For roughly a century, ever since Alexander Fleming's accidental discovery of penicillin in 1928, fungi have proven to be a goldmine for medicines. They've provided treatments for a wide range of diseases, from infections and high cholesterol to organ rejection and even cancer.

However, the process by which fungi synthesize some of their most potent compounds remains opaque.

Researchers at the Warren Center for Neuroscience Drug Discovery, a clinical stage biotech within the Vanderbilt University School of Medicine Basic Sciences, have detailed the successful drug discovery of a phase I single ascending dose clinical trial of VU319, a drug for memory loss in people with Alzheimer's disease and schizophrenia.

"This milestone highlights Vanderbilt’s ability to drive discovery from research to clinical impact," said Provost and Vice Chancellor for Academic Affairs Cybele Raver.

Millions of people take metformin, a Type 2 diabetes medication that lowers blood sugar. The "wonder drug" has also been shown to slow cancer growth, improve COVID outcomes and reduce inflammation. But until now, scientists have been unable to determine how, exactly, the drug works.

A new Northwestern Medicine study has provided direct evidence in mice that the drug reversibly cuts the cell’s energy supply by interfering with mitochondria,

A team of University of Melbourne researchers from the Caruso Nanoengineering Group has created an innovative drug delivery system with outstanding potential to improve drug development.

The team has pioneered a drug delivery system that is a coordination network composed of only metal ions and biomolecules, known as metal-biomolecule network (MBN).

A novel drug holds promise for treating Duchenne muscular dystrophy (DMD), a rare genetic disorder that causes severe muscle degeneration.

McGill University researchers have discovered that an experimental compound called K884 can boost the natural repair abilities of muscle stem cells. Current treatments can slow muscle damage, but don’t address the root problem.

As its 2024 Breakthrough of The Year, Science has named the development of lenacapavir - a promising new injectable drug that prevents HIV infection. The award also recognizes related work surrounding gaining a new understanding of the structure and function of HIV's capsid protein. Despite decades of advancements, HIV continues to infect more than a million people annually, with a vaccine remaining elusive. However, a new injectable drug, lenacapavir, offers hope by providing six months of protection per shot.

New research, led by experts at the University of Nottingham, has found that repeated doses of paracetamol in people aged 65 and over, can lead to an increased risk of gastrointestinal, cardiovascular and renal complications.

The new study, which is published in Arthritis Care and Research, shows that care must be taken when repeated doses are required for chronic painful conditions such as osteoarthritis in older people.

Bayer is today announcing that it plans to acquire HiDoc Technologies GmbH in the first quarter of 2025 and to start commercialization of the digital health application, Cara Care®. Cara Care® is the first prescribed digital health application for the treatment of irritable bowel syndrome (IBS) and offers a novel, holistic therapeutic approach for patients with IBS.(1)

Patients with irritable bowel syndrome have generally already been through a lot by the time they receive a formal diagnosis, and they face numerous daily challenges and obstacles associated with their symptoms that impair their quality of life.

A new gene therapy can reverse the effects of heart failure and restore heart function in a large animal model. The therapy increases the amount of blood the heart can pump and dramatically improves survival, in what a paper describing the results calls "an unprecedented recovery of cardiac function."

Currently, heart failure is irreversible. In the absence of a heart transplant, most medical treatments aim to reduce the stress on the heart and slow the progression of the often-deadly disease.

A group of scientists at VCU Massey Comprehensive Cancer Center has revealed a new genetic code that acts like a cancer ringleader, recruiting and deploying a gang of tumor cells to incite a biological turf war by invading healthy organs and overpowering the normal cells. This discovery - published today, Dec. 9, in Nature Biotechnology - could unveil an entirely different understanding of the origins of cancer within the body, as well as offer groundbreaking insight into new treatment strategies that could target the growth of tumors in their earliest stages.

The Horner-Wadsworth-Emmons (HWE) reaction is a fundamental reaction in organic chemistry, widely used to create conjugated carbonyl compounds. Conjugated carbonyl compounds are used in many industries for synthesizing perfumes, plastics, and pharmaceuticals and are also involved in biological processes. Consequently, methods for improving HWE reactions are an active area of research.

