Beyond Batten Disease Foundation Announces New Funding for Metabolomics to Drive Biomarker Discovery in juvenile Batten disease


AUSTIN, TX –January 9, 2017—Beyond Batten Disease Foundation has launched into high gear in the new year to find ways to measure what is happening to a child’s brain in the throws of juvenile Batten disease. Children across the world are suffering from this devastating illness. The foundation has identified a potential treatment and is preparing to conduct a clinical trial this year.

Before one can conduct a clinical trial to test potential new treatments for a brain disease, one must identify harmless ways of checking up on brain cells and measuring their response to treatment. To ensure progress towards a trial is not slowed due to a lack of ways to measure brain cell responses in juvenile Batten disease, Beyond Batten Disease Foundation (BBDF) has awarded De Montfort and Oxford Universities more than $150,000 to address this problem. Investigators with a rich history of success using advanced analytic tools to measure treatment responses to cancer and other lysosomal storage diseases will apply proton (1H) NMR-linked metabolomic strategies to identify sequential changes in cell activity during disease. Once documented, these changes can be monitored during the course of a clinical trial to confirm that a drug is having its predicted effect or provide early warning signs that something is wrong. Oftentimes, measurements such as these are taken long before a patient would feel different or appear to respond to treatment.

“It is hard to discover what is happening inside individual brain cells. Unlike other parts of the body, we can’t perform biopsies or rely on blood tests to provide that information. It is very elusive.   We have awarded money to a team of researchers with 20+ years of experience and a history of success identifying trial-ready biomarkers so that we can conduct early determination of how effective treatment is while carefully monitoring safety,” said Danielle Kerkovich, PhD, Principal Scientist of the Beyond Batten Disease Foundation.

“We have high hopes because of the stellar records and years of experience of the investigators involved,” said Mary Beth Kiser, President and CEO of Beyond Batten Disease Foundation.

The award, titled “Identification of New Biomarkers for Metabolomics Classification of Juvenile Disease: A Drug-Targeting Strategy”, is part of BBDF’s portfolio to carve a path from here to a cure. The successful completion of this project has the potential to substantially accelerate efforts to treat juvenile Batten disease.

Beyond Batten Disease Foundation (BBDF) is the world’s largest nonprofit organization dedicated to funding research for a treatment and cure for juvenile (CLN3) Batten disease. Since its inception in 2008, over $16 million has been invested in research by leveraging donations, co-funding and strategic partnerships. BBDF is spearheading a unique, cohesive strategy, incorporating independent scientific resources and collaboration with related organizations to drive research in juvenile Batten Disease. Today there is a treatment in sight. BBDF funded research has discovered a drug that slows the progression of the disease in Batten models. The foundation has launched a 24 month $6 million campaign to advance the treatment to clinical trials. More information can be found at