Today is more than showdown day for the functioning of the federal
government. It is the last day to submit nominations for the Stem
Cell Person of 2013.
This is the second year for the contest, which carries a $1,000
prize provided by UC Davis stem cell researcher Paul Knoepfler. He
runs the contest and is the ultimate arbiter on who wins.
You have until midnight to submit your nominations. Here are links to more details.
With more than 3.0 million page views and more than 5,000 items, this blog provides news and commentary on public policy, business and economic issues related to the $3 billion California stem cell agency. David Jensen, a retired California newsman, has published this blog since January 2005. His email address is djensen@californiastemcellreport.com.
Monday, September 30, 2013
California's $70 Million Alpha Stem Cell Clinic Plan Edging Forward
The $3 billion California stem cell agency is predicting that
“human healthcare will be greatly improved” by stem cell research
by 2024 and this week is fleshing out some of the details of an
ambitious plan to help make that happen.
The effort involves what the agency calls “Alpha” stem cell clinics. The agency said its proposed $70 million, Alpha network will support its own projects along with products developed worldwide and brought to California.
The plan will come before the stem cell agency's standards group tomorrow at a meeting in San Francisco. The agency's staff has recommended alteration of some of the agency's regulations to avoid duplication in connection with review and approval of clinical trials.
The staff document said a workshop earlier this year developed a “consensus that it would be beneficial if the CIRM (the stem cell agency) regulations reflected existing state and federal requirements with regard to delegation of responsibility and avoid unnecessary duplication of effort, such as the IRB (institutional review board) responsibility for the risk and benefit assessment.”
The staff is recommending that “CIRM’s regulatory requirements for clinical research should be modified to avoid duplication of IRB’s responsibility for review and approval of clinical trials.”
The staff also said,
Dubbed the “Discuss Project,” its goal is “to initiate a process designed to develop consensus for the use of previously collected specimens for iPSC research" and to publish "final considerations" in early 2014.
Another item on the agenda tomorrow involves an update on the “progress of CIRM iPSC bank and donor consent protocol.”
The effort involves what the agency calls “Alpha” stem cell clinics. The agency said its proposed $70 million, Alpha network will support its own projects along with products developed worldwide and brought to California.
The plan will come before the stem cell agency's standards group tomorrow at a meeting in San Francisco. The agency's staff has recommended alteration of some of the agency's regulations to avoid duplication in connection with review and approval of clinical trials.
The staff document said a workshop earlier this year developed a “consensus that it would be beneficial if the CIRM (the stem cell agency) regulations reflected existing state and federal requirements with regard to delegation of responsibility and avoid unnecessary duplication of effort, such as the IRB (institutional review board) responsibility for the risk and benefit assessment.”
The staff is recommending that “CIRM’s regulatory requirements for clinical research should be modified to avoid duplication of IRB’s responsibility for review and approval of clinical trials.”
The staff also said,
“CIRM’s existing regulatory requirements for notification, review and approval of basic and pre-clinical research appear effective at this time without creating undue burdens. In fact, mature systems appear to be in place to efficiently incorporate ESCRO (embryonic stem cell research oversight) operations into institutional compliance programs.”Also on the standards group agenda are issues dealing with informed consent by providers of somatic cells “obtained under general (biomedical) research protocols" in connection with iPSC (induced pluripotent stem cell) derivation.
Dubbed the “Discuss Project,” its goal is “to initiate a process designed to develop consensus for the use of previously collected specimens for iPSC research" and to publish "final considerations" in early 2014.
Another item on the agenda tomorrow involves an update on the “progress of CIRM iPSC bank and donor consent protocol.”
Tuesday, September 24, 2013
Deadline Approaching on Stem Cell Person of the Year Awards
Only six more days remain to submit nominations for the Stem Cell
Person of the Year.
This is the contest run by UC Davis stem cell researcher Paul Knoepfler, who will give the winner $1,000 of his own, personal cash.
The criteria are nearly wide open although Knoepfler is looking for the “most influential” person in the field. He wrote on his blog,
Knoepfler reported today on his blog that he has received two dozen nominations so far. He is planning on opening up the selection of the semi-finalists to online voting from the list that will be published in early October. Last year he selected the semi-finalists himself.
As for the names he has received so far, Knoepfler said,
This is the contest run by UC Davis stem cell researcher Paul Knoepfler, who will give the winner $1,000 of his own, personal cash.
