Posted: May 7, 2017 at 9:41 am
Oncology Nurse Advisor | CAR T Cells: Keeping Pace With Adverse Effects of an Emerging Therapy Oncology Nurse Advisor Chimeric antigen receptor T cells (CAR T cells) are human T cells that are collected from the patient and genetically modified to express a CAR immunoreceptor. The modified CAR T cells target specific surface proteins on cancer cells. CAR T cell ... |
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CAR T Cells: Keeping Pace With Adverse Effects of an Emerging Therapy - Oncology Nurse Advisor
Posted: May 7, 2017 at 9:40 am
John Webb, Special to The Clarion-Ledger 8:26 p.m. CT May 6, 2017
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Jacob Forester, 6, of Starkville is all smiles after Della Matheson, from the University of Miami, finishes drawing blood to check his glucose levels during a camp held Saturday at Twin Lakes in Florence sponsored by the Diabetes Foundation of Mississippi.(Photo: Kathy Matheny/The Clarion-Ledger)Buy Photo
FLORENCEWhile Caleb Warnock, 6, of Florence has many playmates at camp, his best friend may be the one he carries in his pocket and calls Dexter.
That is Caleb's nickname for his Dexcom, a continuous glucose monitor, or CGM, that transmits his blood sugar to a receiver that he carries in his pocket. The CGM can show which way his glucose is headed and can head off emergencies and help fine-tune glucose control, which can ultimately prevent complications in years to come.
When Dexter says his sugar is high, he takes more insulin, and when its low
I come to Mommy and she gives me a piece of candy, said Caleb, who has had type 1 diabetes for two and a half years and whose father, Christopher, was diagnosed at 18 months.
Caleb was among the 33 children with type I diabetes sharing stories that only others with diabetes could understand, laughing and joking about needles and counting carbs, at the Diabetes Foundation of Mississippis Camp Kandu, held Saturday and Sunday at Twin Lakes campgrounds near Florence.
According to the Mayo Clinic, type 1 diabetes, once known as juvenile diabetes or insulin-dependent diabetes, is a chronic condition in which the pancreas produces little or no insulin, a hormone needed to allow sugar (glucose) to enter cells to produce energy. Rates of type 1 are rapidly increasing, in Mississippi and around the world.
I thought my dad and I were the only ones who had diabetes, but then I came here and found out we werent, said Caleb, who helped his cousin, another type 1, overcome his fears of sticking his finger to test his blood sugar.
If you are a child living with diabetes, that may be the most powerful message of this unusual camp, which has been meeting every spring and fall for 15 years.
Diabetes drives children to help others
Diabetes and children: A balancing act
Suddenly maybe for the first time in your life you find out that youre not the only one. Not the only one having to stop what youre doing to check your blood sugar. (Always, it seems, at the most inopportune moment.) Not the only one having to struggle with what to eat. Not the only one whose life is a series of finger sticks and injections, and who must contend with the complex calculus of a disease that requires constant adjustment of insulin pump doses divided or multiplied by carbohydrates and exercise.
And not the only one trying to be a regular kid in spite of it all. At Kandu, amid the games, races and karate lessons, everyone stopped to check their blood sugar together. A few needed a little juice, and then picked up right where they were.
For some, diabetes is an exclusive club whose members intimately understand one anothers challenges. Sarah Fowler, 11, of Jackson, was diagnosed seven years ago (and is friends with the daughter of the Clarion-Ledger reporter who shares her name). She has come to Camp Kandu for six years and is in training to become a counselor.
There are not that many of us with type 1, so when you meet others you immediately know a lot of things about each other, said Sarah, amid the joyful sound of kids who, often for the first time. are meeting others like themselves. Ive made at least three lifelong friends here, because you have the same struggles and you go through them together. Sometimes you have more of a connection with them than with people without diabetes.
Hoping to remain without diabetes, however, are those being tested for antibodies that would indicate that they are at risk for developing the disease. Under the impassive eyes of the fish and deer adorning a rustic room at the lodge a toddler cries as a researcher tries to take his blood for the Type 1 Diabetes TrialNet Study, which is funded by the National Institutes of Health and is investigating interventions for the prevention or delay of type 1 diabetes.
Families of children with type 1 can learn who among them might have the antibody and how to avoid or postpone the onset of diabetes.
Fowler said she was, as a counselor in training, looking forward to being able to help more people like herself. I had someone last year come to me when their sugar was lowand showed them which foods in their backpack would bring it up to normal but not make it go too high, she said, adding that the magic formula was two glucose tablets.
But its not only children who make connections. Cher Crowley, a territory manager for OmniPod tubeless insulin pumps and pump educator at the camp, encountered a child whose family had lost employment and health insurance and who therefore had not seen an endocrinologist in months.
Nicki Nichols, whose child Bella has type 1, just came in and gave the mother not just hope but also a roadmap, Crowley said. Its frightening to have a child with type 1 and no insurance.
And parents say they finally have a place to share their deepest uncertainties with other parents. Having parents you can text or Facebook with a question as simple as, My child is starting swimming, and how can she check her blood sugar? said Kristen Fowler, mother of Sarah Fowler, a swimmer. We learned we had to give her 15 grams of carbs before practice or shed get low. Unless you have a child with type 1 in that activity you are not able to answer that question.
