Balloting on Stem Cell Person of the Year: Cast Your Vote Now

The countdown has begun for selection of the Stem Cell Person of the Year. You can vote online for your preference at The Niche, a blog operated by UC Davis researcher Paul Knoepfler.

He has put together a list of 20 nominees ranging from a California state legislative aide to the CEO of the New York Stem Cell Foundation, Susan Solomon. Also included is a host of researchers, including Shoukhrat Mitalipov and Alexey Bersenev. The full list can be found here.

Knoepfler is the sole judge of the winner and personally provides a prize of $2,000.

The current voting, which ends Dec. 12, will whittle the list down to 10 from which Knoepfler will pick the winner. Earlier this month, he had this to say about the award,

"Unlike some other science awards out there that shall remain nameless, the Stem Cell Person of the Year prize is not an insider kind of thing, but more of an anti-old boy’s club award. It’s not about who you know, but what you do to help science, medicine, and other people."

Personally, I think the team at the California stem cell agency should be on the list. The folks, past and present, at the agency have labored mightily since 2004 to advance stem cell research. Sometimes the circumstances have been difficult and challenging, but the work continued nonetheless. Maybe next year, as they say in the sports world.

"Cuts," Private Fund-Raising, $5 Billion Bond Measure: Answers to the Critical State of California Stem Cell Finances?

Bob Klein explored a $5 billion bond measure at today's stem cell meeting. 
California Stem Cell Report photo

OAKLAND, Ca. -- Facing the likelihood of a slow and withering death, the California stem cell agency today edged gingerly forward on a path of "cuts" and risky fund-raising in hopes that its research results will soon generate voter support for more billions of dollars.

Two governing board committees of the $3 billion agency, formally known as the California Institute for Regenerative Medicine (CIRM), this afternoon recommended that the full board "entertain" the proposals at its Dec. 14 meeting.

The agency is down to its last $269 million and expects to halt new awards in 2019 unless additional funds are raised between now and the beginning of 2020. It has been running at a $300-million-a-year pace recently and has pumped out about $25,000 an hour, 24 hours a day, seven days a week since it started making awards in 2005, according to calculations by the California Stem Cell Report.

Today's meeting focused on both short and long term finances. The committees examined a proposal to cut its planned clinical awards by $68 million over the next two years by limiting their size. The committees also indicated support for an ambitious effort to raise $222 million privately between now and early 2020.

Longer term, directors staked their hopes on voter approval of a ballot initiative in November 2020 that could total $5 billion. Development of a stem cell therapy that would resonate with voters would be a major key to the success of that effort. The 2004 campaign raised expectations that stem cell cures were right around the corner, but so far the agency has not backed one for widespread use.

Robert Klein, a Palo Alto real estate investment banker who ran the 2004 campaign that created the agency, told the CIRM directors of a private poll that he said showed 70 percent of Californians supported stem cell research and continued funds for the stem cell agency.

Following the meeting, the California Stem Cell Report asked him for a copy of the poll. He declined to provide it or identify the firm that conducted the survey.

Klein told the CIRM board members that the agency has made "remarkable progress" and has created a "moral imperative" to continue the search for stem cell cures. He said,

"The bridge to the future is CIRM and stem cell therapies."

The now less-than-robust finances of CIRM are a product of the ballot initiative, Proposition 71, that created it. CIRM is not funded through the customary process used by most state agencies. The initiative  stipulated that CIRM would be supported by $3 billion in state bonds whose funds would flow directly to the agency and bypass the legislature and the governor. When that cash runs out, the agency is left with no other source of state funding.

Raising $222 million privately over the next two years is no small task, but deep pockets exist that may well be tapped. The Eli and Edythe Broad Foundation, for example, has donated nearly $80 million to stem cell research centers at UCLA, UC San Francisco and the University of Southern California. All three are linked to CIRM. In San Diego, the Sanford Stem Cell Consortium was given $100 million in 2013 by Denny Sanford, another billionaire. The consortium came into being after it was backed by $43 million from CIRM.

Nonetheless, competition for philanthropic cash is heated. Thomas and Klein, however, both indicated that they would collaborate on raising the $222 million, which would allow the agency to add eight new clinical awards in 2020, among other things.

The risk lies in the timetable for bringing in the cash. The agency has a relatively tight schedule for making awards --- a schedule that a major philanthropist may or may not be ready or willing to comply with.

Without more cash, the stem cell agency projected that it would wither away by 2023 as it managed a declining number of old research grants.

For more, recent articles on CIRM's current critical funding situation, see here, here, here and here.

California Stem Cell Agency Examines its Financial Options Long-Term and Short-Term

OAKLAND, Ca. -- A key group of directors of the $3 billion California stem cell agency this afternoon opened a discussion dealing with its short and long-term financial future at 1:04 p.m. The meeting is scheduled to last until 4 p.m. The California Stem Cell Report will file stories as warranted.

