Stem Cell Scientists Awarded Nobel Prize in Physiology and Medicine
Kyodo / Reuters
Kyoto University Professor Shinya Yamanaka (left) and John Gurdon of the Gurdon Institute in Cambridge, England, at a symposium on induced pluripotent stem cells in Tokyo in April 2008
In a testament to the revolutionary potential of the field of regenerative medicine, in which scientists are able to create and replace any cells that are at fault in disease, the Nobel Prize committee on Monday awarded the 2012 Nobel in Physiology or Medicine to two researchers whose discoveries have made such cellular alchemy possible.
The prize went to John B. Gurdon of the University of Cambridge in England, who was among the first to clone an animal, a frog, in 1962, and to Shinya Yamanaka of Kyoto University in Japan who in 2006 discovered the four genes necessary to reprogram an adult cell back to an embryonic state.
Sir John Gurdon, who is now a professor at an institute that bears his name, earned the ridicule of many colleagues back in the 1960s when he set out on a series of experiments to show that the development of cells could be reversed. At the time, biologists knew that all cells in an embryo had the potential to become any cell in the body, but they believed that once a developmental path was set for each cell toward becoming part of the brain, or a nerve or muscle it could not be returned to its embryonic state. The thinking was that as a cell developed, it would either shed or silence the genes it no longer used, so that it would be impossible for a cell from an adult animal, for example, to return to its embryonic state and make other cells.
(MORE: Stem Cell Miracle? New Therapies May Cure Chronic Conditions Like Alzheimers)
Working with frogs, Gurdon proved his critics wrong, showing that some reprogramming could occur. Gurdon took the DNA from a mature frogs gut cell and inserted it into an egg cell. The resulting egg, when fertilized, developed into a normal tadpole, a strong indication that the genes of the gut cell were amenable to reprogramming; they had the ability to function as more than just an intestinal cell, and could give rise to any of the cells needed to create an entirely new frog.
Just as Gurdon was facing his critics in England, a young boy was born in Osaka, Japan, who would eventually take Gurdons finding to unthinkable extremes. Initially, Shinya Yamanaka would follow his fathers wishes and become an orthopedic surgeon, but he found himself ill-suited to the surgeons life. Intrigued more by the behind-the-scenes biological processes that make the body work, he found himself drawn to basic research, and began his career by trying to find a way to lower cholesterol production. That work also wasnt successful, but it drew him to the challenge of understanding what makes cells divide, proliferate and develop in specific ways.
In 2006, while at Kyoto University, Yamanaka stunned scientists by announcing he had successfully achieved what Gurdon had with the frog cells, but without using eggs at all. Yamanaka mixed four genes in with skin cells from adult mice and turned those cells back to an embryo-like state, essentially erasing their development and turning back their clock. The four genes reactivated other genes that are prolific in the early embryo, and turned off those that directed the cells to behave like skin.
(MORE: Ovary Stem Cells Can Produce New Human Eggs)
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Stem Cell Scientists Awarded Nobel Prize in Physiology and Medicine
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CORD:USE Cord Blood Bank is proud to announce the addition of Cord Blood Stem Cell Transplantation Expert, Dr …
ORLANDO, Fla., Oct. 9, 2012 /PRNewswire/ --Vanderson Rocha, M.D., Ph.D., recognizedthroughout the world as a respected leader in the field of cord blood stem cell transplantation, hasjoined the team at CORD:USE Cord Blood Bank. Dr. Rocha's extensive experience and knowledge in transplant medicine and stem cell biology will provide a significant contribution to CORD:USE. "We're excited and honored to have Dr. Rocha, an internationally acclaimed expert in cord blood stem cell transplantation, as a member of our highly esteemed team,"said Edward Guindi MD, President and CEO of CORD:USE.
Dr. Rocha is a professor of Hematology and the Director of the Bone Marrow Transplant Unit at the University of Oxford, UK. He also serves as the Director of the Bone Marrow Transplant Unit, Hospital Sirio Libanes and Children's Hospital of the University of Sao Paulo, Brazil. He is the Scientific Director of the Eurocord Project and is on the Editorial Board of Bone Marrow Transplantation. Dr. Rocha is an internationally renowned speaker regarding the use of unrelated and related hematopoietic stem cells in transplants. He has published more than 200 papers in the New England Journal of Medicine, Blood, Lancet, Journal of Clinical Oncology, British Journal of Hematology, and other peer reviewed publications.
Dr. Rocha continues to contribute significantly to the development and refinement of the therapeutic applications of cord blood stem cells. Due to his expertise, he was elected by the European Transplant Centers as Chairman of the Acute Leukemia Working Party of the European Group for Blood and Marrow Transplantation (EBMT) from 2004 to 2010.
"I am very honored to be a member of the distinguished team at CORD:USE which includes my colleagues who are pioneers in cord blood science, banking and transplantation. I look forward to continuing to work with them to advance the use of cord blood transplantation to treat many more patients in the future," said Dr. Rocha.
Dr. Rocha joins otherhighly respected leaders and pioneers in the field of cord blood stem cell transplantation on the CORD:USE team:
About CORD:USE Cord Blood Bank, Inc.
CORD:USE operates leading public and family cord blood banks. CORD:USE Public Cord Blood Bank is one of the high quality cord blood banks selected and funded by HRSA of the U.S. Department of Health and Human Services to help build the National Cord Blood Inventory (NCBI). CORD:USE Cord Blood Bank has entered into agreements with hospitalsacross the country to provide mothers the option to donate their babies' cord blood. CORD:USE cord blood units are listed in the NCBI through the National Marrow Donor Program's Registry and are distributed to transplanters, throughout the country and the world. CORD:USE Family Cord Blood Bank protects family banked cord blood units utilizing similar high-quality cord blood banking practices and technologies that are used in our leading public cord blood bank in its state-of-the-art laboratory. For more information, please visit our website http://www.corduse.com, or contact Michael Ernst at 407.667.3000.
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CORD:USE Cord Blood Bank is proud to announce the addition of Cord Blood Stem Cell Transplantation Expert, Dr ...
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Foundation Medicine, Eisai Inks Personalized Med Alliance
The J. Craig Venter Institute has appointed Karen Nelson as President and Robert Friedman as chief operating officer. They will report to Craig Venter, who will continue to lead JCVI as founder, CEO, and chairman of the board of trustees. Nelson has been at JCVI since 1996 and will continue to serve as director of the institute's offices in Rockville, Md., and leader of the Human Genomic Medicine Group. She will remain based in Rockville. Friedman has been with the institute since 2003 and he is director of the San Diego facility and head of the JCVI Policy Group. He will keep those duties but will take on more administrative leadership responsibilities.
XDx has named Peter Maag to be its president and CEO, effective immediately, the molecular diagnostics company said this week. Maag previously was president of Novartis Diagnostics, where he ran the company's blood screening business and created a strategy for the company's investment into developing diagnostics for transplantation, infectious disease, and prenatal care. He also was country president for Novartis Pharma in Germany and held other positions at Novartis after working at McKinsey and Company.
The Human Frontier Science Program Organization has awarded Stephen Quake the 2013 Nakasone Award for his work developing technologies in biophysics, biological automation, genome analysis, and personalized medicine. Quake is a professor of bioengineering and a Howard Hughes Medical Institute investigator at Stanford University. He is also a co-founder of Helicos Biosciences and Fluidigm.
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Foundation Medicine, Eisai Inks Personalized Med Alliance
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Foundation Medicine, Eisai Ink Personalized Med Alliance
The J. Craig Venter Institute has appointed Karen Nelson as President and Robert Friedman as chief operating officer. They will report to Craig Venter, who will continue to lead JCVI as founder, CEO, and chairman of the board of trustees. Nelson has been at JCVI since 1996 and will continue to serve as director of the institute's offices in Rockville, Md., and leader of the Human Genomic Medicine Group. She will remain based in Rockville. Friedman has been with the institute since 2003 and he is director of the San Diego facility and head of the JCVI Policy Group. He will keep those duties but will take on more administrative leadership responsibilities.