Its given her confidence knowing there are all these kids like her. Camp has made her more open to trying new things, like new pump infusion sites.
Sarah Joy Sudduth, 13, of Starkville treasures her independence and shares that with friends at camp. I tell them that diabetes is just a part of life and that learning to check their own blood sugar can keep them from having to depend on others, Sara Joy said, on the sidelines as a karate instructor chopped through a stack of flaming boards.
Its an appropriate image for those with diabetes for what may look impossible is in fact something that they can do.
Hence the name, Kandu. We were brainstorming and one of our former employees came up with the spelling of can-do, said Irena McClain, associate director of the Diabetes Foundation of Mississippi.
One camper knows what she wants to do with her life. I want to become a pediatric endocrinologist, because my doctor is one and she is my role model. She has type 1, too.
And that, it seems, is the Kandu spirit.
The Diabetes Foundation of Mississippi cares for all children adults in Mississippi with diabetes. Contact the foundation at 601-957-7878 for more information.
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Kids with diabetes show a 'Kandu' spirit - Jackson Clarion Ledger
Posted: May 7, 2017 at 9:40 am
Hollidaysburg, Blair County, Pa. - The community gathered together in Hollidaysburg to raise awareness and money for diabetes research.
A boy scout made it his mission to make it the best day for everyone there.
14 year-old Aaron Saylor dedicated three months of work to the Blair County Diabetes Walk.
He's doing it in honor of his sister who was diagnosed withtype 1 diabetes 5 years ago.
Saylor put together the kid zone for the event as part as his eagle scout project.
"I made different types of games from Plinko to duck game, 25 multi purposes boxes, the podium and more games."
Saylor says it's an important day for his sister and other kids in the region who suffer from diabetes.
Organizer Llisa Treese agrees.
"All these kids that have type 1 come and meet each other and they know other kids that have type one so they know they're not the only kid that has it."
Treese's son also has diabetes.
He was diagnosed ten years ago and it changed her family's life completely.
"We need other people to understand what they're going through and now that we have all these families here, and the word is getting out other people will understand. The kids don't look sick but their bodies are going through huge adjustments because their bodies can't regulate it."
Saylor says he spent more than 150 hours working on the games for the kid zone, the podium, and the boxes for the basket raffle.
It was all for one common goal.
"I just wanted the kids to have a fun day and just enjoy themselves."
Organizers of the walk hope to raise $10,000.
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Hollidaysburg, Blair County, Pa. - The community gathered together in Hollidaysburg to raise awareness and money for ... - WTAJ
Posted: May 6, 2017 at 3:47 pm
By Lindsey BeverThe Washington Post
Jasmin Floyd was on her way to kindergarten in northeastern Connecticut, buckled into the back seat of her mother's car.
On the way, she called out, "Mommy, my neck hurts," her mother, RoJeanne Doege, recently recalled. Doege said she peered through the rearview mirror and tried to reassure her, "Honey, it's probably just how you slept."
But it wasn't and, not long after that, Floyd's father noticed that their 5-year-old's neck was tilted ever so slightly to the side.
It was not the first time the girl and her family had been confronted with troublesome medical questions. Floyd had been born with an unexplained bunion on her big toe, and by the time she was a toddler, she had developed small bumps on her head and her spine. Doctors said the bumps were extra growths of bone, or osteomas, but that they were nothing to worry about, her mother said. Indeed, as Floyd grew, that bone fused and the bumps disappeared.
By January 1999, Floyd was on another quest for answers and, this time, she got one: a diagnosis of fibrodysplasia ossificans progressiva (FOP), a rare genetic disease that causes muscle tissue and connective tissue to turn into bone gradually forming a second skeleton and making it nearly impossible to move.
"It was the hardest, darkest time of our lives," Doege said. "We were helpless. There was nothing we could do. It was going to take on a life of its own."
Since then, there have been bad mornings when the now-23-year-old from Danielson, Connecticut, has woken up with a tight neck or an elbow locked in place, then slowly but permanently lost the ability to move them.
By the time she was 7 or so, Floyd said, her shoulders had started sticking; gradually, she found herself unable to rotate them. She had to relearn how to ride a bike and soon, how to do simpler tasks, such as switch on lights and turn on water faucets.
She now relies on a "reacher" to pick up things. She uses a long hairbrush to brush her hair and also to help pull on her shirts, laughing about the MacGyver-like skills she has acquired over the years.
"As I got older, I started to learn more about what FOP was," she said. "Now when something happens, I know what's happening and why it's happening."
But it does not make it any easier to live that way.
It was about a year ago, Floyd said, when her jaw started locking, which "has been the most traumatic thing I've ever had to deal with." Now, she said, she can open her mouth about a centimeter.
"I've accepted it the best I can, but it's not something I can put behind me," she said. "I dread brushing my teeth. I never used to have any dietary needs, but now I have to avoid crunchy or chewy foods. My jaw gets tired easily."
She calls each new issue a "new normal."
FOP is a rare and debilitating disorder that plagues about one in 2 million people worldwide, according to data from the National Institutes of Health. It is caused by mutations in a gene called ACVR1, which regulates bone growth and turns cartilage to bone as children grow up.
