UNIVERSITY OF SYDNEY

GSK Launches Landmark Study to Investigate Shingles Vaccine's Potential to Reduce Dementia Risk

2 months ago

• British pharmaceutical company GSK is initiating a groundbreaking four-year study using data from up to 1.4 million NHS patients to determine if its Shingrix vaccine reduces dementia risk. • Previous research suggests the shingles vaccine may lower dementia risk by up to 27%, with a Stanford Medicine study finding vaccinated Welsh adults were 20% less likely to develop dementia over seven years. • If confirmed, this could represent a significant breakthrough in dementia prevention using an existing vaccine already administered through the NHS to adults aged 65 and older.

FDA Approves Blujepa, First New Class of Oral Antibiotic for UTIs in Nearly 30 Years

2 months ago

• GSK's Blujepa (gepotidacin) has received FDA approval for treating uncomplicated urinary tract infections, marking the first new class of oral antibiotics for UTIs in nearly three decades. • The novel triazaacenaphthylene antibiotic demonstrated non-inferiority or superiority to nitrofurantoin in clinical trials, offering a new treatment option amid rising antibiotic resistance concerns. • Blujepa works through a unique dual-enzyme inhibition mechanism that may reduce the potential for resistance development, with commercial availability expected in the second half of 2025.

Novel Triple Immunotherapy Approach Shows Promise in Glioblastoma Treatment

3 months ago

• A groundbreaking experimental treatment combining three checkpoint inhibitor immunotherapies before surgery has shown promising results in glioblastoma, with one patient remaining cancer-free for over 18 months. • The innovative approach, developed by Professor Georgina Long AO, demonstrated increased immune cell activation and diversity in the treated tumor, suggesting an enhanced immune response against cancer cells. • An Australian-led international clinical trial is being planned to evaluate double immunotherapy, with some patients receiving additional chemotherapy, to validate this potential breakthrough in glioblastoma treatment.

Novel Antifungal Drug Fosmanogepix Enters Clinical Trials at Westmead Hospital for Drug-Resistant Infections

3 months ago

• Westmead Hospital launches groundbreaking clinical trials for Fosmanogepix, a new class of antifungal drug designed to combat drug-resistant invasive fungal infections. • The trials target vulnerable patients undergoing cancer treatment, organ transplants, and ICU care, where current mortality rates from resistant infections range from 15% to 70%. • Led by Associate Professor Justin Beardsley, the research aligns with WHO priorities and could revolutionize treatment options for patients with limited alternatives.

FDA Approves Clinical Trials for Pig Kidney Transplants in Humans, Addressing Organ Shortage

4 months ago

• The FDA has approved clinical trials for two companies, United Therapeutics and eGenesis, to transplant genetically modified pig kidneys into humans with kidney failure. • United Therapeutics will begin with six patients in mid-2025, potentially expanding to 50, while eGenesis will start with three patients, monitoring them closely. • These trials aim to address the critical shortage of donor kidneys, with over 550,000 Americans facing kidney failure and approximately 100,000 on the transplant waiting list. • Both companies have genetically modified pig genes to reduce organ rejection and improve compatibility with human bodies, but ethical and safety concerns persist.

Landmark Report Establishes New Diagnostic Framework for Clinical Obesity, Moving Beyond BMI

4 months ago

• A new international commission of 58 experts has redefined clinical obesity as a chronic systemic disease caused by excess adiposity, distinguishing it from preclinical obesity in a groundbreaking Lancet report. • The framework moves beyond traditional BMI measurements, incorporating multiple body measurements and functional criteria to provide more accurate diagnosis and treatment guidance. • The commission emphasizes the need for evidence-based treatments, reduced weight stigma, and updated healthcare policies to improve access to care for patients with clinical obesity.

FDA Approves Spravato (Esketamine) Nasal Spray as Standalone Treatment for Treatment-Resistant Depression

4 months ago

• The FDA has approved Spravato (esketamine) nasal spray as a monotherapy for adults with treatment-resistant depression (TRD). • Spravato demonstrated rapid and superior improvement in depressive symptoms compared to placebo in clinical trials. • The approval was based on a study where 22.5% of patients achieved remission with Spravato compared to 7.6% with placebo. • Due to risks, Spravato is only available through a restricted program under the supervision of a healthcare provider.

