Tag: 25/9/26

Categories : OphthalmologyTags :

Simple Pill Could Prevent Sight Loss in Common Form of Diabetic Retinopathy

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Retina showing reticular pseudodrusen. Although they can infrequently appear in individuals with no other apparent pathology, their highest rates of occurrence are in association with age-related macular degeneration (AMD), for which they hold clinical significance by being highly correlated with end-stage disease sub-types, choroidal neovascularisation and geographic atrophy. Credit: National Eye Institute

Researchers at Trinity, in collaboration with Breye Therapeutics, have found that an oral medication (Danegaptide) can protect against vision-threatening damage in Non-Proliferative Diabetic Retinopathy (NPDR), which is a leading cause of blindness in working-age adults.

The study, published today in leading international journal, Science Translational Medicine, highlights a new therapeutic approach that could replace or complement frequent, highly invasive eye injections for millions of patients with diabetes.

Diabetic retinopathy affects nearly 30% of people living with diabetes and the non-proliferative form (NPDR) represents the vast majority of cases. 

Currently, standard treatments such as intravitreal anti-VEGF injections are reserved for advanced stages of the disease after significant, often irreversible, vision loss has already occurred. Because these injections require regular and direct administration directly into the eye, early intervention has remained impractical.

Key findings

In pre-clinical models of the disease, administering Danegaptide rapidly reduced leakage of blood vessels in the retina, prevented inner blood-retina barrier (iBRB) breakdown, and reversed retinal oedema (swelling) with efficacy comparable to injectable therapies.

Additionally, in an early stage clinical trial involving 24 patients with NPDR, oral Danegaptide was safe and well-tolerated. Notably, 55% of participants demonstrated early signs of biological activity, including marked reductions in retinal leakage, decreased central subfield thickness, and resolution of intra-retinal fluid cysts.

What are the potential impacts of this research?

Global estimates suggest tens of millions of people are living with NPDR, with roughly one-third of adults with diabetes likely to develop this or another form of diabetic retinopathy in their lifetime.  

Given how debilitating the condition can be and the urgent need for better, less invasive treatments, this research offers significant hope that a simple oral medication could make a huge difference in effectively managing the condition and enabling affected people to live more normal lives. 

The findings are extremely promising and the research team now underlines the need to progress with larger Phase II clinical trials. 

“Managing diabetic eye disease before irreversible damage occurs has long been constrained by the burden of invasive injections,” said Professor Matthew Campbell, Chair of Neurovascular Genetics at Trinity College Dublin and senior author on the research paper. 

“Our findings demonstrate that a simple oral pill can effectively target and repair the inner blood-retina barrier. This potentially offers a non-invasive, preventive path forward that could protect vision in both eyes simultaneously, long before severe sight loss takes hold.” 

Dr Pete Adamson, CSO, Breye Therapeutics, added: “The data from this study validate a major paradigm shift in retinal care. By proving that an oral therapy like Danegaptide can directly protect the inner blood-retina barrier, we are opening the door to early, non-invasive intervention for NPDR. The successful Phase Ib results provide a very strong foundation for larger Phase II clinical trials to evaluate long-term efficacy.”

Source: Trinity College Dublin 

Categories : Obstetrics & GynaecologyTags :

Study Proposes Safer Alternative to Hormone Therapy During Menopause

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Liver cells viewed under a fluorescence microscope. On the left are healthy cells, and on the right, fat droplets stand out in yellow. The research revealed that activating the ERβ receptor induces the organ to completely oxidize these lipids to generate energy. Credit: Débora Santos Rocha et al./Comprehensive Physiology

By Maria Fernanda Ziegler  |  Agência FAPESP – When it comes to balancing hormonal changes during the transition to menopause, less can be more. Researchers at the University of São Paulo (USP) in Brazil demonstrated this when testing an alternative strategy to conventional therapy. Rather than replacing oestrogen entirely, the team activated only one type of oestrogen receptor in the cells. This approach achieved a broad and beneficial metabolic effect without stimulating the growth of reproductive tissues, such as the uterus and breasts. This is important for individuals with a genetic predisposition to developing tumours.

