PEDIATRIC NEUROSCIENCE - CHILDREN'S MEMORIAL HERMANN HOSPITAL - UTHealth
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CHILDREN'S MEMORIAL HERMANN HOSPITAL
PE D I AT R I C N E U RO S C I E N C E
SUMMER 2020
JOURNAL A publication of Children's Memorial Hermann Hospital and McGovern Medical School at UTHealth
Fetal Myelomeningocele Repair
Exposed spinal
cord and nerve
Exposed spinal
Edge of myometrium cord and nerve
Uterus
and chorioamniotic
membranes
Closure of
dura mater
Myofascial flap
Prenatal repair of myelomeningocele is a delicate surgical procedure where fetal surgeons open the
uterus and close the opening in the baby's back while still in the womb.
In This Issue Celebrating Firsts
F
FETAL MYELOMENINGOCELE REPAIR: PAST, RESEARCH or every hour we spend with patients, we spend many more in detailed preparation for that
PRESENT, AND FUTURE first meeting. During the exam, we ask questions, listen closely, and consider every diagnostic
12 Novel Studies Seek to Improve Outcomes in
possibility. Afterward, we review our treatment options as a team. When we have executed
02 Faith in a Tiny Patient: The First Fetal Children with Malignant Fourth Ventricular Brain
successful surgical solutions, we’re not afraid to marry them in new procedures that advance pediatric
Myelomeningocele Repair in Texas Tumors
neuroscience. In this issue of the Pediatric Neuroscience Journal, you’ll read about home runs with
04 Independence From Spina Bifida: Toward 14 Nanotechnology for Drug Delivery: The Science
three patients.
a Safe Method of Minimally Invasive of Moving Chemotherapeutic Drugs Directly to
Myelomeningocele Repair Brain Tumors Faith Hagler was the first baby in Texas to undergo in-utero spina bifida repair after the results of
the Management of Myelomeningocele Study (MOMS) were published in the New England Journal
06 Open In-Utero Repair of Severe Spina Bifida
NEWS OF NOTE
of Medicine in 2011. The Haglers, residents of Dallas, learned about the MOMS trial during their
Using a Human Umbilical Cord Patch internet search for the best treatment for their daughter, and their maternal-fetal medicine specialist
07 Fetoscopic Spina Bifida Repair for Small Defects 16 Dr. David Sandberg Receives the American referred them to The Fetal Center at Children’s Memorial Hermann Hospital in Houston. Faith,
Using a Human Umbilical Cord Patch Association of Neurological Surgeons now 9, is a shining star who performs song-and-dance routines at her church and is on the honor roll
Humanitarian Award at her elementary school.
We’re also grateful to the Guidry family of Louisiana and the Medlins of Texas for sharing the stories
CRANIOSYNOSTOSIS 17 Dr. Rachael Sirianni Named UTHealth Women
Faculty Forum Rising Star of their daughters, who were born with unicoronal craniosynostosis. Both babies underwent an
08 Innovation in Surgery for Unicoronal innovative surgery that produced excellent outcomes after their families trusted our team to provide
Craniosynostosis: Presurgical Preparation on a 18 Experts Discuss Innovative Treatment Options
them with the very best care. Using CT scans, Dr. Manish N. Shah, a pediatric neurosurgeon, and
3D Model Ensures Success in Two Cases for Cerebral Palsy
Dr. Phuong Nguyen, a plastic/craniofacial surgeon, designed a 3D model and fine-tuned the surgery
09 What Is Craniosynostosis and When Is Surgery
19 SELECTED PUBLICATIONS
the day before they took each patient to the OR.
Necessary? Three novel studies underway at UTHealth seek to improve the outlook for children with malignant
fourth ventricular brain tumors. In our Research section, we also report on the work of Dr. Rachael
Sirianni and her lab team to encapsulate drugs within biocompatible and biodegradable nanoparticles
that serve as carriers to prolong drug action and target specific tissue sites. Her work complements the
research we’re doing in the two single-center trials for fourth ventricular brain tumors.
We hope you find the articles in this issue of the Pediatric Neuroscience Journal thought-provoking
and useful in your practice. If you have questions about any of our programs, please contact us directly.
With best wishes,
David I. Sandberg, MD, FAANS, FACS, FAAP Ian J. Butler, MD
Professor and Director of Pediatric Neurosurgery Professor and Director of Child and Adolescent Neurology
Dr. Marnie Rose Professor in Pediatric Neurosurgery Jacobo Geissler Distinguished Chair in West Syndrome Research
Department of Pediatric Surgery and Vivian L. Smith Department of Neurosurgery McGovern Medical School at UTHealth
McGovern Medical School at UTHealth, Children’s Memorial Hermann Hospital, 713-500-7142
and Mischer Neuroscience Institute at Memorial Hermann-Texas Medical Center
Co-Director, Pediatric Brain Tumor Program
The University of Texas MD Anderson Cancer Center
713-500-7370
PEDIATRIC NEUROSCIENCE JOURNAL 01
F E TAL M YE L OM E NI NGOCE L E R E PA I R : PA S T, P R E S E N T, A N D FU T U R E
Faith in a Tiny Patient: The First Fetal With support from the National Institutes
of Health, MOMS studied the effects of fetal
Stephen Fletcher, DO,
works with the fetal
Myelomeningocele Repair in Texas surgery for the repair of spina bifida to the stan-
dard-of-care surgery for the disorder after birth.
medicine team to perform
an in-utero spina bifida
The study, which followed 158 women at three repair at Children’s
hospitals, found that if a baby undergoes surgery Memorial Hermann
in utero, the serious complications associated Hospital.
with spina bifida could be reversed or lessened.
Picture the most creative, self-assured 9-year-old you can imagine. Fetal surgery decreased the need for shunting
at 12 months for the infants who participated
Her songs and dance moves come straight from Ariana Grande. She in the study, and nearly half were able to walk
loves bright colors and runs to the mirror to check every outfit. She without crutches by age 2½.
performs at church, is on the honor roll at her elementary school, “MOMS was the first study to show the Thirteen percent had a preterm birth at less than
significant benefits of surgery to unborn babies 30 weeks, one-third delivered between 30 and
and reads at a higher level than her third-grade class. She has the with spina bifida,” Tsao says. “The study was also 34 weeks, and one-third delivered between 35
attitude of an actress, the energy of an entertainer, and the million- important because traditionally, fetal surgery was and 36 weeks, with the remainder delivering at
performed for lethal conditions. With the MOMS
dollar smile of a model. Her goal is to be a superstar dancer and study, researchers conducted the first fetal opera-
37 weeks or later.
Medically, Hagler was a good candidate. “The
actress, or maybe a teacher. tion for a nonlethal condition. Because of the risks couple knew that this was our first in-utero spina
involved in fetal surgery, we tend to limit heroic bifida repair,” Tsao says. “We had performed the
L
ike all babies, Faith Hagler’s story begins of spina bifida, my husband and I started doing interventions to the most severe and life-threaten- various parts of the operation many times with
before she was born. But unlike others, research online.” ing cases – those associated with high mortality other patients, but this was the first time we pulled
her history was complicated by a prenatal A maternal-fetal medicine specialist in Dallas and high morbidity. While myelomeningocele is everything together in one procedure. This was
diagnosis of myelomeningocele – spina bifida – at confirmed the diagnosis and referred her to The associated with high morbidity, the mortality rate something the Haglers really wanted to do. They
Ramesha Papanna, MD, 20 weeks of gestation. Born on the Fourth of July, Fetal Center1 at Children’s Memorial Hermann is relatively low.” had done their research and had a good under-
MPH 2011, Faith was the first child to undergo in-utero Hospital in Houston. A week later, Hagler and Since the 1930s, the first step in the treatment standing of the risks to mom and baby.”
