Cubital Tunnel Syndrome & Spinal Cord Injury – Video
Cubital Tunnel Syndrome Spinal Cord Injury
This is a little awareness to Neuropathy and how it can be easily misdiagnosed Especially in someone who has sustained a Spinal Cord Injury Please visit! htt...
By: Matt Valente
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Cubital Tunnel Syndrome & Spinal Cord Injury - Video
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Stem Cell Therapies for Leukemia: Marching Toward the Clinic – Video
Stem Cell Therapies for Leukemia: Marching Toward the Clinic
Join California #39;s Stem Cell Agency (CIRM) for a live Google Hangout about recent progress in stem cell based treatment strategies for leukemia. Guest experts...
By: California Institute for Regenerative Medicine
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Stem Cell Therapies for Leukemia: Marching Toward the Clinic - Video
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What’s Next for Stem Cells and Regenerative Medicine?
See Inside Mar 19, 2013 |By Christine Gorman
Richard Clark, NIH
Researchers are now experimenting with stem cellsprogenitor cells that can develop into many different types of tissueto coax the bodies of a few individuals to heal themselves. Some of the most advanced clinical trials so far involve treating congestive heart disease and regrowing muscles in soldiers who were wounded in an explosion. But new developments are happening so quickly that investigators have come up with a new nameregenerative medicineto describe the emerging field.
Many of the stem cells being studied are referred to as pluripotent, meaning they can give rise to any of the cell types in the body but they cannot give rise on their own to an entirely new body. (Only the earliest embryonic cells, which occur just after fertilization, can give rise to a whole other organism by themselves.) Other stem cells, such as the ones found in the adult body, are multipotent, meaning they can develop into a limited number of different tissue types.
One of the most common stem cell treatments being studied is a procedure that extracts a few stem cells from a person's body and grows them in large quantities in the laboratorywhat scientists refer to as expanding the number of stem cells. Once a sufficient number have been produced in this manner, the investigators inject them back into the patient.
The bone marrow is a rich source of adult stem cells, containing both the hematopoietic stem cells that give rise to the various types of blood and the so-called mesenchymal cells, which can develop into bone, cartilage and fat. Mesenchymal cells are found in the bone marrow and various other places in the body, although whether all mesenchymal stem cells are truly interchangeable irrespective of origin is unclear.
Scientific American spoke with Mahendra Rao, director of the Center for Regenerative Medicine at the National Institutes of Health in Bethesda, Md., to get a sense of the sorts of new developments that might occur in regenerative medicine in the next five years or so.
[An edited transcript of the interview follows.]
Why is there so much excitement about regenerative medicine? You could say that medicine up until now has been all about replacements. If your heart valve isn't working, you replace it with another valve, say from a pig. With regenerative medicine, you're treating the cause and using your own cells to perform the replacement. The hope is that by regenerating the tissue, you're causing the repairs to grow so that it's like normal.
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What's Next for Stem Cells and Regenerative Medicine?
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21.Spinal Cord Injury(T5-6) Treated by Stem Cell Therapy(Before) – Video
21.Spinal Cord Injury(T5-6) Treated by Stem Cell Therapy(Before)
Patient with T5-6 spinal cord injury: condition before treatment Before treatment, sensation remains only above the waist. Sweating remains only in the upper...
By: Cells Center China
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21.Spinal Cord Injury(T5-6) Treated by Stem Cell Therapy(Before) - Video
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Controversial Stem Cell Company Moves Treatment out of U.S.
Celltex Therapeutics of Houston ceased treatment patients in the U.S. last year after a warning from regulators, and will now send patients for treatments to Mexico
Flickr/GE Healthcare
US citizens who had pinned their hopes on a company being able to offer stem-cell treatments close to home will now need to travel a little farther. Celltex Therapeutics of Houston, Texas, stopped treating patients in the United States last year following a warning from regulators. A 25 January e-mail to Celltex customers indicates that the firm will now follow in the footsteps of many other companies offering unproven stem-cell therapies and send its patients abroad for treatment but only to Mexico.
The stem-cell treatments offered by Celltex involved extracting adult stem cells from a patient, culturing them and then reinjecting them in a bid to replenish damaged tissue. It had been offering the treatment for more than a year with one of its high-profile customers being Texas governor, Rick Perry when the US Food and Drug Administration (FDA) wrote to the company on 24 September 2012 advising it that the stem cells it harvested and grew were more than minimally manipulated during Celltex's procedures. As such, the FDA regarded the cells as drugs, which would require the agency's approval to be used in treatments. The FDA also warned that Celltex had failed to address problems in its cell processing that inspectors from the agency had identified in an April 2012 inspection of its cell bank in Sugar Land, Texas. Shortly after it received the letter, Celltex stopped injecting stem cells into patients.
For customers who still had cells banked at Celltex and were wondering how to get them out, things became more chaotic when Celltex and RNL Bio, a company based in Seoul, South Korea, which operated the processing center and bank in Sugar Land, sued each other over financial disagreements. Celltex had to issue a restraining order just to gain access to the cells.
The January e-mail from Celltex reassures customers that their cells are safely stored in a facility in Houston and adds: We anticipate that we will be able to offer our stem cell therapy services to physicians in Mexico starting very soon! The e-mail also says that the company is building a new laboratory in Houston, to be opened in March.
Celltex adds that it will carry out an FDA-approved clinical trial, to start shortly after a March meeting with the FDA, pending a positive review from the regulator. However, the company had said in a 25 October e-mail to patients that it would start such a trial within two months and that patient enrolment could begin in late November.
Leigh Turner, a bioethicist at the University of Minnesota in Minneapolis, says that the move to Mexico is "not surprising", given the companys difficulties in the United States.
As Celltex's stem culturing and banking technology was licensed from RNL Bio, it is also not clear whether it has the expertise needed to launch a clinical trial on its own, says Turner. "It would have to build a stem-cell company from the ground floor up. I wouldnt say it is anywhere near the starting line."
Celltex did not respond to questions about how it would ship stem cells to Mexico or how it would perform the clinical research needed to seek FDA approval.
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A Change of Heart: Stem Cells May Transform Treatment for Heart Failure
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Stem cells may transform the way doctors treat heart failure
In early 2009 Mike Jones bought a newspaper at a convenience store in Louisville, Ky., and read about a local doctor who wanted to try something unprecedented: healing an ailing heart by harvesting and multiplying its native stem cellsimmature cells with regenerative powers. Jones, then 65, had congestive heart failure: his heart was no longer pumping blood efficiently. He contacted the doctor, Roberto Bolli of the University of Louisville, and in July of that year Jones became the first person in the world to receive an infusion of his own cardiac stem cells.
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A Change of Heart: Stem Cells May Transform Treatment for Heart Failure
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Transplanted human umbilical cord blood cells improved heart function in rat model of MI
PUBLIC RELEASE DATE:
6-Mar-2014
Contact: Robert Miranda cogcomm@aol.com Cell Transplantation Center of Excellence for Aging and Brain Repair
Putnam Valley, NY. (Mar. 6, 2014) When human umbilical cord blood cells were transplanted into rats that had undergone a simulated myocardial infarction (MI), researchers investigating the long term effects of the transplantation found that left ventricular (LV) heart function in the treated rats was improved over those that did not get the stem cells. The animals were maintained without immunosuppressive therapy.