XDx has named Peter Maag to be its president and CEO, effective immediately, the molecular diagnostics company said this week. Maag previously was president of Novartis Diagnostics, where he ran the company's blood screening business and created a strategy for the company's investment into developing diagnostics for transplantation, infectious disease, and prenatal care. He also was country president for Novartis Pharma in Germany and held other positions at Novartis after working at McKinsey and Company.
The Human Frontier Science Program Organization has awarded Stephen Quake the 2013 Nakasone Award for his work developing technologies in biophysics, biological automation, genome analysis, and personalized medicine. Quake is a professor of bioengineering and a Howard Hughes Medical Institute investigator at Stanford University. He is also a co-founder of Helicos Biosciences and Fluidigm.
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Foundation Medicine, Eisai Ink Personalized Med Alliance
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Research and Markets: Competitive Handbook towards Personalized Medicine in Prostate Cancer Report Identifies 227 Drug …
DUBLIN--(BUSINESS WIRE)--
Research and Markets (http://www.researchandmarkets.com/research/m427tb/competitive) has announced the addition of the "Competitive Handbook towards Personalized Medicine in Prostate Cancer" report to their offering.
BioSeeker builds its analysis on a comprehensive base of 394 prostate cancer drugs from within the portfolio of 222 companies world-wide, from Ceased to Marketed. We have identified 227 drug targets, which we have organized into 216 drug target strategies, and assessed them by four levels:
1. Individual Target: Shows you how individual targets tie into different target strategies and their subsequent R&D progress.
2. Developmental Stage: Shows you the progress and maturation of different target strategies. Identifies which target strategies are new and unique from one developmental stage to the next.
3. Compound Type: Shows you the competitive landscape of target strategies from a compound perspective, including crossover analysis of target strategies among different compound types.
4. Company: Provides a cross-examination of each company's entire pipeline on the basis of its defined drug target strategies, including a Competitive Fall-Out analysis and a corporate pipeline ranking based on 15 parameters.
Companies Mentioned:
Abbott
Active Biotech
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Kessler Foundation scientists present rehabilitation research findings at 2012 ACRM-ASNR Conference
Public release date: 8-Oct-2012 [ | E-mail | Share ]
Contact: Carolann Murphy CMurphy@KesslerFoundation.org 973-324-8382 Kessler Foundation
West Orange, NJ. October 8, 2012. Scientists from Kessler Foundation are presenting recent findings during Progress in Rehabilitation Research, the 2012 Conference of the American Congress of Rehabilitation Medicine and the American Society of NeuroRehabilitation (ACRM-ASNR). A.M. Barrett, MD, Amanda Botticello, PhD, Peii Chen, PhD, Abhijit Das, MD, Gail F. Forrest, PhD, Yael Goverover, PhD, Denise Krch, PhD, Karen Nolan, PhD, and Mooyeon Oh-Park, MD, are addressing a variety of topics that represent the Foundation's rehabilitation research in stroke, brain injury, multiple sclerosis and spinal cord injury.
A.M. Barrett, MD, the current president of ASNR, is director of Stroke Rehabilitation Research at Kessler Foundation. Drs. Barrett and Chen will present promising results of prism adaptation treatment for spatial neglect, a common post-stroke hidden disability. Dr. Park, the assistant director of Stroke Rehabilitation Research, will present the Foundation's work on the impact of post-stroke cognitive deficits on patient satisfaction surveys of inpatient rehabilitation. Dr. Chen, research scientist in Stroke Rehabilitation Research, will discuss the Kessler Foundation Neglect Assessment Process (KF-NAP)-a new standardized tool for reliable functional assessment evaluation of spatial neglect. Drs. Chen and Botticello share their findings on the secondary impact of stroke in their study of cognitive decline among caregivers. Dr. Botticello is a research scientist in Outcomes & Assessment Research at Kessler Foundation.
During the symposium, Health Promotion and Disease Prevention across the Lifespan in Spinal Cord Injury, Dr. Forrest will address the potential for electrical stimulation to attenuate the muscle and bone loss that occurs after spinal cord injury. Dr. Forrest directs mobility research at the Foundation, where she is assistant director of Human Performance and Engineering Research. Dr. Nolan, a research scientist in Human Performance and Engineering Research, will present a case report on hemiplegia after stroke illustrating the functional gains after utilization of a foot drop stimulator.
Drs. Krch and Goverover are presenting on cultural adaptations and functional assessment in cognitive rehabilitation. Dr. Krch is a research scientist in Neuropsychology and Neuroscience Research; Dr. Goverover is a visiting scientist from New York University. Their presentations address the use of strategies to improve cognitive and everyday functioning in persons with cognitive impairments.
Dr. Das is one of five young investigators invited to participate in the conference's Young Investigators Panel sponsored by National Institute on Disability and Rehabilitation Research (NIDRR). Dr. Das, a NIDRR-funded post-doctoral fellow in Neuropsychology & Neuroscience Research at Kessler Foundation, will present his findings on the neurobiology of self-reported fatigue in individual with multiple sclerosis.
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Researchers at Kessler Foundation have faculty appointments in the department of physical medicine & rehabilitation at the University of Medicine & Dentistry of New JerseyNew Jersey Medical School. They collaborate closely with clinicians at Kessler Institute for Rehabilitation.
About Kessler Foundation
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Kessler Foundation scientists present rehabilitation research findings at 2012 ACRM-ASNR Conference
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ACT Announces Approval to Treat Additional Stargardt’s Disease Patients with Higher RPE Dosage in Both U.S. and …
MARLBOROUGH, Mass.--(BUSINESS WIRE)--
Advanced Cell Technology, Inc. (ACT; OTCBB: ACTC), a leader in the field of regenerative medicine, announced today that the Data and Safety Monitoring Board (DSMB), an independent group of medical experts closely monitoring the Companys three ongoing clinical trials, has authorized the Company to move forward with enrollment and treatment of second and third additional patients with Stargardts macular dystrophy (SMD) in the second patient cohort of its U.S. trial for the condition. Additionally, the DSMB has authorized the Company to treat all three patients in the second cohort of its European trial for SMD.
The UK Medicines and Healthcare products Regulatory Agency (MHRA) recently approved a protocol modification to the DSMB review, streamlining the process, allowing the company to treat the first patient in a new cohort if the DSMB has allowed this in the US study, and once clearance has been received in the US trial to treat the next two patients in the US cohort. This would also allow for treatment of the UK patients without an additional review by the DSMB. Moreover, according to the protocol for both trials, each patient in the second cohort will be injected with 100,000 human embryonic stem cell (hESC)-derived retinal pigment epithelial (RPE) cells, up from 50,000 in the first cohort.
This authorization to treat the next five patients in the second, higher-dosage cohort in both our clinical trials for SMD represents a significant step forward for our clinical programs, commented Gary Rabin, chairman and CEO of ACT. We are also encouraged with the MHRAs approval of the DSMBs streamlined review process. Clearly this has the potential to help accelerate the pace of our European trial.
ACT is conducting three clinical trials in the U.S. and Europe using hESC-derived RPE cells to treat forms of macular degeneration, SMD and dry age-related macular degeneration (dry AMD). Each trial will enroll a total of 12 patients, with cohorts of three patients each in an ascending dosage format, from 50,000 hESC-derived RPE cells in the first patient cohort to 200,000 in the last and final cohort. These trials are prospective, open-label studies, designed to determine the safety and tolerability of hESC-derived RPE cells following sub-retinal transplantation into patients with dry-AMD or SMD at 12 months, the studys primary endpoint.
