New Gene Therapy Offers Hope for Sickle Cell Anemia Patients
FDA to Consider New Gene Therapy for Sickle Cell Anemia Expert Advisors Weigh In
FDA Advisors to Consider New Gene Therapy for Sickle Cell Anemia
By Cara Murez HealthDay Reporter
MONDAY, Oct. 30, 2023 (HealthDay News) – Brace yourselves, folks! Sickle cell disease patients may soon have not one, but two new treatments to cheer about!
On Tuesday, a U.S. Food and Drug Administration (FDA) advisory committee will take a deep dive into the potential of a cutting-edge gene therapy for this painful, hereditary condition that mainly affects the Black community. Hold your breath, because the agency is expected to make a decision on this therapy in early December, along with another groundbreaking treatment. Fingers crossed, my friends!
So, what’s the deal with this remarkable gene therapy? The treatment in question is based on CRISPR technology, a gene-editing tool that nabbed the inventors a well-deserved Nobel Prize in 2020. Get this: the therapy, named “exa-cel,” is a one-time treatment that permanently changes the DNA in a patient’s blood cells. Talk about the stuff of legends!
But how does it work, you ask? Well, first, they remove some stem cells from the patient’s blood. Then, using CRISPR, they “knock out” a gene that causes the pesky development of those sickle-shaped blood cells. This is followed by a dose of medicine that wipes out those defective cells. And voilà! The patients are given back their own, beautifully altered stem cells. It’s like a magical transformation, my dear readers!
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According to Dr. Allison King, a professor at Washington University School of Medicine in St. Louis, “anything that can help relieve somebody with this condition of the pain and the multiple health complications is amazing.” And trust me, she’s not exaggerating. People with sickle cell disease describe the pain as being “stabbed all over.” Ouch!
Now, I know you’re all on the edge of your seats, so let me tell you what happens next! On Tuesday, the FDA’s advisory panel will huddle together and discuss whether further research is needed to uncover any potential unintended consequences of this new gene therapy. Because hey, we can’t be too careful when it comes to messing with DNA, right?
In documents filed with the advisory committee, Vertex, the company behind the therapy, boasted some impressive stats. Out of the 46 individuals who received the treatment in their study, 30 had 18 months of follow-up. And guess what? Twenty-nine of those lucky souls were free of pain crises for at least a year. And all 30 avoided any hospitalization due to these excruciatingly painful episodes. Can you believe it?
Of course, the FDA panel wants to cover all bases, so they’ve called on outside gene therapy experts to discuss the possibility of “off-target effects.” Translation: they want to know if there are any unexpected changes to a person’s genome because, let’s face it, nobody wants any nasty surprises.
The company, however, has a plan. They’ve proposed a post-approval safety study and a product label that clearly outlines the potential risks. Safety first, you know!
But let’s not forget the human side of all this. Victoria Gray, one of the lucky recipients of this gene therapy, described her experience as feeling “reborn.” Imagine that! Gray had been suffering from excruciating pain since childhood, but now she’s active, spending quality time with her kids, and working full time. Truly, a success story in the making!
Sickle cell disease wreaks havoc on the protein responsible for carrying oxygen in our red blood cells. Due to a genetic mutation, these cells often take on a peculiar crescent shape, blocking blood flow and causing unimaginable pain, organ damage, and even strokes. This devastating disease affects millions worldwide, with approximately 100,000 individuals grappling with it in the United States alone.
Interestingly, the prevalence of sickle cell disease is higher in regions where malaria runs rampant, such as Africa and India. It turns out that being a carrier of the trait could actually protect against severe malaria. Who would’ve thought?
Standard treatments for this condition currently include medications and blood transfusions. However, a bone marrow transplant from a closely matched donor is the only definitive cure. But here’s where the game changes!
The second gene therapy that the FDA will be considering also aims to tackle sickle cell disease. How, you ask? By creating functional copies of a modified gene. This helps our red blood cells produce well-behaved hemoglobin that isn’t misshapen. And guess what? This treatment comes courtesy of Bluebird Bio, another player in the gene therapy game.
Now, I bet you’re all thinking about one thing: the price tag. Sadly, we don’t have that information yet. But here’s a shocker for you! If these gene therapies come with a price of around $2 million, it would actually be considered cost-effective. Why, you ask? Because existing treatments rack up a bill of roughly $1.6 million for women and $1.7 million for men from birth to age 65. So, even though it’s a hefty price tag, can you really put a price on feeling better, avoiding pain, and staying out of the hospital?
My dear readers, we’re witnessing a revolution in the treatment of sickle cell disease. This gene therapy could turn lives around, eradicating pain and restoring hope. The FDA is diligently weighing the potential risks and rewards, consulting with experts, and considering the future of this groundbreaking therapy. So, let’s keep our fingers crossed and hope for a resounding victory in the fight against sickle cell disease!
More information The U.S. Centers for Disease Control and Prevention has more on sickle cell disease.
SOURCES: Associated Press 
QUESTION Sickle cell disease is named after a farming tool. See Answer