“It’s a more targeted strategy. By acting only on that receptor, we were able to rebalance the metabolic effects of the drop in oestrogen during menopause without affecting potentially sensitive areas of the body,” explains the researcher Débora Santos Rocha, a FAPESP postdoctoral fellow at the Institute of Chemistry (IQ-USP) and first author of the article published in the journal Comprehensive Physiology.

The study was conducted on female rats that had their ovaries removed to simulate menopause. In this model, the researchers used an experimental drug to activate only oestrogen receptor beta (ERβ), which is an important regulator of metabolism and does not stimulate reproductive tissues or increase the risk of hormone-sensitive tumours.

As Rocha explains, the significant drop in oestrogen during the transition to menopause disrupts the entire metabolism, potentially promoting the accumulation of visceral fat, insulin resistance, and cardiovascular risk. “In addition to their importance in reproduction, oestrogens play a central role in energy regulation. When their levels decrease, widespread metabolic changes also occur, increasing the risk of metabolic syndrome and diseases such as diabetes and hypercholesterolaemia [high cholesterol],” she states.

Conventional hormone replacement therapy attempts to counteract this loss by providing oestrogen and progesterone through pills, patches, or creams. However, by activating all oestrogen receptors indiscriminately, it may increase the risk of tumour growth in predisposed women. “It isn’t hormone replacement therapy that causes cancer. What happens is that if there are tumour cells in oestrogen-sensitive tissues, such as the uterus, breast, or endometrium, they may respond to the hormone and multiply more quickly,” Rocha explains.

A targeted solution with broad benefits

The results of the study show that the isolated activation of ERβ promoted profound metabolic reprogramming. “With the new approach, we restored fasting blood glucose levels, normalised the lipid profile, and reduced the size of fat cells. The pancreatic islets [cells that produce insulin and other hormones] regained their normal shape, and blood levels of cholesterol, triglycerides, and free fatty acids returned to healthy levels. All of that occurred without any effect on the uterus, which indicates the safety of the approach,” comments Alicia Kowaltowski, a professor at IQ-USP and the research coordinator.

The study primarily focused on the liver. This is because the organ begins to process fats differently during the transition to menopause and in the absence of oestrogen. “Lipid metabolism changes significantly, and the liver accumulates more fat. ‘Bad’ cholesterol [LDL] in the blood increases, and various lipid profiles are altered. However, with the activation of ERβ, those parameters returned to normal,” says Rocha.

The same effect was observed in the analysis of isolated liver cells. “The drug remodels the metabolism of liver cells [hepatocytes], causing them to oxidise fatty acids more [ie, to ‘burn’ the building blocks of lipids to generate energy]. That reduces fat accumulation and improves the overall lipid profile. By activating that specific pathway, we modulate liver metabolism and make circulating lipids healthier,” the postdoctoral fellow explains.

Kowaltowski highlights another important finding from the study. “By activating ERβ, the drug causes liver cells to fully oxidize fatty acids, something that normally doesn’t happen since the liver typically performs only partial oxidation, generating ketone bodies [alternative energy sources that the body creates to compensate for a lack of carbohydrates]. With that ‘complete oxidation,’ fat is fully converted into CO₂, significantly improving the body’s lipid profile. It’s important to note that this pathway can be activated to provide metabolic benefits – something we hadn’t imagined,” she explains.

There is no one-size-fits-all model

Although the treatment did not prevent the weight gain characteristic of menopause, the authors note in the article that it broadly improved metabolic quality and the functioning of the liver, pancreas, and blood.

“Menopause is a phase characterised by a wide variability of symptoms among women, which makes it important to have a variety of therapeutic approaches. If we can modulate specific pathways, we pave the way for more personalized treatments. Perhaps the approach we’re presenting won’t be ideal for one person, but it could be decisive for another. The important thing is to have treatment options,” Kowaltowski emphasizes.