Associate Professor, surgery for spina bifida repair in Texas. her husband, Ivan, were consulting with KuoJen of newborns with the condition has been to The surgery itself was straightforward. A special
Department of Obstetrics,
Gynecology, and Reproductive Spina bifida is a complex and permanently dis- Tsao, MD, co-director of The Fetal Center and close the opening within a few days of birth. stapling device was used to cut through the uterine
Sciences
abling birth defect involving incomplete develop- professor of pediatric surgery at McGovern Med- The surgery moves tissues back into their normal wall and close blood vessels to prevent bleeding.
Director, Fetal Intervention
Fellowship ment of a portion of the spinal cord and associated ical School at UTHealth; Stephen Fletcher, DO, position and prevents further damage to and Once the fetus was exposed, Fletcher closed the
McGovern Medical School at
UTHealth
nerves, as well as the surrounding spinal bones associate professor of pediatric neurosurgery at infection of the nerve tissue, but does not restore gap in her spine, and the team returned the one-
and overlaying muscle. At birth, the incompletely McGovern Medical School; and a maternal-fetal function to the already damaged nerves. The pound baby to the uterus.
developed portion of the spinal cord protrudes medicine specialist on staff at the time. During second step is usually placement of a shunt in the The Haglers say they relied in large part on their
through the open skin. their two-day visit, they also met with other ventricles of the brain, which allows for drainage faith in making the decision to go forward with
Infants born with spina bifida are at risk for a members of Children’s Memorial Hermann of the excess fluid characteristic of spina bifida, the surgery. “I had a lot of anxiety, especially about
range of associated conditions, including hydro- Hospital’s multidisciplinary team to determine relieving pressure on the brain. the anesthesia, but once I learned about the possi- Stephen Fletcher, DO
cephalus, which may require placement of a ven- a treatment plan. The team performed the first “The MOMS study was exciting because it pro- bility of in-utero repair, I wanted to do it,” Colette Associate Professor,
Division of Pediatric Neurosurgery
triculoperitoneal (VP) shunt to relieve pressure in-utero spina bifida repair in Texas, and one of duced greatly improved outcomes,” says Fletcher, Hagler says. “By the time I got to Houston, I had Vivian L. Smith Department of
inside the skull caused by excess fluid on the brain. the first in the nation after the results of the Man- a key member of the pediatric neurosurgery team already made up my mind. We wanted to give our Neurosurgery
McGovern Medical School at
KuoJen Tsao, MD
They are prone to life-threatening infections and agement of Myelomeningocele Study (MOMS) at Children’s Memorial Hermann Hospital and baby the best chance possible.” UTHealth
The Children's Fund, Inc. may suffer loss of sensation or paralysis below the were published. UTHealth Neurosciences. “But in fetal surgery, Hagler gave birth almost nine weeks later on
Distinguished Professor in
Pediatric Surgery, Department
level of the spinal cord defect, as well as ambula- Tsao had begun screening patients for in- there are risks to the mother and the baby, and we July 4, two weeks prior to her 34-week planned
of Pediatric Surgery tory problems, deformities of the hips, and lower utero repair in early 2011, based on outcomes weigh those risks carefully.” delivery date. “We named her Faith because at
Professor and Chief, Division of
Pediatric General and Thoracic
back pain, leading to lifelong disability. reported in a landmark study that found major The physician team was up front with the many points in our journey, it was faith that kept
Surgery “We were devastated when we learned our baby benefits for fetal surgery in patients with spina Haglers about the risks, which include infection, us going,” she says. “When I think back about the
Program Director, Pediatric Surgery
Fellowship
had spina bifida,” says Colette Hagler, a resident bifida. The results of MOMS were published in bleeding, miscarriage, stillbirth, placental abrup- surgery, even now, my lasting impression was of
1
Located within the Texas
Co-Director, The Fetal Center of Dallas. “Our emotions were all over the place. an article that appeared in the March 17, 2011, tion, uterine scar separation/rupture, preterm the outstanding care I received from the doctors. Medical Center, The Fetal Center
McGovern Medical School at We had gone for a routine ultrasound to find issue of the New England Journal of Medicine. ruptured membranes, and preterm birth. For It was much more than a check-in, check-out re- is affiliated with Children’s
UTHealth Memorial Hermann Hospital,
out the gender, and the radiologist noticed an The Haglers learned of the MOMS trial during mothers who had prenatal surgery during the lationship. It was genuine caring, and we’ve been McGovern Medical School at
abnormality. When we learned of the possibility their internet search. trial, the average age of delivery was 34 weeks. singing their praises ever since.” UTHealth, and UT Physicians.
02 PEDIATRIC NEUROSCIENCE JOURNAL PEDIATRIC NEUROSCIENCE JOURNAL 03
F E TAL M YE L OM E NI NGOCE L E R E PA I R : PA S T, P R E S E N T, A N D FU T U R E
Independence From Spina Bifida: Toward a world-renowned surgeon in ocular surface
reconstruction, who was using human amniotic
accomplished something,” she says.
Currently, the research team is working to
“After investigating
many types of patches
a Safe Method of Minimally Invasive membrane and umbilical cord donated by
mothers of healthy infants to repair corneas.
find ways to encourage the skin to heal inside
the uterus and different ways to deploy the patch
in surgical animal
models, we found
Tseng is chief scientific officer and co-founder over the defect site through less invasive means. that using a graft from
Myelomeningocele Repair of TissueTech, a biotechnology company based “We’ve made progress at an incredibly rapid human umbilical cord
in Miami. The research team believes that pace,” Papanna says. “Taking an idea from the promotes regeneration
HUC has the potential to improve the quality lab to human use typically takes about a decade. of the protective layers
of life of children and families with spina bifida, We’ve been able to reduce that time by more than
In 2011, fetal surgeon KuoJen Tsao, MD, and pediatric neurosurgeon around the spinal
which can result in paralysis, urinary and bowel half. We have a good system in place with strong cord after surgery and
Stephen Fletcher, DO, were following the results of a landmark dysfunction, and mental retardation. collaborators, all of whom have track records of improves neurological
clinical trial, Management of Myelomeningocele Study (MOMS), Papanna’s current research builds on his success in their fields.” function.”
laboratory’s experience with cryopreserved “Dr. Papanna is a workhorse for UTHealth
which found that if a fetus undergoes surgery in utero to repair human umbilical cord for in-utero spina and our efforts to further our research in a very
a spina bifida defect, serious complications could be reversed or bifida repair. His findings have been published fast-moving and competitive market,” Fletcher
lessened when compared to infants who underwent repair after in multiple leading peer-reviewed journals says. “We see patients who come from across the
including the Journal of Neurosurgery: Spine,1 country, and when I ask them why they travel
birth. The study found that fetal surgery decreased the need for Journal of Pediatric Neurosurgery,2 Prenatal so far, they say it’s because of the research we’re
ventriculoperitoneal shunting for infants, and nearly half were able Diagnosis,3 AJP Reports,4 Obstetrics & Gynecology,5 doing at UTHealth. We are leaders in the field.
