At Understanding Animal Research, we make it our mission to keep you informed about developments in the world of animal research. To finish the year, here is a pick of our top animal research stories of 2025 that inspire and offer hope for the future.
1. Huntington’s disease successfully treated for the first time
A novel one-time gene therapy, first tested in animals, has successfully treated Huntington’s disease for the first time in humans. The experimental treatment, AMT-130, targets and stops the production of the abnormal huntingtin proteins in the brain responsible for disease progression. It is delivered deep inside the brain during a 12–18 hour surgery. The therapy was successful in slowing the progression of the disease by about 75%, without significant side effects.
Human and monkey cells helped show that AMT-130 remained active as a genetic therapy for at least two years. AMT-130 was then tested in rats and monkeys to show it was safe and achieved widespread distribution throughout the brains of these small and large animals. The gene therapy also significantly reduced both normal and harmful huntingtin protein levels in transgenic minipigs with the disease.
2. New drug tackles chemotherapy-resistant cancers
To guard themselves from chemotherapy, cancer tumours can build protective walls around themselves made of immune cells. These cells act as gatekeepers, stopping cancer-fighting immune cells from coming in and working alongside the chemotherapy treatment. Researchers from King’s College London (KCL) have developed a daily pill designed to break down these immune-cell barriers, so chemotherapy treatments can function in more patients. The drug, named KCL-HO-1i, boosts the effectiveness of chemotherapy in mouse models, making chemotherapy-resistant tumours vulnerable. Clinical trials are aimed to start within the next two years.
news.cancerresearchuk.org/2025/11/04/a-new-drug-to-stop-cancers-resisting-chemotherapy-kcl-ho-1i/
3. A new pill against endometriosis
Endometriosis affects one in ten women of reproductive age. Treatments do exist, but remain limited and are often inadequate. The NHS in England approved this year a new daily pill, Linzagolix, with fewer side effects than other treatments, offering new hope to women who have been unable to find relief with existing options.
Research involved preclinical studies in animal models, mainly monkeys and rats, aimed at elucidating the pharmacokinetic profile, mechanism of action, preliminary toxicology and safety data. Research in dogs and rats also indicated that linzagolix may provide a new treatment option for benign prostatic hyperplasia (BPH) and polycystic ovary syndrome (PCOS). “We need animal models because the reproductive system doesn’t work in a little isolated bubble. It’s a hormone-dependent tissue that needs to be studied in a body,” said Professor Philippa Saunders, Chair of Reproductive Studies at the University of Edinburgh.
www.understandinganimalresearch.org.uk/news/the-animal-research-behind-the-new-endometriosis-pill
4. A new therapeutic target to fight asthma
Current treatments for asthma largely involve controlling the inflammation of lung tissue. This is crucial but not always enough to prevent and reverse structural lung damage that occurs in severe asthma. Four people still die every day in the UK from asthma-related complications. A team of researchers from the University of Aberdeen and the University of Manchester investigated in mice the scarring that occurs in the lungs as a result of asthma and were able to reverse these changes. They found that preventing inflammation alone was not enough to reverse tissue scarring. The action of specific proteins strongly associated with inflammation and tissue damage also needed to be blocked.
This discovery paves the way for a new approach not only to prevent disease progression and even reverse tissue scarring in asthma but also to treat many different diseases in which similar structural changes in tissues occur. Such diseases include conditions such as chronic obstructive pulmonary disease (COPD), chronic heart disease and cirrhosis of the liver and account for approximately 40% of deaths worldwide.
5. New treatment to tackle the commonest form of childhood cancer
Every year, more than 500 people are diagnosed in the UK with B-cell acute lymphoblastic leukaemia (B-ALL), the commonest cancer in childhood (accounting for up to 40% of all childhood cancers) and one that can be particularly difficult to treat in older patients. Although the disease can be curable, the treatment requires over two years of chemotherapy with significant toxic side effects.
Cambridge scientists have developed a new treatment that has the potential to be less toxic than current treatments and to be effective in all age groups. A combination of two drugs improves outcomes and reduces the need for toxic chemotherapy in cell lines and mice. Clinical trials are to begin shortly.
www.cam.ac.uk/stories/B-ALL-new-treatment
6. Nanoparticles reverse Alzheimer’s in mice
UCL researchers have reversed Alzheimer's disease in mice using nanoparticles. The therapy restores the proper function of the blood-brain barrier and helps the brain naturally clear away toxic amyloid (Aβ) proteins.
Researchers used mice that are genetically altered to produce larger amounts of Aβ protein and develop a significant cognitive decline mimicking Alzheimer's disease. They administered three doses of the nanoparticles, and after only one hour, a reduction of 50–60% in Aβ was observed inside the brain. By repairing the critical blood-brain interface, the researchers achieved a reversal of Alzheimer's pathology in animals.
www.ucl.ac.uk/news/2025/oct/nanoparticles-reverse-alzheimers-pathology-mice
7. New rheumatoid arthritis treatment in clinical trials
In rheumatoid arthritis, the immune system goes into overdrive and attacks healthy tissues, causing joint pain, stiffness, redness and swelling of the joints. A new type of treatment called tolerogenic dendritic cell (tolDC) therapy has shown promising results in animal studies. Following these results, Newcastle researchers have determined that tolDC therapy is safe to give patients in a phase I trial. The therapy works by collecting a sample of white blood cells from the patient, which is then sent to a specialist laboratory. The cells are then stabilised and injected back into the body to help calm the overactive immune system. A phase II clinical trial has begun to assess where the treatment should be injected and its effect on the immune system.
