Topics of the Week - Archive

January 4, 2008

Thought of the Week

We all have a few failures under our belt. It's what makes us ready for the successes.
-- Randy K. Milholland

Topic of the Week: Tay-Sachs Gene Therapy Consortium Update

Since forming last summer, the Tay-Sachs Gene Therapy (TSGT) Consortium has been busy working toward the goal of starting a clinical trial for Tay-Sachs and Sandhoff in the next three years. In fact, participating laboratories hired new personnel to accelerate the rate of progress. Currently the consortium is working to answer these (very simplified) questions:

• In mice, what is the relationship between the amount of vector (a vector transports the correct genetic code into the cells) and the therapeutic effect?
• In mice, which is more effective: a vector that carries the corrected Tay-Sachs and Sandhoff gene OR a different vectors for each the Tay-Sachs gene and the Sandhoff gene?
• Demonstrating effectiveness in larger animal models is an important step towards a clinical trial; do we get the same results in cats as in mice?

In the coming months, in collaboration with the Consortium, NTSAD will be sending a Natural History Survey to families and individuals affected by Tay-Sachs and Sandhoff. The survey will provide a detailed description of the diseases from the earliest symptoms to terminal stages. This is a vital component of the clinical trial because it provides a quantified baseline to compare the effectiveness of the gene therapy in patients.

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