Topics of the Week - Archive

July 6, 2007

Thought of the Week

Never deprive someone of hope; it may be all they have.
-- H. Jackson Brown Jr.

Summing up the Research Development of NTSAD's 2007 Fiscal Year

This has truly been a very exciting year for the development of potential therapeutic approaches for the leukodystrophies!

• In February, the NTSAD Research Initiative matched the funds raised by several NTSAD Canavan families to award a grant to Aryan Namboodiri, PhD research project Preclinical Research toward Acetate Supplementation Therapy for Canavan Disease. The central theory of this project is that Canavan disease results in defective myelin because of a lack of acetate derived from N-acetylaspartate. This therapeutic approach would simply provide missing acetate in the form of an oral supplement. Mouse model results have been very promising. A primary goal of this project is to collect the necessary preclinical data to move forward to Phase I clinical trial in children.
  
  • Simplified: Acetate supplement therapy for Canavan disease could be a simple and inexpensive therapeutic approach administered orally in a supplemented infant formula.
  • Dr. Namboodiri will be submitting his 6-month progress report next month, watch future issues of the Topic of the Week for additional information.
    
• Last month Zymenex, a Danish biotech company, launched Phase II clinical trials of Metazym, an enzyme replacement therapy for Metachromatic Leukodystrophy (MLD). At this time the trials are only in Europe and any U.S. patients interested would need to relocate to Europe for 12 – 52 weeks. The Metachromatic Leukodystrophy Foundation is working closely with Zymenex to find a way to include U.S. patients in the trial but the substantial financial increase of adding a trial site in the U.S. continues to be the biggest obstacle.
  
• Researchers published the 3-dimensional shape of aspartoacylase, the enzyme deficiency which causes Canavan disease, in the January issue of Proceedings of the National Academy of Sciences of the USA . Enzymes fold into specific 3-D shapes which provide the enzyme with its functionality. Understanding the correct shape and function of aspartoacylase provides clues to how to make the enzyme work properly in children affected by Canavan disease.

Please, remember that you can call the office and speak with Diana or Kim with any concerns,
questions or suggestions - our door is always open!

Diana Pangonis
Interim Executive Director
Diana@ntsad.org
800-906-8723