Federal Stem Cell Regulation Setback in California; Case Involves Treatments with Fat Cells

Dubious stem cell clinics in California were in the news again this week with a piece in the Los Angeles Times declaring that a federal judge had dealt a blow to regulators trying to crack down on the much-criticized enterprises. 

The column by Michael Hiltzik said,

"A Los Angeles federal judge has rejected a government motion that could have shut down a network of clinics offering customers allegedly unauthorized stem cell treatments.

"The ruling by Judge Jesus G. Bernal potentially deals a setback for the Food and Drug Administration’s campaign against treatments using stem cell preparations that the FDA has not approved. 

"In the ruling published late Monday, Bernal denied the government’s motion for summary judgment in its lawsuit against the Rancho Mirage-based California Stem Cell Treatment Center, the Cell Surgical Network, and the latter’s founders, Mark Berman and Elliott Lander. Bernal ruled that the case was suitable for trial."

UC Davis stem cell researcher Paul Knoepfler also wrote about the case on his blog.

"The reasons why Bernal said he rejected the summary judgment request are concerning and don’t fit with my view of the medical science here as a stem cell biologist. 

"Unfortunately, this all leaves the door potentially still open for hundreds of clinics to sell the unproven fat stem cell product at the heart of this case called 'stromal vascular fraction' or (SVF)."

This lawsuit was first filed in May 2018 and is likely to continue for some time, possibly involving lengthy appeals. 

The controversy over unregulated clinics is of concern to those who support refinancing California's stem cell agency with $5.5 billion via a hoped-for voter approval of a ballot measure in November. The fear is that the flap could create voter confusion and besmirch the entire field. 

The agency was created in 2004 by voters who provided it with $3 billion. It is now down to its last $27 million for research grants. That figure is likely to increase next week as agency directors hear a report on the amount of cash recovered from awards that did not meet benchmarks.

Calfornia's Stem Cell Pie for 2020: State Agency to Slice Up Nearly Final Millions Next Week

It ain't over yet -- at least that's the word from what could be deemed the financial center of stem cell research in California.

The governing board of the California stem cell agency is scheduled to meet Feb. 6 to allocate unspecified additional millions for stem cell research and peer down the road that awaits it beginning Nov. 4 of this year.

That is the day after California voters are expected to deliver their judgment on whether the California Institute for Regenerative Medicine (CIRM), as the agency is formally known, should continue and receive an additional $5.5 billion.

The agency is running out of money. Its initial $3 billion is now down to $27 million for awards. A ballot initiative to give the agency the additional billions is expected to be on the ballot this fall, Nov. 3 to be precise.

But in just 10 days, the CIRM board will reveal the amount of cash that has been recovered from research awards that failed to meet milestones during 2019. The board is scheduled to make decisions on how that money, which is likely to run into tens of millions, is to be allocated during the remainder of this year.

Also on the agenda is a three-word topic: "strategic plan themes."  This is likely to involve possible directions of the agency should it receive additional funding from the ballot initiative. That proposed measure substantially expands the scope of the agency and specifies it move into in certain new areas, including attempting to assure the affordability of agency-financed therapies whose costs could run upwards of $2 million.

The agenda includes an update on most major programs involving CIRM, information that will come widely into play as the next fall's election receives more attention.

The meeting will be based at CIRM's headquarters in Oakland. But it will also be available electronically at a number of other public locations throughout California and via the Internet. Members of the public will be able to comment and ask questions through the Internet. Details are available on the agenda. 

Tiny Poll Heartens Backers of California's Financially Beleaguered Stem Cell Agency

The first public opinion poll of 2020 is out concerning the financial fate of California's $3 billion stem cell agency, and the result is "upbeat." 

However, there is one caveat that is as consequential as the failure of a phase 1 clinical trial. That is the go, no-go stage at which the safety of a stem cell therapy is tested in humans. 

The poll covers only 83 people out of the roughly 20 million California voters. 

The testing of public sentiment was conducted in the past week or so by UC Davis stem cell scientist Paul Knoepfler, who well knows the weakness of the non-scientific sample. But he still thinks it is something of a promising sign. 

Writing on his blog, The Niche, Knoepler reported that 48 percent of his self-selected respondents, who filled in the poll on the blog, believe that the agency will be refinanced with $5.5 billion from California voters come November. 

Sixty-three percent of those polled also said it should be.  

The agency is now down to its last $27 million, which is only a relative drop in the stem cell research bucket. 

Knoepfler described the poll results as "upbeat." He said that he thought that the 63 percent figure was an especially good sign for the agency, known formally as the California Institute for Regenerative Medicine (CIRM). Knoepfler wrote, 

"I actually thought this one might swing the other way since we have many readers here on The Niche who run stem cell clinics or are fans of the clinics, and seem to have a dislike or skepticism for organizations such as CIRM, ISSCR (an international group of stem cell researchers), etc."

Backers of the agency are currently in the process of gathering more than 600,000 voter signatures to qualify the $5.5 billion ballot measure for the fall ballot. 

