At 6th Annual Clinical Trial Supply New England 2017 Conference in Boston Asymmetrex Introduces A First Specific … – Benzinga
By daniellenierenberg
On March 8-9 in Boston, stem cell medicine biotechnology start-up Asymmetrex led attendees at the 6th Annual Clinical Trials Supply New England 2017 conference in discussions about the need for quality controls for the supply of tissue stem cells used for treatments in either FDA-approved clinical trials or unregulated private stem cell clinics. Though these two stem cell treatment settings are often contrasted regarding their safety and effectiveness, Asymmetrex stressed that patient care and research progress is compromised in both because of the lack of essential quality control tests for the number and quality of transplanted tissue stem cells.
Boston, MA (PRWEB) March 14, 2017
At the 6th Annual Clinical Trials Supply New England 2017 conference, held in Boston from March 8-9, James Sherley, M.D., Ph.D., director of Asymmetrex, led discussions that evaluated the quality of U.S. supplies of stem cells used in clinical trials compared to private stem cell clinics. Private stem cell clinics have been criticized for not employing research standards that are necessary to establish the therapeutic effectiveness of treatments with statistical confidence. In part because of this difference in practice, they are also often accused of making unproven claims about the effectiveness of their therapies.
Sherley presented comparisons of key operational elements to argue that, given good intent in both settings, the two different settings of stem cell treatments had both distinct and shared shortcomings. He noted, however, that the most significant shortcoming, which stem cell clinical trials and private stem cell clinics share, was perennially overlooked.
Based on the number of reported stem cell clinical trials and private stem cell clinics, Sherley estimated that close to a quarter-million patients in the U.S. now receive stem cell treatments each year. Though many of these occur within FDA-approved clinical trials, their number is dwarfed nearly 10 times by the number of treatments that occur in private stem cell clinics. It shocked the audience of clinical trial suppliers to learn that there was no stem cell quality control test performed for any of these many treatments.
Even for approved stem cell medicine treatments like bone marrow transplantation and umbilical cord blood transplant, there is no stem cell-specific quality control test available. Counts of total cells are made, but these do not adequately predict stem cell number or function. Biomarkers designated for tissue stem cells are also expressed by stem cells' more abundant non-stem cell products. So, the biomarkers lack sufficient specificity to be used to count and monitor tissue stem cell function.
Without a quality control test for tissue stem cell number, stem cell treatments in all settings proceed without knowing the dose of treating tissue stem cells. This previously unavoidable therapeutic blind spot creates an instant treatment risk. It also precludes effective analyses to optimize treatment procedures, to compare different treatments, or to relate treatment outcomes to tissue stem cell dose. Without knowing stem cell dose, the interpretation of any stem cell treatment in terms of stem cells as the responsible agents is compromised.
In this context, Sherley announced briefly to attendees that Asymmetrex's new AlphaSTEM Test for counting adult tissue stem cells and providing data on their viability and tissue cell renewal function represented the needed first quality control test for tissue stem cell treatments, whether in clinical trials, in private stem cell clinics, or approved therapies. In particular, he indicated that both stem cell treatment patients and progress in stem cell medicine would benefit from existing clinical trial supply companies developing into future private stem cell clinic supply companies to insure the quality of stem cell treatment preparations. Sherley said that, of course, their partnership with Asymmetrex to implement its new stem cell-specific quality control test was an all around best solution for accelerating progress in stem cell transplantation medicine.
About Asymmetrex
Asymmetrex, LLC is a Massachusetts life sciences company with a focus on developing technologies to advance stem cell medicine. Asymmetrex's founder and director, James L. Sherley, M.D., Ph.D. is an internationally recognized expert on the unique properties of adult tissue stem cells. The company's patent portfolio contains biotechnologies that solve the two main technical problems production and quantification that have stood in the way of successful commercialization of human adult tissue stem cells for regenerative medicine and drug development. In addition, the portfolio includes novel technologies for isolating cancer stem cells and producing induced pluripotent stem cells for disease research purposes. Currently, Asymmetrex's focus is employing its technological advantages to develop and market facile methods for monitoring adult stem cell number and function in stem cell transplantation treatments and in pre-clinical assays for drug safety.
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At 6th Annual Clinical Trial Supply New England 2017 Conference in Boston Asymmetrex Introduces A First Specific ... - Benzinga
‘Butterfly Boy’ steels himself for second stem-cell transplant | Ottawa … – Ottawa Sun
By daniellenierenberg
Ottawa Sun | 'Butterfly Boy' steels himself for second stem-cell transplant | Ottawa ... Ottawa Sun Bracing for his second stem-cell transplant in seven months, Jonathan Pitre knows all too well the mountain in front of him, its hardships and precipices. |
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'Butterfly Boy' steels himself for second stem-cell transplant | Ottawa ... - Ottawa Sun
‘Butterfly Boy’ steels himself for second stem-cell transplant – Ottawa Citizen
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Ottawa Citizen | 'Butterfly Boy' steels himself for second stem-cell transplant Ottawa Citizen Bracing for his second stem-cell transplant in seven months, Jonathan Pitre knows all too well the mountain in front of him, its hardships and precipices. So he's doing ... Pitre will face the transplant alongside his mother, Tina Boileau, who will ... |
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'Butterfly Boy' steels himself for second stem-cell transplant - Ottawa Citizen
Applied StemCell Announces the Appointment of Dr. Michele Calos, Stanford Professor and Vice President of the … – Yahoo Finance
By daniellenierenberg
MILPITAS, Calif.--(BUSINESS WIRE)--
Applied StemCell (ASC), a leading stem cell and genome-editing company with a goal to advance genome editing and stem cell technologies for biomedical research and clinical applications, welcomes Dr. Michele Calos as a member of the companys Scientific Advisory Board (SAB).
Dr. Michele Calos is a Professor of Genetics at the Stanford University School of Medicine, Vice President of the American Society of Gene and Cell Therapy, and has served as an Advisory Committee member for the US FDA, grant review panels for the NIH and NSF, and on numerous editorial review committees of scientific journals. She is a leader in the field of molecular genetics and has developed several novel vector systems for genetic manipulation of mammalian cells. In particular, she developed novel methods for sequence-specific integration in mammalian cells using the C31 phage integrase system. A similar integrase system was also successfully used in site-specific integration in human ES and iPS cells. For this work, Dr. Calos holds a joint patent application with Applied StemCells Chief Scientific Officer, Dr. Ruby Yanru Chen-Tsai and several other Stanford researchers. Dr. Calos pioneering work with C31 integrase also set the scientific stage for ASCs TARGATT integrase technology, which was co-developed by Dr. Chen-Tsai and Dr. Liqun Luo of Stanford University for gene modification in mouse models.
We are extremely pleased to have Dr. Calos join as a member of our scientific advisory board. With her impressive background in integrase gene modification technology and gene therapy, Dr. Calos will be an invaluable guide in furthering expansion of our genome editing platforms and our gene/cell therapy pipeline, said Ruby Yanru Chen-Tsai, Ph.D., Co-founder and Chief Scientific Officer of Applied StemCell.
Dr. Calos and her research team are currently focused on gene therapy and genome engineering for the treatment of Duchenne and Limb Girdle Muscular Dystrophies and developing further novel strategies for gene and cell therapy.
About Applied StemCell, Inc.
