March 31, 2015

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4115stbaldricksbefore640Eighteen years ago, Adam Henderson lost his hair as a result of treatments while battling acute lymphocytic leukemia at Texas Children’s Hospital. As of a few days ago, Henderson is once again bald after participating in the St. Baldrick’s event in The Woodlands with his former pediatric oncologist, Dr. Timothy Porea. Both Porea, clinical director of Texas Children’s Cancer and Hematology Centers, and Henderson shaved their heads to raise funds and awareness for pediatric cancer research along with about 60 other participants.

“It is the first time Adam has been bald since he had been treated for his cancer 18 years ago,” said Porea who was a fellow at Texas Children’s Hospital when Henderson was undergoing treatment. “He’s just a great role model for our current patients and their families to show how far you can go when you’re through with these difficult treatments.”

“There were a lot of emotions about being bald again,” Henderson said. “This time, I’m doing it by choice and it’s a joyous occasion because I was able to promote a great cause. When I was sick, I was beat down, it was very difficult when I lost all of my hair.”

Porea has participated in the fundraising and head shaving for the last ten years. He had previously taken part in the events in Virginia. This year, after a move back to Houston, he was excited to call his former patient to join him. Porea and Henderson have kept in touch over the years and are even counselors together at Camp Periwinkle.

4115stbaldricksduring640“It’s more than just a profession to Tim,” Henderson said of his former physician. “It’s true to his character and speaks to where his heart is. He’s an inspiration.”

St. Baldrick’s annual challenge to “Brave the Shave” brings together survivors, patient families, physicians and supporters from across the community to raise funds for childhood cancer research. This year, the nationwide shaving events have raised more than $22 million so far. The foundation was started as a response to the lack in funding for childhood cancer research. According to the organization’s website, while 175,000 children are diagnosed with cancer each year, all types of childhood cancers combined receive only 4 percent of the U.S. federal funding for research. The funds raised through St. Baldrick’s have helped fund 820 grants, at 329 institutions, in 22 countries. Several have been given to researchers at Texas Children’s Cancer and Hematology Centers. For Porea, who sees these patients every day, this research is vital to the discovery of better treatments and the possibility for a cure.

“Seeing Adam next to me for this event means we have succeeded, he’s here!” Porea said. “He’s able to do all of these things despite everything he went through as a child. It helps reinforce to me why we all do what we do here every day.”

For Henderson, his new bald look is a conversation starter. Most importantly, it brings attention to a disease he’s all too familiar with.

“At first, the conversation about being bald starts off as kind of a joke with people commenting on my ‘nice haircut’,” Henderson said. “But when I share my story and specifically talk about St. Baldrick’s, it’s impactful. People take it very seriously and there have been a lot of people that have walked away from these conversations with more knowledge and awareness.”

Asked how long he plans to continue shaving his head for childhood cancer research, Porea didn’t hesitate to answer.

“As long as I have hair.”

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April 2 is World Autism Awareness Day when the international community will “Light It Up Blue” to bring recognition to autism as a rising global health concern. Texas Children’s encourages everyone to wear blue that day or post something blue to one of their personal social media pages to show their support for autism awareness.

Autism spectrum disorder is the fastest-growing developmental disability in the United States, affecting 1 out of every 68 children. It occurs more often among boys than girls. Children with autism often experience significant social, communication and behavioral challenges.

For more than a decade, Natanya Dugall searched for a diagnosis for her son that would explain his vision, speech and movement impairments. She knew something was wrong with Grayson from the moment he was born, but initial genetic testing turned up nothing.

When Grayson turned 13, Dugall received some unexpected news. A laboratory had identified chromosomal deletions on a specific gene in Grayson’s DNA which had been collected years earlier. After the initial find, Grayson participated in a study at the Autism Center at Texas Children’s that examined the effects of people who grew up with either chromosomal deletions or copies on this gene.

When the Dugall family made the 1,120-mile trek from their home in Milwaukee, Wisconsin to Houston in 2011, their quest for answers to Grayson’s puzzling condition was confirmed – he had autism.

“I cannot begin to express my gratitude to the research team from Baylor College of Medicine and Texas Children’s Hospital for funding all of the testing and travel expenses,” Dugall said. “Grayson understands the probability that he can pass this to his children – knowledge that we struggle with but are so grateful to finally know.”

