BioTime Makes Bid for Geron’s Stem Cell Assets
By Dr. Matthew Watson
Biotime, Inc., and two men who were
leading players in history of Geron Corp. today made a surprise,
public bid for the stem cell assets of their former firm.
 |
| Michael West West photo |
 |
| Tom Okarma AP file photo |
Okarma. West founded Geron in 1990 and was its first CEO. West is
now CEO of Biotime. Okarma was CEO of Geron from 1999 to 2011.
Okarma joined Biotime on Sept. 28 to lead its acquistion efforts.
Both Geron, based in Menlo Park, Ca., and Biotime, based in Alameda,
Ca., are publicly traded.
a pitch for the Geron's stem cell assets. Geron jettisoned its hESC
program nearly a year ago and closed its clinical trial program for
spinal injuries. The move shocked the California stem cell agency,
which just a few months earlier had signed an agreement to loan the
firm $25 million to help fund the clinical trial. The portion of the
loan that was distributed was repaid with interest.
sell off the hESC program, but no buyers have surfaced publicly.
Personnel in the program have been laid off or found employment
elsewhere.
said that under the deal,
“Geron would transfer its stem cell
assets to BAC(a new subsidiary of Biotime headed by Okarma), in
exchange for which you along with the other Geron shareholders would
receive shares of BAC common stock representing approximately 21.4%
of the outstanding BAC capital stock. BioTime would contribute to BAC
the following assets in exchange for the balance of outstanding BAC
capital stock:
- “$40 million in BioTime common
shares;- “Warrants to purchase BioTime
common shares (“BioTime Warrants”);- “Rights to certain stem cell
assets of BioTime, and shares of two BioTime subsidiaries engaged in
the development of therapeutic products from stem cells.”
write the firm's board of directors to urge them to approve the
offer.
proposal. Asked for comment, Kevin McCormack, spokesman for the
California stem cell agency, said the deal “had nothing to do with
us.” However, in the past, CIRM has indicated that it could find a
way to transfer the loan to an entity that would continue spinal
injury clinical trial. CIRM President Alan Trounson was also involved
at one point in trying to assist in a deal.
today while Biotime's shares lost four cents to $3.95.
that have appeared so far on the proposed deal: Associated Press, Marketwatch.
Source:
Stem Cell Therapy
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October 21st, 2012
California Stem Cell Agency Boosting Disease Team Program to $543 Million
By Dr. Matthew Watson
Directors of the California stem cell
agency are set to give away $20 million next Thursday and authorize
a handsome addition to their signature disease team effort, bringing
its total to $543 million.
agency's push to develop therapies prior to running out of money for
new grants in 2017.
the first two winners in the agency's new strategic partnership
program. CIRM says the effort is aimed at
creating “incentives and processes that will: (i) enhance the
likelihood that CIRM funded projects will obtain funding for Phase
III clinical trials (e.g. follow-on financing), (ii) provide a source
of co-funding in the earlier stages of clinical development, and
(iii) enable CIRM funded projects to access expertise within
pharmaceutical and large biotechnology partners in the areas of
discovery, preclinical, regulatory, clinical trial design and
manufacturing process development.”
board action even when applicants have identified themselves.
disease team round will come on top of the second, $213 million disease
team awards approved last this summer. The first round, awarded in
2009, totaled $230 million. The size of the new round could be altered by CIRM directors prior to approval. Also before the board is a $40 million
proposal to expand the industry-friendly strategic partnership effort
into a second round.
is to speed the process of establishing clinical trials and to finance
efforts that might founder in what the biotech industry calls a
valley of death – a high risk financial location, so to speak,
where conventional financiers fear to tread.
“co-funding” from applicants but the agency did not specify what
it means by the term. The matter of matching funds has become an issue in awards to StemCells, Inc., of Newark, Ca., in this summer's
disease team round.
contains a plan to tighten review of proposed research budgets in
grant applications, making it clear that CIRM staff will be
negotiating such matters even after the board approves grants and
loans.
public on the budget plan although it could well have a significant
impact on their future efforts.
well at the meeting in Burlingame, which also has a teleconference
location in La Jolla that will be open to the public. The address
and additional material can be found on the agenda.
Source:Stem Cell Therapy
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October 21st, 2012
Los Angeles Times: StemCells, Inc., Award ‘Redolent of Cronyism’
By Dr. Matthew Watson
The Los Angeles Times this
morning carried a column about the “charmed relationship” between
StemCells, Inc., its “powerful friends” and the $3 billion
California stem cell agency.
Pulitzer prize winner and author Michael Hiltzik, who has been
critical of the agency in the past. The piece was the first in the major
mainstream media about a $20 million award to StemCells, Inc., that was approved in September by the agency's board. The bottom line of the
article? The award was “redolent of cronyism.”
StemCells, Inc., now ranks as the leading corporate recipient of cash
from the agency with $40 million approved during the last few months.
on September's $20 million award, which was approved despite being
rejected twice by grant reviewers – “a particularly
impressive” performance, according to Hiltzik. It was the first
time that the board has approved an award that was rejected twice by
reviewers.
“What was the company's
secret? StemCells says it's addressing 'a serious unmet medical need'
in Alzheimer's research. But it doesn't hurt that the company also
had powerful friends going to bat for it, including two guys who were
instrumental in getting CIRM off the ground in the first place.”
who led the ballot campaign that created the agency and became its
first chairman, and Irv Weissman of Stanford, who co-founded
StemCells, Inc., and sits on its board. Weissman, an internationally
known stem cell researcher, also was an important supporter of the
campaign, raising millions of dollars and appearing in TV ads. Klein,
who left the agency last year, appeared twice before the CIRM board
this summer to lobby his former colleagues on behalf of Weissman's
company. It was Klein's first appearance before the board on behalf
of a specific application.
“But private enterprise
is new territory for CIRM, which has steered almost all its grants
thus far to nonprofit institutions. Those efforts haven't been
trouble-free: With some 90% of the agency's grants having gone to
institutions with representatives on its board, the agency has long
been vulnerable to charges of conflicts of interest. The last thing
it needed was to show a similar flaw in its dealings with private
companies too.”
“(Weissman) has also
been a leading beneficiary of CIRM funding, listed as the principal
researcher on three grants worth a total of $24.5 million. The agency
also contributed $43.6 million toward the construction of his
institute's glittering $200-million research building on the Stanford
campus.”
