Istituto di Ricerche Farmacologiche Mario Negri

NORTH CHICAGO, Ill., March 26, 2024 /PRNewswire/ -- BLR Bio, an early stage biotech company in Rosalind Franklin University's Helix 51 biomedical incubator, released new data on its novel cancer therapy recently at the 7th annual Labroots Drug Discovery and Development conference. The data points to a promising strategy to boost the effectiveness of existing drugs in pancreatic cancer and other solid-tumor cancers. According to the American Cancer Society, some 66,000 persons in the U.S. are diagnosed annually with pancreatic cancer, a disease with a very low survival rate that is projected to take 51,000 lives in 2024. BLR Bio's new findings are based on a long-term research relationship between the company and oncology researchers at the Mario Negri Institute for Pharmacological Research in Milan and Bergamo, Italy, and follow work published with that team in the journal Cells in 2020. The current study, "BLR-100: Can a Novel Tumor Stroma Targeting Agent Reduce Chemotherapy Load as a Potential Effective Combination Therapy," demonstrates that the use of BLR's compound — BLR-100 — could provide similar efficacy to a doubling of the dose of existing chemotherapy without increasing side effects of these therapeutics, which often impact patient quality of life during treatment. "The high dose chemotherapy necessary for efficacy in high mortality cancers like pancreatic adenocarcinoma (PNAC) are often complicated and have high rates of toxicity," said BLR Bio CEO Dr. Bruce Riser. "Finding an approach to use lower doses while maintaining efficacy has been a constant search for cancer treatments." "While other approaches are being pursued to decrease the chemotherapy load, our approach is unique," Dr. Riser said. "BLR-100 targets the formation of the protective tumor stroma, a fibrotic-like element that is thought to retard the entry of chemo-therapeutic agents." BLR-100 is also expected to allow better access to normal immune clearance mechanisms as well as the new immuno-oncology drugs, which to date have not been effective in cancers like PNAC. BLR Bio is currently completing IND-enabling studies for this novel compound. Dr. Ronald Kaplan, RFU executive vice president for research, said, "The Mario Negri Institute is a world-renowned cancer research organization, and we are delighted to see this ongoing collaboration with one of our companies to further the development of this novel therapy for such a devastating disease." About BLR Bio: blrbio.com About RFU: rosalindfranklin.edu Office of Marketing and Communications judy.masterson@rosalindfranklin.edu

Studies seek to establish an early-stage blood biomarker, explore novel therapies and support the creation of a cohort of PSP and CBD patients in Argentina. NEW YORK, Sept. 28, 2023 /PRNewswire/ -- CurePSP has awarded its latest Pathway and Pipeline Grants and Urso Student Fellowships, totaling over $422,000. Recipients of the grants are: Dr. Kurt Farrell, assistant professor at the Icahn School of Medicine at Mount Sinai; Dr. Blas Couto, researcher at the Institute of Translational and Cognitive Neuroscience (INCyT) in Buenos Aires; Dr. Luana Fioriti, head of laboratory at the Mario Negri Institute for Pharmacological Research in Milan, alongside Dr. Carmela Tartaglia, associate professor at the University of Toronto and clinician at the Rossy PSP Centre; and Dr. Joseph B. Rayman, assistant professor of medical sciences at Columbia University Irving Medical Center. Recipients of the Urso Student Fellowship Grants are: Hania Qamar (mentor: Dr. Naomi Visanji), a graduate student at the University of Toronto; Hasnat Nuri (mentor: Dr. Stewart Clark), an undergraduate student at SUNY at Buffalo; and Soyeon Park (mentor: Dr. Jonathan Lin), an undergraduate student at Stanford University. The studies will make progress in identifying biomarkers, studying pathways for therapeutics and creating a new database of patients in Argentina. The latest Pathway grants will fund two projects: "Understanding the Roles of Risk Loci Implicated in Genome-Wide Association Studies of PSP" by Dr. Kurt Farrell, and "Epidemiology and Tau Biology in a Longitudinal Cohort of Argentinian Patients with PSP and CBD" by Dr. Blas Couto. Dr. Farrell's project will use data from a previous genetic analysis to identify specific genes and biological mechanisms that cause PSP. Dr. Couto will study a group of Argentinian patients with CBD and PSP to develop a research-ready biobank and database. The newest Pipeline grants will fund "Potential Therapeutic Biomarkers for Progressive Supranuclear Palsy" by Dr. Luana Fioriti and Dr. Carmela Tartaglia, and "Development of Small Molecule Inhibitors of Tau Oligomerization" by Dr. Joseph B. Rayman. Dr. Fioriti and Dr. Tartaglia are investigating a specific type of post-translational modification of tau that could lead to a novel PSP-specific early-stage blood biomarker. Dr. Rayman's work will continue the development of a small molecule that, via its interaction with TIA1 protein, could prevent the pathological oligomerization of the tau protein, therefore opening the way to new therapeutic approaches for tauopathies including PSP, CBD and Alzheimer's disease. Dr. Jennifer Brummet, associate director of scientific affairs and partnerships at CurePSP, hopes that these projects will lead to progress in high-priority research areas. "Together, these grants will help advance our understanding of the fundamental neurobiology of PSP and CBD while also investigating potential biomarkers and therapeutics," Dr. Brummet said. "There is a critical need for biomarkers to help ensure accurate diagnosis of PSP and CBD and for therapeutics to help treat or cure PSP and CBD, and these grants will help move the needle on these goals." The newest Urso Student Fellowship grants were awarded to students conducting projects that focus on the complex barriers facing research of PSP and CBD. "Progressive Supranuclear Palsy: A Disease-Specific Mouse Tauopathy Model" by Hania Qamar (mentor: Dr. Naomi Visanji) recognizes the lack of an animal model as a barrier to understanding PSP and aims to generate a mouse model that would be the closest replica of how PSP presents itself in the human brain. "Investigation of the Role of Non-cholinergic PPTg Neurons in a Model of PSP" by Hasnat Nuri (mentor: Dr. Stewart Clark) seeks to further develop a mouse model of PSP by administering the tau protein in the brain region known as the pedunculopontine tegmentum (PPTg) to investigate the effects of abnormal tau accumulating in this region. "Testing Anti-Tau Properties of Integrated Stress Response Boosters In Vivo" by Soyeon Park (mentor: Dr. Jonathan Lin) will study the effects of injecting mice with Integrated Stress Response (ISR) boosters to see if cell death and tau levels are reduced. The opportunities afforded by these grants will help foster the next generation of researchers dedicated to furthering our understanding of PSP, CBD and related diseases, while contributing essential information to the field. 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 Urso Student Fellowship supports students conducting summer research projects focused on PSP, CBD and related diseases with a goal of encouraging students at all post-secondary levels to pursue research in the field. The next deadline for Pathway and Pipeline Grant applications is October 31, 2023. The next deadline for Urso Student Fellowship Grants is January 31, 2024, and applications will open this fall. Learn more about our research grants here. Grant applications will be submitted to ProposalCentral and can be found 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: Dr. Kristophe Diaz Executive Director and Chief Science Officer 646-725-1453 [email protected] SOURCE CurePSP, Inc.

