Latest news with #neurodegenerative
The Australian
a day ago
- Health
- The Australian
Study backs Alterity tool for tracking MSA
Special Report: Alterity Therapeutics has unveiled promising new research showing its novel brain imaging tool could play a key role in diagnosing and tracking Multiple System Atrophy (MSA), a rare and aggressive neurodegenerative disease. Quality peer-reviewed publication highlights use of Alterity's MSA Atrophy Index developed to diagnose and track MSA disease progression Tool helps pinpoint changes in brain over time, making it easier to detect disease progression and assess treatments MSA Atrophy Index developed as part of Alterity's bioMUSE natural history study with Vanderbilt University Medical Center The peer-reviewed study, published in peer-reviewed journal Annals of Clinical and Translational Neurology, highlights a new MRI-based measure called the MSA Atrophy Index (MSA-AI) – developed as part of Alterity Therapeutics' (ASX:ATH) bioMUSE natural history study. Using artificial intelligence (AI), the tool helps pinpoint changes in the brain over time, making it easier to detect disease progression and assess how well treatments are working. MSA-AI offers a standardised way to measure brain shrinkage in regions affected by the disease, regardless of the subtype. This makes it a useful tool for both diagnosing patients earlier and improving how clinical trial participants are selected. The study combined data from both early-stage and more advanced MSA patients, capturing a wide range of disease severity. The approach has helped researchers confirm that the MSA-AI could reliably track changes in brain volume over time and differentiate MSA from other similar neurological conditions like Parkinson's disease and Lewy body dementia. Importantly, lower MSA-AI scores were linked to more severe symptoms and greater disease progression over 12 months, highlighting the tool's potential value in future clinical trials and patient care. About the bioMUSE study Titled Biomarkers of Progression in Multiple System Atrophy (bioMUSE), the natural history study tracks the progression of individuals with MSA, a Parkinsonian disorder without an approved therapy. The study was conducted in collaboration with Vanderbilt University Medical Center in the US under the direction of Daniel Claassen M.D, M.S, professor of neurology and principal investigator. Alterity noted that natural history studies were important for characterising disease progression in selected patient populations. Insights into early-stage MSA The company said the study had provided rich data for optimising the design of its randomised ATH434-201 phase II clinical trial and had enrolled ~20 individuals with clinically probable or clinically established MSA. The bioMUSE study continues to provide critical insights into early-stage MSA, helping select biomarkers and track disease progression in patients like those in its phase II trial. 'This research used state-of-the-art technology employed in the bioMUSE study that goes above and beyond traditional MRI methods for assessing brain volume in patients with MSA,' Alterity's US-based CEO Dr David Stamler said. 'Based on the creativity and technical skill of our colleagues at Vanderbilt University Medical Center, we now have superior tools for diagnosing MSA and tracking brain atrophy over time. 'Importantly, we observed that statistically significant reductions in brain volume over 12 months correlated with clinical worsening of the disease.' Stamler said the results underscore importance of using advanced neuroimaging methods and analytical tools in evaluating MSA, which Alterity implemented in its phase II clinical program. 'While previous MRI studies have reported brain volume reductions in MSA-affected brain regions, tracking these changes reliably has been challenging,' he said. Stamler said development of the MSA Atrophy Index can enhance the understanding of MSA progression and provide support for using brain atrophy markers for evaluation of disease-modifying therapies. 'These tools offer potential applications in diagnosis, staging, and monitoring of disease severity, contributing to more personalised care in MSA,' he said. 'We look forward to leveraging this invaluable technology for patient selection and disease progression in our phase III clinical trial.' This article does not constitute financial product advice. You should consider obtaining independent advice before making any financial decisions.