$20 million for StemCells, Inc., came on 7-5 vote that also required
the firm to prove that it had a promised $20 million in matching
funds prior to distribution of state cash.
“The problem is that
StemCells doesn't have $20 million in spare funds. Its quarterly
report for the period ended June 30 listed about $10.4
million in liquid assets, and shows it's burning about $5 million per
quarter. Its prospects of raising significant cash from investors
are, shall we say, conjectural.
“As it happens, within
days of the board's vote, the
firm downplayed any pledge 'to raise a specific amount of
money in a particular period of time.' The idea that CIRM 'is
requiring us to raise $20 million in matching funds' is a
'misimpression,' it said. Indeed, it suggested that it might count
its existing spending on salaries and other 'infrastructure and
overhead' as part of the match. StemCells declined my request that it
expand on its statement.
“CIRM spokesman Kevin
McCormack says the agency is currently scrutinizing StemCells'
finances 'to see what it is they have and whether it meets the
requirements and expectations of the board.' The goal is to set
'terms and conditions that provide maximum protection for taxpayer
dollars.' He says, 'If we can't agree on a plan, the award will
not be funded.'"
“The agency shouldn't be
deciding on the spot what does or doesn't qualify as matching funds.
It should have clear guidelines in advance.
“Nor should the board
overturn the judgment of its scientific review panels without
clear-cut reasons....The record suggests that the handling of the
StemCells appeal was at best haphazard and at worst redolent of
cronyism.”
Source:Stem Cell Therapy
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October 21st, 2012
Researcher Alert: Stem Cell Agency to Take Up Grant Appeal Restrictions
By Dr. Matthew Watson
The move by the $3 billion California
stem cell agency to curtail its free-wheeling grant appeal process
will undergo its first public hearing next week.
whose applications are rejected by reviewers will have fewer avenues
to pursue to overturn those decisions. The changes could take effect
as early as next year.
number of appeals this summer that left the board complaining about
“arm-twisting,” lobbying and “emotionally charged presentations.”
criteria for re-review – “additional analysis” – of
applications involved in appeals, also called “extraordinary
petitions.” The plan states that re-review should occur only in
the case of a material dispute of fact or material new information.
(See the end of this item for agency's proposed definitions.)
appeal process, the CIRM directors' Application Review Task Force
will take up questions involving “ex parte communications.” The
agenda for the Oct. 24 meeting did not contain any additional
information on the issue but it likely deals with lobbying efforts on
grants outside of public meetings of the agency. We understand that
such efforts surfaced last summer involving the $$214 million disease
team round and Robert Klein, the former chairman of the stem cell
agency.
the board on one, $20 million application by StemCells, Inc., the
first time a former governing board member has publicly lobbied his former
colleagues on an application. The application was rejected twice by reviewers – once
on the initial review and again later on a re-review – but it was
ultimately approved by directors in September on a 7-5 vote.
its appeal process but last summer's events brought the matter to a
new head. The issue is difficult to deal with because state law
allows anyone to address the CIRM governing board on any subject when
it meets. That includes applicants who can ask the board to approve
grants for any reason whatsoever, not withstanding CIRM rules. The board can also approve a grant
for virtually any reason although it has generally relied on
scientific scores from reviewers.
designed to make it clear to scientists whose applications are
rejected by reviewers that the board is not going to look with favor
on those who depart from the normal appeals procedure.
overturned a positive decision by reviewers, in nearly every round it approves some applications that have been rejected by reviewers. That has
occurred as the result of appeals and as the result of motions by
board members that did not result from public appeals.
diseases that they have been involved with. Sometimes that means
seeking approval of applications with low scientific scores.
“material dispute of fact:”
“A material dispute of fact should
meet five criteria:(1) An applicant disputes the accuracy of a
statement in the review summary;(2) the disputed fact was significant
in the scoring or recommendation of the GWG(grant review group); (3) the dispute pertains
to an objectively verifiable fact, rather than a matter of scientific
judgment or opinion;(4) the discrepancy was not addressed through the
Supplemental Information Process and cannot be resolved at the
meeting at which the application is being considered; and
(5) resolution of the dispute could affect the outcome of the board’s
funding decision."
define “material new information:”
“New information should: (1)be
verifiable through external sources; (2) have arisen since the
Grants Working Group(grant review group) meeting at which the application
was considered; (3) respond directly to a specific criticism or
question identified in the Grants Working Group’s review; and (4)
be submitted as part of an extraordinary petition filed five business
days before the board meeting at which the application is
being considered."
Children's Hospital in Oakland with a teleconference location at UC
Irvine. Addresses can be found on the agenda.
Source:Stem Cell Therapy
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October 21st, 2012
Traditional breeding outperforms genetic engineering – The Hindu
By Dr. Matthew Watson
 The Hindu | Traditional breeding outperforms genetic engineering The Hindu Although exaggerated claims of rise of productivity by GM (genetically modified) crops have been made time and again in India and abroad, on closer examination these have been proved time and again to be untrue. According to a report by eminent ... |
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October 21st, 2012
Pattaya Mail
By JoanneRUSSELL25
BANGKOK, Oct 18 - A Siriraj Hospital medical team today announced its successful isolation of stem cells from amniotic fluid which they said will contribute to treatments of many chronic diseases.
Dr Udom Kachintorn, dean of the Siriraj medical school, said further research will be conducted on the use of stem cells to treat various illnesses including Alzheimers, osteoarthritis, diabetes and spinal cord pain. These chronic ailments are related to deteriorating stem cells in a human body.
A preliminary lab test has been done with animals but it will take some time before the medical team starts testing on human beings, he said.
Dr Udom said stem cells normally spread all over a person's body but they are abundant and pure in an infant's umbilical cord and placenta.
Dr Tassanee Permthai, chief of the stem cell research project, said stem cells from the placenta of a four-month-old baby can be transplanted in a patient like fully-developed stem cells.
Once transplanted in a patients body, the stem cells evolve into cells in different parts of the body, she explained, adding that the transplantation of stem cells can also prevent tumours.
More here:
Pattaya Mail
Researchers discover new blood vessel-generating cell with therapeutic potential
By raymumme
Public release date: 16-Oct-2012 [ | E-mail | Share ]
Contact: Bryan Ghosh bghosh@plos.org 44-122-344-2837 Public Library of Science
Researchers at the University of Helsinki believe they have discovered stem cells that play a decisive role in the growth of new blood vessels. If researchers learn to isolate and efficiently produce these stem cells found in blood vessel walls, the cells could offer new opportunities for developing therapeutics to treat diseases, such as cardiovascular disease and cancer. The study reporting the discovery of these stem cells is published in the open access journal PLOS Biology on October 16.
