A Landmark Global Trial Led from Lebanon: Mitapivat in NTDT Published in The Lancet
The study, titled 'Phase 3 trial of mitapivat in non-transfusion-dependent alpha- or beta-thalassemia,' led by Dr. Ali Taher, professor of medicine at the Division of Hematology and Oncology, director of the Naef K. Basile Cancer Institute, and consultant at the Chronic Care Center served as both global principal investigator and first and corresponding author. It is the first to demonstrate the efficacy of an oral, disease-modifying therapy for both beta and alpha forms of NTDT, positioning mitapivat as a potential game changer in the treatment of this lifelong blood disorder.
'This publication is more than a scientific milestone,' said Dr. Taher. 'It's proof that Lebanon, even in its hardest times, can lead groundbreaking medical advances that change global standards of care. I'm proud this trial was conceived, led, and delivered from AUB and the Chronic Care Center.'
NTDT is a lifelong inherited blood disorder that results in chronic anemia. Patients with NTDT do not require blood transfusions, but still suffer from chronic anemia and associated complications, including iron overload and reduced quality of life. Until now, therapeutic options for these patients have been limited.
Mitapivat is currently under review by the United States Food and Drug Administration (FDA) for potential marketing authorization. If approved, it would become the first FDA-authorized oral disease-modifying treatment for NTDT, offering a transformative option for thousands of patients worldwide.
The phase 3 trial met all of its primary and secondary endpoints, demonstrating statistically and clinically significant improvements in hemoglobin levels, alongside favorable safety and tolerability profiles.
This work represents the culmination of over a decade of research, beginning with redefining the disease and understanding the morbidity and mortality risks of untreated anemia and iron overload. It builds on prior global efforts led from AUB that resulted in the approval of key therapies, including iron chelation through the THALASSA trial and luspatercept through the BEYOND trial. Both studies were led by Dr. Ali Taher, whose vision and leadership have been instrumental in transforming the global approach to thalassemia care. Widely recognized as one of the foremost experts in the field, Dr. Taher has played a central role in advancing treatment standards and improving outcomes for patients worldwide. His long-standing commitment to thalassemia research has not only shaped the current therapeutic landscape, but has also positioned Lebanon and AUB at the forefront of international medical innovation.
This achievement serves as a powerful affirmation of AUB's role as a beacon of academic and clinical excellence, firmly committed to advancing health through research, education, and patient-centered innovation. It also reflects the resilience and determination of Lebanon's medical and scientific communities to contribute meaningfully to global health, despite the formidable challenges faced at home.
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a day ago
- Web Release
A Landmark Global Trial Led from Lebanon: Mitapivat in NTDT Published in The Lancet
In a major advancement for global thalassemia care, the results of a landmark global phase 3 clinical trial of mitapivat in non-transfusion-dependent thalassemia (NTDT) led by the American University of Beirut (AUB) and the Chronic Care Center, the thalassemia center in Lebanon, have been published in The Lancet, one of the world's leading medical journals. The study, titled 'Phase 3 trial of mitapivat in non-transfusion-dependent alpha- or beta-thalassemia,' led by Dr. Ali Taher, professor of medicine at the Division of Hematology and Oncology, director of the Naef K. Basile Cancer Institute, and consultant at the Chronic Care Center served as both global principal investigator and first and corresponding author. It is the first to demonstrate the efficacy of an oral, disease-modifying therapy for both beta and alpha forms of NTDT, positioning mitapivat as a potential game changer in the treatment of this lifelong blood disorder. 'This publication is more than a scientific milestone,' said Dr. Taher. 