Frederick Kaplan, Floyd's doctor and head of the Division of Orthopaedic Molecular Medicine at the University of Pennsylvania's Perelman School of Medicine, said he and his colleagues discovered the FOP gene in 2006. He said FOP patients have an overactive copy of the abnormal gene that sends signals to the body's muscles and soft tissues especially after an injury telling the muscles to repair themselves by forming bone rather than muscle or scar tissue.
"The body thinks the injured muscles are fractures and heals them as if they were fractures," he said. "So the extra bone that forms is normal bone, but it forms in the wrong place and it locks the joints."
Any attempt to surgically remove that extra bone, he said, "leads to catastrophic explosions of new bone formation."
Kaplan said patients with FOP are born with the bunion-like bumps which are not bunions at all but malformations. He said the bumps are not an issue but are "a harbinger of things to come."
Starting in the preschool years, FOP patients usually start to develop severe swellings in the body's skeletal muscles that look like tumors.
These "flare-ups" can be triggered by the mildest injuries, such as bumps and bruises. But about 80 percent of the time, the flare-ups occur for no known reason at all, Kaplan said.
Kaplan explained how it happens: Intense inflammation erupts around the blood vessels in the muscles and destroys muscle tissue. Stem cells harboring the FOP gene are then awakened and start to divide and build a scaffold of sorts from connective tissue. The scaffolding turns to cartilage, on which the new bone begins to grow.
"FOP is a particularly cruel disease because, at a time when children are becoming adolescents and adults, at a time when they're trying to become more independent, their bodies are betraying them because of this mutation and they're becoming more dependent rather than less dependent," he said. "People with FOP have normal minds, but they're trapped in this prison of an extra skeleton.
"The other very difficult part of FOP is that you don't know when the next flare-up is going to occur, how long it's going to last, how painful it's going to feel and how disabling it's going to be."
By 40 or 50, he said, most patients succumb to a restrictive chest wall disease. He said that the extra skeleton severely limits lung capacity and, ultimately, the overworked heart starts to fail.
Floyd, who has chronicled her struggles on her blog, "One Spirit, Two Skeletons," said she has been sharing her story to inspire others and raise awareness about her disease.
At 23, she said, she now stands a bit off-balance and walks with a limp.
When she travels which she likes to do on her own to reclaim some independence she has to use a wheelchair or a scooter to get around, she said. Sometimes, she said, her jaw gets tired and she gets tongue-tied.
She said her greatest fear is the unpredictable and unsparing progression.
"I could wake up and not be able to unbend my leg," she said. "I've had it happen where my elbow will lock at a 90-degree angle."
Floyd said her most sudden flare-up was when she lost mobility in her jaw.
"I had eaten dinner and later that night before bed, I had pain in my jaw. That was it," she said. "It was painful not just physically but also emotionally. It gradually locked throughout the next few months."
But despite her fears, Floyd said, FOP has been a motivation.
"Even though I have fears, I do my best to make things happen so I can experience something," she said. "My motto is to try to as much as I can for as long as I can and not let anything stop me from achieving."
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Woman growing second skeleton, and it's locking her inside her own body - Arkansas News
Posted: May 6, 2017 at 3:46 pm
Human lungs, like all organs, begin their existence as clumps of undifferentiated stem cells. But in a matter of months, the cells get organized. They gather together, branch and bud, some forming airways and others alveoli, the delicate sacs where our bodies exchange oxygen for carbon dioxide. The end result, ideally: two healthy, breathing lungs.
For years, scientists who study lung diseases like cystic fibrosis have tried to track this process in detail, from start to finish, in the hope that understanding how lungs form normally may help explain how things go wrong. Now, scientists at Boston University's Center for Regenerative Medicine (CReM) have announced two major findings that further our understanding of this process: the ability to grow and purify the earliest lung progenitors that emerge from human stem cells, and the ability to differentiate these cells into tiny "bronchospheres" that model cystic fibrosis. Researchers hope that the results, published separately in the Journal of Clinical Investigation and Cell Stem Cell, will lead to new, "personalized medicine" approaches to treating lung disease.
"Sorting these cells to purity is really difficult and important," says Darrell Kotton, director of CReM and co-senior author of both papers, with Brian Davis of UTHealth at the University of Texas. "It's the first step in trying to predict how an individual might respond to existing treatments or new drugs."
"There's a long list of lung diseases for which there are no treatments other than a lung transplant," added Kotton, whose work is funded by the National Institutes of Health (NIH), the Cystic Fibrosis Foundation, and the Massachusetts Life Sciences Center. "It's critically important to develop new tools for understanding these diseases."
CReM scientists work with induced pluripotent stem cells, or iPSCs, which were discovered by Shinya Yamanaka in 2006. Yamanaka figured out how to take an adult cell in the human body -- like a blood cell or skin cell -- and "reprogram" it into a stem cell with the ability to grow into any organ. In recent years, several groups of scientists have grown lung cells from human iPSCs, but the recipes aren't perfect -- the resulting lung cells grow amidst a jumble of liver cells, intestinal cells, and other tissues.