The results of that signature drive, which is being handled by professional signature-gathering firms, may not be known until June. 

Global Growth in 2020 for Regenerative Medicine While California's Stem Cell Program Faces Extinction

ARM graphic

SAN FRANCISCO -- Leaders of the stem cell and regenerative medicine industry gathered yesterday at a sumptuous hotel here, and the talk was of billions of dollars in business, not to mention medical treatments that can cost millions. 

Just 11 miles away across the famed San Francisco Bay, however, was another sort of stem cell scene. It could be found in the not-so-posh, Oakland headquarters of the state stem cell program, known officially as the California Institute for Regenerative Medicine (CIRM) . 

CIRM is going broke. Its original $3 billion has shrunk to $27 million over the last 15 years. 

On the other hand, global financing for therapies and treatments in the regenerative medicine field totaled nearly $10 billion in 2019, marking the second highest year ever, the leading U.S. industry group reported at the session at the Parc 55 hotel.

Janet Lambert, CEO of ARM -- ARM photo

"2019 (was) a year of significant growth in the regenerative medicine sector," said Janet Lambert, CEO of the Alliance for Regenerative Medicine (ARM), told a standing-room-only crowd of more than 500 gathered for its annual briefing. "We enter 2020 poised for continued expansion."

Lambert said,

"The pipeline is robust, with several next-gen technologies entering the clinic and an increase in clinical trials for indications with large patient populations."

Her comments came as CIRM faces a financing shortfall that could mean its virtual extinction. 

The agency, created in 2004 by state voters, is down to its last $27 million of uncommitted cash. CIRM has often spent close to $300 million a year. The agency is hoping voters next November will provide it with $5.5 billion more under the terms of a ballot initiative yet to qualify for the ballot. CIRM's only significant source of funding is bonds issued by the state. 

Lambert's organization has more than 350 members, including CIRM. The largest percentage of ARM's membership comes from cell and gene therapy companies. The global financing it reports is almost entirely private. 

ARM's 2019 survey tracked nearly 1,000 companies globally, including 534 in the United States. The second largest number, 237, includes Europe and Israel. Lambert said the fastest growing area is Asia, which has 180.

ARM counted 94 phase three clinical trials globally, the last stage before a therapy is approved for widespread use by the federal government. 

CIRM is helping to finance 60 clinical trials, including three that are still active in phase three. It has yet to back research that has led to a widely available product.

Here are links to CIRM's phase three trials: BrainStorm Cell Therapeutics,  $16 million, ALS, PI Ralph Kern; Humacyte, Inc., $10 million, kidney failure, PI Jeffrey Lawson; Medeor Therapeutics, $11 million, kidney failure, PI Karen Smith. 

Here is a link to Lambert's slides. In the near future, ARM expects to mount online a video of her presentation including two additional panel sessions that examine the current and future outlook for regenerative medicine.   

Gold-Plated Stem Cell Treatments, Multi-billion Dollar Benefits and Potential Industry Profits

One of the nation's leading regenerative medicine industry groups is touting multi-billion dollar savings that may be achieved with the type of stem cell and gene therapies that are being developed with cash from California's financially beleaguered stem cell program.

The industry group is the Washington, D.C.,-based Alliance for Regenerative Medicine (ARM). It is tackling one of the major issues facing development of commercial stem cell therapies -- sticker shock at their expected prices, running upwards of a $1 million or more.

Without a willingness from health care insurers to cover the costs and provide a pathway to profit, it is unlikely that the biotech industry will embrace production of the therapies.

In a study released last Friday, the group said,

"Advances in molecular biology and genetics are leading to new treatments for rare diseases that require new ways of assessing value. CGTs (cell and gene therapies) are directed at the underlying cause of a condition and offer durable, potentially curative, or near-curative benefits. These transformative therapies create challenges for current reimbursement frameworks as they (the therapies) require significant upfront costs but are expected to provide a lifetime of benefits. The recurring treatment costs of chronically-managed patients can be greatly reduced and even eliminated with a one-time administration or short course of these novel therapies. 

"As CGTs arrive on the market, payers need new models for assessing their value. These treatments could potentially end the patient’s burden of illness, resulting in cost offsets (eliminating or reducing the need for long-term treatment, hospitalizations, and other care) and productivity gains that span a lifetime. Manufacturers incur a high per-patient development cost for these therapies and payers who bear the cost of treatment may not realize the long-term financial benefits due to health plan switching."

The ARM study predicted cost savings of as much as $33.6 billion over about a decade in connection with three afflictions: sickle cell disease (SCD), multiple myeloma (MM) and hemophilia A (Hem A). 

California's stem cell agency was not mentioned in the study, but it has funded research in all three areas. The agency is a member of ARM.

The study, backed by ARM and performed by the Marwood Group, said,

"Access to CGTs for even a modest number of patients with MM, SCD, and Hem A each year can reduce overall disease costs by nearly 23% over a 10-year period. The savings from lowering healthcare costs and raising productivity are considerable, approaching $34 billion by 2029. Of the savings, $31 billion are from a reduction in healthcare costs and $3 billion are from productivity gains."