The growth of new blood vessels, known as neoangiogenesis, occurs during the repair of damaged tissue and organs in adults. However, malignant tumours also grow new blood vessels in order to receive oxygen and nutrients. As such, neoangiogenesis is both beneficial and detrimental to health, depending on the context, requiring therapeutic approaches that can either help to stimulate or prevent it. Therapeutics that aim to prevent the growth of new blood vessels are already in use, but the results are often more modest than predicted.
Adjunct Professor Petri Salvn and his team, from the University of Helsinki, now report that these stem cells can be found among the cellsso-called endothelial cellsthat line the inside of blood vessel walls. He explains, "we succeeded in isolating endothelial cells with a high rate of division in the blood vessel walls of mice. We found these same cells in human blood vessels and blood vessels growing in malignant tumours in humans. These cells are known as vascular endothelial stem cells, abbreviated as VESC. In a cell culture, one such cell is capable of producing tens of millions of new blood vessel wall cells".
From their studies in mice, the team are able to show that the growth of new blood vessels weakens, and the growth of malignant tumours slows, if the amount of these cells is below normal. Conversely, new blood vessels form where these stem cells are implanted.
"The identification and isolation of an entirely new adult stem cell type is a significant discovery in stem cell biology." explains Salvn. "Endothelial stem cells in blood vessels are particularly interesting, because they offer great potential for applications in practical medicine and the treatment of patients."
If an efficient method of vascular endothelial stem cell production could be developed, it could offer new treatment opportunities in situations where damaged tissue or diseases call for new blood vessel growth, or where the constriction or dysfunction of blood vessels deprives tissues of oxygen, for example in cardiac disease. These cells also offer new opportunities for developing therapeutics that seek to prevent new blood vessel growth in malignant tumours.
###
Funding: The work was supported by the Finnish Academy of Sciences. The funders had no role in study design, data collection and analysis, decision to publish,or preparation of the manuscript.
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Researchers discover new blood vessel-generating cell with therapeutic potential
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October 20th, 2012
The Hutch suggests changing common marrow-donation procedure
By NEVAGiles23
Originally published October 18, 2012 at 2:49 PM | Page modified October 18, 2012 at 10:51 PM
The most common method of extracting cells from unrelated donors for patients needing bone-marrow transplants should change in light of new research revealing higher complication rates than an older approach, said the head of clinical research for the Fred Hutchinson Cancer Research Center.
Significant new research published Thursday in the New England Journal of Medicine the first large, randomized study comparing the two sources of bone-marrow stem cells showed patients who received transplants from unrelated donors' blood were significantly more likely to get a complication known as chronic graft-versus-host-disease than those who received cells from donors' pelvic bones.
In an editorial accompanying the study, Dr. Fred Appelbaum, director of clinical research at the Hutch, said the blood-extraction method supplanted the traditional pelvic-bone extraction method about a decade ago. It is now used in more than three-quarters of the unrelated-donor transplants for patients with such blood malignancies as leukemia or lymphoma.
The complication, short-handed as GVHD, means the transplant recognizes the patient's tissues as foreign objects and attacks them. The condition can be relatively mild, or severe and debilitating.
"While this study should change practice, it will be interesting to see if it really does," Appelbaum wrote in his editorial. "The benefits of peripheral blood are seen early, under the watchful eyes of the transplant physician, while the deleterious effects occur late, often after the patient has left the transplant center."
About 5,500 unrelated donor transplants were done in the U.S. last year, and about 20 million potential unrelated donors are typed and listed in the registries worldwide.
The study, led by a former Hutch transplant physician, Dr. Claudio Anasetti, now at the H. Lee Moffitt Cancer Center in Tampa, Fla., looked at short- and long-term survival, transplant success and complications, both acute and chronic.
Patients survived equally well on both types of transplants, and the peripheral blood-derived grafts began doing their job faster and engrafted more reliably.
But GVHD can cause skin rashes, diarrhea and liver problems, sometimes up to three years after the transplant.
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The Hutch suggests changing common marrow-donation procedure
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October 20th, 2012
Peripheral Blood Stem Cell Transplants from Unrelated Donors Associated with Higher Rates of Chronic Graft-Versus-Host …
By NEVAGiles23
Newswise Claudio Anasetti, M.D., chair of the Department of Blood & Marrow Transplant at Moffitt Cancer Center, and colleagues from 47 research sites in the Blood and Marrow Transplant Clinical Trials Network conducted a two-year clinical trial comparing two-year survival probabilities for patients transplanted with peripheral blood stem cells or bone marrow stem cells from unrelated donors. The goal was to determine whether graft source, peripheral blood stem cells or bone marrow, affects outcomes in unrelated donor transplants for patients with leukemia or other hematologic malignancies.
Fifty transplant centers in the United States and Canada participated in this phase III study, which randomized 278 patients to receive bone marrow and 273 patients to receive peripheral blood stem cells as the graft source for transplant. The results of the study are in the Oct. 18 issue of The New England Journal of Medicine.
According to the trial analyses, there were no observed differences in overall survival, relapse, non-relapse mortality, or acute graft-versus-host disease (GHVD) between the patients receiving peripheral blood stem cells or bone marrow stem cells from unrelated donors. GVHD is a serious and often deadly post-transplant complication that occurs when the newly transplanted donor cells attack the transplant recipients body. While engraftment was faster in patients receiving peripheral blood stem cells, there was a higher incidence of overall chronic GVHD in these patients (53 percent) than in those transplanted with bone marrow stem cells (40 percent). Patients receiving transplants of peripheral blood stem cells from unrelated donors also had a higher incidence of chronic GVHD affecting multiple organs (46 percent) than patients who received bone marrow stem cells (31 percent).
Although peripheral blood stem cells from related donors have demonstrated clinical benefits, our trial demonstrates that when these stem cells originate from unrelated donors, they are not superior to bone marrow stem cells in terms of patient survival, and they increase the risk for chronic GVHD, said Anasetti, lead study author. More effective strategies to prevent GVHD are needed to improve outcomes for all patients receiving unrelated donor transplants.
