From Fraud Checks to Unspent Funds: California's Proposed Rules for Stem Cell Researchers

The California stem cell agency today put off adoption of  new rules for the way it will hand out its last $1 billion, speeding the money to researchers and attempting to improve the quality of research proposals.

The agency's staff deferred action on the proposal in order to spend more time refining it prior to action in March. The interim grant administration rules are designed to implement the CIRM 2.0 plan laid out by agency president Randy Mills, who has been on the job since last May. 

The rules will apply to the initial phase of CIRM 2.0, which closes the first of its new, rolling application rounds tomorrow, and is subject to adjustment. CIRM (the California Institute for Regenerative Medicine) expects to extend the new rules in one form or another to all upcoming award rounds.

The agency calls the fast-track effort a radical change from previous years. It is certain to have a major impact on many scientists.

The agency is funded by money that the state borrows (bonds). It has handed out roughly $2 billion and figures its remaining $1 billion will last until about 2020 when it will need additional financing.

The new regulations talk about such things as “real-time course corrections,” background fraud checks, early and stringent budget review, restrictions on appeals by rejected applicants and elimination of paper work.

Here are some excerpts from an agency memo identified as coming from the “legal team.”  It unfortunately does not contain a marked-up version showing language that has been deleted from the previous grant administration policy along with the new language.

Money

“Prior to CIRM 2.0, payments to grantees were made primarily based on the calendar – disbursements keyed off the start date of the project and were periodically made based on some given period of time following that date. Under the proposed GAP(grant administration policy), however, for clinical stage projects, CIRM will shift to a milestone-based payment schedule. Thus, this section of the GAP describes the importance of the milestones and how payments on the grant will only be made upon successful completion of the milestones.

“Additionally, in many circumstances the grantee will be allowed to keep unspent CIRM funds upon successful completion of the project, to be spent on any other project of the grantee’s that is consistent with advancing CIRM’s mission. This new process will incentivize grantees to advance the project in the most efficient and shortest time possible, fulfilling CIRM’s goal to accelerate such projects.”

Background checks

“The eligibility section has been drafted to reflect that applicants will undergo a background check to ensure no prior or pending records of fraud or misuse of funds.” 

Budgets

“This new review will examine the proposed budget to identify where proposed costs diverge from established market rates and where opportunities for budget tightening may be found. To incentivize efficient budgeting, where CIRM determines that a budget differs significantly from market rates, conforming adjustments will have to be made before the application will be brought forward for review by the GWG (the grant review group).” 

Fast-track

“Rather than require submission of extensive documentation regarding compliance with myriad protocols and processes – some CIRM-imposed and others external – the proposed process will rely on certification of compliance by the applicant, with the ability for CIRM to request supporting documentation if cause to do so arises.” 

Elimination of paper work

“Because most prior approval requests were routinely granted, and therefore added little value to the process, prior approval requests for rebudgeting and carryforward have been eliminated. We have also eliminated prior approval requests for no-cost extensions because our new CIRM awards will have project end dates that will be extended automatically as needed to complete the final Operational Milestone. CIRM intends to increase the latitude for grantees to pursue their research, while maintaining visibility into and approval of any changes to key components of clinical trials, manufacturing processes, or any other activities that meaningfully impact milestones or suspension events.” 

Clinical advisory panels (CAP)  for each award

“CAPs will provide real-time course correction and will focus more on acceleration opportunities than pure evaluation. CAPs will be tailored for the needs of each project and will consist of CIRM and external members, more nimbly sized than prior CDAP panels. CAPs will meet on a quarterly basis (instead of annually with CDAP) and examine all relevant information regarding project progression, possible roadblocks, and avenues for progression.” 

Appeals from rejected applicants

“Finally, in light of the rolling nature of the programs which will allow unsuccessful applicants in many instances to reapply with improvements to their applications, CIRM will limit the grounds for appeal of Scientific Review to those based on demonstrable conflicts of interest (as defined in the conflict of interest policy applicable to GWG members).”

Stem Cell Research Training Program Terminated by California

The California stem cell agency today scuttled its first grant program, a $131 million effort to train stem cell scholars at time when researchers were backing away from the field.

The 29-member governing board voted, 14-5, to end the program that began in 2005 as an effort to demonstrate that the research effort was still kicking while legal challenges threatened its existence. The vote came on a recommendation by the agency's staff.

Arlene Chiu, who is now with the City of Hope, was the chief scientific officer for the agency when the program was launched. She told agency directors said it was time to rethink the program, which was aimed at bringing more scientists into the field. She said,

"Its success has removed its necessity."

Chiu said stem cell research is currently attracting a growing wave of interest among younger scientists. The agency said some training would be included in future research programs.

(Editor's note: The chart on the training program was added to this item after it was initially posted.)

