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Telegraph
2 hours ago
- Telegraph
The pioneering therapy that could roll back the rigours of ageing
The clock could be turned back on ageing with a groundbreaking new therapy that aims to recharge cells with 'batteries' from the placenta of newborns. Israel-based Minovia Therapeutics is the first company in the world to start testing mitochondrial transplantation in humans, which could treat incurable diseases and keep bodies younger for longer. Mitochondria are tiny compartments within cells that act like batteries, supplying the cell with the energy it needs to function. In ageing and certain diseases, the mitochondria stop working efficiently, starving the cells of energy, similar to the batteries running down in a machine. But scientists have now shown that it is possible to 'augment' cells with supercharged mitochondria from the placenta. Minovia has already completed clinical trials of the new therapy for patients with Pearson's Syndrome - a mitochondrial disease - and is planning to trial the treatment in elderly people next year. Dr Natalie Yivgi-Ohana, CEO and co-founder of Minovia, said: 'It's actually a very natural process for cells to take up mitochondria when they come in contact with them, but it was usually only one in 1000 cells that would do it. 'In the past 13 years, we've developed a method to maximise cellular take up without harming the cells or the mitochondria, so now more than 50 per cent of cells take up a significant amount of mitochondria. 'We take it from the youngest and healthiest organ, which is the placenta and which is full of super mitochondria and yet it's normally thrown away like garbage. We could find it to be the fountain of youth.' To make the therapy, scientists take the mitochondria from a healthy donated placenta and mix them with blood stem cells, which are then infused back into the patient's bloodstream. Not only do the super mitochondria help to produce more energy in cells, but they also reactivate natural quality control functions inside cells, which keep failing mitochondria at bay. So over time, the level of the body's healthy mitochondria also goes up. Last month, the company released phase 2 results for Pearson Syndrome, which can cause a range of problems in children, including failure to thrive, diabetes and neurological issues. There are currently no approved therapies for the condition, and care is only palliative, with patients dying during childhood. But the new therapy has led to marked improvements. 'Severe energy failure' 'These were all paediatric patients and they suffer severe energy failure, so they are not growing,' added Dr Yivgi-Ohana. 'It took a few months, but we have started observing improvement in their energy and less fatigue, more waking hours. We observed improvement in renal function, improvement in appetite, in growth.' Now the company is turning its attention towards ageing, and is hoping that growing older may one day be seen as a treatable disease. The company is planning to start trials of the treatment for elderly people in Israel next year and is developing biomarkers so they can test whether older people are experiencing mitochondrial dysfunction. It could even help diseases of ageing, such as Parkinson's, which is strongly linked to mitochondrial dysfunction. 'Significant in almost every disease' 'We are all going to suffer mitochondrial disease as we age,' added Dr Yivgi-Ohana. 'Ageing is not considered a disease, but if we have a way to demonstrate that actually, as we age, we become mitochondrially dysfunctional, then that would be the trigger to propose a treatment. 'We want to start next year treating elderly people with mitochondrial dysfunction in longevity clinics and demonstrate that it really helps. ' She added: 'The scalability in terms of the mitochondria is unlimited, there are no limitations of placentas and the amount of mitochondria that we can harvest and cryopreserve.' 'Mitochondria are such powerful organelles, and they can transfer between cells, so their sensing and their information transfer is so significant in almost every disease that you will look at.'
Medical News Today
10 hours ago
- Medical News Today
Experimental drug eliminates aggressive breast, skin cancer in small trial
Scientists have developed a new cancer drug that can be injected directly into tumors. The treatment, an enhanced CD40 agonist, was successful in its first trial of 12 human participants. Six people in the group saw their tumors shrink, while two participants saw their cancer go completely into remission. The National Institutes of Health project that doctors will diagnose at least 2 million new cancer cases and that 600,000 people will die from cancer in 2025 in the United improving cancer death rates, this prediction highlights the need to continue developing more effective treatments. Researchers from Rockefeller University made headway in this area with the completion of their human phase 1 study. They tested a new CD40 antibody drug, 2141-V11, designed to shrink tumors and reduce side new drug had promising results, shrinking tumors in half the patients and completely eliminating cancer in two cases, while not causing serious side effects. The study appears in the journal Cancer Cell.A new approach to cancer immunotherapyMany cancer treatments exist, and sometimes treatment can be as simple as removing the tumor or lesion for localized cancers. In cases where the cancer is metastatic and has spread, more aggressive treatments are necessary. Some types of more aggressive cancer treatments include: radiation therapychemotherapyhormone American Cancer Society describes immunotherapy as a 'treatment that uses the body's immune system, usually your own, to attack cancer.' There are several types of immunotherapy, such as immune checkpoint inhibitors, CAR T-cell therapy, and monoclonal antibodies. Researchers in the new study focused on immunotherapy — specifically, the CD40 agonist antibody drug class, a type of monoclonal antibody treatment. It works by activating the CD40 receptor and causing an immune system response. According to the study authors, research on CD40 has not been successful in human trials in the past and has caused serious side effects. This led the scientists to create an engineered form of CD40 called 2141-V11. The drug was designed to improve the issues with CD40 and reduce systemic inflammation and liver toxicity. The scientists also decided to inject the treatment directly into the tumor instead of administering it intravenously. For the human study, the researchers recruited 12 participants, and everyone in the group had metastatic cancers including melanoma and breast cancer. The participants ranged from ages 42 to participants received injections of 2141-V11 into