The Pazopanib clinical trial has launched, and the road to reach this breakthrough milestone has been more involved and hard-fought than many may realize.

Cure HHT is one of the few patient advocacy organizations that not only helps to facilitate and drive research forward but is directly sponsoring a Phase II/III clinical trial of an investigational new drug product which they own. I wanted to take this moment to 1) explain what this means, 2) reflect on how we reached this point, and 3) share more on why it’s monumental for our community.

What is pazopanib?

Currently, there are no FDA-approved drugs to treat HHT. Some patients with HHT are receiving FDA-approved therapies to treat HHT-related bleeding (like Avastin), but these therapies are what we call ‘off-label’, or approved by the FDA to treat another disease that is not HHT.

Pazopanib (pa-zah-pa-nib) is an antiangiogenic therapeutic (works to stop blood vessel growth) that is typically given orally to patients with cancerous tumors. When given off-label at a greatly reduced dose than the chemotherapeutic dose, pazopanib has had positive effects in HHT patients with outcomes like a reduced need for blood transfusion, iron infusion, and a decreased amount of nose and gastrointestinal bleeding.