Interdisciplinary Stem Cell Institute

Miami, FL, United States

Interdisciplinary Stem Cell Institute

Miami, FL, United States
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News Article | November 2, 2016
Site: www.marketwired.com

12th Annual Stem Cell Action Awards to be presented at World Stem Cell Summit December 7, in West Palm Beach, Florida WEST PALM BEACH, FL --(Marketwired - November 02, 2016) - Four distinguished honorees have been selected for the 2016 Stem Cell Action Awards. For 12 years, the nonprofit organization, now known as Regenerative Medicine Foundation, has honored the stem cell and regenerative medicine community's top innovators, leaders, and champions. The honorees are recognized at a gala reception and dinner on Wednesday, December 7 during the 12th annual World Stem Cell Summit & RegMed Capital Conference at the Palm Beach County Convention Center in West Palm Beach, Florida. This year's Stem Cell Action Award Honorees include a path-finding diabetes researcher motivating the biomedical community to take a stand for cures, two visionary philanthropists strategically building translational research projects and a devoted mother, turned advocate, who is creating awareness about the scourge of childhood cancer. All four have devoted themselves to accelerating stem cell and regenerative medicine to improve health and deliver cures. Meet the 2016 Stem Cell Action Award Honorees: Leadership Award: Bernie Marcus - Stem cell and regenerative medicine benefactor, Bernie Marcus, is a recipient of a "Leadership" award for his visionary strategic philanthropy. The co-founder and former CEO of The Home Depot, has provided grants through the Marcus Foundation, to fund the new Georgia Tech Marcus Center for Therapeutic Cell Characterization and Manufacturing (MC3M), as well as clinical stem cell trials and research in the treatment of autism, stroke and cerebral palsy at Duke, asthma and lung disease at the University of Miami and Case Western Reserve, neonatal heart disease at Emory and Children's Hospital of Atlanta, and cancer cellular therapy trials at City of Hope, Penn, Miami and MD Anderson. The Marcus Foundation has also created centers of excellence in Autism, Stroke, Trauma, Emergency Medicine, Integrative Health, Neuroscience, and Heart disease in Atlanta, Philadelphia, and Boca Raton. Leadership Award: Don Soffer - The celebrated Florida real estate developer Don Soffer, who created the thriving Aventura community, will be honored with a "Leadership" award. Under his stewardship, the Soffer family provided a transformative $25 million gift to the Interdisciplinary Stem Cell Institute (ISCI) at the University of Miami Miller School of Medicine. The contribution has boosted ISCI into the highest orbit of translational regenerative medicine. Advocacy Award: Camillo Ricordi, M.D. - Dr. Ricordi is the director of the Diabetes Research Institute and Cell Transplant Program at the University of Miami Miller School of Medicine. Acknowledged as one of the world's leading scientists in diabetes cure-focused research and cell transplantation, Dr. Ricordi has author over 1,000 scientific publications. He has been awarded 25 patents including the automated method and Ricordi Chamber used to obtain large numbers of insulin producing cells from the human pancreas. The technology was used in the first successful islet cell transplants and is still used worldwide for clinical islet transplantation. He is currently working on immunomodulation and stem cell strategies to reverse autoimmunity, and on biologic replacement platform technologies "BioHUB" for the cure of diabetes without the need for anti-rejection drugs. Dr. Ricordi has also devoted himself to supporting the cause of stem cell research. He is a founding member of the science advisory board of Genetics Policy Institute and in 2004 he spoke at the organization's seminal meeting at the United Nations headquarters. He founded and served as the first president of The Cure Alliance, an important advocacy nonprofit organization whose primary goal is to promote collaborative efforts and help accelerate potential cures from the laboratory to the bedside. Inspiration Award: Sandy Barker and The Gold Rush Cure Foundation - Sandy Barker and her husband Gary founded Gold Rush Cure Foundation in memory of their beloved son, Christian Gordon Barker. Besides supporting children battling cancer via their "Pot of Gold" program, the foundation commits itself to advocating both locally and nationally for increased awareness and research funding for childhood cancer. Sandy will be making her 20th advocacy trip in February 2017. She has distinguished herself as a key spokesperson and advocate supporting an accelerated regulatory pathway for regenerative cellular therapies. Sandy sees the vital need for improvement in this area as she mentors numerous families affected by childhood cancer. Some of the previous Stem Cell Action awardees have included Michael J. Fox, Brooke Ellison, Robert Klein, Bob Nerem, Susan Solomon, Ann Murphy, Mary Ann Liebert, Research!America, National Association of Biology Teachers, Juvenile Diabetes Research Foundation (JDRF), the Huntington's Disease "grassroots" advocacy community, ALS Worldwide, and philanthropists like A. Alfred Taubman and T. Denny Sanford. The World Stem Cell Summit & RegMed Capital Conference is the original translational regenerative medicine conference. The Summit aims to accelerate the discovery and development of lifesaving cures and therapies, bringing global stakeholders together to solve global challenges. The 2016 Summit Organizing Partners are: Regenerative Medicine Foundation (RMF), Center for Advancement of Science in Space (CASIS), Mayo Clinic, Kyoto University Institute for Integrated Cell-Material Sciences (iCeMS), Interdisciplinary Stem Cell Institute at the University of Miami Miller School of Medicine, Wake Forest Institute for Regenerative Medicine, Diabetes Research Institute Foundation, The Cure Alliance, Centre for Commercialization for Regenerative Medicine (CCRM) and Nova Southeastern University Cell Therapy Institute, More than 225 speakers and panelists will discuss the latest scientific discoveries, funding opportunities, translational issues, legal and regulatory solutions, and best practices. The event is expected to attract more than 1,200 attendees from 35 nations. The Summit will be held December 6-9, 2016, at the Palm Beach County Convention Center, West Palm Beach, Florida. Regenerative Medicine Foundation (formerly called Genetics Policy Institute) is a 501(c)(3) nonprofit organization dedicated to accelerating regenerative medicine to improve health and deliver cures. We pursue our mission by producing the World Stem Cell Summit & RegMed Capital Conference, honoring community leaders through the Stem Cell Action Awards, supporting our official journal partner STEM CELLS Translational Medicine published by AlphaMed Press, organizing educational and policy initiatives and fostering strategic collaborations. RMF maintains offices in Florida and California. For more information about RMF, visit www.regmedfoundation.org. Visit worldstemcellsummit.com and follow @WSCSummit on Twitter. For information about sponsoring or attending the World Stem Cell Summit and the Stem Cell Action Awards, contact Alan Fernandez at (650) 847-1640 or email alan@regmedfoundation.org.


