On December 8, 2023, the United States approved a ground-breaking treatment for sickle cell disease, a crippling blood condition, that makes use of the cutting-edge gene editing technology CRISPR.
Over 100,000 Americans, primarily Black, are afflicted with sickle cell disease, a debilitating and potentially fatal condition that has proven difficult for medical science to treat.
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Two gene therapies have been approved by the US Food and Drug Administration (FDA), one of which is named Casgevy and makes use of the CRISPR technology that won the Nobel Prize.
“These treatments represent a major advancement in the field of gene therapy for patients with sickle cell disease,” the FDA Center for Biologics Evaluation and Research’s director, Peter Marks, stated. “The potential these products have to transform the lives of patients living with sickle cell disease is enormous.”
The creators of CRISPR, a gene editing method that will earn a Nobel Prize in 2020, can precisely alter the DNA of plants, animals, and microbes. The technology, which has been hailed for its astounding potential, has transformed the study of molecular life and is already having an impact on crops resistant to drought and experimental cancer treatments.
“This approval means that for the first time, approximately 16,000 patients with SCD may be eligible for a durable one-time therapy that offers the potential of a functional cure for their disease,” Maker Vertex of Casgevy noted in a statement.
Lyfgenia, the second approved medication, modifies genetic material using a virus that is safe to use.
Normal red blood cells pass through blood vessels with ease; however, in sickle cell disease, these cells take on a crescent or “sickle” form that obstructs blood flow and increases the risk of stroke, eye issues, excruciating pain, and even death. A bone marrow transplant has been the only treatment for sickle cell disease up until this point.
The declaration made on Friday, according to US President Joe Biden, “represents the power of medical innovation to improve Americans’ lives.”
“My Administration will continue our efforts to accelerate the development of cures for rare diseases and support the medical research and innovation that achieved this breakthrough,” he remarked.
The CRISPR gene therapy was approved by Britain’s pharmaceutical authority last month, but this authorization marks the first for the US on Friday.
During the treatment, the patient’s blood stem cells are altered using CRISPR genome editing technology and subsequently reintroduced.
The fetal hemoglobin (HbF) produced by the altered blood stem cells is increased, aiding in oxygen delivery and preventing red blood cells from “sickling.” 29 out of 31 individuals in a clinical trial did not have a “pain crisis” for a minimum of a year during a two-year follow-up period.
Vomiting, nausea, stomach pain, and mouth sores were a few of the adverse symptoms.
“Gene therapy holds the promise of delivering more targeted and effective treatments, especially for individuals with rare diseases where the current treatment options are limited,” declared Nicole Verdun, the head of the Office of Therapeutic Products at the FDA.
She stated that follow-up investigations will occur over 15 years, and additional patient monitoring is intended for the remainder of their lives.
Additionally, researchers are working on ways to fix damaged genes in big organs like the brain, which could lead to the development of treatments for debilitating conditions like Huntington’s disease and muscular dystrophy.