to walk without crutches by the age of 30 months. and Ultrasound in Obstetrics and Gynecology.6 We’re moving forward deliberately to answer
Made of the donated outer layer of the umbilical every question and avoid the failures that have
T
sao, co-director of The Fetal Center Designing a Regenerative Patch for In-Utero Repair cord of healthy newborns, the patch has been used occurred in other programs.”
at Children’s Memorial Hermann When skin closure of the spina bifida defect is for repairs of severe defects in cases performed at 1
Mann LK, Won JH, Trenton NJ, Garnett J, Snowise S, Fletcher SA,
Hospital and professor of pediatric not possible, a patch is needed. Fewer than half Children’s Memorial Hermann Hospital. All Tseng SCG, Diehl MR, Papanna R. Cryopreserved human umbilical
surgery at McGovern Medical School at of the patients who undergo in-utero spina bifida cases were approved by the U.S. Food and Drug cord versus acellular dermal matrix patches for in-utero fetal spina
bifida repair in a pregnant rat model. J Neurosurg Spine. 2019 Nov
UTHealth, and his maternal-fetal medicine repair with a patch show improvement in spinal Administration under expanded access, by The 1;1-11. doi: 10.3171/2019.7.SPINE19468. [Epub ahead of print].
colleagues were in the process of screening cord function. For the past decade, maternal- Fetal Center at Children’s Memorial Hermann PMID: 31675701.
patients for the study when Faith Hagler’s fetal medicine specialists Ramesha Papanna, Hospital, and by the UTHealth Institutional 2
Vu T, Mann LK, Fletcher SA, Jain R, Garnett J, Tsao K, Austin MT,
Moise KJ Jr, Johnson A, Shah MN, Papanna R. Suture techniques Lovepreet K. Mann,
parents, Ivan and Colette Hagler of Dallas, MD, MPH; Lovepreet K. Mann, MBBS, and Review Board prior to the surgeries. The patients and patch materials using an in-vitro model for watertight closure MBBS
were referred to The Fetal Center. Based on their research team have been working to gain underwent fetal surgery performed by Fletcher, of in-utero spina bifida repair. J Pediatr Surg. 2019 Jun 19;S0022- Assistant Professor,
3468(19):30409-9. doi: 10.1016/j.jpedsurg.2019.05.024. [Epub Department of Obstetrics,
the MOMS trial protocols, which included greater understanding of the lack of complete an associate professor of pediatric surgery at ahead of print]. PMID: 31255327. Gynecology, and Reproductive
medical, psychological, and social criteria, benefit after fetal surgery. They hope to improve McGovern Medical School, and Tsao. All patients 3
Snowise S, Mann L, Morales Y, Moise KJ Jr, Johnson A, Fletcher S,
Sciences
McGovern Medical School at
the team determined that Hagler was an ideal the neurological outcomes of affected children have good lower-extremity motor and sensory Grill RJ, Tseng SCG, Papanna R. Cryopreserved human umbilical cord UTHealth
candidate for the procedure. versus biocellulose film for prenatal spina bifida repair in a physiologic
through regenerative repair of the defect using function and have been able to walk. rat model. Prenat Diagn. 2017 May;37(5):473-481. doi: 10.1002/
After the fetal surgery, Hagler remained at a patch made of donated cryopreserved human “The HUC patch acts as a watertight scaffold, pd.5035. Epub 2017 Apr 16. PMID: 28295455.
Children’s Memorial Hermann Hospital and umbilical cord (HUC). allowing native tissue to regenerate in an 4
Papanna R, Mann LK, Snowise S, Morales Y, Prabhu SP, Tseng SC,
delivered Faith almost nine weeks later on the “After investigating many types of patches in organized manner,” says Mann, an assistant Grill R, Fletcher S, Moise KJ Jr. Neurological Outcomes after Human
Umbilical Cord Patch for In Utero Spina Bifida Repair in a Sheep
Fourth of July, a date that Hagler describes as “so surgical animal models, we found that using professor in the Department of Obstetrics, Model. AJP Rep. 2016 Jul;6(3):e309-17. doi: 10.1055/s-0036-
symbolic” of the independence her daughter has a graft from human umbilical cord promotes Gynecology, and Reproductive Sciences. “It 1592316. PMID: 27621952; PMCID: PMC5017885.
achieved since her birth. Faith began crawling regeneration of the protective layers around also has anti-scarring and anti-inflammatory 5
Papanna R, Fletcher S, Moise KJ Jr, Mann LK, Tseng SC. Cryo-
preserved Human Umbilical Cord for In Utero Myeloschisis
at 11 months, took her first steps at 21 months, the spinal cord after surgery and improves properties. Preventing the scarring could Repair. Obstet Gynecol. 2016 Aug;128(2):325-30. doi: 10.1097/
and today she’s dancing. neurological function,” says Papanna, an associate prevent spinal cord tethering, a common AOG.0000000000001512. PMID: 27400004.
The nine-year-old fetal surgery program at professor of maternal-fetal medicine at McGovern problem with spina bifida, which in turn can 6
Papanna R, Moise KJ Jr, Mann LK, Fletcher S, Schniederjan R,
Bhattacharjee MB, Stewart RJ, Kaur S, Prabhu SP, Tseng SC. Cryo-
The Fetal Center continues to produce excellent Medical School. “We have compared the HUC prevent further damage to the cord.” preserved human umbilical cord patch for in-utero spina bifida repair.
outcomes with patients chosen based on the patch to conventionally used methods in an effort Mann says the research team is focusing Ultrasound Obstet Gynecol. 2016 Feb;47(2):168-76. doi: 10.1002/
uog.15790. PMID: 26489897.
selection criteria established in the MOMS to reduce scar formation and improve spinal cord on further improving outcomes by pushing
trial. The selection process for in-utero repair is function at and below the defect site.” the boundaries of fetal wound healing and
based on a strict prenatal algorithm emphasizing The researchers’ work is based on preliminary improving outcomes through regenerative repair.
