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From
the physician-in-chief
Texas Children’s Hospital
Therapeutics and Research Institute for Neurologic Disorders
By Ralph D. Feigin, M.D.
For the past nine months, many individuals working at Texas
Children’s Hospital have been involved in assessing the current
status of our institution and planning for additional
programmatic and facility growth and development in the future.
One of the principal areas of potential growth, which we plan to
target, is the entire field of neurologic disorders in infants
and children.
In 2001, the World Health Organization estimated that up to 400
million people worldwide suffer from mental or neurologic
disorders. The Children’s Neurologic Solutions Foundation
reports that 14 million Americans under the age of 19 have
experienced disabling neurologic disorders. Two percent of the
population suffers from mental retardation. One out of every
1,000 children has a language disorder. Autism afflicts as many
as one in every 166 children. More children in the United States
are diagnosed with autistic spectrum disorders yearly than with
diabetes mellitus or with cancer.
At Texas Children’s Hospital, our pediatric neurologists and
neurosurgeons see more than 16,000 children annually. Over a
period of the last half century, we have developed many
clinical, educational and research programs focused on children
with neurologic disease and handicapping conditions.
Nevertheless, we believe the time is appropriate to attempt to
make more rapid strides to improve our understanding of these
disorders and to offer improved care. With this goal in mind,
the hospital has chosen to establish a Therapeutics and Research
Institute for Neurologic Disorders. Mr. Anthony Petrello, a
member of the Board of Trustees of Texas Children’s Hospital,
should be commended for his role in urging the hospital to make
rapid strides in an innovative way in this particular area of
great need. Mr. Petrello and his wife, Cynthia, have personally
made a $5 million commitment to these endeavors and have agreed
to lead a fund-raising effort to support both the program and
facility that such an effort would require.
In our opinion, pharmaceutical companies have not focused
sufficient resources on these disorders. In particular, no one
has addressed pediatric neurologic disorders in the way that we
are proposing and, thus, the new institute will indeed be a
groundbreaking effort. Recent advances have increased our
understanding of the developing brain. The plasticity of stem
cells have opened up the possibility that damaged nerve cells
may even be replaced. Genetic therapies have proven to be
effective in treating the molecular basis of selected diseases
of the liver and blood, and we believe the same potential exists
for neurologic disorders. There also have been dramatic advances
in neurologic imaging capabilities that can be brought to bear
on this effort.
Texas Children’s Hospital and its partner, Baylor College of
Medicine, have been a focus for research advances, with
particular striking advances emanating from the laboratories of
Dr. Huda Zoghbi and from the Cain Pediatric Neurology Foundation
Research laboratories directed by Dr. John Swann. Texas
Children’s is home to more than 23 pediatric neurologists and is
the geographic location of the Blue Bird Clinic for Pediatric
Neurologic Disorders, a clinic which specializes in the
diagnosis, treatment and follow-up care of neurologic disorders
in children. Texas Children’s also has established preschool
programs for children with autistic spectrum disorders (the
Bridges program) and, in collaboration with citizens in this
community, the Rise School for children with Down’s syndrome and
related disorders. The hospital also has been home to the Meyer
Center for Developmental Pediatrics for more than 40 years.
Faculty in the Department of Pediatrics at Baylor College of
Medicine and Texas Children’s have been at the forefront of
identifying genes that cause neurologic disorders and have
described the functional characteristics of those genes.
Examples of genes identified on our campus include: Fragile X
syndrome; Rett syndrome; Angleman’s syndrome; Charcot-Marie-Tooth
disease; Prader-Willi syndrome; spinocerebellar ataxia; and
lissencephaly, a brain malformation.
Opportunities for collaboration
The new institute that we are developing will be similar to
large centers at Texas Children’s, such as the Texas Children’s
Cancer Center and the Texas Children’s Heart Center, which are
consistently ranked among the best in the nation.
The Human Genome Sequencing Center at Baylor College of Medicine
is engaged actively in sequencing many genes, having completed
its participation in the sequencing of the human genome. The
Therapeutics and Research Institute for Pediatric Neurologic
Disorders will enjoy abundant opportunities for collaboration
and partnership with this center.
Neighbors such as Rice University and the University of Houston
enable the newly proposed institute to be in close proximity to
outstanding potential partners in computational biology, physics
and imaging modalities. The Center for Cell and Gene Therapy at
Baylor and Texas Children’s also provides an infrastructure to
rapidly move novel gene and cell therapy protocols from the
laboratory to the clinic. Texas Children’s is home also to the
largest pediatric stem cell transplant unit in the Southwest.
This laboratory provides processing capabilities for patient
and/or donor components and an environment that allows the
integration of progenitor and stem cell component preparation
for transplantation and adaptive immunotherapy and tumor
vaccines.
All of us look forward to the development of this special
institute for the research and therapy of pediatric neurologic
disorders in an innovative way. We thank and applaud the Petrellos for taking a leadership role in helping to bring this
effort to fruition.
Ralph D. Feigin, M.D., is physician-in-chief at Texas
Children’s Hospital and professor and chairman of the Department
of Pediatrics at Baylor College of Medicine.
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