LATimes: 'Secret Life' of Former USC Med School Dean and California Stem Cell Agency Director

Carmen Puliafito (right), a member of the governing board of the California
stem agency, and Robert Klein, then chairman of the agency. USC photo 2009

The headline in the Los Angeles Times this morning said:

"An overdose, a young companion, drug-fueled parties: The secret life of USC med school dean"

Put together by a team of five reporters, the article called Carmen Puliafito a "towering figure," a "renowned eye surgeon" and a prodigious fund raiser, bringing in more than $1 billion for USC by his own estimate. And then it said, 

"During his tenure as dean, Puliafito kept company with a circle of criminals and drug users who said he used methamphetamine and other drugs with them, a Los Angeles Times investigation found.

"Puliafito, 66, and these much younger acquaintances captured their exploits in photos and videos. The Times reviewed dozens of the images."

Puliafito resigned his $1.1 million position as dean in March 2016, declaring he wanted to pursue outside opportunities. He still serves, as a gubernatorial appointee, on the board of the California Institute for Regenerate Medicine (CIRM), as the stem cell agency is formally known. The board position pays $100 a day for meeting attendance. The board meets 10 to 12 times a year. 

USC has received $110 million from CIRM since 2004, ranking No. 6 among all California institutions that have won stem cell awards from the state agency. USC has had a representative on the board since its inception in 2004.

Paul Aisen, San Diego UT photo, Howard Lipin

Puliafito remains on the USC faculty and continues to accept patients. He is a "central witness" in a $185 million, legal wrangle involving UC San Diego researcher Paul Aisen. Puliafito has described himself as the "quarterback" in the effort to hire Aisen away from UC San Diego. The Times wrote,

"Curing Alzheimer’s is a top priority for government agencies and pharmaceutical companies, and Aisen’s lab was overseeing groundbreaking research, including drug trials at 70 locations around the world. More than $340 million in funding was expected to flow to his lab, according to court records.

"UC contended in its suit that its private school rival went beyond the bounds of academic recruiting by targeting professors and labs based on grant funding. The suit accused USC of civil conspiracy, aiding and abetting breach of fiduciary duty and other misconduct."

Puliafito's current term on the stem cell agency board term expired Nov. 3, 2016, according to a governor's office document. However, members of the board may serve until a replacement is named. He was appointed in 2008 by then Gov. Arnold Schwarzenegger and reappointed in 2010, also by Schwarzenegger, according to the governor's office document.

In response to a query, Kevin McCormack, senior director of communications for the agency, said via email,

"We were surprised to read the allegations about Dean Puliafito in the Los Angeles Times. Since being appointed to the CIRM Board by Governor Arnold Schwarzenegger, in late 2008, Dean Puliafito has served with distinction, bringing knowledge, expertise and deep commitment to our mission."

The California Stem Cell Report also asked Brown's office for comment. When it responds, the full text of the comments will be carried.

The reporters who put together the story are Paul Pringle, Harriet Ryan, Adam Elmahrek, Matt Hamilton and Sarah Parvini.

(Editor's notes: An earlier version of this item said that Puliafito was reappointed by Brown in 2010. The re-appointment was by Schwarzenegger. Brown was elected in 2010 but did not take office until 2011.
(This article was picked up Capitol Weekly, a news and information service devoted to California public policy issues. The Capitol Weekly version can be found here.)

Two Rounds of Transplants, 70 Drugs: An Advocate's Journey into Stem Cell Safety

Lukas Wartman
Science magazine, Whitney Curtis photo

The California stem cell agency today related a compelling story concerning a physician named Lukas Wartman who received "a life-saving stem cell treatment that is now threatening his health."

The headline on the item on the agency's blog, The Stem Cellar, said,

"One man’s journey with leukemia has turned into a quest to make bone marrow stem cell transplants safer"

Karen Ring, a scientist herself and overseer of social media at the California Institute for Regenerative Medicine (CIRM), wrote about the case after she read an article by Jon Cohen in Science Magazine concerning Wartman. He is a physician at the Washington University School of Medicine. Ring wrote,

"He was first diagnosed with a type of blood cancer called acute lymphoblastic leukemia (ALL) in 2003. Since then he has taken over 70 drugs and undergone two rounds of bone marrow stem cell transplants to fight off his cancer."

The first transplant came from his brother but the second round was from an unrelated donor and was different. Ring said, 

"While the second transplant and cancer-fighting drugs have succeeded in keeping his cancer at bay, Wartman is now suffering from something equally life threatening – a condition called graft vs host disease (GVHD). In a nut shell, the stem cell transplant that cured him of cancer and saved his life is now attacking his body."

Ring discussed the currently available treatments and continued, 

"Another promising therapy is called Prochymal. It’s a stem cell therapy developed by former CIRM President and CEO, Dr. Randy Mills, at Osiris Therapeutics. Prochymal is already approved to treat the acute form of GVHD in Canada, and is currently being tested in phase 3 trials in the US in young children and adults.

"While CIRM isn’t currently funding clinical trials for GVHD, we are funding a ($20 million) trial out of Stanford University led by Dr. Judy Shizuru that aims to improve the outcome of bone marrow stem cell transplants in patients. Shizuru says that these transplants are “the most powerful form of cell therapy out there, for cancers or deficiencies in blood formation” but they come with their own set of potentially deadly side effects such as GVHD."

Ring said that Wartman's battle with the disease has made him "one of the strongest patient voices advocating for new treatments" for his affliction. Ring said Wartman is an inspiration to funding agencies and other scientists. She said,

"We don’t want these patients to suffer quietly. Wartman’s story is an important reminder that there’s a lot more work to do to make bone marrow transplants safer – so that they save lives without later putting those lives at risk."

