Tag: type 1 diabetes

Categories : Metabolic Disorders PaediatricsTags :

UK Study Proves Effectiveness of Childhood Type 1 Diabetes Screening

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Photo by Pavel Danilyuk

Thousands of families have taken part in a landmark UK study led by researchers at the University of Birmingham which shows that childhood screening for type 1 diabetes is effective, laying the groundwork for a UK-wide childhood screening programme.

Results from the first phase of the ELSA (Early Surveillance for Autoimmune diabetes) study, co-funded by charities Diabetes UK and Breakthrough T1D, have been published in a research letter in The Lancet Diabetes & Endocrinology today.

The findings mark a major step towards a future in which type 1 diabetes can be detected in children before symptoms appear. Currently, over a quarter of children aren’t diagnosed with type 1 diabetes until they are in diabetic ketoacidosis (DKA), a potentially fatal condition that requires urgent hospital treatment. Early detection can dramatically reduce emergency diagnoses and could give children access to new immunotherapy treatments that can delay the need for insulin for years.

We are working towards a future where type 1 diabetes can be detected in a timely manner

Professor Parth Narendran, lead researcher

Launched in 2022, ELSA is the first UK study of its kind, tested blood samples from 17,931 children aged 3-13 for autoantibodies, markers of type 1 diabetes that can appear years before symptoms.

Children without autoantibodies are unlikely to develop type 1 diabetes, while those with one autoantibody have a 15% chance of developing the condition within 10 years. Having two or more autoantibodies indicates the immune system has already started attacking the insulin-producing cells in the pancreas and it is almost certain these children will eventually need insulin therapy. This is known as early-stage type 1 diabetes.

Among the 17,283 children aged 3-13 years who were screened for type 1 diabetes risk at the time of analysis:

  • 75 had one autoantibody, signaling increased future risk.
  • 160 had two or more autoantibodies but did not yet require insulin therapy, indicating early-stage type 1 diabetes.
  • 7 were found to have undiagnosed type 1 diabetes with all needing to start insulin immediately.

Lead researcher, Parth Narendran, Professor of Diabetes Medicine at the University of Birmingham, said: “We are extremely grateful to all the families who have participated in the study and generously given their time to help understand how a UK-wide screening programme could be developed. Together with Diabetes UK, Breakthrough T1D and the National Institute for Health and Care Research, we are working towards a future where type 1 diabetes can be detected in a timely manner, and families appropriately supported and treated with medicines to delay the need for insulin.

“We are also grateful to partners across the Birmingham Health and Life Sciences District and beyond as well as the NIHR for the support they have provided in getting us to where we are.”

Interventions before diagnosis

Families of children found to have early-stage type 1 diabetes received tailored education and ongoing support to prepare for the eventual onset of type 1 diabetes symptoms and to ensure insulin therapy can begin promptly when needed, reducing the chances of needing emergency treatment. Those with one autoantibody also received ongoing support and monitoring.

Some families were also offered teplizumab, the first ever immunotherapy for type 1 diabetes, which can delay the need for insulin by around three years in people with early-stage type 1 diabetes. The first patient was treated at Birmingham Children’s Hospital. Teplizumab was licensed by the Medicines and Healthcare products Regulatory Agency (MHRA) in the UK in August 2025, and is currently being assessed by the National Institute for Health and Care Excellence (NICE) to determine whether it should be available through the NHS.

As of November 2025, more than 37,000 families have signed up to the ELSA programme. Building on this strong foundation, the second phase of the research, ELSA 2, launches today. ELSA 2 will expand screening to all children in the UK aged 2-17 years, with a focus on younger children (2-3 years) and older teenagers (14-17 years). The research team aims to recruit 30,000 additional children across these new age groups.

ELSA 2 will also establish new NHS Early-Stage Type 1 Diabetes Clinics, providing families taking part with clinical and psychological support and creating a clear pathway from screening to diagnosis, monitoring and treatment.

Case study: Knowing what’s coming … has made an enormous difference

Amy Norman, 44, from the West Midlands, was diagnosed with type 1 diabetes at the age of 13. She recently discovered via the ELSA study that her 11-year-old daughter, Imogen, is in the early stages of type 1 diabetes but has been able to slow its progression as the second child in the UK to access a breakthrough immunotherapy drug – teplizumab. She said: “Being part of the ELSA study has helped us as a family to prepare for the future in a way we never expected. Knowing what’s coming – rather than being taken by surprise – has made an enormous difference to our confidence and peace of mind.

“When I was diagnosed, I had no warning and ended up quite poorly in hospital with diabetic ketoacidosis (DKA). When Imogen’s diagnosis arrives, we hope that having this awareness will reduce her chances of experiencing DKA and the added trauma that comes from a sudden illness.

“Imogen took part in the study to further research and help others, but it has helped her too – being forewarned is being forearmed. She was always going to develop type 1 diabetes, but through ELSA we’ve been able to slow down the process and prepare – we know what is coming, but we’re not scared.”

