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Cure HHT Newsletter
2025 Volume 1 - Spring / Summer
Bonus Content
Find full text versions of select articles here
There are no FDA-approved therapeutics for the treatment of HHT. Cure HHT sought to change that. In many ways, the history of pazopanib is not unlike the story of the HHT community. Both are defined by persistence: a long battle for awareness, funding, and ultimately, solutions. Now, after years of dedicated effort, the paths of pazopanib and HHT have converged, moving forward in unison and resulting in the accomplishment of a pazopanib clinical trial filling and closing to enrollment. This is a major step forward for the future of HHT treatment, as we push to bring our community FDA approved therapies for the first time. Here’s a look back at how we got here. WHAT IS PAZOPANIB? The story of pazopanib starts more than 15 years ago. In 2009, the pharmaceutical giant GlaxoSmithKline secured FDA approval to use the drug, marketed under the name Votrient™, for treatment of patients with renal cell carcinoma — a rare kidney cancer. Votrient functions as an angiogenesis inhibitor, meaning it helps to prevent the formation and growth of new blood vessels. When pazopanib was approved to treat kidney cancer, it arrived at a turning point in care—when doctors were shifting from older immune-based treatments…
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Cure HHT helps drive science forward through government advocacy, seed grants and other means and have helped leverage your donations into millions of dollars dedicated to funding new treatments, advancing diagnostic technology, and developing new drugs. See examples of your donations at work and learn about some of the most transformational work being done by clinicians and scientists we support.
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Up Close and Personal with Christopher Hughes, PhD – Cure HHT’s 2020 Robert E. Berkman Leadership Award Recipient by Phyllis Loveland In recognition of Robert E. Berkman’s exemplary leadership, we honor his legacy through the creation of the Robert E. Berkman Leadership Award, to recognize those members of the Cure HHT Community who exhibit the qualities that have made Bob such a valued part of our organization’s mission. Have you ever spoken to someone who is just so darn brilliant that you stand in awe, hanging on their every word? If you haven’t, then you need to meet Chris Hughes, PhD, Chair of the Cure HHT North American Scientific and Medical Advisory Council, Lead Researcher for the Rare as One, Chan Zuckerberg Initiative and this year’s recipient of the Robert E. Berkman Leadership Award (“REBLA”). Dr. Hughes is one of those people who is connected to HHT with a passion and drive that stands out among not only his peers but the entire HHT Community. “He was a unanimous and natural choice for this award,” states Bob Berkman, named Award Honoree. I had the opportunity to chat personally with Dr. Hughes about new advancements in the HHT research world and,…
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We are at once happy and sad to announce the retirement of Reed Pyeritz, MD, PhD, Co-Director of the University of Pennsylvania School of Medicine (“UPenn”) HHT Center of Excellence. Dr. Pyeritz has been an integral part of our organization since 2003 when UPenn became Cure HHT’s 7th HHT Center of Excellence. The knowledge and commitment to the HHT Community that Dr. Pyeritz has contributed over a stellar forty-five-year career as one of this country’s leading geneticists is immeasurable. Starting at the beginning, Dr. Pyeritz began his education in the field of Biochemistry and it was while he was conducting his PhD work that he became interested in the world of genetics. “I was given the opportunity to watch one of the first amniocentesis and thought…wow, this is incredible to be able to diagnosis an unborn child using techniques in the lab,” says Pyeritz. From there, he made the decision to complete his PhD and then continue onto medical school at Harvard University where he graduated and soon after became Board Certified in Internal Medicine and Medical Genetics and Genomics. Dr. Pyeritz conducted his residency at the Brigham and Women’s Hospital in Boston until he joined the Genetics Department at…
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