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EDITORIAL | After Major Research Cuts, SA Charts a New Path

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To limit the damage from the US research cuts, the SAMRC mobilised a rescue fund of about R600 million.

Spotlight Editors

It has been a bruising year or so for medical researchers in South Africa with the US pausing, cancelling, and then resuming some grants. But as bad as things were, what played out wasn’t the worst case scenario, and momentum is now building toward recovery.

For decades, the United States government has been the world’s top funder of medical research. When it started cutting research funding last year, South Africa was caught in the firing line. This is because the US administration decided to specifically target South Africa, but also because South Africa was uniquely exposed due to the sheer volume of US-funded research here.

Over recent decades, South Africa built an impressive network of research groups and infrastructure to support high quality research – all underpinned by a strong regulatory environment, several good universities, and many productive partnerships with research groups from across the world. All this, plus the fact that we have large TB and HIV epidemics, means that South Africa was, and still is, one of the best places in the world to conduct research on these two diseases.

But a weakness of South Africa’s impressive research infrastructure was its overreliance on US funding.

To be clear, this was not an overreliance on aid or charity. South African researchers won grants from the US by coming out on top in rigorous and highly competitive selection processes. Much of the research done here benefited people around the world, including in the US.

Instead, the thing that we overly relied upon was that the US would continue to make medical research grants in a way that is rational and in our common interest.

There was much chaos and uncertainty last year with the pausing, cancellation, and resuming of grants. One small positive is that bad as things were, what played out wasn’t the worst case scenario we seemed to be heading for. At least some projects got their funding flows restored. You can read more about that in this Spotlight article.

But there is no doubt that the situation remains very bleak. While some studies that were already underway will be completed, it seems very unlikely that the US will fund any new studies in South Africa in the coming years. Given the historic scale of US investment here, the total volume of clinical trials conducted in South Africa will almost certainly fall precipitously.

Charting a new course

One ray of light in all this has been the response from the South African Medical Research Council (SAMRC) – probably the best run of all the entities linked to the Department of Health.

To limit the damage from the US research cuts, the SAMRC mobilised a rescue fund of about R600 million. This includes major contributions from National Treasury, the Gates Foundation, the Wellcome Trust and the ELMA Foundation.

Some of this funding has already helped sustain dozens of research projects and protect vital expertise during a period of instability. The current funding supports work in HIV, TB, newborn and child health, as well as non-communicable and other infectious diseases.

One example is a cutting-edge HIV vaccine clinical trial that began in January at the Desmond Tutu Health Foundation’s clinical research site at Groote Schuur Hospital in Cape Town. While still in its early stages, the study aims to help piece together what an effective HIV vaccine might look like.

Beyond the SAMRC’s efforts, universities and research institutions have also stepped in, raising funds to safeguard projects and retain skilled staff whose jobs were at risk.

Even so, we are still facing a massive net loss to money for medical research in South Africa.

What to do?

Funding from international partners will remain vital in South Africa. For now, the US government still invests substantial funds in South Africa, as does several philanthropies and the European Union, through the European & Developing Countries Clinical Trials Partnership. There are also new partnerships such as one we recently reported on between South African and Korean researchers.

Such partnerships are not just about money – science thrives where there is collaboration across national borders. In fact, almost all of the most important TB and HIV clinical trials conducted in South Africa in the last two decades were collaborations between researchers from multiple countries. No matter how you slice it, collaboration with international partners will remain an essential foundation of the medical research landscape in South Africa.

The problem was never that South African researchers took too much money from the US or other donors, or worked too closely with researchers based in other countries. One might quibble on details here and there, but on the whole, US-South African research collaboration in recent decades has been a resounding success.

Rather, the problem was that we invested so little of our own funds that we became overly vulnerable to changes in external funding.

Professor Ntobeko Ntusi, president and CEO of the SAMRC, previously told Spotlight that the SAMRC receives in the region of R2 billion from government per year, including funds from both the Department of Health and the Department of Science and Innovation.

Unlike so many parts of our government, the SAMRC is a well-run entity that got clean audits in each of the last five years. This strongly suggests that money allocated to it won’t be wasted or looted. If we understand recent messaging from the Finance Minister and National Treasury, this is precisely the kind of clean government spending that should be rewarded in future budgets.

Relative to health budgets more generally and to what government has historically spent on entities such as South African Airways, the SAMRC’s budget is tiny. As far as we can tell, the current funding level is largely a product of history – apart from the still widespread atmosphere of austerity, there really isn’t any other reason why the budget shouldn’t be scaled up over the next three years to be double what it is now.

The SAMRC supports a sector in which South Africa has truly world-class capacity – capacity that as we speak remains under threat. More than just the research studies and the jobs for young scientists, what is at stake here is the idea of South Africa as a place where we can do world-class medical research. Allowing funding cuts to extinguish this bright spark, would feel like a victory for Afro-pessimism.

