Latest grants will fund basic research into Parkinson's, epilepsy and other brain disorders
The Max Planck Florida Institute for Neuroscience, the first and only U.S. extension of the prestigious Max Planck Society, today announced it has received approximately $1,257,500 in grant funding from prestigious national and international organizations to fund research into Parkinson-s, epilepsy and other neurological disorders. The new institute focuses solely on basic research that aims to analyze, map, and decode the human brain-the most important and least understood organ in the body.
"The Max Planck Florida Institute for Neuroscience continues on its mission to provide new and more detailed understanding of the structure, development and functional organization of the nervous system," said Max Planck Florida Institute for Neuroscience Scientific Director and CEO Dr. David Fitzpatrick. "These grants and the research they will support will pave the way for new insights, hopefully leading to advances in treatments and cures for brain disorders ranging from Parkinson's to epilepsy."
The Max Planck Florida Institute for Neuroscience was awarded $522,500 over two years in funding from the National Eye Institute, one of 27 institutes and centers of the U.S. National Institutes of Health. The goal of this project is to develop the next generation of molecular tools to probe the structure, function and development of neurons in the living brain. The research builds on recent technological developments from the world of physics that use laser powered microscopes to see into the living brain, literally watching neurons perform their functions. This same technology can be used to control the activity of neurons with light, making it possible to understand how the interactions between specific populations of neurons contribute to brain function. Further progress in using this technology depends on the development of molecular probes that can be targeted to specific classes of neurons in the brain, and this is the specific aim of this project that will be done in collaboration between two of the institute scientists, Dr. Samuel Young, Jr. and Dr. David Fitzpatrick. Working together, they plan to develop and characterize a recombinant viral vector that will allow selective expression of molecular probes in neurons to modulate their function of neurons that use the inhibitory neurotransmitter GABA. These neurons play an important role in ensuring the proper balance of activity in the brain, and alterations in their function have been implicated in a number of brain disorders including epilepsy, schizophrenia, and depression. Success in generating this viral vector which allows for cell-type specific expression will open the door to enable the use of a battery of molecular tools in a cell-type specific manner that has the potential to alter the study of brain function and provide the foundation for new approaches to the diagnosis and treatment of neurological and psychiatric disorders.
Dr. Fitzpatrick added that the National Institutes of Health funding is "precisely the type of innovative research that emerges when scientists with different expertise are brought together in an environment that stimulates creative collaborations."
"We hope that the development of this technology can eventually be turned into therapeutic tools to treat neurological disorders," noted Dr. Young.
Research Group Leader Dr. Hiroki Taniguchi was awarded $100,000 by Citizens United for Research in Epilepsy (CURE) for his efforts to study cellular structures seeking to identify pathways for treatments or cures for epilepsy. Dr. Taniguchi heads a research group that studies the development and function of inhibitory neural circuits and is a leading expert in chandelier cells, the brain's most powerful inhibitory neurons. His work on the origin of these cells in pre-clinical models, recently published in Science Magazine, found that these highly powerful neurons form much later in embryonic development than previously thought. Dr. Taniguchi's work not only adds to the understanding of chandelier cells, but gives direction to future and continuing research on the periods of prenatal neurologic development that might be associated with epilepsy.
Chandelier cells potentially hold the key to new epilepsy treatments with minimal side effects due to their powerful inhibitory effect. To date, the primary treatment for epilepsy is anticonvulsant medications which target neurotransmitter receptors and ion channels. These drugs can have significant side effects and are inefficient for about 30 percent of patients. While the cause of epilepsy is not clear, one of the most plausible hypotheses is there is an imbalance between excitatory and inhibitory neurons. Dr. Taniguchi's research supported by this grant will study the transplantation of chandelier cells seeking to restore a proper balance and revert seizure activities in pre-clinical models of epilepsy.
"The use of chandelier cells has the potential to revolutionize our treatment of epilepsy," said Dr. Hiroki Taniguchi. "If transplanting inhibitory neurons works, we might uncover a cure rather than simply treatments for epilepsy, involving significantly less side effects than anticonvulsant medications used in current treatments."
Dr. Taniguchi was also awarded roughly $488,000 over three years by the Japan Science & Technology Agency to develop cutting-edge methods to track fine details in local circuits of inhibitory neurons that are thought to be linked to epilepsy, autism and schizophrenia. Dr. Taniguchi will utilize state of the art mouse genetics to highlight the connectivity between neurons that specifically use gamma aminobutyric acid (GABA) as a neurotransmitter. If successful, this will allow scientists to visualize neuron connections and ultimately identify defective areas to facilitate continued research on diagnosis, and pathways for potential treatments or cures.
Dr. Samuel Young, Jr. also received two separate awards, totalling nearly $147,000, both from the Michael J. Fox Foundation for Parkinson's Research (MJFF) for his work to develop better models for the study of Parkinson's disease to advance the development of treatments. Dr. Young leads the Research Group of Molecular Mechanisms of Synaptic Function where their research focus is one synapses - the highly specialized contact points within the brain that are the fundamental units of communication where neurons pass electrical and chemical signals to one another.
The first grant, a collaborative research project between Drs. Young and Ron Mandel (University of Florida), will support the development of non-toxic transgene controls for viral vector based Parkinson's disease models. Viral vectors are used by scientists to deliver genetic material into cells. They enable scientists to ask questions such as how a particular gene affects a cell. Control genes are critical in all viral vector studies as they provide a point of comparison to the experimental gene that is expressed, which allows for differentiation of potential artifacts. Recent work points to the fact that many commonly used control genes (i.e. eGFP) are themselves toxic when overexpressed in a cellular environment. Successful identification of an appropriate control gene will enable investigators to more appropriately examine the role that Parkinson's disease-specific genes play in cellular settings and to evaluate their role in disease progression in in vivo models.
"We are thrilled to receive our second round of funding from the Michael J. Fox Foundation - the leading force advancing Parkinson's research," said Dr. Samuel Young, Jr. "Finding the proper control gene is critical for the development of better models that replicate Parkinson's disease and will assist in successfully testing Parkinson's drugs and ultimately find a cure for PD."
The second grant will allow Dr. Young's group to build upon existing research focused on LRRK2. Mutations in LRRK2, which encode a type of protein known as a kinase, have been found to account for the greatest genetic link to Parkinson's disease. The MJFF has previously awarded a grant to Dr. Young in May 2012 under which Dr. Young is leading a multi-investigator team focused on generating and characterizing viral vectors that can express LRRK2 in pre-clinical models. This additional work will ensure that all the LRRK2 viral vectors created are appropriately evaluated for basic quality control measures before they are made available to the general research community.
In addition to these individual grants, the Max Planck Florida Institute for Neuroscience received a contribution of $40 million in December 2012 from representatives from Germany and the Max Planck Society to support additional neuroscience research of the next four years. Prior to the Institute opening, several governmental and educational partners committed $190 million, including the Florida State Governor's Office of Tourism, Trade and Economic Development's Innovation Incentive Fund ($94.1 million) and Palm Beach County ($86.9 million, including $60 million dedicated to the construction of the new research facility). Florida Atlantic University provided a 50-year rent-free lease on a six-acre site, valued at $6.3 million, and the Town of Jupiter waived impact fees with a value of $260,000.
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