A special party for Kendric Cromer, 12 “had to be postponed because he was too groggy to celebrate,” reports the New York Times:
It was meant to mark the first day of his new life — the day he became one of the first children ever to be treated with a newly approved gene therapy that will free him from the sickle cell disease that has stolen his childhood.
On Sept. 11, despite the excitement of the moment, Kendric was unable to keep his eyes open as he lay in his hospital bed at Children’s National Hospital in Washington because of the drugs he had been given in preparation for his treatment.
His life with the disease has been punctuated by episodes of excruciating pain, requiring days in the hospital as doctors tried to control it. Sickle cell eroded his hip bones. It prevented him from riding a bike or playing soccer or even going outside when the temperature was below 55 degrees Fahrenheit because cold often brought on intense pain.
Now he could see a future — in a month or so — without pain from sickle cell. His disease is caused by an inherited genetic mutation that leads to blood cells that form crescent shapes — sickles — instead of discs. Trapped in blood vessels and organs, the cells cause damage and pain. Gene therapy fixes that problem by giving the patient’s blood-forming cells a new, normal hemoglobin gene. An estimated 100,000 people in the United States, most of them Black, have sickle cell disease.
Gene therapy dangles the prospect of normalcy for the estimated 20,000 people in the United States with the most severe forms of the disease — lives without constant pain and continuing damage to organs and bones and joints.
But all is not well in the world of sickle cell gene therapy….The treatment is labor intensive, requiring patients to spend at least a month in the hospital. City of Hope can treat at least one patient a month, Dr. Wang said. Other large medical centers said they could treat only 10 or fewer per year, and some say they can treat just five or six.
The treatment is mind-bogglingly expensive. In December, the FDA “approved the country’s first gene-editing treatment, Casgevy, for use in patients with sickle cell disease. It will cost $2.2 million per patient per treatment, according to NBC. Later that same day, the FDA approved another sickle-cell drug called Lyfgenia. It will cost $3.1 million per patient per treatment. If all of the tens of thousands of people who could benefit from the treatment get it, that will cost tens of billions of dollars, much of it presumably covered by taxpayers, directly or indirectly. Half of all U.S. healthcare spending is by federal, state, or local governments.
But for people in agonizing pain from sickle cell disease, the cost will presumably be worth it. NBC reported that
the approval comes about a decade after the discovery of CRISPR technology for editing human DNA, representing a significant scientific advancement….Casgevy, co-developed by Vertex Pharmaceuticals and CRISPR Therapeutics, uses Nobel Prize-winning technology CRISPR to edit a person’s genes to treat disease. The treatment was approved by U.K. regulators last month….. Casgevy uses CRISPR to make an edit to a person’s DNA that turns on fetal hemoglobin, a protein that normally shuts off shortly after birth, to help red blood cells keep their healthy full-moon shape. In clinical trials, Casgevy eliminated pain crises in most patients. The FDA approved the treatment for people 12 years and older…
While the treatment itself is administered only once, the whole process takes months. Blood stem cells are extracted and isolated before being sent to Vertex’s lab, where they’re genetically modified. Once ready, patients receive chemotherapy for a few days to clear out the old cells and make room for the new ones. After the new cells are infused, recipients spend weeks in the hospital recovering. Vertex will take the lead on launching the drug and estimates about 16,000 people with severe cases of sickle cell will be eligible….Because the procedure is so complex, it will be limited to certain health facilities like academic medical centers. Nine health-care facilities are ready to start administering Casgevy, Vertex said in a release, with more facilities added in the coming weeks….
The FDA also on Friday approved a separate gene therapy by Bluebird Bio, called Lyfgenia that works differently than Casgevy but is administered similarly and is also intended to eliminate pain crises. That therapy was similarly approved for the treatment of sickle cell disease in people 12 years and older.
