Friday, September 12, 2014

ViaCyte: Stem Cell Diabetes Therapy 'Not Possible' Without California Taxpayer Cash

The California stem cell agency hit a couple of firsts this week when it pumped up its investment this week in the “teabag” diabetes therapy being developed by ViaCyte, Inc., of San Diego.

The effort, backed by $55 million from the agency, is the first clinical trial in the United States for a diabetes treatment involving human embryonic cells (hESC).

It is also likely the first time that the state of California has directly invested so much money in a single business.

However, the cash and the research work did not happen overnight. The research began many years ago when ViaCyte was known as Novocell. The stem cell agency entered the game six years ago. Wednesday’s $16.6 million cash infusion came on top of $38.5 million already handed out by the state, which is likely to be a financial player for at least several more years, assuming the ViaCyte clinical trials go well.  

Paul Laikind
ViaCyte photo
Paul Laikind, CEO of the firm, was there as agency directors Wednesday approved ViaCyte’s latest application (AP1-08039).  Following the 8-0-1 vote, he said, 
“I can say without doubt that the progress we have made developing our therapeutic candidate would not have been possible but for the tremendous support we have received from CIRM(the stem cell agency).  Importantly, CIRM’s support has been multiplied, as it has helped us to secure other funding sources that we need to drive this project forward.” 
About 40 patients are being recruited for the clinical trial. UC San Diego this week announced that interested persons can contact them at 858-657-7039. Laikind said other sites are expected to open around the country.

Here is the full text of Laikind’s remarks.   
“Good afternoon, I am Dr. Paul Laikind, president and CEO of ViaCyte.
 “I would like to take this opportunity to thank the members of the ICOC, the Grants Review Working Group, the CIRM staff and especially the citizens of California whom you all represent, for the continued support of the work we are doing at ViaCyte to develop what we all hope will be an important, innovative treatment for diabetes.
 “CIRM has been a partner with us in this endeavor since the early days and your confidence and support has allowed us to make tremendous progress.  Last month we were cleared by the Food and Drug Administration to begin clinical trials evaluating our stem cell-derived islet replacement therapy candidate in patients with type 1 diabetes.  Following up on that exciting news, it was proudly announced just yesterday that the first center to enroll patients in this trial will be the University of California, San Diego School of Medicine.
 “To our knowledge this will be the first time an embryonic stem cell derived cell replacement therapy for diabetes will be tested in the clinic.  This exciting development illustrates the importance of CIRM’s mission for medicine and for California.  CIRM is all about breaking new ground, nurturing promising medical advances and stimulating our great state’s economy.
 “I can say without doubt that the progress we have made developing our therapeutic candidate would not have been possible but for the tremendous support we have received from CIRM.  Importantly, CIRM’s support has been multiplied, as it has helped us to secure other funding sources that we need to drive this project forward.
 “Some will point out that that we are still at an early stage with this project and there is no denying that, there is much left to do and discover.  However, together we have made tremendous progress and increased the odds of success with each milestone achieved.  Whatever the outcome, CIRM has pushed the boundaries of medicine and is step by step bringing us closer to realizing the tremendous promise of regenerative medicine.
 “Thank you again for the very important work you are doing.”
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Wednesday, September 10, 2014

California Stem Cell Meeting Concludes; Links to Stories

BERKELEY, Ca. -- Directors of the California stem cell agency today concluded their meeting at 1:53 p.m. PDT. Look for more coverage of matters discussed at the meeting in the next week or so.

Here are links and excerpts from stories from today's session.

California Bets $55 Million on 'Teabag' Diabetes Treatment

BERKELEY, Ca. -- California today beefed up its investment in a “teabag” therapy for diabetes, bringing the total to $55 million in an effort to develop a “virtual cure” for an affliction that affects 347 million people worldwide.
It is believed to be the largest direct investment that the state has ever made in a company. The therapy also involves the most controversial of stem cell treatments, ones derived from human embryonic stem cells(hESC). 

Revolving Door Policy Tightened at California Stem Cell Agency

BERKELEY, Ca. -- Directors of the California stem cell agency today approved a measure aimed at easing conflicts of interest involving employees who may seek employment with recipients of the agency’s largess.