The Autism Center’s multidisciplinary team of specialists from developmental pediatrics, psychology, psychiatry, neurology and social work provide diagnostic evaluations and ongoing clinical care to 2,000 patients with autism each year. Researchers at the Autism Center participate in a wide range of research studies focused on autism spectrum disorders, collaborating with scientists from other renowned centers across the nation.

In conjunction with the groundbreaking work emerging from the The Autism Center, Dr. Ruth Ann Luna at Texas Children’s Microbiome Center focuses much of her research on the link between disturbances in the gut microbiome and gastrointestinal (GI) symptoms in children with autism. One of her biggest motivators is her 6-year-old son, Kellen, who has autism.

With the support of a $1.4 million, three-year-grant from Autism Speaks, Luna and her Baylor and Texas Children’s colleagues are embarking on a study to determine if a biological connection exists between autism and GI disorders.

“I was in awe when I found out we were awarded this funding to advance autism research,” Luna said. “The data gleaned from our research will benefit my son and other families who have children with autism, especially those children with limited verbal abilities.” Click here to read more about Luna’s autism study.

For more information about “Light It Up Blue, click here and to learn more aobut the Autism Center at Texas Children’s click here.

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Dr. Huda Zoghbi received the 2015 Mortimer D. Sackler, M.D. Prize for Distinguished Achievement in Developmental Psychobiology, awarded by Weill Cornell Medical College and Columbia University College of Physicians and Surgeons.

Zoghbi is the director of the Jan and Dan Duncan Neurological Research Institute at Texas Children’s, a professor of molecular and human genetics, pediatrics, neuroscience and neurology at Baylor College of Medicine, and a Howard Hughes Medical Institute investigator.

As a member of the National Academy of Sciences, Zoghbi has pioneered research on Rett syndrome and other rare brain disorders, and has advanced research of more common conditions including autism and adult neurodegenerative diseases.

In one of her most well-known achievements, Zoghbi provided a definitive genetic diagnosis for Rett syndrome and opened the door to a biological understanding and a search for treatment. Rett syndrome is a genetic neurological disease that usually affects young girls. Girls born with the disease develop normally for one or two years, but then begin to show progressive loss of motor skills, speech and other cognitive abilities.

Zoghbi has also found other genes, including Math1, an essential gene governing the development of cells critical for hearing, balance, and breathing, and the genes for spinocerebellar ataxia 1 and 6. Her work has led to a better understanding of neurodevelopmental and neurodegenerative disorders.

“We’re thrilled that Dr. Zoghbi is this year’s recipient,” said Dr. B.J. Casey, director of the Sackler Institute and the Sackler Professor of Developmental Psychobiology at Weill Cornell Medical College. “She’s dedicated herself to this field for decades and has made remarkable achievements. But we’re also recognizing her leadership in the field of neuroscience and her track record of mentoring young scientists as they embark on their own careers.”

The prize is named for the Dr. Mortimer D. Sackler, one of the most creative scientists in the field of developmental psychobiology. The Mortimer D. Sackler Foundation, Inc. endowed the prize in a gift in 2009.

“To have a prize bearing the name of someone who’s made such remarkable achievements in this field is a great honor,” Zoghbi said. “There are so many deserving scientists and physician-scientists out there who have done fantastic work. I feel truly honored to have been selected.”

Zoghbi plans to donate the $100,000 prize money to a mentorship fund that she established to help young female scientists get their work – specifically research on “bold, new ideas” – off the ground.

March 24, 2015

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Have you ever found your fruit bowl teeming with fruit flies that seemed to appear out of nowhere? While pesky at times, these 6-legged, red-eyed insects have endured as a laboratory staple for more than 100 years, helping scientists unlock the genetic causes of human disease.

In a recently published article in the journal Genetics, fruit fly biologists at the Jan and Duncan Neurological Research Institute at Texas Children’s – Drs. Michael Wangler, Shinya Yamamoto and Hugo Bellen – explore the value of fruit fly research and the need for more funding to accelerate biomedical discoveries using the Drosophila fruit fly.

Unlike mouse models, the authors argue that fruit flies are inexpensive to maintain, they grow quickly from embryo to adult in 12 days, and share many similar genes with humans, making the Drosophila a powerful model organism for the study of genetics.

To study human disease in fruit flies, scientists mutate, or disrupt, the same gene that is known or suspected to cause the disease, and then figure out why mutations with this particular gene leads to disease. However, one can also take an unbiased approach and discover new genes.

