Britain’s medicines regulator has approved the world’s first gene therapy, billed as a potential cure for two inherited blood disorders.
A treatment for sickle cell anemia and beta thalassemia called Casgevy is the first treatment licensed using the gene-editing tool Crispr, for which its inventors received a Nobel Prize in 2020.
The Medicines and Healthcare Products Regulatory Agency (MHRA) said the treatment is intended for patients aged 12 years and over “following a thorough assessment of its safety, quality and effectiveness”.
Scientists said it was a “historic” day and called the decision an “important step” in advancing medical approaches to combating genetic diseases once considered incurable.
Both sickle cell anemia and beta thalassemia are genetic disorders caused by errors in the genes for hemoglobin, which is used by red blood cells to transport oxygen throughout the body.
Sickle cell anemia is especially common among people of African or Caribbean descent, while beta thalassemia primarily affects people of Mediterranean, South Asian, Southeast Asian, and Middle Eastern descent.
Symptoms of sickle cell disease can include severe pain, severe and life-threatening infections, and anemia. People with beta thalassemia may develop severe anemia. In addition to regular injections and medications, patients often require blood transfusions every three to five weeks.
Casgevy is designed to work by editing a faulty gene in a patient’s bone marrow stem cells so that the body produces functional hemoglobin. To achieve this goal, stem cells are taken from the bone marrow, processed in a laboratory, and then reintroduced into a patient, potentially curing people.
The MHRA said clinical trials showed Kasgevy restored healthy hemoglobin production in most people. In a clinical study of sickle cell disease, analysis showed that 28 of 29 patients (97 percent) had no severe pain at least 12 months after treatment.
In a beta thalassemia clinical trial, 39 of 42 patients analyzed (93 percent) did not require red blood cell transfusions for at least 12 months after treatment. In the remaining three cases, the need for red blood cell transfusions was reduced by more than 70 percent.
Julian Beach, interim director of healthcare quality and access at the MHRA, said both conditions are “painful, lifelong conditions that can be fatal in some cases”.
He said: “Until now, the only permanent treatment option has been a bone marrow transplant, which must be obtained from a precisely matched donor and carries the risk of rejection.”
Dr Alena Pans, senior lecturer in genetics at the University of Hertfordshire, said: “This is an important step in advancing medical approaches to tackle genetic diseases that we never thought could be cured.”
“By modifying stem cells from a patient’s bone marrow, immune compatibility problems can be avoided. those. Finding donors who are suitable for the patient and immunosuppression, which is a real cure for the disease, not a cure.
“The most interesting thing about this is the gene editing strategy used, since blood diseases can be caused by many different mutations that are difficult to target individually.”
Crispr is a unique technology that allows geneticists and medical researchers to edit parts of the genome by removing, adding or changing parts of the DNA sequence. It is faster, cheaper and more precise than previous DNA editing methods and has a wide range of potential applications.
Reshma Kewalramani, president of Vertex, which promoted the drug with Crispr Therapeutics, said: “Today is a historic day in science and medicine: the approval of Casgevy in the UK is the first regulatory approval of a Crispr-based therapy in the world. »
The NHS has not yet explored the possibility of wider use of this treatment.
John James, chief executive of the Sickle Cell Society, said: “Sickle cell disease is an incredibly debilitating disease, causing severe pain for those affected and potentially leading to premature death.” “The new treatment appears to be safe and effective and has the potential to significantly improve the quality of life for so many people.”
Source: I News
I’m Raymond Molina, a professional writer and journalist with over 5 years of experience in the media industry. I currently work for 24 News Reporters, where I write for the health section of their news website. In my role, I am responsible for researching and writing stories on current health trends and issues. My articles are often seen as thought-provoking pieces that provide valuable insight into the state of society’s wellbeing.