California's Stem Cell CEO Search: $3 Billion Agency Needs New President

California's $3 billion stem cell research program is looking for a new president to carry the state agency through what may be the last three years of its life.

The search committee of the agency's 29-member governing board meets July 17 to discuss the matter behind closed doors. But members of the public will have a chance to comment during an open portion of the meeting. Or they can email comments directly to the board at info@cirm.ca.gov ahead of the meeting.

So far, no comments have been received from the public concerning the selection of a new president, according to an agency spokesman.

Currently Maria Millan is the interim president and CEO of the 12-year-old program, replacing Randy Mills, who is taking a job in Minneapolis. Mills has said that Millan is his choice as a successor. Millan joined the agency in December 2012 and was vice president for therapeutics before her interim appointment.

Last month, directors of the California Institute for Regenerative Medicine (CIRM), as the agency is formally known, ratified her as interim president, voicing no reservations in public about the action.

The current salary for the position ranges from $311,000 to $575,000 annually.

The main location for next week's session is the Claremont Hotel in Berkeley. Teleconference locations where the public make direct audio comments to the board are available in Sacramento and at UCLA in Los Angeles. Details are available on the agenda.

The agency has projected that it will run out of funds for new awards in June 2020. However, the end of funding could come sooner or later depending on the agency's rate of spending for awards. The agency relies on money borrowed by the state (bonds), which roughly doubles the cost of the research to about $6 billion because of  interest costs.

Earlier, there was talk about a possible $5 billion bond election in 2018 to continue funding the agency. However, that possibility appears to have been delayed until November 2020, which would be beyond the date when the agency is currently expected to run out of cash.

Bob Klein, the Palo Alto real estatement investment banker who led the 2004 ballot campaign that created the stem cell agency, is the chief voice behind the idea of another bond issue. He has not responded to inquiries from the California Stem Cell Report about his latest bond plans. This blog will carry the full text of his comments if he does respond.

Here is a list of the members of the CIRM search group: Deborah Deas, Judy Gasson, David Higgins, Steve Juelsgaard, Al Rowlett, Jeff Sheehy, Jonathan Thomas, Art Torres and Kristiina Vuori. Biographical sketches can be found here.

Alpha Thalassemia to Strokes to Zika: A $64 Million Boost by California for Stem Cell Research

BURLINGAME, Ca. -- The California stem cell agency today awarded more than $64 million for research tackling cancer, stroke, leukemia, heart failure, brain injury, Zika and much, much more

The agency's directors allotted $44 million of the amount for two clinical trials and potentially two more trials, which are the last stages before approval of treatments for use by the general public. Prior to today's action, the agency -- known officially as the California Institute for Regenerative Medicine or CIRM -- was helping to finance 27 active clinical trials.

The agency has yet to develop therapies that would be available for widespread use that voters were led to expect in 2004 when they approved creation of the $3 billion research effort. CIRM expects to run out of cash for new awards in three years. 

The most advanced clinical research approved today involves a phase 2b trial for which CIRM ponied up $20 million. The effort is also backed with co-funding of $22 million by the recipient, SanBio, Inc., a Mountain View, Ca., subsidiary of a Japanese firm, SanBio Co., Ltd. 

The review summary of the application (CLIN2-10344) said the trial had already demonstrated safety and "a trend toward efficacy" involving ischemic strokes. The research is aimed at improving motor function of victims of strokes, which is the leading cause of adult disability.

Directors also approved $5.3 million for work aimed at starting a clinical trial for a treatment that would enhance the brain's ability to create new blood vessels to replace those damaged during a stroke. (The review summary for CLIN-09433 can be found here.)

The funds went to Gary Steinberg of Stanford University.

Maria Millan, interim president and CEO of the agency, said in a news release,

"Today the CIRM Board approved two very different methods, using different kinds of stem cells, to address this need. By funding 'multiple shots on goal' we believe that we have a better chance of finding a way to repair the damage caused by stroke and give people a better quality of life.”

The second phase 2 clinical trial backed by CIRM involves transplantation of maternal bone marrow stem cells into fetuses discovered to have alpha thalassemia major, an affliction that is almost always fatal in utero.

The $12.1 million award (CLIN2-09183) went to Tippi MacKenzie of UC San Francisco. (The review summary can be found here.)

The fourth application (CLIN1-09776) involves acute myeloid leukemia and preparation for a clinical trial application. CIRM awarded $6.9 million to Cellerant Therapeutics, Inc., of San Carlos. The agency reported that $1.7 million in co-funding would be provided by Cellerant. (The review summary can be found here.)

Also approved was $20 million for 13 awards in what the agency calls its Quest program, which is aimed at supporting promising, less developed research that is likely to move on to the next stage of development within two years. Twenty-six applications were rejected.

The review summaries and scores on the awards can be found in this document. The list of recipients can be found in the CIRM press release.

Plans Scrubbed for a 2018 Bond Measure for California Stem Cell Research

BURLINGAME, Ca. -- Plans to ask California voters in 2018 to approve a $5 billion bond issue to finance the California stem cell agency have been shelved,  a director of the agency said today.

Jeff Sheehy, a San Francisco county supervisor, said that the key backer of the proposal had informed him that no bond measure would be offered to voters before 2020, presumably at the presidential general election.

At a meeting of stem agency directors, Sheehy said this morning that Bob Klein, who led the 2004 ballot campaign that created the agency, had told him by telephone that a 2018 bond measure was now off the table.