“Perhaps the approach we’re presenting won’t be ideal for one person, but it could be decisive for another. The important thing is to have treatment options,” emphasises Alicia Kowaltowski, a professor at IQ-USP (photo: Cecília Bastos/USP Imagens)

The research continues in a new project funded by FAPESP that is now focused on the transition to menopause (perimenopause), using an experimental model that more closely mimics human reality.

Rather than abruptly removing the ovaries from female rats, the new study will use a model of gradual ovarian reserve decline, enabling the researchers to observe hormonal change dynamics throughout the process. “We want to understand that transition and identify new therapeutic targets that could serve as alternatives to conventional hormone replacement therapy,” Rocha concludes.

The article “Estrogen receptor beta activation coordinates liver lipid remodeling and metabolic fluxes, preventing lipotoxicity” can be read at onlinelibrary.wiley.com/doi/10.1002/cph4.70228.

Source: FAPESP

Categories : PaediatricsTags :

Probiotics for Very Preterm Babies Linked to Reduced Mortality

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Photo by Hush Naidoo on Unsplash

Probiotic supplementation in very preterm babies was linked to a lower risk of both severe intestinal disease and death. This is shown by a new study from the Karolinska Institutet, published in JAMA Network Open. The findings are based on data from nearly 4700 babies born prematurely in Sweden.

Very preterm babies are at increased risk of developing necrotising enterocolitis (NEC), a serious intestinal disease that can be life-threatening. Since 2020, the Swedish Neonatal Society has recommended that babies born between gestational weeks 28 and 31 receive daily probiotic supplements – that is, live bacteria that can influence the gut microbiota. This  is in line with a European recommendation based on international research.

In the current study, the researchers analysed data from the Swedish nNeonatal care quality register on 4695 babies born between 2017 and 2024. Of these, 1571 babies received a probiotic supplement containing three selected bacterial strains, whilst 3124 babies did not. The researchers investigated whether probiotic supplements could be linked to the risk of death or of developing NEC during their stay in the neonatal ward.

”We found that infants who received probiotics had a significantly lower risk of both NEC and death compared with those who did not receive the supplements. At the same time, mortality was low in both groups,” says Ayoub Mitha, a doctor and researcher at the Department of Medicine, Solna, Karolinska Institutet, who is the study’s corresponding author.

Among children who received probiotics, 0.8% experienced death or NEC, compared with 3.4% among those who did not receive the supplement. The risk of death was 0.3% in the probiotic group compared with 1.4% in the group without probiotics. The risk of NEC was 0.6% and 2.5% respectively.

Similar results regardless method

The researchers used several different statistical methods to account for differences between the groups. The results were similar regardless of the analysis method. Further analyses suggested that a significant proportion of the lower mortality rate was linked to fewer children developing NEC.

However, the researchers found no clear link between probiotic supplementation and the risk of sepsis, a serious infection that can spread throughout the body. The proportion of children affected by sepsis was roughly the same in both groups.

“Our findings contribute to the international body of knowledge and may help healthcare professionals and families make informed decisions regarding probiotic supplements for very preterm infants. At the same time, it is important not to generalise the results to extremely preterm infants – those born before 28 weeks – as this group was not included in the study,” says Ayoub Mitha.

The researchers also emphasise that the study is observational. This means that it can show associations but cannot definitively establish cause and effect.

Source: Karolinska Institutet

Categories : Neurology Pain ManagementTags :

Anti-epileptic Drug May Reduce Migraine Aura

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Photo by Andrea Piacquadio: https://www.pexels.com/photo/woman-in-gray-tank-top-3812757/

A new study by researchers at Lund University in Sweden found that 85 percent of patients with aura-dominant migraine experienced a reduction in monthly aura days by at least half following treatment with the anti-epileptic drug lamotrigine. The findings need to be confirmed in a controlled study, but could eventually be significant for a patient group that currently lacks a specific treatment for the aura itself.

Around 1.5 million people in Sweden suffer from migraine. Migraine affects both individuals’ daily lives and society as a whole. The disease limits the ability to work and participate in social activities, and leads to significant costs through sick leave, reduced productivity and increased healthcare needs. Women are three times as likely to be affected as often as men, a difference that is largely due to hormonal factors.  