"That's a big issue," says Finn Hawkins, a BU School of Medicine (MED) assistant professor of medicine and part of the CReM team. Hawkins is co-first author on the Journal of Clinical Investigation paper, along with Philipp Kramer, formerly of UTHealth. "If you want to use these cells to study the lung, you need to get rid of those others."
First, Hawkins needed a way to identify the lung cells. Previous work by Kotton and other CReM scientists demonstrated that mouse stem cells express a gene called Nkx2-1 at the "fate decision" -- the moment they turn into lung cells. "That's the first gene that comes on that says, 'I'm a lung cell,'" says Hawkins. Kotton built a reporter gene that glowed green when the stem cells first expressed Nkx2-1, and Hawkins engineered the same gene into human cells. Now, he could easily spot and purify the glowing green lung cells.
Using a flow cytometer, Hawkins and his colleagues separated the green cells out from the mix, then grew them in a matrix. The result: tiny green spheres about half a millimeter across, "a population of pure, early lung cells," says Hawkins. The team calls the tiny spheres "organoids," simplified and miniaturized versions of an organ, containing key types of lung cells. The organoids are tools, and they serve at least two important purposes. First, they allow scientists to study, in detail, a critical juncture in human lung development about which very little is known. "We discovered that many of the genes that control lung development in other species, such as mice, are also expressed in these human cells," says Hawkins.
The organoids serve another purpose, as well: scientists can grow them into more mature, specific cell types -- like airway cells or alveolar cells -- that are critical for lung function. "Now we can actually start looking at disease," says Hawkins. That's where Katherine McCauley (MED'17), a fifth-year PhD candidate at CReM, enters the picture.
McCauley's interest is cystic fibrosis, a disease caused by mutations in a single gene, CFTR. The mutation causes a person's lungs to produce a thick, viscous mucus that leads to infection, inflammation, and, eventually, lung failure. For many patients, there is no cure.
McCauley, looking at the earliest stages of the disease, wanted to take Hawkins' purified lung cells to the next step and figure out how they became airway cells. Through many painstaking experiments, she zeroed in on a signaling pathway called Wnt, known to be important in mouse lung development. By turning the pathway off, she guided the immature lung cells into becoming airway cells. Then, she grew them into tiny balls of cells, which she called "bronchospheres."
Like Hawkins' organoids, the bronchospheres don't act like a bronchus; they are simply a collection of specific cells. But their specificity makes them exquisitely useful. "We wanted to see if we could use these to study airway diseases," says McCauley. "That's one of the big goals: to engineer these cells from patients and then use them to study those patients' diseases."
As a proof of concept, McCauley obtained two cell lines from a patient with cystic fibrosis, one in which the CFTR mutation that caused the disease had been corrected, and one in which it hadn't, and grew them into bronchospheres. To see if her recipe worked, she ran a test, applying a drug that should cause spheres made of normal, functioning cells to fill with fluid. It worked: the "fixed" bronchospheres began to swell, while the cystic fibrosis spheres didn't react. "The cool part is that we measured this using high-throughput microscopy, and then we calculated the change in area with time," says McCauley, who published these results in Cell Stem Cell and is lead author on the study. "So now we can evaluate CFTR function in a quantitative way."
The next step, says McCauley, is to improve the test, and scale it up, and create similar tests for other lung diseases. "The end goal is to take cells from a patient, and then screen different combinations of drugs," she says. "The idea that we could take a patient's cells and test not twenty, but hundreds or thousands of drugs, and actually understand how the patient was going to respond before we even give them the treatment, is just an incredible idea."
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Scientists turn human induced pluripotent stem cells into lung cells - Science Daily
Posted: May 6, 2017 at 3:46 pm
Newswise BIRMINGHAM, Ala. The third annual Cardiovascular Tissue Engineering Symposium met at the University of Alabama at Birmingham last month, a gathering of noted physicians and scientists who share the goal of creating new tissues and new knowledge that can prevent or repair heart disease and heart attacks.
Talks ranged from the cutting-edge translational work of Phillippe Menasche, M.D., Ph.D., professor of thoracic and cardiovascular surgery, Paris Descartes University, to the basic biology research of Sean Wu, M.D., Ph.D., an associate professor of medicine, Stanford University School of Medicine. Menasches work pioneers human treatment with engineered heart tissue. Wus work opens the door to generating heart chamber-specific cardiomyocytes from human induced pluripotent stem cells, which act similarly to embryonic stem cells, having the potential to differentiate into any type of cell.
Menasche has placed engineered heart tissue derived from embryonic stem cell-derived cardiac cells onto the hearts of six heart attack patients in France in an initial safety study for this engineered tissue approach. Wu has used single-cell RNA sequencing to show 18 categories of cardiomyocytes in the heart, differing by cell type and anatomical location, even though they all derived from the same lineage.
We are creating a new community of engineer-scientists, said Jay Zhang, M.D., Ph.D., chair and professor of the UAB Department of Biomedical Engineering. In their welcoming remarks, both Selwyn Vickers, M.D., dean of the UAB School of Medicine, and Victor Dzau, M.D., professor of medicine at Duke University School of Medicine and president of the National Academy of Medicine, spoke of the growing convergence between scientists and physicians that is leading to tremendous possibilities to improve patient care.
