Alicja Urbaniak, MSc, PhD’s Post

Inflammation in the gut/brain axis is a key factor in neurodegenerative diseases such as Parkinson’s. We were delighted to learn of the use of TRC research chemicals in preclinical drug repurposing work in which the anti-inflammatory dutasteride was identified as a potential promising drug for the treatment of early Parkinson’s disease. Read the research here, published in the journal Frontiers in Pharmacology: https://okt.to/hyz7tB #Citation #ProductCitation #ResearchChemicals #Parkinsons #ParkinsonsDisease #Neurology #NeurodegenerativeDiseases

Simoa® technology has profound implications for clinical trials in neuropathological diseases. Gain the latest insights into how biomarkers are enhancing drug development. ➡️ https://bit.ly/3Q4xFbD #Neurology #ClinicalTrials #DrugDevelopment

The recent FDA approval of WAINUA™ (eplontersen) by Ionis Pharmaceuticals and AstraZeneca marks a landmark achievement in the field of genetic medicine. Derived from the comprehensive Phase 3 NEURO-TTRansform study, WAINUA is now the first FDA-approved, self-administered therapy for hereditary transthyretin-mediated amyloid polyneuropathy (hATTR-PN). This approval is a testament to the potential of RNA-targeted therapies in addressing complex genetic disorders.   WAINUA stands out not just for its unique mode of administration but also for its effectiveness in slowing the progression of this debilitating neuropathic disease, as validated by the results published in The Journal of the American Medical Association (JAMA). This therapy represents a major step forward in improving the quality of life for patients afflicted with hATTR-PN.   Looking to the future, the scope of WAINUA extends beyond hATTR-PN. The ongoing Phase 3 CARDIO-TTRansform study is exploring the efficacy of eplontersen in treating transthyretin-mediated amyloid cardiomyopathy (ATTR-CM), potentially broadening the impact of this innovative treatment in the realm of cardiac health.   This development underscores the importance of continued investment and innovation in genetic medicine. As a professional community, it is crucial to acknowledge and support advancements that offer new hope and improved treatment options for patients with rare and challenging conditions.   #WAINUA #GeneticTherapy #hATTRPN #FDAApproval #HopeInScience #mRNA #ATTR-CM

📃Scientific paper: The type 1 cannabinoid receptor positive allosteric modulators GAT591 and GAT593 reduce spike-and-wave discharges in Genetic Absence Epilepsy Rats from Strasbourg Abstract: Childhood absence epilepsy (CAE) is a non-convulsive seizure disorder primarily in children characterized by absence seizures. Absence seizures consist of 2.5–5 Hz spike-and-wave discharges (SWDs) detectable using electroencephalography (EEG). Current drug treatments are only partially effective and adverse side effects have spurred research into alternative treatment approaches. Recent research shows that positive allosteric modulation of the type-1 cannabinoid receptor (CB1R) reduces the frequency and duration of SWDs in Genetic Absence Epilepsy Rats from Strasbourg (GAERS), a model that recapitulates the SWDs in CAE. Here, we tested additional CB1R ago-PAMs, GAT591 and GAT593, for their potential in alleviating SWD activity in GAERS. In vitro experiments confirm that GAT591 and GAT593 exhibit increased potency and selectivity in cell cultures and behave as CB1R allosteric agonists and PAMs. To assess drug effects on SWDs, bilateral electrodes were surgically implanted in the somatosensory cortices of male GAERS and EEGs recorded for 4 h following systemic administration of GAT591 or GAT593 (1.0, 3.0 and 10.0 mg/kg). Both GAT591 and GAT593 dose-dependently reduced total SWD duration during the recording period. The greatest effect on SWD activity was observed at 10.0 mg/kg doses, with GAT591 and GAT593 reducing seizure duration by 36% and 34% respectively. Taken together, these results support the continued investigation of CB1R PAMs as a potential therapeutic to... Discover the rest of the scientific article on es/iode ➡️https://etcse.fr/zq1t

[ALA Client News] Axoltis Pharma presents promising results from phase 1b clinical trial of innovative drug candidate for neurodegenerative diseases ☑️ Good safety profile and significant pharmacological effects confirm NX210c drug candidate properties and potential to address broad range of neurodegenerative diseases ☑️Biomarkers reveal significant and sustained effects confirming NX210c’s action on integrity and repair of Blood Brain Barrier (#BBB), as well as fundamental pathways in neuroprotection and neurotransmission ☑️ In 2024, Axoltis plans to initiate phase 2 trial in patients with Amyotrophic Lateral Sclerosis (#ALS) and conduct phase 1b trial in Parkinson’s Disease (#PD) while performing in silico modeling to further evaluate and optimize potential of NX210c more broadly in neurodegenerative diseases 👉 https://lnkd.in/eR4uwvTj Yann GODFRIN, Ph.D. Annette Janus, MD Centre for Human Drug Research InSilicoTrials #pharma #neurology #clinicaltrials

