Tuesday, October 21, 2014

California's $40 Million Alpha Clinic Round: Appeals Filed by Two Rejected Applicants

Two of the three rejected applicants in California’s $40 million-plus Alpha Stem Cell Clinic competition are seeking to overturn the decisions, but none of the cases is expected to be made public.

The appeals are being considered behind closed doors by the staff of the $3 billion state stem cell agency, which will make decisions on whether to proceed further. Directors of the agency are not expected to see the appeals at their public meeting on Thursday in Los Angeles.

Up until last year, appeals were considered in public by the board. The process was altered in the wake of often emotional outpourings involving patient advocates and the public. However, scientists and others can appear before the board on any matter, including applications. Researchers can also choose to disclose publicly their appeals.

The governing board is scheduled to ratify three awards in the Alpha round, which is designed to make the stem cell agency a one-stop, global center for stem cell treatments. The Alpha effort also will help to fund additional clinical trials aimed at afflictions ranging from cancer to heart disease.

The expected winners are the City of Hope, UC San Diego and UCLA, based on an analysis by the California Stem Cell Report. A fourth applicant is on the fence. The agency declines to reveal the names of applicants for fear of embarrassing rejected institutions and researchers. In response to a query, Don Gibbons, a spokesman for the agency, Monday said two applicants had filed appeals.

The agency’s blue-ribbon, out-of-state scientific reviewers make the de facto decisions on funding of applications during closed-door meetings. The agency’s governing  board almost never overturns the reviewers’ positive decision for funding. Occasionally, the board will approve funding for a rejected application.

Summaries of the reviews can be found here.
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Sunday, October 19, 2014

California Stem Cells, College Football and Success

The $3 billion California stem cell agency this week will deal with the sort of odds that often figure in the financial fate of college football players.

The issue at hand involves education, helping young people and setting priorities. Should the Golden State’s stem cell effort train a few hundred college students at $65,000 a pop or spend $9 million to give a piece of advanced research the critical lift to turn it into a cure?

At least that is one way to look at the issues coming up on Thursday at the Los Angeles meeting of the 29-member governing board of the California Institute for Regenerative Medicine (CIRM), as the agency is formally known.

Up for decisions are two training programs on which CIRM has already spent $52.4 million. Costs per student average nearly $65,000 in one effort.

Extension of the high school training program would cost only $550,000 for one year. However, training at community and state colleges would cost $9 million for one year. That is the program that has schooled 782 students at a cost of about $65,000 each.

Extending both programs for one year would mean nearly $10 million less for basic research or support of clinical trials that could lead to actual stem cell treatments. Keep in mind that board has turned its focus sharply towards aiding clinical trials – which are very expensive -- in hopes of fulfilling the promises of the 2004 ballot campaign that led to the creation of the stem cell agency.

Strong cases have been made for continuation of the programs, most recently by Susan Baxter, executive director of the California State University’s system-wide program for biotechnology, who appeared before the board and asked for a review of the college program.

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 (college training) 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.”
Jeanne Loring, director the Scripps stem cell program, also supported the college training program.  She said recently,
“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 of the arguments made by backers is that one of those relatively few college or high school students could become the next Jamie Thomson, the scientist who is widely credited with starting what may be the stem cell revolution.  It is an appealing vision with strong emotional impact.

And it is also where college football comes in. Many persons support such athletic programs, contending that they provide a way for young persons to train and become a high profile athlete who earns tens of millions of dollars annually, possibly leading a pro team to the Super Bowl year after year.

The odds of that happening for an individual college student are supremely low. Only 1.6 percent of college players ever join pro teams – 254 out of 70,174 college players, according to the latest NCAA figures. Even fewer achieve stardom.

The odds of professional success resulting from the college football and CIRM training programs are not too different, this writer suggests.

Does that mean that the agency should not continue the training efforts. Of course not. But directors should be very clear about the message they deliver about their priorities and their limited resources. Funds likely run out in 2020, according the agency’s latest estimate, and it is years away from producing all the cures promised by the 2004 campaign – something that, in fact, may never happen.

Some might ask whether these programs – if they are so valuable – should not be funded by the state or community colleges themselves. The answer is that those systems have been starved for state funding for years and that situation is not likely to change dramatically any time soon. Of course, those institutions can change their own priorities if they truly believe that stem cell training is more valuable than some of their other offerings.

CIRM’s new president, Randy Mills, has not taken a public position on the training efforts. But he has offered four criteria to evaluate funding decisions by the agency.  They are:
“1. Will it speed up the development of treatments for patients?
“2. Will it increase the likelihood of developing a successful treatment for patients?
“3. Will it meet an unmet medical need?
“4. Is it efficient?” 
Come Thursday, California’s stem cell agency will apply those criteria and maybe some others to decide the fate of the two training programs.
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Wednesday, October 15, 2014

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. 
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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. 
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Friday, October 10, 2014

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.”
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Wednesday, October 08, 2014

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.   
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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.
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Wednesday, October 01, 2014

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.)

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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.”
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Wednesday, September 24, 2014

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.

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