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)