News Article | December 13, 2016
Site: www.prweb.com

Scott P. Bruder, M.D., Ph.D., Chairman of the parent company of Amendia, Inc., spoke at the World Stem Cell Summit on December 8 at the Palm Beach County Convention Center in West Palm Beach, Florida. Dr. Bruder’s presentation was titled “Our Journey from Autologous to Allogeneic Stem Cell Therapy: How did we get here & when do we use what?” and was delivered in collaboration with three other leading experts as part of the event’s cell therapy program. The World Stem Cell Summit & RegMed Capital Conference is the original, translation-focused global meeting of industry stakeholders. Now in its 12th year, and with over 1,200 attendees, 225 speakers, and 90-plus hours of programming, the World Stem Cell Summit serves as the choice venue for forging collaborations with worldwide pioneers across multiple disciplines. "This summit is the premier opportunity to illuminate and explore the business of regenerative medicine on a worldwide scale,” said Dr. Bruder. “My presentation on allogeneic cells with Timothy Ganey, Ph.D., Lou Barnes, and Lisa Ferrara, Ph.D. is one of several important and distinguished cell-manufacturing discussions that focus on translation and commercialization for the present and future of stem cell medicine.” Over the course of his 25-year career in healthcare, Dr. Bruder has united principles from basic science, clinical medicine, and industrial development to deliver products that enhance patients’ lives and improve outcomes. As a veteran physician, scientist, and executive, he now leads Bruder Consulting International, LLC, providing a variety of services to medical device, biotechnology, and regenerative medicine entities. Currently, he serves as Chairman of the Board of Directors of the parent company of Amendia, Inc., helping to develop biologic implants and medical devices for spine applications. “Dr. Bruder’s command of the business of regenerative medicine, and influence in healthcare as a whole, is invaluable to the present and future of Amendia’s spine device and biologics innovation,” said Amendia CEO Chris Fair. “We’re honored to have such an insightful luminary on our team.” About Regenerative Medicine Foundation The World Stem Cell Summit is a project of the nonprofit Regenerative Medicine Foundation. Since 2003, Regenerative Medicine Foundation and its predecessor Genetics Policy Institute have built the strongest, most comprehensive and trusted global network of Regenerative Medicine stakeholders, uniting the world's leading researchers, medical centers, universities, labs, businesses, funders, policymakers, experts in law, regulation and ethics, medical philanthropies and patient organizations. Our mission is to accelerate regenerative medicine to improve health and deliver cures. We are committed to the ethical advancement of innovative medicine powered by regenerative, restorative, and curative technologies. Learn more at http://www.regmedfoundation.org. About 2016 WSCS The World Stem Cell Summit is the "window to the world" to stem cell research and regenerative medicine. It is the original, translation-focused global meeting of stakeholders, now celebrating its 12th year. With 1,200+ attendees, 225+ speakers and 90+ hours of programming it is no surprise that attendees travel from 30+ countries in order to attend. WSCS16 is organized by the Center for the Advancement of Science in Space (CASIS), the sole manager of the International Space Station U.S. National Laboratory; Centre For Commercialization Of Regenerative Medicine (Canada); Diabetes Research Institute Foundation; Kyoto University Institute for Integrated Cell Material Science; Mayo Clinic; NOVA Southeastern University Cell Therapy Institute; The Cure Alliance; University of Miami Miller School of Medicine ISCI (Interdisciplinary Stem Cell Institute); and Wake Forest School of Medicine Institute for Regenerative Medicine. Learn more at http://www.worldstemcellsummit.com. For more information on the World Stem Cell Summit & RegMed Capital Conference, and Dr. Bruder’s presentation, visit the event website. About Amendia Headquartered in a state-of-the-art manufacturing facility in Marietta, GA, Amendia is a leading designer, developer, manufacturer and marketer of medical devices used in spinal surgical procedures. Amendia’s mission is to exceed surgeon and patient expectations by creating balanced solutions with disruptive technologies for medical devices paired with biologics and instrumentation. Amendia’s vertically-integrated strategy focuses on improving surgical outcomes and the lives of patients with spinal disorders. For more information, please visit http://www.amendia.com.


