Group Title: Florida tomorrow
Title: McKnight Brain Institute
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Title: McKnight Brain Institute
Series Title: Florida tomorrow
Physical Description: Book
Language: English
Creator: University of Florida Foundation
Publisher: University of Florida Foundation
Place of Publication: Gainesville, FL
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Bibliographic ID: UF00083438
Volume ID: VID00011
Source Institution: University of Florida
Holding Location: University of Florida
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From the Director

It is a special time in the brain sciences.
My experience as executive director of the Evelyn F and William L. McKnight Brain Institute over the past several years has rein-
forced what I have learned in more than three decades as a research scientist and teacher: We are making great discoveries that will
reshape the future of health care, and the need to put these discoveries to practical use grows more urgent by the day.
Exciting new findings including groundbreaking discoveries made at UF tell us that the brain can repair itself after injury or
disease, but it needs assistance from a new field called "regenerative medicine."
A decade ago, scientists thought people were born with a fixed amount of irreplaceable brain cells. We now know our brains produce
cells and connections throughout adulthood. Furthermore, we have shown ordinary human brain cells may share the prized qualities
of self-renewal and adaptability associated with stem cells.
Unprecedented hope now exists to develop treatments for brain diseases, and the public shares this hope. Confidence in biomedical
research has never been stronger.
A major thrust of our efforts is to encourage neurogenesis the generation of brain cells and to find ways to protect existing cells.
By focusing on adult stem cells and exploring causes of disease, we aim to develop therapies to fight ailments such as Alzheimer's
disease, Parkinson's disease, multiple sclerosis, epilepsy, stroke, brain cancer, age-related memory loss and Huntington's disease.
Undoubtedly, the mission is challenging. But the university is equal to the task. For the sake of an aging population of Americans,
we must succeed. It will take expertise in the biological sciences, tissue engineering and clinical practice to accomplish our goals. It will
take strategic investment in scientists, clinicians and facilities. In addition, the financial support of friends, partners and donors from
the Gator Nation and beyond is needed to augment the funding our scientists earn through National Institutes of Health and other
competitive grants.
The reward will come when people are protected from Parkinson's, Alzheimer's, brain cancer and a host of other neurological dis-
eases, and when our own cells can be mustered to fight devastating neurological problems.
That is the McKnight Brain Institute's vision for Florida Tomorrow. Please, share it with us.

Dennis A. Steindler, Ph.D.
Executive Director, McKnight Brain Institute of the University of Florida

Slorida SomSrSoS

The Promise of Tomorrow
The University of Florida holds the promise of the future:
Florida Tomorrow a place, a belief, a day. Florida Tomorrow is
filled with possibilities. Florida Tomorrow is for dreamers and
doers, for optimists and pragmatists, for scholars and entrepre-
neurs, all of whom are nurtured at Florida's flagship university:
the University of Florida, the foundation of the Gator Nation.
What is Florida Tomorrow? Here at the McKnight Brain Institute,
we believe it's an opportunity, one filled with promise and hope.
It's that belief that feeds the university's capital campaign to raise
more than $1 billion.
The Florida Tomorrow campaign will shape the university, cer-
tainly. But its ripple effect will also touch the state of Florida,
the nation and the entire world. Florida Tomorrow is pioneering
research and spirited academic programs. It's a fertile envi-
ronment for inquiry, teaching and learning. It's being at the
forefront to address the challenges facing all of us, both today
and tomorrow.

Evelyn F. and William L. McKnight Brain Institute
Florida Tomorrow Campaign Goal

S. upport and Research $25 mill


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Florida Tomorrow is a place

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where the best researchers and clinicians in the world are rallying to

defeat even the most daunting brain disorders.

