May 15, 2018

The Society for Pediatric Research (SPR) recently gave two Texas Children’s trainees national research awards for their outstanding research on kidney disease at this year’s annual Pediatric Academic Societies’ SPR meeting.

Joseph Alge, a medical resident in the Pediatrician-Scientist Training & Development Program (PSTDP) at Baylor College of Medicine (BCM), received the SPR House Officer Research Award, and Rachel Shenoi, a junior at The University of Houston and participant in the 2017 SMART program at BCM, received the SPR Student Research Award. The SPR annually honors students engaged in pediatric research to encourage pediatricians in training to pursue careers in academic pediatrics.

According to Dr. Michael Braun, Texas Children’s Hospital physician, these accomplishments are worth celebrating because it is rare for an institution to receive one award, let alone two awards in the same research area.

“It’s very exciting,” said Braun. “These awards are not only a recognition of the quality of the science being done, but also the individuals working at Texas Children’s Hospital.”

Dr. Scott Wenderfer, Texas Children’s physician and BCM Assistant Professor, has been Rachel Shenoi’s mentor throughout her research process.

“Rachel is exactly the type of person we want to be bringing into Texas Children’s Hospital and into Pediatric Nephrology,” said Wenderfer. “Investments in ambitious and talented undergraduates will provide exposure to the unmet needs and growing opportunities for Pediatric Research. Her success has been a pleasure to witness as a mentor.”

SPR’s mission is to create a network of multidisciplinary researchers that can connect and collaborate all while working to improve child health.

April 10, 2018

After two independent studies were published two years ago suggesting that the maternal mortality ratio (MMR) in Texas had doubled within the past decade, an updated report released today in the journal Obstetrics & Gynecology found that the MMR in Texas was significantly lower in 2012 than previously reported due to data collection error. The identification of this error illustrates an important role of the maternal mortality review process – ensuring accurate data on which to base interventions.

Comprised of 15 Texas-based multidisciplinary experts and chaired by Dr. Lisa Hollier, chief medical officer at Texas Children’s Health Plan and professor of obstetrics and gynecology at Baylor College of Medicine, the Maternal Mortality and Morbidity Review Task Force used an enhanced methodology that combined data matching and records review for identifying maternal deaths. After meticulously reviewing 2012 data, investigators determined that the MMR in Texas was 14 to 18 deaths per 100,000 versus the 37 deaths per 100,000 previously reported.

“Our analysis found there were data quality issues,” said Hollier, who also is president-elect of the American College of Obstetricians and Gynecologists. “When we examined the data, it showed that some deaths were mistakenly coded as “dying while pregnant,” when there was never a pregnancy. Beginning in 2010, Texas transitioned from paper to electronic death certificates, so unintentional user error in reporting pregnancy status may be responsible for the inaccurate data.”

Texas’ current electronic death registration system displays pregnancy status options as a dropdown list. The “pregnant at time of death” option is directly below the “not pregnant within past year” option. The investigators concluded that this could have led to erroneous selection, and could explain why pregnancy at time of death was reported for nearly 76 percent of the 74 obstetric-coded deaths with no evidence of pregnancy upon review.

The task force determined that a total of 56 Texas resident maternal deaths occurred during pregnancy or within 42 days postpartum in 2012. The most common causes for these deaths were drug overdose and cardiac events.

While the MMR is lower than initially reported, the updated report confirms a vast disparity still exists – black women had a higher MMR (27.8 per 100,000 live births) than did women of other racial and ethnic groups. Black women in Texas have more than double the risk of dying during pregnancy or within 42 days postpartum.

As one of the nation’s premier facilities for women’s, fetal and newborn health, Texas Children’s Pavilion for Women remains actively involved in grassroots advocacy at state and national levels with the goal of improving health outcomes, quality of care and patient safety for women and newborns across Texas and the nation.

Through the efforts of the Maternal Mortality and Morbidity Review Task Force, Texas Children’s maternal-fetal medicine physician leadership is helping to review cases and study trends in maternal deaths so that we can better understand the problem and make recommendations to help reduce the MMR across Texas.

While these results published in the new report likely have national implications – since miscoding of obstetric deaths may occur in other states and affect the accuracy of their MMRs – the updated data demonstrates that Texas is more in the middle of the pack when it comes to MMRs across the nation.

