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UAB DEPARTMENT OF NEUROLOGY PARKINSON’S DISEASE RESEARCH PROGRESS REPORT 2015 Knowledge that will change your world THE UNIVERSITY OF ALABAMA AT BIRMINGHAM

PARKINSON’S DISEASE RESEARCH PROGRESS …...UAB DEPARTMENT OF NEUROLOGY PARKINSON’S DISEASE RESEARCH PROGRESS REPORT 2015 Knowledge that will change your world THE UNIVERSITY OF

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Page 1: PARKINSON’S DISEASE RESEARCH PROGRESS …...UAB DEPARTMENT OF NEUROLOGY PARKINSON’S DISEASE RESEARCH PROGRESS REPORT 2015 Knowledge that will change your world THE UNIVERSITY OF

UAB DEPARTMENT OF NEUROLOGY

PARKINSON’S DISEASE RESEARCH PROGRESS REPORT

2015

Knowledge that will change your world

THE UNIVERSITY OFALABAMA AT BIRMINGHAM

Page 2: PARKINSON’S DISEASE RESEARCH PROGRESS …...UAB DEPARTMENT OF NEUROLOGY PARKINSON’S DISEASE RESEARCH PROGRESS REPORT 2015 Knowledge that will change your world THE UNIVERSITY OF

Haydeh Payami, Ph.D.John T. and Juanelle D. Strain Professor of Neurology

A world-renowned geneticist and scientist, Dr. Payami joined the UAB faculty in February 2015 from New York where she served as the Senior Research Scientist at the New York State Department of Health Wadsworth Center and Professor of Molecular Genetics at the State University of New York. Dr. Payami is focused on prevention and treatments for Parkinson’s and Alzheimer’s diseases, bringing a wealth of

knowledge on the role of genetics in drug response with more than 25 years of research. She is the founding director of the NeuroGenetics Research Consortium and serves as Lead-Investigator. We are most excited about Dr. Payami’s objective of personalized medicine where treatments can be tailored to an individual’s genetic makeup for maximum benefit.

Matthew Goldberg, Ph.D.Charles S. Ackerman Endowed Professor of Neurology

Dr. Goldberg was recruited from the University of Texas Southwestern Medical Center to UAB in fall of 2014 as an Associate Professor of Neurology, joining the Center for Neurodegeneration and Experimental Therapeutics. His research has concentrated on detecting the mechanisms of neuronal degeneration and understanding the roles in inherited forms of Parkinson’s disease. He has made important progress in

these areas by analyzing cell and animal models bearing chromosomal mutations linked to hereditary Parkinson’s disease. Currently, Dr. Goldberg’s laboratory is studying PINK1 knockout and DJ-1 knockout rat models of Parkinson’s disease, supported by two NIH grants, as well as LRRK2 transgenic mice, supported by a grant from the Michael J. Fox Foundation for Parkinson’s Research.

OUR LATEST RECRUITMENTS

Thanks to the generous support of visionary philanthropic partners, researchers at the University of Alabama at Birmingham (UAB) and the Center for Neurodegeneration and Experimental Therapeutics (CNET) are making great progress toward disease-modifying treatments for Parkinson’s disease with the ultimate goal of finding a cure. We are now on the cusp of first-in-man clinical trials that hold the potential for neuroprotective drugs to treat Parkinson’s. Philanthropic support is vital to advancing the most promising research and has played an important role in our success to date. This support also is leveraged to garner additional critical funding from numerous private donors, nonprofit grants, and federal grants, including the National Institutes of Health (NIH). The support of philanthropic partners strengthens our ability to provide unsurpassed care for the more than 5,000 patients served each year by the UAB Movement Disorders Clinic and enables us to draw closer to our ultimate goal of helping the patients who live with the disease and their families.

PHILANTHROPIC IMPACT ON RESEARCH AND PATIENT CARE

Over the past 10 years, UAB has become a national and international leader in Parkinson’s disease research by recruiting the best and brightest physicians and scientists and by strategically investing in research efforts aimed at new treatments and cures.

DRUG DISCOVERY & DEVELOPMENT PATHWAYTraditional purview

of academia “Valley of Death” Traditional purview of industry

Target identification

Target validation

Assay development and small molecule

screening

Drug development: hit to lead and lead

optimizationLRRK2 and 14-3-3

Preclinical candidate (ADME and safety/

toxicology)

First-in-human trials

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Talene Yacoubian, M.D., Ph.D.Parkinson Association of Alabama Scholar in Neurology

Talene A. Yacoubian, M.D., Ph.D., is researching potential neuroprotective effects of drugs that act on 14-3-3 proteins that play a crucial role in inhibiting cell death pathways. Evidence shows overexpressing these proteins could ultimately slow and possibly halt the progression of Parkinson’s disease. Small molecules are being identified that can help increase overexpression of these proteins. Dr. Yacoubian is working with SRI on a pilot high-throughput screening that has already produced 11 small molecules that

can enhance expression of 14-3-3. Candidate molecules identified through this screening will be tested for their ability to reduce toxicity in cellular models of Parkinson’s disease. The most promising molecules will then be examined in animal models. Dr. Yacoubian is also leading an investigation into a possible interaction between 14-3-3s and LRRK2. She hopes to discover if 14-3-3s can regulate LRRK2 function, thereby reducing the toxicity of mutant LRRK2.