Peripheral blood stem cells are stem cells originally found in the bone marrow that have been moved into the blood stream by a special regimen of drugs. Unlike bone marrow stem cells, which must be extracted from the bones in an operating room, peripheral blood stem cells are more easily obtained through apheresis, a process similar to regular blood donation, which collects the peripheral blood stem cells through a tube inserted in a vein. A critical step before the transplant involves finding a donor that is tissue matched to the recipient.
About one-third of patients who need a peripheral blood stem cell or bone marrow transplant for treatment of leukemia or another blood disease are able to secure a related donor. According to the National Marrow Donor Program, for the 70 percent who cannot find a donor within their family, most will be able to find an unrelated donor. Because the majority of transplant patients need cells from unrelated donors, its necessary to better understand the risks associated with transplants of unrelated donor cells.
Clinical trials on related donor transplants have demonstrated that peripheral blood stem cell transplants in patients with leukemia and other blood diseases result in better engraftment, lower relapse rates, and increased survival compared with transplants with bone marrow stem cells. However, those trials also found that peripheral blood stem cell transplants carry an increased risk of GVHD. Patients who survive early post-transplant may develop chronic GVHD, a disabling condition managed with long-term immunosuppressant therapy.
Many transplant centers are increasingly using peripheral blood stem cells as a source for adult stem cells because of their superiority in clinical trials that have directly compared outcomes between peripheral blood stem cells and bone marrow stem cells from related donors. However, there has not been a comparative study of the two transplant sources that has prospectively analyzed patient outcomes in unrelated donor transplants.
The study was funded by the National Heart, Lung and Blood Institute (U10HL069294), the National Cancer Institute and the National Marrow Donor Program.
About Moffitt Cancer Center Located in Tampa, Moffitt is one of only 41 National Cancer Institute-designated Comprehensive Cancer Centers, a distinction that recognizes Moffitts excellence in research, its contributions to clinical trials, prevention and cancer control. Since 1999, Moffitt has been listed in U.S. News & World Report as one of Americas Best Hospitals for cancer. With more than 4,200 employees, Moffitt has an economic impact on the state of nearly $2 billion. For more information, visit MOFFITT.org, and follow the Moffitt momentum on Facebook, twitter and YouTube.
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October 20th, 2012
Leading bone marrow transplant expert recommends significant change to current practice
By raymumme
Public release date: 17-Oct-2012 [ | E-mail | Share ]
Contact: Dean Forbes dforbes@fhcrc.org 206-667-2896 Fred Hutchinson Cancer Research Center
SEATTLE One of the world's leading bone marrow transplant experts is recommending a significant change to current transplant practice for patients who need marrow or adult stem cells from an unrelated donor to treat hematologic malignancies. Fred Appelbaum, M.D., director of the Clinical Research Division at Fred Hutchinson Cancer Research Center, asserts that bone marrow not circulating, peripheral blood, which is the current norm should be the source for unrelated donor adult stem cells for most patients who require a transplant. The reason: because there is less incidence of chronic graft-versus-host disease (GVHD), which can be a debilitating side effect of transplantation.
Appelbaum called for the change in an Oct. 18 editorial in The New England Journal of Medicine in response to a new study, published in the same issue, which compared survival rates and side effects of treating patients with hematopoietic adult stem cells derived from bone marrow versus circulating peripheral blood. The study found a higher incidence of chronic GVHD 53 percent when peripheral blood was the source of stem cells for transplant versus 41 percent when bone marrow is the source.
"For the majority of unrelated transplants following a standard high-dose preparative regimen, bone marrow should be used since survival is equivalent with the two sources but the incidence of chronic graft-versus-host disease, which can be a debilitating complication, is significantly less with marrow," Appelbaum wrote.
GVHD is a common side effect in people who receive cells from an unrelated donor. It occurs when the transplanted cells recognize the recipient's tissues as foreign and attack the tissues. This can cause a variety of problems, including skin rashes, liver problems and diarrhea. Chronic GVHD can develop any time between three months and three years after the transplant and can range from mild to serious in intensity.
Appelbaum said that stem cells derived from peripheral blood should only be used for the minority of patients in whom the benefits outweigh the risks. These include patients in need of rapid engraftment, such as those with life-threatening infections, or patients at high risk for graft rejection, such as those who receive reduced-intensity conditioning that does not include intensive chemotherapy.
For the past 10 years peripheral blood has been the norm as a source of matched related and matched unrelated adult stem cells for transplant because, despite the higher risk of GVHD, they are easier to harvest from the donor, they can be stimulated to grow in large numbers prior to harvesting, and they engraft, or set up shop, quickly inside the recipient's body.
The potential impact if such a practice change were widely implemented is large. Currently, about 75 percent of unrelated donor transplants are done using stem cells that are collected from the peripheral blood of donors. About 70 percent of all patients who undergo a life-saving transplant to treat blood cancers such as leukemia require an unrelated donor. Collecting adult stem cells from bone marrow is a more invasive process than collecting them from the bloodstream.
According to Appelbaum, about 5,500 unrelated donor transplants were performed in the United States last year. More than 20 million potential unrelated donors are typed and listed in registries in the Americas, Europe and Asia.
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Leading bone marrow transplant expert recommends significant change to current practice
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October 20th, 2012
Moffitt researcher says no survival advantage with peripheral blood stem cells versus bone marrow
By LizaAVILA
Public release date: 19-Oct-2012 [ | E-mail | Share ]
Contact: Kim Polacek kim.polacek@moffitt.org 813-745-7408 H. Lee Moffitt Cancer Center & Research Institute
Claudio Anasetti, M.D., chair of the Department of Blood & Marrow Transplant at Moffitt Cancer Center, and colleagues from 47 research sites in the Blood and Marrow Transplant Clinical Trials Network conducted a two-year clinical trial comparing two-year survival probabilities for patients transplanted with peripheral blood stem cells or bone marrow stem cells from unrelated donors. The goal was to determine whether graft source, peripheral blood stem cells or bone marrow, affects outcomes in unrelated donor transplants for patients with leukemia or other hematologic malignancies.
Fifty transplant centers in the United States and Canada participated in this phase III study, which randomized 278 patients to receive bone marrow and 273 patients to receive peripheral blood stem cells as the graft source for transplant. The results of the study are in the Oct. 18 issue of The New England Journal of Medicine.