The reality is that if President Cyril Ramaphosa and National Treasury seizes the opportunity, the shock of the US funding cuts could be turned into a bright new beginning for medical research in South Africa – all at a price that in relative terms is very low. Let’s hope they have the vision and ambition to seize the day.

Disclosure: The Gates Foundation is mentioned in this article. Spotlight receives funding from the Gates Foundation, but is editorially independent – an independence that the editors guard jealously. Spotlight is a member of the South African Press Council.

Republished from Spotlight under a Creative Commons licence.

Read the original article.

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Half of Social Science Research Results Cannot Be Replicated

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Photo by Pexels on Pixabay

A major international collaboration on scientific reliability has been completed and is now presented in three articles in Nature by researchers from institutions including Karolinska Institutet. Around half of previously published research results in the social and behavioural sciences could not be replicated in new experiments.

The research programme, known as SCORE, involved 865 researchers who analysed nearly 3900 scientific articles published between 2009 and 2018 in 62 journals in the fields of criminology, economics, educational science, health sciences, leadership, marketing, organisational behaviour, psychology, political science, public administration and sociology.

In three studies published in Nature, different methods were used to investigate whether the research results are reliable. The questions addressed were whether the results can be reproduced, whether they are robust, and whether they can be replicated.

Replication involves testing the same research question, but with new data. In the replication study, the researchers analysed 164 previously published results in the social and behavioural sciences. Of these, just under half, 49%, could be replicated with a similar result to that of the original study.

Open data is key

In the reproducibility study, the same analysis was carried out on the same data. Reproducibility was hampered by the fact that data was often unavailable; only just under a quarter of the articles studied had shared their data openly. Of the 143 articles analysed, 74% could be reproduced to an approximate degree, and 54% to an exact degree. When the original data and code were shared, these figures increased to 91 and 77%, respectively.

“This shows that transparency is key to achieving credible research results,” says Gustav Nilsonne, associate professor of neuroscience at Karolinska Institutet, who co-led the robustness study and is a co-author of all three papers. “Sharing research data enables outsiders to assess which results are reliable.”

In the robustness study, alternative analyses were tested on the same data in 100 different articles. For each article, at least five researchers analysed the same hypothesis using the same data, but with the analysis method they deemed to be the best.

Same conclusion in most cases

Only a third of the new analyses yielded results very close to those reported in the original study. However, three out of four analyses reached the same overall conclusion as the original article. But in about a quarter of the cases, no clear effects were found at all, and in a few cases (around two per cent), the results pointed in the opposite direction.

“This is the world’s largest research project to date investigating the reliability of reported scientific results, and an example of how large-scale collaborations can address questions that no single research group could answer alone,” concludes Gustav Nilsonne. “I hope we will see systematic replication attempts in more fields of research in the future.”

The collaboration was led by the Center for Open Science and researchers at Pennsylvania State University, TwoSix Technologies, the University of Southern California and Eötvös Loránd University. The programme was funded by the US research council DARPA.

Publications

“Investigating the reproducibility of the social and behavioural sciences”, Olivia Miske et al., Nature, online 1 April 2026, doi: 10.1038/s41586-026-10203-5.

“Investigating the analytical robustness of the social and behavioural sciences”, Balazs Aczel et al., Nature, online 1 April 2026, doi: 10.1038/s41586-025-09844-9.

“Investigating the replicability of the social and behavioural sciences”, Andrew Tyner et al., Nature, online 1 April 2026, doi: 10.1038/s41586-025-10078-y.

Source: Karolinska Institutet

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World’s Longest Running Birth Cohort Study Marks 80 Years

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Participants were children when the study began. Photo: supplied.

The world’s longest continuously running birth cohort study, which follows thousands of participants born in the first week of March 1946 and is hosted by University College London, is celebrating its 80th birthday.

The Medical Research Council (MRC) National Survey of Health and Development (NSHD), also known as the British 1946 birth cohort, has advanced understanding of what affects our health and wellbeing over a lifetime.

Through questionnaires, clinic visits, and home visits, these study members have helped shape our knowledge of developmental milestones, education, diet, exercise, mental and physical health and healthy ageing across the life course.

Findings have helped reveal how early life conditions, schooling and social circumstances influence adult health, chronic disease and later‑life function, and provided evidence on topics ranging from childhood lung infections and later respiratory disease, to the long‑term effects of diet and physical activity, to the lasting effects of childhood social inequalities.

Professor Nishi Chaturvedi, Director of the Unit for Lifelong Health and Ageing at UCL, said: “On the NSHD’s 80th birthday, we want to extend our deepest thanks to every study member. Their lifelong contributions have been invaluable to medical science, and their generosity with their time continues to make this work possible.”

The origins of the study go back to the 1930s, a period when concerns were growing about declining birth rates and the rising cost of having children. Policymakers feared that financial pressures were discouraging families from expanding. The result was the Maternity Survey of 1946, which captured every birth that took place in England, Wales, and Scotland during a single week in March of that year. Its success was immediate and influential, paving the way for nurses to be able to offer pain relief in childbirth.

