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Mammals could regenerate damaged tissue by turning on ‘genetic switch': Chinese team
Mammals could regenerate damaged tissue by turning on ‘genetic switch': Chinese team

South China Morning Post

timea day ago

  • Health
  • South China Morning Post

Mammals could regenerate damaged tissue by turning on ‘genetic switch': Chinese team

Chinese scientists say they have restored the damaged outer ear in mice, a mammal that lacks natural regenerative abilities. The researchers said the pathway they identified could be applied to different tissue types and play a significant role in advancing regeneration in vertebrates. The team found that a mouse's failure to regenerate stemmed from an inability to produce sufficient retinoic acid, a derivative of vitamin A that regulates what cell type emerges during a cell's development and tissue repair. 10:16 Rise in Hong Kong infant eczema cases linked to 'excessive' Covid-19 cleaning habits Rise in Hong Kong infant eczema cases linked to 'excessive' Covid-19 cleaning habits This was because mice had lost the DNA 'remote controls' that switch on the gene that encodes an enzyme to convert vitamin A into retinoic acid, they found. By turning on the 'evolutionarily disabled genetic switch', the scientists could reactivate the regeneration of the mouse ear. 'Our study identified a genetic switch involved in the evolution of regeneration,' said the researchers from the National Institute of Biological Sciences in Beijing, the BGI-Research genomics institute and the Shaanxi Key Laboratory of Molecular Biology for Agriculture. They published their research in the peer-reviewed journal Science on Thursday. Some animals possess the power of regeneration. Salamanders, for example, can regrow tissues, organs and limbs, while teleost fish can regenerate nervous tissue and restore function following injuries to their central nervous system. But mammals, including humans, have limited capacity to rejuvenate tissue and organs.

Woman off insulin for Type 1 diabetes after a single dose of experimental manufactured stem cells
Woman off insulin for Type 1 diabetes after a single dose of experimental manufactured stem cells

CTV News

time21-06-2025

  • Health
  • CTV News

Woman off insulin for Type 1 diabetes after a single dose of experimental manufactured stem cells