According to the trial analyses, there were no observed differences in overall survival, relapse, non-relapse mortality, or acute graft-versus-host disease (GHVD) between the patients receiving peripheral blood stem cells or bone marrow stem cells from unrelated donors. GVHD is a serious and often deadly post-transplant complication that occurs when the newly transplanted donor cells attack the transplant recipient's body. While engraftment was faster in patients receiving peripheral blood stem cells, there was a higher incidence of overall chronic GVHD in these patients (53 percent) than in those transplanted with bone marrow stem cells (40 percent). Patients receiving transplants of peripheral blood stem cells from unrelated donors also had a higher incidence of chronic GVHD affecting multiple organs (46 percent) than patients who received bone marrow stem cells (31 percent).
"Although peripheral blood stem cells from related donors have demonstrated clinical benefits, our trial demonstrates that when these stem cells originate from unrelated donors, they are not superior to bone marrow stem cells in terms of patient survival, and they increase the risk for chronic GVHD," said Anasetti, lead study author. "More effective strategies to prevent GVHD are needed to improve outcomes for all patients receiving unrelated donor transplants."
Peripheral blood stem cells are stem cells originally found in the bone marrow that have been moved into the blood stream by a special regimen of drugs. Unlike bone marrow stem cells, which must be extracted from the bones in an operating room, peripheral blood stem cells are more easily obtained through apheresis, a process similar to regular blood donation, which collects the peripheral blood stem cells through a tube inserted in a vein. A critical step before the transplant involves finding a donor that is tissue matched to the recipient.
About one-third of patients who need a peripheral blood stem cell or bone marrow transplant for treatment of leukemia or another blood disease are able to secure a related donor. According to the National Marrow Donor Program, for the 70 percent who cannot find a donor within their family, most will be able to find an unrelated donor. Because the majority of transplant patients need cells from unrelated donors, it's necessary to better understand the risks associated with transplants of unrelated donor cells.
Clinical trials on related donor transplants have demonstrated that peripheral blood stem cell transplants in patients with leukemia and other blood diseases result in better engraftment, lower relapse rates, and increased survival compared with transplants with bone marrow stem cells. However, those trials also found that peripheral blood stem cell transplants carry an increased risk of GVHD. Patients who survive early post-transplant may develop chronic GVHD, a disabling condition managed with long-term immunosuppressant therapy.
Many transplant centers are increasingly using peripheral blood stem cells as a source for adult stem cells because of their superiority in clinical trials that have directly compared outcomes between peripheral blood stem cells and bone marrow stem cells from related donors. However, there has not been a comparative study of the two transplant sources that has prospectively analyzed patient outcomes in unrelated donor transplants.
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Moffitt researcher says no survival advantage with peripheral blood stem cells versus bone marrow
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October 20th, 2012
No survival advantage with peripheral blood stem cells versus bone marrow, study suggests
By Sykes24Tracey
ScienceDaily (Oct. 19, 2012) Claudio Anasetti, M.D., chair of the Department of Blood & Marrow Transplant at Moffitt Cancer Center, and colleagues from 47 research sites in the Blood and Marrow Transplant Clinical Trials Network conducted a two-year clinical trial comparing two-year survival probabilities for patients transplanted with peripheral blood stem cells or bone marrow stem cells from unrelated donors. The goal was to determine whether graft source, peripheral blood stem cells or bone marrow, affects outcomes in unrelated donor transplants for patients with leukemia or other hematologic malignancies.
Fifty transplant centers in the United States and Canada participated in this phase III study, which randomized 278 patients to receive bone marrow and 273 patients to receive peripheral blood stem cells as the graft source for transplant. The results of the study are in the Oct. 18 issue of The New England Journal of Medicine.
According to the trial analyses, there were no observed differences in overall survival, relapse, non-relapse mortality, or acute graft-versus-host disease (GHVD) between the patients receiving peripheral blood stem cells or bone marrow stem cells from unrelated donors. GVHD is a serious and often deadly post-transplant complication that occurs when the newly transplanted donor cells attack the transplant recipient's body. While engraftment was faster in patients receiving peripheral blood stem cells, there was a higher incidence of overall chronic GVHD in these patients (53 percent) than in those transplanted with bone marrow stem cells (40 percent). Patients receiving transplants of peripheral blood stem cells from unrelated donors also had a higher incidence of chronic GVHD affecting multiple organs (46 percent) than patients who received bone marrow stem cells (31 percent).
"Although peripheral blood stem cells from related donors have demonstrated clinical benefits, our trial demonstrates that when these stem cells originate from unrelated donors, they are not superior to bone marrow stem cells in terms of patient survival, and they increase the risk for chronic GVHD," said Anasetti, lead study author. "More effective strategies to prevent GVHD are needed to improve outcomes for all patients receiving unrelated donor transplants."
Peripheral blood stem cells are stem cells originally found in the bone marrow that have been moved into the blood stream by a special regimen of drugs. Unlike bone marrow stem cells, which must be extracted from the bones in an operating room, peripheral blood stem cells are more easily obtained through apheresis, a process similar to regular blood donation, which collects the peripheral blood stem cells through a tube inserted in a vein. A critical step before the transplant involves finding a donor that is tissue matched to the recipient.
About one-third of patients who need a peripheral blood stem cell or bone marrow transplant for treatment of leukemia or another blood disease are able to secure a related donor. According to the National Marrow Donor Program, for the 70 percent who cannot find a donor within their family, most will be able to find an unrelated donor. Because the majority of transplant patients need cells from unrelated donors, it's necessary to better understand the risks associated with transplants of unrelated donor cells.
Clinical trials on related donor transplants have demonstrated that peripheral blood stem cell transplants in patients with leukemia and other blood diseases result in better engraftment, lower relapse rates, and increased survival compared with transplants with bone marrow stem cells. However, those trials also found that peripheral blood stem cell transplants carry an increased risk of GVHD. Patients who survive early post-transplant may develop chronic GVHD, a disabling condition managed with long-term immunosuppressant therapy.
Many transplant centers are increasingly using peripheral blood stem cells as a source for adult stem cells because of their superiority in clinical trials that have directly compared outcomes between peripheral blood stem cells and bone marrow stem cells from related donors. However, there has not been a comparative study of the two transplant sources that has prospectively analyzed patient outcomes in unrelated donor transplants.
The study was funded by the National Heart, Lung and Blood Institute (U10HL069294), the National Cancer Institute and the National Marrow Donor Program.
