Louis doctor, acknowledged the new treatments would be expensive. By comparison, research earlier this year showed medical expenses for current sickle cell treatments, from birth to age 65, add up to about $1.6 million for women and $1.7 million for men. The companies have not released potential prices for either therapy, but the institute report said prices up to around $2 million would be cost-effective. It aims to add functional copies of a modified gene, which helps red blood cells produce “anti-sickling” hemoglobin that prevents or reverses misshapen cells. The FDA is expected to decide on the second gene therapy for sickle cell, made by Bluebird Bio, before the end of the year. If the treatment is allowed on the market, the company has proposed a post-approval safety study, product labeling outlining potential risks and continuing research. While the agency doesn’t have to follow the group’s advice, it often does. The FDA is looking for advice on whether the company’s research on such effects was adequate to assess the risk or whether additional studies are needed. “My children no longer have a fear of losing their mom to sickle cell disease,” she said.īut the FDA is asking an outside panel of gene therapy experts next week to discuss a lingering issue that often comes up when discussing CRISPR: the possibility of “off-target effects,” which are unexpected, unwanted changes to a person’s genome. Now, she’s able to run around with her kids and work a full-time job. She described feeling she “was being reborn” the day she got the gene therapy. She described suffering with terrible bouts of pain since childhood and receiving high-dose pain medications and sometimes blood transfusions. Victoria Gray, of Mississippi, the first patient to test the treatment, shared her experience with researchers at a scientific conference earlier this year. The company called the treatment “transformative” and said it has “a strong safety profile.” Of 30 who had at least 18 months of follow-up, 29 were free of pain crises for at least a year and all 30 avoided being hospitalized for pain crises for that long. In a briefing document released Friday before the advisory committee meeting, Vertex said 46 people got the treatment in the pivotal study. The treatment has been tested in a relatively small number of patients thus far, the nonprofit Institute for Clinical and Economic Review said in an evidence report. Patients get medicines to kill off other flawed blood-producing cells and then are given back their own altered stem cells. When patients undergo the treatment, stem cells are removed from their blood and CRISPR is used to knock out the switching gene. The goal is to help the body go back to producing a fetal form of hemoglobin - which is naturally present at birth but then switches to an adult form that’s defective in people with sickle cell disease. This new treatment involves permanently changing DNA in a patient’s blood cells. No donor is required for the one-time gene therapy, “exa-cel,” made by Vertex Pharmaceuticals and CRISPR Therapeutics. The only permanent solution is a bone marrow transplant, which must come from a closely matched donor without the disease and brings a risk of rejection. Current treatments include medications and blood transfusions.
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