Following is a listing of peer-reviewed publications related to Ceregene's R&D programs, including Parkinson's disease, Alzheimer's disease, Huntington's disease and ALS (Lou Gehrig's disease)
Parkinson's Disease - CERE-120 (AAV2-neurturin)
• CERE-120 Clinical Publications
Herzog, C.D., Brown, L., Kruegel, B.R., Wilson, A., Tansey, M.G., Gage, F.H., Johnson, E.M., Bartus, R.T.
Enhanced neurotrophic distribution, cell signaling and neuroprotection following substantia nigral versus striatal delivery of AAV2-NRTN (CERE-120)
Neurobiology of Disease, In Press (Doi:10.1016/j.nbd.2013.04.011), 2013
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Bartus, R.T., Baumann, T. L., Siffert, J., Herzog, C. D., Alterman, R., Boulis, N., Turner, D. A., Stacy, M., Lang, A. E., Lozano, A. M., Olanow, C. W.
Safety/Feasibility of Targeting the Substantia Nigra with AAV2-Neurturin in Parkinson's Patients
Neurology, 2013, 80;1698-1701.
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Bartus, R.T., Baumann, T.L., Brown, L., Kruegel, B.R., Ostrove, J.M., Herzog, C.D.
Advancing neurotrophic factors as treatments for age-related neurodegenerative diseases: developing and demonstrating “clinical proof-of-concept” for AAV-neurturin (CERE-120) in Parkinson’s disease.
Neurobiology of Aging, 2013, 34;35-61.
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Bartus, R.T.
Translating the therapeutic potential of neurotrophic factors to clinical ‘proof of concept': A personal saga achieving a career-long quest.
Neurobiology of Disease, 2012, 48:153-178.
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Baumann, T.L., Lang, A.E., Lozano, A.M., Olanow, C.W., Bartus, R.T.
AAV2-neurturin (CERE-120) and Parkinson's disease: The safety and feasibility of combined substantia nigral and putaminal stereotactic targeting via a Phase 1/2b clinical trial in advanced Parkinson's disease.
Abstract No.9, Movement Disorder Society 16th Intl. Congress, Dublin, Ireland. 2012
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Bartus, R.T., Herzog, C.D, Chu, Y., Wilson, A., Brown, L., Siffert, J., Johnson, E.M., Olanow, C.W., Mufson, E.J., Kordower, J.H.
Bioactivity of AAV2-Neurturin Gene Therapy (CERE-120): Differences Between Parkinson’s Disease and Nonhuman Primate Brains
Movement Disorders, 2011, 26:28-36.
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Marks, W.J., Bartus, R.T., Siffert, J., Davis, C.S., Lozano, A., Boulis, N., Vitek, J., Stacy, M., Turner, D., Verhagen, L., Bakay, R., Watts, R., Guthrie, B., Jankovic, J., Simpson, R., Tagliati, M., Alterman, R., Stern, M., Baltuch, G., Starr, P.A., Larson, P.S., Ostrem, J.L., Nutt, J., Kieburtz, K., Kordower, J.H., Olanow, C.W.
Gene delivery of AAV2-neurturin for Parkinson’s disease: a double-blind, randomized, controlled trial.
Lancet Neurology, 2010, 9:1164-1172.
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Marks W.J., Ostrem J.L., Verhagen L., Starr P.A., Larson P.S., Bakay R.A.E., Taylor R., Cahn-Weiner D.A., Stoessl A.J., Olanow C.W., Bartus R.T.
Safety and tolerability of intraputaminal delivery of CERE-120 (adeno-associated virus serotype 2-neurturin) to patients with idiopathic Parkinson's disease: an open-label, phase I trial.
Lancet Neurology, 2008, 7:400-408
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• CERE-120 Related Nonclinical Publications
Bartus, R.T.
Translating the therapeutic potential of neurotrophic factors to clinical ‘proof of concept’: A personal saga achieving a career-long quest.
Neurobiology of Disease, 2012, 48:153-178.
