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CAMBRIDGE, Mass.--(BUSINESS WIRE)--Cydan Development, Inc., an orphan drug accelerator dedicated to creating therapies that improve the lives of people living with rare genetic diseases, today announced its support for Rare Disease Day by making a gift to newly formed Children’s National Rare Disease Institute (CNRDI). This gift will go toward the CNRDI’s Innovation Fund, which supports new projects including diagnostics, devices, research, therapeutics, and process engineering, which will help advance the care and experience for children with rare diseases. “We are grateful for Cydan’s support of the Children’s National Rare Disease Institute,” said Marshall Summar, MD, chief of Genetics and Metabolism at Children’s National and chairman of the board at the National Organization for Rare Disorders (NORD). “With philanthropic investments from companies like Cydan, we will redefine the standard of care for children with genetic disorders, especially those largely unknown to the general medical community.” Chris Adams, Ph.D., co-founder and chief executive officer at Cydan, said, “We are proud to support Children’s National and this first-of-its-kind center in the U.S. focused exclusively on advancing the care and treatment of children and adults with rare genetic diseases. Rare diseases affect approximately 30 million Americans, and it’s heartening to see more resources dedicated to the identification, treatment and cure of these disorders.” As one of the first philanthropic contributions to the CNRDI Innovation Fund, Cydan’s gift will help to serve as a catalyst for rare disease care initiatives and help create an ever-expanding clinical knowledge base that advances the testing and development of rare disease therapies. Rare Disease Day® is an annual awareness day celebrated worldwide. This day is dedicated to elevating public understanding of rare diseases and calling attention to the special challenges faced by patients and their families. Rare Disease Day takes place every year on the last day of February to underscore the scientific, medical and social issues that affect this population. It was established in Europe in 2008 by EURORDIS, the organization representing rare disease patients in Europe, and is now observed in more than 80 nations. Rare Disease Day is sponsored by the National Organization for Rare Disorders® (NORD®), a leading independent, nonprofit organization committed to the identification, treatment, and cure of rare diseases. Cydan is an orphan drug accelerator dedicated to delivering therapies that will significantly improve the lives of people living with rare genetic diseases. Cydan evaluates experimental new therapies and advances those with the highest potential to be disease modifying treatments. Cydan’s goal is to launch new companies focused on developing promising therapies for rare genetic diseases with high unmet medical need. Vtesse, Cydan’s first new company, was launched in January 2015 and is developing a novel therapy for Niemann-Pick Disease Type C (NPC). Imara was launched in 2016 and is developing a novel treatment for Sickle Cell disease. Cydan was founded in 2013 by a management team with extensive drug discovery, clinical development and business development experience and financed by leading life sciences investors NEA, Pfizer Venture Investments, Lundbeckfond Ventures, Bay City Capital and Alexandria Venture Investments. The accelerator is based in Tech Square in Cambridge, Mass. For more information, visit http://www.cydanco.com or contact Cydan at info@cydanco.com. Children’s National Health System, based in Washington, DC, has been serving the nation’s children since 1870. Children’s National is ranked in the top 20 in every specialty evaluated by U.S. News & World Report; one of only four children’s hospitals in the nation to earn this distinction. Designated a Leapfrog Group Top Hospital and a two-time recipient of Magnet® status, this pediatric academic health system offers expert care through a convenient, community-based primary care network and specialty outpatient centers. Home to the Children’s Research Institute and the Sheikh Zayed Institute for Pediatric Surgical Innovation, Children’s National is one of the nation’s top NIH-funded pediatric institutions. Children’s National is recognized for its expertise and innovation in pediatric care and as a strong voice for children through advocacy at the local, regional and national levels. For more information, visit ChildrensNational.org.