From this initial survey, a cohort of 5362 babies was selected for continued follow-up, and remarkably, more than 2000 are still taking part eight decades later.

In recent years, the NSHD has become a flagship study of ageing, with study members taking part in clinical sub-studies to improve our understanding to dementia and poor heart health:

  • Insight 46. This sub-study uses detailed brain scans, memory tests, and cardiovascular measures to identify early brain changes linked to dementia risk. Now in its 10th year, it has increased our understanding on the factors leading to dementia, including the links with the amyloid protein in the brain and dementia; the effects of shift working on the brain; and the effects of air pollution on brain health. It has also shown that a blood test may help diagnose people in the earliest stages of Alzheimer’s disease. (The test is now being trialled in centres across the UK.)
  • MyoFit 46. This sub-study focused on the heart, using cardiac imaging to investigate cardiac ageing. The study has found that even moderately elevated blood pressure in early adulthood increases later heart disease risk. The study has developed a cardiac MRI vest, which non-invasively maps the heart’s electrical activity, enabling safer diagnosis of heart rhythm problems. 

UCL is home to some of the world’s most impactful cohort studies which have shaped our understanding of health. These include the 1958 National Child Development Study, the 1970 British Cohort Study, the Millennium Cohort Study and the most recent study, Generation New Era, which is led by a team at UCL’s Centre for Longitudinal Studies and aims to recruit more than 30 000 babies born this year.

Note: UCL200 
2026 also marks a major milestone for UCL – 200 years since we were founded as the first university in London. UCL200 promises an exciting and varied programme of activities, events and storytelling, aiming to celebrate and reinforce UCL’s commitment to our founding values, highlight the excellence and impact of our groundbreaking work and people, and present an ambitious and inspiring portrait of our future. Highlights of the UCL200 programme include: a major new free exhibition – Two Centuries Here – that explores UCL’s past, present and future; a public art programme; and three specially published books about the histories of UCL, Bloomsbury and students in London.  

Source: University College London

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Scientists Find Hidden Diversity Among T. Gondii

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UC Riverside study reshapes understanding of toxoplasmosis and identifies new paths for treatment

Toxoplasma gondii. Source: Wikimedia CC0

A University of California, Riverside team of scientists has found that Toxoplasma gondii, a common parasite affecting up to one-third of the global population, is far more complex than previously believed. The findings, published in Nature Communications, offer new insight into how T. gondii causes disease and why it has been so difficult to treat.

Humans commonly contract toxoplasmosis by eating undercooked meat or through exposure to contaminated soil or cat faeces. The parasite is best known for its ability to hide in the body by forming tiny cysts in the brain and muscles. 

Most people who are infected never notice any symptoms. However, the parasite remains in the body for life as cysts, which can contain hundreds of parasites. The parasites they lodge can become active again later, however, especially in people with weakened immune systems, leading sometimes to serious problems affecting the brain or eyes. Most people who are infected never notice any symptoms. Infection during pregnancy can cause serious complications for developing babies with limited immune systems. 

Until now, scientists believed that the cysts contained a single, uniform type of parasite lying dormant until it reactivated. But using advanced single-cell analysis techniques, the UC Riverside team discovered that each cyst contains multiple distinct subtypes of parasites, each with different biological roles.

“We found the cyst is not just a quiet hiding place – it’s an active hub with different parasite types geared toward survival, spread, or reactivation,” said Emma Wilson, a professor of biomedical sciences in the UCR School of Medicine who led the study. 

Wilson explained that cysts form slowly under immune pressure and are encased in a protective wall, housing hundreds of slow-replicating parasites called bradyzoites. Although microscopic, cysts are relatively large for intracellular pathogens, reaching up to 80 microns in diameter, with each bradyzoite measuring roughly five microns in length. They reside primarily within neurons but are also commonly found in skeletal and cardiac muscle, which is important since humans are often infected by consuming undercooked meat containing these cysts.

According to Wilson, cysts are clinically and biologically significant for several reasons. They are resistant to all existing therapies and remain in the body once established. They facilitate transmission between hosts. When reactivated, bradyzoites convert into fast-replicating tachyzoites that disseminate throughout tissues, causing severe disease such as toxoplasmic encephalitis (neurological damage) or retinal toxoplasmosis (vision loss).

Image shows a cyst which can contain hundreds of T. gondii parasites. (UCR/Wilson lab)

“For decades, the Toxoplasma life cycle was understood in overly simplistic terms, conceptualised as a linear transition between tachyzoite and bradyzoite stages,” Wilson said. “Our research challenges that model. By applying single-cell RNA sequencing to parasites isolated directly from cysts in vivo, we found unexpected complexity within the cyst itself. Rather than a uniform population, cysts contain at least five distinct subtypes of bradyzoites. Although all are classified as bradyzoites, they are functionally different, with specific subsets primed for reactivation and disease.”

