Source: PR Newswire
LEAPS Project to Advance Encapsulated Cell Delivery of GDNF
NEW YORK, July 1 /PRNewswire/ -- The Michael J. Fox Foundation for
Parkinson's Research (MJFF) announced that it has awarded a grant to a
research team led by Olle Lindvall, MD, PhD, of the University of Lund,
Sweden
to develop a neuroprotective therapy based on the implantation of
encapsulated
cells that produce the growth factor GDNF directly into the brain.
Awarded
under the Linked Efforts to Accelerate Parkinson's Solutions (LEAPS)
program,
the project will receive funding totaling approximately $3 million over
four
years, assuming all milestones are met.
This project was made possible through a lead gift from Anne and
Bernard
Spitzer with matching funds provided by members of the Foundation's Board
of
Directors.
"This project drives forward potentially breakthrough technologies on
two
fronts," stated Deborah W. Brooks, executive director of the Foundation.
"First, should encapsulated cell technology prove safe and effective, the
result would be a novel delivery mechanism with applications for a myriad
of
therapies for Parkinson's patients. In addition, the team will further
assess
the ability of GDNF to slow, halt or even reverse the degenerative
effects of
Parkinson's disease."
The Lindvall team's approach plans to take the encapsulated cell
technology (ECT) delivery of GDNF from a theoretical concept to a
therapeutic
reality. GDNF, or glial-derived neurotrophic factor, is recognized by
researchers for its potentially protective influence on the dopamine
neurons
that typically degenerate in Parkinson's disease. But because GDNF does
not
cross the blood-brain barrier, it cannot be given orally or by injection.
While still experimental, encapsulated cell technology, if successful,
would
enable localized, long-term sustained delivery of GDNF to the brain with
the
primary aim of protecting dopamine neurons and stimulating their
regeneration.
Prior studies of GDNF in Parkinson's disease have shown positive
effects
in animal models as well as in early clinical trials. Recently,
preliminary
results from a phase II trial employing another delivery system showed no
clinical improvement after six months. However, the ability of GDNF to
protect
dopamine neurons and stop the progression of the disease was not tested.
This
LEAPS project will help resolve and advance understanding of GDNF's
therapeutic potential as well as potential delivery system variables.
Full funding for the project is contingent upon the achievement of
predetermined scientific milestones. The team's first milestone is to
generate
human cell lines capable of steadily secreting small amounts of GDNF and
place
these cells in retrievable fiber capsules. Once created, these capsules
will
be tested to assess cell viability, level and duration of GDNF secretion
and
diffusion into the brain, along with general safety issues such as
inflammation and retrievability. In addition, the neuroprotective and
regenerative effect of this type of delivery of GDNF will be studied in
disease models. Should this therapy prove effective, 12 patients will be
enrolled in clinical trials in Sweden, Switzerland, Germany and the U.K.
and
assessed clinically for at least 24 months.
In addition to Dr. Lindvall, members of the team represent seven
academic
institutions, a research foundation and a private company, including:
* Patrick Aebischer, MD, PhD, Federal Institute of Technology (EPFL)
Lausanne, Switzerland
* Anders Bjorklund, MD, PhD, University of Lund, Lund, Sweden
* David Brooks, MD, DSc, FRCP, FMedSci and Paola Piccini, MRCP, MD,
Imperial College, London
* Wolfgang Oertel, MD, Philipps University, Marburg, Germany
* Niall Quinn, MD, FRCP, University College, London
* Jean-Guy Villemure, MD and Francois Vingerhoets, MD, Centre
Hospitalier
Universitaire Vaudois (CHUV), Lausanne, Switzerland
* Lars Wahlberg, MD, PhD, NsGene A/S, Ballerup, Denmark.
To date, The Michael J. Fox Foundation for Parkinson's Research has
funded
more than $45 million in research aimed at finding a cure for the
disease,
either directly or through partnerships. For more information on The
Michael
J. Fox Foundation for Parkinson's Research, visit
http://www.michaeljfox.org.
SOURCE The Michael J. Fox Foundation for Parkinson's Research
Web Site: http://www.michaeljfox.org
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