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Child Dies After Receiving New Experimental Gene Therapy
The first child to receive a new experimental gene therapy for a rare neurological disorder died a few days after dosing, the company sponsoring that trial said Wednesday.
The company, Capsida Biotherapeutics, is one of a group of companies and labs engineering new viruses potentially capable of safely ferrying genes deep into the brain. Its first effort was designed to treat STXBP1 encephalopathy, a condition that can cause seizures, developmental delays, and other symptoms.
The STXBP1 Foundation said in a note to community members that the company and investigators were still working to understand what happened.
COVID Falls From Top 10 U.S. Causes of Death for First Time Since 2020
U.S. News & World Report reported:
For the first time since the pandemic began, COVID-19 dropped out of the nation’s top 10 causes of death in 2024, new U.S. government data shows. Overall U.S. death rates also fell to their lowest level since 2020, according to the National Center for Health Statistics. Heart disease and cancer remained the leading causes of death. In all, 683,037 deaths due to heart disease and 619,812 due to cancer were reported last year — both up slightly from 2023.
Unintentional injuries ranked third, while suicide moved into the top 10 for the first time, displacing COVID-19. “Heart disease is still number one, and it’s definitely not going in the right direction,” Dr. Eric Topol, founder and director of Scripps Research in San Diego, told STAT News. Further, “there are many thousands of people dying of COVID still,” he added. After unintentional injuries, the list continued with stroke, chronic lower respiratory diseases, Alzheimer’s disease, diabetes, kidney disease, chronic liver disease and cirrhosis. Topol said these leading causes are largely preventable through lifestyle changes such as exercise and diet.
“These numbers are not encouraging,” he told STAT News. “We’re talking about diseases that are eminently preventable, just with lifestyle factors, but we have 75% of the population that doesn’t do even the minimum requirement of exercise.”
Most Docs Say It’s Fine to Get Tiny Gifts From Pharma, Survey Finds
Most doctors said it’s appropriate to accept gifts valued at under $50 from the pharmaceutical industry, according to one of the many findings from a longitudinal study that surveyed physicians at the start of their careers in 2011 and then again last year.
Yet a majority of the participants agreed at both points that marketing interactions threaten trust in medicine, with the share who strongly agreed with that idea growing from 5.6% in 2011 to 14.5% in 2024 (P=0.004), reported Aaron Kesselheim, M.D., J.D., M.P.H., of Brigham and Women’s Hospital in Boston, and colleagues.
More than 90% of those surveyed last year said that doctors might prescribe pricier drugs without extra benefits or become unconsciously biased toward a certain product as a result of interactions with pharma reps, according to the findings in JAMA Health Forum.
“Physicians expressed greater concern about pharmaceutical marketing in some spaces, but then also greater recognition that pharmaceutical representative materials are important resources to learn about drugs,” co-author Helen Mooney, M.P.H., also of Brigham and Women’s Hospital, told MedPage Today.
FDA Shares Guidance to Drugmakers Developing Non-Opioid Pain Medicines
While their addictive properties are now well known, opioids remain commonly prescribed because they are effective at neutralizing pain and often available in cheap, generic form. About one out of every five adults suffering from chronic pain takes opioids, according to the U.S. Food and Drug Administration (FDA).
Efforts to develop alternatives have been hampered by the significant challenges in pain research. Scientists don’t fully understand the mechanisms behind pain and analgesia, and two people with the same injury can have completely different experiences. In addition, compounds that look promising in laboratories often fail to translate to safe and effective medicines for people.
The FDA is hoping to kickstart more research by making “regulatory pathways more predictable for innovators and drug developers,” with the guidance, FDA Commissioner Marty Makary said in a statement accompanying the guidance. “Physicians need more alternatives.”
Merck’s Pneumococcal Shot Shows Promise in Children and Teens in Study
Merck’s pneumococcal vaccine showed strong immune responses in children and teens who are at higher risk of serious illness in a late-stage study, the drugmaker said on Thursday. The shot, Capvaxive, triggered immune responses against 21 strains of the bacteria responsible for pneumococcal infections, which can cause severe illnesses including pneumonia, meningitis and sepsis.
Merck was testing the shot against its older vaccine PPSV23 in children aged two years and above and teens aged below 18 years, who have completed a primary pediatric pneumococcal vaccination regimen and have one or more chronic medical conditions that put them at increased risk.
Capvaxive is already approved for adults in the U.S., European Union, Japan and other countries.
Intercept, After Many Setbacks, Pulls Liver Disease Drug Ocaliva in US
After years of regulatory struggles for Intercept Pharmaceuticals and its liver disease drug Ocaliva (obeticholic acid), the U.S. Food and Drug Administration (FDA) has called time on the drug’s run in the U.S. market.
The agency placed Intercept’s trials of obeticholic acid on a clinical hold and requested that the company voluntarily withdraw the drug from the market, where it has been cleared to treat certain patients with primary biliary cholangitis (PBC) since winning an accelerated approval in 2016. Despite Intercept’s arduous efforts to obtain full approval for the drug, the company has obliged and will pull the med, it announced in a Sept. 11 press release.
“While our view of Ocaliva’s benefit-risk profile differs from FDA’s, we respect its request and have made this difficult decision to provide clear guidance for patients and prescribers,” Intercept’s U.S. president Vivek Devaraj said in a statement. “We remain committed to innovation in hepatology and to serving the needs of patients and physicians.”
