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.

“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."

“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."

Source:
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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 ...

Source:
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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

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.

See the rest here:
Researchers discover new blood vessel-generating cell with therapeutic potential

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.

More:
The Hutch suggests changing common marrow-donation procedure

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.

View post:
Peripheral Blood Stem Cell Transplants from Unrelated Donors Associated with Higher Rates of Chronic Graft-Versus-Host ...

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.

More here:
Leading bone marrow transplant expert recommends significant change to current practice

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.

View original post here:
Moffitt researcher says no survival advantage with peripheral blood stem cells versus bone marrow

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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Read more:
No survival advantage with peripheral blood stem cells versus bone marrow, study suggests

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."

See original here:
Parkinson's cells

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."

Read the rest here:
Stem Cells Reveal Defect in Parkinson's Cells

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.

See the original post:
Key player in Parkinson's disease triggered neuron loss pinpointed

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.

Go here to see the original:
Scientists Pinpoint Key Player in Parkinson's Disease Neuron Loss

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.

View post:
Key player in Parkinson's disease neuron loss pinpointed

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?

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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ACT Announces Second Stargardt’s Disease Patient Treated with Higher Dosage of Embryonic Stem Cell-Derived Retinal ...

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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Journal Stem Cell Translational Medicine to Publish Article on FDA Communications and the Regulatory Pathway for Cell ...

Manila, Philippines The Department of Health (DoH) warned yesterday against the proliferation of commercial establishments offering stem cell treatment for medical or aesthetic purposes.

DoH Secretary Enrique T. Ona said the use and effects of stem cell therapy is still being investigated by doctors while the Food and Drugs Administration (FDA) has yet to release the standards and regulations governing the safety of its use. Although this technology holds promise, stem cell therapy is not yet part of standard of care and is considered an investigative procedure for compassionate use. Applications of stem cells for the treatment of malignancies, blood disorders, degenerative diseases (such as Alzheimers Disease), metabolic diseases (e.g. diabetes), and immune cell therapy are still under clinical evaluation and study, Ona said in a statement.

While research has shown that adult stem cells and umbilical cord stem cells are the safest and most ethical, the public is warned that claims of preventive and curative bene ts of stem cell therapy are still on their investigative stage. Thus, the Department of Health (DoH) advises the public to be cautious with stem cell therapies being promoted in the media or through word of mouth, he added.

The DoH noted that there is an increasing demand for the use of stem cells in cancer therapy, endorgan diseases, and regenerative medicine.

But until a conclusive study has been completed, health of cials said the public is strongly advised to avoid stem cell therapies which use the following as sources for stem cells: Embryonic stem cells, aborted fetuses, and geneticallyaltered and animal fresh cells.

The DoH will soon issue guidelines for the use of stem cell therapy and the process of giving license to facilities offering such services, Ona noted. (Jenny F. Manongdo)

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DoH warns on stem cell

MANILA, Philippines - Dapatpa ring mag-ingat ang publiko laban sa nauusong stem cell therapy para sa medical at aesthetic purposes.

Sa isang advisory, sinabi ni Health Secretary Enrique Ona na ang stem cell therapy ay hindi pa rin bahagi ng standard of care at ikinukonsidera pa rin bilang investigative procedure for compassionate use.

Ayon kay Ona, ang aplikasyon ng stem cell bilang lunas sa malignancies, blood disorders, degenerative diseases tulad ngAlzheimers Disease, metabolic diseases tulad ngdiabetes, at immune cell therapy ay isinasailalim pa rin ng clinical evaluation at pag-aaral.

Pinapayuhan rin ni Ona ang publiko na iwasan ang stem cell therapies na gumagamit ng embryonic stem cells, aborted fetuses, genetically-altered at animal fresh cells, bilang sources ng stem cells.

Magpapalabas sila ng guidelines sa paggamit ng stem cell therapy at ng proseso para sa pagpapalisensiya ng mga nag-aalok ng naturang serbisyo.

Ang stem cell bilang therapy sa oncology, end-organ diseases at regenerative medicine ay in demand ngayon at maging sa Pilipinas ay naobserbahan na rin umano ang pagdami ng mga center na nag-aalok ng stem cell at aesthetic purposes.

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DOH nagbabala vs stem cell therapy

Washington, October 17 (ANI): Researchers believe they have discovered stem cells that play a decisive role in new blood vessel growth.

If the researchers at the University of Helsinki, Finland, learn to isolate and efficiently produce these stem cells found in blood vessel walls, the cells offer new opportunities in the treatment of cardiovascular diseases, cancer and many other diseases.

The growth of new blood vessels, also known as angiogenesis, is needed in adults when repairing damaged tissue or organs.

Unfortunately, malignant tumours are also capable of growing new blood vessels to receive oxygen and nutrients. In other words, the treatment of diseases would benefit from two types of methods - ones that help launch the process of angiogenesis and ones that make it possible to prevent the process.

Medications that prevent the growth of new blood vessels have already been introduced, but their effectiveness and long-term efficacy leave much to be desired.

For more than a decade, Adjunct Professor Petri Salven from the University of Helsinki has studied the mechanisms of angiogenesis to discover how blood vessel growth could be prevented or accelerated effectively.

He has examined the birth and origin of endothelial cells, which form the thin layer that lines the interior surface of blood vessels. Endothelial cells are necessary for new blood vessel growth. Where do these highly diversified cells come from? Can their production be prevented or increased?

For a long time, it was assumed that new cells in the blood vessel walls of an adult originate in the bone marrow. In an article published in the PNAS journal in 2008, Salven's research team showed that such stem cells were not found in bone marrow.

Now Salven is ready to reveal where these mysterious stem cells originate.

"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 able to produce tens of millions of new blood vessel wall cells," Salven said.

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New blood-vessel-generating cells with therapeutic potential discovered