California Tightening Stem Cell Research Belt; Size of Research Awards Targeted

The stem cell agency outlined its assessment of its current position
 in this slide prepared for today's meeting. 

OAKLAND, Ca. -- California's $3 billion stem cell agency is preparing to put the squeeze on its research awards over the next two years, cutting the caps on new grants in some areas by 50 percent or more.

It is all part of a strategy to prolong the life of the 13-year-old research effort as it fights to fulfill the expectations of voters who created it when they approved a ballot initiative in 2004. Voters provided only $3 billion in funding, however, and the agency is now expected to run out of cash for awards in 2019 unless it changes its financial ways.

Meeting here today are two key groups, the Transition and Science subcommittees of the governing board of the California Institute for Regenerative Medicine (CIRM), as the agency is formally known.

They are scheduled to wrestle with both the agency's short-term finances and the longer term effort to extend CIRM's life for perhaps an additional 10 years or more via another ballot initiative in 2020. As for next year, based on past discussions and practices it appears certain that the agency's directors will reduce the size of awards to the scientists and businesses who are often hard-pressed to find non-governmental support.

A proposal by the president of CIRM, Maria Millan, calls for reducing clinical program awards by a total of $68 million over the next two years. Here is how the cuts would work compared to the average award this year in each category.

• Phase 1 and phase 1/2 clinical trial grants would see their caps reduced from $20 million to $12 million for nonprofits and $8 million for for-profit enterprises. The average award in 2017 in these categories was $10.3 million. 
• Phase 2 trials would see their caps cut from also $20 million to $15 million for both non-profit and for-profit enterprises. The 2017 average award was $15.3 million. 
• Phase 3 trial awards, which averaged $16.7 million in 2017, would have their caps cut from $20 million to $10 million for both types of enterprises.

Millan's presentation indicated that would her proposals would allow the agency to support 24 new trials and eight trial candidates over the next two years, in keeping with a strategy of taking "more shots on goal" as the agency tries to develop a stem cell therapy for widespread use. Clinical trials, which can take years, are the last stage before a therapy is approved by the federal government. 

Millan's plan would also allot only $10 million for what the agency calls "Discovery" awards (basic research) next year (10 projects) and $9.25 million in 2019 (nine projects). Discovery awards are expected to hit $46 million this year. Education awards are expected to total $1 million this year. Next year they  would receive $750,000 and nothing in 2019. 

Key to the spending plan is the assumption that the agency could raise an additional $220 million from a variety of sources between the end of next year and early 2020. The goal of the fund raising is to bring in $55 million by the final quarter of next year. The rest would be raised between then and the first quarter of  2020. 

CIRM did not release details of its strategy for raising the $220 million. CIRM Chairman Jonathan Thomas, however, has already been on the fundraising trail and reports that he has identified a number of potential donors.

The financial plans and any changes will go to the full board for ratification at a meeting Dec. 14 here at CIRM headquarters.

The California Stem Cell Report will cover today's meeting and file reports as warranted on this web site. Below is another presentation slide from the stem cell agency. 

California Stem Cell Agency Faces Rising Cash Burn Rate; Readying for New, Multibillion Dollar Bond Attempt

Cash is flowing out the door fast at California’s $3 billion stem cell agency, which now expects money to run out for new research awards by the end of 2019 instead of the middle of 2020.

The California Institute for Regenerative Medicine (CIRM), as the agency is formally known, expects to be down to its last $269 million in uncommitted funds by end of this year. And it is working on a new plan to raise $200 million in private funds to sustain its operations through 2020.

The financial projections -- crafted as the agency faces what its leaders call a "critical stage" -- are online this week on the CIRM web site and include proposed lower caps on the size of awards in 2018 and 2019. The agency said the increase this year in the pace of funding was generated by more, higher quality research applications.

Maria Millan, CIRM photo

In documents prepared for a key CIRM meeting next Monday, Maria Millan, CEO of the agency, said her budget scenario would maintain CIRM’s “value proposition as intact as possible” while work moves forward on a proposed, multibillion dollar bond measure in November 2020. She also said enough funds would be available to “wind down” the agency if the bond measure fails.

(See here for Millan's plan and here for longer term bond funding.)

The agency was created by a ballot initiative in 2004, Proposition 71, which financed it with a $3 billion bond measure. No other significant funding was provided. The ballot campaign raised rosy expectations among voters that stem cell therapies were right around the corner.

So far the agency has not backed a therapy that has been approved for widespread use. But it is helping to support 38 clinical trials, the last stage before a therapy is approved by the federal government for widespread use. Many more trials, which can take years, are expected to be funded prior to 2020,

CIRM Chairman Jonathan Thomas, in a presentation prepared for next week’s meeting, pointed to “early successes” such as those involving work by Donald Kohn at UCLA. Thomas also cited “promising early returns” with Asterias Therapeutics’ work on spinal cord injuries.

