Uric Acid Shows Promise as Novel Add-On Therapy for Ischemic Stroke in NIH-Funded Study
• NIH-funded preclinical research demonstrates that uric acid treatment significantly improved sensorimotor function and survival rates in rodents 30 days after ischemic stroke.
• The study, conducted by University of Iowa researchers, showed efficacy across diverse animal groups including different ages, sexes, and those with comorbidities like obesity and hypertension.
• As part of the NIH's Stroke Preclinical Assessment Network (SPAN), uric acid emerged as the only effective agent among six promising stroke treatments tested, suggesting readiness for human clinical trials.
Novel Drug VIP36 Targets CB1 Receptor for Pain Relief Without CNS Side Effects
• NIH-funded researchers have developed VIP36, a new drug targeting cannabinoid receptor type 1 (CB1) that shows promise for treating acute and chronic pain in animal models without harmful side effects.
• The drug overcomes previous CB1-targeting challenges by using computer modeling to create a "peripherally restricted" compound that minimizes central nervous system penetration and reduces tolerance development.
• This breakthrough could lead to non-addictive pain treatments and has broader implications for drug design targeting G-protein-coupled receptors involved in various physiological functions and disease states.
First Patients Receive Groundbreaking Autologous Stem Cell Treatment for Parkinson's Disease in Phase 1 Trial
• Mass General Brigham launches pioneering Phase 1 trial using patients' own reprogrammed stem cells to replace damaged dopamine neurons in Parkinson's disease, with three of six participants treated successfully.
• The innovative treatment converts patients' blood cells into induced pluripotent stem cells (iPSCs), which are then transformed into dopamine neurons, eliminating the need for immunosuppression.
• The FDA-approved trial, based on three decades of research at McLean Hospital, will monitor patients for 12 months to assess safety and potential improvements in Parkinson's symptoms.
UVA Launches $30M Trial to Test Ketamine for Life-Threatening Seizures
• The University of Virginia has secured a $9.3 million NIH grant as initial funding for a $30 million clinical trial investigating ketamine's potential in treating status epilepticus, a life-threatening seizure condition.
• Current standard treatments for status epilepticus are only effective in approximately 47% of adults and 52% of children, highlighting a critical need for improved therapeutic approaches.
• The KESETT trial will evaluate two different ketamine dosages (1mg and 3mg) as add-on therapy to existing treatments across approximately 60 sites, focusing particularly on outcomes in children aged 1 year and older.
uniQure's AMT-162 Gene Therapy for SOD1-ALS Advances to Second Dose Cohort
• uniQure's AMT-162, a gene therapy for SOD1-ALS, has received the green light from the IDMC to proceed with enrollment in the second dose cohort of its Phase I/II EPISOD1 trial.
• The decision follows a review of safety data from the first cohort, which showed no significant safety concerns, marking a positive step in the therapy's clinical development.
• AMT-162 utilizes an AAVrh10 vector to deliver a microRNA designed to silence the mutated SOD1 gene, offering a potential one-time treatment for this rare form of ALS.
• The EPISOD1 trial is ongoing in multiple centers across the U.S. and is evaluating the safety, tolerability, and preliminary efficacy of AMT-162 in SOD1-ALS patients.
FDA Approves Generic Everolimus for Tuberous Sclerosis Complex Brain Tumors
The FDA has granted final approval for generic everolimus tablets for oral suspension, offering a new cost-effective treatment option for patients with tuberous sclerosis complex (TSC)-associated brain tumors. The approval, granted to Breckenridge Pharmaceutical Inc., is based on clinical trial data showing a 35% reduction in tumor volume compared to placebo.
NIH Study Links SARS-CoV-2 Infection to Increased ME/CFS Incidence
• A new NIH-funded study reveals a significant association between SARS-CoV-2 infection and an increased incidence of myalgic encephalomyelitis/chronic fatigue syndrome (ME/CFS).
• The study found that 4.5% of post-COVID-19 participants met the diagnostic criteria for ME/CFS, compared to only 0.6% of those uninfected with SARS-CoV-2.
• Researchers observed that new ME/CFS cases were 15 times higher than pre-pandemic levels, highlighting the potential long-term impact of COVID-19 on chronic conditions.
