London, November 18 (IANS). Britain has approved the world’s first gene therapy to treat blood disorders sickle-cell and thalassemia using the gene-editing tool CRISPR, which earned its inventors the Nobel Prize in 2020.
Until now, bone marrow transplantation has been the only permanent treatment option.
The UK’s Medicines and Healthcare products Regulatory Agency (MHRA) has authorized a new treatment called Casgevi for patients aged 12 and over with sickle cell disease and transfusion beta-thalassemia.
Sickle cell disease and beta-thalassemia are both genetic conditions caused by errors in the gene for hemoglobin, which is used by red blood cells to carry oxygen around the body.
Casgevi is designed to work by editing defective genes in a patient’s bone marrow stem cells so the body can produce functional hemoglobin.
To do this, stem cells are taken out of the bone marrow, edited in a laboratory and then infused back into the patient after which the results are likely to last a lifetime.
In people with sickle cell disease, the genetic error can cause very severe pain, infection, and anemia (which makes it difficult for your body to carry oxygen). In beta-thalassemia patients, it can cause severe anemia. Patients often require blood transfusions and injections and medications every 3 to 5 weeks.
“Sickle cell disease and beta-thalassaemia are both painful, life-long conditions, which can be fatal in some cases,” Julian Beech, interim executive director of Healthcare Quality and Access at the MHRA, said in a statement.
The approval of Casgevi for sickle cell disease was based on a clinical trial of 29 patients, 28 of whom (97 percent) were free from severe pain crisis for at least 12 months after treatment.
In a clinical trial for beta-thalassemia, 39 of 42 patients (93 percent) did not require a red blood cell transfusion for at least 12 months after treatment. The remaining three reduced the need for red blood cell transfusions by more than 70 percent.
Side effects of the treatment were similar to those associated with autologous stem cell transplants, including nausea, fatigue, fever, and increased risk of infection.
The MHRA said no significant safety concerns were identified during the trials, adding that the safety of the treatment will be analyzed further.
Casgevi is currently being evaluated by the US Food and Drug Administration (FDA) and is expected to receive the agency’s approval next month.