vimentin

N-acetylcysteine (NAC) shows promise to treat hereditary cystatin C amyloid angiopathy (HCCAA) HCCAA – a subtype of cerebral amyloid angiopathy (CAA) – is an ultra-rare Icelandic amyloid disease due to a mutation in the CST3 gene encoding for the protease inhibitor, cystatin C. HCCAA causes cerebral hemorrhages in asymptomatic carriers of the L68Q variant due to amyloid aggregation with precipitation in brain vessels and tissue. Findings could guide the development of preventative therapies for related CAAs. REYKJAVIK, Iceland, April 2, 2025 /PRNewswire/ -- Icelandic drug discovery and development company Arctic Therapeutics (ATx) today announced the publication of research in JAMA Neurology, which informs a possible therapeutic strategy to treat a rare form of familial dementia. The publication, titled N-Acetylcysteine for hereditary cystatin C amyloid angiopathy (HCCAA), a nonrandomised clinical trial, details a new treatment approach for HCCAA, a rare and fatal genetic brain disorder that primarily affects young Icelandic adults, with the disease process beginning in childhood. The research provides insights into the potential of N-acetylcysteine (NAC) as a treatment for HCCAA, a disease with no currently approved therapeutic options. The study highlights how NAC may reduce disease-associated biomarkers and slow disease progression. The study is the first phase 2a clinical trial to assess the safety and therapeutic potential of high-dose NAC in patients carrying the L68Q-CST3 sequence variation of HCCAA. Results indicate that NAC is well tolerated, reduces disease specific biomarkers and stabilises disease progression, suggesting NAC may offer a preventative strategy against HCCAA. These findings could accelerate the development of preventative therapies for individuals at risk of HCCAA and other forms of CAAs. "These promising results provide hope to HCCAA patients and their families," Dr Ásbjörg Ósk Snorradóttir, lead author of the study explained. Senior author, Dr. Hákon Hákonarson, director of the Center for Applied Genomics at the Children's Hospital of Philadelphia and founder of Arctic Therapeutics added: "This study provides the first clinical evidence that NAC, by breaking disulfide bonds and other aberrant molecular interactions, can disrupt cystatin C oligomers and prevent amyloid aggregation that ultimately accumulates in brain vessels. By targeting the root biochemical process behind HCCAA pathology, NAC may ultimately offer a novel disease-modifying approach, not only for this devastating condition, but potentially for other forms of CAAs and protein aggregation disorders where toxic oligomers are central to disease progression." The open-label phase 2a clinical trial was conducted at Iceland's Landspitali University Hospital from March 2019 to December 2021. A total of 17 participants were enrolled, all confirmed carriers of the L68Q-CST3 mutation. The study assessed: Safety & tolerability: NAC was well tolerated, with no drug-related severe adverse events. Five participants experienced cerebral hemorrhages, but none resulted in permanent neurological damage. Biomarker reduction: Significant reductions were observed in collagen IV, fibronectin, vimentin, and SMAD levels—key indicators of disease progression. Plasma & skin biomarker changes: NAC was shown to increase reduced glutathione levels, a protective antioxidant, and reduce high-molecular-weight cystatin C aggregates, which are linked to amyloid buildup in the brain. Cognitive stability: No measurable decline in cognitive function was observed during the study period. Building on these findings, as well as internal in-vitro data that demonstrates that NAC-amide (NACA) has more than 10-fold higher potency in preventing or dispersing amyloid complex aggregates than NAC and increased ability to penetrate the blood brain barrier and get into cells involved in the disease pathogenesis, ATx has launched a European Medicines Agency-approved registration study to monitor safety, tolerability, and efficacy using NACA in patients with HCCAA. For more details, read the full study in JAMA Neurology: Original Investigation. About Arctic Therapeutics Arctic Therapeutics is an Icelandic drug discovery and development company established in 2015, which has licensed intellectual property from Children's Hospital of Philadelphia. ATx leverages the transformative power of applied genomics, steering the course of drug development towards safer, more effective treatments for some of the world's most challenging diseases. The company has operations in Iceland, the US and multiple collaborations across Europe. For more information, please visit: and follow us on LinkedIn For media inquires