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Category Archives: Stem Cells
US team aim to make human sperm
Posted: October 9, 2012 at 11:17 am
8 October 2012 Last updated at 22:25 ET By Regan Morris BBC News, Los Angeles
US researchers say they will redouble their efforts to create human sperm from stem cells following the success of a Japanese study involving mice.
A Kyoto University team used mice stem cells to create eggs, which were fertilised to produce baby mice.
Dr Renee Pera, of Stanford University in California, aims to create human sperm to use for reproduction within two years, and eggs within five years.
Infertility affects up to 15% of reproductive-aged couples worldwide.
"I know people think it's Frankenstein medicine, but I think it's not an imagined or lessened health problem - infertility affects your whole life," Dr Pera says.
"To have sex and have a baby would be a super simple decision, but not everybody can do it."
But using embryonic stem cells for research - as Dr Pera's lab at the Institute for Stem Cell Biology and Regenerative Medicine does - is controversial because the embryos are destroyed in order to use them.
Dr Pera's lab uses embryos left over from IVF treatments.
Stem cells have the potential to grow into any cell in the body. Creating eggs in a lab could become mainstream, much like IVF is viewed today.
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US team aim to make human sperm
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Milestones in Stem Cell Science
Posted: October 8, 2012 at 3:18 pm
Gail Martin, PhD
Since 1981, when UCSFs Gail Martin, PhD, co-discovered embryonic stem cells in mice and coined the term embryonic stem cell, UCSF has been a key player in the stem cell field.
The success in 1998 by the University of Wisconsins James Thomson in deriving human embryonic stem cells from embryos propelled the stem cell research field forward.
Beginning in the late 1990s, UCSFs Roger Pedersen, PhD, was one of two University scientists nationwide the other being James Thomson, DVM, PhD, of the University of Wisconsin to pioneer the human embryonic stem cell field. Following Thomsons 1998 discovery of a technique for deriving human embryonic stem cells from donated embryos left over following in vitro fertilization efforts, Pedersens lab derived two of its own lines of cells using the same technique.
In 2006, Shinya Yamanaka, MD, PhD, a senior investigator and the L.K. Whittier Foundation Investigator in Stem Cell Biology at the Gladstone Institute of Cardiovascular Disease and a professor of anatomy at UCSF, developed the method for inducing skin cells from mice into becoming like pluripotent stem cells and called them iPS cells. In 2007, Yamanaka did the same with adult human skin cells.
Shinya Yamanaka, MD, PhD
Yamanakas experiments revealed that adult skin cells, when treated with four pieces of DNA (now called the Yamanaka factors), can induce skin cells to revert back to their pluripotent state. His discovery has since led to a variety of methods for reprogramming adult cells into stem cells that can become virtually any cell type such as a beating heart cell or a neuron that can transmit chemical signals in the brain. This allows researchers to create patient-specific cell lines that can be studied and used in everything from drug therapies to regenerative medicine.In between and since, there has been major progress in scientists understanding of stem cells.
Today, fueled in part by the robust research enterprise at UCSF, the field is burgeoning. Yamanaka now commutes between Japan and San Francisco, where he is a professor of anatomy at UCSF and a senior investigator at the UCSF-affiliated J. David Gladstone Institute for Cardiovascular Disease.
At UCSF, Arnold Kriegstein, MD, PhD, director of the Eli and Edythe Broad Center of Regeneration Medicine and Stem Cell Research at UCSF, leads one of the largest and most comprehensive programs of its kind in the United States.
In about 125 labs, basic science researchers carry out studies in cell culture and animals aimed at understanding healthy cell function and disease progression and developing treatment strategies for a broad spectrum of disorders, including heart disease, diabetes, neurological diseases such as epilepsy, multiple sclerosis, Parkinsons disease and spinal cord injury and cancer. Clinical research teams have begun one of the first early-stage stem cell clinical trials in the United States, and other potential trials are on the horizon.
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Milestones in Stem Cell Science
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Nobel prize to Briton, Japanese for stem cell work
Posted: October 8, 2012 at 3:18 pm
STOCKHOLM (AP) A British researcher and a Japanese scientist won the Nobel Prize in physiology or medicine on Monday for discovering that ordinary cells of the body can be reprogrammed into stem cells, which then can turn into any kind of tissue a discovery that may led to new treatments.