Wilson acknowledged that studying cysts has long been a technical challenge. They grow slowly, are embedded deep within tissues like the brain, and do not form efficiently in standard laboratory cultures. As a result, most genetic and molecular studies of Toxoplasma have focused on tachyzoites grown in vitro, leaving the biology of cyst-resident bradyzoites poorly understood. 

“Our work overcomes those limitations by using a mouse model that closely mirrors natural infection,” Wilson said. “Because mice are a natural intermediate host for Toxoplasma, their brains can harbour thousands of cysts. By isolating these cysts, digesting them enzymatically, and analysing individual parasites, we were able to gain a view of chronic infection as it occurs in living tissue.”

Wilson explained that current treatments for toxoplasmosis can control the fast-growing form of the parasite that causes acute illness, but no existing drugs can eliminate the cysts. 

“By identifying different parasite subtypes inside cysts, our study pinpoints which ones are most likely to reactivate and cause damage,” she said. “This helps explain why past drug development efforts have struggled and suggests new, more precise targets for future therapies.”

Congenital toxoplasmosis remains a major concern when primary infection occurs during pregnancy, potentially leading to severe foetal outcomes. Although prior immunity typically protects the foetus, routine screening is lacking in some countries, reflecting how difficult it is to manage an infection that is common but usually symptom-free.

Despite its prevalence, toxoplasmosis has received relatively little attention compared to other infectious diseases. Wilson hopes her team’s work will help shift that perspective.

“Our work changes how we think about the Toxoplasma cyst,” she said. “It reframes the cyst as the central control point of the parasite’s life cycle. It shows us where to aim new treatments. If we want to really treat toxoplasmosis, the cyst is the place to focus.”

Wilson was joined in the study by Arzu Ulu, Sandeep Srivastava, Nala Kachour, Brandon H. Le, and Michael W. White. Wilson and White are co-corresponding authors of the paper.

The study was supported by grants from the National Institute of Allergy and Infectious Diseases of the National Institutes of Health. 

The title of the paper is “Bradyzoite subtypes rule the crossroads of Toxoplasma development.”

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Preventing Drug Damage to the Vestibular System

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Photo by Dylann Hendricks on Unsplash

The vestibular system is responsible for the sense of balance in the inner ear. Prolonged use of toxic substances, such as certain antibiotics or anticancer drugs, can damage the hair cells that form part of this system, leading to alterations in balance and other motor skills. Now, a team from the University of Barcelona and the Bellvitge Biomedical Research Institute (IDIBELL) has identified the genetic mechanisms involved in the degradation of the vestibular system regarding the damage caused by these ototoxic compounds that affect the vestibule. The results could help improve the diagnosis of chronic vestibular ototoxicity and other pathologies related to the hair cells of the vestibular system.

The study, published in the Journal of Biomedical Science, is led by Jordi Llorens, professor at the UB’s Faculty of Medicine and Health Sciences and researcher at the Institute of Neurosciences (UBneuro) and IDIBELL. Researchers from the National Centre for Genomic Analysis (CNAG) also took part in the study.

The main causes of chronic vestibular ototoxicity are antibiotics of the aminoglycoside family, such as streptomycin – an antibiotic of choice in case of tuberculosis relapses – or anticancer drugs, such as cisplatin. Continued use of these drugs initiates a process of degeneration that causes “the hair cells to detach from the neurons, begin to deform and end up being expelled from their place in the sensory tissue,” explains Llorens.

This is a serious problem because the hair cells of the vestibular system do not regenerate. “We only have the ones we are born with. If we lose them, we also lose our balance, with very diverse consequences: from not being able to ride a bicycle to suffering blurred vision while moving, falls, orientation difficulties, dizziness or vertigo,” explains the UB professor.

Using RNA-seq analysis, i.e. a study of the global expression of genes that reveals which genes are activated or deactivated in the tissues of the vestibular system, the researchers discovered that, in the initial stages of degeneration, the hair cells change the expression of their genes to adapt to the progressive damage caused by otototoxic drugs. “The expression of many genes that define the identity of the hair cell, i.e. those that determine its shape and its ability to respond to movement by generating the signals that are sent to the brain, is reduced,” explains Llorens.

These results, together with the fact, discovered by the researchers, that the damage is reversible during the early stages of the degeneration process, indicate that it is essential to detect the problem as early as possible to stop the toxicity and avoid irreversible damage. “Hair cells become disconnected from neurons and stop sending information to the brain, but if the toxicity is interrupted, the connections can be repaired and function is restored. This increases the chances of avoiding a permanent loss of function,” the researcher stresses.

A potential biomarker

This study may also contribute to advances in the diagnosis and treatment of the pathology, since, according to the researchers, the genetic mechanisms they have identified in response to the stress caused by ototoxic drugs will make it possible, in the future, to “measure this stress and evaluate the effect of possible therapies, such as the development of drugs capable of stopping the process of eliminating hair cells or promoting their repair.”