Amanda Smith celebrates the day, August 1, nearly two years ago, when she stopped taking insulin to manage her type one diabetes, just a few months after getting a dose of experimental stem cells as part of a study. 'I remember, like, being scared and excited, and it's history now,' she said. The 36-year-old nurse and mother is part of a small, but what some call 'milestone study,' of patients with Type 1 Diabetes using manufactured stem cells, designed to grow in the liver and become the full array of islet cells array of pancreatic islet cells that naturally control blood sugar levels. In a study published in the New England Journal of Medicine, researchers report that of the 12 patients who received a single dose of the stem cells, it eliminated the need for insulin in 10 for at least a year and stopped episodes of low blood sugar, or hypoglycemia, which can lead to dangerous complications, even death. For Amanda, the treatment has been a blessing. Diagnosed with late-onset juvenile diabetes when she was 25, she was plagued with sudden bouts of low blood sugar, or hypoglycemia that would leave her faint, despite close monitoring. The risk was a diabetic coma or worse. 'I get emotional because I'm free from those handcuffs ... I don't have that looming over me every day,' she said from her home in London, Ont. 'I took it as a death sentence,' she said. 'I knew, eventually, like the end is always some sort of complication with diabetes,' she said 'We've ... dealt with a lot of patients that have struggled with diabetes. And to be able to see such a transformational change in their life is just amazing,' said Dr. Trevor Reichman, the lead author and the Surgical Director of the Pancreas and Islet Cell Transplant Program at the University Health Network in Toronto. Dr. Trevor Reichman Dr. Trevor Reichman appears for an interview with CTV News. Reichman says it is the first time that scientists have seen this kind of response with implanted stem cell-derived islets. Vertex, a Boston-based biotech company that sponsored the study, derived the cells from embryonic tissue and then found a way to grow them stem cells in large quantities. Researchers, working at centers in the U.S. and at least three transplant hospitals in Canada, infused them into the patient's liver. Over the next four to six months, Reichman said they transformed into the array of hormone-producing cells found in a normal pancreas, and they were monitoring the patients' blood sugar levels in real time. 'In the liver, they're sensing a patient's blood glucose level, and they're secreting the appropriate hormone,' said Reichman. adding that these biological replacements appear to sense changes in 'seconds or milliseconds. Essentially, it's the same as your native islet cells would function.' 'I think the data is just so very exciting, so very, very powerful,' said Dr. Peter Senior, director of the Alberta Diabetes Institute at the University of Alberta. He was not part of this study. 'The primary objective of the study was just to show that the blood sugars were better and that people were not having severe hypoglycemia. They blew past that. Ten of the 12 people are off insulin,' said Senior. ' It's never been done before in history' said Dr. Michael Thompson, director of Vancouver General Diabetes Centre. 'It's first time they a have achieved a high enough levels of insulin in patients,'using a stem cell product 'It's a big advance' he added. But there's a tradeoff. The patients, however, require immune-suppressing drugs for life, so that the immune system doesn't destroy the cells. There are risks to these immunosuppressive medications, including a higher risk of some cancers, infections, and high blood pressure. Amanda says it is nothing like her constant terror that she might slip into a sudden diabetic coma. 'Taking a couple of pills three times a day is nothing. I take it with breakfast, lunch, and dinner. It's easy. No comparison, none,' she said. 