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The goal of this Request for Applications (RFA) is to accelerate the translation of gene transfer methodologies into the clinic. Gene therapy holds tremendous promise for the treatment of neurological disorders. Despite recent advances in this area however, specific scientific, technological, and safety goals must be achieved before gene transfer becomes a viable therapeutic alternative. Current needs include more effective methods for controlling the expression of therapeutic transgenes in the brain, better strategies for vector delivery and monitoring transgene expression, more studies addressing the toxicity of specific vector/transgene combinations, and greater knowledge of the long term effects of expressing specific transgenes in the nervous system. This RFA is intended to encourage research projects that address these or related translational issues in the context of specific neurological disorders.

Deep brain stimulation (DBS) in the thalamus for the treatment of tremor was approved by the FDA in 1997 following the pioneering work of Benabid and colleagues in France. Subsequently it has been found that stimulation in the subthalamic nucleus (STN) or globus pallidus (GPi) can relieve multiple symptoms of Parkinson"s disease (PD). DBS has been successful in restoring significant mobility to a large number of patients with advanced PD. GPi and STN stimulation are currently being considered for use to decrease symptoms of dyskinesia, tremor, postural instability, and/or bradykinesia in individuals with advanced PD.

The Neurodegeneration and Clinical Trial Groups of the National Institute of Neurological Disorders and Stroke (NINDS) request applications for clinical centers to collaborate in the performance of a large, double-blind randomized trial of two or more potential neuroprotective agents in patients early in the course of Parkinson’s disease. Applications for coordinating and statistical centers were requested in a separate solicitation (http://grants.nih.gov/grants/guide/rfa-files/RFA-NS-01-012.html). The trial was called for in the NIH Parkinson Research Agenda (http://www.ninds.nih.gov/about_ninds/nihparkinsons_agenda.htm). The neuroprotectants to be tested in the trial have not yet been chosen and will be selected by an NINDS-appointed Oversight Committee from among those proposed by the applicants who respond to this Request for Applications (RFA) as well as from those suggested by others, including NINDS grantees, pharmaceutical companies, patients, and patient advocates.

The National Institute of Nursing Research and co-sponsoring Institutes and Centers encourage research proposals in the management of chronic pain across the lifespan. Pain is a subjective experience influenced by gender, age, race/ethnicity and psychosocial factors. The management of pain is influenced by patient, health care provider and system factors. Research is needed to determine the most effective interventions to remove barriers to effective treatment, to determine the most effective pharmacological and non- pharmacological therapies including complementary and alternative therapies, to identify assessment tools for patients unable to verbalize their pain, and to identify effective pain management strategies for individuals with disabilities and underserved populations.

The National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK), the National Eye Institute (NEI), the National Heart, Lung and Blood Institute (NHLBI), the National Institute on Aging (NIA), the National Institute of Neurological Disorders and Stroke (NINDS) and the Office of Dietary Supplements (ODS) seek basic and clinical research applications to study the use of vitamin E and other antioxidants in the prevention or amelioration of diabetic complications.

The National Institute of Neurological Disorders and Stroke (NINDS) is interested in promoting collaborations among junior investigators (Postdoctoral Fellows through Assistant Professors, or equivalent) to stimulate translational research in the field of epilepsy. To this end the NINDS invites exploratory/developmental research grant applications (R21) in patient- oriented research, developmental neurobiology, genetics, advanced technology, imaging, pharmacotherapeutics, or other research areas, which are likely to lead to the cure of epilepsy (defined as "the prevention of epilepsy before it occurs in people at risk, and the cessation of seizures without therapy-associated side effects in those who develop the disease"). Emphasis will be placed on cross- disciplinary collaborations, novel hypotheses, and unique approaches in applying fundamental neurobiological concepts to epilepsy research. Special consideration will be given to proposals that enhance the application of scientific knowledge to the understanding and treatment of the disorder. This initiative requires collaborations of two or more junior investigators at different institutions, or in different laboratories within the same institution. Investigators already working together at the same department are not eligible.

The National Institutes of Health Autism Coordinating Committee (NIH/ACC) is implementing the aspects of the Children"s Health Act of 2000 that relate to support of autism research by NIH. The NIH/ACC is composed of the NIH institutes currently funding autism research: NIMH, NICHD, NINDS, NIDCD, and NIEHS. An important aspect of these activities is the establishment of Centers of Excellence in Autism Research, and in this RFA the participating institutes invite research grant applications for such Centers. These Centers will constitute a cohesive program, operating under an NIH cooperative agreement, which will be called the STAART Centers Program (Studies to Advance Autism Research and Treatment).

This Program Announcement (PA) solicits applications for investigator- initiated research related to the prevention and management of cachexia to improve the quality of life for these patients. Cachexia is a condition of severe malnutrition characterized by anorexia, weight loss and muscle wasting that occurs as a consequence of chronic conditions such as cystic fibrosis, cerebral palsy, cancer, AIDS, congestive heart failure, failure to thrive in older populations, end-stage organ failure, neurological degenerative diseases, chronic obstructive lung disease, chronic liver disease, and chronic renal disease. Cachexia has repeatedly been associated with adverse clinical outcomes, and increased morbidity and mortality. Research findings are reported in the literature which address individual symptoms and speculated causes of cachexia but data are not available to provide a scientific base for a multidisciplinary approach to prevent cachexia and manage the associated symptoms to improve the quality of life for patients suffering from cachexia. The goal of this PA is threefold: 1) to stimulate basic and clinical research in cachexia, 2) to examine cachexia in relation to several related symptoms to improve quality of life, and 3) to examine cachexia symptoms in two or more chronic conditions.

The National Institutes of Neurological Disorders and Stroke (NINDS) will award National Research Service Award (NRSA) Short-Term Institutional Training Grants (T35) to eligible institutions to provide research training opportunities for medical students who are preparing for careers in basic or clinical neurological sciences research. The purpose of this program is to help ensure that a highly trained workforce is available to assume leadership roles related to the Nation"s neurological sciences research agenda. The goals of the program are to encourage the interest of potential clinician scientists early in their education, and accelerate entry to independent research.

The National Institutes of Neurological Disorders and Stroke (NINDS) will award individual fellowships (F31) to eligible individuals to provide research training opportunities for medical students who are preparing for careers in basic or clinical neurological sciences research. The purpose of this program is to help ensure that a highly trained workforce is available to assume leadership roles related to the Nation"s neurological sciences research agenda. The goals of the program are to encourage the interest of potential clinician scientists early in their education, and accelerate entry to independent research.