Health
‘Remarkable’ gene-editing treatment for sickle cell disease is approved by FDA
Modern medicine is one step closer to potentially curing sickle cell disease.
On the heels of the U.K.’s authorizing the world’s first gene-edited treatment for sickle cell disease on Nov. 16, the U.S. Food and Drug Administration (FDA) approved the same treatment on Dec. 8.
“CASGEVY’s approval by the FDA is momentous,” Reshma Kewalramani, M.D., chief executive officer and president of Vertex Pharmaceuticals in Boston, said in a statement provided to Fox News Digital.
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“It is the first CRISPR-based gene-editing therapy to be approved in the U.S.”
“These approvals represent an important medical advance with the use of innovative cell-based gene therapies to target potentially devastating diseases and improve public health,” said Peter Marks, M.D., Ph.D., director of the FDA’s Center for Biologics Evaluation and Research, in the agency’s press release.
“Today’s actions follow rigorous evaluations of the scientific and clinical data needed to support approval,” he also said “reflecting the FDA’s commitment to facilitating [the] development of safe and effective treatments for conditions with severe impacts on human health.”
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The CASEVY treatment — also known as exa-cel, or exagamglogene autotemcel — uses technology that “edits” the genes that cause sickle cell disease.
To develop the drug, Boston-based Vertex and CRISPR Therapeutics, which is based in Switzerland, used a gene-editing system known as CRISPR-Cas9 to turn genes on and off, according to the Sickle Cell Disease Association of America website.
“CASGEVY is a first-in-class treatment that offers the potential of a one-time transformative therapy for eligible patients with sickle cell disease,” said Kewalramani.
The inventors of the CRISPR technology won the Nobel Prize in 2020.
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CRISPR stands for “clustered regularly interspaced short palindromic repeats,” which are repetitive sequences of DNA.
“The CRISPR technology [that is] being used with this treatment for sickle cell disease — a devastating inherited illness that profoundly affects survival and quality of life — is remarkable,” Dr. Mikkael Sekeres, chief of the division of hematology of Sylvester Cancer Center at the University of Miami, told Fox News Digital.
He was not involved in the drug’s development.
“The data from the admittedly small study in support of the treatment’s approval by the FDA are impressive, and the therapy was thought safe by a panel of independent experts,” Sekeres added.
“CASGEVY is a first-in-class treatment that offers the potential of a one-time transformative therapy for eligible patients with sickle cell disease.”
The treatment is approved for people 12 and older with severe sickle cell disease who get recurrent pain syndromes known as vaso-occlusive crises, in which blood flow is blocked due to sickle cells.
The FDA also approved Lyfgenia, the first cell-based gene therapy for the treatment of sickle cell disease in people 12 years of age and older, per the agency’s press release.
What to know about sickle cell disease
Sickle cell disease is the most common inherited blood disorder in the United States, affecting an estimated 100,000 Americans, as the National Institutes of Health (NIH) noted on its website.
It disproportionately affects people of Black or African ancestry.
Red blood cells, which are produced in the bone marrow, contain a protein called hemoglobin that helps carry oxygen throughout the body.
The cells are normally a round shape, which allows them to easily travel through small blood vessels, according to the Centers for Disease Control and Prevention (CDC) website.
Patients with sickle cell disease inherit a gene mutation that produces abnormal hemoglobin, which makes it more difficult for the cells to move through the bloodstream, per the NIH.
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The diseased cells eventually get stuck in blood vessels, which blocks blood flow — sometimes leading to painful episodes (known as sickle cell crises), infection, acute chest syndrome or stroke, per the CDC.
The cells begin to take on the shape of the C-shaped farm tool known as a sickle, which led to the disease’s name.
How the therapy works
While in utero, fetuses make a type of hemoglobin called fetal hemoglobin, which does not cause cells to sickle and has been shown to make sickle cell disease milder in those who have it.