WEST PALM BEACH, FL--(Marketwired - November 29, 2016) - Organizers of the Equine World Stem Cell Summit (EWSCS) are pleased to announce a partnership with the North American Veterinary Regenerative Medicine Association (NAVRMA). The Equine World Stem Cell Summit will be held as a dedicated showcase track of the esteemed World Stem Cell Summit on December 7-9 at the Palm Beach County Convention Center in West Palm Beach, FL. Riders, owners, trainers, veterinarians, and more are welcome to attend and learn more about this exciting and wide-ranging topic. Bernie Siegel, Founder & Chair of the World Stem Cell Summit, stated, "We are excited to partner with NAVRMA. They are a committee of some of the most respected research scientists and veterinary practitioners in the industry, and they share our mission to accelerate regenerative medicine to improve health and deliver cures, whether for humans or animals." The EWSCS will welcome Dr. Alan Nixon, Chair of the Board of Directors at NAVRMA, as a speaker at the summit. Dr. Nixon is the Director of the Comparative Orthopaedics Laboratory at Cornell University. Dr. Nixon obtained his veterinary degree from the University of Sydney in 1978 and completed a surgical residency and research degree at Colorado State University in 1983. After five years in the Department of Surgical Sciences at the University of Florida, Dr. Nixon moved to New York in 1988, where he is currently a professor in the Department of Clinical Sciences at Cornell University. Dr. Nixon's research includes joint pathobiology and cartilage repair with growth factor gene-enhanced chondrocyte and stem cell transplantation techniques, genetic characterization of OCD in animals and man using microarray expression studies, and clinical application of growth factor recombinant proteins and gene therapy for improved joint, tendon, and bone repair. "We are excited to participate in the Equine World Stem Cell Summit and believe NAVRMA and EWSCS is a natural partnership," said Dr. Nixon. "We encourage professional improvement and the exchange of knowledge and ideas among people interested in veterinary regenerative medicine. The summit is the perfect place to share information and encourage learning not only for veterinarians and researchers, but for interested owners, riders, trainers, and breeders in the equine industry." Throughout the three-day conference, produced by the non-profit Regenerative Medicine Foundation, attendees will hear from industry-leading veterinarians and researchers. As the single conference uniting the global stem cell community, the WSCS provides a platform for the equine community to interact with leading researchers and institutions, as well as industry, investor, and philanthropic groups. The conference attracts approximately 1,000 attendees from 40 countries with 225 speakers and program participants. Registration for the Equine World Stem Cell Summit is $500 for the three-day track. Sign up online and use the code "EQUINERM" today! To learn more about the 12th Annual World Stem Cell Summit and the Equine World Stem Cell Summit and to find out how you can get involved as a sponsor or attendee, visit www.worldstemcellsummit.com or email Alan Fernandez at alan@regmedfoundation.org. The Equine World Stem Cell Summit is a dedicated track of the World Stem Cell Summit, to be held December 6-9 at the Palm Beach County Convention Center in West Palm Beach, FL. Throughout the conference, leading scientists and veterinarians will present fellow researchers, veterinarians, owners, trainers, riders, and interested public with the latest information on the regenerative medicine that is transforming the care and treatment of horses. The World Stem Cell Summit, produced by the Regenerative Medicine Foundation strives to unite, educate, and empower the global stem cell and regenerative medicine communities and to create a supportive environment for the field. The principal organizing partners for the 2016 World Stem Cell Summit include the Regenerative Medicine Foundation, Mayo Clinic, Kyoto University Institute for Integrated Cell Material Science, Center for Advancement of Science in Space (CASIS), Wake Forest Institute for Regenerative Medicine, Interdisciplinary Stem Cell Institute at the University of Miami Miller School of Medicine, Nova Southeastern University, CCRM and the Cure Alliance. To learn more, visit www.worldstemcellsummit.com.