The model used by ARM assumed CGT prices as high as $2 million. The study said,

 "The model has tested more than 180 different prices across the three potential CGTs that ranged from a minimum test price of $150,000 and up to a maximum price test of $2,000,000. The prices entered into the model created 60 different cost savings curves for all three of drugs in this model. Prices were distributed with more than 50% of test prices in the $100,000-$600,000 price per administration range."

The discussion of the costs of stem cell therapies has special resonance in the Golden State where voters are likely to be asked next fall to give $5.5 billion more to its stem cell agency, known formally as the California Institute for Regenerative Medicine (CIRM).

The agency, funded with $3 billion in 2004, is down to its last $27 million. A new, proposed ballot initiative is focusing hard on affordability of stem cell treatments. The initiative has no specific solutions but stipulates that a new version of CIRM  -- if the ballot measure is approved -- should devise some ways to come up with answers for insurers who are not likely to warm easily to $1 million therapies.

The 2020 Outlook for Regenerative Medicine: Major Industry Briefing Available Monday on Internet

One of more significant conferences of the year involving stem cells and regenerative medicine begins Monday in San Francisco with an overview of the industry and state of research. 

And there is an opportunity for those not on the scene to sample the presentations Monday morning via the Internet, including emerging therapies for cancer and next generation cell and gene therapies. 

The event is a state-of-the-industry presentation by the Alliance for Regenerative Medicine (ARM) in conjunction with the annual J.P. Morgan Health Care Conference.  

Something like 500 persons are expected to attend the ARM event. Below is the agenda for the Monday morning program. All times are PST. A live webcast will be available here, according to ARM.  Also available at the same Internet page location is a video from last year's presentation. 

Look for coverage also of the event next week right here on the California Stem Cell Report. 

8:00 – 8:20am | Introduction & Industry Update
Janet Lambert, CEO, Alliance for Regenerative Medicine

8:20am – 9:05am | Emerging Cell Therapies for Cancer
Claudia Mitchell, Senior VP, Production & Portfolio Strategy, Astellas (moderator)
Pascal Touchon, CEO, Atara Biotherapeutics
Matthew Kane, Co-Founder and CEO, Precision BioSciences
Samarth Kulkarni, CEO, CRISPR Therapeutics

9:05am – 9:50am | Next Generation Gene and Cell Technologies
Timothy Miller, Co-Founder, President, & Chief Scientific Officer, Abeona Therapeutics
Shelia Mikhail, CEO, AskBio
Laurence Cooper, CEO, Ziopharm
Emile Nuwaysir, President & CEO, BlueRock Therapeutics
Tim Lu, Co-Founder and CEO, Senti Bio

Doubters and Hard Choices: Challenges for California's Stem Cell Program

California's 15-year-old, $3 billion stem cell program received a modest smattering of attention during the holidays, first in an article in the Los Angeles Times and another in Bloomberg Law.

The Times, the state's largest circulation newspaper with 2 million readers on Sunday, called it one of eight, global science stories to watch in the coming year. Specifically, the brief item by Karen Kaplan referred to the proposed bond measure to refinance the agency, known formally as the California Institute for Regenerative Medicine (CIRM). She wrote about the impact of the cash handed out by the agency and said, ,

"A CIRM-funded treatment developed at UCLA has led to a cure for dozens of children who were born without a functioning immune system.

"All of this was made possible by CIRM’s initial allocation of $3 billion. Now that money has been spent, and voters will be asked to renew their commitment to stem cell science by approving a $5.5-billion ballot initiative in November 2020."

At Bloomberg Law online, Joyce Cutler wrote that supporters of the agency are refining their pitch and this month will begin seeking more than 600,000 verified signatures of registered voters to place the proposed initiative on the  ballot.  She wrote,

"They will face doubters and lots of competition for voters’ attention and tax dollars. 

"'I think that a funding decision by popular vote that locks in a substantial income stream over a decade over a particular use is not good social policy. It’s not to say that the money isn’t being used for a good purpose,' said Ken Taymor, deputy director of the Forum for Collaborative Research at the University of California Berkeley School of Public Health. 

"The question, said Taymor, is 'can we afford to spend it on what CIRM is doing as opposed to other public health needs?'"

Cutler covers more ground than does the piece in the Times. She has comments from Jeff Sheehy, a CIRM board member since 2004; Arnold Kriegstein, a CIRM grant recipient and director of UC San Francisco’s Developmental and Stem Cell Biology Program, and Zev Yaroslavsky, a specialist in state politics and government and director of the Los Angeles Initiative at UCLA’s Luskin School of Public Affairs, and Jeanne Loring, a CIRM grant recipient and chief scientific officer of Aspen Neuroscience.

Two samples:

Sheehy said he would give the agency an A plus for advancing science. He continued,

"We supported the field, we’ve grown biomedical research and biotech in California tremendously. I think if you look at returns to the state, you might say B minus. And that speaks to one of my frustrations as we try to move forward, as we talk about moving forward, that we really haven’t grappled with the mechanics of ensuring that the state gets a real return on its investment."

