Ultimately, the goal is for this therapy to become simpler, for the principles behind the treatment to be applied to a gene editing method that does not require toxic chemotherapy and a month or more in the hospital. While gene therapy will be undertaken by a minority of the sickle cell population, at least initially, these questions will only become more pressing over time.
As will questions of cost. Looking to recently approved gene therapies for comparison, the treatment could carry a price tag of $1 million or more per person. And if this high-cost treatment and necessary chemotherapy and hospitalization are not covered by Medicaid, then this tremendous scientific advance will only accentuate disparities in a disease where disparities are already too prevalent. Finally, for those who do undergo gene therapy, will the worlds that they re-enter acknowledge the complexity of what it means to be cured? Those who have suffered from sickle cell for years often become accustomed to hefty doses of opiates. Their interactions with the medical system are defined by pain and, for so many, by not being believed. Those experiences will continue to reverberate.
Some of the people who are going for gene therapy have been so tremendously affected by sickle cell that basically their whole lives were about being sick, said Dr. Lewis Hsu, the chief medical officer at the Sickle Cell Disease Association of America and the director of the pediatric sickle cell program at the University of Illinois at Chicago. Youre an adult but youve never applied for a job. Other people dont have friends outside their sickle cell peer group, he continued, extrapolating from the experiences of those who have undergone bone marrow transplants. Some people almost need a society re-entry program. And then there are others who have blossomed, gone off and done wonderful things.
When Mr. Holmes returned to his home in Mobile, Ala., after about 90 days of hospitalization at the N.I.H., he found himself with an energy he had never experienced before, a feeling he describes as the newness of life. Though he expected those in his world to be elated by his newfound health, it was not that simple. His wife, accustomed to her role as the caretaker, was unable to adjust to the shift and they ultimately divorced. The adjustment was hard on Mr. Holmes, too. He realized that he had come to terms with the idea that he would die young, but living with the knowledge of all his missed opportunities was almost harder. No one had prepared him for that.
But he carried on. He connected with others suffering from sickle cell disease in Alabama, to offer education and to encourage them to keep fighting the disease and advocating for themselves, to move cities to find good care if they had to. Though he would not be able to fulfill his long-held goal of serving as a Marine, he could keep a job, and found work in the county jail as a corrections officer. It was traumatizing work at first, but work he is good at and for which he is respected. He writes poetry when he has time. And more recently, Mr. Holmes moved out of Alabama to Austin, Texas.
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Opinion | A New Sickle Cell Treatment Will Change Lives but ... - The New York Times
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