News Article | December 21, 2016
Site: www.biosciencetechnology.com

The 4-month-old on the operating table has a shocking birth defect, nearly half his heart too small or even missing. To save him, surgeons will have to totally reroute how his blood flows, a drastic treatment that doesn't always work. So this time they are going a step further. In a bold experiment, doctors injected donated stem cells directly into the healthy side of Josue Salinas Salgado's little heart, aiming to boost its pumping power as it compensates for what's missing. It's one of the first attempts in the U.S. to test if stem cells that seem to help heart attack survivors repair cardiac muscle might help these tiniest heart patients, too. "We think the young heart is able to be more responsive," said Dr. Sunjay Kaushal, chief of pediatric cardiac surgery at the University of Maryland Medical Center, who is leading the study in partnership with University of Miami researchers. Kaushal bent over the baby's right ventricle, the part of the heart that will take over for the abnormal left side. The surgeon had repaired as much as possible for now. Next he measured where to place eight shots of precious stem cells. Then the bustling operating room went silent as Kaushal helped fellow surgeon Dr. Si M. Pham guide tiny needles into the ventricle's muscle. "We're not saying we're going to cure it," Kaushal said of the birth defect, called hypoplastic left heart syndrome. But, "my whole quest is to see if we can make these little kids do better." Josue's parents knew there was no guarantee the experimental injections would make a difference. But their son had been hospitalized since birth and needed open-heart surgery anyway for a chance to go home. Teary-eyed, they clasped hands and prayed over Josue's crib moments before nurses wheeled him to the operating room. "We are marching ahead with God," said Josue's father, Hidelberto Salinas Ramos, speaking in Spanish through a hospital interpreter. Nearly 1,000 babies are born with hypoplastic left heart syndrome in the U.S. each year. It's the most complex cardiac birth defect. Josue is missing his left ventricle, the main pumping chamber that pushes oxygen-rich blood to the body. Other key structures on his heart's left side are too small or malformed to work. Always lethal until a few decades ago, this defect now is treated with three open-heart surgeries performed between birth and age 3. Doctors route blood around the abnormal left heart and they convert the right ventricle - which normally would shuttle oxygen-poor blood to the lungs - into the main pumping chamber. Today, about 65 percent survive at least five years, and many reach adulthood, said Dr. Kristin Burns, a pediatric cardiologist at the National Institutes of Health. But too many children still die or require a heart transplant because the right ventricle wears out under its increased workload. That's why doctors are conducting this early-stage study of whether stems cells might help that ventricle work better. "This is very different than a surgical approach or giving a medicine just to treat the symptoms. This is trying to treat the underlying problem," said Burns, of NIH's National Heart, Lung and Blood Institute. "I know you're really nervous," Kaushal told Josue's father, placing a hand on his shoulder. "Everything is going to be fine." Just 4 months old, Josue was undergoing his second open-heart surgery. The first operation, a day after his birth, was a temporary fix to keep his heart pumping and create an aorta, the main artery leading to the body, big enough for blood to flow. While he babbled happily at his family and nurses, Josue never got strong enough to be discharged to his Edgewater, Maryland, home. This time, Kaushal would take pressure off Josue's right ventricle - and inject those stem cells. Even in adults, stem cell regeneration is highly experimental. But small studies involving heart attack survivors and older adults with heart failure have found what Dr. Denis Buxton, a stem cell specialist at NIH's heart institute, calls a modest benefit in how well their hearts pump blood. For testing in babies, Kaushal turned to Dr. Joshua Hare at the University of Miami's Interdisciplinary Stem Cell Institute, who researches a specific type of stem cell donated from the bone marrow of healthy adults. Hare's institute freezes these so-called mesenchymal stem cells, which have a long safety record in adult studies, and is providing them free for the baby study. Initially, Kaushal's team tested piglets, whose hearts are similar to humans. When the right ventricle was damaged, they found stem cell injections preserved the piglets' heart tissue. Apparently the cells spurred some of the heart's existing repair mechanisms. Back in the Baltimore operating room, University of Miami researchers helped Kaushal's nurses thaw the frozen stem cells and prepare injections. A few feet away, Kaushal was moving a large vein so it will drain deoxygenated blood from the top of Josue's body straight to his lungs without having to enter that overworked right ventricle. (When he's 3, Josue will need a final operation to similarly reroute blood returning from his lower body.) Then, just before his chest was stitched back up, Josue became the second baby with this defect to receive the experimental bone marrow stem cells. It's an early-stage study that will compare 30 babies, half given stem cells, to see if the strategy is safe and shows any difference over surgery alone. If so, it could open stem cell research for other pediatric heart problems. Other types of stem cells also are being explored for hypoplastic left heart syndrome. Mayo Clinic researchers have tested stem cells taken from affected babies' umbilical cord blood. Kaushal also plans to try stem cells from affected newborns' own heart tissue, something researchers in Japan are pursuing. It will take several years to know if stem cell experiments work. But, like many babies after their second surgery for the heart defect, Josue bounced back - and a week later, finally got to go home.


