In 2020, Jaci Hermstad died at age 26 from a rare and aggressive form of ALS. But her story didn’t end there.
A drug that was created just for her, called Jacifusen, has since been given to others with mutations in the FUS gene, what’s known as FUS-ALS. The results in the first 12 patients, published in May in The Lancet, suggest the therapy slowed disease progression in some cases in what’s considered an untreatable condition. One patient even recovered for a period.
The company that hatched the drug, Ionis Pharmaceuticals, is now sponsoring a late-stage trial that will determine whether the therapy is effective and safe enough to seek approval. Several thousand patients are afflicted by FUS-ALS.
Custom treatments, known as “n-of-1” therapies, were once seen as an end in themselves. But now they’re a launchpad to reach more patients. Beyond Ionis, the model is being pursued by various organizations, from the company La Jolla Labs to Boston Children’s Hospital to the n-Lorem Foundation.
The concept is becoming more popular, especially as new patients are discovered through genetic testing or found to share molecular mechanisms, even if they have different diseases.
