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.
. 
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.  

Former CEO of California Stem Cell Agency Leaves Top Post at Minnesota Non-profit

Randy Mills, the former head of the California stem cell agency who left it in 2017, has resigned as president of a large bone marrow non-profit known as "Be The Match" in Minneapolis.

A brief news release from the organization said he was leaving for "personal reasons."   Local news coverage of Mills' departure in Minnesota was scanty. Be The Match reported income of about $418 million in 2018.

Maria Millan, then vice president for therapeutics at California's stem cell agency, succeeded him as CEO and president of the agency and still holds that position. Mills was highly regarded at the agency and has been credited with helping to sharpen its focus and direction.

Seeking a CEO: $3 Billion California Stem Cell Agency Faces Critical Leadership Challenges

California's 12-year-old stem cell research effort is expected to give away tens of millions of dollars in public this week, but its most important matters -- issues that deal with its survival and future -- likely will be discussed behind closed doors at a meeting Thursday of its governing board

On the table is the leadership of the $3 billion organization, which is scheduled to run out of cash in just three years, which amounts to a mere tick of the clock in the world of biomedical research. Beginning next week the California Institute for Regenerative Medicine (CIRM), as the agency is formally known, will be minus its chief executive officer and its longtime counselor, who even predates the organization's actual creation in 2004.

CIRM directors are scheduled to meet Thursday at the San Francisco Marriott hotel in Burlingame, Ca., to confirm the appointment of Maria Millan, CIRM's vice president of therapeutics, as interim president of the agency. She will assume the duties of Randy Mills, who is leaving CIRM next week to head the National Marrow Donor Progam. 

Mills, who was paid $573,00 last year, also made it clear to the California Stem Cell Report in May that Millan is the appropriate person to take over the agency on a permanent basis after he leaves.

However, the decision is up to the 29-member board, which has scheduled an executive session Thursday to discuss the interim replacement for Mills. He joined the agency only three years ago but has left an impressive mark.

CIRM directors have also scheduled a July 17 meeting of their presidential search subcommittee to deal with the agency's leadership during what could be the last years of its life.

CIRM has a checkered record  in recruiting new presidents for a variety of reasons (see here, here and here). Some candidates have rejected offers. Other search efforts have been excessively prolonged.

Finding a new president from outside CIRM poses difficulties that would not have been in place, for example, five years ago. They include the tenuous future of CIRM along with the time needed for a normal executive search, plus the learning curve for a new CEO.

While CIRM is a small enterprise in some ways (less than 50 employees), it is an unusual mix of government, biotech business and academia, unlike any other state agency.  The combination has raised hurdles in the past.

The clock is running out fast at the agency. Any alterations in the plan put in place by Mills, Millan and company could slow its efforts to fulfill voter expectations that the agency would actually generate a widely available therapy. CIRM is helping to finance 27 current clinical trials, which are the last stages in research prior to a product reaching the market. The agency hopes to add 38 more trials over the next three years. But there are no guarantees that any will be successful.

Millan can step in and pick up the job relatively seamlessly.  Bringing in a CEO from outside could well take six months or more, including relocation. But serving as the head of  an organization that could be out of business in three years may not be appealing to many and could prolong recruitment.

If Millan is bypassed by the board, she may well leave the agency, triggering a cascade of departures as other CIRM employees also look to their own professional futures. An employee drain would hamper the agency's drive to come up with a commercial therapy.

James Harrison, the longtime counsel to the agency, is also leaving at the end of this week, returning to other pursuits at his private practice. Harrison has been a cornerstone of CIRM and has influence well beyond the not-so-simple legal matters involving the agency. He was also one of the authors of the 10,000-word ballot initiative that created the agency in 2004.

Scott Tocher, a longtime veteran of the agency, will replace Harrison. An announcement of the appointment is expected at the Thursday meeting.

Looming in the background is a gossamer plan for another ballot initiative to fund CIRM  beyond 2020. Bob Klein, a Palo Alto real estate investment banker who led the campaign that created CIRM, is talking about a $5 billion bond measure on the ballot as early as November of next year. Some political observers have predicted a less-than-warm-reception for such a proposal, given that the agency has yet to measure up to its 2004 campaign promises.

Another, rival proposal is being mentioned that would, in fact, move stem cell funding away from the agency.

One stem cell scientist, Paul Knoepfler of UC Davis, wrote last week about the agency's presidential search.

Commenting on his blog, Knoepfler said that CIRM directors should pick a "fantastic" person to replace Mills.  Knoepfler said the new president should have "strong leadership skills," a "big picture clinical vision" and "impeccable stem cell credentials," criteria that one could argue have not been met by most CIRM CEOs.

In the past, debate about presidential candidates centered on whether they should be stem cell stars or a leader who can execute an aggressive program that is already approved and in place. Given the current CIRM challenges, other criteria, such as speed and continuity, are also high.

The journal Nature this year said that the agency is in its "last stage." CIRM directors may well have that admonition on their minds as they consider fresh leadership for the program.

NY Times Offers Overview of $3 Billion California Stem Program

The New York Times took a look yesterday at California's $3 billion search for a stem cell therapy in a piece that almost incidentally reported the departure of its president, Randy Mills.

The article was more of a  blog item than the heavily and independently reported news story that is often characteristic of the Times. However, it marked one of the few occasions that the newspaper has attempted an overview of the California Institute for Regenerative Medicine (CIRM), as the stem cell agency is formally known.