Thomas’ proposal, in addition to private fundraising, calls for a “citizen-led” bond measure in the fall of 2020 to sustain CIRM on a more long-term basis. During next week's meeting, Robert Klein, the Palo Alto real estate investment banker who led the 2004 ballot initiative campaign, is expected to address the prospects for another initiative measure, possibly as large as $5 billion.

As back-up options, Thomas identified the possibility of asking the legislature and governor to place a bond measure on the 2020 ballot along with seeking donor funds for co-funding of specific projects.

In Millan's presentation, she said it is “essential to preserve CIRM’s value proposition to increase the probability of and the speed by which stem cell treatments can reach patients.”

The session on Monday involves the Transition and Science subcommittees of the CIRM board. The committees are expected to recommend budget and fundraising plans to be ratified by the full board at its meeting Dec. 14.

In September, the Transition group took a crack at a wider range of possibilities. The latest proposals are a refinement of what was discussed then.

Next week's meeting will be based at CIRM’s headquarters in Oakland with telephonic sites where the public can participate in La Jolla, Duarte, South San Francisco, Los Angeles and San Francisco. Public comments can be emailed to mbonneville@cirm.ca.gov. The session will also be audiocast on a listen-only basis. Instructions can be found on the agenda along with addresses for the telephonic locations. 

Correction

In the Parkinson’s therapy item on Nov. 17, 2017, the California Stem Cell Report incorrectly described the amount of funding involved in the GForce Parkinson’s initiative. The backing includes $52.3 million plus substantial support from BlueRock Therapeutics, which is financed with $225 million from Bayer AG and Versant Ventures. BlueRock, a Cambridge, Mass., firm, says on its web site, “Our most advanced therapeutic candidate, for Parkinson’s disease, will enter the clinic in 2018.”

More California Millions Sought to Support the 'New Era' of Parkinson's Therapy Research

A "new era" in the search for a cure for Parkinson's disease was heralded this month in an article in a prominent scientific journal that explored research involving more than $52 million and an organization called GForce-PD.

The news was accompanied by a cry for more support for Parkinson's research from the $3 billion California stem cell agency, which has pumped $49 million in Parkinson's studies over the last 13 years.

Jeanne Loring, director of the Scripps Center for Regenerative Medicine in La Jolla, Ca., and also a participant in the GForce initiative, said this week that CIRM has not supported Parkinson's research at the level of the other enterprises involved in GForce.

In an item she wrote for The Niche, a blog published by UC Davis researcher Paul Knoepfler, she listed $52.3 million in support plus substantial backing from BlueRock Therapeutics, which is financed with $225 million from Bayer AG and Versant Ventures. BlueRock, a Cambridge, Mass., firm, says on its web site, “Our most advanced therapeutic candidate, for Parkinson’s disease, will enter the clinic in 2018.

Parkinson's is a devastating disease that afflicts 10 million people in the world and 125,000 in California. One of those persons, David Higgins of San Diego, currently serves on the board of the stem cell agency, formally known as the California Institute for Regenerative Medicine (CIRM). Another, Joan Samuelson, was one of the original board members in 2004 and a fervent but often frustrated voice for Parkinson's research at many CIRM board meetings.

Actor Michael J. Fox, who made TV ads for the 2004 ballot initiative that created the stem cell agency, is also among the those living with the disease. Others with the affliction included the late boxing champion Muhammad Ali and famed semiconductor pioneer Andy Grove. 

Loring wrote on The Niche about a meeting in Japan earlier this year dealing with the research teams in the GForce project. She said,

"The Kyoto meeting was unprecedented in my experience.  Instead of competing, the four groups cooperated and shared plans for their proposed clinical trials.  We agreed to harmonize our trials and stay in communication about our progress.  All of us plan to start clinical trials within two years."

Loring continued,

"Since my team has been recognized by the international GForce initiative devoted to safe effective therapy for PD, we hope that CIRM will follow the example of New York, the EU, and Japan, and invest more in our project to provide neuron replacement therapy for Californians with Parkinson’s disease.

"While we hope to gain more support from CIRM, we are determined to follow through with our clinical trial, with or without CIRM.  It will just be more difficult without their help. The patients and their advocates inspire us, and we won’t let them down."

Loring added more information on Sunday (Nov. 19) concerning CIRM funding. She told the California Stem Cell Report. 

"We started our pre-clinical PD studies in 2011, with funding from Summit for Stem Cell. The first and only funding we received from CIRM for PD research was in 2016. 

"Before 2011, CIRM invested $41,838,336. Since 2011, CIRM has invested $7,357,468. This means that the majority of the funding went to projects that didn’t lead to any translational or clinical applications.  