In addition, the study has identified a new gene, Vsig10l2, expressed by hair cells, which significantly reduces its expression in all the models analysed. “This gene is of great interest as a possible marker of chronic ototoxicity in preclinical studies,” says Llorens.

The same response to different toxics

One of the most remarkable elements of the study is that the analysis has been carried out with four different models of chronic ototoxicity, using two different animal species and two different toxins, and then cross-checking the results of all experiments.

This comprehensive analysis has allowed them to determine that the degradation process occurs in response to very different toxins. “It is not a response conditioned by a particular toxin, it is the basic response of hair cells, which is always there, in response to chronic ototoxicity of any kind,” stresses the UB professor.

These results, together with the fact, discovered by the researchers, that the damage is reversible during the early stages of the degeneration process, indicate that it is essential to detect the problem as early as possible to stop the toxicity and avoid irreversible damage.

Impact on other pathologies

The study could have implications for understanding other pathologies, as the researchers suggest that the response they have demonstrated in chronic ototoxicity might represent a general response to chronic stress of any origin. “The results could be relevant to any chronic pathology with progressive loss of vestibular hair cells, including age-related loss of vestibular function. We also hypothesise that auditory hair cells might respond in a similar way, so they could help understanding deafness,” explains Llorens.

In this sense, the research team is studying – within the framework of a project funded by La Marató de TV3 – the possible relevance of the loss of vestibular function in patients with vestibular schwannoma, a tumour of the audiovestibular nerve that appears spontaneously or as a consequence of a minority disease, neurofibromatosis type 2. “Thanks to this project, we have been able to develop a culture model that allows us to study these chronic effects or how the hair cells become progressively more damaged before dying,” he concludes. 

Source: University of Barcelona

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Solving Africa’s Hidden Snakebite Problem with a New Universal Antivenom

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Photo by Nivedh P on Unsplash

In Sub-Saharan Africa, more than 300 000 people are bitten by venomous snakes annually, 3000 of whom die – but with the underreporting that goes hand in hand with the lack of healthcare infrastructure, the real number could be as much as five times higher. Many more face amputations. Even if patients manage to make it to a clinic or hospital in time, there is no guarantee that there will be any anti-venom available to treat them. As a South African case study shows, just having antivenom in the right place is a problem even in Western Cape’s relatively well-developed healthcare system, with antivenom’s three-year shelf life and cold chain failures posing a major problem for rural healthcare centres.

But now, scientists have developed a new kind of antivenom that is effective for 17 different snake species, including mambas, cobras and a rinkhals. The study, published in Nature, makes use of a nanobody-based cocktail that targets common mechanisms across venoms – and which is also more effective at preventing the tissue damage that leads to amputations.

A huge obstacle to creating broad-spectrum antivenoms is the enormous diversity of venomous snakes and the complexity of their venoms – a single species’ venom may contain 100 toxins from multiple different protein families. Listen to our podcast to hear a deep dive into Africa’s snakebite burden and how the international team of researchers accomplished their feat:

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How an Old Drug Could Help Treat Mitochondrial Diseases

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Credit: Pixabay CC0

Oxybutynin is usually prescribed for an unglamorous problem: bladder incontinence. But researchers have discovered a surprising new role for this decades-old drug – one that could open the door to treatments for a devastating class of genetic illnesses known as mitochondrial diseases.

In a paper published Sept. 8 in the American Journal of Physiology-Cell Physiology, a team of Cornell researchers described their finding that the molecule oxybutynin can overcome mitochondrial dysfunction by enhancing cellular glycolysis to improve healthy muscle formation by interacting with a suite of proteins involved in mRNA function. 

“Mitochondria are essential for our body to produce energy,” said Joeva Barrow, assistant professor of nutritional sciences in the College of Human Ecology who led the study. “If mitochondria are damaged and can no longer produce energy, the cells die, the tissues die and, eventually, the person dies.”

Mitochondrial diseases affect about one in every 5000 people and a large proportion of them are children, Barrow said. Patients often experience profound muscle weakness, neurological decline, heart problems and, in the most severe cases, shortened lives. There are no cures and virtually no effective treatments.

“Our approach was to test a series of small molecules that have never been used to treat mitochondrial disease before,” Barrow said. “Previous attempts at small molecules therapy were unsuccessful because of the use of artificial cell systems, but our plan was to use these molecules directly at the source – the muscle stem cells themselves.”

After running a screen of thousands of small molecules, they saw oxybutynin emerge as a clear frontrunner. They found that oxybutynin treatment can help muscle stem cells overcome one of the most severe forms of the condition, Complex III mitochondrial dysfunction. Normally, cells rely on mitochondria to generate ATP, the molecule that powers nearly every biological process. In Complex III disorders, that system grinds down, leaving cells starved.