One potential application of HWE reactions is to develop (E)-isomers of conjugated carbonyl compounds that are useful for synthesizing chemicals called hynapene analogues with promising anti-cancer properties.

GSK plc (LSE/NYSE: GSK) and Medicines for Malaria Venture (MMV) announced that the World Health Organization (WHO) has awarded prequalification to tafenoquine, the first single-dose medicine for the prevention of relapse of Plasmodium vivax (P. vivax) malaria. Tafenoquine, co-administered with chloroquine, is now also included in WHO's updated Guidelines for malaria, in South America, marking the first time the medicine has been recommended by WHO. This milestone is a significant step toward closing the treatment gap for P. vivax malaria.

A new study led by Lund University in Sweden has solved a years-old mystery: which patients with aggressive breast cancer are helped by a targeted cancer treatment that had been under development but was shelved. The study is important since it brings hope that the development of the drug can continue and that it will make it all the way to these patients.

Despite the efforts of the North American researchers, many years of research and development and millions of dollars spent, a medicine named ganitumab that had shown promising results against breast cancer in animal trials was shelved.

Treatment for more advanced and difficult-to-treat head and neck cancers can be improved with the addition of polyvinyl alcohol (PVA), the same ingredient used in children's glue. Researchers found that combining PVA with a boron-containing compound, D-BPA, improved the effects of a type of radiation therapy for cancer, compared to currently clinically used drugs. The PVA made the drug more selective of tumor cells and prolonged drug retention, helping to spare healthy cells from unnecessary radiation damage.

A common heart drug may slow the progression of Huntington's disease (HD), according to a new study by University of Iowa Health Care researchers.

Using clinical information from a large, observational database of over 21,000 people with HD, the UI team found that the use of beta-blocker drugs - commonly used to treat heart and blood pressure issues - was associated with both a significantly later onset of HD symptoms for people in the pre-symptomatic stages, and a slower rate of symptom worsening for those with symptoms.

Researchers from the National Institutes of Health (NIH) have developed an artificial intelligence (AI) algorithm to help speed up the process of matching potential volunteers to relevant clinical research trials listed on ClinicalTrials.gov. A study published in Nature Communications found that the AI algorithm, called TrialGPT, could successfully identify relevant clinical trials for which a person is eligible and provide a summary that clearly explains how that person meets the criteria for study enrollment.

Large language models, a type of AI that analyses text, can predict the results of proposed neuroscience studies more accurately than human experts, finds a new study led by UCL (University College London) researchers.

The findings, published in Nature Human Behaviour, demonstrate that large language models (LLMs) trained on vast datasets of text can distil patterns from scientific literature, enabling them to forecast scientific outcomes with superhuman accuracy.

An injection given during some asthma and COPD attacks is more effective than the current treatment of steroid tablets, reducing the need for further treatment by 30%.

The findings, published today in The Lancet Respiratory Medicine, could be "game-changing" for millions of people with asthma and COPD around the world, scientists say.

Pfizer Inc. (NYSE: PFE) announced that after a comprehensive internal and external selection process, the company is appointing Chris Boshoff, M.D., PhD, as Chief Scientific Officer and President, Research & Development effective January 1, 2025. Dr. Boshoff, who most recently served as Chief Oncology Officer and Executive Vice President, will succeed Dr. Mikael Dolsten whose departure from Pfizer was announced earlier this year. In his new role, Dr. Boshoff will remain a member of Pfizer's Executive Leadership Team reporting to Chairman and Chief Executive Officer, Dr. Albert Bourla, and he will oversee all functions of Research & Development across all therapeutic areas.

With the rapid development of antibiotics in the 1930s, phage therapy - using viruses known as bacteriophages or phages to tackle bacterial infections - fell into oblivion. But as the current rise in antibiotic resistance is making it increasingly difficult to treat bacterial infections, phage therapy is once again sparking interest among physicians and scientists - although it remains complex in practice because of the great diversity and specificity of phages. Against this backdrop, scientists from the Institut Pasteur, Inserm, the Paris Public Hospital Network (AP-HP) and Université Paris Cité have developed a simple and effective new tool that recommends the best possible phage cocktail for a given patient.