Specialty pharmaceutical company, Aytu BioPharma, announced this week that they will be conducting a pivotal clinical trial for the development of treatment for vascular Ehlers-Danlos syndrome (vEDS).
What is vascular Ehlers-Danlos syndrome?
VEDS is estimated to occur between 1 in 50,000 individuals to 1 in 200,000 and results from pathogenic variants in COL3A1, which is responsible for producing chains of type III procollagen, a major protein in the walls of blood vessels and hollow organs. The changes in COL3A1 known to cause vEDS to alter the framework used to build connective tissue, which decreases its capacity to withstand stress. Potentially fatal effects include arterial aneurysm, dissection, and rupture; bowel rupture; and rupture of the uterus when pregnant. Life span is often affected. Currently, the median life span is about 51 years—within a wide range from 10 to 80 years—and it is influenced by the nature of the particular COL3A1 variant. VEDS is a devastating condition, and currently, there are no therapies proven to prevent the life-threatening emergencies that can occur.
What will the clinical trial study?
The trial will be investigating a medication that has the potential to reduce life-threatening events in vEDS. The trial will study the investigational oral compound, enzastaurin, for its inhibition of the protein kinase C (PKC) pathway. The PKC pathway has been implicated in the pathogenesis of vEDS in mouse models. Enzastaurin has been previously studied in over 40 human trials including a range of cancers. The trial will investigate the ability of enzastaurin to reduce the severe complications of vEDS.
“Our community is thirsting for additional research across all types of Ehlers-Danlos syndromes. It’s also a mobilized and well-connected community that is excited to participate in advancing research and clinical development” explains Lara Bloom, President, and CEO for The Ehlers-Danlos Society, “The Ehlers-Danlos Society has been delighted to be part of this project since the start, first meeting with the co-founders Topher Brooke and Nate Massari in 2017 to establish research priorities with the chair of our Medical and Scientific Board, Dr. Clair Francomano, and making the first introduction to the renowned researcher, Dr. Hal Dietz. We are absolutely delighted to see the tremendous vision and commitment of Nate and Topher to improving the lives of those living with EDS come to fruition with this promising trial.”
How can people living with vEDS get involved?
The Ehlers-Danlos Society is delighted to be supporting this study, which gives much-needed hope for those living with vEDS and their families, as part of the key stakeholder group. The Ehlers-Danlos Society will help to facilitate collaboration through the vEDS working group of The International Consortium on Ehlers-Danlos Syndromes and Hypermobility Spectrum Disorders, and with the enrolment of study participants through our global registry.
We will update our community with updates on the trial and how to get involved through the global registry when enrollment begins. Join our CONNECT newsletter to stay up to date.
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