October 15, 2018

The Clinical Research Center/Research Resources Office presented the Clinical Research Award for Third Quarter 2018 to Ananth Iyer, Project Manager, Quality Assurance/Quality Control, Department of Pediatrics – Research Resources Office.

This award was established by the Clinical Research Center in collaboration with the Research Resources Office to recognize and honor individual contributions to protecting the best interest of the research subjects and compliance with applicable rules and regulations.

“I joined the Research Resources Office (RRO) in 2014 as a Senior Research Coordinator and am currently in a Quality Assurance (QA) role,” said Iyer. “I take pride in providing support for good quality and compliance in the clinical trials that we undertake. I’m highly motivated by the care and options that clinical research studies provide to patients and never forget that their safety and protection is our top priority. I enjoy the teamwork with which clinical care and research come together in the RRO and I deeply appreciate the RRO for providing me (a lifelong student) with this opportunity.”

September 4, 2018

The Southwest Pediatric Device Consortium (SWPDC), anchored at Texas Children’s Hospital and Baylor College of Medicine, recently received a prestigious P50 grant from the U.S. Food and Drug Administration. The five-year, $6.75 million grant will begin on September 1 and will allow SWPDC to leverage ongoing activities to expand and accelerate the development of much-needed pediatric medical devices. The five principal investigators include Drs. Chester Koh and Henri Justino of Texas Children’s and Baylor, Dr. Balakrishna Haridas of Texas A&M University, Dr. Maria Oden of Rice University, and Dr. Michael Heffernan of Fannin Innovation Studio.

SWPDC supports pediatric device innovators with the goal of addressing the shortage of needed novel medical devices for children, a public health problem that has been acknowledged by the FDA.

“A great need currently exists for medical devices designed specifically for children,” said Koh, founder of SWPDC and lead principal investigator, as well as a pediatric urologist at Texas Children’s and professor of urology, pediatrics and Ob/Gyn at Baylor. “Pediatric device development is challenging, but with this support from the FDA, our consortium will continue to assist pediatric device innovators along all stages of development with the goal of improving our care of pediatric patients.”

The past decade has been a period of growth in adult medical device innovation. Advances in devices for children, however, have lagged far behind. Why the disparity? Economics are partially to blame. The market for pediatric devices is smaller, and thus the return on investment lower. Then there are the clinical and regulatory challenges. Pediatric device projects may need an extended life cycle before they can be approved and exposed to the external market. As a result, pediatric surgeons and pediatricians have had to make do with what’s available, often using retooled adult medical devices, and without adequate testing in children.

“Significant technical (design and manufacturing), preclinical testing, clinical and regulatory testing challenges exist in the field of pediatric devices,” said Haridas, co-founder and co-PI of SWPDC (lead PI at Texas A&M) and professor of practice in biomedical engineering at Texas A&M. “This FDA-funded SWPDC is uniquely positioned to address these challenges across the pediatric device development and clinical translational cycle to deliver significant advances in treatments tailored for pediatric patients.”

Support from the P50 grant will enable SWPDC to provide services in several areas: unmet needs assessment, prototype development, product and technology acceleration services, and business acceleration services. Consortium members will evaluate and support projects, as well as advise innovators throughout the total product life cycle. Based on individual project needs, the consortium will direct investigators to specific resources, collaborators and industry experts, and will coordinate the services offered by its member programs to identify, evaluate and assist pediatric device projects.

SWPDC includes clinical, scientific/engineering, investment, regulatory and academic partners in the Texas Medical Center, the Greater Houston area and the southwestern U.S. Primary partners include Texas A&M University, Rice University, University of Houston and Fannin Innovation Studio, and includes others such as Biotex Inc., Children’s Hospital of San Antonio, Children’s Health in Dallas and Phoenix Children’s Hospital, with additional future sites. SWPDC was selected as one of five national consortia that are addressing the shortage of pediatric devices.

To learn more about the Southwest Pediatric Device Consortium, visit swpdc.org.

August 13, 2018

Thanks to the more than 10 years of dedication and hard work of neonatologists and neonatal nutrition experts at Texas Children’s, Boston Children’s and the University of California at Los Angeles (UCLA) hospitals, premature babies with liver disease now have better access to a potentially lifesaving drug.