CIRM Press Release on $30 Million in Awards

The California stem cell agency has posted a news release on approval of $30 million for 20 projects to clear out roadblocks hampering the development of stem cell cures. A list of recipients will be forthcoming later, the agency said.

California Okays $30 Million in Awards to Help Remove Stem Cell Roadblocks

Directors of the California stem cell agency today approved $30 million to help eliminate bottlenecks in turning stem cell research into cures.

The action gave the go-ahead to awards that were backed by either its blue-ribbon reviewers from outside of California or the agency’s own staff, plus three additional proposals moved forward by the board.  The agency has already spent $52 million to develop stem cell research tools. Today's round was originally budgeted for $35 million.

After a brief discussion, the 29-member board approved, 9-2, an award to UC Davis researchers who had appealed rejection of a $1.8 million proposal by the staff of the California Institute for Regenerative Medicine (CIRM), as the $3 billion agency is formally known.

The agency did not immediately release the names of the winners. It keeps the names of all applicants secret until after the board ratifies the actions of its reviewers, who make the de facto award decisions for the agency behind closed doors.  The agency is expected to issue a press release on the awards later today. (Here is the link to the press release. It does not include a list of recipients.)

The board did not vote on two applications on which appeals were filed involving material misstatement of facts. Those were deferred  until the March board meeting. The final four numbers of those applications are 7836 and 7678.

Here is a link to the document on this "tools and technology" round submitted to the board by its staff. Summaries of the reviews can be found with the document along with the scores on the winners.

Discussion Underway in $35 Million in California Stem Cell Grant Round

Directors of the California stem cell agency have begun discussion of awards in a $35 million round to remove bottlenecks in stem cell research. Here is a CIRM list of board members that have conflicts on specific applications. Those board members are not allowed to discuss those applications or vote on them.

  Voting Conflict List CIRM Board Meeting 1-29-15 by DavidJensen

California Stem Cell Directors Open Meeting Today in California

Directors of the California stem cell agency opened their meeting today in Burlingame, Ca., at 9 a.m. PST with introduction of new board members.

Former Top Exec at Stem Cell Agency Appointed President of Huntington Medical

The former chief scientific officer of the California stem cell agency, Marie Csete, yesterday was named the president of the $53 million Huntington Medical Research Institutes in Pasadena, Ca.

Marie Csete, Huntington photo

Csete left the stem cell agency in 2009, after serving for a little more than a year. Her departure involved tension between her and former agency President Alan Trounson.  She told the journal Nature,

"When it became clear to me that my considered clinical advice was not respected, I concluded that it made no sense for me to stay at CIRM.”

Csete was the last person to hold the position of chief scientific officer at the agency. Trounson left the agency in July last year.

Huntington reported assets of $53 million in late 2013 and funds a wide variety of research. 

California Stem Cell Appeal: $1.8 Million Needed for Removing Bone Replacement Bottleneck

A UC Davis researcher is making a strong pitch to the California stem cell agency to finance a $1.8 million effort to “resolve bottlenecks in engineering replacement bone and cartilage.”

J. Kent Leach, UC Davis photo

J. Kent Leach, an associate professor in the departments of biomedical engineering and orthopedic surgery, said his proposal addresses “a major hurdle in regenerative medicine of the musculoskeletal system (that) impairs the inherent personalized medicine component of stem cell-based methods.”

In a letter to the agency’s board for its meeting tomorrow, Leach said the research team involved is “arguably the most qualified team in the nation to conduct these studies.” 

Other scientists participating are Laura Marcu and Kyriacos Athanasiou.

Kyriacos Athanasiou
UC Davis photo

Reviewers for the $3 billion agency, who make the de facto decisions on awards, scored the application at 72 and did not approve it for funding. The agency’s staff also nixed the proposal, declaring,

“There is another application recommended for funding in Tier 1 that proposes to optimize and apply the same imaging technology platform to a different test system. In addition, the scientific leadership of the two applications is the same.”

Leach said,

“The co-PI (Dr. Marcu) of this proposal is also PI (principal investigator) on RT3-07879, which is focused on assessing stem cell repopulation and remodeling of engineered vascular tissue constructs. 

Laura Marcu, UC Davis photo

"However, the use of this technology for monitoring the maturation of engineered bone and cartilage, tissues composed of dense matrix reflective of the differentiation of contributing stem cell populations, is substantially different from cardiovascular applications. Of course, both applications involve instrumentation based on optical spectroscopy and ultrasound principles, but the implementation and subsequent commercial hurdles for this technology is very different.”

 (For the summary of reviewer and staff comments, see application RT3-07981 in this document.)

Leach noted that another competing application in the round received an identical 72 score and was approved by the agency’s staff.

