Charlotte and Gwenyth Gray Foundation wrote -
We are working with the world’s leading researchers studying gene therapy, cellular therapy and small molecule approaches to treat and cure a fatal neurodegenerative brain disease that affects young children. Our gene therapy research has progressed through efficacy and safety studies and we are now facing the critical vector production stage. The vector production costs exceed $500,000 and must be successfully completed before garnering approval from the FDA to initiate human clinical trials.
OUR GOAL: The biggest obstacle for treating any brain disease is the inability of nearly all drugs and treatments to cross through the blood brain barrier. Like a fortress, the blood brain barrier prevents the entry of external compounds into the brain. Because of this barrier, potentially lifesaving treatments aren’t able to reach their intended target.
But now, thanks to the truly groundbreaking research supported by the Foundation, scientists have discovered an ingenious way to overcome this barrier. This method relies on nature itself and on the behavior of a particular virus called Adenovirus or AAV. Through millions of years of evolution, AAV has figured out ways of infecting cells, including the brain, and now scientists have devised methods to harness this power while removing its infectivity. The vector production process you are supporting makes it possible to deliver the corrective gene to the brain by inserting it into the AAV virus, allowing nature itself to deliver this potentially lifesaving technology.
OUR STORY: Just a few years after welcoming their two daughters into the world, Gordon and Kristen Gray are faced with the agonizing prospect that neither will live to become a teenager. Charlotte Gray was born just a few weeks before Christmas 2010 and developed at the pace of a typical baby and toddler – walking and talking, with an early passion for gymnastics, dancing and swimming. After Charlotte’s first full year of preschool, her parents noticed that she seemed to hit a plateau developmentally. In March 2015, after a steady stream of questions and tests, Charlotte was diagnosed with Batten disease (Late Infantile NCL Batten Disease CLN6). A geneticist explained that this rare neurodegenerative disorder would leave Charlotte blind, immobile and cognitively impaired, and ultimately, gone between the ages of 6 and 12. The Grays immediately had their younger daughter Gwenyth evaluated and genetic tests revealed the same devastating diagnosis. Due to its rarity, solutions for this presently incurable and fatal disease have received minimal research, focus and funding. Kristen and Gordon’s worlds were shattered, but they were unwilling to accept “there is no cure” for an answer and created The Charlotte and Gwenyth Gray Foundation to Cure Batten Disease to raise the money necessary to fund accelerated medical research to identify the most effective treatment options and find a cure.
THE PROGRESS: Since launching in June 2015, the foundation has raised almost $3 million and has initiated funding into promising gene therapy and small molecule therapy treatments. As with any medical research, progress comes with a price tag and additional funds are needed urgently to maintain the pace necessary to reach the ultimate goal of a full clinical program to save the lives of Charlotte, Gwenyth and all other children battling the disease.
WEBSITE: For more information on the foundation, please visit www.curebatten.org.