Connect Issues Fall 2021

Hope IN R E SEARCH The VEDS Movement is celebrating its second year as a division of The Marfan Foundation. We are thrilled to be celebrating two clinical trials that were announced this year. To have two clinical trials in the works for VEDS is an incredible opportunity to learn more about this condition; they provide hope that one or both may reveal a treatment to prevent life- threatening arterial ruptures and dissections for people with VEDS. THE CLINICAL TRIALS Aytu BioPharama is planning a clinical trial, called the PREVent trial, to test the effectiveness of an investigational medication called enzastaurin (AR101) for its inhibition of the protein kinase C (PKC)

p Bridgette Howe, her husband Travis Howe, and their dog, Stella.

pathway. The PKC pathway has been implicated in the pathogenesis of VEDS in mouse models. Enzastuarin has already been studied in more than 40 human trials across a range of cancers including glioblastoma. This trial will investigate the ability of enzastaurin to reduce severe complications of VEDS, such as arterial ruptures and dissections. The trial is anticipated to enroll patients globally. Acer Therapeutics is also planning a clinical trial, called the DiSCoVer trial, to test the effectiveness of Edsivo (celiprolol) in reducing events in people with VEDS. This trial is anticipated to enroll patients from the US only. Celiprolol is a beta blocker currently used in some countries as the primary treatment for people with VEDS, but it is not approved by the FDA in the US. This clinical trial hopes to reveal whether the medication is definitively effective in reducing events for people with VEDS, and if so, the company will pursue FDA approval. COMMUNITY PERSPECTIVES These clinical trials inspire hope in our community for the future. “Thinking about a clinical trial for a drug for VEDS is just amazing. The help that it would bring to families and friends, the hope... I think the hope is what's more important. If we have hope for a new medicine, then we have hope for tomorrow,” said Annie Barlow. “We have hope for a better life. We have hope for an easier life, with fewer doctor appointments and fewer panic attacks over what could happen if I do this or that. So I'm just so excited for what this could bring for the VEDS community.”

p Annie Barlow

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