UC Davis Nurtures Next Generation for Stem Cell Research – Video
By LizaAVILA
UC Davis Nurtures Next Generation for Stem Cell Research
High school students are learning about stem-cell medicine from researchers in the UC Davis Institute for Regenerative Cures. The students are involved in a rigorous yearlong teen biotech challenge that has placed them in one of the state #39;s most advanced stem cell labs, located at the UC Davis Health System in Sacramento.From:UCDavisViews:549 9ratingsTime:02:31More inEducation
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UC Davis Nurtures Next Generation for Stem Cell Research - Video
FAQ-6 of 19: How Soon Do Stem Cell Therapy Cells WORK in Stem Cell Therapy? – Video
By LizaAVILA
FAQ-6 of 19: How Soon Do Stem Cell Therapy Cells WORK in Stem Cell Therapy?
youtu.be When you choose stem cell therapy, and your board-certified doctor okays you for stem cell therapy, how soon can you expect effects? There #39;s no one-answer-fits-all, but this brief video gets across the gist of stem cell therapy. For more personalized information or more FAQ on stem cell therapy at the only licensed clinic in Thailand, visit StemCell-Asia.infoFrom:Lek WorkerViews:0 0ratingsTime:01:26More inScience Technology
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FAQ-6 of 19: How Soon Do Stem Cell Therapy Cells WORK in Stem Cell Therapy? - Video
FAQ-7 of 19: With Stem Cells, How Long Do Stem Cell Therapy Benefits Last? – Video
By LizaAVILA
FAQ-7 of 19: With Stem Cells, How Long Do Stem Cell Therapy Benefits Last?
youtu.be Briefly explaining how long the benefits of stem cell therapy last, when, why and for what conditions. For more detailed explanations, go to StemCell-Asia.info and find the #39;Apply Now #39; page, httpFrom:John PepperViews:0 0ratingsTime:01:19More inNonprofits Activism
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FAQ-7 of 19: With Stem Cells, How Long Do Stem Cell Therapy Benefits Last? - Video
FAQ-9 of 19: In Stem Cell Therapy Are There Contraindications for Stem Cells? – Video
By LizaAVILA
FAQ-9 of 19: In Stem Cell Therapy Are There Contraindications for Stem Cells?
youtu.be Yes, there ARE some times and some ways that stem cells should NOT be used, so this short video outlines them for you. For more personalized information, visit bit.ly or StemCell-Asia.info now.From:Karridine1Views:0 0ratingsTime:00:54More inPeople Blogs
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FAQ-9 of 19: In Stem Cell Therapy Are There Contraindications for Stem Cells? - Video
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
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
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
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.
Claim of first human stem cell trial unravels
By LizaAVILA
It has been a crazy week for stem cell research. After the high of a Nobel prize for Japan's Shinya Yamanaka, the pioneer of cellular reprogramming, events took an alarming and surreal turn when a little-known compatriot Hisashi Moriguchi claimed to have already run a clinical trial in which similarly reprogrammed cells were injected into people.
But Moriguchi's claims quickly unravelled. "I have not found a single person to say anything concrete indicating that this has really happened," says Paul Knoepfler, a stem cell researcher at the University of California, Davis, who tracked the unfolding story on his blog.
In a poster presented at a meeting of the New York Stem Cell Foundation, Moriguchi who claimed to work at Harvard Medical School and the University of Tokyo described results from a trial in which cardiac muscle cells were grown from induced pluripotent stem (iPS) cells, and transplanted into six US patients with severe heart failure.
The Yomiuri Shimbun newspaper Japan's biggest splashed the story, based on an interview with Moriguchi, who claimed he had received ethical approval from Harvard Medical School's Institutional Review Board (IRB).
This was surprising, given the safety concerns that surround iPS cells adult cells that have been reprogrammed to an embryonic state. Support for the claim quickly disintegrated: within hours, Harvard released a statement noting that Moriguchi had no current affiliation with the university, nor any ethical approval to run a clinical trial.
