This has been a busy summer at the Beyond Batten Disease Foundation (BBDF) and we would like to highlight some of our recent accomplishments:

Suzanne Kho, BBDF executive director, traveled to London to meet with representatives from Batten Disease organizations from seven other countries. At the meeting, the attendees voted to create the Batten Disease International Alliance to share information between member organizations. We are now working to formalize the structure of the alliance and have begun educating each other about our services and/or our research initiatives.   This will help prevent duplication of effort and maximize our efficiency.

The Batten Disease International Alliance

The New York Consortium on Membrane Protein Structure (NYCOMPS) is analyzing the structure of the CLN3 protein.  Dysfunction of this protein results in Juvenile Batten Disease, but little is understood about its actual structure.  Not only will this information elucidate the protein's function, but it is critical for drug design.  The BBDF, along with our colleagues at NCL-Stiftung in Germany, nominated the CLN3 protein for evaluation.  NYCOMPS was formed in 2005 under The Protein Structure Initiative of the National Institute of General Medical Sciences. 

We awarded a new grant to Dr. Jonathan Cooper at King's College London for his research project, Finding New Targets for Therapy: The Brain's Forgotten Cells in Juvenile Batten Disease. Dr. Cooper and his team will look at how sick glial cells affect the function and survival of nerve cells. This information is particularly important because it may open up new therapeutic avenues using drugs to specifically treat these glial cells. Funding for the project was provided by the Will Herndon Fund for Juvenile Batten Disease Research of the BBDF.

Dr. Danielle Kerkovich, principal scientist, and Amy Drew, a volunteer science writer, published a paper in the journal Cerebrum in follow-up to the Drug Discovery in Juvenile Batten Disease conference that we sponsored in February. Their paper, Designing a Plan for Drug Discovery in Rare Pediatric Neurodegenerative Disease, argues that to overcome the gap between basic research and clinical treatment development, researchers in academia and the pharmaceutical industry, supported by government agencies and nonprofits like the BBDF, must come together to share expertise and promote translational research. The full text of the article can be read HERE.