News Article | February 17, 2017
Site: www.eurekalert.org

WASHINGTON, DC - (Feb. 17, 2017) - The cutting-edge biocompatible near-infrared 3D tracking system used to guide the suturing in the first smart tissue autonomous robot (STAR) surgery has the potential to improve manual and robot-assisted surgery and interventions through unobstructed 3D visibility and enhanced accuracy, according to a study published in the March 2017 issue of IEEE Transactions on Biomedical Engineering. The study successfully demonstrates feasibility in live subjects (in-vivo) and demonstrates 3D tracking of tissue and surgical tools with millimeter accuracy in ex-vivo tests. More accurate and consistent suturing helps reduce leakage, which can improve surgical outcomes. Authored by the development team from Sheikh Zayed Institute for Pediatric Surgical Innovation at Children's National Health System and funded by the National Institutes of Health, the study explains the design of the 3D tracking system with near-infrared fluorescent (NIRF) markers and, using robotic experiments, compares its tracking accuracies against standard optical tracking methods. At speeds of 1 mm/second, the team observed tracking accuracies of 1.61 mm that degraded only to 1.71 mm when the markers were covered in blood and tissue. "A fundamental challenge in soft-tissue surgery is that target tissue moves and deforms, becomes occluded by blood or other tissue, which makes it difficult to differentiate from surrounding tissue," says Axel Krieger, Ph.D., senior author on the study and program lead for Smart Tools at the Sheikh Zayed Institute. "By enabling accurate tracking of tools and tissue in the surgical environment, this innovative work has the potential to improve many applications for manual and robot-assisted surgery." The system is made up of small biocompatible NIRF markers with a novel fused plenoptic and near-infrared (NIR) camera tracking system, enabling 3D tracking that can overcome blood and tissue occlusion in an uncontrolled, rapidly changing surgical environment. Krieger explains that the NIR imaging has the potential to overcome occlusion problems because NIR light penetrates deeper than visual light. "This work describes the 'super human eyes' and a bit of 'intelligence' of our STAR robotic system, making tasks such as soft tissue surgery on live subjects possible," explains Peter C. Kim, M.D., vice president and associate surgeon in chief of the Sheikh Zayed Institute. Future work will include further integration and evaluation of the tracking system in image-guided medical interventions such as robotic surgeries. The Sheikh Zayed Institute for Pediatric Surgical Innovation at Children's National Health System is a hub for innovation focused on making pediatric surgery more precise, less invasive and pain free. It was founded in 2010 through a $150 million gift from the Government of Abu Dhabi. By combining research and clinical work in the areas of imaging, bioengineering, pain medicine, immunology and personalized medicine, the institute's physicians and scientists are developing leading-edge knowledge, tools and procedures that will benefit children globally.