Laura A. Volpicelli-Daley, Ph.D.Assistant Professor of Neurology

The goal of Laura Volpicelli-Daley, Ph.D., is to determine how alpha-synuclein pathology found in Parkinson’s disease and dementia with Lewy Bodies impacts membrane traffic in neurons. Protein aggregates of alpha-synuclein define Parkinson’s disease and other synucleinopathies including: dementia with Lewy bodies, the Lewy body variant of Alzheimer’s disease, and other neurodegenerative disorders. She also seeks to determine how other genes implicated in Parkinson’s disease impact the formation alpha-synuclein

inclusions. To accomplish these goals, Dr. Volpicelli-Daley uses a wide range of approaches such as live cell and high resolution imaging in primary cultured neurons, pharmacology, mouse and rat models and behavior.

David G. Standaert, M.D., Ph.D.John N. Whitaker Professor and Chair of Neurology

UAB Department of Neurology Chair, David G. Standaert, M.D.,Ph.D., is studying changes in the immune system that may be critical for the progression of Parkinson’s disease. This novel approach suggests that, while there may be several upstream triggers for Parkinson’s, once the degenerative process starts,

inflammation is responsible for progression. This idea is supported by recent studies showing anti-inflammatory drugs, such as ibuprofen, may reduce risk of Parkinson’s disease slightly. We are now seeking a more powerful and targeted inhibitor of the Parkinson’s disease-specific immune response to halt the progress of the disease.

Andrew B. West, Ph.D.John A. and Ruth R. Jurenko Professor of Neurology

The protein Leucine-rich Repeat Kinase 2 (LRRK2) has been identified as potentially playing a pivotal role in the development of Parkinson’s disease. Using high-throughput drug screening, UAB’s Andrew B. West, Ph.D., in collaboration with Southern Research Institute (SRI), has identified a set of five potential

drugs called LRRK2 inhibitors that may go beyond symptom relief to directly counter the inflammation and nerve cell death at the root of Parkinson’s disease. This is the first time such a drug target has been found for any neurodegenerative disease. The ultimate objective is to find a compound that will inhibit the activity of LRRK2 in Parkinson’s disease patients and those with early stages of the disease, which Dr. West theorizes will halt disease progression. He envisions a treatment that protects and restores neurological pathways, and that will address and correct the underlying mechanisms that cause Parkinson’s disease.

G I V E S O M E T H I N G | C H A N G E E V E RY T H I N G

ACCELERATING RESEARCH AIMED AT A CURE

MAJORACCOMPLISHMENTS

UAB Neurology and Neurosurgery ranked in the top 50 programs in the country by U.S. News & World Report for a second year in a row.

Research funding by neurology at UAB has grown more than 200 percent since 2003, and the numbers continue to rise.

UAB has been designated as an Advanced Center for Parkinson Research by the American Parkinson Disease Association (one of only nine nationally), a designation that provides sup-port for pilot research projects breaking new ground.

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Page 4: PARKINSON’S DISEASE RESEARCH PROGRESS …...UAB DEPARTMENT OF NEUROLOGY PARKINSON’S DISEASE RESEARCH PROGRESS REPORT 2015 Knowledge that will change your world THE UNIVERSITY OF

Knowledge that will change your world

THE UNIVERSITY OFALABAMA AT BIRMINGHAM

PARTNERSHIPS MAKE OUR PROGRESS POSSIBLEThe immense courage displayed by the Parkinson’s patients I have treated over the years continues to bolster my resolve to advance UAB’s research into more effective therapies for Parkinson’s disease. Our ultimate goal remains, as always, finding a cure.

Philanthropic support has allowed our research to progress with astonishing speed, so much so that we now find ourselves within a few years of Phase I clinical trials for potentially life-altering therapies for Parkinson’s patients.

I believe we are in the midst of the “Century of the Brain,” with greater opportunities to make groundbreaking discoveries in neuroscience than ever before. We thank our community partners for enabling our researchers to take full advantage of this unique moment in history. Without them, we could not have advanced to this critical phase.

Sincerely,Ray. L. Watts, M.D.

President, University of Alabama at BirminghamProfessor of Neurology

FOR MORE INFORMATION Kate TullyDirector of Development and Community RelationsDepartment of NeurologyThe University of Alabama at BirminghamSC 360B, 1720 Second Avenue SouthBirmingham, AL 35294-0017(205) [email protected]