8. Pioneering microstent to treat glaucoma
Glaucoma is a leading cause of vision loss, second only to cataracts. Globally, 7.7 million people were blind or visually impaired due to glaucoma in 2020. The condition can cause irreversible damage to the optic nerve, due to increased pressure within the eyeball. Current treatment options – principally surgical – are highly invasive, carry a risk of complications, and have limited durability.
A team of researchers at the University of Oxford have unveiled a pioneering “microstent” which could revolutionise treatment for glaucoma. Initial trials carried out in rabbits found that the microstents lowered eye pressure in less than a month with minimal inflammation and scarring. They achieved a greater reduction of eye pressure than a standard tubular implant. This technique has the potential to transform the landscape of glaucoma therapy by offering an enhanced solution that combines mechanical innovation with biocompatibility, with a minimally invasive glaucoma surgery.
www.ox.ac.uk/news/2025-08-21-oxford-researchers-develop-uniquely-shaped-microstent-combat-glaucoma
9. Key difference between the immune systems of males and females identified
Biological sex affects the function of the immune system. Females and males tend to respond differently to the same infections, allergens, inflammation triggers, or immune therapies – and females are often more severely affected by autoimmune conditions or allergic diseases. Scientists from the University of York may have found one of the reasons why.
They have identified the gene Malat1 as a critical player in regulating immune responses in female immune cells, but not in males. The team discovered that, in female mice, a type of immune cell called Th2 cells did not develop appropriately during lung inflammation when the gene Malat1 was missing. However, this defect was not seen in male mice. Next step: examining these results in humans and exploring how Malat1 works to fine-tune immune responses, in hopes that this will lead to new treatments.
www.york.ac.uk/news-and-events/news/2025/research/genetic-key-immune-responses-male-females/
10. Scientists rewrite textbooks on how cells divide
Scientists from the University of Manchester have changed our understanding of how cells in living organisms divide. We are taught at school that when a cell divides, it generates two daughter cells that are uniform and spherical. The researchers showed that in real living organisms, it is not as simple as that. The resulting daughter cells are often not spherical and not even symmetrical. The scientists used real-time imaging to study the formation of blood vessels in one-day-old transparent zebrafish embryos, allowing them to study a dynamic process inside a living organism. The findings could have far-reaching implications on our understanding of the role of cell division in disease, notably cancer.
www.manchester.ac.uk/about/news/scientists-rewrite-textbooks-on-how-cells-divide/
11. Bird flu viruses are resistant to fever, making them even more dangerous to humans
Unlike human flu viruses, avian influenza viruses tend to thrive at temperatures that can be as high as 40–42 °C, well above what is typically experienced during fever. New research led by the universities of Cambridge and Glasgow used in vivo models – mice infected with influenza viruses – to help explain how fever protects humans and why it may not be enough to protect us against avian influenza.
Although mice do not typically develop fever in response to influenza A viruses, the researchers were able to mimic the fever effect by raising the ambient temperature where the mice were housed (elevating the body temperature of the mice). Raising body temperature to fever levels was effective at stopping human-origin flu viruses from replicating, but it is unlikely to stop avian flu viruses. The research also revealed that the PB1 gene of the virus, important in the replication of the virus genome inside infected cells, plays a key role in setting the temperature sensitivity. Viruses carrying an avian-like PB1 gene were able to withstand the high temperatures associated with fever and caused severe illness in the mice. This is important because human and bird flu viruses can “swap” their genes when they co-infect a host at the same time, for example, when both viruses infect pigs. This phenomenon may even explain why previous flu pandemics caused serious illness in people. It remains crucial to monitor bird flu strains to prepare for potential outbreaks.
12. New vaccine to tackle bovine respiratory syncytial virus in calves
The highly contagious bovine respiratory syncytial virus (BRSV) can cause serious respiratory infection in calves, representing an annual cost to British farmers of around £54 million, with global figures reaching as much as £5.6 billion.
A University of Plymouth spinoff company has developed a novel vaccine candidate using bovine herpesvirus-4 vaccine platform technology to fight BRSV. The vaccines prevented clinical disease in vaccinated calves with no significant virus shedding. This vaccine technology offers several advantages over current commercial vaccines, which use the live or inactivated whole virus of BRSV to stimulate an immune response. It can notably be administered in very young animals, even in the presence of maternal antibodies, further narrowing the window of potential infection. This vaccine candidate represents an opportunity to provide complete protection against a major livestock disease.
www.plymouth.ac.uk/news/university-spinout-highlights-outstanding-performance-of-vaccine-candidate
13. Gut bacteria help control healthy muscle contraction in the colon
A healthy gut contains trillions of microorganisms, which help the digestion of food and promote the fitness of gut tissues. Disturbances of intestinal motility are extremely common and result in many symptoms. New research from the Crick and Bern University using mice, has shown that these micro-organisms in the gut support healthy digestion by helping nerve cells within the intestine to regulate the contraction and relaxation of the muscle wall of the colon. The presence of the microbes activates a specific gene called Ahr in intestinal nerves, resulting in healthy contraction and relaxation of the colon in mice. The Ahr molecule is known to be very important for the function of immune and epithelial cells in the gut. This relationship can be disrupted in cases of intestinal disorders, such as irritable bowel syndrome (IBS). The work helps better understand how nerve cells sense the microbes in the gut and how they could coordinate their function with other gut tissues.
www.crick.ac.uk/news/2020-02-05_gut-bacteria-help-control-healthy-muscle-contraction-in-the-colon
Last edited: 5 January 2026 14:22
