It begins with a series of blood transfusions over three to four months, after which the stem cells are extracted from the patient’s bone marrow and sent off to a lab where they are edited, Hanna said.īefore they can be reinfused into the patient, however, doctors need to make sure no flawed stem cells remain in the body. While technically a one-time treatment, a number of steps that span months are required before the patient actually gets the modified stem cells. Patients can experience excruciating pain, breathing problems and stroke.Ĭasgevy works by editing the DNA in a patient’s stem cells - which are responsible for making the body’s blood cells - so that they no longer produce sickle-shaped cells. This change can cause cells to clump together, leading to clots and blockages in the blood vessels, starving tissues of oxygen. In patients with sickle cell disease, red blood cells, which are usually disk-shaped, take on a crescent or sickle shape. “It’s been eagerly awaited by patients and families, but also by providers and physicians.” How Casgevy works “It’s something families have been aware of in the early research stage, and they’ve been very patiently waiting for years,” Ferdjallah said. “This could be an equalizer for people with sickle cell because many patients cannot pursue career options” because of the illness. Rabi Hanna, a pediatric hematologist-oncologist at the Cleveland Clinic who has previously served on the advisory board for Vertex. “We really have to make sure that it is accessible,” said Dr. What’s more, that price doesn’t include the cost of care associated with the treatment, like a stay in the hospital or chemotherapy. The pricing strategy, experts argue, may place it out of reach for many families. Still, the new therapy is extremely expensive - $2.2 million per patient, Vertex said. “To really reimagine and re-discuss sickle cell disease as a curable disease and not as this painful and debilitating chronic disease is hope enough with this news.” Asmaa Ferdjallah, a pediatric hematologist and bone marrow transplant physician at the Mayo Clinic in Rochester, Minnesota. “The patient is their own donor,” Thompson said. Using CRISPR, it edits the DNA found in a patient’s stem cells to remove the gene that causes the disease. Until now, the only known cure for sickle cell disease was a bone marrow transplant from a donor, which carries the risk of rejection by the immune system, in addition to the difficult process of finding a matching donor.Ĭasgevy, which was approved for people ages 12 and older, removes the need for a donor. Both treatments work by genetically modifying a patient’s own stem cells. The FDA on Friday also approved a second treatment for sickle cell disease, called Lyfgenia, a gene therapy from drugmaker Bluebird Bio. The approval marks the first of two potential breakthroughs for the inherited blood disorder. “It’s been really remarkable how quickly we went from the actual discovery of CRISPR, the awarding of a Nobel Prize, and now actually seeing it being an approved product.” Alexis Thompson, chief of the division of hematology at Children’s Hospital of Philadelphia, who has previously consulted for Vertex. “I think this is a pivotal moment in the field,” said Dr. The therapy, called Casgevy, from Vertex Pharmaceuticals and CRISPR Therapeutics, is the first medicine to be approved in the United States that uses the gene-editing tool CRISPR, which won its inventors the Nobel Prize in chemistry in 2020. The Food and Drug Administration on Friday approved a powerful treatment for sickle cell disease, a devastating illness that affects more than 100,000 Americans, the majority of whom are Black.
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