Inside their laboratory at the NRI, Bellen and his team have performed a large scale forward unbiased genetic screen which allowed them to isolate 165 fruit fly genes that cause neuro-developmental and neuro-degenerative phenotypes when mutated, 90 percent of which have human counterparts. The human counterparts of one third of the genes cause Mendelian disorders. By exploring the remaining two thirds of the genes in patients with rare disease they could identify a few novel human diseases genes, work that they did in collaboration with Drs. Jim Lupski and Richard Gibbs.

A detailed characterization of some of the 165 genes also permitted them to discover novel mechanisms that underlie a variety of known diseases such as the peripheral neuropathy associated with Charcot-Marie-Tooth (2A) disease, Leigh syndrome and some ataxias, including Spinocerebellar Ataxia 6 and Friedreich ataxia.

“By understanding how a particular gene works in the fruit fly, we can dissect the molecular and genetic events that underlie the pathogenesis of human disease so that effective treatments can be developed in the future,” Bellen said.

“We see fruit fly research as an avenue of tremendous importance,” Wangler said. “Federal agencies need to actively encourage collaborative research between fruit fly researchers and human geneticists and clinicians to make the best possible use of dwindling research dollars to promote public health.”

Click here to read their article titled, “Fruit flies in biomedical research,” in the journal Genetics.

March 10, 2015

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Bench and Bedside is a digest of the previous month’s stories about the clinical and academic activities of our physicians and scientists. We welcome your submissions and feedback.

Top news story

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Hope, faith and expertise: Surgical team leads historical Mata conjoined twins surgery

Knatalye Hope and Adeline Faith Mata, conjoined twin girls born at Texas Children’s Pavilion for Women in April 2014, underwent a successful separation surgery on February 17 at Texas Children’s Hospital. Led by surgeon Dr. Darrell Cass, a team of more than 26 clinicians worked together for more than a day to separate the girls who shared a chest wall, lungs, pericardial sac, diaphragm, liver, intestines, colon and pelvis. Watch the video or read the story for a behind-the-scenes look at this monumental undertaking.

February 25

Surgical Seed Grant Award recipients named

Six researchers representing congenital heart surgery, general surgery, ophthalmology and plastic surgery were recently named the recipients of the 2015 Surgical Seed Grant Awards given by the Department of Surgery. This grant program allows surgery researchers to generate the preliminary data necessary for National Institutes of Health (NIH) grant applications and other extramural funding. In total, more than $275,000 in grants will be awarded to the following recipients:

  • Ophthalmologist Dr. Lingun Kong Intravitreal Polymer Nanoparticle Drug Delivery System for Treatment of Retinopathy of Prematurity
  • Congenital Heart Surgeon Dr. Carlos Mery Development of a Novel Open Fetal Cardiac Surgical Intervention for Creation of an Atrial Septal Defect in Patients with Hypoplastic left Heart Syndrome & an Intact or Highly Restrictive Atrial Septum
  • Plastic Surgeon Dr. Edward Buchanan Psychometric Assessment of the Medical Interview in the Pediatric Population
  • Congenital Heart Surgeon Dr. Iki Adachi Pediatric Myocardial Alterations in Response to Ventricular Assist Devices
  • Ophthalmologist Dr. Mohamed Hussein Exploring the Role of the Choroid and the Autonomic Innervation in the Development of Retinopathy of Prematurity
  • Pediatric General Surgeon Dr. Sanjeev Vasudevan A “Pan-Omic” Analysis of Vascular Invasion in Pediatric Solid Tumors

Read more about the Surgical Seed Grant Awards.

February 24

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Luna co-leads GI study to offer hope for son, children with autism spectrum disorders

Have you ever wondered why life leads us down certain career paths? For Dr. Ruth Ann Luna, the answer is obvious – her immense curiosity and passion for research to improve the quality of life for sick children. As director of Medical Metagenomics at Texas Children’s Microbiome Center, Luna focuses much of her research on the link between disturbances in the gut microbiome – bacterial communities in the gastrointestinal (GI) tract – and GI symptoms in children. Read more

February 17

Texas Children’s expands its subspecialty, therapy services to Kingwood

Quality is key, but convenience also plays a big part in the decision-making process when parents are looking for subspecialty and therapy services. That’s why Texas Children’s works to make such services convenient to families throughout the Greater Houston area. The organization’s most recent efforts focus on Kingwood, where Texas Children’s Specialty Care at Kingwood Glen recently, and Texas Children’s Hospital Outpatient Therapy at Kingwood Glen and Texas Children’s Hospital Sports Medicine Therapy at Kingwood Glen opened in October. Read more

