Lithium citrate reduces excessive intra-cerebral N-acetyl aspartate in Canavan disease

Mitra Assadi, Christopher Janson, Dah Jyuu Wang, Olga Goldfarb, Neeti Suri, Larissa Bilaniuk, Paola Leone

Research output: Contribution to journalArticlepeer-review

43 Scopus citations


Our group has previously reported the first clinical application of lithium in a child affected by Canavan disease. In this study, we aimed to assess the effects of lithium on N-acetyl aspartate (NAA) as well as other end points in a larger cohort. Six patients with clinical, laboratory and genetic confirmation of Canavan disease were recruited and underwent treatment with lithium. The battery of safety and efficacy testing performed before and after sixty days of treatment included Gross Motor Function Testing (GMFM), Magnetic Resonance Imaging (MRI) Proton Magnetic Spectroscopy (H-MRS) as well as blood work. The medication was safe without any clinical or laboratory evidence for toxicity. Parental reports indicated improvement in alertness and social interactions. GMFM did not show statistically significant improvement in motor development. H-MRS documented an overall drop in NAA which was statistically significant in the basal ganglia. T1 measurements recorded on MRI studies suggested a mild improvement in myelination in the frontal white matter after treatment. Diffusion Tensor Imaging was available in two patients and suggested micro-structural improvement in the corpus callosum. The results suggest that lithium administration may be beneficial in patients with Canavan disease.

Original languageEnglish (US)
Pages (from-to)354-359
Number of pages6
JournalEuropean Journal of Paediatric Neurology
Issue number4
StatePublished - Jul 2010
Externally publishedYes


  • Canavan disease
  • Lithium
  • NAA


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