The grant round was budgeted for as much as $35 million. Reviewers and CIRM staff recommended approval of awards totaling $29.2 million.

Scientists making appeals directly to the board have not been successful in the last 12 months or so. The board has been more reticent about overturning reviewer and staff recommendations since it changed review and appeal procedures in 2013.

Follow Complete Coverage of Tomorrow's California Stem Cell Meeting

Keep track of all the “thrills and chills” at tomorrow’s meeting of the governing board of the $3 billion California stem cell agency with gavel-to-gavel coverage from the California Stem Cell Report.

This blog will monitor the session via the Internet from our perch in Banderas Bay in Mexico and file stories as warranted. On tap is as much as $35 million in awards. Reviewers and agency staff have approved only $29 million. But three UC Davis researchers are seeking another $1.8 million.  

Also up for action are major changes in the administration of grants – all part of the CIRM 2.0 effort designed to fast-track cash and beef up the quality of applications.

The session, scheduled for eight hours, can also be heard via a listen-only audiocast and Webcast for the slides being used.  Teleconference locations where the public can attend and comment are also available in Sacramento, Irvine and two in La Jolla.  Specific locations and directions for the Internet access are available on the agenda.  

The meeting begins at 9 a.m. PST in Burlingame.

Bump on CIRM 2.0 Speedway: California's Missing Stem Cell Loan Rules

When the applications come in this Friday for the California stem cell agency’s new, $50 million stem cell research round, business applicants will not be seeing a substantial portion of key financial rules for the awards.  

Missing will be the terms of loans that the agency expects to offer to for-profit enterprises. Loans -- rather than grants -- have been the favored method of businesses for receiving awards. The reason is that the agency’s loans have been forgivable if the research does not generate an appropriate amount of cash for the company. Grants, on the other hand, carry requirements for royalty payments. (None have been generated.)

Under the terms of the fast-track, CIRM 2.0 applications, businesses will have the choice of grants or loans. New grant terms are expected to be approved at Thursday’s meeting of the governing board of the $3 billion agency, one day before the deadline for applications for the first round of CIRM 2.0.

Action on new loan rules was originally scheduled for this week but postponed a few days ago after the agenda was posted Jan. 17. The specific reasons are not entirely clear.  The California Stem Cell Report yesterday asked about the delay and for a copy of the latest loan proposal. Kevin McCormack, senior director for communications, replied, 

“We are in the process of revising the (loan policy) and nAeeded additional time to ensure alignment with CIRM 2.0 and the other policy changes so we postponed the meeting.  It will be rescheduled before the March board meeting.
 “And as we are still working on the loan policy it would be premature to share that with you right now, it's still a work in progress.”  

Given that businesses have amounted to a tiny percentage of awards in the past, the delay in the loan rules question may not be significant. But this round is aimed at clinical stage projects, which are more likely to involve businesses and could generate more applications from the private sector.

Some time is available to adopt new rules before CIRM board action on the awards, possibly during an April 23 telephonic meeting. But one of the main points of CIRM 2.0 is to put cash in the hands of recipients significantly faster than in the past. This is especially important for stem cell companies, which are always pretty much cash starved.

Delays in any portion of the aggressive timetable for CIRM 2.0 could mean delays in handing out money.

It is not surprising that some difficulties have arisen in the new award procedures. Randy Mills, CEO of the stem cell agency, last week told an Internet audience of about 200 there would be “some bumps.” He said,

“This is going to be one wild ride.”  

To help smooth it out for both the agency and applicants, it would behoove the agency to post the proposed loan terms in a timely fashion. That means that they should be available to the public when a meeting agenda involving the rules – be it for a subcommittee or full board meeting -- is posted on the CIRM Web site. That is normally 10 calendar days ahead of the session.  The agency, which is costing taxpayers $6 billion including interest, doesn’t need any more mysteries.

$131 Million and 887 Papers: Demise of California's First Stem Cell Grant Program

The $3 billion California stem cell agency on Thursday is expected to put to rest its oldest grant program – an effort that was begun with bravado in 2005 but minus the cash to back it.

It was a matter -- at the time -- of showing the Golden State’s stem cell flag despite legal challenges that had stalled the progress of the agency.  The grants were the agency’s first and came on a late summer day in Sacramento during a governing board meeting marked by confusion and frustration.

David Baltimore
PasadenaNow photo

Board members complained they did not have enough information to make good judgments on the awards. 

Nobel Prize winner David Baltimore, a board member and president of Caltech, objected to what he considered loose standards in evaluating the applications for millions of dollars.

At one point, a crusty reporter from The Associated Press, Paul Elias, jumped up and demanded to know exactly what was going on.

The headline on the story the next day in the California Stem Cell Report said, 

“CIRM Hands Out $39 Million (sort of)….”

