MONASH HEALTH | MedPath

First-in-Human Trial Launches for Novel Extracellular Vesicle Therapy in Crohn's Fistulas

3 months ago

• Hudson Institute researchers advance groundbreaking extracellular vesicle treatment derived from amniotic cells into clinical trials for complex perianal Crohn's disease, offering a potential cost-effective alternative to stem cell therapy. • The first-in-human trial will evaluate the safety and efficacy of hAEC-EV therapy in 15 participants with refractory Crohn's perianal fistulas, marking the second human EV trial in Australia. • With the global Crohn's disease market projected to reach $13.4 billion by 2026, this innovative approach could provide a more accessible treatment option for the one-third of patients who develop fistulas.

PLL Therapeutics to Initiate Phase I/II Trial of PLL001 for Amyotrophic Lateral Sclerosis in Australia

6 months ago

• PLL Therapeutics has received approval to begin a Phase I/II clinical trial in Australia for PLL001, a drug candidate targeting the root cause of Amyotrophic Lateral Sclerosis (ALS). • The trial will assess the safety, tolerability, efficacy, and pharmacodynamics of PLL001 in restoring gut permeability and preventing contaminants from leaking into the brain. • PLL Therapeutics will also evaluate an early-stage diagnostic tool for ALS, aiming to detect the disease at its onset using blood-based biomarkers. • The study is a multi-center, randomized, double-blind, placebo-controlled trial with an optional open-label extension, enrolling patients at Monash Health in Melbourne.

Beyond LDL: Novel Cholesterol Targets and Therapies Show Promise in Reducing Cardiovascular Risk

8 months ago

• Researchers are exploring treatments for lipoprotein(a), a genetically influenced cholesterol type unaffected by statins, with therapies showing potential in early trials. • HDL cholesterol research shifts focus from simply raising levels to improving functionality, exemplified by CSL112, though recent trials showed it did not meet its primary endpoint. • Triglyceride-lowering strategies face scrutiny as studies yield conflicting results, suggesting triglycerides may be indicators rather than direct causes of cardiovascular issues. • Emerging focus on remnant cholesterol highlights its potential role in atherosclerosis, prompting investigation into novel treatments to clear these particles from the bloodstream.