News Article | December 21, 2016
Site: www.chromatographytechniques.com

In a first-in-children randomized clinical study, medical researchers at the University of Maryland School of Medicine (UM SOM) and the Interdisciplinary Stem Cell Institute (ISCI) at the University of Miami Miller School of Medicine have begun testing to see whether adult stem cells derived from bone marrow benefit children with the congenital heart defect hypoplastic left heart syndrome (HLHS). UM SOM surgeons are injecting the cells into the babies’ hearts during open-heart operations at the University of Maryland Medical Center. ISCI is supplying the stem cells for the procedures. Even with extensive surgical treatments, HLHS babies still do not have optimal outcomes. The researchers hope the cells will increase the babies’ chances of survival as HLHS limits the heart's ability to pump blood from the heart to the body. “The premise of this clinical trial is to boost or regenerate the right ventricle, the only ventricle in these babies, to make it pump as strongly as a normal left ventricle,” says lead researcher Sunjay Kaushal, MD, PhD, associate professor of surgery, University of Maryland School of Medicine and director, pediatric cardiac surgery, University of Maryland Medical Center. “We are hoping this therapy will be a game-changer for these patients.” Kaushal says the first two patients, who were both four-months-old when the stem cells were injected, are doing well after their surgery. This is the first HLHS research in the United States to use stem cells known as allogeneic mesenchymal stem cells (MSC). Allogeneic cells can be used in other human beings without creating an immune response, which could cause the body to reject the cells. Additionally, these cells are a type of adult stem cell (found in both children and adults), unspecialized cells that can develop into tissue- or organ-specific cells. MSCs can be harvested in advance, expanded in culture, and stored for use later. The allogeneic nature of the MSCs makes it possible for stem cells from one bone marrow donor to provide all the stem cells for this study. Researchers elsewhere are taking a different approach to strengthen the HLHS heart, with autologous cells, stem cells taken from the HLHS patient's own umbilical cord, for use in that specific patient. In adult patients, MSCs in the heart have been shown to reduce scar tissue, reduce inflammation, cause new small vessels to grow, and stimulate the heart to regenerate itself, causing heart muscle cells and cardiac stem cells to grow. "We've had incredible results in using mesenchymal stem cells to regenerate damaged heart muscle in adults," says Joshua M. Hare, MD, ISCI founding director and sponsor of the study. “This is the first time these types of cells are being used in infants, so this is very exciting.” The Interdisciplinary Stem Cell Institute has grown from a local research center to a national cell manufacturing facility. ISCI provides cells for the Cardiovascular Cell Therapy Research Network, has been named a Production Assistance for Cellular Therapies Center by the National Heart, Lung and Blood Institute, and has been conducting research in stem cell use for cardiovascular repair since 2008. HLHS is one of the most challenging and complex congenital heart diseases to treat. The Centers for Disease Control and Prevention (CDC) estimates that about 960 babies in the United States are born each year with HLHS. For unknown reasons, the heart’s main pumping chamber, the left ventricle, does not develop completely during a critical growth period just prior to birth. The right ventricle normally pumps blood to the lungs at low pressure to be oxygenated, while the left ventricle pushes blood at high pressure through the aorta to the entire body. In children with HLHS, the right heart assumes the extra workload, temporarily supporting the circulation to both the lungs and body. That stress can cause the right heart to fail and the baby to die. Current HLHS treatment options are either a heart transplant or a series of three open-heart reconstructive surgical procedures to connect the left and right sides of the heart. However, even with a transplant or the reconstructive surgical series, children with HLHS have an average five-year survival of only 50 to 60 percent. In this Phase 1 safety and efficacy study, allogeneic MSCs are injected into the heart muscle during the second of the three reconstructive surgeries, typically performed at approximately four months of age. A total of 30 patients with HLHS will be enrolled in the study. Fifteen patients will receive six-to-eight stem cell injections each, based on the size of the heart, while 15 control patients will not receive the cells. This is an open-label trial, in which researchers and participant families will know whether or not the cells are administered. Kaushal laid the groundwork for this trial eight years ago as he began exploring the possibilities of stem cells to strengthen children’s hearts. Kaushal says he and his team developed many models trying to understand how these cells work in the laboratory before moving to a clinical application. “There’s a lot of basic science behind what we’re doing. I want to make sure that what we pursue is rigorous in the laboratory, to make sure that we’re providing the best therapy for these little kids.” Several researchers at the School of Medicine’s University of Maryland Center for Stem Cell Biology & Regenerative Medicine have added their expertise to the effort, collaborating with Dr. Kaushal to understand and develop stem cell therapy for children with heart failure. “Dr. Kaushal and colleagues have discovered that the failing neonatal heart is actually a rich source of cardiac stem cells, but the existing stem cells in the hearts of these babies are not sufficient to overcome HLHS,” says Curt I. Civin, MD, professor of pediatrics and physiology, director of the Center for Stem Cell Biology & Regenerative Medicine, and Associate Dean for Research at the University of Maryland School of Medicine. “We are close to understanding one mechanism underlying this insufficiency. This line of research is a key part of our quest to use stem cells to repair, cure and prevent severe diseases in children and adults.” In previously published research, Kaushal demonstrated that mesenchymal stem cells can restore function in a pre-clinical model replicating many of the features of HLHS. The stem cells remodeled the heart muscle (myocardium) similar to normal myocardium. Stem cells in the heart may also secrete growth factors conducive to forming heart muscle and keeping the muscle from dying. “These key findings suggested these cells would work for HLHS patients,” says Kaushal. While stem cells have been used to regenerate adult hearts, Kaushal says improvements have been marginal. His research suggests results may be better in pediatric hearts: “The heart is able to remodel better in a younger patient than an older patient, because the body is still growing, good things are going on, and things are not deteriorating.” Civin, a pediatric oncologist, says his very first patient as a pediatric intern-in-training years ago was an infant with HLHS. “I’ve seen how devastating HLHS can be for babies and their families. I’m thrilled with the launch of this first-in-children stem cell therapeutic trial, and look forward to the patient outcomes.” The Department of Surgery at the University of Maryland School of Medicine is providing funding for the clinical costs associated with this trial.