The criteria are nearly wide open although Knoepfler is looking for the “most influential” person in the field. He wrote on his blog,
“Nominees can be almost anyone ranging from an advocate to an academic or industry scientist to a stem cell biotech leader to a policy maker to a physician…think outside the box! Surprise me!”Knoepfler initiated the contest last year. The 2012 winner was Roman Reed, a San Francisco Bay Area patient advocate.
Knoepfler reported today on his blog that he has received two dozen nominations so far. He is planning on opening up the selection of the semi-finalists to online voting from the list that will be published in early October. Last year he selected the semi-finalists himself.
As for the names he has received so far, Knoepfler said,
“This group of nominees is very diverse, far more so than last year, and includes more scientists and physicians.”Nominations should be sent to knoefpler@ucdavis.edu. More information about the rules can be found here.
Wednesday, September 18, 2013
Surgeon Expected to be Added to California Stem Cell Agency Board
Julie Freischlag, UC Davis Photo |
Freischlag was named this week as the new vice chancellor for human health services and dean of the UC Davis medical school, replacing Claire Pomeroy, who resigned to become president of the Lasker Foundation.
Pomeroy had served on the stem cell board since 2005. She was named to the board by the UC Davis chancellor because of her position as dean of the medical school.
Freischlag, who is scheduled to begin her new job Feb. 10, is no ordinary surgeon. Currently she is chair of the Department of Surgery at Johns Hopkins. Prior to that she was chief of vascular surgery at UCLA. A Baltimore Sun profile of her in 2003 said she stands out because of her stellar career in “perhaps the most macho specialty in medicine.” The article said women surgeons are outnumbered 5 to 1 by men.
Responding to a query from the California Stem Cell Report, a spokeswoman for the UC Davis chancellor was noncommital about whether Freischlag would be appointed to the stem cell agency board. Luanne Lawrence, associate chancellor for strategic communications at Davis, said,
“This is one of the important and transitional decisions that hasn’t been addressed quite yet.”However, the deans of the other major California medical schools sit on the stem cell agency board, which gives away $300 million a year for research. Given that context, it would seem prudent for the dean of the UC Davis medical school to serve on the board as well.
For more on Freischlag's background, see here and here.
Sunday, September 15, 2013
Inside Stem Cells: A Guide from a UC Davis Researcher
The Sacramento Bee today published an article on UC Davis stem cell researcher Paul Knoepfler and his new book, which explores the basics of stem cells and how to make good judgments concerning
possible treatments.
The book, “Stem Cells: An Insider's Guide,” is aimed at a general audience while at the same time exploring the nuances of stem cell science.
Below is a copy of the article, which appeared on the main page of The Bee Web site early today and which was written by yours truly. In the print version of The Bee, the Knoepfler piece appeared on the same page as a New York Times story dealing with dubious stem cell treatments(Here is a link to the original version of the Times' piece.).
The Knoepfler story was also the No. 1 article that turned up this afternoon when the Google news category was searched using the term “stem cell.” It is likely to be published elsewhere in the coming weeks as it is circulated by the McClatchy News Service.
UC Davis stem cell researcher warns consumers to beware of unproven or dangerous stem cell treatments
In his day job, UC Davis scientist Paul Knoepfler probes the inner workings of stem cells and cancer cells and what makes them behave the way they do.
“Paul is a powerful advocate for helping the public understand what research is being done, and why it is important. He is gifted at taking complex science and turning it into plain English so that anyone can understand what he's talking about.”
The book, “Stem Cells: An Insider's Guide,” is aimed at a general audience while at the same time exploring the nuances of stem cell science.
Below is a copy of the article, which appeared on the main page of The Bee Web site early today and which was written by yours truly. In the print version of The Bee, the Knoepfler piece appeared on the same page as a New York Times story dealing with dubious stem cell treatments(Here is a link to the original version of the Times' piece.).
The Knoepfler story was also the No. 1 article that turned up this afternoon when the Google news category was searched using the term “stem cell.” It is likely to be published elsewhere in the coming weeks as it is circulated by the McClatchy News Service.
UC Davis stem cell researcher warns consumers to beware of unproven or dangerous stem cell treatments
In his day job, UC Davis scientist Paul Knoepfler probes the inner workings of stem cells and cancer cells and what makes them behave the way they do.
On
the side, the father of three daughters blogs about costly, unproven
stem cell treatments and provides guidance for those seeking
experimental therapies.
Knoepfler
is a rare stem cell researcher who regularly explores the most
problematic aspects of stem cell therapies on the Internet in full
public gaze. He considers himself an advocate for patients as well
as a scientist, having survived an aggressive form of prostate
cancer at the age of 42.