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Revolving Door Policy Tightened at California Stem Cell Agency

BERKELEY, Ca. -- Directors of the California stem cell agency today approved a measure aimed at easing conflicts of interest involving employees who may seek employment with recipients of the agency’s largess.

On a unanimous voice voice, the governing board  of the California Institute for Regenerative Medicine (CIRM) set a new revolving door policy that says,
“To prevent even the appearance of a conflict of interest, CIRM employees should contact CIRM’s general counsel or deputy general counsel if the employee has begun discussions with a prospective employer that has received or is currently applying for CIRM funding. CIRM’s attorneys will maintain the confidence of this information and advise the employee of his or her obligations under state law, and the employee will be precluded from participating in any decisions relating to the prospective employer.­­"
The new policy is a slight extension of the state law and was offered in the wake of the appointment of former CIRM President Alan Trounson to the board of directors of StemCells, Inc., of Newak, Ca., which holds $18.4 million in awards from the agency. 

Trounson was named to the board seven days after he left the agency and did not inform the agency he was considering a position with the firm. The news triggered a wave of unfavorable publicity for the agency.
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CIRM Press Release on Viacyte Award on Diabetes

BERKELEY, Ca. -- The California stem cell has posted a press release on today's award to Viacyte, Inc., of San Diego, Ca., to speed up development of a human embryonic stem cell treatment for diabetes. Here is a link to the press release.  Sphere: Related Content

California Bets $55 Million on 'Teabag' Diabetes Treatment

BERKELEY, Ca. -- California today beefed up its investment in a “teabag” therapy for diabetes, bringing the total to $55 million in an effort to develop a “virtual cure” for an affliction that affects 347 million people worldwide.

It is believed to be the largest direct investment that the state has ever made in a company. The therapy also involves the most controversial of stem cell treatments, ones derived from human embryonic stem cells(hESC). 

The impact of the potential therapy could be far-reaching.  About 70,000 persons die each year in this country from diabetes. It is the 7th leading cause of death in the United States.

Forty-three-year-old Maria Torres, who lives in the Sacramento area, is hoping for a positive outcome on the therapy.
Maria Torres
CIRM photo
 “I have three kids, and I know they could have the same thing I have. If they find a cure, for me, that’s peace of mind.”
Torres was featured in a blog post yesterday by the California Institute of Regenerative Medicine(CIRM), the state’s nearly 10-year-old, $3 billion stem cell research program, which is providing the taxpayer funding.

Meeting here today, directors of the agency approved, on an 8-0-1 vote, $16.6 million in awards to Viacycte, Inc., of San Diego, Ca., to advance its work on the therapy.  Over the last six years, the agency has pumped $38.5 million into the company, which has received by far the greatest amount of cash from the agency of any business. A subsidiary of Johnson&Johnson as well recently invested $20 million in Viacyte.

The firm’s treatment is scheduled to begin clinical trials this year. UC San Diego has begun enrolling patients, Viacyte CEO Paul Laikind told CIRM directors this morning.

The therapy involves several, porous, teabag-like packages that are inserted beneath the skin. An individual device is about the length of a credit card but half the width. The firm plans to work on a larger device for single insertion. 

In the CIRM blog post yesterday, Anne Holden, Web content and social media manager for the agency, said the device contains cells that “sense blood sugar levels and produce insulin to reduce them.”  That “allows transfer of blood sugar, insulin, oxygen, and other molecules but keeps (other) cells out, thus avoiding the possible attack and rejection by the patient’s own immune system.”

Regular use of the treatment is years away because of the series of clinical trials that must be run. Additionally, only about one out 10 traditional drugs entering clinical trials reach the marketplace. No therapies involving human embryonic stem cells (hESC) have successfully run the clinical trial gauntlet in the U.S. and secured approval for widespread use.  

Viacyte’s therapy is derived from those controversial cells and is the first hESC trial backed by CIRM.   Some religious groups and others believe the use of the cells is tantamount to murder. Opposition to hESC research has subsided in recent years because of the focus on the possible use of reprogrammed adult stem cells. However, it flared up again in recent weeks because of a flap over the so-called Ice Bucket Challenge, which raised funds for ALS research, some of which involves hESC.

