Kendric Cromer, a 12-year-old kid from a suburb of Washington, has become the first person in the world to receive commercially licensed gene therapy for sickle cell disease.
According to the New York Times, Kendric's successful therapy provides hope to around 20,000 people in the United States who suffer from sickle cell disease. However, it also emphasises the difficulties patients confront in obtaining novel sickle cell medications.
After receiving the therapy, Kendric said, "Sickle cell always steals my dreams and interrupts all the things I want to do." He believes that now he has a chance to live a normal life like other kids.
What is Lyfgenia & how much does the treatment cost?
Based in Somerville, Massachusetts, Bluebird Bio developed a gene therapy treatment called 'Lyfgenia'. The cost of Kendric's treatment is approximately $3.1 million, which was covered by his family's insurance.
The first phase of his treatment at Children's National Hospital in Washington, D.C., where doctors removed his bone marrow stem cells, which the Bluebird will genetically tweak for his treatment. The modified cells will return to his body within three months.
For the treatment, doctors need hundreds of millions of stem cells from the patient, and if the first extraction, which takes six to eight hours, is not enough, the firm will try one or two more extractions.
FDA gives approval to two companies to sell gene therapy
{{/usCountry}}For the treatment, doctors need hundreds of millions of stem cells from the patient, and if the first extraction, which takes six to eight hours, is not enough, the firm will try one or two more extractions.
FDA gives approval to two companies to sell gene therapy
{{/usCountry}}The FDA authorised two firms to sell gene therapy to patients with sickle cell disease, a genetic illness that affects approximately 100,000 Americans, which include mostly Black people.
Bluebird has estimated that it can only treat between 85 and 105 patients per year with sickle cell or beta thalassemia. However, Children's National stated that it can only treat 10 patients with gene therapy each year.
Andrew Obenshain, chief executive officer of Bluebird, said in a statement, “This historic moment comes nearly a century after sickle cell disease was the first genetic disorder to be characterized at the molecular level, and almost a decade after bluebird initiated clinical development for our sickle cell gene therapy.”
Meanwhile, a confirmation from Vertex Therapeutics of Boston is still awaited if it has begun treating any patients with its authorised CRISPR gene-editing medication.
Furthermore, medical centers have a restricted ability to manage gene therapy patients as they need professional and intense care. After the patient's stem cells are treated, they are required to stay in the hospital for a month, during which they get seriously ill due to intense chemotherapy.