News Article | December 20, 2016
Site: www.eurekalert.org

Baltimore, MD - Dec. 20, 2016 - In a first-in-children randomized clinical study, medical researchers at the University of Maryland School of Medicine (UM SOM) and the Interdisciplinary Stem Cell Institute (ISCI) at the University of Miami Miller School of Medicine have begun testing to see whether adult stem cells derived from bone marrow benefit children with the congenital heart defect hypoplastic left heart syndrome (HLHS). UM SOM surgeons are injecting the cells into the babies' hearts during open-heart operations at the University of Maryland Medical Center. ISCI is supplying the stem cells for the procedures. Even with extensive surgical treatments, HLHS babies still do not have optimal outcomes. The researchers hope the cells will increase the babies' chances of survival as HLHS limits the heart's ability to pump blood from the heart to the body. "The premise of this clinical trial is to boost or regenerate the right ventricle, the only ventricle in these babies, to make it pump as strongly as a normal left ventricle," says lead researcher Sunjay Kaushal, MD, PhD, associate professor of surgery, University of Maryland School of Medicine and director, pediatric cardiac surgery, University of Maryland Medical Center. "We are hoping this therapy will be a game-changer for these patients." Kaushal says the first two patients, who were both four-months-old when the stem cells were injected, are doing well after their surgery. This is the first HLHS research in the United States to use stem cells known as allogeneic mesenchymal stem cells (MSC). Allogeneic cells can be used in other human beings without creating an immune response, which could cause the body to reject the cells. Additionally, these cells are a type of adult stem cell (found in both children and adults), unspecialized cells that can develop into tissue- or organ-specific cells. MSCs can be harvested in advance, expanded in culture, and stored for use later. The allogeneic nature of the MSCs makes it possible for stem cells from one bone marrow donor to provide all the stem cells for this study. Researchers elsewhere are taking a different approach to strengthen the HLHS heart, with autologous cells, stem cells taken from the HLHS patient's own umbilical cord, for use in that specific patient. In adult patients, MSCs in the heart have been shown to reduce scar tissue, reduce inflammation, cause new small vessels to grow, and stimulate the heart to regenerate itself, causing heart muscle cells and cardiac stem cells to grow. "We've had incredible results in using mesenchymal stem cells to regenerate damaged heart muscle in adults," says Joshua M. Hare, MD, ISCI founding director and sponsor of the study. "This is the first time these types of cells are being used in infants, so this is very exciting." The Interdisciplinary Stem Cell Institute has grown from a local research center to a national cell manufacturing facility. ISCI provides cells for the Cardiovascular Cell Therapy Research Network, has been named a Production Assistance for Cellular Therapies Center by the National Heart, Lung and Blood Institute, and has been conducting research in stem cell use for cardiovascular repair since 2008. HLHS is one of the most challenging and complex congenital heart diseases to treat. The Centers for Disease Control and Prevention (CDC) estimates that about 960 babies in the United States are born each year with HLHS. For unknown reasons, the heart's main pumping chamber, the left ventricle, does not develop completely during a critical growth period just prior to birth. The right ventricle normally pumps blood to the lungs at low pressure to be oxygenated, while the left ventricle pushes blood at high pressure through the aorta to the entire body. In children with HLHS, the right heart assumes the extra workload, temporarily supporting the circulation to both the lungs and body. That stress can cause the right heart to fail and the baby to die. Current HLHS treatment options are either a heart transplant or a series of three open-heart reconstructive surgical procedures to connect the left and right sides of the heart. However, even with a transplant or the reconstructive surgical series, children with HLHS have an average five-year survival of only 50-60 percent. In this Phase 1 safety and efficacy study, allogeneic MSCs are injected into the heart muscle during the second of the three reconstructive surgeries, typically performed at approximately four months of age. A total of 30 patients with HLHS will be enrolled in the study. Fifteen patients will receive six-to-eight stem cell injections each, based on the size of the heart, while 15 control patients will not receive the cells. This is an open-label trial, in which researchers and participant families will know whether or not the cells are administered. Kaushal laid the groundwork for this trial eight years ago as he began exploring the possibilities of stem cells to strengthen children's hearts. Kaushal says he and his team developed many models trying to understand how these cells work in the laboratory before moving to a clinical application. "There's a lot of basic science behind what we're doing. I want to make sure that what we pursue is rigorous in the laboratory, to make sure that we're providing the best therapy for these little kids." Several researchers at the School of Medicine's University of Maryland Center for Stem Cell Biology & Regenerative Medicine have added their expertise to the effort, collaborating with Dr. Kaushal to understand and develop stem cell therapy for children with heart failure. "Dr. Kaushal and colleagues have discovered that the failing neonatal heart is actually a rich source of cardiac stem cells, but the existing stem cells in the hearts of these babies are not sufficient to overcome HLHS," says Curt I. Civin, MD, professor of pediatrics and physiology, director of the Center for Stem Cell Biology & Regenerative Medicine, and Associate Dean for Research at the University of Maryland School of Medicine. "We are close to understanding one mechanism underlying this insufficiency. This line of research is a key part of our quest to use stem cells to repair, cure and prevent severe diseases in children and adults." In previously published research, Kaushal demonstrated that mesenchymal stem cells can restore function in a pre-clinical model replicating many of the features of HLHS. The stem cells remodeled the heart muscle (myocardium) similar to normal myocardium. Stem cells in the heart may also secrete growth factors conducive to forming heart muscle and keeping the muscle from dying. "These key findings suggested these cells would work for HLHS patients," says Kaushal. While stem cells have been used to regenerate adult hearts, Kaushal says improvements have been marginal. His research suggests results may be better in pediatric hearts: "The heart is able to remodel better in a younger patient than an older patient, because the body is still growing, good things are going on, and things are not deteriorating." Civin, a pediatric oncologist, says his very first patient as a pediatric intern-in-training years ago was an infant with HLHS. "I've seen how devastating HLHS can be for babies and their families. I'm thrilled with the launch of this first-in-children stem cell therapeutic trial, and look forward to the patient outcomes." The Department of Surgery at the University of Maryland School of Medicine is providing funding for the clinical costs associated with this trial. "Dr. Kaushal's research will give families with a devastating diagnosis hope for a better outcome for their child," says Stephen T. Bartlett, MD, the Peter Angelos Distinguished Professor; Chair of the Department of Surgery at the University of Maryland School of Medicine; and Surgeon-in-Chief and Executive Vice President of the University of Maryland Medical System. "The Department of Surgery's funding of this project demonstrates the critical need and the promise this research holds for a very at-risk population who only have a 50/50 shot at survival with current treatment protocols." "This novel therapeutic approach exemplifies how our faculty are unrelenting in their search for new ways to improve the health of some of our tiniest and most vulnerable patients," says E. Albert Reece, MD, PhD, MBA, vice president for medical affairs, University of Maryland; the John Z. and Akiko K. Bowers Distinguished Professor; and dean, University of Maryland School of Medicine." This stem cell therapy may provide a new treatment option not just for patients with HLHS, but also for patients with other congenital heart problems." For more details on this study, see identifier NCT02398604 on clinicaltrials.gov. About the University of Maryland School of Medicine The University of Maryland School of Medicine was chartered in 1807 and is the first public medical school in the United States. It continues today as an innovative leader in accelerating innovation and discovery in medicine. The School of Medicine is the founding school of the University of Maryland and is an integral part of the 11-campus University System of Maryland. Located on the University of Maryland's Baltimore campus, the School of Medicine works closely with the University of Maryland Medical Center and Medical System to provide a research-intensive, academic and clinically based education. With 43 academic departments, centers and institutes and a faculty of more than 3,000 physicians and research scientists plus more than $400 million in extramural funding, the School is regarded as one of the leading biomedical research institutions in the U.S. with top-tier faculty and programs in cancer, brain science, surgery and transplantation, trauma and emergency medicine, vaccine development and human genomics, among other centers of excellence. The School is not only concerned with the health of the citizens of Maryland and the nation, but also has a global presence, with research and treatment facilities in more than 35 countries around the world. http://medschool. . About the University of Maryland Medical Center The University of Maryland Medical Center (UMMC) is comprised of two hospitals in Baltimore: an 800-bed teaching hospital - the flagship institution of the 12-hospital University of Maryland Medical System (UMMS) - and a 200-bed community teaching hospital, UMMC Midtown Campus. UMMC is a national and regional referral center for trauma, cancer care, neurocare, cardiac care, diabetes and endocrinology, women's and children's health, and has one of the largest solid organ transplant programs in the country. All physicians on staff at the flagship hospital are faculty physicians of the University of Maryland School of Medicine. At UMMC Midtown Campus, faculty physicians work alongside community physicians to provide patients with the highest quality care. UMMC Midtown Campus was founded in 1881 and is located one mile away from the University Campus hospital. For more information, visit http://www. .