Applied StemCell, Inc. is a leading stem cell and gene-editing company focused on the development of products and therapeutics that are enabled by its proprietary gene editing platform technologies TARGATT and CRISPR/Cas9. For more information, please visit http://www.appliedstemcell.com.
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Applied StemCell Announces the Appointment of Dr. Michele Calos, Stanford Professor and Vice President of the ... - Yahoo Finance
Neuralstem Announces Publication of NSI-566 Data in a Rodent Model of Traumatic Brain Injury – GlobeNewswire (press release)
By daniellenierenberg
March 09, 2017 07:03 ET | Source: Neuralstem, Inc.
-NSI-566 Achieved Robust Engraftment and Long-Term Survival After Transplantation-
- Data Published in Journal of Neurotrauma-
GERMANTOWN, Md., March 09, 2017 (GLOBE NEWSWIRE) -- Neuralstem, Inc. (Nasdaq:CUR), a biopharmaceutical company focused on the development of nervous system therapies based on its neural stem cell technology, announced the recent publication of preclinical data on NSI-566 spinal cord-derived neural stem cells in Journal of Neurotrauma. These data showed robust engraftment and long-term survival of NSI-566 post transplantation in a rat model of penetrating ballistic-like brain injury (PBBI). NSI-566 is Neuralstems lead stem cell therapy candidate.
The study entitled, Amelioration of penetrating ballistic-like brain injury induced cognitive deficits after neuronal differentiation of transplanted human neural stem cells," was led by Ross Bullock, M.D., Ph.D., The Miami Project to Cure Paralysis, University of Miami School of Medicine. These are the first data from the 4-year proof-of-concept research program, funded by the United States Department of Defense, for NSI-566 in traumatic brain injury.
These data on NSI-566 are encouraging, particularly since researchers have long been challenged to achieve durable engraftment and survival of neural stem cells after transplantation, said Dr. Bullock. No long-term treatment beyond physical therapy is currently available to restore cognition after a traumatic brain injury. Transplantation of stem cells into the injured brain may allow a unique replacement therapy and fill a significant medical need.
Researchers transplanted NSI-566 into rats 7-10 days after PBBI. The rats were immunosuppressed to enable survival of NSI-566 neural stem cells. Robust engraftment with evidence of prominent neuronal differentiation was observed after 4 months, and axons from grafted cells extended a significant distance from the graft site along host white matter tracts.
These data continue to support our research and development platform. The results provide additional insight into our proprietary regionally specific stem cells and their potential benefits in nervous system disorders, said Karl Johe, Ph.D., Chief Scientific Officer, Neuralstem. We look forward to additional preclinical data from this collaboration with Dr. Bullocks group to support the potential use of NSI-566 in traumatic brain injury.
About Neuralstem Neuralstems patented technology enables the commercial-scale production of multiple types of central nervous system stem cells, which are being developed as potential therapies for multiple central nervous system diseases and conditions.
Neuralstems technology enables the discovery of small molecule compounds by systematic screening chemical compounds against its proprietary human hippocampal stem cell line. The screening process has led to the discovery and patenting of molecules that Neuralstem believes may stimulate the brains capacity to generate new neurons, potentially reversing pathophysiologies associated with certain central nervous system (CNS) conditions.
The company has completed Phase 1a and 1b trials evaluating NSI-189, a novel neurogenic small molecule product candidate, for the treatment of major depressive disorder or MDD, and is currently conducting a Phase 2 efficacy study for MDD.
Neuralstems stem cell therapy product candidate, NSI-566, is a spinal cord-derived neural stem cell line. Neuralstem is currently evaluating NSI-566 in three indications: stroke, chronic spinal cord injury (cSCI), and Amyotrophic Lateral Sclerosis (ALS).
Neuralstem is conducting a Phase 1 safety study for the treatment of paralysis from chronic motor stroke at the BaYi Brain Hospital in Beijing, China. In addition, NSI-566 was evaluated in a Phase 1 safety study to treat paralysis due to chronic spinal cord injury as well as a Phase 1 and Phase 2a risk escalation, safety trials for ALS. Subjects from all three indications are currently in long-term observational follow-up periods to continue to monitor safety and possible therapeutic benefits.
Cautionary Statement Regarding Forward Looking Information This news release contains forward-looking statements made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements relate to future, not past, events and may often be identified by words such as expect, anticipate, intend, plan, believe, seek or will. Forward-looking statements by their nature address matters that are, to different degrees, uncertain. Specific risks and uncertainties that could cause our actual results to differ materially from those expressed in our forward-looking statements include risks inherent in the development and commercialization of potential products, uncertainty of clinical trial results or regulatory approvals or clearances, need for future capital, dependence upon collaborators and maintenance of our intellectual property rights. Actual results may differ materially from the results anticipated in these forward-looking statements. Additional information on potential factors that could affect our results and other risks and uncertainties are detailed from time to time in Neuralstems periodic reports, including the Annual Report on Form 10-K for the year ended December 31, 2015, and Form 10-Q for the nine months ended September 30, 2016, filed with the Securities and Exchange Commission (SEC), and in other reports filed with the SEC. We do not assume any obligation to update any forward-looking statements.
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Neuralstem Announces Publication of NSI-566 Data in a Rodent Model of Traumatic Brain Injury - GlobeNewswire (press release)
Sickle cell anemia patient ‘cured’ by gene therapy, doctors say – FOX31 Denver
By daniellenierenberg
FOX31 Denver | Sickle cell anemia patient 'cured' by gene therapy, doctors say FOX31 Denver Essentially, researchers extracted bone marrow from the patient, harvested the stem cells and altered the genetic instructions so that they would make normal hemoglobin. Next, they treated the patient with chemotherapy for four days to eliminate his ... Gene therapy shows early promise against sickle cell Doctors Claim They've Cured a Boy of a Painful Blood Disorder ... Will Sickle Cell Be the Next Disease Genetic Engineering Cures? |
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Sickle cell anemia patient 'cured' by gene therapy, doctors say - FOX31 Denver
This simple method can predict a stem cell’s fate – Futurity: Research News
By daniellenierenberg
Scientists have created an easy way to identify the state and fate of stem cells earlier than previously possible.
Understanding a stem cells fatethe type of cell it will eventually becomeand how far along it is in the process of development can help scientists better manipulate cells for stem cell therapy.
Having the ability to visualize a stem cells future will take some of the questions out of using stem cells to help regenerate tissue and treat diseases.
The beauty of the method is its simplicity and versatility, says Prabhas V. Moghe, a professor of biomedical engineering and chemical and biochemical engineering at Rutgers and senior author of a study published recently in the journal Scientific Reports. It will usher in the next wave of studies and findings.
Existing methods look at the overall population of cells but arent specific enough to identify individual cells fates. But when implanting stem cells (during a bone marrow transplant following cancer treatment, for example), knowing that each cell will become the desired cell type is essential.
Also, many protein markers used to distinguish cell types dont show up until after the cell has transitioned, which can be too late for some applications.
To identify earlier signals of a stem cells fate, scientists used super-resolution microscopy to analyze epigenetic modifications. Epigenetic modifications change how DNA is wrapped up within the nucleus, allowing different genes to be expressed.
Some modifications signal that a stem cell is transitioning into a particular type of cell, such as a blood, bone or fat cell. Using the new method, the team of scientists was able to determine a cells fate days before other techniques.
