The United Kingdom has become the first country to grant regulatory approval to a medical treatment involving a gene-editing tool: CRISPR.
The country’s Medicines and Healthcare products Regulatory Agency (MHRA) said on Thursday it has given the green light to a treatment known as Casgevy, which will be used to treat sickle cell disease and beta thalassemia.
Both genetic conditions are caused by errors in the genes for hemoglobin, which is used by red blood cells to carry oxygen around the body. There is still no successful treatment in the world for any of the disorders.
Sickle cell disease, which can result in debilitating pain crises, is more common in people of African or Caribbean family origin. Beta thalassemia mainly affects people of Mediterranean, South Asian, Southeast Asian and Middle Eastern origin, the statement said.
“Both sickle cell disease and β-thalassemia are painful, lifelong conditions that, in some cases, can be fatal. To date, a bone marrow transplant – which must come from a compatible donor and carries a risk of rejection – has been the only permanent treatment option,” said Julian Beach, acting executive director of healthcare quality and access at the MHRA. , in a statement.
“I am pleased to announce that we have authorized an innovative and pioneering gene-editing treatment called Casgevy, which, in testing, was found to restore healthy hemoglobin production in the majority of participants with sickle cell disease and transfusion. -β-dependent thalassemia, relieving the symptoms of the disease.”
The promise of CRISPR-Cas9
The CRISPR-Cas9 gene editing technique allows scientists to make very precise changes to DNA. Its inventors — Emmanuelle Charpentier and Jennifer A. Doudna — won the Nobel Prize in Chemistry in 2020.
Casgevy is not a simple pill or injection. The treatment, manufactured by Vertex Pharmaceuticals, is administered by taking stem cells from a patient’s bone marrow and editing a gene in the cells in the laboratory.
Patients must then undergo a “conditioning treatment”, which may involve an immunosuppressant drug, radiotherapy or chemotherapy, to prepare the bone marrow before the modified cells are infused back into the patient, according to the MHRA.
“After that, patients may need to spend at least a month in a hospital while the treated cells take up residence in the bone marrow and begin producing red blood cells with the stable form of hemoglobin,” according to the statement.
The US Food and Drug Administration (FDA) is evaluating the same treatment and is expected to make a decision on whether or not to accept it by December 8.
“This is a huge step forward in advancing medical approaches to combating genetic diseases that we never thought possible to cure,” said Alena Pance, senior lecturer in genetics at the University of Hertfordshire, in a statement released by the Science Media Center, which provides information to journalists.
“Modifying the patient’s bone marrow stem cells avoids the problems associated with immunological compatibility, that is, looking for donors compatible with the patient and following immunosuppression, constituting a true cure for the disease and not a treatment,” added Pance.
The MHRA statement did not say how much the treatment would cost, but it is likely to 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 cutting-edge technology was marred by controversy after Chinese scientist He Jiankui announced in 2018 that he had created the world’s first gene-edited babies.
Scientists say this powerful technique should not be used to manipulate human genes that will be passed from one generation to the next.
See also: Government receives medicine that simplifies HIV treatment
Source: CNN Brasil
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