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Herzog, C.D., Bishop, K.M., Brown, L., Wilson, A., Kordower, J.H., Bartus, R.T.
Gene transfer provides a practical means for safe, long-term, targeted delivery of biologically active neurotrophic factor proteins for neurodegenerative diseases
Drug Delivery and Translational Research, 2011, 5:361-382.
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Bartus, R.T., Brown, L., Wilson, A., Kruegel B., Siffert, J., Johnson, E.M., Kordower, J.H., Herzog, C.D.
Properly scaled and targeted AAV2-NRTN (neurturin) to the substantia nigra is safe, effective and causes no weight loss: Support for nigral targeting in Parkinson's disease
Neurobiology of Disease, 2011, 44:38-52.
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Wilson, A., Herzog, C. D., Brown, L., Kruegel, B., Bartus, R. T.
Enhanced neuroprotection by neurturin (NRTN) in 6-OHDA rats following combined substantia nigral plus striatal delivery of AAV2-NRTN (CERE-120).
Abstract, Society for Neuroscience Meeting, 2010
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Herzog, C.D., Brown, L., Gammon, D., Kruegel, B., Lin, R., Wilson, A., Bolton, A., Printz, M., Gasmi, M., Bishop, K.M., Kordower, J.H., Bartus, R.T.
Expression, bioactivity, and safety 1 year after adeno-associated viral vector type 2-mediated delivery of neurturin to the monkey nigrostriatal system support cere-120 for Parkinson's disease.
Neurosurgery. 2009, 64:602-612.
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Herzog, C.D., Dass, B., Gasmi, M., Bakay, R., Stansell, J.E., Tuszynski, M., Bankiewicz, K., Chen, E.Y., Chu, Y., Bishop, K., Kordower, J.H., Bartus, R.T.
Transgene expression, bioactivity, and safety of CERE-120 (AAV2-neurturin) following delivery to the monkey striatum.
Molecular Therapy. 2008,16:1737-1744.
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Herzog C.D., Dass B., Holden J.E., Stansell J., Gasmi M, Tuszynski M. Bartus R.T., Kordower J.H.
Striatal delivery of CERE-120, an AAV2 vector encoding human neurturin, enhances activity of the dopaminergic nigrostriatal system in aged monkeys.
Movement Disorders, 2007, 22:1124-1132.
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Bartus, R.T., Herzog, C.D., Bishop, K., Ostrove, J.M., Tuszynski, M., Kordower, J.H., Gasmi, M.
Issues regarding gene therapy products for Parkinson's disease: the development of CERE-120 (AAV-NTN) as one reference point.
Parkinsonism and Related Disorders. 2007, 13:469-477
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Gasmi, M., Brandon, E.P., Herzog, C.D., Wilson, A., Bishop, K.M,, Hofer, E.K., Cunningham, J.J., Printz, M.A., Kordower J.H., Bartus, R.T.
AAV2-mediated delivery of human neurturin to the rat nigrostriatal system: long-term efficacy and tolerability of CERE-120 for Parkinson's disease.
Neurobiology of Disease, 2007, 27:67-76.
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Gasmi, M., Herzog, C.D., Brandon, E.P., Cunningham, J.J., Ramirez, G.A., Ketchum, E.T., and Bartus, R.T.
Striatal delivery of neurturin by CERE-120, an AAV2 vector for the treatment of dopaminergic neuron degeneration in Parkinson's disease.
Molecular Therapy, 2007, 15:62-68.
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Kordower, J.H., Herzog, C.D., Dass, B., Bakay, R.A.E., Stansell, J., Gasmi, M., Bartus, R.T.
Delivery of neurturin by AAV2 (CERE-120) mediated gene transfer provides structural and functional neuroprotection and neurorestoration in MPTP-treated monkeys.
Annals of Neurology, 2006, 60:706-715.
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• CERE-120 Related Publications by others
Lewis, T.B. and Standaert, G.D.
Parkinson’s Disease, Primates, and Gene Therapy: Vive la Differénce?
Movement Disorders, 2011, 26:2-3.
