Drug repurposing for treatment of congenital vascular malformations


Reference #: 1659

The University of South Carolina is offering licensing opportunities for Drug Repurposing for Treatment of Congenital Vascular Malformations.


Congenital vascular malformations (CVM) are a major threat to public health with a 1.5% incident rate in the general population. Congenital capillary malformation (CM), a.k.a., port-wine birthmarks or stains (PWB or PWS), are the most common type of CVM with an estimated prevalence of 0.3–0.5% per live births. The pulsed dye laser (PDL) or photodynamic therapy (PDT) is the treatment of choice for PWB. The current laser-based modalities result in inadequate efficacies which urgently needs a solution for patients.

Invention Description:

This invention is for repurposing of a panel of FDA-approved drugs or investigational compounds for treatment of blood vessel abnormalities in patients with CVM. This modality can be laser independent.

Potential Applications:

This invention serves as a laser-independent treatment option for PWB. The pulsed dye laser (PDL) or photodynamic therapy (PDT) is the treatment of choice for PWB. Unfortunately, complete removal of PWB occurs in less than 10% of patients. Additionally, about 20% of lesions have no response to laser treatment. Between 16.3% and 50% of patients experience re-darkening of their PWB as early as 5 years after multiple PDL treatments.

Advantages and Benefits:

Some MAPK inhibitors such as axitinib have also been investigated for this purpose. However, the efficacy is unsatisfactory. Additionally, the side effects are a major concern for pediatric patients and the costs are high. The method used in this invention has a good safety profile which will be applicable to pediatric patients. In addition, the cost is low, which will not cause a big economic burden to patients.


Patent Information:
For Information, Contact:
Lacie Cottrill
Technology Associate
University of South Carolina
Wenbin Tan
Vi Nguyen
Chao Gao
Anil Jegga
Yunguan Wang
Sudhir Ghandikota
Marcelo Hochman
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