After a baby is born, the BCL11A gene instructs the red blood cells to stop producing fetal hemoglobin. The gene is usually turned off within six months of birth, a Vertex spokesperson told Fox News Digital.
CASEVY works by targeting — and “turning off” — the BCL11A gene.
This enables continued production of fetal hemoglobin and significantly reduces the damage from sickle cells, according to Vertex.
Although the treatment may not “cure” sickle cell disease, as patients will still have the sickle cell mutation, it could potentially eliminate most of the signs and symptoms, even if a small subset of the patient’s red blood cells continue to sickle, the Vertex spokesperson told Fox News Digital.
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To receive the treatment, patients will undergo a bone marrow transplant.
First, the patient will receive medication to help stem cells move from the bone marrow into the bloodstream. Next, the blood will be collected and the stem cells isolated.
The cells then will be shipped to a laboratory, where genetic technology will be used to edit their DNA.
The patient will then receive chemotherapy to destroy all potential stem cells, ideally, that could produce sickle cells in the future.
The edited cells then will be infused back into the patient under the monitoring of health care professionals, who will ensure that healthy cells are produced in the bone marrow.
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The FDA recommends that patients be monitored for health issues for 15 years after treatment.
Potential limitations and cautions
More research is needed to determine the treatment’s long-term effectiveness, experts say.
It’s unclear whether the treated stem cells will continue to produce red blood cells that don’t sickle for the rest of the patient’s life, or if the cells will die off after a certain number of years, the Sickle Cell Disease Association of America cautioned on its website.
As the patients also receive chemotherapy, potential adverse effects could include infertility, secondary cancer or infectious complications.
The cost of the therapy and eligibility restrictions may also limit who can receive it.
The treatment could cost millions of dollars per patient, according to recent reports.
“The million-dollar question is how people with the disease will be able to afford this life-altering treatment, and how the health care system can sustain such costs,” Sekeres told Fox News Digital.
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Health
Mom’s Gripes About Sister-in-Law Put Daughter in a Bind
My mother is hypercritical of my brother’s wife, to the point that she blames my sister-in-law for my brother’s “failings” (not getting a better job, not taking better care of his health, etc.). It has gotten worse now that there are grandchildren. My mother constantly criticizes how my sister-in-law is raising the kids, who are lovely and adore their grandparents.
Although my mother will occasionally raise criticisms with my sister-in-law and brother, I am mostly her audience.
I have a great relationship with my sister-in-law, and when my mother goes off on one of her rants, I defend her. I tell my mother how lucky she is to have such wonderful grandchildren, and point out that my brother is an adult who makes his own decisions. This just leads to an argument between my mother and me.
When I finally told my mother how much it hurts me to hear her say these things about my sister-in-law, she said that she needed to air her frustrations with someone. I want to be there for my mother, but I don’t like being put in this position. How do I navigate this?
From the Therapist: The short answer to your question is that you can navigate this by no longer engaging in these conversations. But I imagine you already know this. What you might be less aware of is that you aren’t being “put in this position” of supportive daughter, protective sister-in-law and unwilling confidante. You’ve chosen it, and it’s worth examining why you’ve signed up for a job you don’t want — and what makes it hard to resign.
Usually when we find ourselves repeatedly engaging in uncomfortable family patterns, it’s because they echo familiar roles from our childhood. It sounds as if you’re struggling with enmeshment, a relationship pattern in which boundaries between family members become blurred or are nonexistent.
Think of enmeshment as being like two trees that have grown so close together that their branches have become intertwined. While this might look like closeness, it actually prevents either tree from growing in a healthy way. In your case, your mother’s emotions and grievances have become so entangled with your own emotional life that it’s hard to distinguish where her feelings end and yours begin.
