TY - JOUR
T1 - Issues regarding gene therapy products for Parkinson's disease
T2 - The development of CERE-120 (AAV-NTN) as one reference point
AU - Bartus, Raymond T.
AU - Herzog, Christopher D.
AU - Bishop, Kathie
AU - Ostrove, Jeffrey M.
AU - Tuszynski, Mark
AU - Kordower, Jeffrey H.
AU - Gasmi, Mehdi
N1 - Funding Information:
All of the work involved with the development of CERE-120 was funded by Ceregene, Inc. The authors thank Lee Ann Forrest for help with the preparation of the manuscript.
Funding Information:
Portions of the clinical trials have been supported by the Michael J. Fox Foundation for PD (to RTB).
PY - 2007
Y1 - 2007
N2 - Purpose: To develop CERE-120 (AAV-NTN) as a novel therapy for Parkinson's disease (PD) that might restore function of degenerating dopamine neurons and prevent further degeneration. Scope: A nonclinical program demonstrated that NTN expression can be predictably controlled following CERE-120 administration, provides clear evidence of efficacy in numerous animal models and is safe at dose multiples that far exceed those required for efficacy. Preliminary, open label evidence in PD subjects offers corroborative support for these observations. Conclusions: CERE-120 may represent an important, novel therapy for PD, though the clinical data require confirmation with additional clinical tests, including an ongoing multi-center, double-blinded controlled trial.
AB - Purpose: To develop CERE-120 (AAV-NTN) as a novel therapy for Parkinson's disease (PD) that might restore function of degenerating dopamine neurons and prevent further degeneration. Scope: A nonclinical program demonstrated that NTN expression can be predictably controlled following CERE-120 administration, provides clear evidence of efficacy in numerous animal models and is safe at dose multiples that far exceed those required for efficacy. Preliminary, open label evidence in PD subjects offers corroborative support for these observations. Conclusions: CERE-120 may represent an important, novel therapy for PD, though the clinical data require confirmation with additional clinical tests, including an ongoing multi-center, double-blinded controlled trial.
KW - Disease progression
KW - Neuronal restoration
KW - Neuroprotection
KW - Neurotrophic factors
KW - Neurturin
UR - http://www.scopus.com/inward/record.url?scp=38949196405&partnerID=8YFLogxK
UR - http://www.scopus.com/inward/citedby.url?scp=38949196405&partnerID=8YFLogxK
U2 - 10.1016/S1353-8020(08)70052-X
DO - 10.1016/S1353-8020(08)70052-X
M3 - Article
C2 - 18267286
AN - SCOPUS:38949196405
SN - 1353-8020
VL - 13
SP - S469-S477
JO - Parkinsonism and Related Disorders
JF - Parkinsonism and Related Disorders
IS - SUPPL. 3
ER -