We are eagerly anticipating treating these final two patients in the second cohort of our U.S. trial for SMD, and all three patients in the second cohort of our E.U. trial, commented Robert Lanza, M.D., ACTs chief scientific officer. We are encouraged by the preliminary data in the first patient in this second, higher-dosage cohort and look forward to gathering more data.
Further information about patient eligibility for ACTs SMD studies in the U.S. and E.U. as well as its dry AMD study are available atwww.clinicaltrials.gov,with the following Identifiers: NCT01345006 (U.S. SMD), NCT01469832 (E.U. SMD), and NCT01344993 (dry AMD).
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 Approval to Treat Additional Stargardt’s Disease Patients with Higher RPE Dosage in Both U.S. and ...
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Regenerative Medicine Biotech Company, Eqalix, Names Scientific Advisory Board
Eqalix Inc., an emerging regenerative medicine company, announces its Scientific Advisory Board (SAB). This SAB gives Eqalix a depth and breadth of experience necessary to take it to the next level.
Reston, VA (PRWEB) October 09, 2012
"We are very pleased to bring together these key thought leaders to establish the Eqalix Scientific Advisory Board," stated Joseph P. Connell, Eqalix CEO and Chairman of the Board. "I have worked with Drs. Gold and Goldman for years and have always admired their abilities. Dr Lelkes technologies will make a profound impact upon aesthetic dermatology, wound healing and regenerating blood vessels, nerve endings and damaged organs with the guidance of this distinguished panel. It is not clich in any manner when I say that we are thrilled to work with this team. We look to their guidance, industry knowledge and network to help deliver these therapies into clinic and prospective patients as soon as possible, as I am confident our technologies will make a difference, said Connell.
The members of the Eqalix Scientific Advisory Board are:
Peter I. Lelkes, PhD: Chief Scientific Advisor; Dr. Lelkes is the Laura H. Carnell Professor and Founding Chair of the Department of Bioengineering in the College of Engineering at Temple University and the Inaugural Director of the Institute for Regenerative Medicine and Engineering (TIME) at Temple Universitys School of Medicine. While at Drexel, Prof. Lelkes directed an interdisciplinary program in tissue engineering and regenerative medicine, focusing on nanotechnology-based biomaterials and soft tissue engineering, employing developmental biological principles to enhance the tissue-specific differentiation of embryonic and adult stem cells. Dr. Lelkes has organized several Keystone conferences and published more than 160 peer-reviewed papers and 45 book chapters and made more than 400 presentations nationally and internationally.
Dr. Lelkes basic and translational research has been support by federal (NIH, NSF, NASA, DOE) and state funding agencies, (NTI and Dept. of Commerce, Tobacco Settlement Funds) and private Foundations, including the Coulter Foundation. Most recently, Dr. Lelkes has been named Director of the Surgical Engineering Enterprise, one of the major initiatives of the strategic plan of Drexel Universitys College of Medicine. In addition, Dr. Lelkes has been the team leader for tissue engineering at the Nanotechnology Institute of Southeastern Pennsylvania (NTI) and is the Co-Director of PATRIC, the Pennsylvania Advanced Textile Research and Innovation Center, focusing on BioNanoTextiles and Stem Cell Biology.
Dr Lelkes stated, "I am delighted and excited to partner with Eqalix to translate our inventions from the bench to the bedside in a timely fashion.
Mitchel P. Goldman, MD, Scientific Advisor, Founder and Medical Director of Goldman Butterwick Fitzpatrick, Groff & Fabi, Cosmetic Laser Dermatology. A graduate of Boston University, Summa Cum Laude, and the Stanford University Medical School, Dr. Goldman is a Volunteer Clinical Professor in Medicine/Dermatology at the University of California, San Diego. Dr Goldman is Board Certified by both the American Board of Dermatology and the American Board of Cosmetic Surgery.
He is a fellow of the American Academy of Dermatology, American Society for Dermatologic Surgery, American Society for Laser Medicine and Surgery, American Academy of Cosmetic Surgery and the American Society of Liposuction Surgery. He is former President of the American College of Phlebology and President-Elect of the American Society for Dermatologic Surgery. He presently serves on the Board of Trustees for the American Academy of Cosmetic Surgery. He also has authored and/or co-authored 21 Textbooks on Dermatology, Sclerotherapy, Ambulatory Phlebectomy, Cutaneous Laser Surgery, Cellulite and Dermatologic Surgery as well as over 300 peer-reviewed publications and textbook chapters.
Dr Goldman added: I am very interested and excited to work with the Eqalix team to make these technologies a success. I believe that my background lends well to truly shaping the successful commercialization of these products for my patients to improve outcomes.
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Regenerative Medicine Biotech Company, Eqalix, Names Scientific Advisory Board
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Stem cell therapy a miracle cure? Not quite yet
The techniques pioneered by the winners of this years Nobel Prize in medicine, John B. Gurdon and Shinya Yamanaka, have already allowed scientists to generate stem cells and clone animals.
But it is the potential these discoveries hold that truly boggles the mind. If stem cells the primitive cells that develop into tissue like skin, blood, nerves, muscle and bone can be harnessed, the belief is they can be used as a repair kit for the body.
In theory, a few skin cells could be harvested to rebuild a spinal cord damaged by trauma, to replace brain cells destroyed by dementia, to rebuild heart muscle damaged by a heart attack or to grow a new limb ravaged by diabetes. It is the stuff of science fiction, so close we can taste it.
But these dreams of miracle cures must be tempered with a strong dose of realism.
Despite billions of dollars in investment in research, from government agencies and biotech companies, there is little evidence that stem cell therapies work.
Yes, some hearing has been restored in gerbils and there have been modest improvements in paralyzed lab rats using stem cell treatments, but these are baby steps. In humans, the gains have been far more modest.
We can treat some forms of cancer, like leukemia and multiple myeloma, with stem cell transplants. But this is simply a refinement of an earlier technique, bone marrow transplant. And to perform such a transplant, the immune system must, for all intents and purposes, be destroyed a punishing regime with a significant mortality rate.
It is a far cry from the notion of an injection of magic stem cells that allow people to walk again or restore their memories.
The International Society for Stem Cell Research says that while there are hundreds of conditions that can purportedly be treated with stem cells, the treatments that have actually been shown to be beneficial are extremely limited. Aside from the cancer treatments mentioned above, some bone, skin and corneal conditions have been treated by grafting stem cells, growing them in the lab and transplanting them.
But in all these cases, the stem cells are tissue-specific, meaning the cells are carrying out a function they were designed to do. This is very different from the notion that undifferentiated stem cells can be used to treat a broad range of conditions.(And we wont delve into potential problems, such as rejection and the concern that stem cells could grow out of control and cause cancerous tumours.)
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Stem cell therapy a miracle cure? Not quite yet
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UK, Japan scientists win Nobel for stem cell breakthroughs
STOCKHOLM (Reuters) - Scientists from Britain and Japan shared a Nobel Prize on Monday for the discovery that adult cells can be transformed back into embryo-like stem cells that may one day regrow tissue in damaged brains, hearts or other organs.
John Gurdon, 79, of the Gurdon Institute in Cambridge, Britain and Shinya Yamanaka, 50, of Kyoto University in Japan, discovered ways to create tissue that would act like embryonic cells, without the need to harvest embryos.
They share the $1.2 million Nobel Prize for Medicine, for work Gurdon began 50 years ago and Yamanaka capped with a 2006 experiment that transformed the field of "regenerative medicine" - the field of curing disease by regrowing healthy tissue.
"These groundbreaking discoveries have completely changed our view of the development and specialisation of cells," the Nobel Assembly at Stockholm's Karolinska Institute said.
All of the body's tissue starts as stem cells, before developing into skin, blood, nerves, muscle and bone. The big hope for stem cells is that they can be used to replace damaged tissue in everything from spinal cord injuries to Parkinson's disease.