Moriguchi's poster describing the clinical trial was taken down after the New York Stem Cell Foundation learned of Harvard's statement but a summary was published on Knoepfler's blog. This suggested an improvement of 41.5 per cent in "ejection fraction" a measure of heart output in patients whose hearts were injected with iPS-derived cells, compared to 4.1 per cent in a placebo group.
That would have been an astonishing claim, says Michael Laflamme at the University of Washington in Seattle, who is working to develop cell therapies for heart attack: "I'm not aware of any clinical trial that reported anything of this magnitude."
Indeed, similar studies involving adult stem cells have typically found improvements of less than 5 per cent (European Journal of Hearth Failure, doi.org/crq5k6).
Moriguchi did not respond to emails from New Scientist. But on Saturday he admitted to reporters that for five of the patients he was actually describing "planned" procedures. Still, Moriguchi maintained that he had transplanted cells into one patient at an unidentified hospital in Boston.
New Scientist's enquiries raise further questions about Moriguchi's work. In papers published earlier this year, he described experiments on freezing human ovarian tissue (Scientific Reports, doi.org/jht), and a remarkable claim to be able to eliminate liver tumour cells using a reprogramming technique (Scientific Reports, doi.org/jhv). Both gave Harvard and University of Tokyo affiliations, and claimed ethical approval from each institution.
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Claim of first human stem cell trial unravels
Director of Operations for GeneCell International, Jose Cirino, Accentuates the Importance Surrounding the Minority …
By LizaAVILA
Miami, FL (PRWEB) October 15, 2012
Leveraging more than a decade of experience in the biotech industry and a founding member of GeneCell International, Jose Cirino, Director of Operations, is an industry expert for expanding awareness in the field of adult stem cells worldwide. Cirino plays a pivotal role in GeneCell Internationals success, managing all operational aspects of a company thats at the forefront of the biotech industry. While providing leadership and direction for the company, he is responsible for all strategic planning to help advance GeneCells mission and objectives, as well as the expansion of product, service and development at the national and international levels. Currently, Cirino is not only working on the expansion and awareness of cord blood services, but on the implementation of other adult stem cells sources, such as umbilical cord tissue, dental pulp, and adipose (fat) tissue into other countries. He has presentation talks in the advantage of adult cord blood banking to health and biotech industry organizations, conferences and small group meetings (both English and Spanish). Cirino was a key player in the expansion of GeneCell International into Miami, Florida, as the first and only stem cell laboratory of its kind in the South Floridian market and the gateway to international countries.
Through his field of work, Cirinos aspiration and passion is being able to assist individuals in potentially saving their life when a debilitating immune deficiency or disorder arises. Though, Cirino continuously asks himself, Why isn't everyone banking these cells?
His best assumption is that people are not informed about stem cell banking and what is most disheartening, some have never even heard of it. Most people are not aware they have stem cells in their body. Others believe that stem cells only come from only human embryos since this is whats mainly discussed in politics and the news today. May this be the reason they are choosing to have no part in it and ignore it? If so, this is not the case, these cells are found in adults and there are not controversial, moral, ethical or have any political issues surrounding them. The amazing thing about these cells, aside from their potential to treat a variety of different diseases, is that for the most part they can be harvested from the individual through relatively minimally invasive procedures and can be cryogenically frozen (at a temperature of -321 F (-196 C)) and stored for decades until a disease manifests itself or the needed for cell-based therapies arises," said Cirino.
Due to this lack of awareness, there is a massive shortage of stem cell units stored for future treatments. This shortage, or lack of availability, is mostly affecting patients of African, Asian, Hispanic and Native American Indian descent. I, being a minority member of this group, am very concerned by this shortage. Since patients who need a transplant are more likely to find a match within their own genetic background, Cirino adds it is important that the pool of donors reflects the overall community.
