Category: Diseases, Syndromes and Conditions

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Kaitlin and Lihle’s Fight Against a Rare Blood Disease

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Photo by National Cancer Institute on Unsplash

At 25, Kaitlin should be living independently. At 18, Lihle should be finishing school. Instead, both are fighting for their lives against aplastic anaemia (AA), a rare blood disease that leaves patients vulnerable to infections, uncontrolled bleeding, and severe anaemia. A stem cell transplant gives approximately 80% of patients a real chance at recovery, but for around 70% of those patients, that match will not come from within their family. It will come from a generous stranger.

“AA strikes hardest between 15 and 25 – the years nobody expects to spend fighting for their life,” says Palesa Mokomele, Head of Community Engagement and Communication at DKMS Africa. “We want South Africans to understand that registering as a stem cell donor is a simple act that could give someone like Kaitlin or Lihle their life back. Every person who registers increases their chances of finding a match.”

A long road to the right diagnosis: Kaitlin’s story

For years, nobody could tell Kaitlin from KwaZulu-Natal what was wrong. She experienced prolonged and excessive bleeding and severe fatigue, which was repeatedly misattributed to gynaecological issues. She kept going back to the hospital and kept being sent home. It was only in August 2025, when her condition deteriorated dramatically, and the bleeding would not stop despite ongoing treatment, that she was finally referred to a haematologist. A bone marrow biopsy told them what years of tests had missed: Kaitlin had AA.

Before this, she was working full-time and living independently. Today, she cannot work. She cannot manage basic daily tasks. She requires weekly blood transfusions simply to stay alive. Medication trials have yielded no response, and her doctors have been clear: a stem cell transplant is her only path to recovery.

Through it all, Kaitlin has held on. “I draw strength from my faith and from the people I love most – my nephews and siblings, who show up for me even on the hardest hospital days. I just want my life back, and a matching donor could make that possible.”

Sudden illness, endless resilience: Lihle’s story

Lihle was 14 years old when his life changed overnight. It started with severe nosebleeds in November 2021. Then one night, the bleeding became uncontrollable. He lost consciousness. After two months in hospital, the diagnosis came: Severe Aplastic Anaemia (SAA). That same year, his father passed away.

The eldest of four children, Lihle grew up fast. Hailing from Butterworth in the Eastern Cape and raised in Carletonville, Gauteng, he has always felt the weight of being the firstborn – the one his younger siblings look up to. Their mother cares for them all – while also carrying the emotional weight of losing her husband and watching her son fight for his life.

Lihle shares that he is determined to finish his education, set an example, and one day return to the football pitch. Like Kaitlin, all he needs is a matching donor to make that possible.”

How you can help

“No family should have to face what Kaitlin’s and Lihle’s are going through – knowing that a cure exists, but that the donor hasn’t been found yet. For patients from Black, Coloured and Indian/Asian backgrounds, that search is even harder, because the registry does not yet reflect the diversity of our population. We are calling on all South Africans to register. It costs nothing. It takes minutes. And it could mean everything,” concludes Mokomele.

Signing up could be the most important thing you ever do. If you are aged 17 – 55 and in good health, please register today at: https://www.dkms-africa.org/save-lives

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Key International Mpox Trial Finds No Clinical Benefit from Tecovirimat 

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Mpox (monkeypox) virus. Source: NIH

An international, randomised, double‑blind, placebo‑controlled phase 3 study, the largest of its kind for mpox, found that tecovirimat did not improve clinical outcomes for adults with clade II mpox compared with placebo, while demonstrating a similar safety profile. Results of the STOMP/A5418 trial, published in the New England Journal of Medicine underscore both the urgent need for alternative therapeutics and the critical importance of randomised trials during public health emergencies.  

This Phase 3 study randomised 412 participants (344 with laboratory‑confirmed mpox), to receive either tecovirimat or a matching placebo for 14 days. Randomisation was stratified by early versus later symptom onset and by the presence of severe pain. Participants had active skin or mucosal lesions and self‑reported daily symptoms, pain scores, and lesion status through Day 29, with confirmatory clinical assessments at scheduled visits. Biospecimens, including lesion swabs, oral and rectal swabs, and blood samples, were collected at multiple time points to assess viral DNA clearance. The primary endpoint was time to clinical resolution of all lesions, and key secondary endpoints included pain reduction, complete lesion healing, and virologic response.  

Conducted across seven countries at 49 sites, the phase 3 study showed that tecovirimat did not shorten the time to lesion resolution, reduce pain, or speed viral clearance compared with placebo. These results align with interim findings released in December 2024, which led the trial’s independent Data and Safety Monitoring Board to halt further enrolment due to statistical futility.  