Daily Mail
3 days ago
- Health
- Daily Mail
Scientists discover two drugs already approved by the FDA can reverse Alzheimer's
Two drugs already approved by the FDA for cancer treatment may hold the key to reversing Alzheimer's disease in patients, experts say. Researchers from the University of California, San Francisco (UCSF) believe that letrozole, a hormone-based breast cancer drug, and irinotecan, a lung and colon cancer chemotherapy medication, can help reverse brain damage caused by the incurable neurodegenerative disease. In an animal study, the UCSF experts found that both cancer drugs were seen to reduce brain degeneration in mice and even improve their memory and learning capacity. Alzheimer's disease is one of the most common forms of dementia and mostly affects adults over the age of 65. About 7million Americans live with the condition and over 100,00 die from it annually. The disease is believed to be caused by the development of toxic amyloid proteins and/or tau proteins in the brain, which can accumulate and damage cells responsible for memory and learning. Amyloid protein molecules stick together in brain cells, forming clumps called plaques. While tau proteins twist together in fiber-like strands called tangles. As of now, there is no cure for AD and only two FDA-approved therapies, Lecanemab (Leqembi) and Donanemab (Kisunla), are available for early-stage Alzheimer's treatment. However, because letrozole and irinotecan are already approved for other treatments, this could fast-track clinical trials and the potential approval for use in Alzheimer's patients. Co-senior author Dr Marina Sirota, a professor at UCSF, said: 'Alzheimer's disease comes with complex changes to the brain, which has made it tough to study and treat, but our computational tools opened up the possibility of tackling the complexity directly. 'We're excited that our computational approach led us to a potential combination therapy for Alzheimer's based on existing FDA-approved medications.' In Alzheimer's patients, the plaques and tangles block the ability of the brain's neurons to send electrical and chemical signals back and forth. Over time, this disruption causes permanent damage in the brain that leads to Alzheimer's and dementia, causing patients to lose their ability to speak, care for themselves and interact with the world around them. While the exact mechanisms of how the Alzheimer's-related brain damage begins are still under investigation, age and genetics are known risk factors. Experts also believe that lifestyle factors such as physical inactivity and high blood pressure can also contribute to the development of Alzheimer's. Despite rigorous preclinical and clinical research efforts, drug development for dementia faces significant challenges, with a 98 percent failure rate in recent decades. Neuroscientist Dr Yadong Huang, co-author of the study and professor of neurology at UCSF, explained: 'Alzheimer's is likely the result of numerous alterations in many genes and proteins that, together, disrupt brain health. 'This makes it very challenging for drug development – which traditionally produces one drug for a single gene or protein that drives disease.' However, researchers at USCF believe their discovery can help reduce or reverse the cognitive decline caused by the disease. First, the team looked at how dementia changes gene expression in the brain. Then, they scoured a database of over 1,300 drugs, including antipsychotics, antibiotics, antifungals and chemotherapy drugs, to determine which, if any, reversed any of these gene expressions. If any existing drugs were found to be effective, they could be repurposed to treat the condition in a reduced the time in which the drugs could be made available to patients. During their search, the team specifically looked for drugs that would target the harmful Alzheimer's-related changes in neurons and in brain cells called glia that are responsible for supporting the nervous system. Then, the researchers analyzed millions of digital medical records to find patients who took some of these drugs as part of cancer treatments and their likelihood of developing Alzheimer's. Ultimately, they identified letrozole and irinotecan as the best candidates to lower Alzheimer's risk in patients. By combining the two drugs together, the researchers were able to target different types of brain cells affected by the disease. They noted that letorozole could counter the effect of Alzheimer's on neurons and irinotecan helped reverse damage on the glia cells. When the combination was tested on mice, the scientists saw that the harmful clumps of tau protein were reduced significantly and the mice showed improvements in learning and memory tasks. The study authors noted that it remains unclear how the cancer drugs are able to reverse the damage. However, they theorized that it was possible that letrozole blocks the production of estrogen, a hormone that controls the working of a large number of genes, which therefore reduces the genetic risk factor of developing Alzheimer's. Additionally, they believe that irinotecan may also block inflammation in the brain by preventing the rapid reproduction and DNA damage of glial cells. As this was an animal study, the researchers hope to test the drugs in a clinical trial with human Alzheimer's patients. Dr Huang said of the results: 'Developing a new drug can take hundreds of millions, or even billions, of dollars, on average take more than 10 years. For this repurposed drug, usually it just takes two or three years, and then you can go to the clinical trial and the cost is much, much lower. 'We still haven't generated or produced any very effective drugs that can really slow dramatically the cognitive decline.' However, despite their groundbreaking discovery, risks continue as letrozole is known to cause hot flashes in patients while irinotecan can cause severe diarrhea. Both drugs can also lead to nausea and vomiting. Dr Sirota said: 'These drugs have huge side effects, so you need to always balance and figure out whether those types of side effects would be amenable to somebody with Alzheimer's. It's not that it's a slam dunk.'
