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Clinical Trials Updates: Annovis Bio, Inc. announces positive phase II results for their Alzheimer's Disease small molecule Alzheimer's has definitely been the primary focus of the neurology world for 2023/2024, after Eisai US launch last summer. Yesterday, Annovis Bio, Inc. shared encouraging results from its study of buntanetap for Alzheimer's disease (AD). Patients receiving buntanetap showed significant cognitive improvement compared to those on a placebo, with a notable reduction in plasma Tau protein levels, a marker of AD progression. This is bacuse Buntanetap can selectively inhibit transcriptions of harmful proteins associated with the disease, effectively reducing their production. The study involved over 700 patients, with those receiving buntanetap showing better cognitive function after 12 weeks. These findings are encouraging, leading Annovis Bio to plan a larger phase III trial in early-stage AD patients. Congrats to them for reaching this importnat milestone! #Neuro #medicalaffairs #clinicaldevelopment #clinicaltrials #medicaljobs #clinicaljobs https://lnkd.in/eYtpzEd5

The Alzheimer’s disease research community have been trying to identify predictive biomarkers for early detection of neurodegeneration for many years. Here, the authors report that, in a multicenter human cohort, brain-derived phosphorylated-tau (BD-tau - a structural component of axons) in blood increases concomitant with Aβ and neurodegenerative abnormalities detected in cerebrospinal fluid. They demonstrate that blood-based BD-tau is a biomarker for identifying Aβ-positive individuals at risk of short-term cognitive decline and atrophy. This biomarker will be useful for future clinical trials, especially those testing anti-Aβ therapies. Plasma brain-derived tau is an amyloid-associated neurodegeneration biomarker in Alzheimer’s disease https://lnkd.in/gXMnsYzh

More than 100,000 Canadians currently live with Parkinson’s disease. A novel drug molecule being studied by researchers from McGill University could reactivate housekeeping functions in brain cells of young Parkinson’s patients, paving the way for potential future treatments for this incurable, degenerative disease. “We are excited about this drug compound because it raises the possibility of a cure for Parkinson’s disease for a subset of patients,” said Kalle Gehring, a biochemistry professor in the McGill School of Biomedical Sciences. Developed by the biotech company Biogen, the new compound has shown promising results activating parkin, a key protein in the brain responsible for “cleaning up” and recycling damaged mitochondria – the energy powerhouse of the cell. When parkin doesn’t work properly, these damaged mitochondria accumulate, leading eventually to Parkinson’s disease. In studies published in 2013 and 2018, Gehring shed light on the functions of parkin based on data collected at the CLS. In this new follow-up study, Gehring used the CMCF beamline at the CLS to determine how the Biogen compound activates parkin. They found that it glues together parkin and a natural activator present in the cell. This molecular-level information is essential for the drug's future development. “The way the drug molecule turns on parkin is through a secondary route, which is effective for specific mutations of parkin that occur in younger patients,” he said. After turning proteins into tiny crystals, Gehring and his team used a technique called protein crystallography to identify their 3D structures and learn where the novel drug binds and how it affects the proteins. The results are published in the journal Nature Communications. “We need quality data to solve the protein structures and see their 3D pictures. It takes a facility like the CLS to take Canadian research to an international level,” said Gehring. While Parkinson’s symptoms – slowed movements, tremors, and balance problems – often appear in people in their 60s, onset at an earlier age is not uncommon and five to 10 per cent of people diagnosed are affected before they turn 40. “The hope is that one day we’ll find compounds that can treat Parkinson’s disease in general,” said Gehring. “As the population in Canada is aging and better treatments for other diseases are becoming available, neurodegenerative diseases such as Parkinson’s will be a major health concern.” This study involved Biogen, Gehring, and McGill research groups led by professors Edward A. Fon and Jean-François Trempe. This project was sparked from a research exchange organized by the The Michael J. Fox Foundation for Parkinson's Research, which provided funding for the project along with the Canadian Institutes of Health Research | Instituts de recherche en santé du Canada and the Canada Research Chairs program. https://bit.ly/47Aee1P #parkinsons #parkin #moleculeglue

Advancements in Redox-Active Nanozymes! This study highlighted nanozymes' role in modulating cellular redox status, offering promising avenues for addressing oxidative-stress-related disorders. The latest findings on nanozymes' ability to emulate antioxidant enzymes in mammalian cells were discussed, showcasing their therapeutic potential in conditions like cancer, neurodegeneration, and cardiovascular diseases. These data shed light on the potential of nanozymes to revolutionize therapeutic interventions in oxidative-stress-mediated disorders, offering hope for improved treatment modalities in the future. 🌐 Read more: https://lnkd.in/dDGrxR3c #RedoxMedicine 2024 will introduce you to the latest redox innovations. Don't miss out! Join us this June in Paris to learn more! #RedoxMedicineSociety #RedoxMedicine #biomarkers #redoxbiology #redox #antioxidants #innovation #oxidativestress #Nanozymes #RedoxResearch #BiomedicalInnovation

