The Johns Hopkins. the consequences of brain injuries from stroke, traumatic brain injury, or infection.

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1 The nervous system (consisting of the brain, spinal cord, and peripheral nerves) is dauntingly complex and, unfortunately, susceptible to many devastating disorders. Some neurologic diseases and disorders arise from genetic mutations, for example epilepsy that runs in a family, but often neurological conditions appear sporadically, or out of the blue. Some neurologic diseases and disorders worsen steadily over time, while others are episodic; some, such as Alzheimer s or Parkinson s disease, are household words, whereas others are rare, causing a further sense of isolation and despair. All neurologic diseases and disorders, however, profoundly affect the lives of patients, their families, and other loved ones. 1

2 rising to the challenge A Call to Action The impact of neurologic conditions is far-reaching Rising to the Challenge: The Campaign for learning and social opportunities, and More than 10 million Americans suffer from neuro-degenerative and related neurologic diseases including multiple sclerosis (MS) and Huntington Disease, Lou Gehrig s Disease (ALS), ataxias, Parkinson s disease and Parkinsonian syndromes, and various conditions that lead to dementia. One out of every 150 children in the United States today carries a diagnosis of autism; recent data suggest that this number may be as high as one out of every 88 children. Each year approximately 795,000 strokes are reported in this country one every 40 seconds. Nearly 20 million Americans endure peripheral neuropathy, and half a million have MS, the most common cause of disability in individuals under the age of 45. new programs that will enhance student- The list goes on faculty interactions, ensure diversity on The incidence of neurologic diseases is increasing. According to the World Health Organization, by the year 2020 mental and neurological disorders will account for 14% of the global burden of disease. In developed countries, this increase is fueled by demographic trends, especially population aging. By 2030, approximately one in five Americans will be over the age of 65, and more than 12 million will suffer from age-related neurodegenerative diseases including Alzheimer s, Parkinson s, and peripheral neuropathies. Many more will be living with the consequences of brain injuries from stroke, traumatic brain injury, or infection. Johns Hopkins will raise unprecedented levels of support to attract, sustain, and further empower the people of Johns Hopkins our students, faculty, and researchers who through their work improve the lives of millions around the world. Together with our philanthropic partners we will: Advance Discovery and Creativity through support of our exceptional faculty and researchers. Their innovative work drives the development of new knowledge, new forms of expression, and new ways to save lives and improve health, and furthers progress across our core disciplines in science and technology, the humanities and arts, and public health and medicine. Enrich the Student Experience by investing in scholarships and fellowships, inspirational spaces for collaborative campus, link learning in the classroom to life after graduation, and strengthen connections between our students and our surrounding communities. Solve Global Problems as One University by creating new cross- disciplinary solutions in crucial areas such as sustaining global water resources, revitalizing America s cities, advancing individualized health, understanding how we learn and teach, and attacking the root causes of global health problems. Department of Neurology is committed to playing a key role in the success of the campaign. Please join with us in this important mission. 2 Johns Hopkins Neurology The Department of Neurology at Johns Hopkins is recognized around the world for excellence in clinical care and research. Each year, we provide in excess of 30,000 outpatient consultations for pediatric and adult patients. The Department has grown rapidly since its founding in Our faculty now includes more than 140 neurological clinician-investigators and scientists engaged in disease-oriented discovery research, and we continue to expand our labs and facilities. A central focus of the Department is improving the treatments available for neurologic diseases. There are currently no curative therapies to offer children with autism; instead, many clinical interventions focus on controlling symptoms such as hyperactivity. There is, as yet, no cure for Parkinson s disease. For many other neurologic conditions, better treatments are desperately needed but there is hope. Novel advances in our research laboratories are starting to pay off. In recent discoveries, we have identified new ways of stimulating the plasticity of the brain and nervous system; this progress is allowing us to more effectively treat patients with stroke, traumatic brain and spine injury, and MS. Johns Hopkins Neurology is poised to make many similarly transformational advances that will improve the diagnosis 1

