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Innovative therapies
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AR101 – Enzastaurin

About AR101 (enzastaurin)

Enzastaurin is a well characterized PKCβ inhibitor that has been evaluated in over 50 clinical trials, with more than 3300 patients. This includes a Phase 3 study of nearly 500 patients with 3 years of enzastaurin treatment.1 Mutations in the COL3A1 gene have been linked to the loss of structural integrity of the extracellular matrix and increased clinical presentation of VEDS related symptoms, including arterial dissection and/or rupture. Recent findings from animal studies, in a VEDS mouse model, with similar Col3A1 mutations have shown that the mutation is a key mediator in increased PKC/ERK pathway signaling. Additionally, in this model, treatment with an inhibitor of PKCβ significantly prevented death due to spontaneous aortic rupture.2 Further investigation will be necessary to determine the potential of PKC inhibition as a treatment option.

VEDS/COL3A1 Overview

About Vascular Ehlers-Danlos Syndrome (VEDS)

Vascular Ehlers-Danlos Syndrome (VEDS) is an inherited connective tissue disorder, typically caused by a mutation in the COL3A1 gene. This mutation leads to defects in type III procollagen, a major protein in vessel walls and hollow organs. Patients with this diagnosis are at significant risk for serious vascular events like dissections, pseudoaneurysms, and ruptures throughout the vasculature.

Diagram 1

VEDS affects about 1 in 50,000 people worldwide. Nearly 50% of patients with this devastating condition die before the age of 50 years old. Currently, there are no FDA-approved therapies, and after diagnosis, the current standard of care is “watchful waiting.”

Living with VEDS

VEDS changes lives, but it does not define the people who have it. Watch the stories of those confronting VEDS with courage, conviction, and above all, hope for a better future.

Annie

Bridgette

Katie

The Tays Family

References

1. https://www.clinicaltrials.gov/ct2/show/NCT00332202. 2. Bowen CJ et al. Targetable cellular signaling events mediate vascular pathology in vascular Ehlers-Danlos Syndrome. J Clin Invest. 2020 Feb 3;130(2):686-698.

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