The awards created a training program for stem cell scientists. It was aimed at attracting more researchers into a field that was atrophying because of the Bush administration’s restrictions on stem cell research funding. 

Without adequate financial resources, the California Institute for Regenerative Medicine (CIRM), as the agency is formally known, appealed to the recipient institutions to advance the funds with the expectation they would be reimbursed later.

Zach Hall, who was appointed president of the agency the same day, called the awards “historic.” Robert Klein, the chairman of the CIRM board, said he was thrilled by the program.

One of the major goals of the board action was to show that the agency and that its tiny staff (something less than 20) was still kicking despite the legal dispute that blocked issuance of the bonds that provide funding for the agency. 

In that regard, CIRM was more than successful.  The meeting was well-covered by major newspapers in the state, a far cry from the situation today. The message in the stories was largely favorable. The California Stem Cell Report wrote on Sept.11, 2005,

“Subordinated was the reality that the agency does not yet have the money. Even more deeply subordinated were complaints about the grant process from critics.”

The initial awards ultimately grew into a nine-year, $131 million effort. In a Jan. 20 memo to the CIRM board, the agency’s current president, Randy Mills, said,

“The programs have trained 859 CIRM Scholars. These trainees have worked in 436 different laboratories on a broad range of research projects, with the majority focused on basic research. CIRM Scholars have been authors on 887 scientific publications.”

The average cost for each “scholar” was about $152,000, Mills reported. The California Stem Cell Report calculated an average expense of about $148,000 for each scientific article, although such articles usually have more than one author. Mills said 34 percent of the recipients were graduate students, 48 percent post-doctoral and 18 percent clinical fellows.

Not noted by Mills was the fact that nearly all the awards went to institutions linked to members of the agency’s governing board. Virtually no other institutions existed, however, in California that could provide the training.

In December 2013, the board asked the agency staff to prepare a new version of the training program, a task that was not completed before the board hired Mills last spring.

In his memo last week, Mills said that “the CIRM team determined that a new training grant program is no longer an optimal method of supporting the education and training of stem cell scientists.” He continued,

“CIRM believes that supporting the training of new stem cell scientists is best accomplished by bolstering funding for our research grants programs, particularly the earlier discovery and translational phases where each program can be individually evaluated for its merit and contribution to CIRM’s mission.”

Mills said he will present concept plans for new discovery and translation rounds, including training within them, during the next six months. And he asked the board to approve his recommendation to drop the plans continue the old, once-hailed training program.

A California Stem Cell Mystery is Solved

The California stem cell agency today cleared up one of the mysteries involving next Thursday’s meeting of its governing board and likely action on a training program.

The agency last Saturday posted the agenda for the meeting that included a cryptic description that appeared to involve the abolition of one of its several training program. The agency did not respond to a query last Monday about exactly what was meant by the agenda item, making it nearly impossible for affected parties to respond in an appropriate and timely fashion.

Following the posting yesterday on this blog of an item describing the situation -- "The California Stem Cell Agency and Its Unnecessary Mysteries" -- the agency added a memo early today from CIRM President Randy Mills to the Thursday agenda. The memo explained that what was involved was the CIRM Scholars Program.  

Look for an item on this Web site early Monday on the expected end to the program.

The California Stem Cell Agency and Its Unnecessary Mysteries

For the second month in a row, the California stem cell agency, which sits on top of $3 billion in taxpayer cash, is playing a mystery game with the public.

Coming up next Thursday is a meeting of the 29-member board of the agency. These sessions are the most important public events involving the agency, which operates outside of normal state oversight. They deserve full transparency.

Without it, affected parties -- not to mention the people who pay the $6 billion total bill (including interest) for the stem cell research – do not have a genuine opportunity to comment and make suggestions about matters that involve their livelihoods and organizations.   

The latest problem primarily involves a cryptic agenda item for next week's board meeting that says only “consideration of the CIRM team’s determination not to present concept proposal for training program at this time.”

The agenda containing the item was posted last Saturday on the agency's Web site. The agency has not posted anything further on the matter and has not responded to a query five days ago seeking more information.

Training issues have been a subject of considerable concern and interest in the stem cell community, both at the state college level, which has received little of the agency’s cash, and universities and research institutions.

Backed by 11 top researchers, Jeanne Loring, head of the stem cell program at Scripps, has said the “Bridges” shared laboratory and training program has “provided the infrastructure upon which California’s reputation as the center of the stem cell universe was built.” (See also here.) 

Susan Baxter, executive director of the California State University’s system-wide program for biotechnology, said,

“Despite the Great Recession, Bridges graduates have succeeded in landing jobs and gaining admittance to graduate and medical schools at much higher rates than peer groups.” 