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Multidisciplinary care enhances outcomes for women with placenta accreta

Due to our success in treating the most severe cases of morbidly adherent placenta, Texas Children’s Pavilion for Women continues to attract numerous patients from across the country. With the rise of caesarean births in the U.S., this potentially life-threatening condition is becoming more common among pregnant women. Morbidly adherent placenta – known as placenta accreta, increta or percreta, depending on the depth of uterine invasion – occurs when the placenta and its blood vessels grow deeply into the wall of the uterus and is unable to detach after childbirth. “The number of women we care for with morbidly adherent placenta is rapidly escalating as our outcomes continue to attract a growing number of referrals,” said Texas Children’s OB/GYN-in-Chief Dr. Michael Belfort, a world-renowned placenta accreta expert and founder of the Morbidly Adherent Placenta Program at Baylor College of Medicine. Read more

Texas Children’s Pediatrics opens sixth Community Cares practice

In February, Texas Children’s Pediatrics announced Texas Children’s Pediatrics Kingsland as its newest practice. Located at 21715 Kingsland Blvd., Suite 103, in Katy, Texas Children’s Pediatrics Kingsland is among six practices in the Texas Children’s Pediatrics Community Cares Program. The Community Cares Program provides trusted, high-quality pediatric medical services for children who otherwise would seek care from emergency rooms or possibly go without care or treatment due to low family incomes and/or lack of health insurance. Dr. Punita Sunder serves as the primary physician at Texas Children’s Pediatrics Kingsland. Board certified by the American Board of Pediatrics, Sunder received her undergraduate degree from Rice University and earned her medical degree from Baylor College of Medicine. She earned her doctorate in philosophy from The University of Texas Medical Branch and completed her residency at Baylor. Read more

February 10

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3-D Model of Mata twins helps surgeons prepare for girls’ separation

In the months before the separation surgery of conjoined twin girls Knatalye Hope and Adeline Faith Mata, comprehensive, multidisciplinary planning was underway. With help from a Dallas printing company, Chief of Radiology Research and Cardiac Imaging Dr. Rajesh Krishnamurthy built a 3-D model of the twins’ anatomy to help surgeons plan for the girls’ separation. The model was built using a computerized image data set of the twins and a 3-D printer. The detailed model includes a detachable, transparent liver and was especially helpful in the planning of the pelvic portion of the surgery, which presented an interesting and challenging anatomical situation because each girl had her own pelvic organs but each of those organs received blood supply from the other girl. Watch the video or read the story about the 3-D model. Read more

Friends, colleagues attend Singleton tribute ceremony

Family, friends and former colleagues of the late Dr. Edward B. Singleton, Chief Emeritus of the Edward B. Singleton Department of Pediatric Radiology, gathered to celebrate the life of the late physician and relive their favorite memories of the jovial Singleton. Browse the photo gallery to see some of the moments from this tribute ceremony. View the photo gallery.

February 9

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Milewicz named chief surgical officer at Texas Children’s Hospital West Campus

In February, Texas Children’s Hospital West Campus announced Dr. Allen Milewicz as chief surgical officer. In his new role, Milewicz will be responsible for organizing Texas Children’s surgery within the community, focusing on Texas Children’s Hospital West Campus. Additionally, he will be working in conjunction with the entire Texas Children’s Department of Surgery to optimize surgical service for patients, families and referring physicians. “I look forward to expanding surgical services in the community while ensuring that each patient receives the experience, expertise and clinical outcomes for which Texas Children’s is known,” Milewicz said. Read more

February 6

Cutting Edge of Pediatrics conference draws more than 100 participants

The first “Cutting Edge of Pediatrics” conference sponsored by the Department of Surgery was held in January, providing 138 Houston-area pediatricians, family practice physicians and advanced practice providers an opportunity to learn about common pediatric surgical conditions from Texas Children’s surgeons. The surgeons and pediatric providers engaged in lively discussions of the topics, which included anesthesia, ethics, adolescent gynecology, ophthalmology, orthopedics, otolaryngology, pediatric general surgery, plastic surgery and urology. Following a welcome from Surgeon-in-Chief Dr. Charles D. Fraser, Jr., presenters included Dr. Dean Andropoulos, Dr. Jennifer Bercaw-Pratt, Dr. David Coats, Dr. Nicolette Janzen, Dr. Edward Lee, Dr. Mark Mazziotti, Dr. Julina Ongkasuwan, Dr. Scott Rosenfeld and Dr. Veeral Shah. Read more

