Lifeboat Foundation: Safeguarding Humanity
Toggle light / dark theme

Blog

Category: biotech/medical

Gene therapy improves movement in kids with spinal muscular atrophy

A single-dose gene replacement therapy is found to improve movement ability in children over 2 years of age and teenagers with spinal muscular atrophy, according to research published in Nature Medicine. The results of this phase 3 clinical trial, involving 126 children and adolescents, could support an alternative to lifelong, repeat-dose treatments for people living with spinal atrophy beyond the age of 2 years.

Spinal muscular atrophy is a rare genetic condition that causes muscle weakness and loss of movement over time. It develops because the body cannot make enough of a protein, called survival motor neuron, needed for healthy nerve cells.

Onasemnogene abeparvovec is a gene therapy that restores production of this missing protein in a single treatment. However, it is currently approved in the U.S. and Europe only as a single intravenous treatment for children under 2 years of age. Therefore, those older than 2 years of age can receive treatments only to slow the disease, and these must be taken regularly, either by injection or orally.

Mice Generated with Induced Pluripotent Stem Cells Derived from Mucosal-Associated Invariant T Cells

The function of mucosal-associated invariant T (MAIT) cells, a burgeoning member of innate-like T cells abundant in humans and implicated in many diseases, remains obscure. To explore this, mice with a rearranged T cell receptor (TCR) α or β locus, specific for MAIT cells, were generated via induced pluripotent stem cells derived from MAIT cells and were designated Vα19 and Vβ8 mice, respectively. Both groups of mice expressed large numbers of MAIT cells. The MAIT cells from these mice were activated by cytokines and an agonist to produce IFN-γ and IL-17. While Vβ8 mice showed resistance in a cancer metastasis model, Vα19 mice did not. Adoptive transfer of MAIT cells from the latter into the control mice, however, recapitulated the resistance.

Ozempic can reduce metabolic risks in schizophrenia patients, multicenter study finds

Semaglutide medications like Ozempic and Wegovy can help lower the risk of heart and metabolic diseases in people with schizophrenia spectrum disorders, according to a study published in JAMA Psychiatry.

Glucagon-like peptide–1 receptor agonist (GLP-1RAs) drugs, such as semaglutide, mimic the natural gut hormone GLP-1 that regulates hunger and food intake. By activating GLP-1 receptors in the brain, the drug reduces hunger and slows gastric emptying, helping one feel full longer. It also enhances glucose-dependent insulin secretion, thereby improving blood sugar control.

Researchers in Denmark recruited 73 adults taking antipsychotic medications who were showing early signs of diabetes and had an average BMI of 36, which falls in the category of obesity. The participants, aged 18 to 65 years, were randomly assigned to receive either weekly semaglutide injections or a placebo for 6.5 months.

Unified EEG imaging improves mapping for epilepsy surgery

A new advance from Carnegie Mellon University researchers could reshape how clinicians identify the brain regions responsible for drug-resistant epilepsy. Surgery can be a life-changing option for millions of epilepsy patients worldwide, but only if physicians can accurately locate the epileptogenic zone, the area where seizures originate.

Bin He, professor of biomedical engineering, and his team have developed a unified, machine learning-based approach called spatial-temporal-spectral imaging (STSI) to assist. It is the first technology capable of analyzing every major type of epileptic brain signal within a single computational framework.

Their work, published in PNAS, presents a technical breakthrough and promising new direction for noninvasive presurgical planning.

Do ICIs Have Long-Term Survival Benefit?

A cross‑sectional analysis found that few registration trials assessing immune checkpoint inhibitors (ICIs) cancer therapies report long-term overall survival data. Among nearly 90 FDA-approved ICIs indications in the metastatic setting, less than one third of trials had overall survival data at 3 years, and even fewer (about 11%) reported overall survival data at 5 years.

Most registration trials for ICI cancer therapies don’t report long term overall survival data, a cross-sectional analysis finds.

Abstract: Introducing a new player in GLP-1-regulated insulin secretion…

Jeffrey R. Schelling & team find inhibition of the fatty acid transport protein FATP2 exerts glucose-lowering effects through GLP-1 secretion by alpha cells:

The figure shows FATP2 expression localizes to pancreatic alpha cells in mouse islets.

1Department of Physiology and Biophysics, Case Western Reserve University School of Medicine, Cleveland, Ohio, USA.

2Louis Stokes Cleveland VA Medical Center, VA Northeast Ohio Healthcare System; Department of Ophthalmic Research, Cole Eye Institute, Cleveland, Ohio, USA.

3Department of Medicine and Naomi Berrie Diabetes Center, Vagelos College of Physicians and Surgeons of Columbia University, New York, New York, USA.

Researchers Use Virus To Trace Psilocybin’s Effects Across the Entire Brain

An international collaboration led by Cornell researchers used a combination of psilocybin and the rabies virus to map how – and where – the psychedelic compound rewires the connections in the brain.

Specifically, they showed psilocybin weakens the cortico-cortical feedback loops that can lock people into negative thinking. Psilocybin also strengthens pathways to subcortical regions that turn sensory perceptions into action, essentially enhancing sensory-motor responses.

The findings published Dec. 5 in Cell. The lead author is postdoctoral researcher Quan Jiang.

/* */