3 rising to the challenge and treatment of neurological conditions. Our motivation is strong because, every day, we care for the very patients whose illnesses we tackle through research, in pursuit of a cure. Repair and regeneration of the nervous system after injury Until recently, widespread belief held that the brain and nerves could not repair themselves, that these cells and tissues, when damaged, permanently lost their capacity for normal function. This belief is being overturned. Our research seeks to capitalize on early advances, to develop new and more effective methods for protecting and promoting repair of the injured nervous system. Ultimately, these methods will enable patients to recover more completely their critical neurological functions, including those governing movement, cognition, and balance. In collaboration with the Johns Hopkins Institute for Cell Engineering, neurology research teams are unlocking the mysteries of neuronal cell death and survival, cell fate specification, embryonic and adult stem cells, synapse formation, neuronal development, gene expression, and the molecular biology of Parkinson s disease, stroke, and vision. Disease targets include MS, stroke, traumatic brain injury, spinal cord injury, and neuropathic pain. Future treatments, just now envisioned, will coax healthy cells in the brain and nervous system to repair and replace injured or damaged ones. Personalized Neurological Medicine Personalized Medicine is healthcare that, using a full array of individual health information, customizes care to each patient. Central to Personalized Medicine is an understanding of the individual s genetic profile, which helps identify the person s unique health risks and predict his or her likely response to treatments. Other complementary methods include describing the individual s proteome (the set of proteins which are produced based on the genome, and which implement geneencoded instructions) and metabolome (the unique chemical fingerprint resulting from specific cellular processes). A new program in Personalized Neurological Medicine will provide a platform for studies of genetic variation across many different neurological disorders. This program will amplify current collaborations between the Department and the McKusick-Nathans Institute of Genetic Medicine at Johns Hopkins to describe genetic variability between individuals, identify individual variations in disease susceptibility, and to predict drug response based on individual characteristics. Exquisitely fine-tuned knowledge will enable neurologists to tailor treatment to the individual patient, so that the care that each person receives has the greatest like lihood of success. Prevention and prediction of neurological disease For many neurological diseases and disorders, early detection and intervention is the key to minimizing the condition s impact. In the future, we will be able to detect an individual s risk before the disease or disorder appears, and to intervene with an effective preventive measure. To gain this predictive ability, our faculty are working to develop chemical tags called biomarkers (examples include specific molecules or genes that can be identified and tracked in blood or spinal fluid) and imaging techniques to assess brain function. In clinical practice, biomarkers in conjunction with imaging can be used to predict, diagnose, and track neurological disorders, and to better test the impact of new therapies. Research in the Department encompasses developing new strategies for disease prevention, which can be applied to protect patients once they have been identified as at risk. Sophisticated imaging technologies, such as functional magnetic resonance imaging (fmri), offer powerful tools for observing and improving our understanding of neurological conditions and open new horizons for diagnosis and management of neurological disease. Drug discovery and clinical trials: resources for neurological therapies The process of developing a new pharmaceutical treatment for a neurological condition is demanding and intensive. It requires a constellation of ingredients: brilliant scientists who pool their ideas and efforts, state-ofthe-art facilities, staff and resources to support research, an environment conducive to creative thinking and intellectual freedom, time, dedication, and hard work. The Department of Neurology seeks to create this optimal environment for research to leverage the resources of the NeuroTranslation Program of the Brain Science Institute at Johns Hopkins Medicine, leading to novel neurological therapeutics. Designed to expedite the trajectory from basic discovery to new treatment and better patient outcomes, our research program accelerates preclinical testing, and links the results of these laboratory studies to efficient testing of new drugs in clinical trials involving patients. Educational opportunities that prepare the next generation of neurologists The future of Neurology depends on next generations of talented individuals; it merits careful planning. We now know that the brain continuously creates and recreates itself based on the inputs it receives, and the process of strengthening certain capacities while winnowing others occurs at a rapid pace during youth. In young people, cultural and social norms also conspire to support or discourage an inquiring mind. Unless we provide the right inputs and context, we will never know which student might evolve into a brilliant scientist. To develop a pipeline of highpotential youth who can advance the state of the science in Neurology, the Department intends to create a continuum of programs that offer motivated students a pathway into scientific careers. This continuum begins with science education at the high school and college levels, to attract The Department has grown rapidly since its founding in Our faculty now includes more than 140 neurological clinician-investigators and scientists. 2 3