The study will be published in a future issue of Cell Transplantation but is currently freely available on-line as an unedited early e-pub at: http://www.ingentaconnect.com/content/cog/ct/pre-prints/content-ct0860Chen.
"Myocardial infarction induced by coronary artery disease is one of the major causes of heart attack," said study co-author Dr. Jianyi Zhang of the University of Minnesota Health Science Center. "Because of the loss of viable myocardium after an MI, the heart works under elevated wall stress, which results in progressive myocardial hypertrophy and left ventricular dilation that leads to heart failure. We investigated the long term effects of stem cell therapy using human non-hematopoietic umbilical cord blood stem cells (nh-UCBCs). These cells have previously exhibited neuro-restorative effects in a rodent model of ischemic brain injury in terms of improved LV function and myocardial fiber structure, the three-dimensional architecture of which make the heart an efficient pump."
According to the authors, stem cell therapy for myocardial repair has been investigated extensively for the last decade, with researchers using a variety of different animal models, delivery modes, cells types and doses, all with varying levels of LV functional response. They also note that the underlying mechanisms for improvement are "poorly understood," and that the overall regeneration of muscle cells is "low."
To investigate the heart's remodeling processes and to characterize alterations in the cardiac fiber architecture, the research team used diffusion tensor MRI (DTMRI), used previously to study myofiber structure in both humans and animals.
While most previous studies have been focused on the short term effects of UCBCs, their study on long term effects not only demonstrated evidence of significantly improved heart function in the treated rats, but also showed evidence of delay and prevention in terms of myocardial fiber structural remodeling, alterations that could have resulted in heart failure.
When compared to the age-matched but untreated rat hearts with MI, the regional myocardial function of nh-UCBC-treated hearts was significantly improved and the preserved myocardial fiber structure may have served as an "underlying mechanism for the observed function improvements."
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Transplanted human umbilical cord blood cells improved heart function in rat model of MI
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Establishing standards where none exist; Harvard researchers define 'good' stem cells
PUBLIC RELEASE DATE:
6-Mar-2014
Contact: B.D. Colen bd_colen@harvard.edu 617-413-1224 Harvard University
After more than a decade of incremental and paradigm shifting, advances in stem cell biology, almost anyone with a basic understanding of life sciences knows that stem cells are the basic form of cell from which all specialized cells, and eventually organs and body parts, derive.
But what makes a "good" stem cell, one that can reliably be used in drug development, and for disease study? Researchers have made enormous strides in understanding the process of cellular reprogramming, and how and why stem cells commit to becoming various types of adult cells. But until now, there have been no standards, no criteria, by which to test these ubiquitous cells for their ability to faithfully adopt characteristics that make them suitable substitutes for patients for drug testing. And the need for such quality control standards becomes ever more critical as industry looks toward manufacturing products and treatments using stem cells.
Now a research team lead by Kevin Kit Parker, a Harvard Stem Cell Institute (HSCI) Principal Faculty member has identified a set of 64 crucial parameters from more than 1,000 by which to judge stem cell-derived cardiac myocytes, making it possible for perhaps the first time for scientists and pharmaceutical companies to quantitatively judge and compare the value of the countless commercially available lines of stem cells.
"We have an entire industry without a single quality control standard," said Parker, the Tarr Family Professor of Bioengineering and Applied Physics in Harvard's School of Engineering and Applied Sciences, and a Core Member of the Wyss Institute for Biologically Inspired Engineering.
HSCI Co-director Doug Melton, who also is co-chair of Harvard's Department of Stem Cell and Regenerative Biology, called the standard-setting study "very important. This addresses a critical issue," Melton said. "It provides a standardized method to test whether differentiated cells, produced from stem cells, have the properties needed to function. This approach provides a standard for the field to move toward reproducible tests for cell function, an important precursor to getting cells into patients or using them for drug screening."
Parker said that starting in 2009, he and Sean P. Sheehy, a graduate student in Parker's lab and the first author on a paper just given early on-line release by the journal Stem Cell Reports, "visited a lot of these companies (commercially producing stem cells), and I'd never seen a dedicated quality control department, never saw a separate effort for quality control." Parker explained many companies seemed to assume that it was sufficient simply to produce beating cardiac cells from stem cells, without asking any deeper questions about their functions and quality.
"We put out a call to different companies in 2010 asking for cells to start testing," Parker says, "some we got were so bad we couldn't even get a baseline curve on them; we couldn't even do a calibration on them."
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Establishing standards where none exist; Harvard researchers define 'good' stem cells
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3D printing helps create tailor-made wrap-around heart sensor array
Researchers have used 3D-printed models of the heart to create a personalized wrap-around heart sensor array which can transmit highly detailed information on a patients cardiac health and may thus help to predict and prevent serious medical problems.
The buzz surrounding 3D printing sometimes gives the impression that the technology provides a miracle solution for making any manufactured product more cheaply. In fact the main advantage of the technology is to be able to produce prototypes cheaper and faster or to customize products and components. The medical sector may well be among the first to benefit from this latter approach by using the technique, formally known as additive layer manufacturing (ALM), to produce tailor-made surgical implants. At the moment, medical researchers are focusing on highly ambitious projects such as printing replacement organs from a persons own stem cells, but this procedure will take years of development before it can be widely used on patients. Recently researchers have used 3D printing to help create a rather more modest device which could be incorporated fairly quickly into treatment procedures. Every heart has its own unique size and shape, and medical procedures need to be adjusted accordingly in order to deliver fully personalised treatment. Now researchers Igor Efimov of WashingtonUniversity in St Louisand John Rogers at the University of Illinoishave demonstrated a new type of tailor-made cardiac sensor array which increases the quantity and improves the quality of the information gathered, and thus help prevent certain cardiac problems.
Efimov, a cardiac physiologist and bioengineer, and Rogers, a materials scientist, used optical images of rabbits hearts to demonstrate the concept of creating an ALM model of the heart in order to make the sensor array. In fact CT or MRI scans of each persons heart would be used to make devices for human patients. Having 3D-printed the model of the heart, they then built a stretchy electronic mesh structure a sort of envelope to wrap round the model. The stretchy material can then be peeled off the printed model and wrapped around the real heart in a perfect fit. This technique enables a far more precise approach than has hitherto been feasible and the research team were able to integrate an unprecedented number of components into the device, including embedded sensors, oxygenation detectors, thermometers and electrodes that can, if need be, deliver electric shocks to stimulate a flagging heart. Although the device has been developed specifically to treat ventricular deformation andcardiacarrhythmia, it could incorporate different types of sensors in order to improve treatment for a number of other heart conditions, inter alia enabling medicines to be delivered to the exact spot where they are needed.
