This technology identifies proteasome inhibitors and PI3K/mTOR inhibitors as therapeutic interventions for patients with pathologic vascular malformations.
Current patients suffering from vascular malformations have limited treatment options, with no available FDA-approved therapy. While several clinical trials are exploring various candidates including sirolimus, an mTOR inhibitor, these treatment options are expected to require substantially high doses. These doses can exacerbate unwanted side effects, necessitating more precise therapeutic options.
This technology identifies proteasome inhibitors and PI3K/mTOR inhibitors such as omipalisib as therapeutics for treating vascular malformations. These targets were identified through high-throughput screening of patient-derived endothelial cells, suggesting that treatments will be much more germane to specific molecular pathology. Targeting the proteasome and PI3K/mTOR were revealed to be effective at reducing outgrowth of pathogenic endothelial cells, underscoring them as potential treatments.
This technology has been validated in patient-derived endothelial cell lines.
IR CU20315
Licensing Contact: Cynthia Lang