4 rising to the challenge THE JOHNS HOPKINS Department of neurology future clinician-scientists into Neurology, and then sustains scientific growth through new mentoring and funding mechanisms for junior physicianscientists, to help them develop highly productive careers in Neurology. Why Johns Hopkins? We are the #1 ranked Neurology program at the hospital ranked #1 in the United States for 21 consecutive years. Within this setting of excellence, the Johns Hopkins Department of Neurology continues to improve clinical care, research and training. Our mortality rates are among the lowest in the world. Our patient care is unsurpassed. We commit ourselves to providing excellent patient care through individualized treatment. Our 38 clinical programs address a broad range of neurological diseases, creating a spectrum of specialized, disease-specific centers. Although our expertise is focused, the care we provide is comprehensive, typically pulling together faculty from diverse disciplines to best address the complexity of each patient s condition. Our closest collaboration is with the Department of Neurosurgery; Neurology and Neurosurgery faculty work together every day to provide coordinated medical and surgical services, so our patients experience seamless care. We understand the power of teamwork. In Neurology, we place great value on working together. We know that teamwork is the most effective way to spark insights, and to move visions into reality. By tapping each other s expertise, and blending laboratory-based and clinical research, we accelerate both discovery and its translation into new ways to benefit patients. To provide the best possible patient care, the Neurology team draws upon the outstanding resources at Hopkins in anesthesiology and critical care medicine, physical medicine and rehabilitation, psychiatry, geriatrics, and pulmonary medicine. We have a history of pushing the leading edge of research and treatment. Based on studies in the 1980s and 90s of disease mechanisms in neurological disorders, our team quickly became leaders in neurologic research. Since then, we have maintained a steady record of scientific contribution. We were first to open a neurologic critical care unit, and to pioneer other clinical advances such as: plasma pheresis for treating Guillain- Barré Syndrome and other neuroimmune nerve disorders; hemispherectomy and ketogenic diet for refractory seizures in children; cerebral spinal fluid drainage, to select candidates with normal pressure hydroceophalus for shunting procedures; new therapeutic agents for MS, including nataluzimab, fingolimod, and high-dose cyclophosphamide; imaging techniques for identifying individuals at high risk for progression to Alzheimer s Disease, and who might benefit from early intervention, and; telemedicine for treating chronic diseases such as Parkinson s. This is a time of rapid progress. Research conducted in the Department of Neurology is yielding progress on myriad fronts, from Alzheimer s Disease and autism to neurological cancers and stroke. We are at the forefront of revolutionary changes in how we diagnose and treat neurological diseases and brain or nervous system injuries. Now is an optimal time for investment the field of Neurology is ripe for scientific breakthroughs, eading to vastly better treatments. Philanthropy plays a vital role Philanthropy accelerates scientific discovery and advances clinical breakthroughs. With its inherent flexibility, philan-thropy supports innovation and discovery in ways that no other type of funding can: it pushes forward the most novel ideas from their initial concept to new understanding, new approaches to neurological conditions, and, ultimately, new treatments leading to improved results for patients and families. Our strategic priorities highlight areas of research that are poised for discovery. Their broad scope allows multiple opportunities for our donors, doctors, and scientists to dream big and then through determination, collaboration, hard work, and genius to make those dreams a reality. Philanthropic funding is the optimal type of support. It enables clinicianresearchers to move nimbly and creatively, to rapidly advance the science, and to bring new discoveries into clinical practice. Ultimately, it is our most efficient and effective way to help the millions of people affected by neurologic diseases and disorders. WHAT WILL IT TAKE? Priority Specific Funding needs Current Use Endowment Funds needed Nervous System Injury: Repair and Regeneration Personalized Neurological Medicine Prevention and Prediction of Neurological Disease Drug Discovery and Clinical Trials The Next Generation of Neurology Equipment $2,500,000 $2,500,000 New Research Space $1,000,000 $1,000,000 Imaging Equipment and Capital Resources $2,500,000 $2,500,000 Education and Training $1,500,000 $3,000,000 $4,500,000 Capital Equipment and Support $3,000,000 $3,000,000 Faculty Support $500,000 $2,000,000 $2,500,000 Education and Training $500,000 $500,000 High School Program $500,000 $1,000,000 $1,500,000 Total $125,000,000 $35,000,000 $160,000,

5 Rising to the Challenge: The Campaign for Johns Hopkins Department of Neurology 100 North Charles Street, 4th Floor Baltimore, Maryland hopkinsmedicine.org/neuro rising.jhu.edu rev. 03/03/14 6

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