Mike McPhate did the online article as part of his California Today column, which is provided online subscribers. The article began:

"More than a decade ago, Californians made a $3 billion wager on the healing potential of stem cell research.
"Today, with that money projected to start running out in the next few years, what does the state have to show for it?"

After recounting a bit of CIRM history, McPhate answered, 

• "More than 750 grants distributed
• "A dozen research facilities constructed
• "Roughly 2,000 scholarly papers published
• "More than 2,400 students and young scientists trained
• "About 30 projects that include clinical trials funded
"Still, the agency has yet to finance a therapy approved for commercial use."

McPhate concluded by saying that Robert Klein is eyeing another bond measure to continue the work at CIRM, which says it will run out of cash for new awards in about three years.

McPhate wrote,

"It would appear on the 2018 ballot. The ask this time? $5 billion."

More Media: San Diego Weighs in With Story on Randy Mills' Departure from Stem Cell Agency

The San Diego Union-Tribune, which covers the large biotech community in its area, today carried a hefty piece on the departure of Randy Mills as CEO of the $3 billion California stem cell agency.

Bradley Fikes wrote the story, which was headlined,

"Amid uncertain future, state's stem cell agency loses transformational leader "

Fikes, the only reporter on a major daily, California newspaper to regularly cover stem cell matters, reported that Mills said the agency will do fine without him. Fikes wrote, 

"'If me leaving CIRM is a problem, then I didn’t do a good job at CIRM,' Mills said. 'Whether it’s because I’m going to be the head of the National Marrow Donor Program or I get hit by a car, the success of this organization, or any organization that’s healthy and functional, should never pivot on one person,'  Mills said. 'I’ve assembled a team at CIRM that I have absolute, absolute confidence in.'"

The article also said,

"Jeanne Loring, a CIRM-funded stem cell scientist at The Scripps Research Institute, said Mills made the agency friendlier and more predictable for the scientists it funds.

"'The first and most dramatic thing he did was to end the process of independent grants,' Loring said. Under that process, each grant proposal was considered on its own, with no consideration for success under a previous grant for an earlier stage of the research.

"'It was always very troubling to people, I think, that they could do very well with CIRM money on an early-stage grant, and that would earn them nothing in a further application to continue the work,' Loring said."

Mills' Departure: Surprise and Concern About California Stem Cell Agency

Disappointment, shock and surprise are surfacing in the wake of the news that Randy Mills is leaving his post as CEO of the California stem cell agency. However, the news drew little attention in the media.

Mills, 45, yesterday announced that he will depart at the end of June to become head of the National Bone Marrow Donor Program in Minneapolis. Maria Millan, vice president for therapeutics, will take over on an interim basis while the agency's  board decides on a permanent successor.

The agency is entering what will be the last three years of its life unless it can round up additional funding. It has relied almost entirely on money borrowed by the state (bonds) which is expected to run out by June 2020.

Mills' unexpected move triggered expressions of dismay and amazement. "Wow!" was the one-word, email remark from a Southern California scientist, who declined to be identified.

Jeanne Loring, head of the stem cell program at the Scripps Research Institute in La Jolla, said in an email,

"I don’t understand why Randy Mills would leave CIRM now, when the rebirth of CIRM that he initiated is beginning to succeed.  I would have expected him to follow through on his vision.  I’m disappointed."

Paul Knoepfler, a UC Davis stem cell researcher, said in an email,

"Overall CIRM has prospered under Mills’ leadership with important, concrete accomplishments during his tenure. The agency's current trajectory is also very positive. He deserves a lot of credit for the positive impact he has brought to CIRM in just three years. The timing of his departure probably isn’t ideal as CIRM looks to the future with some challenges such as the nature of future funding for the agency and three years is a short tenure, but just as Mills brought in a new, helpful vision, the next leader may likewise provide new ideas and energy to successfully tackle the next phase for CIRM. Who that new leader ends up being could make all the difference for CIRM’s future so it’s a crucial decision. I’ll be curious how the Board approaches this challenge, and I’m excited to see what develops."

Knoepfler also posted more of his thoughts in an item on his blog.

John M. Simpson, stem cell maven for Consumer Watchdog of Santa Monica, Ca., said,

"Dr. Mills made substantial contributions to the agency during his tenure, improving both efficiency of the grant making process and transparency of CIRM's operations. Given the uncertain future as CIRM's current funding winds down, it is not at all surprising that he has opted to move onto another opportunity.

"Significantly, unlike the departure of his predecessor Dr. Alan Trounson, this move does not appear to raise any  conflicts of interest."

Another scientist who could not be identified said Mills' departure could lead to the loss of others of the 46 agency employees.

On the media side, Ron Leuty of the San Francisco Business Times wrote a straightforward piece on Mills' departure. Alex Lash of Xconomy offered a lengthier take. He covered some of the history of the agency along with the status of some its current projects. The Sacramento Bee carried a freelance piece by yours truly as well.

California's $3 Billion Stem Cell Agency Loses CEO Randy Mills

C. Randal Mills
CIRM photo

In a surprise move, the president of California's $3 billion stem cell research effort this morning announced his resignation as the program enters what some are calling its "final test."

C. Randal Mills said that he has taken a position as president of the National Marrow Donor Program in Minneapolis, Minn. He said he will leave the California Institute for Regenerative Medicine (CIRM), as the stem cell agency is formally known, at the end of June.