Lilly's Kisunla™ (donanemab-azbt) Approved by the FDA for the Treatment of Early Symptomatic Alzheimer's Disease TRAILBLAZER-ALZ 2 is a Phase 3, double-blind, placebo-controlled study to evaluate the safety and efficacy of N3pG antibody (donanemab) in participants with early symptomatic AD (prodromal AD and mild dementia due to AD) with the presence of brain amyloid and tau pathology. Active arm: Participants received 700 milligram (mg) Donanemab every 4 weeks (Q4W) x 3 doses, then 1400 mg Q4W given intravenously (IV) for up to 72 weeks Those individuals treated with Kisunla who were less advanced in their disease showed a significant slowing of decline of 35% compared with placebo on the integrated Among the overall population of participants, Kisunla reduced amyloid plaques on average by 61% at 6 months, 80% at 12 months, and 84% at 18 months compared to the start of the study. One of the treatment goals of the study was to remove amyloid plaques to minimal levels consistent with a visually negative scan using amyloid positron emission tomography (PET) Kisunla can cause amyloid-related imaging abnormalities (ARIA), which is a potential side effect with amyloid plaque-targeting therapies that does not usually cause symptoms. It can be detected via magnetic resonance imaging (MRI) scans and, when it does occur, may present as temporary swelling in an area or areas of the brain, which usually resolves over time, or as small spots of bleeding in or on the surface of the brain. Infrequently, larger areas of bleeding in the brain can occur.1, 2 ARIA can be serious, and life-threatening events can occur. The price of each vial of Kisunla is $695.65, 18 months of treatment costs USD 48,696. https://lnkd.in/gcj6ReB7

📃Scientific paper: Inhibition of colony stimulating factor-1 receptor (CSF-1R) as a potential therapeutic strategy for neurodegenerative diseases: opportunities and challenges Abstract: Microglia are specialized dynamic immune cells in the central nervous system (CNS) that plays a crucial role in brain homeostasis and in disease states. Persistent neuroinflammation is considered a hallmark of many neurodegenerative diseases, including Alzheimer’s disease (AD), Parkinson's disease (PD), Huntington’s disease (HD), amyotrophic lateral sclerosis (ALS) and primary progressive multiple sclerosis (MS). Colony stimulating factor 1-receptor (CSF-1R) is predominantly expressed on microglia and its expression is significantly increased in neurodegenerative diseases. Cumulative findings have indicated that CSF-1R inhibitors can have beneficial effects in preclinical neurodegenerative disease models. Research using CSF-1R inhibitors has now been extended into non-human primates and humans. This review article summarizes the most recent advances using CSF-1R inhibitors in different neurodegenerative conditions including AD, PD, HD, ALS and MS. Potential challenges for translating these findings into clinical practice are presented. Continued on ES/IODE ➡️ https://etcse.fr/mCx ------- If you find this interesting, feel free to follow, comment and share. We need your help to enhance our visibility, so that our platform continues to serve you. #amyotrophiclateralsclerosis #als #charcot

Engineering CAR-T therapies for autoimmune disease and beyond In this Review, the authors assess the current state of the field, challenges, and emerging concepts that will unleash the potential of CAR-Ts in autoimmunity and other therapeutic areas. - CAR-T technology enables precision targeting and elimination of pathogenic cells. - The landmark successes in late-stage CD19+ and BCMA+ hematological malignancies have inspired efforts to extend the application of CAR-Ts to other indications, including early-stage hematological cancers and solid tumors. - Recent seminal proof-of-concept publications in SLE and MG have catalyzed new efforts to apply CAR-T therapy in autoimmune diseases. - With 59 registered clinical trials for CAR-T therapy in autoimmune diseases as of April 2024, the pace of innovation in CAR-T development for autoantibody-driven disease is continuing to accelerate. - With the promise in autoimmune indications, CAR-T therapies are also being explored for other areas outside of oncology, such as cardiovascular disease, infectious disease, and neurology. https://lnkd.in/eVT-nZuA #CART #Celltherapy #autoimmunity #autoimmunedisease

Wonderful overview of cell therapies and their evolution.

Alzheimer's Disease: Survey of 268 Neurologists in the US and Europe: 1. Amyloid-targeted therapies: * 51% support; * 41% remain skeptical. 2. Diagnosing Alzheimer's based on blood biomarkers even before symptoms appear and prescribing the new drugs: * ~50% are ready; * 50% are skeptical. 3. Clinical benefit of biomarkers: * 5% of neurologists consider changes in biomarkers (such as imaging or blood tests that measure amyloid plaque reduction) to be a measure of clinical benefit; * 95% of neurologists overall did not consider changes in biomarkers to be a measure of clinical benefit. 4. Adverse effects of new treatments: * 71% of neurologists expressed significant concern. The study authors emphasize the importance of transparency in research and disclosure of clinical trial data. This would allow clinicians and researchers to more critically assess the benefits and risks of new treatments. My comment: The field of Alzheimer's treatment is at a time of great change and debate. New treatments targeting amyloid offer hope for patients, but also pose significant challenges. It is critical that researchers, clinicians, and regulatory agencies work together to develop safe and effective treatments for Alzheimer's disease. https://lnkd.in/dnE5HrMG

Unlocking the Future of Alzheimer’s Treatment: A Must-Read Blog by colleagues at Worldwide Clinical Trials. Alzheimer’s disease remains a formidable challenge in medicine due to its complex nature and the critical need for early detection. This blog explores groundbreaking innovations in AD therapeutics, including the integration of fluid and imaging biomarkers that are reshaping clinical trial designs. These advancements could enhance treatment windows and allow for FDA accelerated approvals. Dive into the latest breakthroughs, such as the FDA’s regulatory adaptations, providing a comprehensive overview of the current landscape and future directions in AD research. Read the full article here. #AlzheimersResearch #Biomarkers #ClinicalTrials #FDA