Levofloxacin Shows Promise in Preventing Multidrug-Resistant Tuberculosis

4 months ago

• A clinical trial in Vietnam demonstrated that a six-month course of daily levofloxacin significantly reduced the risk of developing multidrug-resistant tuberculosis (MDR-TB). • The VQUIN trial found a 45% reduction in MDR-TB among adults and adolescents who received levofloxacin compared to placebo, highlighting its potential for prevention. • Combined analysis with the TB-CHAMP trial in South Africa suggests levofloxacin can curtail the global impact of MDR-TB among household contacts, showing a roughly 60% reduction in TB incidence. • The study indicated that levofloxacin was generally safe and well-tolerated, with no major adverse effects or new acquired resistance observed during the trial.

Semaglutide Shows Promise in Reducing Knee Pain in Osteoarthritis Patients

6 months ago

• A recent study in the New England Journal of Medicine suggests semaglutide can significantly improve knee pain in individuals with obesity and osteoarthritis. • Participants taking semaglutide experienced a 14-point reduction in pain on a 0-100 scale, compared to placebo, along with improved knee function and greater weight loss. • Semaglutide, a GLP-1 receptor agonist, may reduce knee pain through weight loss and potential anti-inflammatory properties, offering a new approach to managing osteoarthritis symptoms. • While promising, further independent studies are needed to confirm these findings, considering the limitations of the current study's population and funding source.

Colchicine and Herbal Supplements Fail to Show Benefit in Osteoarthritis Trials

6 months ago

• A trial found colchicine no more effective than placebo for pain relief or functional improvement in knee osteoarthritis, despite its immunomodulatory effects. • A study of herbal supplements, including curcumin and Boswellia serrata, showed no significant advantage over placebo in treating hand osteoarthritis. • Both studies, presented at the American College of Rheumatology (ACR) annual meeting, highlight the ongoing challenge of finding effective non-surgical treatments for osteoarthritis. • Limitations in both trials, including short durations and external factors like the COVID-19 pandemic, may have influenced the results.

Tarantula Venom Shows Promise for Treating Drug-Resistant Epilepsy in Children

6 months ago

• Researchers are testing spider venom proteins as potential treatments for epilepsy, supported by a $4.1 million grant from the Medical Research Future Fund. • Preliminary research on a tarantula peptide, Hm1a, has shown to reduce seizures and early death in mice with Dravet syndrome, a severe form of epilepsy. • The study utilizes brain organoids derived from patients' blood to test the venom's efficacy on various genetic epilepsies, offering a personalized approach. • Scientists aim to develop more specific medications for different types of epilepsy, potentially leading to clinical trials and new treatments for drug-resistant cases.

Regorafenib Shows Survival Benefit in Late-Stage Gastric Cancer INTEGRATE IIa Trial

7 months ago

• Phase 3 INTEGRATE IIa trial demonstrates regorafenib extends median overall survival to 4.5 months versus 4.0 months with placebo in refractory advanced gastric/GEJ cancer patients. • Treatment with regorafenib significantly delayed disease progression and quality-of-life deterioration, with a progression-free survival of 1.8 months compared to 1.6 months for placebo. • The study validates regorafenib's potential as a treatment platform, supporting its investigation in combination therapies including ongoing trials with nivolumab and chemotherapy.

GS-441524 Now Available in the US for Feline Infectious Peritonitis Treatment

7 months ago

• GS-441524, an antiviral medication, is now available in the US for treating feline infectious peritonitis (FIP), a historically fatal disease in cats. • Clinical studies have demonstrated high survival rates (over 80%) when FIP is treated with GS-441524, offering a significant advancement in veterinary medicine. • The FDA allows compounding of GS-441524, making it accessible to US veterinarians for prescription and use as office stock in most states. • Treatment with GS-441524 involves a minimum 84-day course, with dosage adjustments based on the type of FIP, the cat’s weight, and clinical responses.

Cancer Drug Conatumumab Shows Promise in Treating Peripheral Artery Disease

7 months ago

• Preclinical studies demonstrate that Conatumumab, previously tested as a cancer drug, effectively treats Peripheral Artery Disease (PAD). • The drug was tested in preclinical models of PAD and in vitro using cells and arteries from PAD patients who underwent amputation. • Safety and toxicology studies already completed for Conatumumab in cancer treatment expedite its potential transition to human trials for PAD. • Researchers are optimistic about rapidly advancing to clinical trials, given the established safety profile and promising preclinical results.