Maria Millan, CIRM photo

Maria Millan, vice president of therapeutics, will become interim president of the 12-year-old agency in July. The governing board of the agency plans a meeting of its search committee in July to consider its options regarding the appointment of a permanent president for CIRM, which expects to run out of cash for new awards in three years.

Just last week, the prestigious journal Nature described the Oakland-based agency as entering its "final test" and "last stage."

Mills, 45, joined the agency almost exactly three years ago and promptly launched a more focused effort than previously existed to fulfill the promises and expectations created by the 2004 ballot initiative campaign that created the agency.

Jonathan Thomas, CIRM chairman and who recruited Mills, said in a press release,

“CIRM has experienced a remarkable transformation since Randy’s arrival. He has taken the agency to a new level by developing and implementing 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.

"CIRM’s mission is to accelerate stem cell treatments to patients with unmet medical needs, and under Randy’s leadership, CIRM has done just that. The opportunity for Randy to lead a tremendously important organization such as NMDP is consistent with the values he demonstrated at CIRM, which put the well-being of patients above all else."

In an interview with the California Stem Cell Report, Mills said the offer to lead the donoro program "came out of the blue." He said the opportunity to join the world's largest bone marrow effort was unique. The organization, he said "does not do anything that doesn't save lives."

Mills said in the interview that Millan was an obvious choice to succeed him on a permanent basis. In the agency's press release, Mills said,

"What this team has been able to accomplish in that time is remarkable by any objective measure and I thank them for their 'All In' attitude and effort. As a trailblazing institute, CIRM is setting new highs in productivity and efficiency and will continue to deliver on its mission under the leadership of Dr. Millan."

Millan, a physician, has been with the agency since 2012, joining it from StemCells, Inc., where she was acting medical officer and vice president. Prior to that, she was an associate professor of transplant surgery for eight years at Stanford University.

Thomas said,

“One of the most valuable contributions Randy has made at CIRM is the strength of the team he has assembled. Maria is exceptionally well qualified and completely engaged in the operations of CIRM, having worked with Randy as member of the leadership team that created and is executing the strategic plan. With her leadership, we are well positioned for continued success,

Millan was paid $281,000 last year, according to The Sacramento Bee's state worker database. Mills was paid $573,000.

Mills' departure comes as supporters of the agency are concerned about whether its work will effectively end in 2020. However, its first chairman, Bob Klein, is talking about asking California voters for another $5 billion. Klein also led the $34 million ballot campaign.

Klein's organization, Americans for Cures, is planning to conduct a poll this fall to determine the level of public support for CIRM. He has said that if support is in the 70 percent range he would mount a bond issue in 2018. Otherwise, he might try in 2020, a presidential election year, with a larger voter turnout.

The organization that Mills is joining is "the world's largest the world's largest registry of unrelated adult donors and umbilical cord blood (UCB) units," according to Nature. It has been heavily funded by the federal government with an annual budget of $383 million, according to 2014 figures, and has about 1,000 employees. CIRM currently has 46 employees and has ranged up into the middle 50s.

Accompanying Mills in the move to Minneapolis will be his wife, Anna, and two children, Elise, 13, and Chase, 10.

(The agency posted an item on its blog, The Stem Cellar, dealing with Mills' resignation shortly after this item was posted.)

Ambitious, $150 Million, California Plan for Stem Cell 'Powerhouse' Stalls

Highlights

$75 million state loan

Pick of stem cell research

Agency vague on problems

The California stem cell agency's bold plan to create a unique, $150 million, public-private company to speed development of stem cell therapies has hit a significant snag, stalling its progress indefinitely.

The agency, however, says it hasn't given up and will try to find solutions to the undisclosed problems.

Under the proposal -- first advanced a year ago -- California would provide a $75 million loan to a partner that would also provide $75 million. The goal would be to create a "powerhouse" that would likely be one of the landmark legacies of the $3 billion agency. The partner would be expected to pay back only 50 percent of the loan plus interest. The new company would also have the pick of the 94 percent of the agency research that doesn't already have a business partner. 

The public-private biotech partnership  -- dubbed ATP3 -- would be the first in state history and the first of such magnitude nationally.

The agency has been working for much of 2016 to recruit applicants and to address potential concerns. The difficulties began to surface last fall at the Oct. 31 deadline for applications. The agency declined to disclose to the California Stem Cell Report even the number of applications it had received. A spokesman said only that the agency was checking to see if they were "eligible."

The chairman of the agency, Jonathan Thomas, went public with slightly more information at last week's meeting of the agency's governing board.

He referred to a "number of applicants" that were involved. He said that issues still exist and that the application(s) that were submitted to the agency will not be given to the agency's reviewers as scheduled. Thomas said the request for applications may extended and some criteria adjusted.

Randy Mills, president of the agency, said later in the meeting that an effort would be made to make the plan "compliant with state requirements and palatable to taxpayers." He described it as an "ongoing challenge."

Mills said,

"We haven’t given up but we haven’t nailed it yet."

No public questions were raised at the meeting by 29-directors of the agency, which is officially known as the California Institute for Regenerative Medicine (CIRM).

It was created 12 years ago by a ballot initiative approved by voters and relies on money borrowed by the state(bonds). The ballot campaign indicated that stem cell therapies were close on the horizon. However, the agency has not developed a therapy for widespread use.