Scientists once thought it was impossible to turn adult tissue back into stem cells, which meant that new stem cells could only be created by harvesting embryos - a practice that raised ethical qualms in some countries and also means that implanted cells might be rejected by the body.
In 1958, Gurdon was the first scientist to clone an animal, producing a healthy tadpole from the egg of a frog with DNA from another tadpole's intestinal cell. That showed developed cells still carry the information needed to make every cell in the body, decades before other scientists made headlines around the world by cloning the first mammal, Dolly the sheep.
More than 40 years later, Yamanaka produced mouse stem cells from adult mouse skin cells, by inserting a few genes. His breakthrough effectively showed that the development that takes place in adult tissue could be reversed, turning adult cells back into cells that behave like embryos. The new stem cells are known as "induced pluripotency stem cells", or iPS cells.
"The eventual aim is to provide replacement cells of all kinds," Gurdon's Institute explains on its website.
"We would like to be able to find a way of obtaining spare heart or brain cells from skin or blood cells. The important point is that the replacement cells need to be from the same individual, to avoid problems of rejection and hence of the need for immunosuppression."
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UK, Japan scientists win Nobel for stem cell breakthroughs
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Nobel Winners Unlocked Cells' Unlimited Potential
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John B. Gurdon (left) and Shinya Yamanaka will share the prize, worth about $1.2 million.
The two scientists who won this year's Nobel Prize in Physiology or Medicine discovered that cells in our body have the remarkable ability to reinvent themselves. They found that every cell in the human body, from our skin and bones to our heart and brain, can be coaxed into forming any other cell.
The process is called reprogramming, and its potential for new drugs and therapies is vast. If neurons or heart cells are damaged by disease or aging, then cells from the skin or blood potentially could be induced to reprogram themselves and repair the damaged tissue.
The winners John Gurdon of the Gurdon Institute in Cambridge, England, and Shinya Yamanaka of Kyoto University in Japan and the Gladstone Institute in San Francisco made their discoveries more than 40 years apart.
In 1962, Gurdon proved that a cell from a frog's stomach contained the entire blueprint to make a whole frog. When he took the cell's nucleus and popped it into a frog egg, the egg developed into a normal frog.
This method eventually was used to clone all sorts of animals, including cats, dogs, horses and, most famously, Dolly the sheep the first mammal cloned from an adult cell. Gurdon, 79, continues to study reprogramming and was working in his lab when he received the call from the Nobel committee.
But a major obstacle stood in the way of further development of these stem cells: Getting the frog's stomach cell to strip away its specialization and turn into one of the 200 or so cell types known to exist in animals always required the use of an egg.
A question hung over the field for decades: Could a specialized cell reprogram itself all on its own?
In 2006, Yamanaka and graduate student Kazutoshi Takahashi found the answer, and it sent shockwaves through biology and medicine. They demonstrated that any cell could be reset and induced to develop into another cell type. And, even more remarkably, that it took little to get the job done.
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Nobel Winners Unlocked Cells' Unlimited Potential
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Nobel Prize awarded for stem cell breakthroughs
Reuters
This undated handout photo shows iPS cells derived from adult human dermal fibroblasts released by Kyoto University Professor Shinya Yamanaka at Center for iPS Cell Research and Application of Kyoto University in Kyoto, western Japan.
By Reuters
Scientists from Britain and Japan shared a Nobel Prize on Monday for the discovery that adult cells can be transformed back into embryo-like stem cells that may one day regrow tissue in damaged brains, hearts or other organs.
John Gurdon, 79, of the Gurdon Institute in Cambridge, Britain and Shinya Yamanaka, 50, of Kyoto University in Japan, discovered ways to create tissue that would act like embryonic cells, without the need to harvest embryos.
They share the $1.2 million Nobel Prize for Medicine, for work Gurdon began 50 years ago and Yamanaka capped with a 2006 experiment that transformed the field of "regenerative medicine" - the field of curing disease by regrowing healthy tissue.
"These groundbreaking discoveries have completely changed our view of the development and specialization of cells," the Nobel Assembly at Stockholm's Karolinska Institute said.
Photoblog: Click for a close-up viiew of the Nobel Prize-winning stem cell research
All of the body's tissue starts as stem cells, before developing into skin, blood, nerves, muscle and bone. The big hope for stem cells is that they can be used to replace damaged tissue in everything from spinal cord injuries to Parkinson's disease.
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Nobel Prize awarded for stem cell breakthroughs
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Stem Cell Science Q & A
Shinya Yamanaka MD, PhD
Here are answers to frequently asked questions about induced pluripotent stem cells, or iPS cells, the type of cell that has been reprogrammed from an adult cell, such as a skin or blood cell.
What are induced pluripotent stem cells?
Induced pluripotent stem cells, or iPS cells, are a type of cell that has been reprogrammed from an adult cell, such as a skin or blood cell. iPS cells are pluripotent cells because, like embryonic stem cells, they can develop into virtually any type of cell. iPS cells are distinct from embryonic stem cells, however, because they are derived from adult tissue, rather than from embryos. iPS cells are also distinct from adult stem cells, which naturally occur in small numbers in thehuman body.
In 2006, Shinya Yamanaka developed the method for inducing skin cells from mice into becoming like pluripotent stem cells and called them iPS cells. In 2007, Yamanaka did the same with adult human skin cells.
Yamanakas experiments revealed that adult skin cells, when treated with four pieces of DNA (now called the Yamanaka factors), can induce skin cells to revert back to their pluripotent state. His discovery has since led to a variety of methods for reprogramming adult cells into stem cells that can become virtually any cell type such as a beating heart cell or a neuron that can transmit chemical signals in the brain. This allows researchers to create patient-specific celllines that can be studied and used in everything from drug therapies to regenerative medicine.
How are iPS cells different from embryonic stem cells?
iPS cells are a promising alternative to embryonic stem cells. Embryonic stem cells hold tremendous potential for regenerative medicine, in which damaged organs and tissues could be replaced or repaired. But the use of embryonic stem cells has long been controversial. iPS cells hold the same sort of promise but avoid controversy because they do not require the destruction of human embryos. Nor do they require the harvesting of adult stem cells. Rather, they simply require a small tissue sample from a living human.
Why is iPS cell technology so important?
In addition to avoiding the controversial use of embryonic stem cells, iPS cell technology also represents an entirely new platform for fundamental studies of human disease. Rather than using models made in yeast, flies or mice for disease research, iPS cell technology allows human stem cells to be created from patients with a specific disease. As a result, the iPS cells contain a complete set of the genes that resulted in that disease and thus represent the potential of a farsuperior human model for studying disease and testing new drugs and treatments. In the future, iPS cells could be used in a Petri dish to test both drug safety andefficacy for an individual patient.
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Stem Cell Science Q & A
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UK, Japan scientists win Nobel for adult stem cell discovery
STOCKHOLM (Reuters) - Scientists from Britain and Japan shared a Nobel Prize on Monday for the discovery that adult cells can be transformed back into embryo-like stem cells that may one day regrow tissue in damaged brains, hearts or other organs.
John Gurdon, 79, of the Gurdon Institute in Cambridge, Britain and Shinya Yamanaka, 50, of Kyoto University in Japan, discovered ways to create tissue that would act like embryonic cells, without the need to collect the cells from 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 search for ways to cure disease by growing 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 starts as stem cells, before developing into tissue like skin, blood, nerves, muscle and bone. The big hope is that stem cells can grow to replace damaged tissue in cases from spinal cord injuries to Parkinson's disease.
Scientists once thought it was impossible to turn adult tissue back into stem cells. That meant new stem cells could only be created by taking them from embryos, which raised ethical objections that led to research bans in some countries.
