Vascarta Inc. has commenced a first-in-human Phase 1 clinical trial of VAS-101, a topical curcumin gel formulation, for patients with sickle cell disease (SCD). The proof-of-concept study is being conducted at the Foundation for Sickle Cell Disease Research (FSCDR) in Hollywood, Florida, under the supervision of principal investigator Dr. Gershwin Blyden.
The clinical trial will enroll ten patients with SCD who will receive VAS-101 treatment twice weekly over a four-week period, for a total of eight treatments. Five patients will receive topical application on the forearm, while the other five will receive sublingual (under the tongue) administration. The study will span 29 days with weekly blood draws to monitor various parameters.
Study Objectives and Assessments
The primary objectives of the trial are twofold: to evaluate the safety and tolerability of VAS-101, and to assess its effects on impaired blood flow dynamics, including adhesion molecule expression and erythrocyte fragility parameters. Functional Fluidics, a specialty laboratory in Detroit, Michigan, will conduct proprietary assays to objectively assess VAS-101's impact on red blood cell health.
Secondary objectives include evaluating VAS-101's effect on inflammatory markers associated with sterile inflammation activation over the 28-day period. Additionally, the Biophysical Chemistry Section at the National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) in Bethesda, Maryland, will determine changes in red blood cell sickling kinetics and oxygen dissociation curves. The study will also assess the pain-relieving benefits and potential opioid-sparing effects of VAS-101.
Dr. Lanetta Bronté-Hall, President of FSCDR, emphasized the significance of this research: "I'm proud that we are taking part in Vascarta's pilot study, which explores a new transdermal approach to pain management for individuals living with Sickle Cell Disease. Pain remains one of the most challenging and life-disrupting aspects of this disease, and new approaches are urgently needed."
VAS-101: A Novel Approach to SCD Treatment
VAS-101, developed by Vascarta Inc. (Summit, NJ), represents an innovative approach to SCD treatment. The formulation employs a patented transdermal delivery technology designed to increase the bioavailability of curcumin, which has traditionally been limited when administered orally.
A study published in the PNAS Nexus Journal indicated that VAS-101 "has the potential to ameliorate chronic pain, improve RBC stability, and reduce inflammatory consequences of SCD." While curcumin possesses beneficial properties including anti-sickling, anti-inflammatory, and antioxidant effects, its clinical potential has been constrained by poor bioavailability from oral administration.
Dr. Joel Friedman, Professor at the Department of Microbiology & Immunology at Albert Einstein College of Medicine and Vascarta Scientific Founder, stated, "The preclinical 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 if not most of the clinical consequences of SCD." Dr. Friedman is the inventor of VAS-101, which is exclusively licensed to Vascarta from the Albert Einstein College of Medicine.
Addressing Critical Unmet Needs in SCD
Sickle cell disease is the most common inherited genetic disorder affecting primarily African American and non-Hispanic Black individuals in the United States. The condition is caused by a single point mutation in the globin gene, leading to sickling of red blood cells. SCD is characterized by severe pain, inflammation, oxidative stress, and organ damage, which contribute to poor quality of life and reduced survival.
Current estimates indicate there are more than 165,000 cases of SCD in the USA and 45,000 in European Union countries. The estimated life expectancy for individuals with sickle cell disease in the USA is more than 20 years shorter than the average expected lifespan.
Despite recent therapeutic advances, most approved SCD therapies do not adequately address pain management, and patients often require multiple medications with undesirable side effects. The unmet medical need remains significant, with a clear demand for better therapies that improve outcomes, optimize compliance, minimize the need for blood transfusions, and reduce dependence on potentially harmful pain medications.
Future Implications
Dr. Richard Prince, Vascarta Chairman, CEO & President, expressed optimism about the potential impact of VAS-101: "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."
If successful, this novel transdermal approach could represent a significant advancement in SCD treatment, particularly for pain management—one of the most debilitating aspects of the disease. The study's focus on both topical and sublingual administration will provide valuable insights into the optimal delivery method for this innovative therapy.
The results of this Phase 1 trial will be crucial in determining the path forward for VAS-101 and could potentially open new avenues for treating not only SCD but also other inflammatory conditions. Vascarta is also exploring applications of its transdermal delivery technology for osteoarthritis, further expanding the potential impact of this platform.