A persons blood stem cell type is inherited, which means a patient is more likely to find a matched donor from within their own ethnic group, more than half of cord blood donations and privately banked cord blood in the United States are from Caucasians while minorities remain underrepresented, significantly. By increasing awareness of the advantages of cord blood among minorities, there is a potential for increased access to therapies for more people.
Umbilical cord blood preservation is a process by which blood is collected from the umbilical cord of a newborn baby and is stored cryogenically in a specially-designated bank. According to the National Marrow Donor Program, cord blood contains cells that can be transfused to a patient to treat various diseases, including lymphoma and leukemia. Currently, there are approximately 80 treatable diseases and the list of illnesses continues to grow. Cord blood is rich in stem cells and because certain immune cells found in the cord blood are not mature, there is less risk for the recipients immune system to reject these cells. Cord blood can be used to treat the child from whom the blood was collected as well as some first-degree relatives who are a close genetic match, such as immediate family members. Additionally, patients can get the treatment in about three weeks - as opposed to six to eight for bone marrow from an adult donor.
Prior to founding GeneCell, Cirino served as the President of the International Division to a cord blood laboratory in Boston, Massachusetts, where he was responsible for identifying, evaluating and selecting international representatives for affiliate programs to expand the services internationally. In doing so, he coordinated laboratory development protocol license agreements and implemented these programs throughout various international countries. After the expansion into other countries, Cirino would manage the company owned offices as well as provide support to the affiliate offices, from Mexico and South America, to the UK and the Middle East. He also represented the company at international health and biotech industry conferences, implemented new sales tools and processes for all international divisions of the company, and oversaw all accounting tasks as a method of monitoring its sales projections. Cirino joined the company as the Accounting Manager, where he was responsible for all aspects of U.S. and international accounting functions. He is a seasoned accounting professional, holding various accounting positions within large companies such as Sir Speedy Printing Centers of Boston and Harvard Institute for International Development. He has served as a member within various industry organizations including the International Cord Blood Society, and New England Fertility Society, as well as participated in the International Federation of Gynecology and Obstetrics (FIGO), The Mexican Federation of Ultrasounds, The World Cord Blood Congress, and Stem Cells USA-Regenerative Medicine conferences.
In addition to cord blood, Cirinos implementation projects of other adult stem cells sources, in the U.S. and other countries, include Cord Tissue Segment, Dental Pulp and Adipose Tissue:
About Cord Tissue Segment - A gelatinous substance, which functions as the primary connective tissue of the umbilical cord and is referred to as Whartons Jelly. This segment contains an important amount of Mesenchymal stem cells. These cells are an excellent candidate for regenerative medicine and tissue engineering applications. Mesenchymal stem cells have shown great promise in the potential treatment of diseases such as heart attack, Parkinsons disease, Alzheimers disease, type I diabetes, assist in bone and dental regeneration and expedite wound healing. In the past, the umbilical cord has been viewed as medical waste and discarded, resulting in the loss of this potential life-saving resource. By storing the stem cells extracted from your umbilical cord tissue segment along with your babys cord blood, youll have access to a wider variety of stem cells as new scientific discoveries are made.
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Director of Operations for GeneCell International, Jose Cirino, Accentuates the Importance Surrounding the Minority ...
Anmore boy needs bone marrow transplant
By LizaAVILA
METRO VANCOUVER -- It started with a routine blood test after a bout of pneumonia.
But since finding two rare bone marrow conditions, the family of 11-year-old Jonathan Barnes has been campaigning to shore up the list of potential marrow donors, and to keep local blood banks stocked.
Without finding a bone marrow donor who will match Jonathan's criteria, the Anmore youngster will likely end up with leukemia. Despite the frightening prospect, Jonathan and his family are meeting the challenge with poise.
"He knows he has a condition called myelodysplasia. He knows that he needs to have a bone marrow transplant," said Mariam Barnes, Jonathan's mother.
"He knows he will get cancer if he doesn't have the transplant. But in the way that children are always so wonderfully unique, he's not fazed by that."