Scientists want to build on the work by John Gurdon and Shinya Yamanaka to create replacement tissues for treating diseases like Parkinson's and diabetes, and for studying the roots of diseases in the laboratory without the ethical dilemma posed by embryonic stem cells.
In announcing the 8 million kronor ($1.2 million) award, the Nobel committee at Stockholm's Karolinska Institute said the discovery has "revolutionized our understanding of how cells and organisms develop."
Gurdon showed in 1962 the year Yamanaka was born that the DNA from specialized cells of frogs, like skin or intestinal cells, could be used to generate new tadpoles. That showed the DNA still had its ability to drive the formation of all cells of the body.
At the time, the discovery had "no obvious therapeutic benefit at all," Gurdon told reporters in London.
"It was almost 50 years before the value the potential value of that basic scientific research comes to light," he said.
In 1997, the cloning of Dolly the sheep by other scientists showed that the same process Gurdon discovered in frogs would work in mammals.
More than 40 years after Gurdon's discovery, in 2006, Yamanaka showed that a surprisingly simple recipe could turn mature cells back into primitive cells, which in turn could be prodded into different kinds of mature cells.
Basically, the primitive cells were the equivalent of embryonic stem cells, which had been embroiled in controversy because to get human embryonic cells, human embryos had to be destroyed. Yamanaka's method provided a way to get such primitive cells without destroying embryos.
"The discoveries of Gurdon and Yamanaka have shown that specialized cells can turn back the developmental clock under certain circumstances," the committee said. "These discoveries have also provided new tools for scientists around the world and led to remarkable progress in many areas of medicine."
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Nobel prize to Briton, Japanese for stem cell work
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Stem-Cell Pioneers Gurdon, Yamanaka Win Nobel Prize
Posted: October 8, 2012 at 3:18 pm
John B. Gurdon transferred DNA between a tadpole and a frog to clone the first animal. Shinya Yamanaka used Gurdons concept to turn ordinary skin into potent stem cells. Both won the Nobel Prize for medicine today.
Gurdon, 79, a researcher at the University of Cambridge in the U.K., and Yamanaka, 50, a professor at Kyoto University in Japan, will share the 8 million-kronor ($1.2 million) prize, the Nobel Assembly said today in Stockholm. The pairs findings have created new opportunities to study diseases and develop methods for diagnosis and therapy, the assembly said in a statement.
Gurdons feat, in 1962, paved the way in 1996 for the cloning of Dolly the sheep and, 10 years later, for Yamanaka, who turned mouse skin cells into stem cells with the potential to become any cell in the body. That achievement was lauded by some politicians and religious figures as a more ethical way to make stem cells because it doesnt destroy human life.
This field has had a long history, starting with John Gurdon, Yamanaka, who was born the same year Gurdon published his achievement, said in an interview on the Nobel Assemblys website. I was able to initiate my project because of his experiments 50 years ago.
Stem cells are found in human embryos and in some tissues and organs of adults, and have the potential to develop into different types of cells. Thats spurred scientists to look at ways of harnessing their power to treat diseases such as Alzheimers, stroke, diabetes and rheumatoid arthritis, according to the U.S. National Institutes of Health.
Gurdon showed that mature cells from specific parts of an animals body retain all the genetic information they had as immature stem cells. He took a cell from a tadpoles gut, extracted the nucleus, and inserted it into the egg cell of an adult frog whose own nucleus had been removed. That reprogrammed egg cell developed into a tadpole with the genetic characteristics of the original tadpole, and subsequent trials yielded adult frogs.
Gurdon overturned the prevailing view that as cells differentiate, they lose genes and their ability to generate other cells of any kind, said Alan Colman, the executive director of the Singapore Stem Cell Consortium, who gained his doctorate under Gurdon at Cambridge.
Hes amazingly passionate, Colman said in an interview before the award was announced. He was the sort of supervisor who you found it difficult to get appointments with, not because he was flying around the world, but because he was doing experiments all the time.
Gurdon was answering e-mails in his laboratory when he received the call from Sweden today about the prize, he said in an interview on the Nobel Assemblys website. His first reaction was, Its amazing if its really true, he said. Could it be that someones pulling your leg? That has happened before.
Gurdon will celebrate at a reception that his institute is hosting today, and then hell be back to work early tomorrow, he said at a London news conference today.