The researchers tested oxybutynin on mouse and human muscle stem cells, the cells responsible for repairing and growing new muscle. These cells, normally stunted by the disease, began multiplying and forming muscle fibers again when treated with the drug.

The effect didn’t come from fixing the broken mitochondria. Instead, oxybutynin rewires the cellular energetic pathways to perform glycolysis: the quick-burning process of breaking down glucose. That backup system provided just enough energy to revive growth.

Using a high-tech small molecule binding protein analysis method, the team discovered that oxybutynin binds to proteins involved in RNA processing – the machinery that fine-tunes how cells interpret their genetic code. That interaction set off a cascade of changes, including a boost in amino acid and glucose transport into the cells.

In other words, the drug seems to rewire how diseased muscle cells fuel themselves, finding clever ways to survive without fully functioning mitochondria.

The results held true not only for mouse stem cells but also for human ones. Treated muscle stem cells grew stronger, produced more muscle fibres and maintained higher energy levels than untreated controls.

“Translating these findings to children with mitochondrial disease is happening in real time at the Children’s Hospital of Philadelphia with collaboration with Dr Marni Falk,” Barrow said. Dr Marni Falk, is the executive director of the Mitochondrial Medicine Frontier Program at the Children’s Hospital of Philadelphia. “Their team performs biopsies with kids with mitochondrial diseases, and they are currently testing oxybutynin with those cells.”

While this is still far from a clinical therapy – no human patients have yet received oxybutynin for mitochondrial disease – the findings raise hopes that an old, inexpensive drug might be repurposed for a devastating illness. “Oxybutynin already has FDA approval for treatment of bladder disorders” she said. 

For families facing mitochondrial disease, even small advances can be a lifeline. Most patients today rely only on supportive care, managing symptoms without any way to slow or reverse the disease.

If further studies confirm its benefits, oxybutynin could speed its way into trials, bypassing years of costly development, Barrow said.

Source: Cornell University

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South African Hunters Chewed the Kanna Plant for Endurance: New Study Tests its Effects on Mouse Brain Chemistry

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Sceletium tortuosum – Kougoed. Source: Wikimedia Commons.

Catherine H Kaschula, Stellenbosch University

Sceletium tortuosum is a little succulent plant that grows in the semi-arid Karoo and Namaqualand regions of South Africa. It has a long history of traditional use among the hunter-gatherers of the region.

The plant, known as kanna or kougoed by the San and Khoikhoi people, was mainly chewed or smoked to stay alert and suppress appetite during long hunts. The San were traditionally hunter-gatherers, while the Khoikhoi were pastoralists who herded livestock.

The name kanna (meaning “eland” in the click language of the San), has a symbolic reference to this large antelope, as the “trance animal”, which was called upon during religious and spiritual gatherings. Kougoed is Afrikaans for “something to chew”. The plant can be chewed after being dried and fermented, which is believed to intensify its effects.

The first colonial governor of the Cape colony, Simon van der Stel, in 1685 wrote about kanna in his journal:

They chew mostly a certain plant which they call Canna and which they bruise, roots as well as the stem, between the stones and store and preserve in sewn-up sheepskins.

I’m part of a group of scientists from different disciplines with an interest in this plant and we pooled our expertise to understand its effects on neurochemical concentrations in different parts of the brain.

Our studies were done in mice, so there is caution about establishing effectiveness on humans. Still, the results are striking.

As a chemist with an interest in natural products, I wanted to know which alkaloids in the plant were important in bringing about these effects.

Our latest study explored the effects of Sceletium tortuosum extracts on mouse brain chemistry.

We found that Sceletium increased the levels of certain brain chemicals which may balance mood and reduce stress. These findings lend support to the calming and mood-enhancing use of this plant in traditional medicine.

Plant chemistry

Our study examined how extracts from different chemotypes of Sceletium tortuosum can have different effects on brain chemistry. Chemotypes are groups of the same plant species that differ in the alkaloids they produce. This is because plants often produce alkaloids in response to external cues such as the weather or the presence of a plant-eating animal or pathogen.

Alkaloids are carbon-based compounds produced by plants. They are often toxic or taste bitter, making the plants less appealing or even harmful to the predators or invaders that want to eat or inhabit them. Alkaloids generally have physiological effects of use to humans. Some commonly used ones include caffeine, morphine and quinine.

We harvested two chemotypes of kanna from the Touwsrivier and De Rust regions of South Africa. These areas were chosen because of their interesting and unusual alkaloid profiles. The chemotypes were given to healthy mice as a supplement once a day for one month. The mice were monitored every day for behavioural or unexpected adverse reactions but none were noted.

At the end of the month, the levels of chemicals in the mouse brain were measured. Both the chemotypes were found to cause a marked increase in noradrenaline and a decrease in GABA in all brain regions studied. Both molecules are neurotransmitters that transmit nerve signals in the brain affecting memory, mood, attention and sleep.

