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Globe and Mail
12 hours ago
- 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
06-06-2025
- 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?
Telegraph
03-06-2025
- 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.'
NHK
27-05-2025
- Health
- NHK
Things You Should Do To Prevent CKD - Medical Frontiers
A large-scale study in Japan has shown that improving one's diet and lifestyle can slow the progression of chronic kidney disease. Clinical trials in regenerative medicine are also underway. Dialysis is usually done three times a week, with each session lasting four hours Small changes to diet and lifestyle can make a big difference Diabetes medication is effective for CKD Administering stem cells to mice reduced kidney inflammation
The National
22-05-2025
- Health
- The National
Abu Dhabi researchers hopeful of Type 1 diabetes breakthrough within two years
Researchers at the Abu Dhabi Stem Cells Centre are hopeful of a breakthrough in their work on a cure for Type 1 diabetes 'in the next couple of years'. The most promising development is focused on regenerating the pancreas using stem cells and is part of a collaboration with Kyoto University and research company Rege Nephro. In Type 1 diabetes, the pancreas progressively reduces the amount of insulin (the hormone that regulates blood glucose levels) it produces until it stops producing any at all. If glucose in the blood is too high, it can seriously damage the body's organs. It is hoped pancreas regeneration can reverse this process. 'We're very optimistic that in the next couple of years, we'll have something solid to offer patients,' Prof Yendry Ventura, the centre's chief executive, said. 'This isn't just stem cell therapy – it's an approach to regenerate the pancreas itself using a specific type of engineered cell.' The process is still in the pre-clinical stage but hopes remain high. Research and development 'takes time', Prof Ventura added. 'You need to stabilise a strong cell line, trial it in the lab, test it on animals and only then can you move into human trials. Right now, we're preparing to begin animal testing, after extensive lab work with our Japanese partners.' The work is being showcased at Expo 2025 in Osaka, Japan. The fair provides an opportunity to highlight the centre's scientific achievements and the UAE's growing presence in cutting-edge health care, Prof Ventura said. 'We're using Osaka Expo as a platform to present the progress we've made with our partners Kyoto University and [biotech firm] Rege Nephro,' he added. 'It's also a chance to showcase what we've built in Abu Dhabi and how we're working alongside some of the world's most respected institutions.' Though ADSCC has collaborative relationships, it handles its own cell production and storage. 'We have our own infrastructure. Cells intended for therapies need special storage conditions,' Prof Ventura said. 'There's a difference between storing tissue for studies and storing it for treatment purposes.' He said the UAE's capabilities complement those of its international partners. 'We're focused on developing specific stem cells that can later become any kind of tissue. That's different from just storing samples, but the two approaches work hand in hand.' The project is being developed in close co-operation with Kyoto University's Centre for iPS Cell Research and Application. 'Their contribution has given us access to expertise and speed,' said Prof Ventura. 'At the same time, the UAE is emerging as a leader in stem cell research across the region.' ADSCC is not new to breakthroughs in this area. In January, the centre announced the successful treatment of a 20-year-old Emirati patient with Type 1 diabetes, using umbilical cord-derived mesenchymal stem cells. The procedure, developed and carried out entirely at ADSCC, resulted in significantly improved blood sugar levels and a drop in diabetes-related antibodies within one month of treatment. The treatment is a major step forward in using regenerative medicine to manage diabetes. Regenerative medicine focuses on the body's ability to heal itself at cellular and organ levels. It includes treatments that use stem cells, engineered tissues and even bio-manufactured organs to restore or replace damaged parts of the body. Mesenchymal stem cells, known for their ability to modulate immune responses and repair tissue, are gaining attention as a possible way to restore pancreatic function and reduce complications in diabetic patients. 'Being able to do this entirely in the UAE, using cells produced locally, is a milestone for us,' Prof Ventura said. The country's participation at the expo in Japan reflects a broader national commitment to innovation in health care, said Dr Fatima Al Kaabi, executive director of the Abu Dhabi Bone Marrow Transplant Programme. 'We're a young centre, only six years since inception, but we're already standing alongside global leaders,' she said. 'It's an honour to represent the UAE and show our standards meet the highest benchmarks.' She highlighted the power of pluripotent stem cells, which have the ability to turn into almost any type of tissue. 'Whether it's heart, liver, brain or pancreas, these cells hold incredible potential. The challenge now is to harness that potential and find real solutions to some of the world's most devastating diseases.' Dr Al Kaabi said regenerative medicine is not only the future of health care, it's already reshaping how doctors approach treatment. 'If we can unlock what these cells can do, we can transform the way we treat cancer, autoimmune conditions and traumatic injuries. With the vision and talent we have at ADSCC, I believe we're on the right path.'
