ICYMI, we’ve been seeing some cool stuff with stem cells recently. The latest is a possible treatment for macular degeneration—the leading cause of blindness in the UK. Essentially, the part of your eye that lets you see what’s directly in front of you (the macula) gets worse over the years like an iPhone hardware update. You know, subtle changes at first, then it’s just objectively worse than it was before (stages of macular degeneration here.) But using an embryonic stem cell patch inserted into the back of the patients’ eye, failing macula cells can be replaced to restore partial vision. Two patients in a Phase I study have experienced improved vision for about a year, and eight more patients will be part of the ongoing study
What is “it,” you ask? Spark Therapeutics’ new gene therapy Luxturna which can cure blindness in a single treatment. The condition causing the blindness only affects a few thousand people, so the FDA has designated it as an orphan drug. The single dose and orphan drug aspects of this gene therapy combine for a rather expensive R&D bill, and translate into a bit of sticker shock at the selling price: $425,000 per eye. Spark is trying out some interesting commercialization practices to get the drug to patients, like giving rebates to patients whose eyes don’t see better over time. They’re also considering selling the therapy directly to insurance companies so that health care providers don’t have to pay and store the treatment without a guarantee it’ll ever be used.