A Blue Sky Vision for the Future of Neuroscience: Online Request for Information

As an initial step in its strategic planning process, the NINDS sought to develop a "Blue Sky" vision for the future of neuroscience. Between May 22, 2007 and October 14, 2007, an online Request for Information (RFI) encouraged academic and industrial neuroscience researchers, clinicians, patient groups, and other members of the public with an interest in neuroscience research to provide input into this vision. In particular, the RFI asked about anticipated advances in neuroscience and neurology, infrastructural and technological needs, challenges and research questions that if addressed could revolutionize understanding of the nervous system and prevention and treatment of neurological disorders, as well as emerging ethical, legal and social issues associated with neuroscience research. In addition, the RFI solicited recommendations on the role NINDS should play in neuroscience research and how it could best collaborate with other members of the research community, including other NIH Institutes and Federal agencies, the pharmaceutical and biotechnology industries, academic and private research institutions, and non-profit organizations such as patient advocacy groups and professional societies. (The full text of the questions posed in the RFI appears at the end of this document.)

A total of 124 responses were submitted to the RFI, including 41 from academic researchers, 24 from clinicians, 23 from patients and caregivers, 18 from student or postdoctoral researchers, 5 from advocacy or professional organizations, and 13 from individuals who did not report an affiliation. The respondents touched on a broad range of topics, and many of their comments were relevant across more than one of the questions asked. Therefore, in order to best capture the variety of input received, the responses were summarized and grouped into the following categories: basic science research priority areas; disease-related and clinical research priority areas; clinical practice goals and priorities; promising translational and therapeutic approaches; resource, infrastructure and technological needs; research and professional training needs; public education areas; process or policy recommendations; and ethical, legal and social issues. The lists below are ordered according to how commonly the different types of responses appeared, and an asterisk indicates topics with more than 5 similar responses. The four planning modules received this summary of the RFI input, and original responses in different categories or topics were available upon request.

Basic science research priority areas

Brain mapping and connectivity, using imaging, EEG/MEG localization *
Stem cells, neural stem cells *
Learning and memory *
Effects of stress, environmental influences; gene-environment interactions; epigenetics *
Neural activity patterns, including those underlying perception, cognition, complex and natural behaviors *
Plasticity and neuromodulatory mechanisms, intrinsic repair mechanisms *
Integrated view of neurobiology; interaction with body; systems biology approach; multidisciplinary research *
Non-neuronal cell types (glia, vascular, immune, epithelial)
Pain (basic mechanisms)
Gender differences; endocrine, hormone effects
Gene expression regulation in the brain; targeted genetic manipulation; siRNA
Nervous system development
Nervous system aging
Mitochondria
Primate evolution
Placebo effect
Basis of religious belief
Neural basis of personality and behavioral differences
Postsynaptic function and structure; cell-cell communication

Disease-related and clinical research priority areas

Headache/migraine and chronic pain disorders *
Alzheimer's disease, Parkinson's disease, frontotemporal dementia, triplet repeat disorders, other
neurodegenerative and age-related disorders and dementias *
Stroke *
Multiple sclerosis *
Psychiatric and mood/affective disorders (depression, PTSD, ADHD); role of stress, reasons for prevalence, as comorbid conditions *
Genetic susceptibility to disease; large-scale genetic and epidemiological studies *
Epilepsy
Inflammation in neurological disease
Biomarkers and outcome measures
Disease phenotyping (e.g., stroke subtypes), genotype-phenotype relationships in disease, clinical course and outcomes
Environmental exposures, gene-environment interactions
Cumulative effects of repeated injury or psychological stress on the brain
Nervous system interactions with body and endocrine system in disease (pain, obesity, diabetes)
Plasticity, as intrinsic repair mechanism, role in disease and recovery, changes with age/disease/intervention
Role of viruses in neurological disease
Autism (reasons for increased prevalence, treatments)
Complementary medicine, validity of alternative therapies (religious, cultural)
Brain injury, spinal cord injury
Addictive, toxic potential of medications
Cross-fertilization of research on different diseases
Orphan diseases, movement disorders, cerebral palsy, myasthenia gravis, peripheral neuropathies

Clinical practice goals and priorities

Prevention and early intervention *
Personalized medicine *
Earlier, improved and more accessible diagnostic capabilities *
Quality of life: treatments for current sufferers, in home care *
Health disparities (disease risk, access to care)
Studies on how to implement research-tested advances into clinical practice

Translational and therapeutic approaches

Prostheses, brain-machine interfaces, implantable devices, neurofeedback applications *
Exploit opportunities in bioengineering, nanotechnology, robotics *
Targeted drug delivery, blood-brain barrier, implantable devices and drug delivery systems *
Interventions derived from plasticity and learning and memory research, including behavioral interventions and rehabilitative strategies *
Stem cells, other regenerative strategies, tissue implants (including stem cells for research) *
Neuroprotective strategies (stroke, neurodegenerative disorders)
Nutritional/dietary interventions
Targeted genetic manipulation, siRNA
Non-neuronal cell types
Recapitulate developmental processes
Systems/network-level approach to therapy development

