What the HHT?
A blog for the HHT community
Posts by Ashley Watkins
PAZ Clinical Trial Now Recruiting
HHT Centers of Excellences across the U.S. are now enrolling patients to participate in the new randomized clinical trial to assess the effectiveness of Pazopanib in reducing the severity of nose bleeds in adults with HHT.
To learn more about the trial and to understand if participation may be right for you, please visit the clinical trials section of our website.
By participating in clinical research, you can play a role in helping the development of new treatments and therapeutics – which have the potential to lead to a higher quality of life for generations of HHT patients.
Read MoreA major milestone for HHT treatment!
I am reaching out to share the news of a truly momentous milestone for both our organization and our community; the Pazopanib clinical trial has officially launched!
This means Cure HHT is now one of the few patient advocacy organizations in the world that is directly sponsoring a Phase II/III clinical trial of an investigational drug product that we own. I wanted to explain what this means, reflect on how we reached this point, and share why this is so monumental!
Read MorePazopanib 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 MoreAngiogenesis Publishes 2022 Conference Summaries
The 14th HHT International Scientific Conference brought together 300 in-person attendees and 50 virtual viewers to present and discuss the most recent and impactful scientific and clinical advances in HHT.
The meeting had a record number of 209 original research abstracts submitted and the top scoring abstracts were selected for oral presentations. The Executive Summary and Abstract Summary published in Angiogenesis highlight the high-caliber HHT research presented in Portugal from trainees, scientists, and clinicians around the world.
Read MoreStanford HHT CoE Receives $2.8M Grant
Over the years and thanks to the support of our community, we’ve been able to grow the number of clinicians and researchers focusing on advancing our understanding and treatment of HHT.
We’re excited to announce another major step forward in that fight! Dr. Edda Spiekerkoetter and her scientific colleagues at the Stanford HHT Center of Excellence were recently awarded a $2.8 million (yes, you read that right!) NIH/ NHLBI grant focused on the understanding and targeting of molecular and cellular events responsible for the development, growth, and regression of pulmonary AVMs.
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