Gulli Arnason, Chief Strategy Officer [email protected] +354 660 0053 This information was brought to you by Cision The following files are available for download: WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Patent covers proprietary method using methylation of the CCNA1 gene to help detect esophageal precancer and cancer NEW YORK, Oct. 15, 2024 /PRNewswire/ -- Lucid Diagnostics Inc. (Nasdaq: LUCD) ("Lucid" or the "Company"), a commercial-stage, cancer prevention medical diagnostics company, and subsidiary of PAVmed Inc. (Nasdaq: PAVM), today announced that it has received a Notice of Allowance from the United States Patent and Trademark Office (USPTO) for a patent application covering its proprietary method of using methylation of the cyclin-A1 (CCNA1) gene to help detect esophageal precancer and cancer, a key component of its EsoGuard® Esophageal DNA Test. "This patent strengthens the already robust intellectual property portfolio underlying our groundbreaking technologies and will provide strong protection for the CCNA1 methylation assay method which is at the heart of the EsoGuard assay," said Lishan Aklog, M.D., Lucid's Chairman and Chief Executive Officer. "The allowance validates our strategy of focusing on the laboratory methods of our assay—a strategy we will continue to aggressively pursue for other components of the assay." EsoGuard utilizes next-generation sequencing (NGS) to assess DNA methylation at 31 sites on two genes, vimentin (VIM) and cyclin-A1 (CCNA1). Such methylation has been shown to be strongly associated with conditions along the spectrum from early esophageal precancer (non-dysplastic Barrett's Esophagus or BE), to late precancer (dysplastic BE), to cancer (esophageal adenocarcinoma). Although VIM methylation had been previously associated with gastrointestinal neoplasias, the association of CCNA1 methylation with esophageal neoplasia is novel and appears to be more specific. The technology behind EsoGuard and the EsoCheck® Esophageal Cell Collection Device was developed at Case Western Reserve University (CWRU) by faculty members Sanford Markowitz, M.D., PhD, Amitabh Chak, M.D., and Joseph Willis, M.D., with the support of research funds from the National Institutes of Health (NIH) and elsewhere. Lucid holds the exclusive worldwide license to commercialize this technology. The successful transfer of the assay from the academic research laboratory and its subsequent commercialization was the result of close, ongoing collaboration between Lucid and CWRU. About Lucid Diagnostics Lucid Diagnostics Inc. is a commercial-stage, cancer prevention medical diagnostics company and subsidiary of PAVmed Inc. (Nasdaq: PAVM). Lucid is focused on the millions of patients with gastroesophageal reflux disease (GERD), also known as chronic heartburn, who are at risk of developing esophageal precancer and cancer. Lucid's EsoGuard® Esophageal DNA Test, performed on samples collected in a brief, noninvasive office procedure with its EsoCheck® Esophageal Cell Collection Device, represent the first and only commercially available tools designed with the goal of preventing cancer and cancer deaths through widespread, early detection of esophageal precancer in at-risk patients. For more information about Lucid, please visit and for more information about its parent company PAVmed, please visit . Forward-Looking Statements This press release includes forward-looking statements that involve risk and uncertainties. Forward-looking statements are any statements that are not historical facts. Such forward-looking statements, which are based upon the current beliefs and expectations of Lucid's management, are subject to risks and uncertainties, which could cause actual results to differ from the forward-looking statements. Risks and uncertainties that may cause such differences include, among other things, volatility in the price of Lucid's common stock; general economic and market conditions; the uncertainties inherent in research and development, including the cost and time required to advance Lucid's products to regulatory submission; whether regulatory authorities will be satisfied with the design of and results from Lucid's clinical and preclinical studies; whether and when Lucid's products are cleared by regulatory authorities; market acceptance of Lucid's products once cleared and commercialized; Lucid's ability to raise additional funding as needed; and other competitive developments. In addition, Lucid continues to monitor the COVID-19 pandemic and the pandemic's impact on Lucid's businesses. These factors are difficult or impossible to predict accurately and many of them are beyond Lucid's control. In addition, new risks and