How much will I lose? Asking for more information on sickle cell transplants and gene therapy in the United States – an example from the United Kingdom
“What’s going to happen to me 10 to 20 years out? Will the products fail? They won’t stay in my system for a long time. Will there be any secondary leukemias? Hsu says. We don’t have any good information right now because nobody has been there for that long.
The risk of developing bone disease is higher in people with sickle-cell disease. The chemotherapy regimen can also raise the risk of cancer. DeBaun is optimistic that the new transplant protocols use lower doses of cancer drugs, so they will reduce the chance of these side effects.
Woolford, who lives in a suburb of Baltimore, Maryland, reached out to a few cancer charities that offer assistance to try and retain fertility for women undergoing chemotherapy. “I don’t have cancer, but I’m getting chemo and radiation,” she explained. “Maybe you can squeeze me in?” The answer was no.
Before Teonna got a stem-cell transplant she wanted six children, but the price was too high. Every other week she was in the hospital due to her sickle cell disease. She’d had both hips replaced because of bone damage caused by impaired blood flow and her liver was failing. Her doctors told her to consider a stem-cell transplant.
Stem cell transplants and gene therapy can take place without the use of chemotherapy. Chemotherapy is time-consuming and risky. Women who go through it are often left infertile. Eggs can be frozen, but in the US they can cost more than $10,000 to Harvest, freeze and store.
A stem-cell transplant can cost between $100,000 and $400,000, with a list price of up to $2 million, according to the University of Massachusetts. Such price tags mean that these therapies are out of reach for many people with sickle-cell disease around the world. In the United States, an effort is under way to make cell and gene therapies available to people who receive publicly funded health care. The programme will take effect in 2025, but is not known how many states will participate. The developers were asked for more data by the National Institute for Health Care and excellence in the United Kingdom, after they determined that Casgevy was not cost-effective for treating sickle-cell disease. The country’s public health-care system doesn’t yet have available the treatment for scurvy-cell disease.
At the same time, gene-targeting therapies have also progressed. One therapy that can be used to treat people with the disorder has a working version of one of the genes. The other therapy, called Casgevy (exagamglogene autotemcel), uses CRISPR–Cas9 genome editing to reactivate a form of haemoglobin that is normally inactivated soon after birth. Fetal haemoglobin helps to compensate for the dysfunctional version.
How do transplant recipients cope with the stigma surrounding genetic treatments of Sickle Cell Disease? The Case of Jones and Supacell: A Black Londoner’s Support Group
Over time, vessels can cause strokes and damage organs such as the heart and kidneys. In the United States, people with sickle cell disease can expect to live only 20 years shorter than people without it.
But the success stories that make headlines belie the uncertainties and struggles that still surround the treatments. The same can be said of cell and gene therapies, which can help with mental, financial and physical health. People who have been cured of leukaemia are often missing out on support and awareness because of the discrimination they face in access to healthcare. “There is a lack of education among health-care providers on how these patients should be managed,” says Akshay Sharma, a specialist in bone-marrow transplants at St. Jude Children’s Research Hospital in Memphis, Tennessee.
The FDA approved the first genetic treatments for sickle cell, a disease that disproportionately affects black people, in late 2023, and Adekanbi was thrilled.
Jones’s support group share stories and experiences, sifting out misinformation they encounter along the way In a recent virtual gathering, the group marvelled over Supacell, a drama series on streaming service Netflix in which a group of Black Londoners with sickle-cell disease in their families develop superpowers. They groaned in sympathy when one member recalled their struggles to get physicians to take their condition seriously.
Similar concerns weigh on people who have received gene therapies for other conditions, and that number is growing rapidly. Over 600 other gene therapies are undergoing clinical testing around the world, and 30 have been approved in the United States. Clinicians and patients are now wrestling with how to support people after their treatment. Anirban is an economist with the University of Washington in Seattle. This is coming.
But a lingering question is how to capture those data in the first place. Some recipients of cell and gene therapy stay in the care of their transplant team for a while after their treatment. But they eventually return to their usual health-care providers, who may not know the full history of their patients or reporting expectations for someone who has received a gene therapy.
And there are mental-health challenges to navigate, too. After noticing that many transplant recipients seemed to struggle after their procedures, Elisabeth Dovern, a haematologist at Amsterdam University Medical Center, and her colleagues conducted a survey of health challenges that arise after a transplant.
When she sought medical help for her pain, physicians sometimes turned her away because blood tests showed she had a different disease than they thought. “They say, ‘Well, why are you here?’” she says. “Basically, you have to suffer alone at home.”
For most of her life, Genesis Jones’s daily routine revolved around her illness, the painful blood disorder known as sickle-cell disease. Each time she left the house, she ran through a mental checklist: did she have her pain medications? What level of energy was she? Would she be able to make it through the day?