It estimates that it has about $692 million left for awards. The agency's funds are projected to run out in 2020. No additional funding is in sight.

Voting Underway for Stem Cell Person of 2016

The latest tally of voting for the stem cell person of the year shows that patient advocate Ted Harada, who died in October, is leading with 47 percent of the vote with the nearest contender at 11 percent.

However, only one vote counts since this is not a democratic election. The sole individual choosing the stem cell person of the year is Paul Knoepfler, a stem cell scientist at UC Davis.

Knoepfler originated the award a few years back and personally puts up $2,000 in prize money. He has compiled a list of 20 possible honorees, ranging from patient advocates to researchers. Voting is advisory only. Deadline for the ballot is 11:59 p.m. Dec. 15.

Knoepfler wrote on his blog last month about Harada, who was an ALS patient advocate. Knoepfler said,

"Ted approached all that life threw at him, whether it was ALS or a brain tumor, with a characteristic passion, sense of humor and classiness. I don’t recall anyone dealing with adversity as well as Ted did and I never heard him say anything like, 'why me.' Instead there was always kindness and grace."

Distant second to Harada this morning was Randy Mills, president of the $3 billion California stem cell agency.

California's Randy Mills: Flood of Demand for Stem Cell Therapies Requires New Development Pathways

The president of the $3 billion California stem cell agency today told federal regulators that patient demand is "very real" for stem cell therapies and called for "alternative" pathways for development.

Randy Mills at FDA hearing

Randy Mills said patients are "really suffering and their needs are not being met." He said the demand is like a flood of water and will find its way around any blockages that rise up.

Mills spoke during the first day of a two-day hearing by the Food and Drug Administration(FDA) into possible new regulation of the stem cell field.

The session came as the Washington Post published online a lengthy piece about unregulated stem cell therapies and the increasing scrutiny they are facing from regulators. Reporter Laurie McGinley wrote about a clinic in Beverly Hills with a self-described "Time Machine" box.

Mark Berman, who was described as the co-founder of the "biggest network of commercial stem cell clinics" in the country, processes stems cell through the box for a procedure that he says doesn't require FDA approval.

McGinley wrote,

"In mini-liposuction procedures, Berman extracts fat tissue from patients and puts it into the Time Machine box, where a centrifuge separates stem cells from fat. The cells are then injected back into the patients to supposedly treat a variety of health problems, such as arthritic knees, heart disease, amyotrophic lateral sclerosis and even autism."

The treatments run about $8,900 each. Berman said his network has treated about 5,000 persons, McGinley reported.

The Post piece said that such treatments and their regulation or lack of regulation has "ignited a fierce debate among physicians, patients, scientists and politicians about whether the (FDA) should crack down on therapies that critics deride as snake oil but that some patients swear by. And it is fueling a broader, longer-term debate over how cellular therapies should be regulated."

Stem cell researcher Paul Knoepfler of UC Davis , who is quoted in the Washington Post article, took in today's FDA hearing from his lab in California. Writing on his blog, he delivered some "take-homes" after the session concluded. Here are a few excerpts from what Knoepfler wrote.

• "Some speakers advocated for a 'middle ground' between 'nothing' in the way of regulation for instance of the dubious clinics and the perceived 'mountainous' extreme of the current FDA oversight system. The middle ground was, for example, argued for by Janet Marchibroda of the BPC and then also in the next talk by CIRM President Randy Mills. Mills, who is a very powerful speaker, argued that there is a harmful binary system now and we need a middle ground between the extremes of little-to-no regulation and the current in his view constricted pathway."
• "Several speakers invoked patient autonomy and patient rights, and argued that they are doing what they are doing as practitioners of stem cell therapies simply for the betterment of patients. There’s the pesky problem there with this argument of probably millions of dollars in profit being made from the patients by some clinics though. If it’s all about patients, I challenge the clinics to do the transplants strictly 'at cost' with no profit margin."
• "Many parties asked the FDA for more clarity and consistency on oversight. Also, a number of speakers asked for concrete, real world examples of how definitions would apply.  I’ve been pushing the FDA for increased clarity for years including lay abstract summaries of their main guidelines and statements of current thinking. I really like the idea of flowcharts."

A Scientist's Comment: Deal Directly with FDA Rather Criticizing It in the Media

The following comment on FDA regulation of stem cell research was submitted via email by a longtime scientist in the field, who asked to remain anonymous. 

"Many advertised  'stem cell' treatments use hematopoietic or blood stem cells which are run under a different set of regulations from pluripotent stem cells since they are umbrella-ed under regulations designed to cover blood transfusions and bone marrow transplants.  Pluripotent stem cells are very new, with many unknowns and the potential for triggering tumor growth.   In contrast, clinical use of blood stem cells has been ongoing for over 50 years with special regulations (or lack thereof) grandfathered in.   Unfortunately, the newly touted 'treatments' are not for homologous use of blood stem cells, are unlikely to be therapeutic and may even be dangerous. 

"The FDA has published new draft guidances addressing these issues that will be discussed in open forum this September.  I think it best to bring concerns about FDA regulations to these meetings and to initiate change by talking directly with the agency rather than criticizing them in the press or through Congress.  Like their oversight over the IRS and other federal agencies, Congress’s actions are counterproductive when they add broad, unfunded mandates while cutting funding at the same time.  But perhaps this is their way of shrinking the government and simultaneously dinging them for not doing their work.  A sure route to failure."