You mention wanting to “be there” for your mom even though these conversations hurt you. Many adult children who struggle to say no to their parents grew up serving as their parents’ emotional support system, or absorbing their parents’ feelings, even at the expense of their own. When you told your mother how much her venting hurt you, she responded not by acknowledging your feelings, but by asserting her need to “air her frustrations.” Her response reveals something important: She sees you as a vessel for her emotional overflow rather than as someone with valid feelings of your own. And yet, despite your hurt, you’re still more concerned about her feelings than yours.
You’re asking how to navigate this situation, but I think the deeper question is: How can you begin to value your own emotional needs?
You can start by reframing what it means to make a reasonable request, which is essentially what setting a boundary is. A boundary isn’t about pushing someone away. Instead, it’s about making a bid for connection. It’s saying: “I want to feel good being close to you, but when you do X, it makes me want to avoid you. Help me come closer.”
Establishing a boundary consists of three steps:
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State the issue and the desire to come closer (what will make this possible): “Mom, I love you and want to support you, but these conversations about my sister-in-law put me in an impossible position and make me want to avoid talking with you, which I know isn’t what either of us wants. I’m happy to talk about other things together, but in order to keep our relationship strong, I need this topic to be off limits.”
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Set the boundary (what you will do): “If you’re struggling with their choices, I’m happy to support you in finding a therapist who can help you work through these feelings. But if you bring up these frustrations with me, I’m going to end the conversation and we can talk another time about other things.”
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Hold the boundary (do what you say): A boundary isn’t about what the other person will or won’t do. A boundary is a contract with yourself. If you say you’ll end the conversation when your mom brings up your sister-in-law, you need to hold that boundary every single time. If you end the conversation only 90 percent of the time, then why would the other person honor your request when 10 percent of the time, you can’t honor it yourself? Honoring your request might sound like: “Mom, I’m going to end the conversation now because I’m not comfortable talking about my sister-in-law. I love you, and we’ll talk later.”
If you start to feel guilty, remember that just because someone sends you guilt doesn’t mean you have to accept delivery. Remind yourself that when you become your mother’s outlet for criticism of your sister-in-law, you’re participating in a cycle that strains loyalties and causes you personal distress. And keep in mind that being a good daughter means setting boundaries that encourage our parents to grow, rather than enabling patterns that harm our family relationships.
Want to Ask the Therapist? If you have a question, email askthetherapist@nytimes.com. By submitting a query, you agree to our reader submission terms. This column is not a substitute for professional medical advice.
Health
Cancer death rates decline yet new diagnoses spike for some groups, says report
A major annual cancer report has revealed a mix of good news and points of concern.
Cancer diagnoses are expected to exceed two million in 2025, with approximately 618,120 deaths predicted, according to the American Cancer Society’s annual cancer trends report, which was published today in CA: A Cancer Journal for Clinicians.
ACS researchers compiled data from central cancer registries and from the National Center for Health Statistics.
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While mortality rates have declined, certain groups are seeing a spike in diagnoses, the report noted.
“Continued reductions in cancer mortality because of drops in smoking, better treatment and earlier detection is certainly great news,” said lead author Rebecca Siegel, senior scientific director of surveillance research at the ACS in Georgia, in a press release.
“However, this progress is tempered by rising incidences in young and middle-aged women, who are often the family caregivers, and a shifting cancer burden from men to women, harkening back to the early 1900s, when cancer was more common in women.”
Overall decline in death rates
Cancer death rates dropped 34% between 1991 and 2022, according to the ACS report.
That equates to approximately 4.5 million deaths avoided due to early detection, reductions in smoking, and improvements in treatment, the report stated.
Cancer death rates dropped 34% between 1991 and 2022.
Several factors likely contributed to this decline, noted John D. Carpten, Ph.D., chief scientific officer at City of Hope, a national cancer research and treatment organization in California.
“I think a big one is smoking cessation and the battle against lung cancer, which has always been the most common form of cancer and is tied to tobacco use,” Carpten told Fox News Digital in an on-camera interview.
“But without a doubt, I think new and better methods for early detection, and screening for colorectal cancer and other forms of the disease, have also allowed us to see a decrease.”