The tissue engineering field is moving fast.
Cardiac progenitor cells that can contribute to growth or repair injury in the heart were only discovered in 2003, says symposium presenter Michael Davis, Ph.D., associate professor of Medicine, Department of Biomedical Engineering, Georgia Tech College of Engineering and Emory University School of Medicine. In 2006, the Japanese scientist Shinya Yamanaka first showed how to transform adult cells into induced pluripotent stem cells. This potentially provides feedstock for tissue engineering using either pluripotent cells or specific progenitor cells for certain tissue lineages.
One example of the pace of change was given by Bjorn Knollman, M.D., Ph.D., professor of medicine and pharmacology at Vanderbilt University School of Medicine. Knollman noted an ugly truth that everyone recognized in 2013 that cardiomyocytes derived from induced pluripotent stem cells were nothing like normal adult cardiomyocytes in shape, size and function.
He described the improved steps like culturing the derived cardiomyocytes in a Matrigel mattress and giving them a 14-day hormone treatment that have led to derived cardiomyocytes with greatly improved cell volume, morphology and function. His take-home message: In just four years, from 2013 to 2017, researchers were able to remove the differences between induced pluripotent stem cell-derived cardiomyocytes and normal adult cardiomyocytes.
In other highlights of the symposium, Joo Soares, Ph.D., a research scientist for the Center for Cardiovascular Simulation, University of Texas at Austin, explained how subjecting engineered heart valve tissue to cyclic flexure as it is grown in a bioreactor leads to improved quantity, quality and distribution of collagen, as opposed to tissue that is not mechanically stressed.
Sumanth Prabhu, M.D., professor and chair of the Division of Cardiovascular Disease, UAB School of Medicine, talked about the role of immune cells in cardiac remodeling and heart failure. He noted the distinct phases after a heart attack acute inflammation and dead tissue degradation, zero to four days; the healing phase of resolution and repair, four to 14 days; and the chronic ischemic heart failure that can occur weeks to months later. Prabhu described experiments to show how specialized spleen macrophages specifically marginal-zone metallophilic macrophages migrate to the heart after a heart attack and are required for heart repair to commence.
Nenad Bursac, Ph.D., professor of Biomedical Engineering, Duke University School of Medicine, described his advances in engineering vascularized heart tissue for repair after a heart attack. Bursac said a better understanding of how to grow the tissue from heart tissue progenitor cells has allowed formation of mature giga patches up to 4 centimeters square that have good propagation of heartbeat contractions and spontaneous formation of capillaries from derived-vascular endothelial and smooth muscle cells. These patches are being tested in pigs.
Duke Universitys Victor Dzau gave a perspective of the paracrine hypothesis over the past 15 years. In 2003, researchers had seen that applying mesenchymal stem cells to a heart attack area led to improved heart function, with beneficial effects seen as early as 72 hours. However, there was little engraftment and survival of the stem cells. Thus was born the hypothesis, which has been worked out in detail since then that stem cells do their work by release of biologically active factors that act on other cells, similar to the way that paracrine hormones have their effect only in the vicinity of the gland secreting it.
Joseph Wu, M.D., Ph.D., professor of radiology, Stanford University School of Medicine, showed how heart cells derived from induced pluripotent stem cells could be used to develop personalized medicine approaches for cancer patients. The problem, he explained, is that some cancer patients are susceptible to a deadly cardiotoxicity when treated with the potent drug doxorubicin. Hence, the drug has a black box warning, the strictest warning mandated by the Food and Drug Administration. Wu was able to use a library of induced pluripotent stem cell-derived cardiomyocytes to associate certain genotypes and phenotypes with doxorubicin sensitivity, in what he called a clinical trial in a dish. From this knowledge, it will be possible to look at the transcriptome profile in patient-specific cardiomyocytes derived from induced pluripotent stem cells to predict patient-specific drug safety and efficacy, thus fulfilling the definition of precision medicine the right treatment at the right time to the right person.
In all, UABs Cardiovascular Tissue Engineering Symposium included more than 30 presentations. The entire symposium will be summarized in a paper for the journal Circulation Research, expected to be published shortly, Zhang says.
Presentations of the 2015 Cardiovascular Tissue Engineering Symposium were published in the journal Science Translational Medicine, and the presentations of the 2016 Cardiovascular Tissue Engineering Symposium were published in the journal Circulation Research.
At UAB, Zhang holds the T. Michael and Gillian Goodrich Endowed Chair of Engineering Leadership, Vickers holds the James C. Lee Jr. Endowed Chair for the Dean of the School of Medicine, and Prabhu holds the Mary Gertrude Waters Chair of Cardiovascular Medicine.
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Fixing Broken Hearts Through Tissue Engineering - Newswise (press release)
Posted: May 6, 2017 at 3:45 pm
Jane S. Lebkowski, Ph.D., Asterias' President of R&D and Chief Scientific Officer, will be one of the presenters during the Presidential Symposium session scheduled on Friday, May 12, 2017 at 1:00pm Eastern Time. Dr. Lebkowski's presentation, titled "498 - Safety and Efficacy of Human Embryonic Stem Cell Derived Oligodendrocyte Progenitor Cells (AST-OPC1) in Patients with Subacute Cervical Spinal Cord Injury," is expected to begin at 2:15pm Eastern Time. The abstract for Dr. Lebkowski's presentation at the ASGCT meeting is available online at: http://www.abstractsonline.com/pp8/#!/4399/presentation/1996.