Influential Scientific Journal Rips Effort to Loosen Stem Cell Research Rules

The prestigious journal Nature today editorialized against easing federal regulation of stem cell treatments, a major blow to the campaign by the California stem cell agency to speed such therapies to the marketplace.

The headline on the piece said,

"FDA should stand firm on stem cell treatments."

The unsigned editorial declared that those who contend that the Food and Drug Administration (FDA) is holding back "effective therapies" are peddling a false narrative. Nature declared,

"The claim that regulation is too harsh wrongly implies that the FDA is holding back therapies that work. Critics point to decades of preclinical and clinical work with stem cells and the pipelines of stem-cell treatments. With circular logic, they argue that, because the treatments have not been approved, there is something wrong with the approval system."

The $3 billion California stem cell agency has been lobbying for months for changes in FDA regulation. Randy Mills, president of the California Institute for Regenerative Medicine(CIRM) as the agency is formally known, said back in December that "patients are dying" because the FDA is  being "so careful about safety."

Mills has said the fast-track process in Japan is worth considering. However, Nature said,

 "It is not a fit and proper model to export, chiefly because it grants 'conditional approval' to treatments with minimal safety data and little attention to efficacy."

Nature was also critical of the Regrow Act, a legislative effort to overturn the FDA's current processes. CIRM has not taken a position on that bill.

Nature concluded,

"The FDA should strive to keep this debate on the proper topic — how to create a more efficient system that still scientifically evaluates whether treatments are safe and efficacious. To fall short would be a setback for science, and for patients."

The journal's editorial did not mention CIRM or Mills by name. It did cite the recent article by UC Davis stem cell researcher Paul Knoepfler and Leigh Turner, an associate professor of bioethics at the University of Minnesota, documenting the existence of 570 dubious stem cell clinics in the United States.

California Stem Cell Hooha: Testy Media Exchange Triggered by Criticism of the State's Agency

A sharp exchange erupted this week on a mainstream California media site after the state's $3 billion stem cell agency was taken to task for lobbying the federal government to ease its regulation of research on stem cell therapies.

The flap involved allegations of lies and questionable motives. Along the way certain death was mentioned, and one writer was accused of "stealing hope" from seriously ill persons. Personal animus charges were raised along with questions about ill-informed rhetoric.

The hooha was set off by an op-ed piece by UC Davis stem cell scientist Paul Knoepfler who wrote an op-ed in the San Francisco Chronicle (see here and here). It chastized the agency and its president, Randy Mills, for a risky and "political" effort to change regulations by the federal Food and Drug Administration. Knoepfler said patients could be put at risk.

The piece triggered harsh comments that were carried by the Chronicle at the end of the article. In addition to Knoepfler, individuals involved in the exchange included Jeanne Loring, head of the stem cell program at the Scripps Institute in La Jolla; Judy Roberson, a Sacramento patient advocate for Huntington's disease, and a person identified only as "Jeff." He wrote,

"What's Knoepler's real beef?....Poor Paul is not getting the money from CIRM he used to get. So his attitude is 'screw patients,' I want my lab funded like it used to be. Nauseating!!!"

CIRM is the abbreviation for the formal name of the state stem cell agency, officially known as the California Institute for Regenerative Medicine. Its board includes Jeff Sheehy, who told the California Stem Cell Report that he is not the Jeff commenting on Knoepfler's article.

The anonymous "Jeff" said, 

"Has any of (Knoepfler's) research led to a development candidate that might lead to a treatment in the future? Or is he just a stem cell scientist opining about something he knows very little or nothing about(the FDA)? Maybe he 'heard' something from somebody and that gave him just the hook he needed to attack CIRM and Mills, both of whom he obviously has personal animus towards."

Loring largely sided with Knoepfler. She also wrote,

"Jeff, why are you questioning Paul's motives? What are your motives? Do you work for CIRM? As you know, a person's profession doesn't define his/her sphere of knowledge. If something is important to you, you can research it and gain expertise."

Roberson, who favors the CIRM effort to loosen regulations to bring therapies into widespread use, said patient safety is important. She also said,

"But for patients and their families with a 100 percent fatal genetic disease like Huntington's disease, we can withstand some risk because we are facing a gut-wrenchingly long, gruesome death anyway. We have no chemotherapy, no surgery, no radiation and sadly, no hope. We have NOTHING! And we'll continue to have nothing for many more decades if the FDA continues doing business as usual."

Another reader, William Barnes, said,

"I suggest that Dr. Knoepfler consider how it feels to be suffering and near certain death and be denied a possible cure because Dr. Knoepfler doesn't think it's in their best interest....Let the individual patient decide! It’s their life! Why should the decision about someone’s life, possibly your loved one, be made by a distant and dispassionate academic?"

In addition to his research, Knoepfler produces a blog, The Niche, on stem cell science, regulation and policy. He took up the blog after a nasty bout with prostate cancer when he was in his 40s. Knoepfler is also the author of "Stem Cells: An Insider's Guide," a book aimed at helping patients understand stem cell science. 

The Case of the FDA's $330,000 Stem Cell Pigs

Judy Roberson at podium, Bob Klein, former chairman of the California stem cell agency at left, Claire Pomeroy, former
UC Davis vice chancellor and agency director at right. UC Davis photo

Judy Roberson is a registered nurse from Sacramento, Ca., who has long been active with the California stem cell agency on behalf of patients with Huntington's disease. She lost her husband to the always fatal, inherited brain disease, and members of her family are at risk.

