“A Year In, 1^st Patient To Get Gene Editing For Sickle Cell Disease Is Thriving”, Rob Stein2020-06-23 (gene editing; similar)⁠:

…as the one-year anniversary of her landmark treatment approaches, Gray has just received good news: The billions of genetically modified cells doctors infused into her body clearly appear to be alleviating virtually all the complications of her disorder, sickle cell disease. “It’s wonderful. It’s the change I’ve been waiting on my whole life”, Gray told NPR, which has had exclusive access to chronicle her experience over the past year.

…The last time NPR spoke with Gray—in November—her doctors had just gotten the first hints the treatment might be working. Now, after nine months of careful testing, the treatment shows no signs of waning, making her doctors more confident than ever the experiment has been a success.

…The researchers conducting the study Gray started caution that it’s too soon to reach any firm conclusions about the long-term safety and effectiveness of the approach. Gray is just one patient who has been followed for what is still a relatively short period of time, they noted. But Gray’s experience so far, along with two other patients who received the same treatment for a similar disorder, indicate the therapy has been effective for her and may work for other patients as well, they said…At a meeting of the European Hematology Association on June 12, Frangoul and other researchers presented the latest results of their latest testing of Gray as well as two study subjects with a related condition, beta thalassemia. The latter also appear to be benefiting…The researchers also reported that the first patient to receive the same treatment for beta thalassemia in Germany has now been able to live without blood transfusions for 15 months. Previously, the researchers had reported data for that patient for nine months. In addition, four other beta thalassemia patients have been treated, including one who has been transfusion-free for five months, the researchers reported. While Gray and the beta thalassemia patients experienced some health complications following their procedures, none appears to have been due to the gene-edited cells and all recovered, according to the researchers.

“A huge change”: Perhaps most importantly, the changes appear to have translated into substantial health benefits for Gray. She hasn’t had any severe pain attacks since the treatment and hasn’t required any emergency room treatments, hospitalizations or blood transfusions. In each of the previous two years, Gray had required an average of seven hospitalizations and emergency room visits due to severe pain episodes as well as requiring regular blood transfusions. She has also been able to reduce substantially her need for powerful narcotics to alleviate her pain.

“It’s a very big deal for me”, Gray said. “It’s a huge change.”

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