Lifeboat Foundation

A study by the Global Antibiotic Research & Development Partnership and Innoviva Specialty Therapeutics found a single dose of a first-in-class oral antibiotic called zoliflodacin was as safe and effective as standard therapy for uncomplicated urogenital gonorrhea. NIAID contributed financial and scientific support to the development of zoliflodacin and applauds its non-governmental and private sector partners on successfully conducting the study. Read the NIH statement on these results: https://go.nih.gov/Wquuct

A single dose of a novel oral antibiotic called zoliflodacin has been found to be as safe and effective as standard therapy for uncomplicated urogenital gonorrhea in an international Phase 3 non-inferiority clinical trial, according to the Global Antibiotic Research & Development Partnership (GARDP), the study sponsor. Gonorrhea treatment options are increasingly limited due to antimicrobial resistance seen in Neisseria gonorrhoeae, the bacteria that cause gonococcal infection.

Because of the imperative to expand the gonococcal therapeutic pipeline, the National Institute of Allergy and Infectious Diseases (NIAID), part of the National Institutes of Health, has contributed financial and scientific support to the development of zoliflodacin and applauds its non-governmental and private sector partners on successfully conducting the Phase 3 study. This research has generated important new evidence for a field in urgent need of alternative therapeutic options. Specifically, zoliflodacin may offer an alternative to current therapy for uncomplicated urogenital gonococcal infection.

Continue reading “NIH Statement on Preliminary Efficacy Results of First-in-Class Gonorrhea Antibiotic Developed Through Public-Private Partnership” »

Tall 10-year-olds may be more at risk of developing an irregular heart rate in later life than their shorter counterparts, but less at risk of having a stroke.

By David Cox

A cancer drug in the final stages of clinical trials may be able to help treat a range of inflammatory diseases including gout, heart failure, cardiomyopathy, and atrial fibrillation, according to scientists at the University of Cambridge.

Their findings are published in the Journal of Clinical Investigation in an article titled, “PLK1 inhibition dampens NLRP3 inflammasome-elicited response in inflammatory disease models.”

“Unabated activation of the NLR family pyrin domain–containing 3 (NLRP3) inflammasome is linked with the pathogenesis of various inflammatory disorders. Polo-like kinase 1 (PLK1) has been widely studied for its role in mitosis,” wrote the researchers. “Here, using both pharmacological and genetic approaches, we demonstrate that PLK1 promoted NLRP3 inflammasome activation at cell interphase. Using an unbiased proximity-dependent biotin identification (Bio-ID) screen for the PLK1 interactome in macrophages, we show an enhanced proximal association of NLRP3 with PLK1 upon NLRP3 inflammasome activation. We further confirmed the interaction between PLK1 and NLRP3 and identified the interacting domains.”

Researchers hope dimeric IgA-based immunotherapy could be more precise, long-lasting, and less toxic than current anticancer treatments.

Engineers at the University of California San Diego have developed modular nanoparticles that can be easily customized to target different biological entities such as tumors, viruses or toxins. The surface of the nanoparticles is engineered to host any biological molecules of choice, making it possible to tailor the nanoparticles for a wide array of applications, ranging from targeted drug delivery to neutralizing biological agents.

The beauty of this technology lies in its simplicity and efficiency. Instead of crafting entirely new nanoparticles for each specific application, researchers can now employ a modular nanoparticle base and conveniently attach proteins targeting a desired biological entity.

In the past, creating distinct nanoparticles for different biological targets required going through a different synthetic process from start to finish each time. But with this new technique, the same modular nanoparticle base can be easily modified to create a whole set of specialized nanoparticles.

Researchers at the National Institutes of Health have developed a way to potentially increase the effectiveness of T cell–based immunotherapy treatments, such as CAR T-cell therapy, against solid tumors. T cells are specialized white blood cells of the immune system that eliminate infected or abnormal cells. In animal studies, the enhanced T-cell therapies were effective against cervical cancer and neuroblastoma, a common solid tumor in children. The findings, by scientists at the National Cancer Institute (NCI), part of NIH, appear in Clinical Cancer Research.

CAR T-cell therapy is a form of cellular immunotherapy that involves engineering T cells in the laboratory so they can specifically target and kill tumors. CAR T-cell therapy has been successful in treating blood cancers, but it hasn’t worked well for solid tumors. To improve the effectiveness of T-cell therapy against solid tumors, researchers at NCI’s Center for Cancer Research engineered T cells (CAR T cells and another form of cellular immunotherapy called TCR T cells) to carry cytokines, which are proteins that can boost T-cell function.

In laboratory studies, CAR and TCR T cells modified to express the cytokines IL-15 and IL-21 on their surface killed far more cancer cells than T cells carrying just one of these cytokines or neither of them. Previous research has found that treating patients with large amounts of cytokines caused severe, potentially fatal, side effects. The new approach aims to deliver this cytokine boost in a much more targeted way.

Transplant is the only option now for liver failure, but it’s not for everyone. One Mayo Clinic M.D./Ph. D. student and her mentor are researching ways to regenerate liver cells as a possible treatment for end-stage liver disease.

“Research provides us with the understanding to develop tools to make big changes in clinical problems like those facing patients with liver failure,” says Nguyen. “I want to be on the forefront of developing medical technologies that provide alternatives. I derive a lot of passion for research through thinking about the future patients I will be treating in the clinic.”

Nguyen is a fifth-year M.D.-Ph. D. student in the Mayo Clinic Alix School of Medicine, who is also completing her Ph.D. in regenerative sciences through the Mayo Clinic Graduate School of Biomedical Sciences.

Continue reading “Engineering stem cells to treat liver disease” »

Year 2020 face_with_colon_three

Ah, Memories! They can be some of our best assets or our most painful tormentors. Good memories give us a sensation of warmth and hope for better times, but bad memories can cause serious trauma.

In this study we aimed to detect epigenetic and genetic loci associated with PTSD in a homogeneous cohort of traumatized police officers. Both a genome-wide and hypothesis-driven replication approach did not result in DMPs between PTSD patients and trauma-exposed controls. GSE analysis on the top 100 DMPs showed, however, a plausible association of the dopaminergic neurogenesis pathway with PTSD. Furthermore, we observed one DMR located at the PAX8 gene suggesting consistent hypermethylation in PTSD patients. Genetic analyses yielded three CpG-SNPs significantly associated with PTSD. Of these, one CpG-SNP, located at the CACNA1C locus, was also significantly associated with PTSD in an independent replication sample of trauma-exposed children. Notably, this result shows that the Illumina 450K array is not restricted to epigenetic surveys but can provide informative genetic data as well.

Although our sample was small, it was highly homogenous as all participants were former or current police officers, and cases and controls were matched for sex, age, education, and years of police service. All participants reported multiple prior traumatic events, without significant group differences in reported types of traumatic experiences. PTSD patients fulfilled current diagnostic criteria for PTSD, while our trauma-exposed controls had minimal PTSD symptoms and did not report lifetime PTSD or other trauma-related psychiatric disorders. Thus our controls were apparently resilient to adverse mental health outcome of trauma. This study design, including extreme phenotypes following similar trauma load, was considered to favor detection of PTSD-associated loci, as also suggested by others [22]. Nevertheless, our genome-wide survey clearly remains limited in statistical power.