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No survival advantage with peripheral blood stem cells versus bone marrow, study suggests
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October 20th, 2012
Parkinson's cells
By Sykes24Tracey
The nuclei of brain stem cells in some Parkinson's patients become misshapen with age. The discovery opens up new ways to target the disease.
Nubby nucleus: Brain cells from a deceased Parkinsons patient have deformed nuclei (right) compared with normal brain cells from an individual of a similar age. Merce Marti and Juan Carlos Izpisua Belmonte
Stem cells in the brains of some Parkinson's patients are increasingly damaged as they age, an effect that eventually diminishes their ability to replicate and differentiate into mature cell types. Researchers studied neural stem cells created from patients' own skin cells to identify the defects. The findings offer a new focus for therapeutics that target the cellular change.
The report, published today in Nature, takes advantage of the ability to model diseases in cell culture by turning patient's own cells first into so-called induced pluripotent stem cells and then into disease-relevant cell typesin this case, neural stem cells. The basis of these techniques was recognized with a Nobel Prize in medicine last week.
The authors studied cells taken from patients with a heritable form of Parkinson's that stems from mutations in a gene. After growing several generation of neural stem cells derived from patients with that mutation, they saw the cell nuclei start to develop abnormal shapes. Those abnormalities compromise the survival of the neural stem cells, says study coauthor Ignacio Sancho-Martinez of the Salk Institute for Biological Studies in La Jolla, California.
Today's study "brings to light a new avenue for trying to figure out the mechanism of Parkinson's," says Scott Noggle of the New York Stem Cell Foundation. It also provides a new set of therapeutic targets: "Drugs that target or modify the activity [of the gene] could be applicable to Parkinson's patients. This gives you a handle on what to start designing drug screens around."
The strange nuclei were also seen in patients who did not have a known genetic basis for Parkinson's disease. The authors suggest this indicates that dysfunctional neural stem cells could contribute to Parkinson's. While that conclusion is "highly speculative," says Ole Isacson, a neuroscientist at Harvard Medical School, the study demonstrates the "wealth of data and information that we now can gain from iPS cells."
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Parkinson's cells
Stem Cells Reveal Defect in Parkinson's Cells
By NEVAGiles23
The nuclei of brain stem cells in some Parkinson's patients become misshapen with age. The discovery opens up new ways to target the disease.
Nubby nucleus: Brain cells from a deceased Parkinsons patient have deformed nuclei (right) compared with normal brain cells from an individual of a similar age. Merce Marti and Juan Carlos Izpisua Belmonte
Stem cells in the brains of some Parkinson's patients are increasingly damaged as they age, an effect that eventually diminishes their ability to replicate and differentiate into mature cell types. Researchers studied neural stem cells created from patients' own skin cells to identify the defects. The findings offer a new focus for therapeutics that target the cellular change.
The report, published today in Nature, takes advantage of the ability to model diseases in cell culture by turning patient's own cells first into so-called induced pluripotent stem cells and then into disease-relevant cell typesin this case, neural stem cells. The basis of these techniques was recognized with a Nobel Prize in medicine last week.
The authors studied cells taken from patients with a heritable form of Parkinson's that stems from mutations in a gene. After growing several generation of neural stem cells derived from patients with that mutation, they saw the cell nuclei start to develop abnormal shapes. Those abnormalities compromise the survival of the neural stem cells, says study coauthor Ignacio Sancho-Martinez of the Salk Institute for Biological Studies in La Jolla, California.
Today's study "brings to light a new avenue for trying to figure out the mechanism of Parkinson's," says Scott Noggle of the New York Stem Cell Foundation. It also provides a new set of therapeutic targets: "Drugs that target or modify the activity [of the gene] could be applicable to Parkinson's patients. This gives you a handle on what to start designing drug screens around."
The strange nuclei were also seen in patients who did not have a known genetic basis for Parkinson's disease. The authors suggest this indicates that dysfunctional neural stem cells could contribute to Parkinson's. While that conclusion is "highly speculative," says Ole Isacson, a neuroscientist at Harvard Medical School, the study demonstrates the "wealth of data and information that we now can gain from iPS cells."
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Stem Cells Reveal Defect in Parkinson's Cells
Key player in Parkinson's disease triggered neuron loss pinpointed
By raymumme
London, October 20 (ANI): By reprogramming skin cells from Parkinson's patients with a known genetic mutation, researchers have identified the damage to neural stem cells as a powerful player in the disease.
The scientists from the Salk Institute for Biological Studies found that a common mutation to a gene that produce the enzyme LRRK2, which is responsible for both familial and sporadic cases of Parkinson's disease, deforms the membrane surrounding the nucleus of a neural stem cell.
Damaging the nuclear architecture leads to destruction of these powerful cells, as well as their decreased ability to spawn functional neurons, such as the ones that respond to dopamine.
The researchers checked their laboratory findings with brain samples from Parkinson's disease patients and found the same nuclear envelope impairment.
"This discovery helps explain how Parkinson's disease, which has been traditionally associated with loss of neurons that produce dopamine and subsequent motor impairment, could lead to locomotor dysfunction and other common non-motor manifestations, such as depression and anxiety," Juan Carlos Izpisua Belmonte, lead researcher of the study, said.
"Similarly, current clinical trials explore the possibility of neural stem cell transplantation to compensate for dopamine deficits. Our work provides the platform for similar trials by using patient-specific corrected cells. It identifies degeneration of the nucleus as a previously unknown player in Parkinson's," Belmonte said.
Although the researchers say that they don't yet know whether these nuclear aberrations cause Parkinson's disease or are a consequence of it, they say the discovery could offer clues about potential new therapeutic approaches.
For example, they were able to use targeted gene-editing technologies to correct the mutation in patient's nuclear stem cells. This genetic correction repaired the disorganization of the nuclear envelope, and improved overall survival and functioning of the neural stem cells.
They were also able to chemically inhibit damage to the nucleus, producing the same results seen with genetic correction.
"This opens the door for drug treatment of Parkinson's disease patients who have this genetic mutation," Belmonte said.
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Key player in Parkinson's disease triggered neuron loss pinpointed
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October 20th, 2012
Scientists Pinpoint Key Player in Parkinson's Disease Neuron Loss
By LizaAVILA
Stem cell study may help to unravel how a genetic mutation leads to Parkinson's symptoms
Newswise LA JOLLA, CA----By reprogramming skin cells from Parkinson's disease patients with a known genetic mutation, researchers at the Salk Institute for Biological Studies have identified damage to neural stem cells as a powerful player in the disease. The findings, reported online October 17th in Nature, may lead to new ways to diagnose and treat the disease.
