No Appeals Filed Yet in California's $44 Million Alpha Stem Cell Clinic Round

None of the rejected California universities seeking multimillion dollar grants to join the Golden State’s new, ambitious network of Alpha Stem Cell Clinics has yet filed an appeal of the decisions, the state stem cell agency said today.

That word comes from Don Gibbons, a spokesman for the $3 billion enterprise. In response to a query from the California Stem Cell Report, he said, however, the deadline for appeals is Friday.

If appeals are filed as expected, they will handled behind closed doors by the agency’s staff. Under state law, the applicants can also appear publicly before the agency’s governing board on Oct. 23 in Los Angeles to make their case. The board, however, has not responded favorably to most such pitches in the last year or so.

The Oct. 23 meeting will be the first public vetting of the proposals, which are outlined briefly in review summaries. The summaries were prepared by the agency's staff following closed-door meetings in which out-of-state scientific reviewers voted the applications up or down. The board almost never rejects a positive decision by its reviewers. Occasionally, it will approve an application that is rejected by reviewers.

Three applications survived the private review and total about $33.6 million. A fourth is on the fence for $11 million more. The agency refuses most of the time to disclose the names of applicants. But based on the review summaries and other information, the City of Hope, UC San Diego and UCLA appear to be the top-ranked applications. 

California to Spend Up to $44 Million for Ambitious Alpha Stem Cell Clinic Plan

California late next week is expected to plunk down as much as $44 million to help make the Golden State the global leader in stem cell research as well as a go-to location worldwide for patient stem cell therapies.

The ambitious proposal comes from the state's $3 billion stem cell agency which aims to create high-powered Alpha Stem Cell Clinics at major universities around the state. The clinics would be one-stop centers for stem cell treatment and would be designed to attract patients from throughout the world.

Afflictions under attack include cancer and heart disease along with diabetes and spinal cord injury.

Just who will get the cash will be determined on Oct. 23 when directors of the agency will meet in Los Angeles to consider seven proposals. The directors will be working from summaries of the closed-door review and subsequent decisions on the applications by the agency’s blue-ribbon panel of out-of-state reviewers. 

The reviewers approved three proposals for a total of $33.6 million. A fourth, $11 million application received partial support but fell short of a flat recommendation for funding, which agency directors almost never reject. However, the board could decide to back the proposal despite reviewers' concerns. 

All of the applications come from the cream of California universities. The agency withholds the identities of grant applicants in nearly all cases until after the board acts. However, proposals from the City of Hope and UC San Diego appear to be ranked No. 1 and No. 2. UCLA is ranked No. 3, based on the review summaries and other information.

Other likely applicants include USC,  UC San Francisco and Children’s Hospital Oakland,  UC Davis, Stanford and UC Irvine. Some of the proposals brought together two or three institutions. 

All of the institutions have representatives on the 29-member governing board of the California Institute of Regenerative Medicine(CIRM), as the agency is formally known. They will not be allowed to vote on or discuss applications involving their institutions or businesses.

Viacyte, Inc., of San Diego, also appears to be a beneficiary because of its involvement in a trial with UC San Diego, one of the three top applicants (see the review summary for application 7764.) Viacyte has already received $55 million from the agency.

All of the clinical trials proposed or underway are early stage efforts. With early trials, the general odds of a specific therapy becoming available for widespread use are slim and could take a decade or more. 

The three apparent winners are all located in Southern California, leaving Northern California unrepresented, which poses a ticklish scientific-political problem. If the 4th-ranked application is located in Northern California, that fact could well push it into approval.

However, the review summary of the application said,

"The PD and institution have ties to one of the lead clinical trials, which could result in the appearance of a conflict of interest. Reviewers commented that policies should be in place to ensure that the relationships are clearly defined and separated."

The candidates rejected outright by reviewers may well appeal the decisions. The California Stem Cell Report has queried the agency concerning appeals, but the agency has moved much of the appeals process behind closed doors to be handled by its staff. Previously appeals often came directly to the board in public. However, applicants still have the right to appear before the board on any matter.  

Also undisclosed is the full amount of matching funds and other commitments offered by the competing applicants, which appear to be substantial. One applicant (application 7650) mentioned $10 million in its review summary.  Another applicant, which appears to be UC San Diego, touted a single, large private donor. Multibillionaire Denny Sanford has funneled $100 million into stem cell research through a linkage with the San Diego university.

