title: GM1 ganglioside in Parkinson's disease: Pilot study of effects on dopamine transporter binding.
abstract: OBJECTIVE
  GM1 ganglioside has been suggested as a treatment for Parkinson's disease (PD), potentially having symptomatic and disease modifying effects. The current pilot imaging study was performed to examine effects of GM1 on dopamine transporter binding, as a surrogate measure of disease progression, studied longitudinally.
  
  
  METHODS
  Positron emission tomography (PET) imaging data were obtained from a subset of subjects enrolled in a delayed start clinical trial of GM1 in PD [1]: 15 Early-start (ES) subjects, 14 Delayed-start (DS) subjects, and 11 Comparison (standard-of-care) subjects. Treatment subjects were studied over a 2.5 year period while Comparison subjects were studied over 2 years. Dynamic PET scans were performed over 90 min following injection of [(11)C]methylphenidate. Regional values of binding potential (BPND) were analyzed for several striatal volumes of interest.
  
  
  RESULTS
  Clinical results for this subset of subjects were similar to those previously reported for the larger study group. ES subjects showed early symptomatic improvement and slow symptom progression over the study period. DS and Comparison subjects were initially on the same symptom progression trajectory but diverged once DS subjects received GM1 treatment. Imaging results showed significant slowing of BPND loss in several striatal regions in GM1-treated subjects and in some cases, an increased BPND in some striatal regions was detected after GM1 use.
  
  
  CONCLUSIONS
  Results of this pilot imaging study provide additional data to suggest a potential disease modifying effect of GM1 on PD. These results need to be confirmed in a larger number of subjects.
authors.affiliation: Department of Pathology, Anatomy and Cell Biology and Parkinson's Disease Research Unit, Thomas Jefferson University, Philadelphia, PA 19107, United States. Electronic address: jay.schneider@jefferson.edu.
authors.forename: Jay S
authors.initials: JS
authors.lastname: Schneider
authors.affiliation: Dept. of Neurology, University of Pittsburgh School of Medicine and Pittsburgh VAMC, Pittsburgh, PA 15213, United States.
authors.forename: Franca
authors.initials: F
authors.lastname: Cambi
authors.affiliation: Division of Neurology, Lankenau Medical Center, Wynnewood, PA 19096, United States.
authors.forename: Stephen M
authors.initials: SM
authors.lastname: Gollomp
authors.affiliation: Division of Nuclear Medicine, Russell H. Morgan Department of Radiology and Radiological Science, Johns Hopkins School of Medicine, Baltimore, MD 21287, United States.
authors.forename: Hiroto
authors.initials: H
authors.lastname: Kuwabara
authors.affiliation: Division of Nuclear Medicine, Russell H. Morgan Department of Radiology and Radiological Science, Johns Hopkins School of Medicine, Baltimore, MD 21287, United States.
authors.forename: James R
authors.initials: JR
authors.lastname: Brašić
authors.affiliation: Department of Pharmacology and Experimental Therapeutics, Thomas Jefferson University, Philadelphia, PA 19107, United States.
authors.forename: Benjamin
authors.initials: B
authors.lastname: Leiby
authors.affiliation: Department of Pathology, Anatomy and Cell Biology and Parkinson's Disease Research Unit, Thomas Jefferson University, Philadelphia, PA 19107, United States.
authors.forename: Stephanie
authors.initials: S
authors.lastname: Sendek
authors.affiliation: Russell H. Morgan Department of Radiology and Radiological Science, Johns Hopkins University School of Medicine, Baltimore, MD 21287, United States; Department of Psychiatry and Behavior Sciences, Johns Hopkins University School of Medicine, Baltimore, MD 21287, United States; Solomon Snyder Department of Neuroscience, Johns Hopkins University School of Medicine, Baltimore, MD 21287, United States.
authors.forename: Dean F
authors.initials: DF
authors.lastname: Wong
mesh_terms: D000328:Adult; D000368:Aged; D000369:Aged, 80 and over; D000978:Antiparkinson Agents / Q000627:therapeutic use*; D002247:Carbon Isotopes / Q000493:pharmacokinetics; D003342:Corpus Striatum / Q000000981:diagnostic imaging / Q000187:drug effects; D050483:Dopamine Plasma Membrane Transport Proteins / Q000378:metabolism*; D018765:Dopamine Uptake Inhibitors / Q000493:pharmacokinetics; D005260:Female; D005500:Follow-Up Studies; D005677:G(M1) Ganglioside / Q000627:therapeutic use*; D006801:Humans; D007091:Image Processing, Computer-Assisted; D008279:Magnetic Resonance Imaging; D008297:Male; D008774:Methylphenidate / Q000493:pharmacokinetics; D008875:Middle Aged; D010300:Parkinson Disease / Q000000981:diagnostic imaging / Q000188:drug therapy* / Q000473:pathology; D010865:Pilot Projects; D049268:Positron-Emission Tomography; D011485:Protein Binding / Q000187:drug effects; D013997:Time Factors
chemical_list: D000978:Antiparkinson Agents; D002247:Carbon Isotopes; D050483:Dopamine Plasma Membrane Transport Proteins; D018765:Dopamine Uptake Inhibitors; D008774:Methylphenidate; D005677:G(M1) Ganglioside
keywords: Caudate; Dopamine transporter; GM1 ganglioside; PET; Parkinson's disease; Putamen