Globe and Mail
4 days ago
- Health
- Globe and Mail
Amyotrophic Lateral Sclerosis Pipeline Insight 2025: 75+ Companies Accelerating Innovation Across Genetic and Neuroinflammatory Targets
The ALS pipeline is gaining momentum, with over 75 companies advancing R&D across key targets like SOD1 mutations, TDP-43, C9orf72 expansions, and neuroinflammation. Approaches include ASOs, gene therapies, mitochondrial modulators, neuroprotective agents, and immune biologics. A major milestone was the FDA's 2023 accelerated approval of Biogen and Ionis's Tofersen for SOD1-ALS. DelveInsight's ' Amyotrophic Lateral Sclerosis (ALS) – Pipeline Insight, 2025 ' offers a detailed analysis of the evolving therapeutic landscape for ALS, a fatal neurodegenerative disease characterized by progressive loss of motor neurons, leading to muscle weakness, paralysis, and eventually respiratory failure. With most patients succumbing to the disease within 3–5 years of symptom onset, there remains a critical unmet need for effective disease-modifying treatments. Several late-stage candidates are progressing through global Phase II/III studies, including Amylyx's AMX0035, which received full FDA approval in September 2022 as RELYVRIO, and is now being evaluated in confirmatory trials. Additionally, investigational programs from companies like Clene Nanomedicine, QurAlis, Wave Life Sciences, and BrainStorm Cell Therapeutics are advancing promising assets in both sporadic and familial ALS subpopulations, often with orphan drug or fast-track designations. The regulatory landscape continues to support innovation in ALS with flexible pathways and growing biomarker guidance, including neurofilament light chain (NfL) levels, digital endpoints, and functional scores. Patient advocacy, real-world evidence generation, and adaptive trial designs are also helping de-risk development strategies and accelerate access. As 2025 unfolds, the ALS pipeline reflects a shift from symptomatic care to precision medicine and disease modification, driven by robust collaboration among academia, biotech, regulatory bodies, and patient communities. With over 80 therapies in the pipeline, the future of ALS treatment is poised for transformative change. Key Takeaways from the Amyotrophic Lateral Sclerosis Pipeline Report • DelveInsight's amyotrophic lateral sclerosis pipeline analysis depicts a strong space with 75+ active players working to develop 80+ pipeline drugs for amyotrophic lateral sclerosis treatment. • The leading amyotrophic lateral sclerosis companies include Ionis Pharmaceuticals, 1ST Biotherapeutics, Scholar Rock, Revalesio, QurAlis Corporation, Sanofi, MediciNova, Helixmith, Verge Genomics, UCB, and others are evaluating their lead assets to improve the amyotrophic lateral sclerosis treatment landscape. • Key amyotrophic lateral sclerosis pipeline therapies in various stages of development include ION363, FB418, SRK-015, RNS60, QRL-201, SAR443820, MN-166, VM202, VRG-50635, Zilucoplan, and others. • In July 2025, Neurizon Therapeutics submitted a formal response to the FDA addressing the clinical hold on its IND application for NUZ-001, its lead ALS therapy. • In July 2025, Klotho Neurosciences received FDA Orphan Drug Designation for KLTO-202 ( its novel gene therapy targeting ALS. • In June 2025, Amylyx Pharmaceuticals, Inc. (NASDAQ: AMLX) announced that the FDA granted Fast Track designation to AMX0114, an investigational antisense oligonucleotide targeting calpain-2 for treating ALS. • In May 2025, BrainStorm Cell Therapeutics Inc. (NASDAQ: BCLI) announced FDA clearance to begin its Phase IIIb trial of NurOwn® for treating amyotrophic lateral sclerosis (ALS). • In May 2025, Neuralink announced that it received the FDA's " breakthrough" designation for its device aimed at restoring communication for individuals with severe speech impairment. The device, designed to assist patients with conditions like ALS, stroke, spinal cord injury, cerebral palsy, and multiple sclerosis, represents a significant step forward in neurotechnology. • In March 2025, DiagnaMed Holdings Corp. announced a significant milestone in rare disease research, receiving Orphan Drug Designation (ODD) from the FDA for molecular hydrogen in the treatment of ALS. • In January 2025, the FDA granted approval for Zydus Lifesciences to proceed with a randomized Phase IIb trial of its oral NLRP3 