Neurodegenerative diseases are devastating. I want to be able to make a difference.
I have been very fortunate to be able to be in a position to apply my skills as a basic scientist and to team up with John Trojanowski, who is a physician scientist, and to be able to translate the basic research we have done, and to be able to make a difference in the lives of people with neurodegenerative diseases.
Narrator:
A recent feature in the journal Nature Medicine said, "Virginia Lee and John Trojanowski, who together lead the Center for Neurodegenerative Disease Research, CNDR, and the Institute on Aging at the University of Pennsylvania, may well be the only collaborators who run a lab that produces over 30 papers a year, and incredibly, remain happily married."
Dr. Virginia Lee:
In 1985, we decided to do a little experiment to see if we could collaborate and not kill each other.
Narrator:
This powerful partnership produced one of the bedrocks of scientific understanding of Alzheimer's.
John Q. Trojanowski, M.D., Ph.D., Director, Alzheimer's Disease Center, University of Pennsylvania:
In 1991, we were able to solve a major controversy about the composition of the tangles in Alzheimer's disease. Virginia Lee and I were able to show concrete, unequivocal data that these tangles are formed of the proteins tau.
Dr. Virginia Lee:
So that started this long collaboration with John Trojanowski.
The Center for Neurodegenerative Disease Research at the University of Pennsylvania is unique. The signature of our work actually is a little bit different than other labs that work on neurodegenerative diseases which focus on one disease. There is tremendous crossover of these different diseases.
So, we study neurodegenerative disease as a group of diseases that could effect your memory, your emotions and your movement.
Christopher M. Clark, M.D., Director, Penn Memory Center (PMC), University of Pennsylvania:
One of the focuses at the University of Pennsylvania is developing new ways to detect the earliest presence of disease in the brain.
That research area is called, "Biomarkers." Anything from ways of imaging the pathology in the brain to methods of detecting the earliest changes in the spinal fluid that indicate the presence of disease.
Mark Forman, M.D., Ph.D., Director, ADCC Neuropathology, University of Pennsylvania
The same way that a blood-glucose level tells you if you have diabetes, can we have a simple blood test that tells if a patient has Alzheimer's disease?
Dr. John Trojanowski:
The analogy that I use is a pregnancy test. You can take a pregnancy test and with 99.9% certainty know that you are pregnant before you ever feel, as a woman, the effects of the pregnancy, or before anyone else would ever surmise that you are pregnant. That's EXACTLY what we need for Alzheimer's disease.
We are not there yet, but we do have chemicals that we can measure in cerebrospinal fluid, in blood, in plasma, in urine.
In collaborating with Chris Clark, we are able to bring assays of these chemicals into the clinic.
Dr. Clark:
It holds the potential for really speeding up the development of new treatments, additional studies can be done using fewer patients, they can be conducted in a much shorter interval, and it should accelerate the discovery for effective treatments in Alzheimer's disease and all dementing illness.
Dr. John Trojanowski:
But the first order of business is to figure out how the brain is broken.
Narrator:
What's broken in the brains of persons with ALS was a mystery until early 2006, when Virginia Lee, leading an international team of researchers, including John Trojanowski, identified the misfolded protein that causes both ALS and a lesser-known disease, frontotemporal dementia (FTD).
Dr. Virginia Lee
Dr. Virginia Lee:
We were after these lesions that are found in the brains of patients with amyotrophic lateral sclerosis, or ALS, or Lou Gehrig's disease.
We worked very hard for four to five years. Then finally, we were able to identify a protein, TDP-43, and we knew we had something really, really exciting and really hot!
Dr. John Trojanowski:
There is robust activity that reflects major advances in understanding for us at Penn, because of the collaborative culture that brings basic scientists, clinicians, epidemiologists and biostatisticians together. Then in this era of increased interest in translational science, we will be more adept at moving forward with programs like this than many of our peers and competitors.
Narrator:
Ultimately, the vision of Penn's neurodegenerative disease researchers is to help people like Karen Faulkner realize their vision.
Karen:
I am going to write books, I am going to shoot fabulous pictures, travel, I want to go to live on a farm for a year.
Dr. Christopher Clark
Our ambition is really to promote successful aging and to get rid of the illnesses that prevent successful aging. Particularly from my standpoint, certainly, the dementing illnesses.
Dr. John Trojanowski:
I look upon aging as beginning at conception. I think the mechanisms that influence how we age may be in process already when we are teenagers or in our 3rd or 4th decade. My own area as a neuroscientist makes me particularly excited about programs for successful brain aging.
What I mean by that are lifestyle practices that studies indicate are associated with reduced risk for dementia; physical activity, cognitively-stimulating activity, exercise, diet.
Some of the information come from epidemiological studies, patient-oriented studies, animal-model studies, studies that involve many different colored palates, if you will. By that I mean technologies, disciplines, areas of expertise - and these all converge very readily in a place like Penn.
Narrator:
The progress made by Penn CNDR has been extraordinary. To fulfill its promise, CNDR needs to create new partnerships, partnerships that will fuel future advances and discoveries.
Dr. John Trojanowski:
We spend 56 billion dollars a year, according to BusinessWeek, on anti-aging potions, lotions, solves, balms, that have no proven efficacy.
But the Federal government, which is the largest funder of research, only spends $700 million on Alzheimer's disease research.
We spend 2 billion dollars on popcorn, so we spend more on popcorn every year than we do on research.
There is something wrong with this equation. We are the richest country on Earth, facing the largest epidemic of a dementing illness ever in the history of civilization.
And we need to see this as an urgent, actionable item. We can take the steps today to go after a hundred targets at the same time rather than a handful, and it is all a matter of money.
Northwestern University researchers found that brains of people in their 80's with extra-sharp memories had far fewer fiber-like "tau" tangles than average.
Exebryl-1 is entering human trials after its remarkable performance in Alzheimer's transgenic mice. In the lab, Exebryl-1 lowered brain beta-amyloid by 30-50%, along with noticeable improvements in memory.
Researchers are beginning to prove that alternative methods of treatment can be helpful in the fight against Alzheimer’s disease. Daily meditation has just joined the list.
Researchers are beginning to prove that alternative methods of treatment can be helpful in the fight against Alzheimer’s disease. Daily meditation has just joined the list.