education to prepare families to make an data formulated in Papanna’s lab. Their research “If we can make a small change and improve the
informed decision about treatment. led them to Scheffer C.G. Tseng, MD, PhD, quality of life for these children, we have really
04 PEDIATRIC NEUROSCIENCE JOURNAL PEDIATRIC NEUROSCIENCE JOURNAL 05
F E TAL M YE L OM E NI NGOCE L E R E PA I R : PA S T, P R E S E N T, A N D FU T U R E
Clinical Trials: Advances in Regenerative
Patches for Spina Bifida Repair
Open In-Utero Repair of Severe born in the country each year, according Fetoscopic Spina Bifida Repair A patch is made from the
to the National Institute of Neurological donated outer layer of the
Spina Bifida Using a Human Disorders and Stroke. Associated disorders for Small Defects Using a Human umbilical cord of healthy
Umbilical Cord Patch include hydrocephalus and learning disability. Umbilical Cord Patch newborns. When applied to
An estimated 166,000 individuals with spina the neural tube defect, the
R R
amesha Papanna, MD, MPH, associate bifida live in the U.S. esearchers at McGovern Medical School patch allows local tissue to
professor of maternal-fetal medicine In addition to McGovern Medical School at UTHealth and Children’s Memorial grow at the repair site, which
at McGovern Medical School at and The Fetal Center at Children’s Memorial Hermann Hospital are now enrolling may help improve spinal
UTHealth, is the clinical lead principal Hermann Hospital, participating centers include patients in a study to determine the feasibility cord function by reducing the
investigator in a multicenter study of open the University of Colorado Denver; Children’s of fetoscopic surgery to repair spina bifida and effects of scar tissue.
in-utero repair for severe spina bifida using a Hospitals and Clinics of Minnesota; Fetal facilitate vaginal delivery. The single-center study The study, the first to use a meningeal patch to
patch made of cryopreserved human umbilical Care Center Dallas in Medical City Children’s is led by Ramesha Papanna, MD, MPH, an cover the spina bifida defect, will enroll 15 patients
cord (HUC). The patch, developed by Hospital; the University of California, San associate professor of maternal-fetal medicine age 18 and older with a singleton pregnancy, a
TissueTech, a biotechnology company based Francisco; and Cincinnati Children’s Hospital who is internationally recognized for his spina bifida defect of 4 centimeters or less, and
in Florida, is widely used for ocular surface Medical Center. research on improving outcomes following fetal no preterm birth risk factors. Participants also
repair and chronic skin ulcers because its TissueTech will pay for all participant hospi- intervention and investigating methods for the must meet other study qualifications.
innate regenerative properties facilitate faster tal expenses related to research, including the prevention of preterm delivery. A digital image of the fetal repair site will
healing with minimal scarring. patch itself, travel expenses, and a follow-up “Our primary outcome measure for the study be captured immediately after the repair, and
Papanna and his research team have used the MRI at 12 months. Although funding for the is successful surgical closure of the spina bifida efficacy of the fetoscopic repair will be assessed
HUC patch successfully with patients through study will conclude at 12 months, the primary defect with a watertight patch that approximates after birth by three blinded reviewers. Reviewing
the U.S. Food and Drug Administration’s outcome date required for the FDA to ap- native tissue and allows for the natural growth of neurosurgeons are Arthur Day, MD, McGovern
Expanded Access Program, a pathway for prove the patch for clinical indications, data the spinal cord,” Papanna says. “The procedure Medical School and UTHealth Neurosciences
people with life-threatening conditions or will be collected on participating patients for differs from in-utero repair, which requires a in Houston; Bradley Edward Weprin, MD, UT
serious diseases to gain access to investigational 30 months. large incision on the uterus and delivery by Southwestern Medical Center in Dallas; and
medical products for treatment outside of The study is the culmination of 10 years of cesarean section. Instead, we will repair the spina John Honeycutt, MD, Cook Children’s Health
clinical trials, when no satisfactory alternative research conducted by Papanna and Lovepreet bifida defect in two layers through three small Care System in Fort Worth.
therapy options are available. K. Mann, MBBS, assistant professor of maternal- incisions in the uterus using fetoscopes and tiny Patients referred to The Fetal Center at
“Cryopreserved human umbilical cord fetal medicine at McGovern Medical School, surgical tools. The first layer will be closed using a Children’s Memorial Hermann Hospital who
has anti-scarring, anti-inflammatory, and and their team. The researchers found that using NEOX® CORD 1K patch as a meningeal patch intend to undergo open in-utero spina bifida
regenerative properties that makes it an effective a graft of the human umbilical cord after surgery placed over the spinal cord, followed by a second repair will be offered and screened for the
substrate for wound healing,” Papanna says. for spina bifida could promote regeneration of layer of primary closure of the skin. Mothers alternative minimally invasive approach. Women
“These unique properties may eliminate the the protective layers around the spinal cord and will undergo vaginal delivery, unless there is an who participate in the study must agree to deliver
scar formation associated with traditional spina improve neurological function in animal models. obstetrical indication for delivery by C-section.” at Children’s Memorial Hermann Hospital.
bifida repair methods and reduce the need “HUC could be a game changer for spina Developed by Amniox, the NEOX CORD 1K “We have published promising preclinical
for future surgeries for tethered spinal cord, a bifida repair,” Papanna says. “Our ultimate goal patch is made of cryopreserved umbilical cord data and have rigorously tested our techniques
complication of the disorder. Preclinical data is to ensure that babies born with the disorder and amniotic membrane. Extensive laboratory before taking fetoscopic repair to humans,”
have shown that the patch promotes organized can walk and lead normal lives. We also think and clinical research on an ocular wound surface Papanna says. “Our research is changing the way
cell growth, resulting in a spinal cord repair that HUC will lead to new paradigms in fetal wound has shown that placental tissues help manage we approach spina bifida to improve closure,
appears more normal with better function.” healing for other spinal defects and repairs.” inflammation in wounds, facilitate cell proliferation reduce scar tissue formation, reduce neurological
Spina bifida is the most common neural and create an environment for tissue regeneration. deficits, and improve function. With this trial we
tube defect in the U.S., affecting about 1,500 NEOX CORD 1K has demonstrated consistently hope to show that the NEOX CORD 1K patch
to 2,000 of the more than 4 million babies high closure rates in real-world experiences. optimizes long-term outcomes for these kids.”
06 PEDIATRIC NEUROSCIENCE JOURNAL PEDIATRIC NEUROSCIENCE JOURNAL 07
CRANI OSYNOST OSI S
Innovation in Surgery for Unicoronal
Craniosynostosis: Presurgical Preparation on a
3D Model Ensures Success in Two Cases
During the last two decades, treatment for craniosynostosis has
evolved from procedures that produced relatively poor results to What Is Craniosynostosis and When Is Surgery Necessary?
tailored cranial vault reconstruction and cranioplasty.
B
Manish N. Shah, MD,
ut unicoronal craniosynostosis remains cranial vault remodeling provides and a much FAANS
challenging to treat because it affects the better patient experience.” Director of Pediatric Spasticity
and Epilepsy Surgery
skull base, the middle cranial fossa, and the In 2019, the two physicians were approached
NORMAL METOPIC SAGITTAL LAMBDOID BICORONAL UNICORONAL (ALL SUTURES OPEN) Director of Texas Comprehensive
intrafacial fossa, the structure that houses the eyes. by mothers of two girls born with unicoronal Spasticity Center
“The classic presentation of unicoronal craniosynostosis; both families were looking Assistant Professor,
Department of Pediatric Surgery
craniosynostosis is harlequin eye – one eye that for alternatives to the cranial vault procedure One in 2,500 infants per year is born with craniosynos- ture. When one is fused, there is compensatory growth Division of Pediatric Neurosurgery
is larger than the other,” says Manish N. Shah, and the prospect of wearing a helmet 23 hours tosis, a relatively rare birth defect of the skull marked on the other side of the skull. Coronal craniosynostosis McGovern Medical School at
UTHealth
Photo courtesy of
MD, FAANS, assistant professor in the Division a day. Nguyen and Shah offered them a newer, by premature closure of one or more of the cranial su- may involve one or both coronal sutures.