Roberson appeared before the governing board of the $3 billion California Institute for Regenerative Medicine (CIRM), as the agency is formally known, earlier this month to applaud its efforts and those of its CEO, Randy Mills, to persuade the federal Food and Drug Administration to ease its regulation of proposed stem cell therapies. Here is the text of her statement. 

"The Northern California Huntington's disease (HD)  advocacy community says BRAVO to President Randy Mills for his editorial directed at the FDA, "Give Us Our Cures."

"Over 40 HD advocates joined the CIRM Stem Cell Champion campaign in April, promoted by Kevin McCormack (the agency's director of communications), in the hopes that pressure from affected families will prompt the FDA to become open to stem cell therapies and allow the increased risks that naturally go along with new therapies.

"For people with Huntington's disease, which is 100 percent fatal and has zero therapies, we are willing to take on more risk since we're dying anyway.

"The FDA has delayed the fully enrolled, CIRM funded, first-in-human clinical trial using adult stem cells at UC Davis with Drs. Vicki Wheelock and Jan Nolta. The NIH RAC committee enthusiastically approved their novel therapeutic clinical trial. Then the FDA asked for additional animal studies, this time with three pigs at a cost of over $330,000 plus two years of additional research; costs will approach $1 million.

"One HD family from New York has funded one of the pigs, but this gap in funding has shelved this promising research!

"Devastated patients and their families see this add-on research requirement as a delay from the FDA, which means that this fully enrolled trial, with 24 patients who meet today's criteria will progress and may not qualify in two to four years even if this project later receives FDA approval to begin a phase one trial. FDA delays are killing us!

"We need a new FDA 2.0 because doing NOTHING is doing harm!"

California's Battle for Easing Regulation of Stem Cells Attracts Global Notice

Highlights
CIRM CEO Mills mentioned
FDA regulation/Regrow co-mingled
A tale of dubious stem cell treatments

California's $3 billion, 11-year-old effort to produce a stem cell therapy -- largely ignored by the mainstream media -- broke into global cyberspace today in a reasonably significant way.

The agency was featured in a piece on Buzzfeed, an online news operation that has chalked up more than 173 million unique visitors worldwide in the last 30 days.

The article by Dan Vergano focused on the efforts by the California Institute for Regenerative Medicine or CIRM, as the agency is formally known, and others to persuade the FDA to ease up on regulations so that stem cell therapies can be more quickly developed.

Vergano wrote,

"Despite a decade of scientific hype, progress has been slow in proving that these new treatments actually work. Some scientists are particularly frustrated with the slow pace of FDA review. In a Fox News op-ed published last month, for example, C. Randal Mills, the head of the prestigious California Institute for Regenerative Medicine (CIRM), called for the federal government to loosen its safety rules, promising “medical breakthroughs” for arthritis, back pain, and diabetes."

Mills has focused his efforts since last year on FDA regulation. But his campaign is being co-mingled in the media with the Regrow Act, an industry effort that the agency has not taken a position on.

Paul Knoepfler, a UC Davis stem cell researcher, also recently brought FDA regulation and Regrow together in a piece in the San Francisco Chronicle recently. Vergano interviewed Knoepfler for the Buzzfeed article,

"'We have been telling people to cut down the stem cell hype, and then we turn around and have this talk about miracles and beautiful medicine....Wishful thinking here could have a whole slew of dangerous consequences.' Most worrisome, (Knoepfler) said, is that desperately ill patients looking cures might end up with tumors instead."

Jim Gass, NYTimes photo by Carlos Moreno

One such case was chronicled this week by New York Times reporter Gina Kolata. Her hair-raising story told the tale of patient Jim Gass, a 66-year-old lawyer from San Diego. The article began,

"The surgeon gasped when he opened up his patient and saw what was in his spine. It was a huge mass, filling the entire part of the man’s lower spinal column.

"'The entire thing was filled with bloody tissue, and as I started to take pieces, it started to bleed,' said Dr. John Chi, the director of Neurosurgical Spine Cancer at Brigham and Women’s Hospital in Boston. 'It was stuck to everything around it.'"

"He added, 'I had never seen anything like it.'"

Stem Cells and the FDA: California's Lobbying for Easier Approval Triggers Opposition

The $3 billion California stem cell agency came in for some sharp criticism last week in an opinion piece in the San Francisco Chronicle that was written by a UC Davis stem cell researcher who said the agency is engaged in politicking that "risks doing much more harm than good."

Paul Knoepfler, the scientist who also produces "The Niche" stem cell blog,  wrote,

"The California Institute for Regenerative Medicine (CIRM) has become politically active under its new president, C. Randal “Randy” Mills. Mills is lobbying for a radical departure in how the U.S. Food and Drug Administration (FDA) vets experimental stem-cell therapies, but such politicking risks doing much more harm than good."

Knoepfler wrapped in an attack on federal legislation known as Regrow Act. Knoepfler said the proposal would "force the FDA to conditionally approve still experimental stem cell therapies with relatively little data supporting them."

Randy Mills at left, USC photo

Knoepfler reported that Mills said he was not anti-FDA. Knoepfler also noted that the agency has not taken a position on the Regrow Act.  Knoepfler said the FDA effort and the Regrow Act are part of a larger effort to weaken the FDA, which has been under regular criticism for decades for moving too slowly.

