A group of Hollywood heavyweights including producers Marty Bowen, Michael De Luca, Scott Stuber and Rick Yorn will host a Call-A-Thon this Thursday at CAA headquarters. Their mission: to call everyone they know in hopes of shaking loose the $1 million that Miracle film producer Gordon Gray needs by October, to keep going with the gene therapy medical breakthrough that has arrested the deterioration of motor and verbal skills in his 5-year-old daughter Charlotte from Batten CLN6. That is the rare and fatal brain disease that befell Charlotte and also faces Gwenyth. The other daughter of Gray and wife Kristen at 3 also got the experimental gene therapy after she too was diagnosed with the disease. A third patient has also received the treatment, but the continuation and expansion of the treatment to other children will require more funding. That is where the support group of Hollywood insiders comes in, the group that has helped Gray galvanize support from the beginning, and have raised millions of dollars to get this far.
So if they call you Thursday, please pick up the phone. Others who want to donate can do so at www.curebatten.org.
Deadline has been writing about this odyssey since Gray first revealed the terrible diagnosis he and his wife received after their energetic and talkative daughter Charlotte began stumbling, and withdrawing as she lost motor and verbal skills. They were given the death sentence diagnosis by doctors and told there was nothing to be done except prepare for Charlotte to go blind, lose all motor and verbal functions and ability to feed herself, progressing to eventual death between ages 6 and 12. And, they were told, they should prepare to re-live the same nightmare, because Charlotte’s younger sister Gwenyth was also diagnosed with the rare brain disorder. The Grays refused to accept this shattering news, and by his own admission, Gray has hardly slept as he raced against time to find some way to arrest the development of his disease, watching every day as Charlotte eroded. A year later, as Deadline readers might remember, he found his potential miracle.
Last March, the girls were the pioneer recipients of the first gene therapy of its kind in humans, in a clinical trial begun at Nationwide Children’s Hospital in Columbus, OH. Charlotte was given the equivalent of a magic bullet: a virus infused with the healthy CLN6 gene that was missing from her brain, and which allows cells to purge built-up wastes and restore balance in the brain. That virus was introduced intrathecally into Charlotte’s spinal fluid, for a short ride to her brain. Gwenyth was given a preventive dose of the gene therapy clinical trial right after her sister. Gray told Deadline in late April that he was already seeing signs of a rebound in Charlotte. She didn’t show symptoms of the rare disease until age 4, so the Grays won’t know for certain about Gwenyth for another year. Right now, her father says she bounds around the house like a normal child full of energy and optimism.
Months later, the Grays are as cautiously hopeful as ever that this treatment is working: “Charlotte continues to give us hope and real signs to be incredibly optimistic,” Gray told Deadline. “She is not declining at all, and continues to expand her vocabulary by reclaiming a lot of the words she lost. Her motor coordination is not even close to where I would like it to be, though. It is still early, and her brain is still healing. She is in a special school, going to kindergarten, and she loves going to school. From a parent’s point of view, it feels like the decline has been arrested. But scientifically, I can’t really say that definitively because I don’t know the natural progression of the disease. My observation is that since she has been treated, she has not declined.”
As for Gwenyth? “From my perspective, she is completely asymptomatic, and she is attending school right now and is happy and seemingly perfectly healthy,” he said. “But she is only only 3 and it would be too soon to know. We are in the position of looking at Gwenyth and hoping we don’t see the decline, as we continue to look for improvement in Charlotte, who will turn 6 in December.”
Gray continues to develop projects under his Disney deal, but hasn’t made one in awhile as he tries to save his daughters. It is laudable that the studio continues to back him. Maybe his own story would make a pretty good Disney film, but Gray said there is so far too much work to do to simply pronounce that fairy tale ending that was part of so many of the inspiring sports-themed movies he has produced so far in his career. Gray has relied heavily on the support group that has helped him raise millions of dollars to get this far, and hopes they can do it again to keep it going. Gray means to keep his promise to extend this treatment to other children suffering from the rare disease, and it will take continued funding to make that a reality.
“I’m fortunate, to the extent I would use the word fortunate, to have great friends who’ve stepped up to help and have made something incredibly difficult a lot easier,” Gray said. “We need to make more of these drugs to continue to treat additional patients. I can’t say how much, and I don’t want to create a stampede, but we are starting to meet our goals to have enough available for the kids who meet the inclusion criteria. So far, we’ve seen no adverse effects from the treatments, but we need to raise $1 million to meet our commitments to drug manufacturing and making sure that any Battens’ patient who fits the inclusion criteria and needs to be treated. We continue to raise funds and have a great team and structure in place to allow this to happen. We need to continue to be aggressive.”
Bowen said that he and the others who’ve helped raise money and will try again Thursday believe this will help other children with rare brain diseases that up until now have been fatal. “I’m not a political person, but have known Gordon 20 years and with a new family of my own, the first instinct is to help when you hear the tragic circumstances surrounding his daughters,” Bowen said. “We go to these rubber chicken dinners for worthy causes, but there is urgency here and unfortunately Gordon’s children and others don’t have the luxury of time. He has to have that money by October or they won’t be able to continue to do this therapy that has all the eyes of the medical community watching, because it is actually making a difference. It is more expensive than it should be, right now. But what has been accomplished in a year’s time is remarkable. So we thought, what about just doing a version of an old-fashioned telethon, a boiler room version where we just cold call people all day. We know a lot of people, and we will call all of them Thursday afternoon, basically saying, ‘I need you to give some money to help out a friend in our community who is in need.’ “