'And I know it's a huge relief for my family, especially my husband, that's for sure.' The study also reports that two patients in the study died, one likely as a result of complications from that immunosuppression, which Dr. Reichman says underscores the need for patients to be closely monitored at experienced transplant centers. A second patient, according to the study, died of severe dementia. Off-the-shelf live cell therapy The idea of using cells implanted in the body to produce insulin, instead of insulin injections, began in Canada 25 years ago. Researchers in Alberta pioneered the Edmonton Protocol. It uses insulin-producing islet cells removed from diseased organ donors that are implanted into those with hard-to-manage Type 1 Diabetes. Some 2,500 patients have been treated around the world, according to the University of Alberta, which reports 80 per cent were able to stop taking insulin injections for a median time of 95 days. Stem cells pipettes (Image credit: Vertex) But the number of procedures is limited because there aren't enough organ donors to meet the demand, and patients also require long-term immune suppression. So, there have been some two dozen companies around the world looking at other ways of getting manufactured islet cells to regulate blood sugar as a replacement for insulin. The Vertex cells, originally called VX-880, have been renamed Zimislecel. The Boston-based pharmaceutical company (says it is ramping up production, as it 'These are fresh, brand-new cells – they're not 60-year-old cells that have already had a life, and we're repurposing them,' said Senior, who works in Edmonton. It pushes the boundary of therapy forward because there is the potential to create a renewable source of insulin-producing cells instead of waiting for cells from deceased donors,' he added. 'I think we've got a treatment for diabetes where we are no longer constrained by organ donors,' said Senior. 'We've got potentially a limitless source of cells that could be used, and that is a massively huge step forward in terms of a cell therapy becoming a reality.' The next step is for someone to produce stem cells that don't require immune suppression, by either genetically engineering the cells or encapsulating them to make them invisible to immune attack. Several pilot studies are underway. It's a welcome advance, according to Senior. While insulin therapy has been a lifesaver for many since its discovery in Toronto by Banting and Best in 1921, it's never been a cure. Stem Cells (Image credit: Vertex) 'I think people with diabetes deserve some of the transformative treatments we've seen in cancer and other diseases but we've been stuck essentially doing the same thing for 100 years,' said Senior. In Canada, there are some 300000 people with Type 1 diabetes. Thirty-two new cases are diagnosed each day. The number of new cases per year increased by 34 per cent between 2000 and 2022, according to Breakthrough TD1(formerly the Juvenile Diabetes Foundation of Canada). Unlike Type 2 diabetes, which is linked to environmental, dietary, and genetic factors, the exact cause of Type 1 diabetes is still unknown, but doctors say the immune system mistakenly attacks insulin-producing islet cells in the pancreas, resulting in a gradual decline in the availability of insulin to regulate blood sugar. Questions remain The study is being continued to include a total of 50 patients, in Vancouver, Edmonton, Toronto and the U.S., with Dr. Reichman's team actively recruiting candidates. There are still many questions. Dr. Reichman admits that no one knows how long these implanted cells will last. Amanda hasn't required any insulin inections for almost two years, the longest documented period so far. He adds there are no signs that the other nine, who also went off insulin, have gone back on the injections since their stem cell infusion. Unclear also is whether this therapy will reduce the longer-term and burdensome complications of diabetes, including heart problems, amputations, kidney failure, and vision loss. However, data from patients treated with the Edmonton protocol, using tissue from deceased donors, show signs of reduced complications, a promising sign for the newer therapy, according to Dr. Thompson. The other concern is that not all patients with severe hypoglycemia may want to make the same choice as Amanda, swapping the diabetes risks for those that come with the anti-rejection medications. 'Taking a couple of pills three times a day is nothing. It's easy. There's no comparison, none,' she said. Another question is that a single treatment therapy for this disease could come with a very high price tag. 'We'll have to wait and see,' said Reichman. Amanda Smith, meanwhile, tries to enjoy her freedom with her family and her work at a long-term care home, without thinking too far ahead. 'What happens if the cells stop working or something? You know, I just try and live right now, and I feel so blessed.' She debates the question - does she have diabetes anymore? 'I don't take any insulin anymore. I don't take medication for diabetes anymore. So, I feel like a regular person again without diabetes.'

