Former Chief of California Stem Cell Agency Returns to California as Head of Sanford Burnham

C. Randal Mills, who led California's $3 billion stem cell agency for three years, this afternoon was named as chief executive officer of the Sanford Burnham Prebys Medical Discovery Institute in La Jolla, Ca.

Randy Mills
Sanford Burnham photo

The institute said in a news release that Mills brought to Sanford "decades of experience as an entrepreneur and transformational leader in the biomedical industry."

Most recently, Mills was president of the National Marrow Donor Program/Be The Match nonprofit, an international leader in facilitating and supporting bone marrow transplantation.

Mills left Be The Match in February of this year, citing personal reasons.
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Mills, who goes by Randy, was  president and CEO of the California stem cell agency from May 2014 until July 2017. 

During his time at the agency, which is formally known as the California Institute for Regenerative Medicine (CIRM), Mills instituted something he called CIRM 2.0. It streamlined and sped up the work of the agency, sharpening its focus with an emphasis on performance metrics. The structure of the effort remains in place today. 

When Mills left the agency, CIRM Chair Jonathan Thomas, who recruited Mills, said his efforts had created a "bold strategic plan, the results of which include an 82 percent reduction in approval time, a 3-fold increase in the number of clinical trials, and a 65 percent reduction in the time it takes to enroll those trials."

Sanford Burnham has received $39.4 million from CIRM over the years. Its president, Kristiina Vouri, sits on the governing board of the stem cell agency, which has included a Sanford representative since 2004. 

Vouris said in the news release that Mills "comes to us with extensive organizational and executive leadership experience. His diverse background and knowledge of translational research and drug discovery will help us propel the Institute into a bright and successful future.”

Mills will begin his work immediately at Sanford Burnham, which has 700 employees in the San Diego area.  

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."

Solid Hook-Up: California Stem Cell Agency and Calibr Connection

Highlights

Creating pathway to clinic

The Calibr approach

Problematic pharma-academic ties

As of today, the California stem cell agency has a $4 million hook into a San Diego research enterprise that also has ties to a glittering array of partners, ranging from Merck to Bristol-Myers Squibb. 

The connections fit with the state stem cell agency’s new goal of creating a clear pathway from the basic research bench to the use of stem cell cures in clinics for millions of people.

The signal event came routinely and quickly this morning when directors of the California Institute for Regenerative Medicine (CIRM), as the agency is formally known, approved a $1.7 million award.

The cash is going to the California Institute for Biomedical Research (Calibr) and its director, Peter Schultz.

The funds will be used for pushing into clinical trials a treatment that would -- for the first time -- modify the progress of osteoarthritis, which afflicts 27 million Americans. The therapy could also be used to treat damaged cartilage in knees and elbows.

CIRM has now invested $10 million in Schultz, a reknown chemist and biotech entrepreneuer. Six million of that went for research that was performed at Scripps. All of the funds involve the proposed arthritis therapy. Including today’s award to Schultz and funds to other researchers, the agency has now handed out $19.5 million to find a therapy for arthritis.

Schultz founded Calibr three years ago with up to $90 million, promised over seven years by Merck. The Big Pharma firm has first refusal rights on treatments developed by Calibr. If Merck passes, then other enterprises can make an offer. Calibr has additional partnerships, including a $28.9 million, five-year relationship with the Gates Foundation.

Peter Schultz, catching a big one

Schultz says “our mission is a little bit arrogant.” In a lengthy piece in March by Bradley Fikes of San Diego Union-Tribune, Schultz said Calibr has “a very broad spectrum of interest and we hope to have a significant impact on a number of diseases."

Fikes wrote,  

"'There are a lot of discoveries being made in life science, and many of these discoveries are made in academic institutions and basic research institutions,’ Schultz said. ‘The process of translating those discoveries into new medicines has become somewhat difficult.’

“These institutions have trouble collaborating with big pharma because of differences in culture and regulatory challenges of for-profit and not-for-profit collaboration.

"'That's left the universities try to do drug discovery themselves,’ Schultz said. ‘The problem there is they don't have the knowledge, experience or even the processes to do drug discovery, which is a lot different than a cottage industry approach to basic research."

Fikes continued,

“Meanwhile, big pharma is becoming more risk-averse, looking for validated drug targets and in-licensing compounds. That makes pharma collaboration with basic research centers even more problematical, Schultz said.

"’We have to come up with other ways to really facilitate and accelerate the translation of new research into new medicines,’ Schultz said. ‘Universities are trying to set up their own drug discovery units. Scripps has done that in Florida, Vanderbilt, we see Cornell, Rockefeller and Sloan-Kettering teaming up to do that. They're building something from scratch, and usually without having the experience of having been there and done that. And in some cases what they wind up doing is hiring a lot of people from pharma industry and recreating a pharma-like process.’"

