BY JULiE METTENBURG | PHOTOGRAPHY BY MARK MCDONALD
Two research projects at the KU Medical Center may provide
new hope for the treatment of spinal cord injuries: not only to
retain movement in patients’ arms and legs, but also to enable
them to use their hands, stand or even walk again.
An anonymous donor has provided $4 million to support the Spinal Cord Injury Repair Program
in developing two novel approaches in restoring nerve communication using microelectronics and
“To be able to stand up would be a major change
in quality of life for many patients,” said Randolph
J. Nudo, Ph.D., director of the Landon Center on
Aging and one of the program’s lead researchers.
“We want to go beyond that, but one step at a time.”
The donor, a quadriplegic following a spinal cord
injury several years ago, approached KU with a
desire to fund neuroscience research that might lead
to restored function after chronic spinal cord injury.
Smith and colleagues identified five potential projects
based on expertise in KU’s Institute for Neurological
Discoveries (IND) and relevance to the donor. They
narrowed the list to the two that provided the best
chances for improvement.
|One gift, many benefits
This research is inspired and
supported by an individual
donor, but it holds potential
promise for thousands of others.
spinal cord injury is costly,
devastating and, currently,
about 265,000 people in the
united states are living with
its effects, with about 12,000
new patients each year. it
affects primarily males who
average 40 years old. They face
reduced life expectancy and
immense continuing medical
costs. only a third of patients
ever successfully resume
employment, with just 11 percent
working one year after injury.
Peter G. Smith, Ph.D., director of the IND and
the Spinal Cord Injury Repair Program, said the
project brings researchers from basic science disciplines such as physiology, anatomy and pharmacology together with clinicians
in neurosurgery, neurology,
rehabilitation medicine and
more. “This is not just about
KU,” Smith said. “We’ve
brought in key collaborators
at K-State, Case Western
Reserve University, Harvard
University and the University of Washington. This is
about building the best possible research teams to solve
a very complicated problem.”
The brain-spinal cord
interface approach, led by
Nudo, uses microelectronics to provide an artificial
communication link from the brain to the spinal cord, a pathway that is severed
in spinal cord injury. The regeneration strategy, led by
Smith, is to discover a way to place new cells in the
spinal cord that can replace damaged pathways.
“Together, these short- and long-term fixes provide
the greatest hope for individuals with spinal cord
injuries,” Smith said. The early phases of the work
must be performed in animals in order to perfect
techniques and develop rigorous measurements to
determine if therapies are working. The challenge
now, for Nudo’s team, is mapping brain and spinal
cord areas to connect; for Smith’s team, it’s engineering the proper cells to replace injured spinal cord cells.
ANSWERS ON THE HEAD OF A PIN
The next time you curse your cell phone, think twice:
The same technology might hold the key to preserving
motion after spinal cord injuries.
Nudo has previously focused on developing therapies for stroke using neural prosthetics to bypass
damaged areas of the brain. This project brings that
approach to spinal cord injury.
When the neural pathways that connect brain to
limbs are severed, several structures remain intact: the
parts of the brain creating signals, the neurons below
the injury in the spinal cord that relay signals, and the
muscles that would receive the signals. Therefore, a
patient could retain basic motor function if implanted
electrodes could record the brain’s electrical signals
and send them past the damaged area, where they
could activate an external limb, stimulate a muscle
directly or stimulate neurons in the spinal cord.
“Our work is trying to put those two things
together,” Nudo said, “so someone literally would
think about moving a limb, using the same neurons
as before, and trigger the movement.”
Implantable devices must be very small. Ten years
ago, Nudo said, the technologies he’s using would
have required an entire rack of computers. With
microelectronics advances, his team can borrow from
resources like cell phone technology.
“Basically, we’re functionally reconnecting the
brain and spinal cord with electronic devices,” he
said. “We are designing circuits like a computer on
the head of a pin.”
A BRIDGE OF CELLS
Unlike nerves outside the central nervous system, the
nerves in the spinal cord cannot regenerate or repair
themselves. Smith’s team is working on a treatment
involving new cells with the ability to repair the
damage for good.
“A more permanent treatment would be reconnecting those wires, which means replacing dead cells,”
Smith said. “If we can discover the right kinds of
cells and the right technique to move them back into
the spinal cord, we can get them to create new pathways and restore function below the lesion.”
Smith’s team takes adult skin or blood cells and
genetically reprograms them into stem cells, which
can then become any other kind of cell. The IND
partners with KU’s High
Throughput Screening Laboratory in discovering drugs that
can coax these stem cells to
become the right type of neurons for repairing spinal injury;
testing in animals will ensure
they perform appropriately.
The goal is to take a patient’s
own easily obtainable cells,
turn them into stem cells, and then encourage them
to become cells that can be transplanted to repair a
spinal cord injury.
DONOR PARTNERSHIP DRIVES RESEARCH
This project represents an unusual way to fund
research. The donor had expressed interest in supporting research on two levels: He wanted to keep
his support local rather than send it to research
centers in other cities, and he wanted to partner with
a top-notch scientific institution able to conduct cutting edge research related to spinal cord injury.
KU’s Institute for Neurological Discoveries, a
new model developed with just this kind of purpose
in mind, was poised to work with him. To prepare
for potential opportunities, the IND formed in 2008
after identifying research strengths at the medical
center and affiliated regional institutions. Six specialties were identified addressing some 22 conditions,
spinal cord injuries among them. With appropriate
funding, Smith said, the IND could become preeminent in any of these areas of research.
He said relatively few institutions have adopted
this partnership model. “Donors have specific endpoints in mind, and we’ve developed specific milestones that we believe will take us toward those endpoints,” he said. “We’ve done this with the highest
level of scientific integrity, so it’s really good science,
but we also have catered to the needs of the patient.”
Nudo said the scientific community at KU is grateful for the opportunity to develop this program, which
could not have happened without this donor’s help.
“It brought together scientists and
clinicians who normally don’t work
together to work single-mindedly
on a project, and created the focus
for all of us to think about a single
goal,” he said. “It’s bringing a lot
of visibility to the IND and to KU
neuroscience in general.”
Smith said, “Suddenly, you’re
not working on a grant from the
National Institutes of Health. You’re working for
someone—a patient—and you understand the goals,
hopes and desires, the urgency with which they
would like to see some restoration of function.”
He said the current NIH funding situation is
unpromising. Many good projects are not funded,
which doesn’t tend to open up much new exploratory science.
However, donations like this one create opportunity for researchers to work on projects NIH might
deem too risky. And success would likely help KU
and the IND obtain future additional funding from
NIH, the Department of Defense and others, bringing new opportunities.
The IND was set up for just this purpose, Smith
said, with teams preassembled and resources in
place to respond rapidly. “This is the culmination of
exactly what we were trying to accomplish,” he said.
“It serves as a template for people as a way to get
involved: You can make a difference.”
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