The California stem cell agency, ironically, owes its existence to the opposition to hESC research. Former President George Bush restricted federal funding for hESC research because of the religious concerns. The ballot campaign in 2004 to create the agency relied heavily on Bush’s action to demonstrate the need for continuing research into the promising field. 

Here is a link to the CIRM press release on the awards. 
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California Stem Meeting Begins in Berkeley

BERKELEY, Ca. -- Directors of the $3 billion California stem cell agency opened their meeting this morning at 10:04 a.m. PDT with a "teabag" cure for  diabetes on their minds.

Also on tap is consideration of a proposal to ease problems with conflict-of-interest, revolving-door problems at the agency. Sphere: Related Content

Tuesday, September 09, 2014

Coverage of California Stem Cell Meeting, Plus an Ohio Participation Possibility

The California Stem Cell Report will bring you on-the-scene, live, gavel-to-gavel coverage tomorrow of the meeting of the directors of the Golden State’s $3 billion stem cell research program.

Directors are expected to approve $16.6 million for a “teabag” treatment for diabetes that is being developed by Viacycte, Inc., of San Diego.  Also on the agenda is a move to ease revolving-door problems at the agency that may recur as some of its employees seek jobs elsewhere.

The meeting begins at 10 a.m. PDT and will be audiocast on the Internet for those who want to listen. To see the PowerPoint slides being used during the meeting, you can also log on to a WebEx presentation. The agenda has instructions on how to take advantage of these opportunities.

Stem cell fanciers in Ohio will be able to actually take part from a location at the Downtown Hilton at 401 N. High St. in Columbus. The room number was not specified on the meeting agenda, so interested parties should query the agency in advance at info@cirm.ca.gov.  

Remote participation locations in California outside of Berkeley include two in Los Angeles one each in La Jolla, Duarte  and Mountain View.  Remote locations are provided when some directors are unable to attend meetings.
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Monday, September 08, 2014

San Diego's Viacyte Scoring Again with State Funding; Total Now Hits $55 Million

California’s stem cell research agency appears ready to pump another $16.6 million into a diabetes therapy effort that has already received $38.5 million from the Golden State.

At a total of more than $55 million, it is likely the single largest direct investment that the state of California has ever made in a business. The cash is expected to be approved by directors of the $3 billion stem cell agency at their meeting Wednesday in Berkeley.

The agency has a policy of secrecy concerning the names of those seeking public money and did not identify the firm.  However, it was apparent from documents on the Web site of the California Institute for Regenerative Medicine (CIRM), as the agency is known, that the recipient is Viacyte, Inc., of San Diego, Ca. The firm is the most heavily backed business by the stem cell agency. Second is Capricor, Inc., of Beverly Hills, Ca., at $19.8 million.

Viacyte is working on a diabetes treatment that it has described as a “virtual cure” for type 1 diabetes. The company describes the device as akin to a “flat tea bag” that would be implanted beneath the skin.

Last month, the firm announced it had received $20 million (see here and here) from Janssen Pharmaceuticals, a subsidiary of Johnson and Johnson. Viacyte also last month announced it has received a federal go-ahead to begin a clinical trial for its human embryonic stem cell treatment.

Only about one out 10 traditional drugs emerge into the marketplace from clinical trials. No therapies involving human embryonic stem cells (hESC) have successfully run the clinical trial gauntlet in the U.S. and secured approval for widespread use.

This week’s funding for Viacyte involves two awards in application AP1-08039 in the agency’s new, $200 million “accelerated development pathway,” which is designed to speed research into the clinic and marketplace. The agency’s blue-ribbon reviewers examined the application behind closed doors last spring but said in a summary posted publicly last week that the therapy had the potential to “transform” diabetes treatments.

More than two million persons suffer from diabetes in the California and roughly 370 million worldwide.

Only four other applicants were considered in the “pathway” round. Viacyte was the only business. The other applications came from Henry Klassen at UC Irvine, Thomas Kipps of UC San Diego, Donald Kohn of UCLA and Clive Svendsen of Cedars of Sinai. All the institutions have representatives on the CIRM board of directors, but were rejected by reviewers.  As mentioned above, none was identified by the agency but all could be recognized by other means.

All of the agency’s comments and summary of reviewer comments can be found on this document. Klassen’s application is DR2A-05379. Kipps’ application is AP1-08043.  Svendsen’s application is AP1-08047. Kohn’s application is AP1-08048.
  