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Benabid, A.L.
Reflection and Reaction. Gene therapy for Parkinson's disease: do we have the cure?
Lancet Neurology, 2010, 9:1142-1143.
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Alzheimer's Disease - CERE-110 (AAV2-NGF)
• CERE-110 Related Clinical Publications
Bartus, R.T.
On Neurodegenerative Diseases, Models, and Treatment Strategies: Lessons Learned and Lessons Forgotten a Generation Following the Cholinergic Hypothesis.
Experimental Neurology. 2000, 163:495-529.
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• CERE-110 Related Nonclinical Publications
Herzog, C.D., Bishop, K.M., Brown, L., Wilson, A., Kordower, J.H., Bartus, R.T.
Gene transfer provides a practical means for safe, long-term, targeted delivery of biologically active neurotrophic factor proteins for neurodegenerative diseases
Drug Delivery and Translational Research, 2011, 5:361-382.
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Bartus, R.T. and Dean, R.L.
Pharmaceutical treatment for cognitive deficits in Alzheimer’s disease and other neurodegenerative conditions: exploring new territory using traditional tools and established maps.
Psychopharmacology, 2009, 202:15-36.
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Bishop, K.M., Hofer, E.K., Mehta, A., Ramirez, A., Sun, L., Tuszynski, M., Bartus, R.T.
Therapeutic potential of CERE-110 (AAV2-NGF): targeted, stable, and sustained NGF delivery and trophic activity on rodent basal forebrain cholinergic neurons.
Experimental Neurology. 2008, 211:574-84.
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Huntington's Disease - CERE-120 (AAV2-neurturin)
• CERE-120 Related Nonclinical Publications
Ramaswamy, S., McBride, J.L., Han, I., Berry-Kravis, E.M., Zhou, L., Herzog, C.D., Gasmi, M., Bartus, R.T., Kordower, J.H.
Intrastriatal CERE-120 (AAV-Neurturin) protects striatal and cortical neurons and delays motor deficits in a transgenic mouse model of Huntington's disease.
Neurobiology of Disease. 2009, 34:40-50.
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Ramaswamy, S., McBride, J., Herzog, C.D., Brandon, E., Gasmi, M., Bartus, R.T., Kordower, J.H.
Neurturin gene therapy improves motor function and prevents death of striatal neurons in a 3-nitropropionic acid rat model of Huntington's disease.
Neurobiology of Disease, 2007, 26:375-384.
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McBride, J.L., Ramaswamy, S., Gasmi, M., Bartus, R.T., Herzog, C.D., Brandon, E.P., Zhou, L., Pitzer, M.R., Berry-Kravis, E.M., Kordower, J.H.
Viral delivery of glial cell line-derived neurotrophic factor improves behavior and protects striatal neurons in a mouse model of Huntington's disease.
Proceedings of the National Academy of Sciences, 2006, 103:9345-9350.
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ALS (Lou Gehrig’s disease) - CERE-130 (AAV2-IGF1)
• CERE-130 Related Nonclinical Publications
Franz, C.K., Federici, T., Yang, J., Backus, C., Oh, S.S., Teng, Q., Carlton, E., Bishop, K.M., Gasmi, M., Bartus, R.T., Feldman, E.L., Boulis, N.M.
Intraspinal cord delivery of IGF-I mediated by adeno-associated virus 2 is neuroprotective in a rat model of familial ALS.
Neurobiology of Disease. 2009, 33:473-481.
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Lepore, A.C., Haenggeli, C., Gasmi, M., Bishop, K.M., Bartus, R.T., Maragakis, N.J., Rothstein, J.D.
Intraparenchymal spinal cord delivery of adeno-associated virus IGF-1 is protective in the SOD1G93A model of ALS.
Brain Res. 2007, 1185:256-265.
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Kasper, B.K., Llado, J., Sherkat, N., Rothstein, J.D., Gage, F.H.
Retrograde viral delivery of IGF-1 prolongs survival in a mouse ALS model.
Science. 2003, 3:839-842.
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