The DOI matches its Crossref record; 2 of the 2 references it cites were found in a registry or an index.
Randomized, double-blind trial of glial cell line-derived neurotrophic factor (GDNF) in PD
Abstract
OBJECTIVE: To assess the safety, tolerability, and biological activity of glial cell line-derived neurotrophic factor (GDNF) administered by an implanted intracerebroventricular (ICV) catheter and access port in advanced PD. BACKGROUND: GDNF is a peptide that promotes survival of dopamine neurons. It improved 6-OHDA- or MPTP-induced behavioral deficits in rodents and monkeys. METHODS: A multicenter, randomized, double-blind, placebo-controlled, sequential cohort study compared the effects of monthly ICV administration of placebo and 25, 75, 150, 300, and 500 to 4,000 microg of GDNF in 50 subjects with PD for 8 months. An open-label study extended exposure up to an additional 20 months and maximum single doses of up to 4,000 microg in 16 subjects. Laboratory testing, adverse events (AE), and Unified Parkinson's Disease Rating Scale (UPDRS) scoring were obtained at 1- to 4-week intervals throughout the studies. RESULTS: Twelve subjects received placebo and seven or eight subjects were assigned to each of the other GDNF dose groups. "On" and "off" total and motor UPDRS scores were not improved by GDNF at any dose. Nausea, anorexia, and vomiting were common hours to several days after injections of GDNF. Weight loss occurred in the majority of subjects receiving 75 microg or larger doses of GDNF. Paresthesias, often described as electric shocks (Lhermitte sign), were common in GDNF-treated subjects, were not dose related, and resolved on discontinuation of GDNF. Asymptomatic hyponatremia occurred in over half of subjects receiving 75 microg or larger doses of GDNF; it was symptomatic in several subjects. The open-label extension study had similar AE and lack of therapeutic efficacy. CONCLUSIONS: GDNF administered by ICV injection is biologically active as evidenced by the spectrum of AE encountered in this study. GDNF did not improve parkinsonism, possibly because GDNF did not reach the target tissues--putamen and substantia nigra.
Overview
The claims and statistics of this paper haven’t been checked yet.
Run the check — free, no account needed. It reads the open-access text and checks the sources it cites.
Integrity signals
Registry
Cites
Statistics
Verification
Claims the paper makes
The claims and statistics of this paper haven’t been checked yet.
Run the check — free, no account needed. It reads the open-access text and checks the sources it cites.
Self-check
Percentages, sums and reported differences re-computed from the paper’s own numbers.
The self-check needs the paper’s full text.
Citations
Cites
Parkinsonism: onset, progression, and mortality
Hoehn, M. M., & Yahr, M. D. (1967) · Neurology · doi:10.1212/wnl.17.5.427 · Cited by 12,107
Functional recovery in parkinsonian monkeys treated with GDNF
Gash, D. M., Zhang, Z., Ovadia, A., Cass, W. A., Yi, A., Simmerman, L., Russell, D., Martin, D., Lapchak, P. A., Collins, F., et al. (1996) · Nature · doi:10.1038/380252a0 · Cited by 955
Research works printed but not linked: 14
- Gash DM, Zhang Z, Gerhardt GA. Neuroprotective and neurorestortative properties of GDNF. Ann Neurol . 1998; 44 (suppl 1): S121–S125.
- 10.1006/nbdi.1997.0151 · DOI · Paper page
- Zhang Z, Miyoshi Y, Lapchak PA, et al. Dose response to intraventricular glial cell line-derived neurotrophic factor administration in parkinsonian monkeys. J Pharmacol Exp Ther . 1997; 282: 1396–1401. · DOI
- 10.1002/mds.870090403 · DOI · Paper page
- 10.1002/1531-8249(199909)46:3<419::AID-ANA21>3.0.CO;2-Q · doi:10.1002/1531-8249(199909)46:3<419::aid-ana21>3.0.co;2-q
- 10.1016/0197-4580(95)02012-8 · DOI
- 10.1016/0014-4886(95)90045-4 · DOI · Paper page
- 10.1159/000017069 · DOI · Paper page
- 10.1002/ana.410360221 · DOI · Paper page
- 10.1177/107385840100700105 · DOI
- 10.1038/35002084 · DOI · Paper page
- 10.1006/exnr.1999.7175 · DOI
- Gill SS, Patel NK, O’Sullivan K, et al. Intra-parenchymal putaminal administration of glial-derived neurotrophic factor in the treatment of advanced Parkinson’s disease. Neurology . 2002; 58 (suppl 3): A241.Abstract.