Yaroslavsky said,

"They’re (voters) are going to have to make a decision whether roads are more important than stem cell research, is education more important than water infrastructure."

California Stem Cell Report Taking Break

The California Stem Cell Report is on holiday until Jan. 6. Look for a fresh posting then. Happy New Year to all.

Possible Parkinson's Stem Cell Treatment in San Diego, $6.5 Million and Loads of Media Coverage

KPBS 2018 video

A San Diego firm that has $22 million worth of financial roots in the California stem cell agency this month snagged a bonus -- a ton of favorable attention for its efforts to treat Parkinson's disease. 

The fledgling business is Aspen Neuroscience, Inc., which was co-founded by Jeanne Loring, professor emeritus at the Scripps Research Institute, and Andres Bratt-Leal, also formerly of Scripps. 

Earlier this month, Aspen received $6.5 million in seed funding from Domain Associates and Axon Ventures. The announcement of the cash infusion triggered a spate of news articles.

In a piece on the Biocentury web site, writer Elizabeth Eaton quoted Kim Kamdar, a partner at Domain, as saying, 

"Aspen is the only company taking an autologous approach to cell therapy for Parkinson's disease." 

The approach can avoid the immunosuppression necessary for allogeneic therapies. Eaton continued,  

"Such (immunosuppression) treatment, which can last for a year or more to ensure a patient's immune system doesn't react to the transplant, can make patients more susceptible to viral diseases and cancer.

"While companies like BlueRock Therapeutics L.P. are exploring allogeneic treatments, Aspen was able to break into the autologous space thanks to its genomic tools, co-founder and CSO Jeanne Loring told BioCentury.

"Aspen is built on Loring's research at the Scripps Research Institute and the PluriTest, an assay she developed after discovering that pluripotent cells have a distinct genetic signature. The patented test shows whether a cell has achieved pluripotency, which allows researchers to avoid conducting more time-consuming confirmatory tests."

Loring has received nearly $22 milllion from the state stem cell agency, formally known as the California Institute for Regenerative Medicine (CIRM), including $4.5 million as a co-PI. She told the California Stem Cell Report, 

"All of my CIRM awards contributed to this project. We used those funds to develop our genomics tools and databases and to develop improved methods for handling the cells.  The only specific CIRM grants supporting this project were DISC2-09073 and the progress award DISC2P-11595."

Loring also credited Summit for Stem Cell Foundation of San Diego for supporting and financing her work as far back as 2011. Joining Domain in the $6.5 million in funding were Alexandria Venture Investments, Arch Venture Partners, OrbiMed Advisors and Section 32.

Here are links to additional stories on Aspen and the $6.5 million in funding: San Diego Union-Tribune, San Diego Business Journal, Endpoints News, FierceBiotech, Xconomy, BioWorld, Parkinson's News Today and BioSpace. 

Aspen Neurosciences video

Latina Cancer Advocate Ysabel Duron Named to California Stem Cell Board

Ysabel Duron

The governing board of the $3 billion state stem cell program has a new member, Ysabel Duron, an award-winning Latina journalist and president of the Latino Cancer Institute.

Her appointment comes as Sherry Lansing, who was the first woman to head a major Hollywood studio, has left the board. Lansing is president of the foundation bearing her name, which is involved deeply in cancer research, and is a member of the University of California board of regents. 

On its blog, the stem cell agency said,

"Ms. Duron was a journalist and TV news anchor for more than 43 years winning numerous awards, including two EMMYS. She has been inducted into the Hall of Fame of the National Association of Hispanic Journalists and given the Living Legacy Award by the Chicana/Latina Foundation.

"As a journalist she covered her own battle against Hodgkin’s Lymphoma, using her reporting to help raise awareness about the disease and the health disparities involved in treating it in communities of color."

The agency quoted Duron as saying,

"Usually I am looking for the best return for the public health! This appointment gives me a new learning opportunity to understand a very complex issue, and, make it bite size so the public, patients and advocates will understand how these scientific revelations will impact lives in the short term and the long run. As a steward of taxpayer dollars, I also want to make sure there is equity for communities across California, and that the research serves all of us."

Lansing served on the agency's 29-member board since its inception in 2004. In her letter of resignation, she said,

"This is just the beginning for stem cell research, and yet we are at a critical inflection point in the life of this organization - with funds coming to an end and promising research that must wait for the next CIRM(the stem cell agency). It is for this reason that I am stepping down now, so that I can dedicate more time to getting the next initiative on the ballot."

State Controller Betty Yee appointed Duron. The agency's board currently has two vacancies.

'Natural Killer' Cancer Cell Treatment Advances; State Stem Cell Agency Pleased

The California stem cell agency this week touted a possible "off-the-shelf cancer killer" that the state enterprise is helping to develop with $4 million.

The company involved in the treatment is Fate Therapeutics, Inc., of San Diego, a publicly traded firm working on induced pluripotent stem cells (iPSC) that have been genetically engineered to enhance their anti-tumor activity. The product is called FT516.