WASHINGTON--(BUSINESS WIRE)--Emerging data from Children’s National Health System are providing hope that a new approach to cell therapy may effectively harness the cancer-killing potential of the natural immune system as a treatment for patients with blood and bone marrow cancers for whom stem cell transplantation has not worked. These patients typically have few treatment options and experience very high mortality rates. Initial results from the Phase 1 RESOLVE trial, a multi-institutional, Phase 1 dose-ranging study co-led by Kirsten Williams, M.D., and Catherine Bollard, M.D., M.B.Ch.B., Chief of the Division of Allergy and Immunology and Director of the Program for Cell Enhancement and Technologies for Immunotherapy (CETI) at Children’s National, showed that the majority (78 percent) of patients responded to multi tumor-associated antigen specific lymphocytes (TAA-L) treatment, and 44 percent of patients achieved complete remission with limited toxicity. The trial included patients diagnosed with one of four tumors who were treated with TAA-L upon relapse of disease post stem cell transplant. “These initial findings suggest that non-genetically engineered antigen-specific lymphocytes can be isolated, expanded and adoptively transferred to severely ill patients with active disease and positively impact tumor regression,” says Dr. Bollard. “We are encouraged by the promise of these data, which support similar efforts to exploit the immunotherapeutic potential of the natural T-cell repertoire.” The data, presented at the BMT Tandem meeting in Orlando, Florida, was a collaboration between investigators at Children’s National and researchers at Johns Hopkins University and Baylor College of Medicine who are testing a novel approach to treating cancer. Their approach includes introducing TAA-L – a natural T-cell subtype from the immune system – to high-risk patients with advanced hematologic or blood cancers including acute myeloid leukemia (AML)/ Myelodysplastic Syndromes (MDS), B-cell acute lymphoblastic leukemia (ALL) and Hodgkin lymphoma. The trial evaluates the safety and efficacy of both donor and patient-derived TAA-L as a novel treatment for patients with AML/MDS, B-cell ALL or Hodgkin lymphoma who are in active disease relapse pre- or post-allogeneic hematopoietic stem cell transplantation (HSCT). Patients with refractory and relapsed AML, MDS, ALL and Hodgkin lymphoma often have extremely poor clinical outcomes. For patients with these malignancies who relapse after allogeneic stem cell transplantation, the prognosis is even more dismal. Sadly, one-year mortality rates for this population approach 90 percent. The results include patients receiving adoptively transferred TAA-L manufactured cells across all dosing cohorts allowed per protocol (dose levels one through four), 10 patients in total. Preliminary observations conclude that ex vivo manufactured TAA-L, composed primarily of central effector memory T-cells, can be successfully isolated and expanded to clinically relevant numbers, cryopreserved, and safely infused to patients after relapse of disease. There were no cases of graft-versus-host disease or other autoimmune-mediated toxicity reported, and there have been no observations of Cytokine Release Syndrome or neurotoxicity associated with TAA-L treatment. The RESOLVE trial has been and/or is funded by a National Institutes of Health grant (P01CA015396), the Leukemia Lymphoma Society, Ben’s Run and Hyundai Hope on Wheels. The BMT Tandem meeting is the combined annual meetings of the Center for International Blood & Marrow Transplant Research and the American Society for Blood and Marrow Transplantation. Children’s National Health System, based in Washington, DC, has been serving the nation’s children since 1870. Children’s National is ranked in the top 20 in every specialty evaluated by U.S. News & World Report; one of only four children’s hospitals in the nation to earn this distinction. Designated a Leapfrog Group Top Hospital and a two-time recipient of Magnet® status, this pediatric academic health system offers expert care through a convenient, community-based primary care network and specialty outpatient centers. Home to the Children’s Research Institute and the Sheikh Zayed Institute for Pediatric Surgical Innovation, Children’s National is one of the nation’s top NIH-funded pediatric institutions. Children’s National is recognized for its expertise and innovation in pediatric care and as a strong voice for children through advocacy at the local, regional and national levels. For more information, visit ChildrensNational.org, or follow us on Facebook and Twitter.

Almeida L.E.F.,University of Maryland Baltimore County | Almeida L.E.F.,Sheikh Zayed Institute for Pediatric Surgical Innovation | Roby C.D.,University of Maryland Baltimore County | Krueger B.K.,University of Maryland Baltimore County | Krueger B.K.,University of Maryland, Baltimore
Molecular and Cellular Neuroscience | Year: 2014

Human fetal exposure to valproic acid (VPA), a widely-used anti-epileptic and mood-stabilizing drug, leads to an increased incidence of behavioral and intellectual impairments including autism; VPA administration to pregnant rats and mice at gestational days 12.5 (E12.5) or E13.5 leads to autistic-like symptoms in the offspring and is widely used as an animal model for autism. We report here that this VPA administration protocol transiently increased both BDNF mRNA and BDNF protein levels 5-6-fold in the fetal mouse brain. VPA exposure in utero induced smaller increases in the expression of mRNA encoding the other neurotrophins, NT3 (2.5-fold) and NT4 (2-fold). Expression of the neurotrophin receptors, trkA, trkB and trkC were minimally affected, while levels of the low-affinity neurotrophin receptor, p75NTR, doubled. Of the nine 5'-untranslated exons of the mouse BDNF gene, only expression of exons I, IV and VI was stimulated by VPA in utero. In light of the well-established role of BDNF in regulating neurogenesis and the laminar fate of postmitotic neurons in the developing cortex, an aberrant increase in BDNF expression in the fetal brain may contribute to VPA-induced cognitive disorders by altering brain development. © 2014 Elsevier Inc.