- 10.1126/science.290.5492.767 · DOI · Paper page
Cited by
Balestrino, R., & Schapira, A. H. V. (2020) · European Journal of Neurology · doi:10.1111/ene.14108 · Cited by 1,508
Direct brain infusion of glial cell line–derived neurotrophic factor in Parkinson disease
Gill, S. S., Patel, N. K., Hotton, G. R., O'Sullivan, K., McCarter, R., Bunnage, M., Brooks, D. J., Svendsen, C. N., & Heywood, P. (2003) · Nature Medicine · doi:10.1038/nm850 · Cited by 1,290
Pardridge, W. M. (2007) · Drug Discovery Today · doi:10.1016/j.drudis.2006.10.013 · Cited by 1,191
Potential therapeutic uses of BDNF in neurological and psychiatric disorders
Nagahara, A. H., & Tuszynski, M. H. (2011) · Nature Reviews Drug Discovery · doi:10.1038/nrd3366 · Cited by 867
Obeso, J. A., Stamelou, M., Goetz, C. G., Poewe, W., Lang, A. E., Weintraub, D., Burn, D., Halliday, G. M., Bezard, E., Przedborski, S., et al. (2017) · Movement Disorders · doi:10.1002/mds.27115 · Cited by 859
“The preclinical data supporting this delivery approach was limited,(277) and this trial reported no efficacy or significant side effects.(278) A patient who came to autopsy failed to demonstrate any dopamine regeneration, likely a result of poor penetration of the trophic factor from the ventricular space.”
Self-citation
Metadata
- Authors
- Nutt, J. G., Burchiel, K. J., Comella, C. L., Jankovic, J., Lang, A. E., Laws, E. R., Lozano, A. M., Penn, R. D., Simpson, R. K., Stacy, M., Wooten, G. F., & the ICV GDNF Study Group*
- Venue
- Neurology
- Published
- 2003-01-14
- Volume
- 60
- Issue
- 1
- Pages
- 69-73
- ISSN
- 0028-3878 (ISSN-L)
- 1526-632X
- OpenAlex
- W2165526364
- Cited by
- 824
- References
- 2
- FWCI
- 33.13
- Type
- journal article
- Language
- en
- Open access
- closed
About these checks
Registry, retraction, Cites and statistics results are computed, not judged, with one exception: a printed reference that no index lists is read once by a model before it counts as a citation error. Claims are extracted by a model and kept only when their quote appears verbatim in the text; each cited source is then checked, with a model’s help, for whether it says what the claim relies on. Those verdicts can be wrong — read the claim’s quote and the cited source before relying on one.
Statistics and arithmetic are recomputed, not judged.
Confidence bands are shown only for fields calibrated on our annotated test set. None is calibrated yet, so no band is shown.
What counts as a claim ›How we measure confidence ›How we check this ›
Are you an author, or do you see a problem with this page? Request a review
How to cite
Cite CoChat for the checks on this page, and the paper for its own findings.
- This page
- CoChat. (2026). Citation check: Randomized, double-blind trial of glial cell line-derived neurotrophic factor (GDNF) in PD. https://app.cochat.ai/papers/10.1212/wnl.60.1.69
- The paper
- Nutt, J. G., Burchiel, K. J., Comella, C. L., Jankovic, J., Lang, A. E., Laws, E. R., Lozano, A. M., Penn, R. D., Simpson, R. K., Stacy, M., Wooten, G. F., & the ICV GDNF Study Group*. (2003). Randomized, double-blind trial of glial cell line-derived neurotrophic factor (GDNF) in PD. Neurology. https://doi.org/10.1212/wnl.60.1.69
Data sources
Bibliographic data: Semantic Scholar, OpenAlex, Crossref.
Contains information from the Semantic Scholar Academic Graph Datasets, made available under the ODC Attribution License (ODC-BY 1.0).
Semantic Scholar’s data is described in: Kinney, R., et al. (2023). The Semantic Scholar Open Data Platform. arXiv:2301.10140.