Having the ability to visualize a stem cells future will take some of the questions out of using stem cells to help regenerate tissue and treat diseases, says Rosemarie Hunziker, program director for tissue engineering and regenerative medicine at the National Institute of Biomedical Imaging and Bioengineering. Its a relatively simple way to get a jump on determining the right cells to use.
The approach, called EDICTS (Epi-mark Descriptor Imaging of Cell Transitional States), involves labeling epigenetic modifications and then imaging the cells with super resolution to see the precise location of the marks.
Were able to demarcate and catch changes in these cells that are actually not distinguished by established techniques such as mass spectrometry, Moghe says.
He described the method as fingerprinting the guts of the cell, and the results are quantifiable descriptors of each cells organization (for example, how particular modifications are distributed throughout the nuclei).
The team demonstrated the methods capabilities by measuring two types of epigenetic modifications in the nuclei of human stem cells cultured in a dish. They added chemicals that coaxed some of the cells to become fat cells and others to become bone, while another set served as control.
Within three days, the localization of the modifications varied in cells destined for different fates, two to four days before traditional methods could identify such differences between the cells. The technique had the specificity to look at regional changes within individual cells, while existing techniques can only measure total levels of modifications among the entire population of cells.
The levels are not significantly different, but how theyre organized is different and that seems to correlate with the fact that these cells are actually exhibiting different fates, Moghe says. It allows us to take out a single cell from a population of dissimilar cells, which can help researchers select particular cells for different stem cell applications.
The method is as easy as labeling, staining, and imaging cellstechniques already familiar to many researchers, he says. As the microscopes capable of super resolution imaging become more widely available, scientists can use it to sort and screen different types of cells, understand how a particular drug may disrupt epigenetic signaling, or ensure that stem cells to be implanted wont transform into the wrong cell type.
Collaborators are from Stanford University School of Medicine, Case Western Reserve University, Seoul National University, Princeton University, the University of Akron, the University of Pennsylvania, and MIT.
Source: Teal Burrell for the National Institute of Biomedical Imaging and Bioengineering via Rutgers University
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Gene therapy shows early promise against sickle cell – Chicago Tribune
By daniellenierenberg
Researchers are reporting early success using gene therapy to treat, or even potentially cure, sickle cell anemia.
The findings come from just one patient, a teenage boy in France. But more than 15 months after receiving the treatment, he remained free of symptoms and his usual medications.
That's a big change from his situation before the gene therapy, according to his doctors at Necker Children's Hospital in Paris.
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For years, the boy had been suffering bouts of severe pain, as well as other sickle cell complications that affected his lungs, bones and spleen.
Medical experts stressed, however, that much more research lies ahead before gene therapy can become an option for sickle cell anemia.
It's not clear how long the benefits will last, they said. And the approach obviously has to be tested in more patients.
"This is not right around the corner," said Dr. George Buchanan, a professor emeritus of pediatrics at the University of Texas Southwestern Medical Center in Dallas.
That said, Buchanan called the results a "breakthrough" against a disease that can be debilitating and difficult to treat.
Buchanan, who wasn't involved in the research, helped craft the current treatment guidelines for sickle cell.
"This is what people have been wanting and waiting for," he said. "So it's exciting."
Sickle cell anemia is an inherited disease that mainly affects people of African, South American or Mediterranean descent. In the U.S., about 1 in 365 black children is born with the condition, according to the U.S. National Heart, Lung, and Blood Institute.
It arises when a person inherits two copies of an abnormal hemoglobin gene one from each parent. Hemoglobin is an oxygen-carrying protein in the body's red blood cells.
When red blood cells contain "sickle" hemoglobin, they become crescent-shaped, rather than disc-shaped. Those abnormal cells tend to be sticky and can block blood flow causing symptoms such pain, fatigue and shortness of breath. Over time, the disease can damage organs throughout the body.
There are treatments for sickle cell, such as some cancer drugs, Buchanan pointed out, but they can be difficult to manage and have side effects.
There is one potential cure for sickle cell, Buchanan said: a bone marrow transplant. In that procedure, doctors use chemotherapy drugs to wipe out the patient's existing bone marrow stem cells which are producing the faulty red blood cells. They are then replaced with bone marrow cells from a healthy donor.
A major problem, Buchanan said, is that the donor typically has to be a sibling who is genetically compatible and free of sickle cell disease.
"We've known for a long time that bone marrow transplants can work," Buchanan said. "But most patients don't have a donor."
That's where gene therapy could fit in. Essentially, the aim is to genetically alter patients' own blood stem cells so they don't produce abnormal hemoglobin.
In this case, the French team led by Dr. Marina Cavazzana focused on a gene called beta globin. In sickle cell anemia, beta globin is mutated.
First, the researchers extracted a stem cell supply from their teen patient's bone marrow, before using chemotherapy to wipe out the remaining stem cells.
Then they used a modified virus to deliver an "anti-sickling" version of the beta globin gene into the stem cells they'd removed pre-chemo. The modified stem cells were infused back into the patient.
Over the next few months, the boy showed a growing number of new blood cells bearing the mark of the anti-sickling gene. The result was that roughly half of his hemoglobin was no longer abnormal.
In essence, Buchanan explained, the therapy "converted" the patient to sickle-cell trait that is, a person who carries only one copy of the abnormal hemoglobin gene. Those individuals don't develop sickle cell disease.
"This is encouraging," said Dr. David Williams, president of the Dana-Farber/Boston Children's Cancer and Blood Disorders Center.
But, he cautioned, "the caveat is, this is one patient, and 15 months is a short follow-up."
Williams and his colleagues are studying a different approach to sickle cell gene therapy. It aims to restart the body's production of healthy fetal hemoglobin to replace the abnormal "adult" hemoglobin seen in sickle cell.
If gene therapy is proven to work, there will no doubt be practical obstacles to its widespread use, according to Buchanan. It's a high-tech treatment, and many sickle cell patients are low-income and far from a major medical center, he said.
But, Buchanan said, the new findings have now "opened a door."
The study was partly funded by Bluebird Bio, the company developing the therapy.
The results were published in March in the New England Journal of Medicine.
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Gene therapy shows early promise against sickle cell - Chicago Tribune
Doctors Claim They’ve Cured a Boy of a Painful Blood Disorder Using Gene Therapy – Futurism
By daniellenierenberg
Potential Treatment
Gene therapy has been available for quite some time now. Advances in modern medical science, particularly in stem cell research, have made it possible to use DNA to compensate for malfunctioning genes in humans. The therapies haveeven proven effective fortreating rare forms of diseases. Now, a research team in France has shown that gene therapy may be used to cure one of the most common genetic diseases in the world.
The team, led by Marina Cavazzana at the Necker Childrens Hospital in Paris, conducted stem cell treatment on a teenage boy with sickle cell disease. The disease alters theblood through beta-globin mutations, which cause abnormalities in the blood proteinhemoglobin. These abnormalities cause the blood cells (which have an irregular shape, like a sickle, hence their name) to clump together. Patients with sickle cell disease usually need transfusions to clear the blockages their cells cause, and some are able to have bone marrow transplants. About 5 percent of the global population has sickle cell disease,according to the WHO. In the United States alone, the CDC reports that approximately 100,000 people have sickle cell disease.
The patient is now 15 years old and free of all previous medication, Cavazzana saidwhen discussing the outcome of their study. He has been free of pain from blood vessel blockages, and has given up taking opioid painkillers. Their research is published in the the New England Journal of Medicine.
