Rush Parkinson's Disease Program Case For Support

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					The case for philanthropic support

The Rush Parkinson’s Disease Program
Now is the time for the Rush Parkinson’s Disease Program.

Rush has one of the world’s largest teams of Parkinson’s disease specialists
treating more than 2,000 patients. Our clinical success is the result of highly
productive research collaborations. When our scientists make a discovery in
the lab, our clinicians apply the findings to patient care; when our clinicians
make a discovery in patient care, our scientists examine the underlying
mechanisms in the lab.

Our team’s depth and breadth of expertise enable us to simultaneously
pursue promising leads spanning everything from the study of Parkinson’s-
related hallucinations to bacteria in the gut of Parkinson’s patients, but this
work is hampered by slow grantmaking processes and limited government
funding. Today our experts are on the brink of multiple breakthroughs
with the potential to crack the code of Parkinson’s disease and pioneer the
most effective treatments. We need you — our philanthropic partners — to
help us advance this work.

With $30 million in philanthropic investment, we will expand patient
care, research, training opportunities and programmatic resources, all to
the benefit of patients worldwide. Together, you and Rush can fulfill the
promise of a transformation in care, one that significantly enhances the
lives of Parkinson’s disease patients and brings us closer to a cure than we
ever dreamed possible.

Christopher G. Goetz, MD
Director, Rush Parkinson’s Disease and Movement Disorders Program
The United Parkinson’s Foundation Professor of Neurological Science

Jeffrey H. Kordower, PhD
Director, Research Center for Brain Repair
Section Head, Neuroscience
The Jean Schweppe Armour Professor of Neurological Sciences
Why Rush?
U.S. News & World Report consistently ranks Rush University Medical
Center among Illinois’ — and the nation’s — top hospitals. We
have a three-time Magnet-designated nursing staff, the highest
recognition for nursing excellence, and a first-of-its-kind hospital
tower, filled with state-of-the-art technologies. We pioneer
groundbreaking treatments and have educated thousands of health
care professionals. But that’s not what sets Rush apart.

Rush is the kind of place patients turn to when they need hope. Patients
at Rush are treated by collaborative teams of compassionate experts.
They can trust that they’ll receive the most promising treatments backed
by scientific discovery because their physicians and nurses are also
skilled researchers partnering with laboratory scientists. Patients
know that they and their families will be considered trusted partners
in making the health care decisions that affect them so personally.
And they quickly realize that, while some hospitals may shy away from
taking on complex cases, clinicians at Rush thrive on it, motivated to
help all patients and especially those who need solutions the most.

So what makes Rush such an extraordinary place of healing and a
worthwhile philanthropic investment? It’s the fact that everything
we do, from teaching students and researching disease to
offering the latest treatment options, reflects our steadfast
commitment to one goal: providing the very best patient care.
“Facing a diagnosis of Parkinson’s disease is
incredibly challenging, but patients who see the
team in the Rush Parkinson’s Disease Program
come away with a sense of hope. At Rush, patients
and physicians work to fight Parkinson’s disease
together, as a team. I find so much hope in the care
I receive, the very promising research happening
now at Rush and the fundraising I do to support
it; without that, this disease would be beating me.
Instead, I’m beating this disease.”
— Sherry Koppel, patient and donor

Sherry Koppel was diagnosed with Parkinson’s disease in 1999 and has
long been a patient of Christopher G. Goetz, MD, director of the Rush
Parkinson’s Disease Program. She has been an avid fundraiser for Rush’s
Parkinson’s disease research for more than 10 years, stating that these
efforts in support of a cure allow her to find “purpose in [her] illness.”
The Impact of Parkinson’s Disease
Millions worldwide live with the effects of Parkinson’s
disease each day, its symptoms changing lives, families and
entire communities:
                                                                      10 million
                                                                      people worldwide
•	 Parkinson’s disease affects an estimated one in 100 people         live with Parkinson’s
   over the age of 60                                                 disease,1 million of
•	 Onset often occurs in patients’ late 50s, but patients have        them in the U.S.
   been diagnosed as early as age 18
•	 The average patient lives with this chronic, degenerative
   disease for decades
•	 Because frequency of Parkinson’s disease increases with
   age, the number of Americans afflicted is expected to rise                                          60,000
   considerably as our population ages                                                                 Americans are
Early intervention and treatment are essential but until a reliable
                                                                                                       diagnosed with
biomarker or predictor is discovered, diagnosis of the disease must                                    Parkinson’s disease
be based on clinical observation of symptoms, many of which may                                        each year
not appear until the disease has progressed.

