Corticobasal degeneration

NEW YORK, March 14, 2024 /PRNewswire/ -- CurePSP has awarded its latest Pathway and Pipeline Grants, totaling approximately $400,000. Recipients of the grants are: Dr. Kathryn Bowles, group leader at University of Edinburgh; Dr. Maura Malpetti, research fellow at University of Cambridge; Dr. Jessica Rexach, assistant professor at UCLA; and Dr. Yang You, assistant professor at Mayo Clinic Jacksonville. The studies will make progress in understanding the basic neurobiology of progressive supranuclear palsy (PSP) and corticobasal degeneration (CBD) and in developing biomarkers to improve diagnosis and treatment. The latest Pathway grants will fund two projects: "Investigating Mitochondrial Dysfunction as an Early Pathogenic Mechanism of Primary Tauopathies, Using MAPT Splicing Mutation iPSC-organoids" by Dr. Kathryn Bowles, and "Defining the Unique Immunogenetic Landscape of PSP Compared to Related Dementias" by Dr. Jessica Rexach. The newest Pipeline grants will fund two projects: "Blood-based Fingerprints of Neuroinflammation in People with PSP and CBD" by Dr. Maura Malpetti, and "Characterization of Plasma-derived Extracellular Vesicle Proteomics for Biomarker Development of PSP and CBD" by Dr. Yang You. "Together, these grants reflect CurePSP's commitment to funding junior investigators working in high-priority research areas that will help advance our understanding and treatment of PSP and CBD," said Dr. Jennifer Brummet, associate director of scientific affairs and partnerships at CurePSP. "There is a critical need to improve our understanding of PSP and CBD and to improve diagnosis, and these grants will help move the field forward." About CurePSP's Research Grants Pathway and Pipeline Grants provide seed funding to support innovative projects focused on the fundamental neurobiology and mechanisms of tauopathies ("Pathway"), and translational projects focusing on new therapeutics and biomarkers for tauopathies ("Pipeline"). The next deadline for Pathway and Pipeline Grant applications is March 28, 2024. Learn more about our research grants here. About CurePSP CurePSP is the leading nonprofit organization dedicated to the awareness, care and cure for three neurodegenerative diseases: progressive supranuclear palsy (PSP), corticobasal degeneration (CBD) and multiple system atrophy (MSA). As a catalyst for new treatments and a cure, CurePSP establishes important partnerships and funds critical research internationally. Through its advocacy and support efforts, CurePSP enhances education, care delivery and quality of life for people living with PSP, CBD and MSA and their families. Science, community and hope are at the heart of CurePSP's mission and all its services. CurePSP is a registered 501(c)(3) charity within the United States (EIN: 52-1704978). For more information, please visit . Contact: Kristophe Diaz, PhD Executive Director and Chief Science Officer 646-725-1453 [email protected] SOURCE CurePSP, Inc.

Findings Presented at 16th Clinical Trials on Alzheimer's Disease Conference ST. LOUIS--(BUSINESS WIRE)-- Scientists, clinicians, and others taking part in the 16th Clinical Trials on Alzheimer's Disease (CTAD) conference learned the latest insights about C2N Diagnostics, LLC’s Precivity™ line of blood tests to facilitate earlier and more accurate diagnosis of Alzheimer’s disease pathology by healthcare providers. CTAD attendees heard about clinical and analytical data relating to C2N’s proprietary portfolio at 10 sessions and from three scientific posters. PrecivityAD® Blood Test Showed Economic Utility in the Evaluation of Cognitive Impairment C2N Senior Medical Advisor Dr. Mark Monane presented details about how the PrecivityAD® blood test is cost-saving when compared to the current standard approaches for evaluating cognitive impairment in older patients. His data showed a substantial cost savings of $611 per case identified at a 40% adoption rate of testing; this is the first study of economic utility for a blood biomarker used for improving Alzheimer’s disease (AD) diagnosis. The finding underscores the desirable features of the PrecivityAD blood test in the diagnostic pathway of Alzheimer’s disease: this blood biomarker is accessible, budget-conscious, and comparable in test performance to usual care pathways, said Dr. Monane. Dr. Joel Braunstein, CEO of C2N Diagnostics, said, “This year’s CTAD conference was a great success for C2N Diagnostics and the field as a whole. Many independent researchers and collaborators presented important findings about our Precivity line of blood tests, affirming these clinical tools are aiding healthcare providers in the diagnosis and treatment of Alzheimer’s disease. The tests are also helping researchers address key biological questions about the disease, who is at elevated risk, and how blood tests can help in everyday clinical practice. As the healthcare sector faces continuing cost pressures, we’re also proud that our tests are showing their value in providing clear economic benefits.” PrecivityAD2™ Blood Test Demonstrated Analytic and Clinical Validity Tim West, Ph.D., Vice President, Research and Development, C2N Diagnostics, explained that the PrecivityAD2 test, launched in August 2023 for clinical use as a CLIA LDT, has an overall percent agreement with amyloid PET (positron emission tomography) of 88% using a single test cutoff value as well as diagnostic performance that rivals that of visual read interpretation of amyloid PET for predicting neuropathology. The test incorporates the Aβ42/40 ratio and p-tau217/np-tau217 ratio (%p-tau217) to produce the Amyloid Probability Score 2 (APS2), a likelihood score that a patient will have brain amyloidosis as defined by a centiloid value greater than 25. Compared to p-tau217 concentration, %p-tau217 is significantly better at detecting brain amyloidosis. C2N Biomarker Test Data Shared by Collaborators At CTAD, Eisai Co., Ltd. presented data using the PrecivityAD blood test that demonstrated