On July 27, the FDA approved Omegaven®, an omega-3 fatty-acid-rich lipid solution made from fish oil for use in babies suffering from Parenteral Nutrition Associated Cholestasis (PNAC). For over a decade, Texas Children’s and Boston Children’s have been giving Omegaven® to NICU babies with this disease under compassionate use protocols.

Nearly 20 percent of babies in the NICU develop intestinal failure and are dependent on artificial nutrition (total parenteral nutrition or TPN). Of these infants 25 percent to 50 percent of them develop PNAC, a dreaded complication resulting in long term damage to the liver. Prior to the use of Omegaven®, nearly 50 percent of these babies with PNAC developed liver failure and died, or needed a liver transplant. However, since the use of Omegaven®, 85 percent of infants with PNAC have survived long term, rarely needing a liver transplant.

Being first-hand witnesses to this remarkably improved outcome, neonatologists at Texas Children’s long advocated for the approval of Omegaven. Extensive patient data demonstrating the beneficial effects of Omegaven® on the babies with PNAC from Texas Children’s, Boston Children’s and UCLA formed a crucial part of the new drug approval application submitted to the FDA. Now that Omegaven® is approved by the FDA for use in babies with PNAC, hospitals across the country will have access to it, saving many lives.

“This is a game changer,” said neonatologist Dr. Muralidhar Premkumar. “I am very confident that Omegaven’s approval by the FDA will tremendously improve the outcomes of infants with intestinal failure.”

Program Director of Neonatal Nutrition Dr. Amy Hair agreed and said the approval is “huge, and will most definitely save babies’ lives.”

Over the past decade, liver transplants have decreased nearly 25 percent nationwide in babies with PNAC, mainly as a result of improved multi-disciplinary care of infants with intestinal failure and the use of safer lipid solutions such as Omegaven®. Texas Children’s Newborn Center sees about 25 to 30 babies with the PNAC a year. None of these babies in the past five years have needed a liver transplant.

Charlie and Henry Fitzpatrick were two of those babies. The now 4-year-old twin boys were born prematurely at Texas Children’s Hospital Pavilion for Women and spent six months in the NICU. During their stay, both infants suffered from PNAC and were treated with Omegaven®. Since then, both their liver disease and intestinal failure have resolved. Charlie and Henry are now happy healthy preschoolers.

The twins’ mother, Aly Fitzpatrick, said Omegaven® played a big part in saving her children’s lives and that she is pleased to hear that the FDA approved the drug for use in babies like hers.

“The approval opens up a world of possibilities for these babies,” she said. “Now, more hospitals can embrace this without having to join a research study.”

Because of their experience with the drug, Texas Children’s is uniquely positioned to educate staff at other hospitals about the benefits of Omegaven® and train them on how to use it.

“It gives me great pride and satisfaction that we were part of this successful effort,” Premkumar said. “I would like to thank our dieticians, physicians, NNPs, pharmacists, nurses and of course, the babies and their families who supported us in this endeavor.”

Premkumar added that none of this would have happened if Dr. Steven Abrams and Keli Hawthorne, both former Texas Children’s staff, had not brought this project to the hospital.

July 17, 2018

On July 9, Texas Children’s Hospital and the Angelman Syndrome Foundation celebrated the official opening of the Angelman Syndrome Clinic at Texas Children’s, one of only seven Angelman syndrome-specific clinics in the United States – and the first in Texas.

Several patient families gathered on the 16th floor of Wallace Tower to attend the ribbon cutting ceremony. Debbie Sukin, the daughter of the late Dr. Ralph Feigin, delivered the opening remarks and shared how this new clinic will benefit her 16-year-old son Jacob and so many other children diagnosed with Angelman Syndrome.

“The hardest part of advocating and managing a child with Angelman Syndrome is trying to understand what might be wrong when they aren’t feeling right, looking right or acting right,” Sukin said. “We’re confident that this multidisciplinary clinic here at Texas Children’s will provide all children the expertise and best chance at meeting their maximum potential and provide guidance to ensure the best outcome.”

