Canavan Research

To date, there is no treatment or cure for Canavan disease but research is making exciting advances towards a treatment and a cure.  

Gene Therapy

The first clinical trial partially funded and approved by the Food and Drug Administration (FDA) and the National Institute of Health (NIH), in which a new and more potent viral vector is being used to transport the genes into the brains of Canavan children, is ongoing at the Robert Wood Johnson Medical Center. Gene Therapy

Stem Cells

Leading stem cell researchers are exploring the potential therapeutic effects of stem cells in Canavan disease. Stem cell therapy is still an emerging field; potential therapies or clinical trials using stem cell therapy are not immediate. Stem Cell Therapy

Acetate Supplement

Acetate supplement therapy has shown improvements in Canavan rats. Canavan disease causes a deficiency of acetate, an essential building block of myelin. Acetate supplement therapy attempts to replenish the deficiency and improve myelination. Acetate Supplement Therapy for Canavan


Read more about NTSAD-funded Canavan Research projects:

Links to Library Documents

 Canavan Review Article - Making the White Matter Matters: Progress in Understanding Canavan’s Disease and Therapeutic Interventions Through Eight Decades, Seemin S. Ahmed • Guangping Gao

 Canavan Gene Therapy - Final Progress Report, Dr. Gao

 Dr. Gao et al, Canavan Gene Therapy published paper

 Dr. Aryan Namboodiri, GTA Treatment in Tremor Rat Model of Canavan Disease: Effectiveness and Lack of Toxicity

 Maria Traka, Canavan research report (Canavan biochemical pathway)