rapid change in plasma Aβ42/40 after three months using Leqembi®, an FDA-approved treatment for people with mild cognitive impairment (MCI) or mild dementia due to Alzheimer's disease. Robert Rissman, Ph.D., Professor of Physiology and Neuroscience and the W.M. Keck Endowed Professor in Medicine at the Keck School of Medicine of University of Southern California, shared findings from the AHEAD 3-45 global prevention study, which is examining whether intervention with lecanemab initiated during the asymptomatic stage can slow biomarker changes and/or new-onset cognitive impairment. Rissman reported the C2N Aβ42/40 and %p-tau217 measures showed high predictability for amyloid PET in AHEAD 3-45 study participants when used in conjunction with the AHEAD algorithm or C2N’s APS2 algorithm. In contrast, p-tau217 and p-tau181 peptides or ratios alone, without the inclusion of AB42/40, demonstrated lower sensitivity and accuracy in detecting amyloid PET-positive participants. The proposed cut points allow for the detection of participants with >20 centiloids with over 90% accuracy when compared to AB42/40 or the p-tau217 ratio alone. This increased sensitivity can reduce the number of screen failures resulting from negative amyloid PET scans, ultimately lowering the burden and cost for both study sites and participants. The use of plasma prescreening can also enhance the inclusion of diverse groups in the AHEAD study. Findings from this analysis were published Nov. 6 in the online edition of Alzheimer’s & Dementia. Suzanne Schindler, M.D., Ph.D., Associate Professor of Neurology, Washington University School of Medicine, gave an overview of the acceptable performance standards for blood biomarkers of Alzheimer's disease in clinical use for symptomatic patients, including the Precivity portfolio of blood tests. She explained research that found a combination of C2N’s %p-tau217 and Aβ42/40 measures, similar to the APS2 score, accurately classified amyloid status with an AUC of 0.96 as compared to the reference standard of PET biomarkers or cerebrospinal (CSF) biomarkers. The study involved a diverse cohort of 746 older individuals. Dr. Schindler said that race, sex, age, body mass index, and common comorbidities like hypertension and diabetes did not affect the classification of amyloid status by the combination plasma %p-tau217 and Aβ42/40. Black individuals participating in research studies may have a lower rate of amyloid positivity at baseline, which could decrease the proportion of Black individuals eligible for AD prevention trials and treatment trials for AD dementia, according to research from Chengjie Xiong, Ph.D., Professor of Biostatistics, Washington University School of Medicine. He further detailed that there were no significant racial differences in the longitudinal rates of change for plasma Aβ42/40. These findings, if confirmed by larger and more representative studies across the entire spectrum of social determinants of health, may allow future AD prevention and treatment trials to target a shared efficacy endpoint, according to Xiong. The PrecivityAD2™/PrecivityAD® blood tests are intended for use in patients aged 55 and older with signs or symptoms of mild cognitive impairment or dementia who are undergoing evaluation of Alzheimer’s disease or dementia. C2N Introduced for Research Use Only Test That Can Measure Microtubule Binding Region of Tau (MTBR-tau) During CTAD, C2N introduced a much-desired, proprietary biomarker test for Research Use Only (RUO) that can measure the microtubule binding region of tau (MTBR-tau). This analyte measurement was engineered with the intention of uniquely detecting the presence and burden of neurofibrillary “tau” tangles (NFTs) comprised of aggregated tau proteins in the brain and as a potential alternative to invasive, costly, and often inaccessible tau PET tracers. MTBR-tau is critical for tau protein aggregation and is an enriched core component of NFTs, one of the pathological hallmarks that accumulate in the brain of individuals with Alzheimer’s disease (AD). The MTBR-tau fluid biomarker can be used in the diagnosis of AD, in disease staging, and in the development of tau-directed therapeutics. Furthermore, the MTBR-tau biomarkers have the potential to advance the diagnosis of non-AD tauopathies, such as corticobasal degeneration (CBD), frontotemporal dementia (FTD), and progressive supranuclear palsy (PSP). About C2N Diagnostics, LLC C₂N is a specialty diagnostics company with a vision to bring Clarity Through Innovation®. C₂N strives to provide exceptional laboratory services and products in the field of brain health. C₂N’s high-resolution mass spectrometry-based biomarker services and products are used for: clinical decision-making to improve patient care, including diagnosis and treatment monitoring; maximizing the quality and efficiency of clinical trials that test novel treatments for neurodegeneration; and providing innovative tools to help healthcare researchers better understand novel mechanisms of disease, identify new treatment targets, and conduct important epidemiologic studies to improve global public health. C₂N assays have been used in over 100 Alzheimer’s disease and other research studies throughout the U.S. and the world. This includes landmark treatment and prevention trials involving disease-modifying therapies (DMTs) that are changing the trajectory of Alzheimer’s disease. C₂N has ongoing collaborations with multi-national pharmaceutical and biotech companies, leading academic institutions, National Institute on Aging, Alzheimer’s Association, and other non-profits and consortiums. Over 15,000 Precivity™-related biomarker measures have been reported through peer-reviewed publications, with many more manuscripts currently under review. The company acknowledges generous support from National Institute on Aging, GHR Foundation, Alzheimer’s Drug Discovery Foundation, BrightFocus Foundation and Alzheimer’s Association. For more information visit .

