Of all the afflictions that can attack people as they grow older, neurodegenerative disease is one of the most devastating. Parkinson's disease (PD), Alzheimer's disease (AD), amyotrophic lateral sclerosis (ALS, or Lou Gehrig's disease) and related conditions affect not only the person involved, but everyone close to them -- and in the most cruel way imaginable, because these diseases attack the mind as well as the body.  Family and friends are forced to witness the literal dissolution of the very personality and identity of the one they love.

Because of the immense complexity of the human brain and nervous system, along with the fact that these diseases can manifest in confoundingly different ways in each of their victims, understanding these afflictions ranks among medicine's greatest and most important challenges.  Researchers at Penn’s Institute on Aging and Center for Neurodegenerative Disease Research are among the leaders of the worldwide effort to discover how these diseases originate and wreak their damage, and to find ways to mitigate their effects, slow or stop their progression, and one day, cure and prevent them.

It's a multifaceted campaign on a number of fronts, ranging from the genetic markers of neurodegenerative disease, to the molecular mechanisms by which they spread themselves throughout the nervous system of an affected individual, to new possibilities for diagnosis and treatment. Penn Medicine scientists are probing these areas and many more, achieving crucial insights and mapping fresh avenues for research and therapy.

TRACING NEURODEGENERATIVE DISEASE TRANSMISSION

An important part of understanding any disease is knowing how it gains a foothold inside the body -- how does it get started?  Once established, how does it spread itself and consolidate its harmful effects in the body? Cancer spreads through metastatic processes, infectious diseases through the bloodstream, but what about neurodegenerative diseases?  Penn researchers Virginia M.Y. Lee, PhDJohn Trojanowski, MD, PhD, and their colleagues have confirmed that tangled, distorted proteins can be passed from cell to cell within the nervous system, causing new damage to formerly healthy cells. Read more.

THE GENETICS OF ALZHEIMER'S DISEASE

Two of the most promising developments in the battle against neurodegenerative diseases are genetically based:  the increasing ability to identify people who may be at risk, and the creation of new therapies that can help existing patients by targeting the specific genes responsible. Gerard Schellenberg, PhD, director of the Alzheimer's Disease Genetics Consortium (ADGC) and professor of Pathology and Laboratory Medicine, is the driving force for Penn's contributions in these areas. Read more.

SEEKING AN ACCURATE DIAGNOSIS

Even though there's no definitive cure -- yet -- for neurodegenerative diseases such as Parkinson's and Alzheimer's, early and accurate diagnosis is still of utmost importance.  Because these diseases can take very different forms from one person to another, each with their own symptoms, rate of progression, and other characteristics, being able to identify a patient's particular disease variant is important for planning an effective individual treatment approach.  Leslie M. Shaw, PhD, professor of Pathology and Laboratory Medicine; Institute on Aging director John Q. Trojanowski, PhD, and colleagues are developing comprehensive tests that can detect and distinguish among the biomarkers of neurodegenerative disease.  Read more.

DRUG DISCOVERY

Ultimately, all the knowledge we may acquire about the origins, processes, and progression of neurodegenerative disease is of little value if it can't be translated into ways to help and treat patients.  One of the most direct ways to do that is through the development of new pharmacological approaches. Kurt Brunden, PhD, who spearheads the drug discovery efforts at the Center for Neurodegenerative Disease Research, came to Penn from the pharmaceutical industry, with hopes of progressing basic research being conducted at CNDR toward clinical trials.Read more.

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