The particular treatment given to the teenage boy at Necker Childrens Hospitalbegan when he was 13 years old. The team took bone marrow stem cells from the boy and added mutated versions of the gene that codes for beta-globin before putting these stem cells back into the boys body. The mutated genes were designed to stop hemoglobin from clumping together and blocking blood vessels the hallmark of sickle cell disease.
Two years later, the boys outcomelooks promising.All the tests we performed on his blood show that hes been cured, but more certainty can only come from long-term follow-up, Cavazzan said. Her team also treated seven other patients who also showed promising progress.
If the method shows success in larger scale clinical trials, it could be a game changer, saidDeborah Gill at the University of Oxford, The fact the team has a patient with real clinical benefit, and biological markers to prove it, is a very big deal.
Other research involving gene therapy is also showing similar promise. One which has already been approved by the FDA is a potential treatment for blindness. Others look at treating Parkinsons disease or evenprolonging human life. What these studies show is that gene therapyand stem cells may be able togive hope to patients with diseases that have long been considered incurable.
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Doctors Claim They've Cured a Boy of a Painful Blood Disorder Using Gene Therapy - Futurism
Johns Hopkins Medicine, Maryland Stem Cell Research Fund and … – Business Wire (press release)
By daniellenierenberg
SAN CARLOS, Calif. & BALTIMORE--(BUSINESS WIRE)--Johns Hopkins Medicine, the Maryland Stem Cell Research Fund (MSCRF) and BioCardia, Inc. (OTC:BCDA) today announced that the first patient has been treated in the pivotal Phase III CardiAMP clinical trial of a cell-based therapy for the treatment of ischemic heart failure that develops after a heart attack. The first patient was treated at Johns Hopkins Hospital by a team led by Peter Johnston, MD, a faculty member in the Department of Medicine and Division of Cardiology, and principal investigator of the trial at Johns Hopkins.
The investigational CardiAMP therapy is designed to deliver a high dose of a patients own bone marrow cells directly to the point of cardiac dysfunction, potentially stimulating the bodys natural healing mechanism after a heart attack.
The patient experience with CardiAMP therapy begins with a pre-procedural cell potency screening test. If a patient qualifies for therapy, they are scheduled for a bone marrow aspiration. A point of care cell processing platform is then utilized to concentrate the autologous bone marrow cells, which are subsequently delivered in a minimally-invasive procedure directly to the damaged regions in a patients heart.
This cell-based therapy offers great potential for heart failure patients, said Carl Pepine, MD, professor and former chief of cardiovascular medicine at the University of Florida, Gainesville and national co-principal investigator of the CardiAMP trial. We look forward to validating the impact of the therapy on patients quality of life and functional capacity in this important study.
In addition to Dr. Johnston, the CardiAMP research team at Johns Hopkins includes Gary Gerstenblith, MD, Jeffrey Brinker, MD, Ivan Borrello, MD, Judi Willhide, Katherine Laws, Audrey Dudek, Michele Fisher and John Texter, as well as the nurses and technicians of the Johns Hopkins Cardiovascular Interventional Laboratory.
Funding the clinical trial of this cell therapy, which could be the first cardiac cell therapy approved in the United States, is an important step towards treatments, said Dan Gincel, PhD., executive director of the MSCRF at TEDCO. Through our clinical program, we are advancing cures and improving healthcare in the State of Maryland.
The CardiAMP Heart Failure Trial is a phase III, multi-center, randomized, double-blinded, sham-controlled study of up to 260 patients at up to 40 centers nationwide, which includes an optional 10-patient roll-in cohort. The primary endpoint for the trial is a significant improvement in Six Minute Walk distance at 12 months post-treatment. Study subjects must be diagnosed with New York Heart Association (NYHA) Class II or III heart failure as a result of a previous heart attack. The national co-principal investigators are Dr. Pepine and Amish Raval, MD, of the University of Wisconsin.
For information about eligibility or enrollment in the trial, please visit http://www.clinicaltrials.gov or ask your cardiologist.
About BioCardia BioCardia, Inc., headquartered in San Carlos, CA, is developing regenerative biologic therapies to treat cardiovascular disease. CardiAMP and CardiALLO cell therapies are the companys biotherapeutic product candidates in clinical development. For more information, visit http://www.BioCardia.com.
About Johns Hopkins Medicine Johns Hopkins Medicine (JHM), headquartered in Baltimore, Maryland, is one of the leading health care systems in the United States. Johns Hopkins Medicine unites physicians and scientists of the Johns Hopkins University School of Medicine with the organizations, health professionals and facilities of The Johns Hopkins Hospital and Health System. For more information, visit http://www.hopkinsmedicine.org.
About Maryland Stem Cell Research Fund The Maryland Stem Cell Research Act of 2006was established by the Governor and the Maryland General Assembly during the 2006 legislative session and created the Maryland Stem Cell Research Fund. This fund is continued through an appropriation in the Governor's annual budget. The purpose of the Fund is to promote state-funded stem cell research and cures through grants and loans to public and private entities in the State. For more information, visit http://www.MSCRF.org.
Forward Looking Statements This press release contains forward-looking statements as that term is defined under the Private Securities Litigation Reform Act of 1995. Such forward-looking statements include, among other things, references to the enrollment of our Phase 3 trial, commercialization and efficacy of our products and therapies, the product development timelines of our competitors. Actual results could differ from those projected in any forward-looking statements due to numerous factors. Such factors include, among others, the inherent uncertainties associated with developing new products or technologies, unexpected expenditures, the ability to raise the additional funding needed to continue to pursue BioCardias business and product development plans, competition in the industry in which BioCardia operates and overall market conditions, and whether the combined funds will support BioCardias operations and enable BioCardia to advance its pivotal Phase 3 CardiAMP cell therapy program. These forward-looking statements are made as of the date of this press release, and BioCardia assumes no obligation to update the forward-looking statements.
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Johns Hopkins Medicine, Maryland Stem Cell Research Fund and ... - Business Wire (press release)
Super resolution imaging helps determine a stem cell’s future – Phys.Org
By daniellenierenberg
February 27, 2017 Human mesenchymal stem cells were labeled for two epigenetic marks (green and red), and the images were analyzed to forecast the cell developmental fate. Credit: Joseph J. Kim
Scientists at Rutgers and other universities have created a new way to identify the state and fate of stem cells earlier than previously possible.
Understanding a stem cell's fatethe type of cell it will eventually becomeand how far along it is in the process of development can help scientists better manipulate cells for stem cell therapy.
The beauty of the method is its simplicity and versatility, said Prabhas V. Moghe, distinguished professor of biomedical engineering and chemical and biochemical engineering at Rutgers and senior author of a study published recently in the journal Scientific Reports. "It will usher in the next wave of studies and findings," he added.
Existing approaches to assess the states of stem cells look at the overall population of cells but aren't specific enough to identify individual cells' fates. But when implanting stem cells (during a bone marrow transplant following cancer treatment, for example), knowing that each cell will become the desired cell type is essential. Furthermore, many protein markers used to distinguish cell types don't show up until after the cell has transitioned, which can be too late for some applications.
To identify earlier signals of a stem cell's fate, an interdisciplinary team from multiple universities collaborated to use super-resolution microscopy to analyze epigenetic modifications. Epigenetic modifications change how DNA is wrapped up within the nucleus, allowing different genes to be expressed. Some modifications signal that a stem cell is transitioning into a particular type of cell, such as a blood, bone or fat cell. Using the new method, the team of scientists was able to determine a cell's fate days before other techniques.