Discover the power of precision-reprogrammed human microglia from iPSCs, revolutionizing neurodegeneration research and therapy. Learn how a leading research organization employs hiPSC-derived microglia for advanced disease modeling. Dr. Christopher Cook, Concept Life Sciences bit.bio #iPSCs #diseasemodeling #neurodegenerativediseases #microglia #therapeutics

🧠 Exciting research Alert! 🌟 Our team leader Christopher Cook just delivered a thought provoking discussion on the future of drug discovery using Human iPSCs. 🧪🔬 In this discussion he highlighted the game-changing potential of Human iPSCs in revolutionizing the way we approach drug discovery. 🚀 Especially for neuroscientists, these cells open doors to elusive cell types and disease-related traits, unlocking new avenues for research. 💡 With the challenges that neuroscience drug discovery faces, the advancements in iPSC quality and reliability could hold the key to transforming the narrative. 🔄 By harnessing the power of iPSCs, we might just be on the brink of accelerating breakthroughs in understanding and treating neurological diseases. Download the article below. https://lnkd.in/erNk5tzr #research #iPSC #DrugDiscovery #Neuroscience

The study published in the Annals of Neurology, titled "Disease-Modifying Drugs Extend Survival in Hereditary #Transthyretin Amyloid Polyneuropathy," sheds light on the effectiveness of treatments for familial #amyloid polyneuropathy (FAP). 🧑🧑🧒🧒 FAP, caused by mutations in the transthyretin gene, results in the accumulation of toxic protein clumps in nerve tissues, leading to debilitating #symptoms. While there is no cure, treatment options include disease-modifying drugs (DMDs) and liver transplants. 🩺 🔬Researchers analyzed data from 201 FAP patients over three decades, observing significant differences in survival outcomes among those receiving treatment versus those untreated. Notably, nearly all treated #patients were estimated to be alive ten years after disease onset, compared to less than half of untreated patients. 📣 DMDs, such as #Onpattro, #tafamidis, and #diflunisal, showed promising results in improving survival, regardless of the age at disease onset. Even patients with late-onset FAP experienced better survival with DMDs, challenging the notion that liver transplants were the primary treatment for early-onset cases. ☑️ While liver transplants prolonged survival in early-onset FAP, their impact on late-onset cases was less significant. The study highlights the importance of DMDs in improving long-term survival for FAP patients, regardless of disease onset age or symptom type. 📊 However, the analysis is limited to a single center, and further studies are needed to validate these findings across different populations and medical settings. ⚠️ Find the article below👇🏻 https://urlr.me/X1tHm

🔬 Unlocking Insights into Multiple Sclerosis: The Crucial Role of Radiopharmaceuticals A recent study from Brigham and Women's Hospital utilizes 8F-PBR06-PET to provide new insights into the nature of Microglial Activation (MA) in Multiple Sclerosis (MS) Patients. They found "smoldering" inflammation even in patients with highly efficacious disease-modifying treatments (DMTs). Microglial Activity (determined by logarithmically transformed "glial activity load on PET") correlated with clinical disability, serum biomarkers, and cortical degeneration Key Findings: Elevated Glial Activity Load: Cortical gray matter (CoGM) and white matter (WM) showed significantly higher glial activity in MS patients compared to healthy controls, underscoring the pervasive nature of MA in the disease. Impact of Treatment: High-efficacy DMTs exhibited a notable reduction in MA compared to lower-efficacy treatments. However, residual MA persisted, emphasizing the need for next-generation therapies Conclusions The persistence of "residual" MA highlights ongoing challenges in achieving complete remission of MS. Importantly, individualized mapping of translocator protein using PET emerges as a promising imaging biomarker, offering a glimpse into the "smoldering" inflammation characteristic of MS. This can provide a new measure of severity, provides deep insights into MS neuropathophysiology, and will be part of how "complete" remission will be measured #radiopharmaceuticals #multiplesclerosis #MS #microglia #PET #radioimaging #biotech #imaging #brain #neuroinflammation

It is such an honor to work with purpose-driven clients like Quanterix every day.    Recently, the FDA approved “Leqembi”, the only disease-modifying therapy fully approved for Alzheimer’s Disease treatment. Following the approval, in which Quanterix’s ptau-181 assay played a supporting role, Quanterix announced LucentAD, a test to assist in the evaluation of patients experiencing cognitive symptoms consistent with the early signs of Alzheimer’s disease (AD). In their recent press release they shared, “The LucentAD test, which will be available to healthcare providers as an aid in conjunction with other diagnostic tools, provides clinicians with a simplified process to quickly assess the likelihood of a patient having amyloid pathology consistent with AD. This information will help healthcare providers determine appropriate follow up and treatment planning for a suspected Alzheimer’s patient.”   To learn more about Leqembi and the role of ptau-181 check out their blog here: https://lnkd.in/eXkSf-J6   The LucentAD test is run by Quanterix’s CLIA laboratory, which powers many of the clinical trials associated with AD. Lucent Diagnostics is Quanterix’s new healthcare provider-facing portal, launched to meet the needs of patients at the same time a therapy for the disease has become more widely available. To learn more about the LucentAD test, see here: https://lnkd.in/eF5wMhQB #Alzheimers #neurology #biomarkers #LifeAtPAN