Now
46, Knoepfler began his blog in 2010, shortly after his cancer was
diagnosed. His blogging has encountered resistance from some
colleagues, who are uncomfortable with such public endeavors. But he
has polished and expanded the blog to the point that it has received
international recognition. He will receive an award in December at
the World Stem Cell Summit in San Diego for his advocacy efforts.
This
month he moved beyond cyberspace and published “Stem Cells: An
Insider’s Guide” (World Scientific Publishing). The book covers
stem cells from A to Z and is aimed at the general reader,
especially people considering stem cell therapy for themselves, a
family member or friend.
The
book comes during what has been described by Christopher Scott, a
senior research scholar at Stanford University, as “an epidemic of
transplant clinics offering so-called cures and therapies” both
here and abroad. The international stem cell medical tourism
business, widely promoted on the Internet, is taking in roughly $1
billion annually, according to an estimate in Stanford Medicine, a
publication of the Stanford medical school.
Knoepfler
is a believer in the potential and power of stem cells. But he says
that some of the dubious stem cell treatments now being offered have
resulted in deaths and injuries. Other unpleasant issues arise as
well in stem cell research, including the tendency of some stem
cells to generate cancer cells, and Knoepfler wants to talk about
them.
“Too
often in the academic section of the stem cell field, people
pussyfoot around the most important issues or do not even dare talk
about them at all,” says Knoepfler.
He
is “not on some crusade to dissuade people from getting risky stem
cell procedures,” but says safety and training need to be
encouraged. Reckless behavior endangers the entire field, he says.
“There
is no better illustration of the risks of unlicensed stem cell
treatments administered by untrained doctors than the recently
reported case of a woman who received a stem cell facelift, only to
have bone grow in her eye,” he said, referring to a Scientific
American report involving a Beverly Hills clinic.
To
help patients, Knoepfler’s book begins with the basics: What are
stem cells? He moves on to topics ranging from whether stem cells
can treat baldness to whether they can help with afflictions such as
Alzheimer’s, autism and arthritis. Along the way, he discusses the
potential for regenerating limbs, made-to-order organ transplants
and curing spinal paralysis, none of which have reached the stage
where patients can be assured of using the techniques safely and
effectively.
Stem
cell treatments are not exactly new. They were first used in the
1950s in bone marrow transplants. More recently, what has excited
researchers and the public are pluripotent stem cells, which have
the ability to transform themselves into any part of the body. The
full range of stem cells, however, includes adult, fetal, embryonic
and induced pluripotent cells (iPS), which are also known as
reprogrammed adult stem cells.
“Each
type of stem cell has clinical promise for specific diseases, but
also certain weaknesses. Some stem cells also stir controversy. For
example, embryonic stem cells have been at the center of ethical
debates for more than a dozen years,” Knoepfler writes.
Embryonic
stem cells are derived from an embryo only a few days after
fertilization and require destruction of the embryo. It was that
type of process that triggered the federal restrictions – now
rescinded by President Barack Obama – on federal funding of
research using human embryonic stem cells. Some people believe that
the process amounts to killing a human being, while others do not.
Knoepfler
has come up with a battery of questions and “rights” that
persons considering a stem cell treatment should address as they
contemplate spending tens of thousands of dollars for treatments.
His
“patient bill of rights” includes the right to treatment by a
trained provider, the right to continuing follow-up and the right
not to be charged to participate in a clinical trial.
Just
this month, he added on his blog, ipscell.com, the top 10 questions
that patients should ask. They include such areas as the details of
the methods to be used and costs, the cell numbers and types,
regulatory compliance by the provider, data supporting the cellular
product’s effectiveness and data supporting its safety.
As
for mainstream media reports and research papers that seem to
promise miraculous cures, Knoepler says, “My advice to patients is
to be cautious when reading papers on the clinical use of stem
cells. Do not believe everything you read and avoid placing too much
weight on any one paper. If something is real, it should be
reproducible by multiple groups.”
One
of the areas of Knoepfler’s research involves cancer stem cells, a
topic of special interest to him because of his own encounter with
the disease. “Even with something as wonderful as stem cells,
there also can too much of a good thing. Stem cells can cause cancer
and in certain conditions they can make cancer especially hard to
cure,” he explains.
In
the case of embryonic stem cells, he cites their ability to form an
unusual tumor called a teratoma. Knoepfler, who was an English
literature major as an undergraduate, says, “The name ‘teratoma‘
literally means ‘monster tumor,’ and there is good reason for
that nomenclature. These tumors look monstrous when observed by eye,
akin to an animal put into a developmental blender.”