"Having the ability to visualize a stem cell's future will take some of the questions out of using stem cells to help regenerate tissue and treat diseases," says Rosemarie Hunziker, program director for Tissue Engineering and Regenerative Medicine at the National Institute of Biomedical Imaging and Bioengineering. "It's a relatively simple way to get a jump on determining the right cells to use."
The approach, called EDICTS (Epi-mark Descriptor Imaging of Cell Transitional States), involves labeling epigenetic modifications and then imaging the cells with super resolution to see the precise location of the marks.
"We're able to demarcate and catch changes in these cells that are actually not distinguished by established techniques such as mass spectrometry," Moghe said. He described the method as "fingerprinting the guts of the cell," and the results are quantifiable descriptors of each cell's organization (for example, how particular modifications are distributed throughout the nuclei).
The team demonstrated the method's capabilities by measuring two types of epigenetic modifications in the nuclei of human stem cells cultured in a dish. They added chemicals that coaxed some of the cells to become fat cells and others to become bone, while another set served as control. Within three days, the localization of the modifications varied in cells destined for different fates, two to four days before traditional methods could identify such differences between the cells. The technique had the specificity to look at regional changes within individual cells, while existing techniques can only measure total levels of modifications among the entire population of cells.
"The levels are not significantly different, but how they're organized is different and that seems to correlate with the fact that these cells are actually exhibiting different fates," Moghe said. "It allows us to take out a single cell from a population of dissimilar cells," which can help researchers select particular cells for different stem cell applications.
The method is as easy as labeling, staining and imaging cells - techniques already familiar to many researchers, he said. As the microscopes capable of super resolution imaging become more widely available, scientists can use it to sort and screen different types of cells, understand how a particular drug may disrupt epigenetic signaling, or ensure that stem cells to be implanted won't transform into the wrong cell type.
Explore further: Super-resolution imaging can map critical cell changes several days sooner than current method
More information: Joseph J. Kim et al, Optical High Content Nanoscopy of Epigenetic Marks Decodes Phenotypic Divergence in Stem Cells, Scientific Reports (2017). DOI: 10.1038/srep39406
Scientists funded by the National Institutes of Health have developed a new way to identify the state and fate of individual stem cells earlier than previously possible. Stem cells are undifferentiated, serving as building ...
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An International Reserach Team coordinated by Igb-Cnr has discovered a key role of vitamins and amino acids in pluripotent stem cells. The research is published in Stem Cell Reports, and may provide new insights in cancer ...
A method that is used to track the fate of a single stem cell within mouse lung tissue is reported in a study published online this week in Nature Nanotechnology. The method may offer insights into the factors that determine ...
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To date, it has been assumed that the differentiation of stem cells depends on the environment they are embedded in. A research group at the University of Basel now describes for the first time a mechanism by which hippocampal ...
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Super resolution imaging helps determine a stem cell's future - Phys.Org
Former football player raising money for stem cell treatment … – Lexington Herald Leader
By daniellenierenberg
Former football player raising money for stem cell treatment ... Lexington Herald Leader A Danville native who hopes to be able to walk again is raising money for life-changing treatments. |
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Former football player raising money for stem cell treatment ... - Lexington Herald Leader
Durham scientists pioneer innovative stem cell research – Palatinate
By daniellenierenberg
By Jacob Dykes
In Durham, a pioneering technology has been developed which is providing a solution to fundamental issues in tissue engineering and stem cell biology. The development of new innovative technology enables the advancement of the research and discovery process and scientific thinking as a whole. For example, its hard to conceive of a biomedical sphere untouched by the blessing of PCR or DNA sequencing. Technological advancements not only offer solutions to existing obstacles, they open up new avenues of research into previously inconceivable areas.
With the current levels of excitement in the research of stem cell biology, you could be forgiven for envisaging a utopian medical scenario where a process akin to science-fiction allows us to generate complex tissues in a Petri-dish, ready for transplantation into the damaged organism. The scientific community has speculated that the nature of stem cells, in their ability to self-renew and produce cell types of any lineage will eventually provide medical solutions to some of our most vilified tissue diseases.
Transitioning speculation to reality requires time, basic research and technology development. A novel product known as Alvetex has been developed by Reinnervate, a Durham University spin-out company, which enables a new routine approach to study stem cells and their ability to form tissues in the laboratory. The product unlocks the potential of stem cell differentiation by mimicking the natural three-dimensional (3D) microenvironment cells encounter in the body, enabling the formation of 3D tissue-like structures.
Cell behaviour, in general, is guided by the complex 3D microenvironment in which they reside. Dispersal of cell-cell interactions and architectural contacts across the surface of the cell are essential for regulating gene expression, the genetic mechanism by which cells change their character and behaviour. Recreation of this microenvironment in the laboratory is essential to studying physiologically relevant behaviour, and the differentiation process by which cells form new cell types. Alvetex is a micro-engineered 3D polystyrene scaffold into which cells can be impregnated for cultivation. Cells grow within a 200-micron thick membrane of the 3D material bathed in culture medium. The microenvironment enables cells to form 3D contacts with neighbouring cells, recreating the more natural interactions found in real tissues. Overall, this affects the structure and function of the cells, enabling them to behave more like their native counterparts, which in turn improves predictive accuracy when working with advanced cell culture models.
We can take progenitor cells from the skin of donors and produce human skin We can take cell lines from the intestine and reproduce the absorptive lining of the intestine. We can take neural progenitors and recapitulate 3D neural networks.
Stefan Przyborski is a Professor of Cell Technology at Durham University and the founder of Reinnervate. He gave us an insight into his technologys applications;
We can take progenitor cells from the skin of donors and produce a full-thickness stratified human skin model (see image). We can take cell lines from the intestine and reproduce the absorptive lining of the intestine. We can take neural progenitors and recapitulate 3D neural networks to simulate aspects of nervous system function. Each of these models can be used to advance basic research, and extend our understanding of tissue development, and simulate aspects of disease.
Such technology is underpinned by well established fundamental principles such as how cellular structure is related to function, which hails way back to Da Vinci himself. It is well known that if you get the structure and the anatomy correct than the physiology will start to follow.
Alvetex technology has already been used in research that has led the publication of over 60 research papers in the field of tissue engineering and cancer biology. One particular group used the technology to successfully test drugs to prevent glioblastoma dispersal, an innovative application in brain oncology. Another has developed a 3D skin model to better study the development of metastatic melanoma, a persistently incurable invasive tumour of the skin. US scientists have used Alvetex on the International Space Station to study the formation of bone tissue in microgravity conditions.
The technology promises to be a cost-effective and ethical solution to current obstacles in cell culturing methods, producing better quality data relevant to man and reducing the need for animal models. Alvetex technology has offered a generational contribution to the process of tissue engineering research, yet the founder has higher ambitions;
What I would like to see in the next few decades is the increased complexity of the tissues that stem cells can be used to generate. If you consider the structure of an organ, the complexity, arrangement and structural organisation of those cell populations, it is far from where we are today. Advances in technology at the interface between disciplines leads to new innovative ideas to solve problems and open up new opportunities.
