Marie Tornyenu missed more than 100 days of high school while hospitalized repeatedly for sickle cell disease, an inherited blood disorder that causes crippling pain. Somehow, she still managed to complete at least eight advanced placement courses, play clarinet in the school band, and get admitted to Boston University.
Then in December 2021, after transferring to Cornell University, she received an experimental medicine that used a revolutionary gene-editing technology to treat inherited diseases. It freed her from excruciating bouts of pain and the frequent blood transfusions that are a mainstay of sickle cell treatment.
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The life-changing drug, developed by Boston-based Vertex and its Swiss partner CRISPR Therapeutics, is expected to be approved by the Food and Drug Administration by Friday for people with severe cases of the disease. Called Casgevy, it would usher in a new era not only for those with sickle cell but also for medicine: The drug would be the first gene-editing therapy authorized by US regulators, and uses a tool called CRISPR.
The likely approval — Casgevy was cleared by British regulators last month — raises both the promise of cures for diseases as well as the ethical concerns that come with the power to manipulate the building blocks of human life. With an expected price tag in the seven figures, it also touches on issues of equity in medicine.
okay so they take out your stem cells, modify them, put them back in and benefits are expected to be life long
do you then get 100% healthy red blood cells or just partially? I assume not unless they wipe out all the other cells? which they don’t do, as far as I understand
Normal red blood cells only live about 120 days, so they replace themselves naturally within a few months.
yeah but the original unmodified “broken” stem cells that produce the sickle cells will still be there presumably. so I assume they will have a mix of constantly replenishing healthy and sickle cells.