Resource, infrastructure, and technological needs

Research resources:
Molecular and other lab research tools (quality-controlled antibodies, siRNA libraries, ORF libraries, clearinghouse for purchasing/accessing neuroscience research tools) *
Research animals (mouse reporter lines and disease models, mouse repositories, extend genetic technology to rats, other engineered models) *
Databases/data and resource sharing (EEG/MEG, normal functional imaging data, genomic data, amino acid sequence data, protein-protein interactions, protein and gene expression maps) * (responses also included issues related to usage and awareness, interoperability, quality control, role of biologists in developing these resources)
Registry, resources for ongoing device development
Shared core technical facilities for research (mouse behavior, electrophysiology, genomics)
Multimodal imaging centers, other collaborative centers

Clinical research resources:
Databases and data/resource sharing (clinical outcome tracking, patient profiling for clinical trials and treatment protocols, pooling clinical data, biomaterials repositories, human tissue for research) Clinical trial networks, researcher-clinician networks to facilitate clinical trials

Resources/infrastructure for clinical practice:
Telemedicine, centralized specialty centers with telemedicine and telesurgery, other web resources to facilitate patient-physician interactions

Technological needs:
Improved imaging and multimodal monitoring and diagnostic devices (for research and clinical application, high resolution, non-invasive, implantable, portable) *
Drug delivery devices
Computational and analytical tools to assist in systems approach, integrated understanding
Technologies for injury prevention (e.g., more automated cars)
New ways to stimulate the brain

Research and professional training needs

Multidisciplinary training (reciprocal- neuroscience training to engineers, physical scientists; quantitative training to neuroscientists; also training in multidisciplinary experimental techniques) *
Specialists and researchers for headache and chronic pain disorders
Stroke care (new technology)
Training for translational research, FDA application preparation
Focus research training on fundamentals, not techniques
Allied health liaisons to communicate research advances to their disciplines
Movement disorders specialists
Addictive potential and side effects of medications
Determine and meet need for specialists trained in new treatments
Encourage women and minorities in biomedical workforce
Continue F and K award support

Public education areas

Information on treatment options, advances and availability for variety of disorders, including self-care, preventive strategies and lifestyle choices, factual information on alternative therapies, nutritional/dietary interventions *
General information about variety of neurological disorders *
Document and communicate outcomes of biomedical research on health and economy (regular AHRQ-style newsletters, public access to research results) *
Information about clinical trials, including clinical trial process and consent, ongoing trials
Basic scientific literacy (large-scale literacy programs, simple explanations of science behind topical issues, discredit pseudoscience, superstition, mysticism)
Anticipate ethical issues and inform public early about evolving technologies
Environmental contributors to disease
Addictive potential and side effects of medications

Process and policy recommendations

Big science/little science and research principles:
Promote data sharing, collaboration and multidisciplinary research; support large research centers *
Support investigator-initiated research *
Promote innovation/high risk research *
Restrict dollar amount/investigator
Encourage reproducibility of research results

New investigators and workforce issues:
Support new investigators, potentially through partnerships with other agencies or with industry
Report all postdocs on research grants
Examine relationship between NIH funding and fluctuations and sustainable research positions

Balancing the research portfolio:
Align research investment with disease burden *
Focus on applied, outcome-driven research
Strong support for basic science research
More systematic approach to basic, translational, and clinical research; ensure balance across areas
Reduce numbers of experimental animals

Specific recommendations for clinical research and physician scientists:
Improve IRB process, including improved efficiency and full disclosure/careful scrutiny of conflicts of interest
Improve consent process for clinical trials
Improved integration and communication between clinical practice and clinical research
Protected research time for physician scientists
Improved clinical trial design, increased efficiency

Issues related to clinical care:
Focus on implementation and accessibility of advances in practice, disparities in access *
Establish practice guidelines and regulations (particularly for treatments that alter brain activity)

Partnerships, roles of other stakeholders:
Protect clinical research and device development from market forces and industry interests *
Continue to collaborate with patient groups and disease organizations
Establish more partnerships with industry, philanthropies, nonprofit groups
Support device development by small businesses
Improved interagency communication; involve the FDA early in the drug development process
Act at bottleneck between lab and commercial sector
Cost-share with pharmaceutical industry, leave therapy and drug development beyond identifying targets to pharmaceutical industry

ELSI

Genetic testing, other prognostic information: management and patient privacy issues *
Costs and access to care and diagnostic procedures, reimbursement issues * Stem cells *
Drugs and devices that enhance brain function (access, regulation) *
Informed consent, managing expectations about treatment in clinical research
Patient privacy and rights, treatment choice, control of medical record, ownership of genetic/other biomaterial
Balancing extension of life with maintaining quality of life, costs, increased chronicity
Genetic and neurobiological underpinnings of human behavior and brain function; implications for concept of free will, for legal responsibility; misuse, patient privacy issues associated with brain imaging technology
Interference in science from religion, alternative movements
Genetic engineering, including introduction of human DNA into animal embryos
Animal research
Ongoing issues with patent law, proprietary information and technology

Areas of interest for well-represented responder groups

The most prevalent responses (those marked with an asterisk above) tended to be frequent among all groups. Other topics appearing frequently in particular groups are listed below.

