Patented transdermal drug candidate from Vascarta may offer a convenient & cost-effective way to treat sickle cell disease

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February 18, 2025 4:40 PM EST

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SUMMIT, N.J., Feb. 18, 2025 /PRNewswire/ -- An article in the Proceedings of the National Academy of Sciences (PNAS) Nexus journal* by Goel et al. describes studies in which humanized sickle cell mice topically treated with Vascarta's transdermal curcumin gel formulation ("VAS-101") experienced reduced pain and inflammation along with improved red blood cell stability and functionality.

Based in part on these favorable pre-clinical results, a clinical trial using VAS-101 in individuals with sickle cell disease will soon be initiated by the Foundation for Sickle Cell Disease and Research [FSCDR (Hollywood, Florida, USA)]. Patients will be treated with VAS-101 via the topical and sublingual routes of administration.

In affected individuals, unstable red blood cells are prone to hemolysis and sickling causing inflammation, oxidative stress and blood flow stagnation which can produce acute and chronic pain as well as organ damage. There is a critical need for treatments which are cost effective address pain, easy-to-use, and without consequential side effects. Unfortunately, recently approved gene therapies cost up to $3.1 million/person and require a rigorous and toxic pretreatment regimen that still may not cure pain. Further, due to the complexity of SCD pathobiology many drugs are themselves toxic. The FDA approved Voxelotor was withdrawn from the market five years after its 2019 launch due to safety concerns (e.g., higher rate of vaso-occlusive crisis and more deaths in patients with sickle cell disease receiving this drug compared to placebo).

VAS-101 reduced multiple indices of chronic pain in humanized sickle cell mice. In some instances, the reduction occurred within hours of application of the drug candidate. There was a substantial reduction in mast cell activation, a process that directly contributes to the onset and persistence of chronic pain. The topical treatment also resulted in a decrease in multiple markers of inflammation including interleukins 2, 4, and 6 (IL-2, IL-4, IL-6), interferon, and other inflammatory markers. Moreover, red blood cell stability and functionality were improved, with reduced levels of hemolysis, oxidative damage, increased hematocrit and increased levels of ATP (adenosine triphosphate).

VAS-101 is highly bioavailable through a unique delivery route that bypasses drug metabolic roadblocks such as first pass modification by the liver that limit the intrinsic therapeutic efficacy for orally administered curcumin. VAS-101 results in rapid appearance of curcumin in both plasma and circulating blood cells. Uptake of curcumin in circulating red blood cells is postulated to be the likely basis for effective and sustained systemic delivery as well as the phenomena of improved health and stability of the human red blood cell. In the reported study, therapeutic efficacy was achieved by applying a single 0.1 ml drop of VAS-101 (0.23 M in curcumin **) to the abdomen once every two days for 21 days.

These findings add to the list of therapeutic benefits of VAS-101 that are achievable through the transdermal delivery route and that bypass the limitations of oral delivery including the well-known gastrointestinal adverse effects from extended use of high doses of oral curcumin.

Dr. Joel Friedman, Professor, Department of Microbiology & Immunology, Albert Einstein College of Medicine, and Scientific Founder & Chief Scientific Officer of Vascarta, Inc., stated, "These positive sickle cell results are consistent with multiple other preclinical studies that have shown therapeutic benefits of transdermal curcumin for pain, inflammation, cytokine storm, endothelial integrity, hypertension, aging and more." [Dr. Friedman is the inventor of VAS-101 which is licensed to Vascarta from the Albert Einstein College of Medicine (Bronx, New York, USA)].

Vascarta CEO & President Dr. Richard Prince commented that, "These breakthrough findings about the beneficial effects of our lead drug candidate (VAS-101) were generated through collaboration among scientists from many institutions. We will soon be initiating a pilot sickle cell clinical trial evaluating both topical and sublingual routes of administration. To help us speed the progression of VAS-101 into an eventual FDA-approved therapy, we welcome discussions with other pharmaceutical companies and/or strategic partners."

* "Targeting sickle cell pathobiology and pain with novel transdermal curcumin"
https://academic.oup.com/pnasnexus/advance-article/doi/10.1093/pnasnexus/pgaf053/8010891

**Formulated with Curcugen® (Dolcas-Biotech, LLC)

About Sickle Cell Disease

Sickle cell disease is the most common inherited disease that primarily affects millions of individuals globally with the major burden shared by Sub-Saharan Africa and India. It is considered a rare disease in the United States, with majority of affected individuals suffering with debilitating chronic pain and unpredictable and recurrent episodes of acute pain requiring hospitalization and poor quality of life with reduced survival.

About Vascarta

Vascarta is a clinical stage pharmaceutical company exploring efficient transdermal delivery of pharmaceuticals to address several conditions including sickle cell disease, arthritis, hypertension, cancer, and aging. More information regarding Vascarta's existing research or future collaborations can be found at www.Vascarta.com or by email to Vascarta's CEO, Dr. Richard Prince, at [email protected].

Publication Authors & Affiliations

Yugal Goel¹, Mya A. Arellano¹, Raghda T. Fouda¹, Natalie R. Garcia¹, Reina A. Lomeli¹, Daniel Kerr², Donovan A. Argueta¹, Mihir Gupta³, Graham J. Velasco⁴, Richard Prince⁵, Probal Banerjee², Sirsendu Jana⁶, Abdu I. Alayash⁶, Joel Friedman^5,7, Kalpna Gupta^1,8*

¹Hematology/Oncology Division, Department of Medicine, University of California, Irvine, CA, USA. ²Department of Chemistry and Center for Developmental Neuroscience, The College of Staten Island (CUNY), NY, USA. ³Department of Neurosurgery, Yale School of Medicine, New Haven, CT, USA.⁴Pathology Department, VA Long Beach Medical Center, Long Beach, CA, USA. ⁵Vascarta, Inc, Summit, NJ, USA. ⁶Laboratory of Biochemistry and Vascular Biology, Center for Biologics Evaluation and Research, Food and Drug Administration, Silver Spring, MD, USA. ⁷Department of Microbiology and Immunology, Albert Einstein College of Medicine, Bronx, NY, USA. ⁸Division of Hematology, Oncology and Transplantation, Department of Medicine, University of Minnesota, Minneapolis, MN, USA.