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In a groundbreaking decision, the Food and Drug Administration approved the first gene-editing therapy to treat patients with sickle cell anemia, a crippling condition that leaves sufferers with life-altering pain.
On Friday, the administration gave the green light to Casgevy and Lyfgenia, for the treatment of sickle cell anemia in patients 12 years and older.
Casgevy is specifically the first FDA-approved treatment to use a type of new gene editing technology called CRISPR. Lyfgenia uses conventional gene therapy, not gene editing, to treat the condition.
Sickle cell anemia is the general term for a group of inherited conditions that severely affect the shape and function of red blood cells. It affects 100,000 Americans and 15,000 Britons, most of whom are black.
These therapies could bring hope to the tens of thousands of Americans suffering from this condition, which has only been approved for treatment through a bone marrow transplant, an invasive procedure for which most patients do not qualify.
However, while the news brings hope to sickle cell patients, there is concern in the medical community that the therapies will be difficult to access due to the expected high cost and a shortage of hospitals capable of administering them.
Patients with sickle cell anemia, of which there are about 100,000 in the United States, do not adequately produce hemoglobin, a substance in red blood cells that carry oxygen throughout the body. As a result, your red blood cells become rigid and shaped like a crescent (pictured) instead of a disk, which can cause them to die and become trapped in your blood vessels.
Casgevy, made by Boston-based Vertex Pharmaceuticals (pictured) and Crispr Therapeutics in Switzerland, works by editing the defective HBB gene behind both conditions in a patient’s bone marrow stem cells so the body produces functional hemoglobin. .
In a healthy person, red blood cells (produced by stem cells within the bone marrow) are round, concave discs that can bend and flex easily.
However, in people with sickle cell disease, defective stem cells produce red blood cells that are crescent-shaped. These cells are rigid, unable to pass through smaller blood vessels and prone to causing blockages that deprive parts of the body of oxygen, causing immense pain and organ damage.
Until now, a bone marrow transplant was the only approved treatment for this condition. A transplant is a procedure in which healthy blood-forming stem cells from a healthy donor are transplanted to replace the patient’s bone marrow that is not producing enough healthy cells.
Stem cells are the body’s “raw materials,” or cells that can become many different types of specialized cells. They can be used to repair damaged tissues, and researchers believe that stem cell therapies may one day treat conditions such as Alzheimer’s disease and paralysis.
In most bone marrow transplant cases, the donor is a sibling, but even a sibling only has a one in four chance of being a match for the patient. And transplants are often not performed because of risks, which include the transplanted cells attacking other cells in the recipient’s body, which can be life-threatening.
More than 30 FDA-approved gene therapies are used to treat several different types of cancer and the blood disorder hemophilia. However, many of them are largely inaccessible due to their high costs.
Casgevy was recently approved in the United Kingdom to treat sickle cell and transfusion-dependent β-thalassemia, a shortage of red blood cells that causes severe anemia. It is expected to cost the UK government approximately $1.25m (£1m) per patient.
Casgevy, made by Boston-based Vertex Pharmaceuticals and Crispr Therapeutics in Switzerland, is the first licensed drug using the innovative CRISPR gene-editing tool. This process is known as “genetic scissors” and allows scientists to make precise changes to DNA. Its inventors received the Nobel Prize in 2020.
The therapy works by editing the defective HBB gene, which causes sickle cell anemia, in the patient’s bone marrow stem cells, so that the body can make properly functioning hemoglobin, the protein in red blood cells responsible for carrying oxygen to the cells. tissues throughout the body.
To do this, stem cells are extracted from a patient’s bone marrow and edited in a laboratory using molecular “scissors”, which precisely deactivate the defective gene.
The stem cells are then infused back into the patient, who may need to spend a month or more in the hospital while the treated cells begin to produce healthy red blood cells.
Scientists believe the results have the potential to last a lifetime.
An ongoing trial of the drug so far shows that 97 percent of sickle cell patients did not suffer severe pain for at least a year after treatment.