Scientists throughout the world combined forces to map the human genome. Discovering the sequence of genes for the entire set of human chromosomes was a phenomenal task. Constructing the human genomic map has had a dramatic effect on current medicine and future therapies. For instance, it has accelerated our unraveling of basic disease processes and has provided insights that spurred the development of new drugs and biological agents. Discovering the types and amounts of proteins that are produced by gene expression in every region of the brain will permit construction of a proteomic map of the normal brain. Creation of progressively more complete genomic and proteomic maps of the brain will expand the understanding of the basic underpinnings of normal brain function. These normal maps provide benchmarks that can be compared to the genome and proteome of patients to pinpoint mutated genes or abnormal protein expression that underlie or accompany certain brain diseases. Increased knowledge of the process of gene regulation will also be essential to understanding the workings of a healthy brain. Taken together, increased knowledge of the normal genome and proteome will provide a foundation for the development of novel methods for counteracting disease processes of the CNS.
Scientists are striving to design ways to tailor the characteristics of nanomolecular and cellular agents and to restrict their effects to specific organs and sites. Nano-neuro-immunotherapy is a developing field based on development of nanoparticles that bind to a target with immune specificity and that deliver their drug payload there. This highly focused drug delivery and therapy technique contrasts sharply with current drug therapies that distribute their effects equally to areas of normal function and disease, the so-called shotgun approach, which results in systemic exposure and toxicity. Another emerging strategy, nanoparticle-augmented cell therapy, provides a method to simultaneously provide treatment and imaging at the site of brain disease. These strategies have the potential to reduce the morbidity and mortality of diseases of the central nervous system and to improve the quality of life of patients.
In the near future, nanomedicine will participate in the development of personalized medicine. Patients will undergo treatment tailored to their unique genetic makeup. Developments in the fields of pharmacogenomics, nutrigenomics, and ecogenomics will assume increasing importance (Odemir et al., 2009
). Neurosurgeons will be able to consider the individualized gene and brain maps (anatomic, functional, proteomic) of a patient and choose the treatment that should be safest and most effective. Using techniques of image guided therapy and nanoneurosurgery, neurosurgeons will be able to detect, confirm, and treat brain injury with nanostructures. Surgeons will inoculate cells with various types of nanostructures that carry a regimen of drugs, which can be released and act at different steps of a biochemical pathway. In the case of brain tumors, therapeutic cells will be delivered into tumors, where they will distribute their nanoparticles into tumor cells and release drugs to selectively eliminate tumor cells. The imaging contrast produced by therapeutic nanoparticles will permit imaging to monitor the progress of treatment for each patient.
Nanovectors, nanostructures, nanoplatforms, and nanoscale objects hold the potential to bring about less invasive and more selective treatment of brain tumors and other CNS diseases. Reaching this potential will require more research and the development of nanovectors that are less toxic, more versatile, and more biodegradable that current ones. Poor water solubility of some nanoplatforms must be overcome before they can be utilized in the development of nanodrugs. Many groups have functionalized very stable nanoplatforms such as CNT and gold nanoparticles in order to achieve solubility (Bianco et al., 2005b
; Klumpp et al., 2006
; Bartczak and Kanaras, 2010
). Others have designed soluble nanoplatforms such as poly(malic acid) nanoconjugates, which contain various antibodies and oligonucleotides for multitargeted drug delivery (Lee et al., 2002
; Fujita et al., 2006
; Lee et al. 2006a
; Fujita et al. 2007
and Ljubimova et al 2008a
). A new generation of nanovectors could incorporate multi-functional compounds and allow multistage, complex delivery of therapeutic compounds and augmented cellular therapies. The fields of nanomedicine, image-guided drug delivery and therapy, and gene therapy will inevitably converge further and to enable personalized medicine and targeted disease therapy. Advances in each field will drive the development of synergistic, more effective, and less toxic therapies for presently incurable neoplastic and non-neoplastic diseases of the CNS.
A coherent and coordinated effort among multiple US governmental agencies, foundations, and industry with a clear focus on translating nanotechnology could contribute significantly to the development of novel targeted and personalized therapeutics. With $40 billion in government-funded nanotechnology research in 2008, there is a strong imperative to increase the breadth and depth of nanotechnology and avoid unnecessary duplication of research efforts (cientifica, 2009). To improve efficiency in moving nano-technology from the laboratory to the clinic, we propose the creation of a central science, technology, medicine and law–healthcare policy (STML) hub/center that fosters and coordinates collaborative efforts across all institutions while creating policies, which could encourage such interaction. The government, regulators, industry, universities, foundations, and scholarly societies would contribute to the hub in order to maximize the impact of funds allocated by the US government. Such a hub could identify existing gaps between disciplines and direct specific seed funds to those areas. The central hub would encourage cross-disciplinary research that focuses on specific nanotechnology/nanomedicine initiatives. In the US, this would support coordinated efforts across the FDA, NSF, NIH, NCI, NINDS, and NNI. We believe that the creation of the central hub and its efforts will cultivate a spirit of partnership between industry, government, universities, and foundations that will translate innovations in nanotechnology into medicine, where they can be developed and implemented as powerful therapeutics for neurological and other disorders.
We also believe that the US government should dedicate funding to establish international consortia that could be administered under the STML hub/Center. These consortia could bring the finest scientists and research programs together across the world, create a united front for translational medicine, and create jobs in participating countries. Some of the funding for the hub and consortia should be allocated for meetings to educate lawmakers about interdisciplinary medicine. These sessions would provide lawmakers with information that would increase their understanding of medical research and science and assist them in meeting the challenge of evaluating legislative proposals that relate to emerging technologies in medicine. These efforts will significantly support efforts toward more personalized medicine, reduce healthcare cost, and eliminate duplicated research. An STML research hub/Center will increase efficiency in healthcare and help cultivate an environment that fosters the growth and development of biotechnologies and the biotechnology industry (e.g., formation of venture capital-backed startups, joint ventures). This approach can contribute to the economic growth and scientific advancement of the country, as new employees are needed to support the development of nanotechnology and nanomedicine in the newly formed companies.