Occurring in one in 15,000 live births, Angelman syndrome is a neurogenetic disorder often misdiagnosed as autism or cerebral palsy that causes severe neurological impairment. Often times, these patients experience developmental delays, lack of speech, seizures, and walking and balancing disorders.

With the creation of this new clinic established through the generosity of donors and in partnership with the ASF, patients and their families now can access multiple specialists – experts in Angelman syndrome, clinical geneticists, neurologist, psychiatrist, speech language pathologist, genetic counselors and social work – in one setting rather than visiting multiple locations across the nation to manage and treat this disease.

“Thanks to the help of the ASF, we are proud to further our mission of providing very specialized care for patients with Angelman syndrome,” said Chief of Neurology Dr. Gary Clark. “Given the science that’s being developed at the NRI at Texas Children’s in genetics and neurology, we are making great strides in helping these patients.”

The ribbon cutting event also included remarks from Dr. Carlos Bacino, chief of the Angelman Syndrome Clinic, Carrie Rys, assistant vice president, and Eileen Braun, executive director of the Angelman Syndrome Foundation, whose organization donated $50,000 to advance Angelman syndrome research at Texas Children’s.

“Opening this clinic brings us one step closer to our commitment to establish a network of clinics across the country,” Braun said. “We could not be more proud to support Texas Children’s and their remarkable team of professionals in bringing this clinic to life. We look forward to impacting the lives of so many more families.”

Click here to learn more about our Angelman Syndrome Clinic at Texas Children’s.

June 27, 2018

Scientists at Texas Children’s Hospital, Rice University and Baylor College of Medicine have won a prestigious National Institutes of Health grant to study the dynamic processes and cellular players linked to discrete subaortic stenosis (DSS), a congenital heart disease.

The $2.2 million, four-year R01 grant administered by the National Heart, Lung and Blood Institute will allow a team led by Dr. Sundeep Keswani and Jane Grande-Allen to develop computer and tissue-engineered models to predict the recurrence of DSS lesions of the left ventricular outflow tract (LVOT).

Keswani is a surgeon and director of surgical research at Texas Children’s and an associate professor in the Michael E. DeBakey Department of Surgery at Baylor. Grande-Allen is Rice’s Isabel C. Cameron Professor of Bioengineering and chair of the university’s Department of Bioengineering.

In normal hearts, blood passes through the tract from the left ventricle to the aorta. In patients with DSS, a fibrous tissue membrane forms and prevents blood flow from leaving the heart. Heart surgery is the only current option to manage the disease, but up to 30 percent of patients have an aggressive form of DSS in which the membrane recurs, and these patients may have to undergo further cardiothoracic surgery. The researchers believe a better understanding of how the fibrous membranes form will help doctors manage the disease.

“We have found some striking differences between patients who have the aggressive form of the disease and those who do not,” Keswani said. “These findings will give insight into the mechanism of how this membrane forms and perhaps why some humans have a more pro-fibrotic phenotype.

“This project is the just the beginning of understanding how different kinds of biomechanical forces interact with cells in the LVOT to produce fibrosis,” he said.

Working with co-investigator Philippe Sucosky of Wright State University, the research team has developed preliminary computational fluid dynamic models that mimic the complex shear forces and the altered geometry of the tract observed in DSS. The models will help the researchers develop a physical bioreactor to investigate interactions between the major cellular players in DSS: the endocardial endothelial cells that are exposed to shear forces, the cardiac fibroblasts that facilitate fibrosis and the circulating immune cells.

“Understanding the mechanisms of how altered shear forces induce fibrosis in the LVOT is a major gap in our knowledge,” Grande-Allen said. “If we can predict recurrence of DSS lesions of this outflow tract, we can change the way the disease is managed and really improve the quality of life for these children.”

Preliminary computer model data suggested that altered internal geometries in children’s hearts generate turbulence in the flow of blood. That affects the shear forces in the LVOT that in turn trigger an inflammatory response by endothelial cells and encourage the formation of fibrous tissue.

In the future, Grande-Allen and Keswani expect their work will help prevent fibrotic lesions by identifying targets in advance. That ability could also help treat other fibrotic cardiovascular diseases associated with altered flow.