To Tame an Epidemic

They may not be the diseases that grab headlines, but move-
ment disorders such as Parkinson's are an epidemic, says Dr.
Michael Okun.
"These disorders aren't infectious, so people don't get scared
like they do about something like bird flu," he notes. "But the
reality is, they affect many more people worldwide than any of
the infectious diseases you hear about on the nightly news."
Okun, along with Drs. Kelly Foote and Hubert Fernandez,
co-directs UF's Movement Disorders Center at the McKnight
Brain Institute. As the population ages, he says, the burden
created by movement disorders is increasing.
In answer to the growing need, the McKnight Brain Institute is
forging an extraordinary center where patients have access to the
latest clinical trials 25 are already in progress as well as to
leading scientists investigating gene therapies, regenerative tech-
niques and drug treatments.
Scientists are optimistic. Using a commonly occurring virus to
insert corrective genes, MBI researchers have revitalized brain
cells and restored normal movements in primates with a drug-
induced form of Parkinson's disease. Similarly, they've used
gene therapy to completely reverse abnormal movements called

dyskinesias in laboratory rats, which is promising for patients
struggling with the flailing movements that occur during conven-
tional drug treatments for Parkinson's disease.
The next step in the evolution of therapies may involve the
introduction of genetically engineered stem cells into the brain
using the delivery viruses pioneered at UF. Stem cells, whether
they already live in our bodies or come from donors, have a natu-
ral ability to target and repair an injured site.
"The home run is a drug or combination of drugs that will, as
any cell starts to die in your brain, activate your own indigenous
stem cell population," says Dennis Steindler, executive director of
the McKnight Brain Institute.
With molecular biologists researching movement disorders
within arm's length of a patient clinic and drug developers, any-
thing is possible. Tremendous strides have already been made.
"We see children who are suffering so much they have been
given up for lost, and after treatment they are able to get up out of
a wheelchair and lead a much more normal life," Okun says. "The
amazing things you hear about happening very rarely happen
with some frequency around here."

Florida Tomorrow is a day

0 0

when people's own regenerative cells can be mobilized to treat

and cure whatever medical problems are ailing them.

Secrets of Regeneration

Scientists could learn a lot from "the squishies."
That's the nickname Dr. Edward Scott uses to refer to crea-
tures from starfish to salamanders with remarkable
regenerative capabilities.
"We're studying creatures that, if their spinal cords are cut,
will heal themselves and walk again," says Scott, a profes-
sor in the department of Molecular Genetics and Microbiology
and the director of the MBI's program of Stem Cell Biology and
Regenerative Medicine. "We don't regenerate nearly as well."
The Regeneration Project, headed by Scott and MBI Executive
Director Dennis Steindler, seeks to unlock the secrets behind
these animals' healing abilities and to use those findings in
human therapies.
"If a child loses a fingertip, it will grow back," Scott explains.
"After puberty, an injury like that is less likely to regenerate; and
if it happened as an adult, it wouldn't re-grow at all. But the fact
that a child can re-grow a fingertip suggests that some of those
pathways are probably still there."
A major thrust of the effort compares stem cells from simple
systems in invertebrates and non-mammalian vertebrates with
normal and diseased adult human stem cells.
MBI researchers have already discovered 100 genes in a sea
slug called Aplysia an important laboratory model of learn-

ing and memory similar to genes in humans associated with
all major neurological diseases, as well as more than 600 similar
genes that control development.
Scott hopes the project's research will lead to better treatments
for spinal cord injuries and diseases from Alzheimer's to macular
"Once we find specific instances where we don't regenerate
but these species do, we can find out what the differences are and
look at how to overcome them," he says.
Through annual summits and fellowships, the project will
bring together physicians, medical researchers, marine scientists
and biomedical engineers to uncover the mysteries of regenera-
tion and develop treatments based on their discoveries. Scott sees
promise in the project's multidisciplinary approach.
"We have marine scientists who may be working with a mate-
rials science engineer and a neurologist who's helping a patient
recover from a stroke. These are people who normally would
never be in the same room to gether, he says.
"In the next year and a half, I'm hoping to see our research
used in three different clinical trials. Seeing people helped by
research is what keeps me going."



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Florida Tomorrow is a belief ...

that nothing is impossible in terms of curing neurological diseases.