Knoepfler
did not use stem cell therapy to treat his cancer, but he says that
as a cancer survivor he understands all too well why patients turn
to it when they’re dealing with a life-threatening or
life-changing medical condition.
“If
you feel that your disease has put you in a place for which patience
is not an option,” he says, “talk with your physician. Get
second and maybe even third and fourth opinions before deciding
whether or not to proceed.”
Knoepfler’s
advice may not be appreciated by some of the firms he writes about,
but others think well of him.
Jonathan
Thomas, chairman of the $3 billion California stem cell agency, says
the field needs more scientists like Knoepfler.
“It's
hard to overstate the value of what Paul does,” Thomas said in an
email.“Paul is a powerful advocate for helping the public understand what research is being done, and why it is important. He is gifted at taking complex science and turning it into plain English so that anyone can understand what he's talking about.”
Knoepfler
has received $2.2 million from the stem cell agency for his
research, which is also funded by the federal government and other
sources. He has been at UC Davis since 2006, part of the campus’
stem cell program, which has received $131 million in grants from
the state stem cell agency since 2005, generating 333
research-related jobs.
Knoepfler,
whose father was a physician and mother a counselor, sees a bright
future for stem cells, indeed a “medical revolution.”
“Stem
cells are today’s new frontier of medicine that will no doubt have
an unimaginable impact on our lives, but even more so on the lives
of many of our kids and grandchildren,” he said.
Jensen
is a retired Sacramento Bee journalist and has produced the
California Stem Cell Report (californiastemcellreport.blogspot.com)
on the Internet since 2005, writing nearly 3,600 items.
Friday, September 13, 2013
Selling Stem Cell Sizzle: The Future of a $3 Billion Effort
All stem cell research is not created equal, a truism that
found fresh validity this week. Particularly research that could play a role
in whether the California stem cell agency can find more cash to continue its
operations.
The difference was highlighted yesterday by articles on the stem
cell agency’s blog. The pieces dealt with findings – some esoteric and some
not-so-esoteric -- that received international attention. The articles written by CIRM staffers Amy Adams and Don Gibbons were fine as far as they went. But it is one thing to deal with the nuts and bolts of
research and another to look at it from the perspective of whether it resonates
with the public.
The research in question is from Spain and Stanford. Researchers
in Spain reprogrammed adult cells in a
living mouse to become like embryonic stem cells. Those results received much “gee
whiz” attention in the mainstream media, most of which overlooked problematic aspects involving its cancer-linked results(see researcher Paul Knoepfler's take here and Gibbons' item here).
The other findings out of Stanford dealt with people and
Down syndrome, along with cognitive function, aging and Alzheimer’s.
The press release by Krista Conger from Stanford said,
"'Conceptually, this study suggests that drug-based strategies to slow the rate of stem cell use could have profound effects on cognitive function, aging and risk for Alzheimer’s disease in people with Down syndrome,' said co-author Craig Garner, PhD, who is the co-director of Stanford’s Center for Research and Treatment of Down Syndrome and a professor of psychiatry and behavioral sciences.”
Both the press release and the CIRM blog item briefly noted
that funding from CIRM helped to sustain the research. Stanford buried the information at the end of its release. CIRM mentioned it much higher in
its item.
In neither case were specific funding figures mentioned. Nor
was there any attempt to say whether this research would have been slow in
coming or not coming at all without CIRM help.
Why does that matter? The $3 billion state stem cell agency
will run out of funds for new grants in about three years, not very long given the
length of time it takes to develop major funding sources and the rather
deliberate pace at which CIRM works on some matters.
Currently the agency spends about $300 million year on
research and is not likely to be able to renew its funding at that level. But
if it wants to play at even the $50 million level, it will have to generate
some sizzle from the research that it has funded.
Sizzle is what the Stanford research has. It resonates with
people. We all know somebody or a family with issues such Alzheimer’s, Down
syndrome or cognitive problems. Missing largely, however, from the press releases, media
stories and even the CIRM blog is some sort of way of assessing whether CIRM
funding played a KEY role.
And that is the clincher for agency. That is the sizzle that
will sell the agency as absolutely necessary if it truly wishes to turn
stem cells into cures.
(Editor's note: Shortly after this item was posted, we searched the agency's Web site. One of the results disclosed that agency gave $1.4 million to Michael Clarke of Stanford for the research. He has filed two progress reports on his findings.) The research received additional support from CIRM as well, but the amounts were not readily apparent.)