'It's proof that Lebanon, even in its hardest times, can lead groundbreaking medical advances that change global standards of care. I'm proud this trial was conceived, led, and delivered from AUB and the Chronic Care Center.' NTDT is a lifelong inherited blood disorder that results in chronic anemia. Patients with NTDT do not require blood transfusions, but still suffer from chronic anemia and associated complications, including iron overload and reduced quality of life. Until now, therapeutic options for these patients have been limited. Mitapivat is currently under review by the United States Food and Drug Administration (FDA) for potential marketing authorization. If approved, it would become the first FDA-authorized oral disease-modifying treatment for NTDT, offering a transformative option for thousands of patients worldwide. The phase 3 trial met all of its primary and secondary endpoints, demonstrating statistically and clinically significant improvements in hemoglobin levels, alongside favorable safety and tolerability profiles. This work represents the culmination of over a decade of research, beginning with redefining the disease and understanding the morbidity and mortality risks of untreated anemia and iron overload. It builds on prior global efforts led from AUB that resulted in the approval of key therapies, including iron chelation through the THALASSA trial and luspatercept through the BEYOND trial. Both studies were led by Dr. Ali Taher, whose vision and leadership have been instrumental in transforming the global approach to thalassemia care. Widely recognized as one of the foremost experts in the field, Dr. Taher has played a central role in advancing treatment standards and improving outcomes for patients worldwide. His long-standing commitment to thalassemia research has not only shaped the current therapeutic landscape, but has also positioned Lebanon and AUB at the forefront of international medical innovation. This achievement serves as a powerful affirmation of AUB's role as a beacon of academic and clinical excellence, firmly committed to advancing health through research, education, and patient-centered innovation. It also reflects the resilience and determination of Lebanon's medical and scientific communities to contribute meaningfully to global health, despite the formidable challenges faced at home.
Khaleej Times
22-06-2025
- Khaleej Times
Are stem cell therapies are safe to try to fight diseases?
The promise of stem cell therapy is powerful. Scientists can draw versatile cells from the human body and deliver them to repair injuries and fight disease from the inside out. But not all stem cell therapies are created equal. Some, like bone marrow transplants, are supported by decades of clinical research and are considered safe and effective. Others are still in clinical trials, meaning they're promising concepts but have not yet been put into practice. At the same time, medical spas and longevity clinics are increasingly marketing their own stem cell treatments for arthritis, joint pain, Parkinson's disease, A.L.S. and even better skin. Scientists warn that the treatments being touted by these spas and clinics are all unproven, expensive and could come with dangerous side effects. 'This clinic industry poses all kinds of risks to the public,' said Paul Knoepfler, a professor at the University of California Davis School of Medicine who studies stem cells. Some stem cell experts, including Dr. Knoepfler, are worried that the F.D.A. may loosen oversight on stem cell therapy, which could confuse the public about the difference between scientifically supported treatment and untested therapies. Their concerns come in part from Health Secretary Robert F. Kennedy Jr.'s remarks on the subject, including a post on X in October calling for an end to the F.D.A.'s 'aggressive suppression' of stem cell therapies. During a recent podcast with longevity influencer Gary Brecka, Mr. Kennedy said that he had traveled to Antigua to get a stem cell treatment for his throat. Mr. Kennedy, who has a neurological condition that affects his throat and voice, said the therapy helped him 'enormously.' 'Why did I have to go to Antigua for that?' he said, suggesting that he would work to expand access to stem cell treatments and other experimental therapies. How are stem cells regulated right now? Companies developing stem cell therapies can apply to the Food and Drug Administration for authorization to begin studies in humans. If the therapy appears to work, the company can then seek approval from the F.D.A., which reviews the data on the treatment. In addition, F.D.A. has the power to regulate the products offered at private clinics, which have generally not been approved. The agency has previously demanded some clinics stop selling unproven stem cell treatments that could harm patients or cause them to delay more legitimate treatment. Less regulation, though, may mean less oversight of companies 'charging a lot of money for something that's not really been validated,' said Clive Svendsen, a professor of biomedical sciences who leads a stem cell research lab at Cedars-Sinai in Los Angeles. Why are scientists excited about them? Stem cells are the blank cells that all tissues and organs in your