Vascarta Announces NIH Phase I clinical study of VAS-101 in sickle cell disease begins
SUMMIT, N.J., Aug. 19, 2026 /PRNewswire/ -- A second proof of concept phase I clinical study of VAS-101 (Vasceptor®; topical curcumin gel) in sickle cell disease (SCD) is underway. It is entitled: A Phase 1 Study to Evaluate the Safety and Tolerability of Escalating Doses of VAS-101 in Subjects with Stable Sickle Cell Disease. This study is being conducted at the National Institutes of Health (NIH) Clinical Center and will be led by principal investigator, Dr. Swee Lay Thein, Chief, Laboratory of Sickle Cell Genetics and Pathophysiology and Chief, Sickle Cell Branch, at NIH's National Heart, Lung and Blood Institute.
SCD is caused by a single point mutation in the beta hemoglobin gene leading to sickling of red blood cells. It is characterized by severe pain, inflammation, oxidative stress, and organ damage, which contribute to the poor quality of life and reduced survival. Pain crises are the prominent complication of SCD, and management of pain is largely supportive, and patients often require multiple painkillers that have undesirable side effects. Current FDA-approved SCD therapies are not effective in all patients with pain. Better therapies that optimize compliance are appropriate for patients of all ages.
The overall objective of this study is to assess the clinical safety and tolerability of a patented, bioavailability-enhanced, transdermal application of curcuminoids, VAS-101 / Vasceptor® (8.5% curcuminoids transdermal gel), in individuals with stable SCD. Patients enrolled will receive 0.2 mL of VAS-101 topically twice weekly for two weeks, followed by 0.4 mL twice weekly for two weeks, then 0.6mL twice weekly for two weeks, and finally a follow-up four weeks thereafter. Throughout the course of the study, subjects will be monitored for signs and symptoms of adverse events. The effect of VAS-101 on laboratory biomarkers of efficacy will also be studied at specific timepoints at NHLBI. Patients will complete the study within 14 weeks, from screening to follow up on week 10.
About VAS-101
VAS-101 (developed by Vascarta Inc., Summit, NJ) is a patented topical curcumin formulation (Curcugen®) that employs a patented transdermal delivery technology (Vasporta™) designed to increase bioavailability. A published study* in the Journal of Sickle Cell Disease stated: "VAS-101 demonstrated safety and tolerability in the sublingual cohort. Improvements in integrated ePRO and red blood cell (RBC) functional biomarkers were noted suggesting potential mechanistic and patient-centered benefits of VAS-101 in individuals with SCD. The resulting biomarkers shift at low dose supports advancement towards dose optimization. Future studies evaluating higher dose and frequency may yield greater improvements in pain, ePRO measures, and laboratory biomarkers. Pre-selecting a cohort that exhibits a higher adhesive burden, indicative of more inflammation may show further improvements in RBC health normalization."
Minimizing the need for blood transfusions and reducing the need for chronic administration of potentially harmful pain medications are desperately needed for those living with SCD.
About Sickle Cell Disease
Sickle cell disease is the most common inherited genetic disorder that effects primarily African American and non – Hispanic Black individuals in the United States. SCD associated complications include anemia, acute and chronic pain, infections, pneumonia and acute chest syndrome, stroke, and kidney, liver, and heart disease.
Current estimates indicate there are ~ 100,000 and ~ 45,000 individuals with SCD in the USA and European Union countries, respectively. The estimated life expectancy of those with sickle cell disease in the USA is more than 20 years shorter than the average expected lifespan.
Dr. Swee Lay Thein, stated: "Curcumin has shown promise in early laboratory studies, but its use in patients has been limited because it is not easily absorbed by the body when taken orally. VAS-101 is a topical, self-applied treatment designed to help overcome this challenge by avoiding the body's usual breakdown processes. Addressing the unmet medical need for most SCD sufferers is significant."
Dr. Joel Friedman, Emeritus Professor, Department of Microbiology & Immunology, Albert Einstein College of Medicine, and Vascarta Scientific Founder & Head, Scientific Advisory Board, stated, "The preclinical and clinical results to date showing pain reduction and therapeutic efficacy resulting from our novel approach of targeting red blood cell instability, neuro-inflammation and vascular inflammation bodes well for the development of a widely accessible therapy that prevents and treats many of the clinical complications of SCD." Dr. Friedman is the inventor of VAS-101 which is exclusively licensed to Vascarta from the Albert Einstein College of Medicine (Bronx, New York, USA).
Dr. Richard Prince, Vascarta Chairman, CEO & President commented, "We are excited at the prospect of bringing relief to SCD sufferers who have limited therapeutic options. VAS-101 has the potential to become the new standard of care in the management of SCD."
About Vascarta
Vascarta is a clinical stage pharmaceutical company exploring efficient transdermal delivery of pharmaceuticals to address inflammation and pain with an initial focus on sickle cell disease and osteoarthritis. More information can be found at www.vascarta.com or by email to:
- Dr. Richard Prince, Chairman, CEO & President – [email protected]
- Media inquiries: David Hymson at [email protected]
SOURCE Vascarta Inc.
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