Does the training agenda item refer to the concerns of Baxter, Loring and the others? It could be a reference to the agency's high school training effort or something else entirely. It is totally unclear and unnecessarily so.

If the agency is fulfill its promise of maximum transparency, it should post ample background material on matters to be discussed when it posts the agenda 10 calendar days ahead of upcoming meetings. In the absence of that, however, it would have taken only five minutes or less to write a line specifying which training programs are up for consideration.

  

In addition to the mystery about the training programs, not to be found is information about proposed changes in the agency’s loan program. Those are scheduled to be approved next Thursday, only three business days from today. The changes likely will have a significant impact on businesses.  A board subcommittee was scheduled to consider them on Monday, but that meeting has been cancelled for unknown reasons. The agency has not responded to a query on when the session will be rescheduled, but presumably it will happen before the board meeting on Thursday.

Last month, the agency also created a similar mystery. It involved what turned out to be a $50 million program that deserved more timely attention from affected parties and the public.

In years past, the agency was infamous for failing to post material for its meetings in a timely fashion. Even board members sometimes complained about not having information. Sometimes meetings were postponed.

Since Jonathan Thomas became chairman in 2011, the situation has improved.  Randy Mills, the new president of the agency, is stressing "clarity" in what the agency does. However, the mysteries of the past two months do not auger well. The agency can and should do better. 

Webinar Now Available on Fast-Track Plan to Hand Out $1 Billion in California Stem Cell Cash

The ins-and-outs of CIRM 2.0 – at least some of them – are explained by the CEO of the California stem cell agency, Randy Mills, in a Webinar now available on the agency’s Web site.

The Webinar includes presentation slides and a Q&A. About 200 persons logged in for the session earlier this week on the new, fast-track effort to unload the agency’s next $1 billion. Kevin McCormack, senior director for communications, said after the Webinar,

"Of the 234 people who registered and, at some point tuned in today (Jan. 21), 108 were industry. Over the course of the 50 minute webinar some tuned in and dropped out so there was a rolling audience with 200 being the most tuned in at any one time."

If you are one of those who would like some cash from CIRM, this is must viewing. The 51-minute session can also be downloaded for later consumption.

Faster Cash but Tougher Competition for California Stem Cell Researchers

A slide from today's Webinar on CIRM 2.0

Randy Mills, who has led California’s $3 billion stem cell agency just since last May, today told a cyberspace audience that his agency will finance only the “absolute best” proposals under his new “2.0” version of the research effort.

Mills addressed about 200 persons who logged into a Webinar to learn more about the radical changes that began this month at the California Institute of Regenerative Medicine (CIRM), as the agency is formally known. Mills has dubbed the new direction CIRM 2.0.  And he said,

“CIRM 2.0 is going to be highly, highly competitive.”

It was a theme that the CIRM CEO came back to several times. He advised listeners that the competition will be tougher than in the past and that the agency will be more discerning.

CIRM 2.0 is aimed at both improving the quality of applications and at speeding money into the hands of researchers, cutting the time from application to funding from an average of 22 months to four months.   Mills intends to extend the new process to basic research and translational research later this year. 

Mills, the former CEO of Osiris Therapeutics in Maryland, said,

“We’re in the time business at CIRM.”

Mills said he expects the quality of applications to improve as the agency becomes more engaged in working with researchers to build proposals that will meet the agency’s needs.  CIRM will additionally become a partner with successful applicants and provide outside, expert help to overcome such things as logjams involving possibly the FDA or manufacturing problems, Mills said.

One of the major changes in CIRM 2.0 involves timing of grant rounds. Instead of rounds a year or more apart, Mills said,

“The application window is always open.”

The agency kicked off the new program this month with clinical stage programs. The deadline for filing applications is a week from this Friday(Jan. 30). No applications have been filed as of today.  No worries, Mills basically said. Applications will be accepted again next month with a deadline of Feb. 28 and every month thereafter with the deadline falling on the last business day of each month.

The agency has strict legal requirements to limit its spending to California operations. But in a Q&A session during the Webinar, Mills made it clear that a foreign-based company was eligible to seek funding for research activities that were being conducted with the Golden State.

In a brief telephone interview later, Mills said today's session was part of a "massive" effort on the part of the agency to spread the word about research funding opportunities involving CIRM.  He said he has been surprised about the lack of awareness -- outside of a relatively small circle in California -- of the availability of CIRM cash for biotech enterprises that traditionally have been struggling for funding.  

During the Webinar, Mills also acknowledged that CIRM 2.0 is new and is likely to encounter “some bumps” that he said the agency will work to solve. Nonetheless he told the researchers,

“This is going to be one wild ride.”

Mills’ Webinar on CIRM 2.0 is expected to be posted on the CIRM Web site either later today or tomorrow.