Igor Efimov reveals that the next step is a device with multiple sensors, and not just more electrical sensors. Sensors that measure acidity, for instance, could provide an early warning of a blocked coronary artery. So far, the researchers have tested their technology on beating rabbit hearts outside the body. The next stage will be to demonstrate that this approach can work in live animals before it can be tested on people. Although devices made in this kind of custom-manufacturing process would probably be more expensive than mass-produced medical implants, using ALM to print the basic heart model will bring the cost down considerably and help to ensure that the technology becomes available to patients who need it. In any case, argues Stanford University materials scientist Zhenan Bao, for these kinds of life-or-death applications, the market is likely to bear the cost, given the rich information that the device will provide, enabling early treatment of potentially serious conditions. The idea of incorporating IT devices into organs is becoming more commonplace and there could be many medical applications, such as devices to assist bladder control or mitigate conditions of the nervous system. In a less life-and-death field, the technology could also be used for body digitisation with a view to producing tailor-made clothing.
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3D printing helps create tailor-made wrap-around heart sensor array
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Painkillers Could Prove Helpful in Stem-Cell Transplants
Inhibition of a prostaglandin with nonsteroidal anti-inflammatory drugs has been found to cause stem cells to leave marrow, where they could be harvested for patients with blood disorders
Tino Soriano/National Geographic Society/Corbis
Aspirin-like drugs could improve the success of stem-cell transplants for patients with blood or bone-marrow disorders, a study suggests. The compounds coax stem cells from bone marrow into the bloodstream where they can be harvested for use in transplantation and they do so with fewer side effects than drugs now in use.
For patients with blood disorders such as leukemia, multiple myeloma or non-Hodgkins lymphoma, transplantation of haematopoietic stem cells precursor cells that reside in the bone marrow and give rise to all types of blood cell can be an effective treatment.
Previous work has shown that prostaglandin E2, or PGE2, a lipid known to regulate multiple bodily reactions including pain, fever and inflammation, also has a role in keeping stem cells in the bone marrow. In the latest study, researchers show that in mice, humans and baboons, inhibition of PGE2 with non-steroidal anti-inflammatory drugs (NSAIDs) causes stem cells to leave the bone marrow.
Releasing the stem cells The team gave baboons and humans an NSAID called meloxicam. They saw a subsequent increase in the numbers of haematopoietic stem cells in the bloodstream.
The researchers think that the departure of stem cells is caused by the disturbance of a group of bone-forming cells called osteoblasts. These cells secrete a protein called osteopontin that hooks the stem cells to the bone marrow. Inhibiting PGE2 would disrupt the production of osteopontin.
At present, doctors use a drug called filgrastim to mobilize haematopoietic stem cells in donors or in patients undergoing autotransplantation (in which they receive their own stem cells). In patients with multiple myeloma or non-Hodgkins lymphoma, however, and in some donors, stem cells dont mobilize well with filgrastim and other drugs in its class. Using NSAIDs such as meloxicam could enhance filgrastims efficacy, says lead author Louis Pelus of the Indiana University School of Medicine in Indianapolis. The study appears in Nature.
Meloxicam also has comparatively few side effects, says Pelus. He and his colleagues found that other NSAIDs, including aspirin and ibuprofen, can also mobilize haematopoietic stem cells, but these drugs can cause gastrointestinal upset in patients. PGE2 controls the secretion of hydrochloric acid in the stomach, and when you block that youve reduced your ability to control acid secretion. Meloxicam doesnt do that as badly as many of the other [drugs] do, he says.
For Charles Craddock, director of the blood and marrow transplant unit at the Queen Elizabeth Hospital in Birmingham, UK, the results might also hold clues about how to mediate the tricky process of getting cells back to the bone marrow once transplanted. If youre beginning to understand what mediates cells moving out, you might be able to understand what mediates cells moving in. If you can make bone marrow more sticky, when you put cells back, you might be able to keep them in.
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Painkillers Could Prove Helpful in Stem-Cell Transplants
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What's Next for Stem Cells and Regenerative Medicine?
See Inside Mar 19, 2013 |By Christine Gorman
Richard Clark, NIH
Researchers are now experimenting with stem cellsprogenitor cells that can develop into many different types of tissueto coax the bodies of a few individuals to heal themselves. Some of the most advanced clinical trials so far involve treating congestive heart disease and regrowing muscles in soldiers who were wounded in an explosion. But new developments are happening so quickly that investigators have come up with a new nameregenerative medicineto describe the emerging field.
Many of the stem cells being studied are referred to as pluripotent, meaning they can give rise to any of the cell types in the body but they cannot give rise on their own to an entirely new body. (Only the earliest embryonic cells, which occur just after fertilization, can give rise to a whole other organism by themselves.) Other stem cells, such as the ones found in the adult body, are multipotent, meaning they can develop into a limited number of different tissue types.
One of the most common stem cell treatments being studied is a procedure that extracts a few stem cells from a person's body and grows them in large quantities in the laboratorywhat scientists refer to as expanding the number of stem cells. Once a sufficient number have been produced in this manner, the investigators inject them back into the patient.
The bone marrow is a rich source of adult stem cells, containing both the hematopoietic stem cells that give rise to the various types of blood and the so-called mesenchymal cells, which can develop into bone, cartilage and fat. Mesenchymal cells are found in the bone marrow and various other places in the body, although whether all mesenchymal stem cells are truly interchangeable irrespective of origin is unclear.
Scientific American spoke with Mahendra Rao, director of the Center for Regenerative Medicine at the National Institutes of Health in Bethesda, Md., to get a sense of the sorts of new developments that might occur in regenerative medicine in the next five years or so.
[An edited transcript of the interview follows.]
Why is there so much excitement about regenerative medicine? You could say that medicine up until now has been all about replacements. If your heart valve isn't working, you replace it with another valve, say from a pig. With regenerative medicine, you're treating the cause and using your own cells to perform the replacement. The hope is that by regenerating the tissue, you're causing the repairs to grow so that it's like normal.
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What's Next for Stem Cells and Regenerative Medicine?
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A Dangerous Game: Some Athletes Risk Untested Stem Cell Treatments
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Some professional athletes' enthusiasm for certain stem cell treatments outpaces the evidence
Peter Ryan
In 2005, at the age of 32, then Los Angeles Angel Bartolo Coln won the American League Cy Young Award for best pitcher, one of professional baseball's top honors. He stumbled through subsequent seasons, however, after a series of rips and strains in the tendons and ligaments of his throwing arm, shoulder and back. In 2009 he all but quit baseball. Desperate to reclaim his career, Coln flew home to the Dominican Republic in 2010 for an experimental procedure not vetted or approved by the U.S. Food and Drug Administration. Doctors centrifuged samples of Coln's bone marrow and fat, skimmed off a slurry containing a particular kind of stem cellimmature, self-renewing cells that can turn into a variety of tissuesand injected it into his injured shoulder and elbow. Within months of the procedure the then 37-year-old Coln was once again pitching near the top of his game for the New York Yankeescommanding a 93-mile-per-hour fastball.