Calibr has 110 employees, Fikes reported, including 60 postdocs. Its annual operating budget for 50 projects totals $25 million. Calibr is still busy hiring with seven positions open on its Web site as of this afternoon.

More on CIRM's Restriction of Info on Biotech Applicants

The California stem cell agency says its brief statement this week on restriction of information involving biotech companies was generated in an effort to offer a public explanation of its policy.

In response to a query about what led to the statement, Maria Bonneville, executive director of the CIRM board, said,

"In light of CIRM's efforts to work with companies to develop new therapies, we felt it was important to offer a public explanation regarding CIRM's efforts to balance its obligation to provide information to the public with the need to protect the proprietary information of applicants for therapy development projects."

The California Stem Cell Report will have more on this subject in the next week, including a discussion of how it relates to the state's constitution, which states that the public has as "broadly construed" right to information about state government activities.

CIRM Okays First Clinical Trial Round; $50 Million Earmarked

Directors of the California stem cell agency this morning unanimously approved a $50 million foray – its first-ever -- into the financing of clinical trials.

The program appears likely to be of benefit to only a handful of organizations – particularly the three that have pluripotent proposals that are now on hold with the FDA. The names of the enterprises were not disclosed during the board meeting because to do so would raise conflict of interest questions.

The CIRM staff proposed one or two trials that would be limited to “novel cell therapies from pluripotent stem cells.” Both businesses and non-profit institutions could compete, but businesses would be required to take a loan instead of a grant. In both cases, matching funds would be required. During the board discussion, the CIRM staff provided two examples of matching requirements. CIRM would provide a $20 million grant on a $40 million trial with the rest to be provided by the applicant or $25 million on a $75 million trial.

The goal, a CIRM document said, is

“completion of early stage clinical trials within three years that: 1) demonstrate preliminary safety in humans and 2) provide compelling data for proof of mechanistic concept and/or early testing for efficacy that could lead to more definitive efficacy studies.”

Several board members said the clinical proposal, which could lead to awards by the end of this year, represented exactly what CIRM should be doing. Ted Love, a board member and executive vice president of Onyx Pharmaceuticals of Emeryville, Ca., said,

“We don't have the luxury of waiting.”

He and others, such as patient advocate board member Jeff Sheehy, cited the need to bring therapies to the point where they can actually be used to treat suffering persons.

Some board members did express concern about the possible appearance of favoritism and the need to ensure that the proposal contain sufficient funds to monitor clinical trial patients for long periods, perhaps their lifetimes.

CIRM President Alan Trounson also said that he understands that some of the potential applicants are facing serious cash problems and need to find funding soon. He said the clinical trial proposal also might bring out more applicants than the three possibilities he is aware of.

Under the plan, Trounson would be given significant discretion to deal with the needs of applicants.

The ambitious proposal is part of CIRM's drive to bring therapies to market. More funding for clinical trials is expected to be provided by CIRM within the next year or so. The agency has already committed one-third of its $3 billion in resources. It will need to show tangible, publicly appealing results if it is secure additional funding in a few years.

Here is a link to the CIRM press release on the program. 

CIRM Seeking More Industry Experience in Top Science Officer; New Post Proposed

The California stem cell agency wants to put more sizzle into its search for a replacement for Marie Csete, who resigned as its chief scientific officer recently and subsequently said her advice was not respected.

Alan Trounson, president of CIRM, is asking its directors to create a new post called vice president, research and development that will be attractive to scientists with commercial experience. The search comes just as the agency is about to award its critical disease team grants, which, at $210 million, are the largest research grant round in CIRM history.

Csete's resignation and her complaints about the organization have received international attention and raised questions about CIRM's management, which the agency has not responded to publicly.

John M. Simpson, stem cell project director for Consumer Watchdog of Santa Monica, Ca., said Csete's position was the second most important at CIRM. He said,

“Before this new position is created, I think there needs to be a full, public explanation of why the former chief scientific officer, Dr. Marie Csete, resigned after a little more than a year on the job.”

In a memo to CIRM directors, Trounson said the new position would provide an opportunity to “find someone with the skill base to have more of a focus in their role with biotech/pharma – translation – clinical applications, which is where we are moving with our translational, disease teams and clinical grants and where we are thin in capacity.”

The new vice president would report to Trounson and apparently rank at the same level as the vice president for operations, John Robson. The new person would be the third in four years to fill the post or its equivalent.

Trounson's proposal, which will come before CIRM directors Aug. 6 in a special teleconference meeting, did not specify a salary range. Don Gibbons, chief communications officer for CIRM, did not respond to questions about pay. Currently the salary range for Robson and the chief scientific officer tops out at $332,000. Csete was paid $310,000.