Writing this week on the agency's blog, Kevin McCormack, senior director of communications at the agency, said,

"Fate Therapeutics has developed an off-the-shelf therapy (thanks to (agency funding) that could, theoretically, be stored at hospitals and clinics around the country and used whenever it’s needed for anyone who needs it."

McCormack said that a CAR-T approach uses a patient's own "re-engineered" cells to kill cancer tumors. He wrote, 

"But the thing that makes it so appealing – using the patient’s own cells – also makes it really complicated and expensive. Creating a custom-made therapy from each patient’s own cells takes time and costs a lot of money. But now (the Fate Therapeutics) approach could change that."

Fate uses NK (natural killer cells). Maxx Chatsko of the Motley Fool financial web site reported,

"It's the first cell product of any kind derived from a clonal master iPSC line to be evaluated in a clinical trial in the United States. The 'clonal master iPSC line' part essentially means it can be manufactured more easily than immunotherapies requiring cells to be extracted from patients. That's why it's called 'off the shelf.'"

McCormack continued, 

"At this year’s meeting of the American Society of Hematology (ASH) Fate announced that the first patient treated with this new approach seems to be doing very well. The patient had acute myeloid leukemia and wasn’t responding to conventional treatments. However, following treatment with Fate’s FT516 the patient responded quickly and...was able to leave the hospital and spend Thanksgiving with his family. 

"Equally impressive is that 42 days after being treated with FT516, the man showed no signs of leukemia in either his bone marrow or blood."

The product is being licensed exclusively to Fate by the Memorial Sloan Kettering Cancer Center

Fate Therapeutics' stock closed today at $18.79, down 13 cents. Its 52-week high was $22.82. The 52-week low was $11.00. Last September it raised $173 million in a stock offering.

LA Times: $5.5 Billion Measure for California Stem Cell Agency Could Actually Be a 'Downfall'

The Los Angeles Times, California's largest circulation newspaper, carried a piece today that called for withdrawal of the proposed, $5.5 billion ballot initiative that would save the financial life of the state's stem cell agency.

The article by Pulitzer Prize-winning business columnist Michael Hiltzik said the measure would perpetuate many of the flaws in the ballot proposition in 2004 that created the agency, officially known as the California Institute for Regenerative Medicine (CIRM). 

In fact, Hiltzik said, the new measure "makes some of them worse."

"That’s dangerous," Hiltzik continued, "because although the measure could fuel the stem cell program for years to come, it might also prompt a repudiation by voters sensitive to its many imperfections. Such an outcome would be tragic for California and the advanced science already supported by CIRM."

Hiltzik's opinion piece was the first extended look at the proposed stem cell initiative by a major media outlet in California. It appeared on the front page of today's business section of the Times. On the paper's web site, the article was featured as a "weekend read" at the time of this writing. 

The Times says it has about 2 million readers on Sunday. It is a go-to source for other journalists when they are researching stories and trying to understand major issues facing the state. 

Hiltzik has followed the $3 billion agency since its inception and has been highly critical. Last spring, however, he wrote that the agency had "proved its value." In today's piece, he had a lot to say about the good work of the agency. 

In his first two paragraphs, Hiltzik declared that CIRM "has made great strides in advancing what’s known as regenerative medicine and placing California at the center of the developing science." Hiltzik also said that CIRM has financed state-of-the-art laboratories, attracted preeminent scientists and "brought scores of promising treatments for severe chronic diseases to the point of clinical trials."

But he also noted that the agency has "has so far failed to yield a single marketable clinical product." He continued, 

"That’s despite the sales pitch for Proposition 71 in 2004 -- that all that stood in the way of 'cures' for Parkinson’s, Alzheimer’s, spinal cord injuries and other maladies was money."

The greatest flaw of the agency, however, has more to do with public policy than science, Hiltzik wrote. And that is the exemption of the agency from the usual oversight by the legislature and the governor, an exemption contained in the measure that created the agency.

Voters provided CIRM with $3 billion in bond funding 15 years ago. 
Today that money has all but run out. Robert Klein, a real estate developer, first chairman of the agency and who sponsored Proposition 71, is now once again sponsoring the latest initiative to give it billions more. 

Hiltzik concluded,

"(Klein) should withdraw his measure, and CIRM’s leadership should write a new one or work with Gov. Newsom and the Legislature to map out the program’s next act. 

"CIRM’s leadership needs to show the public that it’s capable of taking charge of the program’s destiny. If it’s not willing to make its own case for CIRM’s continued existence, how can it persuade voters to give it one cent more?"

San Diego Biotech Reporter Bradley J. Fikes Dies at 61

Bradley J. Fikes, journalist
San Diego U-T photo

When biotech reporter Bradley J. Fikes died last Wednesday at the age of 61, he was described in his newspaper as "part Dr. Dolittle, part Inspector Gadget."

The piece in the San Diego Union-Tribune was a tribute to Fikes, whose family said he died of natural causes at home. The paper said he was "an ever-on-the-move ball of energy" and beloved by his colleagues.