This effect on noradrenaline supports kanna’s traditional use as an appetite suppressing drug. Increased noradrenergic stimulation is also the basis of many anti-depressants as well as drugs that improve attention and alertness.

We also found an impact on the brain chemicals serotonin and dopamine which may act together to balance mood and reduce stress. Serotonin affects emotional well-being and mood; dopamine motivates feelings of pleasure and satisfaction. These findings lend support to the calming and mood-enhancing use of this plant in traditional medicine.

Importantly, the control kanna extracts that did not have the interesting alkaloid profiles did not cause any of these chemical changes in the mouse brain.

Most studies on kanna have focused on the alkaloid mesembrine. The two specific chemotypes of kanna harvested from the Touwsrivier and De Rust regions of South Africa do have the mesembrine, but they are also packed with some other lesser-known or “minor” alkaloids. These differences in alkaloids may arise from a combination of geographic, environmental and inherent genetic factors found in a particular subset of plants.

Both the Touwsrivier and De Rust plants contained higher levels of alkaloids called mesembrine alcohols, which are different from mesembrine, and were barely present in the control extract. Another minor alkaloid, known as sceletium A4, was also identified as possibly being important. Mesembrine alcohols and sceletium A4 may be the ones responsible for the activity.

This suggests that the source of the plant, and the area in which it is grown, can influence its potential as a natural treatment for mood disorders and sleep.

What the results tell us

Stress, anxiety and depression pose a risk to the ability to lead a meaningful life. The World Health Organization has reported a 25% increase in anxiety and depression worldwide since the emergence of COVID-19.

Our study showed that the plant extracts had a broad noradrenergic effect in mice. But we have to be careful about making connections between results in mice and in humans. We need to explore the behavioural impact of these extracts in both mice and humans, especially in relation to sleep, alertness and mood.

The results also highlighted that without understanding the complex chemical composition of these plants, we risk overgeneralising their benefits, or worse, using them inappropriately.

Our findings have two implications.

First, they point towards a future of precision phytotherapy (use of plants for medicinal purposes), where natural remedies are tailored not just to individuals but to selecting certain plant chemotypes that produce certain combinations of alkaloids. Manipulating the growing conditions and genetic make-up of plants to optimise for alkaloid content is an age-old art.

Second, they remind us of the enormous, still largely untapped potential of African medicinal plants in global health innovation if we invest in research that honours both indigenous knowledge and scientific rigour.

As the world searches for safer, more sustainable ways to treat mental health conditions, South Africa’s kanna plant may hold secrets worth rediscovering.

Catherine H Kaschula, Senior Lecturer, Stellenbosch University

This article is republished from The Conversation under a Creative Commons license. Read the original article.

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Research Findings Offer New Insight into Heparin and Bone Builders

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Photo by Mufid Majnun on Unsplash

The blood thinner heparin is used during and after surgery and is essential to kidney dialysis. Most of today’s heparin comes from pigs, but the Federal Drug Administration is encouraging the use of alternative sources, including cows and synthetic forms of heparin, to diversify the supply chain.

Unfortunately, heparin from animals other than pigs just doesn’t work as well.

The reasons are connected to ongoing questions in modern cell biology. Now, an interdisciplinary Virginia Tech team has uncovered new molecular clues that may explain why some sources of heparin are more effective than others. The findings, published recently in the Proceedings of the National Academy of Sciences, may open doors for designing safer, more reliable heparin therapies.

“The structure of heparin and how that structure impacts function is an ongoing puzzle,” said Brenna Knight, first author of the study and recent graduate student studying in the Department of Chemistry. “Seemingly small differences in the content and arrangement of [chemical entities called] sulfates on the molecule cause substantial differences in the energetics that drive chemical activity.”

From mineralization to medicine

Heparin hails from a family called heparan sulfates, or heparans, present in all living creatures. These chains of sugars are diverse, serve many functions in organisms, and many, including heparin, are incredibly complex.

As a student of Patricia Dove in the Departments of Geosciences and Chemistry, Knight was originally looking at heparans for a completely different reason: to understand how the sulfates could impact biological mineralisation, which is the process by which organisms build crystal-strengthened tissues such as bones, teeth, shells, and corals.

Dove is one of today’s pre-eminent geochemists and was elected to the National Academy of Sciences in 2012. Unravelling the process of biomineralisation has been one of her major passions over the past three decades.  

“Animals grow crystals in specific places, usually to make structures that serve to support, defend, or feed themselves.” said Dove. “It’s a coordinated result of many chemical reactions within the organism and a crowning achievement of biology. We’ve been trying to better understand the reactions that produce these working biomaterials for a long time.”

That mineralization process unexpectedly linked back to medicine.

Heparan sulfates are just one of many different agents that interact with calcium to trigger a diverse portfolio of biochemical operations. One of those operations is integral to blood clotting.

