Neurosciences Annual Report :: Nationwide Children's Hospital

Neurosciences 2014-2015 Annual Report (Neurology and Neurosurgery)

View the Nationwide Children's Hospital 2014-2015 Annual Report.

Physicians of the Section of Neurology at Nationwide Children’s Hospital deliver comprehensive patient care for children with a wide variety of neurological and neuromuscular diseases and provide state-of-the-art neurophysiological diagnostic testing and intraoperative monitoring. The section offers outstanding residency and fellowship opportunities. A variety of ongoing research activities, take place in the section and in collaboration with the Center for Gene Therapy.

Ranked in the top 10 programs in the country by U.S. News & World Report, the Division of Pediatric Neurosurgery at Nationwide Children’s Hospital provides evaluation and treatment for problems of the central nervous system in patients from birth through 21 years and in selected adults with congenital neurological disorders. The staff works closely with members of the departments of Anesthesia, Hematology/Oncology/BMT, Neurology, Physical Medicine and Rehabilitation, Pediatric Surgery, Plastic and Reconstructive Surgery, and other pediatric disciplines.

Neurology Highlights

The section has 33 physicians, all of whom are faculty members at The Ohio State University College of Medicine. The group also includes nine nurse practitioners and a physician assistant. Members of the section oversee comprehensive programs for children with epilepsy, neuromuscular disease, movement disorders, and headache. Other multidisciplinary programs include the country’s first Batten Disease Center of Excellence, the first Intracranial Hypertension (pseudotumor cerebri) Program for children, the Stroke and Vascular Anomalies Program, the Tuberous Sclerosis Clinic, the Neuroimmunology Program focusing on demyelinating disease in children, and a brain tumor program.

Recently the Section recruited a pediatric movement disorders specialist, one of only a few individuals in the country with extensive training in these disorders. Another recent recruit completed fellowships in both child neurology and critical care medicine, allowing us to develop a program for very ill children with neurological impairment. Recruitment of three additional physicians who specialize in epilepsy and clinical neurophysiology allows the group to expand its services for children with epilepsy and further develop its epilepsy surgery program.

The child neurology residency program is certified by the ACGME and continues to attract excellent trainees. The Section also offers fellowship training in clinical neurophysiology and neuromuscular disease. Faculty members publish numerous journal articles and textbooks, especially related to dysfunction of the autonomic nervous system, management of childhood stroke, neuromuscular disease, and epilepsy. Several faculty members serve as international leaders in child neurology, including Dr. Lo, who was recently elected chair of the Section of Child Neurology of the American Academy of Neurology. Dr. Roach is editor of the journal Pediatric Neurology, and members of the Neurology Section also hold nine editorial board positions with other medical journals.

Neurosurgery Highlights

July 2014 to June 2015 represented the first year for Jeffery Leonard, MD, as division chief, and during that time two additional neurosurgeons, Jonathan Pindrik, MD, and Eric Sribnick, MD, PhD, were hired. In partnership with The Ohio State University Wexner Medical Center Department of Neurological Surgery, a transgenerational epilepsy program was created, capable of serving the needs of both institutions. In addition, Dr. Pindrik’s clinical interests in brachial plexus surgery and peripheral nerve injury represent a new direction for the department. Dr. Sribnick’s clinical interests include traumatic brain injury and spine surgery. He is also establishing a basic science lab studying head and spinal cord injury. He, along with the Intensive Care Unit, has revised the head injury-management guidelines that reflect the multidisciplinary care these patients require.

We are also fortunate to have Grace Deyo, RN, PhD, join the practice. She and Renee Carpenter-Wells, RN, MSN, CPNP, AC/PC, who focuses on transition to adult healthcare for patients with spina bifida, have specific research goals for advancement of the care of patients with neurological disease.

Dr. Leonard has continued his focus on brain tumors and improving treatment for childhood brain tumors. He continues his research interest in exosomes and the complicated extra-cellular matrix. Lance Governale, MD, continues to focus on pediatric patients with headshape abnormalities and has written excellent reviews while providing outreach to the area pediatricians.

With our new additions, our reach has also grown. In addition to our significant increases in both surgical cases and clinic visits, we have also established an outreach clinic in Mansfield, Ohio, so that our services are located closer to the people we serve. We will now begin seeing patients twice monthly at the Mansfield Close to Home Center.

