Palisadians Gordon and Kristen Gray delivered an update recently on the progress being made to fund research to treat and cure Batten disease. The couple’s two daughters Charlotte and Gwenyth were diagnosed with the rare and fatal neurodegenerative disease earlier this year. Here is the message they released during Thanksgiving week:
“On this week of Thanksgiving in the U.S., YOU have given us many reasons to be thankful.
Photo courtesy of #CureBatten
Through your support, we continue to work with the world’s leading researchers studying gene therapy, cellular therapy and small molecule approaches to treating Batten disease. Funded by a grant from the Foundation, our partner, The Kaspar Lab at The Research Institute at Nationwide Children’s Hospital, has been pioneering a potential gene therapy for this rare form of Batten disease, and has blazed through mouse efficacy and safety studies. Vector manufacturing is the critical next step in this process before garnering approval from the FDA to initiate human clinical trials and getting treatment to those who need it—the kids!
The biggest obstacle for treating any brain disease is the inability of nearly all drugs and treatments to cross through the blood brain barrier, which 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 corrective gene is delivered to the brain by inserting it into the AAV virus, allowing nature itself to deliver this potentially lifesaving technology.
The costs of vector production, which is necessary to accomplish this next step, exceed $500,000. You have taken us this far, and we know that with your support, we can raise this amount by the end of the year.
This holiday season we are thankful for the tens of thousands of people who stand with us and do not accept “no cure” for an answer. Help us give the gift of a cure. Is there anything greater?”
For more information or to donate, visit curebatten.org.
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