A novel approach to drug discovery is enabling University of Birmingham researchers to overcome the 'valley of death', where projects fail due to the funding gap between original research and commercial investment. The approach, detailed in a feature published in the April issue of Drug Discovery Today, has attracted more than £4m in industry funding, grants and industry awards, on the back of just £0.2m investment from the University's Dynamic Investment Fund (DIF ..read more

Alzheimer's disease (AD) currently afflicts nearly seven million people in the U.S. With this number expected to grow to nearly 13 million by 2050, the lack of meaningful therapies represents a major unmet medical need. Scientists at Sanford Burnham Prebys have now identified promising real-world links between common HIV drugs and a reduced incidence of AD. The study, led by Jerold Chun, M.D., Ph.D., was published in Pharmaceuticals. Chun's new research builds on his lab's landmark publication in Nature in 2018 that described how somatic gene recombination in neurons can produce thousands of n ..read more

Bayer AG and Asklepios BioPharmaceutical, Inc. (AskBio), a gene therapy company wholly owned and independently operated as a subsidiary of Bayer AG, on Sunday April 14 presented results from the 18-month Phase Ib clinical trial for AB-1005, an investigational gene therapy for treating patients with Parkinson's disease (PD).(1,2) The data were presented at the American Academy of Neurology 2024 Annual Meeting in Denver, Colorado, USA. The study met its primary objective, which was to evaluate the safety of a one-time bilateral delivery of AB-1005 directly to the putamen ..read more

Antisense oligonucleotides (ASOs) are next-generation drugs that can treat disease by blocking the transfer of harmful messages from our genes. In people with cancer, ASOs have the potential to block messages that encourage the growth and spread of the tumor. However, ASOs aren’t used for treating cancer yet. They must first get delivered inside cancer cells, but the cancer cells won’t let them in. Finding an effective ASO delivery system is a major challenge. Cancer cells have gatekeeper molecules that stop unwanted substances from entering ..read more

Researchers have developed a functional precision medicine approach that targets cancer by combining genetic testing with a new way to test individual drugs on tumor samples. The results of the clinical study were published today in Nature Medicine. This combined approach, developed by Florida International University cancer researcher Diana Azzam, was used successfully for the first time to guide treatment of relapsed pediatric cancer patients in collaboration with First Ascent Biomedical and Dr. Maggie Fader at the Helen & Jacob Shaham Cancer & Blood Disorders Institute at Nicklaus C ..read more

Thiazolidinediones (TZDs) are a class of drug that can be used to treat type 2 diabetes by reversing insulin resistance, one of the main hallmarks of the disease. While TZDs were extremely popular in the 1990’s and early 2000’s, they have fallen out of use among physicians in recent decades because they were discovered to cause unwanted side effects, including weight gain and excess fluid accumulation in body tissues ..read more

Infection with the influenza virus leads to lung injury through inflammation over-activation that causes collateral damage to cells required for breathing. Such damage can be life-threatening, but scientists have a new preventative treatment. A team from St. Jude Children's Research Hospital, University of Houston, Tufts University School of Medicine and Fox Chase Cancer Center created a drug that can prevent flu-induced lung injury. In a mouse model, the drug achieves a novel balance between shutting down runaway inflammation and allowing the immune system to stop the virus ..read more

Swapping red meat for 'forage fish', such as herring, sardines, and anchovies, could save up to 750,000 lives a year in 2050 and significantly reduce the prevalence of disability as a result of diet-related disease, suggests a data analysis published in the open access journal BMJ Global Health. Adopting this type of diet would be especially helpful for low and middle income countries, where these fish are cheap and plentiful, and where the toll taken by heart disease, in particular, is high, say the researchers ..read more

A new study by Upstate Medical University researchers shows that recreational cannabis use may offer protection against cognitive decline. The study, done by Master of Public Health (MPH) student Zhi Chen and Professor Roger Wong, Ph.D., MPH, MSW, analyzed a large data set from the CDC and found that compared to non-users, non-medical cannabis use, such as for recreational purposes, was significantly associated with 96 percent decreased odds of subjective cognitive decline (SCD ..read more

The lab of Yongchao C. Ma, PhD, at Stanley Manne Children's Research Institute at Ann & Robert H. Lurie Children's Hospital of Chicago discovered a fundamental biological mechanism that could lead to new treatments for neurological diseases, such as spinal muscular atrophy (SMA) and autism, as well as different cancers. The study was published in the journal Human Molecular Genetics. Dr. Ma's team found that chemical modification of RNA (called RNA methylation) regulates mitochondrial function ..read more