A Show-and-Tell Tomorrow on Handing Out California's Next $1 Billion for Stem Cell Research

Want to know more – from the horse’s mouth, so to speak -- about how California’s stem cell agency plans to give away its next $1 billion?

The public and researchers have a chance to hear directly tomorrow, via the Internet, from the agency concerning its plans for CIRM 2.0, the name of its new fast-track plan for handing out cash.

Registration is required, however, for the Webinar, which is scheduled for 12 p.m. PST.  CIRM says,

“Questions may be submitted in advance of the Webinar via email to cschaffer@cirm.ca.gov or via Webex registration.  During the webinar questions may be submitted via the Webex Q&A functionality.”

CIRM 2.0 was initiated by CIRM President Randy Mills as part of his efforts after he was appointed last spring. Mills will be personally presenting the proposal for the Webinar.

The agency says the plan is intended to put money in the hands of researchers with greater alacrity and improve the quality of grant applications. The agency describes the changes in the award process as radical.  Read more about it here, here and here. 

California to Pump $35 Million into Removing Stem Cell Therapy Roadblocks

California is set to give away as much as $35 million later this month to create tools and technology to help researchers develop the stem cell therapies that were promised to voters 11 years ago.

The idea behind the awards is to eliminate bottlenecks that block commercialization of research. California’s $3 billion stem cell program was created by voters late in 2004 with the expectation that it would quickly lead to stem cell cures.  It has spent about $1.9 billion so far but no therapies have reached the marketplace.

The latest, $35 million round is budgeted for as many as 20 awards of up to $1.2 million each to be distributed over a period as long as three years.  The $1.2 million is for "direct" research costs. CIRM would also pay for "indirect" costs that raise the total substantially.

Applications were sought last year from both academics and businesses with possible collaboration involving out-of-state partners, including China, Australia, Germany and Brazil.  Non-California enterprises would not receive funding from the state agency.

Action on the applications is part of the agenda for the Jan. 29 meeting of the 29-member governing board of the California Institute for Regenerative Medicine (CIRM), the formal name of the stem cell agency.  

The round is not part of the new fast-track program, CIRM 2.0, initiated by CIRM President Randy Mills. The “tools” effort contains different timetables, among other things. The work must begin within six months of approval of the awards, considerably longer than the 45 days specified under CIRM 2.0, which began just this month.  The old process also contained a pre-application review that has been eliminated.

The board is additionally expected to approve new rules for CIRM 2.0 awards, including loans to businesses.  Given that the deadline for applying for the first round of CIRM 2.0 is Jan. 31, potential applicants would be well-served to scrutinize the proposed new regulations. Interested parties can make comments or suggestions in advance of the board meeting and the related sessions of the board’s Science Subcommittee on Tuesday and the Intellectual Property (IP) Subcommittee on Jan. 26.  

The grant rules can be found on the Science panel agenda. The new loan rules have not yet been posted on the IP agenda. Comments can be sent by email to mbonneville@cirm.ca.gov or made at one of the public meeting sites. 

Public teleconference sites for the Science Subcommittee session Tuesday include San Francisco, Oakland, Irvine, Duarte, La Jolla and Sacramento. Addresses are on the agenda.  The only public site listed currently for the IP subcommittee is San Francisco.

The Jan. 29 full board meeting will be held in Burlingame but remote teleconference locations are also available where the public can participate. They currently include locations in Irvine and La Jolla, but that could change prior to the meeting.  Addresses are available on the agenda. 

California Stem Cell Agency Seeks Five Executives in $3 Billion Program

California’s $3 billion stem cell agency has embarked on a wave of fresh hiring of well-paid executives to fill top slots in the new structure created by Randy Mills, who has been the chief executive of the program since last May.

Five positions are open with salary ranges that hit $244,204 annually.  All of the announcements are emblazoned with the new CIRM 2.0 logo, emphasizing the “radical” changes underway at the agency. The program is formally known as the California Institute for Regenerative Medicine or CIRM.

The postings amount to the largest number of top level positions opening at the agency in a number of years, perhaps the largest since its inception 10 years ago.  Total employment at research funding program is slightly more than 50 persons and is not expected to increase significantly.

The open positions include “directors” in the following areas:  medical affairs and stem cell centers, blood and cancer therapeutics, neuro/ocular therapeutics, organ systems therapeutics and administration, which includes everything from public relations to information technology.

The agency’s governing board in December approved Mills’ reorganization with little discussion. He said it would flatten the organization, making it speedier and more efficient.

The new structure eliminated a number of positions, including that of Ellen Feigal, who was senior vice president for research and development, then the No. 2 position at the agency. She resigned last October, giving two weeks notice. At the time, she was the fifth person to leave the agency following the hiring of Mills.