2014-2015 Grants Awarded


Center for Gene Therapy

Chicoine, Louis
Carefusion Course
Carefusion 2200
Flanigan, Kevin
Genetic modifiers of Duchenne Muscular Dystrophy
National Institute of Neurological Disorders and Stroke 
Clinical development of a U7snRNA vector for exon 2 skipping: therapy for exon 2 duplications, and for 5’ mutations in the DMD gene
First-in-human rAAVrh74.MCK.GALGT2 DMD clinical trial
National Institute of Arthritis and Musculoskeletal and Skin Diseases 
O'Neill Cure Sanfilippo Foundation
Cure Sanfilippo Foundation
Support for Myology Course
The Ohio State University
Fu, Haiyan
Treating the CNS and Somatic Diseases of MPS IIIB by Systemic Gene Delivery
National Institutes of Health
Production of Clinical Grade rAAV9-CMV-hNAGLU vector for a Phase I/II MPS IIIB Gene Therapy Clinical Trial
The Sanfilippo Research Foundation
Harper, Scott
DUX4 and the P53 pathway in FSHD pathogenesis
National Institute of Arthritis and Musculoskeletal and Skin Diseases
Developing a muscle-specific DUX4 inhibition system for FSHD therapy
Chris Carrino Foundation
PPMO Therapy for FSHD
Friends of FSH
Kaspar, Brian
IND Enabling Studies for a CNS Delivered Gene Therapy for SMA
Families of SMA
Industry Study
Industry Sponsor
A gene therapy consortium to develop and evaluate gene therapy approaches in Rett Syndrome
Rett Syndrome Research Trust
GLT1 up-regulation as a therapeutic strategy for Huntington’s disease
Indiana University
CHDI Foundation
Axons and the Extracellular Matrix in Spinal Cord Injury
Ohio State University Research Foundation
National Institutes of Health (NIH)
SMA Funds - Getty Owl
Getty Owl Foundation
Loiler, Scott
Increasing muscle specific gene delivery by reducing liver tropism of aav vectors
Ohio State University
National Institutes of Health
Martin, Paul
A new DMD model with a humanized glycome
National Institute of Arthritis and Musculoskeletal and Skin Diseases
Glycosyltransferase Therapy for Myopathies
National Institute of Arthritis and Musculoskeletal and Skin Diseases
Translational Studies of GALGT2 Gene Therapy for Duchenne Muscular Dystrophy
Department of Defense
Development of a novel HIBM2 mouse model and therapy
National Institutes of Health
A new DMD model with a humanized glycome
National Institute of Arthritis and Musculoskeletal and Skin Diseases
McCarty, Douglas
Patterns Of R Aav Vector Insertion Associated With Liver Tumors In A Mouse Model
National Cancer Institute
Mendell, Jerry
Phase I/IIa Follistatin Gene Therapy Trial for Duchenne Muscular Dystrophy
Industry Sponsor
Duchenne Alliance
Dual-vector mediated mini-dystrophin restoration of function in mdx model of DMD
National Institutes of Health
Dual-vector mediated mini-dystrophin restoration of function in mdx model of DMD
National Institutes of Health
Open-Label, Multiple-Dose, Efficacy, Safety, and Tolerability Study of study drug in Subjects with Duchenne Muscular Dystrophy
Industry Sponsor
Research Development, and Testing of Therapies and Treatment for Spinal Muscular Atrophy (Clinical Trial Phase I/II)
Industry Sponsor
Dual-vector mediated reconstitution of minidystrophin transgene and restoration of dystrophin function in mdx model of DMD
Jesse's Journey
Restoring dystrophin to the heart in mdx and porcine model of Duchenne/Becker muscular dystrophy
Jesse's Journey
Dual-vector mediated reconstitution of minidystrophin transgene and restoration of dystrophin function in mdx model of DMD
Jesse's Journey
Pediatric toxicty and efficacy in long-term systemic treatment with anti-sense: A case study of personalized medicine
Children’s Research Institute at Children’s National Medical Center
Pediatric toxicity and efficacy in long-term systemic treatment with anti-sense: A case study of personalized medicine
Childrens Research Institute at Childrens National Medical Center
Industry Study
Industry Sponsor
Overcoming Immune Barriers to Gene Correction for Duchenne Muscular Dystrophy
National Institutes of Health
Industry Study
Industry Sponsor
Montanaro, Federica
Defining the role of impaired Hedgehog signaling in DMD.
Muscular Dystrophy Association
Muscle stem cells and cancer cachexia
Ohio State University
National Institutes of Health (NIH)
Mechanisms and efficacy of myostation inhibition in myotubular myopathy
Medical College of Wisconsin
National Institutes of Health (NIH)
Rodino-Klapac, Louise
AV5.Dysferlin Gene Therapy as a Treatment for Dysferlinopathies
Jain Foundation, Inc.
AAV5.Dysferlin Gene Therapy as a Treatment for Dysferlinopathies
Jain Foundation, Inc.
Reducing skeletal muscle fibrosis in DMD with AAV-delivered miR-29
Jesse's Journey
VIP study in Dystrophic Mice II
QTest Labs
Sahenk, Zarife
Lentivirus Transduced Tolerogenic Dendritic Cells For The Treatment Of CIDP
National Institute of Neurological Disorders and Stroke (NINDS)