The scientists found that a common mutation to a gene that produce the enzyme LRRK2, which is responsible for both familial and sporadic cases of Parkinson's disease, deforms the membrane surrounding the nucleus of a neural stem cell. Damaging the nuclear architecture leads to destruction of these powerful cells, as well as their decreased ability to spawn functional neurons, such as the ones that respond to dopamine.
The researchers checked their laboratory findings with brain samples from Parkinson's disease patients and found the same nuclear envelope impairment.
"This discovery helps explain how Parkinson's disease, which has been traditionally associated with loss of neurons that produce dopamine and subsequent motor impairment, could lead to locomotor dysfunction and other common non-motor manifestations, such as depression and anxiety," says Juan Carlos Izpisua Belmonte, a professor in Salk's Gene Expression Laboratory, who led the research team. "Similarly, current clinical trials explore the possibility of neural stem cell transplantation to compensate for dopamine deficits. Our work provides the platform for similar trials by using patient-specific corrected cells. It identifies degeneration of the nucleus as a previously unknown player in Parkinson's."
Although the researchers say that they don't yet know whether these nuclear aberrations cause Parkinson's disease or are a consequence of it, they say the discovery could offer clues about potential new therapeutic approaches.
For example, they were able to use targeted gene-editing technologies to correct the mutation in patient's nuclear stem cells. This genetic correction repaired the disorganization of the nuclear envelope, and improved overall survival and functioning of the neural stem cells.
They were also able to chemically inhibit damage to the nucleus, producing the same results seen with genetic correction. "This opens the door for drug treatment of Parkinson's disease patients who have this genetic mutation," says Belmonte.
The new finding may also help clinicians better diagnose this form of Parkinson's disease, he adds. "Due to the striking appearance in patient samples, nuclear deformation parameters could add to the pool of diagnostic features for Parkinson's disease," he says.
The research team, which included scientists from China, Spain, and the University of California, San Diego, and Scripps Research Institute, made their discoveries using human induced pluripotent stem cells (iPSCs). These cells are similar to natural stem cells, such as embryonic stem cells, except that they are derived from adult cells. While generation of these cells has raised expectations within the biomedical community due to their transplant potential - the idea that they could morph into tissue that needs to be replaced - they also provide exceptional research opportunities, says Belmonte.
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Scientists Pinpoint Key Player in Parkinson's Disease Neuron Loss
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October 20th, 2012
Key player in Parkinson's disease neuron loss pinpointed
By LizaAVILA
ScienceDaily (Oct. 19, 2012) By reprogramming skin cells from Parkinson's disease patients with a known genetic mutation, researchers at the Salk Institute for Biological Studies have identified damage to neural stem cells as a powerful player in the disease. The findings, reported online October 17th in Nature, may lead to new ways to diagnose and treat the disease.
The scientists found that a common mutation to a gene that produce the enzyme LRRK2, which is responsible for both familial and sporadic cases of Parkinson's disease, deforms the membrane surrounding the nucleus of a neural stem cell. Damaging the nuclear architecture leads to destruction of these powerful cells, as well as their decreased ability to spawn functional neurons, such as the ones that respond to dopamine.
The researchers checked their laboratory findings with brain samples from Parkinson's disease patients and found the same nuclear envelope impairment.
"This discovery helps explain how Parkinson's disease, which has been traditionally associated with loss of neurons that produce dopamine and subsequent motor impairment, could lead to locomotor dysfunction and other common non-motor manifestations, such as depression and anxiety," says Juan Carlos Izpisua Belmonte, a professor in Salk's Gene Expression Laboratory, who led the research team. "Similarly, current clinical trials explore the possibility of neural stem cell transplantation to compensate for dopamine deficits. Our work provides the platform for similar trials by using patient-specific corrected cells. It identifies degeneration of the nucleus as a previously unknown player in Parkinson's."
Although the researchers say that they don't yet know whether these nuclear aberrations cause Parkinson's disease or are a consequence of it, they say the discovery could offer clues about potential new therapeutic approaches.
For example, they were able to use targeted gene-editing technologies to correct the mutation in patient's nuclear stem cells. This genetic correction repaired the disorganization of the nuclear envelope, and improved overall survival and functioning of the neural stem cells.
They were also able to chemically inhibit damage to the nucleus, producing the same results seen with genetic correction. "This opens the door for drug treatment of Parkinson's disease patients who have this genetic mutation," says Belmonte.
The new finding may also help clinicians better diagnose this form of Parkinson's disease, he adds. "Due to the striking appearance in patient samples, nuclear deformation parameters could add to the pool of diagnostic features for Parkinson's disease," he says.
The research team, which included scientists from China, Spain, and the University of California, San Diego, and Scripps Research Institute, made their discoveries using human induced pluripotent stem cells (iPSCs). These cells are similar to natural stem cells, such as embryonic stem cells, except that they are derived from adult cells. While generation of these cells has raised expectations within the biomedical community due to their transplant potential -- the idea that they could morph into tissue that needs to be replaced -- they also provide exceptional research opportunities, says Belmonte.
"We can model disease using these cells in ways that are not possible using traditional research methods, such as established cell lines, primary cultures and animal models," he says.
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Key player in Parkinson's disease neuron loss pinpointed
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October 20th, 2012
Blood or Bone Marrow Better for Stem Cell Transplants?
By JoanneRUSSELL25
By Barbara Bronson Gray HealthDay Reporter
WEDNESDAY, Oct. 17 (HealthDay News) -- For people whose bone marrow has been destroyed by chemotherapy, radiation or disease, stem cell transplants offer a potential lifeline back to health.
But a key question has remained unanswered: Is it better to get the stem cells from a donor's blood or from bone marrow?
Now, a new study evaluates the pros and cons of harvesting stem cells from bone marrow rather than blood and suggests there are benefits to both approaches, but no survival differences between the two methods. The research was published Oct. 18 in the New England Journal of Medicine.
The study found that while peripheral blood stem cells may reduce the risk of graft failure, bone marrow may cut the chances of developing chronic graft-versus-host disease (GVHD), a complication that is frequently debilitating.
Over the past 10 years, 75 percent of stem cell transplants from unrelated adult donors have used peripheral blood stem cells rather than those harvested from bone marrow, according to study background information.