Ashley LeBlanc Photography
of Pediatric Neurosurgery and director of pediatric innovative option. tures, the fibrous joints between the bones of the skull. Unicoronal craniosynostosis – one fused coronal su-
Raelynn Guidry in May spasticity and epilepsy surgery at McGovern The most common manifestation of the defect is the ture – results in a flattened forehead on the affected
Medical School at UTHealth. “The standard Raelynn Guidry closure of a single suture. side, with the classic presentation of harlequin eye –
2020 after distraction
surgery for unicoronal surgery is fronto-orbital advancement, an open When Raelynn was 10 days old, her mother, The sutures allow the baby to exit the birth canal, and one larger eye. When both coronal sutures are affected,
craniosynostosis. Inset: cranial vault procedure that involves cutting the Danielle Guidry, BS, RN, NCM, looked down at continue to mold after birth. The infant’s brain grows the presentation is a short, wide head with bulging of
September 2019, two bones of the skull into pieces and putting them her daughter from above and noticed a ridge on the exponentially in the first year of life, and normally the the eyes. Brain growth may be restricted.
weeks before the procedure. together with plates and screws to push the right side of her head. A case management nurse skull expands uniformly to accommodate the growth. Treatment for craniosynostosis consists of surgery
affected side of the skull forward. Across all centers in Louisiana, Guidry suspected craniosynostosis. Premature closure of a single suture restricts the growth performed by a pediatric plastic/craniofacial surgeon Phuong Nguyen, MD,
in the U.S., this is the most common procedure, “My pediatrician confirmed it, but I had no idea in that part of the skull, allowing for growth in other and pediatric neurosurgeon to improve the symmetry FACS, FAAP
Assistant Professor and Director
and it is appropriate for older children from 8 to where to go,” she says. “That evening my sister parts of the skull where the sutures remain open. While and appearance of the head and to relieve pressure of Craniofacial Surgery, Division of
12 months of age. The surgery works to a variable told me her best friend’s son had a rhizotomy for the brain may continue to develop normally, closure of on the brain and cranial nerves. The neurosurgeon Pediatric Plastic Surgery
McGovern Medical School at
degree, but when you look at these kids a year later, cerebral palsy in Houston, and they were happy one suture results in a misshapen skull. If many sutures opens the skull and exposes the area where the plastic UTHealth
the result is not as good as it could be.” with the result and the experience they had.” are fused, the skull cannot expand to accommodate surgeon will remove the bones, reshape them and put
The advent of the endoscope allowed surgeons The friend’s surgeon was Shah, the leading the infant’s growing brain. them back into place. The combined expertise of the
to use smaller incisions to access the cranial neurosurgeon in the region for selective dorsal The most apparent sign of craniosynostosis is an two surgical specialties produces optimal results.
sutures. “Endoscopic-assisted surgery has been rhizotomy and an expert in pediatric epilepsy, abnormally shaped skull. Other signs may include in- For decades, the only surgery was open, with an
used for more than 20 years in the craniofacial craniofacial surgery, and craniocervical spine creased intracranial pressure, developmental delays, or incision from ear to ear, called cranial vault remodel-
skeleton with some degree of success in children surgery. Guidry texted Shah’s office and the impaired cognitive development caused by restriction ing or cranial vault reconstruction. It involves remov-
who are 3 to 6 months of age,” says Phuong following day, July 3, 2019, she, Raelynn, and of brain growth. al of bone by a neurosurgeon to free the area that
Nguyen, MD, assistant professor of surgery and her husband, Andy Guidry, made the four-hour More than half of all cases of craniosynostosis in- has been restricted in growth, and is still the most
director of craniofacial surgery in the Division of drive to Houston. They saw Shah and his team, volve the sagittal suture, which runs from a spot in the common procedure for older children from 8 to 12
Pediatric Plastic Surgery at McGovern Medical followed by Nguyen and his team. The two front of the head to the back of the skull. Fusion of the months of age in most centers across the U.S. When
School. “The bones are much softer at that age surgeons ordered a CT scan and asked the family suture results in a long, narrow skull with or without craniosynostosis is diagnosed early, between 3 and
and the brain is growing exponentially. We can to give them time to consult. bulging of both the front and the back of the head. 6 months of age, treatment options include mini-
take advantage of that time of life by removing “After we saw Raelynn, I talked with Dr. Metopic craniosynostosis results in a narrow, triangular mally invasive procedures that mean less scarring,
the abnormal or fused suture and fitting the Shah about a relatively new minimally invasive forehead with lateral pinching of the temples. There are less blood loss, less patient discomfort, and shorter
infant with a cranial modeling helmet for two procedure for unicoronal craniosynostosis,” two coronal sutures that begin at the ear and continue hospital stays than the traditional corrective cranial
months to a year, depending on the patient. But Nguyen says. “We were upfront with the family to the top of the skull, where they join the sagittal su- vault surgery.
our ultimate goal is a better result than what and told them that we’d done parts of the surgery
08 PEDIATRIC NEUROSCIENCE JOURNAL PEDIATRIC NEUROSCIENCE JOURNAL 09
CRANI OSYNOST OSI S
before, but had never used it for this particular following surgery, after her parents made the first us there was a newer, less invasive procedure that intervention. Everything aligned perfectly. We
application. They said they were willing to take turn of the distractor at 7 a.m. on the day she was had shown good long-term results. He talked with couldn’t have gotten to where we are without
the journey with us.” released. The Guidrys made two turns twice a day me about the distraction technique and told me God sending us where we needed to go. Giving
The surgery they proposed involved the use for a total of 100 turns of the distractor over 25 that he and Dr. Shah were performing their first us a friend was putting the cherry on top. People
of a distractor, a controlled instrument that very days, resulting in 33 millimeters of movement. distraction surgery for unicoronal craniosynostosis who haven’t gone through craniosynostosis can
slowly pulls apart the bone to create space. As new “We’re very happy with the results. We didn’t the following Friday. That was Danielle’s baby.” never really understand.”
bone grows in, it remodels the skull. “We’ve used want a second surgery or a helmet,” says Danielle Kate’s right coronal suture was fused. “The Guidry has been a nurse for a decade. “I’ve
the procedure often for children with bicoronal Guidry, who posted the family’s experience on a right side of her head was not growing and been all over Louisiana, and I have never met
closed craniosynostosis Facebook page, thanking the left side was,” she says. “The bridge of her
her friends for their support. Among those who nose was being pulled to the right, and her
commented was Marisa Medlin, who asked for right eyebrow was raised. She had a perpetual
the names of her surgeons. She was excited to learn surprised look on her face. We had to make the
that Raelynn’s surgeons were the same doctors she decision about whether to do the standard but
and her family had met with the day before. invasive procedure or decide if we were willing
to take a chance on a new surgery they had done
Kate Medlin only one time before us. When Dr. Nguyen
Marisa Medlin noticed that her daughter Kate’s told me they would use her CT results to create
nose was crooked while she was nursing during a 3D printed version of the skull and perform
her first month of life. When she was one month the surgery beforehand, we felt better about it.
