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Breakthroughs In Sickle Cell Treatment You Can't Afford To Miss
Let's take a deep breath and talk about something that hits close to home for many of us, especially in the Black community: Sickle Cell Disease.
If you or someone you love lives with sickle cell, you already know the story. The ER visits. The unpredictable pain. The fatigue that doesn't go away. The fear of strokes, infections, and organ damage. And too often, the feeling of being dismissed or misunderstood in a healthcare system that wasn't built with us in mind.
But here's what you may not have heard enough: there is hope — and it's growing. In recent years, scientists, doctors, and patient advocates have been breaking ground on treatments that aren't just managing symptoms, but aiming to transform lives.
Revolutionary Gene Therapies That Are Changing LivesFor decades, treatment options for sickle cell were limited to blood transfusions, hydroxyurea, and symptom management. These helped, but they weren't a cure. Now, for the first time, gene therapy offers something radically different: a potential one-time treatment that targets the disease at its root.
Exa-cel (Exagamglogene autotemcel) – CRISPR-Based TherapyIn late 2023, the FDA approved Exa-cel, the first-ever CRISPR-based gene therapy for sickle cell disease. This treatment involves taking your own stem cells, editing them with CRISPR technology to fix the gene responsible for sickle-shaped red blood cells, and putting those edited cells back into your body.
In clinical trials, over 90 percent of patients had no more severe pain crises after the therapy.
Lovo-cel (Lovotibeglogene autotemcel) – Another Promising Gene TherapyAlso recently approved, Lovo-cel works differently than CRISPR but with similar outcomes, giving the body a new gene that helps produce normal hemoglobin instead of sickled cells.
These treatments are still new, but they've already changed lives. And while they come with serious risks and aren't for everyone, the progress is undeniable.
Weighing the Pros and Cons: Are These New Treatments Worth It?We must be honest here — gene therapy is not a quick fix. It's complex, intense, and not without side effects. Here's a look at what you'll want to consider if you or a loved one is thinking about it:
ProsStill, the chance at a life without constant pain is worth it for many. But that decision is deeply personal — and should always be made with a trusted medical team who listens to your needs and understands your values.
RELATED: How Does Gene Therapy for Sickle Cell Work? 5 Things You Need to Know
How to Find Out If You're Eligible for Cutting-Edge TherapiesGene therapy isn't for everyone. But knowing your options is the first step toward empowerment.
Here's how to get started:
1. Talk to a hematologist who specializes in SCD.Ask if they're familiar with Exa-cel or Lovo-cel and if you qualify. Generally, patients who experience frequent pain crises or organ damage are prioritized.
2. Find a treatment center conducting gene therapy trials or offering the approved therapies.Use this link to find one near you:ClinicalTrials.Gov – Sickle Cell Gene Therapy Trials
3. Get a full medical evaluation.Gene therapy isn't safe for everyone, especially those with certain organ damage or infections. Your doctor can help assess your candidacy.
4. Ask about financial support and logistics.Many have questions about how they'll afford it, who will care for them after treatment, or what happens if it doesn't work. These are real concerns — and you're not alone in them.
RELATED: I Tried Gene Therapy—and Haven't Needed a Blood Transfusion Since!
First-Hand Stories from Patients Who Took the LeapLet's pause for a second and hear from the people who are actually living this journey. Because it's one thing to read stats. It's another to see real lives transformed.
Marie-Chantal, Philadelphia
I can see myself living in to my 80s and 90s now. I can imagine the life; I'll have for myself the life. I've built for myself And my future family and I'm incredibly excited for it."
Victoria Gray, 39, Nashville, TN
She experienced her first sickle cell crisis at three months old. An emergency room visit led to an unexpected sickle cell disease (SCD) diagnosis. Doctors gave her mother a grim prognosis—a life expectancy of up to seven years.
"My mother initially thought I was inconsolable because my shoulder dislocated during my birth. But after the testing and the doctor's report, she thought I was going to die," Gray said. "Coming from a family of faith, my grandmother encouraged her to trust God. I took on that mentality throughout my entire life." Marcus, 34, New Orleans, LA
Everyone's story is different — and valid. But what unites them is the search for something better. And that search is powerful.