Academic researchers (41 responses)

Integrated view of neurobiological systems and their interactions with the body and environment (relating neural activity to behavior, cognitive functions; integration across scales - molecular to networks to full system)
Prostheses, brain-machine interfaces, implantable devices
Support for individual investigators, new investigators
Promote data sharing, collaboration and multidisciplinary research

Clinicians (24 responses)

Improved communication and integration between clinical research and clinical practice
Improved understanding of disease subtypes and clinical features, including early mechanisms

Patients/caregivers (23 responses)

Information on research outcomes and advances, disorders in general, treatment options, standards of care
Preventive strategies, including dietary and lifestyle interventions
Healthcare costs and access

Students and postdocs (18 responses)

Opportunities through new technologies for research and clinical application (robotics, nanotechnology, imaging, multimodal recording and diagnostic devices)

Advocacy and Professional Organizations (5 responses)

Interest in exploiting plasticity and intrinsic repair mechanisms
Promote collaboration across researchers, research areas, and stakeholders

Full text of questions posed in the online RFI:

Question 1: What advances should we expect in clinical care for neurological disorders over the next fifteen years, based on anticipated progress in biomedical research? What scientific advances will result in a quantum leap in the care of neurologic disorders, and what aspects of care are likely to remain unchanged?

The past fifteen years has seen major shifts in how clinicians diagnose, treat, and prevent neurological conditions:

• Brain imaging and genetic technologies have changed how many neurological conditions are diagnosed.
• The first effective emergency treatment for stroke is transforming acute stroke care, and continued progress in stroke prevention is having a major impact on public health.
• New surgical interventions, such as deep brain stimulation, have emerged, with potentially wide-spread uses.

Fifteen years from now, we hope to have better interventions for all neurological conditions. The NINDS will systematically review basic, translational, and clinical opportunities to accomplish this. Please consider scientific trends and crosscutting medical advances to which NINDS research should contribute.

Question 2: Which major questions need to be answered in order to revolutionize how we understand the nervous system and prevent, diagnose, and treat nervous system disorders?

Several major discoveries over the past fifteen years have revolutionized how we understand the nervous system and its disorders. New insights into the development of the nervous system, brain plasticity, molecular pathology, and the role of non-neuronal cells, for example, are already changing how we think about the brain and treat maladies. What mysteries about how the nervous system develops and functions will be the subject of major advances in the next fifteen years?

Question 3: What new technical capabilities have the potential to revolutionize neuroscience research and clinical practice in the next fifteen years?

Advances in neuroscience knowledge frequently come on the heels of technological advances, providing the means to answer questions that were previously inaccessible. Patch clamp recording, gene splicing, genetically modified mice, and advanced neuroimaging are just a few of the techniques and tools that have opened up remarkable new possibilities in the clinic and at the bench. What major technical roadblocks could be overcome in the next fifteen years, and what might be the impact on research and practice?

Question 4: What will the neuroscience research landscape look like in fifteen years, and how can NINDS best contribute?

The NINDS is only one participant of many in the broad neuroscience research enterprise. In the past fifteen years:

• New NIH Institutes and Centers have been established, others have changed their focus, and trans-NIH programs including the Blueprint for Neuroscience and the Roadmap for Medical Research have taken on important roles.
• New neuroscience research institutes have been formed worldwide.
• The biotechnology sector has dramatically expanded, and the pharmaceutical industry has undergone major restructuring.
• Patient groups have become more active in supporting research.

How will the biomedical enterprise evolve over the next fifteen years, and what niche should the NINDS occupy? How should the NINDS operate in this landscape to support the range of neuroscience research, inform the public about the results of that research, train new scientists, and build the infrastructure required for scientific advancement?

Question 5: What, if any, infrastructural resources are needed to advance clinical or basic neuroscience research?

In the past fifteen years:

• The Human Genome Project has developed resources, such as GenBank, that have transformed research, and the NIH Roadmap for Medical Research is developing common resources to enable research that might otherwise not occur.
• The NINDS has invested in resources such as a publicly accessible genetic database for neurologic diseases, clinical trials networks, microarray centers, a Human Genetics Repository, and a variety of programs with other NIH institutes in the NIH Blueprint for Neuroscience Research.

What resources do you consider valuable, and which are no longer needed? What new neuroscience resources are essential to accelerate the pace of discovery over the next fifteen years?

Question 6: What ethical, legal, and social issues are likely to arise from advances in basic and clinical neuroscience over the next fifteen years, for which the NINDS should be prepared?

Genetic testing, stem cell research, brain imaging, and many other advances have raised serious ethical questions in the last fifteen years. Please consider what ethical, legal, and social issues are likely to follow the major changes in clinical practice, basic and translational research, technological capabilities, and the scientific landscape.

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