The development of stem cell research is an incremental process. We have to remain cautious given the potential of stem cell therapy to cause tumour formation, highlighting the need for more stringent models and controls. However, the clinical transplantation of cultured stem cells in bone and cornea repair demonstrates their enormous potential. Laboratory experiments have also demonstrated the potential of stem cells to produce kidney, pancreatic, liver, cardiac and muscle cells. It is hoped that continued research using more physiologically relevant technologies will increase the complexity of these tissues in the lab, and the diversity of their application.
Innovative technological advances play an important role in the process of biomedical science. Scientists at Durham are instrumental in the development of such new technologies that enable the process of new discoveries.
Photograph: Prof Stefan Przyborski, Durham University
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Durham scientists pioneer innovative stem cell research - Palatinate
Cardiac injury, recovery is topic of Osher lecture – Stowe Today
By daniellenierenberg
Dr. Jeffrey Spees, an associate professor of medicine at the University of Vermonts College of Medicine, will present Rescue and Repair of Cardiac Tissue After Injury: Turning Star Trek into Sesame Street, on Wednesday, March 1, at the Town and Country Resort, 876 Mountain Road, Stowe. Doors open at 1 p.m. and the lecture begins promptly at 1:30 p.m. This is the eighth Osher Lifelong Learning Institute lecture of the winter series.
Spees earned his Ph.D. in physiological and molecular ecology at the University of California, Davis. At UVM he teaches courses in developmental neurobiology, human structure and function and stem cells and regenerative medicine.
Spees has directed the Stem Cell Core in UVMs Department of Medicine and was one of the founding members of the New England Stem Cell Consortium. Spees and his colleagues have developed and applied for a patent for a therapy using a protein complex that is highly protective and keeps cells alive. He will discuss this research and its role in repairing cardiac tissue to improve cardiac function after a heart attack.
Vermont musicologist Joel Najman will present the final lecture of the winter series, Rock n Roll: From Elvis to Lady Gaga, on Wednesday, March 8.
The lecture is $5 and refreshments will be served after the talk. To check on weather cancellations, listen to WDEV 550 AM or WLVB 93.9 FM or call Town and Country Resort at 253-7595. To sponsor a lecture, a series or refreshments, call Dick Johannesen, 253-8475. Information: learn.uvm.edu/osher.
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Cardiac injury, recovery is topic of Osher lecture - Stowe Today
Nanostraw doesn’t destroy cells as it samples their guts – Futurity: Research News
By daniellenierenberg
Cells within our bodies divide and change over time, with thousands of chemical reactions occurring within each cell daily. This makes it difficult for scientists to understand whats happening inside. New nanostraws offer a non-disruptive way to find out.
A problem with the current method of cell sampling, called lysing, is that it ruptures the cell. Once the cell is destroyed, it cant be sampled from again. This new sampling system relies on tiny tubes 600 times smaller than a strand of hair that allow researchers to sample a single cell at a time. The nanostraws penetrate a cells outer membrane, without damaging it, and draw out proteins and genetic material from the cells salty interior.
Its like a blood draw for the cell, says Nicholas Melosh, an associate professor of materials science and engineering at Stanford University and senior author of a paper describing the work in the Proceedings of the National Academy of Sciences.
The nanostraw sampling technique, according to Melosh, will significantly impact our understanding of cell development and could lead to much safer and effective medical therapies because the technique allows for long term, non-destructive monitoring.
What we hope to do, using this technology, is to watch as these cells change over time and be able to infer how different environmental conditions and chemical cocktails influence their developmentto help optimize the therapy process, Melosh says.
If researchers can fully understand how a cell works, then they can develop treatments that will address those processes directly. For example, in the case of stem cells, researchers are uncovering ways of growing entire, patient-specific organs. The trick is, scientists dont really know how stem cells develop.
For stem cells, we know that they can turn into many other cell types, but we do not know the evolutionhow do they go from stem cells to, say, cardiac cells? There is always a mystery. This sampling technique will give us a clearer idea of how its done, says Yuhong Cao, a graduate student and first author on the paper.
The sampling technique could also inform cancer treatments and answer questions about why some cancer cells are resistant to chemotherapy while others are not.
With chemotherapy, there are always cells that are resistant, says Cao. If we can follow the intercellular mechanism of the surviving cells, we can know, genetically, its response to the drug.
The sampling platform on which the nanostraws are grown is tinyabout the size of a gumball. Its called the Nanostraw Extraction (NEX) sampling system, and it was designed to mimic biology itself.
In our bodies, cells are connected by a system of gates through which they send each other nutrients and molecules, like rooms in a house connected by doorways. These intercellular gates, called gap junctions, are what inspired Melosh six years ago, when he was trying to determine a non-destructive way of delivering substances, like DNA or medicines, inside cells. The new NEX sampling system is the reverse, observing whats happening within rather than delivering something new.
Its a super exciting time for nanotechnology, Melosh says. Were really getting to a scale where what we can make controllably is the same size as biological systems.
Building the NEX sampling system took years to perfect. Not only did Melosh and his team need to ensure cell sampling with this method was possible, they needed to see that the samples were actually a reliable measure of the cell content, and that samples, when taken over time, remained consistent.
When the team compared their cell samples from the NEX with cell samples taken by breaking the cells open, they found that 90 percent of the samples were congruous. Meloshs team also found that when they sampled from a group of cells day after day, certain molecules that should be present at constant levels remained the same, indicating that their sampling accurately reflected the cells interior.
With help from collaborators Sergiu P. Pasca, assistant professor of psychiatry and behavioral sciences, and Joseph Wu, professor of radiology, Melosh and coworkers tested the NEX sampling method not only with generic cell lines, but also with human heart tissue and brain cells grown from stem cells. In each case, the nanostraw sampling reflected the same cellular contents as lysing the cells.
The goal of developing this technology, according to Melosh, was to make an impact in medical biology by providing a platform that any lab could build. Only a few labs across the globe, so far, are employing nanostraws in cellular research, but Melosh expects that number to grow dramatically.
We want as many people to use this technology as possible, he says.
Funding for the work came from the National Institute of Standards and Technology, the Knut and Alice Wallenberg Foundation, the National Institutes of Health, Stanford Bio-X, the Progenitor Cell Biology Consortium, the National Institute of Mental Health, an MQ Fellow award, the Donald E. and Delia B. Baxter Foundation, and the Child Health Research Institute.
Source: Jackie Flynn forStanford University
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Nanostraw doesn't destroy cells as it samples their guts - Futurity: Research News
Nanostraws Sample Cells Without Damage – R & D Magazine
By daniellenierenberg
Tiny nanostraws may offer a glimpse into a cells contents without causing any damage to the cell.
The nanostraws were developed by researchers at Stanford University, who devised a method of sampling cell contents without disrupting its natural processes, which is a staple of current cell sampling methods.
The new method relies on tiny tubes 600 times smaller than a stand of hair that allow researchers to sample a single cell at a time. The nanostraws are able to penetrate a cells outer membrane without damaging it and draw out proteins and genetic material from the cells salty interior.
It's like a blood draw for the cell, Nicholas Melosh, an associate professor of materials science and engineering and senior author on a paper, said in a statement.
According to Melosh, this technique will significantly impact the understanding of cell development and could yield much safer and effective medical therapies because it allows for long term, non-destructive monitoring.
What we hope to do, using this technology, is to watch as these cells change over time and be able to infer how different environmental conditions and 'chemical cocktails' influence their developmentto help optimize the therapy process, he said.