Under Mills’ organization, the agency does not have a No. 2 person or a chief scientific officer. The chief scientific officer position was eliminated years ago, although it is common in many biotech companies.

A comparison of the new and old organization can be found here.

Here is a look at some of what the new positions entail:

Director, Medical Affairs and Stem Cell Centers: Oversight of “the Alpha Stem Cell Clinics, the CIRM Stem Cell Genomics Centers of Excellence, and the CIRM hiPSC Initiative with a total budget of over $90 million.”

Director, Blood and Therapeutic Area: Oversight of more than “20 active programs and a budget of over $200 million. Projects in the therapeutic area range from translational research through human clinical trials.”

Director, Neuro/Ocular Therapeutic Area: This area “currently has over 25 active programs and a budget of over $200 million. Projects in the therapeutic area range from translational research through human clinical trials.”

Director, Organ Systems Therapeutic Area: This involves more than “25 active programs and a budget of over $200 million. Projects in the therapeutic area range from translational research through human clinical trials.” 

Director, Administration:  Oversight of Communications, Information Technology (IT), and Human Resource departments; “Importantly, the Director is also responsible for managing all Governing Board activities and communications.”

All of the positions have a salary range of $167,877 to $244,204 and report directly to Mills.

No deadline exists for filing applications. The announcements said applications will remain open until the positions are filled. Mills, however, moves with dispatch. Interested persons should submit applications quickly if they want to be considered.

Fast Cash in California for Stem Cell Research; $50 Million Up for Grabs

The stem cell agency's new logo to promote CIRM 2.0

California’s $3 billion stem cell agency this month is kicking off the first round of radical changes in how it finances research, including funding of the clinical trials that it hopes will push nascent therapies into widespread use.

The money is scheduled to start flowing to scientists in the Golden State as soon as June in a new, fast-track program that is called CIRM 2.0. But only if the researchers file their applications within the next 19 days. Slow-moving researchers, however, will have a chance to file again at the end of next month and each following month.

The effort has all the earmarks of a venture capital program with heavy involvement by the financing party (CIRM), involving both the budget and the science.  The agency, known as CIRM after its formal name of the California Institute for Regenerative Medicine, last Friday repeated its pledge to be an “active investor,” which may or may not be welcomed by some researchers.

Nonetheless, Los Angeles Times columnist Michael Hiltzik recently wrote that scientists would find a “lot to like” in the new direction at the agency. Here is how CIRM put it in a news release Dec. 31 that contained links to the detailed program announcements, 

“CIRM 2.0 is a radical overhaul of the way the agency does business, implementing efficient new systems and programs that place added emphasis on speed, partnerships, and patients.

“CIRM 2.0 makes it easier for both companies and academic researchers with promising projects to partner with CIRM to get the support they need when they need it, reducing the time from application to funding from around two years to just 120 days.”

The release laid out some of the aspects of the new grant review and funding process, which the agency expects to extend to all its remaining $1 billion in award programs, 

“Speed:  In addition in to reducing the time to funding to 120 days, new clinical stage projects may be submitted to CIRM year round instead of only once or twice a year as in the past. Applications simply have to be filed by 5pm PT on the last business day of the month to be eligible for consideration in that round of review. If you miss the deadline one month, you only have to wait 30 days for the next one.

“Partnerships:  Under CIRM 2.0, the agency will not act as a passive funding source, but instead will be an active investor, devoting significant internal resources and leveraging its vast external team of world-class subject matter experts to advance the projects it selects. This will result in a true partnership that both accelerates programs and gives them the greatest opportunity for success.

“Patients: Each project will be partnered with a project-specific Clinical Advisory Panel  (CAP) to help advise and guide it forward.  Importantly, every panel will include at least one patient advisor with first-hand experience of the specific condition, who will provide input, recommendations and the appropriate sense of urgency that can only come from the unique perspective of someone living with the disease.” 

The agency has allotted $50 million to be handed out between now and July for programs involving clinical trials or preparation for clinical trials.  Applicants can ask for as much cash as they desire. But their proposed budgets will be scrutinized by an outside panel and subject to possibly substantial revision. If CIRM does not fancy the researcher’s figures, the application will not be presented to reviewers.  

The budget review is a significant change from past procedures.  It is just one of many changes, small and large, that scientists need to understand about the new process. Others involve restrictions on appeals, solvency requirements for businesses and, in some cases, hefty co-funding requirements that must be documented in advance. In-kind support, for example, will no longer be acceptable as  matching cash or for co-funding.

If scientists want to be competitive, they would be wise to carefully review the program announcements posted at the first of the month. In some cases in years past, applicants have lost out simply because they failed to understand in a timely fashion all the nuances of the process.