As far back as 1962 Gurdon became the first scientist to clone an animal, making a healthy tadpole from the egg of a frog with DNA from another tadpole's intestinal cell. That showed that developed cells carry the information to make every cell in the body - decades before other scientists made world headlines by cloning the first mammal from adult DNA, Dolly the sheep.
More than 40 years later, Yamanaka produced mouse stem cells from adult mouse skin cells by inserting a small number of genes. His breakthrough effectively showed that the development that takes place in adult tissue could be reversed, turning adult tissue back into cells that behave like embryos.
Stem cells created from adult tissue are known as "induced pluripotency stem cells", or iPS cells. Because patients may one day be treated with stem cells from their own tissue, their bodies might be less likely to reject them.
"The eventual aim is to provide replacement cells of all kinds," Gurdon's institute explains on its website.
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UK, Japan scientists win Nobel for adult stem cell discovery
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Stem-Cell Pioneers Gurdon, Yamanaka Win Nobel Prize
John B. Gurdon transferred DNA between a tadpole and a frog to clone the first animal. Shinya Yamanaka used Gurdons concept to turn ordinary skin into potent stem cells. Both won the Nobel Prize for medicine today.
Gurdon, 79, a researcher at the University of Cambridge in the U.K., and Yamanaka, 50, a professor at Kyoto University in Japan, will share the 8 million-kronor ($1.2 million) prize, the Nobel Assembly said today in Stockholm. The pairs findings have created new opportunities to study diseases and develop methods for diagnosis and therapy, the assembly said in a statement.
Gurdons feat, in 1962, paved the way in 1996 for the cloning of Dolly the sheep and, 10 years later, for Yamanaka, who turned mouse skin cells into stem cells with the potential to become any cell in the body. That achievement was lauded by some politicians and religious figures as a more ethical way to make stem cells because it doesnt destroy human life.
This field has had a long history, starting with John Gurdon, Yamanaka, who was born the same year Gurdon published his achievement, said in an interview on the Nobel Assemblys website. I was able to initiate my project because of his experiments 50 years ago.
Stem cells are found in human embryos and in some tissues and organs of adults, and have the potential to develop into different types of cells. Thats spurred scientists to look at ways of harnessing their power to treat diseases such as Alzheimers, stroke, diabetes and rheumatoid arthritis, according to the U.S. National Institutes of Health.
Gurdon showed that mature cells from specific parts of an animals body retain all the genetic information they had as immature stem cells. He took a cell from a tadpoles gut, extracted the nucleus, and inserted it into the egg cell of an adult frog whose own nucleus had been removed. That reprogrammed egg cell developed into a tadpole with the genetic characteristics of the original tadpole, and subsequent trials yielded adult frogs.
Gurdon overturned the prevailing view that as cells differentiate, they lose genes and their ability to generate other cells of any kind, said Alan Colman, the executive director of the Singapore Stem Cell Consortium, who gained his doctorate under Gurdon at Cambridge.
Hes amazingly passionate, Colman said in an interview before the award was announced. He was the sort of supervisor who you found it difficult to get appointments with, not because he was flying around the world, but because he was doing experiments all the time.
Gurdon was answering e-mails in his laboratory when he received the call from Sweden today about the prize, he said in an interview on the Nobel Assemblys website. His first reaction was, Its amazing if its really true, he said. Could it be that someones pulling your leg? That has happened before.
Gurdon will celebrate at a reception that his institute is hosting today, and then hell be back to work early tomorrow, he said at a London news conference today.
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Stem-Cell Pioneers Gurdon, Yamanaka Win Nobel Prize
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Blue Spa and Lifeline® Stem Cell Skin Care Pair up to Promote a Beauty Breakthrough and Scientific Approach to Anti …
Skin care meets science for stem cell education and product introduction to the only human and non-embryonic stem cell skin care line of its kind on October 25th, 2012.
Los Angeles, CA (PRWEB) October 08, 2012
Lifeline Skin Care products feature a unique combination of stem cell extracts, vitamins A, B, E, and antioxidants that work synergistically to create new healthy cells. To date, Lifeline is the only skin care line based on human non-embryonic stem cells, which give skin cells the ability to continually proliferate. The result is firmer, smoother, younger and healthier looking skin. Lifeline Skin Care is based on a patented method for ethically extracting growth factors and peptides from young, human stem cells through the use of non-fertilized eggs and never embryos. Stem cell extracts help to increase skins overall thickness, making skin less vulnerable to premature aging.
Independent clinical studies have proven 73% firmer, tighter skin, 93% improved skin hydration, 63% improved skin tone and brightness, and 67% improved appearance of lines and wrinkles with topical use. With benefits boasting similar to those of collagen injections, Lifeline Skin Care offers a collection of formulas for day and night use. Both the Defensive Day Moisturizer Serum SPF 15 and Recovery Night Moisture Serum feature unique combinations of stem cell extract, vitamins A, B, E, and antioxidants.
Stimulating the skins ability to repair itself, these products along with Blue Spa professional procedures and treatments, make a win-win combination for beauty enthusiasts wanting to achieve optimal skincare results. Owner of Blue Spa, Ronda Nofal, recently stated, We are very pleased to be the first Medi Spa in Los Angeles to offer Lifeline@ Skin Care technology to clients. The science and technology behind this product line is far beyond anything else on the market and the results speak for themselves. Our staff has been using these products for the last two months and they have noticed theyre the perfect compliment to any of our facial laser services: IPL (FotoFacial), Laser Genesis, and Titan Skin Tightening. The skin reacts beautifully when paired with dermal fillers, Vitalize Peels, and Micro-dermabrasion as well.
Members of the press and media are invited for early entry on Thursday, October 25th, 2012 between 1-4 pm for Q& A with Lifeline Skin Care expert, Linda Nelson. Additional hours have been arranged for Friday, October 26th, 2012 from 10 am-12 pm. Please directly contact Blue Spa and Lifeline Skin Cares publicity team at Jade Umbrella, to schedule interviews.
About Blue Spa: Opened in October 1999 and former home to the infamous La Reina Theater, Blue Medi Spa is modern luxury spa combining beauty, science, service, and style. Staying ahead of beauty trends and the most effective treatments, highly trained specialists have the knowledge and a decade of experience in lasers (IPL/ Titan/ Laser Genesis/ Zerona), anti-aging skin cocktails, weight loss, non-invasive body contouring, and one-step-ahead aesthetic options. Where feeling blue, never felt better
Website: http://www.bluespa.com.
About Lifeline Skin Care: Developed in 2010 by the International Stem Cell Corporation (http://www.internationalstemcell.com/), while researching cures for diabetes and Parkinsons Disease, a team of biotech scientists discovered a powerful compound for regenerating skin cells. Lifeline Skin Cares goal is to help improve the look and feel of you skin by combining the latest discoveries in the fields of stem cell biology, nanotechnology and skin cream formulation technology to create the highest quality, scientifically tested, and most effective anti-aging products. Revenue helps to fund further research into finding cures and treatments for Diabetes, Parkinsons, Liver, Eye, and other neurological diseases.
Website: http://www.lifelineskincare.com
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Blue Spa and Lifeline® Stem Cell Skin Care Pair up to Promote a Beauty Breakthrough and Scientific Approach to Anti ...
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Nobel Winner’s Stem Cells to Be Tested in Eye Disease Next Year
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
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Cellerant Awarded SBIR Contract Funding to Develop CLT-009 for Treatment of Thrombocytopenia
SAN CARLOS, Calif.--(BUSINESS WIRE)--
Cellerant Therapeutics Inc., a biotechnology company developing novel hematopoietic stem cell-based cellular and antibody therapies for blood disorders and cancer, announced today that it has been awarded a Small Business Innovation Research (SBIR) Phase 1 contract and a Phase 2 option from the National Cancer Institute (NCI) valued up to $1,683,503. The SBIR Contract funds the development of CLT-009, a first-in-class, human allogeneic Megakaryocyte Progenitor Cell therapy for the treatment of thrombocytopenia in cancer patients and allows the Company to conduct studies to enable an Investigational New Drug (IND) Application to be filed with the FDA in the next two years.