“In the midst of a global public health emergency, the ACTG team rapidly conducted this randomized controlled trial to deliver a clear answer for patients and clinicians,” said William A. Fischer II, MD, associate professor of pulmonary and critical care medicine at the UNC School of Medicine, and director of emerging pathogens research at the UNC Institute for Global Health and Infectious Diseases. “These findings advance our understanding of mpox and help the field refocus efforts on identifying safe, effective and accessible treatment strategies, particularly for people at highest risk of severe disease.” 

Although the trial did not demonstrate efficacy, tecovirimat demonstrated a favourable safety profile, with no major safety concerns identified – an important confirmation as thousands of patients worldwide have already received the drug under expanded access protocols.  

“The STOMP trial provides essential evidence at a critical time and demonstrates why randomized controlled trials are an indispensable part of outbreak response,” said Joe Eron, MD, chief of infectious diseases and chair of the ACTG network. “But now we must keep going to find safe and effective treatment for people as this virus continues to circulate globally.”  

The study’s conclusions are expected to influence clinical practice and public health guidance worldwide. With mpox still causing outbreaks in multiple regions, researchers emphasise that developing and evaluating new antiviral candidates remains a top priority.

Source:

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Ivermectin Was Touted as a Cure for COVID, Now it’s Being Tested for Cancer. But what can it Actually Treat?

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Photo by Halgatewood.com on Unsplash

Nial Wheate, Macquarie University

Ivermectin was originally celebrated as a revolutionary treatment for parasitic disease in humans and animals. It has since evolved into a focal point of misinformation and heated debate.

During the early part of the COVID pandemic, it was touted on social media as a miracle cure for the virus, despite a lack of robust evidence.

Now the United States National Cancer Institute is looking into the drug as a potential cancer treatment, with early human clinical studies underway.

But what can it successfully treat?

What is ivermectin?

The drug is a small organic chemical that can be extracted from the bacterium Streptomyces avermitilis. This bacterium grows in the soil, and was first found near the grounds of a Japanese golf course.

Ivermectin’s discovery in the 1970s was considered so important its discoverers were awarded the 2015 Nobel Prize in Physiology or Medicine.

It was first approved for use in animals in 1981 and in humans in 1987. It’s now available in various brands as tablets and creams you apply to the skin.

Assessing the evidence

Governments use human clinical trials to decide whether to approve a medicine for sale.

But clinical trials aren’t the highest level of evidence to inform best practice and guide decisions. For that, there are Cochrane reviews.

A Cochrane review brings together a panel of experts who collate and assess all the relevant evidence on a medication. It takes data from multiple clinical trials, and other studies, and evaluates it following clear and structured steps. It’s able to examine and critique study designs to identify bias and reject bad data.

Cochrane reviews are also regularly updated to take into account new information. The result is a summary that is considered the highest level of evidence to guide decision-making.

So what do Cochrane reviews say about ivermectin for different conditions?

What can and can’t ivermectin treat?

ConditionDoes it work?Notes
CancerUnclearStudies only just starting
COVIDNoDoes not prevent infection or treat
Gut and lymphatic wormsYesTreatment for various roundworms
MalariaUnclearNot enough evidence to decide
River blindnessUnclearNot enough evidence to decide
RosaceaYesUse the topical formulation
ScabiesYes, but with caveatsNot the most effective
Table: Nial Wheate Source: Cochrane reviews – variousGet the dataCreated with Datawrapper

Gut and lymphatic worms

Ivermectin is used to treat a variety of parasitic worm infections. These include the round worms Ascaris lumbricoides, Strongyloides stercoralis, Wuchereria bancrofti, and Brugia malayi.

The latter two worms cause the disease lymphatic filariasis (or elephantiasis) which causes severe swelling in the arms, legs, breasts and genitals.

When ivermectin is used to treat Strongyloides stercoralis, the Cochrane panel found it is better than albendazole and had fewer side effects than thiabendazole.

For Ascaris lumbricoides, the panel concluded ivermectin was as good as albendazole and mebendazole.

For treating lymphatic filariasis, a Cochrane review found ivermectin or diethylcarbamazine should be standard treatment in combination with albendazole.

Rosacea

The Cochrane review for rosacea evaluated 22 different treatments for this skin condition, including a variety of drugs, as well as light therapy, cosmetics and reducing the intake of spicy food.

It concluded that ivermectin applied to the skin was more effective than a placebo, and a bit better than the other standard medication, metronidazole.

Scabies

Cochrane has two reviews on the use of ivermectin for scabies. One specifically evaluated ivermectin and permethrin as treatments. The other evaluated all available treatments for scabies.