News Article | December 20, 2016
Site: www.rdmag.com

The 4-month-old on the operating table has a shocking birth defect, nearly half his heart too small or even missing. To save him, surgeons will have to totally reroute how his blood flows, a drastic treatment that doesn't always work. So this time they are going a step further. In a bold experiment, doctors injected donated stem cells directly into the healthy side of Josue Salinas Salgado's little heart, aiming to boost its pumping power as it compensates for what's missing. It's one of the first attempts in the U.S. to test if stem cells that seem to help heart attack survivors repair cardiac muscle might help these tiniest heart patients, too. "We think the young heart is able to be more responsive," said Dr. Sunjay Kaushal, chief of pediatric cardiac surgery at the University of Maryland Medical Center, who is leading the study in partnership with University of Miami researchers. Kaushal bent over the baby's right ventricle, the part of the heart that will take over for the abnormal left side. The surgeon had repaired as much as possible for now. Next he measured where to place eight shots of precious stem cells. Then the bustling operating room went silent as Kaushal helped fellow surgeon Dr. Si M. Pham guide tiny needles into the ventricle's muscle. "We're not saying we're going to cure it," Kaushal said of the birth defect, called hypoplastic left heart syndrome. But, "my whole quest is to see if we can make these little kids do better." Josue's parents knew there was no guarantee the experimental injections would make a difference. But their son had been hospitalized since birth and needed open-heart surgery anyway for a chance to go home. Teary-eyed, they clasped hands and prayed over Josue's crib moments before nurses wheeled him to the operating room. "We are marching ahead with God," said Josue's father, Hidelberto Salinas Ramos, speaking in Spanish through a hospital interpreter. Nearly 1,000 babies are born with hypoplastic left heart syndrome in the U.S. each year. It's the most complex cardiac birth defect. Josue is missing his left ventricle, the main pumping chamber that pushes oxygen-rich blood to the body. Other key structures on his heart's left side are too small or malformed to work. Always lethal until a few decades ago, this defect now is treated with three open-heart surgeries performed between birth and age 3. Doctors route blood around the abnormal left heart and they convert the right ventricle — which normally would shuttle oxygen-poor blood to the lungs — into the main pumping chamber. Today, about 65 percent survive at least five years, and many reach adulthood, said Dr. Kristin Burns, a pediatric cardiologist at the National Institutes of Health. But too many children still die or require a heart transplant because the right ventricle wears out under its increased workload. That's why doctors are conducting this early-stage study of whether stems cells might help that ventricle work better. "This is very different than a surgical approach or giving a medicine just to treat the symptoms. This is trying to treat the underlying problem," said Burns, of NIH's National Heart, Lung and Blood Institute. "I know you're really nervous," Kaushal told Josue's father, placing a hand on his shoulder. "Everything is going to be fine." Just 4 months old, Josue was undergoing his second open-heart surgery. The first operation, a day after his birth, was a temporary fix to keep his heart pumping and create an aorta, the main artery leading to the body, big enough for blood to flow. While he babbled happily at his family and nurses, Josue never got strong enough to be discharged to his Edgewater, Maryland, home. This time, Kaushal would take pressure off Josue's right ventricle — and inject those stem cells. Even in adults, stem cell regeneration is highly experimental. But small studies involving heart attack survivors and older adults with heart failure have found what Dr. Denis Buxton, a stem cell specialist at NIH's heart institute, calls a modest benefit in how well their hearts pump blood. For testing in babies, Kaushal turned to Dr. Joshua Hare at the University of Miami's Interdisciplinary Stem Cell Institute, who researches a specific type of stem cell donated from the bone marrow of healthy adults. Hare's institute freezes these so-called mesenchymal stem cells, which have a long safety record in adult studies, and is providing them free for the baby study. Initially, Kaushal's team tested piglets, whose hearts are similar to humans. When the right ventricle was damaged, they found stem cell injections preserved the piglets' heart tissue. Apparently the cells spurred some of the heart's existing repair mechanisms. Back in the Baltimore operating room, University of Miami researchers helped Kaushal's nurses thaw the frozen stem cells and prepare injections. A few feet away, Kaushal was moving a large vein so it will drain deoxygenated blood from the top of Josue's body straight to his lungs without having to enter that overworked right ventricle. (When he's 3, Josue will need a final operation to similarly reroute blood returning from his lower body.) Then, just before his chest was stitched back up, Josue became the second baby with this defect to receive the experimental bone marrow stem cells. It's an early-stage study that will compare 30 babies, half given stem cells, to see if the strategy is safe and shows any difference over surgery alone. If so, it could open stem cell research for other pediatric heart problems. Other types of stem cells also are being explored for hypoplastic left heart syndrome. Mayo Clinic researchers have tested stem cells taken from affected babies' umbilical cord blood. Kaushal also plans to try stem cells from affected newborns' own heart tissue, something researchers in Japan are pursuing. It will take several years to know if stem cell experiments work. But, like many babies after their second surgery for the heart defect, Josue bounced back — and a week later, finally got to go home.