(Editor's note: Shortly after this item was posted, we searched the agency's Web site. One of the results disclosed that agency gave $1.4 million to Michael Clarke of Stanford for the research. He has filed two progress reports on his findings.) The research received additional support from CIRM as well, but the amounts were not readily apparent.)
Shuffling the Files: Changes on the CIRM Web Site
The California stem cell agency has re-organized its
cyberspace book shelves to help make its Web site easier to
navigate.
Nothing has been removed, said Amy Adams, the major domo
for the CIRM’s Internet operation.
Writing on the agency’s blog, she explained one of the major
changes dealing with “our funding” category.
“It turns out that people looking for information about research we've funded weren't thinking to look under 'our funding.' And since no other section of the site seemed like a likely place to find that content, people sent me helpful emails suggesting that I add pages about where our funding has gone (I politely directed them to that content).”
Most of the information on funding has now been moved to “our
progress,” she said, leaving only information on how to get and manage grants under the “our
funding” category. Other changes have occurred as well. You can read all about them here.
Wednesday, September 11, 2013
California Stem Cell Agency Blogs on Questionable Stem Cell Clinics
The
California stem cell agency has taken notice of a piece in the New York Times
that reports on questionable practices of enterprises that say they are stem
cell clinics.
In a blog item written yesterday by Don Gibbons, senior
science and education officer at the agency, the agency said that the practices described in the article generated a cautionary advisory to patients last month by the stem cell agency and 12 other organizations.
Gibbons noted that much of the Sept. 9 Times article by Laura Beil focused on one clinic, the Regenerative Medicine Institute in Tijuana, which is just on the California-Mexico border. Gibbons’ item reported that one U.S. expert says that some of the clinic’s key procedures would not pass muster in this country.
Gibbons noted that much of the Sept. 9 Times article by Laura Beil focused on one clinic, the Regenerative Medicine Institute in Tijuana, which is just on the California-Mexico border. Gibbons’ item reported that one U.S. expert says that some of the clinic’s key procedures would not pass muster in this country.
Gibbons continued,
The Times article quoted UC Davis researcher Paul Knoepfler as saying,“Most important, the clinic has not produced any publishable data. We all have great hope for the promise of stem cell science, but I think it is fair to say that anyone who legitimately cares about that promise wants to find out what is the right type of cell to put in each patient. When is the right time for the transplant, and what is the best method of delivering the cells. We will never learn those things without collecting data in a well-designed clinical trial and sharing that data.”
Gibbons also said that the International Society for Stem Cell Research, which backed away under threats of lawsuits a few years ago from some criticism of dubious stem cell clinics, plans to release this week a new statement on these sorts of treatments.“There is absolutely no legitimate reason for such clinics to be not publishing their data.”
We should note that
Times article, which is syndicated to many newspapers in this country, did not pick
up the widely reported instances of deaths and injuries from these treatments. It
did report that problems exist in this country as well as abroad. The article
said,
“In the United States, too, it is easy to conduct business outside government oversight, said Dr. George Q. Daley, who studies stem cells for blood diseases at Harvard Medical School. Close down one shady operation, he went on, and more seem to randomly pop up.
“Even questionable publicity does not necessarily hurt business. Regnocyte, a company in Florida, posted an unflattering CNN report about it on its Web site under the heading ‘special coverage.’”
Monday, September 09, 2013
Rise Up and Blog! Any Scientist Can!
The old saying has it that you can tell the pioneers
by the arrows in their backs.
As he put it:
Tell that to Paul Knoepfler, the UC Davis stem cell
researcher who has been something of a pioneer in stem cell blogging and still
is -- at least given that almost no other stem cell researcher blogs both as a researcher
and patient advocate in addition to taking on wide-ranging business and public policy
issues.
Knoepfler does have a few arrows in his back, some
from enterprises that are less than happy with his vieww of their conduct and
some from within the stem cell research community itself. But overall he has
found the experience beneficial and rewarding.
In an article in Nature Medicine Sept. 6, Knoepfler
is recruiting more researchers for the stem cell blog brigade. To encourage
them, he recounts his experiences and costs (such as $1,500 for his special
domain name, although a normal domain name can be had for virtually no cost). His
experiences include working late at night and on weekends. We can testify that
the father of three puts in the hours, just based on the nature of the
blog content and frequency of posting.
