Nascent ALS Drug Shows ‘Remarkable’ Progress
Pam Belluck reported last week for The New York Times about a “milestone” ALS treatment drug and its effect in “[helping] some patients improve.” Belluck’s story centers on 58-year-old Amanda Sifford, a school psychologist living in Florida.
“It was May 2023, and the Food and Drug Administration had just approved the first therapy for a genetic form of A.L.S., even though clinical trial results had not yet proven the drug would be effective. The drug, tofersen, made by Biogen and marketed as Qalsody, targets the form of A.L.S. that Ms. Sifford inherited, so Dr. Carberry and Dr. Michael Benatar, the executive director of University of Miami A.L.S. Center, scrambled to establish a clinic to administer it,” Belluck wrote. “She began receiving tofersen monthly, through infusions into her spinal canal.”
Notably, there are caveats to this genuinely good news, Belluck said. The first is tofersen is intended only for “a small subset of patients” as it targets only 2% of ALS patients. Second, the drug is exorbitantly expensive, costing $15,500 per monthly dose. Third, research into tofersen is in its earliest days; again, the sample size is small and the drug only stabilizes or slightly improves—it doesn’t reverse or perform miracles. Nonetheless, The Times’ headline is not clickbait; it is remarkable progress.
Indeed, people like Sifford are “exhausted but grateful” following the injections.
“I’ve gotten a chance to help find treatment for something that killed my father and my grandfather,” she said to Belluck. And “I’m alive.”
I covered ALS extensively over the last several years for my old Forbes column. I interviewed ex-Bachelor contestant Sarah Trott twice about leaving her job as a television news anchor to become a full-time caretaker for her father, who had ALS. Trott called ALS “the absolute worst disease that I’ve seen firsthand” in our November 2022 interview, adding the tolls caregiving takes on family—mentally and physically—can be as disabling and debilitating in its own right. (As someone whose mom died from cancer, I can attest to this. Caretaking is brutal.) Her father died in October 2021.
