Cure HHT has a long history of leveraging* donor gifts into breakthrough research. Through the investment of $50,000 seed research grants, young investigator grants and scientific conferences totaling $2.1 Million since 2004, we are now able to determine the classes of drug therapies that are best targeted to reverse AVM development and prevent AVMs from forming in the future. For the first time in 30 years, the building blocks we have carefully invested in learning how AVMs form and the specific mechanisms related to development of HHT at a cellular and molecular level are falling into place for Advancing A Cure!
Together with you, Cure HHT is committed to radically changing the treatment of HHT and improving the lives of HHT patients and their families around the world.
*Cure HHT has leveraged their $2.1 Million investment to over $51.3 Million in research funding through the National Institutes of Health (NIH), Department of Defense (DOD), Food & Drug Administration (FDA) Orphan Drug, and European Research Agency. That is a 2,397% return on investment (ROI)!
Cure HHT's research program fosters the development of lasting cures for the underlying mechanisms and causes of HHT. To spur development of new ideas leading to novel drug therapies, Cure HHT funds basic science research grants to help learn more about the mechanisms of pathology in HHT. Cure HHT Basic Science Research Grants.
| Researcher | Institution / Country | Grant Type | HHT Funding | Other Funding |
|---|---|---|---|---|
| Beth Roman, PhD | University of Pittsburgh (USA) | Basic Science (2021) | --- | $2,000,000 (DOD) |
| Jennifer Fang, PhD | University of California, Irvine (USA) | Basic Science (2020) | --- | $314,000 (DOD) |
| Stryder Meadows, PhD | Tulane University (USA) | Basic Science (2018) | --- | $1,819,500 (NIH) |
| Christopher Hughes, PhD | University of California, Irvine (USA) | Basic Science (2017) | --- | $1,923,000 (NCATS) |
| Stryder Meadows, PhD | Tulane University (USA) | Basic Science (2016) | --- | $1,785,199 (DOD) |
| Marie Faughnan, MD; Douglas Marchuk, PhD; Philippe Marambaud, PhD; S. Paul Oh, PhD; Beth Roman, PhD | University of Toronto (Canada); Duke University (USA); Feinstein Institute for Medical Research (USA); Barrow Neurological Institute (USA); University of Pittsburgh (USA) | Basic Science and Clinical Science (2016) | --- | $10,000,000 (DOD) |
| Michaela Aldred, PhD | Lerner Research Institute, Cleveland Clinic (USA) | Discovery Award (2015) | --- | $2,600,000 (DOD) |
| Rong Wang, PhD | University of California, San Francisco (USA) | Investigator Initiative Award (2015) | --- | $1,500,000 (DOD) |
| S. Paul Oh, PhD | University of Florida (USA) | Basic Science (2015) | $50,000 | $1,500,000 (NIH / NHLBI) |
| Beth Roman, PhD | University of Pittsburgh (USA) | Genetics (2011) | $45,000 | $2,000,000 (NIH) |
| Rosemary Akhurst, PhD | University of California, San Francisco (USA) | Basic Science (2008) | $50,000 | $1,800,000 (NIH / NHLBI) |
| Christopher Hughes, PhD | University of California, Irvine (USA) | Basic Science (2008) | $50,000 | --- |
| S. Paul Oh, PhD | University of Florida (USA) | Basic Science (2008) | $50,000 | $1,000,000 (NIH) |
| Michelle Letarte, PhD | The Hospital for Sick Children (Canada) | Basic Science (2006) | $50,000 | --- |
| Douglas Marchuk, PhD | Duke University (USA) | Basic Science (2004) | $50,000 | --- |
| Mary Porteous, MD | The University of Edinburgh (Scotland) | Genetics (2004) | $5,000 | --- |
Cure HHT has been instrumental in significant advances in HHT treatment, and will continue to act as a catalyst for improved care for HHT patients. Clinical science research determines the safety and effectiveness of medications, devices, diagnostic products and treatments intended for human use. To facilitate the translation of basic science research into clinical trials and new treatments for HHT, Cure HHT collaborates with industry and academia to test the efficacy of therapeutic drugs, medical devices, and treatment protocols. Cure HHT Clinical Science Research Grants.