Whether the injected stem cells rejuvenated his arm is an open question. The fda and the International Society for Stem Cell Research warn that no rigorous studies have demonstrated that such treatments safely and effectively repair damaged connective tissue in people. The results of related animal studies, though promising, have raised more questions than answers. The term stem cell makes it sound cutting edge and exciting, says Paul Knoepfler, a cell biologist at the University of California, Davis, who also writes frequently on policy surrounding stem cells. But the role of these cells in sports medicine is essentially all hype.
No matter, apparently, to the aging, injured athletes who have followed Coln's lead. Lefty pitcher C. J. Nitkowski, who underwent the same procedure in 2011, told readers of his personal blog that he did not mind the lack of carefully controlled research. My attitude is I don't have the time to wait for the five- or 10-year study to come out, the then 38-year-old relief pitcher wrote, so I'm taking a chance now. Besides, Nitkowski figured, even if the treatment did not work, any health risks ought to be slight because the cells involved were his own.
That might not be such a safe bet. Numerous studies suggest that Coln, Nitkowski and others trying untested stem cell treatments may be risking more than they think. Even a syringe of one's own stem cells taken from one part of the body and squirted into another may multiply, form tumors, or may leave the site you put them in and migrate somewhere else the fda warns on its Web site. More clinical research is needed to define safety procedures, as well as how many cells of which types and what other tissue factors produce the desired results. In some animal studies, for example, the regenerated tissue is not as strong or flexible as the original. In other cases, an overgrowth of scar tissue makes the injected tendon or ligament adhere to the overlying skin. By preventing different tissues from gracefully sliding past one another, these adhesions sometimes pull an even bigger tear in an already serious wound.
In addition, Knoepfler worries that high-profile sports testimonials by Coln, Nitkowski and others will encourage joggers with blown-out knees and the parents of sore-armed Little Leaguers to demand the procedure before it has been thoroughly tested. When celebrities take to a new treatment, many other people follow suit, he says. Such premature enthusiasmor an unforeseen tragedy that results from proceeding too fast too sooncould also prevent serious researchers from getting funding to do the kinds of careful experiments that might eventually lead to safe and reliable treatments.
Seeds of Repair
The need for better ways to reknit damaged tendons and ligaments is painfully apparent to the roughly two million Americans in a given year who seek medical help for tears in their shoulder's rotator cuff, for example, or the 100,000 patients in the same year who undergo surgery in the U.S. to repair a ripped or ruptured anterior cruciate ligament (ACL) of the knee. Tendons and ligaments are tough, fibrous bands, made mostly of collagen, that anchor networks of muscles to a bone or link bones and cartilage across crucial joints. They lend strength, flexibility and stability to your daily twists and turns, whether you are rocketing a baseball across home plate or hefting a suitcase into an overhead bin. Once frayed or snapped, they can take many months or longer to mendeven with surgery.
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A Dangerous Game: Some Athletes Risk Untested Stem Cell Treatments
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Establishing standards where none exist: Researchers define 'good' stem cells
13 hours ago This is Kevin Kit Parker, the Thomas D. Cabot Associate Professor of Applied Science and Associate Professor of Biomedical Engineering, and Harvard Stem Cell Institute Principal Faculty member, has identified standards making it possible to quantitatively judge and compare commercially available stem cell lines. Credit: Jon Chase/Harvard Staff Photographer
After more than a decade of incremental and paradigm shifting, advances in stem cell biology, almost anyone with a basic understanding of life sciences knows that stem cells are the basic form of cell from which all specialized cells, and eventually organs and body parts, derive.
But what makes a "good" stem cell, one that can reliably be used in drug development, and for disease study? Researchers have made enormous strides in understanding the process of cellular reprogramming, and how and why stem cells commit to becoming various types of adult cells. But until now, there have been no standards, no criteria, by which to test these ubiquitous cells for their ability to faithfully adopt characteristics that make them suitable substitutes for patients for drug testing. And the need for such quality control standards becomes ever more critical as industry looks toward manufacturing products and treatments using stem cells.
Now a research team lead by Kevin Kit Parker, a Harvard Stem Cell Institute (HSCI) Principal Faculty member has identified a set of 64 crucial parameters from more than 1,000 by which to judge stem cell-derived cardiac myocytes, making it possible for perhaps the first time for scientists and pharmaceutical companies to quantitatively judge and compare the value of the countless commercially available lines of stem cells.
"We have an entire industry without a single quality control standard," said Parker, the Tarr Family Professor of Bioengineering and Applied Physics in Harvard's School of Engineering and Applied Sciences, and a Core Member of the Wyss Institute for Biologically Inspired Engineering.
HSCI Co-director Doug Melton, who also is co-chair of Harvard's Department of Stem Cell and Regenerative Biology, called the standard-setting study "very important. This addresses a critical issue," Melton said. "It provides a standardized method to test whether differentiated cells, produced from stem cells, have the properties needed to function. This approach provides a standard for the field to move toward reproducible tests for cell function, an important precursor to getting cells into patients or using them for drug screening."
Parker said that starting in 2009, he and Sean P. Sheehy, a graduate student in Parker's lab and the first author on a paper just given early on-line release by the journal Stem Cell Reports, "visited a lot of these companies (commercially producing stem cells), and I'd never seen a dedicated quality control department, never saw a separate effort for quality control." Parker explained many companies seemed to assume that it was sufficient simply to produce beating cardiac cells from stem cells, without asking any deeper questions about their functions and quality.
"We put out a call to different companies in 2010 asking for cells to start testing," Parker says, "some we got were so bad we couldn't even get a baseline curve on them; we couldn't even do a calibration on them."
Brock Reeve, Executive Director of HSCI, noted that "this kind of work is as essential for HSCI to be leading in as regenerative biology and medicine, because the faster we can help develop reliable, reproducible standards against which cells can be tested, the faster drugs can be moved into the clinic and the manufacturing process."
The quality of available human stem cells varied so widely, even within a given batch, that the only way to conduct a scientifically accurate study, and establish standards, "was to use mouse stem cells," Parker said, explaining that his group was given mouse cardiac progenitor cells by the company Axiogenesis.
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Sick Vt. kids highlight need for bone marrow donors
BOSTON -
A bone marrow transplant could be a life-saving move for a little girl from Chester.
Keith McGilvery visited her at Boston Children's Hospital Tuesday and found out she's not the only young Vermonter who's sick on her floor. There are two kids from Vermont-- one from Chester and the other from Colchester. They're neighbors at Children's Hospital hoping that their transplants will make them better.
Tuesday, we visited Lindsey Sturtevant, she's the 12-year-old who just received a second bone marrow transplant to fight off a pre-leukemia condition that's done a number on her blood cells.
During our visit, we learned that Colchester Middle Schooler Le'Ondre Brockington is in the hospital bed next door. The 13-year old is fighting a rare form of acute myeloid leukemia. He's been in the hospital for seven months and his mom says every day has been a battle. Both families are thankful to their transplants.
Lindsey's doctor, Christine Duncan of the Dana-Farber Cancer Institute and Boston Children's Hospital, talked with us about what's involved if you decide to donate.
