Epilepsy, stroke. Alzheimer's, traumatic brain injury, autism.
As different as they are, all neurological disorders have one thing in common: they strike at the core of our identity. They are also shockingly prevalent as a group: a 2007 report from the World Health Organization estimated that one billion people around the world suffer from some form of neurological disease.
Less than a generation ago there was little hope for treating most of these disorders, but we are now beginning to appreciate the brain's resilience and its capacity for renewal. We are acquiring new tools to illuminate neurological function and dysfunction; more scientists and clinicians are studying these disorders than ever before. We are, in short, on the cusp of a new era in biomedicine.
To meet this moment of opportunity, Texas Children's Hospital (TCH) and Baylor College of Medicine (BCM) conceived the Jan and Dan Duncan Neurological Research Institute (NRI). Building on our considerable strengths in genetics, neuroscience, and pediatrics, NRI is carefully designed to foster collaboration among 30-40 basic faculty and clinician-scientists with the goal of developing treatments for childhood neurological diseases.
The Gordon and Mary Cain Pediatric Neurology Research Foundation funds epilepsy research programs at the Texas Children's Hospital in Houston, Texas.The labs are located within the Jan and Dan Duncan Neurological Research Institute (NRI) building. In 2010, extensive preclinical cellular and animal studies by Cain lab researchers culminated in the identification of a rapamycin analog, everolimus, as a potential therapy against severe epilepsy due to brain malformations in Tuberous Sclerosis Complex.
In a small FDA-approved clinical trial conducted at the Texas Children’s and Cincinnati Children’s Hospitals, it was demonstrated that most of the severely epileptic children treated with everolimus had significantly fewer seizures. Encouraged by those positive results, a much larger international clinical trial is now underway. Texas Children’s Hospital and Baylor College of Medicine are major sites for this trial.
The vision of the leaders at the Jan and Dan Duncan Neurological Research (NRI) Institute is to foster a multi-disciplinary, collaborative environment that will faciliate fundamental discoveries in neuroscience with the ultimate goal of translating them to therapies for many incurable neurological and neurodegenerative conditions.
Several features of NRI catalyze progress in translational research:
The Texas Medical Center (TMC) is the largest in the world, and its institutions treat about 6 million patients each year. The 344,000 square foot silver-level LEED-certified NRI building, nestled in the heart of the TMC, relocates Baylor College of Medicine and Texas Children's Hospital researchers working on neurodevelopment and neurological diseases under one roof and provides space for us to recruit investigators with complementary areas of expertise.
The Jan and Dan Duncan Neurological Research Institute also physically links Baylor College of Medicine and Texas Children's Hospital to M.D. Anderson Cancer Center, where University of Texas collaborators lend strength in developmental biology and epigenetics. Most importantly, NRI's location adjacent to the hospitals fosters relationships between the lab and the clinic, which are integral to the translational research enterprise.
Multi-disciplinary approach: exploring common themes in different areas of expertise
The best way to break the grip of a particular discipline on the way a question is framed and answered is to concentrate on themes of importance to several disciplines. A number of NRI-sponsored programs such as monthly disease-specific dinner seminars bring together faculty who might otherwise never encounter one another, which often allows them to discover an unexpected commonality in their respective diseases of interest. These events form the hubs of a dynamic intellectual network in which ongoing conversations serve as mutual catalysts for creativity. We can't predict where the next insight will come from; we just know the insights will come.
Robust core services
At a time when too many medical schools are reducing their investment in basic research infrastructure, the NRI is enriching our environment by providing core labs to specifically support translational neuroscience research. Expertise in creating engineered mice, RNA in situ, neuropathology, slice and in vivo neurophysiology, neuroimaging, animal EEG, mouse behavior, bioinformatics, microscopy (confocal, two-photon and electron), and pharmacology/drug trial design are available to all NRI labs.
Researchers at BCM and TCH, with the support of six NIH institutes, the Howard Hughes Medical Institute, and several disease foundations, have long been at the forefront of understanding numerous neurological and developmental disorders. We discovered or co-discovered the genetic basis of Fragile X (A and E) syndromes, Angelman syndrome, Rett syndrome, myotonic dystrophy, Goltz syndrome, incontinentia pigmenti, several inherited ataxias, several inherited neuropathies, and numerous other neurological and neuropsychiatric syndromes. We have created animal models for many of these disorders, and we are already using some of them to develop therapeutic interventions.
The NRI simply makes the work faster, easier, and a lot more fun.