Parents Fund Breakthrough Therapy That May Treat Daughters' Fatal Brain Disorder: 'We Were Told to Take Them Home and Watch Them Die'
"Until I know with certainty that we've arrested the progression of this disease and can help other families, I won't rest either," Gordon Gray tells PEOPLE
When producer Gordon Gray and his wife Kristen’s two young daughters were diagnosed with a rare and deadly brain disease in March 2015, the couple knew they needed a miracle.
One year after their daughters’ received the devastating diagnosis of Batten disease – a genetic degenerative brain disorder for which there is no cure – the Los Angeles, California, couple’s 5-year-old daughter Charlotte became the first patient in the world to enroll in a clinical trial for an experimental therapy that could save her life – and the lives of many others.
“We’re incredibly hopeful and optimistic for Charlotte, but we know this is something we’re going to continue to fight forever,” Gordon, 51, tells PEOPLE. “We’re in the middle of it. We want to help all the kids with Batten.”
The deadly brain disorder that affects both Charlotte and the couple’s 2-year-old daughter Gwenyth is caused by a mutated gene that disrupts the brain’s ability to dispose of waste. The build-up of waste damages brain cells, causing blindness, seizures, the loss of communication and motor skills and dementia. Children with the Grays’ particular form of Batten disease, CLN6, often die between the ages of 6 and 12.
When doctors came to this diagnosis, they told the Grays there was nothing that could be done to save their girls. “One of the doctors we spoke with said, ‘I would recommend that you live in a single-story house and prepare for wheelchairs and blindness,’ ” Gordon told PEOPLE last June.
It was a recommendation the producer, known for inspirational sports movies like The Rookie and Miracle, refused to accept.
“We were told to just take them home and watch them die,” Gordon says. “That was something that we just couldn t do.”
Instead, the couple sprang into action, launching the Charlotte and Gwenyth Gray Foundation to Cure Batten Disease and enlisting an incredible network of celebrities from Rihanna to Gwyneth Paltrow to help them reach their goal of raising $10 million to cure the deadly disease.
“We’ve received endless support from our communities,” says Kristen, 40. “With their help, we found the means to get this therapy started – not just for our children, but for other children, too.”
One year later, the foundation has raised an estimated $3.5 million to fund research for a cure. The funding helped establish a clinical trial for an investigative gene therapy at Nationwide Children’s Hospital. The investigative treatment replaces Batten sufferer’s mutated gene by delivering a new gene that is carried to the brain by a virus that is not harmful to humans.
“The most direct way to correct a genetic disease is to restore what is missing and that is precisely what we have done in the work with the first patient with this devastating disease enrolled in the trial,” explains Dr. Jerry R. Mendell, a lead investigator in the clinical trial.
Charlotte underwent the one-time procedure in March, and, while it’s too soon to make any official statements about the experimental therapy’s success, her parents believe she is showing improvement.
“From the second she was treated, we saw a glimmer in her eye,” says Kristen. “She was more engaged and connected and you could just tell that there was something there that had changed.”
Earlier this year, the Grays watched helplessly as their daughter began having difficulty walking and struggled to recall certain words.
Now, thanks to speech and occupational therapy, Charlotte seems to have already begun regaining what was lost. “She’s gradually starting to reconnect with her language skills,” says Gordon.
Last Friday, Gwenyth – who has not started to show any symptoms of the disease yet – became the second patient to enroll in the clinical trial and a third child with Batten CLN6 will enroll by June. The Grays are hoping to raise an additional $1 million to reach even more children.
“We want to continue to help other kids with Batten,” Gordon says. “We ‘re looking to raise one million dollars to honor the commitment that we made to help every child that is available for the clinical trial.”
Gordon says he and his wife remain hopeful that this therapy will halt the progression of their daughters’ disease and clear a pathway for treatment for other forms of Batten. But, because the therapy is so new, there’s no test to tell them the therapy has worked – they’ll simply have to wait and see.
“We spend every day looking for signs that it’s working,” Gordon says. “We probably torture the doctors because we have so many questions they can’t answer because this hasn’t been done before.”
For now, all the couple can do is watch their daughters and wait for signs that Charlotte is getting better and that Gwenyth continues to be symptomless.
“From the diagnosis to today feels like one long day,” Gordon says. “I’m grateful we were able to get our girls through the clinical trial in a short time, but it was still too long because this disease never rests.”
“Until I know with certainty that we’ve arrested the progression of this disease and can help other families, I won’t rest either.”