"There are lots of different ways that we collect stem cells. Some are directly from the bone, some are from your blood, most often it is a blood-type donation. For people that are really interested, they can look at the national marrow donor program which is the program that helped us find a donor for Lindsey," Dr. Duncan said.
Matches don't always come from family; Lindsey's first donor came from a 42-year-old woman in Europe and the second came from a 23-year-old man.
Le'Ondre's bone marrow donation came from a 33-year-old man.
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Alzheimer's research team employs stem cells to understand disease processes and study new treatment
PUBLIC RELEASE DATE:
6-Mar-2014
Contact: Jessica Maki jmaki3@partners.org 617-525-6373 Brigham and Women's Hospital
Boston, MA A team of Alzheimer's disease (AD) researchers at Brigham and Women's Hospital (BWH) has been able to study the underlying causes of AD and develop assays to test newer approaches to treatment by using stem cells derived from related family members with a genetic predisposition to (AD).
"In the past, research of human cells impacted by AD has been largely limited to postmortem tissue samples from patients who have already succumbed to the disease," said Dr. Tracy L. Young-Pearse, corresponding author of the study recently published in Human Molecular Genetics and an investigator in the Center for Neurologic Diseases at BWH. "In this study, we were able to generate stem cells from skin biopsies of living family members who carry a mutation associated with early-onset AD. We guided these stem cells to become brain cells, where we could then investigate mechanisms of the disease process and test the effects of newer antibody treatments for AD."
The skin biopsies for the study were provided by a 57-year-old father with AD and his 33 year-old- daughter, who is currently asymptomatic for AD. Both harbor the "London" familial AD Amyloid Precursor Protein (APP) mutation, V7171. More than 200 different mutations are associated with familial AD. Depending on the mutation, carriers can begin exhibiting symptoms as early as their 30s and 40s. APPV7171 was the first mutation linked to familial AD and is the most common APP mutation.
The BWH researchers submitted the skin biopsies to the Harvard Stem Cell Institute, where the cells were converted into induced pluripotent stem cells (or iPSCs). Dr. Young-Pearse's lab then directed the stem cells derived from these samples into neurons specifically related to a particular region of the brain which is responsible for memory and cognitive function. The scientists studying these neurons made several important discoveries. First, they showed that the APPV7171 mutation alters APP subcellular location, amyloid-beta protein generation, and then alters Tau protein expression and phosphorylation which impacts the Tau protein's function and activity. Next, the researchers tested multiple amyloid-beta antibodies on the affected neurons. Here, they demonstrated that the secondary increase in Tau can be rescued by treatment with the amyloid -protein antibodies, providing direct evidence linking disease-relevant changes in amyloid-beta to aberrant Tau metabolism in living cells obtained directly from an AD patient.
While AD is characterized by the presence of amyloid-beta protein plaques and Tau protein tangles, observing living cell behavior and understanding the mechanisms and relationship between these abnormal protein deposits and tangles has been challenging. Experimental treatments for AD are using antibodies to try to neutralize the toxic effects of amyloid-beta, because they can bind to and clear the amyoid-beta peptide from the brain.
This study is the first of its kind to examine the effects of antibody therapy on human neurons derived directly from patients with familial AD.
"Amyloid-beta immunotherapy is a promising therapeutic option in AD, if delivered early in the disease process," said Dr. Young-Pearse. "Our study suggests that this stem cell model from actual patients may be useful in testing and comparing amyloid-beta antibodies, as well as other emerging therapeutic strategies in treating AD."
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Alzheimer's research team employs stem cells to understand disease processes and study new treatment
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Patient-Specific Human Embryonic Stem Cells Created by Cloning
The breakthrough might set up another showdown about cloning for therapeutic purposes
OHSU Photos
From Nature magazine
It was hailed some 15 years ago as the great hope for a biomedical revolution: the use of cloning techniques to create perfectly matched tissues that would someday cure ailments ranging from diabetes to Parkinsons disease. Since then, the approach has been enveloped in ethical debate, tainted by fraud and, in recent years, overshadowed by a competing technology. Most groups gave up long ago on the finicky core method production of patient-specific embryonic stem cells (ESCs) from cloning. A quieter debate followed: do we still need therapeutic cloning?
A paper published this week by Shoukhrat Mitalipov, a reproductive biology specialist at the Oregon Health and Science University in Beaverton, and his colleagues is sure to rekindle that debate. Mitalipov and his team have finally created patient-specific ESCs through cloning, and they are keen to prove that the technology is worth pursuing.
Therapeutic cloning, or somatic-cell nuclear transfer (SCNT), begins with the same process used to create Dolly, the famous cloned sheep, in 1996. A donor cell from a body tissue such as skin is fused with an unfertilized egg from which the nucleus has been removed. The egg reprograms the DNA in the donor cell to an embryonic state and divides until it has reached the early, blastocyst stage. The cells are then harvested and cultured to create a stable cell line that is genetically matched to the donor and that can become almost any cell type in the human body.
Many scientists have tried to create human SCNT cell lines; none had succeeded until now. Most infamously, Woo Suk Hwang of Seoul National University in South Korea used hundreds of human eggs to report two successes, in 2004 and 2005. Both turned out to be fabricated. Other researchers made some headway. Mitalipov created SCNT lines in monkeys in 2007. And Dieter Egli, a regenerative medicine specialist at the New York Stem Cell Foundation, successfully produced human SCNT lines, but only when the eggs nucleus was left in the cell. As a result, the cells had abnormal numbers of chromosomes, limiting their use.
Monkeying around Mitalipov and his group began work on their new study last September, using eggs from young donors recruited through a university advertising campaign. In December, after some false starts, cells from four cloned embryos that Mitalipov had engineered began to grow. It looks like colonies, it looks like colonies, he kept thinking. Masahito Tachibana, a fertility specialist from Sendai, Japan, who is finishing a 5-year stint in Mitalipovs laboratory, nervously sectioned the 1-millimetre-wide clumps of cells and transferred them to new culture plates, where they continued to grow evidence of success. Mitalipov cancelled his holiday plans. I was happy to spend Christmas culturing cells, he says. My family understood.
The success came through minor technical tweaks. The researchers used inactivated Sendai virus (known to induce fusion of cells) to unite the egg and body cells, and an electric jolt to activate embryo development. When their first attempts produced six blastocysts but no stable cell lines, they added caffeine, which protects the egg from premature activation.
None of these techniques is new, but the researchers tested them in various combinations in more than 1,000 monkey eggs before moving on to human cells. They made the right improvements to the protocol, says Egli. Its big news. Its convincing. I believe it.
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Patient-Specific Human Embryonic Stem Cells Created by Cloning
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Produce Woolly Mammoth Stem Cells, Says Creator of Dolly the Sheep
Sir Ian Wilmut proposes an alternative method as a possible means of creating a mammoth--or a hybrid. Such research could lead to major biological discoveries and advances
Wikimedia Commons/Mammut
Editor's note: The following essay is reprinted with permission from The Conversation UK, an online publication covering the latest research.
