This technology is an oral treatment option utilizing sirolimus for infants and children with moderate-to-severe cases of pulmonary vein stenosis (PVS).
Limited treatment options currently exist for pulmonary vein stenosis (PVS), a rare disease with high mortality. Because PVS has a high recurrence rate and progresses to previously unaffected vascular distributions, the available approaches to prevent pulmonary vein blockage are highly invasive (e.g., repetitive surgical procedures like catheterization or stent placement) and only ameliorate the symptoms (e.g., diuretics and antihypertensive drugs). Current anti-proliferative agents have resulted in high rates of significant adverse events and only modest survival outcomes.
This therapeutic agent utilizes a blocker of the mTOR pathway, such as sirolimus or rapamycin, and is thought to have both anti-proliferative and immunosuppressive properties. When administered orally in infants and children with severe pulmonary vein stenosis (PVS), survival rate was greatly improved over the control group with few serious adverse events. This technology also provides a method of grading PVS in a subject by imaging the patient, calculating affected veins, and scoring the PVS by severity.
This technology has been validated in a multi-year study involving pediatric patients.
Patent Pending
IR CU21316
Licensing Contact: Jerry Kokoshka