What the HHT?
A blog for the HHT community
Pazopanib Clinical Trial: Q&A
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.
Read MoreChildren’s Hospital of Philadelphia receives funding for pediatric HHT patients
I had the opportunity to visit our pediatric team at the Children’s Hospital of Philadelphia (CHOP) while in town for a conference. The team was awarded an internal grant of $200,000, and we wanted to highlight the work they propose to do with this project.
Dr. Lauren Beslow (Neurology) kindly volunteered to give me a tour of CHOP. This children’s hospital is one of the most beautiful I’ve seen and is completely designed with kids in mind. Carefully chosen artwork that is cheerfully representative of children with all kinds of abilities, lit-up sculptures of brains, sensory mats on the floor, tabletops with pieces that spin, bookshelves with free books to take home, and colors everywhere truly make CHOP seem like a giant play area where you happen to see a healthcare provider.
Read MoreOne in a Million
One year ago today, I underwent a liver transplant due to a genetic condition called HHT (Hereditary Hemorrhagic Telangiectasia), or Osler-Weber Rendu Syndrome. Roughly 1 in 5,000 people are diagnosed with HHT. Of that 1 in 5,000, up to 75%, have liver involvement. Of that 75%, only 8% become symptomatic. The data gets spotty after this, and from my research, I can only find 59 documented cases since 1985 of liver transplantation related to HHT.
Read MoreCure HHT Welcomes Cassi Friday, PhD
Cure HHT is thrilled to welcome Cassi Friday, PhD as the newest member of our team! Get to know Cassi, her connection to HHT, and learn more about her role as Director of Cure HHT Research Programs & Grants.
Read MoreHelp Keep Us On the Cutting Edge
The turn of the calendar to December marks the kickoff of our year-end appeal, and this year we are asking for your continued support to help keep us #OnTheCuttingEdge. The milestones we have our sights set on next are the kinds of things we could have only dreamed about just a few short years ago. A future exists where therapies – not surgeries – treat the chronic aspects of this disease. And that future is closer than you might realize!
Cure HHT remains a driving force for medical and scientific advancements in HHT, and throughout the month, we will be highlighting the transformational work being done…
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