A game-changer: showing what we can achieve in Birmingham

Professor Neil Hanley, Pro-Vice-Chancellor and Head of the College of Medicine and Health at the University of Birmingham, said, “This is a game-changer. This trial shows we can spare countless children the trauma of an emergency diagnosis, ensure they get early support, and potentially give them access to revolutionary new treatments that could delay or even prevent type 1 diabetes.

“Dr Parth Narendran and his team deserve huge credit; and this breakthrough shows what we can achieve in Birmingham. We have world-class clinicians and scientists working side-by-side, backed by great innovation infrastructure and a vibrant, diverse and affordable city – and, as a result, we are changing lives with next generation diagnostics, therapeutics, and clinical care.”

Rewriting the story of type 1 diabetes

Dr Elizabeth Robertson, Director of Research and Clinical at Diabetes UK, said: “For too many families, a child’s type 1 diabetes diagnosis still comes as a frightening emergency. But that doesn’t have to be the case. Thanks to scientific breakthroughs, we now have the tools to identify children in the very earliest stages of type 1 diabetes – giving families precious time to prepare, avoid emergency hospital admissions, and access treatments that can delay the need for insulin for years.

“The ELSA study, co-funded by Diabetes UK, is generating the evidence needed to make type 1 diabetes screening a reality for every family in the UK. We’re incredibly grateful to the 37,000 families who’ve already signed up and urge others to get involved. Together, we can transform type 1 diabetes care for future generations.”

Rachel Connor, Director of Research Partnerships at Breakthrough T1D, said: “This is about rewriting the story of type 1 diabetes for thousands of families. Instead of a devastating emergency, we can offer time, choices, and hope. By finding children in the earliest stages, we’re not just preparing families, we’re opening the door to treatments that can delay the need for insulin by years. That extra time means childhoods with fewer injections, fewer hospital visits and more normality. Thanks to research like ELSA, what once struck as an unexpected crisis can become an actively managed healthcare process, changing the course of T1D for the better.”

The findings from ELSA’s first phase signal a major step towards a future in which type 1 diabetes can be detected early, managed proactively, and potentially delayed through immunotherapy. ELSA demonstrates that childhood screening in the UK is feasible, acceptable to families, and capable of preventing emergency diagnoses. Continued research through ELSA 2 will assess how screening can be scaled across the NHS and evaluate its cost-effectiveness.

Source: University of Birmingham

Categories : Metabolic DisordersTags :

A Gentle ‘Immune System Reset’ Cured Type 1 Diabetes in Mice

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An “immune system reset” cured autoimmune, or Type 1, diabetes in mice in a Stanford Medicine study. The approach may be useful for other autoimmune conditions as well as organ transplants.

A 3D map of the islet density routes throughout the healthy human pancreas. Source: Wikimedia CC0

A combination of blood stem cell and pancreatic islet cell transplant from an immunologically mismatched donor completely prevented or cured Type 1 diabetes in mice in a study by Stanford Medicine researchers. Type 1 diabetes arises when the immune system mistakenly destroys insulin-producing islet cells in the pancreas.

None of the animals developed graft-versus-host disease – in which the immune system arising from the donated blood stem cells attacks healthy tissue in the recipient – and the destruction of islet cells by the native host immune system was halted. After the transplants, the animals did not require the use of immunosuppressive drugs or insulin for the duration of the six-month experiment.

“The possibility of translating these findings into humans is very exciting,” said Seung K. Kim, MD, PhD, professor of developmental biology, gerontology, endocrinology and metabolism. “The key steps in our study – which result in animals with a hybrid immune system containing cells from both the donor and the recipient – are already being used in the clinic for other conditions. We believe this approach will be transformative for people with Type 1 diabetes or other autoimmune diseases, as well as for those who need solid organ transplants.”

Kim, who directs the Stanford Diabetes Research Center and the Northern California Breakthrough T1D Center of Excellence, is the senior author of the study, which published online Nov. 18 in the Journal of Clinical Investigation. Graduate and medical student Preksha Bhagchandani is the lead author of the research.

Setting the table

The findings in the current report dovetail with those from a 2022 study by Kim and collaborators, in which researchers first induced diabetes in mice by destroying insulin-producing cells in the pancreas with toxins. They then cured them with a gentle pre-transplant treatment of immune-targeting antibodies and low-dose radiation, followed by transplantation of blood stem and islet cells from an unrelated donor.

The current study tackled a more complex problem: curing or preventing diabetes caused by autoimmunity, in which the immune system spontaneously destroys its own islet cells. In people this is called Type 1 diabetes. Unlike in the induced-diabetes study — in which the researchers’ goal was to prevent the recipient’s immune system from rejecting donated islet cells — the transplanted islet cells in the autoimmune mice have two targets on their backs: Not only are they foreign, but they are vulnerable to autoimmune attack by a misguided immune system bent on destroying islet cells regardless of their origin.