Rejected applicants will have an opportunity to apply again in later offerings in the $200 million acceleration pathway program, which is aimed at producing major scientific results by 2017.

Viacyte had applied for $25 million, the upper limit in this week’s awards. However, the reviewers rejected two “modules” in the application worth $8.4 million, suggesting that they were not timely and could be reworked incorporating reviewer suggestions.
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Thursday, September 04, 2014

Tightening the Revolving Door at the California Stem Cell Agency

Directors of the California stem cell agency next week will consider a proposal aimed at partially addressing revolving-door and conflict-of-interest problems at the $3 billion research enterprise.

The proposal comes in the wake of a wave of unfavorable publicity this summer that embarrassed the agency when its former president, Alan Trounson, was named to the board of a stem cell company only seven days after he left the state research effort.

The Newark, Ca., firm, StemCells, Inc.,  is the recipient of $18.4 million in awards from the California Institute of Regenerative Medicine (CIRM), as the agency is formally known.  The news surprised the agency and prompted its new president, Randy Mills, to sign an agreement that he would not accept employment from a CIRM grant or loan recipient for at least a year after he left the agency.

Michael Friedman
CIRM photo
In a memo to CIRM board members yesterday, Mills said he was offering a new proposal on revolving door matters after CIRM Director Michael Friedman, president of the City of Hope, made such a suggestion in July.

Mills wrote,
“Under the proposed policy, CIRM team members (employees) would remain free to pursue other employment opportunities, including with CIRM-funded institutions. To prevent inadvertent violations of California’s conflict of interest laws and to ensure the integrity of CIRM’s decision-making process, however, the policy would request that CIRM employees notify CIRM legal counsel when the employee begins employment discussions with a CIRM grantee or current applicant. CIRM’s legal counsel will maintain the confidentiality of this information and advise the employee of the steps he or she needs to take to remain in compliance with the law. Thus, the policy balances the privacy interests of CIRM employees with the need to protect the integrity of  CIRM’s decisions.”
Mills said,
“As Dr. Friedman recognized, CIRM has a highly talented team. It is therefore understandable that California institutions, including those that receive CIRM funds, would be interested in recruiting them. Currently, there is no prohibition on CIRM team members accepting employment from CIRM funded institutions, however I believe additional clarity regarding this topic would help avoid potential conflict of interest occurrences.”
The board will act on the proposal at its meeting in Berkeley next Wednesday.

 Our comment: The proposal will not eliminate revolving door problems at the nearly 10-year-old agency as it winds down its funding and employees may want to seek other employment.  The proposal contains ambiguities that make it difficult to adhere to, such as the question of when employment discussions begin. It also does not contain any indication of the consequences for violation of the policy. However, coupled with the Mills’ personal declaration on future employment and the deplorable situation involving Trounson, the new policy helps make it clear that actions such as Trounson’s do not measure up to what is now expected at CIRM.  Mills has also produced a fresh perspective on CIRM’s future finances that would stretch them out to 2020 instead of 2017, another action that removes an incentive for employees to consider seeking employment elsewhere.  
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Tuesday, September 02, 2014

California's New $200 Million Stem Cell Spark

Directors of the $3 billion California stem cell agency this month will attempt to supercharge some of their major research programs and push stem cell therapies more rapidly into the marketplace and the clinic.

Sir John Bell
Academy of Medical Sciences photo
The move is part of a $200 million effort approved only last December and was recommended by the agency’s blue-ribbon scientific advisory board, chaired by Sir John Bell of the University of Oxford in the United Kingdom. 

Awards in the first phase of the program – dubbed an “Accelerated Development Pathway” -- will range up to $25 million each. The effort will also involve more assistance and streamlined procedures from the California Institute of Regenerative Medicine (CIRM), as the agency is formally known.

The awards are scheduled to be approved publicly on Sept. 10 at the directors’ meeting at the Claremont Hotel in Berkeley, Ca. Winners will be culled from a select group of researchers already funded by the agency.

The number of applicants was not immediately available from CIRM, but they were limited to individuals and institutions that have already received the agency’s signature disease team or strategic partnership awards. Also unknown was whether any businesses were among the applicants. Applicants rejected in next week’s round will have a chance to apply in upcoming, new “pathway” rounds.