inflammasome inhibitor, Usnoflast, for the treatment of ALS. Request a sample and discover the recent breakthroughs happening in the amyotrophic lateral sclerosis pipeline landscape. Amyotrophic Lateral Sclerosis Overview Amyotrophic Lateral Sclerosis (ALS) is a rare, progressive neurodegenerative disorder that affects nerve cells in the brain and spinal cord. It leads to the loss of muscle control, eventually impacting the ability to speak, move, eat, and breathe. ALS is often classified into sporadic (the most common form) and familial types, with the latter being linked to genetic mutations. Although the exact cause of ALS is unknown in most cases, environmental and genetic factors are believed to contribute to its development. Currently, there is no cure for ALS, but several treatment options aim to slow disease progression and manage symptoms. FDA-approved drugs like riluzole and edaravone offer modest benefits in extending survival or preserving function. Ongoing research is focused on gene therapies, neuroprotective agents, and immune-targeting approaches to better address the underlying mechanisms of the disease and improve patient outcomes. Find out more about amyotrophic lateral sclerosis medication at Amyotrophic Lateral Sclerosis Treatment Analysis: Drug Profile MN-166: MediciNova MN-166 is an orally administered small molecule glial attenuator that reduces neuroinflammation by suppressing pro-inflammatory cytokines such as IL-1β, TNF-α, and IL-6, while potentially increasing the anti-inflammatory cytokine IL-10. It also functions as a toll-like receptor 4 (TLR4) antagonist, contributing further to its anti-inflammatory effects. MN-166 is currently in Phase II/III clinical trials for the treatment of ALS. RNS60: Revalesio RNS60 is designed to offer disease-modifying and potentially regenerative effects in neurological disorders. It works by activating signaling pathways that enhance mitochondrial function and reduce inflammation, offering neuroprotection and immune modulation. The FDA has granted RNS60 both Orphan Drug and Fast Track status for ALS. It is currently in Phase II clinical trials. VM202: Helixmith VM202 is a gene therapy aimed at tissue regeneration by promoting the production of hepatocyte growth factor (HGF) at the site of injection along peripheral nerves. HGF supports nerve protection, neuron growth, and muscle atrophy prevention. The FDA has granted VM202 Orphan Drug and Fast Track designations. It is being evaluated in Phase II trials for ALS treatment. Learn more about the novel and emerging amyotrophic lateral sclerosis pipeline therapies. By Product Type • Mono • Combination • Mono/Combination. By Stage • Late-stage products (Phase III) • Mid-stage products (Phase II) • Early-stage product (Phase I) along with the details of • Pre-clinical and Discovery stage candidates • Discontinued & Inactive candidates By Route of Administration • Intravenous • Subcutaneous • Oral • Intramuscular By Molecule Type • Monoclonal antibody • Small molecule • Peptide Scope of the Amyotrophic Lateral Sclerosis Pipeline Report • Coverage: Global • Key Amyotrophic Lateral Sclerosis Companies: Ionis Pharmaceuticals, 1ST Biotherapeutics, Scholar Rock, Revalesio, QurAlis Corporation, Sanofi, MediciNova, Helixmith, Verge Genomics, UCB, and others. • Key Amyotrophic Lateral Sclerosis Pipeline Therapies: ION363, FB418, SRK-015, RNS60, QRL-201, SAR443820, MN-166, VM202, VRG-50635, Zilucoplan, and others. Explore detailed insights on drugs used in the treatment of amyotrophic lateral sclerosis here. Table of Contents 1. Introduction 2. Executive Summary 3. Amyotrophic Lateral Sclerosis Pipeline: Overview 4. Analytical Perspective In-depth Commercial Assessment 5. Amyotrophic Lateral Sclerosis Pipeline Therapeutics 6. Amyotrophic Lateral Sclerosis Pipeline: Late-Stage Products (Phase III) 7. Amyotrophic Lateral Sclerosis Pipeline: Mid-Stage Products (Phase II) 8. Amyotrophic Lateral Sclerosis Pipeline: Early Stage Products (Phase I) 9. Therapeutic Assessment 10. Inactive Products 11. Company-University Collaborations (Licensing/Partnering) Analysis 12. Key Companies 13. Key Products 14. Unmet Needs 15. Market Drivers and Barriers 16. Future Perspectives and Conclusion 17. Analyst Views 18. Appendix About DelveInsight DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports Pharma companies by providing comprehensive end-to-end solutions to improve their performance. Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platform, PharmDelve. Media Contact Company Name: DelveInsight Contact Person: Jatin Vimal Email: Send Email Phone: +14699457679 Address: 304 S. Jones Blvd #2432 City: Las Vegas State: Nevada Country: United States Website:
Yahoo
6 days ago
- Health
- Yahoo
What to know about Parkinson's disease following Ozzy Osbourne's death
Ozzy Osbourne, lead singer of the legendary heavy metal rock band Black Sabbath, died this week at age 76. Although Osbourne's cause of death hasn't yet been publicly released, he revealed in an exclusive interview with "Good Morning America" in 2020 that he had been diagnosed with Parkinson's disease in February of the previous year. "There's so many different types of Parkinson's. It's not a death sentence by any stretch of the imagination but it does affect certain nerves in your body," Osbourne's wife, Sharon, said during the interview. "And it's like you have a good day, a good day, and then a really bad day." Here's what you need to know about Parkinson's, including what are the symptoms, how it's diagnosed and what treatments are available. MORE: White House doctor offers explanation for Parkinson's expert's visits What is Parkinson's disease? Parkinson's is a neurodegenerative disorder characterized by the weakening of nerve cells, which become damaged and die, according to the National Institute of Neurological Disorders and Stroke (NINDS). The most significant loss of neurons occurs in an area near the base of the brain called the substantia nigra, NINDS said. The neurons in the substantia nigra produce dopamine, which is a neurotransmitter that plays a vital role in controlling movement. "As we grow older, all of us lose dopamine but the people who develop Parkinson's disease lose it faster," Dr. Osama Abu-Hadid, a neurologist specializing in movement disorders at New Jersey's Hackensack Meridian Neuroscience Institute at Hackensack University Medical Center, told ABC News. Parkinson's affects more than 1.1 million people in the U.S., according to the Parkinson's Foundation. That figure is expected to reach 1.2 million by 2030. "Generally, neurodegenerative diseases such as Alzheimer's and Parkinson's disease have been on the increase because people are living longer," Abu-Hadid said. There currently are no specific tests to diagnose Parkinson's disease. The diagnosis is clinical based on a physician's review of a patient's medical history and by performing a physical examination. What are the symptoms of Parkinson's disease? Progression of Parkinson's disease symptoms typically occurs slowly over several years and can vary from person to person, according to the Parkinson's Foundation. Typically, symptoms begin on one side of the body. While Parkinson's will eventually affect both sides of the body, one side will generally exhibit more severe symptoms than the other, according to NINDS. MORE: Parkinson's expert visited White House 8 times in 8 months, met with Biden's doctor Symptoms of Parkinson's can include tremors, muscle stiffness, slowing of spontaneous movement, and problems with gait and balance. Patients may also experience difficulty chewing and swallowing or changes in speech. Some patients can also develop non-motor symptoms, including constipation, hallucinations, depression, loss of smell and sleep problems, Abu-Hadid said. What Parkinson's disease treatments are available? Currently, there is no cure for Parkinson's, but physical therapy can help slow progression of the disease and a healthy diet can promote overall well-being, according to Abu-Hadid. However, there are medications that may improve some Parkinson's symptoms. These include drugs that increase the level of dopamine in the brain, drugs that interact with other neurotransmitters to help ease some symptoms, and medications that can help control the non-motor symptoms, according to NINDS. If medications aren't working, "procedural options include deep brain stimulation, which is an electrode, or electrodes, that are placed in the brain by a neurosurgeon, and then a physician like myself programs the electrodes to give electricity in a certain way," Abu-Hadid said. The electrodes can stimulate the brain and help block signals that cause the motor symptoms of Parkinson's, according to NINDS.