Sheehy did not go into reasons for the delaying the bond measure. However, conventional political wisdom holds that bond measures have a better chance of approval during a general election that attracts a larger voter turnout.

J.T. Thomas, chairman of the agency's board, said he was forming a transition committee to make plans for various alternatives, including shutting down the agency in 2020.  The California Institute for Regenerative (CIRM), as the agency is formally known, currently estimates that it will run out of cash for new awards in just three years.

Other alternatives to be examined include funding from a new bond measure and some sort of private-public partnership.

CIRM Directors Open Morning Meeting

BURLINGAME, Ca. -- Directors of the $3 billion California stem cell agency opened their meeting this morning at 9:06 a.m. with  introduction of the newest member of the board, Linda Malkas of the City of Hope, and a report from Chairman Jon Thomas.

Conflict List for CIRM Directors Clinical and Preclinical Awards

Here is the conflict of interest list for directors of the California stem cell agency compiled by the agency in preparation for voting today on applications for clinical and preclinical awards.  The list identifies the directors with conflicts on specific applications. The directors with conflicts are barred from voting on those applications or discussing them during today's meeting.

  Conflict of Interest List CIRM Directors Clinical Awards June 2017 by DavidJensen on Scribd

Conflict List for CIRM Directors on Quest Awards

Here is the conflict of interest list for directors of the California stem cell agency compiled by the agency in preparation for voting today on applications for Quest research awards. The list identifies the directors with conflicts on specific applications. The directors with conflicts are barred from voting on those applications or discussing them during today's meeting.

  Conflict of Interest List CIRM Directors Quest Awards June 2017 by DavidJensen on Scribd

Check Out All the Breaking California Stem Cell News Tomorrow -- Right Here on This Web Site

The California Stem Cell Report will bring you on-site, gavel-to-gavel coverage of tomorrow's meeting of the governing board of the Golden State's $3 billion stem cell agency.

The board is slated to approve $64 million in new awards for research into afflictions ranging from strokes to liver disease. Also to be approved is the appointment of Maria Millan as the interim president of the stem cell program. The excitement is scheduled to begin at 9 a.m. PDT.

For those who prefer to monitor online the nearly day-long meeting themselves, instructions can be found on the agenda. If you would like to attend, the meeting is in Burlingame at the San Francisco Airport Marriott Hotel. Two other public, telephonic locations can be found in Los Angeles and one each in Beverly Hills, Sacramento and La Jolla.

Specific addresses and more information can be found on the agenda.

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.

Guest Posting: "Stem Cell Options Should be on the Table"

(Editor's note: The following is a guest posting from Joseph Rodota and Bernard Munos, both of whom have been active in California policy matters for some time. More biographical information can be found at the end of the item. The California Stem Cell Report welcomes diverse, well-considered views on California stem cell issues. If you have something that you think should see the light of day, please send it to djensen@californiastemcellreport.com.)

By Joseph Rodota and Bernard Munos

The sponsors of Proposition 71, the 2004 initiative that provided $3 billion in bonds to support stem-cell research, are readying a proposal to keep the agency alive after the last of these funds have been given out. According to recent news reports, a new $5 billion bond could be on the California ballot as early as November 2018.

Researchers supported by the California Institute for Regenerative Medicine (CIRM) have published hundreds of academic articles, but placed fewer than 30 drugs in clinical trials.

Even if all of these clinical trials resulted in drug candidates, they would still come up against the so-called “Valley of Death”—the well-documented shortage of funding for early-stage translational research.

California needs to move regenerative medicine from an academic timeline to a business timeline. The skills needed to turn an academic discovery into a commercial product are very different from the skills needed to be a successful academic scientist.

We proposed an alternative to continuing the current approach -- a state bond with three distinctive features:

A Focus on Entrepreneurs: Funds would be available only to companies, not academics (who would still be able to tap into billions in NIH funding for stem cell research);

A Focus on California: Only companies with a headquarters and a majority of employees in California, the nation’s center of overall innovation, or willing to relocate here; and

A Focus on Breakthrough Medicine: Only companies working on projects that have the potential to greatly impact patient health would qualify.

The University of California is well-qualified to administer this bond and report on its operations to the Legislature and the Governor, without the need for the cumbersome and controversial governance structure put in place by Proposition 71.

In exchange for the funds they receive, companies would tender to the University of California shares of their common stock, with an estimated value as determined by the most recent outside valuation or price set by investors. These shares would become part of the UC endowment -- and the University of California be free to sell or leverage these shares, or acquire additional shares, as it sees fit.

CIRM has over-invested in academic research, and under-invested in translating that research into therapies that cure diseases and prolong heathy lives. California needs to right that balance.

(Joe Rodota served as Cabinet Secretary to former California Governor Pete Wilson and director of policy for Arnold Schwarzenegger’s 2003 recall campaign. Munos is a senior fellow with FasterCures and the founder of the Innothink Center for Research in Biomedical Innovation.).

Here is a summary of the bond proposal.

SUMMARY -- California Next Generation Cures Bond -- June 2017 uploaded by DavidJensen on Scribd

California Stem Cell Agency Reports 'Streak of Good News' on Asterias Spinal Therapy

Asterias video

California's $3 billion stem cell program today reported ongoing rosy results in a clinical trial involving a therapy for severe spinal cord injury.

The treatment is being developed Asterias Biotherapeutics, Inc., of Fremont, Ca., with $14.3 million from the state research program, which is now in its 12th year.

The latest news was reported on The Stem Cellar, the agency's blog, and was based on a news release from Asterias, which is publicly traded. 

