Gordon Gray’s Race For Miracle Batten Disease Cure Update: Jon Hamm Stepped Up. Who’s Next?

On June 9, Deadline revealed how Miracle producer Gordon Gray was racing the clock to raise a minimum of $10 million to run trials for a treatment that could possibly stop the progression of a rare degenerative brain disease in his two daughters. Both 4-year-old Charlotte and her younger sister Gwenyth were diagnosed in March with the rare Late Infantile NCL Batten Disease CLN6, for which there currently is no cure. Rarely has a Deadline article received such an outpouring of sympathy, as the story reported how a long list of stars — some of whom have made movies with Gray — appealed to their vast social media followings to pledge $1. This in the hope of jump-starting trials that could help both Gray girls, as well as children suffering from a disease identified in 1903 but which has made little progress toward treatment because it is so rare.

Three weeks later, the initial Facebook fundraising appeal has raised just north of $1 million. Considering how much of that was derived a buck at a time from working class people moved by the Grays’ story, that is a huge amount of money. Unfortunately, it’s only about half of what is required just to get underway with the necessary trials on three potential courses of treatment that have shown promise in animal testing. There is a long road ahead and the Gray family and the Hollywood establishment is going to have to rise to the occasion here if this effort is to reach its goal.

Jon Hamm, who worked with Gray when he starred in The Million Dollar Arm, was among the first to step up. Last Thursday, he served as emcee for a packed $250 a head fundraiser that featured a special performance by Meghan Trainor, and a silent auction of memorabilia. At the same time, corporations like the family brand Munchkin Inc donated 100% of sale proceeds from its baby product line between June 17-28 to the cause, this after Munchkin CEO Steven Dunn saw the heartbreaking video about Charlotte and Gwenyth that got this whole thing started (see it below).

My colleague Pete Hammond and wife Madelyn bought tickets and attended last week’s fundraiser, which was held at The Atom Factory in Culver City. Pete said that the Mad Men star learned of the crisis from Gray’s former partner Mark Ciardi on the set of Keeping Up With The Joneses, and that when the actor (who is involved with several children’s hospitals) asked one of those experts what they knew about Batten Disease, “They replied ‘nothing,’ ” Hamm said. “That is why time is of the essence to come up with enough money to finance the research for a cure.” He said that among the items auctioned was a Navy SEAL training dog (that raised $50,000) and Hammond noted how heavyweights like Universal’s Ron Meyer and a coterie of producers and studio execs have rallied around Gray, his wife Kristen and the girls. Truth be told, those are the people who will have to use their formidable influence if these girls, and others with Batten, are to have a fighting chance here at raising a daunting amount of money.

What are the odds of finding a cure here? I spoke with Rahul Jandial, M.D., Ph.D, brain surgeon & scientist at City of Hope, and he said that the goal isn’t cure as much as it is treatment that can stop the progression of a fatal disease like Batten, which is basically what happened with HIV and AIDS. Jandial also emphasized that the opportunity here goes far beyond treating two adorable sisters; a breakthrough is a breakthrough and while this form of Batten is rare, a positive development like the one that the Grays are trying to fund can help a large group of children suffering from similarly debilitating brain diseases. “Looking into treatments for Batten Disease isn’t just about those few kids that have it,” Jandial said. “It’s about this whole world of rare diseases and neurodegenerative diseases. Breakthroughs can benefit other brain diseases like ALS, Parkinson’s and dementia.”

Having treated so many children, Jandial knows better than anyone the confounding complexity and mysteries of the brain, and the heartbreaking limitations of medicine. He won’t say that what the treatments the Grays are trying to fund will definitely underwrite the storybook ending that was the hallmark of many of several movies Gray has produced. But it is possible, and only possible if the funding is there. I watched a Ted talk with Sirius XM founder Martine Rothblatt who was told her young daughter, gasping for breath, would soon die when she was diagnosed with the fatal rare disease Pulmonary Arterial Hypertension. Rothblatt tracked down a possible treatment and wouldn’t take no for an answer when the pharmaceutical company that controlled it told her it had ended progress on a possible treatment drug because so few patients had the disease there was no market for it. Rothblatt finally talked Glaxo into licensing her the patent (they gave her a baggie full of powder which was all they developed) for $25,000 against 10% of any revenues. She hired the scientists, and underwrote the research. Long story short, the drug arrested the progression of the disease in her daughter, who is now 30 and still breathing, as are a lot of other former children who would be dead; the same drug generates $1.5 billion per year and the pharmaceutical company gets $150 million of that, year in and year out.

That is a happy ending worthy of a Gordon Gray production and evidence that miracles are possible. But Charlotte is showing the effects of Batten each day, and it is difficult to keep up awareness beyond that initial outpouring of sympathy and support and publicity. Friends of the Gray family told Hammond that Kristen was “a warrior” for her tireless efforts so far, and they said that Gordon Gray “has not gotten off the phone since the diagnosis,” trying to rally the financial support needed to fund what Gray called in the last article his equivalent of the Doug Flutie Hail Mary Pass.

We will check in on the Grays as this campaign continues. For my part, I have only three pieces of Hollywood memorabilia that mean anything to me. One is an actual leather vest from my beloved series Sons Of Anarchy, signed by the cast; there is my machete, hand signed by Danny Trejo; and an autographed framed color photo of Pierce Brosnan, including a few actual celluloid frames from the 007 film Goldeneye. I’m not sure if these would move the needle, but if the Grays think they would help in their next auction, they need only tell me where to ship them. It’s only stuff, and I bet there are people in town with a whole lot more who are just as eager to step up. Here’s the Gray’s video, again:

This article was printed from https://deadline.com/2015/06/gordon-gray-daughters-batten-disease-fundraiser-jon-hamm-meghan-trainor-1201461555/