The European Commission has authorized the first treatment that uses the revolutionary CRISPR technique to modify human DNA. The therapy, called Casgevy, is effective against beta thalassemia and sickle cell disease, two potentially fatal blood diseases. More than 8,000 patients in Europe meet the characteristics to receive this treatment, according to the American company Vertex Pharmaceuticals, developer of the therapy in collaboration with the Swiss company CRISPR Therapeutics. 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, at a price unaffordable for the vast majority of people concerned: 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 to eligible patients as quickly as possible,” the company said in a statement.
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Behind this 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 medicine.
Doctor Elena Cela, head of the pediatric oncology and hematology department at the Gregorio Marañón Hospital in Madrid, urges the authorities to adopt this therapy. “This innovation offers the ability to use a single treatment once, which could potentially transform patients’ lives, and it would be important to bring this treatment option to candidates as quickly as possible,” Cela said on Wednesday. ©rcoles in the press release sent by Vertex Pharmaceuticals itself. Questioned by this newspaper, the pediatrician emphasizes that the price in Spain is still unknown. “Now negotiations with European governments will begin,” he said.
This recalls the case of Zyntéglo, a treatment from the American pharmaceutical company Bluebird Bio which 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 fight over the price of Zynteglo in Germany. In the United States, it amounts to almost three million dollars per patient.
“Money is unfortunately a major obstacle, as we have seen previously with Zynteglo, but gene therapy offers great disease transformation, perhaps something close to a cure, even if we don’t have only a few years of results. and we must remain careful. “, 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 the DNA of genes related to hemoglobin, the protein that carries oxygen in the body. Casgevy therapy involves 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 the patient. The effect of a single treatment can last a lifetime.
American Victoria Gray was the first sickle cell patient to try the experimental therapy Casgevy, in July 2019. Her blood cells were altered and blocked her arteries, causing excruciating pain and a high risk of dying from a stroke. “Thanks to my supercells, my life has completely changed,” he proclaimed a year ago during the third international conference on human genome editing, held in London.
This article is originally published on .nouvelles-du-monde.com