News Article | November 22, 2016
Site: www.marketwired.com

Press Registration & Interviews: To apply for press registration credentials, visit worldstemcellsummit.com/media/. Advance registration is requested. On-site and remote interviews can be arranged. What: A full day of stem cell education designed for patients, doctors, the general public and students. Price for the day is $30 for general admission. Free admission for students. Learn how stem cell scientists conduct research in space; meet an astronaut; enjoy dazzling art created by youth inspired by cell biology; and discover anti-aging technologies, possible cures for macular degeneration, insights into clinical trials, rescuing species from extinction with stem cell technology, and more! What: Celebrating its 12th year, the 2016 World Stem Cell Summit (#wscs16) brings scientists, clinicians, philanthropists, investors and patient advocates from more than 30 countries to share breakthroughs that were once thought impossible. The Summit meets to accelerate the discovery and development of lifesaving cures and therapies. This is an ideal event for health, science, biotech and business reporters to interview the world's stem cell leaders and pioneers. Some interviews may be able to be scheduled in advance of the Summit. The World Stem Cell Summit is a project of the nonprofit Regenerative Medicine Foundation. Since 2003, Regenerative Medicine Foundation and its predecessor Genetics Policy Institute have built the strongest, most comprehensive and trusted global network of Regenerative Medicine stakeholders, uniting the world's leading researchers, medical centers, universities, labs, businesses, funders, policymakers, experts in law, regulation and ethics, medical philanthropies and patient organizations. Our mission is to accelerate regenerative medicine to improve health and deliver cures. We are committed to the ethical advancement of innovative medicine powered by regenerative, restorative, and curative technologies. Learn more at www.regmedfoundation.org. The World Stem Cell Summit is the "window to the world" to stem cell research and regenerative medicine. It is the original, translation-focused global meeting of stakeholders, now celebrating its 12th year. With 1,200+ attendees, 225+ speakers and 90+ hours of programming it is no surprise that attendees travel from 30+ countries in order to attend. WSCS16 is organized by the Center for the Advancement of Science in Space (CASIS), the sole manager of the International Space Station U.S. National Laboratory; Centre For Commercialization Of Regenerative Medicine (Canada); Diabetes Research Institute Foundation; Kyoto University Institute for Integrated Cell Material Science; Mayo Clinic; NOVA Southeastern University Cell Therapy Institute; The Cure Alliance; University of Miami Miller School of Medicine ISCI (Interdisciplinary Stem Cell Institute); and Wake Forest School of Medicine Institute for Regenerative Medicine. Learn more at www.worldstemcellsummit.com.


Li J.,Interdisciplinary Stem Cell Institute | Vargas M.A.X.,University of Connecticut Health Center | Kapiloff M.S.,Interdisciplinary Stem Cell Institute | Dodge-Kafka K.L.,University of Connecticut Health Center
Experimental Cell Research | Year: 2013

The calcium/calmodulin-dependent protein phosphatase calcineurin is required for the induction of transcriptional events that initiate and promote myogenic differentiation. An important effector for calcineurin in striated muscle is the transcription factor myocyte enhancer factor 2 (MEF2). The targeting of the enzyme and substrate to specific intracellular compartments by scaffold proteins often confers specificity in phosphatase activity. We now show that the scaffolding protein mAKAP organizes a calcineurin/MEF2 signaling complex in myocytes, regulating gene transcription. A calcineurin/mAKAP/MEF2 complex can be isolated from C2C12 cells and cardiac myocytes, and the calcineurin/MEF2 association is dependent on mAKAP expression. We have identified a peptide comprising the calcineurin binding domain in mAKAP that can disrupt the binding of the phosphatase to the scaffold in vivo. Dominant interference of calcineurin/mAKAP binding blunts the increase in MEF2 transcriptional activity seen during myoblast differentiation, as well as the expression of endogenous MEF2-target genes. Furthermore, disruption of calcineurin binding to mAKAP in cardiac myocytes inhibits adrenergic-induced cellular hypertrophy. Together these data illustrate the importance of calcineurin anchoring by the mAKAP scaffold for MEF2 regulation. © 2012 Elsevier Inc..


Hatzistergos K.E.,Interdisciplinary Stem Cell Institute | Hare J.M.,Interdisciplinary Stem Cell Institute | Hare J.M.,University of Miami
Circulation Research | Year: 2016

After 2 recent genetic studies in mice addressing the developmental origins1 and regenerative activity of cardiac cKit+ cells,2 2 additional reports by Sultana et al3 and Liu et al4 provide further information on the expression of cKit in the embryonic and adult hearts. Here, we synthesize the findings from the 4 distinct cKit models to gain insights into the biology of this important cell type. ©2016 American Heart Association, Inc.

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