FDA Approves Gene-Editing Treatment for Sickle Cell Anemia
OAN’s Brooke Mallory
3:57 PM – Friday, December 8, 2023
A ground-breaking gene-editing therapy for sickle cell disease, a debilitating ailment that affects around 100,000 Americans, the majority of whom are people of color (POC), was authorized by the U.S. Food and Drug Administration (FDA) on Friday.
The therapy claims to be able to repair the disease-causing gene.
For Johnny Lubin, a 15-year-old from Connecticut who has seen the crippling symptoms of the condition, the discovery provides a glimmer of hope. Since birth, he has struggled with excruciating pain and health issues related to his sickle cell gene, which he genetically received from both of his parents.
Being typically shaped like “doughnuts,” red blood cells bend into stiff sickle shapes that clog blood vessels and impair the body’s ability to absorb oxygen.
Physicians had informed Lubin that he would not live past the age of 40.
“I was starting to get a little bit scared. Like, I actually did want to live past 40,” he said.
Lubin was in and out of hospitals for almost ten years. He claimed that he would keep track of the number of times he visited each hospital room, and that one day he discovered he had visited every room on the floor.
When Fabienne and J.R. Lubin, Johnny’s parents, found out about a state-of-the-art clinical study utilizing gene editing—a procedure that does not require a donor—they were desperate for a cure.
Lubin first had chemotherapy to help eradicate the aberrant cells after stem cells were taken out of his bone marrow.
The protective form of hemoglobin, a protein that absorbs oxygen from the lungs and distributes it throughout the body, was then increased in a lab using the editing technique known as “CRISPR.” Normally, this protective form of hemoglobin decreases after birth. After that, the cells were reinfused into Lubin’s circulation.
The leader of pediatric stem cell transplantation at New York-Presbyterian/Columbia University Irving Medical Center and Johnny’s physician, Dr. Monica Bhatia, explained that altering the cell reprograms it to generate fetal hemoglobin.
“It’s been widely known that fetal hemoglobin is somewhat protective, and those who have higher levels of fetal hemoglobin tend to have less severe symptoms of sickle cell disease,” she said.
“You’re changing somebody’s DNA. So obviously, you wanna make sure that the corrections you’re making are, are the ones you want,” Bhatia added.
Lubin now has much improved health and the possibility of living a longer life after a difficult five weeks in the hospital and a six-month leave from school.
“I thought that was pretty cool how I have like new cells, and I honestly hoped, you know, I could get, you know, some super powers from it, you know, maybe become a superhero, you know, like genetically engineered,” Lubin said.
Vertex Pharmaceuticals and CRISPR Therapeutics, located in Boston, developed the medication known as Casgevy.
Before the specialists declare the medication to be a guaranteed cure, patients must be monitored for an extended period of time. Although gene editing may not be suitable for every sickle cell disease patient, it is anticipated to cost several million dollars per patient.
However, it would not stop the gene from being inherited by future generations.
Stay informed! Receive breaking news blasts directly to your inbox for free. Subscribe here. https://www.oann.com/alerts
From a massive drop in support among the Muslim community to Biden’s re-election campaign scrambling to save face. One America’s Monica Paige breaks down the week at the Biden White House.
A unique store in Scottsboro, Alabama, called unclaimed baggage, leaves little to the imagination with their name.
The President’s son Hunter is slammed with nine new tax-related charges in a new indictment.
West Virginia and Indiana co-lead an amicus brief asking the Michigan Court Of Appeals to keep 45th President Donald Trump on the ballot in both the primary and general elections.
Amazon.com asked a federal court to dismiss a U.S. government antitrust lawsuit which accuses the company of using illegal strategies to boost profits at its online retail empire.
More than 10 million people have signed up for X in December, X CEO Linda Yaccarino said in a post on the social media platform.
Alphabet shares ended 5.3% higher on Thursday as Wall Street cheered the launch of Gemini.
Meta Platforms will add invisible watermarking to its text-to-image generation product imagine with Meta AI chatbot in the coming weeks to enhance transparency.
rnrn
What are the ethical considerations associated with gene-editing therapy for sickle cell disease?
To our newsletter for the latest updates.
The Significance of Gene-Editing Therapy
The authorization of gene-editing therapy by the FDA marks a significant breakthrough in the treatment of sickle cell disease. This debilitating ailment, which primarily affects people of color, has caused immense suffering and decreased life expectancy for thousands of Americans. With the new therapy, there is hope for a better quality of life and a longer lifespan for individuals like Johnny Lubin.
Sickle cell disease is characterized by red blood cells that are abnormally shaped, resembling sickles. These misshapen cells clog blood vessels and hinder the body’s ability to absorb oxygen. People with sickle cell disease experience excruciating pain and are prone to various health issues. Many have been told that their life expectancy is limited, causing anxiety and fear.
However, gene editing offers a promising solution. The therapy works by repairing the disease-causing gene and increasing the production of a protective form of hemoglobin known as fetal hemoglobin. Research has shown that higher levels of fetal hemoglobin can lead to milder symptoms of sickle cell disease.
The successful implementation of gene editing in Johnny Lubin’s case is a testament to its potential. Lubin, who had been in and out of hospitals for years, saw a significant improvement in his health after undergoing the therapy. The procedure involved chemotherapy to eliminate aberrant cells and increase fetal hemoglobin levels, followed by the reinfusion of modified cells into his bloodstream.
Ethical Considerations and Future Prospects
While the approval of gene-editing therapy brings hope for many individuals suffering from sickle cell disease, there are ethical considerations that need to be addressed. Altering a person’s DNA raises concerns about the long-term effects and the potential unintended consequences of manipulating genes.
Medical professionals and researchers emphasize the importance of ensuring that gene corrections are accurate and targeted. This highlights the need for rigorous testing and monitoring of patients who undergo gene editing. The therapy’s high cost also poses a significant barrier to accessibility, with estimates suggesting a price tag of several million dollars per patient.
Additionally, it’s important to note that gene editing does not eliminate the possibility of the disease being inherited by future generations. Therefore, comprehensive genetic counseling and education should accompany the therapy to help individuals make informed decisions about family planning.
Despite these challenges, the authorization of gene-editing therapy for sickle cell disease is a major step forward in the medical field. It offers hope, improved quality of life, and the potential for a longer lifespan for those affected by the disease.
Conclusion
The approval of gene-editing therapy by the FDA provides a glimmer of hope for individuals living with sickle cell disease. By repairing the disease-causing gene and increasing the production of protective hemoglobin, the therapy offers the possibility of a better quality of life and increased life expectancy.
However, ethical considerations and the high cost of the therapy must be addressed to ensure that it is accessible and safe for all patients. Long-term monitoring and genetic counseling are essential components of the treatment process.
As research in gene editing continues to advance, there is hope for further breakthroughs in the treatment of genetic disorders. The authorization of this therapy is a significant milestone, bringing us closer to a future where genetic diseases can be effectively managed and potentially cured.
Sources:
" Conservative News Daily does not always share or support the views and opinions expressed here; they are just those of the writer."
*As an Amazon Associate I earn from qualifying purchases
Now loading...