Dubai, United Arab Emirates (CNN) — The United Kingdom has become the first country to grant regulatory approval to a medical treatment involving the revolutionary gene-editing tool CRISPR.
The country’s Medicines and Healthcare products Regulatory Agency said on Thursday that it has been given the green light for the treatment of “Kaskevi”, which is used to treat sickle cell anemia and beta thalassemia. Both genetic conditions are caused by errors in the genes for hemoglobin, which red blood cells use to carry oxygen around the body. There is no universally known cure for either disease.
Sickle cell disease, which can lead to debilitating pain, is more common in people with African or Caribbean family roots. Beta thalassemia mainly affects people from the Mediterranean region, South Asia, Southeast Asia and the Middle East, the report said.
Julian Beach, interim chief executive of quality and access to healthcare at the Medicines and Healthcare products Regulatory Agency, said: “Sickle cell anemia and beta thalassemia are painful, life-long diseases, and in some cases can be fatal. A bone marrow transplant. A matched donor “closer and rejection. At risk, it remains the only permanent treatment option so far.”
He continued: “I am delighted to announce that we have received approval to use an innovative and first-of-its-kind gene-editing therapy, called CaskV, for beta thalassemia patients with sickle cell disease and an approved blood transfusion, reducing the symptoms of the disease.
CRISPR-Cas9… hope
CRISPR-Cas9 gene editing technology allows scientists to make very precise changes to DNA. Its two inventors, Emmanuel Charpentier and Jennifer A. Doudna, Nobel Prize in Chemistry 2020.
And “Casgevy” treatment is not a simple pill or injection. The treatment, manufactured by Vertex Pharmaceuticals, involves taking stem cells from a patient’s bone marrow and modifying the genes in the cells in a laboratory. According to the Medicines and Healthcare Products Regulatory Agency, patients must undergo “conditioning therapy” that includes immunosuppressive drugs, radiation therapy or chemotherapy to prepare the bone marrow before the transplanted cells can be transplanted back into the patient.
The report added, “After that, patients will need to stay in the hospital for at least a month while the treated cells settle in the bone marrow and start producing red blood cells with stable hemoglobin.”
The US Food and Drug Administration is evaluating the same treatment and is expected to make a decision on whether or not to allow its use by December 8.
Alina Bans, a senior lecturer in genetics at the University of Hertfordshire, said in a statement released by the Center for Science Media, “This is a great advance in medical approaches to treating genetic diseases that we didn’t think were curable.”
Bans added, “Transplantation of stem cells from the patient’s bone marrow avoids the problems associated with immunocompatibility, that is, searching for donors who match the patient and pursuing immunocompetence, and is considered a real treatment of the disease and not a drug.”
The agency’s release did not specify the cost of the treatment, but it could be expensive.
CRISPR-Cas9 has had a major impact on biomedical research, clinical medicine, and agriculture, and is widely used in laboratories around the world.
The advanced technology sparked controversy after Chinese scientist He Jiankui announced in 2018 that he had created the world’s first genetically modified babies. Scientists say this powerful technique should not be used to manipulate human genes that are passed down from generation to generation.
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