| Researcher | Institution / Country | Grant Type | HHT Funding | Other Funding |
|---|---|---|---|---|
| Clifford Weiss, MD | Johns Hopkins University (USA) | Clinical Science (2022) | $49,926 | --- |
| Steven Hetts, MD | University of California, San Francisco (USA) | Clinical Science (2022) | --- | $2,584,000 (DOD) |
| Hanny Al-Samkari, MD | Massachusetts General Hospital (USA) | Clinical Science (2020) | $40,000 | $1,000,000 (NIH) |
| Edda Spiekerkoetter, MD | Stanford University (USA) | Clinical Science (2019) | --- | $1,800,000 (DOD) |
| Marie Faughnan, MD; Douglas Marchuk, PhD; Philippe Marambaud, PhD; S. Paul Oh, PhD; Beth Roman, PhD | University of Toronto (Canada); Duke University (USA); Feinstein Institute for Medical Research (USA); Barrow Neurological Institute (USA); University of Pittsburgh (USA) | Basic Science and Clinical Science (2016) | --- | $10,000,000 (DOD) |
| Amelia Clark, MD | Stanford University (USA) | Clinical Science (2014) | $10,000 | --- |
| Kristen Anton, MS | BioInformatics Service Center, Dartmouth College (USA) | Therapeutic Outcomes (2013) | $165,000 | --- |
| Jonathan Zaroff, MD | Kaiser Foundation (USA) | Prevalance / Undiagnosed (2013) | $30,000 | --- |
| Jeffrey Hoag, MD | Drexel University (USA) | Treatment of Disease (2007) | $50,000 | --- |
| Christine Mummery, PhD | Leiden University Medical Center (Netherlands) | Treatment of Disease (2006) | $50,000 | --- |
| Carmelo Bernabeu, PhD | Centro de Investigaciones Biologica (Spain) | Treatment of Disease (2005) | $50,000 | $500,000 (European Research Agency) |
| Karen Swanson, DO | Mayo Clinic (USA) | Treatment of Disease (2004) | $50,000 | $1,000,000 (FDA Orphan Drug Grant) |
| Stanley Shapshay, MD | Albany Medical Center (USA) | Treatment of Disease (2004) | $5,000 | --- |
| Jamie McDonald, MS, CGC | ARUP Laboratories (USA) | Genetics / DNA Patent Release (2003) | $10,000 | --- |
| Arupa Ganguly, PhD | University of Pennsylvania Genetic Diagnostic Laboratory (USA) | Genetics / DNA Patent Release (2003) | $10,000 | --- |
| Kirk Vandezande | HHT Solutions, Inc. (Canada) | Genetics / DNA Patent Release (2003) | $10,000 | --- |
The HHT community is in desperate need of effective and safe treatments to improve health and quality of life. Clinical trials test the safety and efficacy of potential drug therapies and treatments as well as new applications for FDA approved drugs and/or treatments.
| Researcher | Clinical Trial Description | Study Year | HHT Funding | Other Funding |
|---|---|---|---|---|
| Randomized Trial for Pazopanib in HHT-Related Bleeding | A Phase II/III randomized, placebo-controlled, double-blind study to evaluate the effects of up to 24 weeks of low dose oral pazopanib on hereditary hemorrhagic telangiectasia (HHT) related epistaxis and anemia. | 2022-2025 | $877,824 (FDA) | |
| Randomized Trial for Pazopanib in HHT-Related Bleeding | A Phase II/III randomized, placebo-controlled, double-blind study to evaluate the effects of up to 24 weeks of low dose oral pazopanib on hereditary hemorrhagic telangiectasia (HHT) related epistaxis and anemia. | 2021-2024 | $350,000 | $5,240,964 (DOD) |
| Pomalidomide for the Treatment of Bleeding in Hereditary Hemorrhagic Telangiectasia | A Phase II randomized, placebo-controlled double-blind study of pomalidomide in patients with hereditary hemorrhagic telangiectasia (HHT) with moderate to severe epistaxis who have anemia and/or require parenteral iron infusions or blood transfusions. Treatment with oral pomalidomide or matching placebo for 24 weeks. | 2019-2023 | --- | $3,638,312 (NIH) |
| North American Study of Epistaxis (NOSE) | Multi-center clinical trial designed to test the efficacy of three nasal spray agents as compared to a placebo in reducing nose bleed frequency, duration and increasing quality of life. | 2010 - 2014 | $180,000 | --- |
The Cure HHT Young Scholar Research Program supports scientists* and clinicians* who show exceptional ability and promise for conducting HHT basic science, clinical science or translational science research. The objective of this program is to foster creative HHT research; enhance early career development of outstanding young investigators; and increase opportunities for the young investigator to recognize our mission to FIND, TREAT and CURE HHT. Cure HHT Young Investigator Awards.
