National Heart, Lung, and Blood Institute Partners with Vascarta Inc. for Phase 1 Trial of VAS-101 in Sickle Cell Disease

The National Heart, Lung, and Blood Institute (NHLBI), part of the National Institutes of Health, has announced a collaboration with Vascarta Inc. to conduct a Phase 1 clinical trial of VAS-101, a topical curcumin gel, aimed at treating Sickle Cell Disease (SCD). This groundbreaking clinical study, designed to evaluate the safety and effectiveness of VAS-101, is set to commence later this year under the expert guidance of Dr. Swee Lay Thein, who is the Chief of the Laboratory of Sickle Cell Genetics and Pathophysiology and Chief of the Sickle Cell Branch at NHLBI.

Objectives of the Clinical Trial

The primary focus of this Phase 1 trial is to assess the safety and tolerability of VAS-101 while also monitoring its impact on various biomarkers associated with inflammatory activity and red cell metabolism in patients diagnosed with Sickle Cell Disease. Treatment will occur at the NIH Clinical Center, with laboratory analyses conducted at NHLBI, ensuring rigorous scientific oversight throughout the trial process.

Dr. Richard Prince, Chairman, CEO, and President of Vascarta Inc., expressed enthusiasm regarding this collaboration, noting that NHLBI's interest in funding and managing the trial stemmed from their review of a co-authored publication. This research highlighted the positive effects of Vasceptor®—the active ingredient in VAS-101—on red blood cell health, observing enhancements in both laboratory and clinical settings. Furthermore, the recent FDA orphan drug designation for VAS-101 underscores its potential in addressing the unmet medical needs of patients suffering from SCD.

Mechanism of Action

The development of VAS-101 is significant as it utilizes a patented technology intended to improve the bioavailability and efficacy of curcumin, a natural compound known for its anti-inflammatory and antioxidant properties. According to Dr. Joel Friedman, a key figure in the study and scientific advisor at Vascarta, preclinical findings support a mechanism by which curcumin can be rapidly delivered transdermally into the bloodstream, bypassing traditional metabolic pathways that often limit the effectiveness of oral curcumin dosages.

This innovative approach has shown promise in stabilizing red blood cells and providing swift relief from inflammation and oxidative stress, crucial for managing symptoms associated with SCD. Moreover, preliminary efficacy results have indicated that VAS-101 significantly enhances tissue perfusion and oxygenation, potentially improving the overall well-being of patients and mitigating chronic pain associated with the disease.

The Burden of Sickle Cell Disease

Sickle Cell Disease is recognized as the most prevalent inherited blood disorder, particularly affecting African American and non-Hispanic Black individuals in the United States. The condition is characterized by various health complications, including severe pain episodes, anemia, and a higher susceptibility to infections and stroke. Despite ongoing treatments, many patients experience inadequate pain control and substantial side effects from their medications, highlighting an urgent need for safer, more effective therapeutic options.

Current estimates suggest there are approximately 175,000 cases of SCD in the USA, with affected individuals facing a life expectancy that is notably lower than the general population. Many successful medications fail to alleviate pain effectively, leaving patients to rely on a combination of treatments that often have adverse effects. The NHLBI's commitment to this trial reflects a proactive step towards improving the quality of life for those living with this debilitating condition.

Conclusion

The partnership between the NHLBI and Vascarta Inc. presents a significant opportunity for advancements in the treatment of Sickle Cell Disease. As the clinical trial progresses, the health community will be closely monitoring the outcomes, eager to see if VAS-101 can deliver the hope it offers in healing and management of SCD. The ongoing research strives not only to enhance patient outcomes but also to revolutionize the way this chronic condition is approached.

By initiating this Phase 1 trial, both institutions aim to contribute to a future where the challenges posed by Sickle Cell Disease can be more effectively managed, highlighting the critical role of innovative therapies in transforming healthcare.