A first in human proof of concept phase I clinical study of VAS-101 (Vasceptor®; topical curcumin gel) in sickle cell disease (SCD) is underway. The study is being conducted by the Foundation for Sickle Cell Disease Research (FSCDR) in Hollywood, Florida, under the supervision of the principal investigator, Dr. Gershwin Blyden.
Ten (10) patients with sickle cell disease will be treated with VAS-101 twice per week over 4 weeks for a total of 8 treatments; their blood will be drawn weekly. Five (5) patients will be treated topically on their forearm and five (5) patients will be treated sublingually (under the tongue), the duration of treatment is 29 days.
The primary study objectives are: (1) assess the safety and tolerability of VAS-101; and (2) assess its effects on impaired blood flow dynamics, including adhesion molecule expression and erythrocyte fragility parameters. The objective assessment of VAS-101 impact on red blood cell health will be performed by Functional Fluidics utilizing their proprietary assays at their specialty laboratory in Detroit, Michigan.
Secondary study objectives include (1) an evaluation of the effect of VAS-101 on inflammatory markers associated with the activation of sterile inflammation over 28 days; (2) determination of the mean change in red blood cell sickling kinetics and oxygen dissociation curves will be assessed by The Biophysical Chemistry Section, Laboratory of Chemical Physics at the National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) in Bethesda, Maryland; and (3) assessment of the pain-relieving benefit and opioid-sparing effect of VAS-101.
Dr. Lanetta Bronté-Hall, President of FSCDR, stated, "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 (SCD). Pain remains one of the most challenging and life-disrupting aspects of this disease, and new approaches are urgently needed. While early in development, this research represents an important step toward finding new options that could one day help ease that burden. At FSCDR, we are committed to advancing care and bringing hope to the SCD community through innovation and collaboration."
About VAS101:
VAS-101 (developed by Vascarta Inc., Summit, NJ) is a topical curcumin formulation that employs a patented transdermal delivery technology designed to increase bioavailability. A published study 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 many beneficial attributes such as safety, anti-sickling, anti-inflammatory, and antioxidant properties, its clinical translational potential has been constrained due to limited bioavailability from oral administration.
About Sickle Cell Disease:
Sickle cell disease is the most common inherited genetic disorder that affects 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, kidney, liver, and heart disease.
Current estimates indicate there are >165,000 cases of SCD in the USA and 45,000 in European Union countries. The estimated life expectancy of those with sickle cell disease in the USA is more than 20 years shorter than the average expected lifespan.
SCD is caused by a single point mutation in the globin 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. Recently approved SCD therapies do not mitigate pain, and patients are often on multiple drugs which often have undesirable side effects.
The unmet medical need for most SCD sufferers is significant. Better therapies that improve outcomes, optimize compliance for patients of all ages, minimize the need for blood transfusions, and reduce the need for chronic administration of potentially harmful pain medications are needed.
Dr. Joel Friedman, Professor, Department of Microbiology & Immunology, Albert Einstein College of Medicine, and Vascarta Scientific Founder & Head, Scientific Advisory Board, 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 (Bronx, New York, USA).
For more information please visit, www.vascarta.com.
