NEW SICKLE CELL THERAPIES COST AN ARM & A LEG
TWO milestone treatments for sickle cell disease are being hailed as valuable new weapons against the lifethreatening condition — but the multimillion-dollar costs may put them out of reach! Casgevy and Lyfgenia, the first gene therapies approved by the FDA to combat the inherited blood disorder, work by genetically modifying a patient’s stem cells.
Until now, the only known cure for sickle cell was a $400,000 bone marrow transplant, which faces two major hurdles the difficulty of finding a matching donor and a risk of rejection by the recipient’s immune system.
However, the new treatments do not have those problems since each patient is their own donor.
In people with the disease, red blood cells, which are normally disc-shaped, become crescent- or sickleshaped, leading to clots and blockages in blood vessels. This starves tissues of oxygen, causing patients extreme pain, breathing problems, organ damage, stroke and often early death. Casgevy, the first therapy to be approved in the U.S. that uses the gene-editing tool CRISPR, changes DNA in stem cells of patients, so the body will no longer produce sickle-shaped blood cells. The onetime treatment, which costs $2.2 million, needs months of preparation including chemotherapy and blood transfusions to destroy any flawed stem cells lurking in bone marrow — before the edited stem cells can be reinfused into the patient. In a trial of 44 people, all the treatments worked, and 29 participants went two years without any severe blood-clotting episodes! Lyfgenia, which carries a $3.1 million price tag, uses a lentiviral vector — a tool to stably transfer and express genes. The process for treating the patient is similar to CRISPR as cells are removed, modified and reinfused.
“Anything that can help relieve somebody with this condition of the pain and the multiple health complications is amazing,” says Dr. Allison King, a professor at Washington University School of Medicine.
“It’s horribly painful.
Some people will say it’s like being stabbed all over.”