By Ian Wilmut, University of Edinburgh
It is unlikely that a mammoth could be cloned in the way we created Dolly the sheep, as has been proposed following the discovery of mammoth bones in northern Siberia. However, the idea prompts us to consider the feasibility of other avenues. Even if the Dolly method is not possible, there are other ways in which it would be biologically interesting to work with viable mammoth cells if they can be found.
In order for a Dolly-like clone to be born it is necessary to have females of a closely related species to provide unfertilised eggs, and, if cloned embryos are produced, to carry the pregnancies. Cloning depends on having two cells. One is an egg recovered from an animal around the time when usually she would be mated.
In reality there would be a need for not just one, but several hundred or even several thousand eggs to allow an opportunity to optimise the cloning techniques. The cloning procedure is very inefficient. After all, after several years of research with sheep eggs, Dolly was the only one to develop from 277 cloned embryos. In species in which research has continued, the typical success rate is still only around 5% at best.
Elephant eggs
In this case the suggestion is to use eggs from elephants. Because there is a danger of elephants becoming extinct it is clearly not appropriate to try to obtain 500 eggs from elephants. But there is an alternative.
There is a considerable similarity in the mechanisms that regulate function of the ovaries in different mammals. It has been shown that maturation of elephant eggs is stimulated if ovarian tissue from elephants is transplanted into mice.
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Produce Woolly Mammoth Stem Cells, Says Creator of Dolly the Sheep
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Catalent Announces Agreement for One of the First Regenerative Therapies to Employ iPS Cells in Humans with CiRA …
Somerset, NJ (PRWEB) March 06, 2014
Catalent Pharma Solutions, the global leader in advanced delivery technologies and development solutions for drug, biologic and consumer health products, today announced an agreement with the Center for iPS Cell Research and Application (CiRA) at Kyoto University in Japan to make a major advancement toward one of the first regenerative human therapies with induced pluripotent stem (iPS) cells applicable to humans. Under this agreement, Catalent manufactures an anti-CORIN monoclonal antibody using its proprietary GPEx cell line expression technology for a planned clinical research project to develop an iPS cell-based transplant therapy for Parkinsons disease at CiRA, which is directed by Professor Doctor Shinya Yamanaka, the joint winner of the Nobel Prize in Physiology or Medicine in 2012 for the discovery that mature cells can be reprogrammed to become pluripotent.
The anti-CORIN monoclonal antibody was discovered and developed through collaborative research between CiRA and KAN Research Institute, Inc., a research subsidiary of a major Japanese pharmaceutical company, Eisai Co., Ltd-(http://www.kan-research.co.jp/english/index.html). Catalent has already engineered cell lines producing the anti-CORIN monoclonal antibody for CiRA using their GPEx technology, and the antibody has been shown to be useful for sorting CORIN-expressing cells in in vitro studies at CiRA. Under the agreement, Catalent will conduct further clonal selection and manufacturing of the monoclonal antibody under a properly conditioned environment for CiRA, which will use the antibody to select dopaminergic neurons derived from iPS cells and plans to transplant the selected cells into patients in a possible clinical research program upon receipt of regulatory approval. Catalent will also support CiRA, with formulation, production, and sterile fill/finish of the monoclonal antibody, aspects of the project that could not be handled within academia.
It is a great honor to work with a team led by world renowned Professor Doctor Jun Takahashi, commented Shingo Nakamura, Catalents Director of Biologics, Japan. We are very excited to help accelerate the development of a unique regenerative therapy using our GPEx technology and look forward to working with CiRA to bring better treatments to market faster.
Jonathan Arnold, Vice President and General Manager of Catalent Biologics, added, We are witnessing an increased demand for biologics in the Asia Pacific market. Our GPEx technology, our expertise, and access to Antibody Drug Conjugates, combined with our investment in state-of-the art manufacturing facilities, mean that we are ideally placed to act as a partner to CiRA in this exciting project.
Catalents GPEx technology produces high-yielding, stable mammalian cell lines and has been successfully applied in the manufacture of more than 500 different recombinant proteins, over 30 of which are now undergoing clinical trials or being supplied commercially. Antibiotic selection and traditional gene amplification are not required when using GPEx technology, resulting in shorter clonal cell line development timelines.
About Catalent Catalent Pharma Solutions is the leading global provider of advanced drug delivery technologies and development solutions for drugs, biologics and consumer health products. With over 80 years serving the industry, Catalent has proven expertise in bringing more customer products to market faster, enhancing product performance and ensuring reliable clinical and commercial product supply. Catalent employs approximately 8,500 people, including over 1,000 scientists, at nearly 30 facilities across 5 continents and in fiscal 2013 generated more than $1.8 billion in annual revenue. Catalent is headquartered in Somerset, N.J. For more information, visit http://www.catalent.com.
More products. Better treatments. Reliably supplied.
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Catalent Announces Agreement for One of the First Regenerative Therapies to Employ iPS Cells in Humans with CiRA ...
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stem cell therapy treatment for Spastic Diplegic cerebral palsy by dr alok sharma, mumbai, india – Video
stem cell therapy treatment for Spastic Diplegic cerebral palsy by dr alok sharma, mumbai, india
improvement seen in just 5 days after stem cell therapy treatment for Spastic Diplegic cerebral palsy by dr alok sharma, mumbai, india. Stem Cell Therapy don...
By: Neurogen Brain and Spine Institute
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stem cell therapy treatment for Spastic Diplegic cerebral palsy by dr alok sharma, mumbai, india - Video
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Global Cell Therapy Market to Reach 8 Bln by 2018, Says Kuick Research in Its Report Published at MarketPublishers.com
London, UK (PRWEB) March 06, 2014
In 2012, the world cell therapy market was evaluated at around USD 2.2 billion. The market is predicted to see robust growth in the years ahead, driven primarily by the ongoing advancements in the cell therapy industry along with considerable developments in the world of medicine. The overall cell therapy market is forecast to register a 21% CAGR on average in the upcoming years; and is anticipated to amount to USD 8 billion by 2018. A number of cell-based products and technologies, which are currently in the R&D pipeline, are expected to enter the market during the next five years, thus encouraging an increase in the growth rate of the cell therapies market.
With the cell-based treatment options growing by leaps and bounds, the upcoming years will likely see tremendous technological advancements with respect to the cell-based therapeutics industry. The expanding prevalence of diseases together with the lack of adequate effective treatment option for these illnesses is most likely to spur the advances in the cell-based therapeutics market both in developed and developing countries. The development of sophisticated automation devices is the most prominent emerging trend in the overall cell based therapy market.