de los Reyes, Emily
University of Rochester Medical Center – CTCC
Food and Drug Administration
Industry Study
Industry Sponsor
Lo, Warren
CHAMP: Children with Hemiparesis hand and arm project
Ohio State University
Virginia Polytechnic Institute
The Ohio State University Regional Coordinating Stroke Center
Ohio State University
National Institutes of Health
Brain Vascular Malformation Clinical Research Network (BVMC)
University of California San Francisco
National Institutes of Health
Lowes, Linda
A Prospective Natural History Study in Sporadic Inclusion Body Myositis (sIBM)
Industry Sponsor
TMA Meeting Testing
RTI Health Solutions
Development of an innovative outcome measure to define disease progression in spinal muscular atrophy type 1 for use in the home or clinic
Cure SMA
Establishing normative data for the 100-meter walk run test
Industry Sponsor
sPFAS: Primary Data Collection Study 2
RTI Health Solutions
SMA Biomarkers in the Immediate Post-natal Period of Development
Ohio State University Office of Sponsored Programs
Muscular Dystrophy Association
Mytinger, John
Early Onset Epilepsy Consortium (EOEC)
Lurie Children’s Hospital
Pediatric Epilepsy Research Foundation
Pakalnis, Ann
Study Drug in the Prevention of Childhood Migrane
Children's Hospital Medical Center Cincinnati
National Institutes of Health
Patel, Anup
Epidiolex Dravet
Industry Sponsor
Care Management for Pediatric Epilepsy: Predictive Modeling and Evaluation
Cornell University
Pediatric Epilepsy Research Foundation
Industry Study
Industry Sponsor
Ream, Margie
A Randomized, Active-Controlled, Open-Label, Flexible-Dose Study to Assess the Safety and Tolerability of study drugs in Pediatric Subjects With New or Recent-Onset Epilepsy
Industry Sponsor
Roach, E. Steve
Network of Excellence in Neuroscience Clinical Trial Center at The Ohio State University (U10)
Ohio State University
National Institutes of Health
Twanow, Jaime
A Multicenter, Open-Label Proof-of-Concept Trial of Study Drug in Children with PCDH19 Female Pediatric Epilepsy
Industry Sponsor
Vidaurre, Jorge
Impact of Initial Therapy and Response on Long Term Outcome in Children with CAE
Children's Hospital Medical Center Cincinnati
National Institutes of Health (NIH)

Neurosciences Faculty

E. Steve Roach, MD

Jeffrey R. Leonard, MD

View Our Faculty »

Selected Neurosciences Publications


Chung MG, Lo WD. Noninvasive brain stimulation: the potential for use in the rehabilitation of pediatric acquired brain injury. Archives of Physical Medicine and Rehabilitation. 2015 Apr;96(4 Suppl):S129-37.

Heyer GL. Abdominal and lower-extremity compression decreases symptoms of postural tachycardia syndrome in youth during tilt table testing. Journal of Pediatrics. 2014 Aug;165(2):395-7.

Lo W, Gordon A, Hajek C, Gomes A, Greenham M, Perkins E, Zumberge N, Anderson V, Yeates KO, Mackay MT. Social competence following neonatal and childhood stroke. International Journal of Stroke. 2014 Dec;9(8):1037-44.

Mytinger JR, Weber A, Heyer GL. The response to ACTH is determined early in the treatment of infantile spasms. Epileptic Disorders. 2015 Mar;17(1):52-7.

Rose SC, Weber KD, Collen JB, Heyer GL. The Diagnosis and Management of Concussion in Children and Adolescents. Pediatric Neurology. 2015 Apr. [Epub ahead of print]



Chen YH, McGowan LD, Cimino PJ, Dahiya S, Leonard JR, Lee da Y, Gutmann DH. Mouse low-grade gliomas contain cancer stem cells with unique molecular and functional properties. Cell Reports. 2015 Mar;10(11):1899-912.

Governale LS. Minimally invasive pediatric neurosurgery. Pediatric Neurology. 2015 Apr;52(4):389-97.

Haydon DH, Dahiya S, Smyth MD, Limbrick DD, Leonard JR. Greater extent of resection improves ganglioglioma recurrence-free survival in children: a volumetric analysis. Neurosurgery. 2014 Jul;75(1):37-42.

Qualls D, Leonard JR, Keller M, Pineda J, Leonard JC. Utility of magnetic resonance imaging in diagnosing cervical spine injury in children with severe traumatic brain injury. The Journal of Trauma and Acute Care Surgery. 2015 Jun;78(6):1122-8.

Sribnick EA, Hanfelt JJ, Dhall SS. A clinical scale to communicate surgical urgency for traumatic brain injury: a preliminary study. Surgical Neurology International. 2015 Jan 5;6:1.

View Team Publications »

The Center for Gene Therapy

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