Modern medicine is one step closer to a possible cure for sickle cell disease.
Following the UK's approval of the world's first gene-editing treatment for sickle cell disease on November 16th, the US Food and Drug Administration (FDA) also approved the same treatment on December 8th.
“FDA approval of CASGEVY is extremely important,” said Reshma Kewalramani, MD, CEO and President of Vertex Pharmaceuticals. in bostonsaid in a statement provided to Fox News Digital.
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“This is the first CRISPR-based gene editing therapy It will also be approved in the US.”
Peter Marks, M.D., director of the FDA's Center for Biologics Evaluation and Research, said, “These approvals highlight the use of innovative cell-based gene therapies to target potentially devastating diseases and improve public health. “This represents an important medical advance.” , in an agency press release.
The U.S. Food and Drug Administration has approved the world's first gene-editing treatment for sickle cell disease. (St. Petersburg)
He also said, “Today's action was based on a rigorous evaluation of the scientific and clinical data necessary to support approval,” and “reflects FDA's commitment to expediting approval.” ” he also said. [the] Developing safe and effective treatments for conditions that seriously impact human health. ”
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The CASEVY treatment (also known as exa-cel, or exagamglogene autotemcel) uses technology to “edit” the gene that causes sickle cell disease.
Boston-based Vertex and Switzerland-based CRISPR Therapeutics used a gene-editing system known as CRISPR-Cas9 to develop the drug, according to the American Sickle Cell Disease Association website. He said he turned it on and off.
The CASEVY treatment (also known as exa-cel, or exagamglogene autotemcel) uses technology to “edit” the gene that causes sickle cell disease. (St. Petersburg)
“CASGEVY is a first-in-class treatment that offers the potential for a one-time, transformative therapy for eligible sickle cell disease patients,” said Kewalramani.
The inventors of CRISPR technology were awarded the Nobel Prize in 2020.
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CRISPR stands for “Clustered Regularly Interspaced Short Palindromic Repeats,” which are repeating sequences of DNA.
“CRISPR technology [that is] It is noteworthy that this therapy is being used in conjunction with a treatment for sickle cell disease, a devastating genetic disease that has a significant impact on survival and quality of life.'' said Department Chair Dr. Michael Sekeres. University of Miamihe told FOX News Digital.
The treatment is approved for people 12 years and older with severe sickle cell disease, who suffer from a recurrent pain syndrome known as vaso-occlusive crisis, in which blood flow is blocked by sickle cells. (St. Petersburg)
He was not involved in the development of the drug.
“The data from an admittedly small study supporting the FDA's approval of the treatment are impressive, and the treatment was considered safe by a panel of independent experts,” Sekeres said. he added.
“CASGEVY is a first-in-class therapy that offers the potential for a one-time, transformative treatment for eligible sickle cell disease patients.”
The treatment is approved for people 12 years and older with severe sickle cell disease, who suffer from a recurrent pain syndrome known as vaso-occlusive crisis, in which blood flow is blocked by sickle cells.
The FDA also approved Rifgenia, the first cell-based gene therapy, for the treatment of sickle cell disease in people 12 years of age and older, according to an FDA press release.
What you need to know about sickle cell disease
As the National Institutes of Health (NIH) notes on its website, sickle cell disease is the most common inherited blood disorder in the United States, affecting an estimated 100,000 Americans.
It disproportionately affects people of black or African descent.
Differences in blood flow between sickle cell disease and normal, healthy blood vessels have been shown. (St. Petersburg)
Red blood cells produced in the bone marrow contain a protein called hemoglobin, which helps carry oxygen throughout the body.
The cells are typically round in shape and can easily pass through small blood vessels, according to the Centers for Disease Control and Prevention (CDC) website.
According to the NIH, patients with sickle cell disease inherit a genetic mutation that produces abnormal hemoglobin, which makes it more difficult for cells to move through the bloodstream.
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Diseased cells can eventually become lodged in blood vessels, blocking blood flow and sometimes causing painful seizures (known as sickle cell crisis), infections, and acute chest syndrome. or strokeaccording to the CDC.
The cells begin to take the shape of a C-shaped agricultural tool known as a sickle, which gives the disease its name.
Treatment mechanism
meanwhile in the wombThe fetus produces a type of hemoglobin called fetal hemoglobin, which does not cause sickle cells in cells and has been shown to lessen symptoms in people with sickle cell disease.
After the baby is born, the BCL11A gene tells red blood cells to stop producing fetal hemoglobin. This gene typically turns off within six months of birth, a Vertex spokesperson told FOX News Digital.
Patients with sickle cell disease inherit a genetic mutation that produces abnormal hemoglobin, which makes it more difficult for cells to move through the bloodstream. (St. Petersburg)
CASEVY works by targeting and “turning off” the BCL11A gene.
According to Vertex, this allows for continued production of fetal hemoglobin and significantly reduces damage caused by sickle cells.
This treatment does not “cure” sickle cell disease, but because the patient still has the sickle cell mutation, even if some of the patient's red blood cells continue to become sickle cells, the symptoms It may be possible to eliminate most of the symptoms. a spokesperson told Fox News Digital.
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To receive treatment, patients undergo the following tests: Bone marrow transplantation.
First, patients are given drugs that help stem cells move from the bone marrow into the bloodstream. The blood is then collected and the stem cells isolated.
The cells are then transported to a laboratory, where genetic techniques are used to edit the DNA.
Sickle cell disease is the most common inherited blood disorder in the United States, affecting an estimated 100,000 Americans. (St. Petersburg)
The patient then undergoes chemotherapy to ideally destroy all stem cells that could produce sickle cells in the future.
The edited cells are then injected into the patient under the supervision of a medical professional to ensure that healthy cells are produced in the bone marrow.
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The FDA recommends monitoring patients for health problems for 15 years after treatment.
Potential limitations and caveats
Experts say more research is needed to determine the long-term effectiveness of the treatment.
The American Sickle Cell Association cautioned that it is unclear whether the treated stem cells will continue to produce non-sickle red blood cells for the rest of the patient's life, or whether the cells will die after a certain number of years. Its website.
The diseased cells begin to take on the shape of a C-shaped agricultural tool known as a sickle, which gives the disease its name. (St. Petersburg)
Because the patient is also undergoing chemotherapy, infertility, secondary cancer or infectious complications.
Treatment costs and eligibility restrictions may limit who can receive treatment.
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According to recent reports, treatment can cost millions of dollars per patient.
“The million-dollar question is: How can people with this disease access this life-changing treatment? medical system We can sustain those costs,” Sekeres told FOX News Digital.
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