If researchers gain a better grasp on how a cell works they can address those processes directly.
For stem cells, we know that they can turn into many other cell types but we do not know the evolutionhow do they go from stem cells to, say, cardiac cells? Yuhong Cao, a graduate student and first author on the paper, said in a statement. This sampling technique will give us a clearer idea of how it's done.
A benefit of the sampling method is it could inform cancer treatments and answer questions about why some cancer cells are resistant to chemotherapy while others are not.
With chemotherapy, there are always cells that are resistant, Cao said. If we can follow the intercellular mechanism of the surviving cells, we can know, genetically, its response to the drug.
The nanostraws are grown in a small sampling platform designed to mimic biology called the Nanostraw Extraction (NEX) sampling system.
Cells divide and change over time, with thousands of chemical reactions occurring within each cell every day, which makes it difficult to truly understand the inner workings of cells.
Currently, scientists use a method of cell sampling called lysing, which ruptures the cell. However, once a cell is destroyed it cannot be sampled from again.
Cells in our bodies are connected by a system of gates through which they send each other nutrients and molecules.
Melosh was inspired to develop the new system when he observed the intercellular gates after he was trying to determine a non-destructive way of delivering substances, including DNA or medicines, inside cells.
The new sampling system is the reverse of that process, as scientists are able to observe whats happening within a cell.
When the research team compared their cells samples from the NEX with cell samples taken by breaking the cells open, they found that 95 percent of the samples were congruous.
The team also found that when they sampled from a group of cells day after day, certain molecules that should be present at constant levels remained the same, which indicated that their sampling accurately reflected the cells interior.
The team not only sampled generic cell lines but also with human heart tissue and brain cells grown from stem cells and in each case the nanostraw sampling reflected the same cellular contents as lysing the cells.
The study was published in the Proceedings of the National Academy of Sciences of the United States of America.
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Nanostraws Sample Cells Without Damage - R & D Magazine
Right to try, right to buy, right to test – Washington Post
By daniellenierenberg
By Sally Satel By Sally Satel February 21 at 8:04 PM
(Dr. Sally Satel, who has guest-blogged here before, was kind enough to write up this item on a topic that has long interested me; Im delighted to pass it along: -EV)
In 2007, Eugene Volokh, the host of this site, published an essay in the Harvard Law Reviewtitled Medical Self-Defense, Prohibited Experimental Therapies, and Payment for Organs in which he argued that the government should need a very good reason to prevent sick people from saving their own lives.
That insight impels the Right to Try movement, which seeks to give terminally ill patients the right to try drugs that show promise but not have received FDA approval and which has received sympathetic hearings from President Trump and Vice President Pence. One of the leaders of Right to Try reform, the libertarian Goldwater Institute, said it best: We just fundamentally do not believe that you should have to apply to the government for permission to try to save your own life.
That principle has vital implications for patients needing bone marrow and kidney transplants.
Each year, 2,000 to 3,000 individuals with leukemia and other forms of bone marrow disease die while waiting to receive another persons bone marrow cells. Its not that strangers are indifferent to their plight, but that suitable biological matches are hard to find. And even when a match is found, there is a 1-in-2 chance that the needle-in-a-haystack donor either cant be located by registry personnel or, incomprehensibly, refuses to donate even though he had earlier volunteered to be tested.
We can enlarge the pool of potential donors while increasing the likelihood that compatible donors will follow through if they are paid or if sick patients (or charities acting on their behalf) have the Right to Buy, as I call it.
But there is an obstacle to buying. The 1984 National Organ Transplant Act, or NOTA, bans exchange of valuable consideration that is, anything of material worth for solid organs, such as kidneys and livers, as well as for bone marrow.
The Institute for Justice, a libertarian public-interest law firm, fought the prohibition. It sued the Justice Department on behalf of families afraid their ill loved ones would die because they couldnt get a bone marrow transplant.
In a unanimous 2012 ruling, a three-judge panel of the U.S. Court of Appeals for the 9th Circuit rejected the federal governments argument that obtaining bone-marrow stem cells through a needle in a donors arm violates NOTA. The judges based their decision on the fact that modern bone-marrow procurement, a process known as apheresis, is akin to drawing blood. Indeed, filtered stem cells, they held, are merely components of blood, no different from blood-derived plasma, platelets and clotting factors, all of which are replenished by the body within weeks of a donation. Because its legal to compensate blood donors, its also legal to pay bone marrow donors, the court ruled.
Unfortunately, the Department of Health and Human Services rejected the courts ruling. In 2013, it proposed a rule that would extend the NOTA prohibition to bone marrow stem cells. Under the proposed regulation, anyone who accepted material gain for giving bone-marrow stem cells would be subject to NOTAs penalties, facing imprisonment for up to five years. According to HHS, compensation runs afoul of NOTAs intent to ban commodification of human stem cells and to curb opportunities for coercion and exploitation, encourage altruistic donation and decrease the likelihood of disease transmission.
The solicitor general could have asked the Supreme Court to review the 9th Circuits bone-marrow decision, but he declined. Perhaps he grasped the central folly of HHSs position: How could the agency justify its worry about opportunities for coercion and exploitation and the likelihood of disease transmission when it came to bone marrow cells, yet not apply those same concerns to plasma?
For three years, HHS has been silent on its proposed rule. Meanwhile, people are dying because nonprofits that want to begin paying donors on behalf of needy patients cant move forward until they are assured that the agency cant shut them down. The Institute for Justice is considering a legal challenge over the HHS delay, which is causing needless deaths.
But perhaps the lawsuit can wait. With a new administration that is skeptical of overregulation, HHS Secretary Tom Price could withdraw the proposed rule. Ideally, Congress would thwart future regulatory blockades by amending NOTA to stipulate that marrow stem cells are not organs covered by the act.
Changes to NOTA should also be made for other organs. I feel strongly about this on fundamental grounds of liberty but also because, in 2005, I needed to save my own life. I developed kidney failure but could not find a donor. Thank goodness, an angel, or as some readers know her, Virginia Postrel, heard about my predicament and gave me a kidney. And this summer another living saint, Kimberly Hendrickson, who saw how desperate I was many years ago, offered me one of hers when the first transplant began to fail. Every day, 12 people die because no one wasable to come to their rescue and, had a patient offered money for an organ, both the patient and the donor who accepted the money would face felony charges.
Congress could take the bold step of revising NOTA to permit donors who are willing to save the life of a stranger through kidney donation to receive valuable consideration from governments or nonprofit organizations. Or, lawmakers could take the intermediate step of creating a pilot program allowing doctors to study the effect of such measures, as proposed last May by Rep. Matthew Cartwright (D-Pa.), who introduced the Organ Donor Clarification Act of 2016.
Rather than large sums of cash, potential rewards could include a contribution to the donors retirement fund, an income tax credit or a tuition voucher, lifetime health insurance, a contribution to a charity of the donors choice, or loan forgiveness. Only the government, or a government-designated charity, would be allowed to disburse the rewards. Consequently, all patients, not just those with financial means, could benefit. The funds could potentially come from the savings from stopping dialysis, which costs roughly $80,000 a year per person.
The pilot programs, to be designed by individual medical centers, could also impose a waiting period on prospective donors, thereby cooling any impulsivity. Prospective donors would be fully informed about the risks of surgery and carefully screened for physical and emotional health, as all non-compensated kidney donors are now.