Fikes occupied a special position in California journalism. He was one of the few daily newspaper reporters who regularly covered the biotech industry and its research community. Fikes was also one of the few who delved into the affairs of California's 15-year-old, stem cell research agency. 

Last month Fikes penned a lengthy piece for the San Diego Union-Tribune that involved the agency, known officially as the California Institute for Regenerative Medicine (CIRM). The story dealt with a blood cancer drug called fedratinib and the relentless, years-long journey before the research, which was partly funded by CIRM, came to fruition. 

Fikes began the story like this:

"Cancer patients hold onto hope the way some people hold onto the lap bars of roller coasters: As if their lives depend on it.

"And then they get ready for the ups and downs."

Kevin McCormack, the stem cell agency's senior director of communications, wrote on the agency's blog that Fikes' story captured the setbacks, twists and turns and the years that it took before the treatment ultimately emerged. 

McCormack said yesterday, 

"Everyone at CIRM was stunned and saddened by the news. Brad was a real character, and a real gentleman. His unique sense of fashion made him hard to miss, even at a crowded conference, but it was his curiosity and his clear love of science that really stood out. He didn’t just report on stem cell research as a job, it was something he loved and that came across in his writing. He had a tremendous gift for taking complex science and turning it into everyday English. He was also great fun to hang out with. He had a wonderful sense of humor and a wry way of seeing the world. I shall miss him. We all will."

One of Fikes' colleagues, Michelle Guerrero, a Union-Tribune illustrator and graphics reporter, said in Fikes' obituary, 

“He had the wonderment of a child, the complexity of a scientist and an artful way of coming up with the words to explain it all.”

Fikes cut a colorful figure as he divined the doings at Scripps and Salk and at the other hallowed halls of science.  The Union-Tribune's piece on Fikes said, 

"Fikes was impossible to miss. By his own admission, he was a walking fashion disaster. He wore odd-colored business shirts that clashed with his suspenders, and slacks that never made contact with an iron. At times, cellphone cables hung out of his pockets like limp licorice."

Fikes was a graduate of San Diego State University where he first began writing the news. The campus newspaper wrote its own account of the life and times of Bradley J. Fikes. The piece carried comments from his colleagues including Gary Robbins who sometimes teamed with Fikes on stories. Robbins praised not only Fikes' journalism but his humanity as well. Robbins said, 

"He had poise, he had professionalism, he was a gentleman.”

Media Coverage: California Stem Cell Agency Receives Slight Whack in Los Angeles Times

California's $3 billion stem cell research program, now down to only $27 million, popped up in the Los Angeles Times this week in a piece that dealt with presidential politics, term limits and dubious ballot measures.

The mention consisted of only two paragraphs. But it demonstrated, at least for the state stem cell agency, the flaws of excessive generalization in P.T. Barnum's famous saying that “Any kind of publicity is good publicity as long as they spell your name right.”

Los Angeles Times columnist Michael Hiltzik wrote yesterday about "crowd pleasing" pitches to voters that actually contain "few virtues."  The starting point was term limits for congressmen and women. Hiltzik noted that such measures, in fact, do not serve the public well because they enhance the power of lobbyists. And then he went on to discuss ballot initiatives in California. 

Hiltzik, a Pulitzer Prize winner and author of "Big Science," wrote, 

"It's not unusual for ballot measures to win the approval of voters despite provisions that work directly against voters’ interests. That includes Proposition 71 of 2004, which created California’s $6-billion stem cell program. (See below for a look at the $6 billion figure and the nominal cost of the stem cell effort.)

"Proposition 71 was sold to voters as a gateway to cures for a host of intractable diseases, a promise hopelessly incompatible with the way science is done. Its promoters also tried to make a virtue out of its nearly total independence from legislative oversight, another artifact of anti-government sentiment. The stem cell program has indisputably supported excellent science, but its lack of legislative oversight remains a flaw."

The lack of legislative oversight will continue under the proposed 2020, $5.5 billion ballot initiative that is now before California election officials. If approved by voters in 11 months, the initiative will save the California stem cell agency from financial extinction. 

The new initiative is sponsored by Robert Klein, the first chairman of the state stem cell agency. Klein's measure does not address a number of sharp criticisms of the program, including those found in a $700,000 study of the agency by the prestigious Institute of Medicine. 

Klein's new initiative is sort of a son of Proposition 71. Klein also directed the writing of the 2004 measure, which did not provide any source of stem cell funding beyond the $3 billion in state bonds. 

As for the $6 billion figure cited by Hiltzik, it originated in a 2004, state ballot analysis of Proposition 71. The figure was an estimate of the total cost, including interest, to taxpayers of the stem cell agency, which is financed by $3 billion borrowed by the state. 

The interest estimate did not anticipate the 2008 Great Recession and the related drop in interest rates. The latest estimate is that the total will be about $4 billion. Read more about that estimate here. 

Rare Media Coverage of $5.5 Billion California Stem Cell Ballot Measure

The journal Science is carrying an article about the "delicate negotiation" involving the California stem cell agency and the man who is sponsoring a ballot measure that would keep the research effort alive with $5.5 billion in cash. 