The stem cell agency's attempt to modify the way the FDA approaches potential stem cell therapies has been underway for months. The California Stem Cell Report carried news on the subject as long ago as last winter(see here and here.)

Mills initiated the lobbying effort with the FDA after an informal survey of California "stakeholders" showed that they perceived the FDA as the No. 1 obstacle to making stem cell therapies available for widespread use.

In an item on the agency's blog, The Stem Cellar, last December, Mills said that “patients are dying” because the federal government is “being so careful about safety,"

Yesterday, Knoepfler provided some personal insight -- a "backstory" -- on what led him to write about the all of this. His piece was carried on his blog and emphasized that he is a "big backer" of CIRM and still is. Knoepfler has received $2.2 million from CIRM. The CIRM web site has 60 links to mentions of Knoepfler on its Web site, including this one below. 

Knoepfler wrote,

"Most of the stem cell scientists that I have talked to (admittedly with a few notable exceptions that include some long time stem cell advocates and scholars) are opposed to CIRM’s lobbying on this front, but are uncomfortable speaking out."

Uncomfortable is a rather mild word for it based on the fact that the agency holds the key to a $3 billion vault containing the hopes of funding for the entire California stem cell community.

We should note that lobbying is not new to the agency. Its former chairman, Robert Klein, had a passion for it. At one point in 2009, he engineered the hiring of an exceedingly well-connected lobbyist named Tony Podesta for $200,000.  Mills has not ventured into that sort of game, however.

Knoepfler has much more to say on the subject of regulation, the need for it and the impact on patients and the dubious stem cell clinics that seem to be multiplying throughout the nation, despite the fact that the FDA has never approved a stem cell therapy for widespread use.

The California Stem Cell Report will be visiting the matters surrounding these issues and the California stem cell agency. Look for more here during the next week or so.

The California Stem Cell Agency: A Business with Scientists as Subcontractors?

The following commentary was submitted by a grantee of the California Institute for Regenerative Medicine (CIRM) in response to its $15 million award earlier this week to Quintiles, a clinical research organization. The person who wrote the item asked to remain anonymous.

Comments from readers are always welcome on the California Stem Cell Report.  They can either be filed directly on an item by clicking on "post a comment" at the end of each item or by sending them to djensen@californiastemcellreport.com. Here is the commentary on the direction of the stem cell agency.Scientis

"With the funding of Quintiles the last piece of the puzzle falls into place. Randy Mills is very proud of the fact that he transformed the company Osiris, which was research oriented, into a product-oriented company.

"Everything he has done since he became president of CIRM has been strategy to turn it into a product-oriented business.

"From start to finish, Mills is refashioning CIRM as a business. The grant applications have now become 'partnering opportunities,' and the researchers receiving those grants take on a role as subcontractors under  CIRM’s control rather than independent scientists.

"With grantees as partners and subcontractors, the next stage in commercialization is to hire a CRO (contract research organization) to funnel through all of the promising products, taking them out of the grantee’s hands.  Quintiles is a huge organization and the $15 million grant is insignificant for them -- they subcontract for drug companies to run clinical trials.  They have no stem cell experience.

"Subcontracting a CRO is a business strategy to avoid investing in employees and technologies the company needs.  The selection of grantees has also become business-oriented:  CIRM is recruiting companies outside California to fund so they can be added to its corporate profile.  Companies have also become the preferred recipients of CIRM grants, even if they are virtual companies set up by academics for the purpose of obtaining CIRM funding.

"CIRM is morphing into a corporation with centralized control, with the hope that the product pipeline will yield something that makes money.  The question is:  was this the intention of Prop. 71?  If so, why wasn’t it always a corporation?" 

(Editor's note: Prop. 71 is the ballot initiative that created the stem cell agency in 2004.)

California's Stem Cell Agency Says FDA 'Chilling' Development of Stem Cell Therapies

CIRM graphic

The president of the $3 billion California stem cell agency hit the campaign trail this month but not to head off Donald Trump or Hillary Clinton or even Bernie Sanders.

Randy Mills targeted the Food and Drug Administration (FDA), the agency that has life-or-death power over the introduction of stem cell therapies. "Give us our cures" was Mills' message for federal regulators.

The most recent forum for Mills was Fox News. The headline on an opinion piece that he co-authored with former Republican U.S. Sen. Bill Frist,  said,

"Cell therapy reversed blindness for 47,000 patients in 2015. So why is it against the law?"

And in a presentation earlier this month to the Bipartisan Policy Center in Washington, D.C., Mills said that the current FDA "paradigm" for new stem cell therapies has been inconsistent, selective and chilling.

"By having a system that approves nothing after 15 years, we are neither protecting nor helping those in need."

Mills said that FDA regulations should "be scaled to more accurately reflect the risks, be balanced against the very real consequences of doing nothing and be fairly and consistently applied."

The campaign by the California Institute for Regenerative Medicine (CIRM), as the stem cell agency is formally known, grew out of work last year on its strategic plan. A survey of stakeholders showed that 70 percent of them identified the FDA as an impediment to development of commercial therapies.

Kevin McCormack, senior director of communications for CIRM, wrote about Mills' piece on Fox on the agency's blog, The Stem Cellar. McCormack said,

"A lot of people are frustrated with the US Food and Drug Administration (FDA) and its woefully slow process for approving stem cell therapies. That’s one of the reasons why we started the CIRM Stem Cell Champions campaign, to gather as many like-minded supporters of stem cell research as possible and help to change the way the FDA works, to create a more efficient approval process."