The Alpha Clinic plan is the brainchild of former stem cell agency president, Alan Trounson, who is not expected to attend next week’s meeting. Trounson earlier this year resigned to return to Australia. Seven days after he left the agency, he accepted an appointment to the board of StemCells, Inc., which has received $19 million from CIRM. The agency was shocked by the move and suffered a spate of bad publicity as a result.

Trounson first broached the Alpha concept in 2011.  And in 2013, he told the Los Angeles Times, 

“It will make California a go-to place for stem cell therapies. I want to make sure it's part of our medical fabric."

An article in the journal Nature Medicine said the proposal would create the first-ever “clinical trials network focused around a broad therapeutic platform.”

To dig into the applications and scores, see this document. All of the review summaries are jumbled into the document, but you can scroll through or use a search tool to find specifics. 

Prieto: Cost of Stem Cell Therapy Could Be 'Pretty Good Bargain'

One of the directors of the $3 billion California stem cell agency this week commented on the likelihood of very high costs for therapies that the agency and others are pursuing.

Francisco Prieto, a Sacramento, Ca., physician who has been on the agency’s governing board since 2004, was responding in connection with this Oct. 8 item on the California Stem Cell Report: “Rosy Outlooks, Stem Cell Therapy,Stunning Costs.”

Prieto said in an email,

“Like any new transformative technology, I expect that stem cell treatments will start out quite expensive and (hopefully) decrease as they become more common and the cost of producing them drops. If some of them are cures (as opposed to treatments), then that cost needs to be weighed against the lifetime cost of treatment that would now be eliminated, as well as the gain in productivity and years of life. Even before you start to factor in the cost in human suffering, I predict they will start to look like a pretty good bargain.”

Rosy Outlooks, Stem Cell Therapy and Stunning Costs

It is nearly day three of the Stem Cell Meeting on the Mesa in San Diego, and the word that cannot be uttered has been heard: Expensive!

At least that’s what Bradley Fikes of the San Diego U-T has reported. Earlier this week, he was at the gathering of roughly 700 scientists, business people and investors.

Fikes wrote that “stem cell therapies appear poised to transform medicine.” But he also said that it is “clear that such innovations will be very expensive.”

Much has been written during the past year about the increasingly rosy outlook for stem cell research and possible therapies. Rarely is heard, however, a genuine discussion of the cost of such applications, including during meetings of the governing board of the $3 billion California stem cell agency. Nonetheless, affordability was part of Prop. 71, the measure that created the agency nearly 10 years ago.

Fikes did not offer any specific numbers for likely costs of the 131 stem cell therapies that are now being tested in California. He wrote,

“If the product is demonstrably superior to what’s currently available, cost won’t be an obstacle to reimbursement, said Nicholas Anderson, a medical technology analyst with Health Economics and Outcomes Research.”

Reimbursement, of course, is the industry euphemism for the pathway to a substantial profit.

Good reasons exist for avoiding public discussions of the cost of stem cell therapies, at least in the view of some in the field. One is that it is early in their development and not every financial aspect is fully understood. Another is that offering expensive estimates could trigger early controversy of the sort that has flared up nationally concerning more conventional treatments, such as those for cancer and hepatitis.

Nonetheless, considerable interest exists in the potential cost of stem cell therapy. One indicator is the amount of attention drawn by items on this Web site dealing with the likely expense of a stem cell treatment.

They consistently draw more attention than such matters as conflicts of interest at the stem cell agency and proposals for clinical trials.

A Google search this afternoon on the term “cost of stem cell therapies,” for example, produced 6 million results. No. 2 on the list was this item from 2013 on the California Stem Cell Report(CSCR):  “Cost of a Stem Cell Therapy? An Estimated $512,000”

The item has received 8,756 page views, according to Google statistics.

The article was based on a report in the Wall Street Journal concerning a Japanese stem cell project.

Another example involves an item on a 2009 study on stem cell therapy costs commissioned by the stem cell agency. It received no public discussion by the agency's directors. The item on this blog about the study was seen 2,999 times. But the copy of the study itself has been viewed 15,989 times, nearly three times the attention of any of the other 164 documents posted by this Web site on the Scribd.com library site.

The California stem cell agency is on track to be involved in 10 clinical trials by the end of this year. As they progress, the potential costs of the partially publicly financed therapies could well become a matter for public debate. Particularly if the agency plans to ask California voters for more billions for stem cell research.   

Twenty-six Nominated for 2014 Stem Cell Person of the Year Award

A distinguished list of nominees was revealed today for the Stem Cell Person of the Year award, whose cash prize has doubled from last year.  