The piece by Kelly Servick followed a two-hour meeting last Friday of the governing board of the agency, formally known as the California Institute for Regenerative Medicine (CIRM). Klein was in attendance and agreed to additional changes in the measure beyond those he announced to the California Stem Cell Report. 

There was no disagreement between Klein and the agency concerning the amount of money involved, which is much needed by CIRM. It has only $27 million left, compared to the up to $300 million annually that it normally spends on awards. The $27 million is earmarked for sickle cell anemia. 

The piece in Science represented rare national or even California media attention to refinancing the agency, which has pumped $2.7 billion into stem cell research in the Golden State in the last 15 years. Beyond the refinancing, the Klein initiative would make sweeping changes in the agency and governance. It also does not deal in a significant way with some areas that have been the focus of sharp criticism over the years, such as conflicts of interest.

Servick's article captured some of the highlights of last Friday's CIRM board meeting. She also reported, 

"In July, Americans for Cures conducted a poll of about 1150 likely voters and found a 65% approval rating for re-funding CIRM, Klein says."

Servick wrote, 

"Many researchers credit the agency with creating a strong infrastructure for stem cell research in the state and shepherding dozens of experimental treatments into human trials. But no CIRM-funded stem cell therapy has yet won FDA approval, leading critics of the agency to ask whether the initial 2004 initiative, born from opposition to a ban on federal funding for embryonic stem cell research, promised 'cures' it couldn’t deliver." 

Klein has until 5 p.m. today to file his amended initiative with state election officials. If it qualifies for the ballot with more than 600,000 signatures it will be brought to voters next November.

$5.5 Billion Stem Cell Ballot Measure: Questions in California but Apparently No Foot-Dragging

Some directors of the California stem cell agency this morning expressed a number of reservations about a $5.5 billion ballot initiative to stave off its financial demise, but none appeared likely to oppose the proposal 

In a special, two-hour meeting of the agency's governing board, questions were raised about provisions that would restrict the board or mandate programs, ranging from training to lab equipment. A provision to increase the size of the board from 29 to 35 was criticized and supported. A new effort to deal with the affordability of stem cell therapies was praised. But some aspects were questioned.   

The topic of the day was a complex, 30-page, proposed ballot initiative sponsored by Robert Klein, the first chairman of the agency, who has already announced changes in his proposal. More alterations appeared to be in the works as the result of today's hearing, including elimination of some language dealing with pricing. The meeting did not go into details about that subject.

In addition to requiring the state to borrow $5.5 billion for stem cell and other "vital" research, Klein's proposal would expand the scope of the agency and alter its governance. It would be required to develop and finance efforts at making stem cell research more affordable. Brain disease research would receive special attention with a dedicated $1.5 billion out of the $5.5 billion. Training programs would be mandated and expanded at community and state colleges and much more.

(The CIRM summary of the initiative can be found here. A summary by the California Stem Cell Report can be found here.)

(Here is the text of the initiative as filed in October. Here is the text of the measure with Klein's proposed changes as of Nov. 14.)

The agency began its life in 2004 with $3 billion in state bond funding, authorized by California voters through a ballot initiative.  Known formally as the California Institute for Regenerative Medicine(CIRM), it is down to its last $27 million, which is devoted to a sickle cell program with the federal government. At its peak, the agency was giving away up to $300 million a year.

Today's meeting began with several of the board members declaring that the public should not perceive their questions and comments as indicating they did not support the effort to refinance the agency. Some had expressed concerns that opponents might use board comments to defeat the initiative next November. 

Steve Juelsgaard
CIRM photo

The board moved rapidly through the initiative during the morning meeting. CIRM director Steve Juelsgaard and others raised questions about the size of the board, declaring that large boards become disengaged from an enterprise. Os Stewart, who has served on the board since 2004, said he initially thought 29 members were excessive but feels differently after 15 years. 

Several members expressed concern about mandates that would tie the hands of the board. CIRM Director Francisco Prieto said, 

"The science can surprise us."

Klein told the board that "we have fundamental obligation to give the voters an idea where their money is going." He also said that the mandates do not have legal timetables.

Today's meeting was very lightly attended, according to an agency spokesman, mostly by CIRM staffers. Only one member of the public addressed the board, Don Reed, a longtime patient advocate and supporter of the board.

Questions about the measure arose publicly this month after longtime director

Jeff Sheehy
CIRM photo

Jeff Sheehy authored a 3,300-word critique of the Klein measure, declaring it amounted to "too little and too much."  At the end of today's meeting, Sheehy did not express reservations that would prevent him supporting the measure. 

Klein unofficially modified his measure this week. He has until Monday at 5 p.m. to actually file the changes with state elections officials. The official comment period for the initiative is closed. But Klein said yesterday that interested parties can send comments and suggestions to him through Saturday at this email address: castemcell2020@gmail.com. 

Comments filed directly with Klein are not a public record. If a reader would like to share his/her comments with the public, please send a copy of them to the California Stem Cell Report at djensen@californiastemcellreport. We will carry them verbatim.