Battling Brain Cancer

Dr. William Friedman has always been dedicated to helping
patients fight the devastating effects of brain tumors. But when
his mother died of a brain tumor in 2003, he redoubled his efforts.
"Until that point, I had been focused on ways to give the very
best care to one patient at a time," says Friedman, chairman
of neurosurgery at UF's McKnight Brain Institute. "I realized I
wanted to try to do something that could result in a breakthrough
for hundreds of thousands of brain tumor patients."
Today, Friedman, along with MBI Executive Director Dennis
Steindler, helps oversee a statewide tissue bank of brain tumor
samples the most comprehensive of its kind in the country. UF
scientists believe the tissue, collected from patients at UF and four
sister institutions, will provide data for researchers searching for
treatments and cures.
"The major obstacle in brain tumor research is similar to the
major obstacle in other cancers: We don't have a clear under-
standing of the molecular basis of the tumors," Friedman
explains. "Understanding that puzzle will lead to the discovery of
much more effective treatments and cures. This is an opportunity
to do the molecular genetic research we believe will lead to the
next big breakthrough."
Groundbreaking discoveries have already been made. MBI
regenerative medicine scientists were the first to identify a cancer

stem cell population within human brain tumors, drastically shift-
ing the scientific world's understanding of brain tumor growth.
Further analyses of cancer stem cells promise to reveal how nor-
mal brain cells and brain tumor cells are generated.
About 190,000 Americans are diagnosed with brain tumors
each year, most frequently in children and older adults. Primary
malignant tumors those that begin and tend to stay in the
brain are particularly devastating because of their alarmingly
rapid assault. Many patients die within a year of diagnosis, some
within a few months.
Support recently arrived in the form of a $5 million gift from
the Fort Lauderdale-based Lillian S. Wells Foundation Inc. to
the Brain Institute's brain tumor therapy center now known
as the Preston A. Wells Jr. Center for Brain Tumor Therapy. The
contribution reinforces efforts to recruit world-class doctors and
scientists and test experimental therapies. Eight investigational
treatment protocols are under way.
"Our patients are coming to us when they are most in need of
help, support, someone who cares about them," Friedman says.
As someone who has experienced that heartache firsthand,
he can empathize. But when he and colleagues at the McKnight
Brain Institute look to the future of brain-tumor research at UF,
they can offer something more: hope.

Our Vision

of Tomorrow

Scientists at the Evelyn F and William L. McKnight Brain
Institute of the University of Florida are committed to winning
the battle against brain diseases and injuries.
Already our clinicians and scientists have helped patients chal-
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But to make an even greater impact on patients with neuro-
logical problems, we need to attract even more leading scientists
and ideas, and integrate them into the high-powered discov-
ery machine that is the McKnight Brain Institute. That's why
it is vital to receive support from the Gator Nation and any-
one whose life may have been touched by a friend or loved one
with a neurological disorder. We have begun major initiatives in
brain research for age-related memory loss, Alzheimer's disease,
movement disorders, stroke and chronic pain. The scope of the
projects at the McKnight Brain Institute is truly amazing.
We envision a $60 million expansion of the McKnight Brain
Institute facility to build a supercenterr" to study and treat
movement disorders, including Parkinson's disease and dys-
tonia. We are creating a place where people with neurological

problems come to receive the best, research-guided patient care
in the world.
We are assembling the top minds from UF and the world for
"The Regeneration Project," which will connect scientists who
work with adult human stem cells the building blocks that
exist within our brain, bone marrow and blood with scien-
tists who study how tissues and limbs develop in a variety of
organisms. The payoff will come when we find ways to turn on
long-silent genes to repair or replace lost organs, or find ways to
mobilize and reinforce the body's own supply of adult stem cells
to protect against or fight neurodegenerative diseases.
We are fighting brain cancer. New brain tumors arise in more
than 200,000 Americans each year. People in their 60s face the
highest risk each year. The Florida Center for Brain Tumor
Research at the Brain Institute is working with hospitals state-
wide to collect tissue taken during brain surgeries and share data
on brain tumors. These tumor specimens represent a treasure
trove for basic and clinical scientists who are conducting stud-
ies at the cellular and molecular level to understand how brain
tumors develop and grow, and how we might be able to use this
knowledge to disarm or even destroy them.
Gifts and pledges to the McKnight Brain Institute strengthen
our research, patient-care and educational activities. They may be
designated for endowments that support researchers, clinicians
or students, or to support equipment and facilities. Many gifts are
eligible for matching support from the state of Florida.
With your help, the McKnight Brain Institute of the University
of Florida will change the world.

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