Islet cell transplant trial outcome ‘major milestone' toward cure for diabetes
Islet cell transplant trial outcome ‘major milestone' toward cure for diabetes

Globe and Mail

time20-06-2025

  • Health
  • Globe and Mail

Islet cell transplant trial outcome ‘major milestone' toward cure for diabetes

When Amanda Smith learned at the age of 25 that she had late-onset Type 1 diabetes, she considered the diagnosis a death sentence. The nurse, from London, Ont., had a particularly dim view of the disease because she grew up watching her mother struggle with it. Her mother would slur her words and lose consciousness when her blood sugar bottomed out. Once, Ms. Smith's grandfather had to break a window to reach her mother, who was passed out in her home holding a banana she had tried to consume to raise her blood sugar. 'It literally eats you from the inside out,' said Ms. Smith, now 36. 'It rots your teeth, it destroys your body, your vision. The complications that come with Type 1 diabetes are so extreme.' Ms. Smith is confident now that those complications won't be in her future. In February of 2023, she became the first Canadian to receive a transplant of islet cells – the cells in the pancreas that make insulin – derived from embryonic stem cells. The transplant freed her from insulin and gave her blood sugar control akin to a non-diabetic. It was performed at Toronto General Hospital, the institution where insulin was first tested more than 100 years ago. On Friday, the results of the clinical trial in which Ms. Smith participated were published in the New England Journal of Medicine. The early-phase trial was small, and the procedure has a significant downside in that patients must take anti-rejection drugs for life, just like recipients of organ transplants. For people living with diabetes, the push for medical coverage isn't political, it's personal Nonetheless, the study's outcome counts as 'spectacular,' according to James Shapiro, who holds the Canada Research Chair in transplant surgery and regenerative medicine at the University of Alberta and was not involved in the study. Ten of the 12 participants who received a full dose of the cells were insulin-independent one year later. 'This is a major advance and a major milestone along the journey towards a cure,' he said. Dr. Shapiro was himself responsible for an earlier breakthrough on that path. In 2000, he and his colleagues at the University of Alberta published a seminal paper on a method they developed for safely and effectively transplanting islet cells from deceased donors into patients with Type 1 diabetes. They dubbed their regimen the 'Edmonton Protocol.' 'We used to call it the drive-through transplant,' Dr. Shapiro said, because giving patients islet cells was less risky and burdensome than a whole pancreas transplant. The cells are infused into patients under mild sedation with an IV-drip into the portal vein of the liver. The breakthrough described in the new paper is that the islet cells transplanted into Ms. Smith were derived not from deceased donors but from embryonic stem cells, which function as a type of source code that scientists can coax into becoming any cell in the human body. The supply of such lab-created islets could, in theory, be limitless, unlike the supply of deceased donor islets. Semaglutide, or Ozempic, reduces symptoms of feared diabetes' complication, study finds Trevor Reichman, director of the pancreas and islet transplant program at the University Health Network's Ajmera Transplant Centre, said the most encouraging result is that the islet-cell transplants eliminated dangerous blood sugar 'lows' that can cause confusion, seizures and unconsciousness. He ran the study site at UHN and is an author of the new paper. 'All of them, essentially, were able to get rid of their hypoglycemic episodes,' Dr. Reichman said, 'so all of the real risks to them, all of the lows, were essentially eliminated.' Still, the international trial, funded by Boston-based Vertex Pharmaceuticals, wasn't without its challenges. The company paused it temporarily last year after two participants died, though not from taking Zimislecel, the brand name of the new islet therapy. Vertex expects to apply for regulatory approval of Zimislecel starting next year. One participant died of severe dementia 30 months after the infusion. That patient had pre-existing neurocognitive impairment and, before joining the trial, had suffered a brain injury during a motor-vehicle accident caused by a severe blood sugar low. The other died of cryptococcal meningitis, a fungal infection caused by a sinus surgery. The death was related to the immunosuppressant medication that the participant was taking to preserve the islet transplant, the study said. That death underscores the tradeoff that people with Type 1 diabetes would have to make with Zimislecel, said Rémi Rabasa-Lhoret, director of the Metabolic Diseases Research Unit and The Diabetes Clinic at the Montreal Clinical Research Institute. He wonders how many people with Type 1 would be willing to take anti-rejection drugs for life, especially now that advances such as insulin pumps have made diabetes easier to manage for some patients. 'It's probably going to be a very narrow population,' Dr. Rabasa-Lhoret said. The next step for scientists is finding a way to genetically engineer or encapsulate islets so the immune system won't view them as hostile invaders. In the meantime, Ms. Smith said taking three pills, three times a day to suppress her immune system is a breeze compared with the round-the-clock work of keeping her blood sugar in check. Her 10-year-old daughter, Draya, used to say she wanted to become a doctor to cure diabetes. Now, she talks of being a pilot or engineer. 'I would do this a million times over compared to the maintenance I used to have to do to keep myself alive,' Ms. Smith said.

Medscape at 30: Three Decades of Amazing Breakthroughs
Medscape at 30: Three Decades of Amazing Breakthroughs