their tumors every 3 weeks, with an increased dosage each results with limited toxicityThroughout the 2141-V11 treatments, the researchers monitored the side effects and bloodwork of the participants. They were especially concerned with the participants' platelet count and liver enzymes since these would indicate a treatment-related adverse event (TRAE). By the end of the treatment, ten participants experienced adverse events, and seven experienced TRAEs. However, none of the patients experienced any TRAEs higher than grade 3, and the researchers considered the drug 'well tolerated.' Most of the side effects were mild and included fever and issues where the drug was injected. Three participants had severe adverse events, but the researchers did not consider them TRAEs. One participant had to be hospitalized because of a urinary tract infection, and another had shortness of breath related to chronic heart failure. No serious dose-limiting toxicities occurred, and the participants' liver and platelet levels were stable throughout treatment. Additionally, cancer tumors shrank in six of the participants, and two participants went into complete remission. One of the participants who went into remission had melanoma, and the other had breast cancer. The researchers noted that not only did their tumors shrink, but tumors in the rest of the body disappeared. Wael Harb, MD, a board-certified hematologist and medical oncologist at MemorialCare Cancer Institute at Orange Coast and Saddleback Medical Centers, spoke with Medical News Today and explained how the cancer drug works.'This drug (2141-V11) is an engineered antibody that flips a key 'ON' switch (CD40) on immune cells inside a tumor,' said Harb, who was not involved in the recent trial. 'It's injected directly into one tumor, where it organizes immune cells into mini lymph nodes called tertiary lymphoid structures (TLS).' 'Those act like on-site training camps that activate cancer-killing T-cells, which then travel through the body to attack tumors that weren't injected,' Harb he noted that the drug's safety profile was encouraging, he emphasized that larger trials are needed. 'Twelve patients are too few to draw firm safety conclusions across cancers–larger trials are needed and are already underway,' the oncologist is next for 2141-V11?Study author Juan Osorio, MD, director Of Clinical Operations And Translational Immunology at Rockefeller University, spoke with MNT and discussed the future of the research with 2141-V11. 'We look forward to the results of several ongoing phase 2 studies nationwide, targeting difficult-to-treat cancers, such as malignant gliomas, bladder cancer, and prostate cancer,' said Osorio. Osorio mentioned that early data from the phase 2 studies is 'promising,' but it is not a one-size-fits-all solution for people with cancer. 'Not all patients respond, highlighting the need for rational combination strategies to extend the benefit of this therapy,' Osorio shared. Osorio said the team is currently trying to figure out which biomarkers may predict response to CD40 agonists. 'Furthermore, we want to evaluate in larger cohorts whether TLS or other immune spatial interactions are crucial for effective induction of antitumor immunity. Answering these questions will be critical to fully translate this approach into meaningful and durable clinical benefit for patients.'
The Guardian
20 hours ago
- The Guardian
Exposure to some common Pfas changes gene activity, new study finds
New research suggests exposure to some common Pfas or 'forever chemical' compounds causes changes to gene activity, and those changes are linked to health problems including multiple cancers, neurological disorders and autoimmune disease. The findings are a major step toward determining the mechanism by which the chemicals cause disease and could help doctors identify, detect and treat health problems for those exposed to Pfas before the issues advance. The research may also point toward other diseases potentially caused by Pfas that have not yet been identified, the authors said. The study is among the first to examine how Pfas chemicals impact gene activity, called epigenetics. 'This gives us a hint as to which genes and which Pfas might be important,' said Melissa Furlong, a University of Arizona College of Public Health Pfas researcher and study lead author. Pfas are a class of about 15,000 compounds most frequently used to make products water-, stain- and grease-resistant. They have been linked to cancer, birth defects, decreased immunity, high cholesterol, kidney disease and a range of other serious health problems. They are dubbed 'forever chemicals' because they do not naturally break down in the environment The study checked the blood of about 300 firefighters from four departments across the country who were exposed to high levels of Pfas. The chemicals are the main ingredient in most firefighting foam, and are frequently used in 'turnout gear' worn by firefighters because of their heat repelling properties Furlong said she was surprised to find the number of genes and biological pathways that were impacted by Pfas, which suggests the chemicals may cause or contribute to a broad range of health problems. The study did not prove the chemicals cause certain diseases, but findings point to biological changes that might precede disease. Genes play a range of roles in developing or preventing disease, and Pfas essentially change the way the genes are supposed to act, Furlong said. A gene may act as tumor suppressor, but the Pfas interferes with how it expresses, which affects whether cancer develops, or the type of cancer. For example, PFOS, among the most common and dangerous Pfas compounds, reduces levels of miR-128-1-5p, a gene tied to cancer development. Branched forms of PFOS were linked to changes in five other genes, including some that regulate cancer development. Sign up to Detox Your Kitchen A seven-week expert course to help you avoid chemicals in your food and groceries. after newsletter promotion Different Pfas and chemical structures were found to affect different genes, and were associated with different health outcomes. Not all compounds impacted gene expressions. The research found connections among Pfas-related gene changes and biological pathways involved in leukemia, as well as bladder, liver, thyroid and breast cancers. Other genes and biological pathways were involved in Alzheimer's disease, and autoimmune and infectious diseases such as lupus, asthma and tuberculosis. Furlong said it remains unclear at which step in the biological processes disease is triggered, but the picture is clear enough to point to possible treatments. Pharmaceutical companies are trying to develop drugs that could alter gene activity and potentially prevent Pfas-linked disease from developing.