February 3

Texas Children’s researchers push for universal newborn biliary atresia screening

Texas Children’s pediatric gastroenterologist Dr. Sanjiv Harpavat is working to ensure every newborn is screened for biliary atresia, a rare but deadly liver disease affecting 1 in 10,000 births in the U.S. It’s the number one reason for liver transplants in infants. Biliary atresia occurs when the bile duct connecting the liver to the small intestine is blocked. The buildup of bile in the liver scars the tissue so rapidly that most infants need a liver transplant to survive. “Infants with biliary atresia have more scarring after the first few months of life than a chronic alcoholic’s liver,” said Harpavat. “An important way to improve their outcomes is by detecting and treating the disease early before symptoms appear.” Read more

February 2

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CRC presents research award to Dr. Carla Davis

The Clinical Research Center presented the Clinical Research Award for Fourth Quarter 2014 to Dr. Carla Davis, Pediatrics-Allergy & Immunology, Baylor College of Medicine. The 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.

Submissions for Bench and Bedside

Bench and Bedside is produced monthly by Texas Children’s Corporate Communications team to spotlight recent news about Texas Children’s physicians and scientists. The team welcomes submissions related to speaking engagements, staff awards/recognition, research, clinical work and academic activities. Send your questions or submissions to connectnews@texaschildrens.org.

February 24, 2015

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Have you ever wondered why life leads us down certain career paths? For Dr. Ruth Ann Luna, the answer is obvious – her immense curiosity and passion for research to improve the quality of life for sick children.

As director of medical metagenomics at Texas Children’s Microbiome Center, Luna focuses much of her research on the link between disturbances in the gut microbiome – bacterial communities in the gastrointestinal (GI) tract – and GI symptoms in children.

Her quest for answers goes far beyond her role as an astute scientist. One of her biggest motivators is her 6-year-old son, Kellen, who has autism accompanied by significant GI problems – a common complaint among children with autism spectrum disorders.

22515autisminside495“When Autism Speaks announced its GI and Neurobehavioral Processes grant almost a year ago, I recognized it as the opportunity of a lifetime,” said Luna, an assistant professor of pathology at Baylor College of Medicine. “I was in awe when I found out we were awarded this funding to advance autism research.”

With the support of a $1.4 million, three-year grant, Luna and her colleagues at Texas Children’s and Baylor will embark on a comprehensive, multi-center study to determine if a biological connection exists between autism and GI disorders.

“Previous research has shown that gastrointestinal problems are more common among individuals with autism and may worsen behavioral problems,” said Texas Children’s Pathologist-in-Chief Dr. James Versalovic, the Milton J. Finegold professor of pathology at Baylor and director of Texas Children’s Microbiome Center.

Co-led by Luna and Versalovic, scientists in the Microbiome Center will evaluate behavior, GI symptoms, the microbiome and the metabolome, all in the hopes of identifying biomarkers of abdominal pain, understanding the impact of the gut-brain-microbiome axis and determining metabolic disturbances in autism.

“There are inherent differences in the guts of children with autism,” Luna said. “By analyzing all of these factors, we hope to develop better ways to diagnose and treat gastrointestinal issues in children with autism.”

The autism study will enroll 375 children ages 4 through 12 at Texas Children’s, the University of Texas Southwestern Medical Center in Dallas and Nationwide Children’s Hospital in Columbus, Ohio.

Children with autism – with and without GI symptoms – and their unaffected siblings, and children without autism, with and without GI symptoms, are currently being recruited for the study.

“The data gleaned from our research will benefit my son and other families who have children with autism, especially those children with limited verbal abilities” Luna said. “This amazing opportunity to positively impact lives is what makes my job extremely fulfilling.”

For more information or to enroll in the Microbiome Center’s autism and GI study, contact Luna at Ext. 4-1894 or raluna@texaschildrens.org.

February 3, 2015

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The Clinical Research Center will present the Clinical Research Award for Fourth Quarter 2014 to Dr. Carla Davis, Pediatrics-Allergy & Immunology, Baylor College of Medicine.

The 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.

Dr. Davis’s research activities in the CRC focus on allergy and immunology related issues.

Congratulations to Dr. Davis.