New preclinical data presented at CTAD demonstrates exciting potential for a new drug approach for Alzheimer's disease, reducing brain tau pathology by ~70% CAMBRIDGE, Mass., Oct. 30, 2023 /PRNewswire/ -- Aquinnah Pharmaceuticals announced preclinical research findings for a novel therapeutic designed to slow or stop the progression of Alzheimer's disease and related disorders. Aquinnah is a leader in pharmaceutical approaches targeting stress granule biology, which underlies the pathology of a wide range of neurodegenerative disorders. The Aquinnah team announced today that their lead compound targets the interaction of tau with stress granules and removes ~70% of tau pathology in an Alzheimer's animal model with advanced stage disease, measured using three different markers of pathological tau that also increase in patients as their disease progresses. Importantly, the Aquinnah compound is expected to be administered orally as a pill, which is generally preferred by patients over injections that are required currently for approved Alzheimer's immunotherapies. Administration of the compound once daily in preclinical models was sufficient to achieve a strong reduction of abnormal tau protein, which is the key protein contributing to loss of memory and cognition in Alzheimer's disease. Because Aquinnah's compound acts through a unique approach, this new compound also has the potential to be used in combination with anti-amyloid drugs, recently approved by the FDA. If successful in clinical trials, Aquinnah's treatment could benefit patients suffering from Alzheimer's disease by reducing brain damage, restoring memory capabilities, improving brain function and slowing disease progression. The company's Co-Founder and Chief Scientific Officer, Dr. Benjamin Wolozin MD, Ph.D., presented the research – "Development of Orally Available, Brain Penetrant Compound Reducing Tau Pathology" – at the 2023 Clinical Trials on Alzheimer's Disease (CTAD) conference last week in Boston. "As the number of patients and families impacted by Alzheimer's disease continues to grow worldwide, new medical approaches to target tau pathology, alone or in combination with anti-amyloid therapeutics, will benefit these patients greatly. Research from around the world, including the numerous presentations at CTAD, show that decreasing pathological tau, correlates strongly with improved cognitive function," said Dr. Wolozin. "We're excited about these results and the potential to give Alzheimer's patients a therapeutic agent that will not just delay the disease's progression, but potentially improve their cognitive functioning. Our success with animal models, equivalent to advanced stage Alzheimer's patients, suggests that our compound has exactly this ability to turn the tide for patients suffering from cognitive loss and have or are facing impending dementia caused by Alzheimer's disease." In addition to its application to Alzheimer's disease, Aquinnah's therapeutic is a potential treatment for other diseases with tauopathies, including Progressive Supranuclear Palsy (PSP), Corticobasal Degeneration (CBD), Frontotemporal Lobar Dementia (FTD) and Traumatic Brain Injury (TBI). Aquinnah's CEO, Glenn Larsen, Ph.D., added, "Alzheimer's disease affects millions of patients and their families each year, so these results and their potential impact are a significant and promising step forward in the fight against this pervasive disease. More importantly, our approach contrasts to existing and developing therapies which are administered through injections. As a once-a-day pill, it will be a preferred method for patients, easier to take early in disease to prevent progression and be less costly compared to injectable drugs. Our strong results have attracted significant interest from the industry to collaborate with us in advancing the development of this promising therapeutic." Focused on innovative neurodegenerative research, Aquinnah Pharmaceuticals signed a collaboration agreement with Roche in 2022 to advance oral small molecules for ALS and other neurodegenerative diseases, by modulating TDP-43 pathology, which is the hallmark pathology in more than 95% of ALS patients. Aquinnah has received funding from Pfizer, AbbVie and Takeda, with additional grant funding from the National Institute of Health, the Alzheimer's Association, The Rainwater Foundation and the Mass Life Sciences Center. About Aquinnah Pharmaceuticals Aquinnah Pharmaceuticals is harnessing the power of stress granule biology to develop oral small molecule therapeutics in Neurodegenerative Diseases, focusing on Alzheimer's disease and other tauopathy diseases such as Progressive Supranuclear Palsy and Frontotemporal dementia and ALS, also known as Lou Gehrig's disease, by developing chemical modulators of stress granule pathology. More information is available at . SOURCE Aquinnah Pharmaceuticals