Lifestyle improvements have also helped to decrease mortality, he said, along with the development of new and better therapies for cancer.
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Despite overall declines in mortality, the report revealed that death rates are rising for cancers of the oral cavity, pancreas, uterine corpus and liver (for females).
Some common cancers have also seen an increase in diagnoses, including breast (female), prostate, pancreatic, uterine corpus, melanoma (female), liver (female) and oral cancers associated with the human papillomavirus, the report stated.
Increased diagnoses among certain groups
Diagnoses for many cancer types are increasing among certain groups.
Cancer rates for women 50 to 64 years of age have surpassed those for men, the report revealed. For women under 50, rates are 82% higher than males in that age group.
As far as what is influencing the “disconcerting trend” in women’s cancers, Carpten said it is likely “highly nuanced” and will require additional research.
“The decrease in fertility and increases in obesity that we’ve seen are risk factors for breast cancer, especially in postmenopausal middle-aged women,” he said.
“But there could be other modifiable risk factors at play, like alcohol and physical activity.”
Cancer rates for women 50 to 64 years of age have surpassed those for men.
Another trend in the increase in early cancers is occurring in individuals under the age of 50, Carpten noted.
In particular, the report revealed that diagnoses of colorectal cancer in men and women under 65 and cervical cancer in women between 30 and 44 years of age has increased.
The report also discusses inequities in cancer rates among certain ethnic groups, with Native American and Black people experiencing higher diagnoses of some cancer types.
“Progress against cancer continues to be hampered by striking, wide static disparities for many racial and ethnic groups,” said senior author Dr. Ahmedin Jemal, senior vice president of surveillance and health equity science at the ACS, in the release.
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The report shows mixed trends for children, with diagnoses declining in recent years for patients 14 years of age and younger, but rising for adolescents between 15 and 19.
“Mortality rates have dropped by 70% in children and by 63% in adolescents since 1970, largely because of improved treatment for leukemia,” the ACS stated in the release.
Pancreatic cancer a growing concern
The ACS report also warns about “lagging progress” against pancreatic cancer, the third-leading cause of cancer death in the U.S.
Rates of diagnoses and deaths from the disease type are on the rise.
“Pancreatic is an incredibly deadly form of cancer,” Carpten said.
One of the main issues with pancreatic cancer, he said, is that it sometimes can grow in an individual for up to 10 years before it’s detected.
“If we can identify those cancers when they’re at at a curable stage, we can improve outcomes.”
One of the best opportunities for beating pancreatic cancer is early detection, Carpten said.
“By the time those cancers have advanced, they’ve spread to the liver or other organs, and they’re almost impossible to cure at that stage,” he said.
“If we can identify those cancers when they’re at a curable stage, we can improve outcomes.”
‘It takes a village’
Making progress in fighting cancer “takes a village,” Carpten told Fox News Digital.
“It will require partnerships between the community, the health care system, cancer researchers, government, industry — we all have to work together if we want to continue to see a decrease and an ultimate increase in cures,” he said.
Dr. Wayne A. I. Frederick, interim chief executive officer of the American Cancer Society and the American Cancer Society Cancer Action Network (ACS CAN), stated that the report highlights the need to “increase investment in both cancer treatment and care, including equitable screening programs.”
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“Screening programs are a critical component of early detection, and expanding access to these services will save countless lives,” he said in the release.
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“We also must address these shifts in cancer incidence, mainly among women. A concerted effort between health care providers, policymakers and communities needs to be prioritized to assess where and why mortality rates are rising.”
Fox News Digital reached out to the ACS for further comment.
Health
FDA Moves Forward With Last-Minute Push to Cut Nicotine Levels in Cigarettes
The Biden administration unveiled a proposal on Wednesday to cut the level of nicotine in cigarettes, a last-minute push on a plan that could meaningfully cut cancer rates nationwide and extend the lives of millions of cigarette smokers.