old, Kate was referred to a pediatric ENT, who In the end, we felt it was the right choice for
saw no reason for concern. At her 4-month visit, Kate and our family.”
it was apparent that the facial asymmetry had After the surgery to place a distractor, the
not corrected itself, and her pediatrician referred Medlins turned the device twice a day for 25
the family to Nguyen at UTHealth. days, moving the right front portion of Kate’s
“As soon as he saw her, he recognized the signs skull forward. They stopped turning the
of craniosynostosis, and a CT scan confirmed distractor on Nov. 2, and left it in place for
Manish N. Shah, MD, craniosynostosis and based on the limited it,” says Medlin, who lives in Magnolia, Texas, two more months to allow new bone growth physicians like Dr. Nguyen and Dr. Shah,” she Marisa and Shawn Medlin
left, and Phuong Nguyen, experience of physicians at two other centers, we about an hour north of the Texas Medical to fill the gap. It grew faster than expected, and says. “Not many doctors will give you their cell with their daughter Kate,
MD, prepare for distractor wanted to apply it to unicoronal craniosynostosis Center, where Nguyen and Shah practice. “I Nguyen removed the distractor two weeks early phone numbers and email addresses. They saw us who benefited from a
surgery using a 3D model for better skull shape and cosmetic results,” Shah had never heard of craniosynostosis and was on Jan. 6, 2020. on their off days and came to the hospital when new minimally invasive
they designed and had says. “Because we’re moving the bones slowly, the trying to learn as much as I could about Kate’s they knew we were in Houston for a checkup or procedure for unicoronal
printed at UTHealth. resulting symmetry is better. Later we remove the diagnosis. One source of information was a Friendship Forged Through Shared Experience a procedure. When we were in the hospital, they craniosynostosis.
distractor in a small surgery. The skull correction craniosynostosis Facebook group, which I Parents of children with craniosynostosis go to rounded two or three times a day. They don’t
is done at a much earlier age than with the open joined. I happened to see Danielle’s post that great lengths to find the best possible center for disregard anything you say. No matter what
cranial vault surgery, it’s much less invasive, and her daughter was about to have surgery for right treatment. They do their research, and they’re emotions you’re experiencing, they understand
the children won’t need a later surgery.” unicoronal craniosynostosis with Shah and willing to travel. and empathize.”
Nguyen. I was so excited to connect with her “After Danielle and I found each other, we Medlin is happy that things turned out the way
Precision Through 3D Modeling because her daughter had the same diagnosis texted or spoke every day about what our they did. “We’re so thankful that Dr. Nguyen
Using the CT scans of Raelynn’s skull, the two as Kate and was having the same procedure daughters were going through,” Medlin says. “It and Dr. Shah were willing to challenge the status
surgeons designed a 3D model and had it printed with the same doctors we were considering. I was so comforting for me to talk with another quo and perform an innovative procedure that
at UTHealth. They performed the surgery on messaged her immediately and we have been in mom who was going through the exact same we feel was the best choice for our daughter,”
the model skull the day before they took Raelynn constant communication since then.” process that we were going through. I am so she says. “Kate looks amazing! We’re so happy
to the OR, which allowed them to plan and fine Medlin had done enough research to know incredibly grateful to have met her and to have with her result and so incredibly grateful to Dr.
tune the incisions they would make. that undergoing cranial vault remodeling was not had her to lean on through this experience.” Nguyen and Dr. Shah. In our eyes, they’re rock
“We operate and wait two to three days to the ideal solution for Kate. “It’s a very invasive The two families met in person in late stars. They went above and beyond through this
begin turning the distractor, which slowly procedure, an eight- to nine-hour surgery December 2019 at Santa’s Wonderland in entire process to make sure all of our questions
ratchets the bones out,” Shah says. “After a requiring a blood transfusion and a five-night stay College Station, Texas, where both had planned were answered and that we felt as comfortable as
period of about a month, on average, the in the hospital,” she says. “The thought of this family holiday outings. possible. We hope to help spread awareness of
distractor has done its work.” major surgery being performed on our 5 month- “Marisa and I still text at least every other day,” this treatment option to other families who find
Raelynn was discharged on the third day old baby was very scary for us. Dr. Nguyen told Guidry says. “In my eyes, our meeting was divine themselves in the same situation we were in.”
10 PEDIATRIC NEUROSCIENCE JOURNAL PEDIATRIC NEUROSCIENCE JOURNAL 11
Research
David Sandberg, MD, is
Novel Studies Seek to Improve survival rates are low.
now enrolling patients in
“The current treatments for children and
Outcomes in Children with adults with medulloblastoma are inadequate,” three clinical trials of novel
Malignant Fourth Ventricular says Sandberg. “Children have low survival rates therapies with the potential
to improve outcomes for
Brain Tumors despite salvage therapy, and novel approaches
children with fourth
are needed. This is a new trial of a novel drug,
and we are very hopeful that we can help ventricular brain tumors.
The outlook for children with recurrent
malignant brain tumors originating from the patients overcome this devastating disease.”
posterior fossa is extremely poor. Most clinical The clinical trial follows a successful study
trials offer systemic chemotherapy or re- led by Sandberg in an animal model, which
irradiation, both of which can have serious demonstrated that MTX110 can be safely
side effects and most often fail in children with infused in the fourth ventricle and can achieve
recurrent tumors. In three single-center studies drug levels dramatically higher than intravenous
under way at Children’s Memorial Hermann or oral administration of the same drug. The
David I. Sandberg, MD, Hospital and McGovern Medical School study team at Children’s Memorial Hermann
FAANS, FACS, FAAP Hospital and McGovern Medical School found
Professor, Departments of Pediatric
at UTHealth, researchers are investigating
Surgery and Neurosurgery novel therapies with the potential to improve no neurological deficits after fourth-ventricle
Dr. Marnie Rose Professor in
outcomes for children with fourth ventricular infusions.
Pediatric Neurosurgery
McGovern Medical School at brain tumors, while avoiding systemic toxicity. “Our objective was to test the safety and
UTHealth
These studies are led by David Sandberg, pharmacokinetics of short-term and long-term
Director of Pediatric Neurosurgery
MD, FAANS, FACP, FAAP, professor of infusions of MTX110, a chemotherapeutic
Children's Memorial Hermann
Hospital and Mischer Neuroscience pediatric neurosurgery at McGovern Medical agent that inhibits the growth of medulloblas-
Institute
School and UTHealth Neurosciences, and toma, the most common malignant brain tu-
director of pediatric neurosurgery at Children’s mor in children,” Sandberg says. “In the animal
Memorial Hermann Hospital. “Despite the study group there were no MRI signal changes
advances that have been made in pediatric in the brainstem, cerebellum, or elsewhere in
neuro-oncology, we are still seeing too many the brain. In addition, the cytoarchitecture of
children die of malignant brain tumors,” says the brain was preserved in all of the animals,
Sandberg, who holds the Dr. Marnie Rose with only mild postsurgical changes.