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Stem-Cell Pioneers Gurdon, Yamanaka Win Nobel Prize
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Analysis: Reprogrammed cells open new medical window
Posted: October 8, 2012 at 3:18 pm
LONDON (Reuters) - The Nobel Prize-winning discovery of how to reprogram ordinary cells to behave like embryonic stem cells offers a way to skirt around ethical problems with human embryos, but safety concerns make their future use in treating disease uncertain.
While researchers have already applied the scientific breakthroughs of Britain's John Gurdon and Japan's Shinya Yamanaka to study how diseases develop, making such cells into new treatments will involve a lot more checks.
Stem cells act as the body's master cells, providing the source material for all other cells. They could transform medicine by regenerating tissue for diseases ranging from blindness to Parkinson's disease.
Creating embryo-like stem cells without destroying embryos gets round a key controversy by avoiding the need to process embryos left over at fertility clinics - a system that has led to political objections in the United States and elsewhere.
Reprogrammed cells - known as induced pluripotent stem cells, or iPS cells - offer an ethically neutral alternative. They have been a source of intense research since Yamanaka discovered their potential in 2006, building on work that Gurdon did in frogs and tadpoles 40 years earlier.
SAFETY CONCERNS
Recently, however, different research groups have noticed problems with iPS cells, suggesting they may not be as good as embryonic ones. In one study, iPS cells died more quickly and another found multiple genetic mutations, raising concerns that they could cause tumors.
Despite this, Japanese researchers hope to test iPS cells in clinical trials for a form of blindness as early as next year - catching up with recent successful eye trials using embryonic stem cells.
Researchers in the West are generally more wary.
"There is a bit of a divergence between Japan and the rest of the world on this," Chris Mason, professor of regenerative medicine at University College London, told Reuters.
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Analysis: Reprogrammed cells open new medical window
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UK, Japan scientists win Nobel for stem cell breakthroughs
Posted: October 8, 2012 at 3:18 pm
STOCKHOLM (Reuters) - Scientists from Britain and Japan shared a Nobel Prize on Monday for the discovery that adult cells can be transformed back into embryo-like stem cells that may one day regrow tissue in damaged brains, hearts or other organs.
John Gurdon, 79, of the Gurdon Institute in Cambridge, Britain and Shinya Yamanaka, 50, of Kyoto University in Japan, discovered ways to create tissue that would act like embryonic cells, without the need to harvest embryos.
They share the $1.2 million Nobel Prize for Medicine, for work Gurdon began 50 years ago and Yamanaka capped with a 2006 experiment that transformed the field of "regenerative medicine" - the field of curing disease by regrowing healthy tissue.
"These groundbreaking discoveries have completely changed our view of the development and specialization of cells," the Nobel Assembly at Stockholm's Karolinska Institute said.
All of the body's tissue starts as stem cells, before developing into skin, blood, nerves, muscle and bone. The big hope for stem cells is that they can be used to replace damaged tissue in everything from spinal cord injuries to Parkinson's disease.
Scientists once thought it was impossible to turn adult tissue back into stem cells, which meant that new stem cells could only be created by harvesting embryos - a practice that raised ethical qualms in some countries and also means that implanted cells might be rejected by the body.
[Related: Facts about the Nobel Prize for Medicine]
In 1958, Gurdon was the first scientist to clone an animal, producing a healthy tadpole from the egg of a frog with DNA from another tadpole's intestinal cell. That showed developed cells still carry the information needed to make every cell in the body, decades before other scientists made headlines around the world by cloning the first mammal, Dolly the sheep.
More than 40 years later, Yamanaka produced mouse stem cells from adult mouse skin cells, by inserting a few genes. His breakthrough effectively showed that the development that takes place in adult tissue could be reversed, turning adult cells back into cells that behave like embryos. The new stem cells are known as "induced pluripotency stem cells", or iPS cells.
"The eventual aim is to provide replacement cells of all kinds," Gurdon's Institute explains on its website.
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UK, Japan scientists win Nobel for stem cell breakthroughs
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Nobel Prize awarded for stem cell breakthroughs
Posted: October 8, 2012 at 3:18 pm
Reuters
This undated handout photo shows iPS cells derived from adult human dermal fibroblasts released by Kyoto University Professor Shinya Yamanaka at Center for iPS Cell Research and Application of Kyoto University in Kyoto, western Japan.
By Reuters
Scientists from Britain and Japan shared a Nobel Prize on Monday for the discovery that adult cells can be transformed back into embryo-like stem cells that may one day regrow tissue in damaged brains, hearts or other organs.