Todd Dubnicoff, the agency's multimedia editor, wrote the item which discussed nine-month results for the trial involving six patients paralyzed from the neck down. He said,

"In a nut shell, their improvements in arm, hand and finger movement seen at the earlier time points have persisted and even gotten better at 9 months."

Dubnicoff said that the level of improvement "can mean the difference between needing 24-hour a day home care versus dressing, feeding and bathing themselves."

He said,

"The impact of this level of improvement cannot be overstated. As mentioned in the press release, regaining these abilities, 'can result in lower healthcare costs, significant improvements in quality of life, increased ability to engage in activities of daily living, and increased independence.'"

In the Asterias press release, Edward Wirth, chief medical officer for the company, said,

“The new efficacy results show that previously reported meaningful improvements in arm, hand and finger function in the 10 million cell cohort treated with AST-OPC1 cells have been maintained and in some patients have been further enhanced even 9 months following dosing. We are increasingly encouraged by these continued positive results, which are remarkable compared with spontaneous recovery rates observed in a closely matched untreated patient population.”

The company also reported that no "serious adverse events" have surfaced that could be attributed to the therapy, which was initially developed  by Geron, Inc., which received $6.4 million from the stem cell agency. Geron abandoned the trial for financial reasons, and Asterias acquired the technology.

Aserias' stock price jumped nearly 13 percent today, hitting $3.55. Its 52 week high is $5.80 and its 52-week low is $2.30.

California Stem Cell Report Going Dark for a Few Days

The California Stem Cell Report expects to be offline for perhaps the next five to seven days.

The moving home of this web site, the sailing vessel Hopalong, will be making a passage north from Mazatlan into the Sea of Cortez, formally known as the Gulf of California. We will not have access to cellular or Internet coverage during that time.

A Longer Look at the Golden State's Stem Cell Research Efforts

Here is the overview of the California stem cell agency, published May 23, 2017, on Capitol Weekly and written by yours truly.

  California's Stem Cell Agency Confronts Uncertain Future: Capitol Weekly by DavidJensen on Scribd

Full Text: The California Stem Cell Agency's Response on its Accomplishments

  Stem Cell Agency's Response to Questions About Its Performance From Capitol Weekly_ the Newspaper of Califo... by DavidJensen on Scribd

California's 'Great Ideas,' Stem Cell Awards Target Universal Blood Supply, Alzheimer's and Much More

The California stem cell agency this week awarded a total of $1.4 million to six scientists to jump start their work in what it calls its "great ideas" program.

The awards went for research ranging from creation of a universal blood supply with human stem cells to mitigating Alzheimer's disease, which has seen an increase of 55 percent in its death rate from 1999 to 2014, according to results of a new study released yesterday

The agency said in a press release that the "Inception" program  "provides seed funding for great ideas that have the potential to impact human stem cell research, but need some initial support. It’s hoped this will enable the researchers to test their ideas, and give them the data they need to compete for more substantial funding."

Jonathan Thomas, chairman of the agency's governing board, said,

"This is a high risk, high reward program. We feel that a small investment now could produce enormous benefits later.”

The funding is small indeed. The California Institute for Regenerative Medicine(CIRM), as the agency is formally known, finances some clinical trials at $20 million a crack. The largest award in the "great ideas" program was $265,500.

The blood supply award was a reminder of another program that the agency used to entice star researchers to California. The blood grant went to Tannishtha Reya of UC San Diego. She came to California with her spouse, Robert Wechsler-Reya. He was lured by CIRM in 2010 with $5 million in funding to work at the Sanford-Burnham Medical Research Institute in La Jolla.. He has not received any further funding from the agency. This is the first CIRM award for Reya.

Another round of the Inception program is scheduled to open up in January 2018. Here is a link to the most recent request for applications. 

Here is a list of the winners with their application numbers. The summaries of reviewer comments on each application and their scores can be found here.  All of the institutions have ties to CIRM board members, who are not permitted to vote on applications involving their institutions. However, they can vote on creation of the research grant programs, establishment of their scope and rules.

DISC1-10074

Reprogramming human stem cells for blood cell generation

T. Reya – U.C. San Diego

$232,200

DISC1-10036

Prodrug innovation to target muscle stem cells and enhance muscle regeneration

H. Blau – Stanford University

$235,834

DISC1-10079

An exosome-based translational strategy to mitigate Alzheimer’s disease neuropathology

J. Baulch – U.C. Irvine

$179,911

DISC1-09912

A novel tissue engineering technique to repair degenerated retina

B. Thomas – University of Southern California

$215,133

DISC1-09999

Generation of expandable, self-renewing muscle stem cells for Duchenne Muscular Dystrophy

A. Sacco – Sanford-Burnham Medical Research Institute Letter to board

$265,500

DISC1-09984

Hypo-immunogenic cardiac patches for myocardial regeneration

S. Schrepfer – U.C. San Francisco Letter to board

$238,500

A Look at the California Stem Cell Agency: Its Origins, Its Accomplishments and Its Future

The Capitol Weekly online news and information service this afternoon posted a major overview of the $3 billion California stem cell agency.

The piece covered the agency's origins, recent high water marks and discussed its future. Here is the beginning of the freelance article by yours truly.

"C. Randal Mills, the 45-year-old CEO of California’s $3 billion stem cell research program, is a man who loves his milestones. 

"A private pilot, he charts his course in the air from one specific point to the next. Three years ago, Mills brought that same sort of navigation to the state stem cell agency. Miss one of the agency’s milestones, and — if you’re a stem cell scientist — you may not crash and burn, but you could lose millions of dollars in research funding from the state. 

