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Category: biotech/medical – Page 227

Ganglion Cell Layer Compared With Inner Plexiform Layer Atrophy After Optic Neuritis Associated With NMOSD, MOGAD, and MS

In a phase 3 randomized clinical trial of adults with ParkinsonDisease experiencing motor fluctuations despite stable levodopa therapy, adjunctive tavapadon—a once-daily, selective D1/D5 dopamine agonist—significantly increased daily on-time without troublesome dyskinesia and reduced off-time compared with placebo over 27 weeks.

Most adverse events, including nausea, dyskinesia, and dizziness, were mild to moderate. Tavapadon showed a favorable safety profile and provided clinically meaningful motor improvements as adjunctive therapy.

Question Can adjunctive tavapadon—an oral, once-daily, selective dopamine (D) D1/D5 agonist—improve motor control for people with Parkinson disease (PD) experiencing motor fluctuations while minimizing risk of adverse events?

Findings In this phase 3, double-blind, placebo-controlled, 27-week randomized clinical trial of 507 participants with PD, tavapadon significantly increased daily on-time without troublesome dyskinesia (good-on-time) vs placebo. Most adverse events were mild to moderate in severity with nausea, dyskinesia, and dizziness most common with tavapadon.

Meaning Results show that tavapadon adjunctive to levodopa provided clinically meaningful motor improvements and an acceptable safety profile in adults with PD experiencing motor fluctuations while receiving oral levodopa.

New genetic risk score better predicts diabetes, obesity and downstream complications

Type 2 diabetes (T2D) and obesity are metabolic conditions with many causes, including overlapping and distinct genetic features. A polygenic risk score (PRS) can capture multiple genetic risk factors to provide an estimate for whether a person may develop a complex medical condition and how they might fare long-term.

Building stronger genetic risk scores By integrating genetic findings from several of the world’s largest biobanks, investigators from Mass General Brigham built metabolic PRSs for predicting obesity and T2D, which outperformed existing disease-prediction models and predicted downstream morbidity and clinical interventions. Findings are published in Cell Metabolism.

“Our intention was to not only capture the risk of being diagnosed with obesity or diabetes, but also to better predict health consequences across the life course by integrating many aspects of metabolic function,” said co-first author Min Seo Kim, MD, MSc.

Designed to remember

In a new Science study, researchers report that specific regions dense in cytosine and guanosine dinucleotides are epigenetically modified during inflammation to enable gene expression and that these changes persist during the animal’s lifetime.

The finding has implications for understanding how the genome determines the longevity of memory, which affects tissue fitness.

Learn more in a new Science Perspective.

Specific DNA sequence features encode the persistence of epigenetic memory of inflammation.

Guillaume Blot and Przemyslaw Sapieha Authors Info & Affiliations

Science

Continue reading “Designed to remember” | >

How stimulating the vagus nerve could protect the brain from Alzheimer’s disease

Developing tau tangles doesn’t mean a person has Alzheimer’s disease – in fact, it happens to nearly everyone to varying degrees. But because these changes start in the locus coeruleus, some brain researchers – myself included – see this area as a canary in the coal mine for developing Alzheimer’s disease.

We are exploring whether stopping or slowing down tau tangles in this brain region, or otherwise maintaining its health, may be a way to interrupt how the disease ultimately unfolds and to prevent other aspects of cognitive aging.

Emerging research from my lab and others is investigating the idea that a therapy called vagus nerve stimulation, which is already widely used for other health conditions, could be one way of keeping the locus coeruleus functioning properly.

Longevity Isn’t Equal: Why Life-Extending Treatments May Be a “Biological Lottery”

Extending life is only part of the goal in aging research. Scientists also want more people to reach old age in good health, with fewer differences in when individuals die. This ideal outcome is often described as “squaring the survival curve,” where most deaths are pushed into a narrow window late in life rather than spread out across many years.

To test how close current science comes to that goal, University of Sydney researchers revisited a large meta-analysis of studies in vertebrates. They focused on three widely studied interventions: dietary restriction, rapamycin, and metformin. While all are linked to longevity, they work in different ways.

Dietary restriction involves reducing calorie intake without causing malnutrition. It has been known for more than a century to extend lifespan in animals and is thought to act in part by dialing down a key cellular growth pathway called mTORC1, which helps regulate metabolism and aging. Because strict diets are difficult to maintain, scientists have searched for drugs that mimic these effects. Rapamycin directly blocks mTORC1 activity, while metformin, a common diabetes medication, influences the same pathway indirectly by altering how cells sense energy levels.

Mesothelioma: a systemic therapy clinical trials snapshot

Systemic therapy targets in mesothelioma.

Recent changes to clinical practice have made modest improvements in 1– 2-year survival, but longer-term survival remains unchanged, and durable benefit is very rare.

Combining immunotherapy with chemotherapy, particularly with novel bispecific agents, may result in more therapeutic modalities being administered together in the first-line setting. The current evidence for second-line treatments is sparse.

New targeted-therapy strategies are promising. Early-phase clinical trials are showing signals of efficacy in mesotheliomas harboring MTAP loss or inactivation of the Hippo pathway.

Further studies will be needed to robustly confirm clinical benefit. sciencenewshighlights ScienceMission https://sciencemission.com/Mesothelioma

Mesothelioma is a rare cancer that has seen few incremental improvements in survival over the past two decades. However, a significantly improved understanding of the underlying biology has led to new therapeutic advances with the potential to improve clinical outcomes. In this review, we take a snapshot of the current systemic therapy research landscape, with our goal to forecast the trajectory of drug development for mesothelioma over the next half-decade. In our current census, we identify 106 active trials including systemic therapies: 20 (19%) are molecularly targeted, 26 (25%) include immunomodulation, and 12 (11%) combining immunotherapy with antiangiogenic therapies. Collectively, the landscape of therapeutic innovation for mesothelioma is expanding, bringing hope that improvements in life expectancy may follow.

Continue reading “Mesothelioma: a systemic therapy clinical trials snapshot” | >

Molecular Markers of Blood Cell Populations Can Help Estimate Aging of the Immune System

Aging of the immune system involves functional changes in individual cell populations, in hematopoietic tissues and at the systemic level. They are mediated by factors produced by circulating cells, niche cells, and at the systemic level. Age-related alterations in the microenvironment of the bone marrow and thymus cause a decrease in the production of naive immune cells and functional immunodeficiencies. Another result of aging and reduced tissue immune surveillance is the accumulation of senescent cells. Some viral infections deplete adaptive immune cells, increasing the risk of autoimmune and immunodeficiency conditions, leading to a general degradation in the specificity and effectiveness of the immune system in old age.

Mineral and bone effects of EPT

Highlighting the potential of PTH1R receptor agonist therapy in autosomal dominant hypocalcemia type 1

https://doi.org/10.1172/JCI201759 In this Research Letter, Rajesh V. Thakker & team report on the use of Eneboparatide in mice with ADH1, which increased serum calcium levels without increasing urine calcium.

3Mary Lyon Centre, MRC Harwell, Harwell Science and Innovation Campus, Oxfordshire, United Kingdom.

4Endocrine Unit, Massachusetts General Hospital, and Harvard Medical School, Boston, Massachusetts, USA.

5William Harvey Research Institute, London School of Medicine, Queen Mary University of London, London, United Kingdom.

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