I Received the New Gene-editing Drug for Sickle Cell Disease. It Changed My Life.

upstart writes:

I received the new gene-editing drug for sickle cell disease. It changed my life.:

I'd lived with sickle cell my whole life-experiencing 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.

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's approval in mid-November. I'm one of only a few dozen patients who have ever taken it. In late October, I testified in favor of approval to the FDA's advisory group as it met to evaluate the evidence. The agency will make its decision about exa-cel no later than December 8.

[...] I feel very fortunate to have received exa-cel, but undergoing the treatment itself was an intense, monthslong journey. Doctors extracted stem cells from my own bone marrow and used CRISPR to edit them so that they would produce healthy hemoglobin. Then they injected those edited stem cells back into me.

It was an arduous process, from collecting the stem cells, to conditioning my body to receive the edited cells, to the eventual transplant. The collection process alone can take up to eight hours. For each collection, I sat next to an apheresis machine that vigorously separated my red blood cells from my stem cells, leaving me weakened. In my case, I needed blood transfusions after every collection-and I needed four collections to finally amass enough stem cells for the medical team to edit.

Read more of this story at SoylentNews.