Thrombocytopenia is characterized as a significant reduction in the concentration of circulating platelets. Platelets are crucial in the process of coagulation to stop bleeding, and thrombocytopenia can increase the risk of severe bleeding in patients. It is becoming an increasingly common problem among oncology patients and a significant dose-limiting toxicity, especially in the treatment of hematological malignancies. Chemotherapy and radiation therapy are the most common causes of thrombocytopenia because the platelet-producing cells, megakaryocytes, and their precursors are highly sensitive to myelosuppressive cytotoxics and ionizing radiation. Thrombocytopenia typically occurs during the initial cycles of high-dose chemotherapy and radiation therapy, usually 614 days after administration. According to Datamonitor, the estimated incidence of cancer patients who suffer from significant chemotherapy-induced thrombocytopenia worldwide was approximately 200,000 in 2008.
Occurrence of severe thrombocytopenia may require dose reductions for chemotherapy regimens which can impact subsequent disease control and survival, especially in the treatment of hematological malignancies such as acute leukemia and high-risk myelodysplastic syndrome. Current treatment options include platelet transfusions which are costly and labor intensive and are associated with risks such as contamination and transmission of viral and bacterial infections. Recombinant human interleukin-11 is the only approved agent for chemotherapy induced thrombocytopenia but its use is limited and has only modest efficacy and significant side effects. CLT-009, a human Megakaryocyte Progenitor Cell product, would be an alternative treatment option, providing the critical megakayocyte progenitor cellular support to rapidly produce platelets in vivo and shorten the duration of severe thrombocytopenia following chemotherapy treatment.
We are delighted to receive this contract from NCI to support the development of our novel, off-the-shelf, platelet product and address a high unmet need, said Ram Mandalam, Ph.D., President and Chief Executive Officer of Cellerant Therapeutics. This contract allows us to not only leverage our experience in developing cellular therapies but also provides us with the ability to bring CLT-009 closer to the clinic. Our unique product portfolio, which now includes CLT-009, along with our CLT-008 myeloid progenitor cell product and our therapeutic antibodies targeting cancer stem cells, demonstrates our continued commitment to developing novel products for the benefit of cancer patients.
In addition to this SBIR contract, Cellerant has previously received grants from the National Institute of Health (NIH) in 2008 2010 to conduct research studies in platelet recovery which it has successfully completed. In its previous studies, Cellerant demonstrated that megakaryocyte progenitor cells were able to produce human platelets in preclinical models with in vivo functionality similar to that of normal human platelets.
This program is funded with Federal funds from the National Institute of Health, Department of Health and Human Services, under Contract No.HHSN261201200076C.
About CLT-009
CLT-009 is a unique, off-the-shelf, cryopreserved, cell-based therapy that contains human Megakaryocyte Progenitor Cells derived from adult hematopoietic stem cells that have the ability to mature into functional platelets in vivo. Cellerant is developing CLT-009 as an effective treatment for chemotherapy and radiation-induced thrombocytopenia in cancer patients.
About Cellerant Therapeutics
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Cellerant Awarded SBIR Contract Funding to Develop CLT-009 for Treatment of Thrombocytopenia
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NeoStem Announces Very Small Embryonic-Like Cells (VSEL(TM)) Publication in Stem Cells and Development
NEW YORK, Oct. 8, 2012 (GLOBE NEWSWIRE) -- NeoStem, Inc. (NYSE MKT:NBS), an emerging leader in the fast growing cell therapy market, announced today that data from its collaborative studies with the University of Michigan School of Dentistry further expands the therapeutic potential of its proprietary regenerative cell therapy product, "VSELSTM" (very small embryonic-like stem cells), by demonstrating bone regeneration capabilities in a study published online ahead of print1 in the journal Stem Cells and Development (DOI: 10.1089/scd.2012.0327). The paper highlights that human VSEL stem cells form human bone when implanted in the bone tissue of SCID mice.
VSELs are a population of stem cells found in adult bone marrow with potential regenerative properties similar to those of embryonic stem cells. NeoStem has shown that these cells can be mobilized into the peripheral blood, enabling a minimally invasive means for collecting what NeoStem believes to be a population of stem cells that have the potential to achieve the positive benefits associated with embryonic stem cells without the ethical or moral dilemmas or the potential negative effects known to be associated with embryonic stem cells.
This published controlled study, funded by NIH and led by Dr. Russell Taichman, Major Ash Collegiate Professor and Co-Director of the Scholars Program in Dental Leadership Department of Periodontics & Oral Medicine, University of Michigan and Dr. Aaron Havens, Department of Orthodontics and Pediatric Dentistry at University of Michigan, involved isolating G-CSF mobilized VSEL stem cells from the blood of healthy donors and transplanting them into burr holes made in the cranial bones of SCID mice. After three months, it was observed that the implanted VSEL stem cells had differentiated into human bone tissue in the crania of the mice. Dr. Taichman stated, "I believe this work represents a true partnership between Industry and Academic Institutions. Our findings that VSEL cells can generate human bone in animals would not have been feasible without the help and vision that Dr. Denis Rodgerson and his team at NeoStem brought to the table. It was my privilege to have been a part of this collaborative effort, and I see the resulting data as a significant milestone in stem cell therapy development. It is truly inspiring."
Dr. Robin Smith, Chairman and CEO of NeoStem, added, "This is very exciting data that we believe will be the foundation for future VSEL stem cell studies of bone regeneration in humans. We look forward to moving the development work from the laboratory into the clinic to develop a therapeutic stem cell product to enhance bone formation in humans."
About NeoStem, Inc.
NeoStem, Inc. continues to develop and build on its core capabilities in cell therapy, capitalizing on the paradigm shift that we see occurring in medicine. In particular, we anticipate that cell therapy will have a significant role in the fight against chronic disease and in lessening the economic burden that these diseases pose to modern society. We are emerging as a technology and market leading company in this fast developing cell therapy market. Our multi-faceted business strategy combines a state-of-the-art contract development and manufacturing subsidiary, Progenitor Cell Therapy, LLC ("PCT"), with a medically important cell therapy product development program, enabling near and long-term revenue growth opportunities. We believe this expertise and existing research capabilities and collaborations will enable us to achieve our mission of becoming a premier cell therapy company.
Our contract development and manufacturing service business supports the development of proprietary cell therapy products. NeoStem's most clinically advanced therapeutic, AMR-001, is being developed at Amorcyte, LLC ("Amorcyte"), which we acquired in October 2011. Amorcyte is developing a cell therapy for the treatment of cardiovascular disease and is enrolling patients in a Phase 2 trial to investigate AMR-001's efficacy in preserving heart function after a heart attack. Athelos Corporation ("Athelos"), which is approximately 80%-owned by our subsidiary, PCT, is collaborating with Becton-Dickinson in the early clinical exploration of a T-cell therapy for autoimmune conditions. In addition, pre-clinical assets include our VSELTM Technology platform as well as our mesenchymal stem cell product candidate for regenerative medicine. Our service business and pipeline of proprietary cell therapy products work in concert, giving us a competitive advantage that we believe is unique to the biotechnology and pharmaceutical industries. Supported by an experienced scientific and business management team and a substantial intellectual property estate, we believe we are well positioned to succeed.
Forward-Looking Statements for NeoStem, Inc.