The first review concluded both permethrin and ivermectin were just as effective, regardless of whether the ivermectin was administered orally or directly onto the skin.

In contrast, the second review concluded ivermectin does work but topical permethrin appeared to be the most effective treatment.

Malaria

The Cochrane panel looked specifically at whether ivermectin could reduce transmission of the malaria parasite, rather than as a treatment.

Unfortunately there was just a single clinical trial to use as evidence. In that trial, residents of eight villages were given ivermectin and albendazole together, with follow up doses of just ivermectin. The researchers then looked at the rates of child infection over 18 weeks.

Even though the trial didn’t show ivermectin prevented infection, due to the high risk of bias in it, the Cochrane panel couldn’t conclude either way whether ivermectin worked or not.

River blindness

River blindness is caused by another parasitic worm called Onchocerca volvulus.

The Cochrane review concluded there was a lack of evidence either way to know whether it works to prevent infection-based visual impairment and blindness.

It evaluated the data from four clinical trials and two large community-based studies.

One of the reasons the panel was unable to make a firm conclusion was because it thought the drug may work differently against different strains of the parasite and in people of different ethnicity.

Cancer

There are no Cochrane reviews on ivermectin’s use for cancer because clinical interest in the drug for this condition is just starting.

There is a current clinical trial that is evaluating ivermectin in combination with antibody-based drugs for breast cancer.

Early results showed the combination of antibody drugs with ivermectin was safe to patients, but no efficacy data has been published.

COVID

The Cochrane panel rejected the data for seven clinical trials and included 11 other trials. Rejected trials included those which compared ivermectin against other drugs which were known to not be effective against COVID, such as hydroxychloroquine.

The review concluded there was no evidence to support the use of ivermectin for the treatment or prevention of COVID. In making that conclusion, it evaluated treatments that used invermectin or placebo in combination with standard care and whether treatment reduced death, illness, or the length of the infection.

Nial Wheate, Professor, School of Natural Sciences, Macquarie University

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

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Repurposed Cancer Drug may Aid Recovery from Severe Malaria

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Red Blood Cell Infected with Malaria Parasites
Colourised scanning electron micrograph of red blood cell infected with malaria parasites (teal). The small bumps on the infected cell show how the parasite remodels its host cell by forming protrusions called ‘knobs’ on the surface, enabling it to avoid destruction and cause inflammation. Uninfected cells (red) have smoother surfaces. Credit: NIAID

A new clinical trial led by QIMR Berghofer, in collaboration with University of Sunshine Coast Clinical Trials Network has found a medication currently used for some blood disorders could help the body fight malaria more effectively.

The findings mean the drug, ruxolitinib, could potentially be used alongside standard treatment to boost recovery and strengthen people’s immune systems against future infections.

Malaria kills more than 600 000 people each year and three quarters of those deaths are in children under the age of five.

Current treatments for malaria work by killing the parasite that causes most malaria deaths, Plasmodium falciparum. However, even with these treatments, fatality rates from severe malaria remain high.

Furthermore, while patients develop some immunity after infection, this protection is often incomplete, leaving many vulnerable to reinfection.

Head of QIMR Berghofer’s Clinical Malaria Group Associate Professor Bridget Barber says the research overcomes a key hurdle.

“While antimalarial treatments are effective at killing the parasite, they don’t directly address the inflammation that contributes to severe illness and death. These findings suggest that we may be able to improve clinical outcomes by targeting the host inflammatory response as well as the parasite itself,” she said.

The research, published in Science Translational Medicine, looked at how the immune system responds to malaria via the body’s ‘early warning system’ known as type 1 interferon signalling.

To do this, researchers enrolled 20 healthy adult volunteers who had never been exposed to malaria. Participants were deliberately infected with Plasmodium falciparum under closely monitored conditions. Eight days later, all participants received standard malaria treatment (artemether-lumefantrine), while 11 were also given ruxolitinib. Three months later, participants were re-infected with malaria to test how their immune systems responded to a second infection.

The research revealed ruxolitinib was safe and well-tolerated, compared with the placebo group, and participants who received ruxolitinib showed a lower inflammatory response, and favourable changes in markers linked to disease severity.

QIMR Berghofer’s Program Director of Infection and Inflammation Professor Christian Engwerda says the results are encouraging.

“One of the biggest challenges in efforts to eliminate malaria is the limited efficacy and duration of protection provided by current vaccines. By boosting the immune system without causing detrimental inflammation with drugs like ruxolitinib, we may be able to overcome these challenges,” he said.

The researchers say it’s important to note that the study was conducted in healthy volunteers who did not live in malaria-endemic regions. Further studies in malaria-endemic regions will be needed to determine whether these findings translate into improved outcomes for patients most affected by the disease.