The contest is the brainchild of UC Davis stem cell scientist Paul Knoepfler, who boosted the award from $1,000 to $2,000 this year, all of which comes out his pocket.

Knoepfler this morning posted the names of the 26 nominees, who ranged from Pope Francis to California patient advocate Don Reed. The names were submitted by readers of Knoepfler’s blog, among others.

Nominees included two executives at the $3 billion California stem cell agency, Ellen Feigal and Pat Olson, which expects to have money down on 10 stem cell clinical trials by the end of this year. (Here is a link to the agency's blog item on the contest.)

The list is larded with other worthy nominees including Michael West, CEO of Biotime, Inc., which resurrected the human embryonic stem cell trial abandoned for financial reasons by Geron.

Voting is now underway to whittle the list down to 12 finalists. There is also an “other” category for additional nominations.  The criteria are broad. Knoepfler says the award should go to the “the most positively influential person” in the stem cell field during 2014.

Deadline for voting is Oct. 22. Knoepfler, however, has the only vote that counts for the final selection of the overall winner. During the past two years, he has selected Elena Cattaneo of Italy

and patient advocate Roman Reed of California. (Roman is the son of Don Reed.)

For rules on voting, see here.

The California Stem Cell Report will carry its recommendation for the award later this week.

UC Irvine Mounts a New Stem Cell Research Blog

Randal Berg
UCI photo

California has just scored with another stem cell research blog – this one from UC Irvine and produced by Randal Berg.

Stem cell research blogs are scarce. The only other one we know of in California is written by Paul Knoepfler at UC Davis. Of course, there is the blog of the $3 billion California stem cell agency, which is more of an institutional product. 

Irvine’s Berg wrote his inaugural piece Tuesday, introducing himself and the new blogging effort at Orange County campus, which has received $97.5 million from the stem cell agency.

Berg is chief administrative officer of the UC Irvine Stem Cell Research Center.  As such, it is a fair guess that his blog will not reflect an idiosyncratic point of view. 

Berg’s item dealt with some upcoming events and developments at Irvine. One is an appearance by Randy Mills, the new president of the $3 billion California stem cell agency, on Oct. 8, which is the agency’s Stem Cell Awareness Day.

Berg also said his organization is “eagerly anticipating” the results of the agency’s closed door review of applications in its $65 million Alpha clinic program. Directors of the agency are expected to publicly approve up to five applicants later this year.

(Editor's note: An earlier version of this item did not contain a mention of the stem cell agency's blog.)

California's 'Bridges' Stem Cell Effort Hailed Again

CIRM video

California’s $18 million “Bridges” stem cell training program this week received another endorsement as a “spectacularly successful” effort that is key to supporting stem cell research in California.

UC Davis stem cell scientist Paul Knoepfler said on his blog that it would be “counterproductive” to end the program, which is facing competition for funds as the state’s $3 billion stem cell agency focuses more on pushing research into the clinic instead of training efforts.

The Bridges program involves students from 11 of California’s two-year community colleges and four-year state colleges not associated with the University of California. The idea was to offer an opportunity for persons in those schools to be involved in stem cell research, learn skills and find jobs in the business.

Knoepfler said,

“As a faculty member at UC Davis, I have seen first hand just how powerful the Bridges program has been and continues to be. I have trained and continue to train Bridges students. I have been incredibly impressed with their intellect, energy, and the sheer overall amount they have to contribute to stem cell research in California. The sky is the limit with these young scientists.”

Jeanne Loring, head of the stem cell program at Scripps, also endorsed the program in a comment at the end of this item on the California Stem Cell Report and on Knoepfler’s blog. She said,

“I think it's a tragic loss to mothball the equipment and shut down the training labs just when work in those labs is leading to the cures that are CIRM's mission. Some of our best-trained stem cell researchers are losing their jobs, just when they are most needed.”

One person, Li You Hu, who filed a comment on Knoepfler’s item posted this question,

“Perhaps this can be justified as a continuing effort to train more people for stem cell research, but where is the quantitative justification based on those who have already been trained?”

Loring replied,

“Every one of the more than 20 Bridges interns that have worked in my lab are either in graduate school or have jobs as technicians.”

Stem Cell Training Ending at California State Colleges?

CIRM video on the Bridges program

California’s $3 billion stem cell research effort is turning ever more strongly in the direction of pushing stem cell treatments into the clinic, raising concerns among some about at least one program that appears endangered.