Trounson's memo said that the new position is designed to attract applicants with “academic-commercial R&D experience.” Trounson said,

“Pharma is now wishing to engage with us and we need some experience at this interface which we haven’t had to date. The biotech firms had been a bit put off due to a lack of success in granting but are now very keen to re-engage if we can solve some of the issues that have arisen.“

Trounson said the new VP would have primary responsibility for relations with the FDA and the NIH. He said the person would “oversee the basic science and translational to clinical program and partner with the executive director of scientific activities to support and empower the science team.”

The designation of an “executive” director of scientific activities also appears to be new. The current CIRM organizational chart refers only to a “director of scientific activities.”

The CIRM president's memo said a “number of very good people” have indicated an interest in the new VP post.

The Aug. 6 meeting is open to the public and is available at teleconference locations throughout the state, including San Francisco(3), Los Angeles(3), Sacramento, La Jolla(3), Pleasanton, Healdsburg, Duarte, Stanford, Berkeley, Irvine and Palo Alto. The specific addresses can be found on the agenda.

CIRM Eyeing Major Reliance on Industry Cash

The president of the California stem cell agency, Alan Trounson, says it is considering funding its future with money from the biotech industry.

The possibility of a state government agency relying on industry dollars raises significant and new conflict of interest questions concerning the $3 billion effort. CIRM's governing board is already filled with representatives from institutions that have received the bulk of its funding.

Trounson made his comment June 4 in an interview with Bloomberg News. He also told the financial news service that it “wants to get 10 to 12 new therapies into human testing within four years.”

Both the comment about industry funding and human clinical trials appear to be the first public remarks along those lines by CIRM officials. However, we presume that Trounson may have misspoken on the human clinical trials, although Bloomberg has not run a correction as of today (June 8). The Bloomberg article is being circulated nationwide via email by a variety of persons interested in stem cell research.

The stem cell agency, now the largest funding source in the world for human embryonic stem cell research, is unusual financially in California. CIRM relies on state bond funds for its operations and grants. That source of cash is not subject to cuts by the governor or the legislature, only the ability of the state to sell bonds.

CIRM was sold to voters in 2004 as a 10-year program. However, no sunset provision is in place for the agency. It does have a 10-year limit on its bond funding. We have been told that clock started running with the first issuance of its state bonds in 2007.

Here is how Rob Waters of Bloomberg wrote about Trounson's comments on biotech funding for CIRM,

“In the long run, the agency may try to use industry funding to continue operating once its $3 billion in state bond revenue is exhausted by the end of the next decade, he said.”

As far as we know, no California state department relies on donations from private industry for its funding. But CIRM has the unusual capability of creating a nonprofit agency, rare among state departments. A nonprofit could serve as a vehicle for channeling industry funds to CIRM.

CIRM has been edging closer to industry over the last year with the staff's proposed revision of its strategic plan calling for much tighter ties.

Trounson's comments about future reliance on industry funds casts a new and different light on all of the agency's current actions. Is a decision on a grant being made because it will enhance or harm future funding from industry? Will CIRM's standards for its research grants be altered because of the need to be friendly to an important biotech company or a top researcher tied to a biotech firm? Will CIRM create, as is proposed, a special “biotech advisory group” to help evaluate CIRM interaction with industry?

The advisory group is recommended in the revisions to the CIRM strategic plan and has already generated controversy, along with CIRM's direction away from basic research and towards efforts favored by industry.

(You can find CIRM's plans on industry linkages in the draft of the strategic plan, beginning on page 28.)

CIRM already has several representatives from industry on its 29-member governing board. One wonders what would be gained by creation of this new panel, except that it presumably could meet privately and discuss matters that might be deemed inappropriate for airing at board meetings, which are required by law to be public.

The question of the long-term outlook for CIRM came up last month a meeting of the Little Hoover Commission, the state good government agency which has drafted major recommendations for changes in CIRM's structure. The draft proposals suggested CIRM explicitly lay out its plans for sustainability or shutdown. Representatives from CIRM at that meeting did not respond to the comment.

The question of future funding is already on the agenda of the CIRM directors' Finance Subcommittee this Thursday. It would certainly seem that Trounson's comments are suitable for some discussion in that context. The public can take part in the meeting at a number of teleconference locations throughout the state. Their specific addresses can be found on the agenda.

As for Trounson's remarks on human clinical trials, we assume that Trounson was referring to the $210 million disease team grant round, the second largest in CIRM's history. The RFA calls for up to 12 grants with the objective that grantees file an investigational new drug application within four years of the start of the award. Those applications are one step along the way to human clinical trials.

We have asked Don Gibbons, CIRM's chief communications officer, if CIRM has any comment on Trounson's remarks to Bloomberg. We will carry any remarks if Gibbons responds.