Read the scientific paper here: www.science.org/doi/10.1126/scitranslmed.aea2531

Source: QIMR Berghofer Medical Research Institute

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New Antivirals Could Help Prevent HSV-1 by Changing Cell Structures

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Lab tests confirm that antiviral class known as Pin1 inhibitors could reduce and stop outbreaks of herpes simplex virus-1

Photo by Cottonbro on Pexels

A class of antivirals called Pin1 inhibitors could reduce or stop outbreaks of herpes simplex virus 1 (HSV-1), the common infection behind oral herpes, according to new research published in Antiviral Research.

HSV-1 causes sores around the mouth, commonly called cold sores or fever blisters. Most people are infected with HSV-1 in childhood, and between 50% and 90% of people worldwide have HSV-1. After the initial infection, HSV-1 remains in the body and can reactivate throughout a person’s life. While HSV-1 infections are usually mild, they can be serious and even deadly for people with suppressed immune systems. Finding new, more effective antivirals for this common illness is essential. 

Pin1 inhibitors suppress HSV-1 replication by inhibiting viral protein synthesis and preventing nucleocapsid egress from the nucleus. (Takemasa Sakaguchi/Hiroshima University)

Researchers focused on an enzyme called peptidyl-prolyl cis-trans isomerase NIMA-interacting 1, or Pin1, that regulates protein stability, function, and cellular structure. When this enzyme is dysregulated, it can play a role in a variety of conditions, including obesity, cancer, heart failure, and more. Viruses, such as cytomegalovirus (CMV) and SARS-CoV-2, are known to affect Pin1, and Pin1 inhibitors have been developed to reduce the impact of these viruses. 

Because HSV-infected cells over-express Pin1, researchers wanted to know if Pin1 inhibitors could also be used to treat HSV-1. “This study revealed that the host factor Pin1 is a crucial therapeutic target for the proliferation of HSV-1. Pin1 inhibitors potently suppress HSV-1 replication at low concentrations,” said Takemasa Sakaguchi, a professor at the Graduate School of Biomedical and Health Sciences at Hiroshima University in Hiroshima, Japan. 

In laboratory tests, the Pin1 inhibitor H-77 and the four newly developed Pin1 inhibitors successfully stopped the replication of HSV-1. VeroE6 cells, derived from the kidney of an African green monkey and commonly used in virology research, were infected with HSV-1 and cultured in the presence of different amounts of a Pin1 inhibitor. As the amount of the inhibitor increased, the effects of HSV-1 on the cells became less pronounced and completely disappeared at 1 μM. They also found that any viral particles released from the treated cells were non-infectious. 

The most important finding is how Pin1 inhibitors affect cell structures to prevent the virus from escaping. They do this by stabilising nuclear membrane structure, physically trapping the virus in the cell nucleus. “The nuclear lamina initially functions as a ‘barrier’ when nucleocapsids of progeny viruses, that replicate within the nucleus, bud from the nuclear membrane. Pin1 overexpressed by the virus removes this barrier. However, through the action of the Pin1 inhibitor H-77, this barrier is rather reinforced, forming a thick and robust lamina layer. This demonstrates that H-77 transforms the nuclear lamina into an ‘impregnable defensive wall,’ physically blocking the escape of viruses from the nucleus of the cell,” said Sakaguchi.

Looking ahead, researchers will continue to evaluate the effectiveness of Pin1 inhibitors to treat HSV-1. They will also research how Pin1 inhibitors could be used to treat other viruses. “The ultimate goal for the future is to aim for the clinical application of Pin1 inhibitors as ‘host-directed therapeutics,’ which are less likely to cause drug resistance. To achieve this, we will first evaluate their efficacy against diverse viruses to clarify the treatable range. Simultaneously, research to optimise the compound structure is essential for creating more potent and selective drugs,” said Sakaguchi. 

Source: University of Hiroshima

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Bubonic Plague Treatment Proven Highly Effective and Safe in Global First

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Scanning electron micrograph of Yersinia pestis, which causes bubonic plague, on proventricular spines of a Xenopsylla cheopis flea. Credit: NIAID

Researchers from the UK and Madagascar, in collaboration with Madagascar’s health services and national plague programme, have conducted the world’s first rigorous clinical trial of treatments for bubonic plague.

The IMASOY trial provides the first robust evidence of the efficacy and safety of two treatment regimens. They also found that a ten-day course of an oral antibiotic (ciprofloxacin) is a highly effective and safe alternative to existing treatment with injectable antibiotic requiring hospitalisation.