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements reflect management's current expectations, as of the date of this press release, and involve certain risks and uncertainties. Forward-looking statements include statements herein with respect to the successful execution of the Company's business strategy, including with respect to the Company's or its partners' successful development of AMR-001 and other cell therapeutics, the size of the market for such products, its competitive position in such markets, the Company's ability to successfully penetrate such markets and the market for its CDMO business, and the efficacy of protection from its patent portfolio, as well as the future of the cell therapeutics industry in general, including the rate at which such industry may grow. Forward looking statements also include statements with respect to satisfying all conditions to closing the disposition of Erye, including receipt of all necessary regulatory approvals in the PRC. The Company's actual results could differ materially from those anticipated in these forward- looking statements as a result of various factors, including but not limited to (i) the Company's ability to manage its business despite operating losses and cash outflows, (ii) its ability to obtain sufficient capital or strategic business arrangement to fund its operations, including the clinical trials for AMR-001, (iii) successful results of the Company's clinical trials of AMR-001 and other cellular therapeutic products that may be pursued, (iv) demand for and market acceptance of AMR-001 or other cell therapies if clinical trials are successful and the Company is permitted to market such products, (v) establishment of a large global market for cellular-based products, (vi) the impact of competitive products and pricing, (vii) the impact of future scientific and medical developments, (viii) the Company's ability to obtain appropriate governmental licenses and approvals and, in general, future actions of regulatory bodies, including the FDA and foreign counterparts, (ix) reimbursement and rebate policies of government agencies and private payers, (x) the Company's ability to protect its intellectual property, (xi) the company's ability to successfully divest its interest in Erye, and (xii) matters described under the "Risk Factors" in the Company's Annual Report on Form 10-K filed with the Securities and Exchange Commission on March 20, 2012 and in the Company's other periodic filings with the Securities and Exchange Commission, all of which are available on its website. The Company does not undertake to update its forward-looking statements. The Company's further development is highly dependent on future medical and research developments and market acceptance, which is outside its control.
(1) Human Very Small Embryonic-Like Cells Generate Skeletal Structures, In Vivo. Havens A., et al., Stem Cells and Development.
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NeoStem Announces Very Small Embryonic-Like Cells (VSEL(TM)) Publication in Stem Cells and Development
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Biopharma with personalized cell therapy raises $45M in stock sale, forges collaboration deal
A company with the first FDA-approved personalized cell therapy for reducing wrinkles has raised $45 million in a private stock sale. The financing was sought to improve manufacturing capacity for the therapy and advance other uses for it such as treating burn victims, according to a company statement.
Fibrocell Sciences aesthetic therapeutic, Laviv, secured got the green light from the U.S. Food and Drug Administration last year. Laviv uses individuals fibroblast cells to reduce nasolabial fold wrinkles, creases on the face that start from the outer corners of the nose and go down to the corners of the mouth. It also has an acne therapy in phase 3 clinical trials and a burn scar therapy in phase 2 trials.
The Exton, Pennsylvania biopharmaceutical companys personalized cell development platform technology isolates, purifies and multiplies a patients fibroblast cells, connective skin cells that make collagen.
Additionally, Fibrocell agreed to a strategic collaboration with biotechnology firm Intrexon which can provide genome engineering, cell processing, and cell system engineering, among other services, to help advance Fibrocells personalized cell therapy program.
As part of the financing deal, Third Security LLC will get two seats on Fibrocells board.
Personalized stem cell development fits into the broader category of personalized medicine, regarded as the future of medicine in which therapies will be better targeted to individuals and more effective.
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Biopharma with personalized cell therapy raises $45M in stock sale, forges collaboration deal
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Nuvilex Reports Additional Safety and Efficacy Data From Second Phase 2 Pancreatic Cancer Trial
SILVER SPRING, Md., Oct. 8, 2012 (GLOBE NEWSWIRE) -- Nuvilex, Inc. (NVLX), an international biotechnology provider of cell and gene therapy solutions, announced the presentation today of additional safety and efficacy data from a second Phase 2 pancreatic cancer clinical trial. The new trial data was presented by Dr. John Dangerfield, COO of the Nuvilex subsidiary Austrianova Singapore Pte Ltd (ASPL) at the ISCGT Meeting. The trial data presented used the same encapsulated cytochrome P450 expressing cells followed by chemotherapy to treat pancreatic cancer that were used in the previously published trial and confirmed results generated in the previous trial.
The Phase 2 pancreatic cancer trial approach being advanced by Nuvilex, and its subsidiary ASPL, works through the ability of special, cellulose sulphate encapsulated, living cells expressing cytochrome P450 CYP 2B1. These cells are able to convert the inactive chemotherapy drug ifosfamide to its active form at the site they are placed in. As a result, the encapsulated cells create a high level of drug in the area of the pancreatic tumor where the treatment can provide for the greatest anti-tumor effect, coupled with a significant reduction in side-effects since much lower doses of the chemotherapeutic drug need to be used.
The major findings reported at the ISCGT meeting revealed that safety and efficacy data from a total of 27 patients is now available, approximately doubling the number of patient data previously released. In this expanded patient group no immunological, capsule, or cell-related adverse events from the live cell encapsulated treatment approach were reported. Moreover and most importantly, the data presented confirmed the approximate doubling of median survival when compared to the data available for the current standard treatment, Gemzar. In addition, there was a doubling of the number of 1 year survivors treated with encapsulated cells plus chemotherapy compared to the standard Gemzar treatment. Another significant finding comes from the fact that the treatment procedure was successfully performed at 4 different cancer treatment centers in Europe by three different interventional radiologists.
ASPL's CEO, Dr Brian Salmons, said, "The additional data presented at the ISCGT meeting confirms the safety of the capsule material, the implantation procedure and the long term safety of implanting cells in the body inside the cellulose sulphate encapsulation material. Lastly, the survival benefit of the first trial in terms of median survival and 1 year survival was confirmed and therefore the importance of the treatment was verified."
Dr. Robert F. Ryan, CEO of Nuvilex, said, "This data provides us with additional confidence as we undertake the next steps in the development of the treatment of pancreatic cancer as well as other solid tumors."
About Nuvilex
Nuvilex, Inc. (NVLX) is an international biotechnology provider of live therapeutically valuable, encapsulated cells and services for research and medicine. Substantial progress in multiple areas will be providing the Company with increased potential and we look forward to bringing those forward shortly. Our company's clinical offerings will include cancer, diabetes and other treatments using the company's cell and gene therapy expertise and live-cell encapsulation technology.
The Nuvilex, Inc. logo is available at http://www.globenewswire.com/newsroom/prs/?pkgid=13494
Safe Harbor Statement
This press release contains forward-looking statements described within the 1995 Private Securities Litigation Reform Act involving risks and uncertainties including product demand, market competition, and meeting current or future plans which may cause actual results, events, and performances, expressed or implied, to vary and/or differ from those contemplated or predicted. Investors should study and understand all risks before making an investment decision. Readers are recommended not to place undue reliance on forward-looking statements or information. Nuvilex is not obliged to publicly release revisions to any forward-looking statement, reflect events or circumstances afterward, or disclose unanticipated occurrences, except as required under applicable laws.
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Nuvilex Reports Additional Safety and Efficacy Data From Second Phase 2 Pancreatic Cancer Trial
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Scientists develop 'barcode' blood test for aggressive prostate cancer
Public release date: 8-Oct-2012 [ | E-mail | Share ]
Contact: Jane Bunce, ICR Science Communuications Manager jane.bunce@icr.ac.uk 44-207-153-5106 Institute of Cancer Research
Scientists have designed a blood test that reads genetic changes like a barcode and can pick out aggressive prostate cancers by their particular pattern of gene activity.
A team at The Institute of Cancer Research, London, and The Royal Marsden NHS Foundation Trust found reading the pattern of genes switched on and off in blood cells could accurately detect which advanced prostate cancers had the worst survival.
And the researchers believe the blood test could eventually be used alongside the existing PSA test at diagnosis to select patients who need immediate treatment.