A newly developed robot was able to complete a pig intestine surgery on its own and better than human surgeons, a new research has shown. Advancements in artificial intelligence coupled with robotics are on the rise because of the numerous advantages they pose, but some express concern that they could be replacing human workers, particularly in health care. Every year, more than 50 million people undergo risky surgeries in the U.S., with thousands dying due to errors in surgery. A past study even revealed that half of total post-surgical complications are due to errors of the surgeon. Since robots have previously helped in hospital settings, the researchers programmed a Smart Tissue Autonomous Robot (STAR) which turned out to perform a better pig intestine surgery than human surgeons. Without any help from humans, STAR was able to stitch the pig's bowels consistently — evenly spaced and more durable than what humans can do. Thanks to STAR, the pig is expected to have fewer complications. What Makes STAR Better Than Human Surgeons? Surgeons have good vision, skilful hands, and a wealth of information about which procedures to perform for a particular patient. Kim and his colleagues wanted to clone these skills and apply them to an experimental robot. STAR has autonomous operation, which makes it capable of performing tasks on its own. It has near infrared imaging and 3D camera that allow it to see minute details inside the body that humans cannot. The robot is also equipped with minimally invasive surgical tools and force sensor so it can gauge suture tightness. STAR was also programmed to learn appropriate surgical techniques to perform for several scenarios. By doing the open bowel surgery, STAR performed better than human surgeons in all aspects — whether the human surgeons used hands, laparoscopic, or robot assistant. STAR was able to operate on a scale of as much as 60 percent on the intestines with no adjustments from its human counterpart. "Even though we take pride in our surgical procedures, to have a machine or tool that works with us in assuring better outcome safety and reducing complications is a tremendous benefit," said senior author and pediatric surgeon Dr. Peter Kim. "The purpose wasn't to replace surgeons, but if you have an intelligent tool that works with a surgeon, can it improve the outcome?" asked Dr. Kim, who is also an associate surgeon-in-chief of the Children's National Health System and vice-president of Sheikh Zayed Institute for Pediatric Surgical Innovation. The robot was consistently monitored and guided by surgeons who are also in the operating room. The robotic movements simply complement the actions of the human surgeon. Kim explained that STAR is a mere motorized tool controlled by surgeons. "It has no intelligence whatsoever," Kim said. Senior editor Megan Frisk of the journal Science Translational Medicine where the study was published on May 4 said STAR is a promising development for robotics that could significantly help lower human errors in providing healthcare. More studies should be done to back the development before it can be used for more intricate surgeries, however. Weill Cornell Medical College associate professor of surgery Dr. Rasa Zarnegar said healthcare embracing robotics is inevitable because some of the surgical operations are better performed when automated. People will accept robotic surgeries especially if these provide better and safer outcomes, said Zarnegar. Because STAR was only able to perform four successful pig intestine surgeries, Zarnegar believes that tests must be carried out on a larger scale before these could be considered successful. While STAR has not done any human surgeries so far, Kim believes that the robot surgeon could be in hospital in two to three years' time. © 2016 Tech Times, All rights reserved. Do not reproduce without permission.

Saglio F.,Regina Margherita Childrens Hospital | Hanley P.J.,Sheikh Zayed Institute for Pediatric Surgical Innovation | Bollard C.M.,Sheikh Zayed Institute for Pediatric Surgical Innovation
Cytotherapy | Year: 2014

Adoptive immunotherapy-in particular, T-cell therapy-has recently emerged as a useful strategy with the potential to overcome many of the limitations of antiviral drugs for the treatment of viral complications after hematopietic stem cell transplantation. In this review, we briefly summarize the current methods for virus-specific T-cell isolation or selection and we report results from clinical trials that have used these techniques, focusing specifically on the strategies aimed to broaden the application of this technology. © 2014 International Society for Cellular Therapy.

Merritt S.A.,X-Nav Technologies | Khare R.,Sheikh Zayed Institute for Pediatric Surgical Innovation | Bascom R.,Pennsylvania State Hershey Medical Center | Higgins W.E.,Pennsylvania State University
IEEE Transactions on Medical Imaging | Year: 2013

