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The New England Journal of Medicine, November 21, 2024
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‘Worst imaginable’ childhood brain tumours treated by CAR-T cells
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| The childhood brain cancer known as diffuse intrinsic pontine glioma (DIPG) is usually a death sentence, but engineered immune cells might help to shrink tumours. (Credit: Living Art Enterprises/Science Photo Library) | |||||
CAR T shrinks 'worst imaginable' tumourEngineered chimeric antigen receptor (CAR) T cells can shrink a rare childhood brain tumour known as diffuse intrinsic pontine glioma (DIPG). Researchers designed CAR-T cells to target a molecule called GD2 that sits on the surface of DIPG tumour cells. The therapy shrunk four out of eleven children's tumours by at least 50%, and one person has remained in remission for 30 months. Nine of the eleven people ultimately died, but researchers say the therapy is a positive development in treating what has been called the "the worst imaginable childhood brain tumour" because of its nearly 100% fatality rate. STAT | 6 min read (paywall)Reference: Nature paper | |||||
Stem-cell transplants restore lost visionThree people with severely impaired vision can see significantly better thanks to a stem-cell transplant. These improvements have now lasted more than a year. A fourth person also experienced a boost in their vision, but it did not last. The four are the first to receive a transplant of reprogrammed stem cells to treat damaged corneas, the transparent outer surface of the eye. The team behind the treatment will launch larger clinical trials next year. Nature | 4 min readReference: The Lancet paper | |||||
South Africa floats heritable gene editingSouth Africa has amended its health-research ethics guidelines to include a new section on heritable (or germline) human genome editing. The move could put the nation one step closer to being the first to explicitly allow the controversial technique, which involves making genetic changes to sperm, eggs or embryos that could be passed down to future generations. Such editing could prevent inherited diseases, such as cystic fibrosis and sickle-cell disease, but it poses major ethical concerns and safety challenges. Nature | 5 min read | |||||
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| Researchers made these tomatoes (Solanum lycopersicum) extra sweet by using CRISPR-Cas9 to deactivate two genes that degrade sugar-production enzymes. The modification didn't affect the fruits' size — like other modern cultivated tomatoes, they are up to 100 times larger than their wild counterparts. (Nature | 4 min read) Reference: Nature paper (Credit: Zhang, J., Lyu, H., Chen, J. et al., Nature) | |||||
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How weight-loss drugs will change the world"Short of some crazy unfortunate side effect, this is going to change the world," says gastroenterologist Chin Hur about blockbuster GLP-1 agonist drugs such as semaglutide, which is sold as Ozempic for diabetes and Wegovy for weight loss. Reducing obesity globally could have huge societal advantages, from improving people's quality of life to reducing health and education disparities, but their future benefit is still shrouded in uncertainty. The long-term health impacts of these drugs are unclear, and they remain prohibitively expensive for most people, leaving medical researchers and companies scrambling to gather more data and develop better tools to assess how weight-loss drugs could transform societies. Nature | 11 min read | |||||
Can amyloid therapies treat many diseases?Amyloid, the sticky protein best known for its role in Alzheimer's disease, is involved in many other disorders including amyotrophic lateral sclerosis (ALS) and certain types of heart disease. Researchers have identified 39 'amyloidogenic' proteins that can misfold and accumulate in organs throughout the body. Several therapies — including antibodies, genetic editing systems and small-molecule drugs — are now in late-stage clinical trials for treating the 'systemic amyloidoses' that occur when a misfolded protein creates fibrils that damage an organ. One company is even developing a 'pan-amyloid' drug that targets any protein with a specific molecular structure. Nature Biotechnology | 10 min read | |||||
Cadavers could be bone-marrow donorsA new technique for collecting bone-marrow stem cells from a cadaver's spinal column could provide transplants for people with blood cancers. A company called Ossium Health is creating a bank of cryopreserved marrow from organ donors with diverse genetic backgrounds. Unlike solid organ donors, a bone-marrow donor must be a very close genetic match to the recipient. This presents a particular challenge when trying to find donors for people from racial minority groups, who are underrepresented in donor registries. The cadaver bank could widen the potential donor pool, although it is still unclear whether the freezing process could damage the stem cells. Wired | 8 min read | |||||
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Quote of the week"Cats, lemurs, mice are all useful. We shouldn't focus all our efforts on one."Comparative neuroscientist Christine Charvet argues that non-traditional model organisms offer unique insights into human conditions such as ageing. A study known as Translating Time is collecting health records, blood tests and brain scans from cats to map events along the nonlinear relationship between cat and human ages. (Nature | 5 min read) | |||||
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