• Post-exertional malaise, orthostatic intolerance, and cognitive impairment were the most frequently reported ME/CFS symptoms among the infected participants.
FDA Adds Guillain-Barré Syndrome Warning to Pfizer and GSK RSV Vaccines
• The FDA now requires updated safety labels for Pfizer's Abrysvo and GSK's Arexvy RSV vaccines to include a warning about Guillain-Barré Syndrome (GBS).
• Postmarketing studies suggest an increased risk of GBS within 42 days following vaccination with either Abrysvo or Arexvy, particularly in adults 65 and older.
• The FDA emphasizes that while the data indicates a potential increased risk of GBS, a causal relationship between the vaccines and GBS has not been established.
• Despite the GBS warning, the FDA maintains that the benefits of Abrysvo and Arexvy in preventing RSV-related lower respiratory tract disease continue to outweigh the risks.
FDA Approves Journavx, a Novel Non-Opioid Painkiller, Offering New Hope for Acute Pain Management
• The FDA has approved Journavx (suzetrigine), a first-in-class non-opioid analgesic, for treating moderate to severe acute pain in adults, offering a safer alternative to opioids.
• Journavx works by selectively blocking pain signals in the peripheral nervous system, reducing pain without the addictive potential and severe side effects associated with opioids.
• Clinical trials demonstrated Journavx's effectiveness in reducing pain after surgeries, with a safety profile comparable to placebo, though it didn't outperform opioid-acetaminophen combinations.
• Priced at $15.50 per pill, Journavx faces challenges in accessibility due to its higher cost compared to generic opioids, but it represents a significant step in combating the opioid crisis.
Clinical Trials Advance for Potential Kennedy's Disease Therapies
• Structured exercise programs are under Phase 2 trials to improve muscle strength and function in Kennedy's disease patients.
• Clenbuterol, an asthma drug, is in Phase 2 trials to assess its ability to enhance muscle stamina and quality of life.
• AJ201, a curcumin-based drug, is undergoing Phase 1/2a trials to evaluate safety and its potential to break down faulty androgen receptor protein.
• Leuprorelin acetate, originally for prostate cancer, is in Phase 3 trials to determine its long-term efficacy and safety in slowing Kennedy's disease progression.
GeNeuro's GNK-301 Shows Promise in Precision Medicine Approach for ALS
• GeNeuro's GNK-301, a monoclonal antibody targeting HERV-K ENV, demonstrates potential in a precision medicine strategy for amyotrophic lateral sclerosis (ALS).
• Preclinical studies indicate GNK-301 can detect HERV-K ENV, a neurotoxic protein, in cerebrospinal fluid of ALS patients and neutralize its harmful effects.
• The approach aims to protect neurons and prevent blood-brain barrier dysfunction, both hallmarks of ALS, offering a new treatment avenue.
• GeNeuro is planning for medical-grade production of GNK-301 to enable clinical studies in ALS patients who test positive for HERV-K ENV.
Alterity's ATH434 Shows Promise in Phase 2 Trial for Multiple System Atrophy
• Alterity Therapeutics' ATH434 demonstrated a statistically significant 48% slowing of clinical progression in early-stage MSA patients at the 50 mg dose.
• MRI biomarkers indicated that ATH434 reduced iron accumulation in key brain regions affected by MSA, suggesting a potential disease-modifying effect.
• The Phase 2 trial showed a favorable safety profile for ATH434, with most adverse events being mild to moderate and no treatment-related serious adverse events reported.
• Alterity plans to engage with the FDA to discuss accelerating the development of ATH434, addressing the unmet need for MSA treatments.
AXS-12 Demonstrates Long-Term Efficacy and Safety in Narcolepsy Phase 3 Trial
• Axsome Therapeutics' AXS-12 (reboxetine) significantly reduced cataplexy attacks compared to placebo in the Phase 3 ENCORE trial, meeting its primary endpoint.
• Long-term treatment with AXS-12 showed sustained improvements in cataplexy, excessive daytime sleepiness, and cognition in patients with narcolepsy.
• The ENCORE trial's results support AXS-12 as a potential novel treatment option for narcolepsy, with a favorable long-term safety and tolerability profile.
