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."
Seersha is expected to get the treatment next month. She'll become only the second child in the U.S. in the past five years to receive it; the first child received it earlier this month. "It has not escaped our attention at FDA that there have been some clouds on the horizon in gene therapy," Dr. Peter Marks, head of the U.S. Food and Drug Administration's Center for Biologics Evaluation and Research, said at a biotech conference in California in October."We really want to try to see what we can do to move things forward."
In a statement to CNN, an Orchard spokesperson wrote that"after encountering technical challenges related to the commercial-grade manufacture of this particular therapy, we made the very difficult decision to limit additional investment in [the SCID gene therapy]." The spokesperson spoke on the condition of anonymity.
Weighing 6 pounds and 10 ounces, Seersha appeared perfectly healthy, and her parents took her on family outings, going to Dole Plantation and Waikiki Beach when she was five days old. Not only had they been going everywhere with their new baby, but Seersha's big brother and sister had been affectionately pawing all over her.
The next day, Shayla and Seersha were on a military medical transport flight to Los Angeles. Seersha was in an incubator in the front of the plane, and the other patients were kept in the back to decrease the chances they could get her sick. Testing showed that any of her family members could donate to her, but none of them was a particularly good match. This was bad news for two reasons: One, the transplant likely wouldn't be as effective. Two, Seersha would be more likely to suffer complications.
She brought it up to Kohn, the UCLA gene therapy researcher. He told her that not only had he seen the episodes, but he'd given"Grey's Anatomy" staffers a tour of his lab and answered their questions while they were preparing the story arc, which aired in 2014. He still had the"Grey's Anatomy" mug and note pads they gave him as a thank you.
For her first year of life, Seersha hardly ever left her bedroom, which her parents filled with toys to entertain her. She had contact with only her parents and Shayla's parents — not even her siblings — and the adults changed into clean clothes before coming near her. The Sulacks were shocked when four months later, the company issued a news release saying it had a net loss of $50.6 million in the previous quarter and would"reduce investment" in its SCID gene therapy and prioritize research for other diseases.
Now 68, Kohn first started researching gene therapy for ADA-SCID when he was 31 and a fellow at the National Institutes of Health, working in the lab of Dr. Michael Blaese, a pioneer of gene therapy. "He said, 'this is my life's work,' basically, and there's no way I'm not going to get this back," Shayla remembers."He said, 'this is mine, this is my thing and I'm not going to let it die.'"'A desperate call to save young lives'Once Orchard abandoned the treatment, Kohn's mission became to get the license for the therapy back so he could start using it to treat Seersha and the other children.
But the company kept the license, and the treatment continued to sit on the shelf. In February, 2021, the parents of more than 20 children who were waiting for the gene therapy treatment, including the Sulacks, wrote a letter to Gaspar. "He really lit a fire and kept the story going," Smith said."He kept it on the radar, so Orchard knew we were not just going to away quietly."
Urnov, the director of technology and translation at Berkeley's Innovative Genomics Institute, noted that the gene therapy"cured 48" of the 50 patients in the study. Some parents doubted whether manufacturing issues were the real reason Orchard had abandoned SCID gene therapy. They say the company's true motivation was profit — that Orchard saw its revenues decreasing and chose not to pursue the ADA-SCID treatment so they could focus on therapies for more common diseases with likely more lucrative markets.