Bronchoscopy is a major step in lung cancer staging. To perform bronchoscopy, the physician uses a procedure plan, derived from a patient's 3D computed-tomography (CT) chest scan, to navigate the bronchoscope through the lung airways. Unfortunately, physicians vary greatly in their ability to perform bronchoscopy. As a result, image-guided bronchoscopy systems, drawing upon the concept of CT-based virtual bronchoscopy (VB), have been proposed. These systems attempt to register the bronchoscope's live position within the chest to a CT-based virtual chest space. Recent methods, which register the bronchoscopic video to CT-based endoluminal airway renderings, show promise but do not enable continuous real-time guidance. We present a CT-video registration method inspired by computer-vision innovations in the fields of image alignment and image-based rendering. In particular, motivated by the Lucas-Kanade algorithm, we propose an inverse-compositional framework built around a gradient-based optimization procedure. We next propose an implementation of the framework suitable for image-guided bronchoscopy. Laboratory tests, involving both single frames and continuous video sequences, demonstrate the robustness and accuracy of the method. Benchmark timing tests indicate that the method can run continuously at 300 frames/s, well beyond the real-time bronchoscopic video rate of 30 frames/s. This compares extremely favorably to the 1 s/frame speeds of other methods and indicates the method's potential for real-time continuous registration. A human phantom study confirms the method's efficacy for real-time guidance in a controlled setting, and, hence, points the way toward the first interactive CT-video registration approach for image-guided bronchoscopy. Along this line, we demonstrate the method's efficacy in a complete guidance system by presenting a clinical study involving lung cancer patients. © 1982-2012 IEEE.

Peters C.A.,Sheikh Zayed Institute for Pediatric Surgical Innovation
Journal of Endourology | Year: 2011

The emergence of robot-assisted surgical technology has permitted application of laparoscopic pyeloplasty to the pediatric age group to a much wider degree than previously possible. The challenging learning curve for conventional laparoscopic pyeloplasty, particularly in infants, has hindered its widespread application. Robot-assisted pyeloplasty in children has been clearly shown to provide an equally effective, safe, and reasonably efficient means to repair ureteropelvic junction obstruction with more rapid hospital discharge and less postoperative analgesic requirements. Precise port placement, adjusted to the child's anatomy and size, delicate anastomosis, and use of postoperative stent appear to be important elements for successful repair. The procedure has become reproducible and in some centers is exclusively used over open repair. The specific procedural steps are detailed and the potential limitations and complications are reviewed, as well as the limited available data in the literature. Copyright 2011, Mary Ann Liebert, Inc.

Schwartz J.A.T.,Sheikh Zayed Institute for Pediatric Surgical Innovation
Journal of Pediatric Surgery | Year: 2014

Innovations are indispensable to the practice and advancement of pediatric surgery. Children represent a special type of vulnerable population and must be protected since they do not have legal capacity to consent, and their parent's judgment may be compromised in circumstances when the child is very ill or no adequate therapy exists. In an effort to protect patients, legislators could pass and enforce laws that prohibit or curtail surgical innovations and thus stifle noble advancement of the practice. The goals of this paper are, 1) To clearly define the characteristics of surgical innovation types so interventions may be classified into 1 of 3 distinct categories along a continuum: Practice Variation, Transition Zone, and Experimental Research, and 2) To propose a practical systematic method to guide surgeon decision-making when approaching interventions that fall into the "Transition Zone" category on the Surgical Intervention Continuum. The ETHICAL model allows those that know the intricacies and nuances of pediatric surgery best, the pediatric surgeons and professional pediatric surgical societies, to participate in self-regulation of innovation in a manner that safeguards patients without stifling creativity or unduly hampering surgical progress. © 2014 Elsevier Inc.

Hanley P.J.,Sheikh Zayed Institute for Pediatric Surgical Innovation | Bollard C.M.,Sheikh Zayed Institute for Pediatric Surgical Innovation
Viruses | Year: 2014

Cytomegalovirus, of the Herpesviridae family, has evolved alongside humans for thousands of years with an intricate balance of latency, immune evasion, and transmission. While upwards of 70% of humans have evidence of CMV infection, the majority of healthy people show little to no clinical symptoms of primary infection and CMV disease is rarely observed during persistent infection in immunocompetent hosts. Despite the fact that the majority of infected individuals are asymptomatic, immunologically, CMV hijacks the immune system by infecting and remaining latent in antigen-presenting cells that occasionally reactivate subclinically and present antigen to T cells, eventually causing the inflation of CMV-specific T cells until they can compromise up to 10% of the entire T cell repertoire. Because of this impact on the immune system, as well as its importance in fields such as stem cell and organ transplant, the relationship between CMV and the immune response has been studied in depth. Here we provide a review of many of these studies and insights into how CMV-specific T cells are currently being used therapeutically. © 2014 by the authors; licensee MDPI, Basel, Switzerland.

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