While finding an exact match is extremely difficult, getting on the donor list is easier than most people might think, Barnes said.
"A lot of people don't do it because they think it's involving needles but the beginning step is just a mouth swab. It comes to you in the post and you post it back, and they put you on the register," she said.
"What we didn't know and what I don't think many people know, is that they're desperately short of young male donors . There's 19 million people on the transplant register across the world, but only 10 per cent of those are the groups that they need - ethnically diverse young men."
It would be easier to find a match for the family if they could use donated stem cells from umbilical chords, but that procedure won't be available in Canada until next year and the Barnes don't have that much time, she said.
"We're just praying and hoping that someone, somewhere in the world, will put forward a match that will fit with Jonathan."
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Anmore boy needs bone marrow transplant
Springettsbury toddler needs a bone marrow transplant
By LizaAVILA
Joey Duffy of Springettsbury Township is looking for a match.
Two-year-old Joey Duffy yawns as his mother, Maura, vents his stomach via a feeding tube after he was fed at their Springettsbury Township home on Friday. Joey, who has previously had esophageal stricture, has been in and out of the hospital all summer and is in need of a bone marrow transplant. (DAILY RECORD/SUNDAY NEWS - CHRIS DUNN)
Two-year-old Joey Duffy played with his "Sesame Street" doll Ernie, watched the television show "Yo Gabba Gabba" and occasionally called out "mamma" while his parents talked about a bone marrow transplant he needs.
The toddler was diagnosed about five weeks ago with Myelodysplastic Syndromes, also known as MDS, a blood and bone marrow disorder. It's the same ailment that Robin Roberts of "Good Morning America" is receiving treatment for currently.
The disease can progress to leukemia, parents Tom and Maura Duffy said at their Springettsbury Township home. They are lucky that doctors at Johns Hopkins in Baltimore caught the condition when they did for their youngest son.
"We're ahead of the game," Maura Duffy said. "We caught this very early."
The only cure is a bone marrow transplant, and the parents as well as their two older sons, 5-year-old Tommy and 4-year-old Mick, have already submitted a cheek swab to see if they will be a match for Joey. His brothers are the best chance, Maura Duffy said.
Meanwhile, the family is organizing an Oct. 21 donor drive at their church, Saint Andrews Episcopal Church in Spring Garden Township. The idea came about as family and friends asked how they could get tested to see if they are a match, Tom Duffy said.
The process only takes about 15 minutes, said Sarah Brooks Horan, an account executive for the National Marrow Donor Program, also known as "Be The Match." A cotton swab is used to swab the cheek.
These days, donating stem cells can be as simple as giving blood, Horan said.
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Springettsbury toddler needs a bone marrow transplant
Nobel Prize for Physiology or Medicine Goes to Stem Cell Researchers
By LizaAVILA
The Nobel Prize for Physiology or Medicine was announced on Monday. The award this year went to Sir John B. Gurdon and Dr. Shinya Yamanaka. The two men were awarded the Nobel Prize jointly, for their individual work in cloning and stem cell research.
Monday's recognition marked the awarding of the first Nobel Prize for 2012. The rest of the Nobel Prize recipients will be announced throughout the next two weeks.
Here is some of the key information regarding Gurdon and Yamanaka's work and Monday's Nobel Prize announcement.
* Yamanaka and Gurdon did not work together or present shared research, even though they both concentrate their studies on a similar area of research.
* Gurdon is actually being honored for work he did back in 1962. According to a New York Times report, he was the first person to clone an animal, a frog, opening the door to further research into stem cells and cloning.
* Gurdon was able to produce live tadpoles from the adult cells of a frog, by removing the nucleus of a frog's egg and putting the adult cells in its place.
* This "reprogramming" by Gurdon laid the groundwork for Yamanaka's work four decades later. Yamanaka's work, which dates back only six years, to 2006, focused on the mechanisms behind Gurdon's results.