Where to Find Support and Financial Assistance for TreatmentThis is one of the biggest questions people have: How do I pay for this? And it's a fair question. The total cost of gene therapy can reach two million or more. But there's help, and you deserve access to it.
Support Programs and Organizations:
You don't have to navigate this alone. Social workers, advocates, and community health leaders want to walk with you every step of the way.
RELATED: Beyond the Pain: 4 Unseen Obstacles Sickle Cell Warriors Face Every Day
Your Life Is Worth Fighting ForLet's be honest. Sickle cell has been overlooked for far too long. For decades, it got less funding, less attention, and fewer treatment options — although it affects millions, especially Black families across the diaspora.
But that's changing. Because people spoke up. Because researchers pushed forward. Because patients refused to be forgotten.
You deserve to live without constant pain. You deserve a future where you can work, travel, dance, and fall in love — without fear.
Gene therapy isn't a cure for everyone. But it's a light in a space that's been dark for far too long.
In A First For St. Louis, Patient Completes New Gene Therapy To Cure Sickle Cell Disease
ST. LOUIS — Martin Mwita sat on the crumpled exam table paper covered in stars and moons, his hair gone and skin patchy. His mom, Jacinta Mwita, stood by his side.
They were about to learn what kind of blood cells were pumping through Martin's veins.
"It's almost like you need a drumroll," said Dr. Shalini Shenoy, a Washington University hematologist and oncologist at St. Louis Children's Hospital.
Shenoy's news: 85% of Martin's red blood cells were normal. His sickled cells were disappearing.
Jacinta, 51, threw up her hands.
"No way," she cried. "No way!"
Shelani Shenoy, a Washington University pediatric hematologist and oncologist, discusses progress and the dangers ahead with Martin Mwita, 20, on Tuesday, April 22, 2025, at St. Louis Children's Hospital. Mwita, of Omaha, is receiving a new gene therapy treatment for sickle cell disease.
Christian Gooden, Post-DispatchShenoy went on, telling them how Martin's new cells are still growing, how the hope is that the level will soon reach 100%.
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"You're definitely making the good hemoglobin on your own," Shenoy said.
Martin, 20, was the first patient in St. Louis and among the first across the country to receive a new gene therapy for sickle cell disease.
The monthslong therapy involves extracting a patient's own stem cells (which produce red blood cells), genetically modifying them in a lab and transplanting them back to the patient.
In sickle cell disease, a mutation in hemoglobin genes results in crescent-shaped red blood cells instead of round ones. The sickled cells get stuck together, bringing on episodes of severe pain. The clumps can damage organs and cause strokes. Patients tend to die in their mid-40s.
Jacinta Mwita checks her son Martin Mwita's blood pressure on Sunday, April 20, 2025, at their temporary apartment in St. Louis. "She's always driven me to appointments. She's always been bedside. She's always stayed overnight with me," Martin Mwita said. "Whatever it might have been, she was always there."
Zachary Linhares, Post-DispatchAn estimated 100,000 people in the United States have sickle cell. Most are Black. People are born with the disease when they inherit the mutated gene from each parent.
For many patients, the new therapy is their only hope of living a normal life.
Gene therapy to treat sickle cell disease was approved for patients ages 12 and older by the Food and Drug Administration at the end of 2023. Two therapies hit the market — Lyfgenia, made by Bluebird Bio, and Casgevy made by Vertex Pharmaceuticals.
The first commercial patient in the U.S. Completed treatment in October at Washington, D.C.-based Children's National Hospital. With the therapy costing up to $3.1 million, getting insurance approval takes weeks.
A handful of hospitals have announced they've begun treating patients. St. Louis Children's Hospital appears to be the first in the region. Martin and his mom traveled to St. Louis from their home near Omaha, Nebraska.
Though promising, the treatment does not come without risk.
Before the modified cells are transplanted, the patient must undergo an intensive chemotherapy treatment to wipe out the bone marrow — where stem cells are made — so the new stem cells can take hold.