“Just like in human Type 1 diabetes, the diabetes that occurs in these mice results from an immune system that spontaneously attacks the insulin-producing beta cells in pancreatic islets,” Kim said. “We need to not only replace the islets that have been lost but also reset the recipient’s immune system to prevent ongoing islet cell destruction. Creating a hybrid immune system accomplishes both goals.”

Unfortunately, the inherent features that lead to autoimmune diabetes in these mice also make them more challenging to prepare for a successful blood stem cell transplant.

The solution the researchers found was relatively simple: Bhagchandani and Stephan Ramos, PhD, a postdoctoral fellow and study co-author, added a drug used to treat autoimmune diseases to the pre-transplant regimen the researchers had discovered in 2022. Doing so, then transplanting blood stem cells, resulted in an immune system made up of cells from both the donor and the recipient and prevented development of Type 1 diabetes in 19 out of 19 animals. Additionally, nine out of nine mice that had developed long-standing Type 1 diabetes were cured of their disease by the combined blood stem cell and islet transplantation.

Because the antibodies, drugs and low-dose radiation the researchers administered to the mice are already used in the clinic for blood stem cell transplantation, the researchers believe that translating the approach to people with Type 1 diabetes is a logical next step.

Where the concept began

The study builds on the work of the late Samuel Strober, MD, PhD, a professor of immunology and rheumatology, and his colleagues, including study co-author and professor of medicine Judith Shizuru, MD, PhD. They and other Stanford researchers had shown that a bone marrow transplant from a partially immunologically matched human donor allowed formation of a hybrid immune system in the recipient, and subsequent long-term acceptance of a kidney transplant from the same donor. In some cases, Strober and colleagues showed that transplanted donor kidney function lasted for decades, without the need for drugs to suppress rejection.

A blood stem cell transplant can be used to treat cancers of the blood and immune system, such as leukemia and lymphoma. But in those settings, high doses of chemotherapy drugs and radiation needed to treat the cancer and replace the recipient blood and immune system often result in severe side effects. Shizuru and colleagues have devised a safer, gentler avenue to prepare recipients with non-cancerous conditions such as Type 1 diabetes for donor blood stem cell transplantation — knocking their bone marrow back just enough to provide a foothold for the donated blood stem cells to settle in and develop.

“Based on many years of basic research by us and others, we know that blood stem cell transplants could also be beneficial for a wide range of autoimmune diseases,” Shizuru said. “The challenge has been to devise a more benign pre-treatment process, diminishing risk to the point that patients suffering from an autoimmune deficiency that may not be immediately life-threatening would feel comfortable undergoing the treatment.” 

Judith Shizuru

Judith Shizuru

“Now we know that the donated blood stem cells re-educate the recipient animal’s immune system to not only accept the donated islets, but also not attack its healthy tissues, including islets,” Kim said. “In turn, the donated blood stem cells and the immune system they produce learn to not attack the recipient’s tissues, and graft-versus-host disease can be avoided.”

What comes next?

Challenges remain using this approach to treat Type 1 diabetes. Pancreatic islets can be obtained only after death of the donor, and the blood stem cells must come from the same person as the islets. It is also unclear whether the number of islet cells typically isolated from one donor would be enough to reverse established Type 1 diabetes.

But the researchers are working on solutions, which could include generating large numbers of islet cells in the laboratory from pluripotent human stem cells, or finding ways to increase the function and survival of transplanted donor islet cells.

In addition to diabetes, Kim, Shizuru and their colleagues expect that the gentler pre-conditioning approach they developed could make stem cell transplants a viable treatment for autoimmune disease such as rheumatoid arthritis and lupus, and non-cancerous blood conditions like sickle cell anemia (for which current blood stem cell transplant methods remain harsh), or for transplants of mismatched solid organs.

“The ability to reset the immune system safely to permit durable organ replacement could rapidly lead to great medical advances,” Kim said.

Source: Stanford Medicine

Categories : GastrointestinalTags :

New Research May Help Doctors Identify Coeliac Disease Earlier in Kids with T1D

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Photo by cottonbro studio

Children diagnosed with type 1 diabetes (T1D) face more than just managing their blood sugar. They are also at a higher lifelong risk of developing coeliac disease. Because of this, regular screening is recommended to catch coeliac disease early in kids with T1D.

A new article published in Clinical Chemistry takes a closer look at how blood tests for coeliac disease can be used more effectively in this group of patients. In celiac disease, elevated TTG-IgA antibodies are often the first step in the diagnostic process. If blood levels of TTG-IgA are elevated, patients are typically sent for a biopsy to confirm coeliac disease. In kids with T1D, this can be a bit more complicated. In children with T1D, TTG-IgA antibody levels can fluctuate, and there hasn’t been a clear cutoff for when a biopsy is truly necessary.