Knoepfler continues his pitch in his piece in
Nature, which is read primarily by researchers,
“To all this a reader might say, ‘Okay, this all sounds well and good, but you can't have your cake and eat it, too! Your science must have suffered from all this.’ In fact, I have lost some sleep, but I have not seen much in the way of negative scientific consequences. My lab has done very well during the past three years, and we have published many important papers, including one earlier this year that demonstrated the molecular similarities between induced pluripotency in stem cells and oncogenic transformation in cancer cells. I even secured tenure during this time.Knoepfler’s bottom line pitch? Any scientist can!
“Reflecting on my personal transformation to the role of scientist−advocate, I have come to notice many tangible positive outcomes. In a general sense, my work has served to build bridges and stimulate new dialogue between industry and academia in the stem cell field. At the same time, I couple these efforts to accountability. Today, if someone does an Internet search for 'stem cell blog', that person will find my site, with all its educational outreach resources, at the top of the results list. A few years ago, the same search would mostly have yielded sites published by opponents of stem cell research or proponents of sketchy, for-profit endeavors to attract stem cell 'tourism'. I have also interacted with more than 100 patients and caregivers, helping them make more educated and, I believe, safer, stem cell−related medical decisions with their physicians.”
As he put it:
“Any scientist can, and should, do it. It is only logical that scientists would adapt to today's reality—a funding-poor environment that is nonetheless rich with opportunities for communication—by becoming advocates. I predict that any scientist who devotes a tiny bit of time to advocacy endeavors will find that the payoff is greatly multiplied. I will even help. Drop me an e-mail. Or better yet, leave a comment on my blog or message me on Twitter. Let's get the conversation going.”
Tuesday, September 03, 2013
WARF Stem Cell Challenge: Appeal Says Patent Involves Cells Not 'Markedly Different' Than Found in Human Body
The battle over whether excessive protection of stem
cell IP stifles research that can lead to cures was engaged once more today
with a broadside against the powerful Wisconsin Alumni Research Foundation(WARF).
The attack came from California’s Consumer Watchdog
organization and New York’s Public Patent Foundation which have been tussling
with WARF for seven years. The dispute over
intellectual property (IP) centers on a patent on human embryonic stem cells
held by WARF and which the other organizations are challenging in a federal
appellate court in Washington, D.C.
More specifically, the patent involves research by
Jamie Thomson of the University of Wisconsin, and now also of UC Santa Barbara,
in which he isolated human embryonic stem cells.
Consumer Watchdog of Santa Monica, Ca., this morning
issued a news release concerning the organizations’ appellate brief that was filed last
week. It cited the U.S. Supreme Court ruling earlier this year that said genes
cannot be patented because they exist in nature. The lead attorney in that successful case, Dan
Ravicher of the Public Patent Foundation, is also handling the challenge to WARF.
The news release said that Thomson deserved credit
for being first to isolate and maintain human embryonic stem cells, but “his achievement was not the result of
his having created a patentable invention.” The brief said that the work involved
was “obvious.” One of the main reasons for Thomson’s achievement, the news
release said, was that “he had
access to human embryos and financial support that other researchers did not
have.”
The brief said,
“The claims at issue here cover human embryonic stem (hES) cells that are not markedly different from those in our bodies. Thus, the claims are invalid under 35 U.S.C. § 101 for covering ineligible subject matter, an issue the Court may and, as a matter of judicial economy and public policy, should address.”
The challenge to the WARF
patent has drawn impressive support in the scientific community, including Jeanne Loring, now director of the Center for
Regenerative Medicine at The Scripps Research Institute, who was involved from
the start. In 2007, Loring wrote in Nature that she became involved in the case
because “scientists have an obligation not only to perform research but to make
sure that our research can benefit the society that supports it.”
The news release said,
“Later in the case Dr. Alan Trounson, then of Australia’s Monash University and now president of the California Institute for Regenerative Medicine, Dr. Douglas Melton of Harvard and Dr. Chad Cowan of Harvard filed affidavits supporting the challenge.”
Friday, August 30, 2013
Tracking the Fruits of California Stem Cell Agency Research
The California stem
cell agency yesterday shed some interesting light on the awards in its $41
million round this week and their pathway to actually producing a product that
can be used to treat persons who are suffering from diseases.
It is a difficult and
long journey to generate usable therapies, a process poorly understood by the
public, which was promised in 2004 that the stem cell agency would produce
cures for ailments afflicting half the population of the state.
Writing on the agency’s blog, Amy Adams, CIRM communications manager, dealt with the issue indirectly.
