so far, There are only nine centers across the United States. currently offers Casgevy, which may limit who can access it. Vertex says the number of participating sites will increase in the coming weeks and months.
And despite the promise of a pain-free future, the grueling process of getting Casgeby may deter some.
Collecting stem cells from blood can take several hours, and multiple sessions may be required to get enough cells to edit. This is followed by rigorous conditioning. Patients must undergo chemotherapy to kill remaining diseased cells and make room in the bone marrow for the newly edited cells. Chemotherapy can cause mouth sores, fatigue, hair loss, nausea, and other unpleasant side effects. It can also cause infertility. Vertex will also offer fertility support to commercially insured patients, but that benefit will not extend to Medicaid recipients. In the United States, freezing eggs and sperm can cost thousands of dollars, not to mention the cost of IVF.
Patients also need to be hospitalized for several weeks until the edited cells reach the bone marrow and begin producing new blood cells.Oragile I spent a total of 17 weeks in the hospital. To get Kathgevi.
“We know that there are many patients who don’t go down this path because of fertility issues or the need to stay in the hospital for a while,” says Shaar Azar, medical director of the Comprehensive Sickle Cell Disease Treatment Center. Massachusetts General Hospital was one of the first centers to offer Cassibee. For those patients, he says, resources such as housing, child care and food will be needed to help them and their families. Nichols, the Vertex spokeswoman, said the company will assist with travel and lodging and may cover certain costs such as hotels, transportation and meals.
Sickle Cell and Beyond
Sickle cell may be the first disease treated with Crispr, but it won’t be the last. Researchers are deploying gene-editing tools for cancer, HIV, and other genetic diseases. However, not all diseases can be treated yet.
First, it remains difficult to introduce the Crispr system into the cells or organs you want to edit. Kasgeby gets around this problem by removing the cells from the body and editing them in his lab. However, this approach is expensive, complex, and of limited use. In another method, he uses an IV drip to deliver Crispr in tiny bubbles called lipid nanoparticles that are taken up by the liver. However, only some diseases can be treated with this method. Ideally, Crispr would be administered as an injection or pill instead of a complicated cell transplant.