Plague has scourged humanity for millennia. Though cases have been declining, it remains a high-threat pathogen of pandemic potential, due to its widespread animal reservoir and potential for being weaponised via deliberate release. Most continents see sporadic cases of bubonic plague every year, with Madagascar reporting approximately 80% of global cases. Bubonic plague is a life-threatening disease with mortality ranging from 15-25%.

The results from the trial, published in the New England Journal of Medicine, are the outcome of a hugely ambitious clinical trial conducted over the last five years in rural Madagascar.

With many cases occurring in remote villages and with outbreaks being unpredictable, the team deployed dozens of research assistants, and trained over 230 local doctors and nurses and 1300 village health workers. The trial was embedded within Madagascar’s national health service and was conducted with the support of the Ministry of Public Health.

Several treatment options are included in the current international and national guidelines, but none of them have been rigorously tested in humans. Regulatory approval has been based solely on data from animal studies and human safety data.

Researchers designed the IMASOY trial to test two treatments for plague. Patients were randomly allocated to receive either a ten-day oral regimen of ciprofloxacin (an antibiotic tablet which can be taken at home), or a regimen requiring three days’ injectable gentamicin followed by seven days’ ciprofloxacin, (with the intravenous (IV) therapy requiring patients to be hospitalised).

450 patients with clinically suspected bubonic plague were enrolled between 2020-2024 at 47 sites in 11 districts in Madagascar and treated with either regimen. Of these, 222 plague infections were confirmed by laboratory testing.

Key findings:
•    Both regimens were found to be highly effective and safe, with overall treatment success rates of about 90% and an overall mortality rate of about 4%.
•    A ten-day ciprofloxacin regimen has many advantages over a regimen requiring three days of injectable antibiotics. It alleviates bed space (particularly in health centres with minimal bed availability for the surrounding villages), reduces healthcare worker workload, and is much cheaper – costing about one-tenth of the current treatment regimen, depending on the route of administration.

With funding from Wellcome and the UK Foreign, Commonwealth, and Development Office, researchers from the University of Oxford, Institut Pasteur de Madagascar, Centre Hospitalier Universitaire Joseph Raseta Befelatanana, Centre d’Infectiologie Charles Mérieux, London School of Hygiene and Tropical Medicine, along with Madagascar’s health services and national plague programme, collaborated through the International Severe Acute Respiratory and emerging Infection Consortium (ISARIC) to fill this gap in knowledge.

Piero Olliaro, Professor at the Pandemic Science Institute, University of Oxford and senior author of the study said: ‘Despite its deadly history, we’ve had little clinical evidence on treating bubonic plague – until now. Thanks to the patients and healthcare workers in the trial, we now have real-world proof of effective, safe treatment. Ongoing data analysis will deepen our understanding of the disease, including risk factors, symptoms, and diagnostics.’

Mihaja Raberahona, physician at CHU Joseph Raseta Befelatanana and researcher at the Centre d’Infectiologie Charles Mérieux Madagascar said: ‘In Madagascar, where plague cases occur in remote rural locations with limited healthcare infrastructure, taking a straightforward oral antibiotic is vastly preferable to a treatment requiring injections. It alleviates healthcare worker workload and is also much cheaper – costing about one-tenth of the current treatment regimen.’

Rindra Vatosoa Randremanana, Medical epidemiologist at Institut Pasteur de Madagascar said: ‘The trial was a massive operational undertaking. Many patients were in remote villages and the unpredictable nature of the outbreaks made the research very challenging. Recruiting the 450 patients, of whom about half were confirmed with bubonic plague, took five plague transmission seasons and an army of research assistants, doctors and community health workers. We are hugely grateful to everyone who took part.’

The impact of this study is far-reaching. As a result of the trial, the researchers will be working with national and international bodies like the World Health Organization (WHO) to provide the evidence that may be required to update clinical guidelines, thereby translating the results into practice and saving lives.

The full paper, ‘Ciprofloxacin versus Aminoglycoside-Ciprofloxacin for Bubonic Plague‘, is published in the New England Journal of Medicine.

Source: University of Oxford

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South Africa Marked World Hepatitis Day with a Call to Eliminate Viral Hepatitis by 2030

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Hepatitis C virus. Credit: Scientific Animations CC4.0

On the 28th of July, South Africa joined the global community in marking World Hepatitis Day 2025, which is observed annually to raise awareness of viral hepatitis and to call for urgent action to eliminate it as a public health threat.