New research report Global Cell Therapy Market & Pipleine Insight worked out by Kuick Research is now available at MarketPublishers.com
Report Details:
Title: Global Cell Therapy Market & Pipleine Insight Published: March, 2014 Pages: 500 Price: US$ 2,400.00 http://marketpublishers.com/report/healthcare/therapy/global-cell-therapy-market-pipleine-insight.html
The report provides detailed coverage of all the important aspects of the cell therapy market. It features the market growth resistors and stimulators, outlines and discusses the emerging challenges and opportunities, sheds light on the significant industry developments. The research study contains a summary of the existing cell therapy products and technologies, includes a comprehensive assessment of the cell therapy market on the basis of geography, target indicators, clinical phase and segments. The report describes the competitive climate, scrutinizes the actual cell therapy pipeline, uncovers data on the market performance between 2010 and 2012, and also offers an insightful market future outlook through 2018.
Report Scope:
More new research reports by the publisher can be found at Kuick Research page.
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The Global Cell Therapy Market to Grow at a High Rate of 20-22% by 2018, Says a New Report at ReportsnReports.com
Dallas, Texas (PRWEB) March 06, 2014
The global market for cell therapy was valued at close to USD 2.5 Billion in 2012. With continuous advances and developments in this field of medicine, the global cell therapy market is expected to grow at a high rate of 20-22% to reach approximately USD 8 Billion by 2018. Several products and technologies of cell-based therapies are in the R&D pipeline are expected to enter the market during the forecast period, thus resulting in an increased growth rate. It is most likely that the new improved technologies would revolutionize the area of bio-pharma and personalized medicine. Development of sophisticated automation devices for cell expansion and culture process for use in the treatment is one of the emerging trends of ACT market.
For the past 3-5 years, a more complex therapeutic modality has been is emerging as cell therapies which have been developed to treat diseases which are not amenable to treatment with more classical pharmaceutical or biopharmaceutical products. This Global Cell Therapy market offers the promise of successfully regenerating damaged tissues and organs in the body by replacing the damaged tissue and/or by stimulating the bodys own repair mechanisms to heal previously irreparable tissues. With their rising popularity, these therapies are becoming more cost effective and efficient.
Research in cell therapy is transforming the future of medicine. As the life of a human being begins as a cell, these cells undergo a highly complex set of events and finally those few stem become capable of self-renewal and differentiation and develop into the specialized cells in the body. Cell therapy research also offers significant potential for restructuring the method of medical practice.
With the cell-based therapies growing by leaps and bounds, it is expected that the future years would witness significant advancements in technology in the cell therapy market. Increasing incidence of diseases along with lack of adequate effective treatment for these diseases is most likely to drive the cell therapy technology in developed and developing nations. Among the emerging trends of the global market, the most prominent one is the development of sophisticated automation devices for cell expansion and culture process which could be used in the treatment of life threatening diseases.
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Global Cell Therapy Market & Pipeline Insight Report Highlights & Findings:
Table of Contents 1. Introduction to Cell Therapy 2. Global Cell Therapy Market Overview 3. Global Cell Therapy Market Dynamics 4. Cell Therapy Key Developments 5. Cell Therapy Pipeline by Phase, Country & Target Indications 6. Pancreatic Beta Cell Replacements Therapy Pipeline by Phase, Country & Target Indications 7. Dopaminergic Cell Replacements Therapy Pipeline by Phase, Country & Target Indications 8. Competitive Landscape List of Tables Following Information for Each Peptide Profile is Covered in More than 400 Tables in Report: List of Figures
Browse related report on 2014 Deep Research Report on Global and China Solar Cell Paste Industry at http://www.reportsnreports.com/reports/274518-2014-deep-research-report-on-global-and-china-solar-cell-paste-industry.html.
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Gene therapy locks out HIV, paving the way to control virus without antiretroviral drug
PUBLIC RELEASE DATE:
5-Mar-2014
Contact: Steve Graff stephen.graff@uphs.upenn.edu 215-349-5653 University of Pennsylvania School of Medicine
PHILADELPHIAUniversity of Pennsylvania researchers have successfully genetically engineered the immune cells of 12 HIV positive patients to resist infection, and decreased the viral loads of some patients taken off antiretroviral drug therapy (ADT) entirelyincluding one patient whose levels became undetectable. The study, appearing today in the New England Journal of Medicine, is the first published report of any gene editing approach in humans.
The phase I study was co-authored by researchers at Penn Medicine, the Albert Einstein College of Medicine and scientists from Sangamo BioSciences, which developed the zinc finger nuclease (ZFN) technology, the T cell therapy approach used in the clinical trial.
"This study shows that we can safely and effectively engineer an HIV patient's own T cells to mimic a naturally occurring resistance to the virus, infuse those engineered cells, have them persist in the body, and potentially keep viral loads at bay without the use of drugs," said senior author Carl H. June, MD, the Richard W. Vague Professor in Immunotherapy in the department of Pathology and Laboratory Medicine at Penn's Perelman School of Medicine. "This reinforces our belief that modified T cells are the key that could eliminate the need for lifelong ADT and potentially lead to functionally curative approaches for HIV/AIDS."
June and his colleagues, including Bruce L. Levine, PhD, the Barbara and Edward Netter Associate Professor in Cancer Gene Therapy in the department of Pathology and Laboratory Medicine and the director of the Clinical Cell and Vaccine Production Facility at Penn, used the ZFN technology to modify the T cells in the patientsa "molecular scissors," of sorts, to mimic the CCR5-delta-32 mutation. That rare mutation is of interest because it provides a natural resistance to the virus, but in only 1 percent of the general population. By inducing the mutations, the scientists reduced the expression of CCR5 surface proteins. Without those, HIV cannot enter, rendering the patients' cells resistant to infection.
For the study, the team infused the modified cells known as SB-728-Tinto two cohorts of patients, all treated with single infusionsabout 10 billion cellsbetween May 2009 and July 2012. Six were taken off antiretroviral therapy altogether for up to 12 weeks, beginning four weeks after infusion, while six patients remained on treatment.
Infusions were deemed safe and tolerable, the authors report, and modified T cells continued to persist in the patients when tested during follow up visits. One week after the initial infusion, testing revealed a dramatic spike in modified T cells inside the patients' bodies. While those cells declined over a number of weeks in the blood, the decrease of modified cells was significantly less than that of unmodified T cells during ADT treatment interruption. Modified cells were also observed in the gut-associated lymphoid tissue, which is a major reservoir of immune cells and a critical reservoir of HIV infection, suggesting that the modified cells are functioning and trafficking normally in the body.
The study also shows promise in the approach's ability to suppress the virus. The viral loads (HIV-RNA) dropped in four patients whose treatment was interrupted for 12 weeks. One of those patients' viral loads dropped below the limit of detection; interestingly, it was later discovered that the patient was found to be heterozygous for the CCR5 delta-32 gene mutation.
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Gene therapy locks out HIV, paving the way to control virus without antiretroviral drug
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Injections, gene therapy and treatment for infants raise hope for fighting AIDS
GWEN IFILL: For all of the progress made in the fight against AIDS, it still takes a terrible toll. More than 35 million people are infected with HIV around the world. More than two million people are newly infected each year. And well over a million die from it annually.
But research released at an AIDS conference this week is raising hope about new inroads into treating it and preventing infections.
Jeffrey Brown has the story.