Some studies have suggested that using peripheral blood cells rather than bone marrow was associated with more severe GVHD. Other research has found that some people with transplants from peripheral blood stem cells had a lower relapse rate and improved survival.
Bone marrow offers the same chances of survival as does peripheral blood but tends to be associated with more severe side effects of treatment, explained study author Dr. Claudio Anasetti, a professor of medicine at the University of South Florida.
"With bone marrow, you have the same survival, but less long-term morbidity," Anasetti said.
Anasetti said the research shows that both approaches are acceptable, but "it's not a one-choice-for-all situation."
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Blood or Bone Marrow Better for Stem Cell Transplants?
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October 20th, 2012
ACT Announces Second Stargardt’s Disease Patient Treated with Higher Dosage of Embryonic Stem Cell-Derived Retinal …
By Dr. Matthew Watson
MARLBOROUGH, Mass.--(BUSINESS WIRE)--
Advanced Cell Technology, Inc. (ACT; OTCBB: ACTC), a leader in the field of regenerative medicine, today announced treatment of the fifth patient, the second in the second patient cohort, in its U.S. clinical trial for Stargardts Macular Dystrophy (SMD). The patient was injected with 100,000 human embryonic stem cell-derived retinal pigment epithelial (RPE) cells, as compared with the 50,000 cell dose used in the three patients of the first cohort. The patient successfully underwent the outpatient transplantation surgery yesterday, and is recovering uneventfully.
We continue to make steady progress in our ongoing clinical trials, commented Gary Rabin, chairman and CEO. We look forward to completing the third and final patient in this cohort in the U.S. clinical trial for SMD in the near future, which will mark the halfway point. We have now mapped out the series of patients to complete this cohort and the second cohort in the European trial, and have done the same in the US AMD trial, pending DSMB review. With all four trial centers in the US and both trial centers in the UK now trained and ready to treat patients, combined with the streamlined process in the European trial protocol, we anticipate an accelerated pace of generating patient data.
Initiated in July of last year, the Phase I/II trial is designed to determine the safety and tolerability of hESC-derived RPE cells following sub-retinal transplantation in patients with SMD at 12 months, the studys primary endpoint. It will involve a total of 12 patients, with cohorts of three patients each in an ascending dosage format. The company is concurrently conducting a second trial for SMD in Europe and another for dry age-related macular degeneration in the U.S.
We continue to be very encouraged with how this clinical trial is progressing, said Robert Lanza, M.D., chief scientific officer. We are confident of continued momentum in our other two trials, as well.
Further information about patient eligibility for ACTs SMD study and the concurrent studies in the U.S. and Europe (for dry age-related macular degeneration and SMD, respectively) are available at http://www.clinicaltrials.gov, with the following Identifiers: NCT01345006 (U.S. SMD), NCT01344993 (dry AMD), and NCT01469832 (E.U. SMD).
About Stargardts Disease
Stargardts disease or Stargardts Macular Dystrophy is a genetic disease that causes progressive vision loss, usually starting in children between 10 to 20 years of age. Eventually, blindness results from photoreceptor loss associated with degeneration in the pigmented layer of the retina, called the retinal pigment epithelium, which is the site of damage that the company believes the hESC-derived RPE may be able to target for repair after administration.
About Advanced Cell Technology, Inc.
Advanced Cell Technology, Inc. is a biotechnology company applying cellular technology in the field of regenerative medicine. For more information, visit http://www.advancedcell.com.
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October 20th, 2012
Journal Stem Cell Translational Medicine to Publish Article on FDA Communications and the Regulatory Pathway for Cell …
By LizaAVILA
WASHINGTON, DC--(Marketwire - Oct 17, 2012) - The Alliance for Regenerative Medicine (ARM), the international organization representing the interests of the regenerative medicine community, announced the publication today of an article on FDA communications to help companies developing cell-based therapies by clarifying the development pathway. The article, entitled "Communications with the FDA on the Development Pathway for a Cell-Based Therapy: Why, What, When, and How?" will be published in the journal Stem Cells Translational Medicine. It is co-authored by representatives from ARM, Janssen R&D, GE Healthcare and Life Technologies, with the lead author from the California Institute for Regenerative Medicine (CIRM).
"There are a number of ways cell-based therapy companies can communicate with FDA that will help them navigate the road from the bench to a regulatory submission," said Michael Werner, Executive Director of ARM. "We hope that our combined experience as co-authors, and our attempt to create a single source of guidance on the regulatory process, will help companies bring new cell-based therapies through clinical trials and the regulatory review process more quickly so they can reach patients faster," added Mr. Werner.
Lead author Ellen Feigal, MD, Senior Vice President for Research and Development at the California Institute for Regenerative Medicine (CIRM) commented, "Cell-based therapies represent a fundamentally new way to treat or cure disease, but developing a new therapy is costly, time consuming and fraught with uncertainty. Our paper takes a practical approach to clarifying the path to market."
"Communications with the FDA on the Development Pathway for a Cell-Based Therapy: Why, What, When, and How?" provides detailed information on options for communicating with the FDA at different stages; the official communications tied to each stage of development; and the most common reasons regulatory applications are delayed. The article can be accessed at: http://stemcellstm.alphamedpress.org/content/early/recent
About CIRM: CIRM was established in November 2004 with the passage of Proposition 71, the California Stem Cell Research and Cures Act. The statewide ballot measure, which provided $3 billion in funding for stem cell research at California universities and research institutions, was overwhelmingly approved by voters, and called for the establishment of an entity to make grants and provide loans for stem cell research, research facilities, and other vital research opportunities. A list of grants and loans awarded to date may be seen here: http://www.cirm.ca.gov/for-researchers/researchfunding.
About ARM: The Alliance for Regenerative Medicine is a Washington, DC-based multi-stakeholder advocacy organization that promotes legislative, regulatory and reimbursement initiatives necessary to facilitate access to life-giving advances in regenerative medicine. ARM also works to increase public understanding of the field and its potential to transform human healthcare, providing business development and investor outreach services to support the growth of its member companies and research organizations. Prior to the formation of ARM in 2009, there was no advocacy organization operating in Washington, DC to specifically represent the interests of the companies, research institutions, investors and patient groups that comprise the entire regenerative medicine community. Today ARM has more than 120 members and is the leading global advocacy organization in this field. In March 2012, ARM launched a sister organization in Europe -- the Alliance for Advanced Therapies. For more information go to http://www.alliancerm.org.
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October 20th, 2012