* According to the Los Angeles Times, Yamanaka was sharply criticized at first for his own work, in which he sought to discover how cells are able to reprogram themselves the way that Gurdon's work first suggested that they could.
* Ultimately, Yamanaka was able to isolate just four cells that were needed in order to be able to reprogram other cells back to an embryonic state, allowing them to be manipulated into developing into any particular kind of cell that was needed. These cells have now been dubbed "induced pluripotent stem cells," or iPS cells, according to reports by CNN and other media outlets.
* Scientists are reproducing Yamanaka's technique in their own labs to be able to replicate disease cells, like those of Alzheimer's or Parkinson's, in order to study them and even to test the effects of potential new treatments.
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Nobel Prize for Physiology or Medicine Goes to Stem Cell Researchers
Dr. Eva Feldman, Principal Investigator, Updates Interim Data On Completed Neuralstem ALS Phase I Trial
By LizaAVILA
ROCKVILLE, Md., Oct. 9, 2012 /PRNewswire/ --Neuralstem, Inc. (NYSE Amex: CUR) announced that Eva Feldman, MD, PhD, principal investigator of the Phase I trial to test Neuralstem's NSI-566 spinal cord stem cells in the treatment of amyotrophic lateral sclerosis (ALS or Lou Gehrig's disease), updated data on the trial at the American Neurological Association annual meeting in Boston, MA, yesterday. (http://www.aneuroa.org/i4a/pages/index.cfm?pageid=3311). Dr. Feldman, who is President of the American Neurological Association, presented interim results on all 18 procedures in 15 patients, including the last three patients from earlier cohorts who received second procedures. The trial will conclude six months after the last patient was treated, which was in August.
(Logo: http://photos.prnewswire.com/prnh/20061221/DCTH007LOGO )
"This has been a very successful trial so far," said Dr. Feldman, Director of the A. Alfred Taubman Medical Research Institute and Director of Research of the ALS Clinic at the University of Michigan Health System. "With the transplantation of these neural stem cells, we are exploring a paradigm shift in the treatment of ALS. We have demonstrated that intraspinal transplantation is feasible and well-tolerated. Although this phase of the trial was not powered to demonstrate efficacy, we appear to have interrupted the progression of the disease in one subgroup of patients. We are anxious to move to future trial phases to examine therapeutic efficacy." Dr. Feldman is an unpaid consultant to Neuralstem.
"The purpose of this trial was to assess the safety of both the intraspinal transplantation procedure, the first in the world, and of the cells themselves, " said Karl Johe, PhD, Chairman of the Board and Chief Scientific Officer of Neuralstem, Inc. "All assessments show both to be safe. Additionally, we believe we are seeing evidence of a treatment effect in some patients over a sustained period of time. We need now to move forward to more advanced, larger trials to increase the dosage and more effectively look at possible efficacy."
About the Trial
The Phase I trial to assess the safety of Neuralstem's NSI-566 spinal cord neural stem cells and intraspinal transplantation method in ALS patients commenced in January 2010, and consisted of 18 treatments in 15 patients. The trial was designed to follow a risk escalation paradigm. The first 12 patients were each transplanted in the lumbar (lower back) region of the spine, beginning with non-ambulatory and advancing to ambulatory cohorts.
The trial then advanced to transplantation in the cervical (upper back) region of the spine. The first cohort of three was treated in the cervical region only. In an amendment to the trial design, The Food and Drug Administration (FDA) approved the return of previously-treated patients to this cohort. Consequently, the last cohort of three patients received injections in the cervical region in addition to the lumbar injections they had received earlier. All injections delivered 100,000 cells, for a dosing range of up to 1.5 million cells. The last patient was treated in August, 2012. The entire trial concludes six months after the final surgery.