Medscape

time06-06-2025

  • Health
  • Medscape

Medscape at 30: Three Decades of Amazing Breakthroughs

Medscape turns 30 in 2025. As part of the Medscape 2050 project speculating on what the field will look like 25 years from now, we're looking back at medical history as told through Medscape news coverage. A walk down memory lane, for sure, but also a celebration of the breakthroughs, headlines, and reporting that helped define medicine since 1995. July 5, 1996: Dolly the Sheep Cloned The birth of the first cloned animal led to some interesting cloning coverage over the years, whether from claims of a cloned human baby, ethical concerns surrounding human cloning, and advances in animal cloning as more than just a curiosity: Single Genetic Difference May Make Humans Easier to Clone Knockout Pigs Heralded as Future Organ Source FDA: Meat of Cloned Animals Safe to Eat 1998: Discovery of Human Embryonic Stem Cells A groundbreaking and polarizing discovery. Just over a year later, Medscape would look at the fallout, and advances would attempt to eliminate ethical concerns by tricking mouse and human cells to exhibit all the traits of embryonic stem cells. Coverage would span many conditions over the years: Is New Stem Cell Research Major Step Toward Diabetes Cure? Stem Cell Therapy Among Hot Topics at AAO 2017 Promising New Data on Stem Cell Therapy for Parkinson's 1997: da Vinci Robotic Surgical System Begins Development Minimally invasive robotic surgery via the da Vinci system was approved by the FDA in 2000, and by 2011, it was in use in more than 2000 hospitals around the world. Between 2011 and 2012, however, adverse event reports rose 34%, prompting this detailed Medscape slideshow, Robotic Surgery: Too Much, Too Soon? 2000: First Draft of the Human Genome Announced It wasn't perfect, it had gaps and wasn't fully understood, but the turn of the century marked the first map of the human genome, paving the way for what we now see as precision and personalized medicine. In 2020, Medscape commissioned a thorough charting of the genome over its first 20 years to see what grew from that first announcement. Early 2000s: Rise of Direct-to-Consumer (DTC) Genetic Testing (eg, 23andMe) Inexpensive genetic testing led to the advent of offering tests to consumers, and the most famous purveyor, 23andMe, debuted in 2006. This marked a shift in how people access and interpret genetic data — and raised concerns. It's been a bumpy ride: Ethical Considerations in DTC Genetic Testing 23andMe Relaunches Lower-Risk DTC Genetic Tests FDA OK's First DTC Genetic Risk Tests 23andMe Notifies Customers of Data Breach Into Its 'DNA Relatives' Feature 23andMe's Business Woes Raise Genetic Data Privacy Concerns: Ethicist 2009: Electronic Health Record (EHR) Adoption Incentivized by HITECH Act EHRs, now ubiquitous, started slow in 2009, with one report estimating adoption in US hospitals at less than 2%. All that changed of course, with Medscape eventually releasing annual reports on physicians and EHRs. And in 2024, we even asked if physicians should be compensated for their time using EHRs. Don't Get Too Hung Up on 'Meaningful Use' in an EHR EHR Adoption Grows Rapidly in Office-Based Physician Groups 2012: CRISPR-Cas9 Gene Editing Discovered 2016: First CRISPR Clinical Trial in Humans Gene editing, and the ethical arguments that went with it, took the medical world by storm a little over a decade ago. Medscape has been covering its potential therapeutic (and controversial) applications ever since including the 2018 birth of 'gene-edited babies' in China. 5 Things to Know About CRISPR New CRISPR Tools More Precise, but Targeting Mishaps a Worry CRISPR Gene Editing in Limelight at ASHG 2016 Bioethicist: History Tells Us CRISPR Fears Are Overblown 2016: Semaglutide Shows Superior Glucose Control and Weight Loss in SUSTAIN-6 Trials The hype begins. Not only shown effective for glucose control, semaglutide also demonstrated marked cardiovascular risk reduction in 2016. While that was nearly 10 years ago, it feels like we haven't had a day without GLP-1 news since. Oral GLP-1 Agonists Could Be Game Changers for Obesity Treating Obesity: Will New Miracle Drugs End the Crisis? GLP-1s Treat and Even Reverse Some Forms of Liver Disease GLP-1 Agonists Reduce Recurrent Atrial Fibrillation July-October 2017: CAR T-Cell Therapy Approved for Leukemia The FDA approved tisagenlecleucel-T (Kymriah, Novartis) and soon after a xicabtagene ciloleucel (Yescarta, Kite Pharma), the first two CAR T-cell therapies in 2017, opening the door for personalized cancer treatment and further progress beyond treating blood cancers. CARs Motor Through Leukemia, Part 2: 'Landscape Changing' CAR T Cells and Beyond: Breakthroughs and Challenges Ahead Outpatient CAR T: Safe, Effective, Accessible Next-Gen CAR T-Cell Therapy: Expanding Beyond Blood Cancers December 2020: mRNA Vaccines Approved After effectiveness of the Pfizer and Moderna COVID vaccines was documented, the FDA granted emergency use of both vaccines within a week of each other. This opened the door for further mRNA usage, including potential cancer vaccines. 4 Things to Know About Moderna's mRNA Cancer Vaccine New mRNA Vaccines in Development for Cancer and Infections 'Encouraging' Early Data for mRNA Vaccine in Glioblastoma New mRNA Vaccine May Shield Against C difficile Infections 2023: FDA Approvals in Medical Artificial Intelligence (AI), Especially Diagnostics, Increase While AI was not invented in 2023, that year became a tipping point in everything AI — from diagnostics to drug development to medical scribes, inspiring the first Medscape Physicians and AI Report, done annually since. A sampling of the flood of Medscape coverage: Minding the Machine: Assessing the Case for AI Regulations in Healthcare Harnessing FDA-Approved AI Technologies in Your Medical Practice The Protein Problem: The Unsolved Mystery of AI Drug Dev This Bill Could Make It Legal for AI to Prescribe Medicine AI vs Physicians in 2050: Happy Future or No Future?