If finalized, the proposal would require cigarette makers to significantly reduce the levels of nicotine in their products in an effort to make smoking less addictive and less satisfying. Research has suggested that the move would result in fewer people taking up the habit and would help the nation’s roughly 30 million smokers quit or switch to less harmful alternatives like e-cigarettes.
The policy is a centerpiece of antismoking initiatives by Dr. Robert Califf, commissioner of the Food and Drug Administration, who has recounted treating cardiology patients ravaged by smoking during his medical career.
“It’s the biggest thing I’ve ever seen in terms of societal benefit, cost saving and lives saved, and strokes prevented and cancers prevented,” Dr. Califf said.
The policy’s companion effort to ban menthol cigarettes has been set aside indefinitely after vehement opposition from cigarette makers and other opponents, including convenience store retailers.
Whether the nicotine reduction plan would survive the incoming administration of President-elect Donald J. Trump is unclear. Mr. Trump has traditionally been industry friendly and opposed to heavily regulating businesses. In addition, he has had the support of tobacco companies, including Reynolds American, which contributed at least $8 million to Mr. Trump’s main super PAC during the presidential campaign. Reynolds has already expressed its opposition to the proposed requirement.
Mr. Trump’s campaign co-chair and incoming chief of staff, Susie Wiles, is a former lobbyist for Swisher, a company that makes cigars. The rule applies to cigarettes, roll-your-own tobacco, pipe tobacco and cigars (though not premium cigars).
Some public health advocates are holding out hope that the Trump administration will allow the proposal to move forward, given that a previous version was considered by the F.D.A. during his first term. At minimum, officials could continue to allow the public to comment on the initiative without killing it or putting it into effect.
The F.D.A.’s proposal includes projections that by 2100, the nicotine reduction measure would prevent an estimated 48 million young people from starting to smoke. By 2060, the agency also estimates that 1.8 million tobacco-related deaths would be prevented, and that $30 trillion in benefits would accrue over 40 years, mostly from the generation that would not begin smoking.
“We do have an extremely toxic and addictive product with cigarettes that remain on the marketplace, that still kills almost a half a million people a year,” said Dorothy Hatsukami, a tobacco researcher from the University of Minnesota who has studied low-nicotine cigarettes for about 15 years. “So it’s really kind of an unfortunate situation that we haven’t really done anything dramatically about it.”
In 2022, Dr. Califf released an updated proposal to lower nicotine levels, and opposition began to grow almost immediately.
Tobacco companies have viewed the initiative as a major threat to their business. Luis Pinto, a spokesman for Reynolds American, said the proposal would “effectively eliminate legal cigarettes and fuel an already massive illicit nicotine market.”
“These actions would also have a significant negative economic impact on farmers, retailers and others,” he added.
Convenience store retailers have also opposed earlier versions of the proposal, saying they would sustain substantial losses in revenue from a projected decline in cigarette sales.
Congressional Republicans have also tried to thwart restrictions on nicotine levels. In 2023, members of an influential House subcommittee passed a measure that would have prevented the F.D.A. from spending any money to advance limits on nicotine, with nearly all of the supporting votes by Republicans. The Senate did not include the provision in a final budget package.
Still, supporters of the plan point to signs that incoming public health officials may be receptive to it, including to the popularity of Robert F. Kennedy Jr.’s pledge to tackle chronic diseases and improve the health of Americans if he is confirmed to lead the nation’s top health agency. Mr. Trump himself has said that he is personally opposed to cigarette smoking.
“Given these enormous benefits, we urge the incoming Trump administration to move forward in finalizing and implementing this rule,” Yolonda C. Richardson, the president of Campaign for Tobacco-Free Kids, said in a statement. “Few actions would do more to fight chronic diseases such as cancer and cardiovascular disease that greatly undermine health in the United States, and that the incoming administration has indicated should be a priority to address.”
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