Under the theme “Let’s Break It Down,” this year’s campaign urged governments, healthcare systems, and communities to dismantle the financial, social, and systemic barriers that hinder progress—particularly stigma, underdiagnosis, and lack of access to testing and treatment.1

More than 304 million people globally are living with chronic hepatitis B or C, yet the majority remain undiagnosed until it is too late. In South Africa alone, over one million new cases are reported each year—despite the fact that hepatitis B is vaccine-preventable and hepatitis C is curable with available therapies. 1,2

Dr Neliswa Gogela, hepatologist, commented: “Hepatitis B and C are silent killers. People often do not know they’re infected until severe liver damage or cancer develops. But this is a crisis we can stop. We have vaccines, we have treatment, and we have the tools – we simply need to scale up access, embed hepatitis care into our health system, and break the stigma so people are not afraid to get tested or treated.”

Although hepatitis is preventable, treatable, and often curable, only 45% of babies globally received the hepatitis B birth dose vaccine within 24 hours of birth in 2022—a critical early intervention. South Africa has made notable strides, yet challenges remain in ensuring equitable access, particularly in rural and underserved areas. 1,2

Understanding the Disease

Hepatitis refers to inflammation of the liver, most often caused by a viral infection. The most common types, hepatitis B (HBV) and hepatitis C (HCV), are both blood-borne and can lead to chronic liver disease, liver failure, and liver cancer.

  • Hepatitis B is spread through contact with infected blood or bodily fluids, unprotected sex, and from mother to child at birth. It is preventable through vaccination, which has been available for over four decades.1
  • Hepatitis C is commonly spread through unsafe medical practices, contaminated injections, or sharing needles. While there is no vaccine, hepatitis C is curable in most cases with a class of medicines known as direct-acting antiviral medications.1

Because symptoms often only appear in advanced stages, early testing and diagnosis are vital to preventing life-threatening complications.

Time to Act – Before It’s Too Late. Speak to your healthcare practitioner for more information.

Viral hepatitis causes an estimated 1.3 million deaths each year—a figure comparable to that of HIV/AIDS. Yet countries such as Egypt have proven that elimination is achievable through aggressive, integrated screening and vaccination efforts.2

South Africa has the science, tools, and expertise to respond effectively. What is now needed is national commitment, adequate investment, and a public health approach that embeds hepatitis services into primary care.

World Hepatitis Day 2025 served as a timely reminder: the elimination of viral hepatitis is within reach—but only if we act now.

Source – accessed 24 July 2025:

  1. World Health Organization.  World Hepatis Day 2025.  Hepatitis Lets Break it down.  Available from: World Hepatitis Day 2025. Fact sheets
  2. World Hepatitis Alliance.  What is Viral Hepatitis.  Available from: Home – World Hepatitis Alliance. What is Viral Hepatitis – World Hepatitis Alliance
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New Research Finds that Ivermectin Reduces Malaria Transmission

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Photo by Ekamelev on Unsplash

A collaborative new study involving KEMRI-Wellcome Trust researchers has highlighted a new way to control malaria transmission. The study found that ivermectin, a drug normally used for neglected tropical diseases, led to a 26% reduction in new malaria infections among children aged 5-15 by killing feeding mosquitoes.

Malaria remains a global health challenge, with 263 million cases and 597 000 deaths reported in 2023. Current vector control methods, such as long-lasting insecticidal nets and indoor residual spraying, have become less effective due to insecticide resistance and behavioural adaptations in mosquitoes to bite outdoors and during dusk or dawn, when people are not protected by these measures. This underscores the urgent need for innovative solutions to combat malaria.

The BOHEMIA trial, the largest study on ivermectin for malaria to date, showed a 26% reduction in new malaria infection on top of existing bed nets,providing strong evidence of ivermectin’s potential as a complementary tool in malaria control. Coordinated by the Barcelona Institute for Global Health (ISGlobal) – an institution supported by the “la Caixa” Foundation – in collaboration with the Manhiça Health Research Centre and the KEMRI-Wellcome Trust Research Programme, the study has been published in The New England Journal of Medicine.

Ivermectin is a drug traditionally used to treat neglected tropical diseases like onchocerciasis which causes river blindness and lymphatic filariasis which causes elephantiasis. It has now been shown to reduce malaria transmission by killing the mosquitoes that feed on treated individuals. Given the rising resistance to conventional insecticides, ivermectin could offer an effective new approach totackle malaria transmission, especially in regions where traditional methods have become less effective.

The Unitaid-funded BOHEMIA project (Broad One Health Endectocide-based Malaria Intervention in Africa) conducted two Mass Drug Administration (MDA) trials in the high-burden malaria regions: Kwale County (Kenya) and Mopeia district (Mozambique). The trials assessed the safety and efficacy of a single monthly dose of ivermectin (400mcg/kg) given for three consecutive months at the start of the rainy season in reducing malaria transmission. In Kenya, the intervention targeted children aged 5–15, while in Mozambique it focused on children under five.