body are created from. Unlike many other cells, certain stem cells can also regenerate. Because of that, scientists think they could offer an endless supply of cells that can be manipulated to replace other types of cells in your body that have been damaged. Some treatments rely on adult stem cells harvested from blood or bone marrow. Others use regular skin or blood cells which are reprogrammed into stem cells in a lab. For approved therapies, these stem cells are typically manipulated in a lab to become a certain type of cell, and then delivered into specific tissues in the hopes that they replace lost or injured cells, said Tenneille Ludwig, director of the WiCell Stem Cell Bank in Wisconsin, which distributes cells used in research and clinical trials. What treatments are available and what's being studied? Only a handful of stem cell therapies are currently approved by the F.D.A., mainly for treating blood cancer and immune system disorders. (Some therapies, for conditions like corneal damage or arterial disease, are approved in other countries but not in the United States.) In bone marrow transplants, one of the F.D.A.-approved treatments, a donor's stem cells are injected into a patient, where they become blood or immune cells that can replace the ones damaged by chemotherapy or radiation treatment. One ongoing clinical trial is investigating whether stem cells can replace the dopamine-producing neurons that die out during Parkinson's disease, which could potentially reverse the symptoms. Another is looking at whether insulin-producing cells derived from stem cells can be used as a diabetes treatment. And there's other research that some scientists see as promising. Studies have suggested that stem cells that have been reprogrammed into blank cells and then into heart cells may be able to restore some cardiac function. Larger trials on neurological and cardiac treatments — including for A.L.S. and heart failure — are ongoing, but have so far fallen 'short of expectations,' said Jeffrey Karp, chair of the anesthesiology department at Mass General Brigham and Harvard Medical School. What about the experimental treatments at private clinics? Thousands of clinics around the country now offer what they claim are stem cell treatments for a wide range of diseases. These treatments are not covered by insurance, and can cost between a few thousand to tens of thousands of dollars per injection, Dr. Svendsen said. Typically, these clinics draw stem cells from the patients' body or a donor, sometimes from their fat, and inject them back into the patient without any manipulation, experts said. (This can allow companies to skirt the F.D.A., which does not regulate some stem cell treatments if providers say they are only minimally manipulating a person's own cells.) If these treatments do happen to offer some relief, 'it's very likely that it's a placebo,' Dr. Svendsen said, adding that there's no sound scientific explanation for why stem cells that haven't been manipulated into more specific cell types would offer any health benefits. 'They're just asking you for $7,000 to have your cells infused back into your body.' There are other risks to consider. First, there's no guarantee that clinics use true stem cells, Dr. Karp said: While they may be collecting cells from a patient or donor's bone marrow or fat, there's no assurance that the cells they collected are the ones that regenerate. Clinics also aren't required to report where the donor stem cells came from, which means they could be contaminated, Dr. Karp said. In addition, improper injections — and contaminated cells — could lead to serious health problems like clots or cells traveling to the wrong organs in the body, the experts said. The F.D.A. has previously warned of reports of blindness, tumor formations and infections after unapproved stem cell treatments. In 2017, three patients in Florida had permanent vision damage after stem cells were injected into their eyes in an attempt to treat macular degeneration. 'Of course you're going to get a lot of charlatans, and you're going to get people who have bad results,' Mr. Kennedy acknowledged on the recent podcast appearance. 'And ultimately, you can't prevent that either way.' Dr. Karp said that 'the most dangerous scenarios tend to occur at for-profit clinics operating without oversight.' 'These clinics often make exaggerated claims and skip crucial safety protocols,' he said. While people with chronic pain or other illness may be desperate for relief, these unproven treatments pose a 'serious possibility of injury,' Dr. Ludwig said, adding that patients should ask what regulatory protocols clinics follow, and the extent to which scientific literature supports their treatments. 'I would be very wary.'