The idea of the government standing between a dying person and his salvation is deeply troubling. I know. We need to at least test better ways to recruit more marrow and kidney donors.
Dr. Sally Satelis a resident scholar at the American Enterprise Institute and editor ofWhen Altruism isnt Enough: The Case for Compensating Kidney Donors.
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Right to try, right to buy, right to test - Washington Post
Jonathan Pitre battles blood, lung infections before second stem cell … – Ottawa Sun
By daniellenierenberg
Ottawa Sun | Jonathan Pitre battles blood, lung infections before second stem cell ... Ottawa Sun Jonathan Pitre is back in a Minneapolis hospital with blood and lung infections complications that will likely delay his second stem-cell transplant. Pitre and ... |
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Jonathan Pitre battles blood, lung infections before second stem cell ... - Ottawa Sun
What’s the Catch: The Fountain of Youth – Paste Magazine
By daniellenierenberg
Scientist Juan Carlos Izpisua Belmonte from the Salk Institute in La Jolla, California, claims that the aging process may reversible: Our study shows that aging may not have to proceed in one single direction. With careful modulation, aging might be reversed.
Izpisua Belmonte attests that he implemented a new form of gene therapy on mice that were given a genetic disorder called progeria. After six weeks of treatment, the animals looked youngerand not only that, they had straighter spines, better cardiovascular health, healed more quickly when injured and actually lived longer.
How Its Done The rejuvenating treatment performed on the mice manipulates adult cells, such as skin cells, and turns them back into powerful stem cells (similar to what is seen in embryos). These powerhouses are referred to as induced pluripotent stem (iPS) cells and have the ability to multiply and transform into any cell type in the body; in fact, in trial tests, Izpisua Belmonte says iPS cells are being designed to provide organs and limbs for patients. He claims that his latest study is the first to show that the same technique can be used on other cells to rewind the clock and make them look younger. Izpisua Belmonte explains, The treatment involved intermittently switching on the same four genes that are used to turn skin cells into iPS cells. The mice were genetically engineered in such a way that the four genes could be artificially switched on when the mice were exposed to a chemical in their drinking water.
What This Means: This finding at the Salk Institute suggests that aging may not have to proceed in one directionin fact, Izpisua Belmonte states that it may actually be reversible. Although tests have not been conducted on humans yet, he predicts that applications via creams or injections are a decade away.
This rejuvenating treatment may not lead to immortality, but due to a growing body of evidence, scientists at the Salk Institute theorize that aging is driven by an internal genetic clock that actively causes our body to enter a state of decline. In developing this technology, it is hoped that future treatments designed will slow the ticking of this internal clock and ultimately increase life expectancy.
Whats the Catch? Dr. Sidney Chiu, a 5th year resident at the University of Toronto, thinks this information should be taken with a grain of salt: The findings are promising, but nowhere near ready for the front lines of healthcare. These experiments were done in highly controlled settings on genetically modified mice. If this finding were true, it would be worthy of a Nobel Prize because it would be akin to uncovering the Holy Grail. Chiu elaborates, If you can induce iPS cells, you have the basic building blocks to regenerate anything in the body. But this is far beyond any current medical science we have.
There are also numerous issues to address concerning the study: firstly, the mice are bred in labs for these types of tests, so the variables are controlled from the outset to attain desired results. Chiu adds, In the real world, you cannot turn specific genes on and off using treated water on mice in the wild, let alone humans. There isnt one specific gene for aging; I would be cautious about this scientists claims that isolating merely four could unlock the key to anti-aging. Even if we were just talking about reviving skin tissue, if his findings were true, it would be a breakthrough.
Chiu says that while it is technically possible to alter genetic material when humans are in an embryonic state, that wasnt done here (gene editing research in human embryos is currently allowed in Sweden China, and the United Kingdom. The United States doesnt currently have any legal prohibitions against it).
But its not to say that all of this is in the realm of science fiction; Chiu offers knowledge of research being conducted specifically for telomeres and their relationship to aging. Think of telomeres as the plastic caps that protect your shoelaces from fraying. The laces would be our chromosomes, the recipe for making a living thing. In fact, telomeres have an important role; they protect genetic material from damage that could otherwise lead to diseases or cell death. But because the number of cell divisions in telomeres is finite, once they become shorter (in length) and can no longer reproduce, it causes tissues to degenerate and eventually die. It is theorized that this process may contribute to the human aging process. So scientists are trying to find ways to extend the length of telomeres.
Izpisua Belmonte says that chemical approaches (via creams or injections) might be in human clinical trials to rejuvenate skin, bones and muscle within the next decade. However, from his perspective as a frontline healthcare worker, Chiu believes that we may just have to wait a bit longer than that before such innovations are accessible to everyone.
Main Photo by Thomas Rydberg, CC-BY
Tiffany Leigh is a Toronto-based food, travel, and science writer.
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What's the Catch: The Fountain of Youth - Paste Magazine
Mayo doc’s stem cell experiment blasts into space – Post-Bulletin
By daniellenierenberg
JACKSONVILLE, Fla. As a boy growing up in Kano, Nigeria, Dr. Abba Zubair dreamed of going to space.
On Sunday, his work hitched a ride with a private rocket blasting off from NASA's Kennedy Space Center in Cape Canaveral, Fla., on a trip to the International Space Station.
Dr. Zubair, an associate professor of laboratory medicine and pathology at the Mayo Clinic's Florida campus, prepared a science package involving stem cells as part of a resupply mission to the ISS aboard a SpaceX Falcon 9 rocket.
"It was my first rocket launch view," said Dr. Zubair, who was on hand to watch and listen to the deafening sound as his experiment rode into space. "It was incredible."
The stem cells -- specialized cells derived from bone marrow come from Dr. Zubair's lab. Dr. Zubair, according to a report from the Mayo Clinic, specializes in cellular treatments for disease and regenerative medicine. He hopes to find out how the stem cells hold up in space and if they can be more quickly produced in microgravity.
More specifically, Zubair said, he is hoping the research can help in treatment of patients who have suffered a stroke-related brain injury.
"Stem cells are known to reduce inflammation," he said in a press release. "We've shown that an infusion of stem cells at the site of stroke improves the inflammation and also secretes factors for the regeneration of neurons and blood vessels."
The problem with such a treatment and studying the treatment is generating enough stem cells for the job. Based on current regenerative medicine studies, patients need at least 100 million stem cells for an effective dose. However, reproducing stem cells can be time consuming since the cells naturally limit their numbers.
"Scalability is a big issue," Dr. Zubair said. "I've been interested in a faster way to make them divide."
And on earth, everything is impacted by gravity, from how high we grow to our bone size and other physiological traits. "So, how can we use the effect of gravity to impact how the cells divide?" he asked.
Experiments that simulate stem cell growth in microgravity, thus far, have shown cells do grow more quickly than experimental controls, he said. So he began working toward getting an experiment into space. The experiment needed to be designed so the crew onboard the space station could run the experiment with some simple training, and Dr. Zubair will be able to watch the experiment in real time via a video connection. "We'll get some data as early as next week," he said.
If all goes well, growing stem cells in space something Dr. Zubair admits sounds like a dream of the distant future might become a reality more quickly than many people think.
"There are some companies interested in floating labs," he said. "I think the future is bright. There are a lot of possibilities in the area of regenerative medicine."
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Mayo doc's stem cell experiment blasts into space - Post-Bulletin