About Neuralstem
Neuralstem's patented technology enables the ability to produce neural stem cells of the human brain and spinal cord in commercial quantities, and the ability to control the differentiation of these cells constitutively into mature, physiologically relevant human neurons and glia. Neuralstem has recently treated the last patient in an FDA-approved Phase I safety clinical trial for amyotrophic lateral sclerosis (ALS), often referred to as Lou Gehrig's disease, and has been awarded orphan status designation by the FDA.
In addition to ALS, the company is also targeting major central nervous system conditions with its NSI-566 cell therapy platform, including spinal cord injury, ischemic stroke and glioblastoma (brain cancer). The company has submitted an IND (Investigational New Drug) application to the FDA for a Phase I safety trial in spinal cord injury.
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Dr. Eva Feldman, Principal Investigator, Updates Interim Data On Completed Neuralstem ALS Phase I Trial
Electric fish at NMSU activate stem cells for regeneration
By LizaAVILA
Imagine the horror of a soldier losing a limb on the battlefield, or a loved one having a body part amputated due to diabetes. But, what if they could restore their limbs by activating their stem cells?
New Mexico State University biologist Graciela Unguez and a team of researchers found that electric fish, a vertebrate animal just like humans, can regenerate their tails following amputation after activating their stem cells. The findings were published in the May 2012 edition of the scientific journal, PLOS One.
"What's surprising is that as humans, we're one of the few animal species that do not readily regenerate limbs, organs or most tissues," Unguez said. "So, there's a lot of interest in how these fish do it, and what's preventing us from doing it."
Regeneration is the process of restoring lost cells, tissues or organs. According to Unguez, most animals have the ability to regenerate eyes and tails and some animals may be able to regenerate up to half of their bodies.
The researchers discovered that when they cut off up to one third of an electric fish's tail, including the spinal cord, vertebrae, muscles, skin, connective tissues and nerves, the fish would regenerate it. Unguez said the more tissue cut off, the longer the regeneration takes, but for the purpose of her study, it takes about three weeks.
"It's really exciting to us because, here's an example of an animal that can regenerate a lot of tissue types that are also found in humans," Unguez said. "So it puts into question this previous idea that those animals that can regenerate losses of many tissues do it because they do it differently than humans."
Unguez has used the electric fish as a model system to investigate the role that the nervous system plays in the fate of electrically excitable cells like muscle cells for 15 years. She noted that for many years, scientists have thought that highly regenerative animals use a mechanism of regeneration that does not involve stem cells, and this stem cell-based mechanism is well known in humans. In contrast, the stem cell-independent mechanism found in highly regenerative animals is not normally active in humans.
Unguez explained that stem cells are a small population of cells that do not mature and stay with us throughout our life, and then when called upon, they reenter the cell cycle to become muscle cells, neurons, skill cells and such.
But, what Unguez and her collaborators discovered was the opposite. The electric fish actually activated its own muscle and electric organ stem cells to regenerate. She said the adult fish regenerated unendingly with the activation of their stem cells.
"It does not negate other mechanisms, but it definitely showed that it was largely due to an activation of stem cells, just like humans have," Unguez said. "So maybe it's not as far apart, maybe some of the mechanisms involved or the events that need to be activated are more closely related than we thought."
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Electric fish at NMSU activate stem cells for regeneration
Charter Medical Launches New EXP-Pak(TM) Cell Expansion Containers for Cellular Therapy Applications
By LizaAVILA
MANCHESTER, Conn., Oct. 9, 2012 (GLOBE NEWSWIRE) -- Charter Medical, Ltd., a division of Lydall, Inc., (LDL) announced today that it has recently launched the new EXP-Pak(TM) cell expansion containers intended for the expansion and culture of non-adherent cells. The launch of this exciting new product family allows Charter Medical to provide enabling technology critical to the rapidly growing cellular therapy market. The family of closed-system cell expansion containers offers a broad size range from 500mL to 5L and end-user validated cell expansion rates and recovery.