uncertainties may arise from time to time and are difficult to predict. For a further list and description of these and other important risks and uncertainties that may affect Lucid's future operations, see Part I, Item 1A, "Risk Factors," in Lucid's most recent Annual Report on Form 10-K filed with the Securities and Exchange Commission, as the same may be updated in Part II, Item 1A, "Risk Factors" in any Quarterly Report on Form 10-Q filed by Lucid Diagnostics after its most recent Annual Report. Lucid disclaims any intention or obligation to publicly update or revise any forward-looking statement to reflect any change in its expectations or in events, conditions, or circumstances on which those expectations may be based, or that may affect the likelihood that actual results will differ from those contained in the forward-looking statements. SOURCE Lucid Diagnostics WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

BORDEAUX, France--( BUSINESS WIRE )--RebrAIn, a pioneering precision neuro-surgery platform company, has recently received its second FDA 510(k) clearance for its OptimMRI software, which now includes a new machine learning model to assist in targeting the infero-lateral part of the VIM for lesioning techniques such as MRgFUS and Radiosurgery. This new software version already features FDA-cleared machine learning models for the STN and VIM regions of interest to assist in targeting Deep Brain Stimulation (DBS). RebrAIn has secured its second FDA 510(k) clearance in the past year, expanding the software's capabilities to include the infero-lateral part of the VIM. RebrAIn’s software as a medical device (SaMD) is designed to revolutionize neurosurgical target planning by combining advanced AI algorithms, trained on clinical data, with MRI sequences to provide neurosurgeons with the most optimal targets for lesioning and electrode placement. David Caumartin, CEO of RebrAIn, stated, “Our extension to US customers to better plan targets for lesioning is a significant step in RebrAIn’s growth and strategic transformation. The US market represents the largest opportunity to enable personalized targeting for neurological disorders such as Essential Tremor. The ability to offer AI clinical targeting to neurosurgical suites will open many collaborations nationwide, which today are treated with DBS and MR-guided focused ultrasound. Since our recent discussions at US congresses, it was necessary for us to offer neurosurgeons a complete service.” RebrAIn’s co-founders, Prof. Emmanuel Cuny MD and Prof. Nejib Zemzemi PhD, will be presenting on “VIM Lesions in Radiosurgery and Focused Ultrasound: Assessment of the Accuracy of Two Targeting Models Based on Machine Learning” at the World Society for Stereotactic and Functional Neurosurgery (WSSFN) biennial meeting in Chicago, IL, on September 5, 2024. “Our extension to US customers to better plan targets for lesioning is a significant step in RebrAIn’s growth and strategic transformation,” said David Caumartin, CEO of RebrAIn. “The US market is the largest, and the ability to offer AI clinical targeting will open many national collaborations.” To learn more, visit us at Booth #9 during the WSSFN meeting, check out our website www.rebrain.eu , or contact us directly. About RebrAIn RebrAIn is a France-based company that enables precise targeting for DBS and lesioning treatments for patients suffering from severe Parkinson's or essential tremor disease. Its SaaS solution helps neurosurgeons to precisely identify the target area for surgical intervention. Its novel approach uses machine learning of clinical patient data to predict the optimal treatment zone in each patient’s brain. For more information, visit www.rebrain.eu . About the World Society for Stereotactic and Functional Neurosurgery Since 1961, the WSSFN has been regularly held and continues to be a leading neurosurgery conference. At the biennial meetings, the top leaders in the field present the most recent innovations in clinical and basic research as well as new and effective ways to provide material support. Meta Description: "RebrAIn receives its second FDA 510(k) clearance for OptimMRI software, enhancing targeting in stereotactic and functional neurosurgery for Deep Brain Stimulation and lesioning techniques. The company will present VIM targeting advancements at the WSSFN congress." Tags: FDA 510(k) Clearance, Deep Brain Stimulation (DBS), Stereotactic Neurosurgery, Functional Neurosurgery, Medical Device Software, Machine Learning in Healthcare, MR-guided Focused Ultrasound (MRgFUS), Radiosurgery, Neurotechnology, WSSFN Congress, RebrAIn, Essential Tremor Treatment, AI in Healthcare, Precision Neurosurgery, Healthcare Innovations.