#United States #Sickle Cell #VAS-101 #Summit #transdermal curcumin

Innovative Treatment for Sickle Cell Disease

In a significant breakthrough in the treatment of sickle cell disease (SCD), Vascarta Inc. has announced findings from a new study led by researchers from the U.S. Food and Drug Administration's (FDA) Center for Biologics Evaluation and Research. This research, conducted in collaboration with esteemed institutions such as the University of California, Irvine, and Albert Einstein College of Medicine, highlights the potential of a novel transdermal curcumin formulation identified as VAS-101. The formulation has demonstrated remarkable effectiveness in ameliorating cardiac mitochondrial function in a humanized mouse model of sickle cell disease.

Understanding Sickle Cell Disease and Mitochondrial Dysfunction

Sickle cell disease is a genetic condition characterized by chronic oxidative stress, hemolysis, and inflammation, leading to multi-organ damage and serious cardiopulmonary complications. Mitochondrial dysfunction is recognized as a contributing factor to the pathophysiology of these complications. The study utilized Berkeley sickle mice, known for closely mimicking human SCD conditions, to investigate the effects of VAS-101 on cardiac health.

Key findings revealed that the cardiac muscle mitochondria in these mice exhibited impaired respiration, reduced electron transport chain (ETC) activity, and increased oxidative stress markers. The research team noted specific deficiencies in complex I and complex V of the ETC, highlighting severe energy metabolism disruptions within the mitochondria of these sickle mice.

Impact of VAS-101 Treatment

After 21 days of treatment with the curcumin formulation, significant improvements were observed. Notable reductions in oxidative stress markers, such as lipid peroxidation and protein carbonylation, were recorded. Additionally, VAS-101 treatment led to an increase in mitochondrial bioenergetic function, with improvements in enzymatic activities of the critical ETC complexes. These findings suggest that VAS-101 not only mitigates oxidative damage but also enhances mitochondrial function, invigorating cellular respiration processes crucial for cardiac health.

The researchers found that VAS-101 corrected abnormalities in various mitochondrial proteins involved in processes such as β-oxidation and calcium handling. This broad efficacy emphasizes the protective attributes provided by the transdermal curcumin, proposing it as a multifaceted treatment for addressing the underlying mitochondrial impairments associated with sickle cell disease.

Expert Insights

Dr. Joel Friedman, a key figure in the development of VAS-101, expressed the significance of these findings: "These results confirm mitochondrial dysfunction as a main component of cardiac issues in sickle cell disease. Our study indicates that transdermal curcumin interventions like VAS-101 have the potential to restore mitochondrial balance and enhance the overall functioning of the heart in SCD patients."

These conclusions build upon earlier studies indicating that VAS-101 reduces hemolysis and inflammation while providing enhanced bioavailability compared to traditional oral formulations, often challenging in achieving effective systemic delivery of curcumin. By bypassing the gastrointestinal tract and liver, VAS-101 maximizes curcumin’s health benefits, particularly in managing chronic pain and inflammation associated with SCD.

Moving Forward: Vascarta’s Vision

Vascarta Inc. is dedicated to advancing innovative healthcare solutions, focusing on enhancing life quality for those suffering from chronic conditions. The company is currently progressing with VAS-101 through clinical studies, including explorations into osteoarthritis and its effects on neuro-inflammation due to cancer treatments. As they move forward with additional clinical trials and pursue Series A financing, Vascarta aims to bring its first-in-class therapies to market, transforming the treatment landscape of various inflammatory diseases and cancers.

For more information about Vascarta and their innovative products, visit Vascarta's website or follow their updates on LinkedIn.