Therapeutic interventions for treating vascular malformations
This technology identifies proteasome inhibitors and PI3K/mTOR inhibitors as therapeutic interventions for patients with pathologic vascular malformations.
Unmet Need: Treatment options for resolving 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.
The Technology: Proteasome inhibitors and PI3K/mTOR inhibitors to treat vascular malformations
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.
Applications:
- Treatment for vascular malformations
- Treatment for other types of endothelial proliferative diseases
- Inhibition of endothelial cell proliferation for research studies
Advantages:
- Greater expected efficacy than repurposed therapeutics
- More precise than emerging standard of care
- Lower dose is required for efficacy
- Opportunity for combinatorial therapeutics
Lead Inventor:
Patent Information:
Related Publications:
Tech Ventures Reference:
IR CU20315
Licensing Contact: Cynthia Lang