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Highly bioavailable transdermal gel from Vascarta may offer a safe and effective new way to treat sickle cell disease - presentation at American Society of Hematology Conference

Wednesday, December 11, 2024 Genetics & Stem Cells News
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PR Newswire

SAN DIEGO, Dec. 10, 2024

SAN DIEGO, Dec. 10, 2024 /PRNewswire/ -- Vascarta Inc. ("Vascarta") reported new research findings at the 66th Annual American Society of Hematology meeting in San Diego showing that their novel transdermal preparation of curcumin reduces nerve and organ damage while improving red blood cell health in humanized sickle cell mice.
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Based on these and previous results in pre-clinical studies showing reduced pain, Vascarta's transdermal curcumin gel will be clinically tested next year in human sickle cell disease (SCD) sufferers by the Foundation for Sickle Cell Disease and Research (FSCDR (Hollywood, Florida, USA).
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In SCD, red blood cells (RBCs) are unstable. The unstable RBCs tend to release free hemoglobin and heme which sets in motion pro-inflammatory and oxidative processes that promote nerve and organ damage. There is a critical need for an FDA-approved treatment that is safe, prevents and treats SCD sequelae, and is easy to self-administer.

As described in the American Society of Hematology meeting poster*, when humanized sickle cell mice were exposed to inflammation and oxidative stress associated with SCD, they experienced nerve, organ, and RBC damage, leading to pain.  However, when scientists at the University of California – Irvine applied Vascarta's transdermal curcumin gel to humanized sickle cell mice, and to nerve tissue removed from these mice, they demonstrated reduced nervous tissue damage. Improvements were seen in the hippocampal neurons of the brain in the central nervous system and in dorsal root ganglia neurons in the peripheral nervous system.  There was also clear evidence of reduced inflammation-induced activation of microglia in the brain - which is an essential step in protecting the central nervous system from the consequences of SCD - and reduced oxidative damage to the spinal cord.

Experiments conducted by the FDA's Center for Biologics Evaluation and Research (CBER) showed the transdermal curcumin treatment of SCD mice i) improved the mitochondrial bioenergetic parameters in the heart, ii) reduced oxidative stress and iii) increased bioenergetic capacity in RBCs, all of which contribute to the complex constellation of symptoms observed in SCD patients.

Principal investigator, Professor Kalpna Gupta (University of California – Irvine), stated that: "These breakthrough findings about the beneficial effects of the transdermal curcumin gel on nervous system, organs, and red blood cells were generated through collaboration among scientists from several institutions, including the University of California – Irvine, the US Food and Drug Administration (FDA), and Albert Einstein College of Medicine."

Professor Joel Friedman (Albert Einstein College of Medicine and inventor of Vascarta's patented transdermal drug delivery technology) explained that: "The positive sickle cell results are consistent with multiple other preclinical studies that have shown, for example, therapeutic benefits of transdermal curcumin for inflammation, pain, cytokine storm, endothelial integrity, hypertension, aging."

Dr. Richard Prince, Vascarta Chairman and CEO, commented that: "We are excited that these findings, along with other favorable pre-clinical results showing reduced pain, give us the confidence to progress into human clinical testing among sickle cell patients."

*" Mechanism-based treatable targets for organ damage and pain in sickle cell disease"

About Sickle Cell Disease

Sickle cell disease is the most common inherited disease that primarily affects African Americans and Hispanics in the United States. Individuals with sickle cell disease may experience serious health complications, such as chronic pain, stroke, lung problems, eye problems, infections, and kidney disease, along with periodic acute pain crises.

About Vascarta Inc.

Vascarta is a clinical-stage pharmaceutical company developing 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.

Publication Authors & Affiliations

Yugal Goel1, Sirsendu Jana2, Mya A. Arellano1, Reina A. Lomeli1, Graham J. Velasco3, Donovan A. Argueta1, Richard Prince4, Abdu I. Alayash2, Joel Friedman4,5, Kalpna Gupta1,6*

1Hematology/Oncology Division, Department of Medicine, University of California, Irvine, CA, USA; 2Laboratory of Biochemistry and Vascular Biology, Center for Biologics Evaluation and Research, Food and Drug Administration, Silver Spring, MD, USA; 3Pathology Department, VA Long Beach Healthcare System, Long Beach, CA; 4Vascarta Inc, Summit, NJ; 5Department of Microbiology and Immunology, Albert Einstein College of Medicine, Bronx, NY, USA; 6Division of Hematology, Oncology and Transplantation, Department of Medicine, University of Minnesota, Minneapolis, MN, USA.

Corresponding Publication Author Email:  [email protected]

Vascarta contact:

Richard Prince, PhD – Chairman & Chief Executive Officer  Email: [email protected]



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