We have some amazing news to share!

 

Anyone who doesn't believe in miracles should fly to Ohio… Just over a year ago, Gordon and Kristen Gray were told that both of their daughters wouldn't live to become teenagers. They were diagnosed with Batten disease and told it was fatal and no treatment or cure was available.Within six months, Charlotte Gray couldn't walk unassisted or sit up without falling. Her vocabulary, once extensive, had shrunk to 40 words. Batten disease had completely hijacked her CLN6 gene - and Charlotte was running out of time.

Thanks to the boundless generosity of countless supporters and donors like you, we are immensely proud to announce that hope was found for Charlotte in Columbus, Ohio at Nationwide Children's Hospital.

There, on an accelerated timeline, a team led by Dr. Brian Kaspar worked around the clock to produce almost 1,000 pages of safety and efficacy studies to gain FDA approval for a human clinical trial for patients with Batten disease CLN6. This therapy, which we were initially told was years away, employs the AAV9 virus to cross the blood-brain barrier and deliver a replacement CLN6 gene to the brain of Batten patients.

 

Just one week following FDA approval, Nationwide's Dr. Jerry Mendell, Dr. Emily De Los Reyes, and Dr. Samiah Al-Zhaidy made Charlotte the first recipient of this novel and historic type of gene therapy. Dr. Jill Weimer of Sanford Research also played a pivotal role in reaching this milestone.

There are no prior case studies to compare to, and the path to recovery will be difficult to track. But one month after receiving this innovative and ground-breaking investigational therapy, Charlotte is providing reason to be optimistic. Her younger sister, Gwenyth, was also recently enrolled in the trial.

In other words, there is hope - for Charlotte, Gwenyth, and the other children around the world whose parents have been told that Batten is both incurable and fatal and that gene therapy was "years away." Years are not a luxury afforded to children with Batten disease.

Reaching this extraordinary milestone in such an aggressive timeframe has been made possible by the donations, posts, blogs, lemonade stands, and prayers of everyone who rallied around the Gray Family and the Batten community upon learning of Charlotte's diagnosis last year. Thank you! We are forever grateful.

 

But our work is far from over. Charlotte has miles yet to go.

And the families of other Batten children need your help to gain access to the same kind of care. The Charlotte and Gwenyth Gray Foundation is committed to raising the funds necessary to bring this potentially life-saving treatment to all children who need it. Additional patients are being recruited, and it is expected that the first three patients will receive the therapy before June, and three additional children will be enrolled before the end of the year. In addition to sponsoring the clinical trial at Nationwide, we are offering scholarships to help with living and travel expenses for children enrolled in the trial and their families. We also continue to fund research into additional complementary approaches to treating the disease. We're seeking your support once again to keep this progress advancing and get treatment to all children who so desperately need it.

They deserve a chance at a miracle as well.

Details of the trial can be found at clinicaltrials.gov; CLN6 families are encouraged to contact Nationwide Children's Hospital for more information.

 

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