BBDF is pleased to announce that funds are available to conduct research directly relevant to the juvenile or CLN3 form of Neuronal Ceroid Lipofuscinosis (JNCL, juvenile Batten disease), a rare, fatal, lysosomal neurodegenerative disorder that strikes children. Research is needed across a broad spectrum addressing the function of normal CLN3 protein and the underlying mechanisms of neural dysfunction and degeneration in its absence.
Investigators are invited to submit Letters of Intent in the following areas or submit ideas of their own. BBDF does not presently support proposals that fall into the category of social science, psychology or family services.
- Neuroscience/Neurobiology of Batten disease. Many questions in juvenile Batten disease beg investigation in neurobiology, neurochemistry, neurophysiology, neuropharmacology, etc. BBDF seeks proposals that answer key questions important for understanding disease pathogenesis to design effective treatments.
- CLN3 Gene and Protein. Loss of function of CLN3 protein is responsible for juvenile Batten disease and while the gene was isolated in 1995, the function of subsequent CLN3 protein is poorly understood. BBDF seeks proposals that focus on CLN3 gene expression, transcription, CLN3 protein structure, function, functional partners and regulation
- Drug Discovery/Design. While many questions remain regarding the pathogenesis of juvenile Batten disease, BBDF also seeks proposals that focus on target validation, development and testing of novel of screening assays, the testing of potentially disease-modifying lead compounds and medical chemistry on lead compounds. The goal of the foundation is to fund an unbroken and iterative spectrum of activity from basic discovery through clinical implementation.
- Translation Support. We are in an exciting time in juvenile Batten disease research. As the number of discoveries is increasing, their potential to be important drug targets is being explored, and the number of compounds available to evaluate for their potential to treat juvenile Batten disease is expanding. In order to take full-advantage of these opportunities to advance juvenile Batten disease research toward clinical trial success and commercial development, BBDF will help investigators with promising results obtain resources for advancing their research beyond basic discovery. Examples include engaging FDA consultants to prepare for Investigational New Drug status, reaching out to research methodologists and statisticians to improve clinical trial designs, and locating commercial partners
A note on research priorities:
In addition to scientific merit, proposals are also scored for their potential significance to provide information important for the development of therapies to treat the primary effects of juvenile Batten disease.
To see a list of our research partners, click here.