I continue to think about the news that a gene therapy for Stargardt disease is headed for clinical trials. According to Columbia University Medical Center, Stargardt disease is “an inherited form of early-onset macular dystrophy that affects approximately 1 in 10,000.”
When I was diagnosed with Stargardt disease in the 1970s, my retina specialist explained that it was a rare disease. Does “1 in 10,000” sound rare? Probably not, if your imagination dwells on the 1 rather than the 10,000. When the incidence ratio is translated to the scale of today’s U.S. population – if my mental math sans calculator is correct, that is approximately 30,000 people with Stargardt disease out of a population of 300 million Americans. With a market of 30,000 potential customers, how much is this gene therapy likely to cost if it is commercialized?
Wait a minute. I’m the 1 in 10,000 who has this disease. I’m supposed to hope for the cure, right? That’s the master narrative when it comes to research and rare diseases. Never give up hope. Someday science will find a cure.
Well, for now, let’s just say that I’m skeptical about most master narratives, including this one.
I do not subscribe to utilitarianism as an ideology. I resist the notion that some bean-counter or free market bully will calculate the “utiles” of my value/cost to society. But I’m as pragmatic as William James when it comes to problem-solving in everyday life. It’s hard for me to believe that I would pay what this gene therapy will cost, if it were proven effective in my lifetime. Nor would I want society to pay for gene therapy for me. I’d rather that society invested in a little more tolerance, flexibility, and accessibility so people with Stargardt disease can pursue lives of equal opportunity.
After 40 years of living with Stargardt disease, I’ve learned a lot from it about how to pursue such a life. I’ve made a lot of adaptations and negotiated a lot of accommodations along the way. It’s an unfinished project, something I call improvising on the genome. I wouldn’t trade that life experience now for the hope of a cure. But hey, I’m an old graybeard, and I won’t impose my choices on others. Stargardt disease typically manifests in childhood and early adolescence. If gene therapy is proven to be safe, effective, and enduring, it could have an impact throughout one’s life. I expect the decision to have (and continue) such a gene therapy would need to be revisited throughout a lifespan, and calculating the risk/benefit ratio would likely be different every time.