Professorship in Pediatric Neurosurgery at “We are really excited about the promising
UTHealth. “The treatments currently available data from these experiments,” says Sandberg,
are not satisfactory for children. We believe we who is lead author of an article detailing results Combination Intraventricular Chemotherapy Pilot and etoposide infusions into the fourth ventricle
can do better.” in the Journal of Neurosurgery: Pediatrics.1 Study and Infusion of 5-Azacytidine in children with recurrent posterior fossa brain
The pilot study, which has been approved by Two additional clinical trials of infusions into the tumors. The trial is open to patients age 1 to 80
Clinical Trial of High-Dose MTX110 (Soluble Pano- the U.S. Food and Drug Administration, will years with recurrent medulloblastoma, recurrent
fourth ventricle or resection cavity of children
binostat) Begins After Safe Administration into the enroll five patients with recurrent medulloblasto- ependymoma, or recurrent atypical teratoid/
with brain tumors are currently enrolling at
Fourth Ventricle in a Non-Human Model ma at Children’s Memorial Hermann Hospital. rhabdoid tumors of the brain and/or spine.
McGovern Medical School at UTHealth and
A novel clinical trial of MTX110, a new Children’s Memorial Hermann Hospital. Both “Our primary objective is to determine if
The study is listed at clinicaltrials.gov at https:// combination intraventricular infusions of
formulation of soluble panobinostat from build on translational models of direct infusion
clinicaltrials.gov/ct2/show/NCT04315064. For more
Midatech Pharma, is now enrolling patients. of chemotherapy into the fourth ventricle of the these two agents are safe and do not result in
information, please contact Bangning Yu, MD, PhD,
According to the American Cancer Society, brain in animal models, developed by Sandberg. neurological toxicity,” he says. “We are also
at bangning.yu@uth.tmc.edu or 713-500-7363.
about 500 children are diagnosed every year with Neither involves simultaneous systemic assessing the antitumor activity of these infusions
medulloblastoma, the most common malignant chemotherapy. in the hope that they will yield even more robust
brain tumor in children. Current treatments are 1
Sandberg DI, Kharas N, Yu B, Janssen CF, Trimble A, Ballester LY, Patel R, In “A Combination Intraventricular Chemo- treatment responses than those observed in
Mohammad AS, Elmquist WF, Sirianni RW. High-dose MTX110 (soluble
often associated with considerable toxicity, and panobinostat) safely administered into the fourth ventricle in a non-human
therapy Pilot Study,” Sandberg and his research previous single-agent trials.”
when tumors recur despite these treatments, primate model. J Neurosurg: Pediatr. 2020 May 1:1-9. [Epub ahead of print] team are investigating combined methotrexate The other trial involves infusion of 5-Azacyti-
12 PEDIATRIC NEUROSCIENCE JOURNAL PEDIATRIC NEUROSCIENCE JOURNAL 13
RE SE ARCH
Nanotechnology for Drug To address this challenge, Sirianni and her team medulloblastoma. Her laboratory will evaluate
are working on novel approaches. “Pediatric brain the safety and efficacy of these new approaches,
Delivery: The Science of tumors have a tendency to metastasize along the as well as test whether delivery of drugs from
Moving Chemotherapeutic surfaces of the brain and spinal cord. This is called nanoparticles can reduce the radiation dose
Drugs Directly to Brain Tumors leptomeningeal metastasis, and it remains very needed to treat metastases.
difficult to treat,” she says. “Instead of delivering In July 2019, Sirianni received a second five- Rachael Sirianni, PhD
Sandberg’s research Assistant Professor,
Only a very small amount of the chemothera- nanoparticles intravenously, we’re working toward year, $4.5 million R01 from the Eunice Kennedy Vivian L. Smith Department of
focuses on infusions of
peutic drugs given systemically for the treatment administering them directly to the cerebrospinal Shriver National Institute of Child Health and Neurosurgery
chemotherapeutic agents McGovern Medical School at
fluid that moves across these lesions to deliver Human Development to study intrathecal delivery
directly into the fourth of pediatric brain tumors actually reaches the UTHealth
more drug with less overall toxicity. Currently we’re of radiation-sensitizing nanoparticles in pediatric
ventricle or resection cavity brain, due to the blood-brain barrier’s efficiency
focused on engineering nanoparticles to possess the neuro-oncology.
of children with malignant at excluding the entry of most agents that circu-
right properties to accumulate selectively within “High-dose craniospinal radiation damages a
brain tumors to avoid late in the blood. As a result, the current outlook
these metastatic lesions.” child’s developing nervous system, and few other
systemic toxicity. for children with recurrent malignant brain
Because the polymers her laboratory uses are treatment options are available once malignant
dine (5-AZA) into the fourth ventricle or resec- tumors is extremely poor. Most clinical trials of- cells have metastasized to the leptomeninges,”
nontoxic and readily cleared by the body, degrading
tion cavity in children with recurrent posterior fer systemic chemotherapy or radiation therapy, over weeks to months, there is potential to design she says. “Our preliminary data demonstrate With funding from the
fossa ependymoma. 5-AZA is a DNA meth- both of which have side effects and often fail in new, safer chemotherapy for patients. that intrathecally administered histone deacetyle NIH, Rachael Sirianni,
ylation inhibitor that has been infused in children with recurrent tumors. Bioengineer and In May 2019 Sirianni was awarded a five- inhibitor quisiwnostat distributes readily across PhD, and her team are
an animal model with no neurological toxicity, research scientist Rachael Sirianni, PhD, aims to year, $2.7 million R01 grant by the National brain and spinal cord surfaces and localizes to moving toward delivering
while achieving substantial and sustained cere- change that by bringing novel nanomedicine Institute of Neurological Disorders and Stroke slow the growth of leptomeningeal metastasis nanoparticles directly to the
brospinal fluid levels. approaches to the clinic to improve outcomes. to tackle exactly this problem: designing in medulloblastoma. We expect these studies to CSF to deliver more drug
“Recent studies have demonstrated that DNA “There are many drugs available to treat nanoparticles that can target drug delivery advance new nanotechnology toward the clinic for to brain tumors with less
methylation inhibitors are logical therapeutic brain tumors, but most don’t go directly to the to leptomeningeal metastasis in pediatric better treatment of pediatric brain tumors.” toxicity.
candidates for ependymomas originating in the site where they provide the most benefit,” says
posterior fossa,” Sandberg says. “We’ve shown Sirianni, an assistant professor in the Vivian L.
in the laboratory that 5-AZA kills ependymoma Smith Department of Neurosurgery at McGovern
cells and hope to establish the safety of direct Medical School at UTHealth and faculty member
administration of 5-AZA into the fourth ventricle, of MD Anderson Cancer Center UTHealth
and also demonstrate the clinical efficacy of these Graduate School of Biomedical Sciences. “My
infusions. Moreover, low-dose infusions in a pilot science is the science of drug delivery and
trial we conducted showed shrinkage of some understanding ways to circumvent these barriers.”
tumors in the brain. We are hopeful that higher Sirianni and her team encapsulate drugs within
doses and more frequent dosing can lead to even biocompatible and biodegradable nanoparticles,
more robust responses.” which serve as carriers to prolong drug action
The study is open to patients age 1 to 21 years and target specific tissue sites. “Early on scientists
old with recurrent ependymoma that originated discovered that nanoparticles have the capability
in the posterior fossa of the brain. to slide in between the spaces of the tumor’s
If you have questions about the clinical trials or vasculature, such that they selectively accumulate
would like more information about enrollment, within the tumor,” she says. “This enhanced
contact Bangning Yu, MD, PhD, at bangning.yu@uth. permeation retention enables nanoparticles to
tmc.edu or call 713-500-7363. deliver encapsulated drugs preferentially to large
tumors that are highly vascularized. However,
there are some kinds of tumors and parts of
1
Sandberg DI, Kharas N, Yu B, Janssen CF, Trimble A, Ballester LY,
Patel R, Mohammad AS, Elmquist WF, Sirianni RW. High Dose tumors that do not receive a good blood supply.