John Gurdon, 79, of the Gurdon Institute in Cambridge, Britain and Shinya Yamanaka, 50, of Kyoto University in Japan, discovered ways to create tissue that would act like embryonic cells, without the need to harvest embryos.
They share the $1.2 million Nobel Prize for Medicine, for work Gurdon began 50 years ago and Yamanaka capped with a 2006 experiment that transformed the field of "regenerative medicine" - the field of curing disease by regrowing healthy tissue.
"These groundbreaking discoveries have completely changed our view of the development and specialization of cells," the Nobel Assembly at Stockholm's Karolinska Institute said.
Photoblog: Click for a close-up viiew of the Nobel Prize-winning stem cell research
All of the body's tissue starts as stem cells, before developing into skin, blood, nerves, muscle and bone. The big hope for stem cells is that they can be used to replace damaged tissue in everything from spinal cord injuries to Parkinson's disease.
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Nobel Prize awarded for stem cell breakthroughs
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Researcher Alert: California Stem Cell Agency Tightening Budget Oversight on Grants
Posted: October 7, 2012 at 4:06 pm
Some of California's top stem cell
researchers are going to have to sharpen their spreadsheets if they
want to win money from the state's $3 billion stem cell agency.
The agency is moving to beef up
scrutiny of the high-profile, big-ticket grant applications
that it will consider during the next several years. The effort may well extend to all grant programs. The move also makes
it clear to researchers that the CIRM staff is in the driver's seat
when it comes to budgeting on research projects.
scrutiny of the high-profile, big-ticket grant applications
that it will consider during the next several years. The effort may well extend to all grant programs. The move also makes
it clear to researchers that the CIRM staff is in the driver's seat
when it comes to budgeting on research projects.
The plan was laid out this week in a memo to directors of the California Institute for Regenerative Medicine (CIRM) by Ellen Feigal, the agency's senior vice
president for research and development. She said,
president for research and development. She said,
“Increasing the importance of
budgetary review will encourage applicants to propose rigorous,
realistic and vetted budgets, and will further our mission to be good
stewards of taxpayer dollars. These additions will not significantly
increase the workload burden on GWG members (grant reviewers) and
explicitly acknowledge that program goals, scientific plans, accurate budgeting and prudent spending are inextricably linked.”
The proposal comes before the CIRM
directors' Science Subcommittee next Monday and would alter the
closed-door grant review process in the following manner, according
to Feigal's memo.
directors' Science Subcommittee next Monday and would alter the
closed-door grant review process in the following manner, according
to Feigal's memo.
• “To assist GWG review,
appropriate expertise on budget and financial matters (e.g., this
could be in the form of a specialist reviewer, or can also be
assigned to a GWG reviewer with the appropriate background and
expertise), will review applications for sound budgeting and provide
comments or questions to the GWG for consideration by the reviewers
before the reviewer’s final scores are entered.
• “If the financial/budgetary
matter potentially directly impacts on the design or feasibility of
conducting the project, the GWG may consider this issue in the
scoring; otherwise, budgetary and financial issues and questions will
not contribute to the scientific score.
• “As appropriate, review summaries
sent to the ICOC (the CIRM governing board) will identify scientific
as well as budget or other issues. To the extent endorsed by the
GWG, the review summaries will also identify potential resolution
should the ICOC approve a given award with budget issues.
• “CIRM officers should be provided
explicit discretion to consider the budget comments, as well as
budget or other issues. To the extent endorsed by the GWG, the
review summaries will also identify potential resolution should the
ICOC approve a given award with budget issues.”
Feigal's memo clearly indicates that
CIRM staff has experienced push-back from recalcitrant researchers
when efforts have been made to bring costs under control. She noted that
the agency's staff examines a research project's budget during the
“prefunding” review that follows board approval. However, Feigal
said, at that stage, “It is often challenging to make substantive
changes to the budget, based on appropriateness of study activities
and costs, given the ICOC approval at a given budget amount.”
CIRM staff has experienced push-back from recalcitrant researchers
when efforts have been made to bring costs under control. She noted that
the agency's staff examines a research project's budget during the
“prefunding” review that follows board approval. However, Feigal
said, at that stage, “It is often challenging to make substantive
changes to the budget, based on appropriateness of study activities
and costs, given the ICOC approval at a given budget amount.”
The agency has already examined some
budgets prior to board approval. One grant review in a $200
million-plus round this summer, for example, declared that costs to
prepare regulation packages had “overlap” and were “excessive,”
along with costs dealing with manufacturing and per patient expenses.