"Mills has left an indelible stamp on the agency with his emphasis on concrete, measurable results. But he is resigning from the research program at the end of June in the midst of what some say is its “last stage.” His surprise departure to head the world’s largest bone marrow donor organization shocked many in California’s stem cell community. And it added to the unease about its future along with the future of possible stem cell therapies."  

(For those of you who read a brief item this morning about how this blog was going to go dark for a few days while it was on an ocean passage in the Sea of Cortez, we had a minor setback. Our floating home, the sailing vessel Hopalong, suffered a mechanical problem that we could not fix at sea, so we returned to port for repairs. The voyage begins anew tomorrow morning.)

Major Overview of California Stem Cell Upcoming This Afternoon

Look for a major overview of the $3 billion California stem cell agency later today on Capitol Weekly, a well-respected online news and information service that focuses on state government and politics.

The piece was written by yours truly on a freelance basis for Capitol Weekly and includes the latest developments at the agency, including what departing president Randy Mills leaves behind.

Given the vagaries of the Internet and news, publication of the article cannot be totally guaranteed this afternoon. So if it doesn't pop up today, try again later this week.

Meanwhile, the California Stem Cell Report is going dark for a number of days while it makes an ocean voyage in its maritime home, the sailing vessel Hopalong, in the Sea of Cortez.  Coverage of the agency is expected to resume perhaps by this weekend when an Internet connection can be found in Baja California.

California Stem Cell Agency Has Opportunity for 'All-in" Executive Assistant

Looking for a great job with a $3 billion operation headquartered in downtown Oakland? You will be able to share in the progress of one of the hottest biomedical fields in the country and perhaps help save some lives.

The job is executive assistant to the president of the California stem cell agency. The current president, Randy Mills, is leaving at the end of June. Maria Millan, now vice president for therapeutics, is taking over as the interim CEO. She is line to succeed Mills, but there is no guarantee on that.

The job is no walk-in-the-park. The agency is small -- only 46 employees -- despite its reach. Long hours could be the order of the day.

The job posting on the agency's web site says the position requires an "all-in" commitment to the goals of the agency. Salary can range up to $10,433 a month.

Bones, Stem Cells and Bridging the Gap

California's $3 billion stem cell research effort chalked up a small score yesterday with the announcement that a $5.2 million investment is making progress towards development of a therapy to regenerate broken bones. 

Writing on the the state stem cell agency's blog, Karen Ring, social media manager for the agency, said,

"Scientists from Cedars-Sinai Medical Center have developed a new stem cell-based technology in animals that mends broken bones that can’t regenerate on their own. Their research was published today in the journal Science Translational Medicine and was funded in part by a CIRM Early Translational Award."

The award went to Dan Gazit and Hyun Bae at Cedars. Their year one and two progress reports involving the adult stem cell therapy can be found at the link in the above paragraph. 

Ring's  blog item also carried a nifty graphic on the work and a link to a video on the research. 

Ring wrote,

 "Over two million bone grafts are conducted every year to treat bone fractures caused by accidents, trauma, cancer and disease. In cases where the fractures are small, bone can repair itself and heal the injury. In other cases, the fractures are too wide and grafts are required to replace the missing bone.

"It sounds simple, but the bone grafting procedure is far from it and can cause serious problems including graft failure and infection. People that opt to use their own bone (usually from their pelvis) to repair a bone injury can experience intense pain, prolonged recovery time and are at risk for nerve injury and bone instability."

Read All About It! Bad News for The Stem Cell 'Other'

It is a sad day for the stem cell "other."

Only 11 percent of the readers of one stem cell blog say they fit that category. That was a big drop from the early returns that showed the "other" with 21 percent.

(Never mind that we don't know the precise number of eyeballs actually represented by that 11 percent.)

All of this is the product of The Niche, the blog of UC Davis stem cell researcher Paul Knoepfler. He mounted a poll last week for readers to determine both their interests and general identity.

Initially "other" sprang into the lead. But as more readers responded, the reader category of academic scientists moved to the top with 32 percent. Industry scientists followed with 19 percent and patient or patient advocates with 17 percent.

What were they interested in? Investigations, 25 percent, newsy items, 20 percent, and journal club-like paper reviews, 18 percent.  Less than four percent of readers identified themselves as interested laypersons.

If you are feeling left out, there is still time to respond by going to this link. A chance at free stem cell swag is being offered. And the swag is better than a used petri dish.

$20 Million, California-backed Stem Cell Trial Discloses Disappointing News

Capricor Therapeutics, Inc., today announced some bad news about its state-of-California financed clinical trial for a cardiac, stem cell therapy and said that it planned to lay off an unspecified number of employees.

Capricor's stock price plummeted 62 percent following what the company called the "unexpected" news, dropping from $1.89 to $1.16. California's stem cell agency has backed the trial with $20 million, plus an additional $7 million for earlier, related research.

The agency, formally known as the California Institute for Regenerative Medicine (CIRM), said on its blog,

"Obviously this is disappointing news for everyone involved, but we know that not all clinical trials are going to be successful. CIRM supported this research because it clearly addressed an unmet medical need and because an earlier Phase 1 study had showed promise in helping prevent decline in heart function after a heart attack."

In response to a question, Kevin McCormack, senior director of communications, said the agency is talking to Capricor about the next steps. The agency regularly halts funding of awards when recipients do not meet milestones. McCormack did not respond to a question about how much money the firm had already received from CIRM.

Capricor, which is headquartered in Beverly Hills, said in a press release that an interim analysis on the phase two trial has "has demonstrated a low probability (futility) of achieving a statistically-significant difference in the 12-month primary efficacy endpoint." The company said there was "no notable difference" between treatment groups.