Researchers studied nearly 600 children with T1D and found that using a cutoff of six times the normal limit gave the best balance of accuracy, which performed better than the older cutoff of 11 times the normal limit. The study also showed that kids who developed celiac disease soon after being diagnosed with T1D often had higher antibody levels right away, while those who developed celiac disease later had lower levels in the beginning. This highlights the importance of testing at the time of T1D diagnosis and continuing to monitor regularly, even if early test results aren’t extremely high.

“Our article looked at recent work investigating serologic assays’ efficacies in pediatric patients with type 1 diabetes. We found that recent data such as those seen in Muller et al.’s study (2025) suggest that lower transglutaminase elevations can portend coeliac disease in this population than previously thought, and the disease remains considerably prevalent among patients with T1d. As such it is crucial to actively evaluate for CeD in T1d patients, and the serologies provide an excellent tool for doing so,” said study author Dr Andrew M. Ford.

For families, the key takeaway is that regular screening matters, and doctors are working to fine-tune how these tests are used so children with both T1D and coeliac disease can be diagnosed as early and accurately as possible.

Source: Celiac Disease Foundation

Categories : Metabolic DisordersTags :

Roche Launches First AI-enabled Continuous Glucose Monitor in South Africa

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This new technology offers people with diabetes two-hour and overnight predictive notifications

  • The Accu-Chek SmartGuide® continuous glucose monitoring (CGM) solution provides 14 days accuratereal-time glucose values for adults living with diabetes1      
  • Artificial intelligence (AI)-enabled algorithms can predict glucose levels up to two hours ahead and overnight, empowering users to take action before a glucose excursion occurs2 proactively
  • Over 4.2 million people living with diabetes were recorded in 2024, in South Africa people living with diabetes could benefit from the Accu-Chek SmartGuide® CGM solution 3

Johannesburg, 4 November 2025 – Roche (SIX: RO, ROG; OTCQX: RHHBY) announced today that its Accu-Chek® SmartGuide continuous glucose monitoring (CGM) solution is now available in South Africa. This significant milestone means that people living with diabetes over the age of 18 can now benefit from the solution’s enhanced glucose monitoring and artificial intelligence (AI)-enabled predictive insights1,2.     

Diabetes is one of today’s most urgent healthcare challenges. In South Africa, 4.2 million people are living with the condition as of 2024 3. If left unmanaged, diabetes can lead to secondary complications including cardiovascular disease, kidney disease, nerve damage, blindness, lower limb amputation and mental health issues4. In a study conducted by GWI in partnership with Roche, 58% of people living with diabetes stated that they feel a mental burden associated with managing their diabetes.

Since it was introduced, CGM technology has significantly improved diabetes care5, yet a significant number of people still have difficulty keeping their glucose in the right range when using current systems.6 Hypoglycaemia is common amongst persons who have type 1 diabetes, with an annual incidence of severe hypoglycemia ranging from 3.3% to 13.5%.7 

“The Accu-Chek SmartGuide CGM solution is a significant step towards providing greater peace of mind for people living with diabetes, due to the AI-enabled predictive capabilities it offers. This empowers patients to take action before undesired events occur,” said Merilynn Steenkamp, General Manager, Southern Africa, Roche Diagnostics. “Our new Accu-Chek SmartGuide® CGM solution puts the power of prediction into the hands of people living with diabetes with its integrated AI-enabled algorithms that predict glucose levels for the next 2 hours as well as imminent and nocturnal hypoglycaemia. People with diabetes should have the freedom to plan the day ahead and get a good night’s sleep at the end of it all.”

Nighttime hypoglycaemia can present a particular challenge for people with diabetes. Approximately 2-5% of deaths in people with type 1 diabetes under the age of 40 are due to episodes of very low glucose during the night8,9. The unpleasant symptoms and negative consequences of hypoglycaemia can result in actual fear of hypoglycaemia with possible significant implications for the diabetes management, metabolic control and subsequent health outcomes.10

The Accu-Chek SmartGuide® CGM solution uses AI-enabled algorithms to predict where an individual’s glucose levels are likely headed in the next 30 minutes, 2 hours, and even overnight. This means people with diabetes can move from a reactive to a proactive approach to their daily management, preparing for and managing potential episodes in advance, rather than waiting for alerts when they happen.

The Accu-Chek SmartGuide® CGM solution includes a wireless, water-resistant sensor, worn on the back of the upper arm, and the apps where the users can see their current glucose levels and their future glucose development through predictive analytics. Every five minutes, the sensor sends glucose values measured in real-time to the Accu-Chek SmartGuide app. The Accu-Chek SmartGuide® predict app then uses those glucose values and other optional information entered by the user such as food intake and insulin doses to detect patterns and predict future glucose levels. Supported by AI-trained algorithms, it provides key predictions of likely hypoglycaemia within 30 minutes as well as general estimated predictions of glucose levels within 2 hours and during a defined 7-hours nighttime period.11

Clinical studies have demonstrated the new Roche CGM solution’s high system accuracy, with an overall mean absolute relative difference (MARD) of 9.2% and 99.8% of measured glucose values falling within zones A and B on the Parkes Error Grid.1, 12 The evaluation of the predictive capabilities showed that all advanced predictive features exceeded high performance requirements as e.g. accuracy, sensitivity and specificity.13

Roche is committed to bringing improved and proactive relief to diabetes management across South Africa with the immediate availability of the Accu-Chek SmartGuide® CGM solution.