That was for a high-scoring application by Antoni Ribas of UCLA, and
he was not alone.
budgets prior to board approval. One grant review in a $200
million-plus round this summer, for example, declared that costs to
prepare regulation packages had “overlap” and were “excessive,”
along with costs dealing with manufacturing and per patient expenses.
That was for a high-scoring application by Antoni Ribas of UCLA, and
he was not alone.
In her memo, Feigal listed other cases
of budgetary shortcomings in recent applications:,
of budgetary shortcomings in recent applications:,
• “Budget does not align with the
program deliverables and milestones. For example, the budget
includes activities not relevant to project objective(s) or that are
out of scope.
•”Budget does not contain adequate
expenses for known costs. For example, an applicant may budget
$100,000 for a GMP manufacturing run of a biologic in which it is
generally accepted knowledge that the actual expenses are typically
much greater.
•“Budget item significantly exceeds
a known cost or seems excessive without adequate justification. For
example, an applicant may propose a surgical expense of $100,000 per
patient for a procedure with Medicare reimbursement set at $15,000.
•“Cost allocations are not done
properly. For example, an applicant is developing the same
therapeutic candidate for 3 indications, and is applying for CIRM
funding for 1 of the 3, but is charging CIRM for the cost of the
entire manufacturing run.”
Initially, the budgetary review would
be used in disease team, early translational, strategic partnership
rounds, and any new rounds “as deemed appropriate.” Feigal said,
however, that “all applications for CIRM awards should be
carefully examined for budgetary appropriateness.”
be used in disease team, early translational, strategic partnership
rounds, and any new rounds “as deemed appropriate.” Feigal said,
however, that “all applications for CIRM awards should be
carefully examined for budgetary appropriateness.”
Our take: This seems to be a
well-advised move, albeit one that is not likely to find favor with
researchers accustomed to loose oversight. It moves budgetary review
to an earlier stage and gives the CIRM directors a chance to weigh in
on those matters prior to approval of grants, instead of creating a
sense of entitlement on the part of recipients that may pop up
following board approval of their applications. Indeed, the plan
makes such good sense that it raises the question why it was not in
place years ago.
well-advised move, albeit one that is not likely to find favor with
researchers accustomed to loose oversight. It moves budgetary review
to an earlier stage and gives the CIRM directors a chance to weigh in
on those matters prior to approval of grants, instead of creating a
sense of entitlement on the part of recipients that may pop up
following board approval of their applications. Indeed, the plan
makes such good sense that it raises the question why it was not in
place years ago.
A final note: Feigal's memo is an
excellent example of the type of information that clarifies issues
and helps CIRM directors make the best possible decisions. It
provides some history, good evidence for a change and an explanation
of benefits. Additionally, the memo is timely, having been posted on
the CIRM website sufficiently in advance of next week's meeting to give affected parties and others time to comment
and make constructive suggestions. The memo is also far superior to
the Power Point presentations that are often submitted to the board
minus any nuanced, written discussion of the issue at hand.
excellent example of the type of information that clarifies issues
and helps CIRM directors make the best possible decisions. It
provides some history, good evidence for a change and an explanation
of benefits. Additionally, the memo is timely, having been posted on
the CIRM website sufficiently in advance of next week's meeting to give affected parties and others time to comment
and make constructive suggestions. The memo is also far superior to
the Power Point presentations that are often submitted to the board
minus any nuanced, written discussion of the issue at hand.
Next week's meeting will be based in
San Francisco but also has teleconference locations in Irvine (2), La
Jolla, Stanford, Pleasanton, Oakland and Los Angeles where the public
and researchers can participate. The specific addresses can be found on the agenda.
San Francisco but also has teleconference locations in Irvine (2), La
Jolla, Stanford, Pleasanton, Oakland and Los Angeles where the public
and researchers can participate. The specific addresses can be found on the agenda.
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UCD’s Knoepfler’s ‘Somewhat Provocative Paper’ on iPS
Posted: October 7, 2012 at 4:06 pm
UC Davis researcher Paul Knoepfler is
the rare stem cell scientist who blogs about his work as well as
writing about issues in the field.
Over the weekend, he posted an item on
what he described as a “somewhat provocative paper” published by his lab in
“Stem Cells and Development.” He said the paper argued
that iPS cells “are very similar in some ways to cancer cells.”
what he described as a “somewhat provocative paper” published by his lab in
“Stem Cells and Development.” He said the paper argued
that iPS cells “are very similar in some ways to cancer cells.”