LONDON, May 12 (Reuters) - New highly-effective weight loss drugs such as Novo Nordisk's (NOVOb.CO) Wegovy are not a “silver bullet” for addressing the rapid rise in global obesity rates, the World Health Organization’s nutrition chief told Reuters, as the agency conducts its first review of obesity management guidelines in more than 20 years. The global health body is first revising guidelines for treating children and adolescents with obesity, and will then update recommendations for adults, said Francesco Branca, WHO director of nutrition and food safety. The WHO last issued global guidelines on the topic in 2000, which are used as a blueprint for countries without the resources to draft their own plans. As part of the work, the WHO has commissioned the Mario Negri Institute for Pharmacological Research, in Milan, Italy, to assess the evidence for the use of all drugs for children and adolescents – from older options like GSK's (GSK.L) Xenical to newer, more effective treatments like Wegovy and Eli Lilly and Co’s (LLY.N) Mounjaro, Branca told Reuters. “The kind of communication that has been done around these drugs – ‘We’ve found a solution’ – that’s wrong,” said Branca. Drugs for obesity are important but must be “part of a comprehensive approach,” he said. “This is not a silver bullet.” Branca said that other interventions, including diet and exercise, remain critical to help manage obesity. The latest WHO data shows that the percentage of children and adolescents aged 5 to 19 who are obese or overweight has risen to just over 18% in 2016 from 4% in 1975, and this now represents more than 340 million people. Novo Nordisk and Eli Lilly did not immediately respond to a Reuters request for comment. Wegovy and Mounjaro were originally developed for type 2 diabetes to help control blood glucose. More recently, they have been shown to help people lose around 15% of their body weight, capturing the attention of patients, investors and even celebrities. Part of a class of drugs known as GLP-1 agonists, they are given by a weekly injection and work by affecting hunger signals to the brain and slowing the rate at which a person’s stomach empties, making them feel full longer. Studies suggest people are likely to have to take the drugs for the rest of their lives to keep the weight off. Wegovy is approved for weight-loss in the United States and Europe, while Mounjaro is expected to receive U.S. approval later this year. The enormous demand for the drugs is expected to be worth $100 billion in annual sales within a decade, with as many as 10 different drugs on the market. U.S. medical groups are also reviewing their obesity treatment guidelines to consider the best use of Wegovy and similar drugs, with some specialists advocating broad use while others recommend prioritising them for high-risk patients with health conditions, like diabetes or heart disease, that are exacerbated by excess weight. The American Academy of Pediatrics has recommended using such medicines in children age 12 or older with obesity, even though the long-term impacts have yet to be studied. MORE ROBUST The WHO said its revised guidelines will be based on more robust methodology than previous iterations and include up-to-the-minute science. The first draft of the new management guidelines for children and adolescents are expected by the end of this year. Branca said the researchers at Mario Negri, as well as other institutions working on the guidelines, had been extensively vetted to avoid conflict of interest concerns. Novo Nordisk was suspended earlier this year from the Association of the British Pharmaceutical Industry for its marketing practices, such alleged funding of health professionals and providing training that the association alleged was intended to promote its drug. “We really screen the potential conflicts of interest,” said Branca. He described obesity as a "rising epidemic". "There are multiple reasons why we really have to take much more serious and bolder action,” he said. Our Standards: The Thomson Reuters Trust Principles. Thomson Reuters Jen reports on health issues affecting people around the world, from malaria to malnutrition. Part of the Health & Pharma team, recent notable pieces include an investigation into healthcare for young transgender people in the UK as well as stories on the rise in measles after COVID hit routine vaccination, as well as efforts to prevent the next pandemic. She previously worked at the Telegraph newspaper and Channel 4 News in the UK, as well as freelance in Myanmar and the Czech Republic.