Later this spring, a little girl in California who essentially has no immune system will receive a lifesaving treatment for “bubble boy disease” thanks to the persistence of a dogged group of parents, a pediatrician, a veteran newsman and a few episodes of “Grey’s Anatomy.” Five-year-old Seersha Sulack has the same rare disease portrayed in the 1976 John Travolta movie, “The Boy in the Plastic Bubble.
By 2016, when Orchard launched, the clinical trial results looked promising, with 100% survival in 32 patients, according to a company news release at the time. Four years later, when Orchard announced it would reduce investment in the gene therapy, Shayla Sulack called Kohn asking if Seersha would ever get the treatment. “He said, ‘this is my life’s work,’ basically, and there’s no way I’m not going to get this back,” Shayla remembers.