The UK Medicines and Healthcare Products Regulatory Agency, in a statement released on Thursday, said: Has announced the approval of Kaskevi. This revolutionary drug, the first to be licensed using the CRISPR gene editing tool, is eligible for the Nobel Prize in 2020, and is approved for the treatment of sickle cell disease and thalassemia patients over 12 years of age.
Until now, bone marrow transplants, extremely difficult procedures with unpleasant side effects, were the only long-term treatment.
Europe and the US are in the final stages of giving the green light to CRISPR therapy. The US Food and Drug Administration (FDA) has until December 8 to issue its ruling. On October 31, an FDA advisory panel determined that the treatment was safe for patients.
Progress in the United Kingdom
Both of them Sickle cell disease as Thalassemia They are caused by errors in the genes that produce hemoglobin, the protein in red blood cells that carries oxygen.
In people with sickle cells, especially those of African or Caribbean descent, the genetic mutation causes the cells to become crescent-shaped, blocking blood flow and causing severe pain, organ damage, stroke and other complications.
Genetic editing and transplantation, a union that seeks to save lives
In people with thalassemia, the genetic mutation can cause severe anemia, requiring blood transfusions every few weeks, as well as lifelong injections and medications. Thalassemia mostly affects people of South and Southeast Asia, as well as people of Middle Eastern descent..
Caskavi works by attacking the problematic gene in the patient’s bone marrow stem cells so that the body can produce 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 genome. The modified cells are then injected back into the patient for permanent treatment. Patients must be hospitalized at least twice: once for the stem cell collection and once to receive the transplanted cells.
Kaskavi is a joint venture between Vertex Pharmaceuticals (Europe) Ltd and CRISPR Therapeutics.
An expensive treatment
Millions of people worldwide are affected by sickle cell disease, including about 100,000 people in the United States. It occurs most often in people from places where malaria is present or common, such as Africa and India, and is also more common in certain ethnic groups, such as those of African, Middle Eastern, and Indian descent. Scientists believe that sickle cell is a carrier of traits Helps protect against severe malaria.
The British regulator’s decision to approve gene therapy for sickle cell disease was based on a study of 29 patients, 28 of whom reported no severe pain problems for at least a year after treatment. In a thalassemia study, 39 of 42 patients who received treatment did not require red blood cell transfusions after at least one year.
However, the problem is cost. Gene therapy treatments can cost millions of dollars, and experts have previously raised concerns that they could be out of reach for the people who would most benefit.
Vertex Pharmaceuticals said it has yet to set a price for the treatment in Britain and is working with health authorities to do so: drugs and treatments in Britain must be prescribed by a government watchdog before they can be made available free to patients on the National Health Service. system.