The chemotherapy can cause organ damage and wipes out the immune system, leaving patients susceptible to infections that can be deadly. Patients get extremely ill and develop sores in their mouth and throats. Long-term, chemotherapy raises the risk of cancer and infertility.
It's been a long and difficult journey for the Mwita family. On Tuesday, they finally got the news they've hoped for — that Martin's blood cells weren't going to hurt him anymore.
"The lung, the brain, the kidney — all these should feel much better because you have the normal hemoglobin circulating," Shenoy told them. "This is what you did all this for. All those weeks that you put up with the side effects — this is what it was for."
Children's Hospital nurse Page Myers changes the mainline dressing in Martin Mwita's chest, alongside his mother, Jacinta Mwita on Tuesday, April 22, 2025, in St. Louis.
Christian Gooden, Post-DispatchOptions carry risks
The current standard of care for patients with sickle cell disease includes medications and blood transfusions to temporarily ease symptoms. The only treatment that can lead to a cure is a stem cell (also known as bone marrow) transplant.
Transplants have been mostly limited to those younger than 18 — even as young as age 5 — whose tissues matched a sibling donor or matched a donor on the donor registry list. But finding a match is difficult. Using a half-matched donor is possible, but it carries more risk.
A recipient's immune system can reject and attack the donor cells. To prevent that, recipients must take immune-suppressing drugs, leaving them at risk of getting a serious infection.
With gene therapy, a patient's own cells are used, which eliminates the need to find a donor and risk of rejection.
Each option carries risk, Shenoy said, and which is best depends on each individual's circumstances. Patients must be sick enough to warrant the need for the transplant, but not too sick to where they can't tolerate the chemotherapy.
"It can be toxic to the lung, it can be toxic to the kidney, to the heart, to the liver," Shenoy said. "So, we have to make sure that all those organs are reasonably functioning before we expose them to the (chemotherapy)."
Patients also face the risk of infertility, and freezing embryos or sperm may be too costly.
"We sit down and talk to them about both options, and the pluses and minuses," she said.
"But the exciting part is that there are many pathways that can get them to what we would consider a cure at this point," Shenoy said, "something I would not have been able to tell you 15 or 20 years ago."
Missing out
Martin nearly died when he was about 18 months old and fluid filled his lungs, said his mom. He spent a lot of time as a child in the hospital dealing with sudden bouts of pain in different parts of his body. He had to get frequent blood transfusions.
Martin said he loves to play sports and mostly hated things like not being able to play football with friends during recess or "just being too physical in general."
He had to have a port placed in his chest to receive the frequent transfusions. When the veins around it became weak, doctors would close it and put in another. He's had three.
"I did miss out on a lot of stuff," he said, without dwelling too much on the negative.
Pictures of Martin Mwita and his friends are seen in a scrap book made by Martin's family on Sunday, April 20, 2025, at their temporary apartment in St. Louis.
Zachary Linhares, Post-DispatchMartin Mwita and his mother, Jacinta Mwita, flip through a scrap book of memories on Sunday, April 20, 2025, in St. Louis.
Zachary Linhares, Post-DispatchIn high school, his blood transfusions turned into monthly blood exchanges — which doesn't just add healthy donor blood but also takes out an equal amount of his own blood. The exchanges helped his symptoms a lot, but their continued use can cause a dangerous buildup of iron or the body to start rejecting the donor blood.
Martin never took prescription narcotics for pain, which is unusual. Instead, he worked with a psychologist and learned to cope using breathing techniques, playing games, watching videos and listening to music. He loves the Beatles.
The skills helped him stay positive as he recovered from his five days of chemotherapy, which took his hair, peeled his skin, made him constantly spit mucus and throw up, and gave him nose bleeds and mouth sores.
His mom surprised him with a scrapbook his sister and friends made — full of pictures, encouraging notes and sweet messages. That helped a lot too.
They also relied on their faith and the words in Psalm 23, "Though I walk through the valley of the shadow of death, I shall fear no evil, for thou art with me."
"It reminded me that sometimes you have to walk through the valley … but he'll walk with you through it," Jacinta said. "And just reminding ourselves that we were not alone. And I think God showed up for us in so many ways."
