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Thursday, October 23, 2014
From California's new Alpha clinics to threatened training programs, catch it all today with updates from the meeting of the governing board of the $3 billion California stem cell agency.
The California Stem Cell Report will be monitoring the session via the Internet from its post in Banderas Bay in Mexico and will file stories as warranted. Today's meeting in Los Angeles begins at 9 a.m. PDT.
If you are interested in following the session directly, the meeting agenda has directions on how to listen in.
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Wednesday, October 22, 2014
The $3 billion California stem cell agency today put the squeeze on applicants in its ambitious Alpha stem cell clinic round, which is aimed at helping to make the Golden State a global leader in stem cell treatment and research.
The action came as one rejected applicant, UC Davis, publicly appealed to the agency’s governing board to overturn tomorrow a negative decision by the agency’s out-of-state reviewers. Cedar-Sinai in Los Angeles also filed a letter aimed at beefing up consideration of its application, which wound up on the reviewers’ funding fence.
A proposal by Randy Mills, the agency’s new president, however, contained the most surprising news – a plan to cut millions from the budgets of the expected winning applicants – City of Hope (application AC1-07659), UC San Diego (AC1-07764) and UCLA (AC1-07675).
The cuts were specified in a slide presentation posted yesterday or today for the board meeting in Los Angeles tomorrow. The presentation recommended approval of the three applications but with revised budgets to hit a funding cap of $8 million each. The reviewers had recommended approval of the three with budgets ranging from about $11 million to $11.7 million.
The presentation and another document based on the closed-door review indicated that reviewers were concerned about duplicative clinical trial costs and expenses associated with a proposed $10 million information center, which was trimmed earlier this year from $15 million. The request for applications to create the center is being rewritten to sharpen its focus.
The other document from the review represented the first time that such a commentary has been prepared and released publicly. It was requested by an unidentified agency board member who was present at the review session. (When queried, the agency later identified the member as Os Steward, director of the Reeve-Irvine Research Center at UC Irvine.)
The unusual document said, among other things,
The unusual document said, among other things,
“Some reviewers had questions regarding the wording of the RFA and the potential for duplication of costs. Specifically, there was confusion over the portion of the RFA that listed ‘Clinical Trial Costs’ as specifically being outside the scope of the RFA, while simultaneously permitting a funded Alpha Clinic to ‘defray some costs, such as clinical operations.’”As originally proposed, the Alpha plan would have hit $70 million with up to five clinics, although there was no requirement to fund that many. It is now down to something around $40 million if four applications are approved tomorrow at $8 million each, and the information center moves forward with $10 million.
The four-page letter from the Cedars-Sinai Medical Center in Los Angeles to the directors of the stem cell agency was signed by Timothy Henry, director of the division of cardiology. Reviewers gave the $11 million application (AC1-07650) a scientific score of 68 and said,
“Given the large expansion into regenerative medicine that the institution has undertaken, many of the activities described as elements of the (Alpha clinic) have already been initiated or will soon be put in place. Some reviewers questioned whether the additional resources provided by (Alpha) funding would substantially supplement ongoing activities and if the (Alpha clinic) would add significant value."
Henry’s letter did not appear to break new ground but emphasized the strengths of Cedars-Sinai, declaring that one of the clinical trials proposed is “the furthest advanced of any CIRM-funded trials.”
The score for the UC Davis’ $10.8 million application (AC1-07637) was not released by the agency. Reviewers said,
“While some reviewers felt that the large number of trials ongoing or planned would clearly benefit from additional resources, others questioned the added value of establishing a (an Alpha clinic) at this site and did not think the application clearly conveyed how CIRM funds would be used to leverage and enhance the already ongoing activities.”
The three-page Davis appeal letter was signed by Ted Wun, chief of hematology oncology, Jan Nolta, director of the UC Davis stem cell program, and Frederick Meyers, vice dean of the UC Davis School of Medicine.
The letter said that since the review of its application, it has received a favorable FDA judgment on a proposed clinical trial for HIV that would begin in 2015. In an attempt to deal with concerns about personnel, Davis said the Alpha clinic would pull over the most experienced researchers with their old positions being “backfilled.”
The letter also noted Davis’ location in Northern California. All of the top-ranked applications are situated in Southern California, presenting a scientific-geopolitical issue for the statewide stem cell agency.
(The stem cell agency does not release the names of winning applicants prior to board action. However, they were derived by the California Stem Cell Report from a variety of sources. All of the reviews can be found on this document.)Sphere: Related Content
Tuesday, October 21, 2014
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.Sphere: Related Content
Sunday, October 19, 2014
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.
“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.Sphere: Related Content
Wednesday, October 15, 2014
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.Sphere: Related Content
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,
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.
Friday, October 10, 2014
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.”
Wednesday, October 08, 2014
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.Sphere: Related Content
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.Sphere: Related Content