MTX110 (Soluble Panobinostat) Safely Administered into the Fourth Delivery to these kinds of tumors remains a
Ventricle in a Non-human Primate Model. J Neurosurery: Pediatrics.
[In press] major challenge.”
14 PEDIATRIC NEUROSCIENCE JOURNAL PEDIATRIC NEUROSCIENCE JOURNAL 15
NE W S OF NOT E
News of Note
Dr. David Sandberg Receives Neuroscience Institute at Memorial Hermann- made two trips to Guatemala with Neil Feldstein, Dr. Rachael Sirianni Named
Texas Medical Center, as well as co-director of the MD, director of the Division of Pediatric Neuro-
the American Association Pediatric Brain Tumor Program at MD Anderson surgery at Columbia University Medical Center/
UTHealth Women Faculty
of Neurological Surgeons Cancer Center. Fellowship trained in pediatric New York-Presbyterian Hospital, performing sur- Forum Rising Star
Humanitarian Award neurosurgery with a special clinical and research gery on children with spina bifida. As a resident in Rachael Sirianni, PhD, was recognized with the
interest in pediatric brain tumors, Sandberg neurosurgery, he used his vacation time to spend 2019 Rising Star Award given by the Women
David I. Sandberg, MD, FAANS, FACS, FAAP, specializes in minimally invasive endoscopic a month in Tegucigalpa, Honduras. There, with Faculty Forum at McGovern Medical School at
has received the American Association of Neu- approaches to brain tumors, hydrocephalus, and the help of Mark Souweidane, MD, director of UTHealth. The excellence awards reception was
rological Surgeons 2019 Humanitarian Award, arachnoid cysts, as well as surgical management pediatric neurosurgery at Weill Cornell Brain and held Oct. 7, 2019.
one of the highest honors bestowed by the orga- of arteriovenous malformations of the brain, Spine Center, he brought an 8-year-old girl with a Trained as a biomedical engineer in the field
nization. The award recognizes his extraordinary congenital spinal anomalies, Chiari malformations, brain tumor to the U.S. for treatment. She was the of polymeric drug delivery, Dr. Sirianni earned
work with children suffering from neurosurgical and craniofacial anomalies. The recipient of first of several patients he brought to the U.S. her PhD from Yale University in 2008, and
disorders throughout the world. numerous research grants, he is currently principal Sandberg has been on the Board of Directors completed a postdoctoral fellowship in diagnostic
The award recognizes Sandberg is professor and director of pediatric investigator of three single-center trials at Children’s of the Foundation for International Education in radiology at the Yale School of Medicine.
Sandberg’s extraordinary neurosurgery at McGovern Medical School at Memorial Hermann Hospital and McGovern Neurological Surgery (FIENS) since 2004 and Sirianni’s research program, which has
work with children UTHealth, where he holds the Dr. Marnie Rose Medical School investigating direct administration served as secretary of FIENS from 2013 to 2018. received funding from the Department of
suffering from Professorship in Pediatric Neurosurgery. He is also of chemotherapy into the fourth ventricle for Through FIENS, he made multiple neurosurgical Defense and the National Institutes of Health,
neurosurgical disorders director of pediatric neurosurgery at Children’s treatment of malignant brain tumors that originate humanitarian trips to Honduras, Peru, and has designed creative approaches for treating
throughout the world. Memorial Hermann Hospital and Mischer from that ventricle. Guatemala. In 2006, he traveled to Uganda with central nervous system disease via encapsulation
Sandberg is a magna cum laude graduate of CURE International. and tissue-specific delivery of drugs from
Harvard University. He received his medical Since 2007 he has worked with Project polymeric nanoparticles. She also has developed
degree at the Johns Hopkins University School of Medishare, a Miami-based nonprofit organization biomaterial approaches for studying and
Medicine and completed neurosurgery training with a 20-plus-year history of empowering manipulating the behavior of cells in engineered
at Weill Medical College of Cornell University/ Haitians to provide quality health care through microenvironments mimicking the brain. These
New York-Presbyterian Hospital. During community-based, Haitian-led programs. approaches have demonstrated preclinical success
residency, he was awarded the Resident Traveling Through medical volunteers, the organization in applications ranging from neuro-oncology to
Fellowship in Pediatric Neurosurgery by the treats more than 180,000 people annually. neurodegeneration and neural engineering.
American Association of Neurological Surgeons Sandberg leads a team of pediatric neurosurgeons, Sirianni’s research methods are poised to make
and the Congress of Neurological Surgeons, pediatric anesthesiologists, nurses, and surgical a significant impact in the field of pediatric
which he completed at the Hospital for Sick scrub technologists from Children’s Memorial neuro-oncology. Her goal at UTHealth is to
Children in Toronto, Canada. He completed Hermann Hospital and McGovern Medical develop nanoparticle systems that can effectively
pediatric neurosurgery fellowship training at School; they make the trip annually. treat central nervous system infiltration and
the Children’s Hospital in Los Angeles. Before “Education is a major focus for all of our metastasis in children affected by recurrent
joining McGovern Medical School, Sandberg mission trips to Haiti,” he says. “We work with malignant brain tumors.
was an associate professor of clinical neurological a local neurosurgeon, Dr. Yudy Lafortune, to Her professional accomplishments include
surgery and pediatrics at the University of Miami teach him modern neurosurgical care for pediatric invitations to speak at major research conferences,
Miller School of Medicine. patients. This is the ‘teach-a-man-to-fish’ concept. service on an NIH study section, book editorship,
He began his medical mission work in high We’re training Dr. Lafortune to be the pediatric and extensive publications in the fields of
school and college, accompanying his father, neurosurgeon for the children of Haiti.” biomaterials, drug delivery, and imaging.
Miami ophthalmologist Joel Sandberg, MD, on Sandberg grew up in a family with a strong The Women Faculty Forum at McGovern
trips to Jamaica, Antigua, and the Dominican commitment to public service. “I think how Medical School advocates on behalf of women
Republic. His father was his inspiration to lucky I am to have been born into my family in faculty, provides opportunities for professional
practice medicine and also to contribute to the United States, and to be able to contribute development, and offers an opportunity for
the health of the world through international in a small way to helping children in other networking among women faculty, as well as
mission work. countries,” he says. “This award is a huge honor with leaders in academic science and medicine
As a junior neurosurgery resident, Sandberg for me, and I’m humbled to receive it.” within and outside the institution.
16 PEDIATRIC NEUROSCIENCE JOURNAL PEDIATRIC NEUROSCIENCE JOURNAL 17You can also read