Martin Mwita and his mother Jacinta participate in a virtual Easter service on Sunday, April 20, 2025, at their temporary apartment in St. Louis. "It does bring me peace of mind to know that I'm going to end up OK, and God's going to use that for his glory," Martin said.
Zachary Linhares, Post-DispatchThose ways included the hospital's physical, occupational, art and music therapists, which Martin relied heavily on.
"They'll have them come by your room, and they'll, you know, get you up and they'll do something with you," like walking, drawing and playing instruments, he said. "It's the things that kind of seem small that we might take for granted. It's just like everyday stuff. But when you're in that state, that's not stuff that's easy to do."
Martin even earned a medal for walking a 10K — more than 6 miles — measured off in the short hallways outside his hospital room.
He and his mom said they felt like gene therapy was their only choice. Martin didn't have a sibling match. His Kenyan ancestry made it even more difficult to wait for finding a donor match. They also feared the possibility that his body would attack the donor cells.
Luckily, Jacinta's employer insurance covered the treatment. He was able to freeze sperm in case his fertility was affected.
"At the end of the day, it's not, I wouldn't say it's an easy decision. I'm not the one going through it," Jacinta said. "As a parent, when you're making a decision on behalf of your child, it adds another level of complexity. What if it's the wrong one? What if something goes wrong? And especially when you're the first one."
She said, "It takes a lot of faith."
'It's freedom'
"Oh, we can't do that here," Jacinta recalled her doctor in Omaha telling her. He got Martin an appointment with Shenoy, whose department does about 10 to 15 stem cell transplants a year for sickle disease patients.
"So, we made our six-and-a-half-hour trip," Jacinta said, "and the rest is history."
That was nearly a year ago. In December, Martin started the gene therapy process, which involved infusing a drug that would free stem cells from his bone marrow and into his blood, where they could be drawn out.
The process took four of five hours a day, over the course of three days. (Sometimes, this effort to harvest hundreds of millions of stem cells may not work.)
His stem cells, in two containers the size of soda cans, were then shipped to Bluebird's lab in New Jersey, where technicians added healthy hemoglobin genes to correct the mutated ones and test their quality. The corrected cells arrived back a few months later.
Martin began his chemotherapy on March 4, and he received his new cells intravenously less than a week later. On April 11, he was released from the hospital. He and his mom are staying in a nearby apartment for the rest of his appointments.
Evidence that the new stem cells had settled into the bone marrow and were already producing lots of healthy red blood cells came Tuesday.
Martin still maintained his even keel. "I think it's pretty cool," he said.
Martin Mwita and his mother Jacinta Mwita, of Omaha, Neb., pose on Sunday, April 20, 2025, at their temporary apartment in St. Louis.
Zachary Linhares, Post-DispatchMartin said he's looking forward to returning to classes at the University of Nebraska-Omaha and his job at Club Car Wash, where he enjoyed selling memberships and making sure members were happy.
"Just, I guess, to be like, outside and around school without having to really take extra safety measures," he said. "Yeah, I think it'll be really exciting when that happens."
His immune system is still recovering, so he needs to avoid crowds and wear a mask for the next couple months. He'll have to get all his vaccinations again. He doesn't yet have the green light to attend a Cardinals game, but definitely when he returns for a follow-up appointment.
Jacinta said she is looking forward to being able to travel and see family members Martin has never seen. "He's going to be able to go wherever he wants to go," she said. "No restrictions."
Shenoy said she hopes the first gene therapy patients will continue to lead normal lives long-term. She also hopes the treatments will continue to improve and become less expensive.
"We'll probably be able to just walk through a transplant much easier than we ever could. We are heading there," Shenoy said. "To be able to offer them gene therapy without that high-dose chemotherapy — if that becomes possible, that would be such a big step forward."
Jacinta said she will likely seek gene therapy for her 14-year-old daughter, who also has sickle cell and has suffered from brain aneurysms. Martin's result was worth the tribulation.
"It validated all the work that we put in, that he put in. It paid off big-time," she said. "To imagine that going forward, he won't have any sickling. I mean, it's freedom."
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