• Axsome Therapeutics plans to submit a New Drug Application (NDA) to the FDA for AXS-12 based on the positive Phase 3 results.
Newer Epilepsy Medications During Pregnancy Show No Impact on Children's Neurodevelopment at Age 6
• A recent study indicates that newer anti-seizure medications, like lamotrigine and levetiracetam, pose no significant risk to children's language development when taken during pregnancy.
• The research followed 387 children up to age 6, assessing their verbal abilities and finding no difference between those exposed to the drugs in utero and those who were not.
• Findings suggest that controlling seizures with newer medications during pregnancy is a safe approach, contrasting with older medications like valproate, which have known risks.
• The study also highlights the potential benefits of folate supplementation during early pregnancy for improved cognitive and behavioral outcomes in children.
NIH to Advance Gene Therapy Trial for Rare Metabolic Disorder After Biotech Pullout
• NIH and NHGRI collaborate to advance a gene therapy trial for methylmalonic acidemia (MMA) after Selecta Biosciences halted the project due to financial reasons.
• The trial, expected to begin in fall 2025, will use AAV8 to deliver a healthy copy of the _MMUT_ gene, offering hope for MMA patients with limited treatment options.
• NCATS is committing $2.2 million over five years and leveraging its expertise in gene therapy to support the trial, which could streamline future rare disease trials at NIH.
• The MMA-101 trial may inform other gene therapy initiatives like PaVe-GT and BGTC, potentially lowering costs and increasing accessibility of gene therapies.
NIH Collaboratory Adds APA-SM Trial to Evaluate Acupressure for Chronic Pain Management
• The NIH Pragmatic Trials Collaboratory welcomes the APA-SM trial, focusing on personalized auricular point acupressure for chronic pain self-management in rural areas.
• The APA-SM trial will conduct a pragmatic study of a 4-week auricular point acupressure intervention in rural Texas and South Carolina.
• Primary outcomes include pain intensity, pain interference, and function, with secondary outcomes assessing implementation, cost-effectiveness, and predictive factors.
• Funded by the NIH HEAL Initiative, APA-SM aims to reduce opioid prescribing through effective pain management strategies.
Gene Therapy and GPR6 Inverse Agonist Show Promise in Parkinson's Disease Trials
• AAV-GAD gene therapy demonstrated safety and improved motor scores by boosting GABA levels in the subthalamic nucleus in a Parkinson's disease trial.
• CVN424, a GPR6 inverse agonist, was safe and well-tolerated, showing a clinically meaningful reduction in daily "off" time at 150 mg/day in a phase II Parkinson's study.
• The MIND diet was associated with slower cognitive decline in older adults, with relationships varying based on lifestyle and vascular factors.
• Modafinil, cognitive behavioral therapy, and their combination showed similar reductions in multiple sclerosis fatigue at 12 weeks in the COMBO-MS trial.
FDA Approves AbbVie's Vyalev for Advanced Parkinson's Disease
• The FDA has approved AbbVie's Vyalev (foscarbidopa and foslevodopa) as a 24-hour subcutaneous infusion for motor fluctuations in advanced Parkinson's disease.
• Vyalev is the first and only levodopa-based therapy delivered as a continuous 24-hour infusion, offering a non-surgical treatment option for patients.
• Clinical trials showed Vyalev significantly increased "on" time without troublesome dyskinesia and decreased "off" time compared to oral carbidopa/levodopa.
• The most common adverse reactions were infusion site events, hallucinations, and dyskinesia, with Medicare coverage expected in the second half of 2025.
uniQure Doses First Patient in Phase I/II Trial of AMT-162 for SOD1-ALS
• uniQure has dosed the first patient in its Phase I/II EPISOD1 clinical trial evaluating AMT-162 for SOD1-ALS, a rare form of amyotrophic lateral sclerosis.
• AMT-162 is an AAVrh10-based gene therapy designed to reduce the expression of mutated SOD1 protein, which is toxic to motor neurons.
• The EPISOD1 trial is a multi-center, open-label study in the U.S. assessing the safety, tolerability, and exploratory efficacy of AMT-162 with three dose-escalating cohorts.
• AMT-162 has received Orphan Drug and Fast Track designations from the FDA, potentially accelerating its development for this devastating disease.
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