“We believe future efforts should focus on improving the quality of death certificate data, especially pregnancy status,” Hollier said. “It is also critically important we don’t lose the unfortunate truth that even this updated data demonstrates a huge disparity between the MMRs faced by black women compared to women of other racial and ethnic groups. We need to continue work to identify why this disparity exists and create strategies to remedy it.”

Click here to read the report in the medical journal Obstetrics & Gynecology.

March 27, 2018

Dr. Susan Blaney, deputy director of Texas Children’s Cancer and Hematology Centers, recently received a Director’s Service Award from the National Cancer Institute for her outstanding and dedicated service to the Institute and the Clinical Trials and Translational Research Advisory Committee from 2015 to 2017.

Blaney is the vice chair of the Children’s Oncology Group, the National Cancer Institute (NCI) supported clinical trials group and the world’s largest organization devoted exclusively to pediatric cancer research.

Blaney served as the co-director of the Developmental Therapeutics Program at Texas Children’s Cancer Center where she performs pre-clinical and clinical studies of new antineoplastic agents. A primary focus of her research is the development of new drugs for the treatment of central nervous system tumors in children with a particular emphasis on the development of new agents for intrathecal administration.

For more information about Dr. Susan Blaney, visit https://www.texaschildrens.org/find-a-doctor/susan-m-blaney-md.

March 20, 2018

Dr. Huda Zoghbi, director of the Jan and Dan Duncan Neurological Research Institute at Texas Children’s, Howard Hughes Medical Institute investigator and professor at Baylor College of Medicine, has been awarded the sixth annual Ross Prize in Molecular Medicine by The Feinstein Institute for Medical Research at Northwell Health.

The Ross Prize is awarded annually by Molecular Medicine to scientists who have made a demonstrable impact in the understanding of human diseases pathogenesis and/or treatment, and who hold significant promise for making even greater contributions to the general field of molecular medicine.

“It is an honor to be recognized by Molecular Medicine and to join the prestigious roster of past Ross Prize recipients,” said Zoghbi, professor in the Departments of Pediatrics, Molecular and Human Genetics, Neurology and Neuroscience at Baylor. “I look forward to discussing my work and furthering a dialog that I hope will encourage young trainees to join the fields of neurobiology and molecular medicine.”

The prize, which includes a $50,000 award, will be presented to Zoghbi on June 5 at the New York Academy of Sciences in New York City, followed by lectures from Zoghbi and other eminent researchers. The Ross Prize is awarded through the Feinstein Institute’s peer-reviewed, open-access journal, Molecular Medicine, and made possible by the generosity of Feinstein Institute board members Robin and Jack Ross.

“Huda Zoghbi’s examination of the genetic causes for neurological diseases, such as spinocerebellar ataxia and Rett syndrome, has led to a better understanding of neurobiology,” said Feinstein Institute President and Chief Executive Officer Dr. Kevin J. Tracey, who also serves as editor emeritus of Molecular Medicine. “It is through her discoveries that researchers are able to identify new, potential therapies for these conditions that currently have no cure.”

Zoghbi’s research focuses on identifying the genetic causes of neurodegenerative and neurodevelopmental diseases and a broader understanding of neurobiology. Her lab, along with Harry Orr’s team at the University of Minnesota, discovered that excessive repeats of the DNA segment, CAG, in the ATAXIN-1 gene causes the neurodegenerative disease spinocerebellar ataxia type 1 (SCA1). Over time, SCA1 may cause mental impairment, numbness, tingling or pain in the arms and legs and uncontrolled muscle tensing, wasting and twitches. Understanding the genetic cause for SCA1 has inspired additional research that may identify a therapeutic strategy for this condition that affects coordination and balance, difficulties with speech and swallowing, and weakeness in the muscles that control eye movement.

Zoghbi’s lab also has identified the genetic mutations that cause Rett syndrome. Rett syndrome mostly targets young girls and is a postnatal neurological disorder which causes problems in diverse brain functions ranging from cognitive, sensory, emotional, and motor to autonomic functions. These can affect learning, speech, sensations, mood, movement, breathing, cardiac function and even chewing, swallowing and digestion. Zoghbi’s discoveries provide a framework for understanding this disorder as well as the MECP2 duplication disorder and for charting a path for potential therapeutic interventions.

February 20, 2018

The Clinical Research Center/Research Resources Office presented the Clinical Research Award for First Quarter 2018 to Dr. Daniel Leung, associate professor of Pediatrics in the Division of Gastroenterology, Hepatology, and Nutrition where he serves as Director of Clinical Research.

