Austin, Texas – Beyond Batten Disease Foundation (BBDF) and the New York Academy of Sciences (NYAS) are working together in a new and innovative way to find a cure for a rare disease. While diseases like cancer and Alzheimer’s are widely known, rare diseases like juvenile Batten lack the attention and resulting funds needed to research, develop, and implement treatments. Juvenile Batten even lacks awareness among the medical and research community itself.

To raise awareness among research thought leaders and focus their collective energies on a key topic in juvenile Batten disease research, BBDF Principal Scientist, Danielle Kerkovich, PhD, proposed a unique think-tank of top experts from the Parkinson’s, Alzheimer’s, cancer and Lysosomal Storage Diseases (LSDs) fields to focus on our common drug target—a cell’s lysosome. Lysosomes are the recycling centers of the cell playing vital roles in the removal of cellular waste and keeping critical cell systems in balance. In diseases like juvenile Batten, other LSDs and adult forms of neurodegeneration, lysosome dysfunction compromises multiple cell systems eventually resulting in cell death.

If researchers from these fields pool their resources and work together to identify therapeutic strategies to manipulate lysosome function, their collective results have the potential to benefit children with juvenile Batten, other LSDs, cancer patients and millions of adults with dementia. Cancer researchers, who search for ways to create lysosomal dysfunction to selectively destroy cancerous cells, bring critical perspectives and experience to the discussion.

At this working conference, academic and pharmaceutical researchers challenged one another to overcome hurdles faced while drug targeting the lysosome. The results of this 3 day workshop are being released in a new podcast that helps bring to light the need for and potential benefits of juvenile Batten disease research.

“By appreciating commonly shared research objectives across rare and common illnesses, we are saving money and shortening the timeline from here to cures for juvenile Batten and other devastating illnesses.” said Danielle M. Kerkovich, PhD, BBDF Principal Scientist.

Experts participating in the podcast include: Mr. T. Craig Benson and Dr. Danielle Kerkovich of the Beyond Batten Disease Foundation; Dr. Steven Walkley of the Albert Einstein College of Medicine; Dr. Rainer Kuhn of Evotec AG and the NCL Foundation, Germany; Dr. Matthew Miscenyi of Biogen Idec, Inc.; Dr. Forbes Porter of the National Institutes of Health; and Dr. Susan Cotman of Massachusetts General Hospital.

Disease research is suffering. Since 2004, the US National Institutes of Health (NIH) budget has decreased by more than 20 percent. In 2012 alone, 16 percent or 3,500 scientists lost their sustaining NIH research grants. For rare diseases like juvenile Batten, badly in need of attention in the best of financial times, losses in funding can destroy progress completely. Therefore, finding creative ways to pool collective resources, learn from one another, and leverage funding is critically important. We must work together.

A lack of attention, funding, and research for rare diseases is why Charlotte and Craig Benson established Beyond Batten Disease Foundation in August 2008 after their then five-year-old daughter, Christiane, was diagnosed with juvenile Batten disease. Juvenile Batten disease is a rare, inherited neurological disorder that strikes young children, first causing vision loss and seizures, then cognitive and motor impairment, and ultimately death by the late teens or 20s. BBDF is working tirelessly with organizations like the New York Academy of Sciences, to create a brighter future for Christiane and others faced with devastating illness.

“By 2009 we were able to award the largest single grant in the history of Batten disease, which was $2.5 million. We realize while we started out as an advocate for our daughter, we have become the voice for all children with Batten disease,” said Craig Benson, co-founder of the Beyond Batten Disease Foundation.

Listen to the full podcast here.

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 Batten disease. Batten disease is a rare, inherited pediatric neurological disorder, which begins with vision loss and seizures, followed by cognitive and motor impairment, and ultimately death by the late teens or 20s. Since its inception in 2008, BBDF has raised over $14.6 million for research through donations, co-funding, leveraging and partnerships. BBDF is spearheading a unique, cohesive strategy, incorporating independent scientific resources and collaboration with related organizations to drive research in Batten Disease. For more information, visit

Leave a Reply

Your email address will not be published. Required fields are marked *