Joe Petrosky, Vice President of Global Marketing and Sales for Charter Medical, stated, "We are excited with the launch of the EXP-Pak(TM) cell expansion product family. The EXP-Pak(TM) containers complement our closed-system solution approach and play a key role in supporting the development of new cellular therapies."
Dale Barnhart, President and CEO of Lydall, stated, "I am pleased with the launch of this product family for cellular therapy which represents a strategic growth opportunity. It further demonstrates our commitment to being the global supplier of choice as we grow our presence in this emerging segment."
About Lydall, Inc.
Lydall, Inc. is a New York Stock Exchange listed company, headquartered in Manchester, Connecticut. The Company, with operations in the U.S., France, and Germany and offices in Europe and Asia, focuses on specialty engineered products for the thermal/acoustical and filtration/separation markets. Charter Medical, Ltd., a Lydall subsidiary, is a vital fluids management company focused on providing products to separate, contain and transport vital fluids in the blood and cell therapy market and the biotech and pharmaceutical industries. Lydall(R) is a registered trademark of Lydall, Inc. in the U.S. and other countries. All product names are trademarks of Lydall, Inc. or Charter Medical, Ltd.
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Charter Medical Launches New EXP-Pak(TM) Cell Expansion Containers for Cellular Therapy Applications
Nobel Prize In Medicine Awarded To Stem Cell Researchers
By LizaAVILA
The Nobel Prize in Medicine or Physiology for 2012 was awarded jointly to British scientist John B. Gurdon and Japanese scientist Shinya Yamanaka for their work in stem cell research, the Karolinska Institute in Stockholm announced Monday.
The announcement opens the prestigious award season for this year while the speculation over literature and peace prizes is rife.
"These groundbreaking discoveries have completely changed our view of the development and specialization of cells," the Nobel Assembly at Sweden's Karolinska Institute said in a statement on its website.
We now understand that the mature cell does not have to be confined forever to its specialized state. Textbooks have been rewritten and new research fields have been established. By reprogramming human cells, scientists have created new opportunities to study diseases and develop methods for diagnosis and therapy," the statement said.
Gurdon discovered in 1962 that the specialization of cells is reversible. Yamanaka discovered more than 40 years later in 2006 how the intact mature cells in mice could be reprogrammed to become immature stem cells. These groundbreaking discoveries have completely changed our view of the development and cellular specialization, the institute has said.
Gurdon was born in 1933 in Dippenhall, the U.K, and received his Doctorate from the University of Oxford in 1960 and was a postdoctoral fellow at the California Institute of Technology. Gurdon is currently at the Gurdon Institute in Cambridge.
Yamanaka was born in Osaka, Japan, in 1962 and received his MD in 1987 at Kobe University and was trained as an orthopedic surgeon. Yamanaka obtained his PhD at Osaka University in 1993. Yamanaka is currently Professor at Kyoto University and is also affiliated to the Gladstone Institute.
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Nobel Prize In Medicine Awarded To Stem Cell Researchers
UK, Japan scientists win Nobel for stem cell breakthroughs
By LizaAVILA
STOCKHOLM - Scientists from Britain and Japan shared a Nobel Prize on Monday for the discovery that adult cells can be transformed back into embryo-like stem cells that may one day regrow tissue in damaged brains, hearts or other organs.
John Gurdon, 79, of the Gurdon Institute in Cambridge, Britain and Shinya Yamanaka, 50, of Kyoto University in Japan, discovered ways to create tissue that would act like embryonic cells, without the need to harvest embryos.
They share the $1.2 million Nobel Prize for Medicine, for work Gurdon began 50 years ago and Yamanaka capped with a 2006 experiment that transformed the field of "regenerative medicine" - the field of curing disease by regrowing healthy tissue.
"These groundbreaking discoveries have completely changed our view of the development and specialization of cells," the Nobel Assembly at Stockholm's Karolinska Institute said.
All of the body's tissue starts as stem cells, before developing into skin, blood, nerves, muscle and bone. The big hope for stem cells is that they can be used to replace damaged tissue in everything from spinal cord injuries to Parkinson's disease.