About the Accu-Chek SmartGuide® CGM solution

Accu-Chek SmartGuide® is a continuous glucose monitoring (CGM) solution developed by Roche providing accurate1 real-time glucose readings and AI-enabled predictions for different timeframes7. The solution includes three elements: the Accu-Chek SmartGuide® CGM sensor, the Accu-Chek SmartGuide® App and the Accu-Chek SmartGuide® Predict App. With an all-in-one applicator and 14-day wear time, Accu-Chek SmartGuide® is designed for people living with diabetes, 18 years of age and older. It aims at empowering people living with diabetes to be prepared for the future development of glucose levels and take preventive action by making the appropriate therapy adjustments in good time.

The Accu-Chek SmartGuide® CGM solution can be seamlessly integrated with the Accu-Chek® Care platform, offering healthcare professionals (HCPs) access to comprehensive and accurate1 therapy-relevant data provided by the CGM solution. This integration allows HCPs to analyse together with their patients how lifestyle and therapy impact their glucose levels and make more informed decisions.

About Roche

Founded in 1896 in Basel, Switzerland, as one of the first industrial manufacturers of branded medicines, Roche has grown into the world’s largest biotechnology company and the global leader in in-vitro diagnostics. The company pursues scientific excellence to discover and develop medicines and diagnostics for improving and saving the lives of people around the world. We are a pioneer in personalised healthcare and want to further transform how healthcare is delivered to have an even greater impact. To provide the best care for each person we partner with many stakeholders and combine our strengths in Diagnostics and Pharma with data insights from the clinical practice.

For over 125 years, sustainability has been an integral part of Roche’s business. As a science-driven company, our greatest contribution to society is developing innovative medicines and diagnostics that help people live healthier lives. Roche is committed to the Science Based Targets initiative and the Sustainable Markets Initiative to achieve net zero by 2045. 

Genentech, in the United States, is a wholly owned member of the Roche Group. Roche is the majority shareholder in Chugai Pharmaceutical, Japan. 

For more information, please visit www.roche.com.

All trademarks used or mentioned in this release are protected by law.


References
[1] Mader, J. K., Waldenmaier, D., Mueller-Hoffmann, W., Mueller, K., Angstmann, M., Vogt, G., Rieger, C. C., Eichenlaub, M., Forst, T., & Freckmann, G. (2024). Performance of a Novel Continuous Glucose Monitoring Device in People With Diabetes. Journal of diabetes science and technology, 18(5), 1044–1051. https://doi.org/10.1177/19322968241267774

[2] Simulation study, Data on file, Roche Diabetes Care GmbH, 2025.

[3] Ngassa Piotie P, Rheeder P. UP Expert Opinion: South Africa has more than 4 million people living with diabetes – many aren’t getting proper treatment. University of Pretoria Diabetes Research Centre News. 2024 Mar 11 [cited 2025 Oct 8]. Available from: https://www.up.ac.za/diabetes-research-centre/news/post_3214595-up-expert-opinion-south africa-has-more-than-4-million-people-living-with-diabetes-many-arent-getting-proper-treatment

[4] Tomic D, Shaw JE, Magliano DJ. The burden and risks of emerging complications of diabetes mellitus. Nat Rev Endocrinol. 2022 Sep;18(9):525-539. doi: 10.1038/s41574-022-00690-7. Epub 2022 Jun 6. PMID: 35668219; PMCID: PMC9169030.

[5] Carlson AL, et al., Diabetes Technol Ther., 2017 May;19(S2):S4-S11. doi: 10.1089/dia.2017.0024

[6] DeSalvo DJ, et al., Journal of Diabetes Science and Technology. 2023;17(2):322-328.

[7] Nakhleh A, Shehadeh N. Hypoglycemia in diabetes: An update on pathophysiology, treatment, and prevention. World J Diabetes. 2021 Dec 15;12(12):2036-2049. doi: 10.4239/wjd.v12.i12.2036. PMID: 35047118; PMCID: PMC8696639.

[8] Secrest AM et al. Characterizing sudden death and dead in bed syndrome in Type 1 diabetes: analysis from two childhood onset Type 1 diabetes registries. Diabet. Med. 2011. 28, 293–300.

[9] Jones J et al. Dead in bed – A systematic review of overnight deaths in type 1 diabetes. Diabetes Res Clin Pract. 2022. 191:110042. 

