Neurological Medical Research at GUMC, where Glimmers of Hope Become Beacons of Possibility

Alzheimer’s and Parkinson’s diseases, stroke, and traumatic brain injury are neurodegenerative diseases and neurotraumas, medical conditions with sometimes unclear origins and no known cures.

Patients appear lost within themselves, unreachable in their own lives. Deterioration envelops them. Most do not exhibit their symptoms until they already are hostage to the disease.

The prevalence and cost to society of these age-related abductors of normalcy are soaring as the nation’s population ages. More than 5 million Americans suffer from Alzheimer’s. About 1.5 million are victims of Parkinson’s, a number that is expected to double to within 20 years. Every 40 seconds, someone in the US suffers a stroke. Most are 50 and older.

Aging is the biggest factor in Alzheimer’s, Parkinson’s, and stroke.

On the other end of life’s spectrum, traumatic brain injury is often a tragedy of youth and sudden violence. Wars, sports injuries, and vehicle crashes provide a steady stream of patients, many of them young people aged 18 to 25.

The need for therapies and cures is urgent. What limited treatments are available suppress symptoms or make sufferers more comfortable. Yet they are used only after the disorder, deep-rooted and full-fledged, already has done significant damage.

Research-driven and clinically active, Georgetown University Medical Center (GUMC), embedded in the Jesuit university in the nation’s capital where it enjoys unique and collegial access to ethical, policy, legal, and regulatory expertise, is ready to respond.

Led by renowned neuroscientist and physician Howard Federoff, executive vice president for health sciences, GUMC’s strategy is to create a neurological institute, shored up by research and clinically based centers, which takes full advantage of research strengths, attracts extraordinary faculty and students and provides the infrastructure and resources needed to excel.

Envisioned as a collection of centers of research, clinical trials, and academic excellence designed to promote collaboration among researchers and clinicians, the focus will be on Federoff’s guiding vision of systems medicine – a whole body, environment, and gene inclusive, patient-specific approach to prevent or identify and treat human diseases.

"There is great potential for Georgetown to be a leader in biomedical sciences, not just locally, but nationally and internationally, and there is a remarkably fertile foundation to build upon here," Federoff says. "Investigators at GUMC have made truly outstanding contributions that have helped to shape the manner in which medicine is taught and clinical care is delivered."

Researchers at GUMC have made some remarkable neurological discoveries that are transforming glimmers of hope into beacons of possibility. Georgetown scientists here have found:

• The key to repairing the cell damage that causes profound alteration in patients with AD, PD and TBI – as well as with spinal cord injuries and Multiple Sclerosis -- lies in the similarities in how cells live or die in these diseases.
• Blood tests can show patients’ sensitivities to these diseases, an early warning to enable physicians to reduce the damage before it spreads.
• Some drugs for AD also reduce the harm done by TBI in animals and so could stem the long-term and continuing decline that usually follows these injuries.
• The interplay of the patient’s genes and the environment cause most cases of AD and PD, making these diseases particularly suited to GUMC’s systems approach to medicine.

GUMC’s partnership with MedStar Georgetown University Hospital, also known for its commitment to and clinical expertise in neuromedicine, completes a powerful bench to bedside translational research curve in which researchers and physicians can crack the code of how the brain develops and functions normally and how to intervene, prevent, and fix disease or trauma damage when it does not.

The Department of Neurology has four renewed National Institute of Health training grants, the highest number of neurology training grants of any medical university in the US. Already underway at GUMC are the first-ever neurosurgical and gene therapy focused Alzheimer’s clinical trials in the nation.

Existing and future resource facilities, large-scale computational capacities, genomics/proteomics proficiencies, drug discovery competencies, ongoing clinical trials, and extraordinary scientific research and scientists combine uniquely to enhance GUMC’s demonstrated potential to break-through medical barriers such as those currently blocking brain disease and disorder cures and therapies.

As part of Georgetown University, research into brain diseases and disorders is edified by a broader intellectual, academic, and action framework that imbues research, education, and patient care with policy, ethics, and legal considerations, guided by Jesuit principals of service to others and care for the whole person, or, curas personalis.

And, as the nation tackles the confounding problems of crafting a national healthcare policy, GUMC’s proximity to and relationships with lawmakers and federal and elected officials enables our experts to participate boldly in the medical community that informs the shaping of regulatory policies.

The genesis of these diseases and disorders is still largely uncharted territory. Their genetic complexities, and similar characteristics appear to hold the promise of synergistic scientific breakthroughs. Shared core resources and essential collaboration and cross talk among physicians and scientists will create radical, intellectual synergies to advance against these diseases, which share genetic complexity, characteristics, and staggering human costs.

GUMC can galvanize important research findings, empower scientists by allowing long-term research, shored up by state-of-art technology and collaborative access to other researchers and clinicians, and speed translation of advances into therapies and cures for aging generations suffering from neurodegenerative diseases and younger generations hobbled by devastating neurotraumas.

"Medicine will be most effective when the focus of health care changes from treatment of the symptoms of disease to holistic prevention of it," Federoff says. "Instead of being reactive – waiting for sick patients to walk through the clinic door – physicians will be proactive, advising and treating the healthy so that they stay that way."

Victoria Churchville, GUMC Science Writer