It is the $17.5 million “Bridges” training project that involved 11 state and community colleges, schools where advanced research does not usually take place.

Once lauded by top officials of the California Institute for Regenerative Medicine (CIRM), as the stem cell agency is formally known, the Bridges program was aimed at expanding the number of trained personnel and providing a way for trainees to connect with stem cell labs in academia and industry.

In 2009, when the program was funded, Robert Klein, then chairman of the stem cell agency, said,

 “Training young people is critical to our mission of developing new therapies. As California’s stem cell industry continues to grow the state will face a critical shortage for biomedical laboratory workers trained in state-of-the-art techniques required by stem cell research labs. People who graduate from our Bridges programs will be ready to fill these positions and help California industry and academic labs maintain momentum in their search for cures.”

Earlier this month, Susan Baxter, executive director of the California State University’s system-wide program for biotechnology, warned that unless the board extends the program, the agency “will lose significant momentum in its efforts to build and inspire a professional stem-cell-related workforce in California.”

She said,

“The number one workforce need in this industry is hands-on practice and participation in multi-disciplinary, team-based research projects.  Research experience is baked into the Bridges program; as a result, graduates have many career options.  Despite the Great Recession, Bridges graduates have succeeded in landing jobs and gaining admittance to graduate and medical schools at much higher rates than peer groups.”

Baxter asked that the board review and consider extending the Bridges program at its meeting late next month.

Her remarks came as the agency is aiming its cash at clinical trials and activities leading to clinical trials, all of which requires large sums. Some of the agency’s earlier programs will necessarily be discontinued as the money shrinks.

The agency previously said funds for new awards will run out in 2017. However, after examining the agency’s financing assumptions, Randy Mills, CIRM’s new president, says the cash will last until about 2020.

For the schools involved in the Bridges program see the list at the end of the press release here. For those not familiar with California's higher education structure, the colleges listed are not part of the University of California system and generally do not award Ph.D. degrees.

Here is the text of Baxter’s remarks to the CIRM board on Sept. 10.

   California Stem Cell Bridges Program

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.”

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.

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.

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. 

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. 

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.

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.

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.

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.  

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.

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.”

California's Stem Cell Trial for Spinal Cord Injury Moves Forward

A California firm, backed by $14.3 million in state cash, today announced that it has received a federal go-ahead to advance its clinical trial involving a human embryonic (hESC) therapy for cervical spinal cord injury.

Asterias Biotherapeutics, Inc., of Menlo Park, said in a press release that it is currently selecting clinical trial sites and expects to enroll patients beginning early next year. The trial is the continuation of an effort that Geron abandoned in 2011 for financial reasons.

Pedro Lichtinger, president and CEO of Asterias, said,

  “We are especially enthusiastic about working with our new partner, (the California Institute for Regenerative Medicine or CIRM), in executing this clinical trial. The FDA clearance provides Asterias with imminent access to the previously announced $14.3 million CIRM grant, which provides non-dilutive funding to support both the clinical trial and other product development activities for AST-OPC1.”

The phase 1/2a trial will involve doses of up to 10 times higher than what was used earlier. Up to 13 patients will be treated within 14 to 30 days after their injury occurred. The hope is that it will be easier to detect the efficacy of the treatment with such dosages.

As for the early stage trial involving five patients, the company said,

“These five patients were administered a low dose of two million AST-OPC1 cells and have been followed to date for 2 to 3 years. No serious adverse events were observed associated with the delivery of the cells, the cells themselves, or the short-course immunosuppression regimen used.  There was no evidence of expanding masses, expanding cysts, infections, cerebrospinal fluid leaks, increased inflammation, neural tissue deterioration or immune responses targeting AST-OPC1 in these patients.  In four of the five subjects, serial MRI scans performed throughout the 2 to 3 year follow-up period indicate that reduced spinal cord cavitation may have occurred and that AST-OPC1 may have had some positive effects in reducing spinal cord tissue deterioration.”

Asterias was awarded the cash by the California stem cell agency in May. (See here and here.)

Randy Mills, president of the stem cell agency, said today,

“This is exactly the type of treatment, focusing on an unmet medical need, that CIRM was created to address.”

Katie Sharify, one of the patients involved in the trial when it was started by Geron, said in the CIRM press release,

“A lot remains unknown about human embryonic stem cells and that's exactly why this research is so important. The scientific community is going to have a much greater understanding of these stem cells from the data that will be collected throughout the study and I'm glad to have been a part of this advancement."