The European Commission has authorized the first treatment that uses the revolutionary CRISPR technique to edit human DNA. The therapy, called Casgevy, is effective against beta thalassemia and sickle cell anemia, two potentially fatal blood diseases. More than 8,000 patients in Europe qualify to receive this treatment, according to the American company Vertex Pharmaceuticals, developer of the therapy together with the Swiss company CRISPR Therapeutics. The European authorization opens a new era in medicine, with the promise of saving millions of lives.
The United Kingdom was the first country to approve this treatment, on November 16. The United States also authorized it on December 8, with a price inaccessible to the vast majority of those affected: two million euros per patient. Eight million people live with sickle cell anemia worldwide, 75% of them in sub-Saharan Africa. “Vertex is already working closely with national health authorities to ensure access for eligible patients as soon as possible,” the company said in a statement.
Behind the advance are French biochemist Emmanuelle Charpentier and American chemist Jennifer Doudna, who won the Nobel Prize in Chemistry in 2020 for developing CRISPR tools, a kind of molecular scissors for editing DNA. Charpentier co-founded CRISPR Therapeutics in 2013. The European Commission authorized Casgevy this Monday, in record time in the world of medicines.
Doctor Elena Cela, head of Pediatric Oncology and Hematology at the Gregorio Marañón hospital in Madrid, urges the authorities to adopt the therapy. “This innovation offers the possibility of using a single treatment only once, which could potentially transform the lives of patients, and it would be important to offer this therapeutic option to candidates as soon as possible,” Cela stated this Wednesday in the statement. sent by Vertex Pharmaceuticals itself. When asked by this newspaper, the pediatrician emphasizes that the price in Spain is still unknown. “Now the negotiation with the governments in Europe will begin,” she says.
Cela recalls the case of Zynteglo, a treatment from the American pharmaceutical company Bluebird Bio that uses a modified virus to introduce a functional gene into the blood cells of patients with beta thalassemia. The company refused to sell its drugs in the European Union after a brawl over the price of Zynteglo in Germany. In the United States it is close to three million dollars per patient.
“Money is, unfortunately, a big obstacle, as previously seen with Zynteglo, but gene therapy offers a great transformation in the disease, perhaps something similar to a cure, although we have only a few years of results and we must remain cautious” , reflects Cela, coordinator of the Erythropathology group of the Spanish Society of Pediatric Hematology and Oncology.
Sickle cell anemia and beta thalassemia are caused by errors in DNA in genes linked to hemoglobin, the protein that carries oxygen around the body. Casgevy therapy consists of extracting red blood cell precursor cells from the patient, using CRISPR scissors to correct the DNA error in the laboratory, and reintroducing these modified cells into patients. The effect of a single treatment can persist for a lifetime.
American Victoria Gray was the first patient with sickle cell anemia to try the experimental Casgevy therapy, in July 2019. Her blood cells were altered and clogged her arteries, causing unbearable pain and a high risk of dying from a stroke. “Thanks to my supercells, my life has completely changed,” she proclaimed a year ago at the third International Congress on Human Genome Editing, held in London.
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