She said,
“Many scientists who receive our early translation awards first got their idea for a therapy while carrying out research with one of our other awards. In fact, eight of the scientists in this round of funding had previous CIRM funding for an earlier stage of research. If a scientist's early translation award provides good results, the scientists are then able to apply for one of our disease team awards, which fund the effort of compiling data to convince the Food and Drug Administration to allow them to test it in people. Other organizations fund only early discovery research or only preclinical research. Under those conditions, researchers continually pause their projects to look for new sources of funding as the project moves through the phases toward clinical trial.”
One of the virtues of
the California stem cell agency is its promise of a continued stream of
funding. Former Chairman Robert Klein used to tout that particular aspect of
the agency, particularly in light of limited federal resources.
Adams’ comments
implicitly raise important questions concerning CIRM’s entire portfolio. How
many CIRM grants have led to additional funding from CIRM? How many are
basically one-off shots that have not led to research that has advanced the
development of stem cell therapies, either via CIRM or other funding. What is the
therapeutic and scientific significance of the research that is linked by more
than one CIRM award? What previously
funded CIRM research could be fruitfully funded again to advance the science
and not necessarily through the traditional grant rounds, which sometimes have
awkward timing?
Unmentioned in Adams’
item is an application from a UC Irvine researcher that came up at Wednesday’s
meeting of the governing board of the stem cell agency. The woman, whose name
was not clearly audible on the Internet audiocast, publicly appealed rejection
of her application by reviewers. She noted that it was an extension of work
that was previously funded by the agency. She also noted that the score on her
review was all but identical to work that was funded. The board, however,
turned her appeal aside, which had already been rejected behind closed doors by
CIRM staff.
Hers is not the only
such case in CIRM history. But they are virtually impossible to track systematically
because of the structure of the CIRM grant-making progress. It is also not
clear whether the agency itself is tracking its research awards to determine if
they result in continuing, fruitful research in a specific area. Nonetheless,
the matter deserves some public attention.
Wednesday, August 28, 2013
UCLA Takes Four of 13 Awards Today; One Business Wins
The California stem cell agency has
made it official, sending out its press release on the $41 million in grants
approved today for institutions throughout the state.
Most of the 13 awards, as usual, went to organizations represented on the governing board of
the agency. Individual board members, however, are barred from voting on specific grants
to their organizations.
UCLA topped the list with four grants. No other
institution received more than one, including only one business, Numerate, Inc., of San Bruno,
via John
Griffin, the firm's chief scientific officer. The lack of awards
to businesses has long been a sore subject in the biotech community.
The only news story so far was written by Bradley Fikes of the San Diego U-T, which circulates in an area that is a hotbed of biotech research. Institutions there snagged $12.6 million in four grants. Fikes also
identified one of the five researchers who lost their appeals on negative grant
review decisions. He is Evan
Snyder, leader of stem cell research at Sanford-Burnham Medical Research
Institute in La Jolla, who
had a $5 million request before the agency.
CIRM Board Meeting Adjourns
Today's meeting of the directors of the California stem cell agency concluded at 4:11 p.m. PDT. We do not anticipate posting additional news items today.
Stem Cell Agency Seeks Stronger Ties with Possible Industry Funding Partners
The California stem cell agency today triggered a new program aimed at recruiting major biotech and venture capital firms to assist in providing tens of millions of dollars for research by California enterprises.
The effort, part of an $80 million business-friendly initiative, was approved by the agency's governing board on a voice vote.
The effort, part of an $80 million business-friendly initiative, was approved by the agency's governing board on a voice vote.
Participating companies will have a special relationship with the state agency, according to a staff document. The "industry collaborators" will have early input into concept funding proposals prior to their presentation to the agency's governing board. The companies will also be able to attend agency workshops and meetings involving hundreds of grant recipients.
Other aspects of the proposal call for special event-hosting arrangements aimed at creating more collaborations along with posting of information from the selected collaborators on the CIRM website.
California Stem Directors Still in Closed Session
Directors of the California stem cell agency today are still huddled behind closed doors, discussing an evaluation of CIRM President Alan Trounson. It is not clear when the public meeting will resume. Normally, little is publicly disclosed about such private personnel sessions, which are permitted under state law.
UCLA Snags $3.6 Million from California Stem Cell Agency
UCLA scored today with at least two grants, totaling $3.6 million, from the California stem cell agency.
Seeking the cash were Donald Kohn, application 6823, and Gerald Lipshutz, application 6831. Both of the grants are for $1.8 million each.
Seeking the cash were Donald Kohn, application 6823, and Gerald Lipshutz, application 6831. Both of the grants are for $1.8 million each.
Their applications were initially in the agency's tier two category, which means that CIRM's reviewers did not approve them outright for funding. CIRM staff, however, did under a new procedure, and the agency's governing ratified the recommendation.