Yahoo
6 days ago
- Business
- Yahoo
Amprion Announces Strategic Growth Initiatives, Expanding Access to Breakthrough SAAmplify Test
Company Launches New Website, Scales Test Access Alongside Mayo Clinic, & Looks to Expand International Presence SAN DIEGO, July 23, 2025--(BUSINESS WIRE)--Amprion, a global leader advancing diagnosis of neurodegenerative disorders through seed amplification testing, has announced several strategic growth initiatives to broaden access to its SAAmplify-ɑSYN test. These initiatives include a comprehensive website refresh, expanded test availability via Mayo Clinic's network of hospitals and clinical sites, and ongoing preparations for international expansion. As part of its continuing growth and commitment to advancing early and accurate diagnosis, Amprion will showcase its latest developments and momentum at the Alzheimer's Association International Conference (AAIC), July 27-31, 2025, in Toronto, Canada. Attendees can connect with the Amprion team at booth #616. Following its recent collaboration with Mayo Clinic Laboratories, Amprion launched a newly redesigned website to meet the growing demand for its SAAmplify-ɑSYN test, which aids in the diagnosis of synucleinopathies. The breakthrough test can detect the presence of aggregates of alpha-synuclein protein, the underlying pathology of Parkinson's disease (PD), Lewy body dementia (LBD), multiple system atrophy (MSA), and Alzheimer's disease with Lewy bodies (AD + DLB), in cerebrospinal fluid. Launched earlier this month, the updated website is designed to provide patients, clinicians, and industry leaders with streamlined access to Amprion's latest research, information, and more. "As awareness and demand for our SAAmplify-ɑSYN test grow, we're working to ensure clinicians, researchers, and patients have better access to the tools and information they need," said Dr. Russ Lebovitz, CEO and co-founder of Amprion. "Our refreshed website and expanding partnerships are steps toward that goal, and we look forward to connecting with the global neuroscience community at AAIC." Due to this surge in awareness, Amprion is actively exploring international expansion opportunities, with plans to bring its test to European and global markets. Initial announcements regarding country-specific launch strategies are expected in Q4 2025. For more information or to schedule a meeting with Amprion at AAIC 2025, please visit About SAAmplify-ɑSYN (formerly SYNTap®) Amprion's SAAmplify-ɑSYN test is a first-in-class-qualitative Laboratory Developed Test (LDT) and the only seed amplification assay available to aid the diagnosis of synucleinopathies such as Parkinson's disease (PD), Lewy body dementia (LBD/DLB), and Alzheimer's disease (AD) with Lewy body co-pathology. The U.S. Food and Drug Administration (FDA) granted Amprion a Breakthrough Device Designation in 2019 for use of the test as an aid in the diagnosis of PD. The test became commercially available in the US in 2021. About Amprion Amprion is the global leader advancing diagnosis of neurodegenerative disorders through seed amplification testing. Amprion's intellectual property surrounding SAA methodology extends to research, drug development, and commercialization. SAAmplify-ɑSYN (formerly SYNTap®) is the only seed amplification assay available to aid the diagnosis of synucleinopathies such as Parkinson's disease, Lewy body dementia, and Alzheimer's disease with Lewy body co-pathology. Amprion is also accelerating precision medicine for neurodegenerative disorders by helping biopharma partners identify new drug candidates and underlying pathologies. Learn more at or find us on LinkedIn. View source version on Contacts Media Contacts Liz RobinsonCG Lifelrobinson@ Karen SharmaCG Lifeksharma@ Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