Most of his item deals with the
technical details and background of the research. But at the end of
this item, Knoepfler wrote,
technical details and background of the research. But at the end of
this item, Knoepfler wrote,
“So what does this mean in the big
picture?
“I believe that iPS cells and cancer
cells are, while not the same, close enough to be called siblings. As
such, the clinical use of iPS cells should wait for a lot more study.
Even if scientists do not use iPS cells themselves for transplants,
but instead use differentiated derivatives of iPS cells, the risk of
patients getting malignant cancers cannot be ignored.
“At the same time, the studies
suggest possible ways to make iPS cells safer and support the notion
of reprogramming cancer cells as an innovative new cancer therapy.
“Stay tuned in the next few days for
part 2 where I will discuss what this paper went through in terms of
review, etc. to get published. It wasn’t a popular story for some
folks.”
The UC Davis press release on the
research, which was financed by the California stem cell agency and the NIH, was picked up by several online sites, including Redorbit,
Medicalexpress and geekosystem.
research, which was financed by the California stem cell agency and the NIH, was picked up by several online sites, including Redorbit,
Medicalexpress and geekosystem.
Posted in Stem Cells, Stem Cell Therapy
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Researcher Alert: California Stem Cell Agency Tightening Budget Oversight on Grants
Posted: October 7, 2012 at 4:05 pm
Some of California's top stem cell
researchers are going to have to sharpen their spreadsheets if they
want to win money from the state's $3 billion stem cell agency.
The agency is moving to beef up
scrutiny of the high-profile, big-ticket grant applications
that it will consider during the next several years. The effort may well extend to all grant programs. The move also makes
it clear to researchers that the CIRM staff is in the driver's seat
when it comes to budgeting on research projects.
scrutiny of the high-profile, big-ticket grant applications
that it will consider during the next several years. The effort may well extend to all grant programs. The move also makes
it clear to researchers that the CIRM staff is in the driver's seat
when it comes to budgeting on research projects.
The plan was laid out this week in a memo to directors of the California Institute for Regenerative Medicine (CIRM) by Ellen Feigal, the agency's senior vice
president for research and development. She said,
president for research and development. She said,
“Increasing the importance of
budgetary review will encourage applicants to propose rigorous,
realistic and vetted budgets, and will further our mission to be good
stewards of taxpayer dollars. These additions will not significantly
increase the workload burden on GWG members (grant reviewers) and
explicitly acknowledge that program goals, scientific plans, accurate budgeting and prudent spending are inextricably linked.”
The proposal comes before the CIRM
directors' Science Subcommittee next Monday and would alter the
closed-door grant review process in the following manner, according
to Feigal's memo.
directors' Science Subcommittee next Monday and would alter the
closed-door grant review process in the following manner, according
to Feigal's memo.
• “To assist GWG review,
appropriate expertise on budget and financial matters (e.g., this
could be in the form of a specialist reviewer, or can also be
assigned to a GWG reviewer with the appropriate background and
expertise), will review applications for sound budgeting and provide
comments or questions to the GWG for consideration by the reviewers
before the reviewer’s final scores are entered.
• “If the financial/budgetary
matter potentially directly impacts on the design or feasibility of
conducting the project, the GWG may consider this issue in the
scoring; otherwise, budgetary and financial issues and questions will
not contribute to the scientific score.
• “As appropriate, review summaries
sent to the ICOC (the CIRM governing board) will identify scientific
as well as budget or other issues. To the extent endorsed by the
GWG, the review summaries will also identify potential resolution
should the ICOC approve a given award with budget issues.
• “CIRM officers should be provided
explicit discretion to consider the budget comments, as well as
budget or other issues. To the extent endorsed by the GWG, the
review summaries will also identify potential resolution should the
ICOC approve a given award with budget issues.”
Feigal's memo clearly indicates that
CIRM staff has experienced push-back from recalcitrant researchers
when efforts have been made to bring costs under control. She noted that
the agency's staff examines a research project's budget during the
“prefunding” review that follows board approval. However, Feigal
said, at that stage, “It is often challenging to make substantive
changes to the budget, based on appropriateness of study activities
and costs, given the ICOC approval at a given budget amount.”