"There are currently three active grants working on Parkinson’s disease, for a total of $4.9 million. We have the only translational grant, and it will expire in March 2018. All but $650,000 runs out by the spring of 2018. 

"There are no more grants forthcoming for our work. The Scaled Biolabs grant awarded this year is a partnership with us. Birgitt Schuele’s grant is basic research, not a cell therapy. (Below are the three active grants identified by Loring.) 

"Parkinson's Institute, Birgitt Schuele, Quest - Discovery Stage Research Projects, CRISPR/dCas9 mutant targeting SNCA promoter for downregulation of alpha-synuclein expression as a novel therapeutic approach for Parkinson’s disease, $1,931,495 

"Scripps Research Institute, Jeanne Loring, Quest - Discovery Stage Research Projects, Autologous cell therapy for Parkinson’s disease using iPSC-derived DA neurons, $2,354,226 and later $4,285,721  

2017
"Scaled Biolabs Inc., Justin Cooper-White, Quest - Discovery Stage Research Projects, A tool for rapid development of clinical-grade protocols for dopaminergic neuronal differentiation of Parkinson’s Disease patient-derived iPSCs, $657,528 "

(Editor's note: An earlier version of this item contained incorrect figures on the GForce initiative.) 

'Critical Stage' for $3 Billion California Stem Cell Effort and its Search for More Cash

California's $3 billion stem cell research program later this month is expected to unveil detailed plans for extending its life beyond the middle of 2020 in hopes of avoiding a lingering death.

The latest proposals, which are not yet public, are scheduled to be discussed Nov. 27. Possibilities range from another multi-billion dollar bond measure to private fundraising to possible merger with some sort of private entity.  

The stem cell agency, known formally as the California Institute for Regenerative Medicine (CIRM), projects its cash for new awards will run out in about 2 1/2 years. At that point, unless more money is forthcoming, CIRM will only be overseeing the dwindling number of awards whose terms extend beyond June of 2020.

The agency's fate was dictated by Proposition 71, which created CIRM in 2004. It also provided $3 billion in state bond funding, which flows directly to CIRM without oversight by the governor or the legislature. No additional, significant resources were contained in the ballot initiative.

CIRM's future has been an occasional topic for its board for some time. But the issue has taken on more urgency this year. At a meeting in September of a newly formed Transition Subcommittee of the governing board, CIRM Chairman Jonathan Thomas said in what may have been an understatement,

"CIRM, as we know, is at a critical stage of its mission here." 

The meeting on Nov. 27 will additionally involve the board's Science Subcommittee. What emerges from the session will go to the full, 29-member board in December for ratification.

Options in September included a multi-billion dollar bond ballot measure in 2020 and a possible merger, which was described as something of a last resort. Whatever path is chosen, it likely would lead to changes in the agency, which has been criticized for conflict of interest issues and its dual executive arrangement, among other matters.

The agency has awarded $2.3 billion in 919 grants during its 13-year history. About 90 percent of the awards has gone to institutions with links to members of the governing board, past and present, according to calculations by the California Stem Cell Report.

So far the agency has not fulfilled expectations of voters that it would generate a widely available therapy. Something may emerge in the next couple of years from the 38 clinical trials currently backed by the agency. (Forty-three have been funded but five were terminated.) The trials, which can take years, are the last stage before a therapy is approved by the Food and Drug Administration for widespread use. CIRM plans to add more trials in the next couple of years.

This month's meeting will be based at CIRM's Oakland headquarters with teleconference locations elsewhere in the state where the public can take part. It is scheduled to begin at 1 p.m. PST and run until 4 p.m.

The California Stem Cell Report will provide full coverage of the meeting that day with advance information as it is posted on the CIRM web site.

(Here is a link to the transcript of the September meeting. Here is a link to Thomas' slide presentation.)

Financial Examination of California Stem Cell Agency: Fireworks Not Expected

The only state panel that has formal oversight of the $3 billion California stem cell agency meets this Thursday in Los Angeles for what is expected to be a routine meeting.

The body is the Citizens Financial Accountability Oversight Committee, chaired by the state controller, Betty Yee.  The 10 a.m. meeting in Los Angeles City Hall has nothing on the agenda that appears to raise a red flag. However, some past sessions have been lively.

Leaders of the agency are expected to brief the committee, which was created by Proposition 71 in 2004, which also created the research effort itself.

The ballot initiative specifically exempted the agency, known formally as the California Insitute for Regenerative Medicine, from control by the governor and legislature.

Here is a link to names of the members of the committee.

The session will be available on a listen-only basis via the internet or an 800 number. Here are instructions:
Public call-in number (listen only): (877) 260-8900
Confirmation number: 433267

Listen to the meeting online: https://im.csgsystems.com/cgi-bin/confCast
Enter conference ID number 433267, then click “Go.”