JEFFREY BROWN: Three reports attracted attention. One involved injections of drugs into monkeys that helped stop infections. A second revealed promising news of a baby born with the virus and given aggressive treatment. A third concerned so-called gene editing, altering cells to resist HIV.
The NIHs Institute of Allergy and Infectious Diseases has been funding much of this work. Dr. Anthony Fauci is its longtime director, and he joins me now.
And welcome back.
So, lets walk through some of this. First, the injections of long-lasting drugs into monkeys, explain the work and why its so important.
DR. ANTHONY FAUCI, National Institutes of Health: Well, the reason the reason the work is important is that we know, in human studies, several human studies, that if you give a drug to an uninfected person whos practicing risk behavior, we call it preexposure prophylaxis, that if they take the drug every day, it absolutely works and prevents infection in over 90 percent of the people.
The problem with the approach is that people dont like to take medicine every day or before or after a sexual encounter. So, a modality of prevention that you know works 90-plus percent doesnt work that well, purely because people dont adhere.
The experiments that have been reported recently now show that, in a monkey model, if you take a long-acting drug, a drug thats used in a different form to treat HIV infection, in a monkey model, and give an injection every so often, like every couple of months, you can actually prevent challenging that monkey with infection with the monkey version of HIV by exposing them rectally or vaginally.
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Injections, gene therapy and treatment for infants raise hope for fighting AIDS
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Gene-editing method tackles HIV in first clinical test
NIBSC/Science Photo Library
HIV attacks a type of immune cell known as a T cell (shown here) using a protein encoded by the CCR5 gene.
A clinical trial has shown that a gene-editing technique can be safe and effective in humans. For the first time, researchers used enzymes called zinc-finger nucleases (ZFNs) to target and destroy a gene in the immune cells of 12 people with HIV, increasing their resistance to the virus. The findings are published today in The New England Journal of Medicine1.
This is the first major advance in HIV gene therapy since it was demonstrated that the Berlin patient Timothy Brown was free of HIV, says John Rossi, a molecular biologist at the Beckman Research Institute of the City of Hope National Medical Center in Duarte, California. In 2008, researchers reported that Brown gained the ability to control his HIV infection after they treated him with donor bone-marrow stem cells that carried a mutation in a gene called CCR5. Most HIV strains use a protein encoded by CCR5 as a gateway into the T cells of a hosts immune system. People who carry a mutated version of the gene, including Brown's donor, are resistant to HIV.
But similar treatment is not feasible for most people with HIV: it is invasive, and the body is likely to attack the donor cells. So a team led by Carl June and Pablo Tebas, immunologists at the University of Pennsylvania in Philadelphia, sought to create the beneficial CCR5 mutation in a persons own cells, using targeted gene editing.
The researchers drew blood from 12 people with HIV who had been taking antiretroviral drugs to keep the virus in check. After culturing blood cells from each participant, the team used a commercially available ZFN to target the CCR5 gene in those cells. The treatment succeeded in disrupting the gene in about 25% of each participants cultured cells; the researchers then transfused all of the cultured cells into the participants. After treatment, all had elevated levels of T cells in their blood, suggesting that the virus was less capable of destroying them.
Six of the 12 participants then stopped their antiretroviral drug therapy, while the team monitored their levels of virus and T cells. Their HIV levels rebounded more slowly than normal, and their T-cell levels remained high for weeks. In short, the presence of HIV seemed to drive the modified immune cells, which lacked a functional CCR5 gene, to proliferate in the body. Researchers suspect that the virus was unable to infect and destroy the altered cells.
They used HIV to help in its own demise, says Paula Cannon, who studies gene therapy at the University of Southern California in Los Angeles. They throw the cells back at it and say, Ha, now what?
In this first small trial, the gene-editing approach seemed to be safe: Tebas says that the worst side effect was that the chemical used in the process made the patients bodies smell bad for several days.
The trial isnt the end game, but its an important advance in the direction of this kind of research, says Anthony Fauci, director of the US National Institute of Allergy and Infectious Diseases in Bethesda, Maryland. Its more practical and applicable than doing a stem-cell transplant, he says, although it remains to be seen whether it is as effective.
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Gene-editing method tackles HIV in first clinical test
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EarthTalk / Synthetic vanilla may be just first of many 'synbio' additives
Dear EarthTalk: Should those of us who care about our health and the planet be concerned about the new trend in genetic engineering called synthetic biology?
Chrissie Wilkins
New Bern, N.C.
"Synthetic biology" (or "synbio") refers to the design and fabrication of novel biological parts, devices and systems that do not otherwise occur in nature. Many see it as an extreme version of genetic engineering. But unlike genetic engineering, whereby genetic information with certain desirable traits is inserted from one organism into another, synbio uses computers and chemicals to create entirely new organisms.
Proponents of synbio -- which include familiar players such as Cargill, BP, Chevron and DuPont -- tout its potential benefits. According to the Synthetic Biology Engineering Research Center, a consortium of leading U.S. researchers in the field, some promising applications of synthetic biology include alternatives to rubber for tires, tumor-seeking microbes for treating cancer, and photosynthetic energy systems. Other potential applications include using synbio to detect and remove environmental contaminants, monitor and respond to disease and develop new drugs and vaccines.
While these and other applications may not be widely available for years, synthetic biology is already in use for creating food additives that will start to show up in products on grocery shelves later this year. Switzerland-based Evolva is using synthetic biology techniques to produce alternatives to resveratrol, stevia, saffron and vanilla. The company's "synthetic vanillin" is slated to go into many foods as a cheaper and limitless version of real vanilla flavor. But many health advocates are outraged that such a product will be available to consumers without more research into potential dangers and without any warnings or labeling to let consumers know they are eating organisms designed and brought to life in a lab.
"This is the first major use of a synbio ingredient in food, and dozens of other flavors and food additives are in the pipeline, so synbio vanilla could set a dangerous precedent for synthetic genetically engineered ingredients to sneak into our food supply and be labeled as `natural,' " reports Friends of the Earth, a leading environmental group. "Synthetic biology vanillin poses several human health, environmental and economic concerns for consumers, food companies and other stakeholders."
For example, FoE worries that synbio vanilla (and eventually other synthetic biology additives) could exacerbate rainforest destruction while harming sustainable farmers and poor communities around the world. "Synbio vanilla ... could displace the demand for the natural vanilla market," reports FoE. "Without the natural vanilla market adding economic value to the rainforest in these regions, these last standing rainforests will not be protected from competing agricultural markets such as soy, palm oil and sugar." Critics of synbio also worry that releasing synthetic life into the environment, whether done intentionally or accidentally, could have adverse effects on our ecosystems.
Despite these risks, could the rewards of embracing synthetic biology be great? Could it help us deal with some of the tough issues of climate change, pollution and world hunger? Given that the genie is already out of the bottle, perhaps only time will tell.
EarthTalk is by Roddy Scheer and Doug Moss of E -- The Environmental Magazine (www.emagazine.com). Send questions to earthtalk@emagazine.com.
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EarthTalk / Synthetic vanilla may be just first of many 'synbio' additives
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