[10] Wild, Diane et al. “A critical review of the literature on fear of hypoglycemia in diabetes: Implications for diabetes management and patient education.” Patient education and counseling vol. 68,1 (2007): 10-5. doi:10.1016/j.pec.2007.05.003

[11] Glatzer et al., 2024. Journal of diabetes science and technology, 18(5), 1004–1008. https://doi.org/10.1177/19322968241267765

[12] Parkes Error Grid – a tool to evaluate the accuracy of glucose monitoring systems (BGM and CGM).

[13] Herrero, P., Andorrà, M., Babion, N., Bos, H., Koehler, M., Klopfenstein, Y., Leppäaho, E., Lustenberger, P., Peak, A., Ringemann, C., & Glatzer, T. (2024). Enhancing the Capabilities of Continuous Glucose Monitoring With a Predictive App. Journal of diabetes science and technology, 18(5), 1014–1026. https://doi.org/10.1177/19322968241267818.

Categories : Metabolic Disorders UrogenitalTags :

Promising New Breakthrough for Preventing Kidney Damage in Type 1 Diabetes

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Chronic kidney disease (CKD). Credit: Scientific Animations CC4.0

A new gene therapy approach aimed at protecting people with type 1 diabetes from developing diabetic kidney disease – a serious and common complication of the condition, has shown promising results in a University of Bristol study.

One in three people with type one diabetes will develop kidney damage during their lifetime, which can develop silently over many years, often going undetected until it becomes severe.

Current treatments can slow kidney damage but there are none that act on the root cause: a tiny filter called the glomerulus. A new study published in Molecular Therapy, demonstrated a 64% reduction in a damage indicator for kidney disease, paving the way for a potential new treatment.

The study, driven by first author, Dr Aldara Martin Alonso and led by Dr Rebecca Foster, Associate Professor of Microvascular Medicine at Bristol Medical School: Translational Health Sciences, explored the potential of delivering a protein called VEGF-C directly into kidney cells.

Previous studies have shown VEGFC could protect against kidney disease as it helps keeps blood vessels in the kidney filter healthy, repairing early signs of diabetes-related kidney damage.

To test whether this new approach could be used to treat or slow down kidney disease, the team used a harmless virus to deliver VEGF-C directly into the kidney cells of diabetic mice.

Their results showed that this approach not only helped the kidneys work better, but also protected a key part of the kidney filter that normally helps prevent damage. It led to a 64% reduction in albuminuria. Importantly, this reduction is more than twice the reduction recommended by the American Diabetes Association to slow the progression of chronic kidney disease.

Dr Foster, the study’s senior author, explained: “Currently, there are no drugs specifically available to protect people with type 1 diabetes from kidney disease, despite their higher risk of developing kidney disease. This gap in treatment highlights the urgent need for new therapeutic approaches. Our goal was to investigate whether gene therapy could offer a viable solution by delivering VEGFC in a more targeted way.”

Dr Foster added: “This gene therapy approach has not been explored before in pre-clinical models and offers a long-term solution for these patients who are at risk of developing kidney disease.”

Source: University of Bristol

Categories : Metabolic DisordersTags :

Unmasking a New Diabetes Subtype in Sub-Saharan Africa

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Photo by Wes Dissy on Unsplash

In this month’s podcast, QuickNews looks at a new Lancet study, “Non-autoimmune, insulin-deficient diabetes in children and young adults in Africa.” In this study, researchers report that a significant subset of what has previously been classified as Type 1 diabetes in sub-Saharan Africa may in fact be a distinct, novel form of the disease.

The individuals in this subset did not exhibit the typical autoimmune markers (islet autoantibodies) usually found in classic Type 1 diabetes in other parts of the world. The researchers instead identified a novel, non-autoimmune, insulin-deficient subtype of diabetes that is also distinct from Type 2 diabetes.

Further evidence of this new subtype was found in Black individuals in the USA, albeit less frequently, but not in White individuals. The discovery throws a spotlight on the heterogeneity of diabetes diagnoses in sub-Saharan Africa, and points to the need to consider alternative causes and explore new prevention and treatment strategies for this distinct form of the disease.

Categories : Implants and Prostheses Metabolic DisordersTags :

Implant Treats Type 1 Diabetes by Oxygenating Insulin-producing Cells

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A 3D map of the islet density routes throughout the healthy human pancreas. Source: Wikimedia CC0

Cornell researchers have developed an implant system that can treat Type 1 diabetes by supplying extra oxygen to densely packed insulin-secreting cells, without the need for immunosuppression. The system could also potentially provide long-term treatment for a range of chronic diseases.

The findings appear in Nature Communications. The co-first authors were former postdoctoral researcher Tung Pham and doctoral candidate Lora (Phuong) Tran.

The technology builds off previous implantable encapsulation devices developed in the lab of Minglin Ma, professor of biological and environmental engineering in the College of Agriculture and Life Sciences (CALS) and the paper’s senior author.