The company said it would cut its workforce to focus more sharply on its treatment of Duchenne muscular dystrophy, which the stem cell agency is also backing with $3.4 million. The Duchenne treatment had better news the last month, clearing its phase one trial with no adverse effects.

Timothy Henry and Rajenda Makkar of the Cedars-Sinai Heart Institute in Los Angeles are the principal investigators for the clinical trial.

The highest price for Capricor stock over the last 12 months was $5.40 and the lowest was $1.13.

Here are links to additional news stories today on Capricor: BiopharmaDive, MarketWatch, Genetic Engineering News, 

The "Other" and Stem Cell Blogs

Ever wonder who reads stem cell blogs? The "other" are the predominant readers of the one produced by a UC Davis stem cell researcher. At least that is the latest result from a survey that Paul Knoepfler is conducting about his cyberspace effort.

Knoepfler posted the survey request today. The results are still coming as you read this item. The initial tally this afternoon showed that 21 percent of respondents placed themselves in the "other" category when asked about their background. Next were academic and industry scientists, both with 18 percent, and physicians, 15 percent.

Most liked posts? Investigations, 33 percent; opinion pieces, 21 percent; newsy items, 17 percent.

The results are ever-changing as more readers respond. You can express your own preferences by going to this item. Respondents will be entered in a drawing for a stem cell T-shirt and signed copies of Knoepfler's two books.

Embedded in a Stem Cell Lab: Melton, Diabetes and Keeping Cells Happy

Yi Yu, a research assistant at the Melton lab at Harvard with flasks containing
human embryonic stem cells -- Photo Chloé Hecketsweiler/Undark

From a "mouse house" to growth cocktails, a web site called Undark has it all in a piece headlined "A Month in the Life of a Stem Cell Lab."

The photo essay was prepared by Chloé Hecketsweiler, a Paris-based journalist with Le Monde. She recorded events and people during six weeks this year in the laboratory of Douglas Melton at the Harvard Stem cell Institute.

Melton is digging into diabetes. A firm he co-founded, Semma Therapeutics, is the recipient of a $5 million award from the California stem cell agency, formally known as the California Institute for Regenerative Medicine.

About Melton's lab, Hecketsweiler wrote,

"I watched their experiments, learned about the complex science of stem cells, and talked with the researchers about their work and hopes. I was allowed to take pictures, and for this photo essay I tried to pick out moments and details that I found revealing, although scientists may see them as business as usual."

Her photos are first-rate, her reporting personal and the presentation strong. One member of Melton's team, Maria Keramari, told her,

"You have to keep the cells happy before you keep yourself happy."

It echoed an axiom from America's old family farming days when livestock was fed and cared for as the sun rose, long before before the family sat down for breakfast.

Another Melton researcher, Ornella Barrandon, said,

"We spend so much time on our projects, they are like our babies."

Hecketsweiler's work tells a science story in a way not regularly seen. It is a good example of making stem cell research accessible to a wide audience and leaving them wanting more.

A Scripps Scientist Deflates Cancer-Stem Cell Nexus

The sky is not falling, says a Scripps Research Institute scientist, despite headlines that seem to link "mutation" and "cancer" and "stem cells."

That comes from Jeanne Loring, head of the stem cell program at Scripps, who was writing on the blog of UC Davis stem cell researcher Paul Knoepfler. Loring said,

"'Mutation' and 'cancer' are eye-catching words for a headline; add 'stem cells' and there is a good chance that a lot of people will hear about it. These words have been liberally used in the press to describe the results of a recent publication: 'Human pluripotent stem cells recurrently acquire and expand dominant negative P53 mutations.'"

Loring said she has been on a soapbox on this issue since 2000.  She said, 

"Every time a scientific report suggests that human stem cells are dangerous, I feel the need to reassure both scientists and non-scientists that we should not panic.  The sky is NOT falling (contrary to Henny Penny), and pluripotent stem cells remain valuable for cell replacement therapies."

Loring went into the rather technical reasons for her position as well as identifying issues having to do with not knowing enough about the cells used in research. She also provided some tools for researchers to use to identify cells with "functionally important mutations."

Loring's bottom line to researchers:

"Don't panic! Check your cells instead."

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.)

Asterias and Capricor: Good News for California's Stem Cell Research Effort

Pat Furlong, CEO of Parent Project Muscular Dystrophy, spoke earlier 

this year about losing her two sons to Duchenne muscular dystrophy

California's $3 billion stem cell agency last week reported encouraging results from two of its clinical trials, news that came as the prestigious journal Nature was declaring that the state effort was in its "last stage."

The trials are being conducted by Capricor Therapeutics, Inc., of Beverly Hills, Ca., and Asterias Biotherapeutics, Inc., of Fremont, Ca. Both are publicly traded firms.

The news was reported by The Stem Cellar, the blog of the California Institute for Regenerative Medicine (CIRM), as the Oakland-based agency is formally known.

CIRM declared,

"Asterias Biotherapeutics continues to keep a steady pace and to hit the proper milestones in its race to develop a stem cell-based treatment for acute spinal cord injury."

The agency has pumped $20.7 million into the company's program, including $6.4 million when the research was being performed by Geron Corp. CIRM said that the latest data show that the treatment is safe and should continue with 10 million and 20 million cell doses with new trial participants. CIRM  blog author Todd Dubnicoff said,

"About a month ago, Asterias reported that six of the six participants who had received a 10 million cell dose – which is transplanted directly into the spinal cord at the site of injury – have shown improvement in arm, hand and finger function nine months after the treatment. These outcomes are better than what would be expected by spontaneous recovery often observed in patients without stem cell treatment. So, we’re hopeful for further good news later this year when Asterias expects to provide more safety and efficacy data on participants given the 10 million cell dose as well as the 20 million cell dose."

