2023-11-17 12:43:00
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The UK Medicines and Healthcare products Regulatory Agency, in a statement issued on Thursday, has announced the approval of Casgevy. This revolutionary drug, the first to be licensed using the CRISPR gene editing tool, worthy of the Nobel Prize in 2020, has been approved for the treatment of patients with sickle cell disease and thalassemia over 12 years of age.
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To date, bone marrow transplants, extremely arduous procedures accompanied by unpleasant side effects, had been the only long-term treatment.
Europe and the United States are in the final stretch to give the green light to CRISPR therapy. The United States Food and Drug Administration (FDA) has until December 8 to issue its verdict. On October 31, an FDA advisory committee determined that the treatment is safe for patients.
PROGRESS IN THE UNITED KINGDOM
Both the sickle cell disease as the thalassemia They are caused by errors in the genes that produce hemoglobin, a protein in red blood cells that carries oxygen.
In individuals with sickle cells, particularly common among people of African or Caribbean descent, a genetic mutation causes the cells to become crescent-shaped, blocking blood flow and causing extreme pain, organ damage, stroke and other problems.
Genetic editing and transplants, a union that seeks to save lives
In those with thalassemia, the genetic mutation can cause severe anemia, requiring blood transfusions every few weeks, as well as lifelong injections and medications. Thalassemia predominantly affects people of South and Southeast Asian, as well as Middle Eastern, descent..
Casgevy operates by attacking the problematic gene in the patient’s bone marrow stem cells so the body can make functional hemoglobin. Patients first receive chemotherapy, then stem cells are extracted from their bone marrow and laboratory gene editing techniques are used to repair the gene. The modified cells are infused back into the patient for permanent treatment. Patients must be hospitalized at least twice: once for stem cell collection and once to receive the altered cells.
Casgevy is a joint creation of Vertex Pharmaceuticals (Europe) Ltd. and CRISPR Therapeutics.
AN EXPENSIVE TREATMENT
Millions of people around the world, including about 100,000 in the United States, have sickle cell disease. It occurs most often among people from places where malaria is or was common, such as Africa and India, and is also more common in certain ethnic groups, such as people of African, Middle Eastern, and Indian descent. Scientists believe that being a carrier of sickle cell trait helps protect against severe malaria.
The British regulator’s decision to authorize gene therapy for sickle cell disease was based on a study of 29 patients, of whom 28 reported having had no serious pain problems for at least a year after treatment. In the thalassemia study, 39 of 42 patients who received the therapy did not need a red blood cell transfusion for at least a year afterwards.
The problem, however, is the cost. Gene therapy treatments can cost millions of dollars, and experts have previously raised concerns that they could remain inaccessible to the people who would benefit most from them.
Vertex Pharmaceuticals has said it has not yet set a price for the treatment in Britain and is working with health authorities to do so: drugs and treatments in Britain must be recommended by a government watchdog before they are freely available to patients in the national health care system.
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