In Kwale County, Kenya, children who received ivermectin experienced a 26% reduction in malaria infection incidence compared to those who received the control drug. The trial involved over 20 000 participants and more than 56 000 treatments, demonstrating that ivermectin significantly reduced malaria infection rates – particularly among children living further from cluster borders or in areas where drug distribution was more efficient. Moreover, the safety profile of ivermectin was favourable, with no severe drug-related adverse events and only mild, transient side effects already seen with ivermectin in campaigns against neglected tropical diseases.

Professor Marta Maia, Associate Professor at the Centre for Tropical Medicine and Global Health and Medical Entomologist based at the KEMRI-Wellcome Trust Research Programme, said: ‘The findings suggest that ivermectin MDA could be a valuable complementary strategy for malaria control, particularly in areas where mosquito resistance to insecticides is a growing concern.’

Dr Joseph Mwangangi, Senior Principal Research Scientist at the KEMRI-Wellcome Trust Research Programme, added: ‘These results align with the World Health Organization’s (WHO) criteria for new vector control tools.’

Carlos Chaccour, co-principal investigator of the BOHEMIA project said: ‘We are thrilled with these results. Ivermectin has shown great promise in reducing malaria transmission and could complement existing control measures. With continued research, ivermectin MDA could become an effective tool for malaria control and even contribute to elimination efforts.’

In contrast, the implementation of the Mozambique trial in the rural district of Mopeia faced severe disruptions due to Cyclone Gombe in 2022 and a subsequent cholera outbreak, which significantly disrupted operations.

Francisco Saúte, director of the Manhiça Health Research Centre said: ‘One of the most important lessons we learned from the trial in Mopeia is thatstrong community engagement is essential. Building trust with local communities and fostering close collaboration with the Health Ministry, National Malaria Control Program, and local authorities was key to ensuring acceptance of the ivermectin MDA.’

In addition to reducing malaria transmission, ivermectin MDA offers significant collateral benefits. The BOHEMIA team found an important reduction in the prevalence of skin infestations such as scabies and head lice in the ivermectin group in Mozambique, and the community reported a major reduction in bed bugs in Kenya. These effects are particularly valuable when ivermectin is integrated into existing delivery systems, maximising its impact on public health.

The study is part of a larger global effort to assess ivermectin’s potential in malaria control. The findings have been reviewed by the WHO vector control advisory group, which concluded that the study had demonstrated impact and recommended further studies. Findings were also shared with national health authorities as they evaluate the potential inclusion of ivermectin in malaria control programmes.

Regina Rabinovich, BOHEMIA PI and Director of ISGlobal’s Malaria Elimination Initiative said: ‘This research has the potential to shape the future of malaria prevention, particularly in endemic areas where existing tools are failing. With its novel mechanism of action and proven safety profile, ivermectin could offer a new approach using a well-known, safe drug that can add to the effect of other mosquito control tools available today.’

Source: Nuffield Department of Medicine, University of Oxford

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Toxoplasma Gondii’s Disruption of the Brain Gives Clues to New Treatments

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Source: Wikimedia CC0

A team of scientists at the University of California, Riverside, explains in a paper published in PLoS Pathogens how the microscopic parasite Toxoplasma gondii can significantly disrupt brain function, even when it infects only a small number of neurons. The team found the parasite interferes with essential communication between brain cells — research that can offer new ways to detect and treat chronic brain infections.

Toxoplasma gondii can infect nearly any warm-blooded animal and prefers to live inside brain cells, forming cysts in neurons that can persist for life. The researchers report that they found infected neurons release fewer extracellular vesicles (EVs) — tiny, membrane-bound packets used by cells to exchange information. 

“We found this disruption in EV signalling can interfere with how neurons and glial cells, especially astrocytes, maintain a healthy brain environment,” said Emma H. Wilson, a professor of biomedical sciences in the UC Riverside School of Medicine who led the research team. “Even a handful of infected neurons can shift the brain’s neurochemical balance. This suggests that communication between neurons and supporting glial cells is not only critical, but also vulnerable to hijacking by parasites.”

Approximately 10–30% of people in the United States are infected with Toxoplasma gondii, often without knowing it. The parasite is typically contracted through undercooked meat or exposure to cat feces. Although the immune system typically keeps the infection in check, the parasite can lie dormant in the brain for decades. In individuals with weakened immunity, it can reactivate and cause serious illness.

Current diagnostic tools can only detect whether someone has been exposed to Toxoplasma gondii by identifying antibodies. The tools cannot confirm whether the parasite is still present in the brain or how it may be affecting brain function.

“Our research opens the door to using EVs as biomarkers, which can be isolated from blood,” Wilson said. 

The study was conducted using mouse models and human cells in a laboratory setting.