*Residents, Postdoctoral/Clinical Fellows, PhD Candidates or Early Stage Investigator who are within 3 years of completing their terminal research degree or within 3 years of completing their medical residency/sub-specialty fellowship
| Researcher | Institution / Country | Grant Type | HHT Funding |
|---|---|---|---|
| HHT International Scientific Conference | Young Investigator Travel Awards and Presentation Awards | 2022 | $42,000 |
| HHT International Scientific Conference | Young Investigator Travel Awards and Presentation Awards | 2019 | $20,250 |
| HHT International Scientific Conference | Young Investigator Travel Awards and Presentation Awards | 2017 | $15,500 |
| Veronique Vorselaars, PhD | St. Antonius Hospital (Netherlands) | 2017 | $1,200 |
| HHT International Scientific Conference | Young Investigator Presentation Awards | 2003 - 2015 | $14,000 |
| Hongyu Tian, PhD | Duke University (USA) | Basic Science (2015) | $30,000 |
| Wan Zhu, PhD | University of California, San Francisco (USA) | Basic Science (2015) | $30,000 |
| Simon Tual-Chalot, PhD | Newcastle University (UK) | Basic Science (2015) | $30,000 |
| Whitney Wooderchak-Donahue, PhD | University of Utah / ARUP Laboratories (USA) | Genetics (2015) | $30,000 |
| Chadwick Davis, PhD | Recursion Pharmaceuticals (USA) | Drug Therapy (2015) | $30,000 |
| Pinar Bayrak-Toydemir, MD, PhD | University of Utah / ARUP Laboratories (USA) | Genetics (2008) | $20,000 |
| Sebastian Velthius, MD, PhD | St. Antonius Hospital (Netherlands) | 2014 | $2,000 |
| Sabine Bailly, MD | CEA-Grenoble (France) | NIH Travel Award (2006) | $1,000 |
| Terri DiMaio, PhD | University of Wisconsin (USA) | NIH Travel Award (2006) | $1,000 |
| Africa Fernandez, PhD | Centro de Investigaciones Biologicas (Spain) | NIH Travel Award (2006) | $1,000 |
| Eric Finkelstein, PhD | Syracuse University (USA) | NIH Travel Award (2006) | $1,000 |
| Tassilo Forg, PhD | University of Applied Sciences (Germany) | NIH Travel Award (2006) | $1,000 |
| Arupa Ganguly, PhD | University of Pennsylvania (USA) | NIH Travel Award (2006) | $1,000 |
| Fatima Govani, PhD | University of Oxford (UK) | NIH Travel Award (2006) | $1,000 |
| Jose Miguel Lopez-Novoa, PhD | Universidad de Salamanca (Spain) | NIH Travel Award (2006) | $1,000 |
| Mourad Toporsian, PhD | McGill University (Canada) | NIH Travel Award (2006) | $1,000 |
| Liana Zucco | University College London (UK) | NIH Travel Award (2006) | $1,000 |
This award was established in recognition of Dr. Robert I. White Jr.’s outstanding 
contribution to HHT care and to mentoring other clinicians to develop expertise in HHT care and research. This award is given annually to a clinician (physician or health professional), who is within the first years of work involving HHT and who embodies Dr. White’s qualities as a compassionate and dedicated clinician, devoted to improving HHT care.
| Researcher | Institution / Country | Award Year | HHT Funding |
|---|---|---|---|
| Vivek Iyer, MD, MPH | Mayo Clinic (USA) | 2019 | $2,500 |
| Steven Hetts, MD | University of California, San Francisco (USA) | 2017 | $2,500 |
| Marcelo Serra, MD | Hospital Italiano de Buenos Aires (Argentina) | 2015 | $2,500 |
| Justin McWilliams, MD | University of California, Los Angeles (USA) | 2011 | $2,500 |
| Takeo Nishida, PhD | Yale University (USA) | 2010 | $2,500 |
| Els Marie De Gussem, MD | Grace Hospital (Canada) | 2009 | $2,500 |
| Sophie Dupuis-Girod, MD | CHU de Lyon (France) | 2008 | $2,500 |
| Roberto Zarrabeitia, MD | Hospital Sierrallana (Spain) | 2007 | $2,500 |
| Pinar Bayrak-Toydemir, MD, PhD | University of Utah / ARUP Laboratories (USA) | 2006 | $2,500 |
| Gaetan Lesca, MD | CHU de Lyon (France) | 2005 | $2,500 |
| Tom Letteboer, MD, PhD | University Medical Centre Utrecht (Netherlands) | 2004 | $2,500 |
Cure HHT has long acted as a catalyst by making personal connections within the relevant communities and hosting scientific and medical conferences. To date, Cure HHT has partnered with NAVBO, NIH, CDC, American Academy of Otolaryngology and many other organizations to educate physicians and scientists about HHT.
| Event | Institution / Country | Award Year | HHT Funding |
|---|---|---|---|
| HHT International Scientific Conference | Conference Host (Portugal) | 2022 | TBD |
| Christopher McMahon Memorial International HHT Guidelines Conference | Conference Host (Canada) | 2019 | $175,000 |
| HHT International Scientific Conference | Conference Host (Puerto Rico) | 2019 | $27,000 |
| HHT International Scientific Conference | Conference Host (Croatia) | 2017 | $61,500 |
| North American Vascular Biology Organization (NAVBO) | Sponsor - HHT Symposia | 2016 | $5,000 |
| HHT International Scientific Conference | Conference Host (USA) | 2015 | $33,000 |
| HHT International Scientific Conference | Sponsor - Speaker Travel Awards | 2001 - 2013 | $70,500 |
| North American Vascular Biology Organization (NAVBO) | Sponsor - HHT Symposia | 2014 | $10,000 |
| North American Vascular Biology Organization (NAVBO) | Sponsor - HHT Genetics Research Presentation | 2009 | $10,000 |
| National Institutes of Health (NIH) | Sponsor - Brain AVM Presentation | 2008 | $2,000 |
| North American Vascular Biology Organization (NAVBO) | Sponsor - HHT Symposia | 2007 | $2,000 |
| HHT Clinical Guidelines Conference | Sponsor - Expert Travel Awards | 2006 | $60,000 |
| National Institutes of Health | Sponsor - Physician Training | 2006 | $5,000 |