The regulatory authorities in the United States approved two gene therapies for sickle cell disease on Friday, which doctors hope will be able to treat this painful hereditary illness that primarily affects Black individuals in the United States.
Sickle Cell Disease
Sickle cell disease is considered a rare and devastating illness that threatens the lives of patients and causes them many problems. This disease is characterized by distorted red blood cells that carry oxygen, with these cells taking on a crescent shape, which can hinder blood flow and cause severe pain, organ damage, stroke, and other complications.
Approved Gene Therapies
These approved gene therapies are the first therapies approved in the United States for sickle cell disease. The U.S. Food and Drug Administration had previously approved 15 gene therapies for other conditions. The gene therapies involve a permanent change in the DNA of the patient’s blood cells. The treatment developed by Vertex Pharmaceuticals and CRISPR Therapeutics aims to help the body produce a type of fetal hemoglobin that is present at birth – as the adult form of hemoglobin is defective in individuals with sickle cell disease. The CRISPR technology is used to disable a gene in stem cells collected from the patient.
The treatment developed by Bluebird Bio aims to add modified copies of a gene that helps red blood cells produce “anti-sickling” hemoglobin that prevents or reverses cell distortion.
Effects of Gene Therapies
Studies conducted to test these therapies indicate they work well. Among the individuals treated in the main study conducted by Vertex, 29 out of 31 people were free from pain crises for at least a year. In the study conducted by Bluebird, 28 out of 32 patients had no severe pain or organ damage between six and eighteen months after treatment.
However, doctors indicate that there are potential side effects and that the long-term results of the treatment are unknown. For both therapies, the necessary chemotherapy comes with risks such as infertility, hair loss, and susceptibility to serious infections. Leukemia occurred with the treatment developed by Bluebird, prompting the FDA to state that a “black box” warning regarding this risk will be placed on the packaging. For the treatment developed by Vertex, some scientists are concerned that CRISPR technology could lead to the possibility of “unintended effects” on a person’s genome.
Challenges of Gene Therapies
Doctors say they do not expect all medical centers to offer gene therapies because they require a lot of equipment and coordination among specialists. They also do not expect many people to seek the treatments immediately. Dr. Benjamin Watkins, director of the stem cell and cell therapy program at Children’s Hospital New Orleans, stated that some may want to wait until more people receive the treatments.
Experts also warned that cost may be an obstacle. Bluebird Bio’s price is $3.1 million, and Vertex’s price is $2.2 million. What patients pay will depend on health insurance coverage and other factors. To help cover the costs, the Centers for Medicare and Medicaid Services in the United States announced a plan aimed at establishing partnerships with Medicaid agencies and pharmaceutical companies.
Nevertheless, Dr. Monica Bhatia, who treats children with sickle cell disease at NewYork-Presbyterian Hospital, said that gene therapy “could be revolutionary and truly change the landscape of sickle cell disease.” Galen Matthews, who volunteers at the Sickle Cell Association in Kentuckiana, expressed optimism that the therapies will have a significant impact.
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Source: https://apnews.com/article/sickle-cell-fda-approval-vertex-crispr-8d85279d7de0c60888d37afbfa06d39e
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