CIRM's Karen Ring reported on the good news from Capricor, which has received $23.2 million from CIRM, not all of it for Duchenne muscular dystrophy, however. The disease follows a devastating course and significantly reduces life expectancy. Ring wrote,

"The company reported positive results from their Phase I/II HOPE trial that’s testing the safety of their cardiosphere stem cell-based therapy called CAP-1002. The trial had 25 patients, 13 of which received the cells and 12 who received normal treatment. No serious adverse effects were observed suggesting that the treatment is 'generally safe' thus far."

Linda Marban, president of the company said that it plans to seek permission from the FDA to move into one of the agency's programs to expedite making the treatment available.

The announcements on the trial results came during a week when Nature, which reports only intermittently on CIRM, did something of an overview of the California research program. The Nature headlines said,

"California’s $3-billion bet on stem cells faces final test
"Major investment in regenerative medicine enters its last stage — and the money might run out before treatments are ready."

Capricor's stock closed at $3.12 Friday with a 52-week price range of $2.12 to $5.40. Asterias closed at $3.70 with a 52-week range of $2.30 to $5.80.

Below is an Asterias video on its research.

The Journal Nature Says: 'Last Stage' for California's Stem Cell Research Program

The prestigious journal Nature this week is calling it the "final test" for California's $3 billion plunge into development of stem cell therapies for afflictions ranging from cancer to arthritis.

The California Institute for Regenerative Medicine (CIRM), as the stem cell agency is formally known, expects to run out of cash for new awards in June 2020. The 12-year-old agency was created by a ballot initiative campaign that led voters to believe that nearly miraculous stem cell treatments were just around the corner. So far the agency has not backed a stem cell therapy that is widely available.

For Nature, the situation merited this headline yesterday on its web site,

"California’s $3-billion bet on stem cells faces final test
"Major investment in regenerative medicine enters its last stage — and the money might run out before treatments are ready."

Amy Maxmen wrote the article. She said,

"Now, the pot of money — one of the biggest state investments in science — is running dry before treatments have emerged, raising questions about whether Californians will pour billions more into stem-cell research."

Maxmen continued,

"If they don’t, that could leave hundreds of scientists without support, and strand potentially promising therapies before they reach the market. 'It’s an issue of great concern,' says Jonathan Thomas, chair of the board for the California Institute for Regenerative Medicine (CIRM) in Oakland." 

Maxmen also noted the critical assessment of CIRM in 2012 by the Institute of Medicine and some subsequent changes made by the agency's board. She said,

"Jim Lott, a member of the state board that oversees CIRM’s finances, says that he is not satisfied with the changes. He also argues that CIRM may not have been strategic enough in directing research. 'Some people say if they had a better focus, they might have achieved cures.'"

The Nature piece covered familiar ground for readers of the California Stem Cell Report. But she had further news from Bob Klein, a Palo Alto, Ca., real estate investment banker. Maxmen wrote,

"Bob Klein, the property developer who put Proposition 71 on the ballot and established CIRM, isn’t waiting for the money to run out. He leads an advocacy group, Americans for Cures, which will soon poll voters  to see whether they would approve another $5 billion in funding. If it looks like at least 70 percent of Californians support that plan, he’ll start a campaign to put another initiative on the ballot in 2018.

"Klein hopes that Californians will rise in support of science at a time when the Trump administration has proposed drastic cuts to the NIH budget. If public enthusiasm is not so strong, Klein says, he’ll aim for the 2020 elections, when voter turnout should be higher because it will coincide with the next presidential race."

Maxmen concluded with this comment from Eric Verdin, president of the Buck Institute on Aging in Novato, Ca., which has received nearly $35 million from CIRM.

“It would be a catastrophe for California if people say CIRM did not do what it was expected to do. They’ve built the foundation for the field and attracted people from around the world — you can’t just now pull the plug.”

California Stem Cell Agency Plumps Its Program as It Eyes Need for More Funding

California's 12-year-old stem cell agency today launched a "statewide outreach tour" that is aimed "partly" at building support for pumping $5 billion more into the program which is nearing the end of its financial life.

The agency has billed the one-hour, public program in San Diego as a "patient advocate event." Reporter Bradley Fikes of the San Diego Union-Tribune discussed the event in an article this morning that was headlined:

"Should Californians give more money for stem cell research?"

In addition to being sponsored by the California Institute for Regenerative Medicine (CIRM),  as the agency is formally known, the event is backed by UC San Diego, which has received $177 million from the agency.

Fikes said the event is the first in a series that is "is partly meant as a way to persuade voters to further support the institute with more funding."

He continued,

"Jonathan Thomas, CIRM’s chairman, said the San Diego event and others like it in other parts of the state are meant to update patients and all Californians about how their money has been spent, and to hear from the public."

Robert Klein, the multimillionaire real estate investment banker who led the 2004 ballot campaign that created the $3 billion agency, said last month that he expects that a public opinion poll this fall will show strong support for adding $5 billion to the effort. It is scheduled to run out of cash for new awards in June 2020 and perhaps sooner.

The 2004 campaign cost $34 million. Klein has not publicly discussed his plans to raise money for the ballot effort.

The agency has yet to finance a commercially available stem cell therapy.

UC Irvine Stem Cell Blog: Troubling New Federal Law on Stem Cell Research

The stem cell blog at UC Irvine this week published a piece that warned that a new federal law aimed at speeding commercialization of stem cell therapies is worrisome and could lead to harm to patients and damage the entire field of research.