Ma has explored a variety of ways to address Type 1 diabetes, which is typically managed through daily insulin injections or insulin pumps, but even with that treatment, patients still suffer the devastating effects of the disease.

Ma’s previous implantable devices have proved effective in controlling blood sugar in diabetic mice, but they can only last so long.

“One of the major challenges is the implant itself often dies due to the lack of oxygen after implantation,” Tran said. “In our lab, they had success in mice that lived over one year, and they controlled the diabetes very effectively with some small capsules without oxygen generation. However, when we scale up, we need more cells, we need more density, especially. We need a higher dose. If we implant without generating oxygen, the cells often die within two weeks.”

Ma’s team developed the new system in collaboration with electrochemical researchers from Giner Inc, including co-author Linda Tempelman, PhD ’93.

The key components of the system are a cylindrical capsule with a ring-shaped cross-section that contains transplanted insulin-secreting cells, and an electrochemical oxygen generator that is roughly the size of a dime and removeable. A nanofibrous membrane outside the capsule protects the cells from the host body’s immune system; a permeable membrane in the core of the capsule allows the central supply of oxygen to reach the ring of cells.

“We have to meet two requirements,” Tran said. “The first is immune protection. And second, you have to maintain mass transfer, like the glucose and other nutrients and molecules that can go in and out.”

Working with Dr James Flanders, associate professor emeritus in the College of Veterinary Medicine, the researchers successfully tested the system in rat models.

“It’s the proof of concept. We really proved that oxygenation is important, and oxygenation will support high cell-density capsules,” Tempelman said. “The capsules are immune protective and last for a long time without having some kind of fouling of the membrane. The body never likes when you put a foreign substance in. So that’s the engineering in the Ma Lab, to look for materials and coatings for the materials that are immune protective, but also don’t invoke excess response from the body because of the material.”

The new system would enable a much greater number of the 2 million people who suffer from Type 1 diabetes in the US to now have an islet transplant or cell therapy without requiring immune suppression, which is considered too dangerous for routine use. Also, the new system can provide much tighter sugar control, effectively curing the disease and enabling the person to eat, drink and exercise like everyone else. 

The next step will be to implant the system in a pig model, and also test it with human stem cells. The researchers are interested in eventually trying to use the system for implanting different cell types in humans for long-term treatment of chronic diseases, according to Tempelman, who is CEO of Persista Bio Inc., a new startup she founded with Ma and Flanders that is licensing these technologies.

“We see an age where people will be getting implants with allogeneic cells from other human beings, from stem cell lines, and using it long term to treat things that your body is missing,” Tempelman said. “Here we’re missing insulin. In pain control, maybe you need more endorphins or some other molecule. In enzyme replacement therapy, you need more enzymes. We’re interested in things like other autoimmune diseases where there’s inflammation that’s out of control.

“So maybe you could put a small molecule in that would treat inflammation, and then someone, like a Lupus patient, wouldn’t have to take an oral medication. They could just have a low-level implant that provides a low amount of it.”

Source: Cornell University

Categories : Metabolic Disorders PaediatricsTags :

Clinical Trial Shows Improved Health Outcomes for Teens with Type 1 Diabetes

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A new study in teenagers with type 1 diabetes shows promise in reducing chronic kidney disease and informing future precision care.

Photo by Nataliya Vaitkevich on Pexels

A clinical trial involving adolescents with type 1 diabetes (T1D) has found a combination therapy may reduce chronic kidney disease and improve health outcomes. The findings could help guide more precision care for young people with T1D. 

Led by Dr Farid Mahmud, Associate Scientist in the Translational Medicine program and Staff Physician in the Division of Endocrinology at The Hospital for Sick Children (SickKids), and published in Nature Medicine, the study evaluated a therapy that combines standard insulin treatment with the investigational drug dapagliflozin. Results of this combination therapy showed improved blood sugar control and kidney function, and reduced weight gain in adolescents with T1D. 

While most people with T1D are diagnosed as adults, the condition often starts in childhood and early adolescence. The lifelong insulin therapy needed can lead to side effects such as weight gain and chronic kidney disease. In the trial, participants who received dapagliflozin alongside insulin had fewer of these side effects and better overall health outcomes. 

“Our findings showed that adolescents who received this combination therapy were able to improve many symptoms typically associated with insulin-managed type one diabetes,” says Mahmud. “This could inform a new early intervention strategy for the growing population of teenagers with type one diabetes.” 

Patient partner key to trial success 

While previous research has shown similar results in adults, Mahmud’s team focused on designing a clinical trial specifically for teenagers, a group often underrepresented in clinical trials. Hormonal changes, psychological development, and the shared responsibility between teens and their parents for managing treatment protocols can make trial participation more complex for this age group. 