Endangered species could be saved by faeces, say scientists
Endangered species could be saved by faeces, say scientists

Telegraph

time03-06-2025

  • General
  • Telegraph

Endangered species could be saved by faeces, say scientists

Animal dung could be used to save endangered species, scientists believe. A new trial by the University of Oxford and Chester Zoo is aiming to harvest living cells from faeces in the hope they could be transformed into stem cells and eventually sperm and eggs. If successful, it could allow conservationists to collect genetic samples of rare and endangered animals from their waste without having to bother, or even see, the creature itself. The cells could be used to inseminate or clone animals and bring them back from the brink of extinction. Dr Rhiannon Bolton, carrying out research on dung from lions, elephants, giraffes and okapi at Chester Zoo, said: 'If this works, it could be a total game-changer. Right now, UK veterinary guidelines mean you can only collect genetic samples in very specific circumstances or following death, which limits the samples you can collect. 'It's early days yet, but this would allow us to collect cells from a far broader range of animals than we currently can, both individuals within a species and in terms of different species. 'It would make collecting and storing living animal cells non-invasive, cost-effective and efficient.' Oxford is currently trialling new techniques to isolate, wash and culture living cells to store them for conservation purposes. The faecal matter is processed to strip away anything left by the animal's diet or bacteria, allowing scientists to separate out living cells from the donor animal, which can then be extracted and cultured. These living cells have the potential to be used to create stem cells further down the line. Stem cells in turn could become sperm and egg cells, which would be used in conservation breeding or even cloning. The International Union for Conservation of Nature estimates that more than 41,000 species are currently under threat of extinction. Dr Bolton said: 'The keepers and scientists at the zoo pick up samples left in animal habitats. Because these are new techniques, we're trying to collect the freshest samples possible, so I'll get a delivery to the zoo lab, which is processed here. 'The clean cells are then posted to Oxford on the same day. We're looking at a range of animals as the cells from some species might be harder to culture than others. It's likely that the techniques will have to be tweaked for different species.' Currently, the samples must be collected quickly to ensure their freshness, so zoo animals are the perfect donors, but this technique could one day be used in the field, preserving cells from rare animals in the wild. The goal is to bank as much material as possible to prevent extinctions.' Chester Zoo already monitors the dung of its animals to track their health and breeding potential. By testing hormones in animal scat, scientists at the zoo can tell whether animals are ready to mate. Dr Sue Walker, the head of science at Chester Zoo, said: 'Faecal testing is integrated into the daily care of our animal species. We use it to monitor the reproductive and overall health of the animals. 'For instance, we track progesterone in the females which allows us to predict their cycles and lets us know when they are likely to be receptive to a breeding partner, and hormonal analysis also gives us a picture of how they respond to their environment. It helps us offer them optimal care. 'As a conservation zoo, many of the animals we look after are critically endangered. This means their genetics are extremely valuable to the survival of a species as a whole. As science advances, these methods could offer a lifeline to species on the brink.'

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