Antidepressant Use for Pain Relief in Seniors Lacks Evidence, Posing Risks

8 months ago

• A new review indicates limited evidence supports the use of antidepressants for pain relief in older adults, raising concerns about potential overuse. • Researchers found that seniors using antidepressants for pain had a higher risk of falls, dizziness, and injury compared to those using other methods. • International guidelines supporting antidepressants for chronic pain are primarily based on data from younger patients, potentially leading to misinformed prescribing decisions for seniors. • Duloxetine (Cymbalta) showed some efficacy for knee osteoarthritis pain over a few months, but benefits were small and must be weighed against risks.

Certepetide Shows Promise in Metastatic Pancreatic Cancer: Lisata Therapeutics Announces Encouraging Phase 2 Results

8 months ago

• Lisata Therapeutics reported positive trends in overall survival for certepetide plus chemotherapy in metastatic pancreatic cancer patients. • The Phase 2 ASCEND trial's Cohort A showed a median overall survival of 12.68 months in the certepetide arm versus 9.72 months in the placebo arm. • Complete responses were observed in 6.2% of patients treated with certepetide, compared to none in the placebo group. • Lisata plans to advance certepetide to Phase 3 trials in early 2026, based on these results and expectations for Cohort B data.

Intensified Therapy Reverses Poor Prognosis in Prostate Cancer Patients with HSD3B1 Mutation

8 months ago

• A new study identifies that intensified treatment with androgen deprivation therapy (ADT) plus enzalutamide improves survival for prostate cancer patients with the adrenal-permissive _HSD3B1_ allele. • The research, analyzing the ENZAMET trial, demonstrates that upfront treatment intensification can effectively block adrenal androgens, reversing the poorer prognosis typically associated with the _HSD3B1_ mutation. • Findings suggest that genetic analysis of _HSD3B1_ could help tailor more effective therapies for advanced prostate cancer, potentially reducing mortality and improving survival outcomes. • The discovery offers a significant step forward in understanding the genetics of advanced prostate cancer and focusing future clinical trials on targeted treatments.

PharmAla Biotech to Supply LaNeo MDMA for Clinical Trials at Mt. Sinai, Harvard, and Johns Hopkins

8 months ago

• PharmAla Biotech will supply its GMP LaNeo MDMA to Mt. Sinai Health System for a clinical trial, supporting research into MDMA-assisted therapy. • Harvard Medical School's McLean Hospital has contracted PharmAla to provide MDMA for a clinical trial focused on borderline personality disorder. • Johns Hopkins Medicine has also contracted with PharmAla Biotech to supply MDMA for clinical trials, expanding the reach of their MDMA supply. • PharmAla Biotech emphasizes its commitment to regulatory compliance and high-quality MDMA production for clinical research purposes.

Pioneering Immunotherapy Approach Keeps Brain Cancer at Bay for Australian Doctor Who Helped Develop It

a year ago

• Professor Richard Scolyer, diagnosed with terminal stage 4 glioblastoma in 2023, remains cancer-free one year after receiving a first-of-its-kind immunotherapy treatment he helped develop. • The groundbreaking approach combined immunotherapy drugs administered before and after surgery, along with the world's first personalized vaccine tailored to his tumor's characteristics. • Despite initial complications including seizures, liver issues, and pneumonia, Scolyer has returned to his daily routine, though researchers caution that while promising, the treatment requires further study in larger patient populations.

Gene Therapy for Hemophilia A Shows Sustained Clotting Factor Expression and Reduced Bleeding

4 years ago

• A novel gene therapy, SPK-8011, demonstrates stable coagulation factor VIII expression in hemophilia A patients, offering a potential long-term treatment option. • The phase 1/2 trial showed a 91.5% reduction in bleeding episodes among participants, with most maintaining FVIII expression for the duration of the study. • The therapy involves a recombinant AAV vector engineered to produce FVIII in liver cells, providing a one-time, disease-altering approach. • While most participants responded well, two experienced a loss of expression due to an immune response, highlighting the need for further research.