Wilson explained that in healthy mouse brains astrocytes regulate neurotransmitters like glutamate, ensuring that neurons do not become overexcited. But when neurons infected with Toxoplasma gondii stop sending the right EV signals, this regulation breaks down. The result is elevated glutamate levels, which can lead to seizures, neural damage, or altered brain connectivity.

“The parasite may play a larger role in neurological and behavioural conditions than we previously thought,” she said.

Wilson’s research team is now working to analyse samples from human blood banks to look for EVs linked to Toxoplasma gondii brain infection. The team also hopes to better understand how glial cells detect and respond to parasite proteins — insights that could one day lead to new therapies or even vaccines.

“Our brains have built-in defences that may recognise and respond to neurons infected by Toxoplasma gondii,” Wilson said. “If we can learn how to support or enhance that process, we may be able to better protect people, especially the most vulnerable.”

Despite its potential impact, Toxoplasma gondii is often misunderstood, Wilson added. 

“There’s no need to avoid someone who is infected; most people live their entire lives without symptoms,” she said. “Pregnant individuals should be cautious as the parasite can cause serious birth defects if contracted for the first time during pregnancy. The most effective prevention is proper food handling and hygiene. Cook meat thoroughly, wash vegetables, and always wash your hands after handling cat litter, especially from young cats, which are more likely to shed the parasite.”

Source: University of California, Riverside

Categories : Diseases, Syndromes and Conditions GeneticsTags :

Baby with Rare, Incurable Disease is First to Receive Personalised Gene Therapy

On By ModernMedia

NIH-supported gene-editing platform lays groundwork to rapidly develop treatments for other rare genetic diseases.

Photo by Sangharsh Lohakare on Unsplash

A research team supported by the National Institutes of Health (NIH) has developed and safely delivered a personalised gene editing therapy to treat an infant with a life-threatening, incurable genetic disease. The infant, who was diagnosed with the rare condition carbamoyl phosphate synthetase 1 (CPS1) deficiency shortly after birth, has responded positively to the treatment.

The process, from diagnosis to treatment, took only six months and marks the first time the technology has been successfully deployed to treat a human patient. The technology used in this study was developed using a platform that could be tweaked to treat a wide range of genetic disorders and opens the possibility of creating personalised treatments in other parts of the body.

A team of researchers at the Children’s Hospital of Philadelphia (CHOP) and the Perelman School of Medicine at the University of Pennsylvania (Penn) developed the customised therapy using the gene-editing platform CRISPR. They corrected a specific gene mutation in the baby’s liver cells that led to the disorder. CRISPR is an advanced gene editing technology that enables precise changes to DNA inside living cells. This is the first known case of a personalised CRISPR-based medicine administered to a single patient and was carefully designed to target non-reproductive cells so changes would only affect the patient.

“As a platform, gene editing – built on reusable components and rapid customisation – promises a new era of precision medicine for hundreds of rare diseases, bringing life-changing therapies to patients when timing matters most: Early, fast, and tailored to the individual,” said Joni L. Rutter, Ph.D., director of NIH’s National Center for Advancing Translational Sciences (NCATS).

CPS1 deficiency is characterized by an inability to fully break down byproducts from protein metabolism in the liver, causing ammonia to build up to toxic levels in the body. It can cause severe damage to the brain and liver. Treatment includes a low protein diet until the child is old enough for a liver transplant. However, in this waiting period there is a risk of rapid organ failure due to stressors such as infection, trauma, or dehydration. High levels of ammonia can cause coma, brain swelling, and may be fatal or cause permanent brain damage.

The child initially received a very low dose of the therapy at six months of age, then a higher dose later. The research team saw signs that the therapy was effective almost from the start. The six-month old began taking in more protein in the diet, and the care team could reduce the medicine needed to keep ammonia levels low in the body. Another telling sign of the child’s improvement to date came after the child caught a cold, and later, had to deal with a gastrointestinal illness. Normally, such infections for a child in this condition could be extremely dangerous, especially with the possibility of ammonia reaching dangerous levels in the brain.

“We knew the method used to deliver the gene-editing machinery to the baby’s liver cells allowed us to give the treatment repeatedly. That meant we could start with a low dose that we were sure was safe,” said CHOP pediatrician Rebecca Ahrens-Nicklas, MD, PhD.

“We were very concerned when the baby got sick, but the baby just shrugged the illness off,” said Penn geneticist and first author Kiran Musunuru, MD, PhD. For now, much work remains, but the researchers are cautiously optimistic about the baby’s progress.

The scientists announced their work at the American Society of Gene & Cell Therapy Meeting on May 15th and described the study in The New England Journal of Medicine.

Source: NIH/Office of the Director