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UK, Japan scientists win Nobel for stem cell breakthroughs
Nobel Winner’s Stem Cells to Be Tested in Eye Disease Next Year
By LizaAVILA
Thomas Perlmann of Karolinska Institute presents Sir John B. Gurdon of Britain and Shinya Yamanaka of Japan as winners of the 2012 Nobel Prize in medicine or physiology. The prize committee at Stockholms Karonlinska institute said the discovery has revolutionized our understanding of how cells and organisms develop. Photograph by Bertil Enevag Ericson/Scanpix/AP Photo
Stem cells derived from a mouses skin won Shinya Yamanaka the Nobel Prize yesterday. Now researchers in Japan are seeking to use his pioneering technology for an even greater prize: restoring sight.
Scientists at the Riken Center for Developmental Biology in Kobe plan to use so-called induced pluripotent stem cells in a trial among patients with macular degeneration, a disease in which the retina becomes damaged, resulting in blindness, Yamanaka told reporters in San Francisco yesterday.
Companies including Marlborough, Massachusetts-based Advanced Cell Technology Inc. (ACTC) are already testing stem cells derived from human embryos. The Japanese study will be the first to use a technology that mimics the power of embryonic cells while avoiding the ethical controversy that accompanies them.
The work in that area looks very encouraging, John B. Gurdon, 79, a professor at the University of Cambridge who shared the Nobel with Yamanaka yesterday, said in an interview in London.
Yamanaka and Gurdon shared the 8 million Swedish kronor ($1.2 million) award for experiments 50 years apart that showed that mature cells retain in latent form all the DNA they had as immature stem cells, and that they can be returned to that potent state, offering the potential for a new generation of therapies against hard-to-treat diseases such as macular degeneration.
In a study published in 1962, Gurdon took a cell from a tadpoles gut, extracted the nucleus, and inserted it into the egg cell of an adult frog whose own nucleus had been removed. That reprogrammed egg cell developed into a tadpole with the genetic characteristics of the original tadpole, and subsequent trials yielded adult frogs.
Yamanaka, 50, a professor at Kyoto University, built on Gurdons work by adding four genes to a mouse skin cell, returning it to its immature state as a stem cell with the potential to become any cell in the body. He dubbed them induced pluripotent stem cells.
The technology may lead to new treatments against diseases such as Parkinsons by providing replacement cells.
The implications for regenerative medicine are obvious, R. Sanders Williams, president of the Gladstone Institutes in San Francisco, where Yamanaka is a senior investigator, said in a telephone interview. Skin cells can be converted to any other cell you want -- skin to brain or skin to heart, skin to insulin-producing.
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Nobel Winner’s Stem Cells to Be Tested in Eye Disease Next Year
Stem Cell Researchers Share Nobel Medicine Prize
By LizaAVILA
British researcher John Gurdon and Shinya Yamanaka from Japan have shared the Nobel prize for medicine or physiology.
The two pioneers of stem cell research were awarded the prize for transforming specialised cells into stem cells, which can become any other type of cell in the body.
John Gurdon discovered in 1962 that the specialisation of cells is reversible. In a classic experiment, he replaced the immature cell nucleus in an egg cell of a frog with the nucleus from a mature intestinal cell. This modified egg cell developed into a normal tadpole. The DNA of the mature cell still had all the information needed to develop all cells in the frog.
Shinya Yamanaka discovered more than 40 years later, in 2006, how intact mature cells in mice could be reprogrammed to become immature stem cells. Surprisingly, by introducing only a few genes, he could reprogram mature cells to become pluripotent stem cells, i.e. immature cells that are able to develop into all types of cells in the body.
These groundbreaking discoveries have completely changed our view of the development and cellular specialisation.
By reprogramming human cells, scientists have created new opportunities to study diseases and develop methods for diagnosis and therapy.
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Stem Cell Researchers Share Nobel Medicine Prize