Up to a third of people with migraine experience aura – a temporary neurological symptom that can cause visual disturbances, loss of vision, sensory disturbances, balance problems, and speech and language difficulties, among other symptoms. For some patients, aura is the most debilitating symptom of the disease, but there is currently no established treatment that specifically targets it. 

“Around 50,000 people in Sweden suffer from aura-dominant migraine. Aura is thought to be linked to cortical spreading depolarisation (CSD), a slowly progressing wave of altered nerve cell activity in the cerebral cortex,” says Gürdal Sahin, a researcher at Lund University and a specialist in neurology at the Skåneuro private clinic. 

Migraine with aura is also associated with a slightly increased risk of ischaemic stroke, particularly when combined with other vascular risk factors. It is therefore important to reduce modifiable risk factors. 

“There are now several effective treatments for migraine, including new medicines that block the neurotransmitter CGRP, which plays a key role in migraine attacks. Botulinum toxin is also used as a preventative treatment for chronic migraine. However, as there is no established treatment that specifically targets the aura itself, migraines with and without aura are often treated in a similar way, even though the aura appears to be partly based on mechanisms other than the headache itself,” Gürdal Sahin continues. 

The medicine lamotrigine is approved for the treatment of epilepsy and bipolar disorder, but not for migraine. However, as both epilepsy and migraine aura involve temporary changes in nerve cell activity, the researchers wanted to investigate whether the drug could also prevent aura. Lamotrigine affects the electrical activity of nerve cells by acting on sodium and potassium channels, and also reduces the release of, amongst other things, the neurotransmitter glutamate. In this way, the medicine can reduce the excessive excitability of the nerve cells. In theory, these mechanisms could help to prevent the emergence and spread of CSD. 

“In our study, we examined 81 patients with frequent or particularly troublesome migraine aura and treated them with lamotrigine. Among the patients included the number of days with aura fell on average from 6.9 to 1.4 days per month, representing a reduction of around 80 per cent,” says Sena Uzun, a doctoral student at Lund University. 

While previous studies involving lamotrigine have shown mixed results, the results of this study were particularly promising for patients with aura-dominant migraine. In this group, 85 per cent of patients experienced at least a 50 per cent reduction in the number of days with aura. Another interesting observation was that a slower dose escalation was associated with fewer side effects, which may be relevant when the treatment is evaluated in future clinical trials. 

“As the study is retrospective and lacks a control group, the results need to be confirmed. We are now proceeding with a national, randomised, double-blind, placebo-controlled trial of lamotrigine. If the results can be confirmed, the drug could become a relatively inexpensive and well-established treatment option for a group of patients who currently have no treatment for the aura itself,” concludes Sena Uzun.

Original written by Åsa Hansdotter

Source: Lund University

Categories : HospitalsTags :

World Pharmacists Day – 25 September 2026

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Pharmacists are the most accessible healthcare professionals for many South Africans. Image created with AI.

Astrid Stealey, Area Managing Director, South Africa & Sub-Saharan Africa, Kenvue

World Pharmacists Day is an opportunity to recognise the contribution pharmacists make to the health of communities across South Africa. But recognition must go hand in hand with ensuring they have the training, resources and support to meet the demands being placed on them.

Pharmacists are often among the most accessible healthcare professionals in their communities. They play an important role in helping people navigate everyday self-care, understand the responsible use of medicines and recognise when symptoms require referral to a doctor or another healthcare service. That guidance can help people make more informed decisions about their health, but it should never be taken for granted.

Kenvue’s previously published research found that 82% of surveyed South African pharmacists regularly provide advice to people who cannot access a GP. At the same time, 76% reported increased demands on their time.

These findings highlight both the value pharmacists provide and the pressure many are working under. Supporting them means investing in continuous professional education, access to credible, evidence-based information and systems that recognise both the potential and the limits of their role.

This year’s World Pharmacists Day 2026 theme, “Empowering pharmacists for healthier futures,” captures that challenge well. A healthier future depends on pharmacists being equipped and supported to use their expertise confidently, responsibly and sustainably.