Three years ago, the technology was awarded the Nobel Prize, a research that was partly developed at the University of Umeå. Now Britain has become the first country in the world to approve the so-called gene scissors for the treatment of two serious blood disorders.
The gene therapy means that the patient is first given chemotherapy, before doctors extract stem cells from the patient’s bone marrow. But using the so-called crispr technology, often described as the gene scissors, the cells can be treated in a laboratory before being returned to the patient.
The treatment has been approved for patients over twelve years of age with sickle cell anemia and thalassemia. Both are diseases that are currently treated with complicated blood transfusions or bone marrow transplants with severe side effects as a result.
“The word ‘cure’ has so far been incompatible with sickle cell anemia and thalassemia,” says Helen O’Neill, geneticist at University College London.
She considers the approval to be a “historic, positive moment in history”.
— The future of life-changing medicines lies in crispr-based technology.
The approval for sickle cell anemia is based on a study with 29 patients. 28 of them had no serious problems from the disease one year after the treatment. For thalassemia, 39 of the 42 patients in the study did not need any transfusions.