Lipshutz also appeared before the board along with several patient advocates who made emotional appeals for funding. Lipshutz's research deals with urea cycle disorders, which occur in one out of 8,200 births. Current treatment is arduous and can involve liver transplants.
Kohn's research deals with sickle cell disease, which afflicts primarily African-Americans. His efforts are aimed at correcting the sickle gene defect in the blood stem cells before transplanting them back into the patient.
$41 Million in California Stem Cell Grants Virtually Approved
Directors of the California stem cell agency today all but approved about $41 million in early translational grants, rejecting all appeals by applicants and accepting staff recommendations on marginal grants.
The roll call vote was held open this morning to record a vote by one board member who was not present at the time. It is virtually certain that the member will vote in favor of affirmative action on the applications in question.
One member of the board, Joan Samuelson, abstained from voting on any of the applications. She said she did not think the board had adequate information on its total grant portfolio, particularly in view of the declining amount of money available.
The agency has about $600 million in uncommitted funds and is scheduled to run out of cash for new grants in 2017.
The research acted on today is aimed at “proof of concept for development of a therapy candidate and/or studies to select a development candidate. The approved grants can be found on this CIRM website page and are listed in tier one and tier two. Identities of the applicants are withheld by CIRM to avoid embarrassing rejected candidates and to avoid disclosing the names of applicants to board members before they vote. However, applicants often appear before the board, as they did today, and identify themselves.
Five applicants appealed negative decisions on their applications by grant reviewers. The agency declined to disclose the appeal letters or identify the applicants, information that was a public record under the previous appeal procedures. New processes were put in place this spring that moved the appeals behind closed doors and made them subject to staff instead of board review. Nonetheless, rejected researchers have a legal right to address the board on appeals or any other matters.
At the request of the California Stem Cell Report, the agency provided the numbers of the grants on which appeals were filed. They are: 06787, 06888, 06761, 06793 and 06830. Review summaries on the applications can be found here.
We have asked the agency to provide its legal and policy justification for now withholding information that was once a public record.
The roll call vote was held open this morning to record a vote by one board member who was not present at the time. It is virtually certain that the member will vote in favor of affirmative action on the applications in question.
One member of the board, Joan Samuelson, abstained from voting on any of the applications. She said she did not think the board had adequate information on its total grant portfolio, particularly in view of the declining amount of money available.
The agency has about $600 million in uncommitted funds and is scheduled to run out of cash for new grants in 2017.
The research acted on today is aimed at “proof of concept for development of a therapy candidate and/or studies to select a development candidate. The approved grants can be found on this CIRM website page and are listed in tier one and tier two. Identities of the applicants are withheld by CIRM to avoid embarrassing rejected candidates and to avoid disclosing the names of applicants to board members before they vote. However, applicants often appear before the board, as they did today, and identify themselves.
Five applicants appealed negative decisions on their applications by grant reviewers. The agency declined to disclose the appeal letters or identify the applicants, information that was a public record under the previous appeal procedures. New processes were put in place this spring that moved the appeals behind closed doors and made them subject to staff instead of board review. Nonetheless, rejected researchers have a legal right to address the board on appeals or any other matters.
At the request of the California Stem Cell Report, the agency provided the numbers of the grants on which appeals were filed. They are: 06787, 06888, 06761, 06793 and 06830. Review summaries on the applications can be found here.
We have asked the agency to provide its legal and policy justification for now withholding information that was once a public record.
Labels:
appeals,
Grant-making,
ICOC,
openness,
translational
Stem Cell Board Moves into Closed Door Lunch Session
Today's meeting of the California stem cell agency governing board is now in recess for a closed-door lunch session involving the evaluation of CIRM President Alan Trounson. It is not clear when the open session will resume, but probably it will be at least 90 minutes.
UCLA Application for $1.8 Million Moves Ahead at Stem Cell Board Meeting
Directors of the California stem cell agency this morning moved forward on a $1.8 million application from UCLA that the agency's reviewers declined to approve outright.
The application (No. 6831) was moved into a category that will be approved routinely later today by the board. CIRM staff recommended that the application be approved.
Advocates speaking on behalf of the application identified UCLA as the institution involved. A researcher also spoke, but his name was not clearly audible on the Internet audiocast.
The application (No. 6831) was moved into a category that will be approved routinely later today by the board. CIRM staff recommended that the application be approved.
Advocates speaking on behalf of the application identified UCLA as the institution involved. A researcher also spoke, but his name was not clearly audible on the Internet audiocast.
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