Newsroom Archives: Press Releases

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.

ABOUT BEYOND BATTEN DISEASE FOUNDATION
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 www.beyondbatten.org.

For Immediate Release
Contact: Mary Beth Kiser – 512-275-2600 – mbkiser@beyondbatten.org
Amy Phillips – 614-657-7707 – amy.r.phillips1@gmail.com

Columbus Family Creates Fund to Support Research for Fatal Childhood Illness: Beyond Batten Disease Foundation Announces the Addition of Hugs for Hudson Fund, Founded by the Family of Hudson Phillips.

Austin, Texas – April 6, 2016 – BBDF is proud to welcome the Hugs for Hudson Fund. BBDF, the world’s largest funder of juvenile Batten disease research, is able to make informed decisions about research projects that have the highest likelihood of becoming therapies. When a fund is created through BBDF, founders are offered an array of projects that are in line with their goals of driving research to finding treatments and discovering a cure, and are able to apply funding to the research areas that interest them the most.

October 19, 2015 – Austin, TX – Beyond Batten Disease Foundation (BBDF) has begun funding Susan Cotman, PhD, an Assistant Professor of Neurology at Harvard Medical School and Assistant in Neuroscience at the Massachusetts General Hospital. The focus of Dr. Cotman’s study is to determine the cellular location and function of the CLN3 protein. Although the CLN3 gene and associated mutations responsible for juvenile Batten disease were discovered 20 years ago, the primary function of the protein remains elusive. Utilizing recent advances in proteomics and biochemistry along with new antibodies and cell models developed by BBDF, Dr. Cotman hopes to better understand the CLN3 protein.

The successful completion of this project will substantially accelerate juvenile Batten disease research because key reagents and information for the study of CLN3 function and dysfunction will be established. Dr. Cotman’s work will increase our understanding of CLN3, a critical step in the path towards the creation of rational, targeted therapies.

ABOUT BEYOND BATTEN DISEASE FOUNDATION

Beyond Batten Disease Foundation (BBDF) is the world’s largest organization dedicated to funding research for a treatment or cure for juvenile (CLN3) Batten disease. Since its inception in 2008, BBDF has applied over $15.5 million towards juvenile Batten disease research by direct donations, leveraging donor-funded successes, and forming 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. For more information, visit www.beyondbatten.org

Austin, TX – July 15, 2015 – Beyond Batten Disease Foundation (BBDF) is pleased to announce Craig Benson’s appointment to the Jan and Dan Duncan Neurological Research Institute (NRI) International Advisory Board. Consistent with BBDF’s strategy and goals to connect our research with top-ranked institutions, Craig is eager to join the NRI in its mission.

The NRI at Texas Children’s Hospital aims to reduce the temporal and conceptual gap between initial gene discovery and clinical applications by providing the context, core services, and community to support interdisciplinary collaborations between physicians, scientists, and patients. Designed to advance the understanding of neurological disorders, such as Batten disease, through basic and translational research and ultimately mitigate the impact of these devastating disorders on human life, the NRI has been integral in BBDF’s success.

Austin, Texas – June 9, 2015 – Beyond Batten Disease Foundation (BBDF) announces groundbreaking research published in Nature Cell Biology. The lysosome biology research is leading the way to a cure for juvenile Batten Disease.

“We think this research is our strongest candidate for therapeutic development for juvenile Batten Disease and understanding lysosome biology is the way to find a cure for this devastating disease,” said Danielle M. Kerkovich, PhD, BBDF Principal Scientist.

Austin, Texas – May 12, 2015 – Beyond Batten Disease Foundation has become a participating organization of The Research Acceleration and Innovation Network (TRAIN), a program of FasterCures, bringing the total number of venture philanthropies in TRAIN to 80.

FasterCures established TRAIN in 2005 to connect innovators in the disease research space with the vital resources, tools and relationships to catalyze development of new therapies and cures. Through the network, innovation in one disease area is translated to another in order to achieve treatment breakthroughs for all.

“We are pleased to expand our fight against juvenile Batten disease by becoming a partner in TRAIN to transform juvenile Batten disease research and accelerate our timeline to a cure,” said Danielle M. Kerkovich, PhD, BBDF Principal Scientist.