Keswani said Texas Children’s, which U.S. News & World Report ranked No. 1 in the nation this week for pediatric cardiology and heart surgery, is uniquely suited to take on the project. “This world-class facility and tremendous volume have driven this project from the bedside to the bench, and this award will hopefully allow us to return to the bedside armed with new tools to help these patients,” he said. “This collaboration is the perfect weave between bioengineering and surgery.”

“Bioengineers bring a unique way of approaching surgical challenges and surgeons bring the surgical insight and the science, resulting in a synergistic relationship,” Keswani said. “In addition, it has been absolutely essential to the success of this project to have the support of our surgical leadership, who are strong advocates for surgical research as a means to develop innovative care for patients.”

Keswani is the principal investigator for Texas Children’s Laboratory for Regenerative Tissue Repair and a pediatric and fetal surgeon. His laboratory investigates the interaction of inflammation and the extracellular matrix that drives fibrosis with the goal of developing anti-fibrotic, regenerative therapies. Grande-Allen’s Integrative Matrix Mechanics Laboratory specializes in studying the composition and behavior of biological tissues, with a particular interest in heart valves.

The project also received support from the Virginia and L.E. Simmons Family Foundation Mini-Collaborative Research Fund and a gift from Lew and Laura Moorman.

June 19, 2018

Dr. Zhandong Liu, assistant professor at Baylor College of Medicine and researcher at the Jan and Dan Duncan Neurological Research Institute (NRI) at Texas Children’s has received a distinguished service award from the International Association for Intelligent Biology and Medicine (IAIBM).

As a non-profit organization, the IAIBM’s mission is to promote bioinformatics, systems biology and intelligent computing among scientists of diverse backgrounds through discussion, network communication, collaborations and education.

The Liu laboratory has developed many new software tools (e.g. MARRVEL and CRISPRcloud) that have helped biomedical scientists analyze complex bioinformatics data faster and with ease. In addition, Liu’s collaborations with neuroscientists have led to many important biological discoveries that have far-reaching implications for various neurological and neurodegenerative diseases.

Dr. Mingshan Xue, assistant professor of neuroscience at Baylor College of Medicine and a researcher with the NRI at Texas Children’s has been awarded the prestigious 2018 McKnight Scholar Award.

The McKnight Scholar Awards are granted to young scientists who are in the early stages of establishing their own independent laboratories and research careers and who have demonstrated a commitment to neuroscience.

Out of 64 applicants, Xue was one of six awardees. The recipients were chosen for their creativity, technological, precise and rigorous new approaches to overcome barriers in answering long-standing neuroscience questions.

The McKnight Endowment Fund for Neuroscience is an independent organization funded solely by the McKnight Foundation of Minneapolis, Minn., and led by a board of prominent neuroscientists from around the country. Each of the six McKnight Scholar Award recipients receives $75,000 per year for three years.

Dr. Hsiao-Tuan Chao, a child neurologist and postdoctoral researcher at the NRI, has been awarded the highly-competitive 2018 Burroughs Wellcome Fund Career Award for Medical Scientists.

The Burroughs Wellcome Fund is an independent private foundation dedicated to advancing the biomedical sciences by supporting research and other scientific and educational activities. The main goals of this fund are to help early-stage scientists develop into independent scientists and to advance undervalued areas of basic biomedical research. To fulfill that mission, the Career Award for Medical Scientists provides $700,000 of research funding spread over five years to physician-scientists who are currently receiving advanced postdoctoral/fellowship training. This award is intended to help physician-scientists embark on independent academic careers.

Chao, who is also an instructor in the department of pediatrics at Baylor College of Medicine, associate program director of the child neurology residency program and clinical child neurologist, is one of the 13 physician-scientists from leading academic institutions in the US to receive this honor in 2018. This award will help Chao establish an independent research program to understand the mechanisms underlying transcriptional dysregulation of inhibitory signaling pathways in childhood neurodevelopmental disorders.

