief chronicle of the affair can be found on his website here."

Hiltzik concluded:

"The Food and Drug Administration has its hands full monitoring these claims and treatments. The agency has issued multiple warnings to steer the public away from such clinics and has taken administrative and legal action against some of them. The signs point to more exploitation and more danger to the public health. Important journals such as Nature shouldn’t be participating in a race to the bottom."

For the record, Knoepfler has been critical in the past about some of Hiltzik's commentary, including the columnist's views about the state stem cell agency.  

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

The California stem cell agency has posted online the 24-page transcript from last week's meeting during which the agency's governing board raised sharp questions about Stanford University and its obligation to provide co-funding on a $19 million award to one of its scientists.

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

Just before casting his vote, Art Torres, vice chairman of the board and a former longtime state lawmaker, said, 

"I do think that we have put forward this motion in order to get Stanford's attention as to what their commitment has been in the past, and what it will continue to be given the nature of the arrangement that we made with them."

Stanford researcher Judith Shizuru sought $6 million for continuation of a phase one clinical trial to develop a potentially "transformative" product that would eliminate the toxic impact of chemotherapy for a number of diseases. 

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

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

Correction

An early version of the Stanford item today incorrectly reported the deadline for co-funding of the award in question. The correct date is May 1 -- not April 1. Judith Shizuru's last name was also incorrectly spelled.