The impact is broad, and the problems associated with the disease
are many. Patients with Parkinson’s disease cannot afford to          $23 billion:
wait for solutions that may take decades to evolve.                   the annual national
                                                                      medical care
At Rush, those solutions are within our grasp right now.              costs for patients
                                                                      with Parkinson’s

A World-Class Parkinson’s Disease Program

Rush — one of only three designated research centers for the Parkinson’s
Disease Foundation and one of the world’s oldest dedicated Parkinson’s disease
programs — is truly a leader in Parkinson’s disease treatment, research
and training.

The following is just a snapshot of what sets us apart:

•	 A wide array of targeted medical therapies and surgical treatments for
   motor and nonmotor symptoms from the disease’s earliest stages to moderate
   and advanced stages
•	 Pioneering expertise in the area of brain stimulation, a surgical advance to
   block the signals that cause Parkinson’s symptoms, often reducing the need
   for medication
•	 Complementary therapies and services, including a range of support groups
   and exercise programs designed to optimize the patient and family experience
•	 Patient access to clinical trials testing the latest treatments and new drug
   therapies to enhance care throughout every stage of disease progression
•	 A wide range of laboratory research with six skilled teams who use
   biochemical, cellular and animal models to study the underlying mechanisms
   of Parkinson’s disease
•	 A highly productive fellowship training program that has served as a
   model for American Parkinson’s disease fellowship training and is one of three
   programs responsible for training more than half of the world’s directors of
   Parkinson’s disease programs
Our Vision for the Future
The Rush Parkinson’s Disease Program is poised to develop
solutions for patients and their families.

With your support, Rush will approach the disease from
every angle:
•	 Expanding its program to establish the cellular and genetic
   mechanisms that unleash Parkinson’s disease
•	 Defining whether the disease starts inside or outside the brain
•	 Developing novel treatments to stop the disease as early as
•	 Providing new therapies focusing on both motor and
   nonmotor disease aspects at every stage of progression

Our vision is to crack the code of Parkinson’s disease, easing
its symptoms, slowing its progression and ultimately finding
a cure.

The highly skilled, highly committed team at Rush is
well-positioned to make significant advances.

We need your support to make this vision a reality.

                                                                     $9.1 million
Risk and Cause of Parkinson’s
Disease: Does Parkinson’s Disease
Start in the Gastrointestinal
Directors: Kathleen M. Shannon, MD, and Jeffrey H. Kordower, PhD

Parkinson’s disease slowly takes over the brain, killing dopamine-
producing cells and causing irreversible damage to the central
nervous system. But what if we could prove that Parkinson’s disease
originated elsewhere in the body and then stop it from reaching the
brain in the first place?

Working toward just that, researchers at Rush will expand upon their
recent groundbreaking studies suggesting that Parkinson’s patients
have increased intestinal permeability (or a leaky gut) and have
elevated levels of alpha-synuclein, a toxic protein that is considered
a hallmark of Parkinson’s disease, in the gastrointestinal tract years
before the disease manifests.

