For the fourth consecutive year, Taylor’s Tale has made a significant financial commitment to the advancement of Batten disease research. We awarded a $79,654 grant at the annual Batten Disease Support & Research Association (BDSRA) conference in Chicago, Ill. on Saturday, July 31. 

The project we are supporting passed a thorough evaluation by BDSRA’s scientific peer review committee. It will be led by Sandra Hofmann, MD, PhD, of the University of Texas Southwestern Medical Center in collaboration with Jonathan D. Cooper, PhD, of the Institute of Psychiatry at King’s College, London. It is a continuation of a project begun in July 2007; Taylor’s Tale has been a major contributor since its inception, giving about $200,000. 

The work done by Dr. Hofmann over the past several years has yielded promising results. The goal of the project is to develop enzyme replacement therapy for infantile NCL, a form of Batten disease. During the course of this project, Dr. Hofmann has successfully produced PPT1, the enzyme that children with infantile Batten disease lack, and has also shown it to have characteristics that make it ideal for mass production. More recently, she has conducted mouse studies using the PPT1 enzyme, work that has also made significant progress. Dr. Hofmann is currently addressing the need to deliver the enzyme across the blood brain barrier, a common problem in the treatment of brain-based diseases such as Batten disease.  She is attempting to fuse the enzyme with various peptide (protein) combinations in order to chaperone the enzyme into the brain, where it is needed. 

Dr. Hofmann and Dr. Cooper will provide a six-month progress report in February 2011.