Team science

To better understand how heparan sulfates help facilitate biomineralisation, Dove and Knight teamed with Kevin Edgar, professor in the Department of Sustainable Biomaterials, who was interested in heparans from the healthcare angle. To study interactions of calcium with heparin, they worked with Michael Schulz and graduate student Connor Gallagher in the chemistry department.  

When they applied their combined expertise to calcium-heparin interactions, they found that slight variations in heparin’s molecular composition changed how effective it was at binding calcium. These differences could affect its ability to form biominerals and blood thinners.  

“This paper provides insights for how to bioengineer synthetic pathways to effective heparin products for applications in therapeutics and drug delivery,” Edgar said.

Source: Virginia Tech

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Synthetic Torpor has the Potential to Redefine Medicine

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A team of researchers at Washington University in St. Louis is in pursuit of translating induced, or synthetic, torpor into potential solutions for humans, such as when there is reduced blood flow to tissues or organs, to preserve organs for transplantation or to protect from radiation during space travel. (Credit: Chen lab)

Nature is often the best model for science. For nearly a century, scientists have been trying to recreate the ability of some mammals and birds to survive extreme environmental conditions for brief or extended periods by going into torpor, when their body temperature and metabolic rate drop, allowing them to preserve energy and heat.

Taking inspiration from nature, Hong Chen, professor of biomedical engineering in the McKelvey School of Engineering and of neurosurgery at WashU Medicine, and an interdisciplinary team induced a reversible torpor-like state in mice by using focused ultrasound to stimulate the hypothalamus preoptic area in the brain, which helps to regulate body temperature and metabolism. In addition to the mouse, which naturally goes into torpor, Chen and her team induced torpor in a rat, which does not. Their findings, published in 2023 in Nature Metabolism, showed the first noninvasive and safe method to induce a torpor-like state by targeting the central nervous system.

Now, the team is in pursuit of translating induced, or synthetic, torpor into potential solutions for humans, such as when there is reduced blood flow to tissues or organs, to preserve organs for transplantation or to protect from radiation during space travel.

Conventional medical interventions focus on increasing energy supply, such as restoring blood flow to the brain after a stroke. Synthetic torpor seeks to do the opposite by reducing energy demand.

“The capability of synthetic torpor to regulate whole-body metabolism promises to transform medicine by offering novel strategies for medical interventions,” said Chen in a Perspectives paper published in Nature Metabolism July 31, 2025. 

Synthetic torpor has been used successfully in preclinical models with medications and specialised targeting of the neural circuit, but there are challenges to adapting these methods for humans. Previous human trials with hydrogen sulfide were terminated early due to safety concerns.

“Our challenges include overcoming metabolic differences among animals and humans, choosing the correct dose of medication and creating ways to allow a reversible torpor-like state,” said Wenbo Wu, a biomedical engineering doctoral student in Chen’s lab and first author of the Perspectives paper, a collaboration between Chen’s team and Genshiro Sunagawa from the RIKEN Center for Biosystems Dynamics Research in Japan. “Collaboration among scientists, clinicians and ethicists will be critical to develop safe, effective and scalable solutions for synthetic torpor to become a practical solution in medicine.”

Chen’s team, including Yaoheng (Mack) Yang, who was a postdoctoral research associate in her lab and is now assistant professor of biomedical engineering at the University of Southern California, targeted the neural circuit with their induced torpor solution in mice. They created a wearable ultrasound transducer to stimulate the neurons in the hypothalamus preoptic area. When stimulated, the mice showed a drop in body temperature of about 3 degrees C for about one hour. In addition, the mice’s metabolism showed a change from using both carbohydrates and fat for energy to only fat, a key feature of torpor, and their heart rates fell by about 47%, all while at room temperature.

“Ultrasound is the only noninvasive energy modality capable of safely penetrating the skull and precisely targeting deep brain structures,” Chen said. “While ultrasound neuromodulation lacks cell-type specificity compared with genetic-based neuromodulation, it provides a noninvasive alternative for inducing synthetic torpor without the need for genetic modifications.”

Chen and her team indicate that synthetic torpor offers a promising therapeutic strategy with additional applications, including inhibiting tumour growth and potential development of new therapies for tau protein related diseases, such as Alzheimer’s disease. However, much remains unknown about how brain regions, peripheral organs and cellular pathways coordinate metabolic suppression and arousal. Researchers also need to study the long-term risks and potential side effects and call for more preclinical studies and technological innovations that will facilitate a dual approach, which would include modulating neural circuits associated with hypometabolism and influencing peripheral metabolic pathways through systemic interventions, such as with drugs or peripheral neuromodulation.

“Synthetic torpor is no longer just a theoretical concept – it is an emerging field with the potential to redefine medicine,” Chen said. “Bridging fundamental neuroscience, bioengineering and translational medicine will be key to overcoming current challenges and advancing synthetic torpor toward real-world applications. Synthetic torpor could transition from a scientific curiosity to a human reality through interdisciplinary collaborations.”

Source: Washington University McKelvey School of Engineering