A blood test could one day offer a highly sensitive and inexpensive approach to detect early cancer in people with neurofibromatosis type 1 (NF1), according to work from researchers at the National Cancer Institute’s (NCI) Center for Cancer Research and Washington University School of Medicine in St. Louis. This blood…

Almost half of adults and children with neurofibromatosis type 1 (NF1) responded to mirdametinib, a MEK1/2 inhibitor, in a Phase IIb trial, ReNeu. The patients had symptomatic neurofibromatosis type 1-associated plexiform neurofibroma. Mirdametinib is an investigational, highly selective, potent, allosteric, central nervous system (CNS)–penetrant, small-molecule MEK1/2 inhibitor. The study appears…

A new combination of experimental drugs may help treat malignant peripheral nerve sheath tumors (MPNST) with fewer harmful side effects, according to preliminary animal studies led by investigators at Johns Hopkins. The study, published in the journal Molecular Cancer Therapeutics, shows that combining an experimental drug that blocks the metabolism of…

Research led by Oregon Health and Science University and Stanford University School of Medicine suggests blocking a protein known as alpha-5 integrin could have therapeutic potential for treating the fatal neurological disease amyotrophic lateral sclerosis (ALS). When the researchers gave a monoclonal antibody that blocks alpha-5 integrin activity to mice…

Artificial intelligence (AI) has the potential to improve many areas of medicine, with drug development as a key focus. Many companies are now working to apply the power of AI to design better drugs, but six companies are leading the race with candidates already in clinical trials. Over the last…

Digital biology drug discovery company Recursion has secured a commitment of up to $12 billion from Roche and its Genetech subsidiary to apply its Recursion Operating Systemt (OS) to advance therapies across 40 program that include neuroscience and an undisclosed oncology indication the companies announced today. Recursion OS applies machine…

About a decade before becoming NIH Director—a position from which he will step down at year’s end—Francis S. Collins, M.D., Ph.D., was at the center of one of the hottest debates in the nascent days of genomics: How many protein-coding sequences comprise the human genome? Collins, then leading the Human…

Twelves years after taking the helm of the NIH and straddling the administrations of three presidents, Francis S. Collins, M.D., Ph.D., announced yesterday he will step down as director of the NIH, ending the longest tenure of any person in that post. “It has been an incredible privilege to lead…

Brigham and Women’s Hospital has launched the Preventive Genomics Clinic, a facility designed to provide comprehensive DNA sequencing, interpretation and reporting of disease-associated genes. The clinic is intended to help healthy adults and their children understand and reduce their risk of future disease, according to Brigham & Women’s, which said…