The April 10 article was written by Navied Akhtar, a Ph.D. candidate in the biomedical engineering department.

Akhtar's topic was the 21st Century Cures Act. He wrote that the law, signed by President Obama and which had a broad range of supporters,

"...utilitizes evidence from clinical experience to 'help to support the approval of a new indication for a drug approved under [accelerated approval]' and 'to help to support or satisfy post approval study requirements.' The only requirements written in for accelerated approval past a 'reasonable likelihood' that there will be clinical benefit, is that one or both of two requirements are met: (1) That the manufacturer conducts studies after accelerated approval to verify the predicted effect on mortality or other clinical benefit and (2) That the manufacturer submits copies of marketing materials for the drug during the preapproval period." 

Akhtar said that the law 

"will allow for what is effectively anecdotal evidence to be used as actual evidence to support the requirement for accelerated approval. Furthermore, the verbiage surrounding the term 'surrogate endpoint' is loose at best. The idea of a surrogate endpoint is to produce a clinically relevant point in which to be able to measure the efficacy and safety of a drug. In the Cures Act, the definition of what constitutes a surrogate endpoint is left rather open-ended. It reads as such: "The term ‘surrogate endpoint’ means a marker, such as a laboratory measurement, radiographic image, physical sign, or other measure, that is not itself a direct measurement of clinical benefit, and is known to predict clinical benefit and could be used to support traditional approval of a drug or biological product; or is reasonably likely to predict clinical benefit and could be used to support the accelerated approval of a drug or biological product.' It is worrisome to write that this endpoint can be a marker that is not a direct measurement of clinical benefit, but is known to predict clinical benefit. This leaves open a large workaround for accelerated approval of drugs that may have no business being approved."

Akhtar goes on to cite cases of drugs that ultimately proved harmful even after clearing the usual approval process. 

In the case of novel stem cell therapies, he argued, serious mishaps or death because of loose federal standards could endanger progress in the entire field. 

New Top Lawyer Likely Coming Soon to California's $3 Billion Stem Cell Agency

California's $3 billion stem cell agency appears set to announce a new general counsel, a key position that pays nearly $400,000 a year.

The agency has closed applications and has only one candidate to replace James Harrison, who has announced he will be leaving the agency's service at the end of June.

A job opening notice was posted last month seeking applicants for the position, which has an annual salary range of $208,483 to $384,537.

Harrison worked as outside contractor with the Oakland-based agency in an agreement that totalled $575,000 this fiscal year. The agency is expected to run out of funds for new awards in June 2020.

Harrison has been the top lawyer for the agency since its inception in 2004.

California's Unregulated Stem Cell Clinics: Will State Regulators Step In to Cure Abuses?

The state of California has laws regarding substandard and questionable medical care, and now questions are being raised about whether it should step in to deal with the unregulated stem cell clinics that appear to flourishing in the Golden State.

The concerns come as the Los Angeles Times reported during the weekend about an enterprise in La Jolla, StemGenex Medical Group, which describes itself as "the premiere leader in the United States for regenerative medicine." 

The Times' Pulitzer Prize-winning columnist Michael Hiltzik wrote about the case of Jim Durgeloh, 59, a construction contractor from Washington state, who was looking for a solution to his hip problems, as well as other cases. Durgeloh paid StemGenex $15,000 for a treatment.

Hiltzik interviewed Durgelogh who was in Southern California with his wife following treatment. Hiltzik wrote,

"They were preparing to fly home, infused with the hope communicated by the clinic staff, who 'seemed very optimistic,' Durgeloh told me.

"A lawsuit in San Diego federal court suggests that StemGenex may have given the Durgelohs nothing but hope. Three StemGenex patients — two with diabetes and one with lupus — say they were misled by the medical group’s marketing pitch to pay $14,900 each in 2015 and 2016 for therapies that have had no effect.

"The lawsuit, which seeks class-action status, claims that StemGenex has made its money by 'targeting the ill and the elderly' with 'false, fabricated and purposefully misleading' claims about patient satisfaction."

Hiltzik continued,

"StemGenex, in its reply to the ... lawsuit, asserts that the plaintiffs 'cannot prove' that its 'representations regarding the efficacy of its stem cell treatments are actually false.' The plaintiffs, it continues, 'do not cite to a single scientific study that disproves [StemGenex’s] advertised claims.'"

UC Davis stem cell scientist Paul Knoepfler wrote yesterday on his blog about Hiltzik's article. Knoepfler was the co-author of a 2016 study that attracted nationwide attention with the disclosure that 570 unregulated stem cell clinics exist in the United States. California leads the way with 113.

Knoepfler focused on Hiltzik's disclosure that one of the StemGenex physicians, Scott Sessions, was placed on three years probation last February involving treatments not related to StemGenex. Sessions was accused of negligence "related to cosmetic surgery and other procedures he performed on two patients at an unrelated facility in 2011 and 2013."

Knoepfler continued,

"Sessions photo was up on the Stemgenex website...and then suddenly it wasn’t. Hiltzik also mentions that Stemgenex has had other questionable information on its web site in the past.

"With California having the most stem cell clinics selling non-FDA approved interventions of any state I hope the state medical board here will wake up to the fact that it needs to give this arena more attention."

The state has wide-ranging authority to regulate physicians. A document on the state Department of Consumer Affairs web site says that physicians can be disciplined for such things as  "not using accepted, effective treatments or diagnostic procedures," "not referring a patient to a specialist when
appropriate" and "continuing to use a procedure that is unnecessary."