{"id":368663,"date":"2023-12-04T08:30:00","date_gmt":"2023-12-04T13:30:00","guid":{"rendered":"https:\/\/platohealth.ai\/i-received-the-new-gene-editing-drug-for-sickle-cell-disease-it-changed-my-life\/"},"modified":"2023-12-04T13:00:13","modified_gmt":"2023-12-04T18:00:13","slug":"i-received-the-new-gene-editing-drug-for-sickle-cell-disease-it-changed-my-life","status":"publish","type":"post","link":"https:\/\/platohealth.ai\/i-received-the-new-gene-editing-drug-for-sickle-cell-disease-it-changed-my-life\/","title":{"rendered":"I received the new gene-editing drug for sickle cell disease. It changed my life.","gt_translate_keys":[{"key":"rendered","format":"text"}]},"content":{"rendered":"
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On a picturesque fall day a few years ago, I opened the mailbox and took out an envelope as thick as a Bible that would change my life. The package was from Vertex Pharmaceuticals, and it contained a consent form to participate in a clinical trial for a new gene-editing drug to treat sickle cell disease.<\/p>\n

A week prior, my wife and I had talked on the phone with Haydar Frangoul, an oncologist and hematologist in Nashville, Tennessee, and the lead researcher of the trial. He gave us an overview of what the trial entailed and how the early participants were faring. Before we knew it, my wife and I were flying to the study site in Nashville to enroll me and begin treatment. At the time, she was pregnant with our first child.<\/p>\n<\/p><\/div>\n<\/div>\n

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I\u2019d lived with sickle cell my whole life\u2014experiencing chronic pain, organ damage, and hopelessness. To me, this opportunity meant finally taking control of my life and having the opportunity to be a present father.<\/p>\n

The drug I received, called exa-cel, could soon become the first CRISPR-based treatment to win approval from the US Food and Drug Administration, following the UK\u2019s approval in mid-November. I\u2019m one of only a few dozen patients who have ever taken it. In late October, I testified in favor of approval to the FDA\u2019s advisory group as it met to evaluate the evidence. The agency will make its decision about exa-cel no later than December 8.<\/p>\n<\/div>\n<\/div>\n