To address these challenges, the research team worked closely with patient partner Lynne McArthur. Together, they enrolled 98 participants between 12 and 18 years old in the study, known as the ATTEMPT study, across three sites. 

McArthur’s involvement in research began when one of her twin sons was diagnosed with T1D following a trip to the SickKids emergency department at just 18 months old. A few years later, his twin was also diagnosed. That experience led McArthur to become more involved in research efforts to improve diagnosis and treatment options for families like hers. 

“Deciding to participate in a clinical trial is an important decision, but my goal has always been disease prevention. I knew that our participation could help build a future where children don’t get T1D.”

Lynne McArthur

Now that her sons are older, McArthur continues to be involved as a patient advisor. She reviews recruitment materials and provides feedback on trial design, helping ensure the research stays connected to the lived experience of people managing T1D. 

“Participating in research, whether in a trial or as an advisor, is hugely rewarding. With my experience as trial participant, I can see how the plans on paper would impact the real lives of people living with diabetes,” explains McArthur.  

Source: SickKids News

Categories : Cardiovascular Disease Metabolic DisordersTags :

Adult-onset Type 1 Diabetes Increases Cardiovascular Risk, Especially in 40s and Older

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Source: Pixabay CC0

A new study in the European Heart Journal shows that people who develop type 1 diabetes in adulthood have an increased risk of cardiovascular disease and death, and that those diagnosed later in life do not have a better prognosis than those diagnosed earlier. The study, conducted by researchers at Karolinska Institutet, points to modifiable factors – smoking, poor glucose control and obesity – as the main risk factors.

Type 1 diabetes used to be called childhood diabetes but can start at any time during life – a situation for which there is limited research. The researchers behind the current study wanted to investigate the risk of cardiovascular disease and death in this group, particularly for those diagnosed after the age of 40.

The registry-based study identified 10 184 people diagnosed with type 1 diabetes in adulthood between 2001 and 2020 and compared them to 509 172 matched people in the control group.

The study shows that these people with adult-onset type 1 diabetes had a higher risk of cardiovascular disease and death from all causes, including cancer and infections, compared to the control group.

“The main reasons for the poor prognosis are smoking, overweight/obesity and poor glucose control. We found that they were less likely to use assistive devices, such as insulin pumps,” says first author Yuxia Wei, postdoctoral fellow at the Institute of Environmental Medicine, Karolinska Institutet.

The prognosis can be improved 

The results emphasise the seriousness of type 1 diabetes, even when it starts later in life, the researchers say. But the prognosis can improved by avoiding smoking and obesity, especially for those diagnosed later in life.

The researchers plan to continue investigating adult-onset type 1 diabetes, including risk factors for developing the disease and the prognosis of other outcomes, such as microvascular complications. Optimal treatment in adult-onset type 1 diabetes, including the effect of pump use and other advanced technologies, also needs to be explored.

Source: Karolinska Institutet

Categories : Metabolic DisordersTags :

Dual-action Approach Targeting Inflammation Shows Potential as Type 1 Diabetes Treatment

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Photo by Nataliya Vaitkevich on Pexels

A study co-led by Indiana University School of Medicine researchers presents a potential new strategy to prevent or slow the progression of Type 1 diabetes by targeting an inflammation-related protein known to drive the disease. The findings, recently published in eBioMedicine, may help inform clinical trials of a drug that is already approved by the U.S. Food and Drug Administration for psoriasis as a treatment for Type 1 diabetes.

In laboratory studies using human cells and mouse models, the researchers found that applying a molecular method to block inflammation signalling through the tyrosine kinase 2 (TYK2) protein reduced harmful inflammation in the pancreas. This strategy not only protected the beta cells in the pancreas but also reduced the immune system’s attack on those cells. A medication that inhibits TYK2 is already approved for the treatment of psoriasis, an autoimmune condition that causes skin inflammation.

“Our study showed that targeting TYK2 could be a powerful way to protect insulin-producing beta cells while calming inflammation in the immune system at the same time,” said Carmella Evans-Molina, MD, PhD, co-author of the study and director of the Indiana Diabetes Research Center and the Eli Lilly and Company Professor of Pediatric Diabetes at the IU School of Medicine. “This finding is exciting because there is already a drug on the market that does this for psoriasis, which could help us move more quickly toward testing it for Type 1 diabetes.”

Past genetic studies have already shown that people with naturally lower TYK2 activity are less likely to develop Type 1 diabetes, further supporting the group’s approach for future treatments using this TYK2 inhibitor approach.

“Our preclinical models suggest that the treatment might work in people as well,” said Farooq Syed, PhD, lead author of the study and assistant professor in the Department of Diabetes-Immunology at the Arthur-Riggs Diabetes and Metabolic Research Institute of the City of Hope. “The next step is to initiate translational studies to evaluate the impact of TYK2 inhibition alone or in combination with other already approved drugs in individuals at-risk or with recent onset Type 1 diabetes.”

Source: Indiana University