Rush will pursue the following:
•	 Establishing whether pathological expression of alpha-synuclein in
   the gut transfers to the brain and then causes the degeneration
   of dopamine-producing brain cells
•	 Determining which form of the alpha-synuclein protein transfers
   from the gut to the brain
•	 Identifying a core biomarker we can use to screen for the disease
   prior to the onset of symptoms
Genetic Underpinnings of
Parkinson’s Disease
Director: Deborah A. Hall, MD, PhD
Collaborator: Elizabeth Berry-Kravis, MD, PhD

The role of genetics is incredibly complex in a complicated disease
like Parkinson’s disease, potentially comprising multiple genes and
numerous environmental factors. Using advanced gene analysis,
researchers at Rush will attempt to identify a genetic “fingerprint” for
Parkinson’s disease, or a series of gene variations that are specific to
Parkinson’s. Identifying this fingerprint will help researchers refine the
mechanisms of disease, fine-tune current therapies and potentially
lead to treatments that slow, or even halt, disease progression.

Rush will pursue the following:
•	 Applying advanced genetic analysis to target 50 different areas of
   the genome that are linked to Parkinson’s so we can define the
   disease at the cellular level
•	 Studying the expression of RNA — a biological molecule that helps
   code, decode, regulate and express genes — in order to test how
   various treatment approaches affect Parkinson’s disease at the
   cellular level
Challenges in Parkinson’s Disease
Director: Jennifer G. Goldman, MD, MS
Collaborators: Brandon R. Barton, MD, MS; Bryan Bernard, PhD; Melany
Danehy, MD; Christopher G. Goetz, MD; and Glenn T. Stebbins, PhD

The nonmotor aspects of Parkinson’s disease — including cognitive
impairment, hallucinations, depression and impulse control disorders —
present major challenges for patients and their caregivers, but these issues
are often under-recognized or incompletely understood and lack optimal

To address these issues, Rush will build upon our expertise in these areas
as we develop a comprehensive, interdisciplinary clinical, research and
educational program focusing on the nonmotor challenges of Parkinson’s

Rush will pursue the following:
•	 Identifying clinical and biomarker predictors of cognitive decline in
   Parkinson’s disease, including specific brain changes detected with
   advanced neuroimaging techniques and genetic or neurochemical
   signatures identified with blood and cerebrospinal fluid analyses
•	 Pinpointing the underlying cellular and neuropathological mechanisms of
   cognitive impairment and dementia in Parkinson’s disease
•	 Conducting clinical research to effectively treat cognitive impairment and
   dementia and prevent onset of mild cognitive impairment in Parkinson’s
•	 Using advanced neuroimaging techniques to identify changes in the brain
   associated with hallucinations and determine whether treatments will
   reverse the hallucination symptoms and changes on brain scans
•	 Developing clinical and electrophysiological screening tools to identify
   patients with impulse control disorders, as well as protocols for new
   treatment interventions
New Medical and Surgical Therapies
Directors: Katie Kompoliti, MD; Leo Verhagen, MD, PhD;
Roy A.E. Bakay, MD; and Brandon R. Barton, MD

Already one of the world’s premier sites for Parkinson’s research, Rush
will expand its capacity to carry out early-phase clinical trials and
provide patients with a wide range of new treatments and surgical

The expanded research program will serve as a “research home” for
patients, addressing both motor and nonmotor aspects and all phases
of disability. This research home will enable patients to participate in
clinical trials throughout the course of their illness and benefit from
the most promising treatments before they become widely available.

Rush will pursue the following:
•	 Creating a clinical research unit, enabling Rush to conduct early-
   phase clinical trials to determine safety, tolerability and efficacy
   of new drugs and positioning Rush as a leading site for the initial
   evaluation of new therapies
•	 Providing a wide array of research programs applicable to both
   motor and nonmotor aspects of the disease and tailored to
   all disease phases; options will include pharmacological and
   nonpharmacological treatments and a range of minimally invasive
   surgical options
•	 Using advanced imaging techniques to develop more precise, more
   effective and gentler approaches for deep brain stimulation (DBS)
   and other surgical therapies, while minimizing side effects
•	 Expanding our already highly respected surgery program in order
   to continue providing patients with the most leading-edge clinical
   and research opportunities, including deep brain stimulation, gene
   transfer and cell-based therapies
Exercise and Parkinson’s Disease:
University Research and Community
Director: Cynthia L. Comella, MD
Collaborator: Deborah A. Hall, MD, PhD

Clinicians in the Rush Parkinson’s Disease Program — home to the first
controlled study of exercise as a therapy for Parkinson’s disease — already
know that exercise lessens the severity of motor symptoms associated with
Parkinson’s disease. What we don’t know is why or what amount of exercise
is optimally effective.

