Thursday, June 29, 2017

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.
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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. Sphere: Related Content

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.



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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.
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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.
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Wednesday, June 28, 2017

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. Sphere: Related Content

Tuesday, June 27, 2017

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 herehere 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. Sphere: Related Content

Thursday, June 15, 2017

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.

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Tuesday, June 13, 2017

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. Sphere: Related Content