Download Parkinson`s Disease Research White Paper

UVA HEALTH SYSTEM
RETHINKING PARKINSON’S DISEASE RESEARCH & CARE
AT UVA, OUR CLINICAL RESEARCH INTO PARKINSON’S DISEASE IS FOCUSED ON THE AREAS OF GREATEST IMPACT FOR
OUR PATIENTS. What can we learn from patients today? Why does Parkinson’s disease progress differently in each
person? By answering these questions, UVA can design new treatments that are more effective, providing patients with
a greater quality of life.
Our research is tied to the intimate connection we build with our patients. Not only does UVA provide compassionate,
state-of-the-art care for a large population of Parkinson’s patients, we have collected standardized data on our
patients—including family history, symptoms, the progression of the disease, and the effectiveness of medications in
alleviating symptoms. With this data we can begin to answer important questions about the disease.
DEVELOPING NEW TREATMENTS. Patients thrive under the care of physicians who are specialists in their fields. They
also benefit from having access to the latest treatment and research options available.
Focused Ultrasound
UVA neurosurgeon Jeffrey Elias, MD, serves as the principal investigator in three ongoing clinical trials for patients
who have Parkinson’s disease with tremor that is resistant to standard medical therapy. Each patient in the trials
will undergo a procedure that delivers more than 1,000 ultrasound beams—guided by a magnetic resonance
scanner—to a single, millimeter-sized treatment point deep in the brain. The energy delivered to that point causes
a thermal rise, essentially creating a small cauterization, which researchers hope will lead to a reduction in
Parkinson’s-related symptoms. The procedure involves no incision and no anesthesia, allowing patients to remain
alert and interactive with surgeons. UVA leads the world in the use of this focused ultrasound therapy for the
treatment of Parkinson’s and other movement disorders. In the lab, researchers are also exploring ways to use the
noninvasive aspects of ultrasound to map the brain.
Improved Drug Delivery
If researchers can identify which neurotransmitters are responsible for the various symptoms of Parkinson’s and
other movement disorders, they can design more selective therapies that will optimize symptom relief while
minimizing associated side effects. A critical step in this process is the precise therapeutic delivery of compounds to
specific regions of the brain. Convection-enhanced delivery bypasses the blood-brain barrier and permits precise,
safe targeting with a variety of new drug compounds.
This two-phase trial is led by Dr. Elias. In the first phase, investigators will measure the distribution of a temporary
neural inhibitor, injected during deep brain stimulation surgery, over a short period of time. If the compound shows
promise, the team will administer the drug over a longer period of time, measuring clinical results and comparing
the new drug to standard treatments used today. Imaging tracers can also be co-infused with therapeutic agents to
accurately determine drug distribution in real-time, allowing doctors to more quickly refine treatments and
improve results.
New Surgical Interventions
Deep brain stimulation (DBS) has become a standard treatment for Parkinson’s. Now UVA has invested in an MRI
surgical suite, an operating room within an MRI machine. This new technology gives neurosurgeons like Dr. Elias the
chance at never-before precision and accuracy in surgical intervention. Unlike traditional DBS, this technique is
performed while a patient is asleep, leading to greater comfort and improved outcomes. UVA has patented using
enhanced-MRI during stereotactic surgeries.
Easing Movement & Stiffness
Neurologist Binit Shah, MD, works closely with Dr. Elias and other researchers conducting groundbreaking clinical
trials at UVA. He supervises clinical care, assessing function and managing medications. At the same time, he is
preparing for a clinical trial to help patients mitigate the slowness and stiffness they experience with the disease. If
approved by the FDA, this new treatment could change the way Parkinson’s patients are cared for, especially
patients who are not candidates for surgical intervention. In addition, Dr. Shah has opened a new trial for focused
ultrasound that promises to help with slowness and stiffness, as well as tremor. He is also part of a NIH-funded trial
of a novel agent to slow Parkinson’s progression in patients with early symptoms.
Gait Disorders & Balance
Gait disorders and balance impairments are some of the most incapacitating symptoms of Parkinson’s disease.
Recent studies have confirmed the high rate and high risk of falls for patients with Parkinson’s. At UVA, our doctors
work closely with physical therapy to detect at-risk patients and to develop protocols to help prevent falls. This
team approach allows us to treat not only the physical symptoms of the disease, but also the accompanying
cognitive and emotional symptoms. Our psychologists also work closely with physicians to assess and treat mood
issues and cognitive changes.
UNDERSTANDING THE BUILDING BLOCKS OF PARKINSON’S DISEASE. Several factors can severely impact a patient’s
prognosis and progression of the disease. At UVA, our researchers are investigating two key areas:
Genetics
Neurologist Matt Barrett, MD, MSc, examines the link between genetics and memory problems in Parkinson’s
disease patients. He believes that genetics is the key factor determining when a Parkinson’s patient might develop
dementia. Earlier research has linked certain genes to an increased risk for Alzheimer’s disease and dementia.
Barrett’s research team will examine whether those same genes increase the risk for memory problems in
Parkinson’s patients, the first step toward tailoring treatments for memory problems that are based on an
individual’s specific genetic makeup.
Heredity
UVA researchers are partnering with investigators in Sweden who are studying the role heredity plays in cognition
deficits among Parkinson’s patients. This collaboration gives our investigators access to vast amounts of data and
speeds their research into answering two vital questions. Why do some patients develop problems with cognition,
while others, even in the same family, do not? Is it possible to predict which patients will lose cognitive ability
before symptoms occur? This research helps us identify risk factors for debilitating symptoms, impacting our ability
to deliver more effective care.
CARING FOR PATIENTS TODAY, AND IN THE FUTURE. UVA offers Parkinson’s patients access to state-of-the-art
multidisciplinary care, from the point of diagnosis throughout the course of their disease. With help from friends and
benefactors, UVA can expand support services for individuals and their families, develop novel diagnostic tests to
measure the progression of the disease, and explore the best ideas for future treatment—treatments that will provide
real and lasting hope for our patients and their families.
For more information, please contact:
Jas Heim
Associate Director of Development
[email protected]
434.982.0313