In his presentation in Washington, Mills was careful to point out that CIRM is not opposed to the FDA or its regulation. He said CIRM just wants better regulation that will help bring therapies to patients by speeding their approval and balancing risk.

In response to a question about how Fox News happened to carry the Mills' piece, McCormack, replied,

"It was something Randy and Senator Frist had been talking about doing for a while, and why not Fox News, it has a big audience."

Other big audiences for CIRM's message will be in the San Francisco Bay area next month: BIO, the annual biotech industry convention, which is expected to draw more than 15,000 from June 6 to June 9, and the annual meeting June 22-25 of the International Society for Stem Cell Research, which expects to see more than 4,000 attendees. CIRM is expected to have a strong presence, relatively speaking, at both events.

The stem cell agency says FDA practices lead to selective
enforcement of its regulations.                     CIRM graphic

A "Chicken in Every Pot" -- Stem Cells and the Latest Warnings About Hype

Let's face it, folks, without some hype, California's $3 billion stem cell research effort would not exist.

That's because it is a creature of a popular vote in 2004, and the Golden State's voters needed to be persuaded to pony up their billions for something that they were told would pay off and pay off relatively quickly.

Old news, right? But not entirely. Last week the world's foremost stem cell research organization, the International Society for Stem Cell Research (ISSCR), issued stern warnings about "communications" involving stem cell research. "Circumspect and restrained" were the watchwords from the more than 4,000-member group. Seek "timely corrections" of misleading information in the media, the world's stem cell researchers were told, among other things.

All of which is interesting coming from the ISSCR, which loaned its considerable clout in 2004 to the $36 million effort to convince Californians to create the state's stem cell agency with billions borrowed by the state. The effort was also endorsed by a host of individual, top researchers. The campaign is widely regarded as over-promising results on an unrealistic timetable. (See here and here.)

That said, electoral campaigns are not science. Think about promises of a "chicken in every pot," and you will have a good idea of what needs to be said to win an election. And there's the rub.

To generate cash from citizens, it is necessary to create some excitement. Otherwise, it is ho-hum time. One Canadian writer, Kelly Crowe, put it this way in a piece on the public relations guidelines from the ISSCR,

"Would you read a story if this was the headline: 'New study raises questions about an experimental treatment that might not work and won't be ready for a long time.'"

Beyond public perceptions, there is the small matter of stimulating business interest in turning stem cell research into cures, the mission of the California stem cell agency. Businesses are often portrayed as daring innovators bringing fresh, exciting stuff to all of our homes. The reality is that businesses are more often timid, unwilling to take risks that might affect their financial well-being.

So they too must be shown the stem cell light by the agency and its backers so that industry will cough up the considerable cash necessary to bring a stem cell therapy to market and fulfill the promises made to voters 12 years ago.

Just how far should stem cell advocates go in promoting their cause? The ISSCR has its new guidelines. Others may disagree. One person's hype is another person's honest belief. It is unlikely that the ISSCR guidelines will settle the questions.

Little doubt exists that stem cell hype is rampant, some of it from the scientific community and some from enterprises offering untested procedures and treatments. The hype has a natural audience. The public tends to want to believe in scientific and medical miracles, and stem cells smack of miracles.

Randy Mills, the president of the California Institute for Regenerative Medicine (CIRM), as the agency is formally known, regularly brings the facts of "risk" to his dealings at the agency. It is one of his finer innovations.

Last year, his plan for the agency's next five years contained three pages of "risks," including inadequate health benefits, foot-dragging by the federal regulators and safety issues.

Mills exudes excitement for stem cell research and its potential, but at the same time he has a keen eye for the obstacles. It could be called realism. Unless you see the obstacles, you cannot hurdle them. Nor can the public be expected to be patient if it is oversold and under-delivered. 

(A final note: Kelly Crowe's piece is a dandy and involves much more than stem cell research. The headline: "It's not just stem cell research that's overhyped— medical science spin is a widespread problem.")

California's Latest, $4 Million Effort to Transform Human Stem Cell Research

The California stem cell agency's "great ideas" program is set to give away $4 million next week to 18 researchers to help them pursue fledgling pathways to a new therapy or cures for stroke, bladder injury, eye problems, Parkinson's disease and more.

In its solicitation for applications, the agency said the seed funding was aimed at supporting "exploration of transformational ideas that hold the potential to greatly impact the field of human
stem cell research." The expectation is that these awards will provide more information that will help generate more funding to carry the efforts forward.

"Great ideas" is how Gil Sambrano, the agency's director of portfolio and review, described the round in a slide prepared for the meeting of the governing board of the agency next Thursday. Board approval of the positive recommendations by the reviewers is routine. At that point, the agency will identify the winners but not the unsuccessful applicants.

The field of applicants began at 101 earlier this year and was winnowed down to 60 before the final 18 were selected.

Approval of the applications was performed behind closed doors by the agency's blue-ribbon, out-of-state reviewers. The identities of reviewers in a specific round are not disclosed nor are their professional and economic interests.

None of the awards exceeds $238,000. Researchers will receive only a maximum of $150,000 for "direct costs." The excess goes to the institution or business involved for a variety of overhead costs. Duration of the awards varies.

Here is a link to summaries of the scientific reviews of the applications, including comments scores on both winning and losing applicants.