New developments in crossing the blood-brain barrier bring human trials closer
BOSTON, MA—April 6, 2011—Breakthroughs in crossing the blood-brain barrier could lead to new treatments for Tay-Sachs, Canavan and other neurodegenerative diseases.  The findings were presented Thursday at the National Tay-Sachs & Allied Diseases (NTSAD) Science Symposium by leading researchers working together to find a treatment.

New methods of delivering missing enzyme to the brain show improvement in animal studies. Human trials may begin as soon as next year for Tay-Sachs, one of nearly 100 lysosomal storage diseases.

“Several speakers described inflammation in the brain as a hallmark of all neurodegenerative diseases.  Therefore, treatments for Tay-Sachs disease could lend insights to advancing treatments for Alzheimer’s Disease and other diseases affecting the central nervous system, as well as the other way around,” said Dr. Miguel Sena-Esteves, Associate Professor of Neurology at the University of Massachusetts Medical School and the project manager of the Tay-Sachs Gene Therapy Consortium, an international collaboration of scientists committed to translating current gene therapy results into human clinical trials.

In a keynote address, Dr. Marc Patterson of the Mayo Clinic cited the personal and...