June 12, 2018

As Texas Children’s continues to expand its physical footprint as a leader in pediatric care, Texas Children’s Neurology Program is growing by leaps and bounds. As one of the largest pediatric neurology service providers in the nation, more than 30,000 patient encounters occur each year at Texas Children’s Neuroscience Center.

Ranked No. 4 nationally in neurology and neurosurgery by U.S. News & World Report, patients are treated for a number of neurological conditions in our 13 specialty clinics including epilepsy, cerebral palsy, Rett syndrome, Batten disease and movement disorders. Many patient families are referred to Texas Children’s for our integrated expertise and multidisciplinary team approach in diagnosing and treating childhood neurological disorders. Recently, the Neurology division expanded its services with the opening of the Angleman Syndrome Clinic to treat children with this very rare, neurogenetic disorder.

“Over the last decade, our neuroscience programs have experienced phenomenal growth,” said Chief of Neurology and Developmental Neuroscience Dr. Gary Clark. “In 2004, the division had 5,000 patient encounters per year. Since then, we have more than quadrupled our patient encounters to 31,500 in 2017. More than half of these patients are seen in the community, allowing for greater access to care.”

In 2017, the neurology service received 1,300 new patient referrals each month which is a 30 percent increase from last year. To meet the increased demand for these services and to accommodate the growing patient population, the pediatric neurology division faculty expanded from 9 to 52 neurologists.

While enhancing access to patient care remains a top priority for Texas Children’s, one way the Neurology service is helping to achieve this collaborative milestone is through the implementation of telemedicine, an interactive telecommunications system that uses real-time video technology to link patients with their primary care and specialty providers. Instead of making the trek to the Texas Medical Center, patients can now “see” their neurologist without ever leaving their primary care clinic.

“Through an HRSA grant, we have seen children with epilepsy in one of our medical homes via this new technology and completed more visits than any other grant recipient,” said Brian Cordasco, practice administrator for Neurology. “This successful experiment will serve as a blueprint for future growth of telemedicine at Texas Children’s. Six faculty offices are now equipped with telemedicine capabilities.”

To balance outpatient services with the increasing demand for inpatient care, the Neurology Service has also developed an inpatient service rotation for physicians at Texas Children’s Hospital West Campus. Just like Texas Children’s Medical Center Campus and Texas Children’s Hospital The Woodlands, neurologists and epileptologists take turns working out of clinic to take neurology consults at the hospital.

Partnerships advance clinical research in neurology

The neurology division’s partnership with the Jan and Dan Duncan Neurological Research Institute (NRI) at Texas Children’s under the leadership of Dr. Huda Zoghbi also has helped to advance clinical and basic science research leading to the development of new treatments for neuro and developmental disabilities.

Nearly $185 million has been raised to build and partially endow the NRI, and the hospital’s long-time philanthropic partner, The Blue Bird Circle, has raised funds (initial gift $1M for four years) to form and partially endow the Blue Bird Circle Clinical Research Center in which NRI discoveries are realized through clinical trials.

The Blue Bird Circle has been the catalyst for so much of what has happened in pediatric neurology in the past 20 years at Texas Children’s. Clinical research protocols have grown from 9 to 63 over the 8-year existence of the Blue Bird Circle CRC. The Blue Bird Circle has raised in excess of $1.2 million annually to directly fund clinical treatment, training and research in pediatric neurology at Texas Children’s.

Through these collaborative research partnerships, the neurology team has made significant progress:

  • Texas Children’s is one of the few sites in the country that provides therapy to patients with an enzyme deficiency in the form of Batten disease by surgically injecting the synthesized enzyme directly into the brain thereby halting this degenerative form of Batten disease.
  • Texas Children’s treats patients with spinal muscular atrophy, a disease where the spinal cord degenerates due to a defective SMN1 gene. This treatment involves injecting a DNA molecule into the spinal fluid that turns a latent gene into an effective one that makes the deficient protein.

“We are contemplating similar treatments for Angelman and Rhett syndromes,” Clark said. “We are so grateful to Texas Children’s Blue Bird Circle Research Center in collaboration with the Jan and Dan Duncan Neurological Research Institute at Texas Children’s for helping us make these research breakthroughs possible, and I am excited about what the future will hold for pediatric neurology.”