Here is a link to a guide to the stem cell initiative information maze, which is updated as other key information surfaces.

California Stem Cell Agency's Summary of $5.5 Billion Measure to Save its Financial Life

The California stem cell agency late yesterday posted its own summary of a proposed $5.5 billion ballot initiative to keep it from basically going out of business next year.

The summary is about 1 1/2 pages long compared to the 30 pages of the actual initiative, which is the subject of a hearing this morning by the governing board of the agency. 

The California Institute for Regenerative Medicine (CIRM), as the agency is formally known, is down to its last $27 million. It came into being in 2004 with $3 billion in state bond financing. 

Also posted on the meeting's agenda is a five-page memo from 2017 discussing what the agency can and cannot do in connection with election activities such as a ballot initiative campaign.  Last month the board was given a seven-page memo about such activities. Both memos confirm that the agency may use public funds to draft its own initiative.

Neither of the memos discuss the pros and cons of the ballot measure sponsored by Robert Klein, who ran the ballot campaign that created the stem cell agency 15 years ago this month.

Here is an updated guide to the key documents and information concerning the initiative.

Robust Defense of $5.5 Billion California Stem Cell Measure; State Hearing Scheduled for Tomorrow Morning

The man behind the $5.5 billion California stem cell initiative this afternoon vigorously defended the measure and released details of fresh changes in the proposal, which will be scrutinized tomorrow morning by the directors of the financially strapped state stem cell agency.

Robert Klein file photo by California Stem Cell Report

Despite the fact that agency faces its fiscal demise next year, the initiative is the only proposal around to continue the agency's funding. It ran afoul last week of questions from some of the agency's directors. One of those directors wrote a 3,300-word critique of the proposal by Robert Klein, the first chairman of the agency and who directed the writing of the 2004 ballot initiative that created the agency. A special meeting was called for tomorrow morning.

In addition to providing $5.5 billion in state bonds, Klein's proposal would significantly expand the reach and change the governance of what is formally known as the California Institute for Regenerative Medicine(CIRM). The measure would require the agency to launch a major effort to make "affordable" what are extremely expensive stem cell and genetic treatments. The proposal would also enlarge its governing board from 29 to 35. The board has been criticized sharply for the size of its board.

Klein said in an emailed statement that the changes he made in the wake of CIRM directors' concerns basically follow the outlines of what he released yesterday. He stoutly defended the agency's current work and his plans to expand its scope.

He said in an eight-page statement to the California Stem Cell Report.

"Patients and their families should not have to suffer more delays because of ill-conceived agendas that obviously do not fit into the new initiative, with a scope that has already been court validated."

Klein added,

"The federal government remains a threat to scientists and physicians investigating the best cell sources for developing therapies for patients. Just within the last nine months there have been new restrictions imposed by the federal government based upon ideological grounds."

Klein, a Palo Alto, Ca., real estate developer, is under a state deadline of 5 p.m. on Monday to make any changes. As sponsor of the 2020 initiative, he is the only person legally entitled to make changes. The measure then will go through a lengthy state elections process. More than 600,000 registered voters will have to sign petitions for the measure if it is to make it onto the November 2020 ballot.

Klein noted that the state deadline has passed for officially making public comments. But he said suggestions and comments can be sent to him through Saturday Nov. 16 at this address: castemcell2020@gmail.com.  Any such comments are not a public record. If a reader would like to share his/her comments with the public, please send a copy of them to the California Stem Cell Report at djensen@californiastemcellreport. We will carry them verbatim.

The public can listen in and participate in tomorrow's meeting via the Internet or at telephonic locations throughout California. An updated list of those locations is here. Information on Internet access is on the meeting agenda.

Below is the text of the statement Klein sent this afternoon to the California Stem Cell Report. In it, he says the full text of the current version of the actual initiative is available on the Americans for Cures web site, a stem cell patient advocacy group that he founded and chairs.

   2020 Update – the California Stem Cell Research, Treatments, And Cures Initiative of 2020 11.14.19 by Anonymous 2ZGCBei on Scribd

A Guide to Information on the $5.5 Billion California Stem Initiative

Trying to wade through the information maze surrounding the proposed, $5.5 billion California stem cell initiative? Here are links to key documents, online postings and information dealing with the proposal and the ballot initiative process.

This list of information sources was updated Nov. 14, 2019, at 8:54 PM PST.

How ballot initiatives work in California

• Ballotpedia on initiative law
• Wikipedia overview of California initiative process
• How changes can be made in an initiative

Text of the 30-page, proposed $5.5 billion measure

Text of the revised version of initiative as of Nov. 14, 2019

A summary of the measure from the California Stem Cell Report

A summary of the measure from the stem cell agency

A critique of the proposal by CIRM board member Jeff Sheehy

A critique of the proposal by CIRM grant recipient Jeanne Loring

An eight-page statement from Robert Klein, author of the initiative, to the California Stem Cell Report.

Proposed changes in the initiative offered by its sponsor, Robert Klein

How CIRM can write its own initiative

An article on CIRM board reaction to the initiative, complaints about "handcuffs"