Primate Study Demonstrates Safety and
Feasibility of Cell Transplantation Approach
Currently in Phase 1/2a First-in-Human Parkinson's Disease
Clinical Trial
SAN
DIEGO, July 29, 2024 /PRNewswire/ --
The Journal of Neurosurgery has published online a
study by the Wisconsin National Primate Research Center at the
University of Wisconsin, Madison
regarding a novel cell transplantation approach being used for
delivery of ANPD001, an autologous, dopaminergic neuronal cell
replacement under investigation by Aspen Neuroscience as a
potential treatment for Parkinson's Disease.
Study Demonstrates Safety &
Feasibility of Cell Transplant Approach Currently in Phase 1/2a
Parkinson's Clinical Trial
The study by the Wisconsin National Primate Research Center
demonstrated the safety and feasibility of the treatment approach
for ANPD001 in non-human primates. Aspen is currently investigating ANPD001 in
the ASPIRO trial, a first-in-human, open-label Phase 1/2a clinical
trial in people with moderate to severe Parkinson's
disease.
The study was also incorporated into Aspen's Investigational New Drug (IND)
application with the U.S. Food & Drug Administration (FDA),
enabling the company to proceed with the clinical trial of
ANPD001.
University of Wisconsin researcher
Marina Emborg, M.D, PhD, professor,
Department of Medical Physics, led the study. A previous study by
Dr. Emborg, and her colleagues, published in 2021, demonstrated
that an autologous neuronal replacement therapy reversed
Parkinsons-like symptoms in primates.
"The results of the study established that this approach can be
used to safely target neuronal replacement therapy with precision
to the brain and supports its clinical investigation as a procedure
for cell replacement therapy in neurodegenerative diseases,"
concluded Dr. Emborg.
"The study was an important step in our work to bring the
promise of a cell-replacement therapy to people with Parkinson's
disease," said Andrés Bratt-Leal, PhD, study co-author, Aspen
Neuroscience co-founder and senior vice president of research and
development. "The results were instrumental in opening our
first-in-human trial and informing how we deliver patients' own
cells."
ANPD001 begins with a small sample of the patient's own skin
cells, followed by reprogramming to induced pluripotent stem cells
(iPSCs) which can become any type of cell. The iPSCs are then
differentiated into dopaminergic neuron precursor cells (DANPCs),
which are transplanted under real-time MRI guidance into the
putamen region of the brain, replacing cells that were lost or
damaged due to disease.
"By the time of diagnosis, it is common for people with
Parkinson's to have lost the majority of dopaminergic neurons,
leading to progressive loss of motor and neurological function,"
explained Edward Wirth III, MD, PhD,
study co-author and Aspen's chief
medical officer. "To replace these lost cells, we must target a
very specific area of the brain with a high degree of surgical
precision. Utilizing the latest advances in intraoperative MRI
guided techniques, the patient's new cells are transplanted, a few
microliters at a time, to the exact area where they are most
needed."
Affecting more than one million Americans, PD is a
neurodegenerative disorder that causes walking and motor problems,
as well as impaired balance and coordination. Existing therapies
alleviate symptoms but do not treat the underlying disease process,
leading to a significant unmet medical need for those suffering
from this chronic condition.
About the ASPIRO Trial
The Autologous-derived Study of a Parkinson's Investigational
Regenerative therapy in an Open-label trial (ASPIRO) is a Phase
1/2a clinical trial to assess the safety, tolerability, and
potential efficacy of ANPD001 in patients with moderate to severe
Parkinson's disease. The primary study endpoint is safety and
tolerability of ANPD001. Secondary endpoints include improvement in
"on" time, when patients experience periods of symptom control, and
improvements in motor symptoms and quality of life based on
standard Parkinson's disease rating scales.
About Aspen Neuroscience
Aspen Neuroscience, Inc., headquartered in San Diego, is dedicated to autologous
regenerative medicine. The company's patient-derived iPSC platform
is used to create personalized therapies, a crucial step in
addressing diseases with high unmet medical needs, starting with
autologous neuron replacement for Parkinson's disease.
Aspen combines cell biology with the latest machine learning and
genomic approaches to investigate patient-specific, restorative
cell treatments. The company's unwavering commitment to quality is
evident in its best-in-class platform to create and optimize
pluripotent-derived cell therapies, which includes in-house
bioinformatics, manufacturing, and quality control.
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SOURCE Aspen Neuroscience, Inc.