Boston-based Massachusetts Eye and Ear on Tuesday performed the first post-approval injection of a new gene therapy for an inherited disease—that comes with a hefty price tag.
About the treatment
Spark Therapeutics' Luxturna treats vision loss and blindness caused defective versions of the RPE65 gene that prevents patients from producing a protein needed to help the eyes process light. In some cases, patients experience diminished vision after dusk, while others are legally blind.
FDA approved the breakthrough therapy in December 2017 after decades of development and research. The treatment is priced at $425,000 per eye, which according to STAT News is a record for the industry.
The treatment requires a doctor to inject a healthy, lab-grown copy of the RPE65 gene into cells to replace the defective gene. According to the Boston Business Journal, only eight U.S. medical centers are certified to inject Luxturna in patients, and Mass. Eye and Ear is the only approved facility in New England.
The first post-approval procedure
The surgery at Mass. Eye and Ear was one of several Luxturna procedures scheduled across the country last week—but the facility's surgery on a 13-year-old boy from New Jersey had the earliest start time. The boy, Jack Hogan, had a visual impairment that prohibited him from seeing outside at night, meaning he couldn't play baseball, which often has evening games.
At 9:15 a.m. Tuesday, Jason Comander, the associate director of Mass. Eye and Ear's inherited retinal disorders service, sat down to perform the first of two procedures—Tuesday's surgery was for Hogan's left eye, and Mass. Eye and Ear this week will conduct a separate surgery on Hogan's right eye.
Before entering the OR pharmacy staff at Mass. Eye and Ear thawed and diluted vials of Luxturna. However, staff were faced with an unexpected conundrum: how to safely get the syringes out of the plastic bag in which they arrived. Tracy Condron, the center's nurse director, said, "Everybody's nervous about taking out the medication, because it's so expensive."
Ultimately, Comander carefully grabbed the syringes with forceps and placed them on a table. "That's $425,000 right there," he said.
Back in the OR, Comander's first step was to insert plastic ports in the white sclera to protect the eye from the hair-thin instruments he used to perform the procedure and to pump a saline-like solution over the eye to keep it from collapsing.
Next, Comander injected a white powder to better view the clear gel that fills the back of the eye, covering the retina. Using a foot petal, Comander turned on the suction that removed the gel, giving him access to the retina. At this point, Comander cleared the OR of all non-essential staff to concentrate on finding the best injection spot.
"If you don't put just the right amount of pressure up against the retina, and if the assistant doesn't put just enough pressure on the plunger, the fluid can be blocked and not go anywhere … or it can be wasted by being injected into the center of the eye, where it can cause inflammation," Comander told STAT News' Eric Boodman.
Comander's first few attempts were not successful, then after about 10 minutes he saw a sign of success—a blister formed, Boodman writes.
While Comander has deemed the first surgery a success, STAT News reports that it's still unclear how effective the treatment would be. According to STAT News, clinical trial results have largely been good, with patients in some cases saying they could see stars in the night sky for the first time. However, given the cost and level of uncertainty surrounding the treatment, Spark has created a refund program for those who the treatment does not help.
Prior to the surgery, Jeanette Hogan, Jack's mother, said, "Once this is all said and done and Jack tells me he can see better, that's when we'll celebrate," adding, "There's still so many unanswered questions. Is it going to work? Will there be a difference? That's what I'm waiting to see" (Boodman, STAT News, 3/21; Stendahl, Boston Business Journal, 3/20).
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