According to the request for applications (RFA) on the CIRM Web site, the agency is seeking “high potential” research that can achieve “clinical demonstration of an acceptable safety profile and proof of concept during or before 2017.”

The RFA continued,
“For example, a team could propose additions to an ongoing CIRM-funded clinical trial that would accelerate development decisions such as including the testing of a biomarker they identified in correlated research work or adding a patient group based on data from an unblinded safety study. Other examples of accelerating activities could include changes in manufacturing processes or delivery devices, based on novel ideas that emerged from the activities of the initial award or related research that could require comparability studies to facilitate development of the candidate therapeutic. In addition, the Accelerated Development Pathway would also consider the possibility of funding a future clinical trial to demonstrate clinical proof of concept in the approved therapeutic indication, ‘subject to satisfaction of milestones and conditions.’”
The “pathway” program was approved by CIRM directors after its new Scientific Advisory Board recommended its creation. The advisors said the agency should move “at speed” to turn research into treatments.

Creation of the panel of advisors was recommended by the Institute of Medicine (IOM) in a $700,000 study funded by CIRM. The IOM report said the panel would be invaluable in helping the agency to “make fundamental decisions about dealing with challenges that cut across particular diseases, decide which discoveries should progress toward the clinic and determine how best to engage industry partners in developing new therapies.”

(See here for the names of the members of the advisory panel. The full text of their report can be found at the end of this link.)

The panel has held only one meeting, and that was behind closed doors.
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Thursday, August 28, 2014

Asterias Stock Price Jumps Nearly 13 Percent Today Following hESC Therapy News

The California stem cell agency and Asterias Biotherapeutics today picked up a modicum of news coverage in connection with an advance on a spinal injury therapy that was once hailed as historic.

The news about the Menlo Park, Ca., firm’s clinical trial received major attention in the San Francisco Chronicle and more modest coverage in the San Francisco Business Times and on the ipscell.com blog.  

The news also helped to push the Asterias stock price up nearly 13 percent since yesterday to close at $3.08 today. The stock closed at $2.43 Tuesday, the day before the company released the clinical trial news, according to Google Finance.

Stephanie Lee’s piece in the Chronicle contained some history about the potential therapy, dating back to when it was developed by Geron and then abandoned. Geron was the first firm to win approval of a clinical trial for a human embryonic stem cell (hESC) treatment.

Lee also had a couple of interesting tidbits, including the fact that the stem cell agency’s $14.3 million award to support the trial will cover half its costs. Lee also reported,
“Geron treated severe injuries in the thoracic region of the spinal cord, which runs along the back. Asterias is targeting injuries that originate in the neck, citing an outside study that suggests injuries in this area are easier to treat.”
Enal Razvi
Select Biosciences photo
Lee additionally quoted Enal Razvi, managing director of Select Biosciences U.S., an international life sciences consulting firm with its U.S. headquarters in Fremont, Ca.,  as saying,
"This is just the start of a trial, not the approval of a drug, which are two very, very different things in this space…(but) this helps things go to the next level." 
The Chronicle story was the No. 1 story late afternoon today in Google news search results using the term "stem cell," ranking ahead of the STAP news out of Japan.

Paul Knoepfler, a UC Davis scientists who writes the ipscell blog, carried a Q&A with Jane Lebkowski, president of research and development at Asterias, who discussed another hESC product. She said,
“A second Asterias product is AST-VAC2, which are human embryonic stem cell derived dendritic cells. These cells are modified to express telomerase, a protein typically expressed in cancer cells. The aim is to use these telomerase expressing dendritic cells to stimulate immune responses against cancer cells. We are now preparing for clinical trials with this product.” 
That effort could well find its way to additional funding from the California stem cell agency if it meets the four-point criteria of the new president, Randy Mills, of the $3 billion research program.

In the San Francisco Business Times piece, Ron Leuty noted that the initial five-patient safety trial showed that “spinal cord injuries in four the patients had shrunk.” Leuty wrote,
“Whether that means Geron’s treatment is working in those patients is an open question. Geron’s study looked only at acute, or new, spinal cord injuries, so some of the results could be connected to normal healing over time.”
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