Jones found out a month after her transplant that she had cancer. Three more rounds of chemotherapy and other treatments drove her cancer into remission, but she still struggles with chronic pain in her back and legs caused by decades of tissue and nerve damage from sickle-cell disease. She is concerned that signs of mild cardiac inflammation mean her new stem cells are attacking her heart.
It’s difficult for the wider field of gene-therapy to follow long-term. Jack Grehan, a videographer in Manchester, UK, was treated for a blood-clotting disorder called haemophilia A more than five years ago. He went back to the hospital every time he was asked to come after receiving therapy for a few years. But now he lives two hours away from the treatment centre and is juggling childcare and a new job. He stopped going to check-ups for two years because he had not had any bleeding episodes since the treatment. He says he’s willing to give them more once his life returns to normal.
As the therapies expand from clinical trials to hospitals, there is an opportunity to collect data that would address these questions, LaBelle says. The FDA believes that data from the recipients of the therapy should be collected by the manufacturers for at least 15 years. Researchers outside those companies are also setting up a registry to track recipients of sickle-cell gene therapies, he adds.
One of the patients is DeShawn Chow, who lives in Irvine, Calif. He started treatment at the Los Angeles City of Hope Children’s Cancer Center. He doesn’t worry about the effect the treatment will have on his ability to have children.
“A lot of people are suffering — and not just suffering, but dying — every day,” says Gray, who’s now 39 and works full time at a Walmart. There is something we can do to stop it. I want people to be free of this type of fear, worry and the level of pain that’s indescribable.”
The challenge of treating sickle cell gene therapies in the United States and beyond: How do patients get the treatment they need, and how to get them?
In addition, the companies are working to help patients afford the treatments and related care, and more government and private insurers are paying for it.
“We do see a lot of traction pretty much on par with what we thought would be the interest level. Andrew Obenshain, the chief executive officer of bluebird bio, says they are encouraged by what they are seeing. There are hospitals ready to treat. The payors will pay for it. And the patients are interested.”
But getting all the costs covered can be tricky. And it remains far from clear how the majority of patients who suffer from these genetic blood disorders will ever get them, given that they live in economically disadvantaged countries in places like Africa and Asia where the new therapies remain unavailable.
“It’s almost like I’m battling myself,” says Adekanbi, 29, who lives in Boston. It feels like a dark place, but I don’t know if you could call it evil within.
Source: Sickle cell gene therapies roll out slowly
The Sickle Cell Gene Therapies Roll Out Slowly: A Reflection from Angienical Bethe Antolian Adekanbi
The rare genetic blood disease is caused by a genetic mutation that causes red blood cells to become deformed, sickle-shaped. These cells cause havoc by damaging vital organs and making attacks of pain.
Sometimes it gets to the point where you realize that you can no longer live the way you are. “You feel like you’re losing your mind. Because sometimes I just can’t move. I just lay in one spot and try to distract myself from the pain.”
She hopes to have children in the future. So I’m nervous about how that will affect fertility when you go through the gene therapy process.
Adekanbi is far from alone in wondering what to do. There is much excitement for the treatments among patients with certain diseases, but only a small number have begun the process.
Adekanbi says she would try to freeze some of her eggs if she decides to proceed. She and other potential patients are more worried about what’s happening in their lives. The treatments also are grueling and complicated in other ways.
Source: Sickle cell gene therapies roll out slowly
How do you get out of the hospital, or how to prepare for the next step? – Connie Chung’s “Coney”
Creary, who works for the University of Michigan School of Public Health, says it could take a while to get out of the hospital. You must be nearby the hospital to be in the state that you are in if you are not in the hospital. There is a very complex process of follow-up for many, many months, if you don’t live in that state, after therapy is finished.
Patients worry about long-term risks associated with their disease, according to Dr. Lewis Hsu, Chief Medical Officer of the Association of America.
Both the therapies that they make appear to be safe for now according to Vertex Pharmaceuticals of Boston.
And while it’s not surprising that it’s taking time to get the treatments widely accepted, given how complicated and expensive they are, both companies say interest is increasing fast.
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NPR Meets Survivor: How a Live Reality Game Changes Nina Fill: Growing a Close Community in the Presence of Trump’s Claims
NPR project coordinator Nina Fill discovered Survivor at 12 years old and her life was changed forever. Her obsession over the competition show is still there at 36. Though she pondered going on the show, 26 days of minimal sleep and food just wasn’t for her. She was accepted into the live game of Survivor Bloomington a year ago, and the gods were happy about it. She met her tribe in a dense wooded backyard as a production crew filmed her. Fill knew that live reality games, or LRGs, existed, but finding one so close to her home was a game-changer. Here‘s how they built up her confidence and brought her a new community.
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The threat level that election officials are concerned about is going to be decided in the general election. This presidential election is the first since Donald Trump tried to overturn the results. Over the last four years, officials have worked to improve the system of voting, educate the people of the community about the risks of voter fraud and convince them that the process is secure.
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