ASGCT is the primary professional membership organization for gene and cell therapy. The Society's members are scientists, physicians, patient advocates, and other professionals. Its members work in a wide range of settings including universities, hospitals, government agencies, foundations, biotechnology and pharmaceutical companies. Its mission is to advance knowledge, awareness, and education leading to the discovery and clinical application of gene and cell therapies to alleviate human disease.
About the SCiStar Trial
The SCiStar trial is an open-label, single-arm trial testing three sequential escalating doses of AST-OPC1 administered at up to 20 million AST-OPC1 cells in as many as 35 patients with sub-acute, C-5 to C-7, motor complete (AIS-A or AIS-B) cervical SCI. These individuals have essentially lost all movement below their injury site and experience severe paralysis of the upper and lower limbs. AIS-A patients have lost all motor and sensory function below their injury site, while AIS-B patients have lost all motor function but may retain some minimal sensory function below their injury site. AST-OPC1 is being administered 14 to 30 days post-injury. Patients will be followed by neurological exams and imaging procedures to assess the safety and activity of the product.
The study is being conducted at six centers in the U.S. and the company plans to increase this to up to 12 sites to accommodate the expanded patient enrollment. Clinical sites involved in the study include the Medical College of Wisconsin in Milwaukee, Shepherd Medical Center in Atlanta, University of Southern California (USC) jointly with Rancho Los Amigos National Rehabilitation Center in Los Angeles, Indiana University, Rush University Medical Center in Chicago and Santa Clara Valley Medical Center in San Jose jointly with Stanford University.
Asterias has received a Strategic Partnerships Award grant from the California Institute for Regenerative Medicine, which provides $14.3 million of non-dilutive funding for the Phase 1/2a clinical trial and other product development activities for AST-OPC1.
Additional information on the Phase 1/2a trial, including trial sites, can be found at http://www.clinicaltrials.gov, using Identifier NCT02302157, and at the SCiStar Study Website (www.SCiStar-study.com).
About AST-OPC1
AST-OPC1, an oligodendrocyte progenitor population derived from human embryonic stem cells, has been shown in animals and in vitro to have three potentially reparative functions that address the complex pathologies observed at the injury site of a spinal cord injury. These activities of AST-OPC1 include production of neurotrophic factors, stimulation of vascularization, and induction of remyelination of denuded axons, all of which are critical for survival, regrowth and conduction of nerve impulses through axons at the injury site. In preclinical animal testing, AST-OPC1 administration led to remyelination of axons, improved hindlimb and forelimb locomotor function, dramatic reductions in injury-related cavitation and significant preservation of myelinated axons traversing the injury site.
In a previous Phase 1 clinical trial, five patients with neurologically complete, thoracic spinal cord injury were administered two million AST-OPC1 cells at the spinal cord injury site 7-14 days post-injury. They also received low levels of immunosuppression for the next 60 days. Delivery of AST-OPC1 was successful in all five subjects with no serious adverse events associated with AST-OPC1. No evidence of rejection of AST-OPC1 was observed in detailed immune response monitoring of all patients. In four of the five patients, serial MRI scans indicated that reduced spinal cord cavitation may have occurred. Based on the results of this study, Asterias received clearance from FDA to progress testing of AST-OPC1 to patients with cervical spine injuries, which represents the first targeted population for registration trials.
About Asterias Biotherapeutics
Asterias Biotherapeutics, Inc. is a biotechnology company pioneering the field of regenerative medicine. The company's proprietary cell therapy programs are based on its pluripotent stem cell and immunotherapy platform technologies. Asterias is presently focused on advancing three clinical-stage programs which have the potential to address areas of very high unmet medical need in the fields of neurology and oncology. AST-OPC1 (oligodendrocyte progenitor cells) is currently in a Phase 1/2a dose escalation clinical trial in spinal cord injury. AST-VAC1 (antigen-presenting autologous dendritic cells) is undergoing continuing development by Asterias based on promising efficacy and safety data from a Phase 2 study in Acute Myeloid Leukemia (AML), with current efforts focused on streamlining and modernizing the manufacturing process. AST-VAC2 (antigen-presenting allogeneic dendritic cells) represents a second generation, allogeneic cancer immunotherapy. The company's research partner, Cancer Research UK, plans to begin a Phase 1/2a clinical trial of AST-VAC2 in non-small cell lung cancer in 2017. Additional information about Asterias can be found at http://www.asteriasbiotherapeutics.com.