Other changes include the following:

• Businesses are limited to 10 percent indirect costs compared to 20 percent nonprofits, a requirement backed by CIRM board members representing nonprofits.  The agency said no legal conflict of interest existed for board members from nonprofits who voted for the bigger indirect allotment for their enterprises.
• Budgets must contain contingency plans for financing should expenses exceed initial estimates.
• Applicants must be ready to work within 45 days of CIRM board approval of their applications.
• The agency – not grant reviewers -- will determine whether applications meet eligibility requirements. If they do not, they will not be reviewed. An appeal by an applicant may be made behind closed doors to the grant review group.
• No public appeals are formally allowed. However, state law permits all members of the public, including researchers, to speak directly to the board on any issue. That avenue for seeking funding, however, has not been very successful in recent months. 

The grant review group is likely to continue to make -- behind closed doors -- virtually all the de facto decisions on application approvals. Their actions will come in three "recommendations" to the board: fund the application, do not fund but allow re-submission or do not fund and do not allow resubmission.  The CIRM board will then ratify in public the group’s decision, as it has almost invariably in the past. The board has the legal authority to do anything it wants with applications, but it has rarely overturned positive decisions by the board. Infrequently, it would approve some lower ranked applications. The new procedures would seem to make any public appeals to the board by researchers even less likely to be successful.

Randy Mills, CIRM photo

CIRM 2.0 is the brainchild of Randy Mills, who took over the presidency of the agency last May. He has launched a major effort to spread the word about the move. It includes a Jan. 21 “webinar” conducted by Mills to discuss CIRM 2.0. Advance registration is required.

On Jan. 20, the CIRM directors Science Subcommittee is scheduled to vote on new grant administration rules involving CIRM 2.0. That is another matter that researchers who wish to be successful in securing funds should be following and making recommendations on.  Sites where they can participate are in San Francisco, Oakland, Irvine, Duarte, La Jolla and Sacramento. See the agenda for specific addresses.

Gil Sambrano continues to head the review process, albeit with a new title: director of portfolio development and review. His phone number and email can be found on the program announcements posted at the first of the month (see here, here and here). 

For additional news stories on CIRM 2.0, see here and here.

LA Times: Researchers Sure to Find 'Lot to Like' in CIRM 2.0

The Los Angeles Times, the newspaper with the largest circulation in California, today took a crack at the California stem cell agency and its “reboot” as CIRM 2.0.

The piece was written by Michael Hiltzik, the Pulitzer Prize-winning columnist who has often been a critic of the $3 billion state research program.  The springboard for the article was the restructuring of the agency’s grant process, which its new president, Randy Mills, has dubbed CIRM 2.0.  The abbreviation comes from the formal name of the agency, the California Institute for Regenerative Medicine (CIRM).

Hiltzik wrote,

“Biomedical researchers are sure to find a lot to like about CIRM 2.0, especially Mills' commitment to streamline the program's grant and loan approval process for projects aimed at clinical trials of potential therapies. Reviews of applications take about 22 months on average; Mills hopes to cut that to about three months.”

Hiltzik said the new effort, which began just this month, was initiated by Mills after “he reached the conclusion that 'there was a lot of room for improvement.’”  Hiltzik wrote,

“That's a striking admission for a program that already has allocated roughly two-thirds of its original $3-billion endowment.” 

Hiltzik discussed the pressure to fulfill the public expectations for quick development of stem cell cures that were generated by the 2004 ballot campaign that created the agency in 2004. He also addressed the pressure to find new sources of funding by 2020, when the money is expected to run out.  Hiltzik wrote,  

“Mills says winning approval for more public funding isn't the goal of CIRM 2.0. ‘It's not our job at CIRM to extend the life of CIRM,’ he told me. Instead, he couches the need for urgency in terms of serving patients.”

Hiltzik continued, 

“As head of CIRM, however, Mills can't escape the tyranny of public expectations. CIRM's mandate, as he describes it, is ‘to accelerate stem cell treatments to patients with unmet needs.’ That presupposes that stem cell treatments exist or will be found for the major medical conditions at which Proposition 71 was aimed. What if science and nature don't cooperate?”

Hiltzik also touched on the competition involving funding of clinical trials and funding of basic research.

Arnold Kriegstein in front of CIRM-financed stem cell
research building -- UCSF photo

“Academic researchers may have a legitimate concern about too much money shifting toward late-stage research prematurely. ‘The field is so young,’ says Arnold Kriegstein, director of the stem cell lab at UC San Francisco, ‘that it's unreasonable to expect that fully transformational therapies are just a few years away. This is the time to continue basic research programs, not cut them off.’”

Hiltzik concluded, 

“Despite the program's unquestionably positive impact on stem cell science, especially in California, it still lacks a coherent sense of its proper role. CIRM 2.0 is the latest effort to find that role, but it may not be the last.”