The test, described in The Lancet Oncology today (Tuesday 9 October), is unique because it assesses changes in the pattern of gene activity in blood cells triggered by a tumour elsewhere in the body.
Study senior author Professor Johann de Bono, leader of the prostate cancer targeted therapy team at The Institute of Cancer Research, London, and honorary consultant at The Royal Marsden NHS Foundation Trust, said: "Prostate cancer is a very diverse disease some people live with it for years without symptoms but for others it can be aggressive and life-threatening so it's vital we develop reliable tests to tell the different types apart.
"We've shown it is possible to learn more about prostate cancers by the signs they leave in the blood, allowing us to develop a test that is potentially more accurate than those available now and easier for patients than taking a biopsy. Our test reads the pattern of genetic activity like a barcode, picking up signs that a patient is likely to have a more aggressive cancer. Doctors should then be able to adjust the treatment they give accordingly."
Researchers scanned all the genes present in blood samples from 100 patients with prostate cancer at the ICR's and The Royal Marsden's joint Drug Development Unit in London and The Beatson West of Scotland Cancer Centre in Glasgow. They included 69 patients with advanced cancer and 31 control patients thought to have low-risk, early-stage cancer, who were being managed by active surveillance.
Using statistical modelling, they divided the patients into four groups reflecting their pattern of gene activity the barcode. When they reviewed all the patients' progress after almost two-and-a-half years, they found patients in one group had survived for significantly less time than patients in the others. Further modelling identified nine key active genes that were shared by all patients in the group.
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Scientists develop 'barcode' blood test for aggressive prostate cancer
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Scientists discover gene 'bursting' plays key role in protein production
Scientists at the Gladstone Institutes have mapped the precise frequency by which genes get turned on across the human genome, providing new insight into the most fundamental of cellular processesand revealing new clues as to what happens when this process goes awry.
In a study being published this week online in the Proceedings of the National Academy of Sciences, Gladstone Investigator Leor Weinberger, PhD, and his research team describe how a gene's on-and-off switchingcalled "bursting"is the predominant method by which genes make proteins. By gaining an understanding of this underlying mechanism, this discovery has the potential to vastly help researchers learn what happens at the molecular level when this mechanism is disruptedsuch as in cancer or when exposed to a particular drug.
The manufacture, or synthesis, of proteins takes place inside every cell. DNA and geneswhich house the instructions for making proteinsare stored within the nucleus of each cell. When a gene is switched on, those instructions are transcribed as a copy onto RNA, another type of genetic material that then directs the protein synthesis. Proteins perform a variety of functions within the cellfrom the breaking down and digesting fats to resisting foreign invaders, such as bacteria or viruses. The timing and frequency with which a particular protein is synthesized is crucial to maintaining the health of the cell.
"Much like flicking on a light switch, genes get 'switched on' at specific intervals to initiate the fundamental biological process of protein synthesis," said Dr. Weinberger, who is also an associate professor at the University of California, San Francisco (UCSF), with which Gladstone is affiliated. "Until recently, the process was thought to be continuousonce a gene is switched on, it stays on, churning out protein products at a steady pace like a garden hose. But recently, some studies have suggested the oppositethat DNA produces RNA molecules in rapid-fire 'staccato' bursts. We decided to investigate how common this rapid-fire bursting was across the genome."
In laboratory experiments, Dr. Weinberger and his team inserted a green fluorescent protein, or "vector," into the DNA of Jurkat T lymphocytesa type of white blood cell that helps maintain a healthy human immune system. From this they generated new cells in which the vector was integrated into any one of thousands of gene segmentswith each segment glowing green when it was activated, or "switched on." This allowed the researchers to see exactly how gene activation occurred across the entire human genome.
"Our analysis reveals support for the "bursting" hypothesisthe genes acted as a sort of strobe lighttranscribing RNA in rapid-fire bursts," said Roy Dar, PhD, a Gladstone postdoctoral fellow and one of the paper's lead authors. "We observed that the bursting frequency increases until, over time, it reaches a particular threshold. At that point higher protein levels are reached by increasing the size of the bursts, eventually coming to a halt when no more protein product is needed. These results are a huge step towards understanding the basic molecular mechanism behind gene regulation."
"Dr. Weinberger and colleagues have shown that there is a single rule governing the behavior of all genes in the genome. Their findings in human cells complement and extend similar findings made recently in other organisms," said Arjun Raj, PhD, assistant professor of bioengineering at the University of Pennsylvania and an expert in imaging single molecules within cells.
The team believes that this new-found understanding of this fundamental biological processthat genomic bursts account for the majority of instances of protein productionholds clues to discovering how the disruption of these bursts could be harmful.
"For example, in certain cancers, genes may be switched on at the wrong times, eventually leading to the formation of tumors," said Brandon Razooky, a Gladstone and UCSF graduate student and the paper's other lead author. "This is also a good example of how the basic science being performed here at Gladstone provides a solid foundation with which to prevent, treat and ultimately cure some of the world's most devastating diseases."
Journal reference: Proceedings of the National Academy of Sciences
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Maynooth geneticists make MS breakthrough
By Ed Carty
Monday, October 08, 2012
Scientists at NUI Maynooth have pinpointed how control and regulation of a specific gene could combat debilitating diseases like multiple sclerosis.
After three years of research the team is in the early stages of trying to uncover how to manipulate the Pellino3 gene to tackle serious illnesses.
Research published in the US journal Nature Immunology showed the genes critical function is to regulate the amount of protective proteins released by immune systems to fight a virus.
Professor Paul Moynagh said it was a significant breakthrough in viralimmunology.
"The research has much potential for the treatment of major auto-immune diseases and the next step is to determine how the exploitation of Pellino3 can physiologically impact on specific conditions," he said.
NUI Maynooth discovered that Pellino3 acts like a brake to stop overproduction of interferons, the protective proteins.
If the gene is not working as designed, overproduction of interferons can lead to debilitating inflammatory diseases such as lupus while those of us who do not produce enough are more at risk of MS and hepatitis.
The research paper says controlling production linked to Pellino3 is critical in the treatment of viral disease.
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Maynooth geneticists make MS breakthrough
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Gene variant linked with reduced lung cancer risk
ScienceDaily (Oct. 8, 2012) A variant in a gene involved with inflammation and the immune response is linked with a decreased risk of lung cancer. That is the finding of an analysis published early online in CANCER, a peer-reviewed journal of the American Cancer Society. The results add to the growing body of literature implicating these processes in the development of lung cancer.
Meredith Shiels, PhD, MHS and Anil Chaturvedi, PhD, of the National Cancer Institute in Rockville, MD, and their colleagues analyzed 1,429 variants in inflammation- and immunity-related genes from 378 patients with lung cancer and 450 healthy controls from the Prostate, Lung, Colorectal and Ovarian (PLCO) cancer screening trial. The investigators observed a significant link between lung cancer and 81 single nucleotide polymorphisms (SNPs) located in 44 genes.
They then compared these results with observed or imputed data from four recently completed genome-wide association studies (GWAS) that included 5,739 lung cancer cases and 5,848 controls. Of the 81 SNPs, one in particular -- named rs4648127 and located within the NFKB1 gene -- was associated with lung cancer in both analyses. This SNP was linked with an estimated 44 percent reduced risk of lung cancer in the cancer screening trial and a 21 percent reduced risk in the combined GWAS analysis.
The NF-B, or nuclear factor kappa B, protein that is produced in part from the NFKB1 gene is known to play an important role in immunity and inflammation by regulating gene expression, cell death, and cell proliferation. Also, previous research has shown that immunity and inflammation may affect the development of lung cancer. "Our study provides further evidence that inflammation may be associated with lung cancer risk," said Dr. Shiels. She added that future studies should further examine the NFKB1 gene and its relationship with lung cancer risk.
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Gene variant linked with reduced lung cancer risk
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