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.

The preclinical study, published in Cardiovascular Diabetology, found LXA4, which is known for its "calming agent" action in turning off the body’s inflammatory response and preventing chronic inflammation, could also serve as a potential new treatment for diabetes-induced heart disease.

Heart conditions like atherosclerosis, heart attacks and heart failure are the leading killers of people with diabetes, driving a growing global health crisis.

Researchers at Karolinska Institutet have found a molecule that can both help the intestines to heal after damage and suppress tumour growth in colorectal cancer. The discovery could lead to new treatments for inflammatory bowel disease (IBD) and cancer. The results are published in the journal Nature.

Many patients with inflammatory bowel disease (IBD) such as Crohn's disease or ulcerative colitis do not respond to available treatments, highlighting the need to identify novel therapeutic strategies.

Malaria, particularly in its severe forms, remains a global health and economic burden. It causes the deaths of more than 600,000 people every year - most of them African children under five. In a new study, published in the journal Nature, researchers from EMBL Barcelona, the University of Texas, the University of Copenhagen, and The Scripps Research Institute have discovered human antibodies that can recognise and target some of the proteins that cause severe malaria. This breakthrough could pave the way for future vaccines or anti-malaria treatments.

AstraZeneca has announced $3.5 billion of capital investment in the United States focused on expanding the Company's research and manufacturing footprint by the end of 2026. This includes $2 billion of new investment creating more than a thousand new, high-skilled jobs contributing to the growth of the US economy.

Researchers at the Schulich Faculty of Chemistry at the Technion have developed a new chemical process to produce raw materials for the manufacture of polymers, pharmaceuticals, and agricultural compounds. In a paper about the process in Nature Chemistry, the researchers detail how they developed the new process and conducted a computational analysis to explain its mechanisms and key stages. The study was led by doctoral students Alexander Koronatov and Deepak Ranolia, and postdoctoral researcher Pavel Sakharov, under the guidance of Prof. Mark Gandelman.

A multitude of genes have been linked to the development of Alzheimer's disease. Specifically how those genes might influence the progression of neurodegeneration remains something of a black box though, in part because of the challenges of examining in molecular detail the brain of a living patient.

Using cerebrospinal fluid (CSF) collected from living patients, a team of researchers at Washington University School of Medicine in St. Louis has for the first time linked disease-related proteins and genes to identify specific cellular pathways responsible for Alzheimer's genesis and progression.

The U.S. Food and Drug Administration approved Kebilidi (eladocagene exuparvovec-tneq), an adeno-associated virus vector-based gene therapy indicated for the treatment of adult and pediatric patients with aromatic L-amino acid decarboxylase (AADC) deficiency. Kebilidi is the first FDA-approved gene therapy for treatment of AADC deficiency.

Scientists at the University of Copenhagen have discovered a new weight loss drug target that reduces appetite, increases energy expenditure, and improves insulin sensitivity without causing nausea or loss of muscle mass. The discovery was reported in the journal Nature and could lead to a new therapy for millions of people with both obesity and type 2 diabetes who do not respond well to current treatments.

Millions of people around the world benefit from weight loss drugs based on the incretin hormone GLP-1.