CIRM staff has experienced push-back from recalcitrant researchers
when efforts have been made to bring costs under control. She noted that
the agency's staff examines a research project's budget during the
“prefunding” review that follows board approval. However, Feigal
said, at that stage, “It is often challenging to make substantive
changes to the budget, based on appropriateness of study activities
and costs, given the ICOC approval at a given budget amount.”
The agency has already examined some
budgets prior to board approval. One grant review in a $200
million-plus round this summer, for example, declared that costs to
prepare regulation packages had “overlap” and were “excessive,”
along with costs dealing with manufacturing and per patient expenses.
That was for a high-scoring application by Antoni Ribas of UCLA, and
he was not alone.
budgets prior to board approval. One grant review in a $200
million-plus round this summer, for example, declared that costs to
prepare regulation packages had “overlap” and were “excessive,”
along with costs dealing with manufacturing and per patient expenses.
That was for a high-scoring application by Antoni Ribas of UCLA, and
he was not alone.
In her memo, Feigal listed other cases
of budgetary shortcomings in recent applications:,
of budgetary shortcomings in recent applications:,
• “Budget does not align with the
program deliverables and milestones. For example, the budget
includes activities not relevant to project objective(s) or that are
out of scope.
•”Budget does not contain adequate
expenses for known costs. For example, an applicant may budget
$100,000 for a GMP manufacturing run of a biologic in which it is
generally accepted knowledge that the actual expenses are typically
much greater.
•“Budget item significantly exceeds
a known cost or seems excessive without adequate justification. For
example, an applicant may propose a surgical expense of $100,000 per
patient for a procedure with Medicare reimbursement set at $15,000.
•“Cost allocations are not done
properly. For example, an applicant is developing the same
therapeutic candidate for 3 indications, and is applying for CIRM
funding for 1 of the 3, but is charging CIRM for the cost of the
entire manufacturing run.”
Initially, the budgetary review would
be used in disease team, early translational, strategic partnership
rounds, and any new rounds “as deemed appropriate.” Feigal said,
however, that “all applications for CIRM awards should be
carefully examined for budgetary appropriateness.”
be used in disease team, early translational, strategic partnership
rounds, and any new rounds “as deemed appropriate.” Feigal said,
however, that “all applications for CIRM awards should be
carefully examined for budgetary appropriateness.”
Our take: This seems to be a
well-advised move, albeit one that is not likely to find favor with
researchers accustomed to loose oversight. It moves budgetary review
to an earlier stage and gives the CIRM directors a chance to weigh in
on those matters prior to approval of grants, instead of creating a
sense of entitlement on the part of recipients that may pop up
following board approval of their applications. Indeed, the plan
makes such good sense that it raises the question why it was not in
place years ago.
well-advised move, albeit one that is not likely to find favor with
researchers accustomed to loose oversight. It moves budgetary review
to an earlier stage and gives the CIRM directors a chance to weigh in
on those matters prior to approval of grants, instead of creating a
sense of entitlement on the part of recipients that may pop up
following board approval of their applications. Indeed, the plan
makes such good sense that it raises the question why it was not in
place years ago.
A final note: Feigal's memo is an
excellent example of the type of information that clarifies issues
and helps CIRM directors make the best possible decisions. It
provides some history, good evidence for a change and an explanation
of benefits. Additionally, the memo is timely, having been posted on
the CIRM website sufficiently in advance of next week's meeting to give affected parties and others time to comment
and make constructive suggestions. The memo is also far superior to
the Power Point presentations that are often submitted to the board
minus any nuanced, written discussion of the issue at hand.
excellent example of the type of information that clarifies issues
and helps CIRM directors make the best possible decisions. It
provides some history, good evidence for a change and an explanation
of benefits. Additionally, the memo is timely, having been posted on
the CIRM website sufficiently in advance of next week's meeting to give affected parties and others time to comment
and make constructive suggestions. The memo is also far superior to
the Power Point presentations that are often submitted to the board
minus any nuanced, written discussion of the issue at hand.
Next week's meeting will be based in
San Francisco but also has teleconference locations in Irvine (2), La
Jolla, Stanford, Pleasanton, Oakland and Los Angeles where the public
and researchers can participate. The specific addresses can be found on the agenda.
San Francisco but also has teleconference locations in Irvine (2), La
Jolla, Stanford, Pleasanton, Oakland and Los Angeles where the public
and researchers can participate. The specific addresses can be found on the agenda.
Posted in Stem Cells, Stem Cell Therapy
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