Penn is committed to addressing public health issues related to the aging population through research programs focusing on normal aging and aging-related diseases, particularly brain diseases. They’ve established a network of partners to carry out this research and translate its findings to the clinic as well as the public-at-large.
At CNDR, the hypothesis that diverse neurodegenerative diseases arise from spreading and aggregation of toxic brain proteins links the CNDR research programs. Clarification in any one disorder has a profound impact on understanding the mechanisms that underlie all of these disorders and on efforts to develop novel therapies to treat them.
To solve the intricate puzzle of neurodegenerative disease, scientists at CNDR take a variety of approaches:
- Basic Science
Led by Dr. Virginia M.-Y. Lee, the basic science program at CNDR aims to try to understand what goes wrong at the most basic, molecular, and cellular level.
Some neurodegenerative diseases originate from mutations in genes. Even those diseases that are not strictly 'genetic' have some genetic component. At CNDR, the neurogenetics program is led by Dr. Jerry Schellenberg and Dr. Vivianna Van Deerlin.
Much of what is known about neurodegenerative disease comes from studies of brain tissue from both affected people and animal models of the disease. Researchers can request to obtain samples from the Biosamples and Brain Bank for study.
Biomarkers are characteristics that can be objectively measured and indicate the presence or state of a disease. They are extremely important for diagnosing disease and monitoring response to treatment. The search for biomarkers of neurodegenerative diseases at CNDR is led by Dr. Leslie Shaw, who is also the Biomarker Core Co-Director of the Alzheimer's Disease Neuroimaging Initiative (ADNI) and Penn's Biomarker Core center for the Michael J. Fox Foundation's Parkinson's Progression Markers Initiative. Alice Chen-Plotkin, MD, is also now the leader of Penn’s Alzheimer’s Disease Core Center (ADCC) Biomarker Core.
- Translational Medicine
The focus of the Translational Medicine Program is to translate basic research discoveries into treatment approaches that will be useful in the clinical setting. At CNDR, this effort is led by Dr. Virginia M.-Y. Lee.
- Drug Discovery
The long process of developing new drugs that will be useful in treating neurodegenerative diseases begins with the discovery of targets that, if modified, might affect the course of the disease. It includes the development of assays for evaluating the effect of different substances on those targets, the screening of hundreds of thousands of compounds to identify one or a few possible drug candidates, the optimization of these candidate drugs, and the testing of potential drugs in animal models of the disease and then in humans. The drug discovery effort at CNDR is led by Dr. Kurt Brunden and Dr. Virginia Lee.
- Clinical Research
Research discoveries made in the laboratory must be tested in clinical settings in order to determine their effectiveness. Clinical research in Alzheimer's disease at CNDR is led by Dr. David Wolk and Dr. Jason Karlawish. Clinical research in frontotemporal degeneration and related conditions is led by Dr. David Irwin, and clinical research in Parkinson's disease is led by Dr. Daniel Weintraub.
- Biostatistics and Bioinformatics
Biomedical research in the 21st century results in the acquisition of enormous amounts of data. At CNDR, the management and analysis of data collected is led by Dr. Sharon Xie and Dr. Li-San Wang. The team is working on disease-related bioinformatics (aging, neurodegenerative diseases, and cancer), human genetics (including genome-wide association studies and whole-genome/whole exome resequencing studies), and computational methods for next-generation sequencing technology. They work collaboratively to analyze the Integrative Neurodegenerative Disease Database (INDD) from the Center for Neurodegenerative Disease Research (CNDR) that tracks ~11,000 patients who attended one of four neurodegenerative disease centers at Penn. This project hopes to lead to better characterization of disease progression, robust identification of controls and AD patients for clinical studies, and more sensitive early diagnosis of conditions such as Alzheimer's disease.