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April 05, 2016; 86 (16 Supplement) April 18, 2016

PRE-CELL: Clinical and Novel Biomarker Measures of Disease Progression in a Lead-In-Observational Study for a Planned Phase 1 Trial of Genetically-Modified Mesenchymal Stem Cells Over-Expressing BDNF in Patients with Huntington’s Disease (S25.004)

Vicki Wheelock, Teresa Tempkin, Alexandra Duffy, Amanda Martin, Lisa Mooney, Ashok Dayananthan, Lorin Scher, Sarah Farias, David Swadell, Charles DeCarli, James Brunberg, Chin-Shang Li, Yu Liu, Mark Yarborough, Julie Stout, miriam Moscovitch-Lopatin, Steven Hersch, Kyle Fink, Geralyn Annett, Jan Nolta
First published April 4, 2016,
Vicki Wheelock
UC Davis Department of Neurology Sacramento CA United StatesUC Davis Department of Neurology Sacramento CA United States
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Teresa Tempkin
United States
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Alexandra Duffy
Sacramento CA United States
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Amanda Martin
UC Davis Department of Neurology Sacramento CA United StatesUC Davis Department of Neurology Sacramento CA United States
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Lisa Mooney
UC Davis Department of Neurology Sacramento CA United StatesUC Davis Department of Neurology Sacramento CA United States
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Ashok Dayananthan
Sacramento CA United States
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Lorin Scher
Department of Psychiatry & Behavioral Sciences University of California Davis Sacramento CA United States
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Sarah Farias
University of California, Davis Sacramento CA United States
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David Swadell
UC Davis Department of Neurology Sacramento CA United StatesUC Davis Department of Neurology Sacramento CA United States
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Charles DeCarli
Department of Neurology Sacramento CA United States
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James Brunberg
UC Davis Medical Center Sacramento CA United States
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Chin-Shang Li
UC davis Department of Public Helath Davis CA United States
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Yu Liu
UC Davis Department of Public Health Davis CA United States
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Mark Yarborough
UC Davis School of Medicine Sacramento CA United States
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Julie Stout
Monash University Melbourne Australia
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miriam Moscovitch-Lopatin
Harvard University Boston MA United States
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Steven Hersch
Massachsuetts General Hospital Charlestown MA United States
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Kyle Fink
Stem Cell Program and Institute for Regenerative Cures Sacramento CA United States
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Geralyn Annett
Stem Cell Program and Institute for Regenerative Cures Sacramento CA United States
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Jan Nolta
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Citation
PRE-CELL: Clinical and Novel Biomarker Measures of Disease Progression in a Lead-In-Observational Study for a Planned Phase 1 Trial of Genetically-Modified Mesenchymal Stem Cells Over-Expressing BDNF in Patients with Huntington’s Disease (S25.004)
Vicki Wheelock, Teresa Tempkin, Alexandra Duffy, Amanda Martin, Lisa Mooney, Ashok Dayananthan, Lorin Scher, Sarah Farias, David Swadell, Charles DeCarli, James Brunberg, Chin-Shang Li, Yu Liu, Mark Yarborough, Julie Stout, miriam Moscovitch-Lopatin, Steven Hersch, Kyle Fink, Geralyn Annett, Jan Nolta
Neurology Apr 2016, 86 (16 Supplement) S25.004;

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Abstract

Objective: To describe baseline characteristics and longitudinal rate of change in multiple clinical, novel biomarker and structural MRI measures for a cohort of subjects with early-stage Huntington’s disease (HD). Background: There are no existing disease-modifying therapies for HD. Mesenchymal stem cells (MSCs) engineered to overexpress brain-derived neurotrophic factor (BDNF) is a proposed therapeutic for neuroprotection in HD. PRE-CELL is the lead-in study for a future planned Phase I trial of MSC/BDNF in HD. Design/Methods: Subjects with genetically-confirmed early-stage HD, without dementia, unstable psychiatric symptoms, contraindications to MRI or neurosurgery were assessed every 6 months for 12 - 24 months. A linear mixed effects model was used to analyze the repeated measures of selected outcome measures in the cohort. Results: Forty-two subjects have been screened and 32 enrolled (38[percnt] females, mean age of 52, range, 23 - 74). The estimated annual change rates for selected clinical measures include: UHDRS Total Functional Capacity score, -0.7884 (p-value < 0.0001); Independence Score, -6.2546 (p-value < 0.0001); Total Motor Score 9.1038 (p-value < 0.0001); HD-Quality of Life score, 17.089 (p-value = 0.0078), E-COG total score, 6.6312 (p-value = 0.0035). Rates of change in the HD Cognitive Assessment Battery will be presented. Biomarker analysis indicates a strong linear relationship between serum and CSF mutant Huntingtin protein levels (correlation of 0.87774, p-value <0.0001). Analysis of serum and CSF BDNF levels will be presented. Structural MRI analysis shows significant reduction in striatal volume detected at 6 months. Conclusions: The PRE-CELL study has enrolled a cohort of subjects with early-stage HD and has characterized the rate of change in clinical, imaging and novel serum and CSF biomarker measures. This data will be used as a baseline for subjects who are candidates for the future planned Phase 1 trial once regulatory approval has been obtained.

Disclosure: Dr. Wheelock has nothing to disclose. Dr. Tempkin has received personal compensation for activities with Lundbeck Pharmaceuticals. Dr. Duffy has nothing to disclose. Dr. Martin has nothing to disclose. Dr. Mooney has nothing to disclose. Dr. Dayananthan has nothing to disclose. Dr. Scher has received personal compensation for activities with Lundbeck, Inc. as honorarium. Dr. Farias has nothing to disclose. Dr. Swadell has nothing to disclose. Dr. DeCarli has nothing to disclose. Dr. Brunberg has nothing to disclose. Dr. Li has nothing to disclose. Dr. Liu has nothing to disclose. Dr. Yarborough has nothing to disclose. Dr. Stout has nothing to disclose. Dr. Moscovitch-Lopatin has nothing to disclose. Dr. Hersch has nothing to disclose. Dr. Fink has nothing to disclose. Dr. Annett has nothing to disclose. Dr. Nolta has nothing to disclose.

Monday, April 18 2016, 3:30 pm-5:30 pm

  • Copyright © 2016 by AAN Enterprises, Inc.

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