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.

Leung’s research activities in the CRC focus on caring for children with a variety of liver disorders. He is an active investigator in the NIH-supported CFLD and ChiLDReN Networks which study cholestatic and cystic fibrosis-related liver disease and led the recently completed Cystic Fibrosis Foundation sponsored multi-center Baby Observational Nutritional Study (BONUS). Leung also oversees the Viral Hepatitis Program at Texas Children’s Hospital which offers cutting edge antiviral therapies through several clinical trials to children throughout the state who have Hepatitis B and C. Virtually all these patients are seen for study treatment and long-term follow up with the support of CRC staff. With the new pipeline of oral direct acting antivirals (DAAs) against hepatitis C, children with chronic HCV can now be cured in as short as 12 weeks with minimal to no side effects. Leung credits the outstanding nursing staff in the CRC and research coordinators through the RRO as “true co-laborers in helping cure and eradicate viral hepatitis in children and breaking the vicious cycle of these viruses.”

January 30, 2018

Dr. Huda Zoghbi was awarded the National Order of the Cedar, Knight grade by Lebanese President General Michel Aoun at a ceremony held in January at the Presidential Palace in Baabda.

Zoghbi is the founder and director of the Jan and Dan Duncan Neurological Research Institute (NRI) at Texas Children’s Hospital and Baylor College of Medicine, a Howard Hughes Medical Institute investigator, and holds the Ralph D. Feigin, MD, Endowed Chair in Pediatrics.

This prestigious honor touches close to home as Zoghbi was born and raised in Beruit, Lebanon’s capital and largest city. She received her Bachelor of Science degree from American University Beirut (AUB).

“It is an honor for AUB and indeed for Lebanon that the President of the Republic of Lebanon has conferred on Huda Zoghbi the Order of the Cedar,” said American University Beirut President Fadlo R. Khuri. “Thus, the greatest and most decorated scientist to have completed her undergraduate education at our university over the last several decades, a woman whose work is transforming the science and medicine of some of the world’s most serious neurological diseases, is now recognized by her native country’s highest award.”

Zoghbi’s hard work and dedication to the medical field spans through Texas Children’s and beyond. The National Order of the Cedar is a public service award and Lebanon’s highest honor.

“I am deeply honored and humbled to receive this great honor,” Zoghbi said. “I owe a lot of gratitude to my family who shaped me and to the institutions that educated me: Makassed and the American University of Beirut. I feel fortunate that I grew up and spent my formative years in Lebanon and I hope the culture that inspired me to seek knowledge will continue to inspire and empower the youth of Lebanon.”

Texas Children’s Hospital recently received a $1.9 million grant from The Robert J. Kleberg Jr. and Helen C. Kleberg Foundation to support Texas Children’s Hospital Center for Vaccine Development (CVD), advancing the efforts to develop a vaccine against Chagas disease.

Chagas is a tropical disease caused by a parasite transmitted to people and animals through insects. The most impacted people are in the poorer areas of Latin America, and the Americas, including Texas. Texas Children’s Hospital and Baylor College of Medicine are working together to find ways to eliminate this disease.

“Because of its link to poverty, new interventions for Chagas disease are not generally of great interest to the industry, so it falls to organizations like our Texas Children’s Center for Vaccine Development to take the lead on developing a vaccine,” said Dr. Peter Hotez, director of the CVD.

Hotez is a distinguished physician with a passion for tropical medicine. He took on this project alongside CVD Deputy Director Dr. Maria Elena Bottazzi to insure that quality research and solid outcomes are developed when it comes to neglected tropical diseases.

“To tackle Chagas disease, the evaluation of novel and innovative technologies is an essential step. The support from the Kleberg Foundation will be transformational and will allow to accelerate the development of important control tools against this devastating disease,” Bottazzi said.

The Robert J. Kleberg Jr. and Helen C. Kleberg Foundation awards grants to institutions that further the vision of the Klebergs, such as improving quality of life through community support, innovation and scientific research.

“We are extremely grateful for our Kleberg Foundation funding, which will allow us to harness the innovative capacity of the Texas Medical Center in order to apply it towards the first vaccine for this devastating condition,” Hotez said.

The first steps have been taken in the vaccine development process, and the CVD hopes to advance the first vaccine candidate to final manufacture and clinical testing within the next few years.