Rush will expand its work with exercise as a highly effective therapy for
Parkinson’s patients, studying the science behind its positive effects and
making targeted exercise therapy more widely available and more convenient
for patients.

Rush will pursue the following:
•	 Implementing a multicenter study to assess how and why physical exercise
   impacts the clinical course of Parkinson’s disease
•	 Developing a clinical exercise program through which movement
   disorders, motor control and rehabilitation specialists take a
   comprehensive approach to exercise that addresses the needs of individual
   Parkinson’s patients
•	 Launching a community-based training program for physical and
   occupational therapists, aimed at making effective exercise therapy more
   widely available to Parkinson’s patients at convenient locations closer to
Gene Transfer and Stem Cell
Treatments of Parkinson’s Disease
Directors: Jeffrey H. Kordower, PhD, and Christopher G. Goetz, MD

As Parkinson’s disease progresses, dopamine-producing cells are
killed. By combining two of the most promising nonpharmacological
approaches — therapeutic genetic manipulation and stem cell
treatments — researchers at Rush hope to enhance dopamine
production in the brain.

While current Parkinson’s treatments cannot yet reverse progressive
cell death, this new combined therapy offers the very realistic
outcome of converting patients’ own cells or implanting new cells to
therapeutically undermine the progression of Parkinson’s disease.

Rush will pursue the following:
•	 Developing models in which to test gene transfer and stem cell
   treatments’ ability to reverse and forestall Parkinson’s before
   moving these techniques into human clinical trials
•	 Designing clinical trials for Parkinson’s patients using the best-
   established and safest gene transfer and/or stem cell therapies
•	 Exploring related and more novel approaches, including the use
   of vaccines and antibody-targeting toxic brain proteins, and
   preparing to translate these approaches from the lab to the clinic
                                                     Achieving the Vision
                                                      Patients and families affected by Parkinson’s disease need more effective
                                                      solutions. With your support, they won’t have to wait much longer.
                                                      Today, new scientific understanding of Parkinson’s disease is coalescing
                                                      into immense opportunity. Nowhere is this more true than at Rush.

                                                      We are on the verge of meaningful discoveries that could forever
                                                      change the way we treat — or even prevent — Parkinson’s disease.
                                                      We’re closer than ever before to pinpointing what causes the disease
                                                      and why; to diagnosing and intervening earlier, potentially before
                                                      symptoms appear; to more effectively treating Parkinson’s, improving
                                                      patients’ quality of life; and to ensuring that these breakthroughs
                                                      will continue to grow with the next generation of Parkinson’s disease
                                                      neurologists. We know how to make significant advances — your
                                                      partnership can be the catalyst that gets us there.

                                                      Now is the time to make a meaningful investment in the future
                                                      of Parkinson’s disease treatment. Your support will transform
                                                      the Rush Parkinson’s Disease Program and the very trajectory of
                                                      Parkinson’s disease treatment worldwide. Please participate in our
                                                      vision by investing today.

A generous gift from Khurram and Sameera Hussain is allowing researchers at Rush to shed light on previously unstudied non-
motor effects of Parkinson’s disease, as well as the link between Parkinson’s disease and the gastrointestinal system. Allocated to
both research and fellowship training, their support is also helping to educate the next generation of physician-researchers in the
area of movement disorders.
                     PLEASE CONTACT:

                    Bernadette O’Shea
              Senior Director of Development
              1700 W. Van Buren St., Suite 250
                  Chicago, IL 60612-3244
                      (312) 942-8710