FORWARD-LOOKING STATEMENTS
Statements pertaining to future financial and/or operating and/or clinical research results, future growth in research, technology, clinical development, and potential opportunities for Asterias, along with other statements about the future expectations, beliefs, goals, plans, or prospects expressed by management constitute forward-looking statements. Any statements that are not historical fact (including, but not limited to statements that contain words such as "will," "believes," "plans," "anticipates," "expects," "estimates") should also be considered to be forward-looking statements. Forward-looking statements involve risks and uncertainties, including, without limitation, risks inherent in the development and/or commercialization of potential products, uncertainty in the results of clinical trials or regulatory approvals, need and ability to obtain future capital, and maintenance of intellectual property rights. Actual results may differ materially from the results anticipated in these forward-looking statements and as such should be evaluated together with the many uncertainties that affect the businesses of Asterias, particularly those mentioned in the cautionary statements found in Asterias' filings with the Securities and Exchange Commission. Asterias disclaims any intent or obligation to update these forward-looking statements.
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SOURCE Asterias Biotherapeutics, Inc.
http://www.asteriasbiotherapeutics.com
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Presidential Symposium at the American Society of Gene and Cell ... - PR Newswire (press release)
Posted: May 6, 2017 at 3:44 pm
Study measures effects of apps on weight reduction.
As the prevalence of obesity continues to rise in the United States (35% in males, 40% in females in 2016), the risks of type 2 diabetes rises as well, supporting the need for ongoing weight control as a simple means of reducing diabetes risk. This concern is especially important in the aging adult population, where at least 66% of those over 65 meet current Centers for Disease Control (CDC) criteria for obesity or being overweight, while 51% of the same population is at risk for type 2 diabetes (US Census Bureau, 2014). The practice of healthy eating and regular exercise in overweight patients reduces the risk of progression from prediabetes or metabolic syndrome to diabetes. Several studies that support this notion have led to the American Association of Clinical Endocrinologists and the American College of Endocrinology including these lifestyle modifications in their first clinical practice guidelines in 2016.
Over time, lifestyle intervention programs were traditionally conducted in a face-to-face environment, typically with an in-person coach. While highly effective, patient barriers to participation (lack of access or lack of interest in this type of program) have demonstrated lack of response in patients who were unable to partake when compared to outcomes in patients who were able to do face-to-face sessions. Recent advances in technology have afforded patients with tools to remotely achieve high-level interaction with a live coach for behavioral counselling, group support, self-paced education, and development of important skills geared toward success. However, very few data are available looking at the Diabetes Prevention Program combined with technology use in older adults. With the rise of Internet availability to the aging population, C.M. Castro-Sweet et al. retrospectively observed a group of Medicare prediabetic patients, examining the effects of an Internet-based DDP digital application on weight status and progression to diabetes.
Subjects were selected from the Humana Medicare Advantage insurance program in the United States. High-risk participants were invited through a 3-week marketing campaign of direct mail, phone call and email contacts describing eligibility for the study as a benefit of their insurance plan. A total of 9,498 members were contacted, of which 501 eligible patients were enrolled. Eligibility was defined as presence of metabolic syndrome, or a combination of three of the following: prediabetes, hypertension, hyperlipidemia, and obesity. Weights were measured and electronically uploaded with an electronic scale. Using Internet-enabled devices (smart phones, computers, tablets), the participants partook in a 16-week intensive curriculum of weekly interactive lessons. Each module was available to all participants for one week at a time, allowing the patients to learn asynchronously but on the same schedule. The program application allowed participants to track weight loss, physical activity, and daily food intake. Following the 16-week weight loss program, a 36-week program focused on weight maintenance was started, for a total intervention length of 12 months. Throughout the duration of the study, all patients were monitored for psychological markers and overall progress using three different approved surveys: The World Health Organization-5 (WHO-5) Well-Being Index (measures subjective well-being), the Patient Health Questionnaire for Depression and Anxiety (PHQ-4), and the Summary of Diabetes Self-Care Activities (SDSCA) scale (reports self-management behaviors relating to diabetes).
Of the 501 participants (mean age 68.8 years), 95% completed at least 4 weekly lessons, and 92% completed 9 or more lessons in the initial 16-week phase. Only 2% failed to complete at least one lesson. Weight change from baseline was found to be significant (mean loss: -6.5%, SD 4.0 at 16 weeks, -8.0%, SD 7.7 at 6 months, and -7.5%, SD 7.8 at 12 months; p=0.01 for all). In the subset of patients (n=96) with available HbA1C and cholesterol measurements, the declines from baseline were also statistically significant (12-month improvement: HbA1c -0.14%, p=0.001; cholesterol -7.08 mg/dl, p=0.0001). As the self-reported surveys were optional, 57% (n=285) provided the surveys. The Who-5 Well-Being Index showed improvement in scores (+12%, p=0.0001), while the PHQ-4 Depression and Anxiety showed improvement in depression scores (-0.26, p=0.0001), but not in anxiety scores (-0.10, p=0.1922). The SDSCA also showed improvement in all 5 markers of healthy lifestyle, with statistical significance, with healthy modifications (better diet, exercise habits) increasing, and unhealthy behaviors (fast food consumption) decreasing.
This study found that willing participants enjoyed a high degree of success, meaningful engagement, and important benefits. Weight loss at for 6 and 12 months (8% and 7.5%) exceeded the standards set by the CDCs National Diabetes Prevention Program (5% reduction). Population presence in 37 states shows good geographic generalizability, supporting the scale that this approach could achieve in reaching patients. Limitations included a lack of a control group, although on average the subjects were not meeting physical activity standards, but by week 16, were all meeting the recommendations. A control group would have provided a better comparator for all data collection points in the 12-month period.