The National
20-06-2025
- The National
More than a third of children spend over seven hours a day on screens, UAE study finds
More than one third of children in the UAE spend over seven hours each weekday on screens, according to new research. The findings, published as schools in the country take tougher action to restrict mobile phone use, also highlighted a link between the excessive use of electronic devices and a lack of exercise. Carried out by University of Sharjah and American University of Beirut, the research reported that 37.7 per cent of students spent more than seven hours on screens each weekday, outside of lessons. 'High screen time was associated with lower physical activity levels, as 68.8 per cent of children who exceeded seven hours of screen time did not participate in any physical activity,' the study said, referring to time outside of lessons during the school week. Based on data about mobile phone, laptop, television and tablet use submitted by parents of 300 children in the UAE aged from four to 17, the paper also found that screen time increased with age. What can parents do? The findings highlighted, the researchers said, 'the need for families to take proactive measures to limit screen time and encourage more physical activity in their children'. 'Parents should be encouraged to set clear limits on screen time and promote alternative activities, such as sports, outdoor play and family outings,' the researchers said. With more than six in 10 children eating sweets while using screens, the researchers said that 'addressing dietary habits is crucial'. The new paper is titled, 'Assessing the effect of screen time on physical activity in children based on parent-reported data: a cross-sectional study,' and is published in the Cureus Journal of Medical Science. Aine McGlue, head of secondary at the British International School Abu Dhabi, said that previous research showed that today's children were less active than young people from previous generations. 'So much of it is caused by inappropriate use of digital media,' she said. 'To expect a child to have the social and emotional maturity to detach themselves from a phone is unreasonable … Schools have to take a proactive stance.' In August, at the start of the 2024/25 academic year, BISAD, a Nord Anglia school, introduced a ban on mobile phone use by pupils during school hours. It is one of many UAE schools to have brought in such restrictions. Ms McGlue said that the policy was 'quite contentious at first', but parents were invited to coffee mornings during which the rule was explained, with the school highlighting research around the link between reduced screen time and improved mental and physical health. The rationale was also explained to pupils. 'Kids need to know that we're not implementing this as a punishment: there's research and evidence behind this to say this is in their best interests,' Ms McGlue said. 'The policy is clear: if you're seen with your phone, the phone is confiscated. If your phone is taken a second time, it's the parents' responsibility to come in and get the phone themselves.' Alternatives to screen time By, for example, installing table tennis tables, the school has given students additional things to do outside of lessons, instead of using phones, she said. 'At lunchtime, when kids might be feeling a little unsure about how they're going to use this time, they're actually out now playing football, they're out playing basketball,' Ms McGlue said. 'We've set up open mic sessions so that kids can creatively perform using their music skills. They have an audience around the things that they're doing.' Research from many other nations has highlighted the potential harms linked to excessive use of screen devices. In a study based on data from New Zealand, Dr Ladan Hashemi, of City St George's, part of the University of London, found that when families had stricter rules about using screens when children were of pre-school age, obesity rates were lower later on in childhood. Dr Hashemi told The National that excessive screen time, poor diet, lack of physical activity and poor sleep were all linked. Using screens too often can, for example, encourage unhealthy snacking, while late-night screen use can disrupt sleep. 'Families who had better regulation around the use of screen devices, their children were more likely to have better sleep, longer sleep, and they were less likely to spend time on screen devices, which means they have more time for physical activity. These are strong contributors to reducing childhood obesity,' Dr Hashemi said. She said that for school-age children, less than two hours per day on screens was widely recommended – but also that parents should set an example. 'You can't just tell children to abandon devices if you as a parent are spending too much time on screens. You need, as a parent, to set an example as a role model,' she said. Dr Hashemi said that parents may 'use screen devices as a babysitter' to occupy their children, so cutting down on screen time meant that parents should provide alternative things to do. 'If you restrict the children's screen time, you need to give them more means to spend their time – family time, activity time, school-based activities, community-based activities,' she said. The American Academy of Paediatrics recommends that for children aged two to five, non-educational screen time should be limited to about one hour per weekday and three hours per day at weekends. For children aged six and older, the academy recommends that parents 'encourage healthy habits and limit activities that include screens'. Screens should be turned off during family meals and outings, the academy suggests, and turned off and removed from bedrooms at least half an hour before lights out. In March, the UAE's first digital detox clinic for children has opened in Abu Dhabi offering a programme to address the growing problem of screen addiction in young people.