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Reference:
Castro Sweet CM, Chiguluri V, Gumpina R, et al. Outcomes of a Digital Health Program With Human Coaching for Diabetes Risk
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Diabetes Risk Reduction Seen with a Digital Health Program, Human Coaching - Diabetes In Control
Posted: May 6, 2017 at 3:44 pm
Stay active and track your reactions
Starstuff/ShutterstockWhen David Weingard was diagnosed with type 1 diabetes at the age of 36, he faced with some tough adjustments. From taking his new medication to monitoring his blood sugar, he fought to stay active and fit, eventually founding his diabetes coaching company, Fit4D. For Weingard, exercising had to remain a part of his life and he encourages other diabetics to do the same.
"Exercise is critical to long-term physical and mental health. Mentally, we need positive energy (and endorphins) to combat the 24/7 strain of the condition. Physically, we need to help our bodies stay strong and avoid the long-term effects and complications of diabetes," he says.
But to figure out how much you can withstand and what works for your body, he also notes that keeping track your reactions will help create a plan that works uniquely for you. "Detailed record keeping is a key factor in realizing the benefits of exercise and minimizing blood sugar swingsespecially highs and lows. You can reference these records to repeat workouts and your body should yield similar results most of the time," he says. Find out what the best exercises are for people with diabetes.
g stockstudio/ShutterstockThough Rachel Zucker is only 24 years old, she's been managing her type 1 diabetes diagnosis since she was four years old, making her quite the expert. She described diabetes as a full-time job: She had to accept that there are no days off, no breaks or vacations. That's why she recommends having supportive friends and family around you who will move with your highs and lowsthey're essential to keeping a good attitude and mindset. Instead of hiding your diagnosis, Zucker says wear it with pride. "I tell anybody and everybody close to me that I'm diabetic. Making sure people around you know you're diabetic can be life-saving in an emergency situation. In college, I made sure everybody around me knew I had type 1 diabetes, so when I went out to a party or to a sorority fundraising event, there was always someone looking out for me. Some people are afraid or embarrassed to tell others about their medical condition; I would highly encourage them not to be. Nobody has to do this alone," she says. Find out how fruit can lower yourdiabetes risk.
Andrey Popov/ShutterstockNow 67, Carol Geewasn't diagnosed with type 2 diabetes until her late 50s. Although her new life was scary at first, she says that leaning into the unknown helped her manage her new lifestyle and adjust her habits, ensuring that she lived vibrantly throughout middle age. "Diabetes is scary, but with knowledge comes power. Take the medications the way you are supposed to and it will get easier. I was afraid of needles, so I considered it a great victory when I injected myself without passing out. Know that you 'can' survive and thrive with diabetes. You just have to say itand more importantlybelieve it." (Learn what interval training can do for diabetes.)
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Sergey Nivens/ShutterstockAuthor and diabetes advocate, Quinn Nystrom first watched her younger brother get diagnosed with type 1 diagnosis. Then, two years later, she got the news herself at the age of 13. While that day was one of the worst of her life, she says, it's taught her many life lessons and led her to help others. Even though living with diabetes is a 24/7 job, she encourages those who are newly diagnosed to not let it define them. Instead, she says, allow it to refine you. "We are not just a broken down pancreas. We're more than a label that a doctor gives us, society tells us, and even sometimes what we tell ourselves. Seek to understand how the diagnosis of diabetes has brought light into your life. Find the positives," she says.
wavebreakmedia/ShutterstockThough it can be difficult to focus on anything but your blood sugar, Mella Barnes has found that keeping all parts of her health top of mind helps keep her more satisfied and balanced. She discovered her type 1 diabetes at the age of eight, and has been managing the illness ever since. She says that focusing on each day has been helpful. She also discovered that staying in touch with her emotions is crucial. "Take care of your mental and emotional health. This impacts your diabetes more than you think! Stress causes a lot of issues as well as a lack of sleep. If you're depressed or anxious about your diabetes, find a therapist or free support group. Do something that makes you happy every day," she says.
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Rawpixel.com/ShutterstockFred Winchar discovered he was a type 2 diabetic six years ago and has worked hard to manage his diagnosis. A successful businessman who knows the importance of good advice, he quickly realized that he needed to talk to someone who had been through the struggle. "When I first was diagnosed, I told a friend who was a type 1 diabetic, and he helped me learn how to test and monitor my sugars. He was one of the most energetic and happy people I have ever met. He was delighted to help another person on the same journey. Not only did I learn but I was able to bond in a special way with someone who knew what I was going through," he says.
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Simple Tricks for Living Well with Diabetesfrom People Who Have It - Reader's Digest
Posted: May 5, 2017 at 11:44 pm
Biotechin.Asia | 3D-printed patch could lead to new treatments for patients after a heart attack Biotechin.Asia In this study, researchers from the University of Minnesota-Twin Cities, University of Wisconsin-Madison, and University of Alabama-Birmingham used laser-based 3D-bioprinting techniques to incorporate stem cells derived from adult human heart cells on ... |
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3D-printed patch could lead to new treatments for patients after a heart attack - Biotechin.Asia
